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Investing.com -- Dyne Therapeutics, Inc. (Nasdaq:DYN) stock fell 23% after the company announced an updated plan for obtaining U.S. Accelerated Approval for DYNE-101 in myotonic dystrophy type 1 (DM1) following a Type C meeting with the FDA.
The clinical-stage neuromuscular disease company revealed that the FDA has granted Breakthrough Therapy Designation to DYNE-101 for the treatment of DM1. Based on FDA feedback and analysis of new long-term functional data, Dyne has submitted a revised protocol for the ongoing Registrational Expansion Cohort of the ACHIEVE trial with video hand opening time (vHOT) as the primary endpoint for potential Accelerated Approval.
According to the updated timeline, Dyne plans to complete enrollment in the Registrational Expansion Cohort in Q4 2025, with data expected by mid-2026 to support a potential U.S. Accelerated Approval submission in late 2026. The company also plans to initiate a confirmatory Phase 3 clinical trial in the first quarter of 2026.
The revised protocol for the Registrational Expansion Cohort includes a primary endpoint measuring change from baseline in middle finger myotonia using vHOT at 6 months compared to placebo. Secondary endpoints include changes in splicing, muscle strength, performance tests, and patient-reported outcomes. The cohort is expected to enroll 60 participants randomized 3:1 to receive DYNE-101 or placebo.
Dyne also reported new long-term data from the multiple ascending dose portion of the ACHIEVE trial, showing sustained improvement in myotonia and other endpoints at 12 months for patients receiving the registrational dose of 6.8 mg/kg Q8W.
The company expects its cash, cash equivalents and marketable securities as of March 31, 2025, totaling $677.5 million, will fund operations into the fourth quarter of 2026.
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