TRAVERE THERAPEUTICS, INC., 10-K filed on 2/19/2026
Annual Report
v3.25.4
Cover - USD ($)
 12 Months Ended
Dec. 31, 2025
Feb. 13, 2026
Jun. 30, 2025
Cover [Abstract]      
Document Type 10-K    
Document Annual Report true    
Document Period End Date Dec. 31, 2025    
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     $ 1,309,524,306
Entity Common Stock, Shares Outstanding   92,241,550  
Documents Incorporated by Reference DOCUMENTS INCORPORATED BY REFERENCE: Portions of the Proxy Statement for the registrant’s 2026 Annual Meeting of Stockholders, to be filed within 120 days after the conclusion of the registrant's fiscal year ended December 31, 2025, 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 2025    
Document Fiscal Period Focus FY    
v3.25.4
Audit Information
 12 Months Ended
Dec. 31, 2025
Audit Information [Abstract]  
Auditor Firm ID 42
Auditor Name Ernst & Young LLP
Auditor Location San Diego, California
v3.25.4
CONSOLIDATED BALANCE SHEETS - USD ($)
$ in Thousands
Dec. 31, 2025
Dec. 31, 2024
Current assets:    
Cash and cash equivalents $ 93,035 $ 58,535
Marketable debt securities, at fair value 229,761 312,166
Accounts receivable, net 80,134 27,116
Inventory 5,875 6,200
Prepaid expenses and other current assets 28,760 12,685
Total current assets 437,565 416,702
Long-term inventory 30,280 35,656
Property and equipment, net 4,022 5,336
Operating lease right-of-use assets 10,576 14,295
Intangible assets, net 113,868 103,974
Other assets 8,880 18,162
Total assets 605,191 594,125
Current liabilities:    
Accounts payable 24,800 23,534
Accrued expenses 126,035 86,028
Convertible debt, current portion 0 68,678
Operating lease liabilities, current portion 5,875 5,405
Other current liabilities 3,194 17,106
Total current liabilities 159,904 200,751
Convertible debt, less current portion 311,724 310,310
Operating lease liabilities, less current portion 11,134 17,191
Other non-current liabilities 7,601 6,796
Total liabilities 490,363 535,048
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, 2025 and 2024 0 0
Common stock $0.0001 par value; 200,000,000 shares authorized; 90,922,868 and 87,452,835 issued and outstanding as of December 31, 2025 and 2024, respectively 9 9
Additional paid-in capital 1,588,721 1,506,315
Accumulated deficit (1,472,713) (1,447,167)
Accumulated other comprehensive loss (1,189) (80)
Total stockholders' equity 114,828 59,077
Total liabilities and stockholders' equity $ 605,191 $ 594,125
v3.25.4
CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares
Dec. 31, 2025
Dec. 31, 2024
Statement of Financial Position [Abstract]    
Preferred stock, par value (in dollars per share) $ 0.0001 $ 0.0001
Preferred stock, authorized (in shares) 20,000,000 20,000,000
Preferred stock, issued (in shares) 0 0
Preferred stock, outstanding (in shares) 0 0
Common stock, par value (in dollars per share) $ 0.0001 $ 0.0001
Common stock, authorized (in shares) 200,000,000 200,000,000
Common stock, issued (in shares) 90,922,868 87,452,835
Common stock, outstanding (in shares) 90,922,868 87,452,835
v3.25.4
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Total revenue $ 490,728 $ 233,175 $ 145,238
Operating expenses:      
Cost of goods sold 10,339 7,744 11,450
Research and development 206,011 217,496 244,990
Selling, general and administrative 337,202 264,119 265,542
In-process research and development 0 65,205 0
Restructuring 0 2,438 11,394
Total operating expenses 553,552 557,002 533,376
Operating loss (62,824) (323,827) (388,138)
Other income, net:      
Interest income 12,721 17,817 21,768
Interest expense (10,748) (11,182) (11,334)
Other income (expense), net 11,578 (3,318) 1,594
Total other income, net 13,551 3,317 12,028
Loss from continuing operations before income tax provision (49,273) (320,510) (376,110)
Income tax provision on continuing operations (988) (120) (223)
Loss from continuing operations, net of tax (50,261) (320,630) (376,333)
Income (loss) from discontinued operations, net of tax 24,715 (915) 264,934
Net loss $ (25,546) $ (321,545) $ (111,399)
Basic and diluted:      
Net loss from continuing operations, basic (in dollars per share) $ (0.57) $ (4.07) $ (5.07)
Net loss from continuing operations, diluted (in dollars per share) (0.57) (4.07) (5.07)
Net income (loss) from discontinued operations, basic (in dollars per share) 0.28 (0.01) 3.57
Net income (loss) from discontinued operations, diluted (in dollars per share) 0.28 (0.01) 3.57
Net loss per common share, basic (in dollars per share) (0.29) (4.08) (1.50)
Net loss per common share, diluted (in dollars per share) $ (0.29) $ (4.08) $ (1.50)
Weighted average common shares outstanding, basic (in shares) 89,211,813 78,888,861 74,267,418
Weighted average common shares outstanding, diluted (in shares) 89,211,813 78,888,861 74,267,418
Comprehensive loss:      
Net loss $ (25,546) $ (321,545) $ (111,399)
Unrealized gain (loss) on defined benefit pension plan 219 328 (374)
Foreign currency translation (loss) gain (945) 1,422 (1,871)
Unrealized (loss) gain on marketable debt securities (383) (374) 3,696
Comprehensive loss (26,655) (320,169) (109,948)
Net product sales      
Total revenue 410,460 226,707 127,537
Operating expenses:      
Cost of goods sold 5,813 7,446 8,406
License and collaboration revenue      
Total revenue 80,268 6,468 17,701
Operating expenses:      
Cost of goods sold $ 4,526 $ 298 $ 3,044
v3.25.4
CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY - USD ($)
$ in Thousands
Total
Underwritten Equity Offering
Common Stock
Common Stock
Underwritten Equity Offering
Additional Paid-in Capital
Additional Paid-in Capital
Underwritten Equity Offering
Accumulated Other Comprehensive Loss
Accumulated Deficit
Beginning balance (in shares) at Dec. 31, 2022     64,290,570          
Beginning 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 purchase and expense (in shares)     326,521          
Employee stock purchase program purchase and expense 4,853       4,853      
Equity offering, net of issuance costs (in shares)       9,703,750        
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 purchase and expense (in shares)     395,768          
Employee stock purchase program purchase and expense 3,566       3,566      
Equity offering, net of issuance costs (in shares)       8,984,375        
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)
Increase (Decrease) in Stockholders' Equity [Roll Forward]                
Share based compensation 43,480       43,480      
Issuance of common stock under the equity incentive plan and proceeds from exercise (in shares)     3,254,517          
Issuance of common stock under the equity incentive plan and proceeds from exercise 34,745       34,745      
Employee stock purchase program purchase and expense (in shares)     215,516          
Employee stock purchase program purchase and expense 4,181       4,181      
Unrealized (gain) loss on defined benefit pension plan 219           219  
Foreign currency translation adjustments (945)           (945)  
Unrealized (loss) gain on marketable debt securities (383)           (383)  
Net loss (25,546)             (25,546)
Ending balance (in shares) at Dec. 31, 2025     90,922,868          
Ending balance at Dec. 31, 2025 $ 114,828   $ 9   $ 1,588,721   $ (1,189) $ (1,472,713)
v3.25.4
CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY (Parenthetical) - USD ($)
$ in Millions
 12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Pre-funded common stock warrants, issuance costs   $ 1.6
Underwritten Equity Offering    
Issuance costs $ 9.0 $ 12.6
v3.25.4
CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Cash Flows From Operating Activities:      
Net loss $ (25,546) $ (321,545) $ (111,399)
Net income (loss) from discontinued operations 24,715 (915) 264,934
Net loss from continuing operations (50,261) (320,630) (376,333)
Adjustments to reconcile net loss to net cash provided by (used in) operating activities:      
Depreciation and amortization 60,743 43,555 38,530
Share based compensation 44,868 36,913 44,246
In-process research and development 0 65,205 0
Loss on allowance for inventory 684 2,819 3,039
Amortization of debt discount and issuance costs 1,640 1,725 1,718
Amortization of discounts on investments (4,436) (4,535) (7,456)
Other (1,457) 7,944 (3,626)
Changes in operating assets and liabilities:      
Accounts receivable (27,822) (6,001) (11,264)
Inventory 5,016 (3,771) (39,420)
Income taxes 849 (41) (35)
Prepaid expenses and other current and non-current assets (7,658) (3,164) (4,657)
Change in lease assets and liabilities, net (622) (949) (986)
Accounts payable (85) (17,563) 24,763
Accrued expenses 28,840 (31,061) 22,321
Deferred revenue, current and non-current (2,815) (6,116) (15,779)
Other current and non-current liabilities (9,700) 5,646 (418)
Net cash provided by (used in) operating activities - continuing operations 37,784 (230,024) (325,357)
Net cash (used in) provided by operating activities - discontinued operations 0 (7,451) 45,336
Net cash provided by (used) in operating activities 37,784 (237,475) (280,021)
Cash Flows From Investing Activities:      
Proceeds from the sale and maturity of marketable debt securities 323,595 326,334 334,575
Purchase of marketable debt securities (237,142) (125,757) (443,942)
Purchase of intangible assets (58,157) (36,212) (41,591)
Payment of milestone 0 (65,000) 0
Other (404) (40) (668)
Net cash provided by (used in) investing activities - continuing operations 27,892 99,325 (151,626)
Net cash provided by investing activities - discontinued operations 0 0 207,402
Net cash provided by investing activities 27,892 99,325 55,776
Cash Flows From Financing Activities:      
Payment of guaranteed minimum royalty (2,100) (2,100) (2,100)
Repayment of 2025 convertible senior notes (68,904) 0 0
Proceeds from the issuance of common stock, net of issuance costs 0 134,738 191,198
Proceeds from the issuance of pre-funded warrants, net of issuance costs 0 0 24,630
Proceeds from exercise of stock options 34,745 4,452 3,240
Proceeds from the issuances under the employee stock purchase plan 2,792 2,332 3,166
Net cash (used in) provided by financing activities - continuing operations (33,467) 139,422 220,134
Net cash used in financing activities - discontinued operations 0 0 (1,382)
Net cash (used in) provided by financing activities (33,467) 139,422 218,752
Effect of exchange rate changes on cash 2,291 (913) 1,981
Net increase (decrease) in cash and cash equivalents 34,500 359 (3,512)
Cash and cash equivalents, beginning of year 58,535 58,176 61,688
Cash and cash equivalents, end of year 93,035 58,535 58,176
Supplemental Disclosure of Cash Flow Information:      
Operating cash flows used for operating leases 6,578 6,392 6,315
Cash paid for interest 8,838 8,838 8,838
Non-cash investing and financing activities:      
Accrued royalty in excess of minimum payable to the sellers of Thiola $ 14,280 $ 15,436 $ 16,206
v3.25.4
DESCRIPTION OF BUSINESS
 12 Months Ended
Dec. 31, 2025
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"). In August 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. Mirum achieved the first such milestone based on its annual net sales in 2025, and Travere recognized a milestone payment of $25.0 million for the year ended December 31, 2025 in discontinued operations, as a result of such achievement. 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)
In September 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 had previously been granted accelerated approval in February 2023 based on the surrogate marker of proteinuria. Full approval was 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.
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).
In September 2021, the Company entered into a license and collaboration agreement (the "CSL Vifor License Agreement") with Vifor (International) Ltd. ("CSL Vifor"). In April 2024, the Company and CSL Vifor announced that the European Commission had 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 ("UPCR") ≥0.75 g/g), and in April 2025, the Company and CSL Vifor announced that the European Commission had converted the CMA into a standard marketing authorization (“MA”) for FILSPARI for the treatment of adults with primary IgAN with a urine protein excretion ≥1.0 g/day (or UPCR ≥0.75 g/g). The MA was granted for all member states of the European Union, as well as in Iceland, Liechtenstein and Norway. As a result of the standard MA approval, the Company received a regulatory milestone payment of $17.5 million in May 2025 under the terms of the CSL Vifor License Agreement. 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 had granted temporary marketing authorization for FILSPARI for the treatment of adults with primary IgAN with a urine protein excretion ≥1.0 g/day (or UPCR ≥0.75 g/g). In April 2025, the Medicines and Healthcare products Regulatory Agency ("MHRA") in the UK converted its conditional approval of FILSPARI in IgAN to standard approval. During the third quarter of 2025, the Company recognized a $40.0 million market access milestone from CSL Vifor; payment was received in the fourth quarter of 2025.
In January 2024, the Company entered into an exclusive licensing agreement (the "Chugai License Agreement") with Renalys Pharma, Inc. ("Renalys"), to bring sparsentan for the treatment of IgAN to patients in Japan and other countries in Asia. 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 in November 2024. In the fourth quarter of 2025, Renalys announced positive topline results from its Phase 3 study of sparsentan in Japanese patients with IgAN. Renalys has also announced that it has reached an agreement with the PMDA regarding development plans for two other Phase 3 clinical trials of sparsentan, one investigating the use of sparsentan in focal segmental glomerulosclerosis (FSGS) and the other in Alport syndrome, in Japan. In the fourth quarter of 2025, Renalys was acquired by and merged into Chugai Pharmaceutical Co., Ltd. (“Chugai”). Through the acquisition, Chugai gained exclusive rights to develop and commercialize sparsentan in Japan, South Korea, and Taiwan. As a minority shareholder in Renalys, Travere received $10.2 million at the closing of the transaction and Travere is also eligible to receive multiple milestones according to the progress of sparsentan regulatory approval, and consideration linked to sparsentan's net sales in the applicable territory. Under the terms of the licensing agreement, Chugai is responsible for development, regulatory matters, and commercialization in the licensed territories. Chugai plans to file for the regulatory approval for sparsentan in Japan in 2026.
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 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 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 in March 2025, the Company announced that it had submitted an sNDA to the FDA seeking traditional approval of FILSPARI for the treatment of FSGS. In May 2025, the Company announced that the FDA accepted the sNDA, assigned a Prescription Drug User Fee Act ("PDUFA") target action date of January 13, 2026, and initially indicated that it planned to hold an advisory committee meeting to discuss the application. In September 2025, following further review of the sNDA, the FDA informed the Company that an advisory committee meeting was no longer needed. In January 2026, the FDA extended the review timeline of the sNDA, and that the new PDUFA target action date is April 13, 2026. The extension followed the recent submission of responses requested by the FDA to further characterize the clinical benefit of FILSPARI. The FDA determined that the additional responses constituted a Major Amendment to the sNDA and extended the action date accordingly. The sNDA remains under review by the FDA with a PDUFA target action date of April 13, 2026.
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 was enacted following the Company's determination that the desired drug substance profile was not achieved in the initial scale-up process, and it enabled the Company to address necessary process improvements in manufacturing scale-up to support initial commercial scale manufacturing as well as full enrollment in the HARMONY Study. Currently enrolled patients will be able to continue on study medication as scheduled for the duration of the trials in which they are participating. Following further optimization of the manufacturing process in 2025, the Company restarted enrollment activities for the pivotal Phase 3 HARMONY Study in the first quarter of 2026.
The Company acquired pegtibatinase as part of the November 2020 acquisition of Orphan Technologies Limited.
v3.25.4
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES
 12 Months Ended
Dec. 31, 2025
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.
Inventory, net of reserves, consisted of the following at December 31, 2025 and 2024 (in thousands):
December 31, 2025December 31, 2024
Raw materials$24,169 $30,552 
Work in process2,348 7,625 
Finished goods9,638 3,679 
Total inventory$36,155 $41,856 
Classified as:
Inventory$5,875 $6,200 
Long-term inventory30,280 35,656 
Total inventory$36,155 $41,856 
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, 2025. The Company maintains levels of these inventories beyond a one-year production plan to limit exposure to potential supply disruption.
