Wave Life Sciences Announces Positive Phase 1 Results for WVE-210201 in Duchenne Muscular Dystrophy (DMD) | DuchenneXchange

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Wave Life Sciences Announces Positive Phase 1 Results for WVE-210201 in Duchenne Muscular Dystrophy (DMD)


WVE-210201 safety and tolerability profile supports Phase 2/3 clinical trial initiation in boys with DMD amenable to exon 51 skipping

Delivery of interim efficacy data from ongoing open-label extension study expected in H2 2019

CAMBRIDGE, Mass., Dec. 06, 2018 (GLOBE NEWSWIRE) — Wave Life Sciences Ltd. (NASDAQ: WVE), a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases, today announced that the safety and tolerability data from the WVE-210201 Phase 1 clinical trial in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping support initiation of a Phase 2/3 clinical trial. Based on results from four ascending dose cohorts in the Phase 1 trial and pending final analysis, Wave has selected a dose for its planned Phase 2/3 clinical trial of WVE-210201, which it intends to initiate in 2019. In parallel, the independent Safety Monitoring Committee of the Phase 1 clinical trial has endorsed moving forward with a higher dose of WVE-210201 to be studied in a fifth cohort. Wave plans to present the results from the WVE-210201 Phase 1 clinical trial, as well as details of the Phase 2/3 study design, at upcoming scientific meetings.

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