0001070081-26-000017 | 10-Q | XBRL Viewer

Capitalization - Narrative (Details) - Sales Agreement - USD ($) $ in Millions	3 Months Ended		6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025	Aug. 31, 2019
Capitalization
Number of shares issued in transaction (in shares)	0	0	0	0
Maximum
Capitalization
Value of shares available to be issued and sold in transaction					$ 125.0
Aggregate value of remaining shares to be issued and sold			$ 93.0

Summary of significant accounting policies

6 Months Ended

Jun. 30, 2026

Summary of significant accounting policies

Summary of significant accounting policies

2. Summary of significant accounting policies

The Company’s complete listing of significant accounting policies is set forth in Note 2 of the notes to the Company’s audited financial statements as of December 31, 2025 included in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (the "SEC") on February 19, 2026 (the "2025 Form 10-K"). There were no changes to significant accounting policies for the three and six months ended June 30, 2026. Selected significant accounting policies are discussed in further detail below.

Basis of presentation

The accompanying financial information as of June 30, 2026 and for the three and six months ended June 30, 2026 and 2025 has been prepared by the Company, without audit, pursuant to the rules and regulations of the SEC. Certain

information and footnote disclosures normally included in financial statements prepared in accordance with generally accepted accounting principles in the United States ("GAAP") have been condensed or omitted pursuant to such rules and regulations. These interim financial statements should be read in conjunction with the Company’s audited financial statements as of December 31, 2025 and notes thereto included in the 2025 Form 10-K.

In the opinion of management, the unaudited financial information as of June 30, 2026 and for the three and six months ended June 30, 2026 and 2025 reflects all adjustments, which are normal recurring adjustments, necessary to present a fair statement of financial position, results of operations, stockholders’ deficit, and cash flows. The results of operations for the three and six months ended June 30, 2026 are not necessarily indicative of the results to be expected for the year ended December 31, 2026 or for any other interim period or for any other future year.

Use of estimates

The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Significant estimates in these consolidated financial statements have been made in connection with the calculation of net product sales, certain accruals related to the Company’s research and development expenses, valuation procedures for liability for sale of future royalties, and the provision for or benefit from income taxes. Actual results could differ from those estimates. Changes in estimates are reflected in reported results in the period in which they become known.

Restricted cash

Restricted cash included in deposits and other assets on the consolidated balance sheet contains an unconditional, irrevocable and transferable letter of credit of $3.0 million in connection with an amendment and restatement of the Company’s lease in Hopewell Township, New Jersey. Restricted cash also contains an unconditional, irrevocable and transferable letter of credit of $10.0 million in connection with obligations for the Company’s facility lease in Warren, New Jersey. If after July 1, 2027, the Company is not in default of the lease agreement and meets certain creditworthiness guidelines, then the letter of credit will be reduced to $5.0 million. If after December 31, 2028, the Company is not in default of the lease agreement and meets certain creditworthiness guidelines, then the letter of credit will be further reduced to $2.5 million. Both letters of credit are classified within deposits and other assets on the consolidated balance sheet due to the long-term nature of the letters of credit. Restricted cash also includes a collateral account of $2.8 million in connection with the Company’s corporate card program. The collateral account is classified within deposits and other assets on the consolidated balance sheet due to its long-term nature. Restricted cash also includes a bank guarantee of $0.7 million denominated in a foreign currency.

The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the consolidated balance sheet that sum to the total of the same amounts shown in the consolidated statement of cash flows:


		End of			Beginning of
		period-			period-
		June 30,			December 31,
		2026			2025
Cash and cash equivalents		$	1,049,887		$	984,648
Restricted cash included in deposits and other assets			16,445			13,686
Total Cash, cash equivalents and restricted cash per consolidated statement of cash flows		$	1,066,332		$	998,334

Revenue recognition

Net product revenue

The Company’s net product revenue primarily consists of sales of Sephience for the treatment of PKU. Net product revenues also includes sales of Translarna in territories outside of the U.S. for the treatment of nmDMD and sales of Emflaza in the U.S. for the treatment of DMD. The Company recognizes revenue when its performance obligation with

its customers has been satisfied. The Company’s performance obligation is to provide products based on customer orders from distributors, hospitals, specialty pharmacies or retail pharmacies. The performance obligation is satisfied at a point in time when the Company’s customer obtains control of the product, which is typically upon delivery. The Company invoices its customers after the products have been delivered and invoice payments are generally due within 30 to 90 days of the invoice date. The Company determines the transaction price based on fixed consideration in its contractual agreements. Contract liabilities arise in certain circumstances when consideration is due for goods the Company has yet to provide. As the Company has identified only one distinct performance obligation, the transaction price is allocated entirely to product sales. In determining the transaction price, a significant financing component does not exist since the timing from when the Company delivers product to when the customers pay for the product is typically less than one year. Customers in certain countries pay in advance of product delivery. In those instances, payment and delivery typically occur in the same month.

The Company records product sales net of any variable consideration, which includes discounts, allowances, rebates related to Medicaid and other government pricing programs, and distribution fees. The Company uses the expected value or most likely amount method when estimating its variable consideration, unless discount or rebate terms are specified within contracts. The identified variable consideration is recorded as a reduction of revenue at the time revenues from product sales are recognized. These estimates for variable consideration are adjusted to reflect known changes in factors and may impact such estimates in the quarter those changes are known. Revenue recognized does not include amounts of variable consideration that are constrained.

In relation to customer contracts, the Company incurs costs to fulfill a contract but does not incur costs to obtain a contract. These costs to fulfill a contract do not meet the criteria for capitalization and are expensed as incurred. The Company considers any shipping and handling costs that are incurred after the customer has obtained control of the product as a cost to fulfill a promise. Shipping and handling costs associated with finished goods delivered to customers are recorded as a selling expense.

Collaboration, license, and royalty revenue

The terms of these agreements typically include payments to the Company of one or more of the following: nonrefundable, upfront license fees; milestone payments; research funding and royalties on future product sales. In addition, the Company generates service revenue through agreements that generally provide for fees for research and development services and may include additional payments upon achievement of specified events.

At the inception of a collaboration arrangement, the Company needs to first evaluate if the arrangement meets the criteria in Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) Topic 808 “Collaborative Arrangements” to then determine if ASC Topic 606 is applicable by considering whether the collaborator meets the definition of a customer. If the criteria are met, the Company assesses the promises in the arrangement to identify distinct performance obligations.

For licenses of intellectual property, the Company assesses, at contract inception, whether the intellectual property is distinct from other performance obligations identified in the arrangement. If the licensing of intellectual property is determined to be distinct, revenue is recognized for nonrefundable, upfront license fees when the license is transferred to the customer and the customer can use and benefit from the license. If the licensing of intellectual property is determined not to be distinct, then the license will be bundled with other promises in the arrangement into one distinct performance obligation. The Company needs to determine if the bundled performance obligation is satisfied over time or at a point in time. If the Company concludes that the nonrefundable, upfront license fees will be recognized over time, the Company will need to assess the appropriate method of measuring proportional performance.

For milestone payments, the Company assesses, at contract inception, whether the development or sales-based milestones are considered probable of being achieved. If it is probable that a significant revenue reversal will occur, the Company will not record revenue until the uncertainty has been resolved. Milestone payments that are contingent upon regulatory approval are not considered probable of being achieved until the applicable regulatory approvals or other external conditions are obtained as such conditions are not within the Company’s control. If it is probable that a significant revenue reversal will not occur, the Company will estimate the milestone payments using the most likely amount method. The

Company will reassess the development and sales-based milestones each reporting period to determine the probability of achievement. The Company recognizes royalties from product sales at the later of when the related sales occur or when the performance obligation to which the royalty has been allocated has been satisfied. If it is probable that a significant revenue reversal will not occur, the Company will estimate the royalty payments using the most likely amount method.

The Company recognizes revenue for reimbursements of research and development costs under collaboration agreements as the services are performed. The Company records these reimbursements as revenue and not as a reduction of research and development expenses as the Company has the risks and rewards as the principal in the research and development activities.

Allowance for doubtful accounts

The Company maintains an allowance for estimated losses resulting from the inability of its customers to make required payments. The Company estimates uncollectible amounts based upon current customer receivable balances, the age of customer receivable balances, the customer’s financial condition and current economic trends. The Company also assesses whether an allowance for expected credit losses may be required which includes a review of the Company’s receivables portfolio, which are pooled on a customer basis or country basis. In making its assessment of whether an allowance for credit losses is required, the Company considers its historical experience with customers, current balances, levels of delinquency, regulatory and legal environments, and other relevant current and future forecasted economic conditions. For the three and six months ended June 30, 2026 and 2025, no allowance was recorded for credit losses. The allowance for doubtful accounts was $15.6 million as of June 30, 2026 and $15.2 million as of December 31, 2025. For the three and six months ended June 30, 2026, bad debt expense was $0.1 million and $0.8 million, respectively. For the three and six months ended June 30, 2025, bad debt expense was $5.9 million and $7.8 million, respectively.

Income Taxes

On December 22, 2017, the U.S. government enacted the 2017 Tax Cuts and Jobs Act (“TCJA”), which significantly revised U.S. tax law by, among other provisions, lowering the U.S. federal statutory corporate income tax rate to 21%, imposing a mandatory one-time transition tax on previously deferred foreign earnings, and eliminating or reducing certain income tax deductions. The Global Intangible Low-Taxed Income (“GILTI”) provisions of the TCJA require the Company to include in its U.S. income tax return foreign subsidiary earnings in excess of an allowable return on the foreign subsidiary’s tangible assets. The Company has elected to account for GILTI tax in the period in which it is incurred, and therefore has not provided any deferred tax impacts of GILTI in its consolidated financial statements for the period ended June 30, 2026.

On December 15, 2022, the European Union member states formally adopted the EU’s Pillar Two Directive, which generally provides for a minimum effective tax rate of 15%, as established by the Organization for Economic Co-operation and Development Pillar Two Framework that was supported by over 130 countries worldwide. The EU effective dates were January 1, 2024, and January 1, 2025, for different aspects of the directive. A significant number of other countries are also implementing similar legislation. As a result, the tax laws in the U.S. and other countries in which PTC and its affiliates do business could change on a prospective or retroactive basis and any such changes could materially adversely affect the Company’s business. The Company is continuing to evaluate the potential impact on future periods of the Pillar Two Framework, pending legislative adoption by additional individual countries, including those within the EU.

Since 2022, TCJA amendments to Internal Revenue Code (“IRC”) Section 174 have no longer permitted an immediate deduction for research and development expenditures in the tax year that such costs were incurred. Instead, these IRC Section 174 development costs have had to be capitalized and amortized over a 5-year or 15-year period for domestic and foreign expenditures, respectively. On July 4, 2025, the U.S. government enacted the One Big Beautiful Bill Act (“OBBBA”) which generally extended the tax provisions previously enacted by the TCJA that were set to expire, as well as made other changes to federal tax law for multinational corporations. Effective beginning with PTC’s 2025 tax year, the OBBBA restored immediate deductibility of domestic expenditures, while foreign expenditures will continue to be capitalized and amortized over 15 years. Additionally, IRC Section 174A, enacted as part of the OBBBA, allows for the immediate expensing of all remaining unamortized domestic costs either upfront in 2025, or ratably in 2025 and 2026.

Deferred tax assets and liabilities are recognized for the future tax consequences attributable to differences between the financial statement carrying amounts of existing assets and liabilities and their respective tax bases and net operating loss and credit carryforwards. Deferred tax assets and liabilities are measured at rates expected to apply to taxable income in the years in which those temporary differences and carryforwards are expected to be recovered or settled. The effect on deferred tax assets and liabilities of a change in tax rates is recognized in the statement of operations in the period that includes the enactment date. A valuation allowance is recorded when it is not more likely than not that all or a portion of the net deferred tax assets will be realized.

Recently issued accounting standards

In November 2024, the FASB issued ASU 2024-03, Income Statement-Reporting Comprehensive Income- Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses. ASU 2024-03 enhances financial reporting by requiring additional information about specific expense categories in the notes to financial statements at interim and annual reporting periods. The guidance is effective for public business entities for annual periods beginning after December 15, 2026, and interim reporting periods beginning after December 15, 2027. Early adoption is permitted. The Company is currently planning to adopt this guidance when effective. The Company is assessing the impact of the adoption on the Company’s consolidated financial statements and accompanying footnotes but expects the impact will be enhanced disclosures related to income statement expenses.

In September 2025, the FASB issued ASU 2025-06, Intangibles—Goodwill and Other— Internal-Use Software (Subtopic 350-40): Targeted Improvements to the Accounting for Internal-Use Software. ASU 2025-06 enhances accounting for software costs that are accounted for under Subtopic 350-40, Intangibles—Goodwill and Other—Internal-Use Software (referred to as “internal-use software”). The guidance is effective for public business entities for annual periods beginning after December 15, 2027, and interim reporting periods within those annual reporting periods. Entities have three transition options: prospective, retrospective, or modified prospective adoption. Early adoption is permitted as of the beginning of an annual reporting period. Instead of following prescriptive project stages for capitalization of internal-use software, entities will capitalize costs when management has authorized and committed funding for the software project and it is probable that the project will be completed and used as intended. The Company is assessing the impact of the adoption on the Company’s consolidated financial statements and accompanying footnotes.

In December 2025, the FASB issued ASU 2025-11, Interim Reporting (Topic 270): Narrow-Scope Improvements. ASU 2025-11 clarifies and reorganizes existing guidance related to interim financial reporting and disclosures. The guidance is effective for public business entities for interim reporting periods with annual reporting periods beginning after December 15, 2027. The Company is assessing the impact of the adoption on the Company’s consolidated financial statements and accompanying footnotes but expects the guidance will not have a material impact, as the guidance does not change the recognition or measurement of financial statement amounts.

In December 2025, the FASB issued ASU 2025-12, Codification Improvements. ASU 2025-12 makes targeted improvements to the FASB Accounting Standards Codification by clarifying language, correcting technical errors, and addressing unintended codification applications across various topics in the U.S. GAAP. The guidance is effective for public business entities for interim reporting periods with annual reporting periods beginning after December 15, 2026. The Company is assessing the impact of the adoption on the Company’s consolidated financial statements and accompanying footnotes but expects the guidance will not have a material impact, as the guidance does not change the recognition or measurement of financial statement amounts.

Impact of recently adopted accounting pronouncements

In July 2025, the FASB issued ASU 2025-05, Financial Instruments-Credit Losses (Topic 326): Measurement of Credit Losses for Accounts Receivable and Contract Assets. ASU 2025-05 addresses challenges encountered when applying the guidance in Topic 326, Financial Instruments—Credit Losses, to current accounts receivable and current contract assets arising from transactions accounted for under Topic 606, Revenue from Contracts with Customers. The guidance is effective for public business entities for annual periods beginning after December 15, 2025, and interim reporting periods within those annual reporting periods. Early adoption is permitted in both interim and annual reporting periods in which financial statements have not yet been issued or made available for issuance. In developing reasonable and supportable

forecasts as part of estimating expected credit losses, the guidance provides that all entities may elect a practical expedient that assumes that current conditions as of the balance sheet date do not change for the remaining life of the asset. The Company adopted this guidance January 1, 2026. The adoption of this guidance did not have a material impact on the Company’s consolidated financial statements and accompanying footnotes.

In April 2024, the FASB issued ASU 2024-04, Debt—Debt with Conversion and Other Options (Subtopic 470-20): Induced Conversions of Convertible Debt Instruments. ASU 2024-04 improves the relevance and consistency in application of the induced conversion guidance in Subtopic 470-20, Debt—Debt with Conversion and Other Options. The guidance is effective for all entities for annual reporting periods beginning after December 15, 2025, and interim reporting periods within those annual reporting periods. Early adoption is permitted for all entities that have adopted the amendments in Update 2020-06. The Company adopted this guidance January 1, 2026. Refer to Note 9 for additional details and the impact on the Company’s consolidated financial statements.

The Company	6 Months Ended
Jun. 30, 2026
The Company
The Company	1. The Company PTC Therapeutics, Inc. (the “Company” or “PTC”) is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. PTC’s strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for its patients and other stakeholders. PTC has a diversified therapeutic portfolio pipeline that includes several commercial products and product candidates in various stages of development, including clinical, pre-clinical and research and discovery stages, focused on the development of new treatments for multiple therapeutic areas for rare diseases relating to neurology and metabolism. The Company has developed Sephience™ (sepiapterin), a product for the treatment of phenylketonuria (“PKU”), a rare inherited metabolic disease characterized by the body’s inability to break down an essential amino acid called phenylalanine, and which can result in neurological and other symptoms. In June 2025, Sephience was granted marketing authorization by the European Commission (“EC”) for the treatment of children and adults living with PKU within the European Economic Area (“EEA”). In July 2025, Sephience was approved by the U.S. Food and Drug Administration (“FDA”) for the treatment of pediatric and adult patients living with PKU in the United States age one month and above. In December 2025, Sephience was approved by the Japanese Ministry of Health, Labor, and Welfare (“MHLW”) for the treatment of children and adults living with PKU in Japan. In February 2026, Sephience was approved by ANVISA, the Brazilian health regulatory authority, for the treatment of children and adults living with PKU in Brazil. Sephience is also approved in additional geographies. The Company has two products, Translarna™ (ataluren) and Emflaza® (deflazacort), for the treatment of Duchenne muscular dystrophy (“DMD”), a rare, life-threatening disorder. While Translarna previously had conditional approval in the EEA, in March 2025, the EC adopted the negative opinion of the Committee for Medicinal Products for Human Use, or CHMP, of the European Medicines Agency, or EMA, to not renew the conditional marketing authorization of Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy (“nmDMD”). However, the EC indicated that individual countries within the European Union, can leverage Articles 117(3) and 5(1) of the EU Directive 2001/83 to allow continued commercial use of Translarna. Translarna has marketing authorization in additional geographies outside of the EEA, but the EC adoption of the CHMP negative opinion may affect future reauthorizations. Emflaza is approved in the United States for the treatment of DMD in patients two years and older. Translarna is an investigational new drug in the United States. In 2017, the Company filed a new drug application (“NDA”) for Translarna for the treatment of nmDMD over protest with the FDA and the Office of Drug Evaluation I of the FDA issued a complete response letter for the NDA, stating that it was unable to approve the application in its current form. The Company re-submitted the NDA in July 2024 and in October 2024, the FDA accepted for review the resubmission of the NDA for Translarna for the treatment of nmDMD. Following feedback from the FDA, the Company withdrew the NDA resubmission for Translarna in February 2026. Further development of Translarna for the treatment of nmDMD in the United States is not planned. The Company has developed Upstaza™ (eladocagene exuparvovec), a gene therapy used for the treatment of Aromatic L-Amino Acid Decarboxylase (“AADC”) deficiency (“AADC deficiency”), a rare central nervous system (“CNS”) disorder arising from reductions in the enzyme AADC that results from mutations in the dopa decarboxylase gene. In July 2022, the EC approved Upstaza for the treatment of AADC deficiency for patients 18 months and older within the EEA. In November 2022, the Medicines and Healthcare Products Regulatory Agency approved Upstaza for the treatment of AADC deficiency for patients 18 months and older within the United Kingdom. In November 2024, the Company’s biologics license application for its gene therapy treatment of AADC deficiency was approved by the FDA. This gene therapy is marketed under the brand name Kebilidi™ in the United States. The Company holds the rights for the commercialization of Tegsedi® (inotersen) and Waylivra® (volanesorsen) for the treatment of rare diseases in countries in Latin America and the Caribbean pursuant to the Collaboration and License Agreement (the “Tegsedi-Waylivra Agreement”), dated August 1, 2018, by and between the Company and Akcea Therapeutics, Inc. (“Akcea”), a subsidiary of Ionis Pharmaceuticals, Inc. Tegsedi has received marketing authorization in the United States, the European Union (the “EU”) and Brazil for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (“hATTR amyloidosis”). In August 2021, ANVISA, the Brazilian health regulatory authority, approved Waylivra as the first treatment for familial chylomicronemia syndrome (“FCS”) in Brazil. Waylivra has also received marketing authorization in the EU for the treatment of FCS. In December 2022, ANVISA approved Waylivra for the treatment of familial partial lipodystrophy. The Company also has a spinal muscular atrophy (“SMA”) collaboration with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc. (referred to collectively as “Roche”) and the Spinal Muscular Atrophy Foundation (“SMA Foundation”). The SMA program has one approved product, Evrysdi® (risdiplam), which was approved by the FDA in August 2020 for the treatment of SMA in adults and children two months and older and by the EC in March 2021 for the treatment of 5q SMA in patients two months and older with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. Evrysdi has also received marketing authorization for the treatment of SMA in over 100 countries. In May 2022, the FDA approved a label expansion for Evrysdi to include infants under two months old with SMA. In August 2023, the EC approved an extension of the Evrysdi marketing authorization to include infants under two months old in the EU. In addition to the Company’s SMA program, the Company’s splicing platform also includes votoplam (PTC518), which is being developed for the treatment of Huntington’s disease (“HD”). The Company initiated a Phase 2 study of votoplam for the treatment of HD in the first quarter of 2022, which consisted of an initial 12-week placebo-controlled phase focused on safety, pharmacology and pharmacodynamic effects followed by a nine-month placebo-controlled phase focused on votoplam biomarker effect. In June 2024, the Company announced interim results from the full Phase 2 study of votoplam. At month 12, votoplam treatment demonstrated durable dose-dependent lowering of mutant HTT (“mHTT”) protein in the blood and dose-dependent lowering of mHTT protein in the cerebrospinal fluid in the interim cohort of stage 2 patients. In addition, favorable trends were demonstrated on several relevant HD clinical assessments. Furthermore, following 12 months of treatment, votoplam continued to be well tolerated. In September 2024, the FDA granted Fast Track designation to the votoplam program for the treatment of HD. In December 2024, the Company held a Type C meeting with the FDA to discuss whether huntingtin protein lowering could be considered a surrogate endpoint for accelerated approval of votoplam. The FDA was aligned on the scientific rationale and asked to see additional data supportive of an association between huntingtin protein lowering and changes in clinical outcome scores. In May 2025, the Company announced that the Phase 2 study of votoplam met its primary endpoints of blood HTT lowering and safety. The results on the full study population are consistent with the previously reported evidence of dose-dependent HTT lowering, favorable safety profile and early signals of dose-dependent clinical effect at 12 months in Stage 2 patients. In addition, at 24 months of treatment, there were continued trends of dose-dependent favorable clinical effect relative to a propensity-matched natural history cohort as well as dose-dependent Neurofilament light (“NfL”) lowering. In November 2024, the Company entered into a License and Collaboration Agreement (the “Novartis Agreement”) with Novartis Pharmaceuticals Corporation (“Novartis”), relating to its votoplam HD program which included related molecules. This transaction closed in January 2025, triggering a $1.0 billion upfront cash payment to the Company. In April 2026, Novartis announced that it had commenced the global Phase 3 clinical trial, which is expected to enroll approximately 770 individuals with early symptomatic disease, randomized 3:2 to receive votoplam 10 milligrams or placebo, and includes an interim analysis. Pursuant to the Novartis Agreement, the initiation of the Phase 3 clinical trial triggered a $50.0 million milestone payment to the Company. The $50.0 million development milestone is recorded as collaboration and license revenue for the three and six months ending June 30, 2026. Also in April 2026, the Company reported positive topline results from the 24-month interim analysis of the PIVOT-HD long-term extension study, with favorable dose-dependent effects on disease progression for Stage 2 HD patients following 24 months of votoplam treatment compared to an external natural history cohort, with 52% slowing of disease progression on the Composite Unified Huntington’s Disease Rating Scale at the 10 milligram dose level. While the Phase 3 clinical trial remains the base case for votoplam approval, Novartis and the Company are finalizing a plan to engage with FDA to discuss the 24-month results in the second half of 2026. The Company’s inflammation and ferroptosis platform consists of small molecule compounds that target oxidoreductase enzymes that regulate oxidative stress and inflammatory pathways central to the pathology of a number of CNS and non-CNS diseases. The most advanced molecule in the Company’s inflammation and ferroptosis platform is vatiquinone. The Company announced topline results from a registration-directed Phase 3 trial of vatiquinone in children and young adults with Friedreich’s ataxia (“FA”), called MOVE-FA, in May 2023. While the study did not meet its primary endpoint, vatiquinone treatment did demonstrate significant benefit on key disease subscales, including the upright stability subscale, as well as on other disease relevant endpoints. In October 2024, the Company announced that the pre-specified endpoint for two different FA long-term extension studies was met, with statistically significant evidence of durable treatment benefit on disease progression. In December 2024, the Company submitted an NDA to the FDA for vatiquinone for the treatment of children and adults living with FA. In February 2025, the FDA accepted for filing the NDA. In August 2025, the FDA issued a complete response letter related to the NDA stating that substantial evidence of efficacy was not demonstrated for vatiquinone and that an additional adequate and well-controlled study would be needed to support NDA resubmission. The Company met with the FDA in the fourth quarter of 2025 to discuss the vatiquinone development program, at which time the FDA suggested an additional study be conducted to support NDA resubmission. In April 2026, the Company again met with FDA to discuss the design of a new trial to provide additional data to support NDA resubmission. Based on the meeting discussion and written feedback, the Company plans to initiate the PROVE-FA open label study using matched natural history control in the third quarter of 2026. This study is expected to enroll approximately 120 patients ages 7 to 21 and the study primary endpoint is the change in mFARS from baseline to month 24. During the quarter ended June 30, 2026, the Company initiated a Phase 1 study of PTC612, its oral NLRP3 inhibitor, and completed several of the single and multiple ascending dose treatment cohorts. Notably, this healthy volunteer study includes a cohort of individuals with obesity and cardiovascular disease, which the Company expects will provide an early view of pharmacokinetics and pharmacodynamics. The Company expects to initiate a Phase 2a study of PTC844, its next-generation DHODH inhibitor, in the third quarter of 2026. The PTC844 study will be a 12-week pharmacokinetics and pharmacodynamics study in which the Company will assess treatment effect on biomarkers related to T-cell and B-cell immunity. The Company expects that the results of this study will help inform the ultimate target indications for PTC844. In addition, the Company has a pipeline of product candidates and discovery programs that are in early clinical, pre-clinical and research and development stages focused on the development of new treatments for multiple therapeutic areas for rare diseases. As of June 30, 2026, the Company had an accumulated deficit of approximately $2,883.5 million. The Company has financed its operations to date primarily through the private offerings of convertible senior notes (see Note 9), public and “at the market” offerings of common stock, proceeds from royalty purchase agreements (see Note 9), private placements of its convertible preferred stock and common stock, collaborations, bank and institutional lender debt, other convertible debt, grant funding and clinical trial support from governmental and philanthropic organizations and patient advocacy groups in the disease areas addressed by the Company’s product candidates. The Company has also relied on revenue generated from net sales of its products, the revenue associated with milestone and royalty payments from Roche pursuant to the License and Collaboration Agreement (the “SMA License Agreement”) dated as of November 23, 2011, by and among the Company, Roche and, for the limited purposes set forth therein, the SMA Foundation, under its SMA program, and the revenues associated with the Novartis Agreement. The Company expects that cash flows from the sales of its products, and milestone payments from Novartis, together with the Company’s cash, cash equivalents and marketable securities, will be sufficient to fund its operations for at least the next twelve months after the issuance date of these financial statements.

