Sarepta Therapeutics Announces Major Restructuring and Pipeline Shift Amid Safety Concerns

Sarepta Therapeutics, a leading gene therapy company, has unveiled a significant restructuring plan that includes substantial layoffs and a strategic pivot in its research focus. This move comes as the company grapples with safety concerns surrounding its flagship product, Elevidys, for Duchenne muscular dystrophy (DMD).

Workforce Reduction and Financial Restructuring

Sarepta has announced the layoff of 500 employees, representing 36% of its workforce. This drastic measure is part of a broader restructuring effort aimed at reducing operating expenses and improving financial flexibility. The company expects these changes to result in annual savings of approximately $400 million.

The restructuring also involves leadership changes, with several key executives taking on new roles. Patrick Moss has been appointed as the new Chief Commercial Officer, replacing outgoing Chief Customer Officer Dallan Murray. Other notable appointments include Ian Estepan as Chief Operating Officer, Louise Rodino-Klapac, Ph.D., as President of R&D and Technical Operations, Ryan Wong as Chief Financial Officer, and Rachael Potter, Ph.D., as Chief Scientific Officer.

Strategic Pipeline Shift

In a significant pivot, Sarepta is discontinuing development of several gene therapy programs, including most of those targeting limb-girdle muscular dystrophy (LGMD). Instead, the company will prioritize its siRNA platform, focusing on investigational treatments for myotonic dystrophy type 1, idiopathic pulmonary fibrosis, and Huntington's disease.

Sarepta will continue its partnership with Arrowhead Pharmaceuticals to develop therapies for skeletal muscle diseases. For deprioritized programs, the company plans to explore strategic alternatives, including potential partnerships.

Elevidys Safety Concerns and FDA Action

The restructuring announcement coincides with the FDA's request for a black box warning for acute liver injury to be added to the label of Elevidys, Sarepta's gene therapy for DMD. This action follows the deaths of two teenage patients who developed acute liver failure within two months of receiving the treatment.

In response to these safety concerns, Sarepta has halted Elevidys administration to non-ambulatory patients in the U.S. commercial setting. The company is also seeking FDA approval for enhanced risk mitigation measures, including the use of the immunosuppressant sirolimus to manage liver toxicity.

Additionally, Sarepta has voluntarily paused dosing in its phase 3 ENVISION trial for DMD. The company is working on a new protocol with an enhanced immunosuppressive regimen for non-ambulatory patients, which will require FDA approval before screening and dosing can resume.