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.
Interesting further information
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