The Company

6 Months Ended

Jun. 30, 2026

The Company

The Company

1. The Company

PTC Therapeutics, Inc. (the “Company” or “PTC”) is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. PTC’s strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for its patients and other stakeholders. PTC has a diversified therapeutic portfolio pipeline that includes several commercial products and product candidates in various stages of development, including clinical, pre-clinical and research and discovery stages, focused on the development of new treatments for multiple therapeutic areas for rare diseases relating to neurology and metabolism.

The Company has developed Sephience™ (sepiapterin), a product for the treatment of phenylketonuria (“PKU”), a rare inherited metabolic disease characterized by the body’s inability to break down an essential amino acid called phenylalanine, and which can result in neurological and other symptoms. In June 2025, Sephience was granted marketing authorization by the European Commission (“EC”) for the treatment of children and adults living with PKU within the European Economic Area (“EEA”). In July 2025, Sephience was approved by the U.S. Food and Drug Administration (“FDA”) for the treatment of pediatric and adult patients living with PKU in the United States age one month and above. In December 2025, Sephience was approved by the Japanese Ministry of Health, Labor, and Welfare (“MHLW”) for the treatment of children and adults living with PKU in Japan. In February 2026, Sephience was approved by ANVISA, the Brazilian health regulatory authority, for the treatment of children and adults living with PKU in Brazil. Sephience is also approved in additional geographies.

The Company has two products, Translarna™ (ataluren) and Emflaza® (deflazacort), for the treatment of Duchenne muscular dystrophy (“DMD”), a rare, life-threatening disorder. While Translarna previously had conditional approval in the EEA, in March 2025, the EC adopted the negative opinion of the Committee for Medicinal Products for Human Use, or CHMP, of the European Medicines Agency, or EMA, to not renew the conditional marketing authorization of Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy (“nmDMD”). However, the EC indicated that individual countries within the European Union, can leverage Articles 117(3) and 5(1) of the EU Directive 2001/83 to allow continued commercial use of Translarna. Translarna has marketing authorization in additional geographies outside of the EEA, but the EC adoption of the CHMP negative opinion may affect future reauthorizations. Emflaza is approved in the United States for the treatment of DMD in patients two years and older.

Translarna is an investigational new drug in the United States. In 2017, the Company filed a new drug application (“NDA”) for Translarna for the treatment of nmDMD over protest with the FDA and the Office of Drug Evaluation I of the FDA issued a complete response letter for the NDA, stating that it was unable to approve the application in its current form. The Company re-submitted the NDA in July 2024 and in October 2024, the FDA accepted for review the resubmission of the NDA for Translarna for the treatment of nmDMD. Following feedback from the FDA, the Company withdrew the NDA resubmission for Translarna in February 2026. Further development of Translarna for the treatment of nmDMD in the United States is not planned.

The Company has developed Upstaza™ (eladocagene exuparvovec), a gene therapy used for the treatment of Aromatic L-Amino Acid Decarboxylase (“AADC”) deficiency (“AADC deficiency”), a rare central nervous system (“CNS”) disorder arising from reductions in the enzyme AADC that results from mutations in the dopa decarboxylase gene. In July 2022, the EC approved Upstaza for the treatment of AADC deficiency for patients 18 months and older within the EEA. In November 2022, the Medicines and Healthcare Products Regulatory Agency approved Upstaza for the treatment of AADC deficiency for patients 18 months and older within the United Kingdom. In November 2024, the Company’s biologics license application for its gene therapy treatment of AADC deficiency was approved by the FDA. This gene therapy is marketed under the brand name Kebilidi™ in the United States.

The Company holds the rights for the commercialization of Tegsedi® (inotersen) and Waylivra® (volanesorsen) for the treatment of rare diseases in countries in Latin America and the Caribbean pursuant to the Collaboration and License Agreement (the “Tegsedi-Waylivra Agreement”), dated August 1, 2018, by and between the Company and Akcea Therapeutics, Inc. (“Akcea”), a subsidiary of Ionis Pharmaceuticals, Inc. Tegsedi has received marketing authorization in the United States, the European Union (the “EU”) and Brazil for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (“hATTR amyloidosis”). In August 2021, ANVISA, the Brazilian health regulatory authority, approved Waylivra as the first treatment for familial chylomicronemia syndrome (“FCS”) in Brazil. Waylivra has also received marketing authorization in the EU for the treatment of FCS. In December 2022, ANVISA approved Waylivra for the treatment of familial partial lipodystrophy.

The Company also has a spinal muscular atrophy (“SMA”) collaboration with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc. (referred to collectively as “Roche”) and the Spinal Muscular Atrophy Foundation (“SMA Foundation”). The SMA program has one approved product, Evrysdi® (risdiplam), which was approved by the FDA in August 2020 for the treatment of SMA in adults and children two months and older and by the EC in March 2021 for the treatment of 5q SMA in patients two months and older with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. Evrysdi has also received marketing authorization for the treatment of SMA in over 100 countries. In May 2022, the FDA approved a label expansion for Evrysdi to include infants under two months old with SMA. In August 2023, the EC approved an extension of the Evrysdi marketing authorization to include infants under two months old in the EU.

In addition to the Company’s SMA program, the Company’s splicing platform also includes votoplam (PTC518), which is being developed for the treatment of Huntington’s disease (“HD”). The Company initiated a Phase 2 study of votoplam for the treatment of HD in the first quarter of 2022, which consisted of an initial 12-week placebo-controlled phase focused on safety, pharmacology and pharmacodynamic effects followed by a nine-month placebo-controlled phase focused on votoplam biomarker effect. In June 2024, the Company announced interim results from the full Phase 2 study of votoplam. At month 12, votoplam treatment demonstrated durable dose-dependent lowering of mutant HTT (“mHTT”) protein in the blood and dose-dependent lowering of mHTT protein in the cerebrospinal fluid in the interim cohort of stage 2 patients. In addition, favorable trends were demonstrated on several relevant HD clinical assessments. Furthermore, following 12 months of treatment, votoplam continued to be well tolerated. In September 2024, the FDA granted Fast Track designation to the votoplam program for the treatment of HD. In December 2024, the Company held a Type C meeting with the FDA to discuss whether huntingtin protein lowering could be considered a surrogate endpoint for accelerated approval of votoplam. The FDA was aligned on the scientific rationale and asked to see additional data supportive of an association between huntingtin protein lowering and changes in clinical outcome scores. In May 2025, the Company announced that the Phase 2 study of votoplam met its primary endpoints of blood HTT lowering and safety. The results on the full study population are consistent with the previously reported evidence of dose-dependent HTT lowering, favorable safety profile and early signals of dose-dependent clinical effect at 12 months in Stage 2 patients. In addition, at 24 months of treatment, there were continued trends of dose-dependent favorable clinical effect relative to a propensity-matched natural history cohort as well as dose-dependent Neurofilament light (“NfL”) lowering. In November 2024, the Company entered into a License and Collaboration Agreement (the “Novartis Agreement”) with Novartis Pharmaceuticals Corporation (“Novartis”), relating to its votoplam HD program which included related molecules. This transaction closed in January 2025, triggering a $1.0 billion upfront cash payment to the Company. In April 2026, Novartis announced that it had commenced the global Phase 3 clinical trial, which is expected to enroll approximately 770 individuals with early symptomatic disease, randomized 3:2 to receive votoplam 10 milligrams or placebo, and includes an interim analysis. Pursuant to the Novartis Agreement, the initiation of the Phase 3 clinical trial triggered a $50.0 million milestone payment to the Company. The $50.0 million development milestone is recorded as collaboration and license revenue for the three and six months ending June 30, 2026. Also in April 2026, the Company reported positive topline results from the 24-month interim analysis of the PIVOT-HD long-term extension study, with favorable dose-dependent effects on disease progression for Stage 2 HD patients following 24 months of votoplam treatment compared to an external natural history cohort, with 52% slowing of disease progression on the Composite Unified Huntington’s Disease Rating Scale at the 10 milligram dose level. While the Phase 3 clinical trial remains the base case for votoplam approval, Novartis and the Company are finalizing a plan to engage with FDA to discuss the 24-month results in the second half of 2026.

The Company’s inflammation and ferroptosis platform consists of small molecule compounds that target oxidoreductase enzymes that regulate oxidative stress and inflammatory pathways central to the pathology of a number of CNS and non-CNS diseases. The most advanced molecule in the Company’s inflammation and ferroptosis platform is vatiquinone. The

Company announced topline results from a registration-directed Phase 3 trial of vatiquinone in children and young adults with Friedreich’s ataxia (“FA”), called MOVE-FA, in May 2023. While the study did not meet its primary endpoint, vatiquinone treatment did demonstrate significant benefit on key disease subscales, including the upright stability subscale, as well as on other disease relevant endpoints. In October 2024, the Company announced that the pre-specified endpoint for two different FA long-term extension studies was met, with statistically significant evidence of durable treatment benefit on disease progression. In December 2024, the Company submitted an NDA to the FDA for vatiquinone for the treatment of children and adults living with FA. In February 2025, the FDA accepted for filing the NDA. In August 2025, the FDA issued a complete response letter related to the NDA stating that substantial evidence of efficacy was not demonstrated for vatiquinone and that an additional adequate and well-controlled study would be needed to support NDA resubmission. The Company met with the FDA in the fourth quarter of 2025 to discuss the vatiquinone development program, at which time the FDA suggested an additional study be conducted to support NDA resubmission. In April 2026, the Company again met with FDA to discuss the design of a new trial to provide additional data to support NDA resubmission. Based on the meeting discussion and written feedback, the Company plans to initiate the PROVE-FA open label study using matched natural history control in the third quarter of 2026. This study is expected to enroll approximately 120 patients ages 7 to 21 and the study primary endpoint is the change in mFARS from baseline to month 24.

During the quarter ended June 30, 2026, the Company initiated a Phase 1 study of PTC612, its oral NLRP3 inhibitor, and completed several of the single and multiple ascending dose treatment cohorts. Notably, this healthy volunteer study includes a cohort of individuals with obesity and cardiovascular disease, which the Company expects will provide an early view of pharmacokinetics and pharmacodynamics. The Company expects to initiate a Phase 2a study of PTC844, its next-generation DHODH inhibitor, in the third quarter of 2026. The PTC844 study will be a 12-week pharmacokinetics and pharmacodynamics study in which the Company will assess treatment effect on biomarkers related to T-cell and B-cell immunity. The Company expects that the results of this study will help inform the ultimate target indications for PTC844.

In addition, the Company has a pipeline of product candidates and discovery programs that are in early clinical, pre-clinical and research and development stages focused on the development of new treatments for multiple therapeutic areas for rare diseases.

As of June 30, 2026, the Company had an accumulated deficit of approximately $2,883.5 million. The Company has financed its operations to date primarily through the private offerings of convertible senior notes (see Note 9), public and “at the market” offerings of common stock, proceeds from royalty purchase agreements (see Note 9), private placements of its convertible preferred stock and common stock, collaborations, bank and institutional lender debt, other convertible debt, grant funding and clinical trial support from governmental and philanthropic organizations and patient advocacy groups in the disease areas addressed by the Company’s product candidates. The Company has also relied on revenue generated from net sales of its products, the revenue associated with milestone and royalty payments from Roche pursuant to the License and Collaboration Agreement (the “SMA License Agreement”) dated as of November 23, 2011, by and among the Company, Roche and, for the limited purposes set forth therein, the SMA Foundation, under its SMA program, and the revenues associated with the Novartis Agreement. The Company expects that cash flows from the sales of its products, and milestone payments from Novartis, together with the Company’s cash, cash equivalents and marketable securities, will be sufficient to fund its operations for at least the next twelve months after the issuance date of these financial statements.

Revenue recognition - Disaggregated Net Product Revenue by Country (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025
Revenue recognition
Total revenues	$ 360,519	$ 178,875	$ 633,070	$ 1,354,971
Product
Revenue recognition
Total revenues	238,819	118,329	464,392	271,755
United States
Revenue recognition
Total revenues	152,184	36,353	288,700	84,142
United States \| Product
Revenue recognition
Total revenues	152,184	36,353	288,700	84,142
Brazil
Revenue recognition
Total revenues	25,581	18,124	28,830	56,638
Russia
Revenue recognition
Total revenues	8,210	40,951	49,422	50,456
All other countries
Revenue recognition
Total revenues	$ 52,844	$ 22,901	$ 97,440	$ 80,519

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

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Amount, excluding tax collected from customer, of revenue from satisfaction of performance obligation by transferring promised good or service to customer. Tax collected from customer is tax assessed by governmental authority that is both imposed on and concurrent with specific revenue-producing transaction, including, but not limited to, sales, use, value added and excise.

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+ Details

Net income (loss) per share - Numerator and Denominator (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025
Numerator:
Net income (loss), basic	$ 83,496	$ (64,849)	$ 80,687	$ 801,713
Add: Interest expense, net of tax, on the Company's 2026 and 2031 Convertible Notes	959		2,045	2,161
Net income (loss), diluted	$ 84,455	$ (64,849)	$ 82,732	$ 803,874
Denominator:
Weighted-average number of shares outstanding, basic	83,006,808	78,151,240	82,765,248	78,438,830
Common stock issuable under the Company's equity incentive plans	3,551,404		3,591,103	2,589,633
Common stock issuable under the Company's 2026 and 2031 Convertible Notes	5,461,797		5,467,922	5,474,115
Weighted-average common shares outstanding, diluted	92,020,009	78,151,240	91,824,273	86,502,578
Net income (loss) per common share, basic	$ 1.01	$ (0.83)	$ 0.97	$ 10.22
Net income (loss) per common share, diluted	$ 0.92	$ (0.83)	$ 0.9	$ 9.29

Net income (loss) per share

6 Months Ended

Jun. 30, 2026

Net income (loss) per share

Net income (loss) per share

7. Net income (loss) per share

Basic and diluted net income (loss) per share is computed by dividing net income (loss) by the weighted-average number of common shares outstanding.

The following tables set forth the computation of basic and diluted net income (loss) per share:


	Three Months Ended June 30,					Six Months Ended June 30,
	2026		2025			2026		2025
Numerator:
Net income (loss), basic	$	83,496	$	(64,849)		$	80,687	$	801,713
Add: Interest expense, net of tax, on the Company's 2026 and 2031 Convertible Notes		959		—			2,045		2,161
Net income (loss), diluted	$	84,455	$	(64,849)		$	82,732	$	803,874
Denominator:
Weighted-average number of shares outstanding, basic		83,006,808		78,151,240			82,765,248		78,438,830
Effect of dilutive securities:
Common stock issuable under the Company's equity incentive plans		3,551,404		—			3,591,103		2,589,633
Common stock issuable under the Company's 2026 and 2031 Convertible Notes		5,461,797		—			5,467,922		5,474,115
Weighted-average common shares outstanding, diluted		92,020,009		78,151,240			91,824,273		86,502,578
Net income (loss) per common share, basic	$	1.01	$	(0.83)	*	$	0.97	$	10.22
Net income (loss) per common share, diluted	$	0.92	$	(0.83)	*	$	0.90	$	9.29

* In the three months ended June 30, 2025, the Company experienced a net loss and therefore did not report any dilutive share impact.

The following table shows historical common share equivalents outstanding, which are not included in the above calculation, as the effect of their inclusion is anti-dilutive during each period. The anti-dilutive shares are calculated as the unweighted outstanding shares as of the reporting period end date that are antidilutive on a year to date basis using the treasury stock method.


	As of June 30,
	2026	2025
Stock options	807,769	3,494,565
Unvested restricted stock units	1,206,529	83,657
Unvested performance based restricted stock units	93,478	—
Total	2,107,776	3,578,222

Summary of significant accounting policies (Tables)

6 Months Ended

Jun. 30, 2026

Summary of significant accounting policies

Schedule of reconciliation of cash


		End of			Beginning of
		period-			period-
		June 30,			December 31,
		2026			2025
Cash and cash equivalents		$	1,049,887		$	984,648
Restricted cash included in deposits and other assets			16,445			13,686
Total Cash, cash equivalents and restricted cash per consolidated statement of cash flows		$	1,066,332		$	998,334

Stock award plan (Tables)

6 Months Ended

Jun. 30, 2026

Stock award plan

Summary of stock option activity


							Weighted-
				Weighted-			average		Aggregate
				average			remaining		intrinsic
		Number of		exercise			contractual		value(in
		options		price			term		thousands)

Outstanding at December 31, 2025		6,396,202		$	43.02
Granted		757,556		$	76.44
Exercised		(506,463)		$	38.80
Forfeited/Cancelled		(21,132)		$	43.72
Outstanding at June 30, 2026		6,626,163		$	47.16		5.84	years	$	227,986
Expected to vest at June 30, 2026		1,593,265		$	54.41		8.62	years	$	43,276
Exercisable at June 30, 2026		4,855,637		$	44.32		4.81	years	$	180,891

Schedule of assumptions used to estimate fair values of grants made on the date of grant


		Six months ended
		June 30, 2026
Risk-free interest rate		3.78% - 4.21%
Expected volatility		54%
Expected term		5.5 years

Summary of information on the Company's restricted stock


	Restricted Stock Units
		Weighted
		Average
		Grant
	Number of	Date
	Shares	Fair Value
Unvested at December 31, 2025	3,667,130	$	38.20
Granted	1,293,798		76.53
Performance based PSUs with conditions satisfied	25,000		46.37
Vested	(1,294,464)		38.16
Forfeited	(169,015)		45.16
Unvested at June 30, 2026	3,522,449	$	52.02

Schedule of information about Performance-based restricted stock units


		Performance-based Restricted Stock Units

	Performance Conditions	Market Conditions	Total
Total PSUs granted for which the performance or market conditions were not yet satisfied at December 31, 2025	25,000	115,625	140,625
PSUs with performance or market conditions granted during the period	37,500	—	37,500
PSUs with performance or market conditions satisfied during the period	(25,000)	—	(25,000)
Total PSUs granted for which the performance or market conditions were not yet satisfied at June 30, 2026	37,500	115,625	153,125

Schedule of share-based compensation expense recorded in the statement of operations


	Three Months Ended June 30,				Six Months Ended June 30,
	2026		2025		2026		2025
Research and development	$	10,547	$	9,030	$	21,677	$	17,693
Selling, general and administrative		12,744		9,513		25,035		18,910
Total	$	23,291	$	18,543	$	46,712	$	36,603

Fair value of financial instruments and marketable securities - Marketable Securities, Balance Sheet Disclosures (Details) - USD ($) $ in Thousands	Jun. 30, 2026	Dec. 31, 2025
Fair value of financial instruments and marketable securities
Marketable securities, Less Than 12 Months	$ 1,135,932	$ 929,127
Marketable securities, More Than 12 Months	11,632
Commercial paper
Fair value of financial instruments and marketable securities
Marketable securities, Less Than 12 Months	134,908	93,112
Corporate debt securities
Fair value of financial instruments and marketable securities
Marketable securities, Less Than 12 Months	498,443	279,262
Marketable securities, More Than 12 Months	11,632
Government obligations
Fair value of financial instruments and marketable securities
Marketable securities, Less Than 12 Months	$ 502,581	$ 556,753

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (aa)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (aaa)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

+ Details

The Company (Details) $ in Thousands	1 Months Ended		3 Months Ended	6 Months Ended
The Company (Details) $ in Thousands	Apr. 30, 2026 USD ($) age item	Jan. 31, 2025 USD ($)	Jun. 30, 2026 USD ($)	Jun. 30, 2026 USD ($) product	Dec. 31, 2025 USD ($)
Collaboration and royalty revenue
Number of products \| product				2
Accumulated deficit			$ (2,883,542)	$ (2,883,542)	$ (2,964,229)
PROVE-FA
Collaboration and royalty revenue
Number of individuals expected to enroll \| item	120
Study duration, clinical trial	84 days
PROVE-FA \| Minimum
Collaboration and royalty revenue
Eligible age, clinical trial \| age	7
PROVE-FA \| Maximum
Collaboration and royalty revenue
Eligible age, clinical trial \| age	21
Novartis
Collaboration and royalty revenue
Development milestone obligation revenue received				50,000
License and Collaboration Agreement \| Novartis
Collaboration and royalty revenue
Cash proceeds from upfront payment on collaboration agreement		$ 1,000,000
License and Collaboration Agreement \| Novartis \| Phase 3 Votoplam trial
Collaboration and royalty revenue
Number of individuals expected to enroll \| item	770
Randomization ratio, clinical trial	1.5
Treatment dosage, clinical trial (milligrams) \| item	10
Development milestone obligation revenue received	$ 50,000		$ 50,000	$ 50,000
Interim analysis period, clinical trial	24 months
Treatment comparison period, clinical trial	24 months
Disease progression slowing percentage	52

Fair value of financial instruments and marketable securities - Hierarchy (Details) - USD ($) $ in Thousands	Jun. 30, 2026	Dec. 31, 2025
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities - available for sale	$ 1,147,564	$ 929,127
Transfers of assets measured between Level 1, Level 2, and Level 3	0	0
Recurring basis
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities - available for sale	1,147,564	929,127
Marketable securities - equity investments	31,631	31,596
Recurring basis \| Quoted prices in active markets for identical assets (Level 1)
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities - equity investments	31,631	31,596
Recurring basis \| Significant other observable inputs (Level 2)
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities - available for sale	$ 1,147,564	$ 929,127

- Definition

Amount of investment in debt security measured at fair value with change in fair value recognized in other comprehensive income (available-for-sale).

