On Friday, Dyne Therapeutics, Inc. DYN revealed new knowledge from its ongoing Section 1/2 ACHIEVE trial of DYNE-101 in sufferers with myotonic dystrophy kind 1 (DM1).
Dyne plans to start a worldwide Registrational Enlargement Cohort with the potential to help a submission for U.S. Accelerated Approval primarily based on biomarker and practical knowledge in H1 2026.
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Dyne reported efficacy knowledge from grownup DM1 sufferers enrolled within the randomized, placebo-controlled MAD portion of the DYNE-101 ACHIEVE trial, together with knowledge from the 6.8 mg/kg Q8W cohort (n=8) at as much as 6 months.
On the 6.8 mg/kg Q8W dose, DYNE-101 resulted in important splicing correction at 3 months in comparison with baseline, which was related to progress in a number of practical endpoints, starting at 3 months and persevering with at 6 months.
- Evaluation of muscle biopsy knowledge for the 6.8 mg/kg Q8W cohort confirmed a considerable knockdown of Drug Metabolism and Pharmacokinetics (DMPK) (DYNE-101 molecular goal) RNA ranges. Splicing correction at 3 months for the 6.8 mg/kg Q8W cohort was strong. It was related to enchancment in a number of practical endpoints, supporting CASI-22 at 3 months as a surrogate endpoint for potential U.S. Accelerated Approval.
- Early and sustained enchancment in myotonia as measured by video hand opening time (vHOT) was seen within the 6.8 mg/kg Q8W cohort and at low doses with modest splicing correction, deepening with extra time on the drug.
- Practical measures confirmed early and sustained medical profit on the 6.8 mg/kg Q8W dose.
Dyne additionally reported security and tolerability knowledge from 56 sufferers. A lot of the treatment-emergent opposed occasions have been delicate or average, and no associated severe treatment-emergent opposed occasions have been recognized.
Primarily based on earlier dialogue with the FDA’s Heart for Drug Analysis and Analysis division, Dyne continues to pursue accelerated approval within the U.S. primarily based on splicing as a surrogate endpoint.
Dyne anticipates finishing enrollment within the Registrational Enlargement Cohort in mid-2025 and submitting for U.S. Accelerated Approval in H1 2026.
Value Motion: DYN inventory is down 35.3% at $14.91 eventually examine Friday.
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