Sarepta Therapeutics, Inc.

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. In response to safety events announced in March and June 2025, the Company suspended all shipments of ELEVIDYS to non-ambulatory patients in the U.S. In response to a request from the FDA that the Company voluntarily stop all shipments of ELEVIDYS in the U.S., it temporarily suspended all shipments of ELEVIDYS in the U.S., effective July 22, 2025. On July 28, 2025, the FDA informed the Company that the FDA recommended the removal of the voluntary hold for ambulatory patients. On July 31, 2025, the Company resumed shipments of ELEVIDYS for ambulatory patients in the U.S. In November 2025, the Company announced a boxed warning for acute liver injury (“ALI”) and acute liver failure (“ALF”) and removal of non-ambulatory population from the Indication and Usage section of the Prescribing Information.

As of December 31, 2025, the Company had $953.8 million of cash, cash equivalents, restricted cash and investments, consisting of $801.3 million of cash and cash equivalents, $139.4 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 and availability under the Company's Revolving Credit Facility (defined in Note 13), 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 POLICIESAND RECENT ACCOUNTING PRONOUNCEMENTS

Basis of Presentation

The accompanying 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. All adjustments of a normal recurring nature necessary for a fair presentation have been reflected.

Estimates and Uncertainties

The preparation of the 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, revenue, expenses and the disclosure of contingent assets and liabilities. Actual results could differ from those estimates.

Correction of Immaterial Errors

During the fourth quarter ended December 31, 2025, the Company recorded an immaterial error correction associated with the accounting for its partial refinancing of its convertible senior notes due on September 15, 2027 (the “2027 Notes”) that was completed on August 28, 2025 (the "August 2025 Exchange"). During the three and nine months ended September 30, 2025, the Company recognized a loss on debt extinguishment of $138.6 million associated with the August 2025 Exchange. Upon further analysis during the fourth quarter of 2025, the Company determined that a gain on debt extinguishment of $3.8 million should have been recognized based on the fair value of the 2030 Notes at issuance. Correspondingly, the related interest expense recognized during the three and nine months ended September 30, 2025 should have been $1.9 million higher and the long-term debt balance should have been $140.5 million lower as of September 30, 2025. The correction of the errors resulted in a net decrease of $140.5 million to the previously reported net loss for both the three and nine months ended September 30, 2025. Accordingly, the previously reported basic and diluted net loss per share decreased by $1.40 and $1.43, respectively, for the three and nine months ended September 30, 2025.

The quarterly financial data required by Item 302(a) of Regulation S‑K, including the three months ended September 30, 2025, has been updated to reflect this correction in Note 24, Quarterly Financial Data.

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.

The fair value of the majority of the Company’s financial assets is categorized as Level 1 within the fair value hierarchy. These assets 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. For additional information related to fair value measurements, please read Note 5, Fair Value Measurements to the consolidated financial statements.

Cash Equivalents

Only investments that are highly liquid and readily convertible to cash and have original maturities of three months or less at the time of acquisition are considered cash equivalents.

Investments

Available-For-Sale Debt Securities

Available-for-sale debt securities are recorded at fair value and unrealized gains and losses are included in accumulated other comprehensive (loss) income in the consolidated statements of stockholders’ equity. Interest income and realized gains and losses are reported in other (expense) income, net, on a specific identification basis. The amortized cost of debt securities is adjusted for amortization of premiums and accretion of discounts to maturity, the net amount of which, along with interest and realized gains and losses, is included in other (expense) income, net in the consolidated statements of comprehensive (loss) income.

Equity Investments

The Company’s equity investments include its strategic investments in both publicly traded and private biotechnology companies and are included in other non-current assets in the Company’s consolidated balance sheets. The strategic investment in the publicly traded biotechnology company has a readily determinable fair value and is carried at fair value. The strategic investments in the privately held biotechnology companies do not have a readily determinable fair value and are measured at cost less any impairment, plus or minus changes resulting from observable price changes for the identical or a similar investment of the same issuer. Any change in the valuation of equity investments is recorded as a gain or loss on the Company’s consolidated statements of comprehensive (loss) income.

Accounts Receivable, Net

The Company’s accounts receivable, net arise from product sales. They are generally stated at the invoiced amount and do not bear interest.

The accounts receivable, net from product sales represents receivables due from the Company’s specialty distributor and specialty pharmacies and sites of care in the U.S., as well as certain distributors in South America, Europe, and the Middle East. Historically, the Company has had no material write-offs of its accounts receivable, net. Payment terms range from 65 to 90 days for sales within the U.S. and 90 and 150 days for the majority of product sales outside the U.S. The Company monitors the financial performance and creditworthiness of its customers so that it can properly assess and respond to changes in the customers’ credit profiles or any specific issues. The Company provides reserves against trade receivables for expected credit losses that may result from a customer’s inability to pay. Amounts determined to be uncollectible are written-off against the established reserve. As of December 31, 2025 and 2024, the credit profiles for the Company’s customers are deemed to be in good standing and an allowance for expected credit losses is not considered necessary.

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 December 31, 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 December 31, 2025, four entities accounted for 33%,26%,20% and 10% of accounts receivable, net, respectively. As of December 31, 2024, three entities accounted for 54%,21%, and 13%, of accounts receivable, net, respectively.

Inventories

Inventories are stated at the lower of cost and net realizable value with cost determined on a first-in, first-out basis. The Company capitalizes inventory costs associated with products following regulatory approval when future commercialization is considered probable and the future economic benefit is expected to be realized. EXONDYS 51, VYONDYS 53, AMONDYS 45 and ELEVIDYS inventory used in clinical development programs is charged to research and development expense when the product enters the research and development process and can no longer be used for commercial purposes.

The Company periodically analyzes its inventories for excess or obsolescence and writes down excess or obsolete or otherwise unmarketable inventory to its estimated net realizable value. Reserves are recorded to reduce the cost basis of inventory when it is determined that inventory on hand is excess or obsolete. The Company determines excess or obsolete inventory based on assumptions about forecasted demand for our products, market conditions and regulatory approval. Additionally, though the Company’s products are subject to strict quality control and monitoring, which the Company performs throughout the manufacturing processes, certain batches or units of product may not meet quality specifications. Expense incurred related to excess inventory, obsolete inventory, or inventories that do not meet the Company's quality specifications is recorded as a component of cost of sales in the Company's consolidated statements of comprehensive (loss) income.

For products which are under development and have not yet been approved by regulatory authorities, purchased drug product is charged to research and development expense upon delivery. Delivery occurs when the inventory passes quality inspection and title and risk of loss transfers to the Company. Nonrefundable advance payments for research and development activities, including production of purchased drug product, are deferred and capitalized until the goods are delivered. If the Company does not expect the goods to be delivered or services to be rendered, the capitalized advanced payment will be charged to expense.

Property and Equipment

Property and equipment are initially recorded at cost, including the acquisition cost and all costs necessarily incurred to bring the asset to the location and working condition necessary for their intended use. The cost of normal, recurring or periodic repairs and maintenance activities related to property and equipment are expensed as incurred. The cost for planned major maintenance activities, including the related acquisition or construction of assets, is capitalized if the repair will result in future economic benefits. Interest costs incurred during the construction period of major capital projects are periodically reviewed, and if determined to be material, capitalized until the asset is ready for its intended use, at which point the interest costs are amortized as depreciation expense over the life of the underlying asset.

The Company generally depreciates the cost of its property and equipment using the straight-line method over the estimated useful lives of the respective assets, which are summarized as follows:

Intangible assets

The Company’s intangible assets, consisting of in-licensed rights, patent costs and software licenses, are included within other non-current assets in the Company’s consolidated balance sheets.

The in-licensed rights primarily relate to agreements with BioMarin Pharmaceutical, Inc. (“BioMarin”), the University of Western Australia (“UWA”), the Research Institute at Nationwide Children’s Hospital (“Nationwide”) and Parent Project Muscular Dystrophy (“PPMD”). The in-licensed rights are being amortized on a straight-line basis over the remaining life of the related patents because the life of the related patents reflects the expected time period that the Company will benefit from the in-licensed rights.

Impairment of Long-Lived Assets

Long-lived assets held and used by the Company, intangible assets with definite lives and right of use (“ROU”) assets are reviewed for impairment whenever events or circumstances indicate that the carrying amount of the assets may not be recoverable. The Company evaluates recoverability of assets to be held and used by comparing the carrying amount of an asset to future net undiscounted cash flows to be generated by the asset. If the asset is considered to be impaired, the impairment to be recognized is measured as the amount by which the carrying amount of the assets exceeds the fair value of the assets. Such reviews assess the fair value of the assets based upon estimates of future cash flows that the assets are expected to generate.

Convertible Debt

The Company accounts for the liability and equity components of convertible debt instruments that can be settled in cash as a single liability measured at its amortized cost, as long as no other features require bifurcation and recognition as derivatives under ASC Topic 815, Derivatives and Hedging(“ASC 815”). Simultaneously with the issuance of the 2027 Notes in September 2022, the Company bought capped call options from certain counterparties to minimize the impact of potential dilution upon conversion. The premium for the capped call options was recorded as additional paid-in capital. For additional information related to the convertible debt transactions, please read Note 13, Indebtedness to the consolidated financial statements.

Revenue Recognition

The Company recognizes revenue when a customer obtains control of promised goods or services, in an amount that reflects the consideration which the Company expects to receive in exchange for the goods or services provided. To determine revenue recognition for arrangements within the scope of ASC Topic 606, Revenue from Contracts with Customers(“ASC 606”), the Company performs the following five steps: (1) identify the contract with the customer; (2) identify the performance obligations in the contract; (3) determine the transaction price; (4) allocate the transaction price to the performance obligations in the contract; and (5) recognize revenue when or as the Company satisfies a performance obligation. The Company only applies the five-step model to contracts when it is probable that the entity will collect the consideration it is entitled to in exchange for the goods or services it transfers or provides to the customer. At contract inception, the Company assesses the goods or services promised within each contract and determines those that are performance obligations, and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when the performance obligation is satisfied. The only performance obligation in the Company’s contracts with customers is to timely deliver drug products to the customer’s designated location.

Product revenues

The Company distributes its products principally through its customers or sells directly to sites of care. When the product is distributed through customers, the customers subsequently resell the products to patients and health care providers. The Company provides right of return to the customers only in cases of shipping error or product defect and other limited rights. Product revenues are recognized when the customers take control of the products, which typically occurs upon delivery. For information related to revenues by product type and region, please read Note 7, Product Revenues, Net, Accounts Receivable, Net and Reserves for Product Revenues to the consolidated financial statements.

Variable Consideration

Product revenues are recorded at the net sales price (transaction price) which includes reserves for variable consideration such as: rebates and chargebacks, distribution fees, prompt pay discounts, patient assistance and return reserves. These reserves, representing the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the contracts, are based on the amounts earned or to be claimed on the related sales and are classified as reductions of accounts receivable if no payments are required of the Company or a current liability if a payment is required of the Company. Where appropriate, the estimates reflect the Company’s historical experience, contractual and statutory requirements, industry data and forecasted customer buying and payment patterns. Actual amounts may differ from the Company’s estimates. If actual results vary, these estimates are adjusted, which could have an effect on revenue in the period of adjustment.

