Dyne Therapeutics announce application to start exon 51 skipping clinical trial

Dyne Therapeutics, Inc. announced on 2nd December 2021 the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) to initiate a clinical trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) amenable to skipping exon 51. Dyne expects to begin dosing patients in clinical trials for DMD by mid-year 2022

The IND application for DYNE-251 includes in vivo data from recent studies in the mdx mouse model and in non-human primates, and outlines plans for a global, randomized, placebo controlled, multiple ascending dose (MAD) clinical trial with a long term extension study. The Phase 1/2 trial aims to enroll approximately 30 to 40 ambulant and non-ambulant male patients ages 4 to 16 with symptomatic DMD and mutations amenable to exon 51 skipping therapy.

Planned endpoints include safety and tolerability, PK/PD, dystrophin expression as measured by Western Blot, and measures of muscle function.  

In addition to DYNE-251, Dyne is building a DMD franchise with programs for patients with mutations amenable to skipping exons 53, 45 and 44. 

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