US RNA specialist Wave Life Sciences (Nasdaq: WVE) has announced positive interim data from the ongoing Phase II 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.
Wave Life noted that the interim analysis was conducted after 24 weeks of 10mg/kg dosing every two weeks (Q2W), and WVE-N531 demonstrated substantial dystrophin expression and that it was safe and well tolerated, with the news sending the firm’s shares up more than 53% to $8.19 by close of trading on Tuesday.
The FORWARD-53 data demonstrated "meaningful" improvements in serum biomarkers for muscle health, according to Wave, with WVE-N531 localizing in myogenic stem cells in the majority of participants and aiding in the regeneration of myofibers. The estimated half-life of WVE-N531 in muscle tissue was 61 days, something Wave says supports a possible monthly dosing regimen moving forward.
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