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Dyne Therapeutics: First DMD Patient Dosed in Phase 1/2 DELIVER Clinical Trial of DYNE-251

September 8, 2022

Dyne Therapeutics has achieved dosing the first patient in their Phase 1/2 clinical trial, DELIVER, evaluating DYNE-251 for the treatment of Duchenne muscular dystrophy mutations amenable to exon 51 skipping, as well as the initiation of their ACHIEVE Phase 1/2 clinical trial evaluating DYNE-101 for the treatment of myotonic dystrophy type 1 (DM1).

The DELIVER trial is a Phase 1/2 global clinical trial evaluating DYNE-251, consisting of 24-week multiple ascending doses (MAD) randomized placebo-controlled period, a 24-week open-label extension and a 96-week long-term extension. The trial, which is designed to be registrational, is expected to enroll approximately 46 ambulant and non-ambulant males with DMD who are ages 4 to 16 and have mutations amenable to exon 51 skipping therapy. The primary endpoints are safety, tolerability and change from baseline in dystrophin levels as measured by Western blot. Secondary endpoints include measures of muscle function, exon skipping, and pharmacokinetics. Dyne anticipates reporting data from the MAD placebo-controlled portion of the DELIVER trial on safety, tolerability and dystrophin in the second half of 2023. 

Trial sites are currently in Georgia and Massachusetts in the United States. Defeat Duchenne Canada will continue to work with Dyne to initiate trial sites in Canada.

Join us at our Family Forum on November 4-6, 2022, where Dyne Therapeutics will provide additional updates and an opportunity for Q&A.