Sarepta Therapeutics, Inc. (SRPT)

CIK: 0000873303. SIC: 2834 Pharmaceutical Preparations. Latest 10-K as of: 2026-03-02.

SIC breadcrumb: Manufacturing > Chemicals And Allied Products > SIC 2834 Pharmaceutical Preparations

SEC company page: https://www.sec.gov/edgar/browse/?CIK=873303. Latest filing source: 0001193125-26-084144.

Selected Fundamentals

Financials

Annual standardized facts from SEC companyfacts as of latest extracted filing date 2026-03-02. Source: https://data.sec.gov/api/xbrl/companyfacts/CIK0000873303.json. Derived margins are computed from the extracted annual SEC facts.

Flow metrics use full-year FY periods from 10-K/10-K/A filings; balance-sheet metrics use FY-end instants. Missing metrics are omitted rather than fabricated.

Financial Charts

Quarterly

Quarterly standardized facts from SEC companyfacts as of latest extracted filing date 2026-05-06. Source: https://data.sec.gov/api/xbrl/companyfacts/CIK0000873303.json.

Flow metrics use discrete quarter-length periods from 10-Q/10-Q/A filings. Q4 revenue and net income are derived only when annual FY and nine-month YTD facts exist for the same fiscal year; derived Q4 values are labeled. EPS Q4 is not derived.

Quarterly Charts

Macro Cross-References

• CPIAUCSL - Consumer Price Index for All Urban Consumers: All Items in U.S. City Average
• UNRATE - Unemployment Rate
• FEDFUNDS - Federal Funds Effective Rate
• CES0500000003 - Average Hourly Earnings of All Employees, Total Private
• DFEDTARU - Federal Funds Target Range - Upper Limit
• DFEDTARL - Federal Funds Target Range - Lower Limit
• DGS3MO - Market Yield on U.S. Treasury Securities at 3-Month Constant Maturity
• DGS2 - Market Yield on U.S. Treasury Securities at 2-Year Constant Maturity
• DGS10 - Market Yield on U.S. Treasury Securities at 10-Year Constant Maturity
• DGS30 - Market Yield on U.S. Treasury Securities at 30-Year Constant Maturity
• T10Y2Y - 10-Year Treasury Constant Maturity Minus 2-Year Treasury Constant Maturity
• CPILFESL - Consumer Price Index for All Urban Consumers: All Items Less Food and Energy
• CPIUFDSL - Consumer Price Index for All Urban Consumers: Food
• CPIENGSL - Consumer Price Index for All Urban Consumers: Energy
• CUSR0000SAH1 - Consumer Price Index for All Urban Consumers: Shelter
• PCEPI - Personal Consumption Expenditures: Chain-type Price Index
• PCEPILFE - Personal Consumption Expenditures Excluding Food and Energy: Chain-type Price Index
• PPIACO - Producer Price Index by Commodity: All Commodities
• T10YIE - 10-Year Breakeven Inflation Rate
• U6RATE - Total Unemployed, Plus All Marginally Attached Workers Plus Total Employed Part Time for Economic Reasons
• PAYEMS - All Employees, Total Nonfarm
• CIVPART - Labor Force Participation Rate
• EMRATIO - Employment-Population Ratio
• UNEMPLOY - Unemployed
• CE16OV - Employment Level
• ICSA - Initial Claims
• JTSJOL - Job Openings: Total Nonfarm
• JTSQUR - Quits: Total Nonfarm
• GDPC1 - Real Gross Domestic Product
• A191RL1Q225SBEA - Real Gross Domestic Product: Percent Change from Preceding Period
• INDPRO - Industrial Production: Total Index
• TCU - Capacity Utilization: Total Index
• HOUST - New Privately-Owned Housing Units Started: Total Units
• PERMIT - New Privately-Owned Housing Units Authorized in Permit-Issuing Places: Total Units
• RSAFS - Advance Retail Sales: Retail Trade
• PCE - Personal Consumption Expenditures
• DSPIC96 - Real Disposable Personal Income
• PSAVERT - Personal Saving Rate
• M2SL - M2
• BOPGSTB - U.S. International Trade in Goods and Services: Balance
• MSPUS - Median Sales Price of Houses Sold for the United States
• HSN1F - New One Family Houses Sold: United States
• RHORUSQ156N - Homeownership Rate in the United States
• TTLCONS - Total Construction Spending: Total Construction in the United States
• RRVRUSQ156N - Rental Vacancy Rate in the United States
• TOTALSL - Total Consumer Credit Owned and Securitized
• REVOLSL - Revolving Consumer Credit Owned and Securitized
• DRCCLACBS - Delinquency Rate on Credit Card Loans, All Commercial Banks
• GDP - Gross Domestic Product
• GPDI - Gross Private Domestic Investment
• GCE - Government Consumption Expenditures and Gross Investment
• PCEC - Personal Consumption Expenditures
• NETEXP - Net Exports of Goods and Services
• GFDEBTN - Federal Debt: Total Public Debt
• GFDEGDQ188S - Federal Debt: Total Public Debt as Percent of Gross Domestic Product
• FYFSD - Federal Surplus or Deficit
• FGRECPT - Federal Government Current Receipts
• FGEXPND - Federal Government: Current Expenditures
• MANEMP - All Employees, Manufacturing
• USCONS - All Employees, Construction
• USTRADE - All Employees, Retail Trade
• USFIRE - All Employees, Financial Activities
• USGOVT - All Employees, Government
• AWHAETP - Average Weekly Hours of All Employees, Total Private
• DGORDER - Manufacturers' New Orders: Durable Goods
• NEWORDER - Manufacturers' New Orders: Nondefense Capital Goods Excluding Aircraft
• BUSINV - Total Business Inventories
• EXPGS - Exports of Goods and Services
• IMPGS - Imports of Goods and Services
• IR - Import Price Index (End Use): All Commodities
• PPIFIS - Producer Price Index by Commodity: Final Demand

Latest quarter (10-Q)

Latest 10-Q source: 0001193125-26-208893.

Extracted between Part I Item 2 and the next Item 3/4 or Part II heading after HTML sanitization. Confidence: high. Filing date: 2026-05-06. Report date: 2026-03-31.

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

The purpose of Management's Discussion and Analysis of Financial Condition and Results of Operations is to provide an understanding of the financial condition, changes in financial condition and results of operations of Sarepta Therapeutics, Inc. This section should be read in conjunction with our unaudited condensed consolidated financial statements and related notes included in Part I, Item 1 of this Quarterly Report on Form 10-Q and the section contained in our Annual Report on Form 10-K for the year ended December 31, 2025 under the caption “Part II-Item 7 — Management’s Discussion and Analysis of Financial Condition and Results of Operations”. This Quarterly Report on Form 10-Q contains certain forward-looking statements, which are often identified by words such as “believe,” “anticipate,” “expect,” “intend,” “plan,” “will,” “may,” “estimate,” “could,” “continue,” “ongoing,” “predict,” “potential,” “likely,” “seek” and other similar expressions, as well as variations or negatives of these words. These statements relate to our future plans, objectives, expectations, intentions and financial performance and the assumptions that underlie these statements. These forward-looking statements include, but are not limited to:

•
our belief that our proprietary technology, technology platforms and collaborations can be used to develop potential therapeutic candidates to treat a broad range of diseases;

