Wave Life Sciences have announced the next steps for WVE-N531, a study for those with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.

Wave plans to initiate Part B of the open-label study to enroll up to ten boys to evaluate dystrophin protein restoration. Boys will be dosed at 10 mg/kg biweekly, and we plan to assess dystrophin protein after 24 and 48 weeks of biweekly dosing. The primary endpoint will be dystrophin protein levels, and the study will also evaluate safety and tolerability, pharmacokinetics, and functional endpoints. Wave expects to share data in 2024.

“In 2023, we are poised to deliver the first RNA editing candidate to the clinic, which will be an exciting moment not just for Wave, but for the entire nucleic acids field. We are initiating Part B of the WVE-N531 study in Duchenne muscular dystrophy to assess dystrophin after 24 and 48 weeks of biweekly dosing, with potential for this program to become a significant commercial opportunity for Wave.”

Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences

This is building off the positive data from the initial cohort of the proof-of-concept open-label study for WVE-N53, which appeared to be safe and well-tolerated and saw the observation of high muscle concentrations of WVE-N531 (mean of 42 micrograms/gram) and exon skipping (mean of 53% as measured by RT-PCR) six weeks after initiating biweekly dosing at 10 mg/kg.