Single infusion of VERVE-102 led to dose-dependent decreases in blood PCSK9 and LDL-C, with mean reduction in LDL-C of 53% and a maximum reduction of 69% observed in the 0.6 mg/kg dose cohort VERVE-102 was well-tolerated with no treatment-related serious adverse events and no clinically significant changes in ALT or platelets observed at any dose level among 14 participants VERVE-102 utilizes a proprietary GalNAc-LNP which has demonstrated a potentially best-in-class safety profile In the second half of 202 ...

Core Insights - Verve Therapeutics has received Fast Track designation from the FDA for its investigational drug VERVE-102, aimed at treating hyperlipidemia and reducing LDL-C levels in patients at high cardiovascular risk [1][2] - VERVE-102 is designed as a single-course treatment that permanently disables the PCSK9 gene in the liver, potentially transforming the management of atherosclerotic cardiovascular disease [1][5] - The company is currently conducting the Phase 1b Heart-2 clinical trial to assess the safety and tolerability of VERVE-102 in patients with heterozygous familial hypercholesterolemia and/or premature coronary artery disease [1][3] Company Overview - Verve Therapeutics is a clinical-stage company focused on developing genetic medicines for cardiovascular diseases, with a goal to shift treatment from chronic therapies to single-course gene editing solutions [5] - The company's lead programs include VERVE-102, VERVE-201, and VERVE-301, targeting key cholesterol drivers associated with atherosclerosis [5] - VERVE-201 aims to turn off the ANGPTL3 gene for patients with refractory hypercholesterolemia, while VERVE-301 targets the LPA gene to reduce Lp(a) levels, an independent risk factor for cardiovascular diseases [5] Clinical Trial Updates - Initial demographic and safety data from the Heart-2 clinical trial are expected to be announced in Q2 2025, including results from three dose cohorts [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]