Wave Life Sciences, a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases, announced the initiation of a global phase I clinical trial for WVE-210201 in Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping. This clinical trial marks the next stage in the company’s ongoing commitment to address the significant unmet needs of patients diagnosed with this devastating disease and it is the company’s third clinical trial initiated in 2017.

“The initiation of WVE-210201’s clinical program is an important milestone in potentially delivering meaningful therapies for DMD patients,” said Michael Panzara, M.D., 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.”

The phase I 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 DMD patients with gene mutations amenable to exon 51 skipping. Data from the phase I 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 I 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 I trial and expected to include an interim efficacy readout of dystrophin expression from muscle biopsies in 2H 2019.

“Wave’s stereopure chemistry platform has enabled the development of WVE-210201 which has shown substantially greater exon 51 skipping efficiency and dystrophin protein restoration in preclinical studies as compared with stereorandom oligonucleotides, including morpholino oligonucleotides,” said Matthew Wood, M.D., Ph.D., Professor of Neuroscience at the University of Oxford and Director of the Oxford Centre for Neuromuscular Disease, Oxford, U.K. “These experimental results suggest the potential for meaningful benefits for DMD patients.”

Wave’s first global clinical trial in DMD is expected to enroll up to 40 patients between the ages of 5 and 18 years. The phase I 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 U.S., with Europe and other regions to follow. Intravenous doses tested in the phase I 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.

“Parent Project Muscular Dystrophy is excited by the progress Wave Life Sciences has made in exon skipping. While advances have been made, there is more to explore within this technology and we appreciate the passion, partnership and understanding the Wave team has shown our Duchenne community. We look forward to continued updates as Wave heads into its phase I clinical trial,” said Pat Furlong, founding President and CEO of Parent Project Muscular Dystrophy.

In addition to its exon 51 program, Wave is leveraging its stereopure chemistry platform to advance investigational therapies targeting additional DMD-related exons. In September 2017, Wave announced that its next DMD development program will target exon 53 and is expected to initiate clinical trials in Q1 2019. In addition, the company is exploring both intravenous and subcutaneous administration for the WVE-210201 and exon 53 programs.