Cost of goods sold includes the cost of inventory sold, third party manufacturing and supply chain costs, product shipping 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 currently consists of the sale of active pharmaceutical ingredients to the Company's collaboration partners, at cost or at cost plus a margin.
The following table summarizes cost of goods sold for the years ended December 31, 2025, 2024 and 2023 (in thousands):
Year Ended December 31,
202520242023
Cost of goods sold - product sales$5,813 $7,446 $8,406 
Cost of goods sold - license and collaboration4,526 298 3,044 
Total cost of goods sold$10,339 $7,744 $11,450 
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, 2025, sales of FILSPARI primarily consisted of zero-cost inventories. As of December 31, 2025 the zero-cost inventory remaining was immaterial. The Company began capitalizing inventory costs associated with FILSPARI following the February 2023 accelerated approval.
For the year ended 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.
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 and year to year.
Advertising Expenses
Advertising costs are expensed as incurred. The Company incurred $14.7 million, $7.8 million and zero in advertising costs during the years ended December 31, 2025, 2024 and 2023, respectively.
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 using the treasury stock method. The treasury stock method reduces the number of shares issuable by the shares 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/(loss) per share calculation. As the Company has reported net losses for all periods presented, all potentially dilutive securities are antidilutive and, accordingly, basic net loss per share equals diluted net loss per share. 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 investments 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, 2025 and 2024, respectively. For the years ended December 31, 2025, 2024 and 2023, provision for credit losses 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. As of December 31, 2025 the Company's accounts receivable balance includes a $25.0 million milestone payment from Mirum. See Note 19 for further discussion.
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 Thiola and 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 and Comprehensive Loss 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 and Comprehensive Loss.
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, 2025 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 Sheets 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, 2025, the Company did not record any basis adjustments. For the year ended December 31, 2024, the Company recognized $3.4 million in other income (expense), net for these basis adjustments and reduced the equity method investment's carrying value 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 $0.3 million, $0.7 million and a gain of $0.5 million arising from foreign exchange transactions is included in other income (expense), net for the years ended December 31, 2025, 2024 and 2023, respectively.
The results and financial position of the Company that have a functional currency different from the U.S. dollar are translated as follows:
a.assets and liabilities presented are translated at the closing exchange rate as of December 31, 2025 and 2024;
b.income and expenses for the statements of operations and comprehensive loss are translated at average exchange rates that are relevant for the respective periods for which the income and expenses occurred; and
c.significant transactions use the exchange rate on the date of the transaction.
All resulting exchange differences arising from such translations are recognized directly in comprehensive income and presented as a separate component of equity.
The following table summarizes the foreign currency translation adjustment included in accumulated other comprehensive loss for the year ended December 31, 2025, 2024 and 2023 (in thousands):
Foreign Currency Translation Adjustments included in Accumulated Other Comprehensive Loss
202520242023
Balance at January 1,$(34)$(1,456)$415 
Foreign currency translation adjustments(945)1,422 (1,871)
Balance at December 31,$(979)$(34)$(1,456)
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 and Comprehensive Loss.
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 and Comprehensive Loss 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. The Company recognized $2.4 million during the year ended December 31, 2024, including $1.2 million related to the impairment of operating lease right-of-use assets and related leasehold improvements, as well as disposal costs associated with the sublease of office space. As of December 31, 2024, the Company had recognized total costs of $13.8 million in connection with the restructuring and no such expenses were incurred for the year ended December 31, 2025.
The following table summarizes the cash payments and accruals, included in accrued expenses of the Consolidated Balance Sheets, related to the restructuring for the year ended December 31, 2024 (in thousands):
2024
Liability balance at January 1,$11,421 
Restructuring expenses2,438 
Non-cash impairment and disposal charges(856)
Payments(12,929)
Foreign currency impact(74)
Liability balance at December 31,$— 
Recently Adopted Accounting Pronouncements
In December 2023, the FASB issued Accounting Standards Update ("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. The Company adopted the standard and applied the disclosure requirements on a prospective basis as required for the year ended December 31, 2025.
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 September 2025, the FASB issued ASU No. 2025-06—Intangibles—Goodwill and Other—Internal-Use Software (Subtopic 350-40): Targeted Improvements to the Accounting for Internal-Use Software. This ASU removed the language around project stages that was used to assess when costs could be capitalized for an internal-use software. The update also requires internal-use software to be disclosed under the ASC 360 Property, Plant, and Equipment guidance. The guidance is effective for annual periods beginning after December 15, 2027. The Company is currently assessing the impact of this standard on the Company’s accounting policies and the financial statements.
In July 2025, the FASB issued ASU No. 2025-05—Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses for Accounts Receivable and Contract Assets. This ASU added a practical expedient that assumes that current conditions as of the balance sheet date do not change for the remaining life of the asset when estimating expected credit losses for current accounts receivable and current contract assets. The guidance is effective for annual periods beginning after December 15, 2025. The Company is currently evaluating the impact of the adoption of this standard on the accounting for credit losses.
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 ASU 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, as clarified by ASU No. 2025-01, for annual reporting periods beginning after December 15, 2026 and interim reporting periods within annual 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.
v3.25.4
REVENUE RECOGNITION
 12 Months Ended
Dec. 31, 2025
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 the Company's 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, 2025, 2024 and 2023, adjustments to net product revenue related to performance obligations satisfied in previous periods were $1.1 million, $0.5 million, and $0.4 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, 2025, 2024 and 2023 (in thousands):
Year Ended December 31,
202520242023
FILSPARI$322,005 $132,222 $29,208 
Tiopronin products88,455 94,485 98,329 
Total net product sales$410,460 $226,707 $127,537 
v3.25.4
COLLABORATION AND LICENSE AGREEMENTS
 12 Months Ended
Dec. 31, 2025
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 the CSL Vifor License Agreement, pursuant to which the Company granted an exclusive license to CSL Vifor for the commercialization of FILSPARI in Europe, Australia and New Zealand. In June 2025, the CSL Vifor License Agreement was amended in order to expand the license to cover additional countries ("CSL Vifor Licensed Territories") and to provide that the license rights to each additional country will revert to the Company if CSL Vifor does not take certain specified actions within specified timelines with respect to such country. CSL Vifor also has first right of negotiation to expand the licensed territories into Canada and/or Mexico.
Under the terms of the CSL Vifor 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 FILSPARI in the CSL Vifor Licensed Territories.
Under the License Agreement, CSL Vifor is responsible for all commercialization activities in the CSL Vifor Licensed Territories. The Company remains responsible for the worldwide clinical development of sparsentan through regulatory approval as defined. 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 CSL Vifor License Agreement will remain in effect, unless terminated earlier, until the expiration of all royalty terms for FILSPARI in the CSL Vifor Licensed Territories. Each party has the right to terminate the CSL Vifor License Agreement for the other party’s uncured material breach, insolvency or if the time required for performance under the CSL Vifor 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 CSL Vifor 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 initial transaction price under the CSL Vifor License Agreement totaled $55.0 million, consisting of the fixed non-refundable upfront payment. The variable regulatory and access related milestones were considered variable consideration and excluded from the transaction price given the substantial uncertainty at inception related to their achievement, based on the most likely amount method. 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 re-evaluates the transaction price for each reporting period and as uncertainties are resolved.
The Company concluded that CSL Vifor represented a customer and applied relevant guidance from ASC 606 to evaluate the accounting under the CSL Vifor License Agreement. In accordance with this guidance, the Company concluded at inception 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 was recognized over the development period based upon the ratio of costs incurred to date to the total estimated costs through June 30, 2025.
For the year ended December 31, 2025, the Company achieved a market access milestone in the UK and European Union of $40.0 million and a regulatory milestone as a result of the European Commission granting standard MA approval of $17.5 million that were added to the transaction price under the most likely amount method and recognized in full.
For the years ended December 31, 2025, 2024 and 2023, royalties on net sales, recorded when the related sales occur, were $5.9 million, $0.6 million and zero, respectively.
For the years ended December 31, 2025, 2024 and 2023, the Company recognized $71.0 million, $6.5 million and $17.7 million, respectively, in license and collaboration revenue associated with the CSL Vifor License Agreement.
Deferred revenue related to the clinical development activities as of December 31, 2025 and 2024 was zero and $2.8 million, respectively, classified as current in the Consolidated Balance Sheets.
The following table sets forth a summary of changes in deferred revenue for the years ended December 31, 2025, 2024 and 2023 (in thousands):
Deferred Revenue
202520242023
Balance at January 1,$2,815 $8,931 $22,907 
License and collaboration revenue(2,872)(5,847)(14,363)
Foreign currency impact57 (269)387 
Balance at December 31,$— $2,815 $8,931 
Licensing Agreement with Chugai
In January 2024, the Chugai License Agreement came into effect, pursuant to which, the Company granted an exclusive license to Renalys (which has since been merged with and into Chugai) for the development and commercialization of sparsentan in Japan and other specified Asian countries ("Chugai Licensed Territories"). In October 2025, the Chugai License Agreement was amended to remove certain specified countries from the Chugai Licensed Territories. Under the terms of the Chugai License Agreement, the Company received a non-refundable upfront payment and will be eligible to receive up to $120.0 million in aggregate regulatory, 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 Chugai 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 had 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 Chugai License Agreement, Chugai is responsible for all development and commercialization activities in the Chugai Licensed Territories. The Company will retain all rights to sparsentan in the United States and rest of world outside of the CSL Vifor Licensed Territories and Chugai Licensed Territories, provided that CSL Vifor has a right of negotiation to expand the licensed territories into Canada and/or Mexico. The Chugai License Agreement will remain in effect, unless terminated earlier, until the expiration of all royalty terms for sparsentan in the Chugai Licensed Territories. Each party has the right to terminate the Chugai License Agreement for the other party’s uncured material breach or insolvency, or if the time required for performance under the Chugai License Agreement by the other party is extended due to a force majeure event that continues for nine months or more. Chugai may terminate the Chugai License Agreement for any reason upon prior written notice to the Company. The Company may terminate the Chugai License Agreement if Chugai abandons development in Japan or South Korea prior to first commercial sales of sparsentan in either Japan or South Korea.
The Company assessed the Chugai 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 a joint steering committee overseeing the development and commercial 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 Chugai 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 under the most likely amount method. 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.
The Company concluded that Chugai represents a customer and applied relevant guidance from ASC 606 to evaluate the accounting under the Chugai License Agreement. In accordance with this guidance, 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 precluded 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 Chugai License Agreement as a financing arrangement. The transaction price was recorded in other current liabilities as a result of the Buyout Right as of December 31, 2024. During the year ended December 31, 2025, the Buyout Right was relinquished and the Company recognized the Renalys deferred revenue amount of $9.3 million in license and collaboration revenue.
See Note 5 for further discussion of VIEs.
v3.25.4
VARIABLE INTEREST ENTITIES
 12 Months Ended
Dec. 31, 2025
Business Combination, Asset Acquisition, Transaction between Entities under Common Control, and Joint Venture Formation [Abstract]  
VARIABLE INTEREST ENTITIES VARIABLE INTEREST ENTITIES
Stock Purchase and Collaboration Agreement with PharmaKrysto
In March 2022, the Company entered into a Collaboration Agreement with PharmaKrysto Limited (“PharmaKrysto”), a privately held pre-clinical stage company and concurrently entered into a Stock Purchase Agreement (together, the "Agreements") whereby the Company acquired 5% of the outstanding common shares of PharmaKrysto. The Agreements granted the Company an option to purchase the remaining outstanding shares of PharmaKrysto for $5.0 million upon the occurrence of a subsequent pre-clinical milestone, which expired on March 8, 2025. As the option was not exercised by the Company prior to expiration, the rights granted to the Company under the Agreements ceased, and the previously purchased common shares were transferred back to PharmaKrysto for immaterial consideration. The Company deconsolidated PharmaKrysto as of March 8, 2025, resulting in an immaterial amount recognized in the Consolidated Statements of Operations and Comprehensive Loss for the year ended December 31, 2025. The consolidated assets and liabilities as of December 31, 2024 were immaterial. The results of operations were not significant for the years ended December 31, 2025, 2024 and 2023.
Licensing Agreement with Chugai
In January 2024, the Chugai License Agreement between the Company and Renalys (which has since been acquired by and merged into Chugai) came into effect and the Company exercised its option to purchase shares of common stock of Renalys. The Company determined that Renalys was a VIE as they could require additional funding to support development and commercial activities. The Company had variable interests in Renalys, including an equity interest, Buyout Right and performance-related payments under the Chugai 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 was not the primary beneficiary as it did not have the power to direct the activities that most significantly impact the economic performance of Renalys and did not have an obligation to absorb losses.
The carrying amount of the liabilities related to the Company’s variable interests was zero and $8.9 million as of December 31, 2025 and 2024, respectively, included in other current liabilities in the Company’s Consolidated Balance Sheets. During the year ended December 31, 2025, the Buyout Right was relinquished and the Company recognized the deferred revenue balance of $9.3 million in license and collaboration revenue. In the fourth quarter of 2025, Renalys was acquired by and merged into Chugai. The Company sold its equity interest in Renalys to Chugai as part of the acquisition and recognized a gain of $10.2 million in other income (expense), net.
v3.25.4
MARKETABLE DEBT SECURITIES
 12 Months Ended
Dec. 31, 2025
Investments, Debt and Equity Securities [Abstract]  
MARKETABLE DEBT SECURITIES MARKETABLE DEBT SECURITIES
The Company's marketable debt securities as of December 31, 2025 and 2024 were composed of available-for-sale commercial paper, corporate debt securities, securities of government-sponsored entities and municipal bonds. 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,
20252024
Marketable debt securities:
Commercial paper$62,988 $73,325 
Corporate debt securities151,777 203,816 
Securities of government-sponsored entities9,993 35,025 
Municipal bonds5,003 — 
Total available-for-sale marketable debt securities$229,761 $312,166 
In addition to funding operations, the decrease in the marketable debt securities balance as of December 31, 2025 is due to the repayment of convertible notes in September 2025. See Note 7 for further discussion.
The following is a summary of short-term marketable debt securities classified as available-for-sale as of December 31, 2025 (in thousands):
Remaining Contractual Maturity
(in years)		Amortized CostUnrealized GainsUnrealized LossesAggregate Estimated Fair Value
Marketable debt securities:
Commercial paperLess than 1$62,974 $20 $(6)$62,988 
Corporate debt securitiesLess than 177,414 12 (33)77,393 
Municipal bondsLess than 15,000 3 — 5,003 
Total maturity less than 1 year145,388 35 (39)145,384 
Corporate debt securities1 to 274,389 69 (74)74,384 
Securities of government-sponsored entities1 to 210,000 — (7)9,993 
Total maturity 1 to 2 years84,389 69 (81)84,377 
Total available-for-sale marketable debt securities$229,777 $104 $(120)$229,761 
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 
During 2025 and 2024, realized gains and losses on marketable debt securities were immaterial. As of December 31, 2025 and December 31, 2024, the accrued interest receivable related to the Company's marketable debt securities was $1.3 million and $2.3 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 aggregated by investment category and length of time those individual securities have been in a continuous unrealized loss position reported as of December 31, 2025 (in thousands):
Less Than 12 Months12 Months or GreaterTotal
Description of SecuritiesFair ValueUnrealized LossesFair ValueUnrealized LossesFair ValueUnrealized Losses
Commercial paper$12,317 $6 $— $— $12,317 $6 
Corporate debt securities118,443 107 — — 118,443 107 
Securities of government-sponsored entities9,993 7 — — 9,993 7 
Total$140,753 $120 $— $— $140,753 $120 
The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses aggregated by investment category and length of time those individual securities have been in a continuous unrealized loss position 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 
As of December 31, 2025 and December 31, 2024, the amortized cost of the available-for-sale marketable debt securities in an unrealized loss position was $140.9 million and $123.7 million, respectively.
