Dyne Therapeutics(US:DYN) Globenewswireยท2025-10-06 22:01Core Insights - Dyne Therapeutics announced one-year data from the Phase 1/2 ACHIEVE clinical trial of zeleciment basivarsen (z-basivarsen) in patients with myotonic dystrophy type 1 (DM1), showing clinically meaningful improvements in function and strength [1][2] Clinical Trial Data - The trial demonstrated robust improvements across various clinical measures, including hand myotonia, functional endpoints, and muscle strength [3][10] - Improvements were reported in both patient-reported outcomes and clinician assessments, indicating a reduction in overall disease burden [1][10] - The selected registrational dose of 6.8 mg/kg administered every eight weeks was evaluated in a randomized, placebo-controlled setting [7] Functional Improvements - Significant enhancements were observed in upper and lower limb function, as measured by tests such as the 10-Meter Walk/Run Test and the 5 Times Sit to Stand Test [3] - New data indicated improvements in the 9-Hole Peg Test, focusing on manual dexterity and coordination [3] - Muscle strength improvements were noted across various quantitative muscle testing scores, including hand grip strength and knee extension [3] Patient-Reported Outcomes - The Myotonic Dystrophy Health Index showed meaningful improvements in multiple subscales, including cognitive impairment, sleep disturbances, and fatigue [3][10] - Patient perceptions of improvements were consistent and encouraging, suggesting a broad and meaningful effect of z-basivarsen treatment [2] Safety and Tolerability - The safety profile of z-basivarsen was favorable, with no serious treatment-emergent adverse events reported among the 56 patients in the trial [10] Future Presentations - Dyne Therapeutics will present additional data at the 30th Annual International Congress of the World Muscle Society, including findings related to z-rostudirsen for Duchenne muscular dystrophy [4][6]