Wave Life Sciences to Present at Parent Project Muscular Dystrophy 2019 Annual Conference
Suvodirsen is currently being studied in an open label extension (OLE) study, initiated in
The presentation slide deck will be available for a limited time on the investor relations page of the
During PPMD, Wave will also present two posters with previously presented data and information on suvodirsen and DYSTANCE 51, the planned Phase 2/3 efficacy and safety clinical trial in DMD patients amenable to exon 51 skipping.
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 a completed Phase 1 clinical trial of suvodirsen demonstrated a favorable safety and tolerability profile, supporting the continued clinical development of the investigational therapy in DMD.
The DYSTANCE 51 Phase 2/3 clinical trial was the first study ever selected by the
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.
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. Worldwide, DMD affects approximately one in 5,000 newborn boys.
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 the Phase 2/3 clinical trial for suvodirsen in DMD, the plans to report interim efficacy data from the ongoing open-label extension study with patients from the Phase 1 clinical trial of suvodirsen in DMD, the belief that the safety and tolerability data from the Phase 1 clinical trial support initiation of a Phase 2/3 clinical trial, Wave’s intention to use the results of the OLE and Phase 2/3 trials to seek various regulatory approvals for suvodirsen. 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. These risks and uncertainties include but are not limited to the following: Wave’s current and planned clinical trials, other studies for suvodirsen, and Wave’s other product candidates may not be successful or may take longer and be more costly than anticipated; product candidates that appeared promising in earlier research and clinical trials may not demonstrate safety and/or efficacy in later-stage or larger-scale clinical trials; and the other risk factors discussed under the heading “Risk Factors” contained in Wave’s Annual Report on Form 10-K for the year ended December 31, 2018 filed with the Securities and Exchange Commission (
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Source: Wave Life Sciences