As of December 31, 2025 and December 31, 2024, 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, 2025 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.
v3.25.4
CONVERTIBLE DEBT
 12 Months Ended
Dec. 31, 2025
Debt Disclosure [Abstract]  
CONVERTIBLE DEBT CONVERTIBLE DEBT
The composition of the Company’s convertible senior notes are as follows (in thousands):
 December 31, 2025December 31, 2024
2.25% convertible senior notes due 2029
$316,250 $316,250 
2.50% convertible senior notes due 2025
— 68,904 
Unamortized debt issuance costs - 2.25% convertible senior notes due 2029
(4,526)(5,940)
Unamortized debt issuance costs - 2.50% convertible senior notes due 2025
— (226)
Total convertible senior notes, net of unamortized debt discount and debt issuance costs$311,724 $378,988 
Classified as:
Convertible debt, current portion$— $68,678 
Convertible debt, less current portion311,724 310,310 
Total convertible debt$311,724 $378,988 
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, 2025 and 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. The Company 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%.
The 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 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 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 2029 Notes at December 31, 2025.
The 2029 Notes are classified as long-term convertible debt on the Company's Consolidated Balance Sheets at December 31, 2025 and December 31, 2024.
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 (collectively, the "2025 Indenture") with respect to the 2025 Notes.
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. 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. After giving effect to the repurchase the total remaining principal amount outstanding under the 2025 Notes was $68.9 million. On September 15, 2025, the 2025 Notes matured and the Company repaid the remaining principal amount outstanding of $68.9 million plus accrued interest.
Interest Expense
The following table sets forth total interest expense recognized related to the 2025 and 2029 Notes (in thousands):
Year Ended December 31,
202520242023
Contractual interest expense$8,336 $8,838 $8,838 
Amortization of debt issuance costs1,640 1,725 1,718 
Total interest expense for the 2025 and 2029 Notes$9,976 $10,563 $10,556 
Total interest expense recognized for the years ended December 31, 2025, 2024 and 2023 was $10.7 million, $11.2 million and $11.3 million, respectively.
v3.25.4
FAIR VALUE MEASUREMENTS
 12 Months Ended
Dec. 31, 2025
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, 2025 and 2024, the fair value of the Company's 2.25% Convertible Senior Notes due 2029, which were issued in 2022, was $448.3 million and $302.1 million, respectively. As of December 31, 2024, the fair value of the Company's 2.50% Convertible Senior Notes due 2025, now repaid, was $68.2 million. 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, 2025 (in thousands):
 As of December 31, 2025Fair Value Hierarchy at December 31, 2025
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$93,035 $93,035 $— $— 
Marketable debt securities, available-for-sale229,761 — 229,761 — 
Total$322,796 $93,035 $229,761 $— 
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 $— 
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, 2025, there were no impairments to long-lived assets. 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.
v3.25.4
INTANGIBLE ASSETS
 12 Months Ended
Dec. 31, 2025
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, 2025, the Company has capitalized $47.2 million for contractual milestones achieved under the Ligand License Agreement. 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, 2025 and 2024, the Company capitalized $54.3 million and $20.3 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 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 and was subsequently amended through 2029. 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 $2.1 million, representing the guaranteed minimum royalty, or 20% of the Company’s net sales of Thiola during each calendar year.
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 $6.6 million and $8.2 million as of December 31, 2025 and 2024, respectively. As of December 31, 2025, the guaranteed minimum royalty current and long-term liability was approximately $2.1 million and $4.5 million, respectively, and $2.1 million and $6.1 million, respectively, as of December 31, 2024, recorded in other current and non-current liabilities, respectively, in the Consolidated Balance Sheets.
The Company has capitalized $185.3 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, 2025 in excess of minimum royalties. In 2025 the Company recorded $14.3 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 future royalties in excess of the annual contractual minimum at December 31, 2025, as such royalties are not yet probable and estimable. The cost of the milestone payments and royalty payments are being amortized to selling, general and administration on a straight-line basis through March 31, 2026.
Amortizable intangible assets as of December 31, 2025 (in thousands):
 Useful LifeGross Carrying AmountAccumulated AmortizationNet Book Value
Ligand license11$108,163 $(13,645)$94,518 
Mission license12185,304 (166,707)18,597 
Total amortizable intangible assets$293,467 $(180,352)$113,115 
Amortizable intangible assets as of December 31, 2024 (in thousands):
Useful LifeGross Carrying AmountAccumulated AmortizationNet Book Value
Ligand license11$53,268 $(5,769)$47,499 
Mission license12171,025 (115,303)55,722 
Total amortizable intangible assets$224,293 $(121,072)$103,221 
The following table summarizes amortization expense for the year ended December 31, 2025, 2024 and 2023 (in thousands):
202520242023
Selling, general and administrative$59,210 $41,739 $29,021 
Research and development— — 7,261 
Total amortization expense$59,210 $41,739 $36,282 
As of December 31, 2025, amortization expense for the next five years and thereafter is expected to be as follows (in thousands):
2026$31,479 
202712,888 
202812,922 
202912,888 
203012,888 
Thereafter30,050 
Total$113,115 
There were no impairments related to finite-lived intangible assets in the years ended December 31, 2025, 2024 and 2023.
v3.25.4
ACCRUED EXPENSES
 12 Months Ended
Dec. 31, 2025
Payables and Accruals [Abstract]  
ACCRUED EXPENSES ACCRUED EXPENSES 
Accrued expenses consist of the following at December 31, 2025 and 2024 (in thousands):
20252024
Compensation related costs$41,515 $35,166 
Sales discounts, rebates, and allowances30,595 10,585 
Accrued royalties22,703 12,309 
Research and development16,027 16,090 
Selling, general and administrative9,818 6,154 
Miscellaneous accrued expenses5,377 5,724 
Total accrued expenses$126,035 $86,028 
v3.25.4
COMMITMENTS AND CONTINGENCIES
 12 Months Ended
Dec. 31, 2025
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 stock purchase 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, 2025, 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.
v3.25.4
STOCKHOLDERS’ EQUITY
 12 Months Ended
Dec. 31, 2025
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 22,384,114 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, 2025, there were 7,135,921 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, 2025, there were 2,780,000 shares authorized and 1,089,373 shares reserved for future issuance under the 2017 ESPP.
Stock Options
The fair values of stock option grants during the years ended December 31, 2025, 2024 and 2023 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,
 202520242023
Risk free rate4.4 %3.9 %3.9 %
Expected volatility62 %59 %50 %
Expected life (in years)6.46.46.4
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, 2025:
Weighted Average
Shares Underlying
Options		Exercise
Price		Remaining
Contractual
Term (in years)		Aggregate
Intrinsic Value
(in thousands)
Outstanding at December 31, 20249,283,331 $20.13 5.51$16,408 
Granted1,484,200 $20.65 
Exercised(1,869,242)$18.63 $23,247 
Forfeited and expired(656,860)$28.29 
Outstanding at December 31, 20258,241,429 $19.91 5.89$150,796 
Vested and expected to vest at December 31, 20258,241,429 $19.91 5.89$150,796 
Exercisable at December 31, 20255,813,961 $20.83 4.77$101,064 
The aggregate intrinsic value of stock options exercised in the years ended December 31, 2025, 2024 and 2023 was $23.2 million, $2.0 million, and $1.4 million, respectively.
The weighted average grant date fair value of options granted was $12.86, $5.10, and $11.66 during the years ended December 31, 2025, 2024 and 2023, 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 $38.21, $17.42 and $8.99 as of December 31, 2025, 2024 and 2023, respectively. Unrecognized compensation cost associated with unvested stock options amounts to $20.7 million as of December 31, 2025, 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, 2025, there was approximately $58.5 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 2.5 years.
The following table summarizes our restricted stock unit activity for the year ended December 31, 2025:
Number of
RSUs		Weighted Average
Grant Date Fair Value
Unvested December 31, 20243,517,263 $15.41 
Granted2,662,490 $20.68 
Vested(1,314,671)$16.72 
Forfeited/cancelled(297,715)$16.69 
Unvested December 31, 20254,567,367 $18.02 
The fair value of restricted stock units vested for the years ended December 31, 2025, 2024 and 2023 was $22.0 million, $22.8 million, and $18.7 million, respectively. The weighted average grant date fair value for stock awards granted during the years ended December 31, 2025, 2024 and 2023 was $20.68, $9.07 and $21.20, respectively.
Performance-based Stock Units
Performance-based stock units ("PSUs") are subject to vest only if certain specified criteria are achieved. A target number of shares is established for each award; however, the actual number of shares that are issued when an award vests may range from zero to 200% of the target amount depending upon the level of achievement of the applicable performance metric. As of December 31, 2025, there was approximately $0.1 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 0.4 years.
The following table summarizes our performance-based stock unit activity for the year ended December 31, 2025:
 Number of
PSUs		Weighted Average
Grant Date Fair Value
Unvested December 31, 2024216,208 $18.34 
Granted88,000 $20.46 
Vested(70,604)$27.54 
Forfeited/cancelled— — 
Unvested December 31, 2025233,604 $16.35 
The fair value of PSUs vested for the years ended December 31, 2025, 2024 and 2023 was $1.9 million, $1.5 million, and $0.3 million, respectively. The weighted average grant date fair value for performance-based stock awards granted during the years ended December 31, 2025, 2024 and 2023 was $20.46, $8.93 and $22.40, respectively.
Share Based Compensation
Total non-cash stock-based compensation expense consisted of the following for the years ended December 31, 2025, 2024 and 2023 (in thousands):
 Year Ended December 31,
 202520242023
Selling, general and administrative expenses$28,187 $22,735 $27,111 
Research and development expenses16,681 14,178 17,135 
Total stock-based compensation expense$44,868 $36,913 $44,246 
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. As of December 31, 2025, the Company has not sold any shares under the ATM Agreement.
v3.25.4
NET LOSS PER COMMON SHARE
 12 Months Ended
Dec. 31, 2025
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,
202520242023
SharesNet (loss) incomeEPSSharesNet lossEPSSharesNet (loss) incomeEPS
Continuing operations89,211,813 $(50,261)$(0.57)78,888,861 $(320,630)$(4.07)74,267,418 $(376,333)$(5.07)
Discontinued operations89,211,813 24,715 0.28 78,888,861 (915)(0.01)74,267,418 264,934 3.57 
Basic and diluted loss per share89,211,813 $(25,546)$(0.29)78,888,861 $(321,545)$(4.08)74,267,418 $(111,399)$(1.50)
For the years ended December 31, 2025, 2024 and 2023, the following weighted-average number of common stock equivalents were excluded because they were anti-dilutive:
For the year ended December 31,
202520242023
Convertible debt11,172,474 11,697,953 11,697,952 
Options9,665,404 10,447,541 10,555,550 
Restricted stock units and performance-based stock units4,437,355 3,780,441 3,417,245 
Total anti-dilutive shares25,275,233 25,925,935 25,670,747 
v3.25.4
INCOME TAXES
 12 Months Ended
Dec. 31, 2025
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,
202520242023
United States$(2,713)$(152,356)$(293,283)
Foreign(46,560)(168,154)(82,827)
Total$(49,273)$(320,510)$(376,110)
The components of the provision for income taxes, in the Consolidated Statements of Operations are as follows (in thousands):
Year Ended December 31,
 202520242023
Current
Federal$— $— $— 
State988 120 223 
Foreign— — — 
Total current988 120 223 
Deferred
Federal— — — 
State— — — 
Total deferred— — — 
Total tax provision$988 $120 $223 
In July 2025 the One Big Beautiful Bill Act was signed into law, which enacts significant changes to U.S. tax and related laws. The legislation did not have a material impact on the Company's income tax expense for the year ended December 31, 2025, nor did it materially change the Company's effective income tax rate for 2025.
The effective income tax rate for the year ended December 31, 2025 differs from the statutory federal income tax rate as follows (in thousands, except percentages):
2025
 AmountPercentage
Statutory rate - federal$(10,347)(21.00)%
State taxes, net of federal benefit*701 1.42 %
Foreign tax effects
Switzerland
Statutory tax rate differences5,831 11.83 %
Changes in valuation allowances3,758 7.63 %
Other207 0.42 %
Other foreign jurisdictions
Other(18)(0.04)%
Tax credits
Orphan drug credits(16,224)(32.93)%
Return to provision adjustments(2,517)(5.10)%
Changes in valuation allowances16,275 33.03 %
Nontaxable or nondeductible items
  Executive compensation1,710 3.47 %
  Share-based awards1,246 2.53 %
  Other442 0.90 %
Change in unrecognized tax benefits(1,456)(2.96)%
Other adjustments
Return to provision adjustments and other true-ups1,380 2.80 %
Effective tax rate$988 2.00 %
* State taxes in California, Michigan and New Jersey for 2025 made up the majority (greater than 50%) of the tax effect in this category.
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 for the years ended December 31, 2024 and 2023:
 20242023
Statutory rate - federal(21.00)%(21.00)%
State taxes, net of federal benefit(1.55)%(3.19)%
Foreign rate differential2.15 %1.29 %
IPR&D4.27 %— %
Nondeductible executive compensation0.44 %1.50 %
Excess tax benefits associated with share-based awards2.92 %0.68 %
Other permanent differences0.23 %0.37 %
Tax credits(4.24)%(1.13)%
Return to provision adjustments and other true-ups(0.80)%4.19 %
Other0.93 %0.57 %
Change in valuation allowance16.69 %16.78 %
Income tax provision0.04 %0.06 %
The significant components of the Company’s deferred tax assets and liabilities as of December 31, 2025 and 2024 are as follows (in thousands):
 20252024
Deferred Tax Assets:
Net operating loss$130,051 $113,303 
Research and development and other tax credits112,093 91,760 
Intangible assets56,052 53,919 
Capitalized research and development27,170 47,880 
Stock based compensation13,971 15,680 
Other accrued expenses21,727 14,266 
Charitable contributions5,699 5,797 
Operating lease liabilities4,164 5,421 
174A state only difference2,397 — 
Depreciation602 435 
Loan costs117 153 
Total deferred tax assets374,043 348,614 
Deferred Tax Liabilities:
Operating lease right of use assets(3,211)(4,259)
Prepaid assets(184)(178)
Total deferred tax liabilities(3,395)(4,437)
Net deferred tax assets before valuation allowance370,648 344,177 
Valuation allowance(370,648)(344,177)
Total deferred tax assets$— $— 
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 $370.6 million as of December 31, 2025 to reflect the estimated amount of deferred tax assets that may not be realized. The Company increased its valuation allowance by $26.5 million for the year ended December 31, 2025, compared to a $53.5 million increase for the year ended December 31, 2024.
As of December 31, 2025, the Company had available unused U.S. federal and state net operating loss (“NOL”) carryforwards of $232.6 million and $214.0 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 2026 unless previously utilized, except for $41.1 million of the state net operating losses that have an indefinite carryforward period. In addition, at December 31, 2025, the Company had federal orphan drug tax credit carryforwards of $122.8 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 $1.3 million that begin to expire in 2030 unless utilized and $11.0 million that have an indefinite carryforward period, and California Competes tax credit carryforwards of $0.8 million that begin to expire in 2026. 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, 2025, the Company had Irish NOL carryforwards of $15.9 million which are fully offset by a valuation allowance and have an indefinite life. The Company also had Swiss NOL carryforwards of $475.2 million which are fully offset by a valuation allowance and begin to expire in 2026, as well as Federal Act on Tax Reform and AHV Financing 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, 2025, the Company had $26.0 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 $26.0 million. To the extent unrecognized tax benefits are recognized at a time when a valuation allowance does not exist, the recognition of the $26.0 million tax benefit would reduce the effective tax rate.
