PepGen said its oligonucleotide therapy for myotonic dystrophy type 1 (DM1) showed promising early signals in a small Phase 2 cohort. The company’s update describes results in six patients treated for 12 weeks, emphasizing a key measure described as the most promising early data to date in the DM1 race. The update arrives amid a competitive landscape that includes late-stage development programs and recent setbacks in other DM1 approaches. PepGen’s data will likely be scrutinized for effect size, durability, and whether biomarkers align with functional improvements. Near-term catalysts include expanded patient follow-up and clarity on dose-response and tolerability as PepGen moves toward later-stage decision points.
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