TRAVERE THERAPEUTICS, INC., 10-K filed on 2/21/2025
Annual Report
v3.25.0.1
Cover - USD ($)
 12 Months Ended
Dec. 31, 2024
Feb. 18, 2025
Jun. 30, 2024
Cover [Abstract]      
Document Type 10-K    
Document Annual Report true    
Document Period End Date Dec. 31, 2024    
Current Fiscal Year End Date --12-31    
Document Transition Report false    
Entity File Number 001-36257    
Entity Registrant Name TRAVERE THERAPEUTICS, INC.    
Entity Incorporation, State or Country Code DE    
Entity Tax Identification Number 27-4842691    
Entity Address, Address Line One 3611 Valley Centre Drive    
Entity Address, Address Line Two Suite 300    
Entity Address, City or Town San Diego    
Entity Address, State or Province CA    
Entity Address, Postal Zip Code 92130    
City Area Code 888    
Local Phone Number 969-7879    
Title of 12(b) Security Common Stock, par value $0.0001 per share    
Trading Symbol TVTX    
Security Exchange Name NASDAQ    
Entity Well-known Seasoned Issuer Yes    
Entity Voluntary Filers No    
Entity Current Reporting Status Yes    
Entity Interactive Data Current Yes    
Entity Filer Category Large Accelerated Filer    
Entity Small Business false    
Entity Emerging Growth Company false    
ICFR Auditor Attestation Flag true    
Document Financial Statement Error Correction false    
Entity Shell Company false    
Entity Public Float     $ 624,140,482
Entity Common Stock, Shares Outstanding   88,739,826  
Documents Incorporated by Reference DOCUMENTS INCORPORATED BY REFERENCE: Portions of the Proxy Statement for the registrant’s 2025 Annual Meeting of Stockholders, to be filed within 120 days after the conclusion of the registrant's fiscal year ended December 31, 2024, are incorporated by reference into Part III of this Annual Report on Form 10-K.    
Entity Central Index Key 0001438533    
Amendment Flag false    
Document Fiscal Year Focus 2024    
Document Fiscal Period Focus FY    
v3.25.0.1
Audit Information
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Audit Information [Abstract]    
Auditor Firm ID 42 243
Auditor Name Ernst & Young LLP BDO USA, P.C.
Auditor Location San Diego, California San Diego, California
v3.25.0.1
CONSOLIDATED BALANCE SHEETS - USD ($)
$ in Thousands
Dec. 31, 2024
Dec. 31, 2023
Current assets:    
Cash and cash equivalents $ 58,535 $ 58,176
Marketable debt securities, at fair value 312,166 508,675
Accounts receivable, net 27,116 21,179
Inventory 6,200 9,410
Prepaid expenses and other current assets 12,685 19,335
Total current assets 416,702 616,775
Long-term inventory 35,656 31,494
Property and equipment, net 5,336 7,479
Operating lease right-of-use assets 14,295 18,061
Intangible assets, net 103,974 104,443
Other assets 18,162 10,661
Total assets 594,125 788,913
Current liabilities:    
Accounts payable 23,534 41,675
Accrued expenses 86,028 118,991
Convertible debt, current portion 68,678 0
Deferred revenue, current portion 2,815 7,096
Operating lease liabilities, current portion 5,405 4,909
Other current liabilities 14,291 5,237
Total current liabilities 200,751 177,908
Convertible debt, less current portion 310,310 377,263
Operating lease liabilities, less current portion 17,191 22,612
Other non-current liabilities 6,796 10,320
Total liabilities 535,048 588,103
Commitments and Contingencies (See Note 11)
Stockholders' Equity:    
Preferred stock $0.0001 par value; 20,000,000 shares authorized; no shares issued and outstanding as of December 31, 2024 and 2023 0 0
Common stock $0.0001 par value; 200,000,000 shares authorized; 87,452,835 and 75,367,117 issued and outstanding as of December 31, 2024 and 2023, respectively 9 7
Additional paid-in capital 1,506,315 1,327,881
Accumulated deficit (1,447,167) (1,125,622)
Accumulated other comprehensive loss (80) (1,456)
Total stockholders' equity 59,077 200,810
Total liabilities and stockholders' equity $ 594,125 $ 788,913
v3.25.0.1
CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares
Dec. 31, 2024
Dec. 31, 2023
Statement of Financial Position [Abstract]    
Preferred stock, par value (in dollars per share) $ 0.0001 $ 0.0001
Preferred stock, shares authorized (in shares) 20,000,000 20,000,000
Preferred stock, shares issued (in shares) 0 0
Preferred stock, shares outstanding (in shares) 0 0
Common stock, par value (in dollars per share) $ 0.0001 $ 0.0001
Common stock, shares authorized (in shares) 200,000,000 200,000,000
Common stock, shares issued (in shares) 87,452,835 75,367,117
Common stock, shares outstanding (in shares) 87,452,835 75,367,117
v3.25.0.1
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Total revenue $ 233,175 $ 145,238 $ 109,460
Operating expenses:      
Cost of goods sold 7,744 11,450 4,420
Research and development 217,496 244,990 227,333
Selling, general and administrative 264,119 265,542 197,520
In-process research and development 65,205 0 0
Restructuring 2,438 11,394 0
Total operating expenses 557,002 533,376 429,273
Operating loss (323,827) (388,138) (319,813)
Other income (expense), net:      
Interest income 17,817 21,768 6,276
Interest expense (11,182) (11,334) (11,014)
Other (expense) income, net (3,318) 1,594 974
Loss on extinguishment of debt 0 0 (7,578)
Total other income (expense), net 3,317 12,028 (11,342)
Loss from continuing operations before income tax provision (320,510) (376,110) (331,155)
Income tax provision on continuing operations (120) (223) (313)
Loss from continuing operations, net of tax (320,630) (376,333) (331,468)
(Loss) income from discontinued operations, net of tax (915) 264,934 52,986
Net loss $ (321,545) $ (111,399) $ (278,482)
Basic and diluted:      
Net loss from continuing operations, basic (in dollars per share) $ (4.07) $ (5.07) $ (5.20)
Net loss from continuing operations, diluted (in dollars per share) (4.07) (5.07) (5.20)
Net income from discontinued operations, basic (in dollars per share) (0.01) 3.57 0.83
Net income from discontinued operations, diluted (in dollars per share) (0.01) 3.57 0.83
Net income (loss) per common share, basic (in dollars per share) (4.08) (1.50) (4.37)
Net income (loss) per common share, diluted (in dollars per share) $ (4.08) $ (1.50) $ (4.37)
Weighted average common shares outstanding, basic (in shares) 78,888,861 74,267,418 63,758,515
Weighted average common shares outstanding, diluted (in shares) 78,888,861 74,267,418 63,758,515
Comprehensive loss:      
Net loss $ (321,545) $ (111,399) $ (278,482)
Unrealized gain (loss) on defined benefit pension plan 328 (374) (368)
Foreign currency translation gain (loss) 1,422 (1,871) 507
Unrealized (loss) gain on marketable debt securities (374) 3,696 (2,484)
Comprehensive loss (320,169) (109,948) (280,827)
Net product sales      
Total revenue 226,707 127,537 97,970
Operating expenses:      
Cost of goods sold 7,446 8,406 4,420
License and collaboration revenue      
Total revenue 6,468 17,701 11,490
Operating expenses:      
Cost of goods sold $ 298 $ 3,044 $ 0
v3.25.0.1
CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY - USD ($)
$ in Thousands
Total
Cumulative Effect, Period of Adoption, Adjustment
Underwritten Equity Offering
At-The-Market Offering
Common Stock
Common Stock
Underwritten Equity Offering
Common Stock
At-The-Market Offering
Additional Paid-in Capital
Additional Paid-in Capital
Cumulative Effect, Period of Adoption, Adjustment
Additional Paid-in Capital
Underwritten Equity Offering
Additional Paid-in Capital
At-The-Market Offering
Accumulated Other Comprehensive Loss
Accumulated Deficit
Accumulated Deficit
Cumulative Effect, Period of Adoption, Adjustment
Beginning balance (in shares) at Dec. 31, 2021         62,491,498                  
Beginning balance at Dec. 31, 2021 $ 302,112 $ (44,720)     $ 6     $ 1,068,634 $ (74,945)     $ (562) $ (765,966) $ 30,225
Increase (Decrease) in Stockholders' Equity [Roll Forward]                            
Share based compensation 37,897             37,897            
Issuance of common stock under the equity incentive plan and proceeds from exercise (in shares)         935,598                  
Issuance of common stock under the equity incentive plan and proceeds from exercise 4,585             4,585            
Employee stock purchase program stock purchase and expense (in shares)         161,874                  
Employee stock purchase program purchase and expense 4,259             4,259            
Issuance of common stock/equity offering, net of issuance costs (in shares)             701,600              
Issuance of common stock/equity offering, net of issuance costs       $ 19,545             $ 19,545      
Unrealized (gain) loss on defined benefit pension plan (368)                     (368)    
Foreign currency translation adjustments 507                     507    
Unrealized (loss) gain on marketable debt securities (2,484)                     (2,484)    
Net loss (278,482)                       (278,482)  
Ending balance (in shares) at Dec. 31, 2022         64,290,570                  
Ending balance at Dec. 31, 2022 42,851       $ 6     1,059,975       (2,907) (1,014,223)  
Increase (Decrease) in Stockholders' Equity [Roll Forward]                            
Share based compensation 43,985             43,985            
Issuance of common stock under the equity incentive plan and proceeds from exercise (in shares)         1,046,276                  
Issuance of common stock under the equity incentive plan and proceeds from exercise 3,240             3,240            
Employee stock purchase program stock purchase and expense (in shares)         326,521                  
Employee stock purchase program purchase and expense 4,853             4,853            
Issuance of common stock/equity offering, net of issuance costs (in shares)           9,703,750                
Issuance of common stock/equity offering, net of issuance costs     $ 191,199     $ 1       $ 191,198        
Issuance of pre-funded common stock warrants, net of issuance costs 24,630             24,630            
Unrealized (gain) loss on defined benefit pension plan (374)                     (374)    
Foreign currency translation adjustments (1,871)                     (1,871)    
Unrealized (loss) gain on marketable debt securities 3,696                     3,696    
Net loss (111,399)                       (111,399)  
Ending balance (in shares) at Dec. 31, 2023         75,367,117                  
Ending balance at Dec. 31, 2023 200,810       $ 7     1,327,881       (1,456) (1,125,622)  
Increase (Decrease) in Stockholders' Equity [Roll Forward]                            
Share based compensation 35,679             35,679            
Issuance of common stock under the equity incentive plan and proceeds from exercise (in shares)         1,455,575                  
Issuance of common stock under the equity incentive plan and proceeds from exercise 4,453       $ 1     4,452            
Employee stock purchase program stock purchase and expense (in shares)         395,768                  
Employee stock purchase program purchase and expense 3,566             3,566            
Issuance of common stock/equity offering, net of issuance costs (in shares)           8,984,375                
Issuance of common stock/equity offering, net of issuance costs     $ 134,738     $ 1       $ 134,737        
Exercise of pre-funded common stock warrants (in shares)         1,250,000                  
Unrealized (gain) loss on defined benefit pension plan 328                     328    
Foreign currency translation adjustments 1,422                     1,422    
Unrealized (loss) gain on marketable debt securities (374)                     (374)    
Net loss (321,545)                       (321,545)  
Ending balance (in shares) at Dec. 31, 2024         87,452,835                  
Ending balance at Dec. 31, 2024 $ 59,077       $ 9     $ 1,506,315       $ (80) $ (1,447,167)  
v3.25.0.1
CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY (Parenthetical) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Dec. 31, 2021
Accounting Standards Update [Extensible List]       Accounting Standards Update 2020-06 Cumulative Effect, Period of Adoption [Member]
Pre-funded common stock warrants, issuance costs   $ 1,600    
Issuance of common stock under the equity incentive plan and proceeds from exercise $ 4,453 3,240 $ 4,585  
Additional Paid-in Capital        
Issuance of common stock under the equity incentive plan and proceeds from exercise 4,452 3,240 4,585  
Underwritten Equity Offering        
Issuance costs $ 9,000 $ 12,600    
At-The-Market Offering        
Issuance costs     $ 600  
v3.25.0.1
CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Cash Flows From Operating Activities:      
Net loss $ (321,545) $ (111,399) $ (278,482)
Net (loss) income from discontinued operations (915) 264,934 52,986
Net loss from continuing operations (320,630) (376,333) (331,468)
Adjustments to reconcile net loss to net cash used in operating activities:      
Depreciation and amortization 43,555 38,530 20,719
Share based compensation 36,913 44,246 38,143
In-process research and development 65,205 0 0
Loss on extinguishment of debt 0 0 7,578
Loss on allowance for inventory 2,819 3,039 1,039
Amortization of debt discount and issuance costs 1,725 1,718 1,622
Amortization of discounts on investments (4,535) (7,456) (700)
Other 7,944 (3,626) 1,346
Changes in operating assets and liabilities:      
Accounts receivable (6,001) (11,264) (1,060)
Inventory (3,771) (39,420) (1,209)
Tax receivable (41) (35) (374)
Prepaid expenses and other current and non-current assets (3,164) (4,657) (5,196)
Change in lease assets and liabilities, net (949) (986) (984)
Accounts payable (17,563) 24,763 2,055
Accrued expenses (31,061) 22,321 22,181
Deferred revenue, current and non-current (6,116) (15,779) (12,929)
Other current and non-current liabilities 5,646 (418) (1,609)
Net cash used in operating activities - continuing operations (230,024) (325,357) (260,846)
Net cash (used in) provided by operating activities - discontinued operations (7,451) 45,336 74,555
Net cash used in operating activities (237,475) (280,021) (186,291)
Cash Flows From Investing Activities:      
Proceeds from the sale and maturity of marketable debt securities 326,334 334,575 381,993
Purchase of marketable debt securities (125,757) (443,942) (385,389)
Purchase of intangible assets (36,212) (41,591) (28,366)
Payment of milestone (65,000) 0 0
Other (40) (668) (791)
Net cash provided by (used in) investing activities - continuing operations 99,325 (151,626) (32,553)
Net cash provided by investing activities - discontinued operations 0 207,402 0
Net cash provided by (used in) investing activities 99,325 55,776 (32,553)
Cash Flows From Financing Activities:      
Payment of guaranteed minimum royalty (2,100) (2,100) (2,100)
Proceeds from issuance of 2029 convertible senior notes 0 0 316,250
Payment of debt issuance costs 0 0 (9,882)
Repurchase of 2025 convertible senior notes including premium 0 0 (211,324)
Proceeds from the issuance of common stock, net of issuance costs 134,738 191,198 0
Proceeds from the issuance of pre-funded warrants, net of issuance costs 0 24,630 0
Proceeds from exercise of stock options 4,452 3,240 4,585
Proceeds from the issuances under the employee stock purchase plan 2,332 3,166 2,978
Net cash provided by financing activities - continuing operations 139,422 220,134 120,052
Net cash used in financing activities - discontinued operations 0 (1,382) (2,479)
Net cash provided by financing activities 139,422 218,752 117,573
Effect of exchange rate changes on cash (913) 1,981 (2,794)
Net increase (decrease) in cash and cash equivalents 359 (3,512) (104,065)
Cash and cash equivalents, beginning of year 58,176 61,688 165,753
Cash and cash equivalents, end of year 58,535 58,176 61,688
Supplemental Disclosure of Cash Flow Information:      
Operating cash flows used for operating leases 6,392 6,315 6,020
Cash paid for interest 8,838 8,838 10,159
Cash paid for income taxes (453) (578) (996)
Non-cash investing and financing activities:      
Accrued royalty in excess of minimum payable to the sellers of Thiola 15,436 16,206 16,067
At-The-Market Offering      
Cash Flows From Financing Activities:      
Proceeds from issuance of common stock $ 0 $ 0 $ 19,545
v3.25.0.1
DESCRIPTION OF BUSINESS
 12 Months Ended
Dec. 31, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]  
DESCRIPTION OF BUSINESS DESCRIPTION OF BUSINESS
Organization and Description of Business
Travere Therapeutics, Inc. (“we”, “our”, “us”, “Travere” and the “Company”) refers to Travere Therapeutics, Inc., a Delaware corporation, as well as its subsidiaries. Travere is a fully integrated biopharmaceutical company headquartered in San Diego, California focused on identifying, developing and delivering life-changing therapies to people living with rare kidney and metabolic diseases. The Company regularly evaluates and, where appropriate, acts on opportunities to expand its product pipeline and approved products through licenses and acquisitions of products in areas that will serve rare disease patients with serious unmet medical need and that the Company believes offer attractive growth characteristics.
Discontinued Operations - Sale of Bile Acid Product Portfolio
In July 2023, Travere entered into an Asset Purchase Agreement (the “Purchase Agreement”) with Mirum Pharmaceuticals, Inc. ("Mirum Pharmaceuticals" or “Mirum”), pursuant to which Mirum agreed to purchase from Travere substantially all of the assets primarily related to Travere’s business of development, manufacture (including synthesis, formulation, finishing or packaging) and commercialization of Chenodal and Cholbam (also known as Kolbam, and together with Chenodal, the “Products”), collectively, the "bile acid business". On August 31, 2023, the Company and Mirum consummated the transactions contemplated by the Purchase Agreement (the “Closing”). In connection with the Closing, Mirum paid Travere an upfront cash payment of $210.0 million. Pursuant to the Purchase Agreement, Travere is eligible to receive up to $235.0 million upon the achievement of certain milestones based on specified amounts of annual net sales (tiered from $125.0 million to $500.0 million) of the Products. The Company has reflected the bile acid business as a discontinued operation in the Consolidated Financial Statements for all periods presented. See Note 19 for further discussion.
Unless otherwise noted, amounts and disclosures throughout the Notes to the Consolidated Financial Statements relate to the Company's continuing operations.
Approved Products:
FILSPARI® (sparsentan)
On September 5, 2024, the FDA granted full approval of FILSPARI® (sparsentan) to slow kidney function decline in adults with primary Immunoglobulin A nephropathy (IgAN) who are at risk of disease progression. FILSPARI is the only oral, once-daily, non-immunosuppressive medication that directly targets glomerular injury in the kidney by blocking two critical pathways of IgAN disease progression (endothelin-1 and angiotensin II).
FILSPARI had previously been granted accelerated approval in February 2023 based on the surrogate marker of proteinuria. Full approval is based on positive long-term confirmatory results from the PROTECT Study demonstrating that FILSPARI significantly slowed kidney function decline over two years compared to irbesartan.
In September 2021, the Company entered into a license and collaboration agreement with Vifor (International) Ltd. ("CSL Vifor"). In April 2024, the Company and CSL Vifor announced that the European Commission has granted conditional marketing authorization (“CMA”) for FILSPARI (sparsentan) for the treatment of adults with primary IgAN with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.75 g/g). The CMA is granted for all member states of the European Union, as well as in Iceland, Liechtenstein and Norway. The European Commission's decision follows the positive opinion from the Committee for Medicinal Products for Human Use (“CHMP”) in February 2024, based on results from the pivotal Phase 3 PROTECT Study of FILSPARI in IgAN. Under the terms of the License Agreement, the Company will be entitled to receive a regulatory milestone payment of $17.5 million upon receipt of full regulatory approval by the European Commission for IgAN, and an additional milestone payment upon achievement of market access initiatives in certain countries. FILSPARI became commercially available in Europe under the CMA in August 2024, with an initial launch in Germany and Austria. In October 2024, the Company and CSL Vifor announced that Swissmedic has granted temporary marketing authorization for FILSPARI for the treatment of adults with primary IgAN with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.75 g/g). In November 2024, the Medicines and Healthcare products Regulatory Agency (MHRA) approved FILSPARI in the United Kingdom.
In January 2024, the Company entered into an exclusive licensing agreement with Renalys Pharma, Inc. ("Renalys"), to bring sparsentan for the treatment of IgAN to patients in Japan and other countries in Asia, for the treatment of IgAN. Renalys will hold regional rights to sparsentan for Japan, South Korea, Taiwan, Brunei, Cambodia, Indonesia, Laos, Malaysia, Myanmar, the Philippines, Singapore, Thailand, and Vietnam. Following successful meetings with the Pharmaceuticals and Medical Devices Agency (PMDA) in 2023, in the second quarter of 2024 Renalys initiated an
open label registration study of sparsentan in Japan to support potential approval of sparsentan in Japan. In July 2024, Renalys announced that the first patient was dosed in the study, and in January 2025, Renalys announced achievement of full enrollment in the study. In December 2024, Renalys announced that sparsentan received Orphan Drug Designation from the Japanese Ministry of Health, Labour and Welfare for the indication of primary IgA nephropathy as of November 27, 2024. Under the terms of the licensing agreement, Renalys will be responsible for development, regulatory matters, and commercialization in the licensed territories.
Thiola® and Thiola EC® (tiopronin tablets)
Thiola® and Thiola EC® (tiopronin tablets) are approved in the United States for the prevention of cystine (kidney) stone formation in patients with severe homozygous cystinuria.
Clinical-Stage Programs:
Sparsentan for the treatment of FSGS
Sparsentan remains a novel investigational product candidate which has been granted Orphan Drug Designation for the treatment of focal segmental glomerulosclerosis (FSGS) in the U.S. and the European Economic Area countries (the “EEA”). In December 2023, the Company announced that it had completed a planned Type C meeting with the FDA to discuss previously reported results from the Phase 3 DUPLEX Study of sparsentan in FSGS. The FDA acknowledged the high unmet need for approved therapies as well as the challenges in studying FSGS but indicated that the two-year results from the Phase 3 DUPLEX Study alone were not sufficient to support an sNDA submission. In February 2025, the Company announced that it had completed a Type C meeting with the FDA and that the Company plans to submit an sNDA around the end of the first quarter of 2025. The sNDA will be based on existing data from the Phase 3 DUPLEX and Phase 2 DUET studies of FILSPARI.
Pegtibatinase
Pegtibatinase is a novel investigational human enzyme replacement candidate being evaluated for the treatment of classical homocystinuria (HCU). Pegtibatinase has been granted Rare Pediatric Disease, Fast Track and Breakthrough Therapy designations by the FDA, as well as orphan drug designation in the United States and European Union. In May 2023, the Company announced positive topline results from cohort 6 in the Phase 1/2 COMPOSE Study. In December 2023, the Company initiated the pivotal Phase 3 HARMONY Study to support the potential approval of pegtibatinase for the treatment of classical HCU. The HARMONY Study is a global, randomized, multi-center, double-blind, placebo-controlled Phase 3 clinical trial designed to evaluate the efficacy and safety of pegtibatinase as a novel treatment to reduce total homocysteine (tHcy) levels. In the beginning of 2024, the first patients were dosed in the HARMONY Study.
In September 2024, the Company announced a voluntary pause of enrollment in the Phase 3 HARMONY Study. The voluntary enrollment pause enables the Company to work to address necessary process improvements in manufacturing scale-up to support commercial scale manufacturing as well as full enrollment in the HARMONY Study. Patients currently enrolled in pegtibatinase studies continue to receive study medication from small scale batches which are unaffected by the scale-up process. Currently enrolled patients will be able to continue on study medication as scheduled for the duration of the trials they are participating in. The voluntary enrollment pause was enacted following our determination that the desired drug substance profile was not achieved in the recent scale-up process. The Company expects to further evaluate the necessary commercial process improvements to enable the continuation of the Phase 3 program.
The Company acquired pegtibatinase as part of the November 2020 acquisition of Orphan Technologies Limited.
Preclinical Programs:
The Company is party to a collaboration agreement with PharmaKrysto Limited and their early-stage cystinuria discovery program, whereby the Company is responsible for funding all research and development expenses for the pre-clinical activities associated with the cystinuria program.
v3.25.0.1
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES
 12 Months Ended
Dec. 31, 2024
Accounting Policies [Abstract]  
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES
A summary of the significant accounting policies applied in the preparation of the accompanying consolidated financial statements follows:
Principles of Consolidation
The consolidated financial statements represent the consolidation of the accounts of the Company, its subsidiaries and variable interest entities for which the Company has been determined to be the primary beneficiary, in conformity with accounting principles generally accepted in the United States ("U.S. GAAP"). All intercompany accounts and transactions have been eliminated in consolidation. See Note 5 for further discussion of variable interest entities (“VIE”) that the Company consolidates.
Use of Estimates
In preparing financial statements in conformity with U.S. GAAP, management is required to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements, as well as the reported amounts of expenses during the reporting period. Due to inherent uncertainty involved in making estimates, actual results reported in future periods may be affected by changes in these estimates. On an ongoing basis, the Company evaluates its estimates and assumptions. These estimates and assumptions include revenue recognition, forecasting probability-weighted cash flows based upon estimates of forecasted revenues, clinical and regulatory timelines and discount rates, valuing equity securities in share-based payments, estimating expenses of contracted research organizations, estimating reserves for inventory, estimating the useful lives of depreciable and amortizable assets, estimating of valuation allowances and uncertain tax positions, and estimates associated with the assessment of impairment for long-lived assets.
Revenue Recognition
The Company recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that an entity determines are within the scope of Accounting Standards Codification ("ASC") 606, Revenue from Contracts with Customers ("ASC 606"), the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company only recognizes revenue from contracts when it is probable that the entity will collect substantially all the consideration it is entitled to in exchange for the goods or services it transfers to the customer. See Note 3 and Note 4 for further discussion.
Payments received under collaboration and licensing agreements may include non-refundable fees at the inception of the arrangements, milestone payments for specific achievements and royalties on the sale of products. At the inception of arrangements that include milestone payments, the Company uses judgment to evaluate whether the milestones are probable of being achieved and estimates the amount to include in the transaction price utilizing the most likely amount method. If it is probable that a significant revenue reversal will not occur, the estimated amount is included in the transaction price. Milestone payments that are not within the Company or the licensee’s control, such as regulatory approvals, are considered to be constrained due to a high degree of uncertainty and are not included in the transaction price until such uncertainty is resolved. At the end of each reporting period, the Company re-evaluates the probability of achievement of development milestones and any related constraint and adjusts the estimate of the overall transaction price, if necessary. The Company recognizes aggregate sales-based milestones and royalty payments from product sales of which the license is deemed to be the predominant item to which the royalties relate, at the later of when the related sales occur or when the performance obligation has been satisfied. Revenue from collaboration and licensing agreements may also include sales of inventory, at cost plus a margin, which is recorded in license and collaboration revenue.
The Company utilizes significant judgment to develop estimates of the stand-alone selling price for each distinct performance obligation based upon the relative stand-alone selling price. Variable consideration that relates specifically to the Company’s efforts to satisfy specific performance obligations is allocated entirely to those performance obligations. The stand-alone selling price for license-related performance obligations requires judgment in developing assumptions to project probability-weighted cash flows based upon estimates of forecasted revenues, clinical and regulatory timelines and discount rates. The stand-alone selling price for clinical development performance obligations is based on forecasted expected costs of satisfying a performance obligation plus an appropriate margin.
If the licenses to intellectual property are determined to be distinct from the other performance obligations identified in the arrangement and have stand-alone functionality, the Company recognizes revenues from non-refundable, upfront fees allocated to the license when the license is transferred to the licensee and the licensee is able to benefit from the license. For licenses that are not distinct from other promises, the Company applies judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue from non-refundable, upfront fees. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the related revenue recognition accordingly.
The selection of the method to measure progress towards completion requires judgment and is based on the nature of the products or services to be provided. Revenue is recorded proportionally as costs are incurred. The Company generally utilizes the cost-to-cost method of progress because it best measures the transfer of control to the customer which occurs as the Company incurs costs. Under the cost-to-cost measure of progress, the extent of progress towards completion is measured based on the ratio of costs incurred to date to the total estimated costs at completion of the performance obligation. The Company uses judgment to estimate the total costs expected to complete the clinical development performance obligations, which include subcontractor costs, labor, materials, other direct costs and an allocation of indirect costs. The Company evaluates these cost estimates and the progress each reporting period and adjusts the measure of progress, if necessary.
Inventory, Related Reserves and Cost of Goods Sold
Inventory, which is recorded at the lower of cost or net realizable value, includes materials and other direct and indirect costs and is valued using the first-in, first-out method. The Company periodically analyzes its inventory levels to identify inventory that may expire prior to expected sale or has a cost basis in excess of its estimated realizable value, and writes down such inventory as appropriate. In addition, the Company's products are
subject to strict quality control and monitoring which the Company’s manufacturers perform throughout their manufacturing process. The Company does not directly manufacture any product. The Company has a single supplier for its product Thiola, and utilizes contract service providers for the manufacture of the active pharmaceutical ingredient for FILSPARI and the manufacture of primary packaging, secondary packaging and serialization for its product FILSPARI. The inventory reserve was $3.7 million and $2.4 million at December 31, 2024 and 2023, respectively.
Inventory, net of reserves, consisted of the following at December 31, 2024 and 2023 (in thousands):
December 31, 2024December 31, 2023
Raw materials$30,552 $33,790 
Work in process7,625 4,727 
Finished goods3,679 2,387 
Total inventory$41,856 $40,904 
Classified as:
Inventory$6,200 $9,410 
Long-term inventory35,656 31,494 
Total inventory$41,856 $40,904 
The balance classified as long-term inventory consists of raw materials, work in process and finished goods for both Thiola and FILSPARI as of December 31, 2024. The Company maintains levels of these inventories beyond a one-year production plan to limit exposure to potential supply disruption. Such inventories are classified as long-term.
Cost of goods sold includes the cost of inventory sold, third party manufacturing and supply chain costs, product shipping, tracking and handling costs, and provisions for excess and obsolete inventory. Cost of goods sold also includes the cost of goods sold under the Company's license and collaboration agreements, which generally consists of the sale of active pharmaceutical ingredients to the Company's collaboration partners, generally at cost plus a margin.
The following table summarizes cost of goods sold for the years ended December 31, 2024, 2023 and 2022 (in thousands):
Year Ended December 31,
202420232022
Cost of goods sold - product sales$7,446 $8,406 $4,420 
Cost of goods sold - license and collaboration298 3,044 — 
Total cost of goods sold$7,744 $11,450 $4,420 
Capitalization of Inventory Costs
Prior to the regulatory approval of the Company's drug candidates, the Company incurs expenses for the manufacture of drug product supplies to support clinical development that could potentially be available to support the commercial launch of those drugs. The Company capitalizes inventory costs associated with its products after regulatory approval, when, based on management’s judgment, future commercialization is considered probable and the future economic benefit is expected to be realized. Until the date at which regulatory approval has been received, costs related to the production of inventory are recorded as research and development expenses as incurred. Any eventual sale of previously expensed ("zero-cost") inventories may impact future margins, for any periods in which those inventories are sold.
Prior to the February 2023 FDA accelerated approval of FILSPARI (sparsentan), the Company expensed the production of active pharmaceutical ingredients purchased to support the commercial launch of FILSPARI, in research and development expenses. For the year ended December 31, 2024, sales of FILSPARI primarily consisted of zero-cost inventories. As of December 31, 2024, the Company had approximately $2.3 million of zero-cost inventory remaining, the majority of which the Company expects will be consumed in 2025. The Company began capitalizing inventory costs associated with FILSPARI following the February 2023 accelerated approval.
At December 31, 2024, the Company's evaluation of excess inventory and obsolescence resulted in a $1.0 million charge to cost of goods sold and an equal write-down of inventory balances due to lower forecasted sales of the Company's tiopronin products. For the year ended December 31, 2024, there were no charges recorded on FILSPARI inventory.
At December 31, 2023, the Company's evaluation of excess inventory and obsolescence considered certain minimum purchase obligations, which in combination with lower forecasted sales of FILSPARI resulted in a $3.2 million charge to cost of goods sold. The charge to cost of goods of sold included a $2.1 million write-down of inventory balances and $1.1 million accrued for firm purchase commitments.
Research and Development Expenses
Research and development includes expenses related to sparsentan, pegtibatinase, and the Company's other pipeline programs. The Company expenses all research and development costs as they are incurred. The Company's research and development costs are composed of salaries and bonuses, benefits, share-based compensation, license fees, milestones under license agreements, costs paid to third-party contractors to perform research, conduct clinical trials, costs to develop drug materials and delivery devices, costs to manufacture drug product supplies to support clinical development, and associated overhead expenses and facilities costs. The Company charges direct internal and external program costs to the respective development programs. The Company also incurs indirect costs that are not allocated to specific programs because such costs benefit multiple development programs and allow us to increase our pharmaceutical development capabilities. These consist of internal shared resources related to the development and maintenance of systems and processes applicable to all of our programs.
Nonrefundable advance payments for goods and services to be received in the future for use in research and development activities are recorded as prepaid expenses. The prepaid amounts are expensed as the related goods are delivered or the services are performed, or when it is no longer expected that the goods will be delivered or the services rendered.