A reconciliation of the Company's unrecognized tax benefits for the years 2025, 2024 and 2023 is provided in the following table (in thousands):
202520242023
Balance as of January 1:$27,404 $22,906 $11,490 
Increase in current period positions3,343 4,248 4,871 
Increase in prior period positions— 250 7,383 
Decrease in prior period positions(4,716)— (838)
Balance as of December 31:$26,031 $27,404 $22,906 
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, 2020 and forward, and the Company's Irish tax returns are open to examination for the years ended December 31, 2021 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, 2025, 2024 and 2023.
The cash taxes paid by the Company for the years ended December 31, 2025, 2024 and 2023 were immaterial.
v3.25.4
EQUITY OFFERINGS
 12 Months Ended
Dec. 31, 2025
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 22,384,114 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, 2025, there were 7,135,921 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, 2025, there were 2,780,000 shares authorized and 1,089,373 shares reserved for future issuance under the 2017 ESPP.
Stock Options
The fair values of stock option grants during the years ended December 31, 2025, 2024 and 2023 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,
 202520242023
Risk free rate4.4 %3.9 %3.9 %
Expected volatility62 %59 %50 %
Expected life (in years)6.46.46.4
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, 2025:
Weighted Average
Shares Underlying
Options		Exercise
Price		Remaining
Contractual
Term (in years)		Aggregate
Intrinsic Value
(in thousands)
Outstanding at December 31, 20249,283,331 $20.13 5.51$16,408 
Granted1,484,200 $20.65 
Exercised(1,869,242)$18.63 $23,247 
Forfeited and expired(656,860)$28.29 
Outstanding at December 31, 20258,241,429 $19.91 5.89$150,796 
Vested and expected to vest at December 31, 20258,241,429 $19.91 5.89$150,796 
Exercisable at December 31, 20255,813,961 $20.83 4.77$101,064 
The aggregate intrinsic value of stock options exercised in the years ended December 31, 2025, 2024 and 2023 was $23.2 million, $2.0 million, and $1.4 million, respectively.
The weighted average grant date fair value of options granted was $12.86, $5.10, and $11.66 during the years ended December 31, 2025, 2024 and 2023, 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 $38.21, $17.42 and $8.99 as of December 31, 2025, 2024 and 2023, respectively. Unrecognized compensation cost associated with unvested stock options amounts to $20.7 million as of December 31, 2025, 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, 2025, there was approximately $58.5 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 2.5 years.
The following table summarizes our restricted stock unit activity for the year ended December 31, 2025:
Number of
RSUs		Weighted Average
Grant Date Fair Value
Unvested December 31, 20243,517,263 $15.41 
Granted2,662,490 $20.68 
Vested(1,314,671)$16.72 
Forfeited/cancelled(297,715)$16.69 
Unvested December 31, 20254,567,367 $18.02 
The fair value of restricted stock units vested for the years ended December 31, 2025, 2024 and 2023 was $22.0 million, $22.8 million, and $18.7 million, respectively. The weighted average grant date fair value for stock awards granted during the years ended December 31, 2025, 2024 and 2023 was $20.68, $9.07 and $21.20, respectively.
Performance-based Stock Units
Performance-based stock units ("PSUs") are subject to vest only if certain specified criteria are achieved. A target number of shares is established for each award; however, the actual number of shares that are issued when an award vests may range from zero to 200% of the target amount depending upon the level of achievement of the applicable performance metric. As of December 31, 2025, there was approximately $0.1 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 0.4 years.
The following table summarizes our performance-based stock unit activity for the year ended December 31, 2025:
 Number of
PSUs		Weighted Average
Grant Date Fair Value
Unvested December 31, 2024216,208 $18.34 
Granted88,000 $20.46 
Vested(70,604)$27.54 
Forfeited/cancelled— — 
Unvested December 31, 2025233,604 $16.35 
The fair value of PSUs vested for the years ended December 31, 2025, 2024 and 2023 was $1.9 million, $1.5 million, and $0.3 million, respectively. The weighted average grant date fair value for performance-based stock awards granted during the years ended December 31, 2025, 2024 and 2023 was $20.46, $8.93 and $22.40, respectively.
Share Based Compensation
Total non-cash stock-based compensation expense consisted of the following for the years ended December 31, 2025, 2024 and 2023 (in thousands):
 Year Ended December 31,
 202520242023
Selling, general and administrative expenses$28,187 $22,735 $27,111 
Research and development expenses16,681 14,178 17,135 
Total stock-based compensation expense$44,868 $36,913 $44,246 
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. As of December 31, 2025, the Company has not sold any shares under the ATM Agreement.
v3.25.4
RETIREMENT PLAN
 12 Months Ended
Dec. 31, 2025
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 $2.8 million, $2.4 million, and $2.7 million for the years ended December 31, 2025, 2024 and 2023, 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, 2025, the projected benefit obligations under the Swiss Plan were approximately $1.7 million, and plan assets were approximately $1.0 million. The funded status of the Swiss Plan is included in other long-term liabilities on the Company's Consolidated Balance Sheets.
v3.25.4
PROPERTY AND EQUIPMENT
 12 Months Ended
Dec. 31, 2025
Property, Plant and Equipment [Abstract]  
PROPERTY AND EQUIPMENT PROPERTY AND EQUIPMENT
Property, plant and equipment, net consisted of the following (in thousands):
December 31,
20252024
Leasehold improvements$8,992 $9,370 
Furniture and fixtures2,397 2,446 
Computers and equipment2,731 2,275 
14,120 14,091 
Less: Accumulated depreciation(10,098)(8,755)
Total property and equipment, net$4,022 $5,336 
Depreciation expense for the years ended December 31, 2025, 2024 and 2023 was $1.5 million, $1.8 million and $2.2 million, respectively.
v3.25.4
LEASES
 12 Months Ended
Dec. 31, 2025
Leases [Abstract]  
LEASES LEASES
As of December 31, 2025, the Company had an 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 five 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.
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.
During the year ended December 31, 2024, the Company identified an indicator of impairment of the 5th floor 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 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, 2025 and 2023.
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, 2025 (in thousands):
December 31, 2025
2026$6,775 
20276,978 
20284,782 
2029 and thereafter— 
Total undiscounted future minimum payments18,535 
Present value discount(1,526)
Total lease liability17,009 
Unamortized lease incentives(2,539)
Cash payments in excess of straight-line lease expense(3,894)
Total ROU asset$10,576 
The weighted-average remaining lease term and weighted-average discount rate of the Company's operating leases are as follows:
December 31,
20252024
Weighted-average remaining lease term in years2.73.7
Weighted-average discount rate6.48 %6.48 %
For the years ended December 31, 2025, 2024 and 2023 the Company recorded $4.4 million, $4.8 million, and $4.9 million, respectively, in expense related to operating leases, including amortized tenant improvement allowances.
v3.25.4
DIVESTITURES
 12 Months Ended
Dec. 31, 2025
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. Mirum achieved the first such milestone based on its annual net sales in 2025, and Travere recognized a milestone of $25.0 million for the year ended December 31, 2025 in discontinued operations. The $25.0 million is included in accounts receivable as of December 31, 2025.
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 year ended December 31, 2024, the Company recognized $0.5 million, under the TSA, included in continuing operations within other income (expense), net. TSA services provided by the Company were substantially complete as of December 31, 2024.
The Company determined that the divestiture represents a strategic shift that will have a major effect on the Company's operations and financial results, and has therefore reflected the bile acid business as a discontinued operation for all periods presented.
Results of discontinued operations are as follows (in thousands):
Year Ended December 31,
202520242023
Net product sales$— $(550)$66,164 
Operating expenses:
Cost of goods sold— (9)1,899 
Research and development— 247 6,118 
Selling, general and administrative— 127 19,500 
Change in fair value of contingent consideration— — (473)
Total operating expenses— 365 27,044 
Operating (loss) income— (915)39,120 
Other income, net:
Interest expense— — (191)
Gain on disposal of discontinued operations, net of tax24,715 — 226,005 
Total other income, net24,715 — 225,814 
Net income (loss) from discontinued operations$24,715 $(915)$264,934 
v3.25.4
SEGMENT INFORMATION
 12 Months Ended
Dec. 31, 2025
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. Long-lived assets located outside the U.S. were immaterial as of December 31, 2025 and 2024. 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.
See Note 3 for further discussion of net product sales and Note 4 for discussion of license and collaboration revenues.
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, 2025, 2024 and 2023 (in thousands):
Year Ended December 31,
202520242023
Revenue$490,728 $233,175 $145,238 
Less:
Cost of goods sold10,339 7,744 11,450 
Research and development:
External research and development107,036 126,303 142,482 
Internal personnel costs80,091 73,843 84,658 
Other research and development18,884 17,350 17,850 
Total research and development206,011 217,496 244,990 
Selling, general and administrative337,202 264,119 265,542 
In-process research and development— 65,205 — 
Restructuring— 2,438 11,394 
Total other income, net13,551 3,317 12,028 
Income tax provision on continuing operations(988)(120)(223)
Income (loss) from discontinued operations, net of tax24,715 (915)264,934 
Net loss$(25,546)$(321,545)$(111,399)
v3.25.4
Insider Trading Arrangements
 3 Months Ended
Dec. 31, 2025
shares
Trading Arrangements, by Individual  
Material Terms of Trading Arrangement
During the fiscal quarter ended December 31, 2025, certain of our directors and/or officers (as defined in Rule 16a-1(f) under the Exchange Act) adopted or terminated a “Rule 10b5-1 trading arrangement” or “non-Rule 10b5-1 trading arrangement,” as those terms are defined in Regulation S-K, Item 408, as set forth below. Each 10b5-1 plan listed below is part of such individual’s long-term asset diversification, tax and/or financial planning strategy, and has been entered into in accordance with the terms of our Insider Trading Policy. Under the terms of the plans, once the plans are in effect, the individuals listed below have no discretion or control over the timing or effectuation of any transactions in our securities pursuant to the plans, and sales will be made only if the stock meets the minimum price thresholds and/or other requirements specified in the applicable plan. Some of the planned sales relate to shares required to be sold by such individual to cover the tax withholding obligation in connection with the settlement of vested restricted stock units and/or performance restricted stock units. Each such director and/or officer continues to be subject to our stock ownership guidelines, and the execution of all potential sales under the plan would not by themselves cause any such individual to be out of compliance with the requirements of our stock ownership guidelines.
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)
Roy Baynes,
member of our Board of Directors
November 17, 2025Rule 10b5-1 Trading Arrangement
Up to 61,500 shares (3)
November 13, 2026
Timothy Coughlin,
member of our Board of Directors
November 19, 2025Rule 10b5-1 Trading Arrangement
Up to 13,250 shares (3)
February 25, 2027
Gary Lyons,
chair of our Board of Directors
November 11, 2025Rule 10b5-1 Trading Arrangement
Up to 8,000 shares (4)
May 17, 2026
Jeffrey Meckler,
member of our Board of Directors
November 17, 2025Rule 10b5-1 Trading Arrangement
Up to 8,000 shares (4)
May 18, 2026
1Each 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.
3
Includes shares underlying stock options expiring in May 2028.
4
Consists of shares of underlying stock options expiring in May 2026.
Non-Rule 10b5-1 Arrangement Adopted false
Rule 10b5-1 Arrangement Terminated false
Non-Rule 10b5-1 Arrangement Terminated false
Roy Baynes [Member]  
Trading Arrangements, by Individual  
Name Roy Baynes
Title member of our Board of Directors
Rule 10b5-1 Arrangement Adopted true
Adoption Date November 17, 2025
Expiration Date November 13, 2026
Arrangement Duration 361 days
Aggregate Available 61,500
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 19, 2025
Expiration Date February 25, 2027
Arrangement Duration 463 days
Aggregate Available 13,250
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 November 11, 2025
Expiration Date May 17, 2026
Arrangement Duration 187 days
Aggregate Available 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 November 17, 2025
Expiration Date May 18, 2026
Arrangement Duration 182 days
Aggregate Available 8,000
v3.25.4
Insider Trading Policies and Procedures
 12 Months Ended
Dec. 31, 2025
Insider Trading Policies and Procedures [Line Items]  
Insider Trading Policies and Procedures Adopted true
v3.25.4
Cybersecurity Risk Management and Strategy Disclosure
 12 Months Ended
Dec. 31, 2025
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 routine tabletop incident response exercises; and evaluating certain threats reported to us. With the help of our managed services provider, we have implemented a Security Operations Center ("SOC") service to provide continuous 24/7 monitoring and incident response across our network infrastructure.
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 cybersecurity risks are integrated into our enterprise risk management program. Cybersecurity risk is addressed as part of our overall risk governance framework, and members of our management team, IT department and other relevant functional teams collaborate to prioritize risk mitigation efforts, address threats that may have a material impact on our business, and report regularly to our board of directors on cybersecurity matters.
We engage third-party service providers to assist us 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
v3.25.4
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies)
 12 Months Ended
Dec. 31, 2025
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, 2025, 2024 and 2023, adjustments to net product revenue related to performance obligations satisfied in previous periods were $1.1 million, $0.5 million, and $0.4 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, 2025, sales of FILSPARI primarily consisted of zero-cost inventories. As of December 31, 2025 the zero-cost inventory remaining was immaterial. The Company began capitalizing inventory costs associated with FILSPARI following the February 2023 accelerated approval.
For the year ended 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 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 currently consists of the sale of active pharmaceutical ingredients to the Company's collaboration partners, at cost or 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.
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 and year to year.
Advertising Expenses
Advertising Expenses
Advertising costs are expensed as incurred. The Company incurred $14.7 million, $7.8 million and zero in advertising costs during the years ended December 31, 2025, 2024 and 2023, respectively.
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 using the treasury stock method. The treasury stock method reduces the number of shares issuable by the shares 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/(loss) per share calculation. As the Company has reported net losses for all periods presented, all potentially dilutive securities are antidilutive and, accordingly, basic net loss per share equals diluted net loss per share. 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 investments 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, 2025 and 2024, respectively. For the years ended December 31, 2025, 2024 and 2023, provision for credit losses 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 Thiola and 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 and Comprehensive Loss 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 and Comprehensive Loss.
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, 2025 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 Sheets 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, 2025, the Company did not record any basis adjustments. For the year ended December 31, 2024, the Company recognized $3.4 million in other income (expense), net for these basis adjustments and reduced the equity method investment's carrying value 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 $0.3 million, $0.7 million and a gain of $0.5 million arising from foreign exchange transactions is included in other income (expense), net for the years ended December 31, 2025, 2024 and 2023, 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, 2025 and 2024;
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 and Comprehensive Loss.
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 and Comprehensive Loss 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.
Recently Adopted and Issued Accounting Pronouncements
Recently Adopted Accounting Pronouncements
In December 2023, the FASB issued Accounting Standards Update ("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. The Company adopted the standard and applied the disclosure requirements on a prospective basis as required for the year ended December 31, 2025.
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 September 2025, the FASB issued ASU No. 2025-06—Intangibles—Goodwill and Other—Internal-Use Software (Subtopic 350-40): Targeted Improvements to the Accounting for Internal-Use Software. This ASU removed the language around project stages that was used to assess when costs could be capitalized for an internal-use software. The update also requires internal-use software to be disclosed under the ASC 360 Property, Plant, and Equipment guidance. The guidance is effective for annual periods beginning after December 15, 2027. The Company is currently assessing the impact of this standard on the Company’s accounting policies and the financial statements.
In July 2025, the FASB issued ASU No. 2025-05—Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses for Accounts Receivable and Contract Assets. This ASU added a practical expedient that assumes that current conditions as of the balance sheet date do not change for the remaining life of the asset when estimating expected credit losses for current accounts receivable and current contract assets. The guidance is effective for annual periods beginning after December 15, 2025. The Company is currently evaluating the impact of the adoption of this standard on the accounting for credit losses.
