Dyne Therapeutics Shares Rise After Announcing Positive Topline Results for z-rostudirsen in Phase 1/2 DMD Trial

Dyne Therapeutics Inc. (NASDAQ:DYN) is one of the high short interest stocks to buy right now. On December 8, Dyne Therapeutics announced positive topline results from the Registrational Expansion Cohort of its Phase 1/2 DELIVER trial. The trial is evaluating zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in individuals with Duchenne muscular dystrophy/DMD amenable to exon 51 skipping.

The DELIVER trial also provided new positive long-term clinical data from its ongoing open-label extension and long-term extension portions. Z-rostudirsen is an investigational therapeutic using Dyne’s FORCE platform, designed to enable the production of near full-length dystrophin by conjugating a phosphorodiamidate morpholino oligomer to an antigen-binding fragment that binds to the transferrin receptor 1.

The drug has received Breakthrough Therapy, Fast Track, and Rare Pediatric Disease designations from the US FDA, as well as Orphan Drug designation from the FDA, EMA, and MHLW in Japan. The REC met its primary endpoint and showed a statistically significant increase in muscle content-adjusted dystrophin expression to 5.46% of normal relative to baseline at 6 months. Functional improvement was observed across all 6 prespecified clinical endpoints at 6 months relative to baseline and declines in the pooled placebo group. Dyne is on track to submit a Biologics License Application/BLA for US Accelerated Approval in Q2 2026.

Dyne Therapeutics Inc. (NASDAQ:DYN) is a clinical-stage neuromuscular disease company that discovers and develops therapeutics for neuromuscular diseases in the US.

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Disclosure: None. This article is originally published at Insider Monkey.