Dyne Therapeutics, Inc. (Nasdaq: DYN), a muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced 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.

“In a little over three years, we’ve been able to move Dyne from concept to clinic because we are steadfast in our commitment to bring potentially transformational therapies to people living with serious muscle diseases,” said Joshua Brumm, president and chief executive officer of Dyne. “The IND submission for DYNE-251 represents a significant milestone. I am so proud of what our team has accomplished, and I want to thank all Dynamos and our partners in the rare muscle disease community. We look forward to an exciting 2022 as we expect to submit an IND for DM1 in the first quarter, begin dosing patients in clinical trials for both our DMD and DM1 programs by mid-year, and submit an IND for our FSHD program in the second half.”

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.