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 ASU 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, as clarified by ASU No. 2025-01, for annual reporting periods beginning after December 15, 2026 and interim reporting periods within annual 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.
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.
v3.25.4
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Tables)
 12 Months Ended
Dec. 31, 2025
Accounting Policies [Abstract]  
Schedule of Inventory, Net of Reserves
Inventory, net of reserves, consisted of the following at December 31, 2025 and 2024 (in thousands):
December 31, 2025December 31, 2024
Raw materials$24,169 $30,552 
Work in process2,348 7,625 
Finished goods9,638 3,679 
Total inventory$36,155 $41,856 
Classified as:
Inventory$5,875 $6,200 
Long-term inventory30,280 35,656 
Total inventory$36,155 $41,856 
Schedule Of Cost Of Goods Sold
The following table summarizes cost of goods sold for the years ended December 31, 2025, 2024 and 2023 (in thousands):
Year Ended December 31,
202520242023
Cost of goods sold - product sales$5,813 $7,446 $8,406 
Cost of goods sold - license and collaboration4,526 298 3,044 
Total cost of goods sold$10,339 $7,744 $11,450 
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,
20252024
Leasehold improvements$8,992 $9,370 
Furniture and fixtures2,397 2,446 
Computers and equipment2,731 2,275 
14,120 14,091 
Less: Accumulated depreciation(10,098)(8,755)
Total property and equipment, net$4,022 $5,336 
Schedule of Foreign Currency Translation Adjustment Included in Accumulated Other Comprehensive Loss
The following table summarizes the foreign currency translation adjustment included in accumulated other comprehensive loss for the year ended December 31, 2025, 2024 and 2023 (in thousands):
Foreign Currency Translation Adjustments included in Accumulated Other Comprehensive Loss
202520242023
Balance at January 1,$(34)$(1,456)$415 
Foreign currency translation adjustments(945)1,422 (1,871)
Balance at December 31,$(979)$(34)$(1,456)
Schedule of Restructuring and Related Costs
The following table summarizes the cash payments and accruals, included in accrued expenses of the Consolidated Balance Sheets, related to the restructuring for the year ended December 31, 2024 (in thousands):
2024
Liability balance at January 1,$11,421 
Restructuring expenses2,438 
Non-cash impairment and disposal charges(856)
Payments(12,929)
Foreign currency impact(74)
Liability balance at December 31,$— 
v3.25.4
REVENUE RECOGNITION (Tables)
 12 Months Ended
Dec. 31, 2025
Revenue from Contract with Customer [Abstract]  
Schedule of Net Product Sales
The following table summarizes net product sales for the year ended December 31, 2025, 2024 and 2023 (in thousands):
Year Ended December 31,
202520242023
FILSPARI$322,005 $132,222 $29,208 
Tiopronin products88,455 94,485 98,329 
Total net product sales$410,460 $226,707 $127,537 
v3.25.4
COLLABORATION AND LICENSE AGREEMENTS (Tables)
 12 Months Ended
Dec. 31, 2025
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, 2025, 2024 and 2023 (in thousands):
Deferred Revenue
202520242023
Balance at January 1,$2,815 $8,931 $22,907 
License and collaboration revenue(2,872)(5,847)(14,363)
Foreign currency impact57 (269)387 
Balance at December 31,$— $2,815 $8,931 
v3.25.4
MARKETABLE DEBT SECURITIES (Tables)
 12 Months Ended
Dec. 31, 2025
Investments, Debt and Equity Securities [Abstract]  
Schedule of Marketable Debt Securities
Marketable debt securities consisted of the following (in thousands):
As of December 31,
20252024
Marketable debt securities:
Commercial paper$62,988 $73,325 
Corporate debt securities151,777 203,816 
Securities of government-sponsored entities9,993 35,025 
Municipal bonds5,003 — 
Total available-for-sale marketable debt securities$229,761 $312,166 
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, 2025 (in thousands):
Remaining Contractual Maturity
(in years)		Amortized CostUnrealized GainsUnrealized LossesAggregate Estimated Fair Value
Marketable debt securities:
Commercial paperLess than 1$62,974 $20 $(6)$62,988 
Corporate debt securitiesLess than 177,414 12 (33)77,393 
Municipal bondsLess than 15,000 3 — 5,003 
Total maturity less than 1 year145,388 35 (39)145,384 
Corporate debt securities1 to 274,389 69 (74)74,384 
Securities of government-sponsored entities1 to 210,000 — (7)9,993 
Total maturity 1 to 2 years84,389 69 (81)84,377 
Total available-for-sale marketable debt securities$229,777 $104 $(120)$229,761 
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 
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 aggregated by investment category and length of time those individual securities have been in a continuous unrealized loss position reported as of December 31, 2025 (in thousands):
Less Than 12 Months12 Months or GreaterTotal
Description of SecuritiesFair ValueUnrealized LossesFair ValueUnrealized LossesFair ValueUnrealized Losses
Commercial paper$12,317 $6 $— $— $12,317 $6 
Corporate debt securities118,443 107 — — 118,443 107 
Securities of government-sponsored entities9,993 7 — — 9,993 7 
Total$140,753 $120 $— $— $140,753 $120 
The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses aggregated by investment category and length of time those individual securities have been in a continuous unrealized loss position 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 
v3.25.4
CONVERTIBLE DEBT (Tables)
 12 Months Ended
Dec. 31, 2025
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, 2025December 31, 2024
2.25% convertible senior notes due 2029
$316,250 $316,250 
2.50% convertible senior notes due 2025
— 68,904 
Unamortized debt issuance costs - 2.25% convertible senior notes due 2029
(4,526)(5,940)
Unamortized debt issuance costs - 2.50% convertible senior notes due 2025
— (226)
Total convertible senior notes, net of unamortized debt discount and debt issuance costs$311,724 $378,988 
Classified as:
Convertible debt, current portion$— $68,678 
Convertible debt, less current portion311,724 310,310 
Total convertible debt$311,724 $378,988 
The following table sets forth total interest expense recognized related to the 2025 and 2029 Notes (in thousands):
Year Ended December 31,
202520242023
Contractual interest expense$8,336 $8,838 $8,838 
Amortization of debt issuance costs1,640 1,725 1,718 
Total interest expense for the 2025 and 2029 Notes$9,976 $10,563 $10,556 
v3.25.4
FAIR VALUE MEASUREMENTS (Tables)
 12 Months Ended
Dec. 31, 2025
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, 2025 (in thousands):
 As of December 31, 2025Fair Value Hierarchy at December 31, 2025
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$93,035 $93,035 $— $— 
Marketable debt securities, available-for-sale229,761 — 229,761 — 
Total$322,796 $93,035 $229,761 $— 
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 $— 
v3.25.4
INTANGIBLE ASSETS (Tables)
 12 Months Ended
Dec. 31, 2025
Goodwill and Intangible Assets Disclosure [Abstract]  
Schedule of Amortizable Intangible Assets
Amortizable intangible assets as of December 31, 2025 (in thousands):
 Useful LifeGross Carrying AmountAccumulated AmortizationNet Book Value
Ligand license11$108,163 $(13,645)$94,518 
Mission license12185,304 (166,707)18,597 
Total amortizable intangible assets$293,467 $(180,352)$113,115 
Amortizable intangible assets as of December 31, 2024 (in thousands):
Useful LifeGross Carrying AmountAccumulated AmortizationNet Book Value
Ligand license11$53,268 $(5,769)$47,499 
Mission license12171,025 (115,303)55,722 
Total amortizable intangible assets$224,293 $(121,072)$103,221 
Schedule of Amortization Expense
The following table summarizes amortization expense for the year ended December 31, 2025, 2024 and 2023 (in thousands):
202520242023
Selling, general and administrative$59,210 $41,739 $29,021 
Research and development— — 7,261 
Total amortization expense$59,210 $41,739 $36,282 
As of December 31, 2025, amortization expense for the next five years and thereafter is expected to be as follows (in thousands):
2026$31,479 
202712,888 
202812,922 
202912,888 
203012,888 
Thereafter30,050 
Total$113,115 
v3.25.4
ACCRUED EXPENSES (Tables)
 12 Months Ended
Dec. 31, 2025
Payables and Accruals [Abstract]  
Schedule of Accrued Expenses
Accrued expenses consist of the following at December 31, 2025 and 2024 (in thousands):
20252024
Compensation related costs$41,515 $35,166 
Sales discounts, rebates, and allowances30,595 10,585 
Accrued royalties22,703 12,309 
Research and development16,027 16,090 
Selling, general and administrative9,818 6,154 
Miscellaneous accrued expenses5,377 5,724 
Total accrued expenses$126,035 $86,028 
v3.25.4
STOCKHOLDERS’ EQUITY (Tables)
 12 Months Ended
Dec. 31, 2025
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,
 202520242023
Risk free rate4.4 %3.9 %3.9 %
Expected volatility62 %59 %50 %
Expected life (in years)6.46.46.4
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, 2025:
Weighted Average
Shares Underlying
Options		Exercise
Price		Remaining
Contractual
Term (in years)		Aggregate
Intrinsic Value
(in thousands)
Outstanding at December 31, 20249,283,331 $20.13 5.51$16,408 
Granted1,484,200 $20.65 
Exercised(1,869,242)$18.63 $23,247 
Forfeited and expired(656,860)$28.29 
Outstanding at December 31, 20258,241,429 $19.91 5.89$150,796 
Vested and expected to vest at December 31, 20258,241,429 $19.91 5.89$150,796 
Exercisable at December 31, 20255,813,961 $20.83 4.77$101,064 
Schedule of Restricted Stock Unit Activity
The following table summarizes our restricted stock unit activity for the year ended December 31, 2025:
Number of
RSUs		Weighted Average
Grant Date Fair Value
Unvested December 31, 20243,517,263 $15.41 
Granted2,662,490 $20.68 
Vested(1,314,671)$16.72 
Forfeited/cancelled(297,715)$16.69 
Unvested December 31, 20254,567,367 $18.02 
Schedule of Performance-based Stock Unit Activity
The following table summarizes our performance-based stock unit activity for the year ended December 31, 2025:
 Number of
PSUs		Weighted Average
Grant Date Fair Value
Unvested December 31, 2024216,208 $18.34 
Granted88,000 $20.46 
Vested(70,604)$27.54 
Forfeited/cancelled— — 
Unvested December 31, 2025233,604 $16.35 
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, 2025, 2024 and 2023 (in thousands):
 Year Ended December 31,
 202520242023
Selling, general and administrative expenses$28,187 $22,735 $27,111 
Research and development expenses16,681 14,178 17,135 
Total stock-based compensation expense$44,868 $36,913 $44,246 
v3.25.4
NET LOSS PER COMMON SHARE (Tables)
 12 Months Ended
Dec. 31, 2025
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,
202520242023
SharesNet (loss) incomeEPSSharesNet lossEPSSharesNet (loss) incomeEPS
Continuing operations89,211,813 $(50,261)$(0.57)78,888,861 $(320,630)$(4.07)74,267,418 $(376,333)$(5.07)
Discontinued operations89,211,813 24,715 0.28 78,888,861 (915)(0.01)74,267,418 264,934 3.57 
Basic and diluted loss per share89,211,813 $(25,546)$(0.29)78,888,861 $(321,545)$(4.08)74,267,418 $(111,399)$(1.50)
Schedule of Anti-dilutive Securities
For the years ended December 31, 2025, 2024 and 2023, the following weighted-average number of common stock equivalents were excluded because they were anti-dilutive:
For the year ended December 31,
202520242023
Convertible debt11,172,474 11,697,953 11,697,952 
Options9,665,404 10,447,541 10,555,550 
Restricted stock units and performance-based stock units4,437,355 3,780,441 3,417,245 
Total anti-dilutive shares25,275,233 25,925,935 25,670,747 
v3.25.4
INCOME TAXES (Tables)
 12 Months Ended
Dec. 31, 2025
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,
202520242023
United States$(2,713)$(152,356)$(293,283)
Foreign(46,560)(168,154)(82,827)
Total$(49,273)$(320,510)$(376,110)
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):
Year Ended December 31,
 202520242023
Current
Federal$— $— $— 
State988 120 223 
Foreign— — — 
Total current988 120 223 
Deferred
Federal— — — 
State— — — 
Total deferred— — — 
Total tax provision$988 $120 $223 
Schedule of Reconciliation of Statutory Federal Income Tax Rate
The effective income tax rate for the year ended December 31, 2025 differs from the statutory federal income tax rate as follows (in thousands, except percentages):
2025
 AmountPercentage
Statutory rate - federal$(10,347)(21.00)%
State taxes, net of federal benefit*701 1.42 %
Foreign tax effects
Switzerland
Statutory tax rate differences5,831 11.83 %
Changes in valuation allowances3,758 7.63 %
Other207 0.42 %
Other foreign jurisdictions
Other(18)(0.04)%
Tax credits
Orphan drug credits(16,224)(32.93)%
Return to provision adjustments(2,517)(5.10)%
Changes in valuation allowances16,275 33.03 %
Nontaxable or nondeductible items
  Executive compensation1,710 3.47 %
  Share-based awards1,246 2.53 %
  Other442 0.90 %
Change in unrecognized tax benefits(1,456)(2.96)%
Other adjustments
Return to provision adjustments and other true-ups1,380 2.80 %
Effective tax rate$988 2.00 %
* State taxes in California, Michigan and New Jersey for 2025 made up the majority (greater than 50%) of the tax effect in this category.