During the three months ended December 31, 2022, the Company recorded an out of period adjustment that decreased research and development expenses and related accrued expenses by $7.7 million, $1.5 million of which was previously recorded in fiscal year 2022 and $6.2 million that was originally recorded in fiscal year 2021. The adjustment was the result of a certain pre-launch inventory contract that was not properly evaluated for accounting implications at inception. The Company evaluated the impact of the adjustment and concluded it is not material, individually and in the aggregate, to 2022 or any prior period financial statements.
Clinical Trial Expenses
The Company records expenses in connection with its clinical trials under contracts with contract research organizations ("CROs") that support conducting and managing clinical trials, as well as contract manufacturing organizations ("CMOs") for the manufacture of drug product supplies to support clinical development. The financial terms and activities of these agreements vary from contract to contract and may result in uneven expense levels. Generally, these agreements set forth activities that drive the recording of expenses such as start-up, initiation activities, enrollment, treatment of patients, or the completion of other clinical trial activities, and in the case of CMOs, costs associated with the production of drug product supplied and the procurement of raw materials to be consumed in the manufacturing process.
Expenses related to clinical trials are accrued based on our estimates of the progress of services performed, including actual level of patient enrollment, completion of patient studies and progress of the clinical trials or the delivery of goods. Other incidental costs related to patient enrollment or treatment are accrued when reasonably certain. If the amounts we are obligated to pay under our clinical trial agreements are modified (for instance, as a result of changes in the clinical trial protocol or scope of work to be performed), the Company adjusts its estimates accordingly on a prospective basis. Revisions to the Company's contractual payment obligations are charged to expense in the period in which the facts that give rise to the revision become reasonably certain.
The Company currently has four Phase 3 clinical trials in process that are in varying stages of activity, with ongoing non-clinical support trials. As such, clinical trial expenses will vary depending on all the factors set forth above and may fluctuate significantly from quarter to quarter.
Share-Based Compensation
The Company recognizes all employee share-based compensation as a cost within research and development expenses and selling, general, and administrative expenses. Equity-classified awards principally related to stock options, restricted stock units (“RSUs”) and performance stock units ("PSUs"), are measured at the grant date fair value of the award. The Company determines grant date fair value of stock option awards using the Black-Scholes option-pricing model. The fair value of RSUs and PSUs are determined using the closing price of the Company’s common stock on the grant date. For service based vesting grants, expense is recognized over the requisite service period. For PSUs, expense is recognized over the implicit service period, once vesting is probable. No expense is recognized for PSUs if it is not probable the vesting criteria will be satisfied. Forfeitures are accounted for as they occur.
Expiration TermVesting Term
Stock Options10 years
3 to 4 years
Restricted Stock Units----
1 to 4 years
Earnings (Loss) Per Share
The Company calculates basic earnings per share by dividing net income/(loss) by the weighted average number of shares outstanding during the period. Pre-funded warrants issued and sold by the Company to purchase shares of its common stock are included in the calculation of basic net loss per common share if the exercise price of the pre-funded warrant represents little consideration and is non-substantive in relation to the price paid for the warrant, and if the warrants are immediately exercisable with no further vesting conditions or contingencies associated with them.
The Company's diluted earnings/(loss) per share computation includes the effect, if any, of shares that would be issuable upon the exercise of outstanding stock options, convertible debt and RSUs, reduced by the number of shares which are assumed to be purchased by the Company from the resulting proceeds at the average market price during the year, when such amounts are dilutive to the earnings per share calculation. In accordance with ASC 260, Earnings per Share, if a company had a discontinued operation, the company uses income from continuing operations, adjusted for preferred dividend and similar adjustments, as its control number to determine whether potential common shares are dilutive.
Cash and Cash Equivalents
The Company considers all highly liquid marketable securities with an original maturity of three months or less to be cash equivalents. Due to the short-term maturity of such investments, the carrying amounts are a reasonable estimate of fair value.
Concentration of Credit Risk
The Company maintains its cash and cash equivalents at insured financial institutions, the balances of which may, at times, exceed federally insured limits. Generally, these deposits may be redeemed upon demand, and the Company believes there is minimal risk of losses on such balances.
The Company monitors its investments with counterparties with the objective of minimizing concentrations of credit risk. The Company's investment policy is to invest only in institutions that meet high credit quality standards and established limits on the amount and time to maturity of investments with any individual counterparty. The policy also requires that investments are only entered into with corporate and financial institutions that meet high quality standards.
Marketable Debt Securities
The Company classified marketable debt securities held as “available-for-sale” and carries them at fair value. The Company classifies these investments as current assets, even if the maturity when acquired by the Company is greater than one year due to the ability to liquidate within the next 12 months. The amortized cost of marketable debt securities is adjusted for amortization of premiums and accretion of discounts to maturity. Such amortization and accretion, as well as interest and dividends, is included in interest income. Unrealized gains and losses on marketable debt securities are recorded as a separate component of stockholders’ equity as accumulated other comprehensive loss, unless an impairment is determined to be the result of credit-related factors or the Company intends to sell the security or it is more likely than not that the Company will be required to sell the security before recovery. Unrealized losses that are determined to be credit-related are recorded as an allowance against the amortized cost basis. Realized gains or losses on debt security transactions and declines in value that are determined to be the result of credit losses, if any, are reported in other income or expense in the Consolidated Statements of Operations and Comprehensive Loss. The cost of securities sold is based on the specific identification method. Marketable debt securities are maintained at one financial institution and are governed by the Company’s investment policy. See Note 6 for further discussion.
Accounts Receivables, Net
Trade accounts receivable are recorded net of reserves for prompt pay discounts and expected credit losses. Estimates for allowances for credit losses are determined based on existing contractual obligations, historical payment patterns and individual customer circumstances. The allowance for credit losses was zero at both December 31, 2024 and 2023, respectively. For the years ended December 31, 2024, 2023 and 2022, bad debt expense recorded in the Consolidated Statements of Operations was immaterial. The Company's evaluation of credit losses for the current period included an assessment of our aged trade receivables balances and their underlying credit risk characteristics. Our evaluation of past events, current conditions, and reasonable and supportable forecasts about the future resulted in an expectation of immaterial credit losses.
Supplier Concentration Risk
The Company has no manufacturing capabilities and relies on third party manufacturers who are sole source suppliers for manufacturing of its products. The Company intends to rely on third-party manufacturers for the long-term commercial supply of FILSPARI and for its development stage product candidates, including sparsentan for the treatment of FSGS and pegtibatinase. The Company expects the manufacturers of each product or product candidate to, at least initially and potentially for a significant period of time, be single source suppliers to the Company.
Property and Equipment, net
Property and equipment are stated at cost, net of accumulated depreciation. Depreciation is computed using the straight-line method over the related estimated useful lives as presented in the table below. Significant additions and improvements are capitalized, while repairs and maintenance are charged to expense as incurred. Property and equipment purchased for specific research and development projects with no alternative use is expensed as incurred.
The major classifications of property and equipment, including their respective expected useful lives, consist of the following:
Computers and equipment3 years
Furniture and fixtures7 years
Leasehold improvementsShorter of length of lease or life of the asset
Leases
The Company determines whether a contract is, or contains, a lease at inception. The Company classifies each of its leases as operating or financing considering factors such as the length of the lease term, the present value of the lease payments, the nature of the asset being leased, and the potential for ownership of the asset to transfer during the lease term. Leases with terms greater than one year are recognized on the Consolidated Balance Sheets as Right-of-use assets and Lease liabilities and are measured at the present value of the fixed payments due over the expected lease term minus the present value of any incentives, rebates or abatement expected to be received from the lessor. Options to extend a lease are typically excluded from the expected lease term as the exercise of the option is typically not reasonably certain. The interest rate implicit in lease contracts is typically not readily determinable. As such, the Company utilizes the appropriate incremental borrowing rate, which is the rate incurred to borrow on a collateralized basis an amount equal to the lease payments over a similar term and in a similar economic environment.
In addition to rent, the leases may require the Company to pay additional amounts for taxes, insurance, maintenance, and other expenses, which are generally referred to as non-lease components. The Company has elected to not separate lease and non-lease components. Only the fixed costs for lease components and their associated non-lease components are accounted for as a single lease component and recognized as part of the Right-of-use assets and Lease liabilities. The Company records expense to recognize fixed lease payments, including payment escalation, on a straight-line basis over the expected lease term. Costs determined to be variable and not based on an index or rate are not included in the measurement of the lease liability and are expensed as incurred.
The Company has made an accounting policy election to not recognize short-term leases, or leases that have a lease term of 12 months or less at commencement date, within its Consolidated Balance Sheets and to recognize those lease payments in the Consolidated Statements of Operations on a straight-line basis over the lease term.
The Company recognizes income from sublet office space on a straight-line basis over the term of the sublease, recorded in other income in the Consolidated Statements of Operations.
Intangible Assets, Net
The Company's intangible assets consist of licenses and purchased technology. Intangible assets with definite lives are amortized on a straight-line basis over their estimated useful lives and are reviewed periodically for impairment.
Intangible Assets with Cost Accumulation Model
In 2014, the Company entered into a license agreement with Mission Pharmacal ("Mission") in which the Company obtained the exclusive right to license the trademark of Thiola ("Mission License Agreement"). The acquisition of the Thiola license qualified as an asset acquisition under the principles of ASC 805, Business Combinations ("ASC 805") in effect at the time of acquisition. The license agreement requires the Company to make royalty payments based on net sales of Thiola. The liability for royalties in excess of the annual contractual minimum is recognized in the period in which the royalties become probable and estimable, which is typically in the period corresponding with the respective sales. The Company records an offsetting increase to the cost basis of the intangible asset under the cost accumulation model ("Thiola Intangible"). The additional cost basis is subsequently amortized over the remaining estimated useful life of the license agreement.
In the second quarter of 2023, the Company reduced the estimated useful life of the Thiola Intangible to better reflect the pattern of projected future cash flows, resulting in incremental expense of $3.7 million recorded in selling, general, and administrative. The change in estimated useful life was accounted for as a change in accounting estimate and the remaining carrying amounts of the Thiola Intangible are being amortized prospectively over the new useful life.
Consistent with all prior periods since Thiola was acquired, the Company has not accrued any liability for future royalties in excess of the annual contractual minimum at December 31, 2024 as such royalties are not yet probable and estimable.
In 2012, the Company entered into an agreement with Ligand Pharmaceuticals, Inc. (“Ligand”) for a worldwide sublicense to develop, manufacture and commercialize sparsentan (the “Ligand License Agreement”). The acquisition of the Ligand License Agreement qualified as an asset acquisition under the principles of ASC 805 in effect at the time of acquisition. The license agreement requires the Company to make royalty payments based on net sales of FILSPARI (sparsentan) and milestone payments. The liabilities for royalties and milestone payments are recognized in the period in which they become probable and estimable, which is typically in the period corresponding with the respective sales or achievement of the milestone.
The Company records an offsetting increase to the cost basis of the intangible asset under the cost accumulation model following the approval of FILSPARI. The additional cost basis is subsequently amortized over the remaining estimated useful life.
Variable Interest Entity
The Company reviews each investment and collaboration agreement to determine if it has a variable interest in the entity. In assessing whether the Company has a variable interest in the entity as a whole, the Company considers and makes judgments regarding the purpose and design of the entity, the value of the licensed assets to the entity, the value of the entity’s total assets and the significant activities of the entity. If the Company has a variable interest in the entity as a whole, the Company assesses whether or not the Company is a primary beneficiary of that VIE, based on a number of factors, including: (i) which party has the power to direct the activities that most significantly affect the VIE’s economic performance, (ii) the parties’ contractual rights and responsibilities pursuant to the collaboration agreement, and (iii) which party has the obligation to absorb losses of or the right to receive benefits from the VIE that could be significant to the VIE. If the Company determines that it is the primary beneficiary of a VIE at the onset of the collaboration, the collaboration is treated as a business combination and the Company consolidates the financial statements of the VIE into the Company’s consolidated financial statements. On a quarterly basis, the Company evaluates whether it continues to be the primary beneficiary of the consolidated VIE. If the Company determines that it is no longer the primary beneficiary of a consolidated VIE, it deconsolidates the VIE in the period in which the determination is made.
Assets and liabilities recorded as a result of consolidating the financial results of the VIE into the Company’s consolidated balance sheet do not represent additional assets that could be used to satisfy claims against the Company’s general assets or liabilities for which creditors have recourse to the Company’s general assets.
Equity Securities
The Company applies the equity method of accounting for investments when it has significant influence, but no controlling interest in the investee. Judgment regarding the level of influence over each equity method investment includes key factors such as ownership interest, representation on the board of directors, participation in joint steering committees and material intercompany transactions. Upon investment, the Company evaluates any basis difference between the carrying value and fair value of the Company's proportionate share of the investee's net assets. Basis differences relating to in-process research and development (IPR&D) are expensed when the investee is not considered a business as defined in ASC 805, Business Combinations, due to substantially all of the estimated fair value of the gross assets being concentrated in a group of similar IPR&D assets with no alternative future use. For the year ended December 31, 2024, the Company recognized $3.4 million in other (expense) income, net in the Company's Consolidated Statements of Operations for these basis adjustments. The equity method investment's carrying value was reduced to zero as the Company's proportionate share of the basis difference exceeded the carrying value. See Note 5 for further discussion. Investments accounted for using the equity method are be reported on a lag of up to three months if the financial statements of the investee are not available in sufficient time for the Company to apply the equity method as of the current reporting date.
Goodwill
Goodwill represents the excess of purchase price over fair value of net assets acquired in a business combination and is not amortized. Goodwill is subject to impairment testing at least annually or when a triggering event occurs that could indicate a potential impairment. The Company has one segment and one reporting unit and as such reviews goodwill for impairment at the consolidated level.
Impairment of Long-Lived Assets
The Company's long-lived assets are primarily comprised of intangible assets, right-of-use assets, and property and equipment. The Company evaluates its finite-lived intangible assets, right-of-use assets, and property and equipment for impairment whenever events or changes in circumstances indicate the carrying value of an asset or group of assets may not be recoverable. If these circumstances exist, recoverability of assets to be held and used is measured by a comparison of the carrying amount of an asset group to future undiscounted net cash flows expected to be generated by the use and eventual disposition of the asset group. If such assets are considered to be impaired, the impairment to be recognized is measured by the amount by which the carrying amount of the assets exceeds the fair value of the assets.
In addition, indefinite-lived intangible assets are reviewed for impairment annually and whenever events or changes in circumstances indicate that it is more likely than not that the asset is impaired by comparing the fair value to the carrying value of the asset. To determine the fair value of the asset, the Company used the multi-period excess earnings method of the income approach. The more significant assumptions inherent in the application of this method include: the amount and timing of projected future cash flows (including revenue, cost of sales, research and development costs, and sales and marketing expenses), and the discount rate selected to measure the risks inherent in the future cash flows. See Note 8 for further discussion of certain long-lived assets measured at fair value on a nonrecurring basis when there are indicators of impairment.
Income Taxes
The Company follows ASC 740, Income Taxes ("ASC 740"), which requires recognition of deferred tax assets and liabilities for the expected future tax consequences of events that have been included in the financial statements or tax returns. Under this method, deferred tax assets and liabilities are based on the differences between the financial statement and tax basis of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. Deferred tax assets are reduced by a valuation allowance to the extent management concludes it is more likely than not that the asset will not be realized.
The standard addresses the determination of whether tax benefits claimed or expected to be claimed on a tax return should be recorded in the financial statements. Under ASC 740, the Company may recognize the tax benefit from an uncertain tax position only if it is more likely than not that the tax position will be sustained on examination by the tax authorities, based on the technical merits of the position. The tax benefits recognized in the financial statements from such a position should be measured based on the largest benefit that has a greater than fifty percent likelihood of being realized upon ultimate settlement. ASC 740 also provides guidance on de-recognition, classification, interest and penalties on income taxes, accounting in interim periods and requires increased disclosures. The Company’s policy is to record estimated interest and penalty related to the underpayment of income taxes or unrecognized tax benefits as a component of its income tax provision.
Foreign Currency Translation
Functional and presentation currency
Items included in the financial statements of each entity comprising the Company are measured using the currency of the primary economic environment in which the entity operates (the functional currency).
Transactions and balances
Foreign currency transactions in each entity comprising the Company are remeasured into the functional currency of the entity using the exchange rates prevailing at the respective transaction dates. Foreign exchange gains and losses resulting from the settlement of such transactions and from the remeasurement at year-end exchange rates of monetary assets and liabilities denominated in foreign currencies are recognized within Other income (expense), net in the Consolidated Statements of Operations and Comprehensive Loss.
An aggregate loss of $0.7 million arising from foreign exchange transactions is included in other (expense) income, net for the year ended December 31, 2024. An aggregate gain of $0.5 million and $1.4 million is included in other (expense) income, net for the years ended December 31, 2023 and 2022, respectively.
The results and financial position of the Company that have a functional currency different from the U.S. dollar are translated as follows:
a.assets and liabilities presented are translated at the closing exchange rate as of December 31, 2024 and 2023;
b.income and expenses for the statements of operations and comprehensive loss are translated at average exchange rates that are relevant for the respective periods for which the income and expenses occurred; and
c.significant transactions use the exchange rate on the date of the transaction.
All resulting exchange differences arising from such translations are recognized directly in comprehensive income and presented as a separate component of equity.
The following table summarizes the foreign currency translation adjustment included in accumulated other comprehensive loss for the year ended December 31, 2024, 2023 and 2022 (in thousands):
Foreign Currency Translation Adjustments included in Accumulated Other Comprehensive (Loss) Gain
202420232022
Balance at January 1,$(1,456)$415 $(92)
Foreign currency translation adjustments1,422 (1,871)507 
Balance at December 31,$(34)$(1,456)$415 
Reclassifications 
Certain reclassifications have been made to the prior year financial statements in order to conform to the current year’s presentation. These reclassifications did not have an impact on total assets or total liabilities and stockholders' equity in the Consolidated Balance Sheets or net loss in the Consolidated Statements of Operations.
Patents
The Company expenses external costs, such as filing fees and associated attorney fees, incurred to obtain issued patents and patent applications pending. The Company also expenses costs associated with maintaining and defending patents subsequent to their issuance in the period incurred. 
Legal Contingencies
The Company may, from time to time, be involved in various claims and legal actions that arise in the ordinary course of business. The Company accrues for legal contingencies when it is determined probable that a liability has been incurred and the amount of the loss can be reasonably estimated. See Note 11 for further discussion.
Discontinued Operations
Discontinued operations is presented when there is a disposal of a component or a group of components that in the Company's judgment represents a strategic shift that will have a major effect on the Company's operations and financial results. Results of operations directly related to discontinued operations are aggregated into a single line item in the Consolidated Statements of Operations for all periods presented. See Note 19 for further discussion.
Restructuring
Restructuring charges consist primarily of employee severance, one-time termination benefits related to the reduction of its workforce, and other costs. Liabilities for costs associated with a restructuring activity are recognized when the liability is incurred and are measured at fair value. One-time termination benefits are expensed at the date the entity notifies the employee, unless the employee must provide future service, in which case the benefits are expensed ratably over the service period. Termination benefits are calculated based on regional benefit practices and local statutory requirements.
In December 2023, the Company initiated a restructuring plan that resulted in a reduction of its workforce, primarily impacting non-field-based employees. Restructuring costs were primarily comprised of one-time termination benefits, including severance, continuation of health insurance coverage, and other benefits for a specified period of time. In 2024, we also recognized charges for impairment of operating lease right-of-use assets and related leasehold improvements, as well as disposal costs on furniture and fixtures associated with available office space that the Company has sublet as a result of the reduction in occupancy. As of December 31, 2024, we are no longer incurring restructuring expenses. Of the $13.8 million recognized to date, $2.4 million was recognized during the year ended December 31, 2024, including $1.2 million related to the impairment and disposal costs and initial direct costs to obtain the sublease.
The following table summarizes the cash payments and accruals, included in accrued expenses of the Consolidated Balance Sheets, related to the restructuring for the years ended December 31, 2024 and 2023 (in thousands):
20242023
Liability balance at January 1,$11,421 $— 
Restructuring expenses2,438 11,394 
Non-cash impairment and disposal charges(856)— 
Payments(12,929)— 
Foreign currency impact(74)27 
Liability balance at December 31,$— $11,421 
Recently Adopted Accounting Pronouncements
In November 2023, the FASB issued ASU No. 2023-07, Improvements to Reportable Segment Disclosures. The FASB amended the guidance in ASC 280, Segment Reporting ("ASC 280"), to require a public entity to disclose significant segment expenses and other segment items on an annual and interim basis and to provide in interim periods all disclosures about a reportable segment's profit or loss and assets that are currently required annually. Public entities with a single reportable segment are required to provide the new disclosures and all the disclosures required under ASC 280. The guidance is applied retrospectively to all periods presented in financial statements, unless it is impracticable. This new guidance is effective for public business entities for annual periods beginning after December 15, 2023, and for interim periods beginning after December 15, 2024. The Company adopted this new standard effective December 31, 2024. See Note 20, Segment Information, for disclosures related to the adoption of ASU 2023-07.
In August 2020, the FASB issued ASU No. 2020-06, Accounting for Convertible Instruments and Contracts in an Entity's Own Equity. The ASU includes amendments to the guidance on convertible instruments and the derivative scope exception for contracts in an entity's own equity in Subtopic 815-40 and simplifies the accounting for convertible instruments which include beneficial conversion features or cash conversion features by removing certain separation models in Subtopic 470-20. Additionally, the ASU requires entities to use the "if-converted" method when calculating diluted earnings per share for convertible instruments. The adoption of the new standard impacted the Company's accounting for its Convertible Senior Notes Due 2025 (2025 Notes), discussed in Note 7, which were previously accounted for using the cash conversion model applied under ASC 470-20, Debt with Conversion and Other Options ("ASC 470-20"). The Company adopted ASU 2020-06 on January 1, 2022 using the modified retrospective method. The cumulative effect of the accounting change as of January 1, 2022 increased the carrying amount of the 2025 Notes by $44.7 million, reduced additional paid-in capital by $74.9 million, and reduced accumulated deficit by $30.2 million.
Recently Issued Accounting Pronouncements
From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board ("FASB") or other standard setting bodies. Unless otherwise discussed, the Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on its consolidated financial position or results of operations upon adoption.
In November 2024, the FASB issued ASU No. 2024-04, Debt with Conversion and Other Options (Subtopic 470-20): Induced Conversions of Convertible Debts Instruments. This ASU clarifies the requirements for determining whether to account for certain early settlements of convertible debt instruments as induced conversions or extinguishments. The guidance is effective for fiscal years beginning after December 15, 2025, with early adoption permitted for entities that have adopted 2020-06. The Company is currently evaluating the impact of the adoption of this standard on the accounting for the Company's convertible notes.
In November 2024, the FASB issued ASU No. 2024-03, Income Statement - Reporting Comprehensive Income - Expense Recognition Disclosures. This ASU will require entities to provide enhanced disclosures related to certain expense categories included in income statement captions. The ASU aims to increase transparency and provide investors with more detailed information about the nature of expenses reported on the face of the income statement. The new standard does not change the requirements for the presentation of expenses on the face of the income statement. Under this ASU, entities are required to disaggregate, in a tabular format, expense captions presented on the face of the income statement — excluding earnings or losses from equity method investments — if they include any of the following expense categories: purchases of inventory, employee compensation, depreciation, intangible asset amortization, and depreciation or depletion. For any remaining items within each relevant expense caption, entities must provide a qualitative description of the nature of those expenses. The new ASU is effective for annual reporting periods beginning after December 15, 2026 and interim reporting periods beginning after December 15, 2027. Early adoption is permitted. The Company is currently evaluating the impact of the adoption of this standard on the related disclosures.
In December 2023, the FASB issued ASU No. 2023-09, Improvements to Income Tax Disclosures. This ASU does not change accounting for income taxes but requires new disclosures focusing on two areas, the effective rate reconciliation and taxes paid. This new standard is effective for public business entities for annual periods beginning after December 15, 2024. Early adoption is permitted. The Company is currently evaluating the impact of the adoption of this standard on the related disclosures.
v3.25.0.1
REVENUE RECOGNITION
 12 Months Ended
Dec. 31, 2024
Revenue from Contract with Customer [Abstract]  
REVENUE RECOGNITION REVENUE RECOGNITION
Product Sales, Net
Product sales consist of FILSPARI and tiopronin products (Thiola and Thiola EC). The Company sells its products to specialty pharmacies and through direct-to-patient distributors worldwide, with the United States representing over 98% of net product sales.
The Company sells FILSPARI to two direct-to-patient specialty pharmacies in the United States. The Company sells its tiopronin products to patients and pharmacies, with distribution facilitated through a single direct-to-patient distributor. Revenues from product sales are recognized in satisfaction of a single performance obligation when the customer obtains control of the Company’s product. For FILSPARI, sales are recognized upon delivery of the product to the specialty pharmacies. The Company receives payments from its FILSPARI sales based on terms that are generally 30 days from shipment of the product to the specialty pharmacy. For the Company's tiopronin products, product sales are recognized upon delivery to the patient. The Company receives payments from sales of its tiopronin products, primarily through third party payers, based on terms that generally are within 30 days of delivery of product to the patient. Contracts do not contain significant financing components based on the typical period of time between performance of services and collection of consideration.
Deductions from Revenue
Revenues from product sales are recorded at the net sales price, which includes provisions resulting from discounts, rebates and co-pay assistance that are offered to customers, payers and other indirect customers relating to the Company’s sales of its products. These provisions are based on the estimates of the amounts earned or to be claimed on the related sales. These amounts are treated as variable consideration, estimated and recognized as a reduction of the transaction price at the time of the sale, using the most likely amount method, and are classified as a reduction of accounts receivable (if the amount is payable to a customer) or as a current liability (if the amount is payable to a party other than a customer). The Company includes these estimated amounts in the transaction price to the extent it is probable that a significant reversal of cumulative revenue recognized for such transactions will not occur. Where appropriate, these reserves take into consideration the Company’s historical experience, current contractual and statutory requirements and specific known market events and trends. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the contract. If actual results in the future vary from the Company’s provisions, the Company will adjust the estimate, which would affect net product revenue and earnings in the period such variances become known. For the years ended December 31, 2024, 2023 and 2022, adjustments to net product revenue related to performance obligations satisfied in previous periods were $0.5 million, $0.4 million, and $0.2 million, respectively.
Government Rebates: The Company calculates the rebates that it will be obligated to provide to government programs and deducts these estimated amounts from its gross product sales at the time the revenues are recognized. Allowances for government rebates and discounts are established based on an estimated allocation of payers and the government-mandated discounts applicable to government-funded programs. Rebate discounts are included in accrued expenses in the accompanying consolidated balance sheets.
Commercial Rebates: The Company calculates the rebates it incurs according to any contracts with certain commercial payers and deducts these amounts from its gross product sales at the time the revenues are recognized. Allowances for commercial rebates are established based on actual payer information, which is reasonably estimated at the time of delivery for applicable products. Rebate discounts are included in accrued expenses in the accompanying consolidated balance sheets.
Prompt Pay Discounts: The Company offers discounts to certain customers for prompt payments. The Company accrues for the calculated prompt pay discount based on the gross amount of each invoice for those customers at the time of sale.
Other Fees: The Company pays service fees to certain customers based on a contractually fixed percentage of the wholesale acquisition cost and fees for data. Other fees are recorded as an offset to revenue based on contractual terms at the time revenue from the sale is recognized.
Product Returns: Consistent with industry practice, the Company offers its customers a limited right to return product purchased directly from the Company, which is principally based upon the product’s expiration date. Historically, returns have been immaterial.
Co-pay Assistance: The Company offers a co-pay assistance program, which is intended to provide financial assistance to qualified commercially insured patients with prescription drug co-payments required by payers. The calculation of the accrual for co-pay assistance is based on an estimate of claims and the estimated cost per claim associated with product that has been recognized as revenue.
The following table summarizes net product sales for the year ended December 31, 2024, 2023 and 2022 (in thousands):
Year Ended December 31,
202420232022
FILSPARI$132,222 $29,208 $— 
Tiopronin products94,485 98,329 97,970 
Total net product sales$226,707 $127,537 $97,970 
v3.25.0.1
COLLABORATION AND LICENSE AGREEMENTS
 12 Months Ended
Dec. 31, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]  
COLLABORATION AND LICENSE AGREEMENTS COLLABORATION AND LICENSE AGREEMENTS
License Agreement with CSL Vifor
In September 2021, the Company entered into a license and collaboration agreement (“License Agreement”) with Vifor (International) Ltd. (“CSL Vifor”), pursuant to which the Company granted an exclusive license to CSL Vifor for the commercialization of sparsentan in Europe, Australia and New Zealand ("Licensed Territories"). CSL Vifor also has first right of negotiation to expand the licensed territories into Canada, China, Brazil and/ or Mexico. Under the terms of the License Agreement, the Company received an upfront payment of $55.0 million and will be eligible for up to $135.0 million in aggregate regulatory and market access related milestone payments and up to $655.0 million in aggregate sales-based milestone payments for a total potential value of up to $845.0 million. The Company is also entitled to receive tiered double-digit royalties of up to 40 percent of annual net sales of sparsentan in the Licensed Territories.
Under the License Agreement, CSL Vifor is responsible for all commercialization activities in the Licensed Territories. The Company remains responsible for the worldwide clinical development of sparsentan through regulatory approval as defined and will retain all rights to sparsentan in the United States and rest of world outside of the Licensed Territories. Development costs for any post regulatory approval development activities, subject to approval by both parties, will be borne by the Company and CSL Vifor as defined, respectively. The License Agreement will remain in effect, unless terminated earlier, until the expiration of all royalty terms for sparsentan in the licensed territories. Each party has the right to terminate the License Agreement for the other party’s uncured material breach, insolvency or if the time required for performance under the License Agreement by the other party is extended due to a force majeure event that continues for six months or more.
The Company assessed the License Agreement and determined that it meets both criteria to be considered a collaborative agreement within the Scope of ASC 808, Collaborative Arrangements of active participation by both parties and exposures to significant risks and rewards dependent on the commercial success of the activities. Both parties participate on joint steering and other committees overseeing the collaboration activities. Also, both parties are exposed to significant risks and rewards based on the economic outcomes of regulatory approvals and commercialization of sparsentan.
The Company determined the transaction price under the License Agreement totaled $55.0 million, consisting of the fixed non-refundable upfront payment. The variable regulatory and access related milestones were excluded from the transaction price given the substantial uncertainty related to their achievement. Sales-based milestone payments and royalties on net sales were excluded from the transaction price and are recognized at the later of when the related sales occur or when the performance obligation to which the sales-based milestone or royalty has been allocated have been satisfied.
The Company concluded that CSL Vifor represented a customer and applied relevant guidance from ASC 606 to evaluate the accounting under the License Agreement. In accordance with this guidance, the Company concluded that the promise to grant the license is distinct from the promise to provide clinical development services resulting in two performance obligations. As a result, the Company allocated $12.0 million of the transaction price, based on the performance obligations' relative standalone selling prices, to the license, which was recognized in full in 2021. The remaining $43.0 million of the transaction price was allocated to the clinical development activities and recorded as deferred revenue, which are being recognized over the development period based upon the ratio of costs incurred to date to the total estimated costs.
For the year ended December 31, 2024, the Company recognized $5.8 million in license and collaboration revenue for clinical development activities, based upon the ratio of costs incurred to total estimated costs, and $0.6 million for royalties earned in 2024 on net sales of FILSPARI in the CSL Vifor Licensed Territories following the August 2024 launch. For the year ended December 31, 2023, the Company recognized a total of $17.7 million in license and collaboration revenue, which consisted of $3.3 million from the sale of active pharmaceutical ingredients to CSL Vifor, and $14.4 million for clinical development activities, based upon the ratio of costs incurred to total estimated costs. A decrease in total costs for clinical development activities due to the restructuring announced in December 2023 resulted in a $2.9 million increase in amortization of deferred revenue under the cost-to-cost model for the year ended December 31, 2023.
Deferred revenue related to the clinical development activities as of December 31, 2024 was $2.8 million, classified as current as the Company estimates that the remainder of the deferred revenue balance will be fully realized by mid-2025. As of December 31, 2023, deferred revenue related
to the clinical development activities was $8.9 million, of which $7.1 million was classified as current. The $1.8 million classified as non-current as of December 31, 2023 is included in other non-current liabilities in the Consolidated Balance Sheets.
