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Wave Life Sciences initiates clinical trial for Exon51 investigational lead program

Posted on: November 8th, 2017 | 0 comments

First clinical trial of WVE-210201, an exon 51 skipping investigational therapy, in ambulatory and non-ambulatory boys 5 to 18 years old

Wave Life Scienves has announced the initiation of a global Phase 1 clinical trial for WVE-210201 in Duchenne muscular dystrophy patients amenable to exon 51 skipping.

“The initiation of WVE-210201’s clinical program is an important milestone in potentially delivering meaningful therapies for DMD patients,” said Michael Panzara, MD, MPH, Neurology Franchise Lead of Wave Life Sciences. “We are grateful to the DMD scientific and patient communities with whom we collaborated in designing our clinical program and will continue our close engagement with these key stakeholders as we advance candidates targeting other exons and explore additional innovative approaches to potentially treat DMD.”

About the study

The Phase 1 study is a multicenter, double-blind, placebo-controlled clinical trial designed to evaluate the safety, tolerability and plasma concentrations of single ascending doses of WVE-210201 administered intravenously in Duchenne patients with gene mutations amenable to exon 51 skipping. Data from the Phase 1 trial for WVE-210201 are expected in Q3 2018 and will facilitate the rapid transition to a double-blind, placebo-controlled, multi-dose efficacy study where dystrophin expression and clinical outcomes will be assessed. The clinical program is designed to allow patient participants in the Phase 1 trial to enroll in an open-label extension study in which dosing with WVE-210201 will continue. The open-label extension study and the planned efficacy study are each intended to follow the Phase 1 trial and expected to include an interim efficacy readout of dystrophin expression from muscle biopsies in 2H 2019.

Wave’s first global clinical trial in Duchenne is expected to enrol up to 40 patients between the ages of 5 and 18 years. The Phase 1 inclusion criteria allow for participation of both ambulatory and non-ambulatory patients, including those previously treated with eteplirsen following an appropriate washout period. The trial has been initiated in the United States, with Europe and other regions to follow. Intravenous doses tested in the Phase 1 trial will escalate through a range expected to be clinically relevant. In the U.S., Wave is required to provide data from ongoing preclinical studies to the FDA in order to progress to the highest dose cohorts and planned multi-dose studies.


About WVE-210201

WVE-210201 is an investigational stereopure antisense oligonucleotide that has been shown to induce skipping of exon 51 of dystrophin pre-mRNA in nonclinical 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 pre-clinical 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, versus approximately 1% when testing other exon-skipping compounds.

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