Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2025 Financial Results and Recent Corporate Developments

• Net product revenues for the full year 2025 totaled $1,864.3 million, consisting of $965.6 million of PMO net product revenue and $898.7 million of ELEVIDYS net product revenue
• Net product revenues for the fourth quarter 2025 totaled $369.6 million, consisting of $259.2 million of PMO net product revenue and $110.4 million of ELEVIDYS net product revenue
• Following refinancing of 2027 notes and corporate restructuring, overall financial position and capital structure strengthened to support full investment in our pipeline and marketed therapies

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the fourth quarter and full-year 2025.

“Following a tumultuous 2025, we entered 2026 from a position of strength, founded on: (a) solid financial footing with a robust and growing cash balance, substantial revenue, and no near-term debt overhang, (b) durable approved therapies that are bringing a better life to patients, with significant yet-tapped opportunity for ELEVIDYS gene therapy, and (c) an exciting, potentially best-in-class siRNA pipeline of advancing therapies for DM1, FSHD, Huntington’s disease, idiopathic pulmonary fibrosis, SCA1, SCA2 and SCA3,” said Doug Ingram, chief executive officer, Sarepta Therapeutics. “In 2025, we streamlined our operations, delivered strong revenue, and ended the year with nearly $1.0 billion in cash—as we anticipate remaining profitable and cash‑flow positive in 2026. ELEVIDYS has emerged from a challenging year with a clear label, traditional approval for ambulatory patients and a plan intended to put us on a potential pathway back to serving the non‑ambulatory community, and we are executing comprehensive plans designed to arm treating physicians and families with accurate and balanced information to unlock the full potential of this transformative and needed therapy. At the same time, our PMO exon-skipping therapies continue to demonstrate durable clinical value, exceptional safety, extraordinary real‑world outcomes, and unwavering support from families and physicians. Finally, with five clinical‑stage RNAi programs and multiple readouts ahead, we are poised for meaningful growth while advancing therapies that can profoundly change the lives of patients living with rare disease.”

Corporate Highlights:

• ELEVIDYS Launched in Japan: In February 2026, Chugai Pharmaceutical Co. announced that it has launched ELEVIDYS Intravenous Infusion in Japan, the country’s first regenerative medical product for Duchenne muscular dystrophy. The launch follows the May 2025 approval of ELEVIDYS by the Japanese Ministry of Health, Labour, and Welfare (MHLW). In Japan, ELEVIDYS is approved for the treatment of Duchenne under the conditional and time-limited approval pathway 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 DMD gene, and who are negative for anti-AAVrh74 antibodies. Sarepta is eligible to receive a $40 million milestone payment upon first commercial sale in Japan.
  
  
• Announced positive three-year results from the EMBARK study: In January 2026, Sarepta reported topline three‑year data from Part 1 of EMBARK (Study SRP‑9001‑301), the global, randomized, Phase 3 trial evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl), the first and only approved gene therapy for Duchenne muscular dystrophy. Three years after treatment, patients who received ELEVIDYS in Part 1 of EMBARK (mean age ~9 years at last assessment) maintained motor function above their baseline NSAA scores, while the matched external control (EC) group demonstrated the expected age-related decline.
  
  ELEVIDYS gene therapy produced clinically meaningful, statistically significant, and durable benefits across all key functional endpoints—North Star Ambulatory Assessment (NSAA), Time to Rise (TTR), and 10‑meter walk/run (10MWR). Treated patients achieved a 73% slowing of disease progression in TTR and 70% slowing in 10MWR relative to the EC group at Year 3, with the performance gap widening between Years 2 and 3. These results further validate and extend the functional improvements observed in the one‑and two‑year EMBARK analyses.
  
  The safety profile of ELEVIDYS remained consistent with previously reported findings, with no new treatment‑related safety signals identified over the three‑year follow‑up period. ELEVIDYS continues to demonstrate a durable treatment effect and strong potential to modify the trajectory of Duchenne by preserving muscle function over time.
  
  
• Approval of CTA for SRP-1005 in Huntington’s: In January 2026, Sarepta announced approval to begin a first‑in‑human Phase 1 trial of SRP‑1005, an siRNA therapeutic for Huntington’s disease, which will assess safety and tolerability in about 24 participants starting in Q2 2026.
  
