Travere Therapeutics, Inc.
For the period ended Sep 30, 2025. Filed Oct 30, 2025.
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Sep 30, 2025 · Filed Oct 30, 2025
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Notes to the Unaudited Consolidated Financial Statements
NOTE 1. 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.
Approved Products:
FILSPARI® (sparsentan)
On September 5, 2024, the FDA granted full approval of FILSPARI® (sparsentan) to slow kidney function decline in adults with primary Immunoglobulin A nephropathy (IgAN) who are at risk of disease progression. FILSPARI is the only oral, once-daily, non-immunosuppressive medication that directly targets glomerular injury in the kidney by blocking two critical pathways of IgAN disease progression (endothelin-1 and angiotensin II).
FILSPARI had previously been granted accelerated approval in February 2023 based on the surrogate marker of proteinuria. Full approval 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.
In September 2021, the Company entered into a license and collaboration agreement (the "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 ≥0.75 g/g), and in April 2025, the Company and CSL Vifor announced that the European Commission has 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 urine protein-to-creatinine ratio ≥0.75 g/g). The MA is 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 License Agreement. Additionally, in September 2025, the Company recognized a $40.0 million milestone for market access initiatives in certain countries; payment of the milestone was received in the fourth quarter of 2025. FILSPARI became commercially available in Europe under the CMA in August 2024, with an initial launch in Germany and Austria. In October 2024, the Company and CSL Vifor announced that Swissmedic has granted temporary marketing authorization for FILSPARI for the treatment of adults with primary IgAN with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.75 g/g). In April 2025, the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK converted its conditional approval of FILSPARI in IgAN to standard approval.
In January 2024, the Company entered into an exclusive licensing agreement with Renalys Pharma, Inc. ("Renalys"), to bring sparsentan for the treatment of IgAN to patients in Japan and other countries in Asia. Renalys holds regional rights to sparsentan for Japan, South Korea, Taiwan, Brunei, Cambodia, Indonesia, Laos, Malaysia, Myanmar, the Philippines, Singapore, Thailand, and Vietnam. Following successful meetings with the Pharmaceuticals and Medical Devices Agency (PMDA) in 2023, in the second quarter of 2024 Renalys initiated an open label registration study of sparsentan in Japan to support potential approval of sparsentan in Japan. In July 2024, Renalys announced that the first patient was dosed in the study, and in January 2025, Renalys announced achievement of full enrollment in the study. In December 2024, Renalys announced that sparsentan received Orphan Drug Designation from the Japanese Ministry of Health, Labour and Welfare for the indication of primary IgA nephropathy as of November 27, 2024. In October 2025, Renalys announced that it had completed data collection for the primary endpoint in the Phase 3 clinical trial of sparsentan for IgAN and that it had reached an agreement with the PMDA regarding development plans for two new 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. Under the terms of the licensing agreement, Renalys is responsible for development, regulatory matters, and commercialization in the licensed territories.
Thiola® and Thiola EC® (tiopronin tablets)
Thiola® and Thiola EC® (tiopronin tablets) are approved in the United States for the prevention of cystine (kidney) stone formation in patients with severe homozygous cystinuria.
Clinical-Stage Programs:
Sparsentan for the treatment of FSGS
Sparsentan remains a novel investigational product candidate which has been granted Orphan Drug Designation for the treatment of FSGS in the U.S. and the European Economic Area countries (the “EEA”). In December 2023, the Company announced that it had completed a planned Type C meeting with the FDA to discuss previously reported results from the Phase 3 DUPLEX Study of sparsentan in FSGS. The FDA acknowledged the high unmet need for approved therapies as well as the challenges in studying FSGS but indicated that the two-year results from the Phase 3 DUPLEX Study alone were not sufficient to support an sNDA submission. In February 2025, the Company announced that it had completed a Type C meeting with the FDA and 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 is no longer needed. The sNDA remains under review by the FDA with a PDUFA target action date of January 13, 2026. The sNDA submission is supported by results from the Phase 3 DUPLEX Study and the Phase 2 DUET Study. If approved, FILSPARI could become the first and only FDA-approved medicine indicated for FSGS.
Pegtibatinase
Pegtibatinase is a novel investigational human enzyme replacement candidate being evaluated for the treatment of classical homocystinuria ("HCU"). Pegtibatinase has been granted Rare Pediatric Disease, Fast Track and Breakthrough Therapy designations by the FDA, as well as orphan drug designation in the United States and European Union. In May 2023, the Company announced positive topline results from cohort 6 in the Phase 1/2 COMPOSE Study. In December 2023, the Company initiated the pivotal Phase 3 HARMONY Study to support the potential approval of pegtibatinase for the treatment of classical HCU. The HARMONY Study is a global, randomized, multi-center, double-blind, placebo-controlled Phase 3 clinical trial designed to evaluate the efficacy and safety of pegtibatinase as a novel treatment to reduce total homocysteine ("tHcy") levels. In the beginning of 2024, the first patients were dosed in the HARMONY Study.
