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Re: DewDiligence post# 210587

Wednesday, 11/15/2017 12:48:29 PM

Wednesday, November 15, 2017 12:48:29 PM

Post# of 257553
WVE up 10%...Wave Life Sciences Initiates Clinical Trial for Lead Program in Duchenne Muscular Dystrophy (DMD)
http://ir.wavelifesciences.com/phoenix.zhtml?c=254233&p=irol-newsArticle&ID=2314450

“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, MD, PhD, Professor of Neuroscience at the University of Oxford and Director of the Oxford Centre for Neuromuscular Disease, Oxford, UK. “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 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.

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.



Wave pipeline...
http://www.wavelifesciences.com/pipeline

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