Sarepta Therapeutics Faces Setbacks: Adds Black Box Warning to Elevidys, Announces Major Layoffs

Sarepta Therapeutics, a Cambridge, Massachusetts-based biotech company, has announced significant changes following recent setbacks with its gene therapy treatment for Duchenne muscular dystrophy (DMD). The company is adding a black box warning to Elevidys and restructuring its workforce and pipeline in response to recent patient deaths and regulatory scrutiny.

Black Box Warning Added to Elevidys

Sarepta has added a black box warning to Elevidys, its gene therapy for DMD, specifically for acute liver injury and acute liver failure. This decision comes after the deaths of two teenage patients with DMD who had received Elevidys treatment. The first death occurred in March and was linked to acute liver failure, a known adverse effect of adeno-associated virus (AAV) vector-based gene therapies. A second teenage patient death in June, also associated with acute liver failure, prompted Sarepta to temporarily halt shipments for non-ambulatory patients.

In response to these events, Sarepta has completed an expert committee review to develop a new protocol for additional prophylactic immunosuppression in non-ambulatory patients. The company plans to submit this protocol to the FDA "imminently" with the aim of resuming Elevidys shipments to this patient group. The FDA has opened an investigation into the gene therapy following the second patient death.

Workforce Reduction and Pipeline Reprioritization

Sarepta has announced a significant restructuring effort, which includes laying off approximately 500 employees, representing more than a third of its workforce. This decision follows a strategic review of the company's operations and pipeline.

The restructuring will involve:

1. Focusing on "high-impact programs" and prioritizing "potentially best-in-class siRNA platform assets," including programs for facioscapulohumeral muscular dystrophy, idiopathic pulmonary fibrosis, and Huntington's disease.
2. Pausing several programs, including most gene therapies in development for limb-girdle muscular dystrophy (LGMD). However, Sarepta still plans to submit a biologics license application for SRP-9003 for LGMD type 2E/R4 in the second half of this year.
3. Generating an estimated $120 million in annual cash cost savings by 2026 through the layoffs.
4. Achieving approximately $300 million in annual non-personnel cost savings through pipeline reprioritization, beginning next year.

CEO Doug Ingram stated, "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."

Despite these challenges, Sarepta's DMD portfolio, including Elevidys, brought in $513 million for the second quarter, according to the company's press release.