In September 2024, the Company announced a voluntary pause of enrollment in the Phase 3 HARMONY Study. The voluntary enrollment pause enables the Company to work to address necessary process improvements in manufacturing scale-up to support commercial scale manufacturing as well as full enrollment in the HARMONY Study. Patients currently enrolled in pegtibatinase studies continue to receive study medication from small scale batches which are unaffected by the scale-up process. Currently enrolled patients will be able to continue on study medication as scheduled for the duration of the trials they are participating in. The voluntary enrollment pause was enacted following the Company's determination that the desired drug substance profile was not achieved in the initial scale-up process. The Company has manufactured the first commercial-scale batches designed to enable the continuation of the Phase 3 program.
The Company acquired pegtibatinase as part of the November 2020 acquisition of Orphan Technologies Limited.
NOTE 2. BASIS OF PRESENTATION AND SIGNIFICANT ACCOUNTING POLICIES
The accompanying unaudited consolidated financial statements should be read in conjunction with the audited consolidated financial statements and notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2024, filed with the SEC on February 21, 2025. The accompanying consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States (“GAAP”) for interim financial information, the instructions for Form 10-Q and the rules and regulations of the SEC. Accordingly, since they are interim statements, the accompanying consolidated financial statements do not include all of the information and notes required by GAAP for annual financial statements, but reflect all adjustments consisting of normal, recurring adjustments, that are necessary for a fair statement of the financial position, results of operations and cash flows for the interim periods presented. Interim results are not necessarily indicative of the results that may be expected for any future periods. The December 31, 2024 balance sheet information was derived from the audited financial statements as of that date. Certain reclassifications have been made to the prior period consolidated financial statements to conform to the current period presentation. These reclassifications did not have an impact on total assets or total liabilities in the Consolidated Balance Sheets or net loss in the Consolidated Statements of Operations and Comprehensive Loss.
A summary of the significant accounting policies applied in the preparation of the accompanying consolidated financial statements follows:
Principles of Consolidation
The unaudited 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 GAAP. All intercompany accounts and transactions have been eliminated in consolidation. See Note 6 for further discussion of variable interest entities (“VIE”) that the Company consolidates.
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.
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.
The following table summarizes cost of goods sold for the three and nine months ended September 30, 2025 and 2024 (in thousands):
| Three Months Ended September 30, | Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2025 | 2024 | 2025 | 2024 | |
| Cost of goods sold - product sales | $1,308 | $1,626 | $4,083 | $5,059 |
| Cost of goods sold - license and collaboration | 277 | — | 3,703 | 132 |
| Total cost of goods sold | $1,585 | $1,626 | $7,786 | $5,191 |
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 three and nine months ended September 30, 2025 and 2024, sales of FILSPARI primarily consisted of zero-cost inventories. As of September 30, 2025, the Company had approximately $0.9 million of zero-cost inventory remaining, the majority of which the Company expects will be consumed in 2025. The Company began capitalizing inventory costs associated with FILSPARI following the February 2023 accelerated approval.
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, and develop drug materials and delivery devices, 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 hasfour 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.
Earnings (Loss) Per Share
The Company calculates basic earnings per share by dividing net income/(loss) by the weighted average number of shares outstanding during the period. Pre-funded warrants issued and sold by the Company to purchase shares of its common stock are included in the calculation of basic net loss per common share if the exercise price of the pre-funded warrant represents little consideration and is non-substantive in relation to the price paid for the warrant, and if the warrants are immediately exercisable with no further vesting conditions or contingencies associated with them.
The Company's diluted earnings/(loss) per share computation includes the effect, if any, of shares that would be issuable upon the exercise of outstanding stock options, convertible debt and RSUs, reduced by the number of shares which are assumed to be purchased by the Company from the resulting proceeds at the average market price during the year, when such amounts are dilutive to the earnings per share calculation. The potential dilutive effect of stock options and RSUs during the period are calculated in accordance with the treasury stock method, but are excluded if the effect is anti-dilutive. The potential dilutive effect of convertible debt outstanding during the period is calculated using the if-converted method assuming the conversion of convertible debt as of the earliest period reported or at the date of issuance, if later, but is excluded if the effect is anti-dilutive. 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.
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.
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 September 30, 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 sheet do not represent additional assets that could be used to satisfy claims against the Company’s general assets or liabilities for which creditors have recourse to the Company’s general assets.
Equity Securities
The Company applies the equity method of accounting for investments when it has significant influence, but no controlling interest in the investee. Judgment regarding the level of influence over each equity method investment includes key factors such as ownership interest, representation on the board of directors, participation in joint steering committees and material intercompany transactions. Upon investment, the Company evaluates any basis difference between the carrying value and fair value of the Company's proportionate share of the investee's net assets. Basis differences relating to in-process research and development ("IPR&D") are expensed when the investee is not considered a business as defined in ASC 805, Business Combinations, due to substantially all of the estimated fair value of the gross assets being concentrated in a group of similar IPR&D assets with no alternative future use. For the three and nine months ended September 30, 2025, the Company didnot have any basis adjustments. For the three and nine months ended September 30, 2024, the Company recognized zero and $3.4 million, respectively, in other (expense) income, net in the Company's Consolidated Statements of Operations and Comprehensive Loss for basis adjustments and reduced the equity method investment's carrying value tozero, as the Company's proportionate share of the basis difference exceeded the carrying value. See Note 6 for further discussion. Investments accounted for using the equity method are 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.