Additional details relating to variable consideration are as follows:

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Rebates and chargebacks: relating to governmental and commercial rebates and chargebacks.

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Governmental rebates, including Medicaid rebates, relate to the Company’s estimated obligations to federal or state governments under established reimbursement arrangements. The commercial rebates relate to arrangements the Company enters into with payors that provide for privately-negotiated rebates. Rebate reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability which is included in accrued expenses.

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Chargebacks, including Public Health Services (“PHS”) chargebacks, relate to the Company’s estimated obligations resulting from contractual commitments to sell products to qualified healthcare providers at prices lower than the list prices that the Company charges to wholesalers. The wholesaler charges the Company for the difference between what the wholesaler pays for the products and the ultimate selling price to the qualified healthcare providers. Chargeback reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and accounts receivable. Chargeback amounts are generally determined at the time of resale to the qualified healthcare provider from the wholesaler, and the Company generally issues credits for such amounts within a few weeks of receiving notification of resale from the wholesaler.

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Distribution fees: relating to fees paid to customers in the distribution channel that provide the Company with inventory management and data and distribution services are generally accounted for as a reduction of revenue. To the extent that the services received are distinct from the Company’s sale of products to the customers, these payments are accounted for as selling, general and administrative expenses. Reserves for distribution fees result in an increase in a liability if payments are required of the Company or a reduction of accounts receivable if no payments are required of the Company.

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Prompt payment discounts: relating to the Company’s estimated obligations for credits to be granted to specialty pharmacies for remitting payment on their purchases within established incentive periods. Reserves for prompt payment discounts are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and accounts receivable.

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Patient assistance: relating to financial assistance programs provided to qualified patients. Reserves for costs related to patient assistance programs are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability which is included in accrued expenses.

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Return reserves: relating to the limited return rights the Company provides to customers. The Company records product return reserve, if appropriate, as a reduction of revenue at the time product sales are recorded. Several factors are considered in determining whether return reserves are required, including the patient population and the customers’ limited return rights. Because of the pricing, the limited number of patients, and the customers’ limited return rights, most customers only carry a limited inventory. Based on these factors and the fact that the Company has not experienced significant product returns to date, return reserves have been immaterial to date.

Collaboration revenue

The Company’s collaboration revenue is primarily generated from its collaboration arrangement with F. Hoffman-La Roche Ltd. (“Roche”). For more information, please read Note 3, License and Collaboration Agreements. At the inception of a collaboration arrangement, the Company first assesses whether the contractual arrangement is within the scope of ASC Topic 808, Collaborative Arrangements to determine whether the arrangement involves a joint operating activity and involves two (or more) parties that are both active participants in the activity and exposed to significant risks and rewards dependent on the commercial success of such activity. Then the Company determines whether the collaboration arrangement in its entirety represents a contract with a customer as defined by ASC 606. If only a portion of the collaboration arrangement is potentially with a customer, the Company applies the distinct good or service unit-of-account guidance in ASC 606 to determine whether there is a unit of account that should be accounted for under ASC 606. For the units of account in the collaboration arrangement that do not represent a vendor-customer relationship, the Company will (i) consider applying other GAAP, including by analogy, or (ii) if there is no appropriate analogy, consistently apply a reasonable and rational accounting policy election.

In general, by analogy to ASC 606, the Company identifies the performance obligations within the collaboration arrangement and identifies and allocates the transaction price the Company expects to receive on a relative standalone selling price basis to each performance obligation. Variable consideration, consisting of development and regulatory milestones, will be included in the transaction price only if the Company expects to receive such consideration and if it is probable that the inclusion of the variable consideration will not result in a significant reversal in the cumulative amount of revenue recognized under the arrangement. Sales-based royalty and milestone payments are excluded from the transaction price the Company expects to receive until the underlying sales occur because the license to the Company’s intellectual property is deemed to be the predominant item to which the royalties or milestones relate as it is the primary driver of value in its collaboration arrangement.

For the recognition of revenue associated with each performance obligation, if the Company determines ASC 606 is not appropriate to apply by analogy, the Company will apply a reasonable, rational and consistently applied accounting policy election to faithfully depict the transfer of services to the collaboration partner over the estimated performance period. Up-front payments from a collaboration partner are recognized as deferred revenue when received and recognized as revenue over the estimated performance period. Reimbursement payments from a collaboration partner associated with cost-sharing provisions in a collaboration arrangement are recognized as the related expense is incurred and classified as an offset to operating expenses. Revenue from sales-based royalty payments is included as collaboration and other revenues on the consolidated statements of comprehensive (loss) income. Revenue from product sold to collaboration partners under a collaboration arrangement via contract manufacturing is included as collaboration and other revenues on the consolidated statements of comprehensive (loss) income.

Valuation of Product Options

The Company's collaboration arrangements may contain options which provide the collaboration partner with the right to obtain additional licenses. If an arrangement contains product options, by analogy to ASC 606, the Company evaluates the product options to determine whether they represent material rights, which may include options to acquire additional goods or services for free or at a discount. If the customer options are determined to represent material rights, they are recognized as a separate performance obligation at inception of the arrangement. The Company allocates a portion of the transaction price of the collaboration arrangement to material rights based on the relative standalone selling price. Amounts allocated to material rights are not recognized as revenue until related options are exercised or expire. Key assumptions to determine the standalone selling price of product options in a collaboration arrangement include, but are not limited to, forecasted revenues, development timelines, incremental costs related to the arrangement, discount rates and likelihood of technical and regulatory success.

Research and Development Expenses

Research and development expenses consist of costs associated with research activities as well as those with the Company’s product development efforts, conducting pre-clinical trials, clinical trials and manufacturing activities. Research and development expenses are expensed as incurred. Up-front fees and milestones paid to third parties in connection with technologies which have not reached technological feasibility and do not have an alternative future use are expensed when incurred.

Direct research and development expenses associated with the Company’s programs include payments made to contract research organizations, clinical trial site costs, clinical manufacturing costs, costs incurred for consultants and other external services, such as data management and statistical analysis support and materials and supplies used in support of clinical programs. Indirect costs of the Company’s research and development programs include salaries, stock-based compensation and an allocation of its facility and technology costs.

When third-party service providers’ billing terms do not coincide with the Company’s period-end, the Company is required to make estimates of its obligations to those third parties, including clinical trial and pharmaceutical development costs, contractual services costs and costs for supply of its drug candidates incurred in a given accounting period, and record accruals at the end of the period. The Company bases its estimates on its knowledge of the research and development programs, services performed for the period, past history for related activities and the expected duration of the third-party service contract, where applicable.

Selling, General and Administrative Expenses

Selling, general and administrative expenses consist of salaries, benefits, stock-based compensation and related costs for personnel in the Company's executive, finance, legal, information technology, business development, human resources, commercial and other general and administrative functions. Other general and administrative expenses include an allocation of the Company's facility- and technology-related costs and professional fees for legal, consulting and accounting services.

Advertising costs are included in selling, general and administrative expenses and are expensed as incurred. The Company considers advertising costs as expenses related to the promotion of the Company's commercial products. For the years ended December 31, 2025, 2024 and 2023, advertising costs totaled $29.5 million, $32.0 million and $28.6 million, respectively.

Stock-Based Compensation

The Company’s stock-based compensation programs include stock options, restricted stock units (“RSUs”), RSU shares with performance conditions (“PSUs”), performance stock awards ("PSAs") and an employee stock purchase program (“ESPP”). Stock-based compensation is recognized based on grant date fair value of stock awards.

The fair value of stock options are estimated on the date of grant using the Black-Scholes-Merton option-pricing model. The fair values of RSUs, PSUs and PSAs are based on the fair market value of the Company’s common stock on the date of the grant. The fair value of stock awards, with consideration given to estimated forfeitures, is recognized as stock-based compensation expense on a straight-line basis over the requisite service period, which is, in most cases, the vesting period of the grants. The Company estimates forfeitures over the requisite service period using historical forfeiture activity. For stock awards with performance-vesting conditions, including PSUs and PSAs, the Company does not recognize stock-based compensation expense until it is probable that the performance condition will be achieved.

Additionally, the Company granted its Chief Executive Officer ("CEO") options with service and market conditions. A market condition relates to the achievement of a specified price of the Company’s common stock, a specified amount of intrinsic value indexed to the Company’s common stock or a specified price of the Company’s common stock in terms of other similar equity shares. The grant date fair value for the options with service and market conditions is determined by a lattice model with Monte Carlo simulations and is recognized as stock-based compensation expense on a straight-line basis over the respective derived service period.

Under the Company’s ESPP, participating employees purchase common stock through payroll deductions. The purchase price is equal to 85% of the lower of the closing price of the Company’s common stock on the first business day and the last business day of the relevant purchase period. The fair value of stock purchase rights is estimated using the Black-Scholes-Merton option-pricing model. The fair value of the look-back provision with the 15% discount is recognized on a graded-vesting basis as stock-based compensation expense over the offering period.

Income Taxes

The Company follows the asset and liability method of accounting for income taxes, which requires the recognition of deferred tax assets and liabilities for expected future tax consequences attributable to differences between the consolidated financial statement carrying amounts of existing assets and liabilities and their respective tax bases. Deferred tax assets and liabilities are measured using enacted tax rates in effect for the year in which the differences are expected to reverse. A valuation allowance is recorded to reduce the net deferred tax asset to zero when it is more likely than not that the net deferred tax asset will not be realized.

The Company recognizes the effect of income tax positions only if those positions are more likely than not of being sustained upon an examination. The amount of the benefit that may be recognized in the financial statements is the largest amount that has a greater than 50% likelihood of being realized. The Company recognizes interest and penalties related to uncertain tax positions within income tax expense.

It is the intention of the Company to reinvest the earnings of its non-U.S. subsidiaries in those operations and not to repatriate the earnings to the U.S. Accordingly, the Company does not provide for deferred taxes on the excess of the financial reporting over the tax basis in its investments in foreign subsidiaries as they are considered permanent in duration.

The Company accounts for Global Intangible Low-Taxed Income (“GILTI”) as a period cost.

Leases

At the inception of an arrangement, the Company determines whether the arrangement is or contains a lease based on the unique facts and circumstances present in the arrangement. Leases with a term greater than twelve months are recognized on the consolidated balance sheets as ROU assets and short-term and long-term lease liabilities, as applicable. The Company has elected not to recognize leases with terms of twelve months or less on the consolidated balance sheets. The Company typically only includes an initial lease term in its assessment of a lease arrangement. Options to renew a lease are not included in the Company’s assessment unless there is reasonable certainty that the Company will renew. The Company monitors its plans to renew its leases quarterly or on an as-needed basis. In addition, the Company’s lease agreements generally do not contain any residual value guarantees or restrictive covenants.