•
our expectation that our partnerships with manufacturers will support our clinical and commercial manufacturing capacity for our products and product candidates, including our PMO, gene therapy, SRP-9003 Limb-girdle muscular dystrophy (“LGMD”), and small interfering RNA (“siRNA”) programs, while also acting as a manufacturing platform for potential future programs, and our belief that our current network of manufacturing partners is able to fulfill the requirements of our commercial plan;

•
the possible impacts of the ELEVIDYS Suspension (as defined herein);

•
the possible impacts of the clinical hold the U.S. Food and Drug Administration (the “FDA") has placed on our investigational use gene therapy clinical trials for LGMD in July 2025 and the revocation of the platform technology designation for our AAVrh74 platform technology previously granted on June 2, 2025;

•
the possible impacts of the results of our ESSENCE confirmatory trial for VYONDYS and AMONDYS, including the timing and outcome of additional results, potential regulatory actions from the FDA, including directives to remove these products from the market or alter labels, patient demand for these products and changes to reimbursement and coverage by insurance companies;

•
the estimated and potential impacts of the strategic restructuring plan announced in July 2025 ("the Restructuring");

•
our expectation that our partnership with Catalent, Inc. (“Catalent”) will support our clinical and commercial manufacturing demand for our Duchenne gene therapy program and SRP-9003 LGMD program, while also acting as a manufacturing platform for any potential future gene therapy programs;

•
our expectation that Aldevron LLC (“Aldevron”) will provide Good Manufacturing Processes (“GMP”)-grade plasmid for our Duchenne muscular dystrophy (“Duchenne”) gene therapy program and our SRP-9003 LGMD program, as well as plasmid source material for future gene therapy programs;

•
the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business, including the addition of a boxed warning for acute liver injury ("ALI") and acute liver failure (“ALF”) and removal of the non-ambulatory population from the Indication and Usage section of the Prescribing Information for ELEVIDYS, as well as the development of our product candidates and our financial and contractual obligations;

•
estimated timelines and milestones for the remainder of 2026 and beyond, including discussions with the FDA regarding ELEVIDYS, VYONDYS 53, AMONDYS 45 and SRP-9003, and sharing data for certain of the Company's siRNA product candidates, SRP-1001 and SRP-1003;

•
our expectations regarding the ongoing study to evaluate the use of sirolimus as an enhanced immunosuppression regimen as part of treatment with ELEVIDYS for non-ambulant individuals living with Duchenne, including discussions with the FDA and possible impacts on the resumption of dosing in the non-ambulatory population;

•
our engagement with regulatory authorities outside of the U.S. including the European Medicines Agency (the “EMA”);

•
our plan to continue building out our network for commercial distribution in jurisdictions in which our products are approved or in which we are seeking approval for our products;

•
our plan to expand our pipeline through internal research and development and through strategic transactions;

•
the timely completion and satisfactory outcome of our post-marketing requirements and commitments, including verification of a clinical benefit for our products in confirmatory trials;

23

•
our ability to further secure long-term supply of our commercial products and our product candidates to satisfy our planned commercial, early access programs (“EAP”) and clinical needs;

•
the possible impact of any executive, legislative or regulatory action and competing products on the commercial success of our products and our product candidates and our ability to compete against such products;

•
our ability to enter into research, development or commercialization alliances with universities, hospitals, independent research centers, non-profit organizations, pharmaceutical and biotechnology companies and other entities for specific molecular targets or selected disease indications and our ability to selectively pursue opportunities to access certain intellectual property rights that complement our internal portfolio through license agreements or other arrangements;

•
our expectation regarding the potential benefits of the partnership, licensing and/or collaboration arrangements and other strategic arrangements and transactions we have entered into or may enter into in the future;

•
our plans and ability to file and progress to issue additional patent applications to enhance and protect our new and existing technologies and programs;

•
the potential benefits of our technologies and programs, including those with strategic partners;

•
our estimates regarding how long our currently available cash and cash equivalents will be sufficient to finance our operations and business plans and statements about our future capital needs;

•
our estimates regarding future revenues, research and development expenses, other expenses, capital requirements and payments to third parties;

•
our expectation regarding the impact of environmental laws and regulations on our business;

•
our expectation regarding the outcomes or impacts of our ongoing litigation that we are currently, or may in the future become, party to;

•
our expectation regarding our ability to satisfy the conditions to borrow under our Credit Agreement; and

•
our beliefs and expectations regarding milestone, royalty or other payments that could be due to third parties under existing agreements.

We undertake no obligation to update any of the forward-looking statements contained in this Quarterly Report on Form 10-Q after the date of this report, except as required by law or the rules and regulations of the U.S. Securities and Exchange Commission (the “SEC”). We caution readers not to place undue reliance on forward-looking statements. Our actual results could differ materially from those discussed in this Quarterly Report on Form 10-Q. The forward-looking statements contained in this Quarterly Report on Form 10-Q, and other written and oral forward-looking statements made by us from time to time, are subject to certain risks and uncertainties that could cause actual results to differ materially from those anticipated in the forward-looking statements, including the risks, uncertainties and assumptions identified under the heading “Risk Factors” in this Quarterly Report on Form 10-Q.

Overview

We are a commercial-stage biopharmaceutical company focused on helping patients through the discovery and development of unique RNA-targeted therapeutics, siRNA knockdown therapies, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. Applying our proprietary, differentiated and innovative technologies, and through collaborations with our strategic partners, we have developed multiple approved products for the treatment of Duchenne and are developing potential therapeutic candidates for a broad range of diseases and disorders, including LGMD. We are also developing potential therapeutic candidates through our partnered program with Arrowhead Pharmaceuticals, Inc. ("Arrowhead"), including Facioscapulohumeral muscular dystrophy ("FSHD"), myotonic dystrophy type 1 ("DM1"), Spinocerebellar ataxia ("SCA"), Idiopathic Pulmonary Fibrosis ("IPF"), and Huntington's disease.

To date, we have developed and commercialized four products that have been approved by the FDA:

•
The PMO Products:

o
EXONDYS 51 (eteplirsen) Injection ("EXONDYS 51"), granted accelerated approval by the FDA in 2016, is indicated for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping. EXONDYS 51 uses our phosphorodiamidate morpholino oligomer ("PMO") chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene.

o
VYONDYS 53 (golodirsen) Injection ("VYONDYS 53"), granted accelerated approval by the FDA in 2019, is indicated for the treatment of Duchenne in patients who have a confirmed mutation of the

24

dystrophin gene that is amenable to exon 53 skipping. VYONDYS 53 uses our PMO chemistry and exon-skipping technology to skip exon 53 of the dystrophin gene.

o
AMONDYS 45 (casimersen) Injection ("AMONDYS 45"), granted accelerated approval by the FDA in 2021, is indicated for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 45 skipping. AMONDYS 45 uses our PMO chemistry and exon-skipping technology to skip exon 45 of the dystrophin gene.

