Sarepta Therapeutics, Inc.

(unaudited)

1. ORGANIZATION AND NATURE OF BUSINESS

Sarepta Therapeutics, Inc. (together with its wholly-owned subsidiaries, “Sarepta” or the “Company”) is a commercial-stage biopharmaceutical company focused on helping patients through the discovery and development of unique RNA-targeted therapeutics, small interfering RNA ("siRNA") platform, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. Applying its proprietary, differentiated and innovative technologies, and through collaborations with its strategic partners, the Company has developed multiple approved products for the treatment of Duchenne muscular dystrophy (“Duchenne”) and is developing potential therapeutic candidates for a broad range of diseases and disorders, including Duchenne, Myotonic Dystrophy type 1 (“DM1"), facioscapulohumeral muscular dystrophy (“FSHD”) and other neuromuscular and central nervous system (“CNS”) disorders.

The Company's products in the U.S., EXONDYS 51 (eteplirsen) Injection (“EXONDYS 51”), VYONDYS 53 (golodirsen) Injection (“VYONDYS 53”) and AMONDYS 45 (casimersen) Injection (“AMONDYS 45”), were granted accelerated approval by the U.S. Food and Drug Administration (the “FDA”) in 2016, 2019 and 2021, respectively. Indicated for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 51, exon 53 and exon 45 skipping, respectively, EXONDYS 51, VYONDYS 53 and AMONDYS 45 (collectively, the “PMO Products”) use the Company’s phosphorodiamidate morpholino oligomer (“PMO”) chemistry and exon-skipping technology to skip exon 51, exon 53 and exon 45 of the dystrophin gene. Exon skipping is intended to promote the production of an internally truncated but functional dystrophin protein.

ELEVIDYS (delandistrogene moxeparvovec-rokl), an adeno-associated virus-(“AAV”) based gene therapy, was approved by the FDA in June 2024 for the treatment of ambulatory patients at least four years old with Duchenne with a confirmed mutation in the Duchenne gene, as well as for non-ambulatory patients under the accelerated approval pathway. ELEVIDYS was previously granted accelerated approval by the FDA in June 2023 for the treatment of ambulatory patients aged four through five years with Duchenne with a confirmed mutation in the Duchenne gene. ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the Duchenne gene. In response to two safety events announced in March and June 2025, the Company suspended all commercial shipments of ELEVIDYS to non-ambulatory patients in June 2025. As of the date of issuance of this Quarterly Report, the Company remains in ongoing discussions with the FDA related to a labeling supplement for ELEVIDYS, including a boxed warning for acute liver injury and acute liver failure and removal of non-ambulatory population from the Indication and Usage section of the Prescribing Information.

As of September 30, 2025, the Company had $865.2 million of cash, cash equivalents, restricted cash and investments, consisting of $613.1 million of cash and cash equivalents, $239.0 million of investments and $13.1 million of non-current restricted cash. The Company believes that its balance of cash, cash equivalents and investments as of the date of the issuance of this report, along with future cash inflows from operations, is sufficient to fund its current operational plan for at least the next twelve months, though it may pursue additional cash resources through public or private debt and equity financings, seek funded research and development arrangements and additional government contracts and establish collaborations with or license its technology to other companies.

2. SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES AND RECENT ACCOUNTING PRONOUNCEMENTS

Basis of Presentation

The accompanying unaudited condensed consolidated financial statements, which have been prepared in accordance with accounting principles generally accepted in the United States (“U.S. GAAP”), reflect the accounts of Sarepta and its wholly-owned subsidiaries. All intercompany transactions between and among its consolidated subsidiaries have been eliminated. Management has determined that the Company operates inone segment: discovering, developing, manufacturing and delivering therapies to patients with rare diseases.

In the opinion of the Company’s management, all adjustments of a normal recurring nature necessary for a fair presentation have been reflected. Certain financial information that is normally included in annual financial statements prepared in accordance with U.S. GAAP, but that is not required for interim reporting purposes, has been omitted. These unaudited condensed consolidated financial statements should be read in conjunction with the consolidated financial statements and related notes for the year ended December 31, 2024, which are contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024, filed with the U.S. Securities and Exchange Commission on February 28, 2025. The results for the three and nine months ended September 30, 2025, are not necessarily indicative of the results to be expected for the full year.

Estimates and Uncertainties

The preparation of the unaudited condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, equity, revenues, expenses and the disclosure of contingent assets and liabilities. Actual results could differ from those estimates.

Concentration of Credit Risk

Financial instruments that potentially subject the Company to concentrations of credit risk consist of cash held at financial institutions, cash equivalents, investments and accounts receivable, net, from customers. As of September 30, 2025, the Company’s cash was concentrated at five financial institutions, which potentially exposes the Company to credit risks. However, the Company does not believe that there is significant risk of non-performance by the financial institutions. The Company also purchases commercial paper, government and government agency bonds, corporate bonds and certificates of deposit issued by highly rated corporations, financial institutions and governments and limits the amount of credit exposure to any one issuer. These amounts may at times exceed federally insured limits. The Company has not experienced any credit losses related to these financial instruments and does not believe to be exposed to any significant credit risk related to these instruments. As of September 30, 2025, three entities accounted for 31%,29% and 23% of accounts receivable, net, respectively. As of December 31, 2024, three entities accounted for 54%,21% and 13% of accounts receivable, net, respectively. For details about the Company’s accounts receivable, please read Note 6, Product Revenues, Net, Accounts Receivable, Net, And Reserves For Product Revenues.

Significant Accounting Policies

For details about the Company's accounting policies, please read Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncementsof the Annual Report on Form 10-K for the year ended December 31, 2024.

Acquired in-process research and development

Acquired in-process research and development (“IPR&D”) is recognized at fair value and initially characterized as an indefinite-lived intangible asset, irrespective of whether the acquired IPR&D has an alternative future use. If the acquired net assets do not constitute a business under the acquisition method of accounting, the transaction is accounted for as an asset acquisition and no goodwill is recognized. In an asset acquisition, the amount allocated to acquired IPR&D with no alternative future use is recorded as an expense at the acquisition date.

Treasury stock

Treasury stock purchases are accounted for under the cost method whereby the entire cost of the acquired stock is recorded as treasury stock. Repurchased shares are held as treasury stock until they are retired or re-issued. The Company uses the weighted average purchase cost to determine the cost of treasury stock that is reissued, if any. Excise taxes incurred on share repurchases and broker fees paid represent direct costs of share repurchases. Excise taxes are recorded as a part of the cost basis of repurchased shares within treasury stock and are paid when due.

There have not been any other material changes to the Company’s accounting policies through September 30, 2025 other than those noted below relating to the adoption of ASU 2024-04, Debt—Debt with Conversion and Other Options (Subtopic 470-20): Induced Conversions of Convertible Debt Instruments("ASU 2024-04").

Recent Accounting Pronouncements

Recently adopted

In November 2024, the Financial Accounting Standards Board ("FASB") issued Accounting Standard Update ("ASU") 2024-04, Induced Conversions of Convertible Debt Instruments, which clarifies the requirements for determining whether certain settlements of convertible debt instruments should be accounted for as an induced conversion. The amendments clarify that, to account for a settlement as an induced conversion, an inducement offer must provide at least the consideration (in form and amount) issuable under the original conversion terms, even for instruments with cash conversion features. The amendments also clarify that the guidance applies to instruments not currently convertible, provided they had a substantive conversion feature at issuance and at the time of the inducement offer. The Company early adopted ASU 2024-04, effective January 1, 2025, as permitted for entities that have previously adopted ASU 2020-06, Accounting for Convertible Instruments and Contracts in an Entity's Own Equity. The Company elected to apply the guidance prospectively to settlements occurring after the adoption date. The Company considered the application of this guidance in the accounting for the debt exchange transaction described in Note 14, Indebtedness.

