In January 2022, Defeat Duchenne Canada shared the news that the U.S. Food and Drug Administration (FDA) placed a clinical hold on the Investigational New Drug (IND) application from Dyne Therapeutics in launching their clinical trial of DYNE-251, which targets mutations amenable to skipping exon 51.
We are pleased to inform you that the FDA has lifted the hold and cleared the IND application. Dyne expects to initiate trial participant dosing in a Phase 1/2 clinical trial evaluating DYNE-251 in mid-2022:
“Today marks a significant step in our journey to build a DMD franchise to serve people across the globe with Duchenne mutations amenable to exon skipping. The clearance of our first IND is an important achievement for Dyne, and we appreciate the partnership with the FDA throughout this process. Our team has worked extensively with key opinion leaders, patient advocacy groups and individuals living with DMD to thoughtfully design and execute our global multiple-ascending dose Phase 1/2 clinical trial of DYNE-251.
We believe we are well-positioned to deliver on our commitment of initiating dosing in both of our DMD and DM1 programs in mid-2022. The entire Dyne team is proud of the progress we have made to advance our mission and address the urgent need to bring new therapeutic options to people living with serious muscle diseases.”Joshua Brumm, President and Chief Executive Officer of Dyne
The Phase 1/2 clinical trial is a global, randomized, placebo-controlled multiple ascending dose (MAD) clinical trial with a long-term extension. Dyne expects to enroll 30-50 ambulant and non-ambulant males aged 4 to 16 with symptomatic DMD and mutations amenable to exon 51 skipping therapy. The study will evaluate the safety, tolerability, dystrophin expression measured by Western Blot, pharmacokinetics and pharmacodynamics, and measures of muscle function. They plan to outline additional details regarding the trial design and data timing upon dosing initiation.
In addition to DYNE-251, Dyne is building a global DMD franchise with preclinical programs for people with DMD mutations amenable to skipping other exons, including 53, 45 and 44.