•
ELEVIDYS (delandistrogene moxeparvovec-rokl), an adeno-associated virus-("AAV") based gene therapy, received traditional approval 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. ELEVIDYS was also approved for non-ambulatory patients under the accelerated approval pathway in June 2024. ELEVIDYS was previously granted accelerated approval by the FDA in June 2023 for the treatment of ambulatory patients aged four through five years with Duchenne with a confirmed mutation in the Duchenne gene. ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the Duchenne gene. In response to safety events announced in March and June 2025, we suspended all shipments of ELEVIDYS to non-ambulatory patients in the U.S. in June 2025. In response to a request from the FDA that we voluntarily stop all shipments of ELEVIDYS in the U.S., we temporarily suspended all shipments of ELEVIDYS in the U.S., effective July 22, 2025, to allow us the necessary time to respond to the FDA's requests for information and complete a labeling supplement process (the "ELEVIDYS Suspension"). On July 28, 2025, the FDA informed us that it recommended the removal of the voluntary hold for ambulatory patients. On July 31, 2025, we resumed shipments of ELEVIDYS for ambulatory patients in the U.S. In November 2025, we announced a boxed warning for ALI and ALF and removal of non-ambulatory population from the Indication and Usage section of the Prescribing Information for ELEVIDYS.

We are in the process of conducting

[Excerpt truncated for page length; source filing is linked above.]

Latest 10-K MD&A

Extracted between Item 7 and the next Item 7A/8 heading after HTML sanitization. Confidence: high. Filing date: 2026-03-02. Report date: 2025-12-31.

Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations.

The purpose of Management's Discussion and Analysis of Financial Condition and Results of Operations is to provide an understanding of the financial condition, changes in financial condition and results of operations of Sarepta Therapeutics, Inc. The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our consolidated financial statements and related notes included elsewhere in this Annual Report on Form 10-K. This discussion contains forward-looking statements that involve risks and uncertainties. Please review our legend titled “Forward-Looking Information” at the beginning of this Annual Report on Form 10-K which is incorporated herein by reference. Our actual results could differ materially from those discussed below. Factors that could cause or contribute to such differences include, but are not limited to, those identified below, and those discussed in the section titled “Risk Factors” included elsewhere in this Annual Report on Form 10-K. Throughout this discussion, unless the context specifies or implies otherwise, the terms “Sarepta”, “we”, “us” and “our” refer to Sarepta Therapeutics, Inc. and its subsidiaries.

This section discusses 2025 and 2024 items and year-to-year comparisons between 2025 and 2024. Discussions of 2023 items and year-to-year comparisons between 2024 and 2023 have been excluded from this Form 10-K and can be found in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in Part II, Item 7 of our Annual Report on Form 10-K for the fiscal year ended December 31, 2024.

Overview

We are a commercial-stage biopharmaceutical company focused on helping patients through the discovery and development of unique RNA-targeted therapeutics, siRNA knockdown therapies, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. Applying our proprietary, differentiated and innovative technologies, and through collaborations with our strategic partners, we have developed multiple approved products for the treatment of Duchenne and are developing potential therapeutic candidates for a broad range of diseases and disorders, including Duchenne and LGMD, as well as those through our partnered programs with Arrowhead, including FSHD, DM1, SCA2, IPF, Huntington's disease and other neuromuscular and skeletal diseases.

We commercialized four products that have been approved by the FDA, including EXONDYS 51, VYONDYS 53, AMONDYS 45, and ELEVIDYS. We are in the process of conducting various clinical trials for our approved products, including studies that are required to comply with our post-marketing FDA requirements/commitments to verify and describe the clinical benefit of these products. On November 3, 2025, we announced top-line results from our ESSENCE trial, a confirmatory trial intended to verify the clinical benefits of AMONDYS 45 and VYONDYS 53. The topline results did not show statistical significance on the study's primary endpoint. We intend to discuss with FDA the potential pathway forward.

Our pipeline includes programs at various stages of discovery, pre-clinical and clinical development. Through our collaborations with our strategic partners, we are expanding into adjacent therapeutic areas. Our pipeline reflects our aspiration to apply our multifaceted approach and expertise in precision genetic medicine to make a profound difference in the lives of patients suffering from rare diseases.

We have developed proprietary state-of-the-art CMC and manufacturing capabilities that allow synthesis and purification of our products and product candidates to support both clinical development as well as commercialization. Our current main focus in manufacturing is to sustain large-scale production of our PMO-based therapies and optimizing manufacturing for gene therapy-based product candidates. We have entered into certain manufacturing and supply arrangements with third-party suppliers and will utilize these capabilities to support production of certain of our products and product candidates and their components.

The likelihood of our long-term success must be considered in light of the expenses, difficulties and delays frequently encountered in the development and commercialization of new pharmaceutical products, competitive factors in the marketplace, the risks associated with government sponsored reimbursement programs and the complex regulatory environment in which we operate.

Critical Accounting Policies and Estimates

The discussion and analysis of our financial condition and results of operations is based upon our consolidated financial statements included elsewhere in this Annual Report on Form 10-K. The preparation of our consolidated financial statements in accordance with U.S. GAAP requires us to make estimates and judgments that affect the reported amounts of assets, liabilities, revenues and expenses and related disclosures of contingent assets and liabilities for the periods presented. Some of these judgments can be subjective and complex, and, consequently, actual results may differ from these estimates. We believe that the estimates and judgments upon which we rely are reasonable based upon historical experience and information available to us at the time that we make these estimates and judgments. To the extent there are material differences between these estimates and actual results, our consolidated financial statements will be affected. Although we believe that our judgments and estimates are appropriate, actual results may differ from these estimates. We believe the following accounting policies to be most critical to the judgments and estimates used in the preparation of our consolidated financial statements:

•
inventory; and

•
income tax.

-76-

Inventory Valuation

Inventories are stated at the lower of cost and net realizable value with cost determined on a first-in, first-out basis. We capitalize 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 that may be used in clinical development programs is charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes.

We periodically analyze our inventories for excess or obsolescence and write 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. Our determination of excess or obsolete inventory is based on assumptions about forecasted demand for our products, market conditions and regulatory approvals. In 2025, we recorded $165.3 million of charges as a component of cost of sales in the consolidated statements of comprehensive (loss) income relating to excess or obsolete inventory. It is reasonably possible that actual results may differ from management’s estimates regarding forecasted demand, market conditions and regulatory approval and such differences could be material to our consolidated balance sheets, consolidated statements of comprehensive (loss) income and consolidated statements of cash flows. Additionally, though our products are subject to strict quality control and monitoring, which we perform 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 our quality specifications is recorded as a component of cost of sales in the consolidated statements of comprehensive (loss) income.

Income Tax

We follow 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. As of December 31, 2025, we continued to maintain a full valuation allowance against all of our deferred tax assets, with the exception of deferred tax assets in certain foreign jurisdictions, based on management's evaluation of all available evidence, including our earnings history.

We will continue to monitor the realizability of our deferred tax assets in future periods. We may release all or a portion of the valuation allowance in the near-term; however, 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, our level of profitability, revenue growth and expectations regarding future profitability. If and when we determine the valuation allowance should be released or reduced, the adjustment would result in a benefit to income tax expense for the period the release is recorded, which could have a material impact on net earnings.

We recognize the effect of income tax positions only if those positions are more likely than not of being sustained upon an examination. The calculation of our tax liabilities (or amount of reduction in our deferred tax assets from net operating loss carryover and research credit carryover) resulting from uncertain tax positions can involve significant judgment. Further, the calculation may involve the application of complex tax regulations in a foreign jurisdiction. Any significant impact as a result of changes in underlying facts, law, tax rates, tax audit, or review could lead to adjustments to our deferred tax asset, income tax expense, our effective tax rate, and/or our cash flow. Although we believe that we have adequately provided for tax liabilities resulting from uncertain tax positions, the actual amounts paid, if any, could have a material impact on our results of operations. Interest and penalties associated with uncertain tax positions are classified as a component of income tax expense.

