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Solid Data For Sarepta’s Next-Gen PPMO Treatment But Future Uncertain

 
• By Jessica Merrill

A Phase II study testing a next-generation exon skipping medicine vesleteplirsen in Duchenne muscular dystrophy amendable to exon 51 outpaced Sarepta’s Exondys 51.

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Sarepta isn't sure about the path ahead for its new exon skipping drug • Source: Shutterstock

Sarepta Therapeutics, Inc.’s next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) treatment SRP-5051, or vesleteplirsen, demonstrated a notable efficacy improvement over the company’s current gold standard for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping, Exondys 51 (eteplirsen), in a Phase II study.

Key Takeaways
  • Vesleteplirsen demonstrated a notable efficacy improvement over Sarepta’s current gold standard for Duchenne muscular dystrophy (DMD), Exondys 51 (eteplirsen), in a Phase II study.
  • Sarepta plans to discuss the data with the FDA in a meeting it expects will occur in the third quarter

The future development, regulatory and commercial path for vesleteplirsen depends on discussions with regulators about the data and the outcome of the US Food and Drug Administration’s ongoing review of Sarepta’s gene therapy for DMD,

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