Recently issued

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"). ASU 2025-06 eliminates accounting considerations of software project development stages and clarifies the threshold applied to begin capitalizing costs. ASU 2025-06 is effective for fiscal years beginning after December 15, 2027 and interim periods within those fiscal years. Early adoption is permitted.

As of September 30, 2025, the Company has not adopted ASU 2025-06 but is evaluating the impact of this guidance on its consolidated financial statements and related disclosures.

In September 2025, the FASB issued ASU 2025-07, Derivatives and Hedging (Topic 815) and Revenue from Contracts with Customers (Topic 606): Scope Refinements("ASU 2025-07"). ASU 2025-07 clarifies the application of derivative accounting to certain contracts and refines the guidance for share-based noncash consideration received from customers. Specifically, ASU 2025-07 introduces a scope exception for contracts that are not exchange-traded and whose underlying is tied to operations or activities specific to one party. It also clarifies that share-based noncash consideration from a customer should initially be accounted for under Topic 606 until the right to receive or retain such consideration becomes unconditional, at which point financial instruments guidance may apply. ASU 2025-07 is effective for fiscal years beginning after December 15, 2027 and interim periods within those fiscal years. Early adoption is permitted. As of September 30, 2025, the Company has not adopted ASU 2025-07 but is evaluating the impact of this guidance on its consolidated financial statements and related disclosures.

3. LICENSE AND COLLABORATION AGREEMENTS

License Agreement with Arrowhead Pharmaceuticals, Inc.

On November 25, 2024, the Company and Arrowhead Pharmaceuticals, Inc. (“Arrowhead”) entered into an exclusive global license and collaboration agreement and a stock purchase agreement (collectively, the “Arrowhead Agreement”), which became effective as of February 7, 2025 (the “Effective Date”), upon the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976.

The Arrowhead Agreement granted the Company an exclusive license under certain of Arrowhead’s intellectual property rights to develop, manufacture and commercialize the lead candidate (and all backup candidates) for the following programs:

In addition, the parties will collaborate on the discovery and development of compounds that are directed to six targets to be selected by the Company during the term (each an “Arrowhead Discovery Program,” and together with the Arrowhead Clinical Programs and Pre-clinical Programs, the “Arrowhead Programs”).

The development and manufacturing activities under the Arrowhead Agreement are governed by a series of committees established to facilitate transition and collaboration between the two parties.

Under the terms of the Arrowhead Agreement, Arrowhead will conduct development activities with respect to the Arrowhead Programs pursuant to agreed-upon development plans. At pre-determined transition points for each Arrowhead Program, Arrowhead will transfer development responsibility to the Company, who will then perform all development activities necessary to obtain and maintain regulatory approvals throughout the world. The Company will have the sole worldwide right to commercialize licensed products.

The Company will reimburse Arrowhead for certain pre-determined development activities for the Arrowhead Clinical Programs. Each party is responsible for the costs and expenses of other development activities under the Arrowhead Agreement. Arrowhead will complete all manufacturing activities necessary for their development activities and provide clinical supply for all Arrowhead Programs and commercial supply for the Arrowhead Clinical Programs. The parties will determine at a later date whether Arrowhead will provide commercial supply for the Arrowhead Pre-clinical Programs and Arrowhead Discovery Programs. Upon the occurrence of certain conditions, Arrowhead will transfer control of manufacturing and supply to the Company.

In connection with the Arrowhead Agreement, Arrowhead appointed the Company’s Chief Executive Officer (“CEO”), Douglas Ingram, as a director, effective February 2025.

When the Arrowhead Agreement became effective, the Company paid Arrowhead an up-front payment of $500.0 million and invested $325.0 million in approximately 11.9 million shares of Arrowhead's common stock at a premium to the valuation on the closing date. Based on the closing price of Arrowhead’s common stock traded on the Nasdaq Global Select Market (“Nasdaq”) on the Effective Date, $241.4 million was allocated to the equity investment in Arrowhead and recorded within strategic investments on the Company’s unaudited condensed consolidated balance sheets. For thenine months ended September 30, 2025, the Company recorded $583.6 million as acquired IPR&D expense, as the remainder of the up-front payments is allocated to the up-front license fee representing rights to potential future benefits associated with research and development activities that have no alternative future use. The Company concluded that it does not have a controlling financial interest in Arrowhead as the Company does not have the power to direct the activities that would most significantly impact the economic performance of Arrowhead. Additionally, the Company concluded that, as it does not have significant influence over Arrowhead, the Company’s investment in Arrowhead is not subject to the equity method of accounting. Accordingly, the investment in Arrowhead’s common stock is recorded at fair value and remeasured each reporting period, with changes recorded to other income (expense), net in the Company’s unaudited condensed consolidated statements of comprehensive (loss) income. The Arrowhead Agreement also provided the Company with the option to exchange its holding of Arrowhead’s common stock into pre-funded warrants (“Warrants”) with an exercise price of $0.001 per Warrant share.

Additionally, the Company will pay Arrowhead an aggregate total of $250.0 million in annual installments of $50.0 million over five years (“Annual Fees”), with the first payment due on the first anniversary of the Effective Date. The Annual Fees are contingent upon the Company not terminating the Arrowhead Agreement. Over the term of the Arrowhead Agreement, the Company may be obligated to make payments to Arrowhead totaling up to $10.3 billion upon the achievement of certain development, regulatory and sales milestones, inclusive of two near-term payments associated with the continued enrollment of certain cohorts of a Phase 1/2 study for the DM1 program (the "Arrowhead DM1 Milestones"), totaling $300.0 million, one of which was achieved as of September 30, 2025. Furthermore, upon commercialization, the Company will be required to make tiered royalty payments based on net sales.

On August 13, 2025, Arrowhead achieved the first of two Arrowhead DM1 Milestones. Consequently, the Company entered into an agreement with Arrowhead to transfer approximately 2.7 million shares of Arrowhead common stock, valued at approximately $50.0 million. The remaining $50.0 million was paid by the Company in cash. As a result, the Company recognized milestone expenses of $100.0 million associated with the achievement of the first Arrowhead DM1 Milestone as research and development expense in the accompanying unaudited condensed consolidated statements of comprehensive (loss) income for the three and nine months ended September 30, 2025.

In August 2025, the Company's contractual sale restriction associated with its Arrowhead common stock expired and the Company sold the remainder of its Arrowhead common stock, representing approximately 9.3 million shares, resulting in gross proceeds of $174.1 million. For the three and nine months ended September 30, 2025, the Company recognized a gain of $35.7 million and loss of $17.3 million, reflecting both the change in fair value and realized loss on the sale of this strategic investment. For the three and nine months ended September 30, 2025, the Company recorded $7.0 million and $15.8 million, respectively, of research and development expense related to reimbursable development costs incurred by Arrowhead.

Collaboration Agreement with F. Hoffman-La Roche Ltd.

Under the license, collaboration and option agreement (the “Roche Collaboration Agreement”) with F. Hoffman-La Roche Ltd. (“Roche”), the Company has granted Roche options to acquire ex-U.S. rights to certain future Duchenne development programs (the “Options”) in exchange for separate option exercise payments, milestone and royalty considerations and cost-sharing provisions. These Options were accounted for as material rights related to individual programs and recorded in deferred revenue at the inception of the Roche Collaboration Agreement. The value assigned to the material rights is included in deferred revenue until such rights expire or are exercised. For the nine months ended September 30, 2025, the Company recognized $175.5 million in collaboration revenue associated with the Roche Collaboration Agreement.

