Wave Life Sciences Reports Positive Data From Phase 2 FORWARD-53 Study Of WVE-N531 In DMD

(RTTNews) - Wave Life Sciences Ltd. (WVE), a clinical-stage biotechnology company, Tuesday announced positive interim data from the Phase 2 FORWARD-53 study evaluating WVE-N531 in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping.

Duchenne muscular dystrophy is a genetic disorder characterized by progressive muscle degeneration, and caused by problem in producing a protein called dystrophin that helps keep muscle cells intact.

In the study, 11 boys amenable to exon 53 skipping are being treated with WVE-N531. Results from the interim analysis of ambulatory boys showed dystrophin expression of 9 percent and mean absolute unadjusted dystrophin expression of 5.5 percent. Eighty nine percent of ambulatory participants achieved muscle content-adjusted dystrophin levels of at least 5 percent. Further, WVE-N531 was safe and well tolerated.

The company expects to report complete data from the study in the first quarter of 2025.