Sarepta Therapeutics Announces Second Quarter 2025 Financial Results and Recent Corporate Developments

• ELEVIDYS shipments have resumed for ambulatory individuals living with Duchenne following notification from the U.S. Food and Drug Administration (FDA); continuing to work with FDA on safety labeling process and risk-mitigation approach for non-ambulatory individuals
• Net product revenues for the second quarter 2025 totaled $513.1 million, a 42% increase over the same quarter of the prior year
• Previously announced restructuring advancing as planned; on track to realize over $100 million in cost savings through the end of 2025
• Multiple clinical data readouts and milestones expected in 2025 and into early 2026 to support siRNA franchise - FSHD, DM1, SCA2, and Huntington’s Disease

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

“We are very pleased that following a rapid review of the safety data, the FDA swiftly recommended that we take the ambulatory patient population off shipment pause and, following that, we have already resumed deliveries. Infusions are taking place for the ambulatory community. We will continue to work with the FDA to define the path and develop the risk mitigation necessary to bring ELEVIDYS back to the non-ambulatory community as well,” said Doug Ingram, chief executive officer, Sarepta. “With the temporary pause lifted, the execution of our restructuring, cost savings and plans designed to meet our financial obligations, and with important siRNA readouts expected later this year, we are well positioned to achieve our strategic objective to remain a patient-centric, financially disciplined organization into the next decade and beyond.”

Corporate Highlights

ELEVIDYS Updates

• On July 28, 2025, the FDA recommended that Sarepta remove its voluntary pause and resume shipments of ELEVIDYS (delandistrogene moxeparvovec) for ambulatory individuals living with Duchenne. The notification came after the FDA completed its review of ELEVIDYS safety data.
• Shipments have resumed so that patient infusions can continue.
• Discussions between FDA and Sarepta are underway on next steps in the safety labeling process for acute liver injury/acute liver failure (ALI/ALF) and risk-mitigation approach for non-ambulatory individuals living with Duchenne.

Financial Updates

• In the second quarter of 2025, the Company delivered strong financial results, reporting both GAAP and non-GAAP operating profit. The quarter was cash flow positive and total cash, cash equivalents and short- and long-term investments increased by $202.8 million from the previous quarter.
• The Company is taking steps to further strengthen its financial foundation and is focused on proactive liability management. Among many things, it is evaluating opportunities to enhance operational efficiency and adjust manufacturing commitments based on latest demand to further strengthen our liquidity position.
• The strategic restructuring announced on July 16, 2025, will sharpen the Company’s focus on high-impact programs, particularly its siRNA platform, while reducing expenses by approximately $400 million annually starting in 2026. These strategic cost reductions and pipeline prioritization are designed to position Sarepta to repay its 2027 convertible notes and continue delivering transformative therapies to patients with rare genetic diseases.

Expected Near-Term Milestones (2025/2026)

• Facioscapulohumeral muscular dystrophy (FSHD)
  • Study Status: Phase 1/2 study of SRP-1001 FSHD1 patients underway; Cohorts 1, 2, and 3 in part 1 are fully enrolled in the single ascending dose (SAD) study
  • Next Milestone: Preliminary data in second half of 2025 focused on safety, DUX4 mRNA knockdown, DUX4 regulated gene expression, and functional assessments
• Myotonic dystrophy type 1 (DM1)
  • Study Status: Phase 1/2 study of SRP-1003 in patients with DM1 underway with recent authorization to dose escalate; Cohorts 1 and 2 in the SAD arm of the study fully enrolled
  • Recently Achieved Collaboration Milestone: In July 2025, the program reached the first of two pre-specified enrollment targets, and the safety review warranted dose escalation in the Phase 1/2 study, triggering $100.0 million milestone payment to Arrowhead Pharmaceuticals, Inc. (Arrowhead)
  • Next Milestone: Preliminary Phase 1 data in second half of 2025; primary endpoint is safety, with key secondary endpoints including DMPK knockdown, DMPK-mediated splice indices, and functional assessments such as Video Hand Opening Time (VHOT)
• Spinocerebellar ataxia type 2 (SCA2)
  • Study Status: Phase 1 study of SRP-1004 in patients with SCA2 underway as a randomized, placebo-controlled, SAD study, and Cohort 1 in this arm of the study has been fully enrolled
  • Next Milestone: On track for First Participant In (FPI), Cohort 2 in the third quarter of 2025
• Huntington’s Disease (HD)
  • Sarepta intends to file its Clinical Trial Application (CTA) for SRP-1005 in HD by the end of 2025 and initiate its clinical trial in the first half of 2026

2025 ASGCT Annual Conference: Sarepta showcased data spanning different age groups including statistically significant functional outcomes for 8- and 9-year-old patients, and new protein expression and safety results in participants 2 years old at the time of treatment.