In June 2025, the Company received a milestone payment of $63.5 million in connection with the receipt of regulatory approval of ELEVIDYS in Japan for individuals ages 3- to less than 8-years-old, who do not have any deletions in exon 8 and/or exon 9 in the Duchenne gene and who are negative for anti-AAVrh74 antibodies. The milestone payment is included in collaboration and other revenues for the nine months ended September 30, 2025. As a result of the milestone received from Roche, the Company recorded a $5.1 million milestone due to Nationwide Children's Hospital (“Nationwide”) in accordance with the Company's license agreement with Nationwide as an in-licensed right intangible asset in its unaudited condensed consolidated balance sheets as of September 30, 2025. As of September 30, 2025, the in-licensed right asset with a net carrying value of $4.9 million is being amortized on a straight-line basis over the remaining life of the relevant patent as it reflects the expected time period that the Company will benefit from this in-licensed right.

In February 2025, an Option for a certain program expired, which resulted in the immediate recognition of $112.0 million of collaboration revenue for thenine months ended September 30, 2025. In February 2024, Roche declinedto exercise its option related toone external, early-stage development program, which resulted in the immediate recognition of $48.0 million of collaboration revenue for thenine months ended September 30, 2024. As of September 30, 2025 and December 31, 2024, the Company had total deferred revenue of $485.4 million and $455.3 million, primarily related to the separate material rights for the Options associated with the Roche Collaboration Agreement, of which $485.4 million and $130.3 million are classified as current, respectively.

In accordance with the Roche Collaboration Agreement, the parties agreed to enter into a supply agreement in order to supply Roche with clinical and commercial batches of ELEVIDYS (the “Roche Supply Agreement”). Roche utilizes the supply for sales of ELEVIDYS in territories outside of the U.S. where Roche has received certain approvals for ELEVIDYS. While the Roche Supply Agreement is in the process of being negotiated at the issuance of this report, the Company delivered commercial ELEVIDYS supply to Roche that were agreed upon on a purchase order-by-purchase order basis. Contract manufacturing revenue and royalty revenue are included in collaboration and other revenues in the unaudited condensed consolidated statementsof comprehensive (loss) income. The following table summarizes certain Roche activity for each of the periods indicated:

The costs associated with co-development activities performed under the Roche Collaboration Agreement are included in operating expenses, with any reimbursement of costs by Roche reflected as a reduction of such expenses when the related expense is incurred. For the three and nine months ended September 30, 2025, costs reimbursable by Roche and reflected as a reduction to operating expenses were $12.2 million and $66.4 million, respectively. For the three and nine months ended September 30, 2024, costs reimbursable by Roche and reflected as a reduction to operating expenses were $61.5 million and $101.1 million, respectively. As of September 30, 2025 and December 31, 2024, there were $128.7 million and $34.6 million, respectively, of collaboration and other receivables included in other current assets on the unaudited condensed consolidated balance sheets.

Milestone Obligations

The Company has license and collaboration agreements in place for which it could be obligated to pay, in addition to the payment of up-front fees upon execution of the agreements, certain milestone payments as a product candidate proceeds from the submission of an investigational new drug application through approval for commercial sale and beyond. As of September 30, 2025, the Company may be obligated to make up to $12.5 billion of future development, regulatory, commercial and up-front royalty payments associated with its collaboration and license agreements. These obligations exclude potential future option and milestone payments for options that have yet to be exercised within agreements entered into by the Company as of September 30, 2025. For the three and nine months ended September 30, 2025, the Company recognized up-front and development milestone expenses of $100.0 million and $683.8 million, respectively, as research and development expense in the accompanying unaudited condensed consolidated statements of comprehensive (loss) income, withno similar activity for thethree and nine months ended September 30, 2024.

4. FAIR VALUE MEASUREMENTS

The Company has certain financial assets and liabilities that are recorded at fair value which have been classified as Level 1, 2 or 3 within the fair value hierarchy as described in the accounting standards for fair value measurements.

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Level 1 — quoted prices for identical instruments in active markets;

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Level 2 — quoted prices for similar instruments in active markets, quoted prices for identical or similar instruments in markets that are not active, and model-derived valuations in which all significant inputs and significant value drivers are observable in active markets; and

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Level 3 — valuations derived from valuation techniques in which one or more significant value drivers are unobservable.

During the three and nine months ended September 30, 2025, there were no transfers into or out of Level 3. The tables below present information about the Company’s financial assets and liabilities that are measured and carried at fair value and indicate the level within the fair value hierarchy of the valuation techniques it utilizes to determine such fair value:

The Company’s assets with a fair value categorized as Level 1 within the fair value hierarchy include money market funds, the Company's strategic investment in a biotechnology company listed on Nasdaq and assets associated with the Company's deferred compensation plan that are held in a trust. The Company's deferred compensation plan allows for certain employees and directors to defer the receipt of compensation until a later date based on the terms of the plan. The Company has recorded an asset within other non-current assets on the Company's unaudited condensed consolidated balance sheets which reflects the participants' investment elections and has been valued at daily quoted market prices.

The Company's assets with a fair value categorized as Level 2 within the fair value hierarchy consist of commercial paper, government and government agency bonds, corporate bonds and certificates of deposit. These assets have been initially valued at the transaction price and subsequently valued at the end of each reporting period. The Company uses observable market inputs to determine value, which primarily consist of reportable trades. Certain highly liquid investments with maturities of less than three months at the date of acquisition are presented as cash equivalents on the unaudited condensed consolidated balance sheets as of September 30, 2025 and December 31, 2024.

The Company’s assets with a fair value categorized as Level 3 within the fair value hierarchy consist of strategic investments in private biotechnology companies whose fair value measurement was based upon significant inputs not observable in the market and therefore represented a Level 3 measurement. At the end of each reporting period, the fair value of the Company's strategic investments that are not listed securities are adjusted if the entities were to issue similar or identical securities or when there is a triggering event for impairment. There were no valuation measurement events related to the fair value of the Company's Level 3 strategic investments during the nine months ended September 30, 2025 or 2024, as no impairment indicators were identified nor were similar securities issued.

The Company’s contingent consideration liability with a fair value categorized as Level 3 within the fair value hierarchy relates to the regulatory-related contingent payments to Myonexus Therapeutics, Inc. (“Myonexus”) selling shareholders as well as to an academic institution under a license agreement that meets the definition of a derivative. For more information related to Myonexus, please read Note 3, License and Collaboration Agreements to the financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024. The contingent consideration liability was estimated using an income approach based on the probability-weighted expected cash flows that incorporated industry-based probability adjusted assumptions relating to the achievement of the milestone and thus the likelihood of making the payments. This fair value measurement was based upon significant inputs not observable in the market and therefore represented a Level 3 measurement. Significant changes which increase or decrease the probabilities of achieving the milestone, or shortening or lengthening the time required to achieve the milestone, would result in a corresponding increase or decrease in the fair value of the liability. At the end of each reporting period, the fair value is adjusted to reflect the most current assumptions through earnings.

The following tables represent a roll-forward of the fair value of Level 3 financial liabilities for each of the periods indicated:

In July 2025, the Company announced a strategic restructuring plan (the “Restructuring Plan”). For more information related to the Restructuring Plan, please read Note 17, Restructuring. As a result of the Restructuring Plan, the development of all LGMD programs with the exception of SRP-9003 (LGMD2E/R4/bidridistrogene xeboparvovec) was suspended. Accordingly, the Company terminated $25.6 million of contingent consideration liabilities associated with all LGMD Programs with the exception of SRP-9003 for thethree and nine months ended September 30, 2025. This decrease was partially offset by a $14.5 million increase in the fair value of the Company's existing contingent consideration liability associated with SRP-9003 for thethree and nine months ended September 30, 2025. The increase to the contingent consideration liability associated with SRP-9003 was a result of updates made to certain inputs and assumptions impacting the probability-weighted expected cash flows. These updates include changes to the expected market price used to estimate future payments, adjustments to the discount rate utilized to determine the present value of the resulting liability and changes to the approval date of SRP-9003. As of September 30, 2025, the remaining contingent consideration was recorded as a current liability on the Company's unaudited condensed consolidated balance sheets as the Company expects that the associated obligation will be settled within the next twelve months.

