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(RTTNews) - Dyne Therapeutics, Inc. (DYN) Monday announced positive data from its Phase 1/2 ACHIEVE study of DYNE-101 in patients with myotonic dystrophy type 1 (DM1) and Phase 1/2 DELIVER study of DYNE-251 in patients with Duchenne muscular dystrophy (DMD).

In the ACHIEVE trial, DYNE-101 showed robust muscle delivery and dose-dependent, consistent splicing correction while also showing improvement in multiple functional endpoints and patient reported outcomes. Additionally, DYNE-101 demonstrated a favorable safety profile with no serious treatment emergent adverse events.

In DELIVER study in patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping, 3.2 percent mean unadjusted dystrophin expression was observed at 6 Months in patients who received DYNE-251 once every four weeks for 6 months. This is 10 times higher level of dystrophin than reported for weekly administered standard of care. Also, DYNE-251 demonstrated trends in functional improvement in North Star Ambulatory Assessment (NSAA), time to rise from floor, 10-meter walk/run time, and stride velocity 95th centile (SV95C).

Dyne said it plans to continue to pursue expedited approvals for both DYNE-101 and DYNE-251 with update on registrational pathways expected by year-end.

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