Dyne Therapeutics has announced that the FDA has lifted the clinical hold and cleared its Investigational New Drug (IND) application to initiate a clinical trial of DYNE-251 in participants with Duchenne amenable to skipping exon 51. Dyne-251 is an exon skipping product that combines a PMO to enable skipping of exon 51 with a fragment antibody (Fab) to increase targeted delivery of the product to muscle tissue.
We look forward to continued updates as Dyne expects to initiate trial participant dosing in a Phase 1/2 clinical trial in mid-2022.
Read the announcement from Dyne:
– Initiation of Dosing in Multiple Ascending Dose Clinical Trial in Patients with Mutations Amenable to Skipping Exon 51 On Track for Mid-2022 –
WALTHAM, Mass., July 05, 2022 (GLOBE NEWSWIRE) — Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold and cleared its Investigational New Drug (IND) application to initiate a clinical trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) amenable to skipping exon 51. The Company expects to begin dosing patients 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,” said Joshua Brumm, president and chief executive officer of Dyne. “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.”
Dyne plans to evaluate DYNE-251 in a global, randomized, placebo controlled, multiple ascending dose (MAD) clinical trial with a long-term extension study. The trial aims to enroll 30 to 50 ambulant and non-ambulant males with Duchenne, who are age 4 to 16 and have mutations amenable to exon 51 skipping therapy. The study will evaluate safety, tolerability, dystrophin expression as measured by Western Blot, pharmacokinetics and pharmacodynamics, and measures of muscle function. Dyne plans to outline additional details regarding the trial design and timing of data upon initiation of dosing.
In addition to DYNE-251, Dyne is building a global DMD franchise with preclinical programs for patients with mutations amenable to skipping other exons, including 53, 45 and 44.