WAVE Life Sciences Announces Plan to Deliver Six Clinical Programs by 2018

Company outlines strategy to become genetic medicine leader

Over 20 stereopure antisense, exon skipping, and RNAi programs in early discovery and development across multiple therapeutic areas that have the potential to be best-in-class

Development efforts focused on neurological and neuromuscular rare diseases; Company plans to file INDs for lead programs in Huntington's disease by end of 2016

CAMBRIDGE, Mass.--(BUSINESS WIRE)--WAVE Life Sciences Ltd. (NASDAQ: WVE), a genetic medicine company focused on advancing potential best-in-class stereopure nucleic acid therapies, today announced its plan to deliver six clinical programs by 2018. The announcement follows WAVE's successful initial public offering in November 2015, in which the company raised approximately $112 million in gross proceeds, bringing the total capital closed in 2015 to approximately $196 million. "Over the next three years, WAVE plans to bring multiple novel therapies into the clinic, offering new hope to patients with rare diseases and their caregivers," said Paul Bolno, M.D., MBA, President and Chief Executive Officer at WAVE Life Sciences. "We believe that we have the leading genetic medicines platform and continue to build both the team and the infrastructure to execute on our vision."

To support its ambitious goals, WAVE's plan includes:

  • First two INDs on track for filing in 2016. WAVE continues to make progress on its first two allele-targeted antisense programs, each distinctly targeting the underlying genetic cause of Huntington's disease (HD), and expects to file two investigational new drug applications (IND) by the end of 2016. Each of WAVE's HD programs enables selective silencing of the disease-associated allele by targeting single nucleotide polymorphisms present only on mutated alleles in subsets of patients with HD. WAVE believes this strategy may increase the therapeutic window relative to current stereo-random oligonucleotide and gene therapy programs that do not distinguish between alleles.
  • Advancement of multiple nucleic acid therapeutic modalities.WAVE's expertise, capabilities, and expansive patent portfolio for its stereopure nucleic acid platform enables the company to advance therapies across multiple modalities, including antisense, exon skipping, and single-strand RNAi, the last of which could potentially unify the broad distribution of antisense with the superior potency of RNAi. In addition, WAVE's pipeline includes allele-specific development programs that hold the promise of targeting the root genetic cause of over a dozen gain-of-function orphan disorders uniquely suited to the company's proprietary chemistry.
  • Expansion of pipeline across rare diseases. WAVE's current pipeline of over twenty discovery programs includes a potentially best-in-class exon-skipping program to treat Duchenne muscular dystrophy (DMD); as well as first-in-class antisense programs targeting KRT14, a key driver of the serious orphan disease Epidermolysis bullosa simplex. Additional programs include therapies to treat central nervous system diseases such as spinal muscular atrophy type 1 and amyotrophic lateral sclerosis, neuromuscular diseases including DMD and myotonic dystrophy type 1, genetic and metabolic liver diseases, and dermatological, eye, and gastrointestinal diseases
  • Launch of patient engagement initiatives; appointment of Wendy Erler as Head of Patient Engagement. Ms. Erler joins WAVE from Biogen where she was responsible for developing the company's rare disease patient advocacy strategies for amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), and hemophilia. Most recently, she led teams during two successful hemophilia commercial product launches and field executions. Ms. Erler will direct WAVE's efforts to institute programs and partnerships with rare disease advocacy communities, to engage their input and continued involvement as the company advances its clinical development programs.
  • Rapid growth of employee base and footprint in Cambridge, Mass.; appointment of Peter Lasky as Head of Human Resources. Mr. Lasky joins WAVE from Sekisui Diagnostics (formerly Genzyme Diagnostics). Prior to this, Mr. Lasky was SVP Head of Global Human Resources at Shire Pharmaceuticals, where he developed and implemented the company's global growth and expansion strategy. WAVE intends to approximately double its employee base this year to support its rapid expansion. The company recently moved its U.S. headquarters to Cambridge, Mass., and now occupies 32,000 square feet of combined office and laboratory space to accommodate this growth.

"WAVE's stereopure drug candidates have the potential to transform the nucleic acid therapeutic class in much the same way as humanization optimized monoclonal antibodies," said Peter Kolchinsky, Ph.D., Managing General Partner of RA Capital and a director of WAVE. "WAVE's progress over the past 12 months has been remarkable. The company has the necessary cash runway to deliver on the promise inherent in its technology. Undoubtedly, WAVE is poised for tremendous growth."

About WAVE Life Sciences

WAVE Life Sciences is utilizing its 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 genetic diseases, delivering new treatment options for patients. Given the versatility of its chemistry platform, WAVE's pipeline is designed to span multiple oligonucleotide modalities including antisense, exon-skipping, and single-stranded RNAi. For more information, please visit www.wavelifesciences.com and follow us on Twitter and LinkedIn.

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; the future performance and results of our programs in clinical trials; our identification of future candidates and their therapeutic potential; the anticipated therapeutic benefits of stereopure therapies compared to other therapies; our advancing of therapies across multiple modalities and the anticipated benefits of that strategy; our future growth; the potential of our stereopure approach and nucleic acid therapeutics; and our cash position supporting our anticipated business activities. 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 studies; 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 Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) 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.


Media Contact:
Feinstein Kean Healthcare
Liz Melone, 617-256-6622
Investor Contact:
Westwicke Partners
Chris Brinzey, 339-970-2843