The following table sets forth a summary of changes in deferred revenue for the years ended December 31, 2024, 2023 and 2022 (in thousands):
Deferred Revenue
202420232022
Balance at January 1,$8,931 $22,907 $36,647 
License and collaboration revenue(5,847)(14,363)(11,490)
Foreign currency impact(269)387 (2,250)
Balance at December 31,$2,815 $8,931 $22,907 
Licensing Agreement with Renalys
In January 2024, the license agreement (“Renalys License Agreement”) between the Company and Renalys Pharma, Inc. (“Renalys”) came into effect. Pursuant to the terms of the Renalys License Agreement, the Company granted an exclusive license to Renalys for the development and commercialization of sparsentan in Japan, South Korea, Taiwan and other specified Asian countries ("Renalys Licensed Territories"). Under the terms of the Renalys License Agreement, the Company received a non-refundable upfront payment and will be eligible to receive up to $120.0 million in aggregate development and sales-based milestones. The Company is also entitled to receive tiered double-digit to mid-20 percent royalties of annual net sales of sparsentan in the Renalys Licensed Territories. In addition, the Company received an option to purchase shares of common stock of Renalys (“Option Agreement”), which it exercised in January 2024. The Company also has the option to purchase all equity securities of Renalys at any time prior to the top-line results of the Phase 3 trial in Japan (“Buyout Right”).
Under the Renalys License Agreement, Renalys will be responsible for all development and commercialization activities in the Renalys Licensed Territories. The Renalys License Agreement will remain in effect, unless terminated earlier, until the expiration of all royalty terms for sparsentan in the Renalys Licensed Territories. Each party has the right to terminate the Renalys License Agreement for the other party’s uncured material breach or insolvency, or if the time required for performance under the Renalys License Agreement by the other party is extended due to a force majeure event that continues for nine months or more. Renalys may terminate the Renalys License Agreement for any reason upon prior written notice to the Company. The Company may terminate the Renalys License Agreement if Renalys abandons development in Japan or South Korea prior to first commercial sales of sparsentan in either Japan or South Korea.
The Company concluded that Renalys represents a customer and applied relevant guidance from ASC 606 to evaluate the accounting under the Renalys License Agreement.
The Company determined the transaction price under the Renalys License Agreement totaled $8.3 million, consisting of the fixed non-refundable upfront payment, milestone payment and estimated fair value of the Option Agreement. The variable development-related milestones were excluded from the transaction price given the substantial uncertainty related to their achievement. Sales-based milestone payments and royalties on net sales were excluded from the initial transaction price and will be recognized at the later of when the related sales occur or when the performance obligation to which the sales-based milestone or royalty has been allocated has been satisfied.
In accordance with ASC 606, the Company concluded that the promise to grant the license is distinct, resulting in one performance obligation as the license has stand-alone functionally at contract inception. The Buyout Right precludes transferring control of the license to Renalys under ASC 606 and the Company’s option to repurchase the common stock at a price greater than the original license premium results in accounting for the Renalys License Agreement as a financing arrangement. The transaction price was recorded in other current liabilities, and will be recognized in revenue upon termination of the Buyout Right.
See Note 5 for further discussion of VIE’s.
v3.25.0.1
VARIABLE INTEREST ENTITIES
 12 Months Ended
Dec. 31, 2024
Business Combination, Asset Acquisition, and Joint Venture Formation [Abstract]  
VARIABLE INTEREST ENTITIES VARIABLE INTEREST ENTITIES
Stock Purchase and Collaboration Agreement with PharmaKrysto
On March 8, 2022, the Company entered into a Collaboration Agreement with PharmaKrysto Limited (“PharmaKrysto”), a privately held pre-clinical stage company related to PharmaKrysto's early-stage cystinuria discovery program, and concurrently therewith entered into a Stock Purchase Agreement with PharmaKrysto (together, the "Agreements"). Pursuant to the terms of the Agreements, the Company paid PharmaKrysto's shareholders $0.6 million in cash to purchase 5% of the outstanding common shares of PharmaKrysto and $0.4 million to PharmaKrysto as a one-time signing fee. Under the Collaboration Agreement, the Company will fund all research and development expenses for the pre-clinical activities
associated with the cystinuria program, which are estimated to be approximately $5.0 million. The Agreements require the Company to purchase an additional 5% of the outstanding common shares for $1.0 million upon the occurrence of a specified pre-clinical milestone, and granted an option to the Company to purchase the remaining outstanding shares of PharmaKrysto for $5.0 million upon the occurrence of a subsequent pre-clinical milestone prior to expiration of the option on March 8, 2025. If the Company elects to exercise the option, it would be required to perform commercially reasonable clinical diligence obligations. In addition, it would be required to make cash milestone payments totaling up to an aggregate $16.0 million upon the achievement of certain development and regulatory milestones, plus tiered royalty payments of less than 4% on future net sales of a product, if approved. The Company has the right to terminate the Agreements and return the shares for a nominal price at any time upon 60 days’ notice, subject to survival of contingent obligations, if any.
The Company determined that PharmaKrysto is a VIE because it lacks the resources to conduct the cystinuria clinical program and the limitation on the residual returns through the Company's option to purchase the remaining outstanding shares. The Company further concluded that it is the primary beneficiary of the VIE due to the Company's ultimate control over the research and development program, and its obligation, subject to continuation of the collaboration, to fund 100% of research and development costs of the program pursuant to the terms of the Collaboration Agreement.
The upfront payments were expensed to research and development and other income (expense), net upon initial consolidation. The consolidated assets and liabilities as of December 31, 2024 and 2023 were immaterial. The results of operations were not significant for the years ended December 31, 2024, 2023 and 2022. The Company is not required to provide additional funding other than the contractually required amounts disclosed above. The creditors and beneficial holders of PharmaKrysto have no recourse to the general credit or assets of the Company.
Licensing Agreement with Renalys
In January 2024, the Renalys License Agreement between the Company and Renalys came into effect and the Company exercised its option to purchase shares of common stock of Renalys. The Company determined that Renalys is a VIE as they could require additional funding to support development and commercial activities. The Company has variable interests in Renalys, including an equity interest, Buyout Right and performance-related payments under the Renalys License Agreement that absorb variability from the performance of Renalys.
In order to determine the primary beneficiary of Renalys, the Company evaluated its variable interest to identify if the Company had the power to direct the activities that most significantly impact the economic performance. Based upon the capital structure, governing documents and overall business operations, the Company determined that it is not the primary beneficiary as it does do not have the power to direct the activities that most significantly impact the economic performance of Renalys and does not have an obligation to absorb losses.
As of December 31, 2024, the carrying amount of the liabilities related to the Company’s variable interests was $8.9 million, recorded in other current liabilities in the Company’s Consolidated Balance Sheets. The Company’s maximum exposure to loss as of December 31, 2024 is zero. The Company is not required to provide additional funding. The creditors have no recourse to the general credit or assets of the Company.
v3.25.0.1
MARKETABLE DEBT SECURITIES
 12 Months Ended
Dec. 31, 2024
Investments, Debt and Equity Securities [Abstract]  
MARKETABLE DEBT SECURITIES MARKETABLE DEBT SECURITIES
The Company's marketable debt securities as of December 31, 2024 and 2023 were composed of available-for-sale commercial paper and corporate and government debt securities. The primary objective of the Company’s investment portfolio is to preserve capital and liquidity while enhancing overall returns. The Company’s investment policy limits interest-bearing security investments to certain types of instruments issued by institutions with primarily investment grade credit ratings and places restrictions on maturities and concentration by asset class and issuer.
Marketable debt securities consisted of the following (in thousands):
As of December 31,
20242023
Marketable debt securities:
Commercial paper$73,325 $34,458 
Corporate debt securities203,816 368,323 
Securities of government-sponsored entities35,025 105,894 
Total available-for-sale marketable debt securities$312,166 $508,675 
The following is a summary of short-term marketable debt securities classified as available-for-sale as of December 31, 2024 (in thousands):
Remaining Contractual Maturity
(in years)		Amortized CostUnrealized GainsUnrealized LossesAggregate Estimated Fair Value
Marketable debt securities:
Commercial paperLess than 1$73,410 $1 $(86)$73,325 
Corporate debt securitiesLess than 1203,395 483 (62)203,816 
Securities of government-sponsored entitiesLess than 134,993 33 (1)35,025 
Total available-for-sale marketable debt securities$311,798 $517 $(149)$312,166 
The following is a summary of short-term marketable debt securities classified as available-for-sale as of December 31, 2023 (in thousands):
Remaining Contractual Maturity
(in years)		Amortized CostUnrealized GainsUnrealized LossesAggregate Estimated Fair Value
Marketable debt securities:
Commercial paperLess than 1$34,450 $25 $(17)$34,458 
Corporate debt securitiesLess than 1133,463 29 (408)133,084 
Securities of government-sponsored entitiesLess than 181,334 36 (274)81,096 
Total maturity less than 1 year249,247 90 (699)248,638 
Corporate debt securities1 to 2233,969 1,444 (174)235,239 
Securities of government-sponsored entities1 to 224,718 106 (26)24,798 
Total maturity 1 to 2 years258,687 1,550 (200)260,037 
Total available-for-sale marketable debt securities$507,934 $1,640 $(899)$508,675 
During 2024 and 2023, realized gains and losses on marketable debt securities were immaterial. As of December 31, 2024 and December 31, 2023, the accrued interest receivable related to the Company's marketable debt securities was $2.3 million and $4.6 million, respectively, and was recorded in prepaid expenses and other current assets on the Consolidated Balance Sheets.
The Company reviews the available-for-sale marketable debt securities for declines in fair value below the cost basis each quarter. For any security whose fair value is below its amortized cost basis, the Company first evaluates whether it intends to sell the impaired security, or will otherwise be more likely than not required to sell the security before recovery. If either are true, the amortized cost basis of the security is written down to its fair value at the reporting date. If neither circumstance holds true, the Company assesses whether any portion of the unrealized loss is a result of a credit loss. Any amount deemed to be attributable to credit loss is recognized in the income statement, with the amount of the loss limited to the difference between fair value and amortized cost and recorded as an allowance for credit losses. The portion of the unrealized loss related to factors other than credit losses is recognized in other comprehensive loss.
The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of December 31, 2024 (in thousands):
Less Than 12 Months12 Months or GreaterTotal
Description of SecuritiesFair ValueUnrealized LossesFair ValueUnrealized LossesFair ValueUnrealized Losses
Commercial paper$68,446 $86 $— $— $68,446 $86 
Corporate debt securities40,112 56 9,969 6 50,081 62 
Securities of government-sponsored entities— — 4,975 1 4,975 1 
Total$108,558 $142 $14,944 $7 $123,502 $149 
The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of December 31, 2023 (in thousands):
Less Than 12 Months12 Months or GreaterTotal
Description of SecuritiesFair ValueUnrealized LossesFair ValueUnrealized LossesFair ValueUnrealized Losses
Commercial paper$24,798 $17 $— $— $24,798 $17 
Corporate debt securities140,802 405 28,775 177 169,577 582 
Securities of government-sponsored entities61,933 217 12,540 83 74,473 300 
Total$227,533 $639 $41,315 $260 $268,848 $899 
As of December 31, 2024 and December 31, 2023, the amortized cost of the available-for-sale marketable debt securities in an unrealized loss position was $123.7 million and $269.7 million, respectively.
As of December 31, 2024 and December 31, 2023, the Company does not intend to sell these investments and it is not more likely than not that the Company will be required to sell the investments before recovery of their amortized cost basis. The decrease in unrealized losses for the year ended December 31, 2024 was primarily due to fluctuations in short-term interest rates. The Company does not believe the unrealized losses incurred during the period are due to credit-related factors. The credit ratings of the securities held remain of the highest quality. Moreover, the Company continues to receive payments of interest and principal as they become due, and our expectation is that those payments will continue to be received timely. Factors unknown to us at this time may cause actual results to differ and require adjustments to the Company’s estimates and assumptions in the future.
v3.25.0.1
CONVERTIBLE NOTES PAYABLE
 12 Months Ended
Dec. 31, 2024
Debt Disclosure [Abstract]  
CONVERTIBLE NOTES PAYABLE CONVERTIBLE NOTES PAYABLE
The composition of the Company’s convertible senior notes are as follows (in thousands):
 December 31, 2024December 31, 2023
2.25% convertible senior notes due 2029
$316,250 $316,250 
2.50% convertible senior notes due 2025
68,904 68,904 
Unamortized debt issuance costs - 2.25% convertible senior notes due 2029
(5,940)(7,348)
Unamortized debt issuance costs - 2.50% convertible senior notes due 2025
(226)(543)
Total convertible senior notes, net of unamortized debt discount and debt issuance costs$378,988 $377,263 
Classified as:
Convertible debt, current portion$68,678 $— 
Convertible debt, less current portion310,310 377,263 
Total convertible debt$378,988 $377,263 
Convertible Senior Notes Due 2029
On March 11, 2022, the Company completed a registered underwritten public offering of $316.3 million aggregate principal amount of 2.25% Convertible Senior Notes due 2029 (“2029 Notes”), which includes $41.3 million aggregate principal amount of 2029 Notes sold pursuant to the full exercise of the underwriters’ option to purchase additional 2029 Notes. The Company issued the 2029 Notes under an indenture, dated as of September 10, 2018, as supplemented by the second supplemental indenture, dated as of March 11, 2022 (collectively, the “2029 Indenture”). The 2029 Notes will mature on March 1, 2029, unless earlier repurchased, redeemed, or converted. The 2029 Notes are senior unsecured obligations of the Company and bear interest at an annual rate of 2.25%, payable semi-annually in arrears on March 1 and September 1 of each year, beginning on September 1, 2022.
The Company received net proceeds from the issuance of the 2029 Notes of $306.4 million, after deducting commissions and offering expenses of $9.9 million. At December 31, 2024, accrued interest on the 2029 Notes of $2.4 million is included in accrued expenses in the accompanying Consolidated Balance Sheets. The 2029 Notes comprise the Company’s senior, unsecured obligations and are (i) equal in right of payment with the Company’s existing and future senior, unsecured indebtedness; (ii) senior in right of payment to the Company’s existing and future indebtedness that is expressly subordinated to the 2029 Notes; (iii) effectively subordinated to the Company’s existing and future secured indebtedness, to the extent of the value of the collateral securing that indebtedness; and (iv) structurally subordinated to all existing and future indebtedness and other liabilities, including trade payables.
Holders may convert their 2029 Notes at their option only in the following circumstances: (1) during any calendar quarter commencing after the calendar quarter ending on June 30, 2022 (and only during such calendar quarter), if the last reported sale price per share of the Company’s common stock for each of at least 20 trading days, whether or not consecutive, during the period of 30 consecutive trading days ending on, and including, the last trading day of the immediately preceding calendar quarter exceeds 130% of the conversion price on the applicable trading day; (2) during the five consecutive business days immediately after any 10 consecutive trading day period (such 10 consecutive trading day period, the “measurement period”) if the trading price per $1,000 principal amount of 2029 Notes for each trading day of the measurement period was less than 98% of the product of the last reported sale price per share of the Company’s common stock on such trading day and the conversion rate on such trading day; (3) upon the occurrence of certain corporate events or distributions of the Company’s common stock; (4) if the Company calls the 2029 Notes for redemption; and (5) at any time from, and including, December 1, 2028 until the close of business on the scheduled trading day immediately before the maturity date. The Company will settle conversions by paying or delivering, as applicable, cash, shares of the Company’s common stock, or a combination of cash and shares of the Company’s common stock, at the Company’s election, based on the applicable conversion rate. The initial conversion rate for the 2029 Notes is 31.3740 shares of the Company’s common stock per $1,000 principal amount of 2029 Notes, which represents an initial conversion price of approximately $31.87 per share. If a “make-whole fundamental change” (as defined in the 2029 Indenture) occurs, then the Company will, in certain circumstances, increase the conversion rate for a specified period of time.
The 2029 Notes will be redeemable, in whole or in part at the Company’s option at any time, and from time to time, on or after March 2, 2026 and, in the case of any partial redemption, on or before the 40th scheduled trading day before the maturity date, at a cash redemption price equal to the principal amount of the 2029 Notes to be redeemed, plus accrued and unpaid interest, if any, to, but excluding, the redemption date but only if the last reported sale price per share of the Company’s common stock exceeds 130% of the conversion price on (1) each of at least 20 trading days, whether or not consecutive, during the 30 consecutive trading days ending on, and including, the trading day immediately before the date the Company sends the related redemption notice; and (2) the trading day immediately before the date the Company sends such notice. However, the Company may not redeem less than all of the outstanding 2029 Notes unless at least $100.0 million aggregate principal amount of 2029 Notes are outstanding and not called for redemption as of the time the Company sends the related redemption notice. In addition, calling any 2029 Note for redemption will constitute a make-whole fundamental change with respect to that 2029 Note, in which case the conversion rate applicable to the conversion of that 2029 Note will be increased in certain circumstances if it is converted after it is called for redemption. If a fundamental change (as defined in the 2029 Indenture) occurs, then, except as described in the 2029 Indenture, holders may require the Company to repurchase their 2029 Notes at a cash repurchase price equal to the principal amount of the 2029 Notes to be repurchased, plus accrued and unpaid interest, if any, to, but excluding, the fundamental change repurchase date. In the event of conversion, holders would forgo all future interest payments, any unpaid accrued interest and the possibility of further stock price appreciation. Upon the receipt of conversion requests, the settlement of the 2029 Notes will be paid pursuant to the terms of the 2029 Indenture. In the event that all of the 2029 Notes are converted, the Company would be required to repay the principal amount and any conversion premium in any combination of cash and shares of its common stock at the Company’s option. In addition, calling the 2029 Notes for redemption will constitute a “make-whole fundamental change."
The Company incurred approximately $9.9 million of debt issuance costs relating to the issuance of the 2029 Notes, which were recorded as a reduction to the 2029 Notes on the Consolidated Balance Sheets. The debt issuance costs are being amortized and recognized as additional interest expense over the expected life of the 2029 Notes using the effective interest method. We determined the expected life of the debt is equal to the seven-year term of the 2029 Notes. The effective interest rate on the 2029 Notes is 2.74%.
Convertible Senior Notes Due 2025
On September 10, 2018, the Company completed a registered underwritten public offering of $276.0 million aggregate principal amount of 2.50% Convertible Senior Notes due 2025 ("2025 Notes"), and entered into a base indenture and supplemental indenture agreement ("2025 Indenture") with respect to the 2025 Notes. The 2025 Notes will mature on September 15, 2025, unless earlier repurchased, redeemed, or converted. The 2025 Notes are senior unsecured obligations of the Company and bear interest at an annual rate of 2.50%, payable semi-annually in arrears on March 15 and September 15 of each year, beginning on March 15, 2019.
The net proceeds from the issuance of the 2025 Notes were approximately $267.2 million, after deducting commissions and the offering expenses of $8.8 million payable by the Company. At December 31, 2024, accrued interest of $0.5 million is included in accrued expenses in the accompanying Consolidated Balance Sheets. The 2025 Notes comprise the Company’s senior, unsecured obligations and are (i) equal in right of payment with the Company’s existing and future senior, unsecured indebtedness; (ii) senior in right of payment to the Company’s existing and future indebtedness that is expressly subordinated to the 2025 Notes; (iii) effectively subordinated to the Company’s existing and future secured indebtedness, to the extent of the value of the collateral securing that indebtedness; and (iv) structurally subordinated to all existing and future indebtedness and other liabilities, including trade payables.
Holders may convert their 2025 Notes at their option only in the following circumstances: (1) during any calendar quarter commencing after the calendar quarter ending on December 31, 2018 (and only during such calendar quarter), if the last reported sale price per share of the Company’s common stock for each of at least 20 trading days, whether or not consecutive, during the period of 30 consecutive trading days ending on, and including, the last trading day of the immediately preceding calendar quarter exceeds 130% of the conversion price on the applicable trading day; (2) during the five consecutive business days immediately after any 10 consecutive trading day period (“measurement period”) if the trading price per $1,000 principal amount of 2025 Notes for each trading day of the measurement period was less than 98% of the product of the last reported sale price per share of the Company’s common stock on such trading day and the conversion rate on such trading day; (3) upon the occurrence of certain
corporate events or distributions on the Company’s common stock; (4) if the Company calls the 2025 Notes for redemption; and (5) at any time from, and including, May 15, 2025 until the close of business on the scheduled trading day immediately before the maturity date. The Company will settle conversions by paying or delivering, as applicable, cash, shares of the Company’s common stock, or a combination of cash and shares of the Company’s common stock, at the Company’s election, based on the applicable conversion rate.
The initial conversion rate for the 2025 Notes is 25.7739 shares of the Company’s common stock per $1,000 principal amount of 2025 Notes, which represents an initial conversion price of approximately $38.80 per share. If a “make-whole fundamental change” (as defined in the 2025 Indenture) occurs, then the Company will, in certain circumstances, increase the conversion rate for a specified period of time.
The 2025 Notes will be redeemable, in whole or in part, at the Company’s option at any time, and from time to time, on or after September 15, 2022 and, in the case of any partial redemption, on or before the 40th scheduled trading day before the maturity date, at a cash redemption price equal to the principal amount of the 2025 Notes to be redeemed, plus accrued and unpaid interest, if any, to, but excluding, the redemption date, but only if the last reported sale price per share of the Company’s common stock exceeds 130% of the conversion price on each of at least 20 trading days during the 30 consecutive trading days ending on, and including, the trading day immediately before the date the Company sends the related redemption notice. If a fundamental change (as defined in the 2025 Indenture) occurs, then, subject to certain exceptions, holders may require the Company to repurchase their 2025 Notes at a cash repurchase price equal to the principal amount of the 2025 Notes to be repurchased, plus accrued and unpaid interest, if any, to, but excluding, the fundamental change repurchase date. In the event of conversion, holders would forgo all future interest payments, any unpaid accrued interest and the possibility of further stock price appreciation. Upon the receipt of conversion requests, the settlement of the 2025 Notes will be paid pursuant to the terms of the 2025 Indenture. In the event that all of the 2025 Notes are converted, the Company would be required to repay the principal amount and any conversion premium in any combination of cash and shares of its common stock at the Company’s option. In addition, calling the 2025 Notes for redemption will constitute a “make-whole fundamental change."
The Company incurred approximately $8.8 million of debt issuance costs relating to the issuance of the 2025 Notes, which were recorded as a reduction to the 2025 Notes on the Consolidated Balance Sheets. The debt issuance costs are being amortized and recognized as additional interest expense over the expected life of the 2025 Notes using the effective interest method. The Company determined the expected life of the debt is equal to the seven-year term of the 2025 Notes. The effective interest rate on the 2025 Notes is 2.98%.
On March 11, 2022, the Company completed its repurchase of $207.1 million aggregate principal amount of 2025 Notes for cash, including accrued and unpaid interest, for a total of $213.8 million. This transaction involved a contemporaneous exchange of cash between the Company and holders of the 2025 Notes participating in the issuance of the 2029 Notes. Accordingly, we evaluated the transaction for modification or extinguishment accounting in accordance with ASC 470-50, Debt – Modifications and Extinguishments on a creditor-by creditor basis depending on whether the exchange was determined to have substantially different terms. The repurchase of the 2025 Notes and issuance of the 2029 Notes were deemed to have substantially different terms based on the present value of the cash flows or significant difference between the value of the conversion option immediately prior to and after the exchange. Therefore, the repurchase of the 2025 Notes was accounted for as a debt extinguishment. The Company recorded a $7.6 million loss on extinguishment of debt on its Consolidated Statements of Operations for the year ended December 31, 2022, which included the write-off of related deferred financing costs of $3.4 million. After giving effect to the repurchase, and as of December 31, 2024, the total remaining principal amount outstanding under the 2025 Notes was $68.9 million.
The 2025 and 2029 Notes are accounted for in accordance with ASC 470-20, Debt with conversion and Other Options (“ASC 470-20”) and ASC 815-40, Contracts in Entity’s Own Equity (“ASC 815-40”). Under ASC 815-40, to qualify for equity classification (or nonbifurcation, if embedded) the instrument (or embedded feature) must be both (1) indexed to the issuer’s stock and (2) meet the requirements of equity classification guidance. Based upon the Company’s analysis, it was determined that the 2025 Notes and the 2029 Notes do not contain embedded features requiring recognition as derivatives and bifurcation, and therefore are measured at amortized cost and recorded as liabilities on the Consolidated Balance Sheets.
The 2025 and 2029 Notes do not contain any financial or operating covenants or any restrictions on the payment of dividends, the issuance of other indebtedness or the issuance or repurchase of securities by the Company. There were no events of default for the 2025 Notes or 2029 Notes at December 31, 2024.
The 2025 and 2029 Notes are classified on the Company's Consolidated Balance Sheets at December 31, 2024 as short-term and long-tern convertible debt, respectively.
Interest Expense
The following table sets forth total interest expense recognized related to the 2025 and 2029 Notes (in thousands):
Year Ended December 31,
202420232022
Contractual interest expense$8,838 $8,838 $8,433 
Amortization of debt issuance costs1,725 1,718 1,622 
Total interest expense for the 2025 and 2029 Notes$10,563 $10,556 $10,055 
Total interest expense recognized for the years ended December 31, 2024, 2023 and 2022 was $11.2 million, $11.3 million and $11.0 million, respectively.
v3.25.0.1
FAIR VALUE MEASUREMENTS
 12 Months Ended
Dec. 31, 2024
Fair Value Disclosures [Abstract]  
FAIR VALUE MEASUREMENTS FAIR VALUE MEASUREMENTS
The Company utilizes a fair value hierarchy that prioritizes the inputs to valuation techniques used to measure fair value. The hierarchy gives the highest priority to unadjusted quoted prices in active markets for identical assets or liabilities (Level 1 measurements) and the lowest priority to unobservable inputs (Level 3 measurements). The three levels of the fair value hierarchy are described below:
Level 1 – Unadjusted quoted prices in active markets that are accessible at the measurement date for identical, unrestricted assets or liabilities;
Level 2 – Quoted prices in markets that are not active or financial instruments for which all significant inputs are observable, either directly or indirectly; and
Level 3 – Prices or valuations that require inputs that are both significant to the fair value measurement and unobservable.
The valuation techniques used to measure the fair value of the Company’s debt securities and all other financial instruments, all of which have counterparties with high credit ratings, were valued based on quoted market prices or model driven valuations using significant inputs derived from or corroborated by observable market data. Based on the fair value hierarchy, the Company classified marketable debt securities within Level 2.
Financial instruments with carrying values approximating fair value include cash and cash equivalents, accounts receivable, and accounts payable, due to their short-term nature. As of December 31, 2024 and 2023, the fair value of the Company's 2.50% Convertible Senior Notes due 2025 was $68.2 million and $58.3 million, respectively. As of December 31, 2024 and 2023, the fair value of the Company's 2.25% Convertible Senior Notes due 2029, which were issued in 2022, was $302.1 million and $212.1 million, respectively. The fair values were estimated utilizing market quotations and are considered Level 2.
The following table presents the Company’s assets, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of December 31, 2024 (in thousands):
 As of December 31, 2024Fair Value Hierarchy at December 31, 2024
Total carrying and estimated fair valueQuoted prices in active markets (Level 1)Significant other observable inputs (Level 2)Significant unobservable inputs (Level 3)
Assets:
Cash and Cash Equivalents$58,535 $51,060 $7,475 $— 
Marketable debt securities, available-for-sale312,166 — 312,166 — 
Total$370,701 $51,060 $319,641 $— 
The following table presents the Company’s assets, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of December 31, 2023 (in thousands):
 As of December 31, 2023Fair Value Hierarchy at December 31, 2023
Total carrying and estimated fair valueQuoted prices in active markets (Level 1)Significant other observable inputs (Level 2)Significant unobservable inputs (Level 3)
Assets:
Cash and Cash Equivalents$58,176 $58,176 $— $— 
Marketable debt securities, available-for-sale508,675 — 508,675 — 
Total$566,851 $58,176 $508,675 $— 
Assets Measured at Fair Value on a Nonrecurring Basis
The Company’s long-lived assets are measured at fair value on a nonrecurring basis when there are indicators of impairment and a loss is recognized. During the year ended December 31, 2024, the Company recorded impairment charges of $1.2 million on right-of-use assets, property and equipment, and other capitalized assets as the fair value of the asset group was less than their carrying value. The fair value of the asset group was determined under the income approach based on projected future cash flows from the operating sublease discounted by a risk adjusted rate of 6.9%. The Company classified the fair value of the asset group as Level 3. See Note 18 for further discussion.
v3.25.0.1
INTANGIBLE ASSETS
 12 Months Ended
Dec. 31, 2024
Goodwill and Intangible Assets Disclosure [Abstract]  
INTANGIBLE ASSETS INTANGIBLE ASSETS
Amortizable Intangible Assets
Ligand License Agreement
In 2012, the Company entered into the Ligand License Agreement with Ligand for a worldwide sublicense to develop, manufacture and commercialize sparsentan. As consideration for the license, the Company is required to make substantial payments upon the achievement of certain milestones, totaling up to $114.1 million. Through December 31, 2024, the Company has capitalized $47.2 million for contractual milestones achieved under the Ligand License Agreement, including $5.8 million for the year ended December 31, 2024. Pursuant to the Ligand License Agreement, the Company is obligated to pay to Ligand (and Bristol-Myers Squibb Company ("BMS")) an escalating royalty between 15% and 17% of net sales of sparsentan, with payments due quarterly. The Company began incurring costs associated with such royalties following the February 2023 approval of FILSPARI (sparsentan). For the years ended December 31, 2024 and 2023, the Company capitalized $20.3 million and $4.4 million, respectively, to intangible assets for royalties owed on net sales of FILSPARI. The cost of the milestone payments and royalty payments are being amortized to selling, general and administration on a straight-line basis through April 30, 2033
Mission License Agreement
In 2014, the Company entered into the Mission License Agreement with Mission in which the Company obtained an exclusive, royalty-bearing license to market, sell and commercialize Thiola (tiopronin) in the United States and Canada, and a non-exclusive license to use know-how relating to Thiola to the extent necessary to market Thiola. The initial term of the license was 10 years.
The Company paid Mission an up-front license fee of $3.0 million and during the term of the agreement will pay the greater of $2.0 million, representing the guaranteed minimum royalty, or 20% of the Company’s net sales of Thiola during each calendar year.
In November 2017, the Company amended its agreement with Mission to extend the term of the current exclusive U.S. and Canada licensing agreement by an additional five years, to 2029. The royalty rate and guaranteed minimum payment were also extended through the new agreement term. Upon execution of the amendment, the Company capitalized an additional $5.9 million in intangible assets and recorded a guaranteed minimum liability for the same amount.
In November 2018, the Company amended its agreement with Mission to remove all territorial restrictions on our license. As consideration for the expanded territory, the Company paid an up-front fee of $0.3 million and will pay the greater of $0.1 million, representing the guaranteed minimum, or 20% of the Company's net sales of Thiola generated outside of the United States during each calendar year. Upon execution of the amendment, the Company capitalized an additional $1.0 million in intangible assets and recorded a guaranteed minimum liability of $0.7 million related to this amendment.
The present value of guaranteed minimum royalties payable using a discount rate ranging from approximately 7% to 11% based on the Company’s then borrowing rate is $8.2 million and $9.7 million as of December 31, 2024 and 2023, respectively. As of December 31, 2024, the guaranteed minimum royalty current and long-term liability was approximately $2.1 million and $6.1 million, respectively, and is recorded as Other Liability in the Consolidated Balance Sheets. As of December 31, 2023, the guaranteed minimum royalty current and long-term liability was approximately $2.1 million and $7.6 million, respectively, and is recorded as Other Liability in the Consolidated Balance Sheets.
The Company has capitalized $171.0 million related to the Thiola intangible asset which consists of the up-front license fee, professional fees, present value of the guaranteed minimum royalties and any additional payment obligations through December 31, 2024 in excess of minimum royalties. In 2024 the Company added $15.4 million to the intangible asset related to royalties in excess of the minimum. Consistent with all prior periods since Thiola was acquired, the Company has not accrued any liability for royalties in excess of the annual contractual minimum at December 31, 2024, as such royalties are not yet probable and estimable.
Amortizable intangible assets as of December 31, 2024 (in thousands):
 Useful LifeGross Carrying AmountAccumulated AmortizationNet Book Value
Mission license12$171,025 $(115,303)$55,722 
Ligand license1153,268 (5,769)47,499 
Total amortizable intangible assets$224,293 $(121,072)$103,221 
Amortizable intangible assets as of December 31, 2023 (in thousands):
Useful LifeGross Carrying AmountAccumulated AmortizationNet Book Value
Mission license12$155,589 $(77,311)$78,278 
Ligand license1127,448 (2,036)25,412 
Total amortizable intangible assets$183,037 $(79,347)$103,690 
The following table summarizes amortization expense for the year ended December 31, 2024, 2023 and 2022 (in thousands):
202420232022
Selling, general and administrative$41,739 $29,021 $12,339 
Research and development— 7,261 6,264 
Total amortization expense$41,739 $36,282 $18,603 
As of December 31, 2024, amortization expense for the next five years and thereafter is expected to be as follows (in thousands):
2025$50,397 
202616,722 
20275,700 
20285,714 
20295,700 
Thereafter18,988 
Total$103,221 
There were no impairments related to finite-lived intangible assets in the years ended December 31, 2024, 2023 and 2022.
