The revised trial protocol that means a delay in filing for U.S. approval of DYNE-101 to treat myotonic dystrophy type 1 (DM1) dented shares of Dyne Therapeutics Inc. (NASDAQ:DYN), which closed June 17 at $10.86, down $2.96, or 21%.
Dyne Therapeutics Inc. is eyeing accelerated approval for its myotonic dystrophy type 1 treatment after reviewing new results from a phase I/II study. DYNE-101, an oligonucleotide antisense and DMPK gene modulator, produced results on disease biomarkers that included DMPK and splicing correction, disease progression reversal on several functional endpoints and a favorable safety profile. The accelerated approval submission could come in the first half of 2026.
Shares of Dyne Therapeutics Inc. (NASDAQ:DYN) closed May 20 at $35.38, up $7.70, or 28%, on word of positive data from the phase I/II Achieve trial of DYNE-101 in myotonic dystrophy type 1 (DM1) and the phase I/II Deliver effort with DYNE-251 in Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. CEO John Cox, who joined Waltham, Mass.-based Dyne eight weeks ago, said he “couldn’t be more proud to be part of this team.” Studies are ongoing, but new data with regard to DM1 as well as DMD showed a “compelling” impact, Dyne said, plus satisfying safety profiles.
With positive initial phase I/II data in hand from two trials, Dyne Therapeutics Inc. plans to report more findings and start enrolling registrational cohorts in both studies by the end of this year for DYNE-101 in myotonic dystrophy type 1 (DM1) and a study called Deliver with DYNE-251 in Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
