Wave Life Sciences announces initiation of dosing in Phase 2 FORWARD-53 trial

Wave Life Sciences announced the initiation of dosing in the Phase 2 FORWARD-53 clinical trial, which is evaluating WVE-N531 as a treatment for boys with Duchenne muscular dystrophy who are amenable to exon 53 skipping. FORWARD-53 is designed to assess functional dystrophin protein at 24 and 48 weeks with every other week dosing of WVE-N531. “Following encouraging data from the WVE-N531 proof-of-concept trial, we believe we are on the right path toward addressing a significant unmet need in DMD – the generation of endogenous dystrophin protein to levels that meaningfully impact the trajectory of the disease,” said Anne-Marie Li-Kwai-Cheung, MChem, MTOPRA, RAPS, Chief Development Officer at Wave Life Sciences. “Our clinical data in boys with DMD amenable to exon 53 skipping demonstrated the highest level of exon skipping ever observed in the clinic, and high muscle concentrations of WVE-N531 in skeletal muscle of 6.1 micromolar. Importantly, in our non-human primate studies, muscle concentrations were higher in the diaphragm and heart than in skeletal muscle. Additionally, while FORWARD-53 is dosing every other week, we have the potential for monthly dosing in the future. For these reasons, we are excited about the potential of WVE-N531. We are grateful to the DMD community for their continued support and look forward to announcing dystrophin expression data in 2024, as well as advancing exon skipping candidates for other mutations if FORWARD-53 is successful.”