  
• Refinance of $291.4 million of 2027 convertible notes: In December 2025, Sarepta exchanged $291.4 million of its 2027 convertible notes for an equal amount of new 2030 convertible notes plus $31.6 million in cash. This latest restructuring of our convertible debt leaves $158.6 million of the original notes outstanding, removing any significant debt overhang this decade.
  
  
• U.S. FDA approval of updated ELEVIDYS prescribing information: In November 2025, Sarepta announced updated prescribing information for ELEVIDYS, adding a boxed warning for serious liver injury, removing the non‑ambulatory indication, and providing expanded immunosuppression, monitoring, and corticosteroid guidance.
  
  
• Sarepta to present new long-term and safety data across gene therapy and exon-skipping programs at 2026 Muscular Dystrophy Association Clinical & Scientific Conference: Sarepta will present new clinical and real‑world evidence at MDA 2026 showing long‑term efficacy, safety, and caregiver‑reported impact of its gene therapy and exon‑skipping treatments for Duchenne muscular dystrophy, reinforcing dystrophin’s role in slowing disease progression and supporting informed treatment decisions.
  
  
• Duchenne Muscular Dystrophy Added to the U.S. Recommended Uniform Screening Panel (RUSP): The inclusion of Duchenne on the RUSP represents a pivotal advancement for the Duchenne community, encouraging broader newborn screening at the state level and empowering more families with early diagnosis and timely information to pursue earlier care, including access to available therapies and clinical trials. Each state administers their own newborn screening programs, and states look to federal evidence-based recommendations – the RUSP – to inform what conditions they add to their panels. Adding a condition to the RUSP signals the importance of early detection and the availability of effective treatments and may accelerate adoption of screening across states.
  
  
• ENDEAVOR Cohort 8 to Evaluate Enhanced Immunosuppression Regimen as Part of ELEVIDYS Gene Therapy for Non-Ambulant Individuals with Duchenne Approved to Begin: 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 an enhanced immunosuppressive regimen as part of treatment with ELEVIDYS for non-ambulant individuals with Duchenne muscular dystrophy.
  
  
• SRP-1003, an Investigational siRNA treatment for Myotonic Dystrophy Type 1, Advances: In November 2025, Sarepta provided an update that the Phase 1/2 multiple ascending dose (MAD) clinical study of SRP-1003 (formerly ARO-DM1), an investigational small interfering RNA (siRNA) therapeutic for the treatment of type 1 myotonic dystrophy (DM1). Cohorts 1 (1.5 mg/kg) and 2 (3 mg/kg) of the study are complete, and cohort 3 (4.5 mg/kg) is fully enrolled and ongoing. Following a positive, pre-specified drug safety committee review, the study is expected to advance with additional drug escalating cohorts.

Conference Call

The event will be webcast live under the investor relations section of Sarepta's website at https://investorrelations.sarepta.com/events-presentations and following the event a replay will be archived there for one year. This event can be accessed using this link.

Q4 2025 Financial Highlights¹

Revenues
Total revenues were $442.9 million for the three months ended December 31, 2025, as compared to $658.4 million for the same period of 2024, a decrease of $215.5 million. The decrease primarily reflects $273.8 million less in net product revenue of ELEVIDYS as a result of lower volume following our decision to suspend shipments of ELEVIDYS to non-ambulatory patients in the U.S. in June 2025. In addition, collaboration and other revenues increased approximately $53.0 million primarily due to contract manufacturing revenues increasing $45.6 million driven by higher volume of shipments of ELEVIDYS to Roche.

Total revenues were $2,198.2 million for the twelve months ended December 31, 2025, as compared to $1,902.0 million for the same period of 2024, an increase of $296.2 million. The increase primarily reflects $77.9 million more in net product revenue of ELEVIDYS as a result of its expanded label approval in June 2024. In addition, collaboration and other revenues increased approximately $219.9 million primarily related to the $63.5 million of collaboration revenue recognized as a result of the regulatory approval in Japan and the $112.0 million of collaboration revenue recognized related to Roche's expiration of an option for a program during the twelve months ended December 31, 2025, as compared to $48.0 million of collaboration revenue recognized in the same period of 2024 related to Roche’s declined option to acquire certain ex-US rights to an external, early-stage Duchenne development program. Furthermore, contract manufacturing revenues and royalty revenues increased $75.0 million and $17.4 million, respectively, associated with an increase in commercial ELEVIDYS supply delivered to Roche as well as royalty revenue from sales of ELEVIDYS by Roche, respectively.

