Wave Life Sciences announces interim data fom ogoing Phase 2 FORWARD-53 study

Wave Life Sciences announced positive interim data from the ongoing Phase 2 FORWARD-53 study of WVE-N531, which is an exon skipping oligonucleotide being investigated in boys with Duchenne muscular dystrophy, DMD, who are amenable to exon 53 skipping. The interim analysis was conducted after 24 weeks of 10 mg/kg dosing every two weeks, Q2W, and WVE-N531 demonstrated substantial dystrophin expression and that it was safe and well tolerated. Results from this interim analysis include: WVE-N531 was safe and well tolerated; Dystrophin results from a pre-specified analysis of ambulatory boys showed: Mean absolute muscle content-adjusted dystrophin expression was 9.0% and mean absolute unadjusted dystrophin expression was 5.5% of normal, The dystrophin expression was quantified from two isoforms consistent with those observed in Becker muscular dystrophy patients who display milder disease, 89% of ambulatory participants achieved muscle content-adjusted dystrophin levels of at least 5%.; WVE-N531 was detected in myocyte nuclei in all participants and in myogenic stem cells in the majority of participants; Participants showed multiple indicators of improvement in muscle health; The muscle tissue half-life of WVE-N531 is estimated to be 61 days