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Wave Life Sciences Announces Suvodirsen Phase 1 Safety and Tolerability Data and Phase 2/3 Clinical Trial Design

Wave Life Sciences Ltd., a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced the final results from its Phase 1 clinical trial of investigational suvodirsen (WVE-210201) in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. The company also announced the design of its planned Phase 2/3 clinical trial of suvodirsen in DMD, DYSTANCE 51. The Phase 1 data and DYSTANCE 51 details will be presented today at the 2019 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in Orlando, Florida.

“We are delighted that suvodirsen’s Phase 1 results demonstrate a favorable safety and tolerability profile that allow us to proceed rapidly into Phase 2/3 clinical development with doses we expect to be within the therapeutic window. Later this year, we expect to report interim efficacy data from the ongoing open-label extension study of suvodirsen and intend to use those data as an important component of a submission for accelerated approval in the United States,” said Michael Panzara, MD, MPH, Chief Medical Officer of Wave Life Sciences. “We truly appreciate the boys and families that made this trial possible.”

Wave Life Sciences Announces Suvodirsen Phase 1 Safety And Tolerability Data And Phase 2/3 Clinical Trial Design