Sarepta to pause development of LGMD programs

Sarepta (SRPT) said it will continue to support its four on-market Duchenne therapies and all associated clinical trial commitments and evidence-generation activities. Revenues from this robust Duchenne portfolio are expected to continue driving profitability and funding a focused pipeline of high-impact development programs primarily leveraging Sarepta’s potentially best-in-class siRNA platform. This strategic pivot emphasizes chronically administered therapies for neurodegenerative and pulmonary diseases. 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 the Biologics License Application for SRP-9003 for LGMD type 2E/R4 in the second half of this year. Sarepta intends to seek strategic alternatives, including partnering, for programs that it no longer intends to fund directly. The siRNA programs include investigational treatments for: Facioscapulohumeral muscular dystrophy; Myotonic dystrophy type 1; Spinocerebellar ataxia type 2; Idiopathic Pulmonary Fibrosis, and; Huntington’s disease.