Please read Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncements to the consolidated financial statements, included elsewhere in this Annual Report on Form 10-K, for a further discussion of our critical accounting policies and estimates.

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The following table sets forth selected consolidated statements of operations data for each of the periods indicated:

For the Year Ended December 31,

2025

2024

Change

Change

(in thousands, except per

share amounts)

$

%

Revenues:

Products, net

$

1,864,296

$

1,787,960

$

76,336

4

%

Collaboration and other

333,941

114,019

219,922

193

%

Total revenues

2,198,237

1,901,979

296,258

16

%

Cost and expenses:

Cost of sales (excluding amortization of in-licensed rights)

839,605

319,099

520,506

163

%

Research and development

1,522,066

804,522

717,544

89

%

Selling, general and administrative

491,716

557,872

(66,156

)

(12

)%

Restructuring charge

42,009

—

42,009

*

Amortization of in-licensed rights

2,622

2,405

217

9

%

Total cost and expenses

2,898,018

1,683,898

1,214,120

72

%

Operating (loss) income

(699,781

)

218,081

(917,862

)

*

Other (loss) income, net:

Other (expense) income, net

(19,306

)

42,693

(61,999

)

(145

)%

Gain on debt extinguishment

16,862

—

16,862

*

Total other (loss) income, net

(2,444

)

42,693

(45,137

)

*

(Loss) income before income tax expense

(702,225

)

260,774

(962,999

)

*

Income tax expense

11,185

25,535

(14,350

)

(56

)%

Net (loss) income

$

(713,410

)

$

235,239

$

(948,649

)

*

(Loss) earnings per share:

Basic

$

(7.13

)

$

2.47

$

(9.60

)

*

Diluted

$

(7.13

)

$

2.34

$

(9.47

)

*

*Not meaningful

Revenues

Revenues from product sales are recorded at the time of sale at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established and which result from rebates, governmental chargebacks including PHS chargebacks, prompt pay discounts, patient assistance programs and distribution fees. These reserves 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 us) or a current liability (if a payment is required of us). Our estimates take into consideration current contractual and statutory requirements. Actual amounts of consideration ultimately received or paid may differ from our estimates.

The following table summarizes the components of our net product revenues, by product, for the periods indicated:

For the Year Ended December 31,

2025

2024

Change

Change

(in thousands)

$

%

PMO Products

$

965,565

$

967,169

$

(1,604

)

(—

)%

ELEVIDYS

898,731

820,791

77,940

9

%

Products, net

$

1,864,296

$

1,787,960

$

76,336

4

%

Net product revenues for our products for 2025 increased by $76.3 million, or 4%, compared with 2024. The change primarily reflects an increase in net product revenues of ELEVIDYS of $77.9 million in 2025 as a result of its expanded label approval in June 2024, partially offset by higher discounts associated with the PHS chargeback program in 2025 due to ELEVIDYS no longer qualifying for pediatric designation, which had previously reduced our rebate obligations to certain payers.

-78-

The following table summarizes the components of our collaboration and other revenues for the periods indicated:

For the Year Ended December 31,

2025

2024

Change

Change

(in thousands)

$

%

Collaboration revenue

$

175,500

$

48,000

$

127,500

*

Contract manufacturing

124,013

49,038

74,975

153

%

Royalty revenue

34,428

16,981

17,447

103

%

Total collaboration and other

$

333,941

$

114,019

$

219,922

193

%

*Not meaningful

Collaboration and other revenues relate to the Roche Collaboration Agreement. For 2025 and 2024, we recognized $333.9 million and $114.0 million of collaboration and other revenues, respectively. In accordance with the Roche Collaboration Agreement, the parties agreed to enter into a supply agreement in order for us 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. We are eligible to receive royalties on these sales.

For 2025, we recognized $175.5 million in collaboration revenue consisting of (1) $112.0 million related to the expiration of an option for a certain program previously recorded as deferred revenue and (2) $63.5 million related to milestone payments received under the Roche Collaboration Agreement from the 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, as compared to $48.0 million in collaboration revenue in 2024 related to Roche’s declined option to acquire the ex-US rights to a certain external, early-stage Duchenne development program previously recorded as deferred revenue. Please refer to Note 3, License and Collaboration Agreements for further discussion of the Roche Collaboration Agreement.

While the Roche Supply Agreement is in the process of being negotiated, we delivered batches of commercial ELEVIDYS supply to Roche that were agreed upon on a purchase order-by-purchase order basis. Contract manufacturing revenue increased by $75.0 million primarily due to increased deliveries of ELEVIDYS to Roche in 2025 and increases in our manufacturing costs driven by greater-than-expected write-offs of (1) batches of our products not meeting our quality specifications as well as (2) certain excess or obsolete inventory attributable to Roche during 2025. In addition, royalty revenue increased by $17.4 million from increased sales of ELEVIDYS by Roche outside of the U.S. in 2025.

Cost of sales (excluding amortization of in-licensed rights)

Our cost of sales (excluding amortization of in-licensed rights) consists of inventory costs that relate to sales of our products and the related overhead costs and royalty payments primarily to BioMarin and UWA for our PMO Products and to Nationwide for ELEVIDYS. Cost of sales also include charges for inventory valuation for excess or obsolete inventory on hand and write-offs of batches of our products not meeting our quality specifications, including any associated costs expected to be reimbursed by Roche. Prior to receiving regulatory approval for our products, we expensed manufacturing and material costs as research and development expenses.

For the PMO Products, all previously expensed manufacturing costs had been fully consumed prior to 2024. For ELEVIDYS sold in 2025, a limited amount of related manufacturing costs incurred had previously been expensed as research and development expenses. For ELEVIDYS sold in 2024, the majority of related manufacturing costs incurred had previously been expensed as research and development expenses. If product related costs had not previously been expensed as research and development expenses prior to receiving FDA approval, the incremental inventory costs related to ELEVIDYS sold, including products sold to Roche under the Roche Collaboration Agreement, would have been approximately $22.9 million and $100.8 million for 2025 and 2024, respectively.

The following table summarizes the components of our cost of sales (excluding amortization of in-licensed rights) for the periods indicated:

For the Year Ended December 31,

2025

2024

Change

Change

(in thousands)

$

%

Product cost of sales (excluding Roche)

$

629,336

$

249,108

$

380,228

153

%

Roche product cost of sales**

159,086

22,247

136,839

*

Royalty payments

51,183

47,744

3,439

7

%

Total cost of sales (excluding amortization of in-licensed rights)

$

839,605

$

319,099

$

520,506

163

%

*Not meaningful

** See revenue section above for further details regarding product supply sold to Roche.

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The cost of sales (excluding amortization of in-licensed rights) for 2025 increased by $520.5 million, or 163%, compared with 2024. The change primarily reflects (1) an increase in the write-offs of certain batches of our products not meeting our quality specifications, (2) depletion of previously expensed ELEVIDYS inventory (3) a $165.3 million increase in our inventory valuation reserve related to excess ELEVIDYS and PMO inventory on hand as of the end of 2025, $24.4 million of which is expected to be reimbursed by Roche through increases in per unit cost of ELEVIDYS on future purchases, (4) increased demand following expanded label approval of ELEVIDYS in June 2024, (5) an increase in products sold to Roche under the Roche Collaboration Agreement, (6) termination costs incurred in association with a side letter agreement entered into with a raw material manufacturer for our PMO Products in 2025 and (7) the impairment of prepaid manufacturing deposits.