Operating lease liabilities and their corresponding ROU assets are recorded based on the present value of future lease payments over the expected remaining lease term at lease commencement. The initial measurement of the lease liability is determined based on the future lease payments, which may include lease payments that depend on an index or a rate (such as the consumer price index or other market index). The Company initially measures payments based on an index or rate by using the applicable rate at lease commencement and subsequent changes in such rates are recognized as variable lease costs. Variable payments that do not depend on a rate or index are not included in the lease liability and are recognized as they are incurred. Lease costs for operating leases are recognized on a straight-line basis over the lease term as an operating expense with unrecognized variable lease payments recognized as incurred. Certain adjustments to the ROU asset may be required for items such as lease prepayments or incentives received. The interest rate implicit in lease contracts is typically not readily determinable. As a result, the Company utilizes its incremental borrowing rate, which reflects the fixed rate at which the Company could borrow on a collateralized basis the amount of the lease payments in the same currency, for a similar term, in a similar economic environment. Components of a lease are bifurcated between lease components and non-lease components. The fixed and in-substance fixed contract consideration identified is then allocated based on the relative standalone price to the lease and non-lease components. The Company adopted a practical expedient provided by ASC Topic 842, Leases, and elected to account for the lease and non-lease components together for existing classes of underlying assets and allocates the contract consideration to the lease component only. In contrast, the Company elected not to apply the practical expedient provided by ASC Topic 842, Leases, for leases embedded in manufacturing and supply agreements with certain of its contract manufacturing organizations and has allocated contract consideration between the lease and non-lease components based on their relative standalone price.

(Loss) Earnings per 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 if-converted method for convertible notes by dividing net income by the weighted-average number of shares of common stock and potentially dilutive common stock equivalents outstanding. Potential common equivalent shares are excluded if their effect is anti-dilutive.

Embedded Derivatives

Effective January 1, 2025, the Company adopted Accounting Standard Update ("ASU") 2025-07, Derivatives and Hedging (Topic 815) and Revenue from Contracts with Customers (Topic 606): Scope Refinements ("ASU 2025-07"), which introduced a scope exception for non-exchange-traded contracts with underlyings based on operations or activities specific to one party. As a result, certain contingent consideration obligations previously accounted for as embedded derivatives no longer meet the definition of a derivative under ASC 815, Derivatives and Hedging (“ASC 815”). See Recent Accounting Pronouncements below related to the cumulative effect of adopting this new accounting guidance.

Contingent Consideration Arising from Asset Acquisitions

Following the adoption of ASU 2025‑07, cash contingent consideration obligations arising from asset acquisitions, which are not accounted for as derivatives, are accounted for under ASC 450, Contingencies("ASC 450"). The Company will recognize a liability when it becomes probable that payment will be required and the amount can be reasonably estimated.

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.

Commitments and Contingencies

The Company records liabilities for legal and other contingencies when information available to the Company indicates that it is probable that a liability has been incurred and the amount of loss can be reasonably estimated. Legal costs in connection with legal and other contingencies are expensed as costs are incurred as selling, general and administrative expenses.

Recent Accounting Pronouncements

Recently adopted

In September 2025, the Financial Accounting Standards Board (the "FASB") issued 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 ASC 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, 2026 and interim periods within those fiscal years. Early adoption is permitted.

Effective in the fourth quarter of 2025, the Company early adopted ASU 2025-07 using the modified retrospective transition method. As a result, two existing contracts that include a settlement feature based on the Company’s operations or activities are now excluded from the scope of ASC 815 and any liabilities will be recognized as such obligations become probable and estimable under ASC 450. The Company recorded the cumulative effect of this accounting change to remove the previously recognized derivative liabilities as of January 1, 2025, reducing the contingent consideration liability by $47.4 million, with an offsetting adjustment to accumulated deficit. The elimination of this derivative liability would result in an increase of $11.1 million to the previously reported net loss for both the three and nine months ended September 30, 2025. Accordingly, the previously reported basic and diluted net loss per share would increase by $0.11 for both the three and nine months ended September 30, 2025. There isno impact to the previously reported net loss for the quarters ended March 31, 2025 and June 30, 2025, respectively.

In November 2024, the FASB issued ASU 2024-04, Induced Conversions of Convertible Debt Instruments("ASU 2024-04"), 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 13, Indebtedness.

In December 2023, the FASB issued ASU 2023-09, Improvements to Income Tax Disclosures. ASU 2023-09 is effective for fiscal years beginning after December 15, 2024. ASU 2023-09 enhances the transparency and decision usefulness of income tax disclosures, primarily related to the rate reconciliation and income taxes paid information. The Company adopted ASU 2023-09 on a retrospective basis for all periods presented. The adoption of ASU 2023-09 had no impact to the Company’s consolidated balance sheets, consolidated statements of comprehensive (loss) income, or consolidated statements of cash flows, as ASU 2023-09 affects disclosures only. Refer to Note 18, Income Taxesfor the related disclosures required by ASU 2023-09.

Recently issued but not yet adopted

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. The Company is evaluating the impact of this guidance on its consolidated financial statements and related disclosures.

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"), which requires public entities to provide disaggregated disclosure of income statement expenses. Public entities are required to disaggregate, in a tabular presentation, each relevant expense caption on the face of the consolidated statements of comprehensive (loss) income such as the following expenses: purchases of inventory, employee compensation, intangible asset amortization, and depreciation. ASU 2024-03 is effective for annual reporting periods beginning after December 15, 2026 and interim periods beginning after December 15, 2027, with early adoption permitted. The Company is evaluating the impact of this guidance on its consolidated financial statements and related disclosures.

3. LICENSE AND COLLABORATION AGREEMENTS

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 Collaboration 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 Collaboration 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 Collaboration Agreement are governed by a series of committees established to facilitate transition and collaboration between the two parties.

Under the terms of the Arrowhead Collaboration 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 Collaboration 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 Collaboration Agreement, Arrowhead appointed the Company’s CEO, Douglas Ingram, as a director, effective February 2025. The Company’s CEO’s is not standing for re-election to Arrowhead’s board of directors and their term will conclude at Arrowhead’s 2026 annual meeting.

When the Arrowhead Collaboration 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 consolidated balance sheets on the Effective Date. For the year ended December 31, 2025, the Company recorded $583.6 million as acquired IPR&D expense, as the remainder of the up-front payments was 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 did not have a controlling financial interest in Arrowhead, as the Company did 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 did not have significant influence over Arrowhead, the Company’s investment in Arrowhead was not subject to the equity method of accounting. Accordingly, the investment in Arrowhead’s common stock was recorded at fair value and remeasured each reporting period through the date of sale in August 2025, with changes recorded toother (expense) income, net in the Company’s consolidated statements of comprehensive (loss) income. The Arrowhead Collaboration 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 Collaboration Agreement. Over the term of the Arrowhead Collaboration 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 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, both of which were achieved during the year ended December 31, 2025, as described below. 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, totaling $100.0 million. 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. On November 24, 2025, Arrowhead achieved the second of two Arrowhead DM1 Milestones, totaling $200.0 million (the "November 2025 Milestone"). In accordance with the Arrowhead Collaboration Agreement, the Company is required to remit the payment within 60 days of achievement of the associated milestone. As of December 31, 2025, the November 2025 Milestone was not settled and the obligation is included in accounts payable in the accompanying consolidated balance sheets. In January 2026, the Company settled the November 2025 Milestone. The Company recognized milestone expenses of $300.0 million associated with the achievement of the Arrowhead DM1 Milestones as research and development expense in the accompanying consolidated statements of comprehensive(loss) income for the year ended December 31, 2025. For the year ended December 31, 2025, the Company recorded $31.5 million of research and development expense related to reimbursable development costs incurred by Arrowhead.

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 year ended December 31, 2025, the Company recognized a loss of $17.3 million, reflecting the realized loss on the sale of this strategic investment.

In December 2025, the Company entered into a clinical supply agreement with Arrowhead (the “Arrowhead CSA”) in connection with the Arrowhead Collaboration Agreement. Under the Arrowhead CSA, Arrowhead is responsible for the manufacturing and supply of certain drug substance required to support the Company's ongoing and future clinical development activities for programs under the Arrowhead Collaboration Agreement. The Arrowhead CSA provides for a jointly prepared clinical supply plan to forecast and update anticipated supply needs and employs statements of work to define required batch quantities, delivery timing and pricing for each batch. The Arrowhead CSA will remain in effect on a program-by-program basis until the earlier of (i) the conclusion of the applicable program's clinical supply term, or (ii) the termination of the Arrowhead Collaboration Agreement.

F. Hoffman-La Roche Ltd.

In December 2019, the Company entered into a license, collaboration and option agreement with Roche and a stock purchase agreement (collectively, the “Roche Collaboration Agreement”) with Roche, providing Roche with exclusive commercial rights to ELEVIDYS outside the U.S. The Company retains all rights to ELEVIDYS in the U.S. and will perform all development activities within the joint global development plan necessary to obtain and maintain regulatory approvals for ELEVIDYS in the U.S. and the EU, unless otherwise agreed to by the parties. Further: (i) research and development expenses incurred under the joint global development plan will be equally shared between the Company and Roche, (ii) Roche is solely responsible for all costs incurred in connection with any development activities (other than those within the joint global development plan) that are necessary to obtain or maintain regulatory approvals outside the U.S, and (iii) the Company will continue to be responsible for the manufacturing of clinical and commercial supplies of ELEVIDYS. The Company has also granted Roche options to acquire ex-U.S. rights to certain future Duchenne-specific programs (the “Options”) in exchange for separate option exercise payments, milestone and royalty considerations, and cost-sharing provisions. The agreement became effective in February 2020. The Roche Collaboration Agreement is governed by a joint steering committee (“JSC”) formed by representatives from Roche and the Company. The JSC, among other activities, manages the overall strategic alignment between the parties, approves any material update to the joint global development plan and budget and oversees the operations of the subcommittees.

The Company received an aggregate of approximately $1.2 billion in cash consideration from Roche, consisting of an up-front payment and an equity investment in the Company. The Company may receive up to $1.7 billion in development, regulatory and sales milestones related to ELEVIDYS. The Company receives tiered royalty payments on net sales of ELEVIDYS outside of the U.S. based on the average cost to manufacture ELEVIDYS. Of the $1.2 billion up-front cash received from Roche, (i) $312.1 million, net of issuance costs, was allocated to the approximately 2.5 million shares of the Company’s common stock issued to Roche based on the closing price when the shares were issued, (ii) $485.0 million was allocated to the Options, and (iii) $348.7 million was allocated to a single, combined performance obligation (“Combined Performance Obligation”) comprised of: (i) the license of IP relating to ELEVIDYS transferred to Roche, (ii) the related research and development services provided under the joint global development plan, (iii) the services provided to manufacture clinical supplies of ELEVIDYS, and (iv) the Company’s participation in the JSC, because the Company determined that the license of IP and related activities were not capable of being distinct from one another.

The value assigned to the Options is reflected as deferred revenue and will not be recognized until an option is either: (i) exercised by Roche, or (ii) expires. If exercised, the value will be aggregated with the option exercise price and recognized over the applicable performance period. If expired, the value will be recognized immediately. The Company recognizes revenue related to the Combined Performance Obligation on a straight-line basis over the expected performance period of the joint global development plan, which extended through the fourth quarter of 2023. Revenue relating to future development, regulatory and sales milestones will be recognized when the milestone is probable of achievement (which is typically when the milestone has occurred). Any royalties payable by Roche will be recognized in the period earned.

