Verve Therapeutics Announces Interim Data for VERVE-101 Demonstrating First Human Proof-of-Concept for In Vivo Base Editing with Dose-Dependent Reductions in LDL-C and Blood PCSK9 Protein in Patients with Heterozygous Familial Hypercholesterolemia

LDL-C Reductions Up to 55% and Blood PCSK9 Protein Reductions Up to 84% Observed After a Single Infusion of VERVE-101 at Potentially Therapeutic Doses

Safety Profile Supports Continued Development of VERVE-101

Enrollment Ongoing in the 0.45 mg/kg and 0.6 mg/kg Cohorts with Plans to Initiate Expansion Cohort in 2024

Company to Host Conference Call and Webcast Today at 6:30 p.m. ET

BOSTON, Nov. 12, 2023 (GLOBE NEWSWIRE) -- Verve Therapeutics, Inc., a clinical-stage biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, today announced first human proof-of-concept data for in vivo base editing from the ongoing heart-1 phase 1b clinical trial of VERVE-101. Treatment with VERVE-101 led to dose-dependent reductions of disease-causing low-density lipoprotein cholesterol (LDL-C) in people living with heterozygous familial hypercholesterolemia (HeFH), a life-threatening inherited disease characterized by lifelong elevations in blood LDL-C and accelerated atherosclerotic cardiovascular disease (ASCVD). VERVE-101 is an investigational, in vivo base editing medicine designed to be a single-course treatment that inactivates the PCSK9 gene in the liver to durably lower blood LDL-C.

“Of the more than three million people with HeFH in the U.S. and Europe, very few are currently at LDL-C goal, due in part to a care model that requires lifetime therapies. This model puts a strain on the healthcare system and is failing our patients,” said Deepak L. Bhatt, M.D., M.P.H., Director of the Mount Sinai Fuster Heart Hospital and the Dr. Valentin Fuster Professor of Cardiovascular Medicine at the Icahn School of Medicine in New York. “I am very encouraged by the initial data from the heart-1 trial that demonstrated the potential for single-course gene editing as a new approach to treat patients with HeFH. The data showed that VERVE-101 could meaningfully and durably lower LDL-C in these patients. This trial enrolled patients with advanced coronary disease, and the cardiovascular adverse events were consistent with what might be expected in this patient population. We’re at an exciting moment for cardiovascular prevention where the management of ASCVD may fundamentally change.”