Exhibit 99.1

 

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Wave Life Sciences Reports Second Quarter 2017 Financial Results and Provides Business Update

CAMBRIDGE, Mass., August 9, 2017 – Wave Life Sciences Ltd. (NASDAQ: WVE), a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases, today announced financial results for the second quarter ended June 30, 2017.

“We recently achieved two major milestones: transitioning to a clinical stage company, with the commencement of our two phase 1b/2a trials in Huntington’s Disease, PRECISION-HD1 and PRECISION-HD2; and the opening of our new cGMP manufacturing facility,” said Paul Bolno, M.D., MBA, President and Chief Executive Officer of Wave Life Sciences. “Our lead DMD Exon 51 program is on track to enter the clinic later this year, and I am excited to announce our next development program targeting C9orf72 mutations in ALS and FTD. Our collaboration with Pfizer continues to advance with multiple programs progressing through in-vivo lead optimization. Within our discovery engine, we continue to generate compelling data for new modalities such as single-stranded RNAi, as well as splice correction. With the progression of our pipeline and advancement in our platform capabilities, we are closer to potentially bringing new hope and precision medicines to patients living with serious, genetically-defined diseases.”

Business Update

 

    Huntington’s disease allele selective programs (WVE-120101, WVE-120102) clinical trials initiated

In July, Wave announced the initiation of the Company’s PRECISION-HD program, which includes PRECISION-HD1 and PRECISION-HD2, two Phase 1b/2a global clinical trials evaluating WVE-120101 and WVE-120102, respectively, for patients with Huntington’s disease (HD). Both PRECISION-HD trials will follow the same protocol, and each will target a single nucleotide polymorphism (SNP), that marks a separate and distinct location on the mutant huntingtin (HTT) gene transcript. Both trials are multicenter, randomized, double-blind, placebo-controlled, and will primarily evaluate the safety and tolerability of single and multiple doses of WVE-120101 and WVE-120102, administered intrathecally in HD patients. PRECISION-HD1 and PRECISION-HD2are the first two clinical trials in the company’s history. Completion of the trials is anticipated for 2H 2019.

 

    DMD Exon 51 (WVE-210201) on track to initiate clinical trials 2H 2017

WVE-210201, Wave’s lead program for Duchenne muscular dystrophy (DMD), is expected to enter the clinic in the second half of 2017 as planned. At the end of the second quarter, Wave presented additional pre-clinical data at the Parent Project Muscular Dystrophy conference, which included additional Western blot data as well as further in-vivo stability data. These data supported previously reported pre-clinical findings, and demonstrated efficient exon 51 skipping, dystrophin protein restoration and high tissue stability.

 

    Expansion of pipeline with C9orf72 targeting program addressing amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD)

Today, Wave is announcing C9orf72 as its next program, aimed at treating the fatal, complex neurodegenerative disorders ALS and FTD. Wave is targeting pathological C9orf72 mutations resulting from repeat expansions in the gene. These expansions are currently known as the largest genetic cause of familial ALS and FTD, accounting for approximately one-third and one-quarter of patients, respectively. Mutations of C9orf72 are also considered to be a strong genetic risk factor for the more common, non-inherited, sporadic forms of ALS and FTD. Wave anticipates clinical trial initiation of ALS and FTD programs in late 2018. Further information on each of these programs will be disclosed and presented later this year.

 

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The following information was filed by Wave Life Sciences Ltd. (WVE) on Wednesday, August 9, 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-Q Quarterly Report statement of earnings and operation as management may choose to highlight particular information in the press release.

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