For the years ended December 31, 2025, 2024 and 2023, the Company recognized $333.9 million, $114.0 million and $98.5 million of collaboration and other revenues, respectively, which primarily consists of collaboration revenue, contract manufacturing revenue and royalty revenue related to 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 "Japan Approval Milestone"). The Japan Approval Milestone payment is included in collaboration and other revenues for the year ended December 31, 2025.

In February 2025, an Option for a certain program expired, which resulted in the immediate recognition of $112.0 million of collaboration revenue during the year ended December 31, 2025. On February 12, 2024, Roche declined to exercise a certain Option related to one external, early-stage development program, which resulted in that Option's expiry and the immediate recognition of the value assigned to that Option as collaboration revenue. As such, the Company recognized $48.0 million of collaboration revenue during the year ended December 31, 2024. As of December 31, 2025 and 2024, the Company had total deferred revenue of $527.3 million and $455.3 million, respectively, of which $443.4 million and $130.3 million was classified as current. The Company's deferred revenue balance primarily relates to the remaining Option in the Roche Collaboration Agreement and the deferral of certain ELEVIDYS shipments for which cash was received in advance of delivery pursuant to the Company's binding forecast with Roche.

In February 2026, an Option for a certain program was declined by Roche, which will result in the immediate recognition of $325.0 million of collaboration revenue during the year ending December 31, 2026.

For the year ended December 31, 2023, the Company recognized $89.2 million of collaboration revenue, which relates to the amortization of the Combined Performance Obligation. The fair value allocated to Combined Performance Obligation was fully amortized as of December 31, 2023.

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. The Company is eligible to receive royalties on these sales. Royalty revenue may be subject to future adjustment based upon the timing of the Company's reconciliation of average ELEVIDYS supply price under the Roche Collaboration Agreement. While the Roche Supply Agreement is in the process of being negotiated at the issuance of this annual 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 accompanying consolidated statements of 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 years ended December 31, 2025, 2024 and 2023, costs reimbursable by Roche and reflected as a reduction to operating expenses were $84.7 million, $127.1 million and $106.9 million, respectively. As of December 31, 2025 and 2024, there were $104.1 million and $34.6 million of collaboration and other receivables included in other current assets on the consolidated balance sheets, respectively.

Myonexus Therapeutics Inc.

In April 2019, the Company completed its acquisition of Myonexus Therapeutics, Inc. (“Myonexus”), a clinical-stage gene therapy biotechnology company that was developing gene therapies for Limb-girdle muscular dystrophy ("LGMD") for $178.3 million. The Company may also be required to make up to $200.0 million in additional payments to selling shareholders of Myonexus based on the achievement of certain sales-and regulatory- related milestones. The acquisition was accounted for as an asset acquisition as substantially all of the fair value of the gross assets acquired is concentrated in a group of similar identifiable assets (the five LGMD gene therapy programs). As part of the consideration for the transaction, the Company is required to make contingent payments to the selling shareholders of Myonexus upon the receipt and subsequent sale of a PRV with respect to a Myonexus product. As of December 31, 2024, the contingent consideration liability associated with Myonexus was $47.1 million. Effective January 1, 2025, upon adoption of ASU 2025‑07, the Company derecognized its contingent consideration liability associated with Myonexus. Future liabilities will be recognized as such obligations become probable and estimable under ASC 450. Refer to Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncementsfor additional information related to the adoption of ASU 2025-07.

Nationwide Children’s Hospital

In December 2016, the Company entered into an exclusive option agreement with Nationwide from which the Company obtained an exclusive right to acquire a worldwide license of the micro-dystrophin gene therapy technology for Duchenne and Becker muscular dystrophy. In October 2018, the Company exercised the option and entered into a license agreement with Nationwide, which granted the Company exclusive worldwide rights to develop, manufacture and commercialize a micro-dystrophin gene therapy product candidate. Under this agreement, the Company is liable for future regulatory milestone, sales milestone and sublicense payments as well as lower single-digit royalties upon commercialization.

During the year ended December 31, 2025, the Company recordedand paid $5.1 million due to Nationwide as a result of the Japan Approval Milestone received from Roche as an in-licensed right intangible asset in its consolidated balance sheets in accordance with the Company's license agreement with Nationwide. As of December 31, 2025, the in-licensed right asset with a net carrying value of$4.8 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.

During the year ended December 31, 2023, the Company recorded $23.0 million as an in-licensed right intangible asset in its consolidated balance sheets, $10.0 million of which related to the regulatory approval of ELEVIDYS, and the remaining related to sales-based milestones as the Company determined that all sales-based milestones were achieved or were probable of being achieved. As of December 31, 2025, the in-licensed right asset with a net carrying value of $19.0 million is being amortized on a straight-line basis over the remaining life of the relevant patent. Royalty payments due to Nationwide associated with commercial sales of ELEVIDYS totaled $38.5 million, $30.6 million, and $6.0 million for the years ended December 31, 2025, 2024 and 2023, respectively, and were recorded as cost of sales in the accompanying consolidated statements of comprehensive (loss) income.

BioMarin Pharmaceutical, Inc.

In July 2017, the Company and UWA entered into a settlement agreement with BioMarin, and simultaneously entered into a license agreement, which was subsequently amended in April 2019 (the “BioMarin Agreement”), with BioMarin and Academisch Ziekenhuis Leiden (collectively with the Company, UWA and BioMarin, the “Settlement Parties”). The BioMarin Agreement provides the Company with an exclusive license to certain intellectual property with an option to convert the exclusive license into a co-exclusive license and the Settlement Parties agreed to stop most existing efforts to continue with ongoing litigation and opposition and other administrative proceedings concerning BioMarin’s intellectual property. BioMarin is also eligible to receive tiered royalty payments, ranging from 4% to 8%, based on the net sales for the three products and product candidates.

In November 2021, the Company entered into a second settlement agreement and second amendment to the license agreement, which waived certain future milestone payments and altered royalty payment terms of the agreement. The royalty terms under the license agreement expired in March 2024 in the U.S. and expired as of December 31, 2024 in the EU and other countries.

For the years ended December 31, 2024 and 2023, the Company recognized royalty expense of $4.4 million and $17.6 million, respectively, which is included in cost of sales in the accompanying consolidated statements of comprehensive (loss) income. For the year ended December 31, 2025 there was no royalty expense recognized related to the agreements with BioMarin. For the years ended December 31, 2025, 2024 and 2023, no regulatory milestones were deemed probable of being achieved and, accordingly, no additional in-licensed rights or expenses have been recognized.

University of Western Australia

In April 2013, the Company and UWA entered into an amendment to an existing exclusive license agreement relating to the treatment of Duchenne by inducing the skipping of certain exons. The agreement was further amended in June 2016. Under the amended agreement, the Company may be obligated to make payments to UWA totaling up to $26.0 million upon the achievement of certain development, regulatory and sales milestones. Additionally, the Company is required to pay a low-single-digit percentage royalty on net sales of products covered by issued patents licensed under the agreements with UWA. For the years ended December 31, 2025, 2024 and 2023, the Company recorded $12.1 million, $12.1 million and $11.8 million in royalty expense, respectively, which is included in cost of sales, related to agreements with UWA. For the years ended December 31, 2025, 2024 and 2023, no development, regulatory or sales milestones were deemed probable of being achieved and, accordingly, no additional in-licensed rights or expenses have been recognized.

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 December 31, 2025, the Company may be obligated to make up to $12.1 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 December 31, 2025. For the years ended December 31, 2025 and 2023, the Company recognized$883.8 million and$13.2 million, respectively, relating to certain up-front, milestone and other payments as research and development expense under these agreements, withno similar activity for the year ended December 31, 2024.

4. RESTRUCTURING

On July 16, 2025, the Company announced a strategic restructuring plan (the "Restructuring"), 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 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 year ended December 31, 2025, the Company incurred restructuring charges of $42.0 million, $34.9 million 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. For the year ended December 31, 2025, the cash costs incurred under the Restructuring Plan were $34.9 million, the majority of which were paid by December 31, 2025. As of December 31, 2025, a $0.8 million liability, which is related to severance obligations and related costs, is recorded for the cash charges incurred that are unpaid, which is included in accrued expenses on the Company's consolidated balance sheets.

The following table summarizes restructuring charges for the year presented:

The following table summarizes the restructuring reserve as of December 31, 2025:

5. FAIR VALUE MEASUREMENTS

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 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 consolidated balance sheets as of December 31, 2025 and 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.

Effective January 1, 2025, upon adoption of ASU 2025‑07, the Company derecognized its contingent consideration liabilities that were previously accounted for as derivative liabilities. Future settlements will be recognized as such obligations become probable and estimable under ASC 450. Refer to Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncements for additional information related to the adoption of ASU 2025-07. As of December 31, 2024, 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 selling shareholders as well as to an academic institution under a separate license agreement that met the definition of a derivative.

The Company assesses its financial assets measured at fair value on a recurring basis and transfers its financial assets between the relevant fair value hierarchies at the end of each reporting period, as needed. There were no transfers into or out of Level 3 during the years ended December 31, 2025 and 2024. An increase of $4.4 million was recorded during the year ended December 31, 2025 to account for new strategic investments in certain private biotechnology companies. The following table represents a roll-forward of the fair value of Level 3 financial assets for each of the periods indicated:

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 years ended December 31, 2025 or 2024, as no impairment indicators were identified nor were similar securities issued.

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

The contingent consideration liability as of December 31, 2024 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 shorten or lengthen the time required to achieve the milestone, would result in a corresponding increase or decrease in the fair value of the liability.

The carrying amounts reported in the 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. For fair value information related to the Company’s debt facilities, please read Note 13, Indebtedness.

6. 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 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 was approximately three and eleven months as of December 31, 2025 and 2024, respectively. All of the Company's non-current investments as of December 31, 2025 and 2024 had maturities betweenone 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:

7. 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:

No individual country outside the U.S. exceeded 10% of total net product revenues for any of the years ended December 31, 2025, 2024 and 2023.

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

The following table summarizes an analysis of the change in reserves for discounts and allowances for the periods indicated:

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

8. INVENTORY

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

For the year ended December 31, 2025, inventory losses for write-downs of excess and obsolete inventory totaled $165.3 million related to excess ELEVIDYS and PMO inventory on hand as of December 31, 2025. Inventory write-downs were immaterial for the years ended December 31, 2024 and 2023.

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 oftwo years. The Company classifies inventory associated with ELEVIDYS as non-current inventory when consumption of the inventory is expected beyond the Company's normal ELEVIDYS inventory operating cycle of approximately 3.5 years. Non-current inventory consists of raw materials and work in progress associated with the PMO Products and raw materials associated with ELEVIDYS.

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

9. 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 years 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.