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 for the years ended December 31, 2024 and 2023:
 20242023
Statutory rate - federal(21.00)%(21.00)%
State taxes, net of federal benefit(1.55)%(3.19)%
Foreign rate differential2.15 %1.29 %
IPR&D4.27 %— %
Nondeductible executive compensation0.44 %1.50 %
Excess tax benefits associated with share-based awards2.92 %0.68 %
Other permanent differences0.23 %0.37 %
Tax credits(4.24)%(1.13)%
Return to provision adjustments and other true-ups(0.80)%4.19 %
Other0.93 %0.57 %
Change in valuation allowance16.69 %16.78 %
Income tax provision0.04 %0.06 %
Schedule of Deferred Tax Assets and Liabilities
The significant components of the Company’s deferred tax assets and liabilities as of December 31, 2025 and 2024 are as follows (in thousands):
 20252024
Deferred Tax Assets:
Net operating loss$130,051 $113,303 
Research and development and other tax credits112,093 91,760 
Intangible assets56,052 53,919 
Capitalized research and development27,170 47,880 
Stock based compensation13,971 15,680 
Other accrued expenses21,727 14,266 
Charitable contributions5,699 5,797 
Operating lease liabilities4,164 5,421 
174A state only difference2,397 — 
Depreciation602 435 
Loan costs117 153 
Total deferred tax assets374,043 348,614 
Deferred Tax Liabilities:
Operating lease right of use assets(3,211)(4,259)
Prepaid assets(184)(178)
Total deferred tax liabilities(3,395)(4,437)
Net deferred tax assets before valuation allowance370,648 344,177 
Valuation allowance(370,648)(344,177)
Total deferred tax assets$— $— 
Schedule of Unrecognized Tax Benefits
A reconciliation of the Company's unrecognized tax benefits for the years 2025, 2024 and 2023 is provided in the following table (in thousands):
202520242023
Balance as of January 1:$27,404 $22,906 $11,490 
Increase in current period positions3,343 4,248 4,871 
Increase in prior period positions— 250 7,383 
Decrease in prior period positions(4,716)— (838)
Balance as of December 31:$26,031 $27,404 $22,906 
v3.25.4
PROPERTY AND EQUIPMENT (Tables)
 12 Months Ended
Dec. 31, 2025
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,
20252024
Leasehold improvements$8,992 $9,370 
Furniture and fixtures2,397 2,446 
Computers and equipment2,731 2,275 
14,120 14,091 
Less: Accumulated depreciation(10,098)(8,755)
Total property and equipment, net$4,022 $5,336 
v3.25.4
LEASES (Tables)
 12 Months Ended
Dec. 31, 2025
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, 2025 (in thousands):
December 31, 2025
2026$6,775 
20276,978 
20284,782 
2029 and thereafter— 
Total undiscounted future minimum payments18,535 
Present value discount(1,526)
Total lease liability17,009 
Unamortized lease incentives(2,539)
Cash payments in excess of straight-line lease expense(3,894)
Total ROU asset$10,576 
Schedule of Weighted-average Remaining Lease Term and Discount Rate
The weighted-average remaining lease term and weighted-average discount rate of the Company's operating leases are as follows:
December 31,
20252024
Weighted-average remaining lease term in years2.73.7
Weighted-average discount rate6.48 %6.48 %
v3.25.4
DIVESTITURES (Tables)
 12 Months Ended
Dec. 31, 2025
Discontinued Operations and Disposal Groups [Abstract]  
Schedule of Results, Assets and Liabilities of Discontinued Operations
Results of discontinued operations are as follows (in thousands):
Year Ended December 31,
202520242023
Net product sales$— $(550)$66,164 
Operating expenses:
Cost of goods sold— (9)1,899 
Research and development— 247 6,118 
Selling, general and administrative— 127 19,500 
Change in fair value of contingent consideration— — (473)
Total operating expenses— 365 27,044 
Operating (loss) income— (915)39,120 
Other income, net:
Interest expense— — (191)
Gain on disposal of discontinued operations, net of tax24,715 — 226,005 
Total other income, net24,715 — 225,814 
Net income (loss) from discontinued operations$24,715 $(915)$264,934 
v3.25.4
SEGMENT INFORMATION (Tables)
 12 Months Ended
Dec. 31, 2025
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, 2025, 2024 and 2023 (in thousands):
Year Ended December 31,
202520242023
Revenue$490,728 $233,175 $145,238 
Less:
Cost of goods sold10,339 7,744 11,450 
Research and development:
External research and development107,036 126,303 142,482 
Internal personnel costs80,091 73,843 84,658 
Other research and development18,884 17,350 17,850 
Total research and development206,011 217,496 244,990 
Selling, general and administrative337,202 264,119 265,542 
In-process research and development— 65,205 — 
Restructuring— 2,438 11,394 
Total other income, net13,551 3,317 12,028 
Income tax provision on continuing operations(988)(120)(223)
Income (loss) from discontinued operations, net of tax24,715 (915)264,934 
Net loss$(25,546)$(321,545)$(111,399)
v3.25.4
DESCRIPTION OF BUSINESS (Details) - USD ($)
$ in Thousands
 1 Months Ended 3 Months Ended 12 Months Ended
Aug. 31, 2023
May 31, 2025
Jan. 31, 2024
Sep. 30, 2021
Dec. 31, 2025
Sep. 30, 2025
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]                  
Revenue             $ 490,728 $ 233,175 $ 145,238
Vifor Pharma | Collaborative Arrangement                  
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]                  
Revenue       $ 55,000          
Chugai | Variable Interest Entity, Not Primary Beneficiary                  
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]                  
Variable interest entity, gain from sale         $ 10,200        
Chugai | Collaborative Arrangement                  
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]                  
Revenue     $ 8,300            
Regulatory Milestone | Vifor Pharma | Collaborative Arrangement                  
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]                  
Revenue   $ 17,500         17,500    
Market Access Milestone | Vifor Pharma | Collaborative Arrangement                  
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]                  
Revenue           $ 40,000 40,000    
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                
Proceeds from milestone payment             $ 25,000    
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                
v3.25.4
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, 2025
USD ($)
clinicalTrial
reporting_unit
segment
Dec. 31, 2024
USD ($)
Dec. 31, 2023
USD ($)
New Accounting Pronouncements or Change in Accounting Principle [Line Items]          
Cost of goods sold     $ 10,339,000 $ 7,744,000 $ 11,450,000
Loss on allowance for inventory     $ 684,000 2,819,000 3,039,000
Number of phase 3 clinical trials in process | clinicalTrial     4    
Advertising costs     $ 14,700,000 7,800,000 0
Accounts receivable, allowance for credit loss     0 0  
Impairment of intangible assets, finite-lived     0 0 0
In-process research and development $ 65,200,000   $ 0 65,205,000 0
Equity method investments       0  
Number of segments | segment     1    
Number of reporting units | reporting_unit     1    
Gain (loss) on foreign exchange transactions     $ (300,000) (700,000) 500,000
Restructuring expenses     0 2,438,000 11,394,000
Impairment charges     0 1,200,000 0
Restructuring costs incurred to date       13,800,000  
Equity Method Investments          
New Accounting Pronouncements or Change in Accounting Principle [Line Items]          
In-process research and development     0 $ 3,400,000  
Mission license          
New Accounting Pronouncements or Change in Accounting Principle [Line Items]          
Impairment of intangible assets, finite-lived   $ 3,700,000      
Impairment, Intangible Asset, Finite-Lived, Statement of Income or Comprehensive Income [Extensible Enumeration]   Selling, general and administrative      
Disposed of by Sale | Bile Acid Product Portfolio          
New Accounting Pronouncements or Change in Accounting Principle [Line Items]          
Proceeds from milestone payment     $ 25,000,000.0    
FILSPARI          
New Accounting Pronouncements or Change in Accounting Principle [Line Items]          
Cost of goods sold         3,200,000
Loss on allowance for inventory         2,100,000
Purchase commitment         $ 1,100,000
v3.25.4
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Inventory, Net of Reserves (Details) - USD ($)
$ in Thousands
Dec. 31, 2025
Dec. 31, 2024
Accounting Policies [Abstract]    
Raw materials $ 24,169 $ 30,552
Work in process 2,348 7,625
Finished goods 9,638 3,679
Total inventory 36,155 41,856
Inventory 5,875 6,200
Long-term inventory $ 30,280 $ 35,656
v3.25.4
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Cost of Goods Sold (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
New Accounting Pronouncements or Change in Accounting Principle [Line Items]      
Cost of goods sold $ 10,339 $ 7,744 $ 11,450
Cost of goods sold - product sales      
New Accounting Pronouncements or Change in Accounting Principle [Line Items]      
Cost of goods sold 5,813 7,446 8,406
Cost of goods sold - license and collaboration      
New Accounting Pronouncements or Change in Accounting Principle [Line Items]      
Cost of goods sold $ 4,526 $ 298 $ 3,044
v3.25.4
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Vesting Award Terms (Details)
 12 Months Ended
Dec. 31, 2025
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
v3.25.4
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Major Classifications of Property and Equipment (Details)
Dec. 31, 2025
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
v3.25.4
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, 2025
Dec. 31, 2024
Dec. 31, 2023
Foreign Currency Translation Adjustments included in Accumulated Other Comprehensive Loss      
Beginning balance $ 59,077 $ 200,810 $ 42,851
Ending balance 114,828 59,077 200,810
Accumulated Foreign Currency Adjustment Attributable to Parent      
Foreign Currency Translation Adjustments included in Accumulated Other Comprehensive Loss      
Beginning balance (34) (1,456) 415
Foreign currency translation adjustments (945) 1,422 (1,871)
Ending balance $ (979) $ (34) $ (1,456)
v3.25.4
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Restructuring and Related Costs (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Restructuring Reserve [Roll Forward]      
Liability balance, beginning of period $ 0 $ 11,421  
Restructuring expenses $ 0 2,438 $ 11,394
Non-cash impairment and disposal charges   (856)  
Payments   (12,929)  
Foreign currency impact   (74)  
Liability balance, end of period   $ 0 $ 11,421
v3.25.4
REVENUE RECOGNITION - Narrative (Details)
$ in Millions
 12 Months Ended
Dec. 31, 2025
USD ($)
pharmacy
Dec. 31, 2024
USD ($)
Dec. 31, 2023
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 | $ $ 1.1 $ 0.5 $ 0.4
United States | Revenue Benchmark | Geographic Concentration Risk      
Disaggregation of Revenue [Line Items]      
Concentration risk, percentage 98.00%    
v3.25.4
REVENUE RECOGNITION - Net Product Sales (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Disaggregation of Revenue [Line Items]      
Total net product sales $ 490,728 $ 233,175 $ 145,238
Net product sales      
Disaggregation of Revenue [Line Items]      
Total net product sales 410,460 226,707 127,537
FILSPARI      
Disaggregation of Revenue [Line Items]      
Total net product sales 322,005 132,222 29,208
Tiopronin products      
Disaggregation of Revenue [Line Items]      
Total net product sales $ 88,455 $ 94,485 $ 98,329
v3.25.4
COLLABORATION AND LICENSE AGREEMENTS - Narrative (Details)
 1 Months Ended 3 Months Ended 4 Months Ended 12 Months Ended
May 31, 2025
USD ($)
Jan. 31, 2024
USD ($)
performance_obligation
Sep. 30, 2021
USD ($)
performance_obligation
Sep. 30, 2025
USD ($)
Dec. 31, 2021
USD ($)
Dec. 31, 2025
USD ($)
Dec. 31, 2024
USD ($)
Dec. 31, 2023
USD ($)
Dec. 31, 2022
USD ($)
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]                  
Revenue           $ 490,728,000 $ 233,175,000 $ 145,238,000  
Deferred revenue           0 2,815,000 8,931,000 $ 22,907,000
License and collaboration revenue                  
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]                  
Revenue           80,268,000 6,468,000 17,701,000  
Vifor Pharma | Collaborative Arrangement                  
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]                  
Upfront payment     $ 55,000,000.0            
Milestone payments (up to)     $ 845,000,000.0            
Percentage of royalty on net sales receives     40.00%            
Force majeure event period threshold     6 months            
Revenue     $ 55,000,000.0            
Number of performance obligations | performance_obligation     2            
Deferred revenue     $ 43,000,000.0     0 2,800,000    
Vifor Pharma | Collaborative Arrangement | Regulatory and Market Access Milestone                  
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]                  
Milestone payments (up to)     135,000,000.0            
Vifor Pharma | Collaborative Arrangement | Sales-based Milestone Payments                  
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]                  
Milestone payments (up to)     $ 655,000,000.0            
Vifor Pharma | Collaborative Arrangement | License and collaboration revenue                  
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]                  
Revenue         $ 12,000,000.0 71,000,000.0 6,500,000 17,700,000  
Vifor Pharma | Collaborative Arrangement | Market Access Milestone                  
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]                  
Revenue       $ 40,000,000.0   40,000,000.0      
Vifor Pharma | Collaborative Arrangement | Regulatory Milestone                  
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]                  
Revenue $ 17,500,000         17,500,000      
Vifor Pharma | Collaborative Arrangement | Royalty                  
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]                  
Revenue           5,900,000 $ 600,000 $ 0  
Chugai | Collaborative Arrangement                  
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]                  
Force majeure event period threshold   9 months              
Revenue   $ 8,300,000              
Number of performance obligations | performance_obligation   1              
Chugai | Collaborative Arrangement | License and collaboration revenue                  
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]                  
Revenue           $ 9,300,000      
Chugai | Collaborative Arrangement | Regulatory, Development And Sales-based Milestone                  
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]                  
Milestone payments (up to)   $ 120,000,000.0              
v3.25.4
COLLABORATION AND LICENSE AGREEMENTS - Changes in Deferred Revenue (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Contract with Customer, Liability [Roll Forward]      
Beginning balance $ 2,815 $ 8,931 $ 22,907
Foreign currency impact 57 (269) 387
Ending balance 0 2,815 8,931
License and collaboration revenue      
Contract with Customer, Liability [Roll Forward]      
License and collaboration revenue $ (2,872) $ (5,847) $ (14,363)
v3.25.4
VARIABLE INTEREST ENTITIES (Details) - USD ($)
 1 Months Ended 3 Months Ended 12 Months Ended
Jan. 31, 2024
Mar. 31, 2022
Dec. 31, 2025
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Other current liabilities     $ 3,194,000 $ 3,194,000 $ 17,106,000  
Revenue       490,728,000 233,175,000 $ 145,238,000
License and collaboration revenue            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Revenue       80,268,000 6,468,000 $ 17,701,000
Variable Interest Entity, Not Primary Beneficiary            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Other current liabilities     0 0 $ 8,900,000  
PharmaKrysto, LTD | Collaborative Arrangement            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Collaborative arrangement, option to purchase remaining shares of VIE, amount   $ 5,000,000.0        
Chugai | Variable Interest Entity, Not Primary Beneficiary            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Variable interest entity, gain from sale     $ 10,200,000      
Chugai | Collaborative Arrangement            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Revenue $ 8,300,000          
Chugai | Collaborative Arrangement | License and collaboration revenue            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Revenue       $ 9,300,000    
PharmaKrysto, LTD            
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]            
Percentage ownership purchased   5.00%        
v3.25.4
MARKETABLE DEBT SECURITIES - Marketable Debt Securities (Details) - USD ($)
$ in Thousands
Dec. 31, 2025
Dec. 31, 2024
Debt Securities, Available-for-sale [Line Items]    
Total available-for-sale marketable debt securities $ 229,761 $ 312,166
