Dosing begins in phase 2 study of Duchenne muscular dystrophy therapy for boys

A clinical-stage RNA medicines firm has announced the initiation of dosing in a phase 2 clinical trial of its investigational therapy for boys with Duchenne muscular dystrophy who are amenable to exon 53 skipping.

According to a press release from Wave Life Sciences, FORWARD-53, a potentially registrational, open label study, is designed to assess functional dystrophin protein at 24 and 48 weeks with every other week IV dosing at 10 mg/kg of WVE-N531, its exon-skipping therapeutic. The primary endpoint is dystrophin protein levels, with secondary endpoints evaluating safety, tolerability and pharmacokinetics. Thus far, per the company, the trial has enrolled 10 boys with the condition.

Commencement of the phase 2 study comes on the heels of Wave’s phase 1b/2a part A proof-of-concept trial, which demonstrated high muscle concentrations of WVE-N531 (mean of 6.1 micromolar or 42 mg/G) and mean exon skipping of 53% (range: 48% to 62%) at 6 weeks, following administration of three doses of 10 mg/kg of WVE-N531 every other week. WVE-N531 appeared safe and well-tolerated, with only mild treatment-related adverse events, according to the release.

Wave additionally said it is on track to deliver data, including dystrophin expression from muscle biopsies, in 2024.

“We believe we are on the right path toward addressing a significant unmet need in DMD [Duchenne muscular dystrophy] — the generation of endogenous dystrophin protein to levels that meaningfully impact the trajectory of the disease,” Anne-Marie Li-Kwai-Cheung, MChem, MTOPRA, RAPS, chief development officer at Wave Life Sciences, said in the release. “We are grateful to the DMD community for their continued support.”