10. PROPERTY AND EQUIPMENT, NET

Property and equipment are recorded at historical cost, net of accumulated depreciation and accumulated impairment losses. The following table summarizes components of property and equipment, net, for each of the periods indicated:

For the years ended December 31, 2025, 2024 and 2023, depreciation expense totaled $51.4 million, $42.7 million, and $42.5 million, respectively.

11. INTANGIBLE ASSETS, NET

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

The in-licensed rights relate to agreements with UWA, Nationwide, BioMarin and PPMD. These in-licensed rights are being amortized on a straight-line basis over the remaining life of the related patent because the life of the related patent reflects the expected time period that the Company will benefit from the in-licensed rights. For the year ended December 31, 2025, the Company recorded $5.1 million in additions of in-licensed rights. The weighted-average remaining amortization period for the in-licensed rights additions during 2025 is 12.3 years. There wereno additions of in-licensed rights during the year ended December 31, 2024. For more information related to the Company's in-licensed rights related to its license or settlement agreements with Nationwide, BioMarin and UWA, please refer to Note 3. License and Collaboration Agreements. For the years ended December 31, 2025, 2024 and 2023, the Company recorded $3.0 million, $2.7 million and $1.9 million, respectively, of amortization expense related to intangible assets.

The following table summarizes the estimated future amortization for intangible assets:

12. ACCRUED EXPENSES

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

13. INDEBTEDNESS

2027 Convertible Notes, 2030 Convertible Notes and Related Transactions

2027 Notes Issuance and Capped Call Transactions

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 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 net proceeds were $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 aggregate issuance of the 2027 Notes includes the issuance of $20.0 million in aggregate principal amount of 2027 Notes to the Michael A. Chambers Living Trust, an entity affiliated with Michael Chambers, a member of the Company’s board of directors.

The conversion rate of the 2027 Notes is 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. As of December 31, 2025, no such conditions or events had occurred, and the notes were not eligible for conversion.

Only on or after September 20, 2025, should certain market conditions be met, the Company may redeem for cash all or any portion of the 2027 Notes at a redemption price equal to the principal amount of the 2027 Notes, plus accrued and unpaid interest. Holders of the 2027 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 2027 Notes. The 2027 Notes contain customary covenants and events of default, occurrence of which permits the holders to accelerate all outstanding obligations, including principal and interest.

In connection with the issuance of the 2027 Notes, the Company entered into privately negotiated capped call transactions with counterparties intended to minimize the impact of potential dilution upon conversion of the 2027 Notes (the “2022 Capped Calls”), which covers approximately 8.1 million shares of the Company’s common stock. The 2022 Capped Calls have an initial strike price of approximately $141.97 per share and a cap price of approximately $210.32 per share. If, upon conversion of the 2027 Notes, the price of the Company’s common stock is between the strike price and the cap price of the capped calls, the counterparties will deliver shares of the Company’s common stock and/or cash with an aggregate value equal to the difference between the price of the Company’s common stock at the conversion date and the strike price, multiplied by the number of shares of the Company’s common stock related to the capped calls being exercised. The Company paid $127.3 million for the 2022 Capped Calls.

Exchange of 2027 Notes

On August 28, 2025 (the “First Closing Date”), the Company completed the August 2025 Exchange. 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 2030 Notes;

(2)

cash payments of $127.3 million, including $4.0 million of accrued interest of the 2027 Notes; and

(3)

5.9 million shares of the Company’s common stock with a fair market value of approximately $104.9 million, net of issuance costs of $2.4 million.

The 2030 Notes issued in association with the August 2025 Exchange were issued at a discount of $142.4 million, based on the fair market value of the 2030 Notes on the First Closing Date. The Company incurred debt issuance costs of $13.4 million associated with the August 2025 Exchange.

On December 18, 2025 (the "Second Closing Date"), the Company completed a second partial refinancing of its 2027 Notes (the “December 2025 Exchange”). The Company exchanged $291.4 millionin aggregate principal amount of the 2027 Notes for the following consideration:

(1)

$291.4 million in aggregate principal amount of the 2030 Notes; and

(2)

cash payments of $31.6 million, including $1.0 million of accrued interest of the 2027 Notes.

The 2030 Notes issued in association with the December 2025 Exchange were issued at a discount of $36.4 million, based on the fair market value of the 2030 Notes on the Second Closing Date. The Company incurred debt issuance costs of $4.0 million associated with the December 2025 Exchange.

The December 2025 Exchange included $20.0 million of 2027 Notes held by the Michael A. Chambers Living Trust. The terms of Mr. Chambers' exchange were on the same basis as all other participating holders. The remaining aggregate principal amount of $158.6 million of the 2027 Notes, which are currently convertible into approximately 1.1 million 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. The December 2025 Exchange did not result in any material changes to the related party debt arrangements.

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 2030 Notes have an initial conversion rate of 16.6667 shares per $1,000 principal amount (approximately 14.9 million 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 of 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.

The Company concluded that neither the August 2025 Exchange nor December 2025 Exchange (collectively, the “Exchange Transactions”) met the criteria for induced conversion. Further, the Company concluded that the refinanced debt in the August 2025 Exchange as well as approximately $182.8 million of the December 2025 Exchange were substantially different from the original debt and, thus, accounted for as debt extinguishments. The Company also determined that the remaining $108.6 million of the refinanced debt in the December 2025 Exchange was not substantially different from the original debt and, therefore, accounted for as debt modification. The following table summarizes the results of the Exchange Transactions:

(1) Please refer to Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncements for additional information.

Revolving Credit Facility

On February 13, 2025 (the “Revolver 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. For the year ended December 31, 2025, the Company incurred $1.8 million in commitment fees related to the unused portion of the Revolving Credit Facility.

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 Revolver 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.

On December 18, 2025, the Company executed the first amendment to its Credit Agreement (the "First Amendment"). The First Amendment primarily updated regulatory representations and reaffirmed the Company’s compliance with existing covenants. No financial terms were amended as a result of the First Amendment. As of December 31, 2025, there were no amounts outstanding under the Revolving Credit Facility and the Company was in compliance with the covenants described above.

2024 Convertible Notes and Related Transactions

Issuance and Capped Call Transactions

In November 2017, the Company issued $570.0 million aggregate principal amount of senior convertible notes due on November 15, 2024 (the "2024 Notes"). The 2024 Notes were senior unsecured obligations of the Company and bore interest at a rate of 1.50% per annum, payable semi-annually in cash on each May 15 and November 15, commencing on May 15, 2018. The net proceeds were $559.4 million after deducting the discounts and offering expenses of $10.6 million. The effective interest rate on the 2024 Notes was 1.9%. The conversion rate of the 2024 Notes was 13.621 shares per $1,000 principal amount, or a conversion price of $73.42 per share, subject to adjustment under certain conditions. In connection with the issuance of the 2024 Notes, the Company entered into privately negotiated capped call transactions with counterparties intended to minimize the impact of potential dilution upon conversion of the 2024 Notes (the “2017 Capped Calls”) which covers approximately 7.8 million shares of the Company’s common stock. The Company paid $50.9 million for the 2017 Capped Calls.

Exchange and Capped Call Settlement

In March 2023, the Company entered into separate, privately negotiated exchange agreements with certain holders of the outstanding 2024 Notes (the “2024 Notes Exchange”), which resulted in an exchange of $313.5 million in aggregate principal value of the 2024 Notes for shares of the Company’s common stock. In connection with the 2024 Notes Exchange, the Company issued approximately 4.5 million shares of the Company's common stock with a fair value of approximately $693.4 million. The Company also incurred approximately $6.9 million in third-party debt conversion costs. The 2024 Notes Exchange was not pursuant to the conversion privileges included in the terms of the debt at issuance and therefore was accounted for as a debt extinguishment and resulted in a loss on debt extinguishment of $387.3 million, inclusive of the $6.9 million in third-party debt conversion costs, which was included in the consolidated statements of comprehensive (loss) income for the year ended December 31, 2023. Corresponding to the 2024 Notes Exchange, the related 2017 Capped Calls were terminated. As a result, the Company received $80.6 million in cash.

Conversion and Capped Call Settlement in 2024

Upon maturity as of November 15, 2024 (“the Maturity Date”), the Company converted the remaining outstanding bonds into approximately 1.2 million shares of its common stock equivalent to an aggregate principal amount of $91.6 million. A de minimis number of bonds were not converted and, as a result, the Company paid less than $0.1 million in cash to those bondholders. As the conversions were completed under the original terms of the 2024 Notes, there is no gain or loss impact on the accompanying consolidated statements of comprehensive (loss) income.

In May 2024, the Company informed remaining holders of the 2024 Notes of its election to settle the 2024 Notes in shares, which, pursuant to the terms of the 2017 Capped Call agreements, would require the Company to settle the 2017 Capped Calls in shares. On September 16, 2024, the Company entered into supplements with each of the counterparties to modify the terms of each of the 2017 Capped Calls to require settlement in cash rather than in shares. As a result of the modification, the Company reclassified the fair value of the 2017 Capped Calls of $43.9 million from stockholders’ equity to derivative assets. As a result of the conversion, the Company ultimately received $45.3 million in cash to settle the remaining 2017 Capped Callson November 15, 2024. The change in the fair value of the derivative assets from September 16, 2024 to Maturity Date was a gain of $1.5 million and recorded to other (expense) income, net in the consolidated statements of comprehensive (loss) income. There wereno derivative assets remaining as of December 31, 2025 or 2024.

Total Debt Obligations

As of December 31, 2025 and 2024, the Company recorded $829.0 million and $1,137.1 million as debt on the consolidated balance sheets, respectively. As of December 31, 2025, all debt was long-term in nature.

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

For the years ended December 31, 2025, 2024 and 2023, the Company recorded $34.8 million, $20.6 million and $22.0 million of interest expense from outstanding debt facilities, respectively. Interest expense from outstanding debt facilities for the years ended December 31, 2025, 2024 and 2023 is inclusive of $12.1 million, $5.0 million and $5.2 million of amortization of debt discounts, respectively. The fair values of the 2027 Notes and 2030 Notes are based on open market trades and are 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:

14. 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 13, 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.

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 year ended December 31, 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 December 31, 2025, the remaining amount authorized under the 2024 Repurchase Program was $475.0 million. There wereno share repurchases of common stock during the year ended December 31, 2024.

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 from the date of repurchase.

15. STOCK-BASED COMPENSATION

Equity Incentive Plan

In June 2018, the Company’s stockholders approved the 2018 Equity Incentive Plan (the “2018 Plan”). The 2018 Plan allows for the grant of stock options, stock appreciation rights (“SARs”), restricted stock awards (“RSAs”), restricted stock units ("RSUs"), performance shares ("PSAs") and performance units ("PSUs"). The 2018 Plan initially authorized 2.9 million shares of common stock to be issued and, in subsequent years, an aggregate 13.1 million shares of common stock were approved by the Company’s stockholders and added to the 2018 Plan. As of December 31, 2025, together with the roll-over shares from the Company’s 2011 Equity Incentive Plan,1.8 million shares of common stock remain available for future grant under the 2018 Plan.

