WAVE Life Sciences Receives Orphan Drug Designation from FDA for its Lead Candidate Designed to Treat Huntington’s Disease

Allele-specific targeting silences mutant HTT allele while leaving healthy HTT allele in tact


On track to file investigational new drug (IND) application in late 2016


CAMBRIDGE, Mass.— June 21, 2016—WAVE Life Sciences Ltd. (NASDAQ: WVE), a genetic medicines company focused on developing stereopure nucleic acid therapies for patients impacted by rare diseases, today announced that its lead candidate WVE-120101, which is being investigated for the treatment of Huntington’s disease (HD), has received Orphan Drug Designation by the U.S. Food and Drug Administration (FDA). WVE-120101 targets rs362307, a Single Nucleotide Polymorphism (SNP) that is associated with the disease-causing mutation in the huntingtin (HTT) gene. WAVE’s approach enables selective silencing of the disease-causing HTT allele, while leaving the healthy HTT allele to produce normally functioning protein.


“We are pleased to receive Orphan Drug Designation for our first Huntington’s disease therapy and to advance what may be the first allele-targeted therapy into clinical trials, particularly as there are no approved disease-modifying treatments for HD,” said Paul Bolno, M.D., MBA, President and Chief Executive Officer of WAVE Life Sciences. “WVE-120101 is the first of our two lead allele-specific antisense programs for HD, and we are on track to file investigational new drug (IND) applications for both in late 2016. Each program distinctly targets the underlying genetic cause of HD and between these two programs, a substantial majority of HD patients would be covered.  Our team at WAVE is driven by a sense of urgency and purpose as we work to advance disease-modifying treatments for HD patients.”


“Each potential new therapy for Huntington’s disease gives fresh hope to the more than 30,000 Americans symptomatic with HD and the 200,000 more who live fearfully at-risk of having inherited it,” said Louise Vetter, Chief Executive Officer of the Huntington’s Disease Society of America.  “We are grateful for the commitment that WAVE Life Sciences has to improve the lives of families affected by this devastating neurological disease.”


The Orphan Drug Act provides for economic incentives to encourage the development of drugs intended to treat, diagnose or prevent rare diseases and conditions affecting fewer than 200,000 people in the United States. Orphan drug designation would entitle seven years of market exclusivity in the United States if market approval is granted for WVE-120101.  Additional incentives may include tax credits related to clinical trial expenses, an exemption from the FDA user fee, and FDA assistance in clinical trial design.


About Huntington’s Disease

Huntington’s disease (HD) is a hereditary neurodegenerative fatal disease that causes the progressive breakdown of nerve cells in the brain. Symptoms of HD typically begin to manifest between the ages of 35-44 years and worsen over the next 10- to 25-year period.  Currently, there are no treatments that can slow or reverse the course of HD.  The National Institutes of Health (NIH) estimates that 30,000 people in the United States have HD. In addition to symptomatic patients, more than 200,000 in the United States carry the gene and are at risk of inheriting the disease.


About WAVE Life Sciences

At WAVE Life Sciences, we are driven by an unwavering passion and commitment to deliver on our mission of confronting challenging diseases by developing transformational therapies and empowering patients. We are utilizing our innovative and proprietary synthetic chemistry drug development platform to design, develop and commercialize stereopure nucleic acid therapeutics that precisely target the underlying cause of rare and other serious genetically defined diseases. Given the versatility of our chemistry platform, WAVE’s deep, diverse pipeline spans multiple modalities including antisense, exon-skipping, and single-stranded RNAi. For more information, please visit www.wavelifesciences.com and follow us on Twitter at @WAVELifeSci and LinkedIn.


Forward Looking Information

This press release contains forward-looking statements concerning our goals, beliefs, expectations, strategies, objectives and plans, and other statements that are not necessarily based on historical facts, including statements regarding the following: our filing of INDs and commencing clinical trials; our belief in the importance of selective silencing; the anticipated therapeutic benefits of stereopure therapies compared to other therapies; the potential of our stereopure approach and nucleic acid therapeutics; and the anticipated benefits of receiving orphan drug designation. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including the following: the ability of our preclinical programs to produce data sufficient to support the filing of INDs and the timing thereof; our ability to continue to build and maintain the company infrastructure and personnel needed to achieve our goals; the clinical results of our programs, which may not support further development of product candidates; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials; our effectiveness in managing future clinical trials and regulatory processes; the success of our platform in identifying viable candidates; the continued development and acceptance of nucleic acid therapeutics as a class of drugs; our ability to demonstrate the therapeutic benefits of our stereopure candidates in clinical trials, including our ability to develop candidates across multiple therapeutic modalities; our ability to obtain, maintain and protect intellectual property; our ability to enforce our patents against infringers and defend our patent portfolio against challenges from third parties; our ability to raise additional capital as needed; and competition from others developing therapies for similar uses, as well as the information under the caption “Risk Factors” contained in our Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March 30, 2016 and in other filings we make with the SEC. We undertake no obligation to update the information contained in this press release to reflect subsequently occurring events or circumstances.

Investor Contact:
Westwicke Partners
Chris Brinzey, 339-970-2843

Media Contact:
Feinstein Kean Healthcare
Jess Rowlands, 202-729-4089