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Dyne Therapeutics Receives FDA Orphan Drug and Rare Pediatric Designations for DYNE-251 for the Treatment of Duchenne Muscular Dystrophy
- Data from the Global, Multiple Ascending Dose Phase 1/2 DELIVER Clinical Trial Anticipated in the Second Half of 2023 - WALTHAM, Mass., March 23, 2023
About this update from Dyne Therapeutics, Inc.
[{"type":"text","content":"- Data from the Global, Multiple Ascending Dose Phase 1/2 DELIVER Clinical Trial Anticipated in the Second Half of 2023 - WALTHAM, Mass., March 23, 2023 (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 DYNE-251, an investigational therapeutic for Duchenne muscular dystrophy (DMD) mutations amenable to exon 51 skipping, was granted U.S. Food and Drug Administration (FDA) orphan drug and rare pediatric disease designations. DYNE-251 is being evaluated in the Phase 1/2 DELIVER clinical trial. “These regulatory designations highlight the urgent and critical need for new and better therapeutic options for people living with this fatal disease,” said Wildon Farwell, M.D., MPH, chief medical officer of Dyne. “We are excited about DYNE-251 which we believe has the potential to transform the lives of people with DMD. We continue to advance our DELIVER clinical trial and look forward to sharing initial clinical data later this year.” Orphan drug designation is granted by the FDA to drugs or biological products intended for treatment, prevention or diagnosis of a rare disease or condition that affects fewer than 200,000 people in the United States. Under the FDA's rare pediatric disease designation program, the FDA may grant a priority review voucher to a sponsor who receives a product approval for a rare pediatric disease. About DYNE-251 and the DELIVER Trial DYNE-251 is an investigational therapeutic being evaluated in the Phase 1/2 global DELIVER trial for people living with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. The DELIVER clinical trial consists of a 24-week multiple ascending dose (MAD) randomized placebo-controlled period, a 24-week open-label extension and a 96-week long-term extension. The trial, which is designed to be registrational, is expected to enroll approximately 46 ambulant and non-ambulant males with DMD who are ages 4 to 16 and have mutations amenable to exon 51 skipping therapy. The primary endpoints are safety, tolerability and change from baseline in dystrophin levels as measured by Western blot. Secondary endpoints include measures of muscle function, exon skipping and pharmacokinetics. Dyne anticipa...