Sarepta Therapeutics announced a strategic restructuring plan aimed at prioritizing high-impact programs and ensuring long-term financial sustainability. The company is focusing its pipeline on potentially best-in-class siRNA platform assets, with the goal of delivering sustainable growth while meeting its 2027 financial obligations.
The restructuring includes reducing operating expenses by approximately $400 million annually and implementing a 36% workforce reduction, affecting around 500 employees. Despite these changes, Sarepta’s Duchenne portfolio continues to perform strongly, with preliminary second-quarter 2025 net product revenue reported at $513 million.
Regarding ELEVIDYS (delandistrogene moxeparvovec), the first approved gene therapy for Duchenne muscular dystrophy, Sarepta has agreed to the U.S. FDA’s request to include a black box warning for acute liver injury and failure, which resolves material issues concerning the ambulant population label. Sarepta paused shipments for non-ambulant patients to explore enhanced immunosuppression regimens and will soon submit a protocol developed by an Expert Committee to the FDA to resume dosing in that group.
“Faced with environmental changes, we have decided to act decisively, implementing a focused strategy to ensure Sarepta remains a vibrant, financially enduring, patient-centric organization dedicated to improving the lives of those with rare genetic diseases,” said Doug Ingram, CEO. He emphasized continued commitment to ELEVIDYS, the three PMOs for Duchenne, and advancing a focused siRNA pipeline while meeting financial targets.
Financial highlights for Q2 2025 include:
- Total net product revenue: $513 million
- ELEVIDYS revenue: $282 million
- RNA-based PMOs revenue: $231 million
- Combined R&D and SG&A expenses (non-GAAP): $294 million
- Cash and investments: approximately $850 million
The company will continue supporting its four approved Duchenne therapies while pivoting toward siRNA programs targeting diseases such as facioscapulohumeral muscular dystrophy, myotonic dystrophy type 1, spinocerebellar ataxias, idiopathic pulmonary fibrosis, and Huntington’s disease. Sarepta will pause most gene therapy programs for limb-girdle muscular dystrophy and seek strategic alternatives for these assets.
Additionally, Sarepta announced key executive appointments, including Ian Estepan as President and COO, Louise Rodino-Klapac, Ph.D., as President of R&D and Technical Operations, and Ryan Wong as CFO, among others.
This restructuring and refocused pipeline strategy position Sarepta for long-term growth while ensuring robust financial health and continued innovation for patients with rare genetic diseases.
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