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Wave Life Sciences Reports Third Quarter 2020 Financial Results and Provides Business Update
Results from all cohorts of PRECISION-HD1 and PRECISION-HD2 clinical trials and initial OLE data on track for 1Q 2021
Dosing in three new clinical trials with novel compounds incorporating PN chemistry and targeting SNP3 in HD, C9orf72 in ALS / FTD and exon 53 skipping in DMD expected in 2021
Alpha-1 antitrypsin deficiency announced as first ADAR editing program, with potential to address both lung and liver manifestations of the disease through correction of single RNA base mutation
Strengthened balance sheet with equity financing; cash runway extended into 2Q 2023
Wave to host investor conference call and webcast at 8:30 a.m. ET today
CAMBRIDGE, Mass., Nov. 9, 2020 (GLOBE NEWSWIRE) 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 third quarter ended September 30, 2020 and provided a business update.
The substantial progress made by Waves research and clinical teams during the third quarter has ushered in a new phase for the company, during which we are rapidly advancing several new programs incorporating our novel PN backbone chemistry modification. We are on track to file clinical trial applications for WVE-003 for Huntingtons disease and WVE-004 for amyotrophic lateral sclerosis and frontotemporal dementia this quarter. In addition, we are announcing today our plan to submit a clinical trial application in the first quarter of 2021 for a third PN backbone-containing molecule, WVE-N531 for patients with Duchenne muscular dystrophy who have mutations amenable to exon 53 skipping, said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. We are also excited to announce our first ADAR editing target, the SERPINA1 gene transcript, where a single base mutation is often the cause of alpha-1 antitrypsin deficiency. Using our unique ADAR editing capability to correct the RNA transcript, we will have the opportunity to address both liver and lung manifestations of the disease.
The 32 mg cohorts in the PRECISION-HD1 and PRECISION-HD2 Phase 1b/2a clinical trials continue to move ahead, and we look forward to sharing results from all cohorts, as well as initial data from the ongoing open-label extension trials, in the first quarter of 2021. We are well-positioned to progress our planned and existing programs with our current cash balance.
Recent Business Highlights
PRECISION-HD programs for Huntingtons disease (HD): Wave is developing a unique portfolio of investigational stereopure oligonucleotides designed to selectively target the mutant allele of the huntingtin (mHTT) gene, while leaving the wild-type (wtHTT) protein relatively intact. Waves approach to HD is guided by the recognition that, in addition to a gain of function of the mHTT protein, people with this disease have lost one copy of the wtHTT allele, leaving them with a smaller protective reservoir of healthy protein than unaffected individuals. A growing body of scientific evidence suggests that preserving as much of this essential protein as possible is important for favorable health outcomes over a lifetime with the disease.
The following information was filed by Wave Life Sciences Ltd. (WVE) on Monday, November 9, 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-Q Quarterly Report statement of earnings and operation as management may choose to highlight particular information in the press release.
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