Dyne Therapeutics Receives Orphan Drug Designation in Japan for Zeleciment Basivarsen (DYNE-101) for Myotonic Dystrophy Type 1

Core Insights - Dyne Therapeutics has received Orphan Drug designation in Japan for zeleciment basivarsen (z-basivarsen) to treat myotonic muscular dystrophy type 1 (DM1), highlighting the urgent need for new therapies in this area [1][2] - Z-basivarsen has shown early and sustained improvements in myotonia, muscle strength, and function, with a favorable safety profile, and has also received similar designations in the U.S. and Europe [2][4] - The ACHIEVE trial is a Phase 1/2 clinical trial evaluating z-basivarsen, with a registrational dose of 6.8 mg/kg administered every eight weeks, aiming for potential regulatory submissions [3][4] Company Overview - Dyne Therapeutics focuses on delivering functional improvement for individuals with genetically driven neuromuscular diseases, including DM1 and Duchenne muscular dystrophy (DMD) [6][7] - The company is advancing clinical programs targeting muscle and the central nervous system to address the root causes of these diseases [7] Clinical Trial Details - The ACHIEVE trial is a global, randomized, placebo-controlled, double-blind study assessing the safety, tolerability, and efficacy of z-basivarsen in DM1 patients [3] - The primary endpoint for the registrational expansion cohort is the change in middle finger myotonia measured by video hand opening time (vHOT) at 6 months compared to placebo [3] Disease Context - Myotonic dystrophy type 1 (DM1) is a rare genetic neuromuscular disease affecting approximately 40,000 people in the U.S. and 55,000 in the EU, characterized by high morbidity and early mortality [5] - Symptoms of DM1 can vary widely and include muscle weakness, myotonia, cognitive impairments, and other systemic issues, with no approved disease-modifying treatments currently available [5]