Verve Therapeutics Announces Positive Initial Data from the Heart-2 Phase 1b Clinical Trial of VERVE-102, an In Vivo Base Editing Medicine Targeting PCSK9

Core Insights - Verve Therapeutics announced positive initial data from the Heart-2 Phase 1b clinical trial of VERVE-102, showing significant reductions in LDL-C and PCSK9 levels with a single infusion [1][2][10] Efficacy - A single infusion of VERVE-102 resulted in a mean LDL-C reduction of 53% and a maximum reduction of 69% in the 0.6 mg/kg dose cohort [1][11] - The trial demonstrated a dose-dependent response, with mean LDL-C reductions of 21% at 0.3 mg/kg, 41% at 0.45 mg/kg, and 53% at 0.6 mg/kg [11] - Among participants receiving a total RNA dose greater than 50 mg, a mean LDL-C reduction of 59% was observed [9][10] Safety and Tolerability - VERVE-102 was well-tolerated with no treatment-related serious adverse events and no clinically significant changes in liver enzymes or platelets [1][6] - One infusion-related reaction was reported, which was transient and resolved with acetaminophen [6] Clinical Trial Design - The Heart-2 trial is an open-label Phase 1b study evaluating the safety and tolerability of VERVE-102 in patients with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD) [4][5] - The trial includes four dose cohorts, with initial data reported from 14 participants across the first three cohorts [5] Future Plans - Verve plans to report final Heart-2 dose escalation data and initiate a Phase 2 clinical trial for VERVE-102 in the second half of 2025 [1][13] - The company expects to receive a decision from Eli Lilly regarding the PCSK9 opt-in in the second half of 2025 [14] Company Overview - Verve Therapeutics is focused on developing genetic medicines for cardiovascular disease, with VERVE-102 targeting the PCSK9 gene to reduce LDL-C levels [16] - The company aims to transform treatment from chronic therapies to single-course gene editing medicines [16]