Dyne Posts 9.48% Dystrophin Increase, On Track for Q2 2026 BLA Submission
Dyne Therapeutics achieved average unadjusted dystrophin production of 9.48% (18.33% content-adjusted) after 67–104 weeks in its Phase 1/2 DELIVER trial with 86 patients and confirmed a favorable safety profile. The company completed a positive pre-BLA meeting for exon 51 DMD therapy, plans BLA submission in Q2 2026 and initiated a global Phase 3 HARMONIA trial.
1. Phase 1/2 DELIVER Dystrophin and Safety Results
Dyne reported average unadjusted dystrophin production of 9.48% of normal (18.33% muscle content-adjusted) after 67–104 weeks in four participants dosed at 20 mg/kg Q4W and observed a favorable safety profile across 86 enrolled individuals, with no related serious adverse events and predominantly mild-to-moderate pyrexia and headache.
2. Pre-BLA Meeting and BLA Submission Timeline
The company completed a positive pre-BLA meeting with the FDA for its exon 51 Duchenne muscular dystrophy candidate and remains on track to submit a Biologics License Application in Q2 2026, aiming for Priority Review and potential U.S. launch in Q1 2027.
3. ACHIEVE Registrational Expansion Cohort Exceeds Enrollment
Dyne reached its target of 60 participants in the Phase 1/2 ACHIEVE registrational expansion cohort of z-basivarsen for myotonic dystrophy type 1, with accelerated screening expected to push total enrollment above 60 by the end of Q2 2026.
4. Initiation of Global Phase 3 HARMONIA Trial
In March 2026, Dyne initiated the global confirmatory Phase 3 HARMONIA trial for z-basivarsen under an FDA-aligned protocol, advancing toward pivotal efficacy data to support a potential BLA submission in early Q3 2027.