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 18 for further discussion. Unless otherwise noted, amounts and disclosures throughout the Notes to the unaudited consolidated financial statements relate to the Company's continuing operations.
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. As of December 31, 2024, the Company had recognized a total of $13.8 million in connection with the restructuring and it is no longer incurring restructuring expenses.
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 Accounting Standards Update ("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 ASU 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 July 2025, the One Big Beautiful Bill Act (“OBBBA”) was signed into law, which enacts significant changes to U.S. tax and related laws. Some of the provisions of the new tax law affecting corporations include but are not limited to expensing of domestic research expenses, calculating the limit on the interest expense based on 30 percent of EBITDA (rather than EBIT), and 100 percent bonus depreciation on eligible property acquired after January 19, 2025. There is no material change to the Company’s effective income tax rate and its net deferred federal income tax assets from the new tax law as the Company maintains a full valuation allowance.
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.
In December 2023, the FASB issued ASU No. 2023-09, Improvements to Income Tax Disclosures. This ASU does not change accounting for income taxes but requires new disclosures focusing on two areas, the effective rate reconciliation and taxes paid. This new standard is effective for public business entities for annual periods beginning after December 15, 2024. The Company plans to adopt the standard as required for the December 31, 2025 annual financial statements and interim periods thereafter. The Company does not expect the adoption of ASU 2023-09 to have a material impact on the consolidated financial statements.
NOTE 3. 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 totwo 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 nine months ended September 30, 2025 and 2024, adjustments to net product revenue related to performance obligations satisfied in previous periods were $1.0 million and $0.5 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 ("WAC") 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 three and nine months ended September 30, 2025 and 2024 (in thousands):
| Three Months Ended September 30, | Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2025 | 2024 | 2025 | 2024 | |
| FILSPARI | $90,900 | $35,619 | $218,668 | $82,578 |
| Tiopronin products | 22,250 | 25,382 | 65,184 | 70,583 |
| Total net product sales | $113,150 | $61,001 | $283,852 | $153,161 |
NOTE 4. COLLABORATION AND LICENSE AGREEMENTS
License Agreement with CSL Vifor
In September 2021, the Company entered into a license and collaboration agreement (“CSL Vifor License Agreement”) with Vifor (International) Ltd. (“CSL Vifor”), 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, among other things, expand the license to cover the following additional countries: Bahrain, Brazil, Chile, Israel, Kuwait, Oman, Qatar, Saudi Arabia and the United Arab Emirates (together with Europe, Australia and New Zealand, the "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. For the three and nine months ended September 30, 2025, the Company recognized $40.0 million for a market access milestone as the amount was probable of being achieved at September 30, 2025, and was subsequently received in the fourth quarter of 2025. For the nine months ended September 30, 2025, the Company recognized a regulatory milestone of $17.5 million as a result of the European Commission granting standard MA approval.
In January 2024, the license agreement (“Renalys License Agreement”) between the Company and Renalys came into effect. Pursuant to the terms of the Renalys License Agreement, the Company granted an exclusive license to Renalys for the development and commercialization of sparsentan in Japan, South Korea, Taiwan and other specified Asian countries ("Renalys Licensed Territories"). Under the terms of the Renalys License Agreement, the Company received a non-refundable upfront payment and will be eligible to receive up to $120.0 million in aggregate 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 Renalys Licensed Territories. In addition, the Company received an option to purchase shares of common stock of Renalys (“Option Agreement”), which it exercised in January 2024. The Company also 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 Renalys License Agreement, Renalys will be responsible for all development and commercialization activities in the Renalys Licensed Territories. The Renalys License Agreement will remain in effect, unless terminated earlier, until the expiration of all royalty terms for sparsentan in the Renalys Licensed Territories. Each party has the right to terminate the Renalys License Agreement for the other party’s uncured material breach or insolvency, or if the time required for performance under the Renalys License Agreement by the other party is extended due to a force majeure event that continues fornine months or more. Renalys may terminate the Renalys License Agreement for any reason upon prior written notice to the Company. The Company may terminate the Renalys License Agreement if Renalys abandons development in Japan or South Korea prior to first commercial sales of sparsentan in either Japan or South Korea.
The Company assessed the Renalys 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 Renalys License Agreement totaled $8.3 million, consisting of the fixed non-refundable upfront payment, milestone payment and estimated fair value of the Option Agreement. The variable development-related milestones were excluded from the transaction price given the substantial uncertainty related to their achievement. Sales-based milestone payments and royalties on net sales were excluded from the initial transaction price and will be recognized at the later of when the related sales occur or when the performance obligation to which the sales-based milestone or royalty has been allocated has been satisfied.
