Dyne Therapeutics has shared new clinical data from the ongoing DELIVER trial of DYNE-251 in patients with Duchenne who are amenable to exon 51 skipping. DYNE-251 is an exon skipping product that combines a PMO to enable skipping of exon 51 with a fragment antibody (Fab) to increase targeted delivery of the product to skeletal muscle.

This most recent data shares efficacy and safety data from patients with Duchenne amenable to exon 51 skipping enrolled in the 10 mg/kg (approximate PMO dose) cohort of the randomized, placebo-controlled multiple ascending dose portion of the DYNE-251 DELIVER trial. Results indicate that DYNE-251 has a favorable safety profile and is well-tolerated.

Additional key findings from the DELIVER trial include:

• Patients treated with 10 mg/kg of DYNE-251 once every four weeks had a mean absolute dystrophin level of 3.22% of normal and a 2.97% change (unadjusted for muscle content) from baseline at six months.

• The 10 mg/kg DYNE-251 once every four week group at six months showed functional improvement in North Star Ambulatory Assessment (NSAA), Stride Velocity 95th Centile (SV95C), 10-Meter Walk/Run Time, and Time to Rise from Floor.

Dyne indicated that the company anticipates sharing program updates in more detail with the community over the coming weeks.

PPMD is pleased to hear this promising news and looks forward to further updates from Dyne.

Read Dyne’s community letter here.