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Dyne Reports Progress Building Next-Generation Exon Skipping Franchise In DMD

 
• By Jessica Merrill

The company reported updated data from the Phase I/II DELIVER trial testing DYNE-251 in patients with Duchenne muscular dystrophy (DMD) and announced leadership changes.

efficacy
Dyne's DMD drug continues to show efficacy in Phase I/II • Source: Shutterstock

The drug development focus in Duchenne muscular dystrophy (DMD) has been on gene therapy recently, but Dyne Therapeutics is working to develop next-generation exon-skipping medications that would compete with Sarepta Therapeutics, Inc.’s established exon-skipping franchise, including Exondys 51 (eteplirsen).

Key Takeaways
  • Updated data from a Phase I/II clinical trial DELIVER testing DYNE-251 in patients with DMD demonstrated greater dystrophin expression than what was seen in clinical trials for Sarepta’s Exondys 51.
  • Dyne’s stock price dropped, however, on the news, with investors possibly shaken by coinciding leadership news

The company announced updated data on 3 September from the ongoing Phase I/II DELIVER trial testing DYNE-251 in patients with DMD who are amenable to exon 51 skipping that demonstrated functional improvement and greater dystrophin expression than what was seen in

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