Clinical TrialJun 3, 2026, 07:31 AM
Dyne Completes Enrollment in DM1 Trial; Topline Data Expected Q1 2027
AI Summary
Dyne Therapeutics announced the completion of enrollment in the registrational expansion cohort of its Phase 1/2 ACHIEVE trial for zeleciment basivarsen (DYNE-101) in patients with Myotonic Dystrophy Type 1 (DM1). Topline data from this cohort are anticipated in Q1 2027, supporting a potential Biologics License Application (BLA) submission for U.S. Accelerated Approval in Q3 2027. The company aims for a potential U.S. launch of z-basivarsen in H1 2028, pending FDA Priority Review and approval.
Key Highlights
- Completed enrollment in the registrational expansion cohort (71 participants) of the Phase 1/2 ACHIEVE trial for zeleciment basivarsen (DYNE-101) in DM1.
- Topline data from the ACHIEVE registrational expansion cohort expected in Q1 2027.
- Potential Biologics License Application (BLA) submission for U.S. Accelerated Approval planned for Q3 2027.
- Anticipated potential U.S. launch of z-basivarsen in H1 2028, assuming FDA Priority Review and approval.
- Z-basivarsen has Breakthrough Therapy, Orphan Drug, and Fast Track designations from the U.S. FDA.
- The ACHIEVE trial evaluates 6.8 mg/kg z-basivarsen administered every eight weeks.
- Confirmatory Phase 3 HARMONIA trial for z-basivarsen in DM1 is ongoing.
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