+ References

Reference 1: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (bbb)(2)(i)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 2: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (bbb)(2)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 3: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (bbb)(1)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 4: http://www.xbrl.org/2003/role/exampleRef
-Name Accounting Standards Codification
-Section 55
-Paragraph 10
-SubTopic 10
-Topic 825
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482881/825-10-55-10

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 825
-SubTopic 10
-Name Accounting Standards Codification
-Section 45
-Paragraph 1A
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482736/825-10-45-1A

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 825
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 11
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482907/825-10-50-11

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 825
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 11
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482907/825-10-50-11

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 825
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 10
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482907/825-10-50-10

Reference 9: http://www.xbrl.org/2003/role/exampleRef
-Topic 825
-SubTopic 10
-Name Accounting Standards Codification
-Section 55
-Paragraph 12
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482881/825-10-55-12

Reference 10: http://www.xbrl.org/2003/role/exampleRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 55
-Paragraph 103
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482078/820-10-55-103

Reference 11: http://www.xbrl.org/2003/role/exampleRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 55
-Paragraph 100
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482078/820-10-55-100

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (aa)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 14: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 45
-Paragraph 1
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481830/320-10-45-1

Reference 15: http://www.xbrl.org/2003/role/disclosureRef
-Topic 942
-SubTopic 210
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.9-03(6))
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147478546/942-210-S99-1

Reference 16: http://www.xbrl.org/2003/role/disclosureRef
-Topic 326
-SubTopic 30
-Name Accounting Standards Codification
-Section 45
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479130/326-30-45-1

+ Details

Summary of significant accounting policies - Reconciliation of cash (Details) - USD ($) $ in Thousands	Jun. 30, 2026	Dec. 31, 2025	Jun. 30, 2025	Dec. 31, 2024
Summary of significant accounting policies
Cash and cash equivalents	$ 1,049,887	$ 984,648
Restricted cash included in deposits and other assets	16,445	13,686
Total Cash, cash equivalents and restricted cash per consolidated statement of cash flows	$ 1,066,332	$ 998,334	$ 1,033,069	$ 795,316

Summary of significant accounting policies - Narrative (Details) - USD ($)		3 Months Ended		6 Months Ended
	Dec. 22, 2017	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025	Dec. 31, 2028	Jul. 01, 2027	Dec. 31, 2025
Summary of significant accounting policies
Collateral account in connection with company's corporate card program		$ 2,800,000		$ 2,800,000
Restricted Cash, Noncurrent, Statement of Financial Position [Extensible Enumeration]		Deposits and Other Assets Noncurrent		Deposits and Other Assets Noncurrent
Allowance for credit loss		$ 0	$ 0	$ 0	$ 0
Allowance for doubtful accounts receivable		15,600,000		15,600,000				$ 15,200,000
Bad debt expense		100,000	$ 5,900,000	800,000	$ 7,800,000
Income taxes
Federal income tax statutory rate	21.00%
Deposits and other assets
Summary of significant accounting policies
Bank guarantee denominated in foreign currency		700,000		700,000
Hopewell Campus \| Deposits and other assets
Summary of significant accounting policies
Letters of credit		3,000,000		3,000,000
Warren Building under lease amendment
Summary of significant accounting policies
Letters of credit						$ 2,500,000	$ 5,000,000
Warren Building under lease amendment \| Deposits and other assets
Summary of significant accounting policies
Letters of credit		$ 10,000,000		$ 10,000,000

Inventory

6 Months Ended

Jun. 30, 2026

Inventory

Inventory

3. Inventory

The following table summarizes the components of the Company’s inventory for the periods indicated:


		June 30, 2026			December 31, 2025
Raw materials		$	10,521		$	2,291
Work in progress			57,775			55,686
Finished goods			23,058			21,670
Total inventory		$	91,354		$	79,647

The Company periodically reviews its inventories for excess amounts or obsolescence and writes down obsolete or otherwise unmarketable inventory to its estimated net realizable value. For the three and six months ended June 30, 2026, the Company recorded inventory write-downs of $0.9 million and $2.7 million, respectively, primarily related to adjustments to inventory reserves and product approaching expiration. For the three and six months ended June 30, 2025, the Company recorded inventory write-downs of $0.8 million and $4.3 million, respectively. Additionally, though the Company’s product is subject to strict quality control and monitoring which it performs throughout the manufacturing processes, certain batches or units of product may not meet quality specifications resulting in a charge to cost of product, collaboration and license sales. For the three and six months ended June 30, 2026 and 2025, these amounts were immaterial. For the six months ended June 30, 2026, the Company recorded a $0.8 million loss related to inventory impairments, which is recorded as tangible asset impairment and losses on transactions, net on the statements of operations. No inventory impairments were recorded for the three months ended June 30, 2026 and for the three and six months ended June 30, 2025.

Revenue recognition (Tables)

6 Months Ended

Jun. 30, 2026

Revenue recognition

Schedule of disaggregation of net product revenue


	Three Months Ended June 30,
		2026					2025
(in thousands)		United States		International		Total		United States		International		Total
Sephience	$	127,550	$	23,760	$	151,310	$	—	$	—	$	—
Translarna		—		42,217		42,217		—		59,470		59,470
Emflaza		24,634		—		24,634		36,353		—		36,353
Upstaza/Kebilidi		—		11,163		11,163		—		11,889		11,889
All other products		—		9,495		9,495		—		10,617		10,617
Total net product revenue	$	152,184	$	86,635	$	238,819	$	36,353	$	81,976	$	118,329



	Six Months Ended June 30,
	2026						2025
		United States		International		Total		United States		International		Total
Sephience	$	239,590	$	36,271	$	275,861	$	—	$	—	$	—
Translarna		—		101,193		101,193		—		145,624		145,624
Emflaza		46,112		—		46,112		84,142		—		84,142
Upstaza/Kebilidi		2,998		17,759		20,757		—		20,547		20,547
All other products		—		20,469		20,469		—		21,442		21,442
Total net product revenue	$	288,700	$	175,692	$	464,392	$	84,142	$	187,613	$	271,755


	Three Months Ended June 30,				Six Months Ended June 30,
(in thousands)	2026		2025		2026		2025
United States	$	152,184	$	36,353	$	288,700	$	84,142
Russia		25,581		18,124		28,830		56,638
Brazil		8,210		40,951		49,422		50,456
All other countries		52,844		22,901		97,440		80,519
Total net product revenue	$	238,819	$	118,329	$	464,392	$	271,755

Fair value of financial instruments and marketable securities - Marketable Securities, Unrealized Gains (Losses) (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025	Dec. 31, 2025
Fair value of financial instruments and marketable securities
Amortized Cost	$ 1,148,927		$ 1,148,927		$ 928,340
Gross Unrealized, Gains	8		8		805
Gross Unrealized, Losses	(1,371)		(1,371)		(18)
Fair Value	1,147,564		1,147,564		929,127
Write downs of available for sale debt securities	0	$ 0	0	$ 0
Increase in allowance for credit losses	0	$ 0	0	$ 0
Commercial paper
Fair value of financial instruments and marketable securities
Amortized Cost	134,989		134,989		93,113
Gross Unrealized, Gains	1		1		8
Gross Unrealized, Losses	(82)		(82)		(9)
Fair Value	134,908		134,908		93,112
Corporate debt securities
Fair value of financial instruments and marketable securities
Amortized Cost	510,870		510,870		279,090
Gross Unrealized, Gains	2		2		181
Gross Unrealized, Losses	(797)		(797)		(9)
Fair Value	510,075		510,075		279,262
Government obligations
Fair value of financial instruments and marketable securities
Amortized Cost	503,068		503,068		556,137
Gross Unrealized, Gains	5		5		616
Gross Unrealized, Losses	(492)		(492)
Fair Value	$ 502,581		$ 502,581		$ 556,753

Accounts payable and accrued expenses - Components (Details) - USD ($) $ in Thousands	Jun. 30, 2026	Dec. 31, 2025	Jun. 30, 2025
Accounts payable and accrued expenses
Employee compensation, benefits, and related accruals	$ 39,303	$ 73,737
Income tax payable		4,677
Consulting and contracted research	16,395	25,007
Sales allowance	119,562	129,197
Sales rebates	109,741	76,704
Royalties	13,580	9,574
Accounts payable	40,447	45,487
Milestone Payable	30,000	416	$ 25,150
Other	13,217	16,473
Total	$ 382,245	$ 381,272

Net income (loss) per share (Tables)

6 Months Ended

Jun. 30, 2026

Net income (loss) per share

Schedule of computation of basic and diluted net income (loss) per share


	Three Months Ended June 30,					Six Months Ended June 30,
	2026		2025			2026		2025
Numerator:
Net income (loss), basic	$	83,496	$	(64,849)		$	80,687	$	801,713
Add: Interest expense, net of tax, on the Company's 2026 and 2031 Convertible Notes		959		—			2,045		2,161
Net income (loss), diluted	$	84,455	$	(64,849)		$	82,732	$	803,874
Denominator:
Weighted-average number of shares outstanding, basic		83,006,808		78,151,240			82,765,248		78,438,830
Effect of dilutive securities:
Common stock issuable under the Company's equity incentive plans		3,551,404		—			3,591,103		2,589,633
Common stock issuable under the Company's 2026 and 2031 Convertible Notes		5,461,797		—			5,467,922		5,474,115
Weighted-average common shares outstanding, diluted		92,020,009		78,151,240			91,824,273		86,502,578
Net income (loss) per common share, basic	$	1.01	$	(0.83)	*	$	0.97	$	10.22
Net income (loss) per common share, diluted	$	0.92	$	(0.83)	*	$	0.90	$	9.29

* In the three months ended June 30, 2025, the Company experienced a net loss and therefore did not report any dilutive share impact.

Schedule of historical dilutive common share equivalents outstanding


	As of June 30,
	2026	2025
Stock options	807,769	3,494,565
Unvested restricted stock units	1,206,529	83,657
Unvested performance based restricted stock units	93,478	—
Total	2,107,776	3,578,222

Revenue recognition - Net Product Revenue (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025
Revenue recognition
Total revenues	$ 360,519	$ 178,875	$ 633,070	$ 1,354,971
United States
Revenue recognition
Total revenues	152,184	36,353	288,700	84,142
Product
Revenue recognition
Total revenues	238,819	118,329	464,392	271,755
Product \| United States
Revenue recognition
Total revenues	152,184	36,353	288,700	84,142
Product \| International
Revenue recognition
Total revenues	86,635	81,976	175,692	187,613
Sephience
Revenue recognition
Total revenues	151,310		275,861
Sephience \| United States
Revenue recognition
Total revenues	127,550		239,590
Sephience \| International
Revenue recognition
Total revenues	23,760		36,271
Translarna
Revenue recognition
Total revenues	42,217	59,470	101,193	145,624
Translarna \| International
Revenue recognition
Total revenues	42,217	59,470	101,193	145,624
Emflaza
Revenue recognition
Total revenues	24,634	36,353	46,112	84,142
Emflaza \| United States
Revenue recognition
Total revenues	24,634	36,353	46,112	84,142
Upstaza/Kebilidi
Revenue recognition
Total revenues	11,163	11,889	20,757	20,547
Upstaza/Kebilidi \| United States
Revenue recognition
Total revenues			2,998
Upstaza/Kebilidi \| International
Revenue recognition
Total revenues	11,163	11,889	17,759	20,547
All other products
Revenue recognition
Total revenues	9,495	10,617	20,469	21,442
All other products \| International
Revenue recognition
Total revenues	$ 9,495	$ 10,617	$ 20,469	$ 21,442

Intangible assets and goodwill - Definite-lived Intangibles (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025	Dec. 31, 2025	Aug. 05, 2025
Definite-lived intangible assets
Accrued royalties	$ 13,580		$ 13,580		$ 9,574
Total definite-lived intangibles, net	402,926		402,926
Definite-lived intangibles
Amortization	$ (11,841)	$ (4,061)	$ (23,422)	$ (7,859)
Weighted Average
Definite-lived intangible assets
Remaining amortization period	10 years 8 months 12 days		10 years 8 months 12 days
Censa Merger Agreement
Definite-lived intangible assets
Maximum Upfront Consideration Agreed To Be Paid	$ 250,000		$ 250,000
Rights Satisfaction Agreement
Definite-lived intangible assets
Maximum Upfront Consideration Agreed To Be Paid						$ 250,000
Akcea \| Waylivra
Definite-lived intangible assets
Royalty payments			2,900
Akcea \| Waylivra \| Accounts payable and accrued expenses
Definite-lived intangible assets
Accrued royalties	400		400
Akcea \| Tegsedi
Definite-lived intangible assets
Royalty payments			3,200
Akcea \| Tegsedi \| Accounts payable and accrued expenses
Definite-lived intangible assets
Accrued royalties	2,000		2,000
Censa \| Waylivra
Definite-lived intangible assets
Royalty payments			2,900
Censa \| Tegsedi
Definite-lived intangible assets
Royalty payments			3,200
Censa \| Sephience
Definite-lived intangible assets
Royalty payments			2,200
Censa \| Sephience \| Accounts payable and accrued expenses
Definite-lived intangible assets
Accrued royalties	2,200		2,200
Censa \| Censa Merger Agreement
Definite-lived intangible assets
Milestone obligation payments recorded			30,000
Definite-lived intangible assets
Definite-lived intangible assets
Total definite-lived intangibles, net	402,926		402,926
Definite-lived intangibles
Beginning balance - Definite-lived assets, Gross			444,591
Additions			38,309
Foreign currency translation			(1,335)
Ending balance - Definite-lived assets, Gross	481,565		481,565
Beginning balance - Accumulated amortization			(55,838)
Amortization	(11,800)	$ (4,100)	(23,422)	$ (7,900)
Foreign currency translation - Accumulated amortization			621
Ending balance - Accumulated amortization	(78,639)		(78,639)
Definite-lived intangible assets \| Waylivra
Definite-lived intangibles
Beginning balance - Definite-lived assets, Gross			18,598
Additions			2,933
Foreign currency translation			(578)
Ending balance - Definite-lived assets, Gross	20,953		20,953
Beginning balance - Accumulated amortization			(8,689)
Amortization			(1,853)
Foreign currency translation - Accumulated amortization			284
Ending balance - Accumulated amortization	(10,258)		(10,258)
Definite-lived intangible assets \| Tegsedi
Definite-lived intangibles
Beginning balance - Definite-lived assets, Gross			24,825
Additions			3,186
Foreign currency translation			(757)
Ending balance - Definite-lived assets, Gross	27,254		27,254
Beginning balance - Accumulated amortization			(10,295)
Amortization			(2,455)
Foreign currency translation - Accumulated amortization			337
Ending balance - Accumulated amortization	(12,413)		(12,413)
Definite-lived intangible assets \| Kebilidi
Definite-lived intangibles
Beginning balance - Definite-lived assets, Gross			10,731
Ending balance - Definite-lived assets, Gross	10,731		10,731
Beginning balance - Accumulated amortization			(1,006)
Amortization			(972)
Ending balance - Accumulated amortization	(1,978)		(1,978)
Definite-lived intangible assets \| Upstaza
Definite-lived intangibles
Beginning balance - Definite-lived assets, Gross			106,937
Ending balance - Definite-lived assets, Gross	106,937		106,937
Beginning balance - Accumulated amortization			(27,437)
Amortization			(7,950)
Ending balance - Accumulated amortization	(35,387)		(35,387)
Definite-lived intangible assets \| Sephience
Definite-lived intangibles
Beginning balance - Definite-lived assets, Gross			283,500
Additions			32,190
Ending balance - Definite-lived assets, Gross	315,690		315,690
Beginning balance - Accumulated amortization			(8,411)
Amortization			(10,192)
Ending balance - Accumulated amortization	$ (18,603)		$ (18,603)

Summary of significant accounting policies (Policies)

6 Months Ended

Jun. 30, 2026

Summary of significant accounting policies

Basis of presentation

Use of estimates

Restricted cash


		End of			Beginning of
		period-			period-
		June 30,			December 31,
		2026			2025
Cash and cash equivalents		$	1,049,887		$	984,648
Restricted cash included in deposits and other assets			16,445			13,686
Total Cash, cash equivalents and restricted cash per consolidated statement of cash flows		$	1,066,332		$	998,334

Revenue recognition

Net product revenue

Collaboration, license, and royalty revenue

Allowance for doubtful accounts

Income Taxes

Recently issued accounting standards

Recently issued accounting standards

Impact of recently adopted accounting pronouncements

- Definition

Disclosure of accounting policy for credit loss on financial instrument measured at amortized cost basis, net investment in lease, off-balance sheet credit exposure, and available-for-sale debt security. Includes, but is not limited to, methodology used to estimate allowance for credit loss, how writeoff of uncollectible amount is recognized, and determination of past due status and nonaccrual status.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 326
-SubTopic 20
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SAB Topic 6.M.2.Q4)
-Publisher FASB
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Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 326
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-URI https://asc.fasb.org/1943274/2147479319/326-20-50-17

+ Details

- Definition

Disclosure of accounting policy for income taxes, which may include its accounting policies for recognizing and measuring deferred tax assets and liabilities and related valuation allowances, recognizing investment tax credits, operating loss carryforwards, tax credit carryforwards, and other carryforwards, methodologies for determining its effective income tax rate and the characterization of interest and penalties in the financial statements.

+ References + Details

- Definition

Disclosure of accounting policy for revenue from contract with customer.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
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-Name Accounting Standards Codification
-Section 50
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-URI https://asc.fasb.org/606/tableOfContent

+ Details

- Definition

Disclosure of accounting policy for the use of estimates in the preparation of financial statements in conformity with generally accepted accounting principles.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 942
-SubTopic 235
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+ Details

Fair value of financial instruments and marketable securities - Marketable Securities (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025
Changes in fair value
Unrealized Gain/(Loss)			$ 1,914	$ 1,538
Investments Purchased			32,976	17,029
Sale			(37,499)	(17,129)
Unrealized Gain/(Loss)				(3,078)
Beginning Balance - Total	$ 48,443	$ 38,824	31,596	42,793
Unrealized Gain/(Loss) - Total	700	808	1,914	(1,540)
Foreign Currency Unrealized Gain/(Loss) - Total	1,083	1,665	2,644	3,731
Investments Purchased - Total	9,019	11,820	32,976	17,029
Sale - Total	(27,614)	(8,233)	(37,499)	(17,129)
Ending Balance - Total	31,631	44,884	31,631	44,884
Marketable securities - equity investments
Changes in fair value
Beginning Balance	48,443	28,187	31,596	29,034
Unrealized Gain/(Loss)	700	764	1,914	1,538
Foreign Currency Unrealized Gain/(Loss)	1,083	1,665	2,644	3,731
Investments Purchased	9,019	11,820	32,976	17,029
Sale	(27,614)	(8,233)	(37,499)	(17,129)
Ending Balance	$ 31,631	34,203	$ 31,631	34,203
ClearPoint Equity Investment
Changes in fair value
Beginning Balance		10,637		13,759
Unrealized Gain/(Loss)		44		(3,078)
Ending Balance		$ 10,681		$ 10,681

Revenue recognition - Net Product Sales (Details) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2026 USD ($) segment Distributor	Jun. 30, 2025 segment Distributor	Jun. 30, 2026 USD ($) segment Distributor	Jun. 30, 2025 segment Distributor	Dec. 31, 2025 USD ($)
Revenue recognition
Number of operating segments \| segment	1	1	1	1
Net product revenue \| Customer concentration risk
Revenue recognition
Number of distributors \| Distributor	3	3	3	2
Percentage of net product sales	10.00%	10.00%	10.00%	10.00%
Product
Revenue recognition
Contract with customer, liability \| $	$ 0		$ 0		$ 0

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

+ Details

Stock award plan - Narrative (Details) - USD ($)		1 Months Ended			3 Months Ended		6 Months Ended		12 Months Ended
Stock award plan - Narrative (Details) - USD ($)	Jun. 08, 2022	Dec. 31, 2022	Jun. 30, 2021	Dec. 31, 2020	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025	Dec. 31, 2024	Apr. 30, 2022	May 31, 2021	Jan. 31, 2020
Stock option plan
Unrecognized compensation cost					$ 206,800,000		$ 206,800,000
Weighted average remaining service period for recognition of unrecognized compensation cost							2 years 10 months 24 days
Share-based compensation expense					23,291,000	$ 18,543,000	$ 46,712,000	$ 36,603,000
Stock option
Stock option plan
Granted (in shares)							757,556
Expected dividend yield (as a percent)							0.00%
Weighted average grant date fair value (in dollars per share)							$ 40.21
Restricted stock units
Stock option plan
Granted (in shares)							1,293,798
Unvested performance based restricted stock units
Stock option plan
Share-based compensation expense					700,000		$ 1,200,000
Unvested performance based restricted stock units \| Chief Executive Officer
Stock option plan
Granted (in shares)							37,500
Performance based \| Chief Executive Officer
Stock option plan
Granted (in shares)							37,500		25,000
Performance period							3 years
Market Conditions
Stock option plan
Share-based compensation expense					1,100,000	$ 300,000	$ 2,300,000	$ 300,000
Market Conditions \| Chief Executive Officer
Stock option plan
Share-based compensation expense							0
Employee Stock Purchase Plan
Stock option plan
Number of shares authorized (in shares)			2,000,000								1,000,000
Award requisite service period			6 months
Purchase price of common stock, percent			85.00%
Employee stock purchase plan, voting percentage limit			5.00%
Share-based compensation expense					$ 500,000		$ 1,000,000
Amended 2013 LTIP
Stock option plan
Number of shares available for issuance					3,408,235		3,408,235
Number of additional shares authorized (in shares)	8,475,000
Amended 2013 LTIP \| Maximum
Stock option plan
Number of shares authorized (in shares)	16,724,212
2020 Inducement Stock Incentive Plan \| Common stock
Stock option plan
Number of shares available for issuance					1,754,494		1,754,494
Number of additional shares authorized (in shares)		1,700,000		1,000,000
2020 Inducement Stock Incentive Plan \| Common stock \| Maximum
Stock option plan
Number of shares authorized (in shares)										1,300,000		1,000,000
2020 Inducement Stock Incentive Plan \| Stock option
Stock option plan
Granted (in shares)							25,755
Stockholder's specified ownership percentage					10.00%		10.00%
Vesting period							4 years
2020 Inducement Stock Incentive Plan \| Stock option \| Minimum
Stock option plan
Stock options granted, exercise price as percentage of the fair market value of common stock at grant date					100.00%		100.00%
2020 Inducement Stock Incentive Plan \| Stock option \| Maximum
Stock option plan
Stock options granted to stockholder with specified ownership percentage, exercise price as percentage of the fair market value of common stock at grant date					110.00%		110.00%
Expiration period							10 years
2020 Inducement Stock Incentive Plan \| Restricted stock units
Stock option plan
Granted (in shares)							30,610

Commitments and contingencies

6 Months Ended

Jun. 30, 2026

Commitments and contingencies

Commitments and contingencies

10. Commitments and contingencies

Under various agreements, the Company will be required to pay royalties and milestone payments upon the successful development and commercialization of products.