Cost of sales (excluding amortization of in-licensed rights)
Cost of sales (excluding amortization of in-license rights) were $398.7 million for the three months ended December 31, 2025, as compared to $132.3 million for the same period of 2024, an increase of $266.4 million. The increase primarily reflects 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 as well as an increase in the write-offs of certain batches of our products not meeting our quality specifications, termination costs incurred in association with a side letter agreement entered into with a raw material manufacturer for our PMO Products in 2025 and an increase in products sold to Roche for the three months ended December 31, 2025, as compared to the same period of 2024.

Cost of sales (excluding amortization of in-license rights) were $839.6 million for the twelve months ended December 31, 2025, as compared to $319.1 million for the same period of 2024, an increase of $520.5 million. The increase primarily reflects the Q4 2025 quarter-to-date increases discussed above, as well as additional write-offs of certain batches of our products not meeting our quality specifications and an increased demand for ELEVIDYS following the expanded label approval in June 2024 for the twelve months ended December 31, 2025, as compared to the same period of 2024.

Operating expenses and others
Research and development expenses were $325.3 million for the three months ended December 31, 2025, as compared to $200.0 million for the same period of 2024, an increase of $125.3 million. The increase is primarily due to a $200.0 million increase in milestone expenses due to our milestone payment to Arrowhead related to the progression of the DM1 program, partially offset by a decrease in manufacturing expense related to our LGMD programs and a decrease in compensation, other personnel, and stock-based compensation expenses, all as a result of our restructuring plan announced in July 2025, with no similar activity for the three months ended December 31, 2024. For the three months ended December 31, 2025, non-GAAP research and development expenses were $308.1 million, as compared to $172.7 million for the same period of 2024, an increase of $135.4 million.

Research and development expenses were $1,522.1 million for the twelve months ended December 31, 2025, as compared to $804.5 million for the same period of 2024, an increase of approximately $717.6 million. The increase primarily reflects a $583.6 million increase in up-front expenses associated with the licensing and collaboration agreement and stock purchase agreement with Arrowhead, as well as the $300.0 million of milestone payments made to Arrowhead related to the progression of the DM1 program for the twelve months ended December 31, 2025. This increase is partially offset by a decrease in manufacturing expenses primarily due to the termination of the Thermo Agreement during the twelve months ended December 31, 2024, as well as a decrease in compensation, other personnel, and stock-based compensation expenses as a result of our restructuring plan announced during the twelve months ended December 31, 2025. For the twelve months ended December 31, 2025, non-GAAP research and development expenses were $1,445.5 million, as compared to $704.5 million for the same period of 2024, an increase of $741.0 million.

Selling, general and administrative expenses were $128.3 million for the three months ended December 31, 2025, as compared to $163.9 million for the same period of 2024, a decrease of $35.6 million. The decrease is primarily driven by lower compensation, other personnel, stock-based compensation, and commercial expenses as a result of our restructuring plan announced in July 2025, with no similar activity for the three months ended December 31, 2024. For the three months ended December 31, 2025, non-GAAP selling, general and administrative expenses were $105.4 million, as compared to $131.6 million for the same period of 2024, a decrease of $26.2 million.

Selling, general and administrative expenses were $491.7 million for the twelve months ended December 31, 2025, as compared to $557.9 million for the same period of 2024, a decrease of $66.2 million. The decrease is primarily driven by reduced headcount as a result of our restructuring plan announced in July 2025 for the twelve months ended December 31, 2025, and a net decrease in stock-based compensation primarily due to the reversal of previously recognized expense related to unvested awards as a result of our restructuring plan, partially offset by the fulfillment of remaining service conditions associated with certain PSUs in March 2025. For the twelve months ended December 31, 2025, non-GAAP selling, general and administrative expenses were $403.0 million, as compared to $438.3 million for the same period of 2024, a decrease of $35.3 million.

Restructuring charges were $1.5 million and $42.0 million for the three and twelve months ended December 31, 2025, respectively, with no similar activity for the same periods of 2024. These charges were primarily related to employee termination benefits, including severance, along with accelerated depreciation for assets impacted by our restructuring plan, announced in July 2025, that 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.