The Company concluded that Renalys represents a customer and applied relevant guidance from ASC 606 to evaluate the accounting under the Renalys License Agreement. In accordance with this guidance, the Company concluded that the promise to grant the license is distinct, resulting inone performance obligation as the license has stand-alone functionally at contract inception. The Buyout Right precludes transferring control of the license to Renalys under ASC 606 and the Company’s option to repurchase the common stock at a price greater than the original license premium results in accounting for the Renalys License Agreement as a financing arrangement. The transaction price was originally recorded in other current liabilities as a result of the Buyout Right. During the three months ended September 30, 2025, the Buyout Right was relinquished and the Company recognized the entire Renalys deferred revenue balance of $9.3 million in license and collaboration revenue on the Consolidated Statements of Operations and Comprehensive Loss.
See Note 6 for further discussion of VIEs.
For the three months ended September 30, 2025, the Company recognized $51.7 million in license and collaboration revenue, which included a market access milestone of $40.0 million associated with the CSL Vifor License Agreement, $9.3 million in license revenue associated with the Renalys License Agreement and $2.4 million for royalties earned on net sales of FILSPARI in the CSL Vifor Licensed Territories. For the nine months ended September 30, 2025 the Company recognized $77.2 million in license and collaboration revenue, which included regulatory and market access milestones totaling $57.5 million associated with the CSL Vifor License Agreement, $9.3 million associated with the Renalys License Agreement, the sale of $3.8 million of active pharmaceutical ingredients, $2.9 million for clinical development activities, based upon the ratio of costs incurred to total estimated costs and $3.7 million for royalties earned on net sales of FILSPARI in the CSL Vifor Licensed Territories.
For the three and nine months ended September 30, 2024, the Company recognized $1.8 million and $5.1 million, respectively, in license and collaboration revenue for clinical development activities, based upon the ratio of costs incurred to total estimated costs.
Deferred revenue related to the clinical development activities as of September 30, 2025 and December 31, 2024 waszero and $2.8 million, respectively.
NOTE 5. MARKETABLE DEBT SECURITIES
The Company's marketable debt securities as of September 30, 2025 and December 31, 2024 were composed of available-for-sale commercial paper and corporate and government debt securities. The primary objective of the Company’s investment portfolio is to preserve capital and liquidity while enhancing overall returns. The Company’s investment policy limits interest-bearing security investments to certain types of instruments issued by institutions with primarily investment grade credit ratings and places restrictions on maturities and concentration by asset class and issuer.
Marketable debt securities consisted of the following (in thousands):
| September 30, 2025 | December 31, 2024 | |
|---|---|---|
| Marketable debt securities: | ||
| Commercial paper | $45,465 | $73,325 |
| Corporate debt securities | 93,134 | 203,816 |
| Securities of government sponsored entities | 5,001 | 35,025 |
| Total available-for-sale marketable debt securities | $143,600 | $312,166 |
In addition to funding operations, the decrease in the marketable debt securities balance as of September 30, 2025 is due to the repayment of the 2025 Notes in September 2025. See Note 10 for further discussion of Convertible Notes Payable.
The following is a summary of short-term marketable debt securities classified as available-for-sale as of September 30, 2025 (in thousands):
| Remaining Contractual Maturity(in years) | AmortizedCost | Unrealized Gains | UnrealizedLosses | Aggregate Estimated Fair Value | |
|---|---|---|---|---|---|
| Marketable debt securities: | |||||
| Commercial paper | Less than 1 | $45,450 | $15 | $— | $45,465 |
| Corporate debt securities | Less than 1 | 88,156 | 40 | (7) | 88,189 |
| Securities of government-sponsored entities | Less than 1 | 5,000 | 1 | — | 5,001 |
| Total maturity less than 1 year | 138,606 | 56 | (7) | 138,655 | |
| Corporate debt securities | 1 to 2 | 4,899 | 46 | — | 4,945 |
| Total maturity 1 to 2 years | 4,899 | 46 | — | 4,945 | |
| Total available-for-sale marketable debt securities | $143,505 | $102 | $(7) | $143,600 | |
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 Cost | Unrealized Gains | UnrealizedLosses | Aggregate Estimated Fair Value | |
|---|---|---|---|---|---|
| Marketable debt securities: | |||||
| Commercial paper | Less than 1 | $73,410 | $1 | $(86) | $73,325 |
| Corporate debt securities | Less than 1 | 203,395 | 483 | (62) | 203,816 |
| Securities of government-sponsored entities | Less than 1 | 34,993 | 33 | (1) | 35,025 |
| Total available-for-sale marketable debt securities | $311,798 | $517 | $(149) | $312,166 | |
For the three and nine months ended September 30, 2025 and 2024, realized gains and losses on marketable debt securities were immaterial. As of September 30, 2025 and December 31, 2024, the accrued interest receivable related to the Company's marketable debt securities was $1.5 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 income (loss).