Goodwill
As of December 31, 2024 and 2023, the Company had goodwill of $0.8 million.
For the years ended December 31, 2024, 2023 and 2022 there were no impairments to goodwill.
v3.25.0.1
ACCRUED EXPENSES
 12 Months Ended
Dec. 31, 2024
Payables and Accruals [Abstract]  
ACCRUED EXPENSES ACCRUED EXPENSES 
Accrued expenses consist of the following at December 31, 2024 and 2023 (in thousands):
20242023
Compensation related costs$35,166 $29,908 
Research and development16,090 26,006 
Accrued royalties12,309 6,991 
Sales discounts, rebates, and allowances10,585 13,730 
Selling, general and administrative6,154 7,190 
Transition services accrual285 12,282 
Accrued restructuring costs— 11,421 
Miscellaneous accrued expenses5,439 11,463 
Total accrued expenses$86,028 $118,991 
v3.25.0.1
COMMITMENTS AND CONTINGENCIES
 12 Months Ended
Dec. 31, 2024
Commitments and Contingencies Disclosure [Abstract]  
COMMITMENTS AND CONTINGENCIES COMMITMENTS AND CONTINGENCIES
Commitments
Certain of the Company's contractual arrangements with contract manufacturing organizations ("CMOs") require binding forecasts or commitments to purchase minimum amounts for the manufacture of drug product supply, which may be material to the Company's financial statements.
Contingencies
In November 2020, the Company completed the acquisition of Orphan Technologies Limited (“Orphan”), including Orphan’s rare metabolic disorder drug pegtibatinase. The Company acquired Orphan by purchasing all of the outstanding shares. Under the Agreement, the Company has also agreed to make contingent cash payments up to an aggregate of $427.0 million based on the achievement of certain development, regulatory and commercialization events as set forth in the Agreement, as well as additional tiered mid-single digit royalty payments based upon future net sales of any pegtibatinase products in the U.S. and Europe, subject to certain reductions as set forth in the Agreement, and a contingent payment in the event a pediatric rare disease voucher for any pegtibatinase product is granted.
In accordance with ASC 450, Contingencies, contingent cash payments will be accrued for when it is probable that a liability has been incurred and the amount can be reasonably estimated. In March 2024, the Company recognized $65.2 million in IPR&D expense upon the achievement of a development milestone, which was paid during the second quarter of 2024 and recorded within investing activities in the Consolidated Statements of Cash Flows. As of December 31, 2024, no contingent cash payments have been accrued.
Legal Proceedings
From time to time in the normal course of business, the Company is subject to various legal matters such as threatened or pending claims or litigation. Although the results of claims and litigation cannot be predicted with certainty, the Company does not believe it is a party to any claim or litigation in which the outcome, if determined adversely to it, would individually or in the aggregate be reasonably expected to have a material adverse effect on its results of operations or financial condition.
v3.25.0.1
STOCKHOLDERS’ EQUITY
 12 Months Ended
Dec. 31, 2024
Equity [Abstract]  
STOCKHOLDERS’ EQUITY STOCKHOLDERS’ EQUITY
Common Stock
The Company is currently authorized to issue up to 200,000,000 shares of $0.0001 par value common stock. All issued shares of common stock are entitled to vote on a 1 share/1 vote basis.
Preferred Stock 
The Company is currently authorized to issue up to 20,000,000 shares of $0.0001 par value preferred stock, of which 1,000 shares are designated Class "A" Preferred shares. Class A Preferred Shares are not entitled to interest, have certain liquidation preferences, special voting rights and other provisions. No preferred stock has been issued to date.
2018 Equity Incentive Plan
The Company's 2018 Equity Incentive Plan (the "2018 Plan") is the successor to and continuation of the Company's 2015 Equity Incentive Plan (the "2015 Plan") and the Company's 2014 Equity Incentive Plan (the "2014 Plan", and together with the 2015 Plan, the "Prior Plans"). Unallocated shares under the Prior Plans are no longer available for issuance under the Prior Plans, and have instead been added to the shares available for issuance under the 2018 Plan. The 2018 Plan, as amended, and including the unallocated shares of the Prior Plans, provides for a total of 18.4 million shares to be issued, plus the Prior Plans' returning shares, if any, which become available for grant under the 2018 Plan from time to time. Options issued under the 2018 Plan will generally expire ten years from the date of grant and vest over a four-year period. As of December 31, 2024, there were approximately 5.7 million shares reserved for future issuance under the 2018 Plan.
2017 Employee Stock Purchase Plan
The 2017 Employee Stock Purchase Plan ("2017 ESPP") originated with 380,000 shares of common stock available for issuance. Beginning on January 1, 2018, and ending on (and including) January 1, 2026, the number of shares of common stock available for issuance under the 2017 ESPP may increase by an amount equal to the lesser of (i) one percent (1%) of the total number of shares of common stock outstanding on December 31st of the preceding calendar year or (ii) 300,000 shares of common stock.
Substantially all employees are eligible to participate in the 2017 ESPP and, through payroll deductions, can purchase shares on established dates semi-annually. The purchase price per share sold pursuant to the 2017 ESPP will be the lower of (i) 85% of the fair market value of common stock on the first day of the offering period or (ii) 85% of the fair market value on the purchase date. Each offering period will span up to six months. Purchases may be up to 15% of qualified compensation, with an annual limit of $25,000. The 2017 ESPP is intended to qualify as an “employee stock purchase plan” under Section 423 of the Internal Revenue Code.
As of December 31, 2024, there were approximately 2,480,000 shares authorized and 1,004,889 shares reserved for future issuance under the 2017 ESPP.
Stock Options
The fair values of stock option grants during the years ended December 31, 2024, 2023 and 2022 were calculated on the date of grant using the Black-Scholes option pricing model. Compensation expense is recognized over the period of service, generally the vesting period. The following weighted average assumptions were used in the Black-Scholes options pricing model to estimate the fair value of stock options for the specified reporting periods:
Year Ended December 31,
 202420232022
Risk free rate3.9 %3.9 %1.7 %
Expected volatility59 %50 %50 %
Expected life (in years)6.26.46.4
Expected dividend yield— — — 
The risk-free interest rate was based on rates established by the Federal Reserve. The Company’s expected volatility was based on analysis of the Company’s historical volatility. The expected life of the Company’s options was determined using the Company's historical exercise activity. The dividend yield is based upon the fact that the Company has not historically paid dividends and does not expect to pay dividends in the foreseeable future.
The following table summarizes our stock option activity and related information for the year ended December 31, 2024:
Weighted Average
Shares Underlying
Options		Exercise
Price		Remaining
Contractual
Term (in years)		Aggregate
Intrinsic Value
(in thousands)
Outstanding at December 31, 202310,211,353 $21.52 4.91$— 
Granted1,524,300 8.63 — — 
Forfeited and expired(2,076,808)20.02 — — 
Exercised(375,514)11.86 — 2,016 
Outstanding at December 31, 20249,283,331 $20.13 5.51$16,408 
Vested and expected to vest at December 31, 20249,283,331 $20.13 5.51$16,408 
The aggregate intrinsic value of stock options exercised in the years ended December 31, 2024, 2023 and 2022 was $2.0 million, $1.4 million, and $5.1 million, respectively.
The following table summarizes our stock options exercisable at December 31, 2024, 2023 and 2022:
Weighted Average
Shares Underlying
Options		Exercise
Price		Remaining
Contractual
Term (in years)		Aggregate
Intrinsic Value
(in thousands)
Exercisable at December 31, 20226,999,669 $20.22 4.62$21,806 
Exercisable at December 31, 20237,942,964 $20.85 3.95$— 
Exercisable at December 31, 20246,892,156 $22.11 4.46$3,249 
The weighted average grant date fair value of options granted was $5.10, $11.66, and $13.45 during the years ended December 31, 2024, 2023 and 2022, respectively. The aggregate intrinsic value for outstanding options is calculated as the difference between the exercise price of the underlying awards and the closing price of the Company’s common stock of $17.42, $8.99 and $21.03 as of December 31, 2024, 2023 and 2022, respectively. Unrecognized compensation cost associated with unvested stock options amounts to $16.2 million as of December 31, 2024, which will be expensed over a weighted average remaining vesting period of 2.4 years.
In connection with the retirement of the Company's former Chief Financial Officer, the Board of Directors approved a modification to extend the deadline to exercise each stock option held to the earlier of three months following the last vesting date or the original expiration date of the option, and to continue vesting on the original schedule of any underlying unvested stock options and restricted stock units. The modification resulted in incremental compensation cost of $2.6 million for the year ended December 31, 2023.
Restricted Stock Units
As of December 31, 2024, there was approximately $35.8 million of unrecognized compensation cost related to restricted stock units ("RSUs") granted. This amount is expected to be recognized over a weighted average period of 2.3 years.
The following table summarizes our restricted stock unit activity for the year ended December 31, 2024:
Number of
RSUs		Weighted Average
Grant Date Fair Value
Unvested December 31, 20232,874,046 $22.97 
Granted2,077,025 9.07 
Vested(1,013,811)22.52 
Forfeited/cancelled(419,997)18.66 
Unvested December 31, 20243,517,263 $15.41 
The fair value of restricted stock units vested for the years ended December 31, 2024, 2023 and 2022 was $22.8 million, $18.7 million, and $11.5 million, respectively. The weighted average grant date fair value for stock awards granted during the years ended December 31, 2024, 2023 and 2022 was $9.07, $21.20 and $26.07, respectively.
Performance-based Stock Units
Performance-based stock units ("PSUs") are subject to vest only if certain specified criteria are achieved. As of December 31, 2024, there was approximately $1.2 million of unrecognized compensation cost related to PSUs granted and deemed probable of vesting. This amount is expected to be recognized over a weighted average period of 0.9 years.
The following table summarizes our performance-based stock unit activity for the year ended December 31, 2024:
 Number of
PSUs		Weighted Average
Grant Date Fair Value
Unvested December 31, 2023175,458 $25.61 
Granted107,000 8.93 
Vested(66,250)22.40 
Forfeited/cancelled— — 
Unvested December 31, 2024216,208 $18.34 
The fair value of PSUs vested for the years ended December 31, 2024, 2023 and 2022 was $1.5 million, $0.3 million, and $0.4 million, respectively. The weighted average grant date fair value for performance-based stock awards granted during the years ended December 31, 2024, 2023 and 2022 was $8.93, $22.40 and $27.54, respectively.
Share Based Compensation
Total non-cash stock-based compensation expense consisted of the following for the years ended December 31, 2024, 2023 and 2022 (in thousands):
 Year Ended December 31,
 202420232022
Selling, general and administrative expenses$22,735 $27,111 $24,368 
Research and development expenses14,178 17,135 13,775 
Total stock-based compensation expense$36,913 $44,246 $38,143 
EQUITY OFFERINGS
Underwritten Public Offering of Common Stock
In November 2024, the Company sold an aggregate of approximately 9.0 million shares of its common stock in an underwritten public offering, at a price to the public of $16.00 per share of common stock. The net proceeds to the Company from the offering, after deducting the underwriting discounts and offering expenses, were approximately $134.7 million.
In February 2023, the Company sold an aggregate of approximately 9.7 million shares of its common stock and pre-funded warrants to purchase 1.25 million shares of its common stock in an underwritten public offering, at a price to the public of $21.00 per share of common stock and $20.9999 per pre-funded warrant. The pre-funded warrants are exercisable immediately, subject to certain beneficial ownership limitations which can be modified by the respective holders with at least 61 days' notice, and are exercisable for one share of the Company's common stock. The exercise price of each pre-funded warrant is $0.0001 per share of common stock. The net proceeds to the Company from the offering, after deducting the
underwriting discounts and offering expenses, were approximately $215.8 million. The pre-funded warrants were exercised in the third quarter of 2024, resulting in the issuance of 1.25 million shares of the Company's common stock.
At-the-Market Equity Offering
In October 2024, the Company filed a prospectus supplement to the prospectus included in its registration statement on Form S-3 (File No. 333-281194), pursuant to which the Company may offer and sell, from time to time through Jefferies LLC, as agent (“Jefferies”), up to $100.0 million of common stock pursuant to an Amended and Restated Open Market Sale Agreement ("ATM Agreement") with Jefferies dated October 2024. The Company did not sell any shares under the ATM Agreement during the year ended December 31, 2024.
v3.25.0.1
NET LOSS PER COMMON SHARE
 12 Months Ended
Dec. 31, 2024
Earnings Per Share [Abstract]  
NET LOSS PER COMMON SHARE NET LOSS PER COMMON SHARE
Basic and diluted net income (loss) per common share is calculated by dividing net loss applicable to common stockholders by the weighted-average number of common shares outstanding during the period. In accordance with ASC 260, Earnings per Share, if a company had a discontinued operation, the company uses income from continuing operations, adjusted for preferred dividend and similar adjustments, as its control number to determine whether potential common shares are dilutive.
As discussed in Note 15, as part of its February 2023 underwritten public offering, the Company issued and sold pre-funded warrants to purchase 1.25 million shares of its common stock at a price to the public of $20.9999 per pre-funded warrant. The pre-funded warrants were immediately exercisable upon issuance, and were exercised in the third quarter of 2024, resulting in the issuance of 1.25 million shares of the Company's common stock. Due to the nominal exercise price of the pre-funded warrants and the lack of any contingencies to exercise, the shares underlying the pre-funded warrants have been included in the calculation of basic net loss per common share since the date the warrants were issued.
The Company’s potentially dilutive shares, which include outstanding stock options, restricted stock units, performance-based stock units, and shares issuable upon conversion of the 2025 Notes and 2029 Notes, are considered to be common stock equivalents and are not included in the calculation of diluted net loss per share because their effect is anti-dilutive.
Basic and diluted net loss per share is calculated as follows (net loss amounts are stated in thousands):
For the year ended December 31,
202420232022
SharesNet (loss) incomeEPSSharesNet (loss) incomeEPSSharesNet (loss) incomeEPS
Continuing operations78,888,861 $(320,630)$(4.07)74,267,418 $(376,333)$(5.07)63,758,515 $(331,468)$(5.20)
Discontinued operations78,888,861 (915)(0.01)74,267,418 264,934 3.57 63,758,515 52,986 0.83 
Basic and diluted loss per share78,888,861 $(321,545)$(4.08)74,267,418 $(111,399)$(1.50)63,758,515 $(278,482)$(4.37)
For the years ended December 31, 2024, 2023 and 2022, the following weighted-average number of common stock equivalents were excluded because they were anti-dilutive:
For the year ended December 31,
202420232022
Convertible debt11,697,953 11,697,952 10,869,000 
Options10,447,541 10,555,550 10,041,249 
Restricted stock units and performance-based stock units3,780,441 3,417,245 2,175,100 
Total anti-dilutive shares25,925,935 25,670,747 23,085,349 
v3.25.0.1
INCOME TAXES
 12 Months Ended
Dec. 31, 2024
Income Tax Disclosure [Abstract]  
INCOME TAXES INCOME TAXES
For financial reporting purposes, net loss from continuing operations before income taxes includes the following components (in thousands):
Year Ended December 31,
202420232022
United States$(152,356)$(293,283)$(268,887)
Foreign(168,154)(82,827)(62,268)
Total$(320,510)$(376,110)$(331,155)
The components of the provision for income taxes, in the Consolidated Statements of Operations are as follows (in thousands):
 202420232022
Current
Federal$— $— $— 
State120 223 314 
Foreign— — (1)
Total current120 223 313 
Deferred
Federal— — — 
State— — — 
Total deferred— — — 
Total tax provision$120 $223 $313 
The following is a reconciliation of the statutory federal income tax rate to the Company’s effective tax rate expressed as a percentage of loss before income taxes:
 202420232022
Statutory rate - federal(21.00)%(21.00)%(21.00)%
State taxes, net of federal benefit(1.55)%(3.19)%(3.62)%
Foreign rate differential2.15 %1.29 %0.81 %
IPR&D4.27 %— %— %
Nondeductible executive compensation0.44 %1.50 %0.41 %
Excess tax benefits associated with share-based awards2.92 %0.68 %(0.17)%
Other permanent differences0.23 %0.37 %0.17 %
Tax credits(4.24)%(1.13)%(5.29)%
Return to provision adjustments and other true-ups(0.80)%4.19 %(0.17)%
Adoption of ASU 2020-06— %— %(3.54)%
Other0.93 %0.57 %— %
Change in valuation allowance16.69 %16.78 %32.49 %
Income tax provision0.04 %0.06 %0.09 %
The significant components of the Company’s deferred tax assets and liabilities as of December 31, 2024 and 2023 are as follows (in thousands):
 20242023
Deferred Tax Assets:
Net operating loss$113,303 $81,531 
Research and development and other tax credits91,760 77,555 
Intangible assets53,919 47,731 
Capitalized research and development47,880 39,907 
Stock based compensation15,680 21,607 
Other accrued expenses14,266 13,187 
Charitable contributions5,797 6,110 
Operating lease liabilities5,421 6,812 
Depreciation435 292 
Loan costs153 62 
Interest expense limitation— 1,679 
Total deferred tax assets348,614 296,473 
Deferred Tax Liabilities:
Operating lease right of use assets(4,259)(5,595)
Prepaid assets(178)(198)
Total deferred tax liabilities(4,437)(5,793)
Net deferred tax assets before valuation allowance344,177 290,680 
Valuation allowance(344,177)(290,680)
Total deferred tax assets$— $— 
The Company has established a full valuation allowance against its U.S. federal, state, and foreign deferred tax assets due to the uncertainty surrounding the realization of such assets in future periods. The ultimate realization of deferred tax assets is dependent upon the generation of future taxable income during the periods in which temporary differences become deductible. Management considers the scheduled reversal of deferred liabilities and tax planning strategies in making this assessment and evaluates the recoverability of the deferred tax assets as of each reporting date. At such time as it is determined that it is more likely than not that deferred assets are realizable, the valuation allowance will be reduced accordingly and recorded as a tax benefit.
The Company has recorded a valuation allowance of $344.2 million as of December 31, 2024 to reflect the estimated amount of deferred tax assets that may not be realized. The Company increased its valuation allowance by $53.5 million for the year ended December 31, 2024, compared to a $2.7 million decrease for the year ended December 31, 2023.
As of December 31, 2024, the Company had available unused U.S. federal and state net operating loss (“NOL”) carryforwards of $223.8 million and $241.8 million, respectively, all of which are fully offset by a valuation allowance. The federal NOL has an indefinite life. The state NOL carryforwards will begin to expire in 2025 unless previously utilized, except for $45.4 million of the state net operating losses that have an indefinite carryforward period. In addition, at December 31, 2024, the Company had federal orphan drug tax credit carryforwards of $104.1 million that begin to expire in 2035 unless utilized, federal research and development tax credit carryforwards of $4.9 million that begin to expire in 2033 unless utilized, state research and development tax credit carryforwards of $1.4 million that begin to expire in 2030 unless utilized and $9.9 million that have an indefinite carryforward period, and California Competes tax credit carryforwards of $1.6 million that begin to expire in 2025. Pursuant to Internal Revenue Code Sections 382 and 383, use of the Company’s federal net operating loss and credit carryforwards may be limited upon a cumulative change in ownership of more than 50% within a three-year period. The Company continues to monitor potential historical ownership changes.
As of December 31, 2024, the Company had Irish NOL carryforwards of $14.2 million which are fully offset by a valuation allowance and have an indefinite life. The Company also had Swiss NOL carryforwards of $359.1 million which are fully offset by a valuation allowance and begin to expire in 2025, as well as Federal Act on Tax Reform and AHV Financing (“TRAF”) cantonal tax benefits of $526.2 million which expire in 2029.
The Company accounts for uncertain tax benefits in accordance with the provisions of ASC 740-10 of the Accounting for Uncertainty in Income Taxes. As of December 31, 2024, the Company had $27.4 million in unrecognized tax benefits, which were recorded as a reduction to the deferred tax assets with a corresponding reduction in the Company’s valuation allowance of $27.4 million. To the extent unrecognized tax benefits are recognized at a time when a valuation allowance does not exist, the recognition of the $27.4 million tax benefit would reduce the effective tax rate. The Company does not anticipate that the amount of unrecognized tax benefits as of December 31, 2024 will change materially within the following 12 months.
A reconciliation of the Company's unrecognized tax benefits for the years 2024, 2023 and 2022 is provided in the following table (in thousands):
202420232022
Balance as of January 1:$22,906 $11,490 $7,825 
Increase in current period positions4,248 4,871 2,056 
Increase in prior period positions250 7,383 1,919 
Decrease in prior period positions— (838)— 
Decrease due to settlements with tax authorities— — (310)
Balance as of December 31:$27,404 $22,906 $11,490 
The Company files income tax returns in the U.S. federal jurisdiction, various state and local, and foreign jurisdictions. With few exceptions, the Company’s income tax returns are open to examination by federal and state authorities for the years ended December 31, 2013 and forward, due to the carryforward of unutilized tax attributes. The Company's Swiss income tax returns are open to examination for the years ended December 31, 2019 and forward, and the Company's Irish tax returns are open to examination for the years ended December 31, 2020 and forward.
The Company recognizes interest and penalties as a component of income tax expense. The Company did not recognize any interest or penalties for the year ended December 31, 2024 and 2023. Interest and penalties for the year ended December 31, 2022 were less than $0.1 million.
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EQUITY OFFERINGS
 12 Months Ended
Dec. 31, 2024
Equity [Abstract]  
EQUITY OFFERINGS STOCKHOLDERS’ EQUITY
Common Stock
The Company is currently authorized to issue up to 200,000,000 shares of $0.0001 par value common stock. All issued shares of common stock are entitled to vote on a 1 share/1 vote basis.
Preferred Stock 
The Company is currently authorized to issue up to 20,000,000 shares of $0.0001 par value preferred stock, of which 1,000 shares are designated Class "A" Preferred shares. Class A Preferred Shares are not entitled to interest, have certain liquidation preferences, special voting rights and other provisions. No preferred stock has been issued to date.
2018 Equity Incentive Plan
The Company's 2018 Equity Incentive Plan (the "2018 Plan") is the successor to and continuation of the Company's 2015 Equity Incentive Plan (the "2015 Plan") and the Company's 2014 Equity Incentive Plan (the "2014 Plan", and together with the 2015 Plan, the "Prior Plans"). Unallocated shares under the Prior Plans are no longer available for issuance under the Prior Plans, and have instead been added to the shares available for issuance under the 2018 Plan. The 2018 Plan, as amended, and including the unallocated shares of the Prior Plans, provides for a total of 18.4 million shares to be issued, plus the Prior Plans' returning shares, if any, which become available for grant under the 2018 Plan from time to time. Options issued under the 2018 Plan will generally expire ten years from the date of grant and vest over a four-year period. As of December 31, 2024, there were approximately 5.7 million shares reserved for future issuance under the 2018 Plan.
2017 Employee Stock Purchase Plan
The 2017 Employee Stock Purchase Plan ("2017 ESPP") originated with 380,000 shares of common stock available for issuance. Beginning on January 1, 2018, and ending on (and including) January 1, 2026, the number of shares of common stock available for issuance under the 2017 ESPP may increase by an amount equal to the lesser of (i) one percent (1%) of the total number of shares of common stock outstanding on December 31st of the preceding calendar year or (ii) 300,000 shares of common stock.
Substantially all employees are eligible to participate in the 2017 ESPP and, through payroll deductions, can purchase shares on established dates semi-annually. The purchase price per share sold pursuant to the 2017 ESPP will be the lower of (i) 85% of the fair market value of common stock on the first day of the offering period or (ii) 85% of the fair market value on the purchase date. Each offering period will span up to six months. Purchases may be up to 15% of qualified compensation, with an annual limit of $25,000. The 2017 ESPP is intended to qualify as an “employee stock purchase plan” under Section 423 of the Internal Revenue Code.
As of December 31, 2024, there were approximately 2,480,000 shares authorized and 1,004,889 shares reserved for future issuance under the 2017 ESPP.
Stock Options
The fair values of stock option grants during the years ended December 31, 2024, 2023 and 2022 were calculated on the date of grant using the Black-Scholes option pricing model. Compensation expense is recognized over the period of service, generally the vesting period. The following weighted average assumptions were used in the Black-Scholes options pricing model to estimate the fair value of stock options for the specified reporting periods:
Year Ended December 31,
 202420232022
Risk free rate3.9 %3.9 %1.7 %
Expected volatility59 %50 %50 %
Expected life (in years)6.26.46.4
Expected dividend yield— — — 
The risk-free interest rate was based on rates established by the Federal Reserve. The Company’s expected volatility was based on analysis of the Company’s historical volatility. The expected life of the Company’s options was determined using the Company's historical exercise activity. The dividend yield is based upon the fact that the Company has not historically paid dividends and does not expect to pay dividends in the foreseeable future.
The following table summarizes our stock option activity and related information for the year ended December 31, 2024:
Weighted Average
Shares Underlying
Options		Exercise
Price		Remaining
Contractual
Term (in years)		Aggregate
Intrinsic Value
(in thousands)
Outstanding at December 31, 202310,211,353 $21.52 4.91$— 
Granted1,524,300 8.63 — — 
Forfeited and expired(2,076,808)20.02 — — 
Exercised(375,514)11.86 — 2,016 
Outstanding at December 31, 20249,283,331 $20.13 5.51$16,408 
Vested and expected to vest at December 31, 20249,283,331 $20.13 5.51$16,408 
The aggregate intrinsic value of stock options exercised in the years ended December 31, 2024, 2023 and 2022 was $2.0 million, $1.4 million, and $5.1 million, respectively.
The following table summarizes our stock options exercisable at December 31, 2024, 2023 and 2022:
Weighted Average
Shares Underlying
Options		Exercise
Price		Remaining
Contractual
Term (in years)		Aggregate
Intrinsic Value
(in thousands)
Exercisable at December 31, 20226,999,669 $20.22 4.62$21,806 
Exercisable at December 31, 20237,942,964 $20.85 3.95$— 
Exercisable at December 31, 20246,892,156 $22.11 4.46$3,249 
The weighted average grant date fair value of options granted was $5.10, $11.66, and $13.45 during the years ended December 31, 2024, 2023 and 2022, respectively. The aggregate intrinsic value for outstanding options is calculated as the difference between the exercise price of the underlying awards and the closing price of the Company’s common stock of $17.42, $8.99 and $21.03 as of December 31, 2024, 2023 and 2022, respectively. Unrecognized compensation cost associated with unvested stock options amounts to $16.2 million as of December 31, 2024, which will be expensed over a weighted average remaining vesting period of 2.4 years.
In connection with the retirement of the Company's former Chief Financial Officer, the Board of Directors approved a modification to extend the deadline to exercise each stock option held to the earlier of three months following the last vesting date or the original expiration date of the option, and to continue vesting on the original schedule of any underlying unvested stock options and restricted stock units. The modification resulted in incremental compensation cost of $2.6 million for the year ended December 31, 2023.
Restricted Stock Units
As of December 31, 2024, there was approximately $35.8 million of unrecognized compensation cost related to restricted stock units ("RSUs") granted. This amount is expected to be recognized over a weighted average period of 2.3 years.
The following table summarizes our restricted stock unit activity for the year ended December 31, 2024:
Number of
RSUs		Weighted Average
Grant Date Fair Value
Unvested December 31, 20232,874,046 $22.97 
Granted2,077,025 9.07 
Vested(1,013,811)22.52 
Forfeited/cancelled(419,997)18.66 
Unvested December 31, 20243,517,263 $15.41 
The fair value of restricted stock units vested for the years ended December 31, 2024, 2023 and 2022 was $22.8 million, $18.7 million, and $11.5 million, respectively. The weighted average grant date fair value for stock awards granted during the years ended December 31, 2024, 2023 and 2022 was $9.07, $21.20 and $26.07, respectively.
Performance-based Stock Units
Performance-based stock units ("PSUs") are subject to vest only if certain specified criteria are achieved. As of December 31, 2024, there was approximately $1.2 million of unrecognized compensation cost related to PSUs granted and deemed probable of vesting. This amount is expected to be recognized over a weighted average period of 0.9 years.
The following table summarizes our performance-based stock unit activity for the year ended December 31, 2024:
 Number of
PSUs		Weighted Average
Grant Date Fair Value
Unvested December 31, 2023175,458 $25.61 
Granted107,000 8.93 
Vested(66,250)22.40 
Forfeited/cancelled— — 
Unvested December 31, 2024216,208 $18.34 
The fair value of PSUs vested for the years ended December 31, 2024, 2023 and 2022 was $1.5 million, $0.3 million, and $0.4 million, respectively. The weighted average grant date fair value for performance-based stock awards granted during the years ended December 31, 2024, 2023 and 2022 was $8.93, $22.40 and $27.54, respectively.
Share Based Compensation
Total non-cash stock-based compensation expense consisted of the following for the years ended December 31, 2024, 2023 and 2022 (in thousands):
 Year Ended December 31,
 202420232022
Selling, general and administrative expenses$22,735 $27,111 $24,368 
Research and development expenses14,178 17,135 13,775 
Total stock-based compensation expense$36,913 $44,246 $38,143 
EQUITY OFFERINGS
Underwritten Public Offering of Common Stock
In November 2024, the Company sold an aggregate of approximately 9.0 million shares of its common stock in an underwritten public offering, at a price to the public of $16.00 per share of common stock. The net proceeds to the Company from the offering, after deducting the underwriting discounts and offering expenses, were approximately $134.7 million.
In February 2023, the Company sold an aggregate of approximately 9.7 million shares of its common stock and pre-funded warrants to purchase 1.25 million shares of its common stock in an underwritten public offering, at a price to the public of $21.00 per share of common stock and $20.9999 per pre-funded warrant. The pre-funded warrants are exercisable immediately, subject to certain beneficial ownership limitations which can be modified by the respective holders with at least 61 days' notice, and are exercisable for one share of the Company's common stock. The exercise price of each pre-funded warrant is $0.0001 per share of common stock. The net proceeds to the Company from the offering, after deducting the
underwriting discounts and offering expenses, were approximately $215.8 million. The pre-funded warrants were exercised in the third quarter of 2024, resulting in the issuance of 1.25 million shares of the Company's common stock.
At-the-Market Equity Offering
In October 2024, the Company filed a prospectus supplement to the prospectus included in its registration statement on Form S-3 (File No. 333-281194), pursuant to which the Company may offer and sell, from time to time through Jefferies LLC, as agent (“Jefferies”), up to $100.0 million of common stock pursuant to an Amended and Restated Open Market Sale Agreement ("ATM Agreement") with Jefferies dated October 2024. The Company did not sell any shares under the ATM Agreement during the year ended December 31, 2024.
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RETIREMENT PLAN
 12 Months Ended
Dec. 31, 2024
Retirement Benefits [Abstract]  
RETIREMENT PLAN RETIREMENT PLAN
401(k) Savings Plan
The Company has a 401(k) defined contribution savings plan for the benefit of all eligible employees. Employer matching contributions were $2.4 million, $2.7 million, and $2.1 million for the years ended December 31, 2024, 2023 and 2022, respectively.
Switzerland Defined Benefit Plan
The Company maintains a defined benefit pension plan covering employees of its Swiss subsidiary, Travere Therapeutics Switzerland GmbH (the "Swiss Plan"). The Swiss Plan is a government-mandated retirement fund that provides employees with a minimum benefit. Employer and employee contributions are made to the Swiss Plan based on various percentages of participants' salaries and wages that vary according to the participants' age and other factors. As of December 31, 2024, the projected benefit obligations under the Swiss Plan were approximately $3.0 million, and plan assets were approximately $2.5 million. The funded status of the Swiss Plan is included in other long-term liabilities on the Company's Consolidated Balance Sheets.
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PROPERTY AND EQUIPMENT
 12 Months Ended
Dec. 31, 2024
Property, Plant and Equipment [Abstract]  
PROPERTY AND EQUIPMENT PROPERTY AND EQUIPMENT
Property, plant and equipment, net consisted of the following (in thousands):
December 31,
20242023
Leasehold improvements$9,370 $9,692 
Furniture and fixtures2,446 2,990 
Computers and equipment2,275 2,071 
Construction-in-progress— 169 
14,091 14,922 
Less: Accumulated depreciation(8,755)(7,443)
Total property and equipment, net$5,336 $7,479 
Depreciation expense for the years ended December 31, 2024, 2023 and 2022 was $1.8 million, $2.2 million and $2.1 million, respectively.
v3.25.0.1
LEASES
 12 Months Ended
Dec. 31, 2024
Leases [Abstract]  
LEASES LEASES
As of December 31, 2024, the Company had operating leases, including one operating lease with Kilroy Realty, L.P. (the "Landlord") for office space located in San Diego, California, which was entered into in April 2019 and subsequently amended in May 2020. Coinciding with the Company's ability to direct the use of the office space and utilizing a discount rate equal to the Company's estimated incremental borrowing rate, the Company established right-of-use assets totaling $34.6 million and lease liabilities totaling $34.5 million. The total right-of-use asset and lease liability at measurement were each offset by lease incentives associated with tenant improvement allowances totaling $7.9 million.
