Sarepta Announces Restructuring as It Lays Off 500 Employees

Rare Daily Staff

Sarepta Therapeutics, which saw its stock tumble following news in June that a second non-ambulatory person had died from acute liver failure after treatment with its Duchenne muscular dystrophy gene therapy Elevidys, announced a restructuring plan to prioritize high-value, high-impact programs, meet its 2027 financial obligations, and support its long-term financial viability.

This plan, which includes laying off about 500 employees, or 36 percent of its workforce, focuses on the opportunities from the company’s siRNA platform. The changes are expected to save Sarepta about $400 million a year.

As a result of this reprioritization, several programs, including most of the gene therapies in development for limb-girdle muscular dystrophy, will be paused. Sarepta expects to submit an application to the U.S. Food and Drug Administration to market SRP-9003 for limb-girdle muscular dystrophy type 2E/R4 in the second half of 2025. It intends to explore strategic alternatives, including partnerships, for programs that it no longer plans to fund directly.

The siRNA programs the company is continuing to pursue include experimental therapies for facioscapulohumeral muscular dystrophy, myotonic dystrophy type 1, spinocerebellar ataxia type 2, idiopathic pulmonary fibrosis, and Huntington’s disease.

In addition to the restructuring plan, the company said that, following previously communicated steps to strengthen the safety profile of Elevidys, the FDA has requested that the label for the gene therapy include a black box warning for acute liver injury and acute liver failure. Sarepta said it agrees with this change, which appears to resolve any material issues with the ambulatory portion of the Elevidys label.

Sarepta paused shipments of Elevidys for non-ambulatory patients while it explored the adoption of additional prophylactic immunosuppression. To that end, Sarepta convened an expert committee of neuromuscular specialists, hepatologists, hematologists, and immunologists to review cases of acute liver failure and explore additional immunosuppression regimens.

The committee agreed on an enhanced immunosuppressive regimen with sirolimus for Elevidys in non-ambulatory patients. Sarepta will submit the findings of the expert panel and proposed protocol to the FDA and will discuss a proposal to gather data on the regimen in a new cohort of the ENDEAVOR study as a pathway to re-establish dosing in the non-ambulatory setting.

Additionally, Sarepta said it is assessing opportunities for generating real-world data in ambulatory patients through investigator-initiated trials.

Collectively, these measures are projected to deliver approximately $400 million in annual cost reductions, significantly lowering the company’s average annual non-GAAP R&D and SG&A expenses to between $800 million and $900 million starting in 2026.

“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 of Sarepta Therapeutics. “These changes will ensure we remain a financially strong and profitable organization built on a sharpened and focused strategy.”

Photo: Doug Ingram, CEO of Sarepta Therapeutics