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
“These results mark an important milestone for the Duchenne community and toward our goal of inducing meaningful, natural dystrophin expression in boys with DMD who are amenable to exon 51 skipping,” said
WVE-210201 is also currently being studied in an ongoing multi-dose, open-label extension (OLE) study initiated in
“PPMD continues to be optimistic about the progress the team at
Wave anticipates initiating a global, placebo-controlled Phase 2/3 efficacy and safety clinical trial of WVE-210201 in DMD patients amenable to exon 51 skipping in 2019. The Phase 2/3 trial is designed to measure clinical efficacy and dystrophin expression, and the company intends to use the results of this trial to seek regulatory approvals globally.
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 in the heart and lungs. Globally, DMD affects approximately one in 5,000 newborn boys.
WVE-210201 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 WVE-210201 in DMD patient-derived myoblasts demonstrated efficient exon 51 skipping and dystrophin protein restoration. Preclinical Western blot studies of WVE-210201 demonstrated 52% dystrophin protein restoration compared with normal skeletal muscle tissue lysates. WVE-210201 has been granted orphan drug designation for the treatment of DMD by the
About WVE-210201 Clinical Trials
WVE-210201 is being studied in an ongoing global, multicenter, double-blind, placebo-controlled Phase 1 clinical trial designed to evaluate the safety, tolerability and plasma concentrations of single ascending doses of WVE-210201 administered intravenously in Duchenne muscular dystrophy patients with gene mutations amenable to exon 51 skipping. The trial is expected to enroll up to 40 patients, including ambulatory and non-ambulatory patients between 5 and 18 years of age. As patients complete the Phase 1 trial, they have the option to enroll in an ongoing open-label extension study in which they receive WVE-210201.
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 initiation of a WVE-210201 Phase 2/3 clinical trial, the plans to present the WVE-210201 Phase 1 clinical trial results and details of the Phase 2/3 study design at upcoming scientific meetings, the expected timing of an interim analysis of dystrophin expression from muscle biopsies in boys receiving WVE-210201 in the OLE study, the belief that the safety and tolerability data from the WVE-210201 Phase 1 clinical trial supports initiation of a Phase 2/3 clinical trial, and Wave’s intention to use the results of that 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
Source: Wave Life Sciences