In March 2024, the Company initiated the 2024 Employment Commencement Incentive Plan (the “2024 Plan”). The 2024 Plan, which authorized 0.6 million shares of common stock to be issued, allows for the grant of stock options, SARs, RSAs, RSUs, PSAs and PSUs. In June 2024, there was an addition of 0.5 million shares to the 2024 Plan. As of December 31, 2025,2.3 million shares of common stock remain available for future grant under the 2024 Plan.

In June 2013, the Company’s stockholders approved the 2013 Employee Stock Purchase Plan (the “2013 ESPP”) which authorized 0.3 million shares of common stock available to be issued. The 2013 ESPP was amended in 2016 and renamed as the 2016 ESPP. In subsequent years, the Company’s stockholders approved an aggregate of 1.4 million shares of common stock available for issuance under the 2016 ESPP. In August 2025, the Company suspended its 2016 ESPP as a result of increased volatility in the Company's stock price (the "ESPP Suspension") and, as a result, the 2016 ESPP is no longer active for future grants.

Stock Options

In general, stock options have aten-year term and vest over afour-year period, with one-fourth of the underlying shares vesting on the first anniversary of the grant and 1/48th of the underlying shares vesting monthly thereafter, such that the underlying shares will be fully vested on the fourth anniversary of the grant, subject to the terms of the applicable plan under which they were granted.

The fair values of stock options granted during the periods presented are measured on the date of grant using the Black-Scholes-Merton option-pricing model, with the following assumptions:

(1)

The risk-free interest rate is estimated using an average of Treasury bill interest rates over a historical period commensurate with the expected term of the option that correlates to the prevailing interest rates at the time of grant.

(2)

The expected dividend yield iszero as the Company has not paid any dividends to date and does not expect to pay dividends in the future.

(3)

The expected term is estimated using historical exercise behavior.

(4)

The expected volatility is the implied volatility in exchange-traded options of the Company’s common stock.

The amounts estimated according to the Black-Scholes-Merton option-pricing model may not be indicative of the actual values realized upon the exercise of these options by the holders.

The following table summarizes the Company’s stock option activity for the period indicated:

The weighted-average grant date fair value per share of stock options granted during the years ended December 31, 2025, 2024 and 2023 was $46.93, $67.31 and $70.94, respectively.

The following table summarizes the Company’s shares vested and stock options exercised for each of the periods indicated:

CEO Option Awards with Market Conditions

The Company granted its CEO 3,300,000 options with service and market conditions which were subject to a five-year cliff vesting schedule in July 2017 and, subsequently in April 2022, modified the vesting conditions of these options. The modification resulted in recognition of an aggregate incremental cost of $123.3 million, which was fully expensed by December 31, 2023. As of December 31, 2025,2.2 million were vested and the remaining 1.1 million did not vest as the market conditions were not met by June 26, 2025.

Excluding the options with market and service conditions granted to the Company’s CEO, the remaining stock options granted during the periods presented in the table have only service-based criteria and vest over four years.

Restricted Stock Units

The Company grants RSUs to members of its board of directors and employees. In general, RSUs vest over four years, with one-fourth of the shares vesting on an annual basis, such that RSUs become fully vested at the fourth anniversary of their respective grant dates.

CEO Awards

On December 7, 2025, the Company granted its CEO 269,542 shares of time-based RSUs ("CEO Time-based Awards") with an aggregate grant date fair value of $6.0 million. These awards vest annually over the three-year period measured from the date of grant subject to the CEO's continued provision of services to the Company. The CEO Time-based Awards will be expensed on a straight-line basis through their attribution period.

Additionally, the Company granted its CEO 123,001 shares of PSUs and 146,541 shares of PSAs (collectively, "CEO Performance Awards") with an aggregate grant date fair value of approximately $2.7 million and $3.3 million, respectively. The CEO Performance Awards have a three-year cliff vesting period and are eligible to be earned based on the achievementof a certain financial performance goal over themeasurement period between January 1, 2026 and December 31, 2027. Dependent upon the level ofperformance achieved, the Company's CEO may be eligible to earn up to 200% of the target value of the CEO Performance Awards. The CEO Performance Awards will become fully vested on December 7, 2028, subject to the Company's CEO's continued provision of services to the Company over such period. When the applicable performance condition is considered probable of being achieved, the corresponding portion of the CEO Performance Awards will be expensed.

The following table summarizes the Company’s RSU activity for the period indicated:

Approximately 3.4 million and 0.1 million RSUs granted in the year ended December 31, 2025 vest over two and three years, respectively. The remaining RSUs vest over four years. The weighted-average grant date fair value of RSUs granted during the years ended December 31, 2024 and 2023 was$129.31 and$151.20, respectively. The fair values of RSUs vested during the years ended December 31, 2025, 2024 and 2023 totaled $82.9 million, $86.9 million and $82.6 million, respectively.

Restricted Stock Units with Performance Conditions

The Company grants PSUs to members of its board of directors and employees. The following table summarizes the Company’s PSU activity for the period indicated:

During the year ended December 31, 2025, the Company granted the following PSUs:

•

In March 2025, the Company granted 120,378 PSUs ("March 2025 PSUs") whose performance conditions 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.

•

In September 2025, the Company granted 225,000 PSUs ("September 2025 PSUs") whose performance condition is related to the achievement of a certain financial performance goal.

•

In December 2025, the Company granted its CEO 123,001 PSUs ("December 2025 PSUs") whose performance condition is related to the achievement of certain financial performance goals.

As of December 31, 2025, the following PSUs became vested or eligible for vesting:

•

Achievement of certain financial performance targets during the year ended December 31, 2025 resulted in 33,215 shares of the PSUs granted in March 2024 becoming eligible for vesting, which is contingent on the fulfillment of remaining service conditions.

•

Achievement of the financial performance goal during the year ended December 31, 2025 resulted in 225,000 shares of the September 2025 PSUs becoming eligible for vesting, which is contingent on the fulfillment of the remaining service condition.

For the year ended December 31, 2025, the Company recorded approximately $12.2 million in stock-based compensation expense related to all PSUs, including PSUs vested in 2025 as well as PSUs outstanding at year-end. As of December 31, 2025, none of performance conditions associated with the remaining PSUs were probable of being achieved. Should these performance conditions become probable of being achieved, the Company will be required to record up to approximately $14.3 million in stock-based compensation expense.

Restricted Stock Awards with Performance Conditions

The Company may grant PSAs to members of its board of directors and employees. The following table summarizes the Company’s PSA activity for the period indicated:

The Company granted its CEO 146,541 PSAs whose performance condition is related to the achievement of a certain financial performance goal.

2016 Employee Stock Purchase Plan

Under the Company’s 2016 ESPP, participating employees purchase common stock through payroll deductions. The purchase price is equal to 85% of the lower ofthe closing price of the Company’s common stock on the first business day and the last business day of the relevant purchase period. In August 2025, the Company suspended its 2016 ESPP. As a result, the 2016 ESPP is no longer active for future grants. The following table summarizes the Company’s ESPP activity for each of the periods indicated:

Stock-based Compensation Expense

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

*Prior to the year ended December 31, 2024, capitalized stock-based compensation costs were not material.

As of December 31, 2025, there was $218.0 million of total unrecognized stock-based compensation expense related to the Company’s stock-based compensation plans, including estimated forfeitures. The expense is expected to be recognized over a weighted-average period of approximatelyone years. Of this amount, $33.7 million relates to options with service conditions only, $14.3 million relates to PSUs with certain performance conditions not met, $3.2 million relates to PSAs with certain performance conditions not met and the remaining $166.8 million relates to restricted stock units with service conditions only.

16. 401(K) PLAN

The Company sponsors a 401(k) Plan in the U.S. and other retirement plans (“the Plan”) in the rest of the world, all of which are defined contribution plans. The Plan is available to all employees who are age 21 or older. Participants may make voluntary contributions and the Company makes matching contributions according to the Plan’s matching formula. Matching contributions fully vest afterone year of service for all employees. The expense related to the Plan primarily consists of the Company’s matching contributions.

Expense related to the Plan totaled $9.4 million, $9.8 million and $8.8 million for the years ended December 31, 2025, 2024 and 2023, respectively.

17. OTHER (LOSS) INCOME, NET

The following table summar izes other (loss) income, net for the years indicated:

• Related to the change in fair value of the contingent consideration derivative liabilities accounted for as derivatives under ASC 815 prior to the adoption of ASU 2025-07 and the 2017 Capped Calls derivative assets. For more information, please read Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncementsand Note 13, Indebtedness.

18. INCOME TAXES

The following table summarizes the (loss) income before the (benefit) provision for income taxes by jurisdiction for the periods indicated:

The following table summarizes the (benefit) provision for income taxes in the accompanying consolidated financial statements for the periods indicated:

The following table summarizes the reconciliation between the Company’s effective tax rate and the statutory income tax rate for each of the periods indicated:

*During the year ended December 31, 2025, state taxes in California, Michigan and Minnesota comprise the majority of this

category. During the years ended December 31, 2024 and 2023, state taxes in Illinois comprised the majority of this category.

The significant items impacting the Company’s effective tax rate for each of the periods primarily includes state taxes, research and development tax credits, valuation allowance, stock-based compensation and foreign rate differential. The statutory income tax rate differential is driven by the Company’s foreign subsidiary, Sarepta Therapeutics Ireland LP, an Irish partnership that has elected to be treated as a corporation for US tax purposes. For the year ended December 31, 2023, the Company's effective tax rate was also significantly impacted by the non-deductible premium paid on the 2024 Notes Exchange.

The Tax Cuts and Jobs Act of 2017 required taxpayers to capitalize research and development costs for tax purposes. This resulted in the Company having taxable profits in 2024 and 2023 and recording federal and state tax expense of $24.8 million and $15.3 million, respectively. The state tax expense is primarily related to the temporary suspension of utilizing net operating loss carryforwards in certain states the Company operates in.

The following table summarizes the deferred tax assets and liabilities for each of the periods indicated:

On July 4, 2025, the U.S. enacted H.R.1, titled “An Act to Provide for Reconciliation Pursuant to Title II of H. Con. Res. 14”, commonly referred to as the One Big Beautiful Bill Act (the OBBBA). The OBBBA includes a broad range of tax reform provisions and extends or modifies several provisions originally enacted under the Tax Cuts and Jobs Act of 2017. Notably, the legislation provides for permanent expensing of U.S. qualifying research and development expenses paid or incurred in tax years beginning after 2024 and allows taxpayers to elect to accelerate the deduction of previously capitalized U.S. research and development costs over a one or two year period. For the year ended December 31, 2025, the favorable research and development provisions reduced taxable income and current tax expense. These provisions did not expected impact the Company’s deferred tax expense as a result of the valuation allowance maintained against its U.S. net deferred tax assets.

