Wave Life Sciences to Present Suvodirsen Phase 1 Safety and Tolerability Data and Phase 2/3 Clinical Trial Design at the Muscular Dystrophy Association Clinical and Scientific Conference
Wave will present two posters focused on suvodirsen (WVE-210201), the company’s investigational stereopure oligonucleotide for boys with DMD who are amenable to exon 51 skipping:
- Safety and Tolerability of Suvodirsen (WVE-210201) in Patients with Duchenne Muscular Dystrophy: Results from a Phase 1 Clinical Trial
- Design of a Phase 2/3 Randomized Controlled Trial of Suvodirsen (WVE-210201) in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Two additional posters providing preclinical data on Wave’s DMD programs will also be presented:
- Potency of Stereopure Antisense Oligonucleotides in Cellular Free-Uptake Model Predicts Exon Skipping and Dystrophin Protein Restoration in mdx23 Mice
- Investigational Stereopure Exon-Skipping Antisense Oligonucleotides Targeting Exons 51 and 53 in Duchenne Muscular Dystrophy
Conference organizers will announce poster numbers and schedules.
About Duchenne Muscular Dystrophy
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. Globally, DMD affects approximately one in 5,000 newborn boys.
About Suvodirsen (WVE-210201)
Suvodirsen is an investigational stereopure oligonucleotide that has been shown to induce skipping of exon 51 of dystrophin pre-mRNA in preclinical studies and is intended for the treatment of Duchenne muscular dystrophy (DMD). 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. Wave preclinical in vitro experiments using gymnotic delivery (free uptake) of suvodirsen in DMD patient-derived myoblasts demonstrated efficient exon 51 skipping and dystrophin protein restoration. Preclinical western blot studies of suvodirsen demonstrated 52% dystrophin protein restoration compared with normal skeletal muscle tissue lysates. Suvodirsen has been granted orphan drug designation for the treatment of DMD by the
About Suvodirsen (WVE-210201) Clinical Trials
Suvodirsen was studied in a global, multicenter, double-blind, placebo-controlled Phase 1 clinical trial designed to evaluate the safety, tolerability and plasma concentrations of single ascending doses of suvodirsen administered intravenously in Duchenne muscular dystrophy patients with gene mutations amenable to exon 51 skipping. In
Suvodirsen is currently being studied in an ongoing multi-dose, open-label extension (OLE) study which was initiated in
Wave anticipates initiating a global, placebo-controlled Phase 2/3 efficacy and safety clinical trial of suvodirsen in DMD patients amenable to exon 51 skipping in 2019. The trial is designed to measure clinical efficacy and dystrophin expression, and Wave intends to use the results of this trial to seek regulatory approvals globally.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the expected timing of an interim analysis of dystrophin expression from muscle biopsies in boys receiving suvodirsen in the Phase 1 OLE study, Wave’s intention to potentially use the interim analysis data to support an accelerated approval in the U.S., the expected timing of initiation for and the anticipated endpoints of a suvodirsen Phase 2/3 clinical trial, and Wave’s intention to potentially use the results of the Phase 2/3 trial to seek regulatory approvals globally. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, the risks and uncertainties described in the section entitled “Risk Factors” in Wave’s most recent Annual Report on Form 10-K filed with the
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Source: Wave Life Sciences