For the three and nine months ended September 30, 2024, the Company recorded a net decrease of $0.8 million and a net increase of $9.3 million, respectively, to account for the change in fair value of the Company's existing contingent consideration liabilities and any liabilities terminated.

The carrying amounts reported in the unaudited condensed consolidated balance sheets for cash and cash equivalents, accounts receivable, net and accounts payable approximated fair value because of the immediate or short-term maturity of these financial instruments.

5. CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES

The following table summarizes the Company’s financial assets with maturities of less than 90 days from the date of purchase included in cash equivalents in the unaudited condensed consolidated balance sheets for each of the periods indicated:

It is the Company’s policy to mitigate credit risk in its financial assets by maintaining a well-diversified portfolio that limits the amount of exposure as to maturity and investment type. The weighted average maturity of the Company's available-for-sale securities as of September 30, 2025 and December 31, 2024 was approximately five and eleven months, respectively. All of the Company's non-current investments as of September 30, 2025 and December 31, 2024 had maturities between one andtwo years.

The following tables summarize the Company’s cash, cash equivalents, short-term investments and non-current investments for each of the periods indicated:

6. PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE, NET, AND RESERVES FOR PRODUCT REVENUES

Net product revenues, which includes revenues associated with the PMO Products and ELEVIDYS, consisted of the following:

For the three and nine months ended September 30, 2025 and 2024, no individual country outside the U.S. exceeded 10% of total net product revenues.

The following table summarizes the Company's net product revenues, by customer, for those customers that exceeded 10% for the periods indicated:

As of September 30, 2025 and December 31, 2024, the Company's accounts receivable, net were $395.7 million and $602.0 million, respectively, both of which were related to product sales, net of discounts and allowances. As of September 30, 2025, the majority of the Company’s accounts receivable arose from product sales in the U.S. and all customers have standard payment terms that generally require payment within 65 to 90 days. Outside of the U.S., the majority of the Company’s customers have payment terms ranging between 90 and 150 days.

The following tables summarize an analysis of the change in reserves for discounts and allowances for each of the periods indicated:

The following table summarizes the total reserves above included in the Company’s unaudited condensed consolidated balance sheets for each of the periods indicated:

7. INVENTORY

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

No material inventory reserves existed as of September 30, 2025 or December 31, 2024. The Company classifies inventory associated with its PMO Products as non-current inventory when consumption of the inventory is expected beyond the Company's normal PMO Product inventory operating cycle of two years. Non-current inventory consists of raw materials and work in progress associated with the PMO Products.

The following table summarizes the balance sheet classification of the Company's inventory for each of the periods indicated:

8. OTHER ASSETS

The following table summarizes the Company’s other current assets for each of the periods indicated:

The following table summarizes the Company’s other non-current assets for each of the periods indicated:

• Restricted cash for both periods relates to (i) letters of credit established under the Company’s various property leases that serve as security for potential future default of lease payments, (ii) a letter of credit established under a certain commercial supply agreement and (iii) collateralized cash for the Company’s credit cards. The restricted cash is unavailable for withdrawal or use for general obligations.

9. ACCRUED EXPENSES

The following table summarizes the Company’s accrued expenses for each of the periods indicated:

10. EQUITY

In August 2025, the Company entered into a privately negotiated subscription agreement (the “Subscription Agreement”) with J. Wood Capital Advisors LLC ("JWCA"), the Company’s financial advisors in connection with the debt exchange transaction defined in Note 14, Indebtedness, for the private placement of approximately 1.1 million shares of the Company's common stock in exchange for $20.0 million in cash. See Note 14, Indebtednessfor additional information about the transactions.

In November 2024, the Board of Directors approved a share repurchase program (the “2024 Repurchase Program”) of up to $500.0 million of the Company’s outstanding common stock over the next 18 months. Correspondingly, the Company entered into a Rule 10b-18 repurchase plan that allows it to conduct open market repurchases periodically up to the remaining authorization under the 2024 Repurchase Program.

During the nine months ended September 30, 2025, the Company purchased a total of 650,876 shares of common stock under the 2024 Repurchase Program at an average price of $38.41 per share for a total cost of $25.3 million, inclusive of approximately $0.3 million in excise taxes and other fees. As of September 30, 2025, the remaining amount authorized under the 2024 Repurchase Program was $475.0 million.

Repurchased shares are held as treasury stock. The amount paid to repurchase shares is recorded as a reduction to stockholders’ equity. As treasury stock is not considered outstanding, these shares are excluded from weighted average common stock outstanding for both basic and diluted earnings per share.

11. STOCK-BASED COMPENSATION

The following table summarizes the Company’s stock awards granted for each of the periods indicated:

(1) Approximately 3.4 million restricted stock units (“RSUs”) granted in thethree and nine months ended September 30, 2025 vest over two years while the remaining vest over four years.

(2) The performance condition for performance stock units (“PSUs”) granted in September 2025 (the "September 2025 PSUs") is related to the achievement of a certain financial performance goal. The September 2025 PSUs have a three-year cliff vesting schedule. As of September 30, 2025, the performance condition associated with the September 2025 PSUs was not probable of being achieved.

(3) In addition to the September 2025 PSUs, included in PSUs for the nine months ended September 30, 2025 are 120,378 shares with performance conditions which are related to the achievement of certain financial performance goals, regulatory development and approval of certain of the Company's product candidates and certain manufacturing achievements (the “March 2025 PSUs”). As of September 30, 2025, none of the performance conditions for the March 2025 PSUs were probable of being achieved.

(4)Performance conditions for the 97,460 shares PSUs granted in March 2024 (the “March 2024 PSUs”) are related to the achievement of certain financial performance goals and regulatory approval of certain of the Company's product candidates. The expanded regulatory approval of ELEVIDYS in June 2024 resulted in the cliff-vesting of 44,300 shares during the year ended December 31, 2024. The achievement of certain financial performance targets during thenine months ended September 30, 2025 resulted in 38,050 shares of the March 2024 PSUs becoming eligible for vesting, which is contingent on the fulfillment of remaining service conditions. As of September 30, 2025, none of the remaining performance conditions associated with the March 2024 PSUs were probable of being achieved.

Stock-based Compensation Expense

For the three months ended September 30, 2025 and 2024, total stock-based compensation expense was $14.9 million and $43.5 million, respectively. For thenine months ended September 30, 2025 and 2024, total stock-based compensation expense was $93.4 million and $134.6 million, respectively.

The following table summarizes stock-based compensation expense by grant type and by function included within theunaudited condensed consolidated statements of comprehensive (loss) income:

In August 2025, the Company suspended its 2013 Employee Stock Purchase Plan (the “2013 ESPP”) as a result of increased volatility in the Company's stock price (the "ESPP Suspension"). As a result of the ESPP Suspension, previously recognized stock-based compensation expense related to shares that were not purchased was reversed, resulting in a reversal of expense of $4.0 million for the three and nine months ended September 30, 2025. As of September 30, 2025,0.3 million shares of common stock remain available for future grant under the 2013 ESPP.

12. OTHER (LOSS) INCOME, NET

The following table summarizes other (loss) income, net for each of the periods indicated:

*Related to the change in fair value of the contingent consideration derivative liabilities. For more information, please read Note 4, Fair Value Measurements.

13. INCOME TAXES

During the three and nine months ended September 30, 2025, the Company recorded an income tax (benefit) expense of $(14.1) million and $6.6 million, representing an effective tax rate of 7.3% and(1.6)%, respectively. During the three and nine months ended September 30, 2024, the Company recorded an income tax expense of $0.4 million and $12.8 million, representing an effective tax rate of 1.2% and 14.4%, respectively. The income tax expense, for the nine months ended September 30, 2025 and 2024 and the three months ended September 30, 2024, primarily relates to the current tax provision on taxable profits, including in certain states which restrict the amount of net operating loss carryforwards which may be utilized to offset taxable income and, prior to the nine months ended September 30, 2025, the requirement to capitalize research and development costs for tax purposes. The income tax (benefit), for the three months ended September 30, 2025, primarily relates to the tax (benefit) recorded on the quarter to date loss which offsets tax expense recorded on profits in prior interim periods.

