Exhibit 99.1

 

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Wave Life Sciences Reports Fourth Quarter and Full Year 2019 Financial Results and Provides Business Update

Innovative pipeline of stereopure oligonucleotides focused on CNS diseases

32 mg data from both PRECISION-HD clinical trials on track for 2H 2020

Two additional CNS programs – SNP3 and C9orf72 – on track to initiate clinical development in 2H 2020

Wave to host investor conference call and webcast at 8:00 a.m. ET today

CAMBRIDGE, Mass., March 2, 2020 – Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced financial results for the fourth quarter and full year ended December 31, 2019 and provided a business update.

“We continue to advance our innovative, CNS-focused pipeline of stereopure oligonucleotides across Huntington’s disease, amyotrophic lateral sclerosis, frontotemporal dementia and other central nervous system diseases. Despite the disappointment of discontinuing our Duchenne program last year, we are advancing more than a dozen programs across discovery and development with several exciting milestones ahead in 2020,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. “In December 2019, we shared the first clinical results from an allele-selective program for Huntington’s disease. Initial results from PRECISION-HD2 demonstrated a reduction in mutant huntingtin protein, with a safety profile that supports the addition of a higher dose cohort, and no change in total huntingtin protein as compared to placebo. In the second half of 2020, we expect to initiate clinical development for our SNP3 program as well as our C9orf72 program, both of which have benefitted from novel advances in our PRISM platform. In addition, we are pleased by recent progress across multiple CNS programs we are working on in collaboration with Takeda. Finally, we presented proof-of-concept data for our RNA-editing program in January, which demonstrated endogenous ADAR engagement in vitro. We expect to have initial in vivo results in 2020, and we look forward to sharing further updates on this exciting new modality.”

Business update

Wave is building a leading genetic medicines company focused on realizing the potential of stereopure oligonucleotides in diseases of the central nervous system, liver, and eye. Wave’s pipeline includes more than a dozen programs across discovery and development, spans multiple modalities and targets, and is intended to deliver transformational medicines to patients and families.

Central nervous system (CNS) diseases

Updates for PRECISION-HD clinical trials of WVE-120101 and WVE-120102 in Huntington’s disease

 

   

WVE-120101 and WVE-120102 allele-selectivity: Investigational WVE-120101 and WVE-120102 are currently the only compounds in clinical development designed to selectively target the mutant allele of the huntingtin (mHTT) gene, while leaving the wild-type (wtHTT) relatively intact. The wtHTT protein is important for neuronal function, and there is increasing evidence that it may be neuroprotective in an adult brain. Additionally, Huntington’s disease (HD) may be caused by a dominant gain of function in mHTT protein and a concurrent loss

 

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The following information was filed by Wave Life Sciences Ltd. (WVE) on Monday, March 2, 2020 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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