Dyne Therapeutics' Registrational Expansion Cohort Of Phase 1/2 DELIVER Trial Of Zeleciment Rostudirsen For Duchenne Muscular Dystrophy Met Primary Endpoint

Benzinga · 1d ago
  • Registrational Expansion Cohort (REC) met primary endpoint, demonstrating statistically significant increase in dystrophin to 5.46% at 6 months (muscle content-adjusted; p<0.0001), replicating the same 7-fold change from baseline previously observed at the registrational dose.
  • Functional improvement was observed across multiple clinical endpoints at 6 months in REC; lung function was preserved at 6 months.
  • New positive long-term results from DELIVER trial showed sustained functional improvement across all assessed endpoints through 24 months.
  • Continued favorable safety and tolerability profile.
  • Submission for U.S. Accelerated Approval on track for Q2 2026