Wave Life Sciences Reports First Quarter 2022 Financial Results and Provides Business Update
Delivered first clinical data demonstrating target engagement and translation of PN chemistry’s impact in clinic; Adapting ongoing Phase 1b/2a FOCUS-C9 clinical trial to optimize dose level and frequency, with additional single and multidose data expected throughout 2022
Clinical data also expected in 2022 from Huntington’s disease (WVE-003) and Duchenne muscular dystrophy (WVE-N531) trials
Robust preclinical datasets for first-in-class AATD program demonstrate restoration of levels of AAT relevant for potential lung protection and reduction of liver-damaging aggregates with GalNAc AIMers; IND enabling toxicology studies for lead AATD candidate on-track to initiate in 3Q 2022
Wave to host investor conference call and webcast at 8:30 a.m. ET today
CAMBRIDGE, Mass., May 12, 2022 (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 first quarter ended March 31, 2022 and provided a business update.
“Thus far in 2022, Wave has achieved several important milestones, with the highlight being our first clinical data demonstrating successful target engagement with WVE-004 in the ongoing FOCUS-C9 clinical trial for C9-ALS and C9-FTD. We observed potent and durable reductions of the poly(GP) biomarker with low single doses of WVE-004, demonstrating that our preclinical data for PN-containing oligonucleotides are beginning to translate in the clinic,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. “These initial results are compelling and reinforce the potential of our unique oligonucleotide platform and our expectation to see the advantages of PN chemistry manifest in our other pipeline programs. We also continue to rapidly advance our WVE-003 program for HD and WVE-N531 program for DMD towards initial data updates later this year. We are also pleased with the recognition we are receiving with our endogenous RNA editing modality, which is being highlighted through scientific presentations and our recent Nature Biotechnology publication. Alpha-1 antitrypsin deficiency (AATD) is uniquely suited for an RNA editing therapeutic, and our AATD program is rapidly advancing towards clinical development with IND enabling studies on track to initiate in the third quarter of this year.”
Recent Pipeline and Business Highlights
Announced first clinical data from ongoing FOCUS-C9 trial of WVE-004 for C9-ALS and C9-FTD demonstrating potent and durable target engagement with low, single doses
Continued to advance clinical trials evaluating WVE-003 targeting SNP3 for Huntington’s disease (HD) and WVE-N531 for Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping
Presented preclinical AIMer data for AATD program supporting the potential for a novel, first-in-class, subcutaneous therapeutic to address both lung and liver manifestations of disease
Scientific publications highlight breadth and potential of Wave’s therapeutic oligonucleotide platform, including novel PN-chemistry and RNA editing modality
Key Anticipated 2022 Milestones
WVE-004 for C9-ALS and C9-FTD:
WVE-003 for HD:
WVE-N531 for DMD:
AIMer GalNAc-conjugated program for AATD:
First Quarter 2022 Financial Results and Financial Guidance
Wave recorded revenue of $1.8 million for the first quarter of 2022, primarily under the Takeda Collaboration. Wave did not record any revenue under the Takeda Collaboration in the first quarter of 2021.
Research and development expenses were $27.5 million in the first quarter of 2022 as compared to $33.4 million in the same period in 2021. The decrease in research and development expenses in the first quarter was primarily due to decreased external expenses related to our previously disclosed discontinued PRECISION-HD programs, partially offset by increased internal and external expenses related to PRISM, including ADAR editing, and other ongoing programs.
General and administrative expenses were $12.4 million in the first quarter of 2022 as compared to $10.1 million in the same period in 2021. The increase in general and administrative expenses in the first quarter of 2022 was primarily due to increases in compensation-related expenses, as well as increases in professional services expenses and other general and administrative operating expenses.
As of March 31, 2022, Wave had $111.7 million in cash, cash equivalents and short-term investments. As of December 31, 2021, Wave had $150.6 million in cash and cash equivalents. This decrease was mainly due to Wave’s year-to-date net loss of $37.8 million.
Wave expects that its existing cash, cash equivalents and short-term investments will enable the company to fund its operating and capital expenditure requirements into the second quarter of 2023.
Investor Conference Call and Webcast
About the FOCUS-C9 Clinical Trial
In an initial data analysis, reductions in poly(GP) were observed across all active treatment groups (10 mg, n=2 patients; 30 mg, n=4 patients; 60 mg, n=3 patients), reaching statistical significance versus placebo (n=3 patients) after single 30 mg doses, with a 34% reduction in poly(GP) at day 85 (p=0.011). At the time of analysis, none of the patients dosed with 60 mg had reached day 85. As the poly(GP) reduction in the 30 mg single dose cohort does not appear to have plateaued, Wave will extend the observation period from approximately three months (85 days) to approximately six months to identify the maximum reduction of poly(GP) and duration of effect of low single doses. Based on the durability and potency observed in the 30 mg cohort, FOCUS-C9 has been adapted to include additional patients receiving 20 mg and 30 mg single doses of WVE-004. Adverse events (AEs) were balanced across treatment groups, including placebo, and were mostly mild to moderate in intensity. Four patients (including one on placebo) experienced severe and/or serious adverse events; three were reported by the investigators to be related to ALS or administration, and one was reported by the investigator to be related to study drug. There were no treatment-associated elevations in CSF white blood cell counts or protein and no other notable laboratory abnormalities were observed.
Support for FOCUS-C9 is provided by the Alzheimer’s Drug Discovery Foundation.
About Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
Frontotemporal dementia (FTD) is a fatal neurodegenerative disease in which progressive nerve cell loss in the brain's frontal lobes and temporal lobes leads to personality and behavioral changes, as well as the gradual impairment of language skills. It is the second most common form of early-onset dementia after Alzheimer’s disease in people under the age of 65. FTD affects as many as 70,000 people in the United States.
A hexanucleotide repeat expansion (G4C2) is the most common known genetic cause of the sporadic and inherited forms of ALS and FTD. The expansion leads to production of modified sense and antisense transcripts that can form nuclear RNA foci and encode dipeptide protein repeats (DPRs), which are believed to drive disease pathology. Additionally, the G4C2 expansion can decrease expression of C9orf72 protein, affecting regulation of neuronal function and the immune system.
In the United States, mutations of the C9orf72 gene are present in approximately 40% of familial ALS cases and ~8-10% of sporadic ALS cases. In FTD, the mutations appear in 38% of familial cases and 6% of sporadic cases.
About Huntington’s Disease
About Duchenne Muscular Dystrophy
About Wave Life Sciences
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