The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of September 30, 2025 (in thousands):
| Less Than 12 Months | 12 Months or Greater | Total | ||||
|---|---|---|---|---|---|---|
| Description of Securities | Fair Value | Unrealized Losses | Fair Value | Unrealized Losses | Fair Value | Unrealized Losses |
| Corporate debt securities | $35,411 | $7 | $— | $— | $35,411 | $7 |
| Total | $35,411 | $7 | $— | $— | $35,411 | $7 |
The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of December 31, 2024 (in thousands):
| Less Than 12 Months | 12 Months or Greater | Total | ||||
|---|---|---|---|---|---|---|
| Description of Securities | Fair Value | Unrealized Losses | Fair Value | Unrealized Losses | Fair Value | Unrealized Losses |
| Commercial paper | $68,446 | $86 | $— | $— | $68,446 | $86 |
| Corporate debt securities | 40,112 | 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 September 30, 2025 and December 31, 2024, the amortized cost of the available-for-sale marketable debt securities in an unrealized loss position was $35.4 million and $123.7 million, respectively.
As of September 30, 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 nine months ended September 30, 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.
NOTE 6. 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 nine months ended September 30, 2025.
In January 2024, the Renalys License Agreement between the Company and Renalys came into effect and the Company exercised its option to purchase shares of common stock of Renalys. The Company determined that Renalys is a VIE as they could require additional funding to support development and commercial activities. The Company has variable interests in Renalys, including an equity interest, Buyout Right and performance-related payments under the Renalys License Agreement that absorb variability from the performance of Renalys.
In order to determine the primary beneficiary of Renalys, the Company evaluated its variable interest to identify if the Company had the power to direct the activities that most significantly impact the economic performance. Based upon the capital structure, governing documents and overall business operations, the Company determined that it is not the primary beneficiary as it does not have the power to direct the activities that most significantly impact the economic performance of Renalys and does not have an obligation to absorb losses.
The carrying amount of the liabilities related to the Company’s variable interests waszero and $8.9 million as of September 30, 2025 and December 31, 2024, respectively, included in other current liabilities in the Company’s Consolidated Balance Sheets. During the three and nine months ended September 30, 2025, the Buyout Right was relinquished and the Company recognized the entire Renalys deferred revenue balance of $9.3 million in license and collaboration revenue on the Consolidated Statements of Operations and Comprehensive Loss. The Company is not required to provide additional funding. The creditors have no recourse to the general credit or assets of the Company.
NOTE 7. LEASES
As of September 30, 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, which occurred in phases over 2020, 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 offive 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 for a portion of the 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 because (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.
The Company also had an operating lease with Esprit Investments Limited for office space located in Dublin, Ireland, which was entered into in October 2022 for a term offive years. In January 2025, the Company entered into a lease assignment for the Dublin office space, which released the Company of its obligations under the original lease. As a result, the Company derecognized the operating lease right-of-use asset and lease liability in the Consolidated Balance Sheets.
The following is a schedule of the future minimum rental commitments for the Company's operating lease reconciled to the lease liability and right-of-use asset as of September 30, 2025 (in thousands):
| September 30, 2025 | |
|---|---|
| 2025 (remaining) | $1,654 |
| 2026 | 6,775 |
| 2027 | 6,978 |
| 2028 | 4,781 |
| 2029 and thereafter | — |
| Total undiscounted future minimum payments | 20,188 |
| Present value discount | (1,809) |
| Total lease liability | 18,379 |
| Unamortized lease incentives | (2,777) |
| Cash payments in excess of straight-line lease expense | (4,138) |
| Total right-of-use asset | $11,464 |
The weighted-average remaining lease term and weighted-average discount rate of the Company's operating leases are as follows:
| September 30, 2025 | December 31, 2024 | |
|---|---|---|
| Weighted-average remaining lease term in years | 2.9 | 3.7 |
| Weighted-average discount rate | 6.47% | 6.48% |
For the three and nine months ended September 30, 2025, the Company recorded $1.1 million and $3.3 million, respectively, in expense related to operating leases, including amortized tenant improvement allowances. For the three and nine months ended September 30, 2024, the Company recorded $1.3 million and $3.7 million, respectively, in expense related to operating leases, including amortized tenant improvement allowances.
