Wave Life Sciences Announces Initiation of Phase 2/3 Clinical Trial 

Wave Life Sciences Ltd. recently announced the initiation of DYSTANCE 51, the Phase 2/3 efficacy and safety clinical trial of suvodirsen, an investigational stereopure oligonucleotide intended for boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

“The initiation of the DYSTANCE 51 Phase 2/3 study represents a significant milestone for Wave and our DMD clinical program, as well as an important development for the families affected by Duchenne,” said Michael Panzara, MD, MPH, Chief Medical Officer of Wave Life Sciences. “We are grateful to be working in collaboration with the U.S. Food and Drug Administration, global regulators and our partners from the Duchenne community on this innovative and exciting trial.  We are proud to have worked with patient groups to incorporate their feedback into the trial design to better address the needs of boys and families living with DMD.”

“We are pleased that Wave Life Sciences has incorporated important input from the Duchenne community in designing their Phase 2/3 clinical trial of suvodirsen. This global study is designed around the needs of families while measuring a robust set of biomarker and functional endpoints. PPMD looks forward to learning more about this stereopure exon-skipping program when Wave presents at our 25th Annual Conference later this week,” said Pat Furlong, founding President and CEO of Parent Project Muscular Dystrophy (PPMD).

DYSTANCE 51 is a global study and is expected to include clinical trial sites in the United States, Europe, Australia, Canada and Japan. Results from this trial are intended to support global regulatory filings for suvodirsen.

DYSTANCE 51 is a global Phase 2/3, multicenter, randomized, double-blind, placebo-controlled clinical trial that will evaluate the efficacy and safety of suvodirsen in ambulatory boys who are between 5 and 12 years of age (inclusive) with a genetically confirmed diagnosis of DMD amenable to exon 51 skipping. The trial will test two dose levels of suvodirsen and is expected to enroll approximately 150 patients. The primary efficacy endpoints of the clinical trial will measure change in dystrophin protein level and change in the North Star Ambulatory Assessment (NSAA) score. In addition to the NSAA, the trial will include other functional outcome measures as secondary efficacy endpoints.

The DYSTANCE 51 clinical trial is the first study ever selected by the U.S. Food and Drug Administration (FDA) for its pilot program for complex innovative trial designs. As a participant in the pilot program, the company has held multiple meetings with FDA staff to discuss the design elements of the trial, including the potential use of Bayesian methods to adapt the trial with the aim of maximizing efficiency while ensuring robust clinical results.

Suvodirsen is an investigational stereopure oligonucleotide currently being evaluated in an ongoing open-label extension (OLE) study for the treatment of boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. Data from the company’s completed Phase 1 clinical trial of suvodirsen demonstrated a favorable safety and tolerability profile after single doses, supporting its continued clinical development, including the launch of DYSTANCE 51 and continuation of the OLE study. Wave remains on track 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.

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.

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. 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.

Duchenne muscular dystrophy (DMD) is a fatal X-linked genetic neuromuscular disorder caused predominantly by out-of-frame deletions in the dystrophin gene, resulting in absent or defective dystrophin protein. Dystrophin protein is needed for normal muscle maintenance and operation. Because of the genetic mutations in DMD, the body cannot produce functional dystrophin, which results in progressive and irreversible loss of muscle function, including the heart and lungs. Worldwide, DMD affects approximately one in 5,000 newborn boys.

Wave Life Sciences is a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases. Wave aspires to develop best-in-class medicines across multiple therapeutic modalities using PRISM, the company’s proprietary discovery and drug development platform that enables the precise design, optimization and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, the Wave team is targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future. To find out more, please visit www.wavelifesciences.com and follow Wave on Twitter @WaveLifeSci.