Research and development expenses

Research and development expenses consist of costs associated with research activities as well as those associated with our product development efforts, conducting pre-clinical trials, clinical trials and manufacturing activities. Direct research and development expenses associated with our programs include clinical trial site costs, clinical manufacturing costs, costs incurred for consultants, up-front fees and milestones paid to third parties in connection with technologies that have not reached technological feasibility and do not have an alternative future use, 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 our programs include salaries, stock-based compensation and allocation of our facility- and technology-related costs.

Research and development expenses represent a substantial percentage of our total operating expenses. We do not maintain or evaluate and, therefore, do not allocate internal research and development costs on a project-by-project basis. As a result, a significant portion of our research and development expenses are not tracked on a project-by-project basis, as the costs may benefit multiple projects.

The following table summarizes our research and development expenses, by project, for each of the periods indicated:

For the Year Ended December 31,

2025

2024

Change

Change

(in thousands)

$

%

Up-front and milestone expenses

$

883,787

$

—

$

883,787

*

SRP-9001

234,645

307,564

(72,919

)

(24

)%

LGMD platform

67,307

99,122

(31,815

)

(32

)%

Eteplirsen (exon 51)

42,315

70,213

(27,898

)

(40

)%

Other gene therapies

35,935

33,272

2,663

8

%

siRNA platform

32,924

—

32,924

*

Casimersen (exon 45)

10,181

14,805

(4,624

)

(31

)%

Golodirsen (exon 53)

7,419

10,062

(2,643

)

(26

)%

PPMO platform

6,453

31,926

(25,473

)

(80

)%

Other projects

9,578

23,917

(14,339

)

(60

)%

Internal research and development expenses

275,432

339,321

(63,889

)

(19

)%

Roche collaboration reimbursement

(83,910

)

(125,680

)

41,770

(33

)%

Total research and development expenses

$

1,522,066

$

804,522

$

717,544

89

%

*Not meaningful

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The following table summarizes our research and development expenses, by category, for each of the periods indicated:

For the Year Ended December 31,

2025

2024

Change

Change

(in thousands)

$

%

Up-front and milestone expenses

$

883,787

$

—

$

883,787

*

Manufacturing expenses

208,830

329,011

(120,181

)

(37

)%

Compensation and other personnel expenses

141,471

164,322

(22,851

)

(14

)%

Clinical trial expenses

124,645

163,565

(38,920

)

(24

)%

Facility- and technology-related expenses

92,455

90,697

1,758

2

%

Stock-based compensation

47,442

74,010

(26,568

)

(36

)%

Professional services

31,454

30,640

814

3

%

Pre-clinical expenses

4,010

6,359

(2,349

)

(37

)%

Research and other

71,882

71,598

284

—

%

Roche collaboration reimbursement

(83,910

)

(125,680

)

41,770

(33

)%

Total research and development expenses

$

1,522,066

$

804,522

$

717,544

89

%

*Not meaningful

Research and development expenses for 2025 increased by $717.5 million, or 89%, compared with 2024. The increase was primarily driven by the following:

•
$883.8 million increase in up-front and milestone expenses primarily due to the $583.6 million in acquired in-process research and development expense associated with our Arrowhead Collaboration Agreement. The remaining increase relates to $300.0 million of milestone payments to Arrowhead, triggered by Arrowhead's achievement of the DM1 Milestones, with no similar activity in 2024;

•
$120.2 million decrease in manufacturing expenses primarily due to costs associated with the termination of the development, commercial manufacturing and supply agreement (the “Thermo Agreement”) related to Brammer Bio MA, LLC, an affiliate of Thermo Fisher Scientific, Inc. (“Thermo”) in 2024, with no similar activity in 2025;

•
$22.9 million decrease in compensation and other personnel expenses primarily due to reduced headcount pursuant to our Restructuring in 2025, partially offset by increased use of temporary personnel;

•
$38.9 million decrease in clinical trial expenses primarily due to our decision to discontinue our PPMO programs in 2024 and the completion of certain SRP-9001 studies;

•
$1.8 million increase in facility- and technology-related expenses primarily due to utilization of our Bedford, Massachusetts facility beginning in 2025, with no similar activity in 2024;

•
$26.6 million decrease in stock-based compensation expense primarily due to the reversal of previously recognized expense related to unvested awards and reduced headcount pursuant to our Restructuring, fulfillment of remaining service conditions associated with certain restricted stock units with performance conditions ("PSUs") in March 2025 and our decision to suspend our employee stock purchase plan ("ESPP Suspension") in 2025. This was partially offset by certain performance conditions being met in June and December 2025 for certain PSUs;

•
$2.3 million decrease in pre-clinical expenses primarily due to a decrease in toxicology studies across our gene therapy programs; and

•
$41.8 million decrease in the offset to expense associated with a collaboration reimbursement from Roche primarily due to reimbursable costs associated with the termination of the Thermo Agreement in 2024, as well as a decrease in reimbursable research & development spend as a result of certain studies concluding in 2024 and a decrease in headcount pursuant to our Restructuring. This was partially offset by an increase in clinical supply costs due to additional SRP-9001 clinical batches released in 2025.

Selling, general and administrative expenses

Selling, general and administrative expenses consist of salaries, benefits, stock-based compensation and related costs for personnel in our 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 our facility- and technology-related costs and professional fees for legal, consulting and accounting services.

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The following table summarizes our selling, general and administrative expenses, by category, for each of the periods indicated:

For the Year Ended December 31,

2025

2024

Change

Change

(in thousands)

$

%

Professional services

$

167,182

$

183,505

$

(16,323

)

(9

)%

Compensation and other personnel expenses

153,494

171,508

(18,014

)

(11

)%

Stock-based compensation

75,954

110,290

(34,336

)

(31

)%

Facility- and technology-related expenses

55,900

50,903

4,997

10

%

Other

40,007

43,093

(3,086

)

(7

)%

Roche collaboration reimbursement

(821

)

(1,427

)

606

(42

)%

Total selling, general and administrative expenses

$

491,716

$

557,872

$

(66,156

)

(12

)%

Selling, general and administrative expenses for 2025 decreased by $66.2 million, or 12%, compared with 2024. The decrease was primarily driven by the following:

•
$16.3 million decrease in professional service expenses primarily due to reduced costs relating to certain litigation matters and reduced commercial spending related to our cost reduction initiative associated with our Restructuring;

•
$18.0 million decrease in compensation and other personnel primarily due to reduced headcount pursuant to our Restructuring;

•
$34.3 million decrease in stock-based compensation expense primarily due to the fulfillment of remaining service conditions associated with certain PSUs in March 2025, reversal of previously recognized expense related to unvested awards and reduced headcount pursuant to our Restructuring and the ESPP Suspension. This was partially offset by certain performance conditions being met in June and December 2025 for certain PSUs;

•
$5.0 million increase in facility- and technology-related expenses primarily due to utilization of our Bedford, Massachusetts facility beginning in 2025, with no similar activity in 2024; and

•
$3.1 million decrease in other expenses primarily due to the timing of charitable contribution activity, partially offset by certain state tax penalties in 2025, with no similar activity in 2024.

