Verve Therapeutics, Inc., (NASDAQ:VERV), a biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, today highlighted key milestones anticipated in 2022 and announced new preclinical data in non-human primates (NHPs) on additional potential dosing regimens for its base editing programs for the treatment of atherosclerotic cardiovascular disease (ASCVD) indications.
“2021 was a year of remarkable progress across all aspects of our company, with data demonstrating robust and durable gene editing and well-tolerated safety profiles with both VERVE-101 and our ANGPTL3 base editor in NHP studies. We successfully executed our IPO and further strengthened our team with several highly talented individuals,” said Sekar Kathiresan, M.D., co-founder and chief executive officer of Verve. “As we look ahead, we expect 2022 will be a transformative year, with additional data expected at scientific and medical meetings throughout the year, leading to our planned transition to a clinical-stage organization in the second half of 2022. As pioneers in the industry for the treatment of cardiovascular disease, we have the opportunity to fundamentally shift how patients with ASCVD are treated and look forward to reporting more progress in the coming months.”
2022 Anticipated Milestones
Verve’s lead program, VERVE-101, is designed to permanently turn off the PCSK9 gene in the liver to reduce disease-driving LDL-C. VERVE-101 is being developed initially for the treatment of patients with heterozygous familial hypercholesterolemia (HeFH), a potentially fatal genetic heart disease. Previously reported data in NHPs demonstrated that a single administration of VERVE-101 led to robust, durable editing of the PCSK9 gene. Key program milestones anticipated in 2022 include:
- Clinical trial application (CTA) and investigational new drug (IND) submissions in the second half of 2022, and
- First HeFH patient treated in a Phase 1 clinical trial in the second half of 2022.
Verve’s second program is designed to permanently turn off the ANGPTL3 gene, a key regulator of cholesterol and triglyceride metabolism. Verve plans to develop this program initially for the treatment of both homozygous familial hypercholesterolemia (HoFH) and HeFH. Previously reported data have demonstrated that a single administration of Verve’s ANGPTL3-targeting base editor led to potent reductions in blood ANGPTL3 protein levels and LDL-C in a novel NHP model of HoFH. Key program milestones anticipated in 2022 include:
- Development candidate selection in the second half of 2022, and
- IND-enabling studies to begin in the second half of 2022.