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Exhibit 99.1
Wave Life Sciences Reports Third Quarter 2017 Financial Results and Provides Business Update
CAMBRIDGE, Mass., November 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 third quarter ended September 30, 2017.
Through the growth of talent and capabilities in our organization and excellent execution throughout the quarter, we achieved our ambitious clinical and research goals. Importantly, we initiated our third clinical program of 2017 with a Phase 1 study investigating our DMD Exon 51 program, WVE-210201, said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. Our first peer reviewed publication was featured in Nature Biotechnology, highlighting the founding principles of Waves stereopure chemistry platform and approach. With the initiation of our first three clinical programs this year and another three programs recently announced, we are on track to meet our objective of six development programs underway by the end of 2018.
Business Update
| DMD Exon 51 (WVE-210201) program initiates clinical trial |
On November 6, Wave announced the initiation of clinical development for WVE-210201, Waves lead program for Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping. We expect that data from the Phase 1 trial for WVE-210201 will be available in Q3 2018 and will facilitate the rapid transition to an open-label extension study and efficacy study. Both studies following the Phase 1 are designed to include an interim efficacy readout of dystrophin expression from muscle biopsies in 2H 2019. The company is exploring both intravenous and subcutaneous administration.
| Huntingtons disease allele selective programs (WVE-120101, WVE-120102) clinical trials on track |
The companys PRECISION-HD program, which includes two Phase 1b/2a global clinical trials evaluating WVE-120101 and WVE-120102 for patients with Huntingtons disease (HD), continues to enroll and it is on track to report topline data in 1H 2019.
| First peer-reviewed publication in Nature Biotechnology |
In August, Wave announced its first peer-reviewed publication in the September issue of Nature Biotechnology, representing a significant scientific advancement for the oligonucleotide field. The paper, entitled Control of phosphorothioate stereochemistry substantially increases the efficacy of antisense oligonucleotides, describes a breakthrough method to produce antisense oligonucleotide (ASO) therapeutics with high stereochemical purity as well as rational drug design to control pharmacologic properties in nucleic acid therapeutics drug development more broadly.
The paper outlines early foundational principles discovered by Wave to engage RNase H1 that can be applied to any ASO sequence, and demonstrated that stereochemistry plays a central role in oligonucleotide drug design, with the potential to improve stability, duration of activity and specificity. The companys highly efficient manufacturing process may allow for these key findings to translate into breakthrough treatments by applying nucleic acid therapeutics. Wave is continuing to leverage these initial findings to further build its knowledge base and expand its platform capabilities beyond antisense, including ongoing work in exon skipping, single stranded RNAi and other modalities.
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