About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a fatalX-linked genetic neuromuscular disorder caused predominantly byout-of-frame deletions in thedystrophin 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.
AboutWVE-210201
WVE-210201 is an investigational stereopure oligonucleotide that has been shown to induce skipping of exon 51 ofdystrophinpre-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 preclinicalin vitro experiments using gymnotic delivery (free uptake) ofWVE-210201 in DMD patient-derived myoblasts demonstrated efficient exon 51 skipping and dystrophin protein restoration. Preclinical Western blot studies ofWVE-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 U.S. Food and Drug Administration (FDA) and the European Commission, as well as rare pediatric disease designation by the FDA.
AboutWVE-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 ofWVE-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 andnon-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 receiveWVE-210201.
About Wave Life Sciences
Wave Life Sciences is a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases. Its chemistry platform enables the creation of highly specific, well characterized oligonucleotides designed to deliver superior efficacy and safety across multiple therapeutic modalities. The company’s pipeline is initially focused on neurological disorders and extends across several other therapeutic areas. For more information, please visitwww.wavelifesciences.com.
Forward-Looking Statements
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 aWVE-210201 Phase 2/3 clinical trial, the plans to present theWVE-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 receivingWVE-210201 in the OLE study, the belief that the safety and tolerability data from theWVE-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 Form10-K filed with the Securities and Exchange Commission (SEC), as amended, and in other filings Wave makes with the SEC from time to time. Wave undertakes no obligation to update the information contained in this press release to reflect subsequently occurring events or circumstances