The initial term of the office lease ends in August 2028, and the Landlord has granted the Company an option to extend the term of the lease by a period of 5 years. At lease inception, it was not reasonably certain that the Company will extend the term of the lease and therefore the renewal period has been excluded from the aforementioned right-of-use asset and lease liability measurements. The measurement of the lease term occurs from the February 2021 occupancy date of the office space.
As of December 31, 2024, the Company also had an operating lease with Esprit Investments Limited for office space located in Dublin, Ireland, which was entered into in October 2022. The initial term of the office lease ends in September 2027. The lease provides the option to extend the term of the lease by a period of 5 years, although at lease inception, it was not reasonably certain that the Company will elect this option and therefore the renewal period has been excluded from the initial lease measurement. Utilizing a discount rate equal to the Company's borrowing rate, the Company established a right-of-use asset and corresponding lease liability of $0.4 million.
During the year ended December 31, 2024, due to the decreased occupancy from the 2023 restructuring along with efforts to optimize a hybrid work model, the Company decided to reduce its use of office space located in San Diego, California. In November 2024, the Company entered into a sublease of its 5th floor premises. The term of the sublease runs from January 2025 through August 2028.
The Company’s sublease arrangement has been classified as an operating lease with sublease income recognized on a straight-line basis over the term of the sublease arrangement. To measure the Company’s periodic sublease income, the Company elected to use a practical expedient under ASC 842 to aggregate non-lease components with the related lease components when (i) the timing and pattern of transfer for the non-lease components and the related lease components are the same and (ii) the lease components, if accounted for separately, would be classified as an operating lease.
Given the circumstances surrounding the 5th floor premises, the Company identified an indicator of impairment of the related operating lease right-of-use assets, property and equipment, and other capitalized assets and compared the carrying value of the asset group to an estimate of the future undiscounted cash flows expected to result from the sublease and eventual disposition of the asset group. The sum of the undiscounted cash flows of the asset group was below the carrying value. Consequently, the Company utilized the present value of the estimated future cash flows attributable to the assets to determine the fair value of the asset group. The resulting fair value of the operating lease right-of-use assets, property and equipment, and other capitalized assets resulted in an impairment of $1.2 million recorded in restructuring expense in the Consolidated Statements of Operations and categorized the aforementioned measurement of fair value as Level 3 within the ASC Topic 820, "Fair Value Measurements" fair value hierarchy. There were no impairments related to right-of-use assets or property and equipment in the years ended December 31, 2023 and 2022.
The following is a schedule of the future minimum rental commitments for the Company's operating leases reconciled to the lease liability and ROU asset as of December 31, 2024 (in thousands):
December 31, 2024
2025$6,665 
20266,889 
20277,064 
20284,781 
Thereafter— 
Total undiscounted future minimum payments25,399 
Present value discount(2,803)
Total lease liability22,596 
Unamortized lease incentives(3,491)
Cash payments in excess of straight-line lease expense(4,810)
Total ROU asset$14,295 
The weighted-average remaining lease term and weighted-average discount rate of the Company's operating leases are as follows:
December 31,
20242023
Weighted-average remaining lease term in years3.74.7
Weighted-average discount rate6.48 %6.48 %
For the years ended December 31, 2024, 2023 and 2022 the Company recorded $4.8 million, $4.9 million, and $5.0 million, respectively, in expense related to operating leases, including amortized tenant improvement allowances.
v3.25.0.1
DIVESTITURES
 12 Months Ended
Dec. 31, 2024
Discontinued Operations and Disposal Groups [Abstract]  
DIVESTITURES DIVESTITURES
Discontinued Operations
Sale of Bile Acid Product Portfolio
On August 31, 2023, the Company closed the sale of its bile acid business to Mirum Pharmaceuticals pursuant to the terms of the Purchase Agreement dated July 16, 2023 between the Company and Mirum. The assets sold consisted of substantially all of the assets primarily related to the Company’s business of development, manufacture (including synthesis, formulation, finishing or packaging) and commercialization of the products, Chenodal and Cholbam (also known as Kolbam). In connection with the Closing, the Company received an upfront cash payment of $210.0 million.
Pursuant to the Purchase Agreement, after the Closing, the Company is eligible to receive up to $235.0 million upon the achievement of certain milestones based on specified amounts of annual net sales (tiered from $125.0 million to $500.0 million) of the Products. The Company will recognize the contingent consideration receivable in earnings when the target annual sales for the milestones are met and the contingency is resolved.
The Company's sale of the bile acid business resulted in a gain, net of tax, of $226.0 million, which was recognized in 2023. The net gain consists of net consideration, including the upfront payment and the deduction of investment banker fees owed upon the Closing, plus the derecognition of the carrying value of the net liabilities included in the transaction and the immaterial tax due on the sale.
The Company and Mirum have also entered into a transition services agreement ("TSA") pursuant to which the Company has agreed to perform certain services for a period of time following the Closing, with respect to Mirum’s use and operation of the assets purchased in the Purchase Agreement. The TSA is designed to ensure and facilitate an orderly transfer of business operations, and the consideration to be received by the Company primarily consists of cost reimbursement. For the years ended December 31, 2024 and 2023, the Company recognized $0.5 million and $1.0 million, respectively, under the TSA, included in continuing operations within other income (expense), net. As of December 31, 2024, the Company collected $1.5 million. The uncollected balance is included in accounts receivable of the Consolidated Balance Sheets. As part of the TSA, the Company is collecting certain receivables related to purchased assets for a period of time and remitting them to Mirum. The transition services accrual as of December 31, 2024 was $0.3 million, and is included in accrued expenses in the accompanying Consolidated Balance Sheets. TSA services provided by the Company are substantially complete as of December 31, 2024.
The Company determined that the divestiture represents a strategic shift that will have a major effect on the Company's operations and financial results, and has therefore reflected the bile acid business as a discontinued operation for all periods presented.
Results of discontinued operations are as follows (in thousands):
Year Ended December 31,
202420232022
Net product sales$(550)$66,164 $102,558 
Total revenue(550)66,164 102,558 
Operating expenses:
Cost of goods sold(9)1,899 3,172 
Research and development247 6,118 8,447 
Selling, general and administrative127 19,500 22,686 
Change in fair value of contingent consideration— (473)15,006 
Total operating expenses365 27,044 49,311 
Operating income(915)39,120 53,247 
Other income (expenses), net:
Interest expense— (191)(261)
Gain on disposal of discontinued operations, net of tax— 226,005 — 
Total other income (expense), net— 225,814 (261)
(Loss) income from discontinued operations before income tax(915)264,934 52,986 
Income tax on discontinued operations— — — 
Net (loss) income from discontinued operations$(915)$264,934 $52,986 
v3.25.0.1
SEGMENT INFORMATION
 12 Months Ended
Dec. 31, 2024
Segment Reporting [Abstract]  
SEGMENT INFORMATION SEGMENT INFORMATION
The Company operates in one business segment focused on identifying, developing and delivering life-changing therapies to people living with rare kidney and metabolic diseases. The determination of a single business segment is consistent with the consolidated financial information regularly provided to the Company’s chief operating decision maker (“CODM”), who is the President and Chief Executive Officer. The CODM uses net loss to monitor budget versus actual results in assessing segment performance and the allocation of resources. The Company’s CODM also utilizes the Company’s long-range plan as a strategic tool to allocate resources according to the Company’s strategic objectives.
The Company sells its products to specialty pharmacies and through direct-to-patient distributors worldwide. Net product sales outside of the United States ("U.S.") were not significant for the years ended December 31, 2024, 2023 and 2022. Long-lived assets located outside the U.S. were $4.5 million as of December 31, 2024. The Company had no long-lived assets located outside the U.S. as of December 31, 2023. The measure of segment assets is reported on the consolidated balance sheets as total assets. The accounting policies of the segment are the same as those described in Note 2, Summary of Significant Accounting Policies.
The following table presents reportable segment loss, including significant expenses regularly provided to the CODM, attributable to the Company’s reportable segment for the years ended December 31, 2024, 2023 and 2022 (in thousands):
Year Ended December 31,
202420232022
Revenue$233,175 $145,238 $109,460 
Less:
Cost of goods sold7,744 11,450 4,420 
Research and development:
External research and development126,303 142,482 129,698 
Internal personnel costs73,843 84,658 75,664 
Other Research and development17,350 17,850 21,971 
Total research and development217,496 244,990 227,333 
Selling, general and administrative264,119 265,542 197,520 
In-process research and development65,205 — — 
Restructuring2,438 11,394 — 
Total other income (expense), net3,317 12,028 (11,342)
Income tax provision on continuing operations(120)(223)(313)
(Loss) income from discontinued operations, net of tax(915)264,934 52,986 
Net loss$(321,545)$(111,399)$(278,482)
v3.25.0.1
Pay vs Performance Disclosure - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Pay vs Performance Disclosure      
Net loss $ (321,545) $ (111,399) $ (278,482)
v3.25.0.1
Insider Trading Arrangements
 3 Months Ended 12 Months Ended
Dec. 31, 2024
shares
Dec. 31, 2024
shares
Trading Arrangements, by Individual    
Material Terms of Trading Arrangement  
During the fiscal quarter ended December 31, 2024, our directors and/or officers (as defined in Rule 16a-1(f) under the Exchange Act) adopted, modified or terminated the “Rule 10b5-1 trading arrangements” or “non-Rule 10b5-1 trading arrangements,” as those terms are defined in Regulation S-K, Item 408, set forth below:
Trading Arrangements Adopted:
Name & TitleDate AdoptedCharacter of Trading Arrangement (1)Aggregate Number of Shares of Common Stock to be Sold Pursuant to Trading ArrangementExpiration Date (2)
Timothy Coughlin,
member of our Board of Directors
November 15, 2024Rule 10b5-1 Trading Arrangement
Up to 40,000 shares (3)
March 31, 2025
Gary Lyons,
chair of our Board of Directors
December 17, 2024Rule 10b5-1 Trading Arrangement
Up to 8,000 shares (4)
June 6, 2025
Jeffrey Meckler,
member of our Board of Directors
December 17, 2024Rule 10b5-1 Trading Arrangement
Up to 8,000 shares (4)
June 9, 2025
1
Each trading arrangement marked as a “Rule 10b5-1 Trading Arrangement” is intended to satisfy the affirmative defense of Rule 10b5-1(c) under the Exchange Act (the “Rule”).
2Each trading arrangement permits transactions through and including the earlier to occur of (a) the completion of all sales and (b) the date listed in the table. Each trading arrangement marked as a “Rule 10b5-1 Trading Arrangement” only permits transactions upon expiration of the applicable mandatory cooling-off period under the Rule.
3Consists of shares of underlying stock options expiring in March 2025.
4Consists of shares of underlying stock options expiring in June 2025.
Non-Rule 10b5-1 Arrangement Adopted false  
Rule 10b5-1 Arrangement Terminated false  
Non-Rule 10b5-1 Arrangement Terminated false  
Timothy Coughlin [Member]    
Trading Arrangements, by Individual    
Name Timothy Coughlin  
Title member of our Board of Directors  
Rule 10b5-1 Arrangement Adopted true  
Adoption Date November 15, 2024  
Expiration Date March 31, 2025  
Arrangement Duration 136 days  
Aggregate Available 40,000 40,000
Gary Lyons [Member]    
Trading Arrangements, by Individual    
Name Gary Lyons  
Title chair of our Board of Directors  
Rule 10b5-1 Arrangement Adopted true  
Adoption Date December 17, 2024  
Expiration Date June 6, 2025  
Arrangement Duration 171 days  
Aggregate Available 8,000 8,000
Jeffrey Meckler [Member]    
Trading Arrangements, by Individual    
Name Jeffrey Meckler  
Title member of our Board of Directors  
Rule 10b5-1 Arrangement Adopted true  
Adoption Date December 17, 2024  
Expiration Date June 9, 2025  
Arrangement Duration 174 days  
Aggregate Available 8,000 8,000
v3.25.0.1
Insider Trading Policies and Procedures
 12 Months Ended
Dec. 31, 2024
Insider Trading Policies and Procedures [Line Items]  
Insider Trading Policies and Procedures Adopted true
v3.25.0.1
Cybersecurity Risk Management and Strategy Disclosure
 12 Months Ended
Dec. 31, 2024
Cybersecurity Risk Management, Strategy, and Governance [Line Items]  
Cybersecurity Risk Management Processes for Assessing, Identifying, and Managing Threats [Text Block]
We have implemented and maintain various information security processes designed to identify, assess and manage material risks from cybersecurity threats to our critical computer networks, third party hosted services, communications systems, hardware and software, and our critical data, including intellectual property, confidential information that is proprietary, strategic or competitive in nature, and data related to patients and clinical trials (“Information Systems and Data”).
Various members of our management team, IT department and other employees, including but not limited to the individuals on our cybersecurity incident management team, help identify, assess and manage our cybersecurity threats and risks, with the assistance of a third-party IT managed
service provider. We manage, identify and assess risks from cybersecurity threats by monitoring and evaluating our threat environment and risk profile using various methods including, for example: through the use of automated tools in certain environments, including but not limited to tools for monitoring, geolocation, remote wiping, threat detection, intrusion detection and prevention (including through the use of machine learning, a form of artificial intelligence), patch management, distributed denial of service (DDoS) protection and forensics; conducting (directly or through third parties) regular audits and threat assessments for internal and external threats; subscribing to reports and services that identify cybersecurity threats; analyzing reports of certain threats and actors; conducting vulnerability assessments in certain environments to identify vulnerabilities; evaluating our and our industry’s risk profile; conducting tabletop incident response exercises; and evaluating certain threats reported to us.
Depending on the environment, we implement and maintain various technical, physical, and organizational measures, processes, standards and policies designed to manage and mitigate material risks from cybersecurity threats to our Information Systems and Data, including, for example: incident response plans and procedures, disaster recovery/business continuity plans, risk assessments, implementation of security standards and certifications, encryption of certain data, network security controls on certain networks, data segregation, Wi-Fi segregation, access controls for certain environments, physical security, asset management, tracking and disposal, systems monitoring, vendor risk management program, employee training and penetration testing.
Our assessment and management of material risks from cybersecurity threats are integrated into our overall risk management processes. For example, cybersecurity risk is addressed as a component of our enterprise risk management program, and members of our management team, IT department and other relevant team members work together to prioritize our risk management processes, mitigate cybersecurity threats that are more likely to lead to a material impact to our business, and report regularly to our board of directors on cybersecurity matters.
We use third-party service providers to assist us from time to time to identify, assess, and manage material risks from cybersecurity threats, including for example managed cybersecurity service providers, threat intelligence service providers, dark web monitoring services, and other cybersecurity software providers.
We use third-party service providers to perform a variety of functions throughout our business, including but not limited to application providers, hosting companies, contract manufacturing organizations and contract research organizations. We have a vendor management program to oversee, identify and manage cybersecurity risks associated with our use of certain of these providers. The program includes a risk assessment for vendors that may include, depending on the vendor and nature of services being performed, security questionnaires, review of the vendor's written security program, review of security assessments, audits and reports, vulnerability scans related to the vendor, security assessment calls with the vendor's security personnel, and the imposition of certain contractual obligations on the vendor, among other elements, in accordance with the processes outlined in our internal vendor selection, management, and oversight process policy and other internal guidelines. More specifically, the level of assessment may depend on the nature of the services provided, the sensitivity of the Information Systems and Data at issue, and the identity of the provider.
For a description of the risks from cybersecurity threats that may materially affect us and how they may do so, see our risk factors under Part 1. Item 1A. Risk Factors in this Annual Report on Form 10-K, including the risk factor captioned “If our information technology systems or data, or those of third parties upon which we rely, are or were compromised, we could experience adverse impacts resulting from such compromise, including, but not limited to, regulatory investigations or actions; litigation; fines and penalties; interruptions to our commercial operations, clinical trials or other operations; harm to our reputation; loss of revenue or profits; loss of sales and other adverse consequences.”
Cybersecurity Risk Management Processes Integrated [Flag] true
Cybersecurity Risk Management Processes Integrated [Text Block]
We have implemented and maintain various information security processes designed to identify, assess and manage material risks from cybersecurity threats to our critical computer networks, third party hosted services, communications systems, hardware and software, and our critical data, including intellectual property, confidential information that is proprietary, strategic or competitive in nature, and data related to patients and clinical trials (“Information Systems and Data”).
Cybersecurity Risk Management Third Party Engaged [Flag] true
Cybersecurity Risk Third Party Oversight and Identification Processes [Flag] true
Cybersecurity Risk Materially Affected or Reasonably Likely to Materially Affect Registrant [Flag] false
Cybersecurity Risk Board of Directors Oversight [Text Block] Our Nominating / Corporate Governance Committee has oversight of our cybersecurity risk management program and reports to our board of directors on cybersecurity matters.
Cybersecurity Risk Board Committee or Subcommittee Responsible for Oversight [Text Block] Our cybersecurity risk assessment and management processes are implemented and maintained by various members of our management team, IT department and other employees, including but not limited to the individuals on our cybersecurity incident management team, which includes individuals who have a diverse combination of relevant expertise, experience, education and training, with representation from our IT, legal, human resources, compliance, risk and privacy functions, among others. Our team includes individuals with relevant experience in enterprise risk management and disclosure controls and procedures. Additionally, certain members of our IT department have experience managing cybersecurity programs and are specifically assigned cybersecurity oversight.
Cybersecurity Risk Process for Informing Board Committee or Subcommittee Responsible for Oversight [Text Block]
The Nominating / Corporate Governance Committee receives regular reports from management concerning our cybersecurity risk management program, and also receives various summaries and/or presentations related to cybersecurity threats, risks and mitigation.
Cybersecurity Risk Role of Management [Text Block]
Our cybersecurity risk assessment and management processes are implemented and maintained by various members of our management team, IT department and other employees, including but not limited to the individuals on our cybersecurity incident management team, which includes individuals who have a diverse combination of relevant expertise, experience, education and training, with representation from our IT, legal, human resources, compliance, risk and privacy functions, among others. Our team includes individuals with relevant experience in enterprise risk management and disclosure controls and procedures. Additionally, certain members of our IT department have experience managing cybersecurity programs and are specifically assigned cybersecurity oversight.
Certain members of our management team and IT department are responsible for hiring appropriate personnel, helping to integrate cybersecurity risk considerations into our overall risk management strategy, communicating key priorities to relevant personnel, approving budgets, helping prepare for cybersecurity incidents, approving cybersecurity processes, and reviewing security assessments and other security-related reports.
Our cybersecurity incident response processes are designed to escalate certain cybersecurity incidents to members of management depending on the circumstances, including in some cases to our executive team. Our cybersecurity incident management team, and other individuals as needed, work to help us mitigate and remediate cybersecurity incidents of which we are notified. In addition, our incident response processes include a procedure for reporting certain cybersecurity incidents to the board of directors and/or the Nominating / Corporate Governance Committee.
Cybersecurity Risk Management Positions or Committees Responsible [Flag] true
Cybersecurity Risk Management Positions or Committees Responsible [Text Block]
Our Nominating / Corporate Governance Committee has oversight of our cybersecurity risk management program and reports to our board of directors on cybersecurity matters.
Our cybersecurity risk assessment and management processes are implemented and maintained by various members of our management team, IT department and other employees, including but not limited to the individuals on our cybersecurity incident management team, which includes individuals who have a diverse combination of relevant expertise, experience, education and training, with representation from our IT, legal, human resources, compliance, risk and privacy functions, among others. Our team includes individuals with relevant experience in enterprise risk management and disclosure controls and procedures. Additionally, certain members of our IT department have experience managing cybersecurity programs and are specifically assigned cybersecurity oversight.
Cybersecurity Risk Management Expertise of Management Responsible [Text Block] Our team includes individuals with relevant experience in enterprise risk management and disclosure controls and procedures. Additionally, certain members of our IT department have experience managing cybersecurity programs and are specifically assigned cybersecurity oversight.
Cybersecurity Risk Process for Informing Management or Committees Responsible [Text Block]
Our cybersecurity incident response processes are designed to escalate certain cybersecurity incidents to members of management depending on the circumstances, including in some cases to our executive team. Our cybersecurity incident management team, and other individuals as needed, work to help us mitigate and remediate cybersecurity incidents of which we are notified. In addition, our incident response processes include a procedure for reporting certain cybersecurity incidents to the board of directors and/or the Nominating / Corporate Governance Committee.
Cybersecurity Risk Management Positions or Committees Responsible Report to Board [Flag] true
v3.25.0.1
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies)
 12 Months Ended
Dec. 31, 2024
Accounting Policies [Abstract]  
Principles of Consolidation
Principles of Consolidation
The consolidated financial statements represent the consolidation of the accounts of the Company, its subsidiaries and variable interest entities for which the Company has been determined to be the primary beneficiary, in conformity with accounting principles generally accepted in the United States ("U.S. GAAP"). All intercompany accounts and transactions have been eliminated in consolidation.
Use of Estimates
Use of Estimates
In preparing financial statements in conformity with U.S. GAAP, management is required to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements, as well as the reported amounts of expenses during the reporting period. Due to inherent uncertainty involved in making estimates, actual results reported in future periods may be affected by changes in these estimates. On an ongoing basis, the Company evaluates its estimates and assumptions. These estimates and assumptions include revenue recognition, forecasting probability-weighted cash flows based upon estimates of forecasted revenues, clinical and regulatory timelines and discount rates, valuing equity securities in share-based payments, estimating expenses of contracted research organizations, estimating reserves for inventory, estimating the useful lives of depreciable and amortizable assets, estimating of valuation allowances and uncertain tax positions, and estimates associated with the assessment of impairment for long-lived assets.
Revenue Recognition
Revenue Recognition
The Company recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that an entity determines are within the scope of Accounting Standards Codification ("ASC") 606, Revenue from Contracts with Customers ("ASC 606"), the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company only recognizes revenue from contracts when it is probable that the entity will collect substantially all the consideration it is entitled to in exchange for the goods or services it transfers to the customer.
Deductions from Revenue
Revenues from product sales are recorded at the net sales price, which includes provisions resulting from discounts, rebates and co-pay assistance that are offered to customers, payers and other indirect customers relating to the Company’s sales of its products. These provisions are based on the estimates of the amounts earned or to be claimed on the related sales. These amounts are treated as variable consideration, estimated and recognized as a reduction of the transaction price at the time of the sale, using the most likely amount method, and are classified as a reduction of accounts receivable (if the amount is payable to a customer) or as a current liability (if the amount is payable to a party other than a customer). The Company includes these estimated amounts in the transaction price to the extent it is probable that a significant reversal of cumulative revenue recognized for such transactions will not occur. Where appropriate, these reserves take into consideration the Company’s historical experience, current contractual and statutory requirements and specific known market events and trends. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the contract. If actual results in the future vary from the Company’s provisions, the Company will adjust the estimate, which would affect net product revenue and earnings in the period such variances become known. For the years ended December 31, 2024, 2023 and 2022, adjustments to net product revenue related to performance obligations satisfied in previous periods were $0.5 million, $0.4 million, and $0.2 million, respectively.
Government Rebates: The Company calculates the rebates that it will be obligated to provide to government programs and deducts these estimated amounts from its gross product sales at the time the revenues are recognized. Allowances for government rebates and discounts are established based on an estimated allocation of payers and the government-mandated discounts applicable to government-funded programs. Rebate discounts are included in accrued expenses in the accompanying consolidated balance sheets.
Commercial Rebates: The Company calculates the rebates it incurs according to any contracts with certain commercial payers and deducts these amounts from its gross product sales at the time the revenues are recognized. Allowances for commercial rebates are established based on actual payer information, which is reasonably estimated at the time of delivery for applicable products. Rebate discounts are included in accrued expenses in the accompanying consolidated balance sheets.
Prompt Pay Discounts: The Company offers discounts to certain customers for prompt payments. The Company accrues for the calculated prompt pay discount based on the gross amount of each invoice for those customers at the time of sale.
Other Fees: The Company pays service fees to certain customers based on a contractually fixed percentage of the wholesale acquisition cost and fees for data. Other fees are recorded as an offset to revenue based on contractual terms at the time revenue from the sale is recognized.
Product Returns: Consistent with industry practice, the Company offers its customers a limited right to return product purchased directly from the Company, which is principally based upon the product’s expiration date. Historically, returns have been immaterial.
Co-pay Assistance: The Company offers a co-pay assistance program, which is intended to provide financial assistance to qualified commercially insured patients with prescription drug co-payments required by payers. The calculation of the accrual for co-pay assistance is based on an estimate of claims and the estimated cost per claim associated with product that has been recognized as revenue.
Collaborative Arrangements
Payments received under collaboration and licensing agreements may include non-refundable fees at the inception of the arrangements, milestone payments for specific achievements and royalties on the sale of products. At the inception of arrangements that include milestone payments, the Company uses judgment to evaluate whether the milestones are probable of being achieved and estimates the amount to include in the transaction price utilizing the most likely amount method. If it is probable that a significant revenue reversal will not occur, the estimated amount is included in the transaction price. Milestone payments that are not within the Company or the licensee’s control, such as regulatory approvals, are considered to be constrained due to a high degree of uncertainty and are not included in the transaction price until such uncertainty is resolved. At the end of each reporting period, the Company re-evaluates the probability of achievement of development milestones and any related constraint and adjusts the estimate of the overall transaction price, if necessary. The Company recognizes aggregate sales-based milestones and royalty payments from product sales of which the license is deemed to be the predominant item to which the royalties relate, at the later of when the related sales occur or when the performance obligation has been satisfied. Revenue from collaboration and licensing agreements may also include sales of inventory, at cost plus a margin, which is recorded in license and collaboration revenue.
The Company utilizes significant judgment to develop estimates of the stand-alone selling price for each distinct performance obligation based upon the relative stand-alone selling price. Variable consideration that relates specifically to the Company’s efforts to satisfy specific performance obligations is allocated entirely to those performance obligations. The stand-alone selling price for license-related performance obligations requires judgment in developing assumptions to project probability-weighted cash flows based upon estimates of forecasted revenues, clinical and regulatory timelines and discount rates. The stand-alone selling price for clinical development performance obligations is based on forecasted expected costs of satisfying a performance obligation plus an appropriate margin.
If the licenses to intellectual property are determined to be distinct from the other performance obligations identified in the arrangement and have stand-alone functionality, the Company recognizes revenues from non-refundable, upfront fees allocated to the license when the license is transferred to the licensee and the licensee is able to benefit from the license. For licenses that are not distinct from other promises, the Company applies judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue from non-refundable, upfront fees. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the related revenue recognition accordingly.
The selection of the method to measure progress towards completion requires judgment and is based on the nature of the products or services to be provided. Revenue is recorded proportionally as costs are incurred. The Company generally utilizes the cost-to-cost method of progress because it best measures the transfer of control to the customer which occurs as the Company incurs costs. Under the cost-to-cost measure of progress, the extent of progress towards completion is measured based on the ratio of costs incurred to date to the total estimated costs at completion of the performance obligation. The Company uses judgment to estimate the total costs expected to complete the clinical development performance obligations, which include subcontractor costs, labor, materials, other direct costs and an allocation of indirect costs. The Company evaluates these cost estimates and the progress each reporting period and adjusts the measure of progress, if necessary.
Inventory, Related Reserves and Capitalization of Inventory Costs
Inventory, which is recorded at the lower of cost or net realizable value, includes materials and other direct and indirect costs and is valued using the first-in, first-out method. The Company periodically analyzes its inventory levels to identify inventory that may expire prior to expected sale or has a cost basis in excess of its estimated realizable value, and writes down such inventory as appropriate. In addition, the Company's products are
subject to strict quality control and monitoring which the Company’s manufacturers perform throughout their manufacturing process. The Company does not directly manufacture any product. The Company has a single supplier for its product Thiola, and utilizes contract service providers for the manufacture of the active pharmaceutical ingredient for FILSPARI and the manufacture of primary packaging, secondary packaging and serialization for its product FILSPARI.
Capitalization of Inventory Costs
Prior to the regulatory approval of the Company's drug candidates, the Company incurs expenses for the manufacture of drug product supplies to support clinical development that could potentially be available to support the commercial launch of those drugs. The Company capitalizes inventory costs associated with its products after regulatory approval, when, based on management’s judgment, future commercialization is considered probable and the future economic benefit is expected to be realized. Until the date at which regulatory approval has been received, costs related to the production of inventory are recorded as research and development expenses as incurred. Any eventual sale of previously expensed ("zero-cost") inventories may impact future margins, for any periods in which those inventories are sold.
Prior to the February 2023 FDA accelerated approval of FILSPARI (sparsentan), the Company expensed the production of active pharmaceutical ingredients purchased to support the commercial launch of FILSPARI, in research and development expenses. For the year ended December 31, 2024, sales of FILSPARI primarily consisted of zero-cost inventories. As of December 31, 2024, the Company had approximately $2.3 million of zero-cost inventory remaining, the majority of which the Company expects will be consumed in 2025. The Company began capitalizing inventory costs associated with FILSPARI following the February 2023 accelerated approval.
At December 31, 2024, the Company's evaluation of excess inventory and obsolescence resulted in a $1.0 million charge to cost of goods sold and an equal write-down of inventory balances due to lower forecasted sales of the Company's tiopronin products. For the year ended December 31, 2024, there were no charges recorded on FILSPARI inventory.
At December 31, 2023, the Company's evaluation of excess inventory and obsolescence considered certain minimum purchase obligations, which in combination with lower forecasted sales of FILSPARI resulted in a $3.2 million charge to cost of goods sold. The charge to cost of goods of sold included a $2.1 million write-down of inventory balances and $1.1 million accrued for firm purchase commitments.
Cost of Goods Sold
Cost of goods sold includes the cost of inventory sold, third party manufacturing and supply chain costs, product shipping, tracking and handling costs, and provisions for excess and obsolete inventory. Cost of goods sold also includes the cost of goods sold under the Company's license and collaboration agreements, which generally consists of the sale of active pharmaceutical ingredients to the Company's collaboration partners, generally at cost plus a margin.
Research and Development Expenses
Research and Development Expenses
Research and development includes expenses related to sparsentan, pegtibatinase, and the Company's other pipeline programs. The Company expenses all research and development costs as they are incurred. The Company's research and development costs are composed of salaries and bonuses, benefits, share-based compensation, license fees, milestones under license agreements, costs paid to third-party contractors to perform research, conduct clinical trials, costs to develop drug materials and delivery devices, costs to manufacture drug product supplies to support clinical development, and associated overhead expenses and facilities costs. The Company charges direct internal and external program costs to the respective development programs. The Company also incurs indirect costs that are not allocated to specific programs because such costs benefit multiple development programs and allow us to increase our pharmaceutical development capabilities. These consist of internal shared resources related to the development and maintenance of systems and processes applicable to all of our programs.
Nonrefundable advance payments for goods and services to be received in the future for use in research and development activities are recorded as prepaid expenses. The prepaid amounts are expensed as the related goods are delivered or the services are performed, or when it is no longer expected that the goods will be delivered or the services rendered.
During the three months ended December 31, 2022, the Company recorded an out of period adjustment that decreased research and development expenses and related accrued expenses by $7.7 million, $1.5 million of which was previously recorded in fiscal year 2022 and $6.2 million that was originally recorded in fiscal year 2021. The adjustment was the result of a certain pre-launch inventory contract that was not properly evaluated for accounting implications at inception. The Company evaluated the impact of the adjustment and concluded it is not material, individually and in the aggregate, to 2022 or any prior period financial statements.
Clinical Trial Expenses
Clinical Trial Expenses
The Company records expenses in connection with its clinical trials under contracts with contract research organizations ("CROs") that support conducting and managing clinical trials, as well as contract manufacturing organizations ("CMOs") for the manufacture of drug product supplies to support clinical development. The financial terms and activities of these agreements vary from contract to contract and may result in uneven expense levels. Generally, these agreements set forth activities that drive the recording of expenses such as start-up, initiation activities, enrollment, treatment of patients, or the completion of other clinical trial activities, and in the case of CMOs, costs associated with the production of drug product supplied and the procurement of raw materials to be consumed in the manufacturing process.
Expenses related to clinical trials are accrued based on our estimates of the progress of services performed, including actual level of patient enrollment, completion of patient studies and progress of the clinical trials or the delivery of goods. Other incidental costs related to patient enrollment or treatment are accrued when reasonably certain. If the amounts we are obligated to pay under our clinical trial agreements are modified (for instance, as a result of changes in the clinical trial protocol or scope of work to be performed), the Company adjusts its estimates accordingly on a prospective basis. Revisions to the Company's contractual payment obligations are charged to expense in the period in which the facts that give rise to the revision become reasonably certain.
The Company currently has four Phase 3 clinical trials in process that are in varying stages of activity, with ongoing non-clinical support trials. As such, clinical trial expenses will vary depending on all the factors set forth above and may fluctuate significantly from quarter to quarter.
