Cambridge, MA — July 16, 2025: Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a leader in precision genetic medicine, has announced a major strategic restructuring as it sharpens operational focus, streamlines its development pipeline, and implements cost-saving measures to ensure long-term sustainability. This decisive move comes as the company adapts to evolving market and regulatory environments, with a renewed emphasis on its promising small interfering RNA (siRNA) platform, robust management of finances, and continuing support for its flagship Duchenne muscular dystrophy (DMD) therapies.

Strategic Restructuring: Cost Control and Workforce Reductions

In one of the most significant transformations in its corporate history, Sarepta will reduce its operating expenses by an anticipated $400 million annually, effective from 2026. Central to this initiative is a sweeping 36% reduction in workforce, involving approximately 500 employees. The company expects this action to generate about $120 million in annual cash savings starting next year. Non-personnel related cost reductions and pipeline reprioritization will contribute a further estimated $300 million in annual savings.

These measures, which include a rigorous realignment of research and development (R&D) as well as selling, general, and administrative (SG&A) expenditures, are designed to reposition Sarepta for prolonged financial health. During a time when capital markets have placed a premium on profitability and operational discipline in biotech, Sarepta’s restructuring signals a strategic pivot toward rigorous financial stewardship and a narrower, high-impact development agenda.

Pipeline Prioritization: Focus on siRNA Technology

Sarepta’s future growth strategy centers on prioritizing its siRNA platform—an innovative gene-silencing approach expected to unlock novel treatments for a range of severe and currently untreatable disorders. While the company continues to support its established Duchenne therapy portfolio—driven by both gene therapy (ELEVIDYS) and RNA-based phosphorodiamidate morpholino oligomers (PMOs)—the primary clinical and commercial investments will now be devoted to siRNA assets.

The focused pipeline includes investigational therapies for key neuromuscular and neurodegenerative diseases, such as:

• Facioscapulohumeral muscular dystrophy (FSHD)
• Myotonic dystrophy type 1 (DM1)
• Spinocerebellar ataxias types 1, 2, and 3 (SCA1, SCA2, SCA3)
• Idiopathic Pulmonary Fibrosis (IPF)
• Huntington’s disease

Notably, most gene therapy programs for limb-girdle muscular dystrophy (LGMD) will be paused, except for a near-term regulatory submission for SRP-9003 in LGMD type 2E/R4. Sarepta also plans to explore strategic partnerships or collaborations for projects no longer at the forefront of its funding priorities, keeping in line with industry trends where large biotechs increasingly turn to external partnerships to extend innovation without increasing financial risk.

ELEVIDYS: Label Update, Enhanced Safety, and FDA Alignment

ELEVIDYS (delandistrogene moxeparvovec-rokl), Sarepta’s flagship single-dose AAV-based gene therapy for DMD, remains a cornerstone of its revenue and innovation engine. As the only approved gene therapy for DMD in the U.S., ELEVIDYS reported strong performance in the second quarter of 2025, generating $282 million in revenue. Sarepta’s PMO therapies also remained strong, bringing in $231 million for the quarter.

On the regulatory front, Sarepta has agreed with the U.S. Food and Drug Administration (FDA) to update the ELEVIDYS label to include a black box warning concerning the risks of acute liver injury and acute liver failure—an increasingly common requirement for AAV-based gene therapies. While such a warning may heighten physician vigilance, the company notes this update resolves material questions about the ambulant population’s eligibility and paves the way for ongoing commercial availability.

Shipments of ELEVIDYS for non-ambulant patients had previously been paused after concerns regarding acute liver injury events. To address this, Sarepta convened a panel of neuromuscular, hepatic, hematologic, and immunologic specialists to review safety data and recommend enhanced immunosuppression protocols, particularly involving sirolimus, for non-ambulant patients. Sarepta is set to submit these expert recommendations and enhanced safety protocols to the FDA and discuss a new clinical cohort (Cohort 8 in ENDEAVOR study SRP-9001-103) as a path toward resuming ELEVIDYS shipments for these patients. Additional investigator-initiated trials and real-world evidence collection are planned for ambulant patient populations.

Financial Results: Q2 2025 Performance and Outlook

Preliminary results for the second quarter ended June 30, 2025, underscore the resilience of Sarepta’s commercial business:

• Total net product revenue: $513 million
• ELEVIDYS revenue: $282 million
• RNA-based PMO therapies: $231 million
• R&D and SG&A expenses (GAAP): $338 million; Non-GAAP: $294 million
• Cash, cash equivalents, and investments: ~$850 million

Sarepta’s robust cash position, coupled with the planned savings from restructuring, should provide ample runway for its renewed strategic focus. The company maintains access to a $600 million revolving credit facility, further supporting liquidity as it manages upcoming obligations, including repayment of its 2027 convertible note.

It’s important to note that these financial results are preliminary; full audited figures will be disclosed in early August 2025.

Leadership Updates and Corporate Governance

As part of the organizational transformation, Sarepta announced several executive changes:

• Ian Estepan as President and COO
• Louise Rodino-Klapac, Ph.D., as President of Research & Development and Technical Operations
• Ryan Wong as Chief Financial Officer
• Rachael Potter, Ph.D., as Chief Scientific Officer
• Patrick Moss, Pharm.D., as Chief Commercial Officer

These appointments are designed to align leadership capabilities with Sarepta’s streamlined mission and the evolving requirements of a large-cap biotechnology company with global aspirations.

Outlook: Balancing Innovation and Operational Discipline

Sarepta Therapeutics’ restructuring reflects a broader trend in the global biopharma industry, where companies are pivoting toward more capital-efficient models, targeted development strategies, and greater risk management after an era of expansive R&D portfolios. The focus on siRNA and strategic pipeline contraction align Sarepta with investor demand for sustainable profitability and innovation with tangible near- and mid-term commercial potential.

As the company continues its mission of delivering life-changing genetic medicines for rare diseases, investors, patients, and partners will be watching closely how the merging of innovation, financial stewardship, and regulatory collaboration shapes Sarepta’s future milestones.

For more information or to access the replay of the July 16 investor call, visit the Sarepta investor relations website at https://investorrelations.sarepta.com/events-presentations.