Gain on debt extinguishment was $13.1 million and $16.9 million for the three and twelve months ended December 31, 2025, respectively, with no similar activity for the same periods of 2024. The gain on debt extinguishment is a result of two partial refinancings of the 2027 Notes through privately negotiated exchange transactions. In August 2025, we exchanged $700.0 million in aggregate principal amount of the 2027 Notes for (1) $602.0 million in aggregate principal amount of new 2030 Notes, net of issuance costs of $13.4 million, (2) cash payments of $127.3 million, including $4.0 million of accrued interests of the 2027 Notes, and (3) the issuance of 5.9 million shares of our common stock with fair market value of approximately $104.9 million, net of issuance costs of $2.4 million (collectively, the "August 2025 Exchange"). In December 2025, we exchanged $291.4 million in aggregate principal amount of 2027 Notes for (1) $291.4 million in aggregate principal amount of the 2030 Notes, net of issuance costs of $4.0 million and (2) cash payments of $31.6 million, including $1.0 million of accrued interests of the 2027 Notes.

Other (expense) income, net for the three months ended December 31, 2025 and 2024, was $(9.2) million and $10.1 million, respectively. The change primarily reflects an increase in interest expense primarily due to a higher interest rate for the 2030 Notes as compared to the 2027 Notes that were exchanged, as well as a decrease in interest income due to lower interest rates and the investment mix of our investment portfolio during the three months ended December 31, 2025. Other (expense) income, net for the twelve months ended December 31, 2025 and 2024, was $(19.3) million and $42.7 million, respectively. The change primarily reflects the decrease in interest income due to lower interest rates and the investment mix of our investment portfolio as well as an increase in the loss on our strategic investments in publicly traded companies, including Arrowhead, during the twelve months ended December 31, 2025.

Income tax expense for the three months ended December 31, 2025 and 2024, was $4.6 million and $12.7 million, respectively. Income tax expense for the twelve months ended December 31, 2025 and 2024, was $11.2 million and $25.5 million, respectively. Income tax expense for the three and twelve months ended December 31, 2025, and December 31, 2024, primarily relates to state, federal and foreign income taxes for which available tax losses or credits were not available to offset.

Use of Non-GAAP Measures
In addition to the GAAP financial measures set forth in this press release, we have included the following non-GAAP measurements:

1. Non-GAAP net (loss) income is defined by us as GAAP net (loss) income excluding interest income/expense, net, depreciation and amortization expense, stock-based compensation expense, restructuring charges, other items, and the estimated income tax impact of each pre-tax non-GAAP adjustment.
2. Non-GAAP net loss per share is defined by us as non-GAAP net loss, as defined above, divided by the weighted-average number of shares of common stock outstanding as the inclusion of dilutive common stock equivalents outstanding is anti-dilutive. Non-GAAP earnings per share is defined by us as non-GAAP net income, as defined previously, divided by the weighted-average number of shares of common stock and dilutive common stock equivalents outstanding, adjusted for the inclusion of additional shares under the “if-converted” method, if applicable and not anti-dilutive.
3. Non-GAAP operating (loss) income is defined by us as GAAP operating (loss) income excluding depreciation and amortization expense, stock-based compensation expense, and restructuring charges.
4. Non-GAAP research and development expenses are defined by us as GAAP research and development expenses excluding depreciation and amortization expense, and stock-based compensation expense.
5. Non-GAAP selling, general and administrative expenses are defined by us as GAAP selling, general and administrative expenses excluding depreciation expense, and stock-based compensation expense.

The following components are used to adjust our GAAP financial measures into the previously defined non-GAAP measurements:

1. Interest, depreciation and amortization - Interest income (expense), net amounts can vary substantially from period to period due to changes in cash and debt balances and interest rates driven by market conditions outside of our operations. Depreciation expense can vary substantially from period to period as the purchases of property and equipment may vary significantly from period to period and without any direct correlation to our operating performance. Amortization expense primarily associated with patent costs are amortized over a period of several years after acquisition or patent application or renewal.
2. Stock-based compensation expenses - Stock-based compensation expenses represent non-cash charges related to equity awards we have granted.

Investor Contacts:
Ian Estepan, 617-274-4052, iestepan@sarepta.com
Ryan Wong, 617-800-4112, rwong@sarepta.com
Tam Thornton, 617-803-3825, tthornton@sarepta.com

Media Contacts:
Tracy Sorrentino, 617-301-8566, tsorrentino@sarepta.com
Kara Hoeger, 617-710-3898, khoeger@sarepta.com