Restructuring charge

The Restructuring, announced in July 2025, was designed to reduce operating expenses and align our cost structure with strategic priorities, aiming to enhance financial flexibility and meet our 2027 financial obligations. This plan included a revised cost structure, program portfolio and a reduction in force. The workforce reduction represented approximately 36% of our workforce. We recorded a restructuring charge of $42.0 million for 2025, primarily related to employee termination benefits, including severance, along with accelerated depreciation for assets impacted by the restructuring plan, with no similar activity in 2024.

Please refer to Note 4, Restructuring for additional information on the Restructuring.

Amortization of in-licensed rights

Amortization of in-licensed rights relates to the agreements we entered into with UWA, Nationwide, BioMarin and Parent Project Muscular Dystrophy in April 2013, December 2016, July 2017 and May 2018, respectively. Each in-licensed right is being amortized on a straight-line basis over the remaining life of the relevant patent from the date the related fee was incurred, either the regulatory approval or the first commercial sale of the applicable product. For 2025 and 2024, we recorded amortization of in-licensed rights of $2.6 million and $2.4 million, respectively.

Gain on debt extinguishment

On August 28, 2025 (the "First Effective Date"), we completed a partial refinancing of the 2027 Notes (the "August 2025 Exchange") and exchanged $700.0 million in aggregate principal amount of 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 our common stock with a fair market value of approximately $104.9 million, net of issuance costs of $2.4 million.

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On December 18, 2025 (the "Second Effective Date"), we completed the second partial refinancing of the 2027 Notes (the “December 2025 Exchange”) and exchanged $291.4 million in 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 Company concluded that neither the August 2025 Exchange nor December 2025 Exchange (collectively, the “Exchange Transactions”) met the criteria for induced conversion. Further, we concluded that the refinanced debt in the August 2025 Exchange as well as approximately $182.8 million of that in the December 2025 Exchange were substantially different from the original debt and, thus, accounted for as debt extinguishments. The gain recognized on the extinguishments for the year ended December 31, 2025 was $16.9 million. 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. Please refer to Note 13, Indebtedness for additional information on the August 2025 Exchange and December 2025 Exchange.

There was no similar activity in 2024.

Other (expense) income, net

Other (expense) income, net primarily consists of the unrealized gain or loss from our investments in our strategic equity investments, interest expense on our 2027 Notes and 2030 Notes, interest income on our cash, cash equivalents and investments and accretion of investment discount. Our cash equivalents and investments consist of money market funds, government and government agency bonds, corporate bonds, commercial paper and certificates of deposit.

Other (expense) income, net for 2025 increased by $62.0 million compared to 2024. The change is primarily due to a $35.2 million decrease in interest income as a result of lower interest rates and the investment mix of our investment portfolio, a $19.7 million increase in interest expense primarily due to the 2030 Notes carrying a higher interest rate than our 2027 Notes and a $13.1 million increase in the loss on our strategic investments in publicly traded companies, including Arrowhead. These changes were partially offset by a $7.8 million loss associated with the change in fair value derivative liabilities in 2024, which primarily relates to our contingent consideration, with no similar activity in 2025 due to the adoption of Accounting Standards Update 2025-07 ("ASU 2025-07"). As a result, two existing contracts that include a settlement feature based on our operations or activities are now excluded from ASC 815, Derivatives and Hedging and any liabilities will be recognized as such obligations become probable and estimable under ASC 450, Contingencies. Refer to Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncements for additional information related to the adoption of ASU 2025-07.

Income tax expense

Income tax expense for 2025 and 2024 was $11.2 million and $25.5 million, respectively. Income tax expense for 2025 primarily relates to state taxes. Income tax expense for 2024 relates to state, foreign and federal taxes for which available tax losses or credits were not available to offset. As of December 31, 2025, we continued to maintain a full valuation allowance against our deferred tax assets, with the exception of deferred tax assets in certain foreign jurisdictions. We continue to monitor the available evidence relative to recovery of our deferred tax assets and whether such evidence would be sufficient to conclude that it is more likely than not that such deferred tax assets may be partially or fully recoverable. If we were to remove our valuation allowance in part or full, any such adjustment could have a material impact on our effective tax rate in the applicable period and beyond. Refer to Note 18, Income Taxes for discussion of the key drivers impacting our effective tax rate.

Liquidity and Capital Resources

In the August 2025 Exchange, we exchanged 2027 Notes for a combination of 2030 Notes, common stock and cash. Concurrently with the issuance of the 2030 Notes during the August 2025 Exchange, we completed the private placement of approximately 1.1 million shares of Common Stock to J. Wood Capital Advisors LLC ("JWCA") at a purchase price per share of $18.07. In the December 2025 Exchange, we exchanged 2027 Notes for 2030 Notes and cash. While the 2030 Notes carry a higher interest rate than the 2027 Notes, the August 2025 Exchange and December 2025 Exchange reduced our total outstanding principal amount of long-term debt by $98.0 million and delayed $893.4 million of the principal payment to 2030. Additionally, our Revolving Credit Facility remains undrawn as of December 31, 2025. Refer to Note 13, Indebtedness for additional discussion of our outstanding indebtedness. We periodically engage in discussions with the lenders under our Revolving Credit Facility regarding business developments and our continued ability to borrow under our Revolving Credit Facility. See Risk Factors—Risks Related to our Financial Condition and Capital Requirements—Our existing and any future indebtedness could adversely affect our ability to operate our business.

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The following table summarizes our financial condition for each of the periods indicated:

For the Year Ended December 31,

2025

2024

Change

Change

(in thousands)

$

%

Financial assets:

Cash and cash equivalents

$

801,282

$

1,103,010

$

(301,728

)

(27

)%

Short-term investments

138,368

251,782

(113,414

)

(45

)%

Non-current investments

1,048

133,163

(132,115

)

(99

)%

Restricted cash

13,125

15,579

(2,454

)

(16

)%

Total cash, cash equivalents and investments

$

953,823

$

1,503,534

$

(549,711

)

(37

)%

Borrowings:

Convertible debt

$

828,974

$

1,137,124

$

(308,150

)

(27

)%

Total borrowings

$

828,974

$

1,137,124

$

(308,150

)

(27

)%

Working capital:

Current assets

$

2,537,938

$

3,073,463

$

(535,525

)

(17

)%

Current liabilities

1,095,290

731,684

363,606

50

%

Total working capital

$

1,442,648

$

2,341,779

$

(899,131

)

(38

)%

For 2025, our principal sources of liquidity were primarily derived from the sales of our products, our collaboration arrangement with Roche and proceeds from the exercise of stock options. Our principal uses of cash for 2025 were our $583.6 million up-front payment to Arrowhead, $241.4 million equity investment in Arrowhead's common stock and the $50.0 million cash component of the $100.0 million of the DM1 Milestones (collectively, the “Arrowhead Payments”), costs incurred as a result of the August 2025 Exchange and the December 2025 Exchange, inventory commitments, research and development expenses, manufacturing costs, selling, general and administrative expenses, investments, capital expenditures, share repurchases under our $500.0 million share repurchase program approved by the Board of Directors in November 2024 (the “2024 Repurchase Program”) and other working capital requirements. Please refer to Note 14, Equity, for further discussion of share repurchases under the 2024 Repurchase Program during the period.

