As an early investor in Dyne Therapeutics, we are pleased to share their exciting news that skipping exon 51 in the dystrophin gene with z-rostudirsen in the registrational expansion cohort met the primary endpoint–a statistically significant increase in dystrophin at 6 months—and was also associated with functional improvements in multiple measurements.
The Details
The DELIVER trial was a randomized, placebo-controlled, double-blind, Phase 1/2 trial that evaluated the safety, tolerability and efficacy of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in individuals with Duchenne who are amenable to exon 51 skipping. DELIVER consisted of two parts:
- Part 1 was a multiple ascending dose (MAD) study, designed to identify the appropriate dose of z-rostudirsen in small numbers of participants
- Part 2 was the Registrational Expansion Cohort (REC), designed to test the selected dose of z-rostudirsen in a larger number of participants to evaluate safety and efficacy. Trial participants who have completed the MAD and the REC continue to receive z-rostudirsen as part of the open label extension portion of the trial.
Along with a favorable safety and tolerability profile, positive topline results from DELIVER Registrational Expansion Cohort (REC) of 32 participants include:
- Dystrophin: The REC met its primary endpoint, demonstrating a statistically significant increase in dystrophin protein expression of 5.46% (muscle content-adjusted) at 6 months. This replicated the same 7-fold change from baseline previously observed.
- Function: Improvement relative to placebo was seen across all six prespecified endpoints at six months. The six endpoints cover upper and lower limb function, as well as lung function.
In addition, new positive long-term results from DELIVER showed sustained functional improvement across multiple endpoints out to 24 months in each of the six endpoints for participants in the MAD cohort.
What comes next?
Dyne plans to submit a potential Biologics License Application (BLA) for U.S. Accelerated Approval in Q2 2026. If the FDA issues priority review and all goes well, the timing could point towards a potential commercial launch in Q1 2027.
Dyne also plans to initiate a global Phase 3 clinical trial of z-rostudirsen in Q2 2026, to support approvals outside of the US.
Link to press release: https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-positive-topline-results-phase-12/
Link to community letter:
https://www.dyne-tx.com/community-letter-deliver-clinical-trial/
The post Dyne announces positive topline results from Phase 1/2 trial of z-rostudirsen in Duchenne amenable to skipping exon 51 appeared first on CureDuchenne.