NOTE 8. 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 counter-parties 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 September 30, 2025, the fair value of the Company's 2.25% Convertible Senior Notes due 2029 was $360.5 million. As of December 31, 2024, the fair value of the Company's 2.5% Convertible Senior Notes due 2025, now repaid, was $68.2 million and the fair value of the Company's 2.25% Convertible Senior Notes due 2029 was $302.1 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 September 30, 2025 (in thousands):
| As of September 30, 2025 | ||||
|---|---|---|---|---|
| Total carrying and estimated fair value | Quoted prices in active markets(Level 1) | Significant other observable inputs (Level 2) | Significant unobservable inputs (Level 3) | |
| Assets: | ||||
| Cash and cash equivalents | $110,930 | $110,930 | $— | $— |
| Marketable debt securities, available-for-sale | 143,600 | — | 143,600 | — |
| Total | $254,530 | $110,930 | $143,600 | $— |
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, 2024 | ||||
|---|---|---|---|---|
| Total carrying and estimated fair value | Quoted 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-sale | 312,166 | — | 312,166 | — |
| Total | $370,701 | $51,060 | $319,641 | $— |
NOTE 9. INTANGIBLE ASSETS
Ligand License Agreement
In 2012, the Company entered into the Ligand License Agreement for a worldwide sublicense to develop, manufacture and commercialize FILSPARI. 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 September 30, 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 FILSPARI and any other products containing FILSPARI or related compounds, with payments due quarterly. The Company began incurring costs associated with such royalties following the February 2023 approval of FILSPARI. For the three and nine months ended September 30, 2025, the Company capitalized royalties owed on net sales of FILSPARI to intangible assets of $15.6 million and $35.9 million, respectively, and $5.4 million and $12.4 million, for the three and nine months ended September 30, 2024, respectively. The cost of historical milestones paid 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, pursuant to which it 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. Under the terms of the Mission License Agreement, as subsequently amended, which runs through May 2029, the Company is obligated to pay Mission the greater of $2.1 million, representing the guaranteed minimum royalty, or 20% of its Thiola net sales generated globally during each calendar year. The Company has capitalized $181.5 million related to the Thiola intangible asset which consists of an up-front license fee, professional fees, present value of the guaranteed minimum royalties and any additional payment obligations through September 30, 2025 in excess of minimum royalties. The Company has not capitalized any royalties in excess of the annual contractual minimum as of September 30, 2025 as royalties are capitalized in the period in which they become probable and estimable, which is typically in the period corresponding with the respective sales.
The following table sets forth amortizable intangible assets as of September 30, 2025 and December 31, 2024 (in thousands):
| September 30, 2025 | December 31, 2024 | |
|---|---|---|
| Finite-lived intangible assets | $271,288 | $224,293 |
| Less: accumulated amortization | (162,606) | (121,072) |
| Net carrying value | $108,682 | $103,221 |
Amortization expense for the three and nine months ended September 30, 2025 was $15.4 million and $41.5 million, respectively, and $10.8 million and $30.1 million, for the three and nine months ended September 30, 2024, respectively, recorded in selling, general and administrative expenses.
As of September 30, 2025 and December 31, 2024, the Company had goodwill of $0.8 million.
NOTE 10. CONVERTIBLE NOTES PAYABLE
The composition of the Company’s convertible senior notes are as follows (in thousands):
| September 30, 2025 | December 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 | (4,880) | (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 | $311,370 | $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 September 30, 2025, accrued interest on the 2029 Notes of $0.6 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 thefive 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 40 th 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 theseven-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 September 30, 2025.
The 2029 Notes are classified on the Company's Consolidated Balance Sheets as long-term convertible debt at September 30, 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.
The following table sets forth total interest expense recognized related to the 2025 and 2029 Notes (in thousands):
| Three Months Ended September 30, | Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2025 | 2024 | 2025 | 2024 | |
| Contractual interest expense | $2,138 | $2,210 | $6,557 | $6,629 |
| Amortization of debt issuance costs | 420 | 432 | 1,286 | 1,293 |
| Total interest expense for the 2025 and 2029 Notes | $2,558 | $2,642 | $7,843 | $7,922 |
Total interest expense recognized for the three and nine months ended September 30, 2025 was $2.8 million and $8.5 million, respectively, and $2.8 million and $8.4 million, for the three and nine months ended September 30, 2024, respectively
NOTE 11. ACCRUED EXPENSES
Accrued expenses at September 30, 2025 and December 31, 2024 consisted of the following (in thousands):
| September 30, 2025 | December 31, 2024 | |
|---|---|---|
| Sales discounts, rebates, and allowances | $29,503 | $10,585 |
| Compensation related costs | 27,587 | 35,166 |
| Accrued royalties | 19,764 | 12,309 |
| Research and development | 16,863 | 16,090 |
| Selling, general and administrative | 8,622 | 6,154 |
| Miscellaneous accrued expenses | 3,423 | 5,724 |
| Total accrued expenses | $105,762 | $86,028 |
NOTE 12. NET INCOME (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 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, and shares issuable upon conversion of the 2025 Notes and 2029 Notes, are considered to be common stock equivalents. For the periods that the Company has reported net losses, all potentially dilutive securities are anti-dilutive and, accordingly, basic net loss per share equals diluted net loss per share.
