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WAVE Life Sciences Reports Fourth Quarter and Full-Year 2016 Financial Results and Provides Business Update
On track to take first three lead programs into the clinic in 2017
Clinical trials expected to commence in Huntingtons disease mid-2017,
Duchenne Muscular Dystrophy second half of 2017
CAMBRIDGE, Mass., March 16, 2017 WAVE Life Sciences Ltd. (NASDAQ: WVE), a genetic medicines company focused on developing targeted therapies for patients impacted by rare diseases, today reported financial results for the fourth quarter and full year ended December 31, 2016, and provided a business update.
Last year, we strengthened our foundation for growth and operational excellence in WAVEs core platform and neurology franchise, adding the leadership of Mike Panzara, M.D., M.P.H., and advancing our first three programs through to clinical candidates, said Paul Bolno, M.D., MBA, President and Chief Executive Officer of WAVE Life Sciences. In 2017, we are poised to enter the clinic with our first three neurology programs and nominate the next three candidates, while also beginning production of drug supply in our cGMP manufacturing facility. At the same time, we will continue to leverage our R&D platform through our hepatic collaboration with Pfizer, and deepen our knowledge and capabilities in novel modalities such as single-stranded RNAi.
Recent Highlights and Outlook
|||Advancing three lead neurological programs into the clinic in 2017. WAVEs two lead programs in Huntingtons disease (HD) and one lead program in Duchenne Muscular Dystrophy (DMD) are expected to advance into clinical trials in 2017. WAVE has made significant progress towards this goal with all three candidates demonstrating strong preclinical data throughout 2016.|
Huntingtons disease (HD): WVE-120101, WVE-120102
WVE-120101 and WVE-120102 each target a distinct patient population, which together account for more than two-thirds of the HD population. Each therapeutic candidate is designed to selectively silence mRNA transcript produced by the disease-causing mutant huntingtin (HTT) allele in order to reduce the mutant HTT protein while leaving the healthy HTT mRNA transcript relatively intact to produce normal protein. Preclinical studies have been completed and both WVE-120101 and WVE-120102 are expected to enter clinical trials in mid-2017. WAVE expects to initiate two simultaneous Phase 1b/2a multi-ascending dose trials to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamic effects, and potentially generate preliminary evidence of therapeutic effect in patients with early manifest HD.
Duchenne Muscular Dystrophy (DMD): WVE-210201
WAVE has selected its lead exon-skipping candidate, WVE-210201, to target deletions of Exon 51. 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. WAVE has initiated cGMP manufacturing, and is working closely with the patient community in development of its clinical trial protocol. WVE-210201 is expected to enter the clinic in the second half of 2017. The company intends to include both ambulatory and non-ambulatory patients, as well as patients previously treated with other exon-skipping compounds after appropriate washout in the trial protocol. Additional exon-skipping programs in preclinical development continue to advance.
|||Investing in R&D platform capabilities. WAVE continues to leverage its proprietary platform to design and develop single, well-characterized medicines that have optimized pharmacology, address underserved patient populations and expand treatment options. WAVEs rational drug design process that produces stereopure oligonucleotides enables reduced use of phosphorothioate (PS) modifications, alongside the naturally occurring phosphodiester (PO), while retaining other important aspects of pharmacology.|
WAVE continues to advance its single-stranded RNAi technology for applications in metabolic disease and expects to expand opportunities to engage RNAi silencing mechanisms in tissues outside the liver. Additional therapeutic areas under development include ophthalmology and dermatology.
Following promising preclinical distribution data in WAVEs CNS programs, and as WAVEs portfolio of programs expands into additional neurological disorders, WAVE is exploring the ability of chemical modifications to direct distribution and characterize productive uptake of nucleic acid cargo to specific cell-types and regions within the CNS. WAVE intends to select an additional three program candidates in 2017 and is on track to deliver six development programs by the end of 2018.
|||Developments in strategic partnerships. In May 2016, WAVE entered a collaboration with Pfizer, focused on the advancement of genetically defined targets for the treatment of metabolic diseases, bringing together WAVEs proprietary drug development platform, across antisense and single-stranded RNAi modalities, along with GalNAc and Pfizers hepatic targeting technology for enhanced delivery to the liver. The company expects to present initial data and learnings from this collaboration in 2017. WAVE continues to make progress under this collaboration with three programs advancing through lead-optimization, including ApoC-III. The remaining two Pfizer collaboration programs are expected to be nominated by November 2017.|
The following information was filed by Wave Life Sciences Ltd. (WVE) on Thursday, March 16, 2017 as an 8K 2.02 statement, which is an earnings press release pertaining to results of operations and financial condition. It may be helpful to assess the quality of management by comparing the information in the press release to the information in the accompanying 10-K Annual Report statement of earnings and operation as management may choose to highlight particular information in the press release.
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