Commercial paper    
Debt Securities, Available-for-sale [Line Items]    
Total available-for-sale marketable debt securities 62,988 73,325
Corporate debt securities    
Debt Securities, Available-for-sale [Line Items]    
Total available-for-sale marketable debt securities 151,777 203,816
Securities of government-sponsored entities    
Debt Securities, Available-for-sale [Line Items]    
Total available-for-sale marketable debt securities 9,993 35,025
Municipal bonds    
Debt Securities, Available-for-sale [Line Items]    
Total available-for-sale marketable debt securities $ 5,003 $ 0
v3.25.4
MARKETABLE DEBT SECURITIES - Short-term Marketable Debt Securities (Details) - USD ($)
$ in Thousands
Dec. 31, 2025
Dec. 31, 2024
Debt Securities, Available-for-sale [Line Items]    
Marketable debt securities, available-for-sale, amortized cost, current $ 145,388  
Marketable debt securities, available-for-sale, unrealized gains, current 35  
Marketable debt securities, available-for-sale, unrealized losses, current (39)  
Marketable debt securities, available-for-sale, current 145,384  
Marketable debt securities, available-for-sale, amortized cost, noncurrent 84,389  
Marketable debt securities, available-for-sale, unrealized gains, noncurrent 69  
Marketable debt securities, available-for-sale, unrealized losses, noncurrent (81)  
Marketable debt securities, available-for-sale, noncurrent 84,377  
Marketable debt securities, available-for-sale, amortized cost 229,777 $ 311,798
Marketable debt securities, available-for-sale, unrealized gains 104 517
Marketable debt securities, available-for-sale, unrealized losses (120) (149)
Total available-for-sale marketable debt securities 229,761 312,166
Commercial paper    
Debt Securities, Available-for-sale [Line Items]    
Marketable debt securities, available-for-sale, amortized cost, current 62,974 73,410
Marketable debt securities, available-for-sale, unrealized gains, current 20 1
Marketable debt securities, available-for-sale, unrealized losses, current (6) (86)
Marketable debt securities, available-for-sale, current 62,988 73,325
Total available-for-sale marketable debt securities 62,988 73,325
Corporate debt securities    
Debt Securities, Available-for-sale [Line Items]    
Marketable debt securities, available-for-sale, amortized cost, current 77,414 203,395
Marketable debt securities, available-for-sale, unrealized gains, current 12 483
Marketable debt securities, available-for-sale, unrealized losses, current (33) (62)
Marketable debt securities, available-for-sale, current 77,393 203,816
Marketable debt securities, available-for-sale, amortized cost, noncurrent 74,389  
Marketable debt securities, available-for-sale, unrealized gains, noncurrent 69  
Marketable debt securities, available-for-sale, unrealized losses, noncurrent (74)  
Marketable debt securities, available-for-sale, noncurrent 74,384  
Total available-for-sale marketable debt securities 151,777 203,816
Municipal bonds    
Debt Securities, Available-for-sale [Line Items]    
Marketable debt securities, available-for-sale, amortized cost, current 5,000  
Marketable debt securities, available-for-sale, unrealized gains, current 3  
Marketable debt securities, available-for-sale, unrealized losses, current 0  
Marketable debt securities, available-for-sale, current 5,003  
Total available-for-sale marketable debt securities 5,003 0
Securities of government-sponsored entities    
Debt Securities, Available-for-sale [Line Items]    
Marketable debt securities, available-for-sale, amortized cost, current   34,993
Marketable debt securities, available-for-sale, unrealized gains, current   33
Marketable debt securities, available-for-sale, unrealized losses, current   (1)
Marketable debt securities, available-for-sale, current   35,025
Marketable debt securities, available-for-sale, amortized cost, noncurrent 10,000  
Marketable debt securities, available-for-sale, unrealized gains, noncurrent 0  
Marketable debt securities, available-for-sale, unrealized losses, noncurrent (7)  
Marketable debt securities, available-for-sale, noncurrent 9,993  
Total available-for-sale marketable debt securities $ 9,993 $ 35,025
v3.25.4
MARKETABLE DEBT SECURITIES - Narrative (Details) - USD ($)
$ in Millions
Dec. 31, 2025
Dec. 31, 2024
Investments, Debt and Equity Securities [Abstract]    
Accrued interest receivable $ 1.3 $ 2.3
Available-for-sale marketable debt securities in an unrealized loss position $ 140.9 $ 123.7
v3.25.4
MARKETABLE DEBT SECURITIES - Debt Securities in Unrealized Loss Position (Details) - USD ($)
$ in Thousands
Dec. 31, 2025
Dec. 31, 2024
Debt Securities, Available-for-sale [Line Items]    
Less than 12 months, fair value $ 140,753 $ 108,558
Less than 12 months, unrealized losses 120 142
12 months or greater, fair value 0 14,944
12 months or greater, unrealized losses 0 7
Total, fair value 140,753 123,502
Total, unrealized losses 120 149
Commercial paper    
Debt Securities, Available-for-sale [Line Items]    
Less than 12 months, fair value 12,317 68,446
Less than 12 months, unrealized losses 6 86
12 months or greater, fair value 0 0
12 months or greater, unrealized losses 0 0
Total, fair value 12,317 68,446
Total, unrealized losses 6 86
Corporate debt securities    
Debt Securities, Available-for-sale [Line Items]    
Less than 12 months, fair value 118,443 40,112
Less than 12 months, unrealized losses 107 56
12 months or greater, fair value 0 9,969
12 months or greater, unrealized losses 0 6
Total, fair value 118,443 50,081
Total, unrealized losses 107 62
Securities of government-sponsored entities    
Debt Securities, Available-for-sale [Line Items]    
Less than 12 months, fair value 9,993 0
Less than 12 months, unrealized losses 7 0
12 months or greater, fair value 0 4,975
12 months or greater, unrealized losses 0 1
Total, fair value 9,993 4,975
Total, unrealized losses $ 7 $ 1
v3.25.4
CONVERTIBLE DEBT - Convertible Senior Notes (Details) - USD ($)
$ in Thousands
Dec. 31, 2025
Dec. 31, 2024
Mar. 11, 2022
Sep. 10, 2018
Classified as:        
Convertible debt, current portion $ 0 $ 68,678    
Convertible debt, less current portion 311,724 310,310    
Total convertible debt 311,724 378,988    
Senior Notes        
Debt Instrument [Line Items]        
Total convertible senior notes, net of unamortized debt discount and debt issuance costs 311,724 378,988    
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 (4,526) (5,940)    
Senior Notes Due 2025 | Senior Notes        
Debt Instrument [Line Items]        
Interest rate percentage       2.50%
Convertible senior notes 0 68,904    
Unamortized debt issuance costs $ 0 $ (226)    
v3.25.4
CONVERTIBLE DEBT - Narrative (Details)
$ / shares in Units, $ in Thousands
 12 Months Ended
Sep. 15, 2025
USD ($)
Mar. 11, 2022
USD ($)
Sep. 10, 2018
USD ($)
Dec. 31, 2025
USD ($)
day
$ / shares
Dec. 31, 2024
USD ($)
Dec. 31, 2023
USD ($)
Debt Instrument [Line Items]            
Debt instrument, repurchase amount Including accrued and unpaid interest   $ 213,800        
Interest expense       $ 10,748 $ 11,182 $ 11,334
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 $ 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      
Debt instrument, repurchase amount   207,100        
Long-term debt, excluding current maturities, repaid if converted   $ 68,900        
Repayments of debt $ 68,900          
v3.25.4
CONVERTIBLE DEBT - Interest Expense Recognized (Details) - Senior Notes Due 2025 and Senior Notes Due 2029 - Senior Notes - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Debt Instrument [Line Items]      
Contractual interest expense $ 8,336 $ 8,838 $ 8,838
Amortization of debt issuance costs 1,640 1,725 1,718
Total interest expense for the 2025 and 2029 Notes $ 9,976 $ 10,563 $ 10,556
v3.25.4
FAIR VALUE MEASUREMENTS - Narrative (Details)
 12 Months Ended
Dec. 31, 2025
USD ($)
Dec. 31, 2024
USD ($)
Dec. 31, 2023
USD ($)
Mar. 11, 2022
Sep. 10, 2018
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]          
Impairment charges $ 0 $ 1,200,000 $ 0    
Measurement Input, Discount Rate          
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]          
Long-lived asset, measurement input 0.069        
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 $ 448,300,000 302,100,000      
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      
v3.25.4
FAIR VALUE MEASUREMENTS - Fair Value on a Recurring Basis (Details) - USD ($)
$ in Thousands
Dec. 31, 2025
Dec. 31, 2024
Assets:    
Marketable debt securities, available-for-sale $ 229,761 $ 312,166
Fair Value, Recurring    
Assets:    
Cash and Cash Equivalents 93,035 58,535
Marketable debt securities, available-for-sale 229,761 312,166
Total 322,796 370,701
Quoted prices in active markets (Level 1) | Fair Value, Recurring    
Assets:    
Cash and Cash Equivalents 93,035 51,060
Marketable debt securities, available-for-sale 0 0
Total 93,035 51,060
Significant other observable inputs (Level 2) | Fair Value, Recurring    
Assets:    
Cash and Cash Equivalents 0 7,475
Marketable debt securities, available-for-sale 229,761 312,166
Total 229,761 319,641
Significant unobservable inputs (Level 3) | Fair Value, Recurring    
Assets:    
Cash and Cash Equivalents 0 0
Marketable debt securities, available-for-sale 0 0
Total $ 0 $ 0
v3.25.4
INTANGIBLE ASSETS - Narrative (Details) - USD ($)
 12 Months Ended 168 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2025
Dec. 31, 2014
Finite-Lived Intangible Assets [Line Items]          
Payments to date under terms of licensing agreement $ 58,157,000 $ 36,212,000 $ 41,591,000    
Gross carrying amount 293,467,000 224,293,000   $ 293,467,000  
Impairment of intangible assets, finite-lived 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       $ 47,200,000  
Useful life 11 years 11 years   11 years  
Gross carrying amount $ 108,163,000 $ 53,268,000   $ 108,163,000  
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 $ 54,300,000 $ 20,300,000      
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 2,100,000     2,100,000  
Accrued royalties 6,600,000 $ 8,200,000   6,600,000  
Gross carrying amount 185,304,000 171,025,000   185,304,000  
Increase in intangible assets 14,300,000        
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 $ 4,500,000 $ 6,100,000   $ 4,500,000  
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%        
v3.25.4
INTANGIBLE ASSETS - Amortizable Intangible Assets (Details) - USD ($)
$ in Thousands
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2014
Finite-Lived Intangible Assets [Line Items]      
Gross Carrying Amount $ 293,467 $ 224,293  
Accumulated Amortization (180,352) (121,072)  
Net Book Value $ 113,115 $ 103,221  
Ligand license      
Finite-Lived Intangible Assets [Line Items]      
Useful Life 11 years 11 years  
Gross Carrying Amount $ 108,163 $ 53,268  
Accumulated Amortization (13,645) (5,769)  
Net Book Value $ 94,518 $ 47,499  
Mission license      
Finite-Lived Intangible Assets [Line Items]      
Useful Life 12 years 12 years 10 years
Gross Carrying Amount $ 185,304 $ 171,025  
Accumulated Amortization (166,707) (115,303)  
Net Book Value $ 18,597 $ 55,722  
v3.25.4
INTANGIBLE ASSETS - Amortization Expense (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Finite-Lived Intangible Assets [Line Items]      
Total amortization expense $ 59,210 $ 41,739 $ 36,282
Selling, general and administrative      
Finite-Lived Intangible Assets [Line Items]      
Total amortization expense 59,210 41,739 29,021
Research and development      
Finite-Lived Intangible Assets [Line Items]      
Total amortization expense $ 0 $ 0 $ 7,261
v3.25.4
INTANGIBLE ASSETS - Amortization Expense for Next Five Years (Details) - USD ($)
$ in Thousands
Dec. 31, 2025
Dec. 31, 2024
Goodwill and Intangible Assets Disclosure [Abstract]    
2026 $ 31,479  
2027 12,888  
2028 12,922  
2029 12,888  
2030 12,888  
Thereafter 30,050  
Net Book Value $ 113,115 $ 103,221
v3.25.4
ACCRUED EXPENSES (Details) - USD ($)
$ in Thousands
Dec. 31, 2025
Dec. 31, 2024
Payables and Accruals [Abstract]    
Compensation related costs $ 41,515 $ 35,166
Sales discounts, rebates, and allowances 30,595 10,585
Accrued royalties 22,703 12,309
Research and development 16,027 16,090
Selling, general and administrative 9,818 6,154
Miscellaneous accrued expenses 5,377 5,724
Total accrued expenses $ 126,035 $ 86,028
v3.25.4
COMMITMENTS AND CONTINGENCIES (Details) - USD ($)
$ in Thousands
 1 Months Ended 12 Months Ended
Mar. 31, 2024
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Nov. 30, 2020
Business Combination [Line Items]          
In-process research and development $ 65,200 $ 0 $ 65,205 $ 0  
Orphan Technologies Limited          
Business Combination [Line Items]          
Contingent consideration, liability         $ 427,000
v3.25.4
STOCKHOLDERS’ EQUITY - Common Stock and Preferred Stock (Details)
 12 Months Ended
Dec. 31, 2025
vote
$ / shares
shares
Dec. 31, 2024
$ / 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  
v3.25.4
STOCKHOLDERS’ EQUITY - 2018 Equity Incentive Plan (Details)
 12 Months Ended
Dec. 31, 2025
shares
2018 Equity Incentive Plan and Prior Plans  
Class of Stock [Line Items]  
Shares remaining available for issuance under the plan (in shares) 22,384,114
2018 Equity Incentive Plan  
Class of Stock [Line Items]  
Shares remaining available for issuance under the plan (in shares) 7,135,921
Expiration Term 10 years
Vesting Term 4 years
v3.25.4
STOCKHOLDERS’ EQUITY - 2017 Employee Stock Purchase Plan (Details) - 2017 ESPP - USD ($)
 12 Months Ended
Dec. 31, 2025
Jan. 01, 2018
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]    
Shares remaining available for issuance under the plan (in shares) 2,780,000 380,000
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) 1,089,373  
v3.25.4
STOCKHOLDERS’ EQUITY - Assumptions used in Black-Scholes Options Pricing Model (Details) - Stock Options
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]      
Risk free rate 4.40% 3.90% 3.90%
Expected volatility 62.00% 59.00% 50.00%
Expected life (in years) 6 years 4 months 24 days 6 years 4 months 24 days 6 years 4 months 24 days
Expected dividend yield 0.00% 0.00% 0.00%
v3.25.4
STOCKHOLDERS’ EQUITY - Share-based Compensation, Stock Options, Activity (Details) - Stock Options - USD ($)
$ / shares in Units, $ in Thousands
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Shares Underlying Options      
Outstanding, beginning balance (in shares) 9,283,331    
Granted (in shares) 1,484,200    
Exercised (in shares) (1,869,242)    
Forfeited and expired (in shares) (656,860)    
Outstanding, ending balance (in shares) 8,241,429 9,283,331  
Vested and expected to vest (in shares) 8,241,429    
Exercisable (in shares) 5,813,961    
Weighted Average, Exercise Price      
Outstanding, beginning balance (in dollars per share) $ 20.13    
Granted (in dollars per share) 20.65    
Exercised (in dollars per share) 18.63    
Forfeited and expired (in dollars per share) 28.29    
Outstanding, ending balance (in dollars per share) 19.91 $ 20.13  
Vested and expected to vest (in dollars per share) 19.91    
Exercisable (in dollars per share) $ 20.83    
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Additional Disclosures [Abstract]      
Weighted average, remaining contractual life (in years) 5 years 10 months 20 days 5 years 6 months 3 days  
Vested and expected to vest (in years) 5 years 10 months 20 days    
Exercisable (in years) 4 years 9 months 7 days    
Aggregate intrinsic value, beginning balance $ 16,408    
Exercised, aggregate intrinsic value 23,247 $ 2,000 $ 1,400
Aggregate intrinsic value, ending balance 150,796 $ 16,408  
Vested and expected to vest, aggregate intrinsic value 150,796    
Exercisable, aggregate intrinsic value $ 101,064    
Weighted average grant date fair value of options (in dollars per share) $ 12.86 $ 5.10 $ 11.66
Closing stock price of stock options outstanding and exercisable (in dollars per share) $ 38.21 $ 17.42 $ 8.99
Compensation expense not yet recognized, stock options $ 20,700    
Weighted average remaining vesting period (in years) 2 years 4 months 24 days    
Chief Financial Officer      
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Additional Disclosures [Abstract]      
Modification expense     $ 2,600
v3.25.4
STOCKHOLDERS’ EQUITY - Restricted Stock Unit Activity (Details) - Restricted Stock Units - USD ($)
$ / shares in Units, $ in Millions
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]      
Unrecognized compensation cost related to RSU's granted $ 58.5    
Weighted average period for unrecognized costs (in years) 2 years 6 months    
Number of RSUs      
Unvested beginning balance (in shares) 3,517,263    
Granted (in shares) 2,662,490    
Vested (in shares) (1,314,671)    
Forfeited/cancelled (in shares) (297,715)    
Unvested ending balance (in shares) 4,567,367 3,517,263  