On a periodic basis, the Company reassesses the valuation allowance on its deferred tax assets, weighing positive and negative evidence to assess the recoverability of such deferred tax assets. In assessing the Company’s ability to realize its net deferred tax assets, the Company considered various factors, including future reversals of existing taxable temporary differences, projected future taxable income, potential carryback claims and tax planning strategies to determine whether it is more likely than not that some portion or all of its net deferred tax assets will not be realized. Based upon these factors, the Company has determined that the uncertainty regarding the realization of these assets is sufficient to warrant the need to maintain a full valuation allowance against its net deferred tax assets as of September 30, 2025. It is possible the Company may release a portion or all of its valuation allowance in future periods. The release of the valuation allowance, as well as the exact timing and the amount of such release, continue to be subject to, among other things, the Company’s level of profitability, revenue growth and expectations regarding future profitability. Release of the valuation allowance would result in a benefit to income tax expense for the period the release is recorded, which could have a material impact on net earnings. The total deferred tax asset balance subject to the valuation allowance was approximately $1,100.5 million as of September 30, 2025. The Company will continue to assess the realizability of its deferred tax assets on a quarterly basis.

On July 4, 2025, the One Big Beautiful Bill Act (“OBBBA”) was enacted in the U.S. The OBBBA contains, among other provisions, changes to the U.S. corporate income tax system, including allowing immediate expensing of U.S. qualifying research and development expenses and allowing taxpayers an election to accelerate the deduction for previously capitalized U.S. research and development costs. The favorable U.S. research and development expenditure provisions are expected to reduce taxable income but are not expected to have a material impact on the Company’s deferred tax expense as a result of the valuation allowance maintained against the Company’s net deferred tax assets.

14. INDEBTEDNESS

2027 Convertible Notes, 2030 Convertible Notes and Related Transactions

Exchange of 2027 Notes

In September 2022, the Company issued $1,150.0 million aggregate principal amount of convertible senior notes due on September 15, 2027 (the “2027 Notes”). The 2027 Notes are senior unsecured obligations of the Company and bear interest at a rate of 1.25% per annum, payable semi-annually in cash on each March 15 and September 15, commencing on March 15, 2023. The Company received net proceeds of $1,126.7 million after deducting the discounts and offering expenses of $23.3 million, which is amortized under the effective interest method and recorded as additional interest expense over the life of the 2027 Notes. The effective interest rate on the 2027 Notes is 1.67%. The 2027 Notes have a conversion rate of 7.0439 shares per $1,000 principal amount of the 2027 Notes, or a conversion price of $141.97 per share. Upon conversion, at its discretion, the Company may pay cash, shares of its common stock or a combination of cash and stock. Prior to March 15, 2027, the holders of the 2027 Notes may convert their 2027 Notes at their option upon achievement of certain market conditions or occurrence of certain corporate events. In connection with the issuance of the 2027 Notes, the Company paid $127.3 million for capped calls with a strike price and a cap price of $141.97 and $210.32, respectively, to minimize the impact of potential dilution upon conversion of the 2027 Notes.

On August 28, 2025 (the “Closing Date”), the Company completed a partial refinancing of its 2027 Notes (the “Exchange Transaction”). The Company exchanged $700.0 million in aggregate principal amount of the 2027 Notes for the following consideration:

(1)

$602.0 million in aggregate principal amount of new 4.875% convertible senior notes due 2030 (the “2030 Notes”);

(2)

cash payments of $127.3 million, including $4.0 million of accrued interest; and

(3)

5.9 million shares of the Company’s common stock with fair market value of approximately $107.3 million, net of issuance costs of $2.4 million.

The remaining aggregate principal amount of $450.0 million of the 2027 Notes, which are currently convertible into 3,169,755 shares of the Company’s common stock, remains outstanding with the respective terms unchanged. Additionally, all capped calls related to the 2027 Notes remain outstanding. Refer to Note 13, Indebtednessof the Company’s Annual Report on Form 10-K for the year ended December 31, 2024 for a description of the key terms associated with the 2027 Notes. The Exchange Transaction did not result in any material changes to the related party debt arrangements disclosed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024.

2030 Notes Issuance

The 2030 Notes are senior unsecured obligations of the Company and bear interest at a rate of 4.875% per annum, payable semi-annually in cash on each March 1 and September 1, commencing on March 1, 2026. The initial carrying value of the 2030 Notes was $588.6 million after deducting offering expenses of $13.4 million, which are amortized under the effective interest method and recorded as additional interest expense over the life of the 2030 Notes. The effective interest rate on the 2030 Notes is 5.38%.

The 2030 Notes have an initial conversion rate of 16.6667 shares per $1,000 principal amount of the 2030 Notes (10,033,386 shares of the Company’s common stock in the aggregate), which represents a conversion price of $60.00 per share, subject to adjustment under certain conditions. Upon conversion, the Company may pay cash, shares of its common stock or a combination of cash and stock, as determined by the Company at its discretion.

The holders of the 2030 Notes may convert their 2030 Notes at their option only in the following circumstances:

(1)

during any calendar quarter commencing after the calendar quarter ending on December 31, 2025, if the last reported sale price per share of common stock exceeds 130% of the conversion price for each of at least 20 trading days during the 30 consecutive trading days ending on, and including, the last trading day of the immediately preceding calendar quarter;

(2)

during the five consecutive business days immediately after any five consecutive trading day period (the “Measurement Period”) in which the trading price per $1,000 principal amount of 2030 Notes for each trading day of the Measurement Period was less than 98% of the product of the last reported sale price per share of our common stock on such trading day and the conversion rate on such trading day;

(3)

upon the occurrence of certain corporate events or distributions on the Company's common stock, as described in the indenture agreement;

(4)

if the Company calls such notes for redemption; and

(5)

at any time from, and including, March 1, 2030 until the close of business on the second trading day immediately before the maturity date.

Prior to September 6, 2028, the 2030 Notes will not be redeemable. On or after September 6, 2028, should certain market conditions be met, the Company may redeem for cash all or any portion of the 2030 Notes at a redemption price equal to the principal amount of the 2030 Notes, plus accrued and unpaid interest. Holders of the 2030 Notes have the right to require the Company to repurchase for cash all or a portion of their notes at 100% of its respective principal amount, plus any accrued and unpaid interest, upon the occurrence of a fundamental change as defined in the indenture agreement for the 2030 Notes. The 2030 Notes contain customary covenants and events of default, the occurrence of which permits the holders to accelerate all outstanding obligations, including principal and interest. As of September 30, 2025, the Company was in compliance with the covenants associated with the 2027 Notes and 2030 Notes.

The Company adopted ASU 2024-04, Debt with Conversion and Other Options, Induced Conversion of Convertible Debt Instruments, and concluded that the Exchange Transaction does not meet the criteria for induced conversion. Further, the Company also concluded that the refinanced debt is substantially different from the original debt and, thus, the transaction was accounted for as a debt extinguishment. Accordingly, on the Closing Date, the Company:

(1)

recorded the carrying value of the 2030 Notes of $588.6 million, net of allocated issuance of $13.4 million;

(2)

recorded the fair value of the issuance of approximately 5.9 million common stock of $104.9 million, net of allocated issuance costs of $2.4 million;

(3)

recorded cash payments of $127.3 million, including payments of accrued interest of $4.0 million;

(4)

reduced the carrying value of the 2027 Notes by $694.0 million; and

(5)

recognized a loss on debt extinguishment of $138.6 million, which included unamortized debt issuance costs of $6.0 million associated with the 2027 Notes.