Pursuant to the Agreement and Plan of Merger, dated as of July 19, 2018 by and among the Company, Agilis Biotherapeutics, Inc. (“Agilis”) and Agility Merger Sub, Inc. (such merger pursuant thereto, the “Agilis Merger”), Agilis equityholders were previously entitled to receive contingent consideration payments from the Company based on (i) the achievement of certain development milestones up to an aggregate maximum amount of $60.0 million, (ii) the achievement of certain regulatory approval milestones together with a milestone payment following the receipt of a priority review voucher up to an aggregate maximum amount of $535.0 million, (iii) the achievement of certain net sales milestones up to an aggregate maximum amount of $150.0 million, and (iv) a percentage of annual net sales for FA and Angelman syndrome during specified terms, ranging from 2%-6%. The Company was required to pay $40.0 million of the development milestone payments upon the passing of the second anniversary of the closing of the Agilis Merger, regardless of whether the applicable milestones have been achieved.

As of June 30, 2026, all of the milestones have either been paid or settled, with the exception of the regulatory milestones and net sales milestones related to FA and Angelman syndrome and the net sales milestones related to Upstaza/Kebilidi. In May 2023, as part of the Company’s strategic portfolio prioritization, the Company decided to discontinue its preclinical and early research programs in its gene therapy platform, which included FA and Angelman syndrome. As a result, the Company does not expect the milestones related to FA and Angelman syndrome to be achieved. In addition, the Company does not expect to pay the 2%–6% royalties on annual net sales related to FA and Angelman syndrome. As of June 30, 2026, the remaining potential sales milestones related to Upstaza/Kebilidi is $50.0 million, however the Company has determined that the probability of triggering these milestones is remote.

On October 25, 2019, the Company completed the acquisition of substantially all of the assets of BioElectron Technology Corporation (“BioElectron”), a Delaware corporation, including certain compounds that the Company has begun to develop as part of its inflammation and ferroptosis platform, pursuant to an asset purchase agreement by and between the Company and BioElectron, dated October 1, 2019 (the “BioElectron Asset Purchase Agreement”). BioElectron was a private company with a pipeline focused on inflammatory CNS disorders. The lead program, vatiquinone, is in late stage development for FA with substantial unmet need and significant commercial opportunity that are complementary to PTC’s existing pipeline.

Subject to the terms and conditions of the BioElectron Asset Purchase Agreement, BioElectron may become entitled to receive contingent milestone payments of up to $200.0 million (in cash or in shares of the Company’s common stock, as determined by the Company) from the Company based on the achievement of certain regulatory and net sales milestones. Subject to the terms and conditions of the BioElectron Asset Purchase Agreement, BioElectron may also become entitled to receive contingent payments based on a percentage of net sales of certain products. As of June 30, 2026, no contingent milestones have been achieved, and therefore, the remaining potential contingent milestone payments is $200.0 million.

Subject to the terms and conditions of the Agreement and Plan of Merger, dated as of May 5, 2020 (the “Censa Merger Agreement”) by and among the Company, Censa Pharmaceuticals, Inc. (“Censa”), Hydro Merger Sub, Inc., the Company’s wholly owned, indirect subsidiary, and, solely in its capacity as the representative, agent and attorney-in-fact of the securityholders of Censa, Shareholder Representative Services LLC (such merger pursuant thereto, the “Censa Merger”), former Censa securityholders may become entitled to receive contingent payments from the Company based on (i) the achievement of certain development and regulatory milestones up to an aggregate maximum amount of $217.5 million for sepiapterin’s two most advanced programs and receipt of a priority review voucher from the FDA as set forth in the Censa Merger Agreement, (ii) $109.0 million in development and regulatory milestones for each additional indication of sepiapterin, (iii) the achievement of certain net sales milestones up to an aggregate maximum amount of $160.0 million, (iv) a percentage of annual net sales during specified terms, ranging from single to low double digits of the applicable net sales threshold amount, and (v) any sublicense fees paid to the Company in consideration of any sublicense of Censa’s intellectual property to commercialize sepiapterin, on a country-by-country basis, which contingent payment shall equal to a mid-double digit percentage of any such sublicense fees.

On August 5, 2025, the Company, certain former securityholders of Censa, and, for the limited purposes set forth in the agreement, Shareholder Representative Services LLC, entered into a Rights Satisfaction Agreement (the “Rights Satisfaction Agreement”) pursuant to which such former securityholders of Censa (the “Participating Rightsholders”) agreed to the cancellation and forfeiture of their rights to receive certain contingent payments from the Company based on worldwide annual net sales by the Company of products containing sepiapterin (“Net Sales of Product”) under the Censa Merger Agreement in exchange for the consideration set forth in the Rights Satisfaction Agreement and further detailed below.

Pursuant to the terms of the Rights Satisfaction Agreement, the Participating Rightsholders canceled and forfeited their rights under the Censa Merger Agreement to receive a percentage of annual net sales during the applicable payment term equal to (i) 8% of annual Net Sales of Product for that portion of annual Net Sales of Product less than or equal to $250.0 million, (ii) 10% of annual Net Sales of Product for that portion of annual Net Sales of Product greater than $250.0 million but less than $500.0 million and (iii) 12% of annual Net Sales of Product for that portion of annual Net Sales of Product greater than $500.0 million (collectively, the “Net Sales Payments”).

In consideration of the foregoing, the Company agreed to pay to the Participating Rightsholders an aggregate amount in cash up to $250.0 million (the “Upfront Consideration”) upon the consummation of the transactions contemplated by the Rights Satisfaction Agreement and potential milestone payments (each an “Additional Milestone Payment”) of up to $100.0 million each (or up to $500.0 million in the aggregate) based on the achievement of specified Net Sales Thresholds (as defined in the Censa Merger Agreement). The amount of the Upfront Consideration and the Additional Milestone Payments was subject to adjustment in the Rights Satisfaction Agreement based on the number of Participating Rightsholders.

At the consummation of the transactions contemplated by the Rights Satisfaction Agreement, based on the participation of former Censa securityholders holding approximately 90% of Censa’s equity securities prior to the consummation of the transactions contemplated by the Censa Merger Agreement, the Company paid an aggregate amount of Upfront Consideration in cash of $225.1 million. Additionally, the Company is obligated to make Additional Milestone Payments in an amount equal to approximately $90.0 million upon achievement of each of the (i) first occurrence of a three or fewer consecutive calendar year period in which aggregate Net Sales of Product are greater than $3.0 billion, (ii) first occurrence of a five or fewer consecutive calendar year period in which aggregate Net Sales of Product are greater than $5.0 billion, (iii) first occurrence of a seven or fewer consecutive calendar year period in which aggregate Net Sales of Product are greater than $7.0 billion, (iv) first occurrence of a nine or fewer consecutive calendar year period in which aggregate Net Sales of Product are greater than $9.0 billion and (v) first occurrence of an 11 or fewer consecutive calendar year period in which aggregate Net Sales of Product are greater than $11.0 billion. If, after the consummation of the transactions contemplated by the Rights Satisfaction Agreement, any additional former securityholder of Censa executes and delivers a joinder to the Rights Satisfaction Agreement and becomes a party thereto, the Company will pay such former securityholder an amount in cash equal to such former securityholder’s applicable pro rata share of the Upfront Consideration (less any Net Sale Payments previously received) and any Additional Milestone Payments that become payable to Participating Rightsholders under the Rights Satisfaction Agreement.

The Rights Satisfaction Agreement has no effect on the Censa Merger Agreement other than to provide for the cancellation and forfeiture of the Participating Rightsholders’ rights to receive the Net Sales Payments described above. As a result, all other rights and obligations under the Censa Merger Agreement remain in effect pursuant to their terms, including, without limitation, the Company’s obligation to pay certain contingent payments upon the achievement of certain development and Net Sales of Product milestones.

In June 2025, Sephience was granted marketing authorization by the EC for the treatment of children and adults living with PKU. Pursuant to the Censa Merger Agreement, the acceptance triggered a $25.0 million regulatory milestone payment to the former Censa securityholders. In July 2025, Sephience was granted FDA approval for the treatment of children and adults living with PKU. Pursuant to the Censa Merger Agreement, the approval triggered a $32.5 million milestone payment to the former Censa securityholders. These milestones were recorded as intangible assets and are being amortized to cost of product sales over their expected useful lives on a straight-line basis. As of June 30, 2026, $152.5 million of the $217.5 million in development and regulatory milestones have been paid to the former Censa securityholders.

As of the quarter ended June 30, 2026, aggregate Sephience global net sales in the prior four consecutive quarters exceeded $250.0 million, which, pursuant to the Censa Merger Agreement, triggered a $30.0 million net sales milestone payment to the former Censa securityholders. This milestone was recorded in accounts payable and accrued expenses and intangible assets on the Company's consolidated balance sheet as of June 30, 2026 and is being amortized to cost of product sales over its expected useful live on a straight-line basis. For the six months ended June 30, 2026, royalties of $2.2 million related payable to the former Censa securityholders were recorded on the consolidated balance sheet within intangible assets, net. Refer to Note 12 for additional details.

The Company also has the Tegsedi-Waylivra Agreement for the commercialization of Tegsedi and Waylivra, and products containing those compounds in countries in Latin America and the Caribbean. Akcea is entitled to receive royalty payments subject to certain terms set forth in the Tegsedi-Waylivra Agreement. For the six months ended June 30, 2026, royalties of $3.2 million and $2.9 million related to Tegsedi and Waylivra, respectively, were recorded on the consolidated balance sheet within intangible assets, net. Refer to Note 12 for additional details.

The Company has employment agreements with certain employees which require the funding of a specific level of payments, if certain events, such as a change in control or termination without cause, occur. Additionally, the Company has royalty payments associated with Translarna, Emflaza, Sephience, and Upstaza/Kebilidi net product revenue, payable quarterly or annually in accordance with the terms of the related agreements.

From time to time in the ordinary course of its business, the Company is subject to claims, legal proceedings and disputes. The Company is not currently aware of any material legal proceedings against it.

Fair value of financial instruments and marketable securities - Convertible senior notes (Details) - USD ($) $ in Thousands	1 Months Ended	3 Months Ended	6 Months Ended
	Jun. 30, 2026	Jun. 30, 2026	Jun. 30, 2026	Dec. 31, 2025	Sep. 30, 2019
Long-term debt
Repayment of convertible notes			$ 327,890
Payments for conversion of debt			12,879
Aggregate principal	$ 55,451	$ 55,451	55,451	$ 286,631
Convertible debt \| 2031 Convertible Notes
Long-term debt
Principal	$ 550,000	$ 550,000	$ 550,000
Interest rate ( as a percent )	0.00%	0.00%	0.00%
Additional amount available for purchase	$ 50,000	$ 50,000	$ 50,000
Convertible debt \| 2031 Convertible Notes \| Significant other observable inputs (Level 2)
Long-term debt
Fair value of convertible notes	579,200	579,200	579,200
Convertible debt \| 2026 Convertible Notes
Long-term debt
Principal					$ 287,500
Interest rate ( as a percent )					1.50%
Additional amount available for purchase					$ 37,500
Repurchased principal amount	222,000	222,000	222,000
Repayment of convertible notes	328,800
Principal amount converted		10,000
Payments for conversion of debt		$ 12,900	10,000
Number of shares issued on conversion of notes		3,506
Aggregate principal	55,500	$ 55,500	55,500
Convertible debt \| 2026 Convertible Notes \| Significant other observable inputs (Level 2)
Long-term debt
Fair value of convertible notes	$ 85,500	$ 85,500	$ 85,500	$ 424,500

Debt - Convertible Notes Interest Expense (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025
Long-term debt
Amortization of debt issuance costs			$ 689	$ 601
Effective interest rate	11.00%		11.00%
Convertible debt \| 2031 Convertible Notes
Long-term debt
Amortization of debt issuance costs	$ 119		$ 119
Total	$ 119		$ 119
Effective interest rate	0.50%		0.50%
Convertible debt \| 2026 Convertible Notes
Long-term debt
Contractual interest expense	$ 830	$ 1,067	$ 1,899	2,135
Amortization of debt issuance costs	265	301	570	601
Total	$ 1,095	$ 1,368	$ 2,469	$ 2,736
Effective interest rate	1.90%	1.90%	1.90%	1.90%

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 815
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 8A
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480434/815-10-50-8A

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
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-SubTopic 235
-Name Accounting Standards Codification
-Section S99
-Paragraph 2
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477314/942-235-S99-2

Reference 3: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 235
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-Name Accounting Standards Codification
-Section S50
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480738/235-10-S50-1

Reference 4: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 235
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-Name Accounting Standards Codification
-Section S50
-Paragraph 4
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480738/235-10-S50-4

Reference 5: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 235
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-Section S99
-Paragraph 3
-Subparagraph (SX 210.12-04(a))
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480678/235-10-S99-3

Reference 6: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 235
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.4-08(f))
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-URI https://asc.fasb.org/1943274/2147480678/235-10-S99-1

Reference 7: http://www.xbrl.org/2003/role/exampleRef
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-Name Accounting Standards Codification
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-URI https://asc.fasb.org/1943274/2147482949/835-30-55-8

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1B
-Subparagraph (a)
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-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1B

Reference 9: http://www.xbrl.org/2003/role/exampleRef
-Topic 470
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-Name Accounting Standards Codification
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-URI https://asc.fasb.org/1943274/2147481568/470-20-55-69B

Reference 10: http://www.xbrl.org/2003/role/exampleRef
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-Name Accounting Standards Codification
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-URI https://asc.fasb.org/1943274/2147481568/470-20-55-69C

Reference 11: http://www.xbrl.org/2003/role/exampleRef
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-URI https://asc.fasb.org/1943274/2147481568/470-20-55-69E

Reference 12: http://www.xbrl.org/2003/role/exampleRef
-Topic 470
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-Name Accounting Standards Codification
-Section 55
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481568/470-20-55-69F

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1B
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1B

Reference 14: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1B
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1B

Reference 15: http://www.xbrl.org/2003/role/exampleRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1B
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1B

Reference 16: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
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-Name Accounting Standards Codification
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-Subparagraph (e)
-Publisher FASB
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Reference 17: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
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-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1B

Reference 18: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
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-Name Accounting Standards Codification
-Section 50
-Paragraph 1B
-Subparagraph (h)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1B

Reference 19: http://www.xbrl.org/2003/role/disclosureRef
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-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1D

Reference 20: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1D

Reference 21: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
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Reference 22: http://www.xbrl.org/2003/role/disclosureRef
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Reference 23: http://www.xbrl.org/2003/role/disclosureRef
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Reference 24: http://www.xbrl.org/2003/role/disclosureRef
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Reference 25: http://www.xbrl.org/2003/role/disclosureRef
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Reference 26: http://www.xbrl.org/2003/role/disclosureRef
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Reference 27: http://www.xbrl.org/2003/role/disclosureRef
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Reference 28: http://www.xbrl.org/2003/role/disclosureRef
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Reference 29: http://www.xbrl.org/2003/role/disclosureRef
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Reference 30: http://www.xbrl.org/2009/role/commonPracticeRef
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-URI https://asc.fasb.org/1943274/2147482925/835-30-45-2

Reference 31: http://www.xbrl.org/2009/role/commonPracticeRef
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-URI https://asc.fasb.org/1943274/2147482900/835-30-50-1

+ Details

Stock award plan - Performance-based Restricted Stock Units (Details) - Chief Executive Officer - shares	6 Months Ended	12 Months Ended
	Jun. 30, 2026	Dec. 31, 2024
Unvested performance based restricted stock units
Number of Shares
Balance at the beginning of the period (in shares)	140,625
PSUs with performance or market conditions granted during the period	37,500
Vested (in shares)	(25,000)
Balance at the end of the period (in shares)	153,125
Performance based
Number of Shares
Balance at the beginning of the period (in shares)	25,000
PSUs with performance or market conditions granted during the period	37,500	25,000
Vested (in shares)	(25,000)
Balance at the end of the period (in shares)	37,500
Market Conditions
Number of Shares
Balance at the beginning of the period (in shares)	115,625
Balance at the end of the period (in shares)	115,625

Debt - 2026 & 2031 Convertible Notes Narrative (Details)	1 Months Ended		3 Months Ended	6 Months Ended
	Jun. 30, 2026 USD ($) D $ / shares	Sep. 30, 2019 USD ($) D $ / shares	Jun. 30, 2026 USD ($) $ / shares shares	Jun. 30, 2026 USD ($) $ / shares
Long-term debt
Repayment of convertible notes				$ 327,890,000
Payments for conversion of debt				12,879,000
Convertible debt \| 2031 Convertible Notes
Long-term debt
Principal	$ 550,000,000		$ 550,000,000	$ 550,000,000
Interest rate ( as a percent )	0.00%		0.00%	0.00%
Additional amount available for purchase	$ 50,000,000		$ 50,000,000	$ 50,000,000
Net proceeds from issuance of convertible notes	$ 535,400,000
Conversion ratio	9.3042
Conversion price per share (in dollars per share) \| $ / shares	$ 107.48		$ 107.48	$ 107.48
Trading days, number \| D	20
Consecutive trading days, period \| D	30
Stock price trigger	130.00%
Business days, period	5 days
Consecutive trading-day period	5 days
Maximum product of the closing sale price of shares of the Company's common stock and the applicable conversion rate for such trading day	98.00%		98.00%	98.00%
Minimum percentage of principal held by convertible debt instrument holders required to issue notice for declaration of principal and unpaid interest payable upon events of default	25.00%
Convertible instruments principal and unpaid interest payable upon events of default	100.00%
Term of the convertible notes	5 years			5 years
Convertible debt \| 2031 Convertible Notes \| Redemption on or after June 20, 2029
Long-term debt
Trading days, number \| D	20
Consecutive trading days, period \| D	30
Stock price trigger	130.00%
Redemption price as percent of principal amount	100.00%
Sinking fund	$ 0		$ 0	$ 0
Convertible debt \| 2026 Convertible Notes
Long-term debt
Principal		$ 287,500,000
Interest rate ( as a percent )		1.50%
Additional amount available for purchase		$ 37,500,000
Net proceeds from issuance of convertible notes		$ 279,300,000
Conversion ratio		19.0404
Conversion price per share (in dollars per share) \| $ / shares		$ 52.52
Minimum percentage of principal held by convertible debt instrument holders required to issue notice for declaration of principal and unpaid interest payable upon events of default		25.00%
Convertible instruments principal and unpaid interest payable upon events of default		100.00%
Term of the convertible notes				2 months 12 days
Repayment of convertible notes	328,800,000
Repurchase of 2026 convertible notes	222,000,000		222,000,000	$ 222,000,000
Excess of the fair value of the repurchase price over the if-converted value	3,400,000
Difference between the if-converted value and the net carrying amount	$ 102,700,000
Principal amount converted			10,000,000
Payments for conversion of debt			$ 12,900,000	$ 10,000,000
Number of shares issued on conversion of notes \| shares			3,506
Reduction to the carrying value			$ 10,000,000
Excess of conversion value over carrying value			$ 2,900,000
Convertible debt \| 2026 Convertible Notes \| Redemption on or after September 20, 2023
Long-term debt
Trading days, number \| D		19
Consecutive trading days, period \| D		30
Stock price trigger		130.00%
Redemption price as percent of principal amount		100.00%
Sinking fund		$ 0

Stock award plan - Assumptions Used (Details) - Stock option	6 Months Ended
	Jun. 30, 2026
Valuation assumptions
Expected volatility (as a percent)	54.00%
Expected term (in years)	5 years 6 months
Minimum
Valuation assumptions
Risk-free interest rate (as a percent)	3.78%
Maximum
Valuation assumptions
Risk-free interest rate (as a percent)	4.21%

Debt

6 Months Ended

Jun. 30, 2026

Debt

Debt

9. Debt

Liability for sale of future royalties

The Company has a royalty purchase agreement, (the “A&R Royalty Purchase Agreement”), with Royalty Pharma. Under the A&R Royalty Purchase Agreement, Royalty Pharma has provided funding to the Company totaling $2.1 billion. In exchange for these fundings, the Company sold Royalty Pharma 100% of the Company's rights to receive sales-based royalty payments on worldwide net sales of Roche’s Evrysdi® (risdiplam) product and any other product developed pursuant to the SMA collaboration with the Company, Roche, and the SMA Foundation. Pursuant to A&R Royalty Purchase Agreement, the Company is entitled to three potential additional purchase price payments of $20.0 million each conditioned upon receipt by Royalty Pharma of more than $347.0 million of Assigned Royalty Payments (as defined in the A&R Royalty Purchase Agreement) in respect of Calendar Year Net Sales (as defined in the A&R Royalty Purchase Agreement) arising in 2027, $363.0 million of Assigned Royalty Payments in respect of Calendar Year Net Sales arising in 2028, and $379.0 million of Assigned Royalty Payments in respect of Calendar Year Net Sales arising in 2029, respectively.

Pursuant to the guidance in ASC 470-10-25-2, the Company determined that these fundings should be classified as debt and are recorded as “liability for sale of future royalties-current” and “liability for sale of future royalties-noncurrent” on the Company’s consolidated balance sheet based on the timing of the expected payments to be made to Royalty Pharma.

The liability is being amortized using the effective interest method over the life of the arrangement, in accordance with the respective guidance, utilizing the prospective method to account for subsequent changes in the estimated future payments to be made to Royalty Pharma and the Company updates the effective interest rate on a quarterly basis.

The following table shows the activity within the “liability for sale of future royalties- current” and “liability for sale of future royalties- noncurrent” accounts for the six months ended June 30, 2026:


		Six Months Ended June 30,
Liability for sale of future royalties- (current and noncurrent)		2026
Beginning balance as of December 31, 2025		$	2,308,366
Less: Non-cash royalty revenue payable to Royalty Pharma			(117,940)
Plus: Non-cash interest expense recognized			131,008
Ending balance		$	2,321,434
Effective interest rate as of June 30, 2026			11.0%

Non-cash interest expense is recorded in the statement of operations within “Interest expense, net”.

2031 Convertible Notes

In June 2026, the Company issued, at par value, $550.0 million aggregate principal amount of 0% convertible senior notes due 2031 (the “2031 Convertible Notes”), which reflects the exercise in full by the initial purchasers of their option to purchase up to an additional $50.0 million in aggregate principal amount of the 2031 Convertible Notes. The net proceeds to the Company from the offering were approximately $535.4 million after deducting the initial purchasers’ discounts and commissions and the estimated offering expenses payable by the Company. The 2031 Convertible Notes do not bear regular interest and the principal amounts of the 2031 Convertible Notes will not accrete.

The 2031 Convertible Notes are governed by an indenture (the “2031 Convertible Notes Indenture”) with U.S Bank Trust Company National Association as trustee (the “2031 Convertible Notes Trustee”).

The 2031 Convertible Notes may bear special interest under specified circumstances relating to the Company’s failure to comply with its reporting obligations under the 2031 Convertible Notes Indenture or if the 2031 Convertible Notes are not freely tradeable as required by the 2031 Convertible Notes Indenture.

Special interest, if any, will be payable semiannually in arrears on June 15 and December 15 of each year, beginning on December 15, 2026 (if and to the extent that special interest is payable). The 2031 Convertible Notes will mature on June 15, 2031, unless earlier converted, redeemed or repurchased pursuant to their terms.

