by: Parent Project Muscular Dystrophy
Wave Life Sciences recently joined PPMD for a community webinar to share an update on the company’s FORWARD-53 study. Wave provided an overview of initial clinical trial results for WVE-N531 in individuals with Duchenne muscular dystrophy who are amenable to exon 53 skipping, as well as an overview of the potentially registrational FORWARD-53 study.
The Wave team highlighted data supporting the safety and tolerability of WVE-N531, as well as dosing intravenous infusion (IV) every other week or potentially less frequent dosing. In Part A of the Phase 1b/2a clinical trial with three patients with Duchenne, WVE-N531 reached high concentrations in skeletal muscle and led to 53% exon skipping following 3 doses of 10 mg/kg every other week. Wave also discussed the company’s new chemistry incorporated in WVE-N531, “PM chemistry,” which changes the shape of the molecule to better penetrate muscle cells.
Wave indicated that the company expects a read-out from FORWARD-53 in Q3 of 2024. Finally, the Wave team answered questions from the community on a variety of topics.