Revolving Credit Facility

On February 13, 2025 (the “Closing Date”), the Company entered into a credit agreement (the “Credit Agreement”) with JPMorgan Chase Bank, N.A., as administrative agent (the “Administrative Agent”) and as collateral agent and the lenders party thereto. The Credit Agreement provides for a five-year, $600.0 million senior secured revolving credit facility (the “Revolving Credit Facility”). The Company's obligations under the Credit Agreement are secured by substantially all of the Company's assets and the assets ofcertain wholly-owned material subsidiaries, subject to certain customary exceptions and exclusions.

Interest rates under the Revolving Credit Facility are variable and equal to the Secured Overnight Financing Rate plus a credit spread adjustment of 0.10% per annum (“Adjusted SOFR”), plus a margin of 1.125% to 1.75% per annum, or, at the Company’s option, at a base reference rate equal to the highest of (a) the federal funds rate plus 0.50%, (b) the rate of interest last quoted by the Administrative Agent as its “base rate” and (c) the one-month Adjusted SOFR rate plus 1.00%, plus a margin of 0.125% to 0.75% per annum. The Company also will pay an unused commitment fee ranging from 0.20% to 0.35% per annum on the unused commitments.

The Credit Agreement contains customary representations and warranties, affirmative covenants, negative covenants, conditions and events of default. The Credit Agreement also contains financial covenants that are assessed on the last day of each of the Company’s fiscal quarters, including certain financial ratios such as a maximum secured net leverage ratio and minimum consolidated interest coverage ratio. The Company may voluntarily prepay the outstanding revolving loans under the Revolving Credit Facility in whole or in part without premium or penalty provided that the prepayment shall be in certain amounts as specified therein.

The Company paid $3.2 million in arrangement and up-front fees on the Closing Date. The arrangement and up-front fees associated with the Revolving Credit Facility are being amortized over thefive-year term of the Revolving Credit Facility. As of September 30, 2025, there were no amounts outstanding under the Revolving Credit Facility and the Company was in compliance with the covenants described above.

Total Debt Obligations

As of September 30, 2025 and December 31, 2024, the Company recorded approximately $1,035.1 million and $1,137.1 million as long-term debt on the unaudited condensed consolidated balance sheets, respectively.

The following table summarizes the Company’s debt instruments for the periods indicated:

For the three months ended September 30, 2025 and 2024, interest expense from debt facilities was $6.6 million and $5.2 million, inclusive of $1.1 million and $1.2 million of amortization of debt discounts, respectively. For the nine months ended September 30, 2025 and 2024, interest expense from debt facilities was $16.2 million and $15.7 million, inclusive of $3.5 million and $3.7 million of amortization of debt discounts, respectively. The fair value of the 2027 Notes and 2030 Notes is based on open market trades and is classified as Level 1 in the fair value hierarchy.

The following table summarizes the total principal and contractual interest payments due under the Company’s debt arrangements:

15. (LOSS) EARNINGSPER SHARE

Basic (loss) earnings per share is computed by dividing net (loss) income by the weighted-average number of shares of common stock outstanding. Diluted earnings per share is computed based on the treasury stock method for stock awards and the if-converted method for convertible debt by dividing net income by the weighted-average number of shares of common stock and dilutive common stock equivalents outstanding. Given that the Company recorded a net loss for the three and nine months ended September 30, 2025, there is no difference between basic and diluted net loss per share since the effect of common stock equivalents would be anti-dilutive and are, therefore, excluded from the diluted net loss per share calculation.

The following table sets forth the computation of basic and diluted (loss) earnings per common share:

The following table summarizes potential shares of common stock that were excluded from the computation of diluted earnings per share as they were anti-dilutive:

(1) As of September 30, 2025, the anti-dilutive common stock issuable under the Company's equity incentive plans includes 0.3 million shares that have performance conditions that were not met as of the end of the period.

(2) As of September 30, 2024, the anti-dilutive common stock issuable under the Company's equity incentive plans excludes 1.2 million shares that are dilutive but have performance or market conditions that were not met as of the end of the period.

16. SEGMENT INFORMATION

The measure of segment profit or loss that the CEO uses to allocate resources and assess performance as the chief operating decision-maker (“CODM”) is the Company’s consolidated net (loss) income. The table below includes information about the Company’s segment, including significant segment expenses, and a reconciliation to net (loss) income:

(a)Research and development-other includes professional services, pre-clinical expenses and research and other expenses.

(b)Selling, general and administrative-other includes professional services and other expenses.

(c)Other segment items included in segment net income (loss) include accretion of investment discount, net, change in fair value of derivatives and other, net, as well as the items separately presented and not defined as significant expenses below.

Significant expense categories that are regularly provided to the CODM include cost of sales (excluding amortization of in-licensed rights), compensation and other personnel expenses, up-front and milestone expenses, manufacturing expenses, clinical trial expenses, facility- and technology-related expenses, restructuring charge, research and development- other and selling, general and administrative- other. The other expense or income information are other segment items and include separate presentation of loss on debt extinguishment, (gain) loss on strategic investments, interest expense, interest income, income tax (benefit) expense, depreciation and amortization and stock-based compensation, which are included in the measure of segment net (loss) income, but are not significant segment expenses.

Assets provided to the CODM for the single segment are consistent with those reported on the unaudited condensed consolidated balance sheets.

17. RESTRUCTURING

On July 16, 2025, the Company announced the Restructuring Plan, designed to reduce operating expenses and align its cost structure with strategic priorities, aiming to enhance financial flexibility and meet its 2027 financial obligations. The Restructuring Plan included the following initiatives:

•

Prioritize siRNA platform assets while reducing other research and development program costs; and

•

Reduce the workforce by 36%, or approximately 500 employees.

For the three and nine months ended September 30, 2025, the Company incurred restructuring charges of approximately $40.5 million, the vast majority of which are related to severance and related costs from the Company’s one-time termination benefits. As a result of discontinuing certain research and development programs, the Company also accelerated depreciation for impacted assets. The costs incurred under the Restructuring Plan are not expected to exceed $40.5 million and are anticipated to be substantially incurred and paid by the end of 2025. As of September 30, 2025, a $2.5 million liability is recorded for the cash charges incurred that are unpaid, which is included in accrued expenses on the Company's unaudited condensed consolidated balance sheets.

The following table summarizes restructuring charges for the periods presented:

The following table summarizes the restructuring reserve as of September 30, 2025:

As of September 30, 2025, the remaining restructuring reserve relates to severance obligations and related costs.

18. COMMITMENTS AND CONTINGENCIES

Manufacturing Obligations

The following table summarizes the aggregate non-cancelable contractual obligations arising from the Company’s manufacturing obligations:

*Total manufacturing commitments include the Catalent, Inc. (“Catalent”) manufacturing and supply agreement, for which the Company has ROU assets and lease liabilities recorded on the unaudited condensed consolidated balance sheets as of September 30, 2025. For more information, please read Note 22, Commitments and Contingencies to the financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024.

**Total manufacturing commitments include obligations related to the global supply of ELEVIDYS, including inventories necessary to supply Roche for sales of ELEVIDYS in territories outside of the U.S. where Roche has received certain approvals for ELEVIDYS.

Catalent, Inc.

In August 2025, the Company entered into a supplemental letter agreement (the “Letter Agreement”) with Catalent in connection with the Company’s manufacturing collaboration agreement and manufacturing and supply agreement entered into with Catalent in October 2018 and February 2019, respectively, and as amended in November 2022 (collectively, the “Catalent Agreements”). The Letter Agreement addresses certain financial and operational matters related to the Company’s suspension of ELEVIDYS shipments for non-ambulatory patients in June 2025 and the temporary suspension of all U.S. ELEVIDYS shipments in July 2025. In addition, the parties agreed to delay delivery of certain ELEVIDYS batches until 2027 and beyond.