Share-Based Compensation
Share-Based Compensation
The Company recognizes all employee share-based compensation as a cost within research and development expenses and selling, general, and administrative expenses. Equity-classified awards principally related to stock options, restricted stock units (“RSUs”) and performance stock units ("PSUs"), are measured at the grant date fair value of the award. The Company determines grant date fair value of stock option awards using the Black-Scholes option-pricing model. The fair value of RSUs and PSUs are determined using the closing price of the Company’s common stock on the grant date. For service based vesting grants, expense is recognized over the requisite service period. For PSUs, expense is recognized over the implicit service period, once vesting is probable. No expense is recognized for PSUs if it is not probable the vesting criteria will be satisfied. Forfeitures are accounted for as they occur.
Expiration TermVesting Term
Stock Options10 years
3 to 4 years
Restricted Stock Units----
1 to 4 years
Earnings (Loss) Per Share
Earnings (Loss) Per Share
The Company calculates basic earnings per share by dividing net income/(loss) by the weighted average number of shares outstanding during the period. Pre-funded warrants issued and sold by the Company to purchase shares of its common stock are included in the calculation of basic net loss per common share if the exercise price of the pre-funded warrant represents little consideration and is non-substantive in relation to the price paid for the warrant, and if the warrants are immediately exercisable with no further vesting conditions or contingencies associated with them.
The Company's diluted earnings/(loss) per share computation includes the effect, if any, of shares that would be issuable upon the exercise of outstanding stock options, convertible debt and RSUs, reduced by the number of shares which are assumed to be purchased by the Company from the resulting proceeds at the average market price during the year, when such amounts are dilutive to the earnings per share calculation. In accordance with ASC 260, Earnings per Share, if a company had a discontinued operation, the company uses income from continuing operations, adjusted for preferred dividend and similar adjustments, as its control number to determine whether potential common shares are dilutive.
Cash and Cash Equivalents
Cash and Cash Equivalents
The Company considers all highly liquid marketable securities with an original maturity of three months or less to be cash equivalents. Due to the short-term maturity of such investments, the carrying amounts are a reasonable estimate of fair value.
Concentration of Credit Risk
Concentration of Credit Risk
The Company maintains its cash and cash equivalents at insured financial institutions, the balances of which may, at times, exceed federally insured limits. Generally, these deposits may be redeemed upon demand, and the Company believes there is minimal risk of losses on such balances.
The Company monitors its investments with counterparties with the objective of minimizing concentrations of credit risk. The Company's investment policy is to invest only in institutions that meet high credit quality standards and established limits on the amount and time to maturity of investments with any individual counterparty. The policy also requires that investments are only entered into with corporate and financial institutions that meet high quality standards.
Marketable Debt Securities
Marketable Debt Securities
The Company classified marketable debt securities held as “available-for-sale” and carries them at fair value. The Company classifies these investments as current assets, even if the maturity when acquired by the Company is greater than one year due to the ability to liquidate within the next 12 months. The amortized cost of marketable debt securities is adjusted for amortization of premiums and accretion of discounts to maturity. Such amortization and accretion, as well as interest and dividends, is included in interest income. Unrealized gains and losses on marketable debt securities are recorded as a separate component of stockholders’ equity as accumulated other comprehensive loss, unless an impairment is determined to be the result of credit-related factors or the Company intends to sell the security or it is more likely than not that the Company will be required to sell the security before recovery. Unrealized losses that are determined to be credit-related are recorded as an allowance against the amortized cost basis. Realized gains or losses on debt security transactions and declines in value that are determined to be the result of credit losses, if any, are reported in other income or expense in the Consolidated Statements of Operations and Comprehensive Loss. The cost of securities sold is based on the specific identification method. Marketable debt securities are maintained at one financial institution and are governed by the Company’s investment policy. See Note 6 for further discussion.
Accounts Receivables, Net
Accounts Receivables, Net
Trade accounts receivable are recorded net of reserves for prompt pay discounts and expected credit losses. Estimates for allowances for credit losses are determined based on existing contractual obligations, historical payment patterns and individual customer circumstances. The allowance for credit losses was zero at both December 31, 2024 and 2023, respectively. For the years ended December 31, 2024, 2023 and 2022, bad debt expense recorded in the Consolidated Statements of Operations was immaterial. The Company's evaluation of credit losses for the current period included an assessment of our aged trade receivables balances and their underlying credit risk characteristics. Our evaluation of past events, current conditions, and reasonable and supportable forecasts about the future resulted in an expectation of immaterial credit losses.
Supplier Concentration Risk
Supplier Concentration Risk
The Company has no manufacturing capabilities and relies on third party manufacturers who are sole source suppliers for manufacturing of its products. The Company intends to rely on third-party manufacturers for the long-term commercial supply of FILSPARI and for its development stage product candidates, including sparsentan for the treatment of FSGS and pegtibatinase. The Company expects the manufacturers of each product or product candidate to, at least initially and potentially for a significant period of time, be single source suppliers to the Company.
Property and Equipment, net
Property and Equipment, net
Property and equipment are stated at cost, net of accumulated depreciation. Depreciation is computed using the straight-line method over the related estimated useful lives as presented in the table below. Significant additions and improvements are capitalized, while repairs and maintenance are charged to expense as incurred. Property and equipment purchased for specific research and development projects with no alternative use is expensed as incurred.
The major classifications of property and equipment, including their respective expected useful lives, consist of the following:
Computers and equipment3 years
Furniture and fixtures7 years
Leasehold improvementsShorter of length of lease or life of the asset
Leases
Leases
The Company determines whether a contract is, or contains, a lease at inception. The Company classifies each of its leases as operating or financing considering factors such as the length of the lease term, the present value of the lease payments, the nature of the asset being leased, and the potential for ownership of the asset to transfer during the lease term. Leases with terms greater than one year are recognized on the Consolidated Balance Sheets as Right-of-use assets and Lease liabilities and are measured at the present value of the fixed payments due over the expected lease term minus the present value of any incentives, rebates or abatement expected to be received from the lessor. Options to extend a lease are typically excluded from the expected lease term as the exercise of the option is typically not reasonably certain. The interest rate implicit in lease contracts is typically not readily determinable. As such, the Company utilizes the appropriate incremental borrowing rate, which is the rate incurred to borrow on a collateralized basis an amount equal to the lease payments over a similar term and in a similar economic environment.
In addition to rent, the leases may require the Company to pay additional amounts for taxes, insurance, maintenance, and other expenses, which are generally referred to as non-lease components. The Company has elected to not separate lease and non-lease components. Only the fixed costs for lease components and their associated non-lease components are accounted for as a single lease component and recognized as part of the Right-of-use assets and Lease liabilities. The Company records expense to recognize fixed lease payments, including payment escalation, on a straight-line basis over the expected lease term. Costs determined to be variable and not based on an index or rate are not included in the measurement of the lease liability and are expensed as incurred.
The Company has made an accounting policy election to not recognize short-term leases, or leases that have a lease term of 12 months or less at commencement date, within its Consolidated Balance Sheets and to recognize those lease payments in the Consolidated Statements of Operations on a straight-line basis over the lease term.
The Company recognizes income from sublet office space on a straight-line basis over the term of the sublease, recorded in other income in the Consolidated Statements of Operations.
Intangible Assets, Net and Intangible Assets with Cost Accumulation Model
Intangible Assets, Net
The Company's intangible assets consist of licenses and purchased technology. Intangible assets with definite lives are amortized on a straight-line basis over their estimated useful lives and are reviewed periodically for impairment.
Intangible Assets with Cost Accumulation Model
In 2014, the Company entered into a license agreement with Mission Pharmacal ("Mission") in which the Company obtained the exclusive right to license the trademark of Thiola ("Mission License Agreement"). The acquisition of the Thiola license qualified as an asset acquisition under the principles of ASC 805, Business Combinations ("ASC 805") in effect at the time of acquisition. The license agreement requires the Company to make royalty payments based on net sales of Thiola. The liability for royalties in excess of the annual contractual minimum is recognized in the period in which the royalties become probable and estimable, which is typically in the period corresponding with the respective sales. The Company records an offsetting increase to the cost basis of the intangible asset under the cost accumulation model ("Thiola Intangible"). The additional cost basis is subsequently amortized over the remaining estimated useful life of the license agreement.
In the second quarter of 2023, the Company reduced the estimated useful life of the Thiola Intangible to better reflect the pattern of projected future cash flows, resulting in incremental expense of $3.7 million recorded in selling, general, and administrative. The change in estimated useful life was accounted for as a change in accounting estimate and the remaining carrying amounts of the Thiola Intangible are being amortized prospectively over the new useful life.
Consistent with all prior periods since Thiola was acquired, the Company has not accrued any liability for future royalties in excess of the annual contractual minimum at December 31, 2024 as such royalties are not yet probable and estimable.
In 2012, the Company entered into an agreement with Ligand Pharmaceuticals, Inc. (“Ligand”) for a worldwide sublicense to develop, manufacture and commercialize sparsentan (the “Ligand License Agreement”). The acquisition of the Ligand License Agreement qualified as an asset acquisition under the principles of ASC 805 in effect at the time of acquisition. The license agreement requires the Company to make royalty payments based on net sales of FILSPARI (sparsentan) and milestone payments. The liabilities for royalties and milestone payments are recognized in the period in which they become probable and estimable, which is typically in the period corresponding with the respective sales or achievement of the milestone.
The Company records an offsetting increase to the cost basis of the intangible asset under the cost accumulation model following the approval of FILSPARI. The additional cost basis is subsequently amortized over the remaining estimated useful life.
Variable Interest Entity
Variable Interest Entity
The Company reviews each investment and collaboration agreement to determine if it has a variable interest in the entity. In assessing whether the Company has a variable interest in the entity as a whole, the Company considers and makes judgments regarding the purpose and design of the entity, the value of the licensed assets to the entity, the value of the entity’s total assets and the significant activities of the entity. If the Company has a variable interest in the entity as a whole, the Company assesses whether or not the Company is a primary beneficiary of that VIE, based on a number of factors, including: (i) which party has the power to direct the activities that most significantly affect the VIE’s economic performance, (ii) the parties’ contractual rights and responsibilities pursuant to the collaboration agreement, and (iii) which party has the obligation to absorb losses of or the right to receive benefits from the VIE that could be significant to the VIE. If the Company determines that it is the primary beneficiary of a VIE at the onset of the collaboration, the collaboration is treated as a business combination and the Company consolidates the financial statements of the VIE into the Company’s consolidated financial statements. On a quarterly basis, the Company evaluates whether it continues to be the primary beneficiary of the consolidated VIE. If the Company determines that it is no longer the primary beneficiary of a consolidated VIE, it deconsolidates the VIE in the period in which the determination is made.
Assets and liabilities recorded as a result of consolidating the financial results of the VIE into the Company’s consolidated balance sheet do not represent additional assets that could be used to satisfy claims against the Company’s general assets or liabilities for which creditors have recourse to the Company’s general assets.
Equity Securities
Equity Securities
The Company applies the equity method of accounting for investments when it has significant influence, but no controlling interest in the investee. Judgment regarding the level of influence over each equity method investment includes key factors such as ownership interest, representation on the board of directors, participation in joint steering committees and material intercompany transactions. Upon investment, the Company evaluates any basis difference between the carrying value and fair value of the Company's proportionate share of the investee's net assets. Basis differences relating to in-process research and development (IPR&D) are expensed when the investee is not considered a business as defined in ASC 805, Business Combinations, due to substantially all of the estimated fair value of the gross assets being concentrated in a group of similar IPR&D assets with no alternative future use. For the year ended December 31, 2024, the Company recognized $3.4 million in other (expense) income, net in the Company's Consolidated Statements of Operations for these basis adjustments. The equity method investment's carrying value was reduced to zero as the Company's proportionate share of the basis difference exceeded the carrying value. See Note 5 for further discussion. Investments accounted for using the equity method are be reported on a lag of up to three months if the financial statements of the investee are not available in sufficient time for the Company to apply the equity method as of the current reporting date.
Goodwill
Goodwill
Goodwill represents the excess of purchase price over fair value of net assets acquired in a business combination and is not amortized. Goodwill is subject to impairment testing at least annually or when a triggering event occurs that could indicate a potential impairment. The Company has one segment and one reporting unit and as such reviews goodwill for impairment at the consolidated level.
Impairment of Long-Lived Assets
Impairment of Long-Lived Assets
The Company's long-lived assets are primarily comprised of intangible assets, right-of-use assets, and property and equipment. The Company evaluates its finite-lived intangible assets, right-of-use assets, and property and equipment for impairment whenever events or changes in circumstances indicate the carrying value of an asset or group of assets may not be recoverable. If these circumstances exist, recoverability of assets to be held and used is measured by a comparison of the carrying amount of an asset group to future undiscounted net cash flows expected to be generated by the use and eventual disposition of the asset group. If such assets are considered to be impaired, the impairment to be recognized is measured by the amount by which the carrying amount of the assets exceeds the fair value of the assets.
In addition, indefinite-lived intangible assets are reviewed for impairment annually and whenever events or changes in circumstances indicate that it is more likely than not that the asset is impaired by comparing the fair value to the carrying value of the asset. To determine the fair value of the asset, the Company used the multi-period excess earnings method of the income approach. The more significant assumptions inherent in the application of this method include: the amount and timing of projected future cash flows (including revenue, cost of sales, research and development costs, and sales and marketing expenses), and the discount rate selected to measure the risks inherent in the future cash flows. See Note 8 for further discussion of certain long-lived assets measured at fair value on a nonrecurring basis when there are indicators of impairment.
Income Taxes
Income Taxes
The Company follows ASC 740, Income Taxes ("ASC 740"), which requires recognition of deferred tax assets and liabilities for the expected future tax consequences of events that have been included in the financial statements or tax returns. Under this method, deferred tax assets and liabilities are based on the differences between the financial statement and tax basis of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. Deferred tax assets are reduced by a valuation allowance to the extent management concludes it is more likely than not that the asset will not be realized.
The standard addresses the determination of whether tax benefits claimed or expected to be claimed on a tax return should be recorded in the financial statements. Under ASC 740, the Company may recognize the tax benefit from an uncertain tax position only if it is more likely than not that the tax position will be sustained on examination by the tax authorities, based on the technical merits of the position. The tax benefits recognized in the financial statements from such a position should be measured based on the largest benefit that has a greater than fifty percent likelihood of being realized upon ultimate settlement. ASC 740 also provides guidance on de-recognition, classification, interest and penalties on income taxes, accounting in interim periods and requires increased disclosures. The Company’s policy is to record estimated interest and penalty related to the underpayment of income taxes or unrecognized tax benefits as a component of its income tax provision.
Foreign Currency Translation
Foreign Currency Translation
Functional and presentation currency
Items included in the financial statements of each entity comprising the Company are measured using the currency of the primary economic environment in which the entity operates (the functional currency).
Transactions and balances
Foreign currency transactions in each entity comprising the Company are remeasured into the functional currency of the entity using the exchange rates prevailing at the respective transaction dates. Foreign exchange gains and losses resulting from the settlement of such transactions and from the remeasurement at year-end exchange rates of monetary assets and liabilities denominated in foreign currencies are recognized within Other income (expense), net in the Consolidated Statements of Operations and Comprehensive Loss.
An aggregate loss of $0.7 million arising from foreign exchange transactions is included in other (expense) income, net for the year ended December 31, 2024. An aggregate gain of $0.5 million and $1.4 million is included in other (expense) income, net for the years ended December 31, 2023 and 2022, respectively.
The results and financial position of the Company that have a functional currency different from the U.S. dollar are translated as follows:
a.assets and liabilities presented are translated at the closing exchange rate as of December 31, 2024 and 2023;
b.income and expenses for the statements of operations and comprehensive loss are translated at average exchange rates that are relevant for the respective periods for which the income and expenses occurred; and
c.significant transactions use the exchange rate on the date of the transaction.
All resulting exchange differences arising from such translations are recognized directly in comprehensive income and presented as a separate component of equity.
Reclassifications
Reclassifications 
Certain reclassifications have been made to the prior year financial statements in order to conform to the current year’s presentation. These reclassifications did not have an impact on total assets or total liabilities and stockholders' equity in the Consolidated Balance Sheets or net loss in the Consolidated Statements of Operations.
Patents
Patents
The Company expenses external costs, such as filing fees and associated attorney fees, incurred to obtain issued patents and patent applications pending. The Company also expenses costs associated with maintaining and defending patents subsequent to their issuance in the period incurred.
Legal Contingencies
Legal Contingencies
The Company may, from time to time, be involved in various claims and legal actions that arise in the ordinary course of business. The Company accrues for legal contingencies when it is determined probable that a liability has been incurred and the amount of the loss can be reasonably estimated.
Discontinued Operations
Discontinued Operations
Discontinued operations is presented when there is a disposal of a component or a group of components that in the Company's judgment represents a strategic shift that will have a major effect on the Company's operations and financial results. Results of operations directly related to discontinued operations are aggregated into a single line item in the Consolidated Statements of Operations for all periods presented.
Restructuring
Restructuring
Restructuring charges consist primarily of employee severance, one-time termination benefits related to the reduction of its workforce, and other costs. Liabilities for costs associated with a restructuring activity are recognized when the liability is incurred and are measured at fair value. One-time termination benefits are expensed at the date the entity notifies the employee, unless the employee must provide future service, in which case the benefits are expensed ratably over the service period. Termination benefits are calculated based on regional benefit practices and local statutory requirements.
In December 2023, the Company initiated a restructuring plan that resulted in a reduction of its workforce, primarily impacting non-field-based employees. Restructuring costs were primarily comprised of one-time termination benefits, including severance, continuation of health insurance coverage, and other benefits for a specified period of time. In 2024, we also recognized charges for impairment of operating lease right-of-use assets and related leasehold improvements, as well as disposal costs on furniture and fixtures associated with available office space that the Company has sublet as a result of the reduction in occupancy. As of December 31, 2024, we are no longer incurring restructuring expenses. Of the $13.8 million recognized to date, $2.4 million was recognized during the year ended December 31, 2024, including $1.2 million related to the impairment and disposal costs and initial direct costs to obtain the sublease.
Recently Adopted and Issued Accounting Pronouncements
Recently Adopted Accounting Pronouncements
In November 2023, the FASB issued ASU No. 2023-07, Improvements to Reportable Segment Disclosures. The FASB amended the guidance in ASC 280, Segment Reporting ("ASC 280"), to require a public entity to disclose significant segment expenses and other segment items on an annual and interim basis and to provide in interim periods all disclosures about a reportable segment's profit or loss and assets that are currently required annually. Public entities with a single reportable segment are required to provide the new disclosures and all the disclosures required under ASC 280. The guidance is applied retrospectively to all periods presented in financial statements, unless it is impracticable. This new guidance is effective for public business entities for annual periods beginning after December 15, 2023, and for interim periods beginning after December 15, 2024. The Company adopted this new standard effective December 31, 2024. See Note 20, Segment Information, for disclosures related to the adoption of ASU 2023-07.
In August 2020, the FASB issued ASU No. 2020-06, Accounting for Convertible Instruments and Contracts in an Entity's Own Equity. The ASU includes amendments to the guidance on convertible instruments and the derivative scope exception for contracts in an entity's own equity in Subtopic 815-40 and simplifies the accounting for convertible instruments which include beneficial conversion features or cash conversion features by removing certain separation models in Subtopic 470-20. Additionally, the ASU requires entities to use the "if-converted" method when calculating diluted earnings per share for convertible instruments. The adoption of the new standard impacted the Company's accounting for its Convertible Senior Notes Due 2025 (2025 Notes), discussed in Note 7, which were previously accounted for using the cash conversion model applied under ASC 470-20, Debt with Conversion and Other Options ("ASC 470-20"). The Company adopted ASU 2020-06 on January 1, 2022 using the modified retrospective method. The cumulative effect of the accounting change as of January 1, 2022 increased the carrying amount of the 2025 Notes by $44.7 million, reduced additional paid-in capital by $74.9 million, and reduced accumulated deficit by $30.2 million.
Recently Issued Accounting Pronouncements
From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board ("FASB") or other standard setting bodies. Unless otherwise discussed, the Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on its consolidated financial position or results of operations upon adoption.
In November 2024, the FASB issued ASU No. 2024-04, Debt with Conversion and Other Options (Subtopic 470-20): Induced Conversions of Convertible Debts Instruments. This ASU clarifies the requirements for determining whether to account for certain early settlements of convertible debt instruments as induced conversions or extinguishments. The guidance is effective for fiscal years beginning after December 15, 2025, with early adoption permitted for entities that have adopted 2020-06. The Company is currently evaluating the impact of the adoption of this standard on the accounting for the Company's convertible notes.
In November 2024, the FASB issued ASU No. 2024-03, Income Statement - Reporting Comprehensive Income - Expense Recognition Disclosures. This ASU will require entities to provide enhanced disclosures related to certain expense categories included in income statement captions. The ASU aims to increase transparency and provide investors with more detailed information about the nature of expenses reported on the face of the income statement. The new standard does not change the requirements for the presentation of expenses on the face of the income statement. Under this ASU, entities are required to disaggregate, in a tabular format, expense captions presented on the face of the income statement — excluding earnings or losses from equity method investments — if they include any of the following expense categories: purchases of inventory, employee compensation, depreciation, intangible asset amortization, and depreciation or depletion. For any remaining items within each relevant expense caption, entities must provide a qualitative description of the nature of those expenses. The new ASU is effective for annual reporting periods beginning after December 15, 2026 and interim reporting periods beginning after December 15, 2027. Early adoption is permitted. The Company is currently evaluating the impact of the adoption of this standard on the related disclosures.
In December 2023, the FASB issued ASU No. 2023-09, Improvements to Income Tax Disclosures. This ASU does not change accounting for income taxes but requires new disclosures focusing on two areas, the effective rate reconciliation and taxes paid. This new standard is effective for public business entities for annual periods beginning after December 15, 2024. Early adoption is permitted. The Company is currently evaluating the impact of the adoption of this standard on the related disclosures.
Fair Value Measurements
The Company utilizes a fair value hierarchy that prioritizes the inputs to valuation techniques used to measure fair value. The hierarchy gives the highest priority to unadjusted quoted prices in active markets for identical assets or liabilities (Level 1 measurements) and the lowest priority to unobservable inputs (Level 3 measurements). The three levels of the fair value hierarchy are described below:
Level 1 – Unadjusted quoted prices in active markets that are accessible at the measurement date for identical, unrestricted assets or liabilities;
Level 2 – Quoted prices in markets that are not active or financial instruments for which all significant inputs are observable, either directly or indirectly; and
Level 3 – Prices or valuations that require inputs that are both significant to the fair value measurement and unobservable.
The valuation techniques used to measure the fair value of the Company’s debt securities and all other financial instruments, all of which have counterparties with high credit ratings, were valued based on quoted market prices or model driven valuations using significant inputs derived from or corroborated by observable market data. Based on the fair value hierarchy, the Company classified marketable debt securities within Level 2.
v3.25.0.1
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Tables)
 12 Months Ended
Dec. 31, 2024
Accounting Policies [Abstract]  
Schedule of Inventory, Net of Reserves
Inventory, net of reserves, consisted of the following at December 31, 2024 and 2023 (in thousands):
December 31, 2024December 31, 2023
Raw materials$30,552 $33,790 
Work in process7,625 4,727 
Finished goods3,679 2,387 
Total inventory$41,856 $40,904 
Classified as:
Inventory$6,200 $9,410 
Long-term inventory35,656 31,494 
Total inventory$41,856 $40,904 
Schedule Of Cost Of Goods Sold
The following table summarizes cost of goods sold for the years ended December 31, 2024, 2023 and 2022 (in thousands):
Year Ended December 31,
202420232022
Cost of goods sold - product sales$7,446 $8,406 $4,420 
Cost of goods sold - license and collaboration298 3,044 — 
Total cost of goods sold$7,744 $11,450 $4,420 
Schedule of Vesting Award Terms
Expiration TermVesting Term
Stock Options10 years
3 to 4 years
Restricted Stock Units----
1 to 4 years
Schedule of Major Classifications of Property and Equipment
The major classifications of property and equipment, including their respective expected useful lives, consist of the following:
Computers and equipment3 years
Furniture and fixtures7 years
Leasehold improvementsShorter of length of lease or life of the asset
Property, plant and equipment, net consisted of the following (in thousands):
December 31,
20242023
Leasehold improvements$9,370 $9,692 
Furniture and fixtures2,446 2,990 
Computers and equipment2,275 2,071 
Construction-in-progress— 169 
14,091 14,922 
Less: Accumulated depreciation(8,755)(7,443)
Total property and equipment, net$5,336 $7,479 
Schedule of Foreign Currency Translation Adjustment Included in Accumulated Other Comprehensive Loss
The following table summarizes the foreign currency translation adjustment included in accumulated other comprehensive loss for the year ended December 31, 2024, 2023 and 2022 (in thousands):
Foreign Currency Translation Adjustments included in Accumulated Other Comprehensive (Loss) Gain
202420232022
Balance at January 1,$(1,456)$415 $(92)
Foreign currency translation adjustments1,422 (1,871)507 
Balance at December 31,$(34)$(1,456)$415 
Schedule of Restructuring and Related Costs
20242023
Liability balance at January 1,$11,421 $— 
Restructuring expenses2,438 11,394 
Non-cash impairment and disposal charges(856)— 
Payments(12,929)— 
Foreign currency impact(74)27 
Liability balance at December 31,$— $11,421 
v3.25.0.1
REVENUE RECOGNITION (Tables)
 12 Months Ended
Dec. 31, 2024
Revenue from Contract with Customer [Abstract]  
Schedule of Net Product Sales
The following table summarizes net product sales for the year ended December 31, 2024, 2023 and 2022 (in thousands):
Year Ended December 31,
202420232022
FILSPARI$132,222 $29,208 $— 
Tiopronin products94,485 98,329 97,970 
Total net product sales$226,707 $127,537 $97,970 
v3.25.0.1
COLLABORATION AND LICENSE AGREEMENTS (Tables)
 12 Months Ended
Dec. 31, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]  
Summary of Changes in Deferred Revenue
The following table sets forth a summary of changes in deferred revenue for the years ended December 31, 2024, 2023 and 2022 (in thousands):
Deferred Revenue
202420232022
Balance at January 1,$8,931 $22,907 $36,647 
License and collaboration revenue(5,847)(14,363)(11,490)
Foreign currency impact(269)387 (2,250)
Balance at December 31,$2,815 $8,931 $22,907 
v3.25.0.1
MARKETABLE DEBT SECURITIES (Tables)
 12 Months Ended
Dec. 31, 2024
Investments, Debt and Equity Securities [Abstract]  
Schedule of Marketable Debt Securities
Marketable debt securities consisted of the following (in thousands):
As of December 31,
20242023
Marketable debt securities:
Commercial paper$73,325 $34,458 
Corporate debt securities203,816 368,323 
Securities of government-sponsored entities35,025 105,894 
Total available-for-sale marketable debt securities$312,166 $508,675 
Schedule of Short-term Marketable Debt Securities
The following is a summary of short-term marketable debt securities classified as available-for-sale as of December 31, 2024 (in thousands):
Remaining Contractual Maturity
(in years)		Amortized CostUnrealized GainsUnrealized LossesAggregate Estimated Fair Value
Marketable debt securities:
