Dyne Therapeutics (DYN) said Friday that new clinical data from its ongoing phase 1/2 trial of DYNE-101 in people with myotonic dystrophy type 1 demonstrated a compelling impact on key disease biomarkers.
This included the knockdown of dystrophin myotonic protein kinase, or DMPK, and splicing correction at the 6.8 mg/kg Q8W dose.
The data also showed a reversal of disease progression across multiple functional endpoints. Improvements were observed in myotonia as measured by video hand opening time, muscle strength through quantitative myometry testing, and dynamic balance.
Dyne said that splicing correction at 3 months supports the use of the composite alternative splicing index as a surrogate endpoint for potential US Accelerated Approval. The data also highlighted a favorable safety profile from 56 patients in the 6.8 mg/kg Q8W cohort.
The company said it plans to initiate a global registrational expansion cohort to support a submission for US accelerated approval based on biomarker and functional data in H1 2026.
Shares of Dyne Therapeutics were down nearly 24% in recent trading.
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