Dyne Therapeutics(DYN) Benzinga·2025-01-10 18:40Core Insights - Dyne Therapeutics, Inc. has revealed new data from its ongoing Phase 1/2 ACHIEVE trial of DYNE-101 for myotonic dystrophy type 1 (DM1) and plans to initiate a global Registrational Expansion Cohort to support U.S. Accelerated Approval submission in H1 2026 [1][4][5] Group 1: Trial Data - Efficacy data from the 6.8 mg/kg Q8W cohort (n=8) showed significant splicing correction at 3 months, linked to improvements in multiple functional endpoints at both 3 and 6 months [2][3] - Analysis of muscle biopsy data indicated a substantial knockdown of DMPK RNA levels, with robust splicing correction at 3 months supporting CASI-22 as a surrogate endpoint for potential U.S. Accelerated Approval [7] Group 2: Safety and Tolerability - Safety data from 56 patients indicated that most treatment-emergent adverse events were mild or moderate, with no serious treatment-emergent adverse events identified [4] - Early and sustained improvement in myotonia was observed, particularly in the 6.8 mg/kg Q8W cohort, with functional measures showing clinical benefits [7] Group 3: Future Plans - Dyne anticipates completing enrollment in the Registrational Expansion Cohort by mid-2025 and aims to submit for U.S. Accelerated Approval in H1 2026 [5]