The initial conversion rate of the 2031 Convertible Notes is 9.3042 shares of the Company’s common stock, per $1,000 principal amount of 2031 Convertible Notes (which is equivalent to an initial conversion price of approximately $107.48 per share). The conversion rate will be subject to adjustment upon the occurrence of certain specified events but will not be adjusted for any accrued and unpaid special interest. In addition, upon the occurrence of a make-whole fundamental change (as defined in the 2031 Convertible Notes Indenture) or an issuance of a notice of redemption, the Company will, in certain circumstances, increase the conversion rate by a number of additional shares for a holder that elects to convert its 2031 Convertible Notes in connection with such make-whole fundamental change or notice of redemption.

Holders may convert all or any portion of their 2031 Convertible Notes at their option at any time prior to the close of business on the business day immediately preceding March 15, 2031 only under the following circumstances: (1) during any calendar quarter commencing after the calendar quarter ending on September 30, 2026 (and only during such calendar quarter), if the last reported sale price of the common stock for at least 20 trading days (whether or not consecutive) during a period of 30 consecutive trading days ending on, and including, the last trading day of the immediately preceding calendar quarter is greater than or equal to 130% of the conversion price on each applicable trading day; (2) during the five business day period after any five consecutive trading day period (the “measurement period”) in which the trading price per $1,000 principal amount of 2031 Convertible Notes for each trading day of the measurement period was less than 98% of the

product of the last reported sale price of the common stock and the conversion rate on each such trading day; (3) if the Company calls any or all of the 2031 Convertible Notes for redemption, at any time prior to the close of business on the second scheduled trading day immediately preceding the relevant redemption date; or (4) upon the occurrence of specified corporate events. On or after March 15, 2031 until the close of business on the second scheduled trading day immediately preceding the maturity date, holders may convert all or any portion of their 2031 Convertible Notes at any time, regardless of the foregoing circumstances. Upon conversion, the Company will satisfy its conversion obligation by paying or delivering, as the case may be, cash, shares of Common Stock or a combination of cash and shares of common stock, at the Company’s election.

The Company may not redeem the 2031 Convertible Notes prior to June 20, 2029. The Company may redeem for cash all or any portion of the 2031 Convertible Notes, at the Company’s option, on or after June 20, 2029 if the last reported sale price of the common stock has been at least 130% of the conversion price then in effect for at least 20 trading days (whether or not consecutive) during any 30 consecutive trading day period (including the last trading day of such period) ending on, and including, the trading day immediately preceding the date on which the Company provides a written notice of redemption at a redemption price equal to 100% of the principal amount of the 2031 Convertible Notes to be redeemed, plus any accrued and unpaid special interest to, but excluding, the redemption date. No “sinking fund” is provided for the 2031 Convertible Notes.

If the Company undergoes a fundamental change (as defined in the 2031 Convertible Notes Indenture) prior to the maturity date, then, subject to certain conditions, holders of 2031 Convertible Notes may require the Company to repurchase for cash all or any portion of their 2031 Convertible Notes at a fundamental change repurchase price equal to 100% of the principal amount of the 2031 Convertible Notes to be repurchased, plus any accrued and unpaid special interest to, but excluding, the fundamental change repurchase date.

The 2031 Convertible Notes are the Company’s general unsecured, senior obligations and will rank senior in right of payment to any of its indebtedness that is expressly subordinated in right of payment to the 2031 Convertible Notes; equal in right of payment with all of the Company’s existing and future unsecured indebtedness that is not so subordinated (including any of the Company’s outstanding 2026 Convertible Notes); effectively junior in right of payment to any of the Company’s senior, secured indebtedness to the extent of the value of the assets securing such indebtedness; and structurally junior to all indebtedness and other liabilities (including trade payables) of the Company’s current or future subsidiaries.

The 2031 Convertible Notes Indenture contains customary events of default with respect to the 2031 Convertible Notes, including that upon certain events of default (including the Company’s failure to make any payment of principal or any special interest on the Notes when due and payable) occurring and continuing, the 2031 Convertible Notes Trustee by written notice to the Company, or the holders of at least 25% in principal amount of the outstanding 2031 Convertible Notes by notice to the Company and the 2031 Convertible Notes Trustee, may (subject to the provisions of the 2031 Convertible Notes Indenture) declare 100% of the principal of and accrued and unpaid special interest, if any, on all the 2031 Convertible Notes to be due and payable. In case of certain events of bankruptcy, insolvency or reorganization, involving the Company or a significant subsidiary, 100% of the principal of and accrued and unpaid special interest, if any, on the 2031 Convertible Notes will automatically become due and payable without any further act or declaration on the part of the holders or the 2031 Convertible Notes Trustee. Upon such a declaration of acceleration, such principal and accrued and unpaid special interest, if any, will be due and payable immediately.

The Company accounted for the 2031 Convertible Notes as a single liability measured at amortized cost. The debt issuance costs were recorded as a direct deduction from the face value of the 2031 Convertible Notes and will be amortized to interest expense over the five-year term of the 2031 Convertible Notes using the effective interest rate method.

The 2031 Convertible Notes consist of the following:




		June 30, 2026			December 31, 2025
Principal		$	550,000		$	—
Less: Debt issuance costs			(14,481)			—

Net carrying amount

535,519

—

As of June 30, 2026, the remaining contractual life of the 2031 Convertible Notes is approximately 5.0 years.

The following table sets forth total interest expense recognized related to the 2031 Convertible Notes:



		Three Months Ended June 30,						Six Months Ended June 30,
		2026			2025			2026			2025
Amortization of debt issuance costs		$	119		$	—		$	119		$	—
Total		$	119		$	—		$	119		$	—
Effective interest rate			0.5	%		—	%		0.5	%		—	%

2026 Convertible Notes

In September 2019, the Company issued, at par value, $287.5 million aggregate principal amount of 1.50% convertible senior notes due 2026, which included an option to purchase up to an additional $37.5 million in aggregate principal amount of the 2026 Convertible Notes, which was exercised in full by the initial purchasers. The net proceeds to the Company from the offering were $279.3 million after deducting the initial purchasers’ discounts and commissions and the offering expenses payable by the Company. The 2026 Convertible Notes bear cash interest at a rate of 1.50% per year, payable semi-annually on March 15 and September 15 of each year, beginning on March 15, 2020. The 2026 Convertible Notes will mature on September 15, 2026, unless earlier repurchased or converted.

The 2026 Convertible Notes are governed by an indenture (the “2026 Convertible Notes Indenture”) with U.S. Bank National Association as trustee (the “2026 Convertible Notes Trustee”).

Beginning March 15, 2026, until the close of business on the business day immediately preceding the maturity date, holders may convert their 2026 Convertible Notes at any time. As of June 30, 2026, the 2026 Convertible Notes are convertible. Upon conversion, the Company will pay or deliver, as the case may be, cash, shares of the Company’s common stock or any combination thereof at the Company’s election.

The conversion rate for the 2026 Convertible Notes was initially, and remains, 19.0404 shares of the Company’s common stock per $1,000 principal amount of the 2026 Convertible Notes, which is equivalent to an initial conversion price of approximately $52.52 per share of the Company’s common stock. The conversion rate may be subject to adjustment in some events but will not be adjusted for any accrued and unpaid interest.

The Company was not permitted to redeem the 2026 Convertible Notes prior to September 20, 2023. The Company may redeem for cash all or any portion of the 2026 Convertible Notes, at its option, if the last reported sale price of its common stock has been at least 130% of the conversion price then in effect on the last trading day of, and for at least 19 other trading days (whether or not consecutive) during, any 30 consecutive trading day period ending on, and including, the trading day immediately preceding the date on which the Company provides notice of redemption, at a redemption price equal to 100% of the principal amount of the 2026 Convertible Notes to be redeemed, plus accrued and unpaid interest to, but excluding, the redemption date. No sinking fund is provided for the 2026 Convertible Notes, which means that the Company is not required to redeem or retire the 2026 Convertible Notes periodically. As of June 30, 2026, the 2026 Convertible Notes were redeemable.

If the Company undergoes a “fundamental change” (as defined in the 2026 Convertible Notes Indenture), subject to certain conditions, holders of the 2026 Convertible Notes may require the Company to repurchase for cash all or part of their 2026 Convertible Notes at a repurchase price equal to 100% of the principal amount of the 2026 Convertible Notes to be repurchased, plus accrued and unpaid interest to, but excluding, the fundamental change repurchase date.

The 2026 Convertible Notes represent senior unsecured obligations and will rank senior in right of payment to the Company’s future indebtedness that is expressly subordinated in right of payment to the notes, equal in right of payment to the Company’s existing and future unsecured indebtedness that is not so subordinated (including any of the Company’s outstanding 2031 Convertible Notes), effectively junior in right of payment to any of the Company’s secured indebtedness to the extent of the value of the assets securing such indebtedness, and structurally subordinated to all existing and future indebtedness and other liabilities (including trade payables) incurred by the Company’s subsidiaries. The 2026 Convertible Notes Indenture contains customary events of default with respect to the 2026 Convertible Notes, including that upon certain events of default (including the Company’s failure to make any payment of principal or interest on the 2026 Convertible Notes when due and payable) occurring and continuing, the 2026 Convertible Notes Trustee by notice to the Company, or the holders of at least 25% in principal amount of the outstanding 2026 Convertible Notes by notice to the Company and the Convertible Notes Trustee, may, and the 2026 Convertible Notes Trustee at the request of such holders (subject to the provisions of the 2026 Convertible Notes Indenture) will, declare 100% of the principal of and accrued and unpaid interest, if any, on all the 2026 Convertible Notes to be due and payable. In case of certain events of bankruptcy, insolvency or reorganization, involving the Company or a significant subsidiary, 100% of the principal of and accrued and unpaid interest on the 2026 Convertible Notes will automatically become due and payable. Upon such a declaration of acceleration, such principal and accrued and unpaid interest, if any, will be due and payable immediately.

Following the issuance of the 2031 Convertible Notes, in June 2026 the Company used approximately $328.8 million of the net proceeds of the 2031 Convertible Notes to repurchase for cash $222.0 million aggregate principal amount of its 2026 Convertible Notes pursuant to privately negotiated transactions with certain holders entered into concurrently with the pricing of the offering of the 2031 Convertible Notes. The repurchase of the 2026 Convertible Notes was accounted for as an induced conversion in accordance with ASC-470-20. The excess of the fair value of the repurchase price over the if-converted value was $3.4 million and was recorded as inducement expense within other expense, net on the consolidated statements of operations. The net carrying amount of the repurchased 2026 Convertible Notes was derecognized, and the difference between the if-converted value and the net carrying amount of $102.7 million was recognized as a reduction in additional paid-in capital within the consolidated statement of stockholders’ deficit.

During the three months ended June 30, 2026, a holder converted a portion of the 2026 Convertible Notes with a principal amount of $10.0 million in exchange for $12.9 million in cash and 3,506 shares of the Company’s common stock. The Company recorded a $10.0 million reduction to the carrying value of the 2026 Convertible Notes, and the excess of $2.9 million was recognized as a reduction in additional paid-in capital within the Company’s statement of stockholders’ deficit.

The 2026 Convertible Notes consist of the following:



		June 30, 2026		December 31, 2025
Principal		$	287,500	$	287,500
Conversion of the 2026 Convertible Notes			(10,000)		—
Repurchase of the 2026 Convertible Notes			(222,000)		—
Less: Debt issuance costs			(49)		(869)
Net carrying amount		$	55,451	$	286,631

As of June 30, 2026, the remaining contractual life of the 2026 Convertible Notes is approximately 0.2 years.

The following table sets forth total interest expense recognized related to the 2026 Convertible Notes:


		Three Months Ended June 30,						Six Months Ended June 30,
	2026			2025			2026			2025
Contractual interest expense	$	830		$	1,067		$	1,899		$	2,135
Amortization of debt issuance costs		265			301			570			601
Total	$	1,095		$	1,368		$	2,469		$	2,736
Effective interest rate		1.9	%		1.9	%		1.9	%		1.9	%

Document and Entity Information - shares	6 Months Ended
Document and Entity Information - shares	Jun. 30, 2026	Jul. 29, 2026
Document and Entity Information
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Jun. 30, 2026
Document Transition Report	false
Entity File Number	001-35969
Entity Registrant Name	PTC Therapeutics, Inc.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	04-3416587
Entity Address, Address Line One	500 Warren Corporate Center Drive
Entity Address, City or Town	Warren
Entity Address, State or Province	NJ
Entity Address, Postal Zip Code	07059
City Area Code	908
Local Phone Number	222-7000
Title of 12(b) Security	Common Stock, $0.001 par value per share
Trading Symbol	PTCT
Security Exchange Name	NASDAQ
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		83,460,802
Entity Central Index Key	0001070081
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2026
Document Fiscal Period Focus	Q2
Amendment Flag	false

Net income (loss) per share - Antidilutive (Details) - shares	6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025
Net income (loss) per share
Total shares excluded from calculation (in shares)	2,107,776	3,578,222
Stock options
Net income (loss) per share
Total shares excluded from calculation (in shares)	807,769	3,494,565
Unvested restricted stock units
Net income (loss) per share
Total shares excluded from calculation (in shares)	1,206,529	83,657
Unvested performance based restricted stock units
Net income (loss) per share
Total shares excluded from calculation (in shares)	93,478

Intangible assets and goodwill

6 Months Ended

Jun. 30, 2026

Intangible assets and goodwill

Intangible assets and goodwill

12. Intangible assets and goodwill

Definite-lived intangibles

Definite-lived intangible assets consisted of the following at June 30, 2026 and December 31, 2025:


			Ending Balance at						Foreign		Ending Balance at
Definite-lived			December 31,						currency		June 30,
intangible assets, gross			2025			Additions			translation		2026
Waylivra			18,598			2,933			(578)		20,953
Tegsedi			24,825			3,186			(757)		27,254
Kebilidi			10,731			—			—		10,731
Upstaza			106,937			—			—		106,937
Sephience			283,500			32,190			—		315,690
Total definite-lived intangibles, gross		$	444,591		$	38,309		$	(1,335)	$	481,565


			Ending Balance at						Foreign			Ending Balance at
Definite-lived			December 31,						currency			June 30,
intangible assets, accumulated amortization			2025			Amortization			translation			2026
Waylivra			(8,689)			(1,853)			284			(10,258)
Tegsedi			(10,295)			(2,455)			337			(12,413)
Kebilidi			(1,006)			(972)			—			(1,978)
Upstaza			(27,437)			(7,950)			—			(35,387)
Sephience			(8,411)			(10,192)			—			(18,603)
Total definite-lived intangibles, accumulated amortization		$	(55,838)		$	(23,422)		$	621		$	(78,639)

Total definite-lived intangibles, net											$	402,926

Akcea is entitled to receive royalty payments subject to certain terms set forth in the Tegsedi-Waylivra Agreement related to sales of Waylivra and Tegsedi. In accordance with the guidance for an asset acquisition, the Company records royalty payments when they become payable to Akcea and increase the cost basis for the Waylivra and Tegsedi intangible assets.

For the six months ended June 30, 2026, royalties of $3.2 million and $2.9 million related to Tegsedi and Waylivra, respectively, were recorded on the consolidated balance sheet within intangible assets, net. As of June 30, 2026, a royalty payable of $2.0 million and $0.4 million for Tegsedi and Waylivra, respectively, was recorded on the consolidated balance sheet within accounts payable and accrued expenses.

As of the quarter ended June 30, 2026, aggregate Sephience global net sales in the prior four consecutive quarters exceeded $250.0 million, which, pursuant to the Censa Merger Agreement, triggered a $30.0 million net sales milestone payment to the former Censa securityholders. The $30.0 million milestone was recorded in accounts payable and accrued expenses on our consolidated balance sheet as of June 30, 2026. These milestones were recorded as intangible assets and are being amortized to cost of product sales over their expected useful lives on a straight-line basis.

The former Censa securityholders may also be entitled to receive other contingent payments subject to certain terms set forth in the Censa Merger Agreement related to sales of Sephience. In accordance with the guidance for an asset acquisition, the Company will record such payments when they become payable to the former Censa securityholders and increase the cost basis for the Sephience intangible asset. For the six months ended June 30, 2026, royalties of $2.2 million were recorded for Sephience. As of June 30, 2026, a royalty payable of $2.2 million for Sephience was recorded on the consolidated balance sheet within accounts payable and accrued expenses.

For the three months ended June 30, 2026 and 2025, the Company recognized amortization expense of $11.8 million and $4.1 million, respectively, related to its intangible assets. For the six months ended June 30, 2026 and 2025, the Company recognized amortization expense of $23.4 million and $7.9 million, respectively, related to its intangible assets.

The estimated future amortization of the Company’s intangible assets is expected to be as follows:


		As of June 30, 2026
2026		$	24,790
2027			49,581
2028			49,581
2029			43,571
2030 and thereafter			235,403
Total		$	402,926

The weighted average remaining amortization period of the definite-lived intangibles as of June 30, 2026 is 10.7 years.

Goodwill

As a result of the Agilis Merger on August 23, 2018, the Company recorded $82.3 million of goodwill. As of June 30, 2026, there have been no changes to the balance of goodwill since the date of the Agilis Merger. Accordingly, the goodwill balance as of June 30, 2026 is $82.3 million.

Stock award plan - Stock Option Activity (Details) - Stock option - USD ($) $ / shares in Units, $ in Thousands	6 Months Ended
	Jun. 30, 2026
Number of options
Outstanding at the beginning of the period (in shares)	6,396,202
Granted (in shares)	757,556
Exercised (in shares)	(506,463)
Forfeited/Cancelled (in shares)	(21,132)
Outstanding at the end of the period (in shares)	6,626,163
Expected to vest at the end of the period (in shares)	1,593,265
Exercisable at the end of the period (in shares)	4,855,637
Weighted- average exercise price
Outstanding at the beginning of the period (in dollars per share)	$ 43.02
Granted (in dollars per share)	76.44
Exercised (in dollars per share)	38.8
Forfeited/Cancelled (in dollars per share)	43.72
Outstanding at the end of the period (in dollars per share)	47.16
Expected to vest at the end of the period (in dollars per share)	54.41
Exercisable at the end of the period (in dollars per share)	$ 44.32
Weighted- average remaining contractual term
Outstanding at the end of the period	5 years 10 months 2 days
Expected to vest at the end of the period	8 years 7 months 13 days
Exercisable at the end of the period	4 years 9 months 21 days
Aggregate intrinsic value (in thousands)
Outstanding at the end of the period (in dollars)	$ 227,986
Expected to vest at the end of the period (in dollars)	43,276
Exercisable at the end of the period (in dollars)	$ 180,891

Insider Trading Arrangements

3 Months Ended

Jun. 30, 2026

Trading Arrangements, by Individual

Non-Rule 10b5-1 Arrangement Adopted

false

Non-Rule 10b5-1 Arrangement Terminated

false

Common Stock Trading Arrangement | Michael Schmertzler

Trading Arrangements, by Individual

Material Terms of Trading Arrangement

Director and Officer Trading Arrangements

A portion of the compensation of our directors and officers (as defined in Rule 16a-1(f) under the Securities Exchange Act of 1934, as amended, or the Exchange Act) is in the form of equity awards and, from time to time, directors and officers engage in open-market transactions with respect to the securities acquired pursuant to such equity awards or other Company securities, including to satisfy tax withholding obligations when equity awards vest or are exercised, and for diversification or other personal reasons.

Transactions in Company securities by directors and officers are required to be made in accordance with our insider trading policy, which requires that the transactions be in accordance with applicable U.S. federal securities laws that prohibit trading while in possession of material nonpublic information. Rule 10b5-1 under the Exchange Act provides an affirmative defense that enables directors and officers to prearrange transactions in Company securities in a manner that avoids concerns about initiating transactions while in possession of material nonpublic information.

The following table describes, for the quarterly period covered by this report, each trading arrangement for the sale or purchase of Company securities adopted or terminated by our directors and officers that is either (1) a contract, instruction or written plan intended to satisfy the affirmative defense conditions of Rule 10b5-1(c), or a “Rule 10b5-1 trading arrangement”, or (2) a “non-Rule 10b5-1 trading arrangement” (as defined in Item 408(c) of Regulation S-K):



Name (Title)	Action Taken (Date of Action)	Type of Trading Arrangement	Nature of Trading Arrangement	Duration of Trading Arrangement	Aggregate Number of Securities
Michael Schmertzler (Director) (by Section Six Partners, L.P., of which Mr. Schmertzler is a general partner and limited partner)	Adoption (May 14, 2026)	Rule 10b5-1 trading arrangement	Sale	Until February 1, 2027, or until such earlier date upon which all transactions are completed	Up to 170,000 shares

Name

Michael Schmertzler

Title

Director

Rule 10b5-1 Arrangement Adopted

true

Adoption Date

May 14, 2026

Expiration Date

February 1, 2027

Aggregate Available

170,000

Fair value of financial instruments and marketable securities - Available-for-sale securities (Details) - USD ($) $ in Thousands	Jun. 30, 2026	Dec. 31, 2025
Fair value of financial instruments and marketable securities
Securities in an unrealized loss position less than 12 months - Unrealized losses	$ (1,371)	$ (18)
Securities in an unrealized loss position less than 12 months - Fair Value	1,067,985	95,374
Total - Unrealized losses	(1,371)	(18)
Total - Fair Value	1,067,985	95,374
Commercial paper
Fair value of financial instruments and marketable securities
Securities in an unrealized loss position less than 12 months - Unrealized losses	(82)	(9)
Securities in an unrealized loss position less than 12 months - Fair Value	120,039	50,306
Total - Unrealized losses	(82)	(9)
Total - Fair Value	120,039	50,306
Corporate debt securities
Fair value of financial instruments and marketable securities
Securities in an unrealized loss position less than 12 months - Unrealized losses	(797)	(9)
Securities in an unrealized loss position less than 12 months - Fair Value	493,847	45,068
Total - Unrealized losses	(797)	(9)
Total - Fair Value	493,847	$ 45,068
Government obligations
Fair value of financial instruments and marketable securities
Securities in an unrealized loss position less than 12 months - Unrealized losses	(492)
Securities in an unrealized loss position less than 12 months - Fair Value	454,099
Total - Unrealized losses	(492)
Total - Fair Value	$ 454,099

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

+ Details

Fair value of financial instruments and marketable securities - Level 3 valuation (Details) $ in Millions	Dec. 31, 2025 USD ($)
Changes in the fair value of warrant liability and SARs liability
Contingent consideration payable	$ 0.0

Segment information

6 Months Ended

Jun. 30, 2026

Segment information

Segment information

13. Segment information

The Company views its operations and manages its business in one operating segment: life science. The Company expects to continue to incur significant expenses as it advances product candidates through all stages of development and clinical trials and, ultimately, seek regulatory approval. The table below summarizes the significant expense categories for the life science segment regularly reviewed by the chief operating decision maker for the three and six months ended June 30, 2026 and 2025:


	Three months ended June 30,				Six months ended June 30,
	2026		2025		2026		2025
Total revenues	$	360,519	$	178,875	$	633,070	$	1,354,971
Less:
Cost of product, collaboration and license sales		10,933		8,093		30,611		15,800
Program spend		42,580		49,705		80,125		97,863
Employee costs		68,348		65,593		144,469		133,292

Manufacturing costs		6,232		19,519		15,317		34,470
Administrative costs		16,547		17,896		33,236		36,310
Occupancy costs		7,493		6,643		15,848		13,825
Other segment items (a)		124,890		76,275		232,777		221,698
Segment net income (loss)	$	83,496	$	(64,849)	$	80,687	$	801,713
Reconciliation of profit or loss
Adjustments and reconciling items		—		—		—		—
Consolidated net income (loss)	$	83,496	$	(64,849)	$	80,687	$	801,713

(a)	Other segment items includes the following:


		Three months ended June 30,				Six months ended June 30,
		2026		2025		2026		2025
Interest income	$	(18,119)	$	(20,991)	$	(36,104)	$	(38,236)
Interest expense		66,600		51,349		133,615		102,686
Income tax expense (benefit)		14,049		(6,203)		14,396		57,063
Depreciation		4,009		3,573		7,815		7,031
Amortization		11,841		4,061		23,422		7,859
All other (b)		46,510		44,486		89,633		85,295
Total other segment items	$	124,890	$	76,275	$	232,777	$	221,698

(b)	All other includes cost of goods sold, royalty, travel and entertainment, distribution costs, bad debt expense, finance costs, contract labor costs, stock compensation expense, change in the fair value of contingent consideration, and other expense.

