Gold prices slip slightly after recent gains; U.S. data eyed
Investing.com -- Dyne Therapeutics Inc (NASDAQ:DYN) stock rose 3.6% after the company announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to its DYNE-251 treatment for Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping.
The designation was based on data from the company’s ongoing DELIVER clinical trial. The breakthrough status provides Dyne with enhanced FDA support, including senior-level involvement, early and frequent communication with FDA reviewers, and eligibility for rolling and priority review, potentially reducing the review timeline from 12 to 8 months.
"This Breakthrough Therapy Designation for DYNE-251 is a testament to its potential as a next-generation therapy designed to bring meaningful functional improvement to individuals with DMD for whom exon 51 skipping can lead to the production of near full-length dystrophin," said Doug Kerr, chief medical officer of Dyne.
The company reported that DYNE-251 has demonstrated sustained functional improvement through 18 months, as assessed by key measures such as time to rise and stride velocity 95th centile.
This marks the second breakthrough therapy designation for Dyne’s lead programs, with DYNE-101 having received the same status for myotonic dystrophy type 1 earlier this year.
Dyne has completed enrollment of 32 patients in the Registrational Expansion Cohort of the DELIVER trial, with data expected in late 2025. The company anticipates a potential Biologics License Application submission for U.S. Accelerated Approval in early 2026.
DYNE-251 has also received Fast Track, Orphan Drug, and Rare Pediatric disease designations from the FDA and Orphan Drug designation from the European Medicines Agency for DMD treatment.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.