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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.

The future development, regulatory and commercial path for vesleteplirsen depends on discussions with regulators about the data and the outcome...

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