- Definition

The entire disclosure for reporting segments including data and tables. Reportable segments include those that meet any of the following quantitative thresholds a) it's reported revenue, including sales to external customers and intersegment sales or transfers is 10 percent or more of the combined revenue, internal and external, of all operating segments b) the absolute amount of its reported profit or loss is 10 percent or more of the greater, in absolute amount of 1) the combined reported profit of all operating segments that did not report a loss or 2) the combined reported loss of all operating segments that did report a loss c) its assets are 10 percent or more of the combined assets of all operating segments.

+ References

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+ Details

Debt (Tables)

6 Months Ended

Jun. 30, 2026

Debt

Schedule of liability for sale of future royalties


		Six Months Ended June 30,
Liability for sale of future royalties- (current and noncurrent)		2026
Beginning balance as of December 31, 2025		$	2,308,366
Less: Non-cash royalty revenue payable to Royalty Pharma			(117,940)
Plus: Non-cash interest expense recognized			131,008
Ending balance		$	2,321,434
Effective interest rate as of June 30, 2026			11.0%

2031 Convertible Notes

Debt

Summary of convertible notes




		June 30, 2026			December 31, 2025
Principal		$	550,000		$	—
Less: Debt issuance costs			(14,481)			—

Net carrying amount

535,519

—

Schedule of interest expense recognized related to the convertible Notes



		Three Months Ended June 30,						Six Months Ended June 30,
		2026			2025			2026			2025
Amortization of debt issuance costs		$	119		$	—		$	119		$	—
Total		$	119		$	—		$	119		$	—
Effective interest rate			0.5	%		—	%		0.5	%		—	%

2026 Convertible Notes

Debt

Summary of convertible notes



		June 30, 2026		December 31, 2025
Principal		$	287,500	$	287,500
Conversion of the 2026 Convertible Notes			(10,000)		—
Repurchase of the 2026 Convertible Notes			(222,000)		—
Less: Debt issuance costs			(49)		(869)
Net carrying amount		$	55,451	$	286,631

Schedule of interest expense recognized related to the convertible Notes


		Three Months Ended June 30,						Six Months Ended June 30,
	2026			2025			2026			2025
Contractual interest expense	$	830		$	1,067		$	1,899		$	2,135
Amortization of debt issuance costs		265			301			570			601
Total	$	1,095		$	1,368		$	2,469		$	2,736
Effective interest rate		1.9	%		1.9	%		1.9	%		1.9	%

Accounts payable and accrued expenses

6 Months Ended

Jun. 30, 2026

Accounts payable and accrued expenses

Accounts payable and accrued expenses

5. Accounts payable and accrued expenses

Accounts payable and accrued expenses at June 30, 2026 and December 31, 2025 consist of the following:


		June 30,			December 31,
		2026			2025
Employee compensation, benefits, and related accruals		$	39,303		$	73,737
Income tax payable			—			4,677
Consulting and contracted research			16,395			25,007
Sales allowance			119,562			129,197
Sales rebates			109,741			76,704
Royalties			13,580			9,574
Accounts payable			40,447			45,487
Milestone payable			30,000			416
Other			13,217			16,473
Total		$	382,245		$	381,272

Revenue recognition - Collaboration and License Revenue (Details) $ in Thousands	1 Months Ended	3 Months Ended		6 Months Ended
	Jan. 31, 2025 USD ($)	Jun. 30, 2026 USD ($)	Jun. 30, 2025 USD ($)	Jun. 30, 2026 USD ($) product	Jun. 30, 2025 USD ($)	Nov. 30, 2011 USD ($)
Revenue recognition
Number of approved products \| product				2
Revenue		$ 360,519	$ 178,875	$ 633,070	$ 1,354,971
Collaboration and license revenue
Revenue recognition
Revenue		50,595	2,941	50,738	989,172
Royalty revenue
Revenue recognition
Revenue		71,105	57,605	117,940	94,044
Novartis
Revenue recognition
Development milestone obligation revenue received				$ 50,000
SMA License Agreement
Revenue recognition
Number of approved products \| product				1
SMA License Agreement \| Collaboration and license revenue
Revenue recognition
Revenue		0	0	$ 0	0
SMA License Agreement \| Royalty revenue
Revenue recognition
Revenue		71,100	57,600	117,900	94,000
License and Collaboration Agreement
Revenue recognition
Refund of revenue related to prior collaboration arrangements					3,500
License and Collaboration Agreement \| Novartis
Revenue recognition
Cash proceeds from upfront payment on collaboration agreement	$ 1,000,000
Percentage share of the income (loss) generated on U.S. sales	40.00%
License and Collaboration Agreement \| Novartis \| Collaboration and license revenue
Revenue recognition
Revenue		50,600	$ 2,900	50,700	$ 992,700
Research And Development Event Milestones \| SMA License Agreement
Revenue recognition
Remaining potential milestones that can be achieved		0		0
Sales Milestones \| SMA License Agreement
Revenue recognition
Remaining potential milestones that can be achieved		$ 150,000		$ 150,000
Maximum \| License and Collaboration Agreement \| Novartis
Revenue recognition
Potential future proceeds contingent on achievement of developmental, regulatory and sales milestones and tiered royalties	$ 1,900,000
Maximum \| Research And Development Event Milestones \| SMA License Agreement
Revenue recognition
Remaining potential milestones that can be achieved						$ 135,000
Maximum \| Sales Milestones \| SMA License Agreement
Revenue recognition
Remaining potential milestones that can be achieved						$ 325,000

Stock award plan - Restricted Stock (Details) - Unvested restricted stock units	6 Months Ended
	Jun. 30, 2026 $ / shares shares
Number of Shares
Balance at the beginning of the period (in shares) \| shares	3,667,130
Granted (in shares) \| shares	1,293,798
Performance based PSUs with conditions satisfied (in shares) \| shares	25,000
Vested (in shares) \| shares	(1,294,464)
Forfeited (in shares) \| shares	(169,015)
Balance at the end of the period (in shares) \| shares	3,522,449
Weighted Average Grant Date Fair Value
Balance at the beginning of the period (in dollars per share) \| $ / shares	$ 38.2
Granted (in dollars per share) \| $ / shares	76.53
Performance based PSUs with conditions satisfied (in dollars per share) \| $ / shares	46.37
Vested (in dollars per share) \| $ / shares	38.16
Forfeited (in dollars per share) \| $ / shares	45.16
Balance at the end of the period (in dollars per share) \| $ / shares	$ 52.02

Intangible assets and goodwill - Future Amortization (Details) $ in Thousands	Jun. 30, 2026 USD ($)
Intangible assets and goodwill
2026	$ 24,790
2027	49,581
2028	49,581
2029	43,571
2030 and thereafter	235,403
Total	$ 402,926

Stock award plan - Share-based compensation expense (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025
Stock option plan
Share-based compensation expense	$ 23,291	$ 18,543	$ 46,712	$ 36,603
Research and development
Stock option plan
Share-based compensation expense	10,547	9,030	21,677	17,693
Selling, general and administrative
Stock option plan
Share-based compensation expense	$ 12,744	$ 9,513	$ 25,035	$ 18,910

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
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-Name Accounting Standards Codification
-Section 50
-Paragraph 2
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Reference 2: http://www.xbrl.org/2003/role/disclosureRef
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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+ Details

Debt - Liability for Sale of Future Royalties Narrative (Details) - A&R Royalty Purchase Agreement $ in Millions	6 Months Ended
	Jun. 30, 2026 USD ($) item
Assigned royalty payments
Aggregate upfront cash consideration received for sale of future royalties	$ 2,100.0
Future royalties sold percentage	100.00%
Potential purchase price payments \| item	3
Royalty Pharma Receives Greater Than $347 Million of Assigned Royalties in 2027
Assigned royalty payments
Contingent purchase price payments	$ 20.0
Assigned royalty payments	347.0
Royalty Pharma Receives Greater Than $363 Million of Assigned Royalties in 2028
Assigned royalty payments
Contingent purchase price payments	20.0
Assigned royalty payments	363.0
Royalty Pharma Receives Greater Than $379 Million of Assigned Royalties in 2029
Assigned royalty payments
Contingent purchase price payments	20.0
Assigned royalty payments	$ 379.0

Consolidated Balance Sheets - USD ($) $ in Thousands	Jun. 30, 2026	Dec. 31, 2025
Current assets:
Cash and cash equivalents	$ 1,049,887	$ 984,648
Marketable securities	1,179,195	960,723
Trade and royalty receivables, net	239,044	181,621
Inventory, net	91,354	79,647
Prepaid expenses and other current assets	57,879	67,309
Total current assets	2,617,359	2,273,948
Fixed assets, net	51,487	55,371
Intangible assets, net	402,926	388,753
Goodwill	82,341	82,341
Operating lease ROU assets	74,783	80,053
Deposits and other assets	21,581	18,301
Total assets	3,250,477	2,898,767
Current liabilities:
Accounts payable and accrued expenses	382,245	381,272
Current portion of long-term debt	55,451	286,631
Deferred revenue	1,192	2,040
Operating lease liabilities- current	11,965	12,484
Finance lease liabilities- current	6,000	3,000
Liability for sale of future royalties- current	314,215	283,000
Total current liabilities	771,068	968,427
Long-term debt	535,519
Operating lease liabilities- noncurrent	87,533	93,583
Finance lease liabilities- noncurrent		3,000
Liability for sale of future royalties- noncurrent	2,007,219	2,025,366
Other long-term liabilities	14,501	13,704
Total liabilities	3,415,840	3,104,080
Stockholders' deficit:
Common stock, $0.001 par value. Authorized 250,000,000 shares; issued and outstanding 83,327,286 shares at June 30, 2026. Authorized 250,000,000 shares; issued and outstanding 81,474,366 shares at December 31, 2025.	82	80
Additional paid-in capital	2,712,080	2,748,335
Accumulated other comprehensive income	6,017	10,501
Accumulated deficit	(2,883,542)	(2,964,229)
Total stockholders' deficit	(165,363)	(205,313)
Total liabilities and stockholders' deficit	$ 3,250,477	$ 2,898,767

Debt - Convertible Notes (Details) - USD ($) $ in Thousands	3 Months Ended	6 Months Ended
Debt - Convertible Notes (Details) - USD ($) $ in Thousands	Jun. 30, 2026	Jun. 30, 2026	Dec. 31, 2025
Long-term debt
Conversion of the 2026 Convertible Notes		$ (12,879)
2031 Convertible Notes \| Convertible debt
Long-term debt
Principal	$ 550,000	550,000
Less: Debt issuance costs	(14,481)	(14,481)
Net carrying amount	535,519	535,519
2026 Convertible Notes \| Convertible debt
Long-term debt
Principal	287,500	287,500	$ 287,500
Conversion of the 2026 Convertible Notes	(12,900)	(10,000)
Repurchase of the 2026 Convertible Notes		(222,000)
Less: Debt issuance costs	(49)	(49)	(869)
Net carrying amount	$ 55,451	$ 55,451	$ 286,631

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

+ Details

- Definition

Amount, after deduction of unamortized premium (discount) and debt issuance cost, of long-term debt. Excludes lease obligation.

+ References

Reference 1: http://fasb.org/us-gaap/role/ref/legacyRef
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-Section S99
-Paragraph 1
-Subparagraph (SX 210.5-02(22))
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+ Details

Segment information - Significant Expenses by Segment (Details) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2026 USD ($) segment	Jun. 30, 2025 USD ($) segment	Jun. 30, 2026 USD ($) segment	Jun. 30, 2025 USD ($) segment
Segment and geographic information
Number of operating segments \| segment	1	1	1	1
Number of reportable segments \| segment	1	1	1	1
Total revenues	$ 360,519	$ 178,875	$ 633,070	$ 1,354,971
Net income (loss) attributable to common stockholders	83,496	(64,849)	80,687	801,713
Income tax expense (benefit)	14,049	(6,203)	14,396	57,063
Amortization	11,841	4,061	23,422	7,859
Operating segments \| Life Science
Segment and geographic information
Total revenues	360,519	178,875	633,070	1,354,971
Cost of product, collaboration and license sales	10,933	8,093	30,611	15,800
Program spend	42,580	49,705	80,125	97,863
Employee costs	68,348	65,593	144,469	133,292
Manufacturing costs	6,232	19,519	15,317	34,470
Administrative costs	16,547	17,896	33,236	36,310
Occupancy costs	7,493	6,643	15,848	13,825
Other segment items	124,890	76,275	232,777	221,698
Net income (loss) attributable to common stockholders	83,496	(64,849)	80,687	801,713
Interest income	(18,119)	(20,991)	(36,104)	(38,236)
Interest expense	66,600	51,349	133,615	102,686
Income tax expense (benefit)	14,049	(6,203)	14,396	57,063
Depreciation	4,009	3,573	7,815	7,031
Amortization	11,841	4,061	23,422	7,859
All other	46,510	44,486	89,633	85,295
Total other segment items	$ 124,890	$ 76,275	$ 232,777	$ 221,698

Consolidated Statements of Operations - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025
Revenues:
Total revenues	$ 360,519	$ 178,875	$ 633,070	$ 1,354,971
Operating expenses:
Cost of product, collaboration and license sales, excluding amortization of acquired intangible assets	19,921	11,420	47,949	24,282
Amortization of acquired intangible assets	11,841	4,061	23,422	7,859
Research and development	99,150	112,990	200,023	221,963
Selling, general and administrative	80,630	85,262	166,813	166,223
Change in the fair value of contingent consideration				(800)
Tangible asset impairment and losses on transactions, net		99	927	176
Total operating expenses	211,542	213,832	439,134	419,703
Income (loss) from operations	148,977	(34,957)	193,936	935,268
Interest expense, net	(48,481)	(30,358)	(97,511)	(64,450)
Other expense, net	(2,951)	(5,737)	(1,342)	(12,042)
Income (loss) before income tax (expense) benefit	97,545	(71,052)	95,083	858,776
Income tax (expense) benefit	(14,049)	6,203	(14,396)	(57,063)
Net income (loss) attributable to common stockholders	$ 83,496	$ (64,849)	$ 80,687	$ 801,713
Weighted-average shares outstanding:
Basic (in shares)	83,006,808	78,151,240	82,765,248	78,438,830
Diluted (in shares)	92,020,009	78,151,240	91,824,273	86,502,578
Net income(loss) per share
Basic (in dollars per share)	$ 1.01	$ (0.83)	$ 0.97	$ 10.22
Diluted (in dollars per share)	$ 0.92	$ (0.83)	$ 0.9	$ 9.29
Net product revenue
Revenues:
Total revenues	$ 238,819	$ 118,329	$ 464,392	$ 271,755
Collaboration and license revenue
Revenues:
Total revenues	50,595	2,941	50,738	989,172
Royalty revenue
Revenues:
Total revenues	$ 71,105	$ 57,605	$ 117,940	$ 94,044

Stock award plan

6 Months Ended

Jun. 30, 2026

Stock award plan

Stock award plan

8. Stock award plan

In May 2013, the Company’s Board of Directors and stockholders approved the 2013 Long-Term Incentive Plan, which became effective upon the closing of the Company’s initial public offering. On June 8, 2022 (the “Restatement Effective Date”), the Company’s stockholders approved the Amended and Restated 2013 Long-Term Incentive Plan (the “Amended 2013 LTIP”). The Amended 2013 LTIP provides for the grant of incentive stock options, nonstatutory stock options, restricted stock units and other stock-based awards. The number of shares of common stock reserved for issuance under the Amended 2013 LTIP is the sum of (A) the number of shares of the Company’s common stock (up to 16,724,212 shares)

that is equal to the sum of (1) the number of shares issued under the 2013 Long-Term Incentive Plan prior to the Restatement Effective Date, (2) the number of shares that remain available for issuance under the 2013 Long-Term Incentive Plan immediately prior to the Restatement Effective Date and (3) the number of shares subject to awards granted under the 2013 Long-Term Incentive Plan prior to the Restatement Effective Date that are outstanding as of the Restatement Effective Date, plus (B) from and after the Restatement Effective Date, an additional 8,475,000 shares of Common Stock. As of June 30, 2026, awards for 3,408,235 shares of common stock are available for issuance under the Amended 2013 LTIP.

In January 2020, the Company’s Board of Directors approved the 2020 Inducement Stock Incentive Plan. The 2020 Inducement Stock Incentive Plan provides for the grant of incentive stock options, nonstatutory stock options, restricted stock awards and other stock-based awards for, initially, up to at the time, an aggregate of 1,000,000 shares of common stock. Any grants made under the 2020 Inducement Stock Incentive Plan must be made pursuant to the Nasdaq Listing Rule 5635(c)(4) inducement grant exception as a material component of the Company’s new hires’ employment compensation. In December 2020, the Company’s Board of Directors approved an additional 1,000,000 shares of common stock that may be issued under the 2020 Inducement Stock Incentive Plan. In April 2022, the Company’s Board of Directors approved a reduction in the total number of shares of common stock that may be issued under the 2020 Inducement Stock Incentive Plan to 1,300,000 shares. In December 2022, the Company’s Board of Directors approved an additional 1,700,000 shares of common stock that may be issued under the 2020 Inducement Stock Incentive Plan. As of June 30, 2026, awards for 1,754,494 shares of common stock were available for issuance under the 2020 Inducement Stock Incentive Plan.

The Board of Directors has the authority to select the individuals to whom options are granted and determine the terms of each option, including (i) the number of shares of common stock subject to the option; (ii) the date on which the option becomes exercisable; (iii) the option exercise price, which, in the case of incentive stock options, must be at least 100% (110% in the case of incentive stock options granted to a stockholder owning in excess of 10% of the Company’s stock) of the fair market value of the common stock as of the date of grant; and (iv) the duration of the option (which, in the case of incentive stock options, may not exceed ten years). Options typically vest over a four-year period.

Stock option awards—From January 1, 2026 through June 30, 2026, the Company issued a total of 757,556 stock options to various employees. Of those, 25,755 were inducement grants for non-statutory stock options, all of which were made pursuant to the 2020 Inducement Stock Incentive Plan. A summary of stock option activity is as follows:


							Weighted-
				Weighted-			average		Aggregate
				average			remaining		intrinsic
		Number of		exercise			contractual		value(in
		options		price			term		thousands)

Outstanding at December 31, 2025		6,396,202		$	43.02
Granted		757,556		$	76.44
Exercised		(506,463)		$	38.80
Forfeited/Cancelled		(21,132)		$	43.72
Outstanding at June 30, 2026		6,626,163		$	47.16		5.84	years	$	227,986
Expected to vest at June 30, 2026		1,593,265		$	54.41		8.62	years	$	43,276
Exercisable at June 30, 2026		4,855,637		$	44.32		4.81	years	$	180,891

The fair value of grants made in the six months ended June 30, 2026, was contemporaneously estimated on the date of grant using the following assumptions:


		Six months ended
		June 30, 2026
Risk-free interest rate		3.78% - 4.21%
Expected volatility		54%
Expected term		5.5 years

The Company assumed no expected dividends for all grants. The weighted average grant date fair value of options granted during the six months ended June 30, 2026 was $40.21 per share.

The expected term of options was estimated based on the Company’s historical exercise data and the expected volatility of options was estimated based on the Company’s historical stock volatility. The risk-free rate of the options was based on U.S. Government Securities Treasury Constant Maturities yields at the date of grant for a term similar to the expected term of the option.

Restricted Stock Units—Restricted stock units are granted subject to certain restrictions, including in some cases service or time conditions (restricted stock). The grant-date fair value of restricted stock units, which have been determined based upon the market value of the Company’s shares on the grant date, are expensed over the vesting period. From January 1, 2026, through June 30, 2026, the Company issued a total of 1,293,798 restricted stock units to various employees. Of those, 30,610 were inducement grants for restricted stock units, all of which were made pursuant to the 2020 Inducement Stock Incentive Plan.

The following table summarizes information on the Company’s restricted stock units, including performance-based restricted stock units (“PSUs”) for which the performance conditions were satisfied:


	Restricted Stock Units
		Weighted
		Average
		Grant
	Number of	Date
	Shares	Fair Value
Unvested at December 31, 2025	3,667,130	$	38.20
Granted	1,293,798		76.53
Performance based PSUs with conditions satisfied	25,000		46.37
Vested	(1,294,464)		38.16
Forfeited	(169,015)		45.16
Unvested at June 30, 2026	3,522,449	$	52.02

Performance-based Restricted Stock Units—The Company has granted Chief Executive Officer, Dr. Matthew Klein PSUs which will vest only if challenging performance goals relating to development and regulatory milestones are achieved which are considered “PSUs with performance conditions”. The Company also granted Dr. Klein PSUs, which will vest only if challenging performance goals relating to stock price goals are achieved. In addition, the Company granted PSUs to Dr. Klein for which the number of PSUs that vest shall be determined based upon the Company’s total shareholder return (“TSR”) over the period beginning on November 14, 2025 and ending on December 31, 2028 relative to the TSR of the group of companies in the Nasdaq Biotechnology Index. Together, the PSUs with stock price goals and the PSUs related to the TSR are considered the “PSUs with market conditions”.

During the six months ended June 30, 2026, 25,000 of the PSUs with performance conditions granted to Dr. Klein in December 2024 were satisfied due to the Company’s achievement of corporate goals and are included in the restricted stock units table above. Also, during the six months ended June 30, 2026, the Company granted Dr. Klein 37,500 PSUs with performance conditions which will only vest if challenging performance goals relating to regulatory milestones are achieved over an approximately three-year performance period. The expenses related to satisfied PSUs were $0.7 million and $1.2 million for the three and six months ended June 30, 2026, respectively. The achievement of the remaining performance goals for the PSUs with performance conditions granted to Dr. Klein has not yet been deemed probable and therefore no expense has been recognized to date. The expenses related to the PSUs with market conditions granted to Dr. Klein were $1.1 million and $2.3 million for the three and six months ended June 30, 2026, respectively, and $0.3 million for each of the three and six months ended June 30, 2025.

The following table summarizes information regarding the Company’s PSUs granted to Dr. Klein:


		Performance-based Restricted Stock Units

	Performance Conditions	Market Conditions	Total
Total PSUs granted for which the performance or market conditions were not yet satisfied at December 31, 2025	25,000	115,625	140,625
PSUs with performance or market conditions granted during the period	37,500	—	37,500
PSUs with performance or market conditions satisfied during the period	(25,000)	—	(25,000)
Total PSUs granted for which the performance or market conditions were not yet satisfied at June 30, 2026	37,500	115,625	153,125

Employee Stock Purchase Plan—In June 2016, the Company established an Employee Stock Purchase Plan (as amended, the “ESPP” or the “Plan”), for certain eligible employees. The Plan is administered by the Company’s Board of Directors or a committee appointed by the Company’s Board of Directors. In June 2021, the Plan was amended to increase the total number of shares available for purchase under the Plan from one million shares to two million shares of the Company’s common stock. Employees may participate over a six month period through payroll withholdings and may purchase, at the end of the six month period, the Company’s common stock at a purchase price of at least 85% of the closing price of a share of the Company’s common stock on the first business day of the offering period or the closing price of a share of the Company’s common stock on the last business day of the offering period, whichever is lower. No participant will be granted a right to purchase the Company’s common stock under the Plan if such participant would own more than 5% of the total combined voting power of the Company or any subsidiary of the Company after such purchase. For the three and six months ended June 30, 2026, the Company recorded $0.5 million and $1.0 million, respectively, in compensation expense related to the ESPP.

