Restructuring Highlights: Workforce Reduction and Pipeline Prioritization

As a result of a rigorous strategic review, Sarepta announced significant organizational changes, including a 36% reduction in workforce—approximately 500 employees—in a bid to cut estimated annual operating costs by $400 million starting in 2026. The reduction affects both personnel and non-personnel costs, with approximately $120 million anticipated in annual cash savings from labor and $300 million from a more focused R&D pipeline.

The cost reduction plan is directly tied to Sarepta’s need to address its 2027 convertible note and to bolster access to its $600 million revolving credit facility—underpinning its path to sustainable profitability and operational resilience.

Financial Performance: Robust Revenues Despite Industry Headwinds

Sarepta continues to deliver solid financial results even amid restructuring. For the second quarter of 2025, the company reported preliminary total net product revenue of $513 million. Of this, the RNA-based PMOs contributed $231 million, while ELEVIDYS, Sarepta’s transformative gene therapy for Duchenne muscular dystrophy (DMD), accounted for $282 million—a testament to the company’s dominant market position in DMD therapeutics.

• Combined research and development (R&D), selling, general and administrative (SG&A) expenses: $338 million GAAP, $294 million non-GAAP (excluding $34 million in stock-based compensation and $10 million in depreciation/amortization).
• Cash reserves: Approximately $850 million in cash, cash equivalents, restricted cash, and investments, providing liquidity to weather industry volatility and fund strategic initiatives.

These preliminary results reflect the company’s ongoing strength in its flagship Duchenne portfolio and lay the foundation for future investments in high-value pipeline assets.

ELEVIDYS Update: Black Box Warning and Safety Enhancements

ELEVIDYS (delandistrogene moxeparvovec), the first and only gene therapy approved for DMD, remains a revenue cornerstone for Sarepta. In light of ongoing safety evaluations and at the request of the U.S. Food and Drug Administration (FDA), the ELEVIDYS label will now include a black box warning for acute liver injury (ALI) and acute liver failure (ALF). Sarepta has cooperated with the FDA, which resolves critical issues for the ambulant (mobile) DMD population that ELEVIDYS serves.

Amid additional scrutiny, Sarepta voluntarily paused shipments of ELEVIDYS for non-ambulant patients pending the adoption of enhanced immunosuppression regimens. An expert committee of leading specialists in neuromuscular, hepatic, hematologic, and immunologic disorders has recommended a customized immunosuppressive protocol using sirolimus for these patients. The company will imminently submit these recommendations to the FDA and seek to resume dosing for non-ambulant DMD populations via a new cohort in its ENDEAVOR (SRP-9001-103) study. This approach underscores Sarepta’s commitment to both safety and regulatory compliance.

In addition to addressing regulatory requirements, Sarepta continues to support real-world data generation for approved indications and to prioritize patient safety through rigorous post-marketing surveillance.

Pipeline Evolution: Strategic Pivot to siRNA Platform

The most consequential change in Sarepta’s pipeline strategy is a decisive focus on high-impact, near- and mid-term programs based around its potentially best-in-class siRNA (small interfering RNA) platform. The siRNA modality has gained momentum in biopharma for its utility in selectively silencing disease-causing genes, enabling more precise, chronic treatments for genetic and neurodegenerative disorders.

Sarepta will continue to commercialize and generate evidence for its marketed Duchenne therapies while concentrating future R&D investments on siRNA-driven treatments. Several lower-priority gene therapies—for example, those developed for limb-girdle muscular dystrophy (LGMD)—will be paused, with the company exploring strategic alternatives such as out-licensing or partnerships for these assets.

Actively advancing programs in the siRNA portfolio include candidates for:

• Facioscapulohumeral muscular dystrophy (FSHD)
• Myotonic dystrophy type 1 (DM1)
• Spinocerebellar ataxia type 2 (SCA2)
• Idiopathic pulmonary fibrosis (IPF)
• Huntington’s disease

Sarepta is also maintaining a robust pipeline of preclinical programs, such as those targeting spinocerebellar ataxia types 1 and 3, and retains an exclusive development partnership with Arrowhead Pharmaceuticals, focused on skeletal muscle and central nervous system targets.

Gene therapy for LGMD type 2E/R4 remains on track, with the Biologics License Application (BLA) for SRP-9003 planned for late 2025, but most other gene therapy programs will be set aside unless viable partnership opportunities arise.

Leadership Realignment to Drive New Strategy

The reorganization goes hand-in-hand with leadership changes meant to guide Sarepta through this new chapter. Newly appointed President and Chief Operating Officer Ian Estepan now oversees company operations, while Dr. Louise Rodino-Klapac steps up as President of Research & Development and Technical Operations. Ryan Wong becomes Chief Financial Officer, and Dr. Rachael Potter is promoted to Chief Scientific Officer. Patrick Moss is named Chief Commercial Officer. The leadership team is tasked with executing the restructuring, accelerating siRNA platform development, and building new collaborative partnerships to monetize legacy assets.

Industry and Market Context

This transformation comes at a pivotal moment for the biotech sector. Increasing R&D costs, price pressure, healthcare policy shifts, and scrutiny over the safety of gene therapies are forcing companies to demonstrate capital efficiency, accelerate time-to-market, and prioritize therapies with clear differentiation and robust addressable markets. Sarepta’s overhaul is consistent with broader biopharma trends, where platform-based approaches are being prioritized for both commercial scalability and pipeline flexibility.

Sarepta’s Duchenne franchise remains resilient, underlining the importance of continuing to deliver on near-term patient needs while maintaining a disciplined focus on innovation. The company’s renewed financial discipline and tight pipeline focus aim to bring high-value treatments to rare disease patients while optimizing returns for shareholders.

Forward-Looking Guidance and Investor Updates

Sarepta will provide more detailed financials and discuss the company’s path forward—in light of these changes—during its scheduled investor webcast on July 16, 2025. Investors and stakeholders can access the event and review archived materials on Sarepta’s Investor Relations website.

The company cautions that all forward-looking statements are subject to risk given fast-moving industry and regulatory conditions. Nonetheless, Sarepta’s overhaul is intended to safeguard its dominant position in rare disease therapeutics, accelerate the most promising treatments to market, and deliver value to patients and investors alike in the years ahead.