• Part 2 analysis of EMBARK study of 8- and 9-year-olds (n=14) demonstrated statistically significant differences across all key endpoints including 4.75 points (P=0.0026) on North Star Ambulatory Assessment (NSAA), 6.87 seconds in time-to-rise (TTR) from the floor (P=0.0010), and 4.76 seconds in 10-meter walk/run (10MWR) (P=0.0097) compared to a well-matched external control cohort
• Results from ENDEAVOR study show treatment in 2-year-old participants with ELEVIDYS for Duchenne muscular dystrophy resulted in mean protein expression of 93.87% as measured by western blot in study participants (n=6)

All the posters and presentations are available on the Company’s website here.

As previously announced, there will be no conference call for this quarterly earnings release.

Q2 2025 Financial Highlights¹

Revenues
Total revenues were $611.1 million for the three months ended June 30, 2025, as compared to $362.9 million for the same period of 2024, an increase of $248.2 million. The increase primarily reflects an increase of $160.1 million in net product revenue of ELEVIDYS as a result of its expanded label approval in June 2024. In addition, collaboration and other revenues increased $95.6 million primarily due to $63.5 million of collaboration revenue recognized related to a milestone payment received from F. Hoffman-La Roche Ltd.'s (“Roche”) for the regulatory approval of ELEVIDYS in Japan (the “Japan Approval Milestone”). Furthermore, contract manufacturing revenues and royalty revenues increased $27.0 million and $5.1 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.

Total revenues were $1,355.9 million for the six months ended June 30, 2025, as compared to $776.4 million for the same period of 2024, an increase of $579.5 million. The increase primarily reflects an increase of $401.2 million in net product revenue of ELEVIDYS as a result of its expanded label approval in June 2024. In addition, collaboration and other revenues increased $174.9 million primarily related to the $63.5 million of collaboration revenue recognized from the Japan Approval Milestone and the $112.0 million of collaboration revenue recognized related to Roche's expiration of an option for a program during the six months ended June 30, 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 $38.6 million and $8.8 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 $152.6 million for the three months ended June 30, 2025, as compared to $44.5 million for the same period of 2024, an increase of approximately $108.1 million. Cost of sales (excluding amortization of in-license rights) were $290.1 million for the six months ended June 30, 2025, as compared to $95.1 million for the same period of 2024, an increase of $195.0 million. The increases in both periods primarily reflect the depletion of previously expensed ELEVIDYS inventory, an increased demand for ELEVIDYS following the expanded label approval in June 2024, an increase in products sold to Roche under the Roche collaboration agreement, as well as an increase in the write-offs of certain batches of products not meeting quality specifications for the periods ended June 30, 2025, as compared to the same periods of 2024.

Operating expenses and others
Research and development expenses were $204.4 million for the three months ended June 30, 2025, as compared to $179.7 million for the same period of 2024, an increase of $24.7 million. The increase is primarily due to an increase in ELEVIDYS clinical material expense as well as $13.0 million of costs associated with our settlement agreement with Brammer Bio MA, LLC (“Brammer”), an affiliate of Thermo Fisher Scientific, Inc., to resolve outstanding claims related to the termination of the development, commercial manufacturing, and supply agreement with Brammer (the “Brammer Settlement”). For the three months ended June 30, 2025, non-GAAP research and development expenses were $181.7 million, as compared to $153.9 million for the same period of 2024, an increase of $27.8 million.

Research and development expenses were $977.8 million for the six months ended June 30, 2025, as compared to $380.1 million for the same period of 2024, an increase of approximately $597.7 million. The increase primarily reflects a $583.6 million increase in up-front and milestone expense associated with the licensing and collaboration agreement and stock purchase agreement with Arrowhead, as well as $13.0 million of costs associated with the Brammer Settlement. For the six months ended June 30, 2025, non-GAAP research and development expenses were $930.9 million, as compared to $332.0 million for the same period of 2024, an increase of $598.9 million.