Commercial paperLess than 1$73,410 $1 $(86)$73,325 
Corporate debt securitiesLess than 1203,395 483 (62)203,816 
Securities of government-sponsored entitiesLess than 134,993 33 (1)35,025 
Total available-for-sale marketable debt securities$311,798 $517 $(149)$312,166 
The following is a summary of short-term marketable debt securities classified as available-for-sale as of December 31, 2023 (in thousands):
Remaining Contractual Maturity
(in years)		Amortized CostUnrealized GainsUnrealized LossesAggregate Estimated Fair Value
Marketable debt securities:
Commercial paperLess than 1$34,450 $25 $(17)$34,458 
Corporate debt securitiesLess than 1133,463 29 (408)133,084 
Securities of government-sponsored entitiesLess than 181,334 36 (274)81,096 
Total maturity less than 1 year249,247 90 (699)248,638 
Corporate debt securities1 to 2233,969 1,444 (174)235,239 
Securities of government-sponsored entities1 to 224,718 106 (26)24,798 
Total maturity 1 to 2 years258,687 1,550 (200)260,037 
Total available-for-sale marketable debt securities$507,934 $1,640 $(899)$508,675 
Schedule of Debt Securities in Unrealized Loss Position
The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of December 31, 2024 (in thousands):
Less Than 12 Months12 Months or GreaterTotal
Description of SecuritiesFair ValueUnrealized LossesFair ValueUnrealized LossesFair ValueUnrealized Losses
Commercial paper$68,446 $86 $— $— $68,446 $86 
Corporate debt securities40,112 56 9,969 6 50,081 62 
Securities of government-sponsored entities— — 4,975 1 4,975 1 
Total$108,558 $142 $14,944 $7 $123,502 $149 
The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of December 31, 2023 (in thousands):
Less Than 12 Months12 Months or GreaterTotal
Description of SecuritiesFair ValueUnrealized LossesFair ValueUnrealized LossesFair ValueUnrealized Losses
Commercial paper$24,798 $17 $— $— $24,798 $17 
Corporate debt securities140,802 405 28,775 177 169,577 582 
Securities of government-sponsored entities61,933 217 12,540 83 74,473 300 
Total$227,533 $639 $41,315 $260 $268,848 $899 
v3.25.0.1
CONVERTIBLE NOTES PAYABLE (Tables)
 12 Months Ended
Dec. 31, 2024
Debt Disclosure [Abstract]  
Schedule of Long-term Debt Instruments
The composition of the Company’s convertible senior notes are as follows (in thousands):
 December 31, 2024December 31, 2023
2.25% convertible senior notes due 2029
$316,250 $316,250 
2.50% convertible senior notes due 2025
68,904 68,904 
Unamortized debt issuance costs - 2.25% convertible senior notes due 2029
(5,940)(7,348)
Unamortized debt issuance costs - 2.50% convertible senior notes due 2025
(226)(543)
Total convertible senior notes, net of unamortized debt discount and debt issuance costs$378,988 $377,263 
Classified as:
Convertible debt, current portion$68,678 $— 
Convertible debt, less current portion310,310 377,263 
Total convertible debt$378,988 $377,263 
The following table sets forth total interest expense recognized related to the 2025 and 2029 Notes (in thousands):
Year Ended December 31,
202420232022
Contractual interest expense$8,838 $8,838 $8,433 
Amortization of debt issuance costs1,725 1,718 1,622 
Total interest expense for the 2025 and 2029 Notes$10,563 $10,556 $10,055 
v3.25.0.1
FAIR VALUE MEASUREMENTS (Tables)
 12 Months Ended
Dec. 31, 2024
Fair Value Disclosures [Abstract]  
Schedule of Fair Value on a Recurring Basis
The following table presents the Company’s assets, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of December 31, 2024 (in thousands):
 As of December 31, 2024Fair Value Hierarchy at December 31, 2024
Total carrying and estimated fair valueQuoted prices in active markets (Level 1)Significant other observable inputs (Level 2)Significant unobservable inputs (Level 3)
Assets:
Cash and Cash Equivalents$58,535 $51,060 $7,475 $— 
Marketable debt securities, available-for-sale312,166 — 312,166 — 
Total$370,701 $51,060 $319,641 $— 
The following table presents the Company’s assets, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of December 31, 2023 (in thousands):
 As of December 31, 2023Fair Value Hierarchy at December 31, 2023
Total carrying and estimated fair valueQuoted prices in active markets (Level 1)Significant other observable inputs (Level 2)Significant unobservable inputs (Level 3)
Assets:
Cash and Cash Equivalents$58,176 $58,176 $— $— 
Marketable debt securities, available-for-sale508,675 — 508,675 — 
Total$566,851 $58,176 $508,675 $— 
v3.25.0.1
INTANGIBLE ASSETS (Tables)
 12 Months Ended
Dec. 31, 2024
Goodwill and Intangible Assets Disclosure [Abstract]  
Schedule of Amortizable Intangible Assets
Amortizable intangible assets as of December 31, 2024 (in thousands):
 Useful LifeGross Carrying AmountAccumulated AmortizationNet Book Value
Mission license12$171,025 $(115,303)$55,722 
Ligand license1153,268 (5,769)47,499 
Total amortizable intangible assets$224,293 $(121,072)$103,221 
Amortizable intangible assets as of December 31, 2023 (in thousands):
Useful LifeGross Carrying AmountAccumulated AmortizationNet Book Value
Mission license12$155,589 $(77,311)$78,278 
Ligand license1127,448 (2,036)25,412 
Total amortizable intangible assets$183,037 $(79,347)$103,690 
Schedule of Amortization Expense
The following table summarizes amortization expense for the year ended December 31, 2024, 2023 and 2022 (in thousands):
202420232022
Selling, general and administrative$41,739 $29,021 $12,339 
Research and development— 7,261 6,264 
Total amortization expense$41,739 $36,282 $18,603 
As of December 31, 2024, amortization expense for the next five years and thereafter is expected to be as follows (in thousands):
2025$50,397 
202616,722 
20275,700 
20285,714 
20295,700 
Thereafter18,988 
Total$103,221 
v3.25.0.1
ACCRUED EXPENSES (Tables)
 12 Months Ended
Dec. 31, 2024
Payables and Accruals [Abstract]  
Schedule of Accrued Expenses
Accrued expenses consist of the following at December 31, 2024 and 2023 (in thousands):
20242023
Compensation related costs$35,166 $29,908 
Research and development16,090 26,006 
Accrued royalties12,309 6,991 
Sales discounts, rebates, and allowances10,585 13,730 
Selling, general and administrative6,154 7,190 
Transition services accrual285 12,282 
Accrued restructuring costs— 11,421 
Miscellaneous accrued expenses5,439 11,463 
Total accrued expenses$86,028 $118,991 
v3.25.0.1
STOCKHOLDERS’ EQUITY (Tables)
 12 Months Ended
Dec. 31, 2024
Equity [Abstract]  
Schedule of Assumptions used in Black-scholes Options Pricing Model The following weighted average assumptions were used in the Black-Scholes options pricing model to estimate the fair value of stock options for the specified reporting periods:
Year Ended December 31,
 202420232022
Risk free rate3.9 %3.9 %1.7 %
Expected volatility59 %50 %50 %
Expected life (in years)6.26.46.4
Expected dividend yield— — — 
Schedule of Share-based Compensation, Stock Options, Activity
The following table summarizes our stock option activity and related information for the year ended December 31, 2024:
Weighted Average
Shares Underlying
Options		Exercise
Price		Remaining
Contractual
Term (in years)		Aggregate
Intrinsic Value
(in thousands)
Outstanding at December 31, 202310,211,353 $21.52 4.91$— 
Granted1,524,300 8.63 — — 
Forfeited and expired(2,076,808)20.02 — — 
Exercised(375,514)11.86 — 2,016 
Outstanding at December 31, 20249,283,331 $20.13 5.51$16,408 
Vested and expected to vest at December 31, 20249,283,331 $20.13 5.51$16,408 
The following table summarizes our stock options exercisable at December 31, 2024, 2023 and 2022:
Weighted Average
Shares Underlying
Options		Exercise
Price		Remaining
Contractual
Term (in years)		Aggregate
Intrinsic Value
(in thousands)
Exercisable at December 31, 20226,999,669 $20.22 4.62$21,806 
Exercisable at December 31, 20237,942,964 $20.85 3.95$— 
Exercisable at December 31, 20246,892,156 $22.11 4.46$3,249 
Schedule of Restricted Stock Unit Activity
The following table summarizes our restricted stock unit activity for the year ended December 31, 2024:
Number of
RSUs		Weighted Average
Grant Date Fair Value
Unvested December 31, 20232,874,046 $22.97 
Granted2,077,025 9.07 
Vested(1,013,811)22.52 
Forfeited/cancelled(419,997)18.66 
Unvested December 31, 20243,517,263 $15.41 
Schedule of Performance-based Stock Unit Activity
The following table summarizes our performance-based stock unit activity for the year ended December 31, 2024:
 Number of
PSUs		Weighted Average
Grant Date Fair Value
Unvested December 31, 2023175,458 $25.61 
Granted107,000 8.93 
Vested(66,250)22.40 
Forfeited/cancelled— — 
Unvested December 31, 2024216,208 $18.34 
Schedule of Non-cash Stock-based Compensation Expense
Total non-cash stock-based compensation expense consisted of the following for the years ended December 31, 2024, 2023 and 2022 (in thousands):
 Year Ended December 31,
 202420232022
Selling, general and administrative expenses$22,735 $27,111 $24,368 
Research and development expenses14,178 17,135 13,775 
Total stock-based compensation expense$36,913 $44,246 $38,143 
v3.25.0.1
NET LOSS PER COMMON SHARE (Tables)
 12 Months Ended
Dec. 31, 2024
Earnings Per Share [Abstract]  
Schedule of Basic and Diluted Net Loss Per Share
Basic and diluted net loss per share is calculated as follows (net loss amounts are stated in thousands):
For the year ended December 31,
202420232022
SharesNet (loss) incomeEPSSharesNet (loss) incomeEPSSharesNet (loss) incomeEPS
Continuing operations78,888,861 $(320,630)$(4.07)74,267,418 $(376,333)$(5.07)63,758,515 $(331,468)$(5.20)
Discontinued operations78,888,861 (915)(0.01)74,267,418 264,934 3.57 63,758,515 52,986 0.83 
Basic and diluted loss per share78,888,861 $(321,545)$(4.08)74,267,418 $(111,399)$(1.50)63,758,515 $(278,482)$(4.37)
Schedule of Anti-dilutive Securities
For the years ended December 31, 2024, 2023 and 2022, the following weighted-average number of common stock equivalents were excluded because they were anti-dilutive:
For the year ended December 31,
202420232022
Convertible debt11,697,953 11,697,952 10,869,000 
Options10,447,541 10,555,550 10,041,249 
Restricted stock units and performance-based stock units3,780,441 3,417,245 2,175,100 
Total anti-dilutive shares25,925,935 25,670,747 23,085,349 
v3.25.0.1
INCOME TAXES (Tables)
 12 Months Ended
Dec. 31, 2024
Income Tax Disclosure [Abstract]  
Schedule of Net Loss from Continuing Operations Before Income Taxes
For financial reporting purposes, net loss from continuing operations before income taxes includes the following components (in thousands):
Year Ended December 31,
202420232022
United States$(152,356)$(293,283)$(268,887)
Foreign(168,154)(82,827)(62,268)
Total$(320,510)$(376,110)$(331,155)
Schedule of Components of Provision (Benefit) for Income Taxes
The components of the provision for income taxes, in the Consolidated Statements of Operations are as follows (in thousands):
 202420232022
Current
Federal$— $— $— 
State120 223 314 
Foreign— — (1)
Total current120 223 313 
Deferred
Federal— — — 
State— — — 
Total deferred— — — 
Total tax provision$120 $223 $313 
Schedule of Reconciliation of Statutory Federal Income Tax Rate
The following is a reconciliation of the statutory federal income tax rate to the Company’s effective tax rate expressed as a percentage of loss before income taxes:
 202420232022
Statutory rate - federal(21.00)%(21.00)%(21.00)%
State taxes, net of federal benefit(1.55)%(3.19)%(3.62)%
Foreign rate differential2.15 %1.29 %0.81 %
IPR&D4.27 %— %— %
Nondeductible executive compensation0.44 %1.50 %0.41 %
Excess tax benefits associated with share-based awards2.92 %0.68 %(0.17)%
Other permanent differences0.23 %0.37 %0.17 %
Tax credits(4.24)%(1.13)%(5.29)%
Return to provision adjustments and other true-ups(0.80)%4.19 %(0.17)%
Adoption of ASU 2020-06— %— %(3.54)%
Other0.93 %0.57 %— %
Change in valuation allowance16.69 %16.78 %32.49 %
Income tax provision0.04 %0.06 %0.09 %
Schedule of Deferred Tax Assets and Liabilities
The significant components of the Company’s deferred tax assets and liabilities as of December 31, 2024 and 2023 are as follows (in thousands):
 20242023
Deferred Tax Assets:
Net operating loss$113,303 $81,531 
Research and development and other tax credits91,760 77,555 
Intangible assets53,919 47,731 
Capitalized research and development47,880 39,907 
Stock based compensation15,680 21,607 
Other accrued expenses14,266 13,187 
Charitable contributions5,797 6,110 
Operating lease liabilities5,421 6,812 
Depreciation435 292 
Loan costs153 62 
Interest expense limitation— 1,679 
Total deferred tax assets348,614 296,473 
Deferred Tax Liabilities:
Operating lease right of use assets(4,259)(5,595)
Prepaid assets(178)(198)
Total deferred tax liabilities(4,437)(5,793)
Net deferred tax assets before valuation allowance344,177 290,680 
Valuation allowance(344,177)(290,680)
Total deferred tax assets$— $— 
Schedule of Unrecognized Tax Benefits
A reconciliation of the Company's unrecognized tax benefits for the years 2024, 2023 and 2022 is provided in the following table (in thousands):
202420232022
Balance as of January 1:$22,906 $11,490 $7,825 
Increase in current period positions4,248 4,871 2,056 
Increase in prior period positions250 7,383 1,919 
Decrease in prior period positions— (838)— 
Decrease due to settlements with tax authorities— — (310)
Balance as of December 31:$27,404 $22,906 $11,490 
v3.25.0.1
PROPERTY AND EQUIPMENT (Tables)
 12 Months Ended
Dec. 31, 2024
Property, Plant and Equipment [Abstract]  
Schedule of Property, Plant, and Equipment, Net
The major classifications of property and equipment, including their respective expected useful lives, consist of the following:
Computers and equipment3 years
Furniture and fixtures7 years
Leasehold improvementsShorter of length of lease or life of the asset
Property, plant and equipment, net consisted of the following (in thousands):
December 31,
20242023
Leasehold improvements$9,370 $9,692 
Furniture and fixtures2,446 2,990 
Computers and equipment2,275 2,071 
Construction-in-progress— 169 
14,091 14,922 
Less: Accumulated depreciation(8,755)(7,443)
Total property and equipment, net$5,336 $7,479 
v3.25.0.1
LEASES (Tables)
 12 Months Ended
Dec. 31, 2024
Leases [Abstract]  
Schedule of Future Minimum Rental Commitments
The following is a schedule of the future minimum rental commitments for the Company's operating leases reconciled to the lease liability and ROU asset as of December 31, 2024 (in thousands):
December 31, 2024
2025$6,665 
20266,889 
20277,064 
20284,781 
Thereafter— 
Total undiscounted future minimum payments25,399 
Present value discount(2,803)
Total lease liability22,596 
Unamortized lease incentives(3,491)
Cash payments in excess of straight-line lease expense(4,810)
Total ROU asset$14,295 
Schedule of Weighted-average Remaining Lease Term and Discount Rate
The weighted-average remaining lease term and weighted-average discount rate of the Company's operating leases are as follows:
December 31,
20242023
Weighted-average remaining lease term in years3.74.7
Weighted-average discount rate6.48 %6.48 %
v3.25.0.1
DIVESTITURES (Tables)
 12 Months Ended
Dec. 31, 2024
Discontinued Operations and Disposal Groups [Abstract]  
Schedule of Results, Assets and Liabilities of Discontinued Operations
Results of discontinued operations are as follows (in thousands):
Year Ended December 31,
202420232022
Net product sales$(550)$66,164 $102,558 
Total revenue(550)66,164 102,558 
Operating expenses:
Cost of goods sold(9)1,899 3,172 
Research and development247 6,118 8,447 
Selling, general and administrative127 19,500 22,686 
Change in fair value of contingent consideration— (473)15,006 
Total operating expenses365 27,044 49,311 
Operating income(915)39,120 53,247 
Other income (expenses), net:
Interest expense— (191)(261)
Gain on disposal of discontinued operations, net of tax— 226,005 — 
Total other income (expense), net— 225,814 (261)
(Loss) income from discontinued operations before income tax(915)264,934 52,986 
Income tax on discontinued operations— — — 
Net (loss) income from discontinued operations$(915)$264,934 $52,986 
v3.25.0.1
SEGMENT INFORMATION (Tables)
 12 Months Ended
Dec. 31, 2024
Segment Reporting [Abstract]  
Schedule of Segment Reporting Information, by Segment
The following table presents reportable segment loss, including significant expenses regularly provided to the CODM, attributable to the Company’s reportable segment for the years ended December 31, 2024, 2023 and 2022 (in thousands):
Year Ended December 31,
202420232022
Revenue$233,175 $145,238 $109,460 
Less:
Cost of goods sold7,744 11,450 4,420 
Research and development:
External research and development126,303 142,482 129,698 
Internal personnel costs73,843 84,658 75,664 
Other Research and development17,350 17,850 21,971 
Total research and development217,496 244,990 227,333 
Selling, general and administrative264,119 265,542 197,520 
In-process research and development65,205 — — 
Restructuring2,438 11,394 — 
Total other income (expense), net3,317 12,028 (11,342)
Income tax provision on continuing operations(120)(223)(313)
(Loss) income from discontinued operations, net of tax(915)264,934 52,986 
Net loss$(321,545)$(111,399)$(278,482)
v3.25.0.1
DESCRIPTION OF BUSINESS (Details) - USD ($)
$ in Millions
Aug. 31, 2023
Apr. 30, 2024
Sep. 30, 2021
Vifor Pharma | Collaborative Arrangement      
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]      
Milestone payments (up to)     $ 845.0
Vifor Pharma | Regulatory Milestone | Collaborative Arrangement      
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]      
Milestone payments (up to)   $ 17.5  
Disposed of by Sale | Bile Acid Product Portfolio      
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]      
Proceeds from sale of business $ 210.0    
Consideration receivable on sale of business (up to) 235.0    
Minimum | Disposed of by Sale | Bile Acid Product Portfolio      
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]      
Milestone thresholds 125.0    
Maximum | Disposed of by Sale | Bile Acid Product Portfolio      
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]      
Milestone thresholds $ 500.0    
v3.25.0.1
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Narrative (Details)
 1 Months Ended 3 Months Ended 12 Months Ended
Mar. 31, 2024
USD ($)
Jun. 30, 2023
USD ($)
Dec. 31, 2022
USD ($)
Dec. 31, 2024
USD ($)
clinicalTrial
segment
reporting_unit
Dec. 31, 2023
USD ($)
Dec. 31, 2022
USD ($)
Dec. 31, 2021
USD ($)
Jan. 01, 2022
USD ($)
New Accounting Pronouncements or Change in Accounting Principle [Line Items]                
Inventory valuation reserves       $ 3,700,000 $ 2,400,000      
Total inventory       41,856,000 40,904,000      
Cost of goods sold       7,744,000 11,450,000 $ 4,420,000    
Loss on allowance for inventory       $ 2,819,000 3,039,000 1,039,000    
Decreased in research and development expenses and related accrued expenses     $ 7,700,000     1,500,000 $ 6,200,000  
Number of phase 3 clinical trials in process | clinicalTrial       4        
Accounts receivable, allowance for credit loss       $ 0 0      
Impairment of intangible assets, finite-lived       0 0 0    
Impairment, Intangible Asset, Finite-Lived, Statement of Income or Comprehensive Income [Extensible Enumeration]   Selling, general and administrative            
In-process research and development $ 65,200,000     65,205,000 0 0    
Equity method investments       $ 0        
Number of segments | segment       1        
Number of reporting units | reporting_unit       1        
Foreign currency transaction gain (loss)       $ (700,000) 500,000 1,400,000    
Restructuring costs incurred to date       13,800,000        
Restructuring expenses       2,438,000 11,394,000 0    
Impairment charges       1,200,000 0 $ 0    
Additional paid-in capital       (1,506,315,000) (1,327,881,000)      
Reduction in accumulated deficit       (1,447,167,000) (1,125,622,000)      
Mission license                
New Accounting Pronouncements or Change in Accounting Principle [Line Items]                
Impairment of intangible assets, finite-lived   $ 3,700,000            
Equity Method Investments                
New Accounting Pronouncements or Change in Accounting Principle [Line Items]                
In-process research and development       3,400,000        
Cumulative Effect, Period of Adoption, Adjustment | Accounting Standards Update 2020-06                
New Accounting Pronouncements or Change in Accounting Principle [Line Items]                
Convertible notes payable               $ 44,700,000
Additional paid-in capital               74,900,000
Reduction in accumulated deficit               $ 30,200,000
FILSPARI                
New Accounting Pronouncements or Change in Accounting Principle [Line Items]                
Total inventory       2,300,000        
Cost of goods sold       1,000,000 3,200,000      
Loss on allowance for inventory       $ 0 2,100,000      
Purchase commitment         $ 1,100,000      
v3.25.0.1
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Inventory, Net of Reserves (Details) - USD ($)
$ in Thousands
Dec. 31, 2024
Dec. 31, 2023
Accounting Policies [Abstract]    
Raw materials $ 30,552 $ 33,790
Work in process 7,625 4,727
Finished goods 3,679 2,387
Total inventory 41,856 40,904
Inventory 6,200 9,410
Long-term inventory $ 35,656 $ 31,494
v3.25.0.1
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Cost of Goods Sold (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
New Accounting Pronouncements or Change in Accounting Principle [Line Items]      
Cost of goods sold $ 7,744 $ 11,450 $ 4,420
Cost of goods sold - product sales      
New Accounting Pronouncements or Change in Accounting Principle [Line Items]      
Cost of goods sold 7,446 8,406 4,420
Cost of goods sold - license and collaboration      
New Accounting Pronouncements or Change in Accounting Principle [Line Items]      
Cost of goods sold $ 298 $ 3,044 $ 0
v3.25.0.1
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Vesting Award Terms (Details)
 12 Months Ended
Dec. 31, 2024
Stock Options  
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]  
Expiration Term 10 years
Stock Options | Minimum  
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]  
Vesting Term 3 years
Stock Options | Maximum  
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]  
Vesting Term 4 years
Restricted Stock Units | Minimum  
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]  
Vesting Term 1 year
Restricted Stock Units | Maximum  
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]  
Vesting Term 4 years
v3.25.0.1
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Major Classifications of Property and Equipment (Details)
Dec. 31, 2024
Computers and equipment  
Property, Plant and Equipment [Line Items]  
Use life (in years) 3 years
Furniture and fixtures  
Property, Plant and Equipment [Line Items]  
Use life (in years) 7 years
v3.25.0.1
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Foreign Currency Translation Adjustment included in Accumulated Other Comprehensive (Loss) Gain (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Foreign Currency Translation Adjustments included in Accumulated Other Comprehensive (Loss) Gain      
Beginning balance $ 200,810 $ 42,851 $ 302,112
Ending balance 59,077 200,810 42,851
Accumulated Foreign Currency Adjustment Attributable to Parent      
Foreign Currency Translation Adjustments included in Accumulated Other Comprehensive (Loss) Gain      
Beginning balance (1,456) 415 (92)
Foreign currency translation adjustments 1,422 (1,871) 507
Ending balance $ (34) $ (1,456) $ 415
v3.25.0.1
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Restructuring and Related Costs (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Restructuring Reserve [Roll Forward]      
Liability balance, beginning of period $ 11,421 $ 0  
Restructuring expenses 2,438 11,394 $ 0
Non-cash impairment and disposal charges (856) 0  
Payments (12,929) 0  
Foreign currency impact (74) 27  
Liability balance, end of period $ 0 $ 11,421 $ 0
v3.25.0.1
REVENUE RECOGNITION - Narrative (Details)
$ in Millions
 12 Months Ended
Dec. 31, 2024
USD ($)
pharmacy
Dec. 31, 2023
USD ($)
Dec. 31, 2022
USD ($)
Disaggregation of Revenue [Line Items]      
Number of direct-to-patient pharmacies sold to | pharmacy 2    
Revenue, performance obligation, description of timing For FILSPARI, sales are recognized upon delivery of the product to the specialty pharmacies. The Company receives payments from its FILSPARI sales based on terms that are generally 30 days from shipment of the product to the specialty pharmacy. For the Company's tiopronin products, product sales are recognized upon delivery to the patient. The Company receives payments from sales of its tiopronin products, primarily through third party payers, based on terms that generally are within 30 days of delivery of product to the patient    
Revenue increase in performance obligations satisfied in previous period | $ $ (0.5) $ 0.4 $ 0.2
United States | Revenue Benchmark | Geographic Concentration Risk      
Disaggregation of Revenue [Line Items]      
Concentration risk, percentage 98.00%    
v3.25.0.1
REVENUE RECOGNITION - Net Product Sales (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Disaggregation of Revenue [Line Items]      
Total net product sales $ 233,175 $ 145,238 $ 109,460
Net product sales      
Disaggregation of Revenue [Line Items]      
Total net product sales 226,707 127,537 97,970
FILSPARI      
Disaggregation of Revenue [Line Items]      
Total net product sales 132,222 29,208 0
Tiopronin products      
Disaggregation of Revenue [Line Items]      
Total net product sales $ 94,485 $ 98,329 $ 97,970
v3.25.0.1
COLLABORATION AND LICENSE AGREEMENTS - Narrative (Details)
$ in Thousands
 1 Months Ended 4 Months Ended 12 Months Ended
Jan. 31, 2024
USD ($)
performance_obligation
Sep. 30, 2021
USD ($)
performance_obligation
Dec. 31, 2021
USD ($)
Dec. 31, 2024
USD ($)
Dec. 31, 2023
USD ($)
Dec. 31, 2022
USD ($)
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Revenue       $ 233,175 $ 145,238 $ 109,460
Deferred revenue     $ 36,647 2,815 8,931 22,907
Increase in amortization of deferred revenue         2,900  
Deferred revenue, current portion       2,815 7,096  
License and collaboration revenue            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Revenue       6,468 17,701 $ 11,490
Vifor Pharma | License and collaboration revenue            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Revenue         17,700  
Vifor Pharma | License and Collaboration - Clinical Development Activities            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Revenue       5,800 14,400  
Vifor Pharma | Royalty            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Revenue       600    
Vifor Pharma | License and Collaboration - Sale of Pharmaceutical Ingredients            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Revenue         3,300  
Vifor Pharma | Collaborative Arrangement            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Upfront payment   $ 55,000        
Milestone payments (up to)   $ 845,000        
Percentage of royalty on net sales receives   40.00%        
Force majeure event period threshold   6 months        
Revenue   $ 55,000        
Number of performance obligations | performance_obligation   2        
Deferred revenue   $ 43,000   $ 2,800 8,900  
Deferred revenue, current portion         7,100  
Deferred revenue, non-current portion         $ 1,800  
Vifor Pharma | Collaborative Arrangement | Regulatory and Market Access Milestone            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Milestone payments (up to)   135,000        
Vifor Pharma | Collaborative Arrangement | Sales-based Milestone Payments            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Milestone payments (up to)   $ 655,000        
Vifor Pharma | Collaborative Arrangement | License and collaboration revenue            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Revenue     $ 12,000      
Renalys Pharma, Inc | Collaborative Arrangement            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Force majeure event period threshold 9 months          
Revenue $ 8,300          
Number of performance obligations | performance_obligation 1          
Renalys Pharma, Inc | Collaborative Arrangement | Regulatory, Development And Sales-based Milestone            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Milestone payments (up to) $ 120,000          
v3.25.0.1
COLLABORATION AND LICENSE AGREEMENTS - Changes in Deferred Revenue (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Contract with Customer, Liability [Roll Forward]      
Beginning balance $ 8,931 $ 22,907 $ 36,647
Foreign currency impact (269) 387 (2,250)
Ending balance 2,815 8,931 22,907
License and collaboration revenue      
Contract with Customer, Liability [Roll Forward]      
License and collaboration revenue $ (5,847) $ (14,363) $ (11,490)
v3.25.0.1
VARIABLE INTEREST ENTITIES (Details) - USD ($)
$ in Thousands
Mar. 08, 2022
Dec. 31, 2024
Dec. 31, 2023
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]      
Other assets   $ 18,162 $ 10,661
Other current liabilities   14,291 $ 5,237
Variable Interest Entity, Not Primary Beneficiary      
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]      
Other assets   0  
Other current liabilities   $ 8,900  
PharmaKrysto, LTD | Collaborative Arrangement      
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]      
Collaboration agreement payment $ 400    
Research and development expenses, estimated 5,000    
Collaborative arrangement, option to purchase additional shares of VIE, amount 1,000    
Collaborative arrangement, option to purchase remaining shares of VIE, amount 5,000    
Milestone payments contingently due $ 16,000    
Royalty payments, percentage (less than) 4.00%    
Agreement termination notice period   60 days  
Percentage of research and development to be funded by the company 100.00%    
PharmaKrysto, LTD      
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]      
Payment to purchase interest in VIE $ 600    
Percentage ownership purchased 5.00%    
PharmaKrysto, LTD | Collaborative Arrangement      
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]      
Additional ownership interest to be purchased upon achievement of certain milestones 5.00%    
v3.25.0.1
MARKETABLE DEBT SECURITIES - Marketable Debt Securities (Details) - USD ($)
$ in Thousands
Dec. 31, 2024
Dec. 31, 2023
Debt Securities, Available-for-sale [Line Items]    
Total available-for-sale marketable debt securities $ 312,166 $ 508,675
Commercial paper    
Debt Securities, Available-for-sale [Line Items]    
Total available-for-sale marketable debt securities 73,325 34,458
Corporate debt securities    
Debt Securities, Available-for-sale [Line Items]    
Total available-for-sale marketable debt securities 203,816 368,323
Securities of government-sponsored entities    
Debt Securities, Available-for-sale [Line Items]    
Total available-for-sale marketable debt securities $ 35,025 $ 105,894
v3.25.0.1
MARKETABLE DEBT SECURITIES - Short-term Marketable Debt Securities (Details) - USD ($)
$ in Thousands
Dec. 31, 2024
Dec. 31, 2023
Debt Securities, Available-for-sale [Line Items]    
Marketable debt securities, available-for-sale, amortized cost, current   $ 249,247
Marketable debt securities, available-for-sale, unrealized gains, current   90
Marketable debt securities, available-for-sale, unrealized losses, current   (699)
Marketable debt securities, available-for-sale, current   248,638
Marketable debt securities, available-for-sale, amortized cost, noncurrent   258,687
Marketable debt securities, available-for-sale, unrealized gains, noncurrent   1,550
Marketable debt securities, available-for-sale, unrealized losses, noncurrent   (200)
Marketable debt securities, available-for-sale, noncurrent   260,037
Marketable debt securities, available-for-sale, amortized cost $ 311,798 507,934
Marketable debt securities, available-for-sale, accumulated gross unrealized gains, before tax 517 1,640
Marketable debt securities, available-for-sale, accumulated gross unrealized losses, before tax (149) (899)
Marketable debt securities, available-for-sale 312,166 508,675
Commercial paper    
Debt Securities, Available-for-sale [Line Items]    
Marketable debt securities, available-for-sale, amortized cost, current 73,410 34,450
Marketable debt securities, available-for-sale, unrealized gains, current 1 25
Marketable debt securities, available-for-sale, unrealized losses, current (86) (17)
Marketable debt securities, available-for-sale, current 73,325 34,458
Marketable debt securities, available-for-sale 73,325 34,458
Corporate debt securities    
Debt Securities, Available-for-sale [Line Items]    
Marketable debt securities, available-for-sale, amortized cost, current 203,395 133,463
Marketable debt securities, available-for-sale, unrealized gains, current 483 29
Marketable debt securities, available-for-sale, unrealized losses, current (62) (408)
Marketable debt securities, available-for-sale, current 203,816 133,084
Marketable debt securities, available-for-sale, amortized cost, noncurrent   233,969
Marketable debt securities, available-for-sale, unrealized gains, noncurrent   1,444
Marketable debt securities, available-for-sale, unrealized losses, noncurrent   (174)
Marketable debt securities, available-for-sale, noncurrent   235,239
Marketable debt securities, available-for-sale 203,816 368,323
Securities of government-sponsored entities    
Debt Securities, Available-for-sale [Line Items]    
Marketable debt securities, available-for-sale, amortized cost, current 34,993 81,334
Marketable debt securities, available-for-sale, unrealized gains, current 33 36
Marketable debt securities, available-for-sale, unrealized losses, current (1) (274)
Marketable debt securities, available-for-sale, current 35,025 81,096
Marketable debt securities, available-for-sale, amortized cost, noncurrent   24,718
Marketable debt securities, available-for-sale, unrealized gains, noncurrent   106
Marketable debt securities, available-for-sale, unrealized losses, noncurrent   (26)
Marketable debt securities, available-for-sale, noncurrent   24,798
Marketable debt securities, available-for-sale $ 35,025 $ 105,894
v3.25.0.1
MARKETABLE DEBT SECURITIES - Narrative (Details) - USD ($)
$ in Millions
Dec. 31, 2024
Dec. 31, 2023
Investments, Debt and Equity Securities [Abstract]    
Accrued interest receivable $ 2.3 $ 4.6
Available-for-sale marketable debt securities in an unrealized loss position $ 123.7 $ 269.7
v3.25.0.1
MARKETABLE DEBT SECURITIES - Debt Securities in Unrealized Loss Position (Details) - USD ($)
$ in Thousands
Dec. 31, 2024
Dec. 31, 2023
Debt Securities, Available-for-sale [Line Items]    
Less than 12 months, fair value $ 108,558 $ 227,533
Less than 12 months, unrealized losses 142 639
12 months or greater, fair value 14,944 41,315
12 months or greater, unrealized losses 7 260
Total, fair value 123,502 268,848
Total, unrealized losses 149 899
Commercial paper    
Debt Securities, Available-for-sale [Line Items]    
Less than 12 months, fair value 68,446 24,798
Less than 12 months, unrealized losses 86 17
12 months or greater, fair value 0 0
12 months or greater, unrealized losses 0 0
Total, fair value 68,446 24,798
Total, unrealized losses 86 17
Corporate debt securities    
Debt Securities, Available-for-sale [Line Items]    
Less than 12 months, fair value 40,112 140,802
Less than 12 months, unrealized losses 56 405
12 months or greater, fair value 9,969 28,775
12 months or greater, unrealized losses 6 177
Total, fair value 50,081 169,577
Total, unrealized losses 62 582
Securities of government-sponsored entities    