For 2024, our principal sources of liquidity were primarily derived from the sales of our products, the maturity and sale of available-for-sale securities, our collaboration arrangement with Roche, proceeds from exercise of stock options and the settlement of capped call options associated with our convertible senior notes due on November 15, 2024. Our principal uses of cash for 2024 were inventory commitments, research and development expenses, manufacturing costs, selling, general and administrative expenses, investments, capital expenditures and other working capital requirements.

The changes in our working capital for both periods primarily reflect use of cash in operating activities, as well as a reduction in our cash, cash equivalents and investments to fund the Arrowhead Payments in 2025. The Arrowhead Collaboration Agreement includes a commitment of $250.0 million in guaranteed payments to be paid in five equal annual installments of $50.0 million, beginning in February 2026. These payments are not contingent on clinical or regulatory milestones. The Arrowhead Collaboration Agreement also includes $300.0 million in payments for the DM1 Milestones. Of this amount, $100.0 million was settled during the year ended December 31, 2025 with a combination of $50.0 million in cash and shares of Arrowhead common stock valued at approximately $50.0 million, and the remaining $200.0 million was paid in January 2026. Refer to Note 3, License and Collaboration Agreements for further discussion of the Arrowhead DM1 Milestones. While our contractual obligations, commitments and debt service requirements over the next several years are significant, we intend to continue to fund our short-term financing needs and working capital requirements from cash flows from operating activities as well as cash on hand and drawing from our Revolving Credit Facility, as needed. Such sources are anticipated to be adequate to fund working capital requirements for at least twelve months from the date these consolidated financial statements were issued.

In response to two reported cases of ALF resulting in death of non-ambulatory patients, we suspended all commercial shipments of ELEVIDYS to non-ambulatory patients in June 2025. In July 2025, we also disclosed a recent death from ALF of a non-ambulatory LGMD patient participating in a Phase I/II trial for SRP-9004, who was not treated with ELEVIDYS. Thereafter, in response to a request from the FDA that we voluntarily stop all shipments of ELEVIDYS in the U.S., including to ambulatory patients, we temporarily suspended all shipments of ELEVIDYS in the U.S., effective July 22, 2025, to allow us the necessary time to respond to FDA's requests for information and complete a labeling supplement process. Although the FDA informed us on July 28, 2025 that it recommended the removal of the voluntary hold for ambulatory patients, and we subsequently resumed shipments of ELEVIDYS for ambulatory patients in the U.S. on July 31, 2025, the ELEVIDYS Suspension could materially impact our near-term revenue generation. In November 2025, the FDA approved dosing in Cohort 8 of ENDEAVOR (Study 9001-103). The purpose of Cohort 8 is to evaluate the use of sirolimus as an enhanced immunosuppressive regimen as part of treatment with ELEVIDYS for non-ambulatory patients. It is currently unclear whether or when we might resume commercial shipments of ELEVIDYS for non-ambulatory patients. We considered the ELEVIDYS Suspension and the partial resumption of shipments when concluding that our

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cash, cash equivalents and investments as of the date of issuance of this report, along with future cash inflows from operations and availability under our Revolving Credit Facility, are sufficient to fund our current operational plan for at least the next twelve months. Additionally, any regulatory action in response to the topline results of our ESSENCE trial that were announced on November 3, 2025 and failed to meet statistical significance on its primary endpoint, could adversely impact our cash flows from operations.

Beyond 2026, our cash requirements will depend extensively on our ability to advance our research, development and commercialization of product candidates. The likelihood of our long-term success must be considered in light of the expenses, difficulties and delays frequently encountered in the development and commercialization of new pharmaceutical products, competitive factors in the marketplace, the risks associated with government sponsored reimbursement programs and the complex regulatory environment in which we operate. We may seek additional financing primarily from, but not limited to, the sale and issuance of equity and debt securities, the licensing or sale of our technologies and entering into additional government contracts and/or funded research and development agreements. Further, while we anticipate that the Restructuring announced in July 2025 will result in a reduction in our future expenditures, actual savings realized may vary and there can be no assurance that the Restructuring will achieve our anticipated cost savings or operational efficiencies. Our future expenditures and long-term capital requirements may be substantial and will depend on many factors, including but not limited to the following:

•
our ability to continue to generate revenues from sales of commercial products and potential future products;

•
our ability to resume commercial shipments of ELEVIDYS for non-ambulatory patients in the U.S.;

•
our ability to realize the benefits of the Restructuring;

•
the risk that our Restructuring and pipeline reprioritization efforts may not generate their intended benefits to the extent or as quickly as anticipated;

•
the impact of potential regulatory actions from the FDA including changes to our drug labels or revocation of accelerated approvals and directives to remove products from the market relating to the topline results of our ESSENCE trial that failed to meet statistical significance on its primary endpoint;

•
the timing and costs associated with repurchases of our common stock under our 2024 Repurchase Program;

•
the timing of payments related to our future inventory commitments and manufacturing obligations;

•
the timing and costs associated with our existing lease obligations and new obligations expected to be entered into in future years;

•
the timing and costs associated with our pre-clinical and clinical trials;

•
the attainment of milestones and our obligations to make milestone payments to Arrowhead, Myonexus Therapeutics, Inc.'s selling shareholders, BioMarin, Nationwide, UWA and other institutions;

•
the timing and repayment of future borrowings on our Revolving Credit Facility;

•
obligations to holders of our 2027 Notes and 2030 Notes; and

•
the costs of filing, prosecuting, defending and enforcing patent claims and our other intellectual property rights.

We cannot provide assurances that financing will be available when and as needed or that, if available, the financings will be on favorable or acceptable terms. If we are unable to obtain additional financing when and if we require, this would have a material adverse effect on our business and results of operations. To the extent we issue additional equity securities, our existing stockholders could experience substantial dilution. Additional information regarding our Revolving Credit Facility is provided in Note 13, Indebtedness to the consolidated financial statements included elsewhere in this Annual Report on Form 10-K. We believe that our existing cash, cash equivalents and investments along with future cash generated from operations and availability under our Revolving Credit Facility will be sufficient to meet the capital requirements of our operations for the next twelve months and foreseeable future.

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We have entered into long-term contractual arrangements from time to time for our facilities, the provision of goods and services, and issuance of debt securities, among others. Additional information regarding our obligations under debt, lease, and manufacturing arrangements is provided in Note 13, Indebtedness, Note 19, Leases and Note 23, Commitments and Contingencies, respectively, to the consolidated financial statements included elsewhere in this Annual Report on Form 10-K. The following table summarizes our total obligations under debt, lease, and manufacturing arrangements:

As of December 31, 2025

Due in less than one year

Due in greater than one year

Total

(in thousands)

Debt obligations (1)

$

41,597

$

1,228,318

$

1,269,915

Lease obligations (2)

21,945

311,023

332,968

Manufacturing obligations (3)

507,440

149,337

656,777

Total obligations under debt, lease and manufacturing arrangements

$

570,982

$

1,688,678

$

2,259,660

(1) Interest payments are included within the future debt obligations.

(2) Lease obligations only include real estate leases that had commenced prior to December 31, 2025.