The Company recorded share-based compensation expense in the statement of operations related to incentive stock options, nonstatutory stock options, restricted stock units, performance-based restricted stock units and the ESPP as follows:


	Three Months Ended June 30,				Six Months Ended June 30,
	2026		2025		2026		2025
Research and development	$	10,547	$	9,030	$	21,677	$	17,693
Selling, general and administrative		12,744		9,513		25,035		18,910
Total	$	23,291	$	18,543	$	46,712	$	36,603

As of June 30, 2026, there was approximately $206.8 million of total unrecognized compensation cost related to unvested share-based compensation arrangements granted under the Company’s equity award plans. This cost is expected to be recognized as share-based compensation expense over the weighted average remaining service period of approximately 2.9 years.

Capitalization

6 Months Ended

Jun. 30, 2026

Capitalization

Capitalization

6. Capitalization

In August 2019, the Company entered into an At the Market Offering Sales Agreement (the “Sales Agreement”) with Cantor Fitzgerald and RBC Capital Markets, LLC (together, the “Sales Agents”), pursuant to which, the Company may offer and sell shares of its common stock, having an aggregate offering price of up to $125.0 million from time to time through the Sales Agents by any method that is deemed to be an “at the market offering” as defined in

Rule 415(a)(4) promulgated under the Securities Act of 1933, as amended. No shares were sold during the three and six months ended June 30, 2026 and 2025. The remaining shares of the Company’s common stock available to be issued and sold, under the At the Market Offering, have an aggregate offering price of up to $93.0 million as of June 30, 2026.

- Definition

The entire disclosure for equity.

+ References

+ Details

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
Pay vs Performance Disclosure - USD ($) $ in Thousands	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025
Pay vs Performance Disclosure
Net Income (Loss)	$ 83,496	$ (64,849)	$ 80,687	$ 801,713

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

+ References

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+ Details

Commitments and Contingencies - Censa agreement (Details) - USD ($) $ in Thousands		3 Months Ended		6 Months Ended
	Aug. 05, 2025	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025	Dec. 31, 2025	Jul. 31, 2025	May 05, 2020
Commitments and contingencies
Revenue		$ 360,519	$ 178,875	$ 633,070	$ 1,354,971
Milestone Payable		30,000	25,150	30,000	25,150	$ 416
Product
Commitments and contingencies
Revenue		238,819	118,329	464,392	271,755
Censa Merger Agreement
Commitments and contingencies
Maximum amount of upfront consideration		250,000		250,000
Rights Satisfaction Agreement
Commitments and contingencies
Maximum amount of upfront consideration	$ 250,000
Maximum amount of additional milestone payable	100,000
Additional milestone payable for right holders	500,000
Payment of upfront consideration	225,100
Additional milestone obligated to be paid	$ 90,000
Rights Satisfaction Agreement \| Annual net sales less than or equal to $250 million
Commitments and contingencies
Percentage on annual net sales	8.00%
Rights Satisfaction Agreement \| Annual net sales greater than $250 million but less than $500 million
Commitments and contingencies
Percentage on annual net sales	10.00%
Rights Satisfaction Agreement \| Annual net sales greater than $500 million
Commitments and contingencies
Percentage on annual net sales	12.00%
Rights Satisfaction Agreement \| First occurrence of a three or fewer consecutive calendar year period
Commitments and contingencies
Threshold consecutive calendar period	3 years
Amount of net sales of product	$ 3,000,000
Rights Satisfaction Agreement \| First occurrence of a five or fewer consecutive calendar year period
Commitments and contingencies
Threshold consecutive calendar period	5 years
Amount of net sales of product	$ 5,000,000
Rights Satisfaction Agreement \| First occurrence of a seven or fewer consecutive calendar year period
Commitments and contingencies
Threshold consecutive calendar period	7 years
Amount of net sales of product	$ 7,000,000
Rights Satisfaction Agreement \| First occurrence of a nine or fewer consecutive calendar year period
Commitments and contingencies
Threshold consecutive calendar period	9 years
Amount of net sales of product	$ 9,000,000
Rights Satisfaction Agreement \| First occurrence of a eleven or fewer consecutive calendar year period
Commitments and contingencies
Threshold consecutive calendar period	11 years
Amount of net sales of product	$ 11,000,000
Minimum \| Rights Satisfaction Agreement \| Product \| Annual net sales greater than $250 million but less than $500 million
Commitments and contingencies
Revenue	250,000
Minimum \| Rights Satisfaction Agreement \| Product \| Annual net sales greater than $500 million
Commitments and contingencies
Revenue	500,000
Maximum \| Rights Satisfaction Agreement \| Product \| Annual net sales less than or equal to $250 million
Commitments and contingencies
Revenue	250,000
Maximum \| Rights Satisfaction Agreement \| Product \| Annual net sales greater than $250 million but less than $500 million
Commitments and contingencies
Revenue	$ 500,000
Censa
Commitments and contingencies
Asset acquisition, development and regulatory milestones								$ 109,000
Censa \| Censa Merger Agreement
Commitments and contingencies
Milestone payments triggered		$ 30,000	$ 25,000	30,000	$ 25,000
Milestone Payable							$ 32,500
Threshold revenue to milestone payment				250,000
Censa \| Maximum
Commitments and contingencies
Asset acquisition, milestone, amount								217,500
Asset acquisition, net sales milestone								$ 160,000
Former Censa Securityholders \| Development and Regulatory Milestones
Commitments and contingencies
Milestone obligation payments made				152,500
Potential milestone payments due				217,500
Former Censa Securityholders \| Rights Satisfaction Agreement
Commitments and contingencies
Ownership percentage prior to merger	90.00%
Sephience \| Censa
Commitments and contingencies
Royalty payments				2,200
Tegsedi \| Censa
Commitments and contingencies
Royalty payments				3,200
Waylivra \| Censa
Commitments and contingencies
Royalty payments				$ 2,900

Inventory (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
Inventory (Details) - USD ($) $ in Thousands	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025	Dec. 31, 2025
Inventory
Raw materials	$ 10,521		$ 10,521		$ 2,291
Work in progress	57,775		57,775		55,686
Finished goods	23,058		23,058		21,670
Total inventory	91,354		91,354		$ 79,647
Inventory Write-down	900	$ 800	2,700	$ 4,300
Loss related to inventory impairments	$ 0	$ 0	$ 800	$ 0

Segment information (Tables)

6 Months Ended

Jun. 30, 2026

Segment information

Schedule of significant expense categories by operating segment


	Three months ended June 30,				Six months ended June 30,
	2026		2025		2026		2025
Total revenues	$	360,519	$	178,875	$	633,070	$	1,354,971
Less:
Cost of product, collaboration and license sales		10,933		8,093		30,611		15,800
Program spend		42,580		49,705		80,125		97,863
Employee costs		68,348		65,593		144,469		133,292

Manufacturing costs		6,232		19,519		15,317		34,470
Administrative costs		16,547		17,896		33,236		36,310
Occupancy costs		7,493		6,643		15,848		13,825
Other segment items (a)		124,890		76,275		232,777		221,698
Segment net income (loss)	$	83,496	$	(64,849)	$	80,687	$	801,713
Reconciliation of profit or loss
Adjustments and reconciling items		—		—		—		—
Consolidated net income (loss)	$	83,496	$	(64,849)	$	80,687	$	801,713

(a)	Other segment items includes the following:


		Three months ended June 30,				Six months ended June 30,
		2026		2025		2026		2025
Interest income	$	(18,119)	$	(20,991)	$	(36,104)	$	(38,236)
Interest expense		66,600		51,349		133,615		102,686
Income tax expense (benefit)		14,049		(6,203)		14,396		57,063
Depreciation		4,009		3,573		7,815		7,031
Amortization		11,841		4,061		23,422		7,859
All other (b)		46,510		44,486		89,633		85,295
Total other segment items	$	124,890	$	76,275	$	232,777	$	221,698

(b)	All other includes cost of goods sold, royalty, travel and entertainment, distribution costs, bad debt expense, finance costs, contract labor costs, stock compensation expense, change in the fair value of contingent consideration, and other expense.

Commitments and Contingencies - Narrative (Details) $ in Millions	6 Months Ended
	Jun. 30, 2026 USD ($)
Agilis
Other Commitments
Development milestone payments which the entity is obligated to pay	$ 40.0
Agilis \| Minimum
Other Commitments
Product sales (as a percent)	2.00%
Agilis \| Maximum
Other Commitments
Development milestone payments which the entity is obligated to pay	$ 60.0
Priority review voucher amount	535.0
Potential sales milestones	$ 150.0
Product sales (as a percent)	6.00%
BioElectron
Other Commitments
Remaining potential contingent milestone	$ 200.0
BioElectron \| Maximum
Other Commitments
Development milestone payments which the entity is obligated to pay	200.0
Emflaza/Kebilidi \| Agilis
Other Commitments
Potential sales milestones	$ 50.0

Fair value of financial instruments and marketable securities

6 Months Ended

Jun. 30, 2026

Fair value of financial instruments and marketable securities

Fair value of financial instruments and marketable securities

4. Fair value of financial instruments and marketable securities

The Company follows the fair value measurement rules, which provideguidance on the use of fair value in accounting and disclosure for assets and liabilities when such accounting and disclosure is called for by other accounting literature. These rules establish a fair value hierarchy for inputs to be used to measure fair value of financial assets and liabilities. This hierarchy prioritizes the inputs to valuation techniques used to measure fair value into three levels: Level 1 (highest priority), Level 2, and Level 3 (lowest priority).

●

Level 1—Unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the balance sheet date.

●

Level 2—Inputs other than quoted prices included within Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs include quoted prices for similar assets and liabilities in active markets, quoted prices for identical or similar assets or liabilities in markets that are not active, inputs other than quoted prices that are observable for the asset or liability (i.e., interest rates, yield curves, etc.), and inputs

that are derived principally from or corroborated by observable market data by correlation or other means (market corroborated inputs).

●

Level 3—Inputs are unobservable and reflect the Company’s assumptions as to what market participants would use in pricing the asset or liability. The Company develops these inputs based on the best information available.

Cash equivalents and marketable securities are reflected in the accompanying financial statements at fair value. The carrying amount of receivables and accounts payable and accrued expenses approximates fair value due to the short-term nature of those instruments.

The Company’s marketable securities consist of both debt securities and equity investments. The Company previously owned common stock in ClearPoint Neuro, Inc. (“ClearPoint”) (formerly MRI Interventions, Inc.), a publicly traded medical device company. The ClearPoint equity investments (collectively, the “ClearPoint Equity Investments”) represented financial instruments, and therefore, were recorded at fair value, which was readily determinable. As of December 31, 2025, the Company sold all of its ClearPoint Equity Investments.

The Company has an investment in mutual funds that is denominated in a foreign currency and is classified as marketable securities on the Company’s consolidated balance sheets. This equity investment is reported at fair value, as it is readily available, and as such is classified as a Level 1 asset. Unrealized holding gains and losses for this equity investment are included as components of interest expense, net within the consolidated statement of operations.

The tables presented below are a summary of changes in the fair value for the Company’s marketable securities – equity investments and ClearPoint Equity Investments for the three and six months ended June 30, 2026 and June 30, 2025:


	Ending				Foreign								Ending
	Balance at				Currency								Balance at
	March 31,		Unrealized		Unrealized			Investments					June 30,
	2026		Gain		Gain			Purchased			Sales		2026
Marketable securities - equity investments	$	48,443		700		1,083			9,019			(27,614)	$	31,631
Total Fair Value	$	48,443	$	700	$	1,083		$	9,019		$	(27,614)	$	31,631


	Ending				Foreign								Ending
	Balance at				Currency								Balance at
	March 31,		Unrealized		Unrealized			Investments					June 30,
	2025		Gain		Gain			Purchased			Sales		2025
Marketable securities - equity investments	$	28,187		764		1,665			11,820			(8,233)	$	34,203
ClearPoint Equity Investments		10,637		44		—			—			—		10,681
Total Fair Value	$	38,824	$	808	$	1,665		$	11,820		$	(8,233)	$	44,884


	Ending				Foreign								Ending
	Balance at				Currency								Balance at
	December 31,		Unrealized		Unrealized			Investments					June 30,
	2025		Gain		Gain			Purchased			Sales		2026
Marketable securities - equity investments	$	31,596		1,914		2,644			32,976			(37,499)	$	31,631
Total Fair Value	$	31,596	$	1,914	$	2,644		$	32,976		$	(37,499)	$	31,631


	Ending				Foreign									Ending
	Balance at				Currency									Balance at
	December 31,		Unrealized		Unrealized			Investments						June 30,
	2024		Gain/(Loss)		Gain			Purchased			Sales			2025
Marketable securities - equity investments	$	29,034		1,538		3,731			17,029			(17,129)		$	34,203
ClearPoint Equity Investments		13,759		(3,078)		—			—			—			10,681
Total Fair Value	$	42,793	$	(1,540)	$	3,731		$	17,029		$	(17,129)		$	44,884

Fair value of marketable securities that are classified as available for sale debt securities is based upon market prices using quoted prices in active markets for identical assets quoted on the last day of the period. In establishing the estimated fair value of the remaining available for sale debt securities, the Company used the fair value as determined by its investment advisors using observable inputs other than quoted prices.

The following represents the fair value using the hierarchy described above for the Company’s financial assets and liabilities that are required to be measured at fair value on a recurring basis as of June 30, 2026 and December 31, 2025:


	June 30, 2026
			Quoted prices		Significant
			in active		other		Significant
			markets for		observable		unobservable
			identical assets		inputs		inputs
	Total		(level 1)		(level 2)		(level 3)
Marketable securities - available for sale	$	1,147,564	$	—	$	1,147,564	$	—
Marketable securities - equity investments	$	31,631	$	31,631	$	—	$	—


	December 31, 2025
			Quoted prices		Significant
			in active		other		Significant
			markets for		observable		unobservable
			identical assets		inputs		inputs
	Total		(level 1)		(level 2)		(level 3)
Marketable securities - available for sale	$	929,127	$	—	$	929,127	$	—
Marketable securities - equity investments	$	31,596	$	31,596	$	—	$	—

No transfers of assets between Level 1, Level 2, or Level 3 of the fair value measurement hierarchy occurred during the three and six months ended June 30, 2026 and year ended December 31, 2025.

The following is a summary of marketable securities accounted for as available for sale debt securities at June 30, 2026 and December 31, 2025:


	June 30, 2026
	Amortized		Gross Unrealized
	Cost		Gains		Losses		Fair Value
Commercial paper	$	134,989	$	1	$	(82)	$	134,908
Corporate debt securities		510,870		2		(797)		510,075
Government obligations		503,068		5		(492)		502,581
Total	$	1,148,927	$	8	$	(1,371)	$	1,147,564


	December 31, 2025
	Amortized		Gross Unrealized
	Cost		Gains		Losses		Fair Value
Commercial paper	$	93,113	$	8	$	(9)	$	93,112
Corporate debt securities		279,090		181		(9)		279,262
Government obligations		556,137		616		—		556,753
Total	$	928,340	$	805	$	(18)	$	929,127

For available for sale debt securities in an unrealized loss position, the Company assesses whether it intends to sell or if it is more likely than not that the Company will be required to sell the security before recovery of its amortized cost basis. If either of the criteria regarding intent or requirement to sell is met, the security’s amortized cost basis is written down to fair value. For the three and six months ended June 30, 2026 and 2025, no write downs occurred. The Company does not intend to sell the 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, which may be maturity. The Company also reviews its available for sale debt securities in an unrealized loss position and evaluates whether the decline in fair value has resulted from credit losses

or other factors. This review is subjective, as it requires management to evaluate whether an event or change in circumstances has occurred in that period that may be related to credit issues. For the three and six months ended June 30, 2026 and 2025, no allowance was recorded for credit losses. Unrealized gains and losses are reported as a component of accumulated other comprehensive income (loss) in stockholders’ deficit.

For the three and six months ended June 30, 2026 and 2025, realized gains from the sale of available for sale debt securities were immaterial. Realized gains and losses are reported as a component of interest expense, net in the consolidated statement of operations. Reclassified amounts from other comprehensive items were determined using the actual realized gains and losses from the sales of marketable securities.

The unrealized losses and fair values of available for sale debt securities that have been in an unrealized loss position for a period of less than and greater than or equal to 12 months as of June 30, 2026 are as follows:


	June 30, 2026
	Securities in an unrealized loss				Securities in an unrealized loss
	position less than 12 months				position greater than or equal to 12 months				Total
	Unrealized losses		Fair Value		Unrealized losses		Fair Value		Unrealized losses		Fair Value
Commercial paper	$	(82)		120,039		—		—		(82)	$	120,039
Corporate debt securities	$	(797)		493,847		—		—		(797)	$	493,847
Government obligations	$	(492)		454,099		—		—		(492)	$	454,099
Total	$	(1,371)	$	1,067,985	$	—	$	—	$	(1,371)	$	1,067,985


	December 31, 2025
	Securities in an unrealized loss				Securities in an unrealized loss
	position less than 12 months				position greater than or equal to 12 months				Total
	Unrealized losses		Fair Value		Unrealized losses		Fair Value		Unrealized losses		Fair Value
Commercial paper	$	(9)		50,306		—		—		(9)	$	50,306
Corporate debt securities	$	(9)		45,068		—		—		(9)	$	45,068
Total	$	(18)	$	95,374	$	—	$	—	$	(18)	$	95,374

Available for sale debt securities at June 30, 2026 and December 31, 2025 mature as follows:


	June 30, 2026
	Less Than		More Than
	12 Months		12 Months
Commercial paper	$	134,908	$	—
Corporate debt securities		498,443		11,632
Government obligations		502,581		—
Total	$	1,135,932	$	11,632


	December 31, 2025
	Less Than		More Than
	12 Months		12 Months
Commercial paper	$	93,112	$	—
Corporate debt securities		279,262		—
Government obligations		556,753		—
Total	$	929,127	$	—

The Company classifies all of its marketable securities as current as they are all either available for sale debt securities or equity investments and are available for current operations.

Convertible senior notes

In June 2026, the Company issued, at par value, $550.0 million aggregate principal amount of 0% convertible senior notes due 2031 (the “2031 Convertible Notes”), which included an option to purchase up to an additional $50.0 million in aggregate principal amount of the 2031 Convertible Notes, which was exercised in full by the initial purchasers. The Company accounted for the 2031 Convertible Notes as a single liability measured at amortized cost, as further discussed in Note 9. The fair value of the 2031 Convertible Notes, which differs from their carrying values, is influenced by interest rates, the Company’s stock price and stock price volatility and is determined by prices for the 2031 Convertible Notes observed in market trading which are Level 2 inputs. The estimated fair value of the 2031 Convertible Notes at June 30, 2026 was $579.2 million.

In September 2019, the Company issued $287.5 million aggregate principal amount of 1.50% convertible senior notes due September 15, 2026 (the “2026 Convertible Notes”). In June 2026, the Company used a portion of the proceeds of the 2031 Convertible Notes to repurchase $222.0 million aggregate principal amount of its 2026 Convertible Notes for approximately $328.8 million, inclusive of accrued interest, as further discussed in Note 9. Additionally, during the three months ended June 30, 2026, a holder converted $10.0 million principal amount of the 2026 Convertible Notes in exchange for $12.9 million in cash and 3,506 shares of the Company’s common stock. Refer to Note 9 for further information. As of June 30, 2026, the remaining aggregate principal of the 2026 Convertible Notes is $55.5 million. The fair value of the 2026 Convertible Notes, which differs from their carrying values, is influenced by interest rates, the Company’s stock price and stock price volatility and is determined by prices for the 2026 Convertible Notes observed in market trading which are Level 2 inputs. The estimated fair value of the 2026 Convertible Notes at June 30, 2026 and December 31, 2025 was $85.5 million and $424.5 million, respectively.

Level 3 valuation

The contingent consideration payable is fair valued each reporting period with the change in fair value recorded as a gain or loss within the change in the fair value of contingent consideration on the consolidated statements of operations. In 2025, the probability of triggering the remaining contingent consideration was determined to be remote, and therefore the balance was written down to zero. Refer to Note 10 for additional details.