The Company has evaluated the positive and negative evidence bearing upon the realizability of its U.S. net deferred tax assets, which are comprised principally of federal and state net operating loss carryforwards, research and development tax credit carryforwards, capitalized research and development costs, stock-based compensation expense, debt discount, and intangibles. Under the applicable accounting standards, management has considered the Company’s history of losses and concluded that it is more likely than not that the Company will not recognize the benefits of its net federal and state deferred tax assets. Accordingly, a full valuation allowance against the U.S. net deferred tax asset is maintained at December 31, 2025 and 2024. 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. The net change in the valuation allowance for deferred tax assets was an increase of $218.6 million and $4.7 million for the years ended December 31, 2025 and 2024, respectively. This increase for the year ended December 31, 2025 was primarily due to the capitalization of license fees, increase of federal and state net operating losses and federal and state tax credits recorded in the current year.

The Company generated foreign deferred tax assets mainly consisting of net operating loss carryforwards, stock-based compensation and unrealized gain/losses. Based upon the income projections in the majority of the foreign jurisdictions, the Company believes it will realize the benefit of its future deductible differences in these jurisdictions. As such, the Company has not recorded a valuation allowance against the net deferred tax assets in these foreign jurisdictions. Brazil and the Netherlands have generated deferred tax assets, which consist primarily of net operating loss carryforwards. The Company has concluded that it is more likely than not that it will not recognize the future benefits of the deferred tax assets in these jurisdictions, and accordingly, a full valuation allowance has been recorded against these foreign deferred tax assets.

As of December 31, 2025, the Company had federal and state net operating loss carryforwardsof $200.8 million and $376.9 million, respectively, available to reduce future taxable income. Federal and state net operating loss carry forwards of $68.9 million and $374.3 million will expire at various dates between 2026 and 2045. Federal and state net operating loss carryforwards of $131.9 million and $2.6 million, respectively, can be carried forward indefinitely. Utilization of these net operating losses could be limited under Section 382 of the Internal Revenue Code and similar state laws based on historical or future ownership changes and the value of the Company’s stock. Additionally, the Company has $59.0 million and $55.9 millionof federal and state research and development credits, respectively, and $308.4 million of federal orphan drug tax credits available to offset future tax liabilities. These federal and state research and development credits expire between 2034 and 2045 and between 2031 and 2040, respectively. The federal orphan drug credits expire between 2034 and 2045. The Company also has foreign net operating loss carryforwards of $15.7 million, mainly derived from the net operating loss generated by its subsidiary in Brazil, which may be carried forward indefinitely.

The Company, or one of its subsidiaries, files income tax returns in the U.S., and various state and foreign jurisdictions. The federal, state and foreign income tax returns are generally subject to tax examinations for the tax years ended December 31, 2022 through December 31, 2025. To the extent the Company has tax attribute carryforwards, the tax years in which the attribute was generated may still be adjusted upon examination by the Internal Revenue Service, state or foreign tax authorities to the extent utilized in a future period.

The following table summarizes the reconciliation of the beginning and ending amount of total unrecognized tax benefits for each of the periods indicated:

The balance of total unrecognized tax benefits at December 31, 2025, if recognized, would not affect the effective tax rate on income from continuing operations, due to a full valuation allowance against the Company’s U.S. deferred tax assets. The Company’s policy is to recognize interest and/or penalties related to unrecognized tax benefits in income tax expense. It hadno accrual for interest or penalties on its consolidated balance sheets at December 31, 2025 or 2024. No interest and/or penalties for unrecognized tax benefits were recognized in 2025, 2024, or 2023.

The Company’s intent is to only make distributions from non-U.S. subsidiaries in the future when they can be made at no net tax cost. Otherwise, the Company considers all of its foreign earnings to be permanently reinvested outside of the U.S. and has no plans to repatriate these foreign earnings to the U.S. The Company has no material unremitted earnings from its non-U.S. subsidiaries.

The following table summarizes the income taxes paid (net of refunds) for the periods indicated:

*The amount of taxes paid during the year does not meet the 5% disaggregation threshold.

19. LEASES

The Company has real estate operating leases in Cambridge, Andover and Bedford, Massachusetts and Columbus, Ohio that provide for scheduled annual rent increases throughout each lease’s term. The Company has also identified leases embedded in certain of its manufacturing and supply agreements as the Company determined that it controls the use of the facilities and related equipment therein. For more information related to the leases embedded in manufacturing and supply agreements with Catalent, Inc. (“Catalent”), please refer to Note 23, Commitments and Contingencies.

Bedford, Massachusetts

On April 22, 2022, the Company entered into a lease agreement (the “Bedford Lease”) for 288,000 square feet of to-be-constructed research and development and manufacturing space in Bedford, Massachusetts. The term of the Bedford Lease commences upon the landlord’s completion of the initial construction of the core and shell of the building, at which time the Company will obtain control of the premises and commence internal construction activities. The initial term of the Bedford Lease is anticipated to terminate on December 31, 2038. The Company has two options to extend the lease for a period often years each, exercisable under certain conditions and at a market rate determined in accordance with the lease agreement. The lease commenced in May 2023 as the Company obtained control of the premises.

The Bedford Lease provides for a tenant improvement allowance from the landlord of $72.0 million to be used towards costs incurred by the Company in the design and construction of the premises, which was recorded as a reduction to ROU assets and lease liabilities. In August 2024, the Company entered into an amendment to the Bedford Lease to extend the term in which the tenant improvement allowance could be reimbursed from the landlord (the “Bedford Amendment”) through December 2, 2025. The Company also agreed to reimburse the landlord for certain costs incurred in order to fund the extension of the reimbursement period associated with the tenant improvement allowance, totaling $2.9 million. The Bedford Amendment was accounted for as a modification to the Bedford Lease.

In May 2022, in connection with the execution of the Bedford Lease, the Company issued a letter of credit collateralized by cash deposits of $8.4 million, which was included in other non-current assets of the Company’s consolidated balance sheets. Such letter of credit shall be reduced to $5.6 million at the commencement of the fourth rent year, provided certain conditions set forth in the Bedford Lease are satisfied. Additionally, the Company is responsible for reimbursing the landlord for the Company’s share of the property’s operating expenses and property taxes.

The Company had a lease liability and ROU asset of $168.9 million and $89.7 million, respectively, on the consolidated balance sheets as of December 31, 2025 related to the Bedford Lease. Tenant improvement costs incurred by the Company that have been reimbursed by the landlord total $69.8 million as of December 31, 2025 and are recorded as an increase to the lease liability within the Company's consolidated balance sheets.

Columbus, Ohio

In December 2018, the Company entered into a lease agreement for a research and development facility in Columbus, Ohio, which was subsequently amended in May 2022 (the “Columbus Amendment,” together with the Columbus Amendment, the lease agreement is referred to as the “Columbus Lease”). The Columbus Lease expands from its current form of approximately 78,000 square feet to 167,000 square feet through a series of expansion spaces commencing at various periods through January 1, 2025.

Each expansion space commences on the date when the landlord will deliver control of that space for the Company to carry out design and construction activities (the “Columbus Commencement Date”). The Company is obligated to pay rent on each expansion space nine months after the Columbus Commencement Date. The Columbus Lease expires on December 31, 2036, and the Company has options to extend the lease byfive years in both 2036 and 2041. Each option is exercisable under certain conditions and at a market rate determined in accordance with the lease agreement.

The Company commenced design and construction activities on areas of the premises of approximately 18,000 square feet (the “Second Expansion Space”),36,000 square feet (the “Initial Expansion Space”),19,000 square feet (the “Third Expansion Space”) and 16,000 square feet (the“Fourth Expansion Space”) on June 1, 2022, October 1, 2022, September 1, 2023 and January 1, 2025, respectively. As a result, it was determined that the lease related to the Second Expansion Space, the Initial Expansion Space, the Third Expansion Space and the Fourth Expansion Space had commenced on those four dates, respectively. The total ROU asset and lease liability associated with the Columbus Lease, inclusive of the Fourth Expansion Space, Third Expansion Space, the Second Expansion Space and the Initial Expansion Space, was $12.9 million and $21.2 million, respectively, as of December 31, 2025.

Lease Obligations

As of December 31, 2025, ROU assets for operating leases were $125.5 million and operating lease liabilities were $210.6 million. The following table contains a summary of the lease costs recognized and other information pertaining to the Company’s operating leases for the periods indicated:

The following table summarizes maturities of lease liabilities and the reconciliation of lease liabilities as of December 31, 2025:

20. (LOSS) EARNINGS PER 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 years ended December 31, 2025 and 2023, 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 December 31, 2025, the anti-dilutive common stock issuable under the Company's equity incentive plans includes 0.2 million shares and awards that are dilutive but have performance conditions that were not met as of the end of the period. These were anti-dilutive as the Company was in a net loss position at the end of the period.

(2) As of December 31, 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.

(3) As of December 31, 2023, the anti-dilutive common stock issuable under the Company's equity incentive plans includes 1.1 million shares that have performance or market conditions that were not met. These were anti-dilutive as the Company was in a net loss position at the end of the period.

21. SEGMENT INFORMATION

The Company, together with its wholly-owned subsidiaries, is a commercial-stage biopharmaceutical company focused on helping patients through the discovery and development of unique RNA-targeted therapeutics, siRNA platform, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. The Company’s research and development organization is responsible for the research and discovery of new product candidates and supports development and registration efforts for potential future products. The Company’s supply chain organization manages the development of the manufacturing processes, clinical trial supply and commercial product supply. The Company’s commercial organization is responsible for worldwide commercialization of EXONDYS 51, VYONDYS 53 and AMONDYS 45 and domestic commercialization of ELEVIDYS. The Company is supported by other back-office general and administration functions. Consistent with this decision-making process, the Company’s CEO, or the chief operating decision maker (the “CODM”), uses consolidated, single-segment financial information for purposes of evaluating performance, forecasting future period financial results, allocating resources and setting incentive targets.

The Company operates inone segment: discovering, developing, manufacturing and delivering therapies to patients with rare diseases. The Company’s reportable segment derives its revenues from sales of its products, which include the PMO Products and ELEVIDYS, as well as through collaboration and other revenues primarily generated from its collaboration arrangement with Roche and other revenues related to the sale of commercial ELEVIDYS supply to Roche and royalty revenue from Roche. The CODM manages and allocates resources to the operations of the Company on a total company basis by assessing the overall level of resources available and how to best deploy these resources across functions and in line with the Company's strategic goals.

The measure of segment profit or loss that the CODM uses to allocate resources and assess performance 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 (loss) income 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 (gain) loss on debt extinguishment, loss (gain) on strategic investments, interest expense, interest income, income tax 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 consolidated balance sheets.

22. GAIN FROM SALE OF PRIORITY REVIEW VOUCHER

In June 2023, the Company entered into an agreement to sell the rare pediatric disease Priority Review Voucher (the “ELEVIDYS PRV”) it received from the FDA in connection with the approval of ELEVIDYS for consideration of $102.0 million, with no commission costs. The closing of the transaction was not subject to the conditions set forth under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, and closed in June 2023. The net proceeds were recorded as a gain from sale of the ELEVIDYS PRV during the year ended December 31, 2023, as it did not have a carrying value at the time of the sale.

