Wave Life Sciences to Present at Parent Project Muscular Dystrophy 2019 Annual Conference

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CAMBRIDGE, Mass., June 12, 2019 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced that Michael Panzara, MD, MPH, Chief Medical Officer, will present at the Parent Project Muscular Dystrophy (PPMD) 2019 Annual Conference in Orlando, Fla. on Saturday, June 29, 2019 at 12:00 p.m. ET. The presentation will include an overview of the ongoing clinical development of suvodirsen, the company’s investigational stereopure oligonucleotide for boys with Duchenne muscular dystrophy who are amenable to exon 51 skipping.

Suvodirsen is currently being studied in an open label extension (OLE) study, initiated in August 2018 with patients from the Phase 1 clinical trial. Wave expects to deliver an interim analysis of dystrophin expression from muscle biopsies in boys receiving suvodirsen in the OLE study in the second half of 2019. Pending positive clinical dystrophin expression data, the company expects to file for an accelerated approval of suvodirsen in the United States in the second half of 2020.

The presentation slide deck will be available for a limited time on the investor relations page of the Wave Life Sciences corporate website at . A live stream of the presentation will be available on the PPMD website during the conference.

During PPMD, Wave will also present two posters with previously presented data and information on suvodirsen and DYSTANCE 51, the planned Phase 2/3 efficacy and safety clinical trial in DMD patients amenable to exon 51 skipping.

In July 2019, Wave expects to initiate DYSTANCE 51, a global, multicenter, randomized, double-blind, placebo-controlled Phase 2/3 efficacy and safety clinical trial of suvodirsen in DMD patients amenable to exon 51 skipping. The company intends to use the results of this trial to seek regulatory approvals globally.

The DYSTANCE 51 Phase 2/3 clinical trial was the first study ever selected by the U.S. Food and Drug Administration (FDA) for the agency’s pilot program for complex innovative trial designs. In addition, suvodirsen has been granted orphan drug designation for the treatment of DMD by the FDA and the European Commission, as well as rare pediatric disease designation by the FDA.

Approximately 13% of DMD patients have genetic mutations that are amenable to treatment with an exon 51 skipping therapy. Exon-skipping technology has the potential to induce cellular machinery to ‘skip over’ a targeted exon and restore the reading frame, resulting in the production of internally truncated, but functional dystrophin protein.

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Globe Newswire: 13:00 GMT Wednesday 12th June 2019

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