Weighted Average Grant Date Fair Value      
Unvested beginning balance (in dollars per share) $ 15.41    
Granted (in dollars per share) 20.68 $ 9.07 $ 21.20
Vested (in dollars per share) 16.72    
Forfeited/cancelled (in dollars per share) 16.69    
Unvested ending balance (in dollars per share) $ 18.02 $ 15.41  
Fair value of restricted stock units vested $ 22.0 $ 22.8 $ 18.7
v3.25.4
STOCKHOLDERS’ EQUITY - Performance-based Stock Unit Activity (Details) - Performance Shares - USD ($)
$ / shares in Units, $ in Millions
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]      
Awards vested, minimum percentage of target 0.00%    
Awards vested, maximum percentage of target 200.00%    
Unrecognized compensation cost related to PSU's granted $ 0.1    
Weighted average period for unrecognized costs (in years) 4 months 24 days    
Number of PSUs      
Unvested beginning balance (in shares) 216,208    
Granted (in shares) 88,000    
Vested (in shares) (70,604)    
Forfeited/cancelled (in shares) 0    
Unvested ending balance (in shares) 233,604 216,208  
Weighted Average Grant Date Fair Value      
Unvested beginning balance (in dollars per share) $ 18.34    
Granted (in dollars per share) 20.46 $ 8.93 $ 22.40
Vested (in dollars per share) 27.54    
Forfeited/cancelled (in dollars per share) 0    
Unvested ending balance (in dollars per share) $ 16.35 $ 18.34  
Fair value of performance-based stock units vested $ 1.9 $ 1.5 $ 0.3
v3.25.4
STOCKHOLDERS’ EQUITY - Non-cash Stock-based Compensation Expense (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]      
Total stock-based compensation expense $ 44,868 $ 36,913 $ 44,246
Selling, general and administrative expenses      
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]      
Total stock-based compensation expense 28,187 22,735 27,111
Research and development expenses      
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]      
Total stock-based compensation expense $ 16,681 $ 14,178 $ 17,135
v3.25.4
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
v3.25.4
NET LOSS PER COMMON SHARE - Basic and Diluted Net Loss Per Share (Details) - USD ($)
$ / shares in Units, $ in Thousands
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Earnings Per Share [Abstract]      
Continuing operations, basic (in shares) 89,211,813 78,888,861 74,267,418
Continuing operations, diluted (in shares) 89,211,813 78,888,861 74,267,418
Discontinued operations, basic (in shares) 89,211,813 78,888,861 74,267,418
Discontinued Operations, diluted (in shares) 89,211,813 78,888,861 74,267,418
Weighted average common shares outstanding, basic (in shares) 89,211,813 78,888,861 74,267,418
Weighted average common shares outstanding, diluted (in shares) 89,211,813 78,888,861 74,267,418
Net (loss) income from continuing operations $ (50,261) $ (320,630) $ (376,333)
Net (loss) income from discontinued operations 24,715 (915) 264,934
Net (loss) income, basic (25,546) (321,545) (111,399)
Net (loss) income, diluted $ (25,546) $ (321,545) $ (111,399)
Net loss from continuing operations, basic (in dollars per share) $ (0.57) $ (4.07) $ (5.07)
Net loss from continuing operations, diluted (in dollars per share) (0.57) (4.07) (5.07)
Net income (loss) from discontinued operations, basic (in dollars per share) 0.28 (0.01) 3.57
Net income (loss) from discontinued operations, diluted (in dollars per share) 0.28 (0.01) 3.57
Net (loss) income per common share, basic (in dollars per share) (0.29) (4.08) (1.50)
Net (loss) income per common share, diluted (in dollars per share) $ (0.29) $ (4.08) $ (1.50)
v3.25.4
NET LOSS PER COMMON SHARE - Anti-dilutive Securities (Details) - shares
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]      
Total anti-dilutive shares (in shares) 25,275,233 25,925,935 25,670,747
Convertible debt      
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]      
Total anti-dilutive shares (in shares) 11,172,474 11,697,953 11,697,952
Options      
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]      
Total anti-dilutive shares (in shares) 9,665,404 10,447,541 10,555,550
Restricted stock units and performance-based stock units      
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]      
Total anti-dilutive shares (in shares) 4,437,355 3,780,441 3,417,245
v3.25.4
INCOME TAXES - Net Loss from Continuing Operations Before Income Taxes (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Operating Loss Carryforwards [Line Items]      
Total $ (49,273) $ (320,510) $ (376,110)
United States      
Operating Loss Carryforwards [Line Items]      
Total (2,713) (152,356) (293,283)
Foreign      
Operating Loss Carryforwards [Line Items]      
Total $ (46,560) $ (168,154) $ (82,827)
v3.25.4
INCOME TAXES - Components of Provision (Benefit) for Income Taxes (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Current      
Federal $ 0 $ 0 $ 0
State 988 120 223
Foreign 0 0 0
Total current 988 120 223
Deferred      
Federal 0 0 0
State 0 0 0
Total deferred 0 0 0
Effective tax rate $ 988 $ 120 $ 223
v3.25.4
INCOME TAXES - Reconciliation of Statutory Federal Income Tax Rate 2025 (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Amount      
Statutory rate - federal $ (10,347)    
State taxes, net of federal benefit* 701    
Statutory tax rate differences 5,831    
Orphan drug credits (16,224)    
Return to provision adjustments (2,517)    
Executive compensation 1,710    
Share-based awards 1,246    
Other 442    
Change in unrecognized tax benefits (1,456)    
Effective tax rate $ 988 $ 120 $ 223
Percentage      
Statutory rate - federal (21.00%) (21.00%) (21.00%)
State taxes, net of federal benefit 1.42% (1.55%) (3.19%)
Statutory tax rate differences 11.83% 2.15% 1.29%
Changes in valuation allowances   16.69% 16.78%
Other   (0.80%) 4.19%
Orphan drug credits (32.93%)    
Return to provision adjustments (5.10%)    
Nondeductible executive compensation 3.47% 0.44% 1.50%
Share-based awards 2.53%    
Other 0.90%    
Change in unrecognized tax benefits (2.96%)    
Effective tax rate 2.00% 0.04% 0.06%
Switzerland      
Amount      
Changes in valuation allowances $ 3,758    
Other $ 207    
Percentage      
Changes in valuation allowances 7.63%    
Other 0.42%    
Other foreign jurisdictions      
Amount      
Other $ (18)    
Percentage      
Other (0.04%)    
United States      
Amount      
Changes in valuation allowances $ 16,275    
Other $ 1,380    
Percentage      
Changes in valuation allowances 33.03%    
Other 2.80%    
v3.25.4
INCOME TAXES - Reconciliation of Statutory Federal Income Tax Rate 2023 , 2024 (Details)
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Income Tax Disclosure [Abstract]      
Statutory rate - federal (21.00%) (21.00%) (21.00%)
State taxes, net of federal benefit 1.42% (1.55%) (3.19%)
Foreign rate differential 11.83% 2.15% 1.29%
IPR&D   4.27% 0.00%
Nondeductible executive compensation 3.47% 0.44% 1.50%
Excess tax benefits associated with share-based awards   2.92% 0.68%
Other permanent differences   0.23% 0.37%
Tax credits   (4.24%) (1.13%)
Return to provision adjustments and other true-ups   (0.80%) 4.19%
Other   0.93% 0.57%
Changes in valuation allowances   16.69% 16.78%
Effective tax rate 2.00% 0.04% 0.06%
v3.25.4
INCOME TAXES - Deferred Tax Assets and Liabilities (Details) - USD ($)
$ in Thousands
Dec. 31, 2025
Dec. 31, 2024
Deferred Tax Assets:    
Net operating loss $ 130,051 $ 113,303
Research and development and other tax credits 112,093 91,760
Intangible assets 56,052 53,919
Capitalized research and development 27,170 47,880
Stock based compensation 13,971 15,680
Other accrued expenses 21,727 14,266
Charitable contributions 5,699 5,797
Operating lease liabilities 4,164 5,421
174A state only difference 2,397 0
Depreciation 602 435
Loan costs 117 153
Total deferred tax assets 374,043 348,614
Deferred Tax Liabilities:    
Operating lease right of use assets (3,211) (4,259)
Prepaid assets (184) (178)
Total deferred tax liabilities (3,395) (4,437)
Net deferred tax assets before valuation allowance 370,648 344,177
Valuation allowance (370,648) (344,177)
Total deferred tax assets $ 0 $ 0
v3.25.4
INCOME TAXES - Narrative (Details) - USD ($)
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Operating Loss Carryforwards [Line Items]        
Valuation allowance $ 370,648,000 $ 344,177,000    
Increase (decrease) in valuation allowance 26,500,000 53,500,000    
Unrecognized tax benefits 26,031,000 27,404,000 $ 22,906,000 $ 11,490,000
Unrecognized tax benefits that would impact effective tax rate 26,000,000.0      
Income tax examination, penalties and interest expense 0 $ 0 $ 0  
Unrecognized Tax Benefits        
Operating Loss Carryforwards [Line Items]        
Increase (decrease) in valuation allowance (26,000,000.0)      
Federal Orphan Drug Tax Credit        
Operating Loss Carryforwards [Line Items]        
Tax credit carryforward 122,800,000      
Tax Competes Tax Credit Carryforward        
Operating Loss Carryforwards [Line Items]        
Tax credit carryforward 800,000      
United States        
Operating Loss Carryforwards [Line Items]        
Available unused NOL carryforwards 232,600,000      
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 214,000,000.0      
Operating loss carryforwards, indefinite carryforward period 41,100,000      
State and Local Jurisdiction | Research Tax Credit Carryforward        
Operating Loss Carryforwards [Line Items]        
Tax credit carryforward, finite carryforward period, amount 1,300,000      
Tax credit carryforward, indefinite carryforward period, amount 11,000,000.0      
Foreign | Irish        
Operating Loss Carryforwards [Line Items]        
Available unused NOL carryforwards 15,900,000      
Foreign | Swiss        
Operating Loss Carryforwards [Line Items]        
Available unused NOL carryforwards 475,200,000      
Tax credit carryforward $ 526,200,000      
v3.25.4
INCOME TAXES - Unrecognized Tax Benefits (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Reconciliation of Unrecognized Tax Benefits, Excluding Amounts Pertaining to Examined Tax Returns [Roll Forward]      
Beginning balance $ 27,404 $ 22,906 $ 11,490
Increase in current period positions 3,343 4,248 4,871
Increase in prior period positions 0 250 7,383
Decrease in prior period positions (4,716) 0 (838)
Ending balance $ 26,031 $ 27,404 $ 22,906
v3.25.4
EQUITY OFFERINGS (Details) - USD ($)
$ / shares in Units, $ in Thousands
 1 Months Ended 3 Months Ended 12 Months Ended 15 Months Ended
Nov. 30, 2024
Feb. 28, 2023
Sep. 30, 2024
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2025
Oct. 31, 2024
Class of Stock [Line Items]                
Proceeds from the issuance of common stock, net of issuance costs       $ 0 $ 134,738 $ 191,198    
Underwritten Equity Offering                
Class of Stock [Line Items]                
Shares sold (in shares) 9,000,000.0 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
v3.25.4
RETIREMENT PLAN (Details) - USD ($)
$ in Millions
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Defined Benefit Plan, Plan Assets, Category [Line Items]      
Defined contribution plan, employer contributions $ 2.8 $ 2.4 $ 2.7
Switzerland Defined Benefit Plan      
Defined Benefit Plan, Plan Assets, Category [Line Items]      
Benefit obligation 1.7    
Plan assets $ 1.0    
v3.25.4
PROPERTY AND EQUIPMENT (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Property, Plant and Equipment [Line Items]      
Property, plant and equipment, gross $ 14,120 $ 14,091  
Less: Accumulated depreciation (10,098) (8,755)  
Total property and equipment, net 4,022 5,336  
Depreciation expense 1,500 1,800 $ 2,200
Leasehold improvements      
Property, Plant and Equipment [Line Items]      
Property, plant and equipment, gross 8,992 9,370  
Furniture and fixtures      
Property, Plant and Equipment [Line Items]      
Property, plant and equipment, gross 2,397 2,446  
Computers and equipment      
Property, Plant and Equipment [Line Items]      
Property, plant and equipment, gross $ 2,731 $ 2,275  
v3.25.4
LEASES - Narrative (Details) - USD ($)
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
May 31, 2020
Lessee, Lease, Description [Line Items]        
Operating lease right-of-use assets $ 10,576,000 $ 14,295,000    
Total lease liability 17,009,000      
Impairment charges 0 1,200,000 $ 0  
Operating lease expense $ 4,400,000 $ 4,800,000 $ 4,900,000  
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      
v3.25.4
LEASES - Future Minimum Rental Commitments (Details) - USD ($)
$ in Thousands
Dec. 31, 2025
Dec. 31, 2024
Leases [Abstract]    
2026 $ 6,775  
2027 6,978  
2028 4,782  
2029 and thereafter 0  
Total undiscounted future minimum payments 18,535  
Present value discount (1,526)  
Total lease liability 17,009  
Unamortized lease incentives (2,539)  
Cash payments in excess of straight-line lease expense (3,894)  
Operating lease right-of-use assets $ 10,576 $ 14,295
v3.25.4
LEASES - Weighted-average Remaining Lease Term and Discount Rate (Details)
Dec. 31, 2025
Dec. 31, 2024
Leases [Abstract]    
Weighted-average remaining lease term in years 2 years 8 months 12 days 3 years 8 months 12 days
Weighted-average discount rate 6.48% 6.48%
v3.25.4
DIVESTITURES - Additional Information (Details) - Disposed of by Sale - Bile Acid Product Portfolio - USD ($)
$ in Thousands
 12 Months Ended
Aug. 31, 2023
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
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      
Proceeds from milestone payment   $ 25,000    
Gain on disposal of discontinued operations, net of tax   $ 24,715 $ 0 $ 226,005
Discontinued operation after disposal, expense     $ 500  
Minimum        
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]        
Milestone thresholds 125,000      
Maximum        
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]        
Milestone thresholds $ 500,000      
v3.25.4
DIVESTITURES - Results of Discontinued Operations (Details) - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Other income, net:      
Income (loss) from discontinued operations, net of tax $ 24,715 $ (915) $ 264,934
Disposed of by Sale | Bile Acid Product Portfolio      
Operating expenses:      
Cost of goods sold 0 (9)  
Cost of goods sold     1,899
Research and development 0 247 6,118
Selling, general and administrative 0 127 19,500
Change in fair value of contingent consideration 0 0 (473)
Total operating expenses 0 365 27,044
Operating (loss) income 0 (915) 39,120
Other income, net:      
Interest expense 0 0 (191)
Gain on disposal of discontinued operations, net of tax 24,715 0 226,005
Total other income, net 24,715 0 225,814
Income (loss) from discontinued operations, net of tax $ 24,715 $ (915) $ 264,934
Discontinued Operation, Gain (Loss) on Disposal, Statement of Income or Comprehensive Income [Extensible Enumeration] Income (loss) from discontinued operations, net of tax Income (loss) from discontinued operations, net of tax Income (loss) from discontinued operations, net of tax
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]      
Net product sales $ 0 $ (550) $ 66,164
v3.25.4
SEGMENT INFORMATION - Narrative (Details)
 12 Months Ended
Dec. 31, 2025
segment
Segment Reporting [Abstract]  
Number of segments 1
Number of reportable segments 1
v3.25.4
SEGMENT INFORMATION - Reportable Segment Loss, Including Significant Expenses (Details) - USD ($)
$ in Thousands
 1 Months Ended 12 Months Ended
Mar. 31, 2024
Dec. 31, 2025
Dec. 31, 2024
Dec. 31, 2023
Segment Reporting Information [Line Items]        
Revenue   $ 490,728 $ 233,175 $ 145,238
Less:        
Cost of goods sold   10,339 7,744 11,450
Research and development:        
Total research and development   206,011 217,496 244,990
Selling, general and administrative   337,202 264,119 265,542
In-process research and development $ 65,200 0 65,205 0
Restructuring   0 2,438 11,394
Total other income, net   13,551 3,317 12,028
Income tax provision on continuing operations   (988) (120) (223)
Income (loss) from discontinued operations, net of tax   24,715 (915) 264,934
Net loss   (25,546) (321,545) (111,399)
Reportable Segment        
Segment Reporting Information [Line Items]        
Revenue   490,728 233,175 145,238
Less:        
Cost of goods sold   10,339 7,744 11,450
Research and development:        
External research and development   107,036 126,303 142,482
Internal personnel costs   80,091 73,843 84,658
Other research and development   18,884 17,350 17,850
Total research and development   206,011 217,496 244,990
Selling, general and administrative   337,202 264,119 265,542
In-process research and development   0 65,205 0
Restructuring   0 2,438 11,394
Total other income, net   13,551 3,317 12,028
Income tax provision on continuing operations   (988) (120) (223)
Income (loss) from discontinued operations, net of tax   24,715 (915) 264,934
Net loss   $ (25,546) $ (321,545) $ (111,399)