Debt Securities, Available-for-sale [Line Items]    
Less than 12 months, fair value 0 61,933
Less than 12 months, unrealized losses 0 217
12 months or greater, fair value 4,975 12,540
12 months or greater, unrealized losses 1 83
Total, fair value 4,975 74,473
Total, unrealized losses $ 1 $ 300
v3.25.0.1
CONVERTIBLE NOTES PAYABLE - Convertible Senior Notes (Details) - USD ($)
$ in Thousands
Dec. 31, 2024
Dec. 31, 2023
Classified as:    
Convertible debt, current portion $ 68,678 $ 0
Convertible debt, less current portion 310,310 377,263
Total convertible debt 378,988 377,263
Senior Notes    
Debt Instrument [Line Items]    
Total convertible senior notes, net of unamortized debt discount and debt issuance costs $ 378,988 377,263
Senior Notes Due 2029 | Senior Notes    
Debt Instrument [Line Items]    
Interest rate percentage 2.25%  
Convertible senior notes $ 316,250 316,250
Unamortized debt issuance costs $ (5,940) (7,348)
Senior Notes Due 2025 | Senior Notes    
Debt Instrument [Line Items]    
Interest rate percentage 2.50%  
Convertible senior notes $ 68,904 68,904
Unamortized debt issuance costs $ (226) $ (543)
v3.25.0.1
CONVERTIBLE NOTES PAYABLE - Narrative (Details)
$ / shares in Units, $ in Thousands
 12 Months Ended
Mar. 11, 2022
USD ($)
Sep. 10, 2018
USD ($)
Dec. 31, 2024
USD ($)
day
$ / shares
Dec. 31, 2023
USD ($)
Dec. 31, 2022
USD ($)
Debt Instrument [Line Items]          
Debt instrument, repurchase amount Including accrued and unpaid interest $ 213,800        
Loss on extinguishment of debt     $ 0 $ 0 $ 7,578
Write off of deferred debt financing costs         3,400
Interest expense     $ 11,182 $ 11,334 $ 11,014
Senior Notes Due 2029 | Senior Notes          
Debt Instrument [Line Items]          
Long-term debt, excluding current maturities 316,300        
Interest rate percentage     2.25%    
Proceeds from issuance of debt 306,400        
Debt issuance costs, net 9,900        
Accrued interest     $ 2,400    
Debt instrument, convertible, threshold percentage of stock price trigger     130.00%    
Debt instrument, convertible, conversion ratio     0.031374    
Debt instrument, convertible, conversion price (in dollars per share) | $ / shares     $ 31.87    
Debt instrument, convertible, prior to maturity date | day     40    
Notes payable     $ 100,000    
Long-term debt, term     7 years    
Debt instrument, interest rate, effective percentage     2.74%    
Senior Notes Due 2029 | Senior Notes | Debt Conversion, Scenario One          
Debt Instrument [Line Items]          
Debt instrument, convertible, threshold trading days | day     20    
Debt instrument, convertible, threshold consecutive trading days | day     30    
Debt instrument, convertible, threshold percentage of stock price trigger     130.00%    
Senior Notes Due 2029 | Senior Notes | Debt Conversion, Scenario Two          
Debt Instrument [Line Items]          
Debt instrument, convertible, threshold trading days | day     5    
Debt instrument, convertible, threshold consecutive trading days | day     10    
Debt instrument, convertible, threshold percentage of stock price trigger     98.00%    
Senior Notes Due 2029, Issued Pursuant To Underwriters Option | Senior Notes          
Debt Instrument [Line Items]          
Long-term debt, excluding current maturities 41,300        
Senior Notes Due 2025 | Senior Notes          
Debt Instrument [Line Items]          
Long-term debt, excluding current maturities   $ 276,000      
Interest rate percentage     2.50%    
Proceeds from issuance of debt   267,200      
Debt issuance costs, net   $ 8,800      
Accrued interest     $ 500    
Debt instrument, convertible, threshold percentage of stock price trigger     130.00%    
Debt instrument, convertible, conversion ratio     0.0257739    
Debt instrument, convertible, conversion price (in dollars per share) | $ / shares     $ 38.80    
Long-term debt, term     7 years    
Debt instrument, interest rate, effective percentage     2.98%    
Debt instrument, repurchase amount $ 207,100        
Long-term debt, excluding current maturities, repaid if converted     $ 68,900    
Senior Notes Due 2025 | Senior Notes | Debt Conversion, Scenario One          
Debt Instrument [Line Items]          
Debt instrument, convertible, threshold trading days | day     20    
Debt instrument, convertible, threshold consecutive trading days | day     30    
Debt instrument, convertible, threshold percentage of stock price trigger     130.00%    
Senior Notes Due 2025 | Senior Notes | Debt Conversion, Scenario Two          
Debt Instrument [Line Items]          
Debt instrument, convertible, threshold trading days | day     5    
Debt instrument, convertible, threshold consecutive trading days | day     10    
Debt instrument, convertible, threshold percentage of stock price trigger     98.00%    
v3.25.0.1
CONVERTIBLE NOTES PAYABLE - Interest Expense Recognized (Details) - Senior Notes Due 2025 and Senior Notes Due 2029 - Senior Notes - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Debt Instrument [Line Items]      
Contractual interest expense $ 8,838 $ 8,838 $ 8,433
Amortization of debt issuance costs 1,725 1,718 1,622
Total interest expense for the 2025 and 2029 Notes $ 10,563 $ 10,556 $ 10,055
v3.25.0.1
FAIR VALUE MEASUREMENTS - Narrative (Details) - USD ($)
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]      
Impairment charges $ 1,200,000 $ 0 $ 0
Measurement Input, Discount Rate [Member]      
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]      
Long-lived asset, measurement input 0.069    
Senior Notes Due 2025 | Senior Notes      
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]      
Interest rate percentage 2.50%    
Fair value of convertible debt $ 68,200,000 58,300,000  
Senior Notes Due 2029 | Senior Notes      
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]      
Interest rate percentage 2.25%    
Fair value of convertible debt $ 302,100,000 $ 212,100,000  
v3.25.0.1
FAIR VALUE MEASUREMENTS - Fair Value on a Recurring Basis (Details) - USD ($)
$ in Thousands
Dec. 31, 2024
Dec. 31, 2023
Assets:    
Cash and Cash Equivalents $ 58,535 $ 58,176
Marketable debt securities, available-for-sale 312,166 508,675
Total 370,701 566,851
Quoted prices in active markets (Level 1)    
Assets:    
Cash and Cash Equivalents 51,060 58,176
Marketable debt securities, available-for-sale 0 0
Total 51,060 58,176
Significant other observable inputs (Level 2)    
Assets:    
Cash and Cash Equivalents 7,475 0
Marketable debt securities, available-for-sale 312,166 508,675
Total 319,641 508,675
Significant unobservable inputs (Level 3)    
Assets:    
Cash and Cash Equivalents 0 0
Marketable debt securities, available-for-sale 0 0
Total $ 0 $ 0
v3.25.0.1
INTANGIBLE ASSETS - Narrative (Details) - USD ($)
 1 Months Ended 12 Months Ended 156 Months Ended
Nov. 30, 2018
Nov. 30, 2017
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Dec. 31, 2024
Dec. 31, 2014
Finite-Lived Intangible Assets [Line Items]              
Payments to date under terms of licensing agreement     $ 36,212,000 $ 41,591,000 $ 28,366,000    
Gross Carrying Amount     224,293,000 183,037,000   $ 224,293,000  
Amortization expense     41,739,000 36,282,000 18,603,000    
Impairment of intangible assets, finite-lived     0 0 0    
Goodwill     800,000 800,000   800,000  
Goodwill, impairment     0 $ 0 $ 0    
Ligand license              
Finite-Lived Intangible Assets [Line Items]              
Payments payable upon achievement of milestones (up to)     114,100,000     114,100,000  
Payments to date under terms of licensing agreement     $ 5,800,000     $ 47,200,000  
Useful Life     11 years 11 years   11 years  
Gross Carrying Amount     $ 53,268,000 $ 27,448,000   $ 53,268,000  
Ligand license | Minimum              
Finite-Lived Intangible Assets [Line Items]              
Annual royalty percentage     15.00%        
Ligand license | Maximum              
Finite-Lived Intangible Assets [Line Items]              
Annual royalty percentage     17.00%        
Royalty Agreements              
Finite-Lived Intangible Assets [Line Items]              
Payments to date under terms of licensing agreement     $ 20,300,000 $ 4,400,000      
Mission license              
Finite-Lived Intangible Assets [Line Items]              
Annual royalty percentage     20.00%        
Useful Life     12 years 12 years   12 years 10 years
Finite lived intangible asset upfront fee     $ 3,000,000.0     $ 3,000,000.0  
Royalty guarantees, commitments, amount     2,000,000.0     2,000,000.0  
Licensing agreement, extension term   5 years          
Gross Carrying Amount   $ 5,900,000 171,025,000 $ 155,589,000   171,025,000  
Payments for the option to acquire business $ 300,000            
Payment of guaranteed minimum royalty $ 100,000            
Minimum royalty, percentage 20.00%            
Amortization expense $ 1,000,000.0            
Guaranteed minimum liability $ 700,000            
Accrued royalties     8,200,000 9,700,000   8,200,000  
Increase in intangible assets     15,400,000        
Mission license | Other Current Liabilities              
Finite-Lived Intangible Assets [Line Items]              
Royalty guarantees, commitments, amount     2,100,000 2,100,000   2,100,000  
Mission license | Other Noncurrent Liabilities              
Finite-Lived Intangible Assets [Line Items]              
Royalty guarantees, commitments, amount     $ 6,100,000 $ 7,600,000   $ 6,100,000  
Mission license | Minimum              
Finite-Lived Intangible Assets [Line Items]              
Discount rate     7.00%        
Mission license | Maximum              
Finite-Lived Intangible Assets [Line Items]              
Discount rate     11.00%        
v3.25.0.1
INTANGIBLE ASSETS - Amortizable Intangible Assets (Details) - USD ($)
$ in Thousands
Dec. 31, 2024
Dec. 31, 2023
Nov. 30, 2017
Dec. 31, 2014
Finite-Lived Intangible Assets [Line Items]        
Gross Carrying Amount $ 224,293 $ 183,037    
Accumulated Amortization (121,072) (79,347)    
Intangible assets finite-lived, net $ 103,221 $ 103,690    
Mission license        
Finite-Lived Intangible Assets [Line Items]        
Useful Life 12 years 12 years   10 years
Gross Carrying Amount $ 171,025 $ 155,589 $ 5,900  
Accumulated Amortization (115,303) (77,311)    
Intangible assets finite-lived, net $ 55,722 $ 78,278    
Ligand license        
Finite-Lived Intangible Assets [Line Items]        
Useful Life 11 years 11 years    
Gross Carrying Amount $ 53,268 $ 27,448    
Accumulated Amortization (5,769) (2,036)    
Intangible assets finite-lived, net $ 47,499 $ 25,412    
v3.25.0.1
INTANGIBLE ASSETS - Amortization Expense (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Finite-Lived Intangible Assets [Line Items]      
Total amortization expense $ 41,739 $ 36,282 $ 18,603
Selling, general and administrative      
Finite-Lived Intangible Assets [Line Items]      
Total amortization expense 41,739 29,021 12,339
Research and development      
Finite-Lived Intangible Assets [Line Items]      
Total amortization expense $ 0 $ 7,261 $ 6,264
v3.25.0.1
INTANGIBLE ASSETS - Amortization Expense for Next Five Years (Details) - USD ($)
$ in Thousands
Dec. 31, 2024
Dec. 31, 2023
Goodwill and Intangible Assets Disclosure [Abstract]    
2025 $ 50,397  
2026 16,722  
2027 5,700  
2028 5,714  
2029 5,700  
Thereafter 18,988  
Intangible assets finite-lived, net $ 103,221 $ 103,690
v3.25.0.1
ACCRUED EXPENSES (Details) - USD ($)
$ in Thousands
Dec. 31, 2024
Dec. 31, 2023
Payables and Accruals [Abstract]    
Compensation related costs $ 35,166 $ 29,908
Research and development 16,090 26,006
Accrued royalties 12,309 6,991
Sales discounts, rebates, and allowances 10,585 13,730
Selling, general and administrative 6,154 7,190
Transition services accrual 285 12,282
Accrued restructuring costs 0 11,421
Miscellaneous accrued expenses 5,439 11,463
Total accrued expenses $ 86,028 $ 118,991
v3.25.0.1
COMMITMENTS AND CONTINGENCIES (Details) - USD ($)
$ in Thousands
 1 Months Ended 12 Months Ended
Mar. 31, 2024
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Nov. 30, 2020
Business Acquisition [Line Items]          
In-process research and development $ 65,200 $ 65,205 $ 0 $ 0  
Orphan Technologies Limited          
Business Acquisition [Line Items]          
Contingent consideration, liability         $ 427,000
v3.25.0.1
STOCKHOLDERS’ EQUITY - Common Stock and Preferred Stock (Details)
 12 Months Ended
Dec. 31, 2024
vote
$ / shares
shares
Dec. 31, 2023
$ / shares
shares
Class of Stock [Line Items]    
Common stock, shares authorized (in shares) 200,000,000 200,000,000
Common stock, par value (in dollars per share) | $ / shares $ 0.0001 $ 0.0001
Number of votes per common share owned | vote 1  
Preferred stock, shares authorized (in shares) 20,000,000 20,000,000
Preferred stock, par value (in dollars per share) | $ / shares $ 0.0001 $ 0.0001
Preferred stock, shares issued (in shares) 0 0
Series A Preferred Stock    
Class of Stock [Line Items]    
Preferred stock, shares authorized (in shares) 1,000  
v3.25.0.1
STOCKHOLDERS’ EQUITY - 2018 Equity Incentive Plan (Details)
shares in Millions
 12 Months Ended
Dec. 31, 2024
shares
2018 Equity Incentive Plan and Prior Plans  
Class of Stock [Line Items]  
Shares remaining available for issuance under the plan (in shares) 18.4
2018 Equity Incentive Plan  
Class of Stock [Line Items]  
Shares remaining available for issuance under the plan (in shares) 5.7
Expiration Term 10 years
Vesting Term 4 years
v3.25.0.1
STOCKHOLDERS’ EQUITY - 2017 Employee Stock Purchase Plan (Details) - 2017 ESPP - USD ($)
 12 Months Ended
Dec. 31, 2024
Jan. 01, 2018
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]    
Shares remaining available for issuance under the plan (in shares) 2,480,000 380,000
Potential increase in shares available for issuance, as a percent of total outstanding common stock 1.00%  
Maximum number of additional shares authorized for issuance (in shares) 300,000  
Purchase price of common stock, percent of fair market value 85.00%  
Stock purchase offering period 6 months  
Employee stock purchase plan, maximum compensation 15.00%  
Employee stock purchase plan annual limit $ 25,000  
Shares reserved for future issuance (in shares) 1,004,889  
v3.25.0.1
STOCKHOLDERS’ EQUITY - Assumptions used in Black-Scholes Options Pricing Model (Details) - Stock Options
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]      
Risk free rate 3.90% 3.90% 1.70%
Expected volatility 59.00% 50.00% 50.00%
Expected life (in years) 6 years 2 months 12 days 6 years 4 months 24 days 6 years 4 months 24 days
Expected dividend yield 0.00% 0.00% 0.00%
v3.25.0.1
STOCKHOLDERS’ EQUITY - Share-based Compensation, Stock Options, Activity (Details) - USD ($)
$ / shares in Units, $ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Additional Disclosures [Abstract]      
Exercised, aggregate intrinsic value $ 2,016    
Shares underlying options (in shares) 6,892,156 7,942,964 6,999,669
Weighted Average, exercise price (in dollars per share) $ 22.11 $ 20.85 $ 20.22
Weighted Average, remaining contractual term (in years) 4 years 5 months 15 days 3 years 11 months 12 days 4 years 7 months 13 days
Aggregate intrinsic value $ 3,249 $ 0 $ 21,806
Closing stock price of stock options outstanding and exercisable (in dollars per share) $ 17.42 $ 8.99 $ 21.03
Stock Options      
Shares Underlying Options      
Outstanding, beginning balance (in shares) 10,211,353    
Granted (in shares) 1,524,300    
Forfeited and expired (in shares) (2,076,808)    
Exercised (in shares) (375,514)    
Outstanding, ending balance (in shares) 9,283,331 10,211,353  
Vested and expected to vest (in shares) 9,283,331    
Weighted Average, Exercise Price      
Outstanding, beginning balance (in dollars per share) $ 21.52    
Granted (in dollars per share) 8.63    
Forfeited and expired (in dollars per share) 20.02    
Exercised (in dollars per share) 11.86    
Outstanding, ending balance (in dollars per share) 20.13 $ 21.52  
Vested and expected to vest (in dollars per share) $ 20.13    
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Additional Disclosures [Abstract]      
Weighted average, remaining contractual life (in years) 5 years 6 months 3 days 4 years 10 months 28 days  
Vested and expected to vest (in years) 5 years 6 months 3 days    
Aggregate intrinsic value, beginning balance $ 0    
Exercised, aggregate intrinsic value 2,000 $ 1,400 $ 5,100
Aggregate intrinsic value, ending balance 16,408 $ 0  
Vested and expected to vest, aggregate intrinsic value $ 16,408    
Weighted average grant date fair value of options (in dollars per share) $ 5.10 $ 11.66 $ 13.45
Compensation expense not yet recognized, stock options $ 16,200    
Weighted average remaining vesting period (in years) 2 years 4 months 24 days    
Stock Options | Chief Financial Officer      
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Additional Disclosures [Abstract]      
Modification expense   $ 2,600  
v3.25.0.1
STOCKHOLDERS’ EQUITY - Restricted Stock Unit Activity (Details) - Restricted Stock Units - USD ($)
$ / shares in Units, $ in Millions
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]      
Unrecognized compensation cost related to RSU's granted $ 35.8    
Weighted average period for unrecognized costs (in years) 2 years 3 months 18 days    
Number of RSUs      
Unvested beginning balance (in shares) 2,874,046    
Granted (in shares) 2,077,025    
Vested (in shares) (1,013,811)    
Forfeited/cancelled (in shares) (419,997)    
Unvested ending balance (in shares) 3,517,263 2,874,046  
Weighted Average Grant Date Fair Value      
Unvested beginning balance (in dollars per share) $ 22.97    
Granted (in dollars per share) 9.07 $ 21.20 $ 26.07
Vested (in dollars per share) 22.52    
Forfeited/cancelled (in dollars per share) 18.66    
Unvested ending balance (in dollars per share) $ 15.41 $ 22.97  
Fair value of restricted stock units vested $ 22.8 $ 18.7 $ 11.5
v3.25.0.1
STOCKHOLDERS’ EQUITY - Performance-based Stock Unit Activity (Details) - Performance Shares - USD ($)
$ / shares in Units, $ in Millions
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]      
Unrecognized compensation cost related to PSU's granted $ 1.2    
Weighted average period for unrecognized costs (in years) 10 months 24 days    
Number of PSUs      
Unvested beginning balance (in shares) 175,458    
Granted (in shares) 107,000    
Vested (in shares) (66,250)    
Forfeited/cancelled (in shares) 0    
Unvested ending balance (in shares) 216,208 175,458  
Weighted Average Grant Date Fair Value      
Unvested beginning balance (in dollars per share) $ 25.61    
Granted (in dollars per share) 8.93 $ 22.40 $ 27.54
Vested (in dollars per share) 22.40    
Forfeited/cancelled (in dollars per share) 0    
Unvested ending balance (in dollars per share) $ 18.34 $ 25.61  
Fair value of performance-based stock units vested $ 1.5 $ 0.3 $ 0.4
v3.25.0.1
STOCKHOLDERS’ EQUITY - Non-cash Stock-based Compensation Expense (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]      
Total stock-based compensation expense $ 36,913 $ 44,246 $ 38,143
Selling, general and administrative expenses      
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]      
Total stock-based compensation expense 22,735 27,111 24,368
Research and development expenses      
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]      
Total stock-based compensation expense $ 14,178 $ 17,135 $ 13,775
v3.25.0.1
NET LOSS PER COMMON SHARE - Narrative (Details) - Underwritten Equity Offering - $ / shares
shares in Thousands
 3 Months Ended
Sep. 30, 2024
Nov. 30, 2024
Feb. 28, 2023
Subsidiary, Sale of Stock [Line Items]      
Sales price per share (in dollars per share)   $ 16.00 $ 21.00
Exercise of pre-funded common stock warrants (in shares) 1,250    
Pre Funded Warrant      
Subsidiary, Sale of Stock [Line Items]      
Number of common stock called by warrants (in shares)     1,250
Sales price per share (in dollars per share)     $ 20.9999
v3.25.0.1
NET LOSS PER COMMON SHARE - Basic and Diluted Net Loss Per Share (Details) - USD ($)
$ / shares in Units, $ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Earnings Per Share [Abstract]      
Continuing operations, basic (in shares) 78,888,861 74,267,418 63,758,515
Continuing operations, diluted (in shares) 78,888,861 74,267,418 63,758,515
Discontinued operations, basic (in shares) 78,888,861 74,267,418 63,758,515
Discontinued Operations, diluted (in shares) 78,888,861 74,267,418 63,758,515
Weighted average common shares outstanding, basic (in shares) 78,888,861 74,267,418 63,758,515
Weighted average common shares outstanding, diluted (in shares) 78,888,861 74,267,418 63,758,515
Net loss from continuing operations $ (320,630) $ (376,333) $ (331,468)
(Loss) income from discontinued operations, net of tax (915) 264,934 52,986
Net loss, basic (321,545) (111,399) (278,482)
Net loss, diluted $ (321,545) $ (111,399) $ (278,482)
Net loss from continuing operations, basic (in dollars per share) $ (4.07) $ (5.07) $ (5.20)
Net loss from continuing operations, diluted (in dollars per share) (4.07) (5.07) (5.20)
Net income from discontinued operations, basic (in dollars per share) (0.01) 3.57 0.83
Net income from discontinued operations, diluted (in dollars per share) (0.01) 3.57 0.83
Net income (loss) per common share, diluted (in dollars per share) (4.08) (1.50) (4.37)
Net income (loss) per common share, basic (in dollars per share) $ (4.08) $ (1.50) $ (4.37)
v3.25.0.1
NET LOSS PER COMMON SHARE - Anti-dilutive Securities (Details) - shares
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]      
Total anti-dilutive shares 25,925,935 25,670,747 23,085,349
Convertible debt      
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]      
Total anti-dilutive shares 11,697,953 11,697,952 10,869,000
Options      
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]      
Total anti-dilutive shares 10,447,541 10,555,550 10,041,249
Restricted stock units and performance-based stock units      
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]      
Total anti-dilutive shares 3,780,441 3,417,245 2,175,100
v3.25.0.1
INCOME TAXES - Net Loss from Continuing Operations Before Income Taxes (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Operating Loss Carryforwards [Line Items]      
Total $ (320,510) $ (376,110) $ (331,155)
United States      
Operating Loss Carryforwards [Line Items]      
Total (152,356) (293,283) (268,887)
Foreign      
Operating Loss Carryforwards [Line Items]      
Total $ (168,154) $ (82,827) $ (62,268)
v3.25.0.1
INCOME TAXES - Components of Provision (Benefit) for Income Taxes (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Current      
Federal $ 0 $ 0 $ 0
State 120 223 314
Foreign 0 0 (1)
Total current 120 223 313
Deferred      
Federal 0 0 0
State 0 0 0
Total deferred 0 0 0
Total tax provision $ 120 $ 223 $ 313
v3.25.0.1
INCOME TAXES - Reconciliation of Statutory Federal Income Tax Rate (Details)
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Income Tax Disclosure [Abstract]      
Statutory rate - federal (21.00%) (21.00%) (21.00%)
State taxes, net of federal benefit (1.55%) (3.19%) (3.62%)
Foreign rate differential 2.15% 1.29% 0.81%
IPR&D 4.27% 0.00% 0.00%
Nondeductible executive compensation 0.44% 1.50% 0.41%
Excess tax benefits associated with share-based awards 2.92% 0.68% (0.17%)
Other permanent differences 0.23% 0.37% 0.17%
Tax credits (4.24%) (1.13%) (5.29%)
Return to provision adjustments and other true-ups (0.80%) 4.19% (0.17%)
Adoption of ASU 2020-06 0.00% 0.00% (3.54%)
Other 0.93% 0.57% 0.00%
Change in valuation allowance 16.69% 16.78% 32.49%
Income tax provision 0.04% 0.06% 0.09%
v3.25.0.1
INCOME TAXES - Deferred Tax Assets and Liabilities (Details) - USD ($)
$ in Thousands
Dec. 31, 2024
Dec. 31, 2023
Deferred Tax Assets:    
Net operating loss $ 113,303 $ 81,531
Research and development and other tax credits 91,760 77,555
Intangible assets 53,919 47,731
Capitalized research and development 47,880 39,907
Stock based compensation 15,680 21,607
Other accrued expenses 14,266 13,187
Charitable contributions 5,797 6,110
Operating lease liabilities 5,421 6,812
Depreciation 435 292
Loan costs 153 62
Interest expense limitation 0 1,679
Total deferred tax assets 348,614 296,473
Deferred Tax Liabilities:    
Operating lease right of use assets (4,259) (5,595)
Prepaid assets (178) (198)
Total deferred tax liabilities (4,437) (5,793)
Net deferred tax assets before valuation allowance 344,177 290,680
Valuation allowance (344,177) (290,680)
Total deferred tax assets $ 0 $ 0
v3.25.0.1
INCOME TAXES - Narrative (Details) - USD ($)
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Dec. 31, 2021
Operating Loss Carryforwards [Line Items]        
Valuation allowance $ 344,177,000 $ 290,680,000    
Increase (decrease) in valuation allowance 53,500,000 (2,700,000)    
Unrecognized tax benefits 27,404,000 22,906,000 $ 11,490,000 $ 7,825,000
Unrecognized tax benefits that would impact effective tax rate 27,400,000      
Income tax examination, penalties and interest expense 0 $ 0 $ 100,000  
Unrecognized Tax Benefits        
Operating Loss Carryforwards [Line Items]        
Increase (decrease) in valuation allowance 27,400,000      
Federal Orphan Drug Tax Credit        
Operating Loss Carryforwards [Line Items]        
Tax credit carryforward 104,100,000      
Tax Competes Tax Credit Carryforward        
Operating Loss Carryforwards [Line Items]        
Tax credit carryforward 1,600,000      
United States        
Operating Loss Carryforwards [Line Items]        
Available unused NOL carryforwards 223,800,000      
United States | Research Tax Credit Carryforward        
Operating Loss Carryforwards [Line Items]        
Tax credit carryforward 4,900,000      
State and Local Jurisdiction        
Operating Loss Carryforwards [Line Items]        
Available unused NOL carryforwards 241,800,000      
Operating loss carryforwards, indefinite carryforward period 45,400,000      
State and Local Jurisdiction | Research Tax Credit Carryforward        
Operating Loss Carryforwards [Line Items]        
Tax credit carryforward, finite carryforward period, amount 1,400,000      
Tax credit carryforward, indefinite carryforward period, amount 9,900,000      
Foreign | Irish        
Operating Loss Carryforwards [Line Items]        
Available unused NOL carryforwards 14,200,000      
Foreign | Swiss        
Operating Loss Carryforwards [Line Items]        
Available unused NOL carryforwards 359,100,000      
Tax credit carryforward $ 526,200,000      
v3.25.0.1
INCOME TAXES - Unrecognized Tax Benefits (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Reconciliation of Unrecognized Tax Benefits, Excluding Amounts Pertaining to Examined Tax Returns [Roll Forward]      
Beginning balance $ 22,906 $ 11,490 $ 7,825
Increase in current period positions 4,248 4,871 2,056
Increase in prior period positions 250 7,383 1,919
Decrease in prior period positions 0 (838) 0
Decrease due to settlements with tax authorities 0 0 (310)
Ending balance $ 27,404 $ 22,906 $ 11,490
v3.25.0.1
EQUITY OFFERINGS (Details) - USD ($)
$ / shares in Units, $ in Thousands
 1 Months Ended 3 Months Ended 12 Months Ended
Nov. 30, 2024
Feb. 28, 2023
Sep. 30, 2024
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Oct. 31, 2024
Class of Stock [Line Items]              
Proceeds from the issuance of common stock, net of issuance costs       $ 134,738 $ 191,198 $ 0  
Underwritten Equity Offering              
Class of Stock [Line Items]              
Shares sold (in shares) 9,000,000 9,700,000          
Sales price per share (in dollars per share) $ 16.00 $ 21.00          
Proceeds from the issuance of common stock, net of issuance costs $ 134,700 $ 215,800          
Exercise of pre-funded common stock warrants (in shares)     1,250,000        
Underwritten Equity Offering | Common Stock              
Class of Stock [Line Items]              
Prefunded warrants, notice period for modifying limitations   61 days          
Pre-funded warrants exercisable for share of common stock (in shares)   1          
Underwritten Equity Offering | Pre Funded Warrant              
Class of Stock [Line Items]              
Sales price per share (in dollars per share)   $ 20.9999          
Number of common stock called by warrants (in shares)   1,250,000          
Exercise price of each pre-funded warrant (in dollars per share)   $ 0.0001          
At-The-Market Offering              
Class of Stock [Line Items]              
Shares sold (in shares)       0      
Aggregate offering amount authorized             $ 100,000
v3.25.0.1
RETIREMENT PLAN (Details) - USD ($)
$ in Millions
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Defined Benefit Plan, Plan Assets, Category [Line Items]      
Defined contribution plan, employer contributions $ 2.4 $ 2.7 $ 2.1
Switzerland Defined Benefit Plan      
Defined Benefit Plan, Plan Assets, Category [Line Items]      
Benefit obligation 3.0    
Plan assets $ 2.5    
v3.25.0.1
PROPERTY AND EQUIPMENT (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Property, Plant and Equipment [Line Items]      
Property, plant and equipment, gross $ 14,091 $ 14,922  
Less: Accumulated depreciation (8,755) (7,443)  
Total property and equipment, net 5,336 7,479  
Depreciation expense 1,800 2,200 $ 2,100
Leasehold improvements      
Property, Plant and Equipment [Line Items]      
Property, plant and equipment, gross 9,370 9,692  
Furniture and fixtures      
Property, Plant and Equipment [Line Items]      
Property, plant and equipment, gross 2,446 2,990  
Computers and equipment      
Property, Plant and Equipment [Line Items]      
Property, plant and equipment, gross 2,275 2,071  
Construction-in-progress      
Property, Plant and Equipment [Line Items]      
Property, plant and equipment, gross $ 0 $ 169  
v3.25.0.1
LEASES - Narrative (Details) - USD ($)
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
May 31, 2020
Lessee, Lease, Description [Line Items]        
Operating lease right-of-use assets $ 14,295,000 $ 18,061,000    
Total lease liability 22,596,000      
Impairment charges 1,200,000 0 $ 0  
Operating lease expense 4,800,000 $ 4,900,000 $ 5,000,000.0  
Esprit Investments Limited        
Lessee, Lease, Description [Line Items]        
Operating lease right-of-use assets 400,000      
Total lease liability $ 400,000      
Operating lease extension term 5 years      
Office Lease 2020        
Lessee, Lease, Description [Line Items]        
Operating lease right-of-use assets       $ 34,600,000
Total lease liability       34,500,000
Tenant improvement allowance       $ 7,900,000
Operating lease extension term 5 years      
v3.25.0.1
LEASES - Future Minimum Rental Commitments (Details) - USD ($)
$ in Thousands
Dec. 31, 2024
Dec. 31, 2023
Leases [Abstract]    
2025 $ 6,665  
2026 6,889  
2027 7,064  
2028 4,781  
Thereafter 0  
Total undiscounted future minimum payments 25,399  
Present value discount (2,803)  
Total lease liability 22,596  
Unamortized lease incentives (3,491)  
Cash payments in excess of straight-line lease expense (4,810)  
Total ROU asset $ 14,295 $ 18,061
v3.25.0.1
LEASES - Weighted-average Remaining Lease Term and Discount Rate (Details)
Dec. 31, 2024
Dec. 31, 2023
Leases [Abstract]    
Weighted-average remaining lease term in years 3 years 8 months 12 days 4 years 8 months 12 days
Weighted-average discount rate 6.48% 6.48%
v3.25.0.1
DIVESTITURES - Additional Information (Details) - USD ($)
$ in Thousands
 12 Months Ended
Aug. 31, 2023
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]        
Transition services accrual   $ 285 $ 12,282  
Disposed of by Sale | Bile Acid Product Portfolio        
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]        
Proceeds from sale of business $ 210,000      
Consideration receivable on sale of business (up to) 235,000      
Gain on disposal of discontinued operations, net of tax   0 226,005 $ 0
Discontinued operation after disposal, expense   500 $ 1,000  
Contingent consideration received on sale of business   1,500    
Transition services accrual   $ 300    
Disposed of by Sale | Bile Acid Product Portfolio | Minimum        
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]        
Milestone thresholds 125,000      
Disposed of by Sale | Bile Acid Product Portfolio | Maximum        
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]        
Milestone thresholds $ 500,000      
v3.25.0.1
DIVESTITURES - Results of Discontinued Operations (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Other income (expenses), net:      
Net (loss) income from discontinued operations $ (915) $ 264,934 $ 52,986
Disposed of by Sale | Bile Acid Product Portfolio      
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]      
Total revenue (550) 66,164 102,558
Operating expenses:      
Cost of goods sold (9)    
Cost of goods sold   1,899 3,172
Research and development 247 6,118 8,447
Selling, general and administrative 127 19,500 22,686
Change in fair value of contingent consideration 0 (473) 15,006
Total operating expenses 365 27,044 49,311
Operating income (915) 39,120 53,247
Other income (expenses), net:      
Interest expense 0 (191) (261)
Gain on disposal of discontinued operations, net of tax 0 226,005 0
Total other income (expense), net 0 225,814 (261)
(Loss) income from discontinued operations before income tax (915) 264,934 52,986
Income tax on discontinued operations 0 0 0
Net (loss) income from discontinued operations (915) 264,934 52,986
Net product sales | Disposed of by Sale | Bile Acid Product Portfolio      
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]      
Total revenue $ (550) $ 66,164 $ 102,558
v3.25.0.1
SEGMENT INFORMATION - Narrative (Details)
 12 Months Ended
Dec. 31, 2024
USD ($)
segment
Dec. 31, 2023
USD ($)
Revenues from External Customers and Long-Lived Assets [Line Items]    
Number of segments | segment 1  
Non U.S    
Revenues from External Customers and Long-Lived Assets [Line Items]    
Long-lived assets | $ $ 4,500,000 $ 0
v3.25.0.1
SEGMENT INFORMATION - Reportable Segment Loss, Including Significant Expenses (Details) - USD ($)
$ in Thousands
 1 Months Ended 12 Months Ended
Mar. 31, 2024
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Segment Reporting Information [Line Items]        
Revenue   $ 233,175 $ 145,238 $ 109,460
Less:        
Cost of goods sold   7,744 11,450 4,420
Research and development:        
Total research and development   217,496 244,990 227,333
Selling, general and administrative   264,119 265,542 197,520
In-process research and development $ 65,200 65,205 0 0
Restructuring   2,438 11,394 0
Total other income (expense), net   3,317 12,028 (11,342)
Income tax provision on continuing operations   (120) (223) (313)
(Loss) income from discontinued operations, net of tax   (915) 264,934 52,986
Net loss   (321,545) (111,399) (278,482)
Reportable Segment        
Segment Reporting Information [Line Items]        
Revenue   233,175 145,238 109,460
Less:        
Cost of goods sold   7,744 11,450 4,420
Research and development:        
External research and development   126,303 142,482 129,698
Internal personnel costs   73,843 84,658 75,664
Other Research and development   17,350 17,850 21,971
Total research and development   217,496 244,990 227,333
Selling, general and administrative   264,119 265,542 197,520
In-process research and development   65,205 0 0
Restructuring   2,438 11,394 0
Total other income (expense), net   3,317 12,028 (11,342)
Income tax provision on continuing operations   (120) (223) (313)
(Loss) income from discontinued operations, net of tax   (915) 264,934 52,986
Net loss   $ (321,545) $ (111,399) $ (278,482)