Selling, general and administrative expenses were $137.9 million for the three months ended June 30, 2025, as compared to $138.8 million for the same period of 2024, a decrease of $0.9 million. The decrease is primarily driven by a net decrease in stock-based compensation primarily due to the achievement of performance conditions related to certain PSUs during the three months ended June 30, 2024, partially offset by an increase in professional services used for the commercialization of ELEVIDYS. For the three months ended June 30, 2025, non-GAAP selling, general and administrative expenses were $113.4 million, as compared to $106.0 million for the same period of 2024, an increase of $7.4 million.

Selling, general and administrative expenses were $271.5 million for the six months ended June 30, 2025, as compared to $265.8 million for the same period of 2024, an increase of $5.7 million. The increase is primarily driven by an increase in compensation and other personnel expenses as well as an increase in professional services used for the commercialization of ELEVIDYS, partially offset by a net decrease in stock-based compensation primarily due to the achievement of performance conditions related to certain PSUs during the six months ended June 30, 2024. For the six months ended June 30, 2025, non-GAAP selling, general and administrative expenses were $220.5 million, as compared to $206.5 million for the same period of 2024, an increase of $14.0 million.

Other income (expense), net for the three months ended June 30, 2025 and 2024, was $38.1 million and $14.3 million, respectively. Other (expense) income, net for the six months ended June 30, 2025 and 2024, was $(45.1) million and $20.8 million, respectively. The changes in each period primarily reflect the fair value adjustments of our investments in publicly-traded companies, including Arrowhead, during the periods ended June 30, 2025.

Income tax (benefit) expense for the three months ended June 30, 2025 and 2024, was $(43.3) million and $7.1 million, respectively. Income tax expense for the six months ended June 30, 2025 and 2024, was $20.7 million and $12.4 million, respectively. Income tax (benefit) expense for all periods presented 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 income (loss) is defined by us as GAAP net income (loss) excluding interest income/expense, net, depreciation and amortization expense, stock-based compensation expense, gain/loss on strategic investments, the estimated income tax impact of each pre-tax non-GAAP adjustment and other items.
2. 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. 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.
3. Non-GAAP operating income (loss) is defined by us as GAAP operating income (loss) excluding depreciation and amortization expense, stock-based compensation expense and other items.
4. Non-GAAP research and development expenses are defined by us as GAAP research and development expenses excluding depreciation and amortization expense, stock-based compensation expense and other items.
5. Non-GAAP selling, general and administrative expenses are defined by us as GAAP selling, general and administrative expenses excluding depreciation expense, stock-based compensation expense and other items.

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. Although these are recurring charges to operations, we believe the measurement of these amounts can vary substantially from period to period and depend significantly on factors that are not a direct consequence of operating performance that is within our control. Therefore, we believe that excluding these charges facilitates comparisons of our operational performance in different periods.
3. Other items - We evaluate other items of expense and income on an individual basis. We take into consideration quantitative and qualitative characteristics of each item, including (a) nature, (b) whether the items relate to our ongoing business operations, and (c) whether we expect the items to continue or occur on a regular basis. These other items include the gain/loss on strategic investments and changes in the fair value of derivatives and may include other items that fit the above characteristics in the future. We exclude from our non-GAAP results:

We use these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. We also believe these non-GAAP measures increase comparability of period-to-period results and are useful to investors as they provide a similar basis for evaluating our performance as is applied by management. These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of our financial results in accordance with GAAP. Use of the terms non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP operating (loss) income, non-GAAP net (loss) income, and non-GAAP diluted (loss) earnings per share may differ from similar measures reported by other companies, which may limit comparability, and are not based on any comprehensive set of accounting rules or principles. All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table “Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures.”

About EXONDYS 51
EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion, or “skipping”, of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein.

EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

EXONDYS 51 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental.

Important Safety Information About EXONDYS 51
Hypersensitivity reactions, including bronchospasm, chest pain, cough, tachycardia, and urticaria have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy.

Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 mg or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended.

The most common adverse reactions from observational clinical studies (N=163) seen in greater than 10% of patients were headache, cough, rash, and vomiting.

Other adverse events may occur.

To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc. at 1-888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

For further information, please see the full US Prescribing Information for EXONDYS 51 (eteplirsen).

About VYONDYS 53
VYONDYS 53 (golodirsen) uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 53 of dystrophin pre-mRNA, resulting in exclusion, or “skipping,” of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein.

VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

VYONDYS 53 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental.

Important Safety In

Investor Contact:
Ian Estepan, 617-274-4052
iestepan@sarepta.com

Media Contact:
Tracy Sorrentino, 617-301-8566
tsorrentino@sarepta.com