Under the terms of the Letter Agreement, the Company:

(1)

agreed to reimburse Catalent for raw materials that had been ordered or received by Catalent and not yet invoiced to the Company as of the effective date of the Letter Agreement. As such, the Company paid $61.8 million of previously billed raw materials associated with the Company's binding payment for the first quarter of 2026, which was recorded as other current assets in its unaudited condensed consolidated balance sheets as of September 30, 2025. The Company and Catalent are currently in discussions to finalize the outstanding balance related to certain unbilled raw materials associated with the Supplemental Letter Agreement. While the outcome of these discussions is uncertain, any resolution may result in adjustments to the Company’s cost of sales in future periods.

(2)

will remain contractually obligated for the full batch price of certain manufacturing batches, including a portion of batches deferred to calendar year 2027. The Company is required to pay 50% of the batch price for the deferred batches by July 1, 2026, and the remaining 50% by June 30, 2027, regardless of whether the batches are manufactured. These commitments are considered purchase obligations and are reflected in the Company’s total manufacturing commitments disclosure, above.

(3)

agreedto reimburse Catalent an amount representing estimated failed batches through June 30, 2025 based on the batch success rate stipulated in the Catalent Agreements. As a result, the Company recorded $14.2 million and $20.8 million within cost of sales in its unaudited condensed consolidated statements of comprehensive (loss) income for the three and nine months ended September 30, 2025, respectively.

Additionally, in connection with changes to the Company's forward-looking production for ELEVIDYS, the Company recognized an impairment charge of $17.0 million related to prepaid deposits, which was recorded within cost of sales in the Company's unaudited condensed consolidated statements of comprehensive (loss) income for the three and nine months ended September 30, 2025.

Thermo Fisher Scientific, Inc.

The Company entered into a development, commercial manufacturing, and supply agreement as related to the Company's adherent manufacturing process for its gene therapy programs in June 2018 and, subsequently, entered into the first, second, third and fourth amendments in May 2019, July 2020, October 2021 and March 2023, respectively, with Brammer Bio MA, LLC (“Brammer”), an affiliate of Thermo Fisher Scientific, Inc. (“Thermo”) (collectively, the “Thermo Agreement”).

On July 18, 2024, the Company issued a termination notice to Thermo to terminate the Thermo Agreement. The termination was effective as of August 21, 2024.

On December 20, 2024, Brammer filed an arbitration demand against the Company relating to the Company’s termination of the Thermo Agreement. Brammer alleged claims for breach of contract, breach of the implied covenant of good faith and fair dealing and a violation of MGL c. 93A, and sought relief including damages, treble damages, and attorneys’ fees and costs. On January 24, 2025, Sarepta filed its answer and asserted counterclaims for declaratory judgment and breach of contract, seeking relief including damages and attorneys’ fees and costs.

On July 12, 2025, the Company entered into a settlement agreement with Brammer to resolve the outstanding claims related to the termination of the Thermo Agreement (the “Brammer Settlement”). Under the Brammer Settlement, the Company agreed to pay Brammer an aggregate amount of $13.0 million. The arbitration was subsequently dismissed. The Brammer Settlement is recorded in the Company's unaudited condensed consolidated statements of comprehensive (loss) income within research and development expense for thenine months ended September 30, 2025.

Litigation

In the normal course of business, the Company from time to time is named as a party to various legal claims, actions and complaints, which have included and may include matters involving securities, employment, intellectual property, arising from the use of therapeutics utilizing its technology, or others. The Company records a loss contingency reserve for a legal proceeding when it considers the potential loss probable and it can reasonably estimate the amount of the loss or determine a probable range of loss. The Company provides disclosure when it considers a loss reasonably possible or when it determines that a loss in excess of a reserve is reasonably possible. The Company provides an estimate of such reasonably possible losses or an aggregate range of such reasonably possible losses, unless the Company believes that such an estimate cannot be made. The Company has not recorded any material accruals for loss contingencies, and in management's opinion, no material range of loss is estimable for the matters described below as of September 30, 2025.

On September 15, 2020, REGENXBIO INC. (“Regenx”) and the Trustees of the University of Pennsylvania (“U-Penn”) filed a lawsuit against the Company and Sarepta Therapeutics Three, LLC, in the U.S. District Court for the District of Delaware. The plaintiffs assert patent infringement of U.S. Patent No. 10,526,617 (“the ’617 Patent”) under 35 U.S.C.§§ 271(a)-(c) based on

Sarepta’s alleged direct or indirect manufacture and use of the patented cultured host cell technology allegedly used to make AAV gene therapy products, including SRP-9001 (approved June 22, 2023 in the U.S. as ELEVIDYS®). Specifically, the Complaint essentially includes the allegation that Sarepta’s use, and the use by its contract manufacturers on its behalf, of a host cell containing a recombinant acid molecule that encodes a capsid protein having at least 95% amino acid identity to AAVrh10 infringes the ’617 Patent asserted by Regenx. Plaintiffs seek injunctive relief, a judgment of infringement and willful infringement, damages that are no less than a reasonable royalty (treble damages), attorneys’ fees and costs, and such other relief as the court deems just and proper. On January 5, 2024, the Court granted Sarepta’s motion for summary judgment on the grounds that the asserted claims of Regenx’s ’617 Patent are invalid because they cover patent ineligible subject matter under 35 U.S.C. § 101. On January 12, 2024, the Court entered judgment and closed the case. Plaintiffs have appealed to the U.S. Court of Appeals for the (the “Federal Circuit”), which heard oral arguments on October 7, 2025.

On June 20, 2023, Regenx and U-Penn commenced a second patent infringement lawsuit against Sarepta and its contract manufacturer, Catalent, asserting alleged patent infringement of U. S. Patent No. 11,680,274 (“the ’274 Patent”). In the second lawsuit, Regenx and U-Penn allege that Sarepta and Catalent’s manufacture, use and commercial launch of ELEVIDYS® (formerly/also known as SRP-9001) infringe the ’274 Patent. Sarepta answered the complaint on August 10, 2023. On February 21, 2024, Sarepta submitted a petition for Inter Partes Review for filing with the Patent Trial and Appeal Board (“PTAB”) at the U.S. Patent and Trademark Office (“USPTO”). The petition seeks to invalidate the ’274 Patent. On March 20, 2024, the court in the patent infringement litigation ordered that the case be stayed pending final resolution of the Inter Partes Review proceedings. On August 22, 2024, the PTAB instituted Inter Partes Review of all challenged claims of the ’274 Patent on all asserted grounds. U-Penn subsequently disclaimed five of the six challenged claims, and on August 20, 2025, the PTAB entered a final written decision in the Inter Partes Review proceedings finding that the sole remaining claim was not unpatentable. On October 3, 2025, the parties submitted a joint status report to the court in the patent infringement litigation, in which they agreed that the stay of the patent litigation should remain in place until Sarepta has exhausted its right to review by the Federal Circuit. On October 20, 2025, Sarepta filed a notice of appeal of the PTAB's decision to the Federal Circuit.