Basic and diluted net income (loss) per share is calculated as follows(net income (loss) amounts are stated in thousands):
| Three Months Ended · Nine Months Ended | |||||||
|---|---|---|---|---|---|---|---|
| September 30, 2025 | |||||||
| Shares · Net Income · EPS · Shares · Net Loss · EPS | |||||||
| Basic income (loss) per share | 89,230,420 | $25,706 | $0.29 | 88,847,209 | $(28,275) | $(0.32) | |
| Interest expense associated with convertible debt, net of tax | 2,558 | ||||||
| Dilutive income (loss) per share | 102,618,560 | $28,264 | $0.28 | 88,847,209 | $(28,275) | $(0.32) | |
| Three Months Ended · Nine Months Ended | |||||||
| September 30, 2024 | |||||||
| Shares · Net Loss · EPS · Shares · Net Loss · EPS | |||||||
| Continuing operations | 77,779,379 | $(54,752) | $(0.70) | 77,473,161 | $(260,362) | $(3.36) | |
| Discontinued operations | 77,779,379 | (59) | — | 77,473,161 | (919) | (0.01) | |
| Basic and diluted loss per share | 77,779,379 | $(54,811) | $(0.70) | 77,473,161 | $(261,281) | $(3.37) | |
The calculation of diluted weighted average common shares outstanding for the three months ended September 30, 2025 was as follows:
| Three Months EndedSeptember 30, 2025 | |
|---|---|
| Basic weighted average common shares outstanding | 89,230,420 |
| Dilutive common share equivalents - restricted stock units | 1,120,629 |
| Dilutive common share equivalents - convertible debt | 11,389,096 |
| Dilutive common share equivalents - options | 878,415 |
| Diluted weighted average common shares outstanding | 102,618,560 |
The following common stock equivalents have been excluded because they were anti-dilutive:
| Three Months Ended September 30, | Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2025 | 2024 | 2025 | 2024 | |
| Convertible debt | — | 11,697,953 | 11,593,869 | 11,697,953 |
| Options | 5,948,756 | 10,235,428 | 9,947,928 | 10,759,644 |
| Restricted stock units | 55,566 | 3,809,142 | 4,368,415 | 3,828,261 |
| Total anti-dilutive shares | 6,004,322 | 25,742,523 | 25,910,212 | 26,285,858 |
NOTE 13. COMMITMENTS AND CONTINGENCIES
Commitments
Certain of the Company's contractual arrangements with 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 September 30, 2025, no contingent 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.
NOTE 14. SHARE-BASED COMPENSATION
Stock Options
The following table summarizes stock option activity during the nine months ended September 30, 2025:
| Shares Underlying Options | Weighted Average Exercise Price | Weighted Average Remaining Contractual Life (years) | Aggregate Intrinsic Value (in thousands) | |
|---|---|---|---|---|
| Outstanding at December 31, 2024 | 9,283,331 | $20.13 | 5.51 | $16,408 |
| Granted | 1,434,200 | 20.50 | ||
| Exercised | (547,616) | 16.20 | 3,790 | |
| Forfeited/canceled | (625,478) | 29.03 | ||
| Outstanding at September 30, 2025 | 9,544,437 | $19.83 | 5.89 | $46,859 |
| Vested and expected to vest at September 30, 2025 | 9,544,437 | $19.83 | 5.89 | $46,859 |
| Exercisable at September 30, 2025 | 6,920,120 | $20.66 | 4.83 | $30,016 |
At September 30, 2025, unamortized stock compensation for stock options was $23.5 million, with a remaining weighted-average recognition period of 2.5 years.
Restricted Stock Units
Service Based Restricted Stock Units
The following table summarizes the Company’s service based restricted stock unit activity during the nine months ended September 30, 2025:
| Number of Restricted Stock Units | Weighted Average Grant Date Fair Value | |
|---|---|---|
| Unvested at December 31, 2024 | 3,517,263 | $15.41 |
| Granted | 2,437,890 | 20.09 |
| Vested | (1,265,220) | 16.61 |
| Forfeited/canceled | (234,788) | 16.47 |
| Unvested at September 30, 2025 | 4,455,145 | $17.57 |
At September 30, 2025, unamortized stock compensation for service based restricted stock units was $60.5 million, with a remaining weighted-average recognition period of 2.6 years.
Performance Based Restricted Stock Units
The following table summarizes the Company’s performance based restricted stock unit activity during the nine months ended September 30, 2025:
| Number of Restricted Stock Units | Weighted Average Grant Date Fair Value | |
|---|---|---|
| Unvested at December 31, 2024 | 216,208 | $18.34 |
| Granted | 88,000 | 20.46 |
| Vested | (70,604) | 27.54 |
| Forfeited/canceled | — | — |
| Unvested at September 30, 2025 | 233,604 | $16.35 |
At September 30, 2025, unamortized stock compensation for performance based restricted stock units was $0.1 million, with a remaining weighted-average recognition period of 0.6 years.
Share-Based Compensation
Total share-based compensation presented in the Consolidated Statements of Stockholders' Equity includes both continuing operations and discontinued operations. The following table sets forth share-based compensation for continuing operations for the three and nine months ended September 30, 2025 and 2024 (in thousands):
| Three Months Ended September 30, | Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2025 | 2024 | 2025 | 2024 | |
| Research and development | $4,101 | $3,321 | $12,817 | $10,752 |
| Selling, general and administrative | 7,162 | 4,700 | 20,587 | 16,946 |
| Total share-based compensation | $11,263 | $8,021 | $33,404 | $27,698 |
NOTE 15. INVENTORY
Inventory consisted of the following at September 30, 2025 and December 31, 2024 (in thousands):
| September 30, 2025 | December 31, 2024 | |
|---|---|---|
| Raw materials | $25,299 | $30,552 |
| Work in process | 1,986 | 7,625 |
| Finished goods | 10,262 | 3,679 |
| Total inventory | $37,547 | $41,856 |
| Classified as: | ||
| Current inventory | $5,548 | $6,200 |
| Long-term inventory | 31,999 | 35,656 |
| Total inventory | $37,547 | $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 September 30, 2025 and December 31, 2024. The Company maintains levels of these inventories beyond a one-year production plan to limit exposure to potential supply disruption.