(3) The leases embedded in a certain supply agreement are included in manufacturing obligations. The decrease in short-term manufacturing commitments is primarily driven by a supplemental letter agreement (the "Letter Agreement") entered into with Catalent in August 2025 to address certain financial and operational matters related to the ELEVIDYS Suspension. Refer to Note 23, Commitments and Contingencies for further discussion surrounding the Letter Agreement with Catalent.

For products and product candidates that are currently approved or are in various research and development stages, we may be obligated to make up to $12.1 billion of future development, regulatory, up-front royalty and sales milestone payments associated with our license and collaboration agreements. Payments under these agreements generally become due and payable upon achievement of certain development, regulatory or commercial milestones. Because the achievement of these milestones is not probable and payment is not required as of December 31, 2025, such contingencies have not been recorded in our consolidated financial statements. Amounts related to contingent milestone payments are not yet considered contractual obligations as they are contingent on the successful achievement of certain development, regulatory approval and commercial milestones.

Cash Flows

The following table summarizes our cash flow activity for each of the periods indicated:

For the Year Ended December 31,

2025

2024

Change

Change

(in thousands)

$

%

Cash (used in) provided by

Operating activities

$

(205,479

)

$

(205,787

)

$

308

(0

)%

Investing activities

69,641

755,561

(685,920

)

(91

)%

Financing activities

(168,344

)

124,806

(293,150

)

(235

)%

(Decrease) increase in cash and cash equivalents

$

(304,182

)

$

674,580

$

(978,762

)

(145

)%

Operating Activities

Cash used in operating activities, which consists of our net (loss) income adjusted for non-cash items and changes in net operating assets and liabilities, totaled $205.5 million and $205.8 million of cash in 2025 and 2024, respectively. Cash used in operating activities in 2025 was primarily driven by the net loss of $713.4 million, adjusted for the following non-cash items:

•
$165.3 million in write-downs for excess and obsolete inventory;

•
$123.4 million in stock-based compensation expense;

•
$50.0 million in-kind milestone payments to Arrowhead;

•
$44.5 million in depreciation and amortization expense;

•
$17.3 million loss on investment in Arrowhead;

•
$17.0 million write-off of prepaid deposits;

•
$16.9 million gain on debt extinguishment;

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•
$14.0 million reduction in the carrying amount of the right of use assets; and

•
$20.6 million in other non-cash items.

These amounts were partially offset by $5.3 million in accretion of investment discount, net.

The net cash outflow from changes in our operating assets and liabilities was primarily driven by the following:

•
$301.1 million increase in inventory primarily due to capitalized inventory related to ELEVIDYS;

•
$203.8 million decrease in accounts receivable primarily due to reduced shipments of ELEVIDYS in the second half of 2025 compared to 2024 due to the ELEVIDYS Suspension;

•
$79.8 million increase in other assets primarily due to the timing of billings to Roche for orders of ELEVIDYS for use outside of the U.S., as well as increased royalty receivables from Roche related to increased ELEVIDYS sales outside of the U.S.;

•
$104.3 million increase in accounts payable, accrued expenses, lease liabilities and other liabilities primarily due to the remaining $200.0 million associated with the Arrowhead DM1 Milestones being included in accounts payable as of December 31, 2025, partially offset by a reduction in accrued employee compensation costs pursuant to our Restructuring and payments on accrued employee compensation costs, a reduction in accrued income taxes and the timing and invoicing of payments with our contract research organizations and contract manufacturing organizations;

•
$72.1 million increase in deferred revenue primarily related to the timing of billings to Roche for orders of ELEVIDYS not yet shipped, partially offset by the recognition of collaboration revenue related to the expiration of an option for a certain program previously recorded as deferred revenue; and

•
$78.8 million decrease in manufacturing-related deposits and prepaids primarily due to the use of raw materials and services previously prepaid with Catalent and Aldevron.

Cash used in operating activities in 2024 was primarily driven by the net income of $235.2 million, adjusted for the following non-cash items:

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$184.3 million in stock-based compensation expense;

•
$62.7 million in non-cash termination charges as a result of the Thermo Agreement termination;

•
$37.7 million in depreciation and amortization expense;

•
$16.2 million reduction in the carrying amount of the right of use assets;

•
$7.8 million charge related to the change in the fair value of derivatives; and

•
$7.1 million in other non-cash items.

These amounts were partially offset by $40.3 million in accretion of investment discount, net.

The net cash outflow from changes in our operating assets and liabilities was primarily driven by the following:

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$395.2 million increase in inventory primarily due to capitalized inventory related to ELEVIDYS;

•
$201.7 million increase in accounts receivable due to an increase in demand for ELEVIDYS following its initial FDA approval in June 2023 and subsequent expanded label approval in June 2024 and an increase in payment terms for product sales related to the PMO Products;

•
$188.6 million increase in manufacturing-related deposits and prepaids primarily due to an increase in prepaids for raw materials and batch fees with Catalent, partially offset by decreases in manufacturing-related deposits and prepaids at Thermo as a result of the termination of the Thermo Agreement during 2024; and

•
$32.2 million decrease in deferred revenue primarily related to the collaboration with Roche.

These amounts were partially offset by a $110.6 million increase in accounts payable, accrued expenses, lease liabilities and other liabilities primarily due to the timing and invoicing of payments with our CROs and CMOs.

Investing Activities

Cash provided by investing activities for 2025 was $69.6 million, compared to $755.6 million in 2024. Cash provided by investing activities in 2025 consisted of $295.9 million from the maturity and sales of available-for-sale securities and $174.1 million in proceeds from the sale of the Arrowhead investment, partially offset by $245.8 million in the acquisition of strategic investments primarily related to Arrowhead as well as purchases of property and equipment, available-for-sale securities and intangible assets of $102.0 million, $44.7 million and $7.9 million, respectively.

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Cash provided by investing activities in 2024 consisted of $2,002.1 million from the maturity and sales of available-for-sale securities, partially offset by purchases of available-for-sale securities, property and equipment and intangible assets of $1,099.6 million, $137.0 million and $10.0 million, respectively.

Financing Activities

Cash used in financing activities was $168.3 million in 2025, compared to $124.8 million of cash provided by financing activities in 2024. Cash used in financing activities in 2025 primarily consisted of the following:

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$158.4 million for the repurchase of a portion of our 2027 Notes;

•
$25.0 million of purchases of our common stock under our 2024 Repurchase Program;

•
$17.1 million of debt issuance costs associated with our 2030 Notes;

•
$4.2 million of payments related to our Revolving Credit Facility; and

•
$2.4 million of issuance costs associated with issuance of our common stock.

These amounts were partially offset by the following items:

•
$20.0 million in proceeds from the private placement of approximately 1.1 million shares of our common stock pursuant to a privately negotiated subscription agreement with JWCA; and

•
$18.7 million in proceeds from exercise of options and purchase of stock under our Employee Stock Purchase Program.

Cash provided by financing activities in 2024 primarily consisted of $79.5 million in proceeds from exercise of options and purchase of stock under our Employee Stock Purchase Program and $45.3 million in proceeds from the settlement of the 2017 Capped Calls.

Other Funding Commitments

We have several on-going clinical trials in various development stages. Our most significant clinical trial expenditures are to CROs. The CRO contracts are generally cancellable at our option. As of December 31, 2025, we had $404.3 million in cancellable future commitments based on existing CRO contracts.

Recent Accounting Pronouncements

Please read Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncements to the consolidated financial statements included elsewhere in this Annual Report on Form 10-K.