- Definition

The entire disclosure for the fair value of financial instruments (as defined), including financial assets and financial liabilities (collectively, as defined), and the measurements of those instruments as well as disclosures related to the fair value of non-financial assets and liabilities. Such disclosures about the financial instruments, assets, and liabilities would include: (1) the fair value of the required items together with their carrying amounts (as appropriate); (2) for items for which it is not practicable to estimate fair value, disclosure would include: (a) information pertinent to estimating fair value (including, carrying amount, effective interest rate, and maturity, and (b) the reasons why it is not practicable to estimate fair value; (3) significant concentrations of credit risk including: (a) information about the activity, region, or economic characteristics identifying a concentration, (b) the maximum amount of loss the entity is exposed to based on the gross fair value of the related item, (c) policy for requiring collateral or other security and information as to accessing such collateral or security, and (d) the nature and brief description of such collateral or security; (4) quantitative information about market risks and how such risks are managed; (5) for items measured on both a recurring and nonrecurring basis information regarding the inputs used to develop the fair value measurement; and (6) for items presented in the financial statement for which fair value measurement is elected: (a) information necessary to understand the reasons for the election, (b) discussion of the effect of fair value changes on earnings, (c) a description of [similar groups] items for which the election is made and the relation thereof to the balance sheet, the aggregate carrying value of items included in the balance sheet that are not eligible for the election; (7) all other required (as defined) and desired information.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (bbb)(1)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 2: http://www.xbrl.org/2003/role/exampleRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 55
-Paragraph 107
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482078/820-10-55-107

Reference 3: http://www.xbrl.org/2003/role/exampleRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 55
-Paragraph 100
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482078/820-10-55-100

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (c)(3)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2E
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2E

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (h)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (f)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (e)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (h)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (g)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 940
-SubTopic 820
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147478119/940-820-50-1

+ Details

Consolidated Statements of Cash Flows - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025
Cash flows from operating activities
Net Income (Loss)	$ 80,687	$ 801,713
Adjustments to reconcile net income to net cash provided by operating activities:
Depreciation and amortization	31,287	14,804
Non-cash operating lease expense	3,553	3,634
Non-cash royalty revenue related to sale of future royalties	(117,940)	(85,099)
Non-cash interest expense on liability related to sale of future royalties	131,008	99,329
Change in valuation of contingent consideration		(800)
Tangible asset impairment and losses on transactions, net	927	176
Inducement expense related to repurchase of 2026 Convertible Notes	3,386
Unrealized loss on ClearPoint Equity Investments		3,078
Unrealized gain on marketable securities - equity investments	(1,914)	(1,538)
Amortization of discounts on investments, net	(8,270)	(6,869)
Amortization of debt issuance costs	689	601
Share-based compensation expense	46,712	36,603
Unrealized foreign currency transaction losses, net	458	4,939
Changes in operating assets and liabilities:
Inventory, net	(13,217)	(8,591)
Prepaid expenses and other current assets	9,544	(12,197)
Trade and royalty receivables, net	(60,118)	(25,891)
Deposits and other assets	(579)	(827)
Accounts payable and accrued expenses	(31,213)	6,864
Other liabilities	(4,125)	(17,320)
Deferred revenue	(804)	3,845
Payments on contingent consideration		(4,684)
Net cash provided by operating activities	70,071	811,770
Cash flows from investing activities
Purchases of fixed assets	(3,389)	(2,971)
Purchases of marketable securities- available for sale	(921,643)	(847,164)
Purchases of marketable securities- equity investments	(32,976)	(17,029)
Sale of marketable securities- available for sale	709,325	247,151
Sale of marketable securities- equity investments	37,499	17,129
Acquisition of product rights and licenses	(5,108)	(3,485)
Net cash used in investing activities	(216,292)	(606,369)
Cash flows from financing activities
Proceeds from exercise of options	19,637	16,885
Proceeds from employee stock purchase plan	3,163	2,827
Repurchase of the 2026 Convertible Notes	(327,890)
Conversion of the 2026 Convertible Notes	(12,879)
Debt issuance costs related to 2031 Convertible Notes	(13,750)
Proceeds from issuance of 2031 Convertible Notes	550,000
Payments on contingent consideration obligation		(6,341)
Net cash provided by financing activities	218,281	13,371
Effect of exchange rate changes on cash	(4,062)	18,981
Net increase in cash and cash equivalents	67,998	237,753
Cash and cash equivalents, and restricted cash beginning of period	998,334	795,316
Cash and cash equivalents, and restricted cash end of period	1,066,332	1,033,069
Supplemental disclosure of cash information
Cash paid for interest	3,017	2,156
Foreign:
Income tax paid, Foreign	487
Total income taxes paid, net of refunds received	6,110	39,188
Supplemental disclosure of non-cash investing and financing activity
Unrealized loss on marketable securities, net of tax	(2,150)	(274)
Right-of-use assets obtained in exchange for operating lease obligations		850
Decrease in right-of-use assets related to lease modifications and termination	1,466
Decrease in operating lease liabilities due to lease modifications and termination	1,547
Acquisition of product rights and licenses	4,566	2,285
Fixed asset additions through tenant improvement allowance		385
Accrued debt issuance fees for 2031 Convertible Notes	850
Capital expenditures unpaid at the end of the period	593
Milestone payable	30,000	$ 25,150
Illinois
State:
Income tax paid, State	4,270
Florida
State:
Income tax paid, State	1,300
Other State
State:
Income tax paid, State	$ 53

Consolidated Statements of Stockholders' Deficit - USD ($) $ in Thousands	Common stock	Additional paid-in capital	Accumulated other comprehensive income (loss)	Accumulated deficit	Total
Balance (in shares) at Dec. 31, 2024	77,704,188
Balance at the beginning of the period at Dec. 31, 2024	$ 77	$ 2,574,611	$ (25,886)	$ (3,646,873)	$ (1,098,071)
Increase (Decrease) in Stockholders' Equity
Exercise of options (in shares)	467,285
Exercise of options		16,885			16,885
Restricted stock vesting and issuance, net (in shares)	1,134,189
Restricted stock vesting and issuance, net	$ 1				1
Issuance of common stock related to stock purchase agreement (in shares)	72,483
Issuance of common stock related to stock purchase agreement		2,827			2,827
Share-based compensation expense		36,603			36,603
Receivable from investor		254			254
Net income (loss)				801,713	801,713
Comprehensive income (loss)			33,242		33,242
Balance (in shares) at Jun. 30, 2025	79,378,145
Balance at the end of the period at Jun. 30, 2025	$ 78	2,631,180	7,356	(2,845,160)	(206,546)
Balance (in shares) at Mar. 31, 2025	79,225,276
Balance at the beginning of the period at Mar. 31, 2025	$ 78	2,608,422	(13,949)	(2,780,311)	(185,760)
Increase (Decrease) in Stockholders' Equity
Exercise of options (in shares)	36,196
Exercise of options		1,134			1,134
Restricted stock vesting and issuance, net (in shares)	44,190
Issuance of common stock related to stock purchase agreement (in shares)	72,483
Issuance of common stock related to stock purchase agreement		2,827			2,827
Share-based compensation expense		18,543			18,543
Receivable from investor		254			254
Net income (loss)				(64,849)	(64,849)
Comprehensive income (loss)			21,305		21,305
Balance (in shares) at Jun. 30, 2025	79,378,145
Balance at the end of the period at Jun. 30, 2025	$ 78	2,631,180	7,356	(2,845,160)	(206,546)
Balance (in shares) at Dec. 31, 2025	81,474,366
Balance at the beginning of the period at Dec. 31, 2025	$ 80	2,748,335	10,501	(2,964,229)	(205,313)
Increase (Decrease) in Stockholders' Equity
Exercise of options (in shares)	506,463
Exercise of options	$ 1	19,636			19,637
Restricted stock vesting and issuance, net (in shares)	1,294,464
Restricted stock vesting and issuance, net	$ 1				1
Repurchase of 2026 Convertible Notes		(102,734)			(102,734)
Conversion of 2026 Convertible Notes (in shares)	3,506
Conversion of 2026 Convertible Notes		(2,899)			(2,899)
Issuance of common stock related to stock purchase agreement (in shares)	48,487
Issuance of common stock related to stock purchase agreement		3,163			3,163
Share-based compensation expense		46,712			46,712
Receivable from investor		(133)			(133)
Net income (loss)				80,687	80,687
Comprehensive income (loss)			(4,484)		(4,484)
Balance (in shares) at Jun. 30, 2026	83,327,286
Balance at the end of the period at Jun. 30, 2026	$ 82	2,712,080	6,017	(2,883,542)	(165,363)
Balance (in shares) at Mar. 31, 2026	82,882,024
Balance at the beginning of the period at Mar. 31, 2026	$ 81	2,778,026	8,466	(2,967,038)	(180,465)
Increase (Decrease) in Stockholders' Equity
Exercise of options (in shares)	344,594
Exercise of options	$ 1	13,233			13,234
Restricted stock vesting and issuance, net (in shares)	48,675
Repurchase of 2026 Convertible Notes		(102,734)			(102,734)
Conversion of 2026 Convertible Notes (in shares)	3,506
Conversion of 2026 Convertible Notes		(2,899)			(2,899)
Issuance of common stock related to stock purchase agreement (in shares)	48,487
Issuance of common stock related to stock purchase agreement		3,163			3,163
Share-based compensation expense		23,291			23,291
Net income (loss)				83,496	83,496
Comprehensive income (loss)			(2,449)		(2,449)
Balance (in shares) at Jun. 30, 2026	83,327,286
Balance at the end of the period at Jun. 30, 2026	$ 82	$ 2,712,080	$ 6,017	$ (2,883,542)	$ (165,363)

Consolidated Balance Sheets (Parenthetical) - $ / shares	Jun. 30, 2026	Dec. 31, 2025
Consolidated Balance Sheets
Common stock, par value (in dollars per share)	$ 0.001	$ 0.001
Common stock, authorized shares (in shares)	250,000,000	250,000,000
Common stock, issued shares (in shares)	83,327,286	81,474,366
Common stock, outstanding shares (in shares)	83,327,286	81,474,366

Accounts payable and accrued expenses (Tables)

6 Months Ended

Jun. 30, 2026

Accounts payable and accrued expenses

Schedule of components of accounts payable and accrued expenses


		June 30,			December 31,
		2026			2025
Employee compensation, benefits, and related accruals		$	39,303		$	73,737
Income tax payable			—			4,677
Consulting and contracted research			16,395			25,007
Sales allowance			119,562			129,197
Sales rebates			109,741			76,704
Royalties			13,580			9,574
Accounts payable			40,447			45,487
Milestone payable			30,000			416
Other			13,217			16,473
Total		$	382,245		$	381,272

Inventory (Tables)

6 Months Ended

Jun. 30, 2026

Inventory

Schedule of Inventory


		June 30, 2026			December 31, 2025
Raw materials		$	10,521		$	2,291
Work in progress			57,775			55,686
Finished goods			23,058			21,670
Total inventory		$	91,354		$	79,647

Revenue recognition

6 Months Ended

Jun. 30, 2026

Revenue recognition

Revenue recognition

11. Revenue recognition

The Company views its operations and manages its business in one operating segment: life science. The life science segment is focused on the discovery, development and commercialization of the Company’s clinically differentiated medicines that provide benefits to patients with rare disorders. The Company derives its revenues through its worldwide net product sales, collaboration and license agreements, and royalty revenues.

Net product sales

During the three and six months ended June 30, 2026 and 2025, net product revenues consisted of the following:


	Three Months Ended June 30,
		2026					2025
(in thousands)		United States		International		Total		United States		International		Total
Sephience	$	127,550	$	23,760	$	151,310	$	—	$	—	$	—
Translarna		—		42,217		42,217		—		59,470		59,470
Emflaza		24,634		—		24,634		36,353		—		36,353
Upstaza/Kebilidi		—		11,163		11,163		—		11,889		11,889
All other products		—		9,495		9,495		—		10,617		10,617
Total net product revenue	$	152,184	$	86,635	$	238,819	$	36,353	$	81,976	$	118,329



	Six Months Ended June 30,
	2026						2025
		United States		International		Total		United States		International		Total
Sephience	$	239,590	$	36,271	$	275,861	$	—	$	—	$	—
Translarna		—		101,193		101,193		—		145,624		145,624
Emflaza		46,112		—		46,112		84,142		—		84,142
Upstaza/Kebilidi		2,998		17,759		20,757		—		20,547		20,547
All other products		—		20,469		20,469		—		21,442		21,442
Total net product revenue	$	288,700	$	175,692	$	464,392	$	84,142	$	187,613	$	271,755

Disaggregated net product revenues by country for the three and six months ended June 30, 2026 and 2025 are as follows:


	Three Months Ended June 30,				Six Months Ended June 30,
(in thousands)	2026		2025		2026		2025
United States	$	152,184	$	36,353	$	288,700	$	84,142
Russia		25,581		18,124		28,830		56,638
Brazil		8,210		40,951		49,422		50,456
All other countries		52,844		22,901		97,440		80,519
Total net product revenue	$	238,819	$	118,329	$	464,392	$	271,755

For three and six months ended June 30, 2026, three of the Company’s distributors each accounted for over 10% of the Company’s net product sales. For three and six months ended June 30, 2025, three and two of the Company’s distributors, respectively, each accounted for over 10% of the Company’s net product sales.

As of June 30, 2026 and December 31, 2025, the Company does not have a contract liabilities balance related to net product sales and has not made significant changes to the judgments made in applying ASC Topic 606.

Collaboration and license revenue

In November 2011, the Company and the SMA Foundation entered into a licensing and collaboration agreement with Roche. Under the terms of the SMA License Agreement, Roche acquired an exclusive worldwide license to the Company’s SMA program.

Under the agreement, the Company is eligible to receive additional payments from Roche if specified events are achieved with respect to each licensed product, including up to $135.0 million in research and development event milestones, up to $325.0 million in sales milestones upon achievement of specified sales events, and up to double digit royalties on worldwide annual net sales of a commercial product.

The SMA program currently has one approved product, Evrysdi, which was approved in August 2020 by the FDA for the treatment of SMA in adults and children two months and older. As of June 30, 2026, the Company does not have any remaining research and development event milestones that can be received. The remaining potential sales milestones that can be received is $150.0 million.

For the three and six months ended June 30, 2026 and 2025, the Company did not recognize collaboration revenue related to the SMA License Agreement with Roche.

In addition to research and development and sales milestones, the Company is eligible to recognize royalties on worldwide annual net sales of a commercial product under the SMA License Agreement. For the three and six months ended June 30, 2026, the Company has recognized $71.1 million and $117.9 million of royalty revenue related to Evrysdi, respectively. For the three and six months ended June 30, 2025 the Company has recognized $57.6 million and $94.0 million of royalty revenue, respectively, related to Evrysdi.

In November 2024, the Company entered into the Novartis Agreement with Novartis related to the Company’s votoplam HD program. The transaction contemplated by the Novartis Agreement closed in January 2025. Under the Novartis Agreement, and upon the closing of the transaction, the Company received an upfront nonrefundable payment of $1.0 billion on the effective date and can receive up to $1.9 billion in development, regulatory and sales milestones, a 40% share of U.S. profits and losses, and tiered double-digit royalties on ex-U.S. sales.

The Company evaluated the Novartis Agreement in order to determine the proper accounting treatment and concluded that the arrangement was not subject to ASC 730 or ASC 808, as the upfront payment was nonrefundable with no obligation for the Company to repay it and as the Company is not exposed to significant risks. The Company evaluated the arrangement under ASC 606, as a contract with a customer was determined to exist.

Pursuant to the Novartis Agreement, the Company determined that there were three material and distinct performance obligations: the transfer of the licenses and know-how, the completion of the Phase 2A clinical trial, and continuing the ongoing open label extension (“OLE”) clinical trial pursuant to its existing development plan, with the goal of transitioning the ongoing OLE clinical trial to Novartis within 12 months after the effective date of the Novartis Agreement. All such performance obligations have been completed. Novartis will be responsible for all other development of licensed compounds and licensed products and the manufacture and commercialization of licensed compounds and licensed products worldwide.

The Company determined that the transaction included the fixed consideration of the $1.0 billion and variable consideration in the form of milestones, profit share, and royalties. Management evaluated the variable consideration under ASC 606 and determined that it would be fully constrained until the contingencies were resolved or any applicable sales occurred. Management allocated the transaction price of $1.0 billion to the performance obligations based on the guidance in ASC 606.

During the three and six months ended June 30, 2026, the Company recognized $50.6 million and $50.7 million in collaboration and license revenues, respectively, primarily related to a development milestone pursuant to the Novartis Agreement for Novartis’s initiation of the first Phase 3 clinical trial for a Licensed Product (as defined in the Novartis Agreement), which triggered a $50.0 million milestone payment to the Company. During the three and six months ended June 30, 2025, the Company recognized $2.9 million and $992.7 million, respectively, in collaboration and license revenues related to performance obligations completed pursuant to the Novartis Agreement. Collaboration and license revenue during the six months ended June 30, 2025 was partially offset by $3.5 million related to a refund for a prior collaboration arrangement in relation to votoplam.

- Definition

The entire disclosure of revenue from contract with customer to transfer good or service and to transfer nonfinancial asset. Includes, but is not limited to, disaggregation of revenue, credit loss recognized from contract with customer, judgment and change in judgment related to contract with customer, and asset recognized from cost incurred to obtain or fulfill contract with customer. Excludes insurance and lease contracts.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 7
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479806/606-10-50-7

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 9
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479806/606-10-50-9

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479806/606-10-50-10

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
-Section 50
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Reference 5: http://www.xbrl.org/2003/role/disclosureRef
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-Section 50
-Paragraph 12
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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
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Reference 8: http://www.xbrl.org/2003/role/disclosureRef
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Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Name Accounting Standards Codification
-Topic 606
-Publisher FASB
-URI https://asc.fasb.org/606/tableOfContent

+ Details

Fair value of financial instruments and marketable securities (Tables)

6 Months Ended

Jun. 30, 2026

Fair value of financial instruments and marketable securities

Schedule of marketable securities


	Ending				Foreign								Ending
	Balance at				Currency								Balance at
	March 31,		Unrealized		Unrealized			Investments					June 30,
	2026		Gain		Gain			Purchased			Sales		2026
Marketable securities - equity investments	$	48,443		700		1,083			9,019			(27,614)	$	31,631
Total Fair Value	$	48,443	$	700	$	1,083		$	9,019		$	(27,614)	$	31,631


	Ending				Foreign								Ending
	Balance at				Currency								Balance at
	March 31,		Unrealized		Unrealized			Investments					June 30,
	2025		Gain		Gain			Purchased			Sales		2025
Marketable securities - equity investments	$	28,187		764		1,665			11,820			(8,233)	$	34,203
ClearPoint Equity Investments		10,637		44		—			—			—		10,681
Total Fair Value	$	38,824	$	808	$	1,665		$	11,820		$	(8,233)	$	44,884


	Ending				Foreign								Ending
	Balance at				Currency								Balance at
	December 31,		Unrealized		Unrealized			Investments					June 30,
	2025		Gain		Gain			Purchased			Sales		2026
Marketable securities - equity investments	$	31,596		1,914		2,644			32,976			(37,499)	$	31,631
Total Fair Value	$	31,596	$	1,914	$	2,644		$	32,976		$	(37,499)	$	31,631


	Ending				Foreign									Ending
	Balance at				Currency									Balance at
	December 31,		Unrealized		Unrealized			Investments						June 30,
	2024		Gain/(Loss)		Gain			Purchased			Sales			2025
Marketable securities - equity investments	$	29,034		1,538		3,731			17,029			(17,129)		$	34,203
ClearPoint Equity Investments		13,759		(3,078)		—			—			—			10,681
Total Fair Value	$	42,793	$	(1,540)	$	3,731		$	17,029		$	(17,129)		$	44,884

Schedule of financial assets and liabilities that are required to be measured at fair value on a recurring basis


	June 30, 2026
			Quoted prices		Significant
			in active		other		Significant
			markets for		observable		unobservable
			identical assets		inputs		inputs
	Total		(level 1)		(level 2)		(level 3)
Marketable securities - available for sale	$	1,147,564	$	—	$	1,147,564	$	—
Marketable securities - equity investments	$	31,631	$	31,631	$	—	$	—


	December 31, 2025
			Quoted prices		Significant
			in active		other		Significant
			markets for		observable		unobservable
			identical assets		inputs		inputs
	Total		(level 1)		(level 2)		(level 3)
Marketable securities - available for sale	$	929,127	$	—	$	929,127	$	—
Marketable securities - equity investments	$	31,596	$	31,596	$	—	$	—

Summary of marketable securities accounted for as available-for-sale debt securities


	June 30, 2026
	Amortized		Gross Unrealized
	Cost		Gains		Losses		Fair Value
Commercial paper	$	134,989	$	1	$	(82)	$	134,908
Corporate debt securities		510,870		2		(797)		510,075
Government obligations		503,068		5		(492)		502,581
Total	$	1,148,927	$	8	$	(1,371)	$	1,147,564


	December 31, 2025
	Amortized		Gross Unrealized
	Cost		Gains		Losses		Fair Value
Commercial paper	$	93,113	$	8	$	(9)	$	93,112
Corporate debt securities		279,090		181		(9)		279,262
Government obligations		556,137		616		—		556,753
Total	$	928,340	$	805	$	(18)	$	929,127

Summary of unrealized losses and fair values of available-for-sale debt securities in a continuous unrealized loss position


	June 30, 2026
	Securities in an unrealized loss				Securities in an unrealized loss
	position less than 12 months				position greater than or equal to 12 months				Total
	Unrealized losses		Fair Value		Unrealized losses		Fair Value		Unrealized losses		Fair Value
Commercial paper	$	(82)		120,039		—		—		(82)	$	120,039
Corporate debt securities	$	(797)		493,847		—		—		(797)	$	493,847
Government obligations	$	(492)		454,099		—		—		(492)	$	454,099
Total	$	(1,371)	$	1,067,985	$	—	$	—	$	(1,371)	$	1,067,985


	December 31, 2025
	Securities in an unrealized loss				Securities in an unrealized loss
	position less than 12 months				position greater than or equal to 12 months				Total
	Unrealized losses		Fair Value		Unrealized losses		Fair Value		Unrealized losses		Fair Value
Commercial paper	$	(9)		50,306		—		—		(9)	$	50,306
Corporate debt securities	$	(9)		45,068		—		—		(9)	$	45,068
Total	$	(18)	$	95,374	$	—	$	—	$	(18)	$	95,374

Schedule of available-for-sale debt securities


	June 30, 2026
	Less Than		More Than
	12 Months		12 Months
Commercial paper	$	134,908	$	—
Corporate debt securities		498,443		11,632
Government obligations		502,581		—
Total	$	1,135,932	$	11,632


	December 31, 2025
	Less Than		More Than
	12 Months		12 Months
Commercial paper	$	93,112	$	—
Corporate debt securities		279,262		—
Government obligations		556,753		—
Total	$	929,127	$	—

- Definition

Tabular disclosure of investment in debt security measured at fair value with change in fair value recognized in other comprehensive income (available-for-sale).

+ References

Reference 1: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 9
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-9

Reference 2: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Reference 3: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 4: http://www.xbrl.org/2009/role/commonPracticeRef
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-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 5: http://www.xbrl.org/2009/role/commonPracticeRef
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-Section 50
-Paragraph 2
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Reference 6: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 7: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 8: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 9: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 10: http://www.xbrl.org/2009/role/commonPracticeRef
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-Section 50
-Paragraph 3
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

+ Details

Consolidated Statements of Comprehensive Income (Loss) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025
Consolidated Statements of Comprehensive Income (Loss)
Net income (loss)	$ 83,496	$ (64,849)	$ 80,687	$ 801,713
Other comprehensive (loss) income:
Unrealized loss on marketable securities, net of tax	(633)	(186)	(2,150)	(274)
Foreign currency translation (loss) gain, net of tax	(1,816)	21,491	(2,334)	33,516
Comprehensive income (loss)	$ 81,047	$ (43,544)	$ 76,203	$ 834,955

- Definition

Amount after tax of increase (decrease) in equity from transactions and other events and circumstances from net income and other comprehensive income, attributable to parent entity. Excludes changes in equity resulting from investments by owners and distributions to owners.

+ References + Details

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

+ References

+ Details

Intangible assets and goodwill (Tables)

6 Months Ended

Jun. 30, 2026

Intangible assets and goodwill

Schedule of definite lived intangible assets


			Ending Balance at						Foreign		Ending Balance at
Definite-lived			December 31,						currency		June 30,
intangible assets, gross			2025			Additions			translation		2026
Waylivra			18,598			2,933			(578)		20,953
Tegsedi			24,825			3,186			(757)		27,254
Kebilidi			10,731			—			—		10,731
Upstaza			106,937			—			—		106,937
Sephience			283,500			32,190			—		315,690
Total definite-lived intangibles, gross		$	444,591		$	38,309		$	(1,335)	$	481,565


			Ending Balance at						Foreign			Ending Balance at
Definite-lived			December 31,						currency			June 30,
intangible assets, accumulated amortization			2025			Amortization			translation			2026
Waylivra			(8,689)			(1,853)			284			(10,258)
Tegsedi			(10,295)			(2,455)			337			(12,413)
Kebilidi			(1,006)			(972)			—			(1,978)
Upstaza			(27,437)			(7,950)			—			(35,387)
Sephience			(8,411)			(10,192)			—			(18,603)
Total definite-lived intangibles, accumulated amortization		$	(55,838)		$	(23,422)		$	621		$	(78,639)

Total definite-lived intangibles, net											$	402,926

Schedule of estimated future amortization of intangible assets


		As of June 30, 2026
2026		$	24,790
2027			49,581
2028			49,581
2029			43,571
2030 and thereafter			235,403
Total		$	402,926

Debt - Liability for Sale of Future Royalties (Details) $ in Thousands	6 Months Ended
	Jun. 30, 2026 USD ($)
Debt
Beginning balance	$ 2,308,366
Less: Non-cash royalty revenue payable to Royalty Pharma	(117,940)
Plus: non-cash interest expense recognized	131,008
Ending balance	$ 2,321,434
Effective interest rate of the liability component	11.00%

Intangible assets and goodwill - Goodwill (Details) - USD ($) $ in Thousands	34 Months Ended
	Jun. 30, 2026	Dec. 31, 2025	Aug. 23, 2018
Goodwill
Goodwill	$ 82,341	$ 82,341
Changes in goodwill	$ 0
Agilis
Goodwill
Goodwill			$ 82,300

- Definition

Amount, after accumulated impairment loss, of asset representing future economic benefit arising from other asset acquired in business combination or from joint venture formation or both, that is not individually identified and separately recognized.

+ References

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+ Details

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

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+ Details

Subsequent events

6 Months Ended

Jun. 30, 2026

Subsequent events

Subsequent events

14. Subsequent events

The Company has evaluated subsequent events and transactions through the filing date. There were no material events that impacted the consolidated financial statements or disclosures.