Wave Life Sciences Receives US Orphan Drug and Rare Pediatric Disease Designations for WVE-210201
“Our team is motivated by a sense of urgency and compassion for the patients, families and caregivers affected by Duchenne muscular dystrophy and other serious, life-threatening conditions with high areas of unmet need,” said
The Orphan Drug Act provides for economic incentives to encourage the development of drugs intended to treat, diagnose or prevent rare diseases and conditions affecting fewer than 200,000 people in
Rare pediatric disease designation by the
"These designations from U.S. regulators represent the significant progress being made by
WVE-210201 is currently 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 DMD 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 the ages of 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 continued treatment with WVE-210201.
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.
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 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 as compared with normal skeletal muscle tissue lysates.
About Wave Life Sciences
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the potential of WVE-210201 to help boys suffering from DMD; the future performance and results of WVE-210201 in clinical trials; the potential of our preclinical data to predict the behavior of WVE-210201 in humans; the expected patient enrollment for our Phase 1 clinical trial of WVE-210201; and the anticipated benefits to Wave from receiving U.S. orphan drug and rare pediatric disease designations for WVE-210201. 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 our most recent Annual Report on Form 10-K filed with the
Source: Wave Life Sciences