NOTE 16. ACCOUNTS RECEIVABLE
Accounts receivable, net of reserves for prompt pay discounts and expected credit losses, was $83.0 million and $27.1 million at September 30, 2025 and December 31, 2024, respectively. The Accounts receivable balance as of September 30, 2025 includes the market access milestone of $40.0 million associated with the CSL Vifor License Agreement, which was received in the fourth quarter of 2025. The total reserves for both periods were immaterial.
The Company's evaluation and accounting for 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.
NOTE 17. EQUITY OFFERINGS
At-the-Market Equity Offering
In October 2024, the Company filed a prospectus supplement to the prospectus included in its registration statement on Form S-3 (File No. 333-281194), pursuant to which the Company may offer and sell, from time to time through Jefferies LLC, as agent ("Jefferies"), up to $100.0 million of common stock pursuant to an Amended and Restated Open Market Sale Agreement ("ATM Agreement") with Jefferies dated October 2024. As of September 30, 2025, the Company hasnot sold any shares under the ATM Agreement.
NOTE 18. DIVESTITURES
Discontinued Operations
Sale of Bile Acid Product Portfolio
In August 2023, the Company closed the sale of its bile acid business to Mirum Pharmaceuticals, Inc. (“Mirum”) pursuant to the terms of an asset purchase agreement dated July 16, 2023 (the "Purchase Agreement"). 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 Chenodal and Cholbam (also known as Kolbam, and together with Chenodal, the "Products"). The Company will recognize the contingent consideration receivable in earnings when the target annual sales for the milestones are met and the contingency is resolved. The Company determined that the divestiture represented a strategic shift that would 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. The Company recognized loss from discontinued operations in the Consolidated Statements of Operations and Comprehensive Loss of $0.1 million and $0.9 million for the three and nine months ended September 30, 2024, respectively.
NOTE 19. SEGMENT INFORMATION
The Company operates inone business segment focused on identifying, developing and delivering life-changing therapies to people living with rare kidney and metabolic diseases. The determination of a single business segment is consistent with the consolidated financial information regularly provided to the Company’s chief operating decision maker (“CODM”), who is the President and Chief Executive Officer. The CODM uses net loss to monitor budget versus actual results in assessing segment performance and the allocation of resources. The Company’s CODM also utilizes the Company’s long-range plan as a strategic tool to allocate resources according to the Company’s strategic objectives.
The Company sells its products to specialty pharmacies and through direct-to-patient distributors worldwide, there were no significant product sales outside of the United States ("U.S.") for the three and nine months ended September 30, 2025 and 2024. License revenues outside of the U.S. were $42.4 million and $61.8 million for the three and nine months ended September 30, 2025, respectively. License revenues outside of the U.S. werenot significant for the three and nine months ended September 30, 2024. Long-lived assets located outside the U.S. were immaterial as of both September 30, 2025 and December 31, 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.
The following table presents reportable segment loss, including significant expenses regularly provided to the CODM, attributable to the Company’s reportable segment for the three and nine months ended September 30, 2025 and 2024 (in thousands):
| Three Months Ended September 30, | Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2025 | 2024 | 2025 | 2024 | |
| Revenue | $164,859 | $62,898 | $361,039 | $158,388 |
| Less: | ||||
| Cost of goods sold | 1,585 | 1,626 | 7,786 | 5,191 |
| Research and development: | ||||
| External research and development | 27,837 | 31,587 | 77,653 | 89,443 |
| Internal personnel costs | 18,959 | 16,783 | 57,276 | 54,478 |
| Other research and development | 5,094 | 3,309 | 13,212 | 11,508 |
| Total research and development | 51,890 | 51,679 | 148,141 | 155,429 |
| Selling, general and administrative | 86,453 | 65,619 | 235,508 | 194,618 |
| In-process research and development | — | — | — | 65,205 |
| Restructuring | — | 123 | — | 1,035 |
| Total other income, net | 783 | 1,313 | 2,188 | 2,920 |
| Income tax (provision) benefit on continuing operations | (8) | 84 | (67) | (192) |
| Loss from discontinued operations, net of tax | — | (59) | — | (919) |
| Net income (loss) | $25,706 | $(54,811) | $(28,275) | $(261,281) |
NOTE 20. SUBSEQUENT EVENTS
In October 2025, Renalys announced that it has entered into a definitive stock purchase agreement with Chugai Pharmaceutical Co., Ltd. (“Chugai”) pursuant to which, upon the closing of the transaction, Chugai will acquire full ownership of Renalys and will gain exclusive rights to develop and commercialize sparsentan in Japan, South Korea, and Taiwan. As a minority shareholder in Renalys, Travere is entitled to receive a portion of the upfront payment at the closing of the transaction, and will be eligible to receive future payments upon the achievement of specified regulatory milestones for sparsentan and royalties on net sales in Japan, South Korea, and Taiwan.
The text of this filing, as the company filed it with the SEC, without its images or exhibits. This is not investment advice.