In the Phase 1b Heart-2 trial, a single intravenous infusion of VERVE-102 produced dose-dependent lowering of PCSK9 and LDL-C, with both reductions sustained over follow-up of up to 18 months in participants at high risk for cardiovascular disease

VERVE-102 is designed to mimic the protective effect of naturally occurring loss-of-function variants in PCSK9, which are associated with markedly lower lifetime risk of coronary heart disease

Lilly plans to begin enrolling the Phase 2 clinical study of VERVE-102 by the end of this year

INDIANAPOLIS, May 25, 2026 /PRNewswire/ -- Eli Lilly and Company (NYSE:LLY) today announced positive Phase 1b Heart-2 study results for VERVE-102, an investigational in vivo base editing medicine designed to durably turn off the PCSK9 gene in the liver and lower blood low-density lipoprotein cholesterol (LDL-C) following a single infusion. The Heart-2 trial is evaluating VERVE-102 in adults with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD). These data were presented as a late-breaking oral presentation at the European Atherosclerosis Society (EAS) Congress and simultaneously published in The New England Journal of Medicine.

In the Heart-2 study, a single intravenous infusion of VERVE-102 resulted in meaningful lowering of circulating PCSK9 protein and corresponding reductions in LDL-C across all evaluated dose levels. In this interim analysis of 35 participants, a single dose of VERVE‑102 resulted in dose-dependent mean reductions in PCSK9 ranging from 51% to 88%, at the lowest 0.3 mg/kg dose to the highest 1.0 mg/kg dose, respectively. Corresponding mean reductions in LDL-C were 9% (0.3 mg/kg), 44% (0.45 mg/kg), 45% (0.6 mg/kg), 33% (0.7 mg/kg), 51% (0.8 mg/kg), and 62% (1.0 mg/kg). These reductions were sustained over time, with durability observed for up to 18 months following treatment.