On July 13, 2021, Nippon Shinyaku Co., Ltd. (“Nippon Shinyaku” or “NS”) filed a lawsuit against the Company in the U.S. District Court for the District of Delaware. NS asserted a claim for breach of contract arising from Sarepta filing seven petitions for Inter Partes Review (“IPR Petitions”) with the PTAB at the USPTO, in which Sarepta sought to invalidate certain NS patents concerning exon 53 skipping technology (U.S. Patent Nos. 9,708,361, 10,385,092, 10,407,461, 10,487,106, 10,647,741, 10,662,217, and 10,683,322, respectively, and collectively the “NS Patents”). In addition, NS asserted claims for patent infringement and willful infringement of each of the NS Patents allegedly arising from Sarepta’s activities, including the sale of, its exon 53 skipping product, VYONDYS 53 (golodirsen). NS further sought a determination of non-infringement by NS alleged to arise from NS’s activities, including the sale of, its exon 53 skipping product, Viltepso (viltolarsen) and invalidity of certain patents licensed to the Company from UWA (U.S. Patent Nos. 9,994,851, 10,227,590, and 10,266,827, collectively the “UWA Patents”). In its complaint, NS sought legal fees and costs, an unspecified amount of monetary relief (treble damages) attributed to Sarepta’s alleged infringement, and such other relief as the court deems just and proper. In January 2022, the PTAB granted institution of all claims of all NS Patents in response to Sarepta’s IPR Petitions and determined that Sarepta demonstrated a reasonable likelihood of success in proving that the NS Patents are unpatentable. NS filed a motion for preliminary injunction solely seeking Sarepta’s withdrawal of the IPR Petitions, which was ultimately granted after the U.S. Court of Appeals for the Federal Circuit reversed and remanded to the district court on February 8, 2022. Sarepta subsequently withdrew the IPRs, which were terminated on June 14, 2022. On December 27, 2021, the district court partially granted and denied the motion to dismiss by Sarepta and ordered NS to file a Second Amended Complaint (“SAC”), which it did on January 14, 2022. In the SAC, NS maintained all claims of the original complaint of July 13, 2021, except a determination of non-infringement of the UWA Patents. On January 28, 2022, Sarepta filed its answer to the SAC, with defenses and counterclaims against NS and NS Pharma Inc. that include infringement of the UWA Patents allegedly arising from their activities concerning, including the sale of, its exon 53 skipping product, Viltepso (viltolarsen) and breach of contract. Sarepta also sought a determination of invalidity of the NS Patents. In its counterclaim complaint, Sarepta sought an award of relief in its defenses to NS’ allegations, a judgment of breach of contract, a determination of invalidity of the NS Patents, a judgment of infringement and willful infringement of the UWA Patents, legal fees and costs, an unspecified amount of monetary relief (treble damages) attributable to NS’ alleged infringement, and such other relief as the court deems just and proper. UWA has since been joined as a Plaintiff in Sarepta’s counterclaims against NS. On August 14, 2023, the Court granted cross motions to amend the pleadings, allowing Sarepta to add a counterclaim against NS for inequitable conduct, and NS to add counterclaims against Sarepta for inequitable conduct and Walker Process fraud. The parties have since stipulated to the dismissal of NS’s claim of infringement of its ’361 Patent and certain claims of the ’322 Patent, and NS’s breach of contract claim. The Court bifurcated the Walker Process fraud claim on April 18, 2024, and granted Sarepta's motion for summary judgment of infringement of the ’851 Patent and NS’s motion for partial summary judgment of infringement of certain NS patents on May 1, 2024. After a jury trial in December 2024, the jury found that NS’s ’092 Patent is invalid as obvious and Sarepta’s and UWA’s ’851 Patent is not invalid. The jury did not find that NS’s infringement was willful. The jury awarded Sarepta approximately $115.2 million in damages for NS’s infringement relating to its sales of Viltepso in the United States, and the parties stipulated to approximately $0.8 millionin reasonable royalty damages for NS's sales of Viltepso outside of the United States, both through December 15, 2024. Judgment was entered on January 7, 2025. On February 14, 2025, Sarepta filed a motion seeking supplemental damages and NS filed post-trial motions challenging the jury's verdict, as well as briefing relating to its inequitable conduct claim, which was tried to the court in December 2024. Those motions remain pending.

On July 26, 2024, Genzyme Corporation ("Genzyme") filed a lawsuit against Sarepta Therapeutics, Inc. and Sarepta Therapeutics Three, LLC, in the U.S. District Court for the District of Delaware. The complaint asserts infringement of United States Patent Nos. 9,051,542 (the “’542 Patent”) and 7,704,721 (the “’721 Patent”) arising from Sarepta’s alleged manufacture and sale of ELEVIDYS® (delandistrogene moxeparvovec-rokl). In its complaint, Genzyme seeks, inter alia, damages for the alleged infringement, including increased damages up to three times the amount found or assessed, together with prejudgment and post-judgment interest and costs. Following a partial motion to dismiss by Sarepta, Genzyme filed its First Amended Complaint on November 21, 2024. In its First Amended Complaint, Genzyme no longer alleges willfulness or indirect infringement before the filing of the complaint. Sarepta answered the First Amended Complaint on December 12, 2024. On May 27, 2025, the court subsequently granted Genzyme’s motion to amend the complaint to include new allegations of infringement related to five patents: United States Patent Nos. 12,031,894 (the “’894 Patent"), 12,013,326 (the “’326 Patent”), 11,698,377 (the “’377 Patent”), 12,123,880 (the “’880 Patent”), and 12,298,313 (the “’313 Patent”). The Court also entered an amended scheduling order, with trial scheduled for June 14, 2027. Sarepta answered the Second Amended Complaint on June 17, 2025. Sarepta has submitted petitions for Inter Partes Review for filing with the PTAB challenging the validity of the ’542 and ’721 Patents, for which institution decisions are expected in January 2026. Proceedings in the patent litigation remain ongoing.

On December 20, 2024, Brammer filed an arbitration demand against the Company relating to the Company’s termination of the Thermo Agreement. Brammer alleged claims for breach of contract, breach of the implied covenant of good faith and fair dealing and a violation of MGL c. 93A, and sought relief including damages, treble damages, and attorneys’ fees and costs. On January 24, 2025, Sarepta filed its answer and asserted counterclaims for declaratory judgment and breach of contract, seeking relief including damages and attorneys’ fees and costs. On July 12, 2025, the parties entered into the Brammer Settlement, pursuant to which the Company agreed to pay Brammer an aggregate amount of $13.0 million. The arbitration was subsequently dismissed.

On June 26, 2025, a putative securities class action complaint was filed against the Company, Chief Executive Officer Douglas Ingram, former Chief Customer Officer Dallan Murray, and President of Research and Development and Technical Operations Louise Rodino-Klapac in the U.S. District Court for the Southern District of New York (the “Securities Action”). The complaint alleges violations of the Securities Exchange Act of 1934 (the “Exchange Act”) and Rule 10b-5 in connection with disclosures made regarding ELEVIDYS’s safety and efficacy and the Company’s financial statements and projections. The plaintiff seeks to represent a class of shareholders who purchased or otherwise acquired the Company’s securities between June 22, 2023 and June 24, 2025. The complaint seeks unspecified damages. On October 17, 2025, the court appointed lead plaintiffs and lead counsel. On October 24, 2025, lead plaintiffs filed a motion to transfer venue to the U.S. District Court for the District of Massachusetts and stay the entry of a case schedule, which Sarepta did not oppose. The parties also filed a stipulation, subject to Court approval, related to the timing of the filing of an Amended Complaint.

On July 15, 2025, a shareholder derivative lawsuit concerning substantially the same disclosures underlying the Securities Action was filed in the U.S. District Court for the Southern District of New York, naming Company directors M. Kathleen Behrens, Richard J. Barry, Kathryn Boor, Michael Chambers, Deirdre Connelly, Stephen L. Mayo, Claude Nicaise, and Hans Wigzell, as well as Douglas Ingram, Dallan Murray, and Louise Rodino-Klapac (the “Individual Defendants”). Two substantially similar shareholder derivative lawsuits were filed in the same court against the same defendants on August 20, 2025 and September 10, 2025, respectively (collectively, the “Derivative Actions”). The Company is named as a nominal defendant in all three lawsuits. The Derivative Actions allege, among other claims, breaches of the Individual Defendants’ fiduciary duties in connection with substantially similar disclosures at issue in the Securities Action and violations of Section 14(a) of the Exchange Act in connection with the Company’s 2024 and 2025 annual proxy statements. The Derivative Action seeks unspecified damages, including costs incurred in defending the Company against the Securities Action. Two of the Derivative Actions also seek an order that the Company take all necessary action to reform and improve its corporate governance and internal procedures. On October 10, 2025, the Court consolidated the Derivative Actions and appointed co-lead counsel (the “Consolidated Derivative Action”).