Verve Therapeutics Announces Clearance of Investigational New Drug Application by the U.S. FDA for VERVE-102, an Investigational Gene Editing Medicine Designed to Durably Lower Cholesterol After a Single Dose

Core Insights - Verve Therapeutics has received FDA clearance for its Investigational New Drug (IND) application for VERVE-102, aimed at treating patients with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD) [1][2] - VERVE-102 is designed as a single-course treatment that inactivates the PCSK9 gene in the liver to durably lower low-density lipoprotein cholesterol (LDL-C) levels [1][2] - The company aims to provide lifelong cholesterol lowering with its gene editing medicines, addressing the limitations of existing cholesterol-lowering therapies [2] Clinical Trial Progress - The ongoing Heart-2 Phase 1b clinical trial for VERVE-102 is evaluating safety and tolerability, with interim clinical data submitted to the FDA as of January 10, 2025 [2][3] - Initial safety and efficacy data from the Heart-2 trial is expected to be announced in the second quarter of 2025, with participants having at least 28 days of follow-up [3] - The final data for the dose escalation portion of the Heart-2 trial is anticipated in the second half of 2025, along with the initiation of the Phase 2 clinical trial for the PCSK9 program [4] Company Overview - Verve Therapeutics is a clinical-stage company focused on developing genetic medicines for cardiovascular disease, transitioning treatment from chronic therapies to single-course gene editing [5] - The company's lead programs include VERVE-102, VERVE-201, and VERVE-301, targeting key cholesterol drivers of atherosclerosis [5] - VERVE-201 aims to turn off the ANGPTL3 gene for refractory hypercholesterolemia, while VERVE-301 targets the LPA gene to reduce Lp(a) levels, an independent risk factor for cardiovascular diseases [5]