23. COMMITMENTS AND CONTINGENCIES

Manufacturing Obligations

The Company has entered into long-term contractual arrangements from time to time for the provision of goods and services. The following table presents non-cancelable contractual obligations arising from long-term contractual arrangements, including obligations related to leases embedded in certain supply agreements:

*Total manufacturing commitments include the Catalent Agreement (defined below), for which the Company determined includes a lease embedded in it and, as such, has ROU assets and lease liabilities recorded on the consolidated balance sheets as of December 31, 2025.

**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.

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 and third amendments in May 2019, July 2020 and October 2021, respectively, with Brammer Bio MA, LLC, an affiliate of Thermo Fisher Scientific, Inc. (“Thermo”) (collectively, the “Thermo Agreement”).

In October 2021, the Company executed a third amendment (the “Third Amendment”) that modified the terms of the Thermo Agreements, which significantly decreased the Company’s right of use of the facility’s capacity and reduced the fixed and in-substance fixed payments due over the remaining term of the agreement. Under the Third Amendment, the Company has committed to guaranteed purchases under the Third Amendment on a take-or-pay basis regardless of whether services or goods are ordered.

In March 2023, the Company executed a fourth amendment (the “Fourth Amendment”) that modified the terms of the Thermo Agreement. The Fourth Amendment removed the previous minimum batch purchase commitment and associated fee for the remaining term of the Thermo Agreement and instead implemented a fee of up to $60.0 million, to be paid in three installments of $20.0 million each by March 1, 2024, December 31, 2024 and December 31, 2025, respectively. During the year ended December 31, 2024, the Company paid the first $20.0 million installment due March 1, 2024, considered a nonrefundable advance payment.

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. The aggregate net impact of the termination was $55.4 million of research and development expense in the accompanying consolidated statements of comprehensive (loss) income. Asthe Company had yet to obtain regulatory approval to produce commercial supply of ELEVIDYS at Thermo manufacturing facilities, the Company recorded the charges incurred as research and development expenses during the year ended December 31, 2024. Included in the net impact noted above are non-cash charges due to the termination of $62.7 million, related to the remaining unamortized nonrefundable advance payments. Additionally, there were unbilled service and material costs ordered by the Company as of the termination date of $29.2 million that were expensed as research and development expense during the year ended December 31, 2024. As related to the termination, costs reimbursable by Roche under the Roche Collaboration Agreement and reflected as a reduction to research and development expenses were $36.5 million.

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 consolidated statements of comprehe nsive (loss) income within research and development expense for the year ended December 31, 2025.

Catalent, Inc.

The Company entered into a manufacturing collaboration agreement and, subsequently, a manufacturing and supply agreement with Catalent, formerly Paragon Biosciences, Inc. in October 2018 and February 2019, respectively (collectively, the “Catalent Agreements”). Pursuant to the terms of the Catalent Agreements, Catalent agreed to provide the Company withtwo dedicated clean room suites and an option to reservetwo additional clean room suites for its gene therapy programs, subject to certain minimum and maximum volume limitations. In September 2019, the Company exercised the option to gain access to the two additional clean room suites. The Company determined that the Catalent Agreements contained a lease because the Company had the right to direct the use of the facility and related equipment therein. The lease on allfour dedicated clean room suites at Catalent commenced during 2020, which is when the dedicated clean room suites became available for use by the Company. In March 2021, the Company modified the terms of the Catalent Agreements, which decreased the Company’s right of use of certain dedicated clean room suites. As of December 31, 2025, the Company controls two clean room suites.

In November 2022, the Company modified certain terms of the Catalent Agreements which extended the term of the agreement through December 31, 2028, which represented a modification of the existing embedded lease over certain clean room suites. The modification resulted in the recognition of additional ROU assets and lease liabilities of $19.2 million, as well as reclassification of $3.9 million between long-term and short-term manufacturing deposits. The modification also removed certain fixed payments due over the remaining term of the agreement. Further, in order to maintain the Company's dedicated clean room suites, it has committed to guaranteed purchases under the amendment on a take-or-pay basis regardless of whether services or goods are ordered.

In October 2025, the Company notified Catalent of its intention to release Catalent from its obligation to dedicate certain clean room suites to the Company, effective October 2026 (the "Dedicated Suites Release"). The Dedicated Suites Release shortened the embedded lease term for those suites and resulted in the derecognition of ROU assets and lease liabilities of $12.3 million, as the related balances were remeasured over the remaining embedded lease term. The Dedicated Suites Release did not change any other terms of the Catalent Agreements.

The Company has the ability to terminate the Catalent Agreements prior to expiration, subject to the payment of additional financial consideration. As of December 31, 2025, the Company believes it is probable that the guaranteed purchase requirements will be met in the normal course of business throughout the term of the Catalent Agreements.

On August 18, 2025 (the “Effective Date”), the Company entered into a supplemental letter agreement (the “Letter Agreement”) with Catalent in connection with 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 and $62.6 million for raw materials associated with the Company's binding payment for the first and second quarter of 2026, respectively. As of December 31, 2025, $40.7 million was classified as manufacturing related deposits and prepaids and $83.7 million was classified as other non-current assets in the consolidated balance sheets, reflecting the expected consumption of these raw materials. The Company and Catalent finalized the outstanding balance related to certain unbilled raw materials associated with the Letter Agreement as of December 31, 2025, resulting in $33.1 million of previously unbilled raw materials being recorded as accrued expenses in the consolidated balances sheets.

(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)

agreed to 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 $23.0 million within costof sales in its consolidated statements of comprehensive (loss) income for the year ended December 31, 2025.

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 consolidated statements of comprehensive (loss) income forthe year ended December 31, 2025.

Aldevron, LLC

The Company entered into a clinical and commercial supply agreement in December 2018, as subsequently amended in June 2020, with Aldevron LLC (“Aldevron”) for the supply of plasmid DNA to fulfill its needs for gene therapy clinical trials and commercial supply (collectively, the “Aldevron Agreements”). Pursuant to the terms of the Aldevron Agreements, Aldevron agreed to reserve a certain amount of manufacturing capacity on a quarterly basis. In return, the Company is required to make advance payments to Aldevron related to the manufacturing capacity.

In January 2025, the Company modified certain terms of the Aldevron Agreements which extended the term of the agreement through December 31, 2028 (the “Aldevron Amendment”). As a result of the Aldevron Amendment, the Company has the option to extend the term of the Aldevron Agreements by one year if the Company delivers a written notice of its intention to extend to Aldevron no later than June 1, 2028. Both parties have the right to early terminate without additional penalty. The Company has determined that the Aldevron Amendment does not contain an embedded lease because it does not convey the right to control the use of Aldevron’s facility or related equipment therein.

Euroapi Germany GmbH

On October 24, 2025, the Company entered into a side letter agreement (the “Euroapi Side Letter”) with Euroapi Germany GmbH (“Euroapi”) in connection with the Company’s manufacturing and supply agreement entered into in October 2020 and as amended in December 2023 and November 2024, respectively (collectively, the “Euroapi Agreement”). The Euroapi Side Letter suspends certain contractual obligations under the Euroapi Agreement. Specifically, manufacturing of certain raw materials associated with the PMO Products will be paused during the term of the side letter.

The Company agreed to pay approximately$10.4 millionto satisfy the remainder of the Company’s 2025 purchase obligations under the Euroapi Agreement. The Company and Euroapi may mutu ally agree to reinstate commercial supply for 2026, which would otherwise require minimum purchase commitments of approximately $15.1 million. Based on its assessment as of December 31, 2025, the Company determined it was not probable that commercial supply will resume in 2026. Accordingly, the Company recorded $15.1 million related to 2026 minimum purchase commitments as cost of sales in the accompanying consolidated statements of comprehensive (loss) income for the year ended December 31, 2025, with the outstanding liability reflected as accrued contract manufacturing costs within accrued expenses in the consolidated balance sheets as of December 31, 2025.

As a result of the Euroapi Side Letter, the total impact to cost of sales for the year ended December 31, 2025 was approximately $25.5 million, representing $10.4 million and $15.1 million for 2025 purchase obligations and 2026 minimum purchase commitments, respectively.

Other Funding Commitments

The Company has several on-going clinical trials in various clinical trial stages. Its most significant clinical trial expenditures are to contract research organizations (“CROs”). The CRO contracts are generally cancellable at the Company’s option. As of December 31, 2025, the Company had approximately $404.3 million in cancellable future commitments based on existing CRO contracts. For the years ended December 31, 2025, 2024 and 2023, the Company recognized $89.8 million,$110.1 million and$112.2 million, respectively, for expenditures incurred by CROs.

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 December 31, 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 (including 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 appealed to the U.S. Court of Appeals for the Federal Circuit (the “Federal Circuit”), which reversed the district court's decision on February 20, 2026, and remanded the case for further proceedings.

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 forinter partes review ("IPR") for filing with the Patent Trial and Appeal Board (“PTAB”) at the U.S. Patent and Trademark Office (“USPTO”). The petition sought 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 IPR proceedings. On August 22, 2024, the PTAB instituted 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 IPR 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, and the appeal is pending.

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 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 million in 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 amotion 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 treble damages, 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 submitted IPR challenging the validity of the ’542 and ’721 Patents, for which the PTAB discretionarily denied institution in November 2025. In November and December 2025, Sarepta submitted IPR petitions challenging the validity of the ’894 Patent, ’326 Patent, ’377 Patent, ’880 Patent, and ’313 Patent. In February 2026, Genzyme filed motions for discretionary denial in all five IPR proceedings. Sarepta subsequently refiled the IPR petition challenging the ‘313 Patent and moved to withdraw the original petition. 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 sought 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, which the court granted on November 6, 2025. On January 22, 2026, the lead plaintiffs filed an amended complaint including a new plaintiff, removing Dallan Murray as a defendant, adding President and Chief Operating Officer Ian Estepan as a defendant, and expanding the class period to shareholders who purchased or otherwise acquired the Company’s securities between June 22, 2023 and November 3, 2025 (the “Amended Complaint”). The Amended Complaint further alleges additional theories of liability, including that Sarepta and the named defendants made false or misleading public statements related to (i) the safety profile of ELEVIDYS and LGMD therapies that utilize the AAVrh74 viral vector; (ii) the status of the development of LGMD therapies; and (iii) the status of the ESSENCE trial. Sarepta intends to file a motion to dismiss the Amended Complaint, which is currently due on March 9, 2026.

On July 15, 2025, a shareholder derivative lawsuit concerning certain of the same 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 certain of the same 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 Actions seek 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”). On November 17, 2025, the court stayed the Consolidated Derivative Action until the Securities Action is resolved.

24. QUARTERLY FINANCIAL DATA (UNAUDITED)

The following tables present the Company’s summarized quarterly financial information for the years ended December 31, 2025 and 2024, including revisions to prior-period amounts as discussed in Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncements. Management believes the information reflects all normal recurring adjustments necessary for a fair presentation. Quarterly results may not be indicative of results for future periods.

*The quarterly financial data for the quarter ended September 30, 2025 has been revised to reflect an immaterial error correction related to the August 2025 Exchange and to reflect the adoption of ASU 2025-07. Refer to Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncementsfor further discussion of the error corrections related to the August 2025 Exchange and the adoption of ASU 2025-07.