Eli Lilly & Co.: VERVE-102 Phase 1 Clinical Trial Success

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.

VERVE-102 was well tolerated across all dose levels with no treatment related serious adverse events (AEs) and no dose limiting toxicities reported. AEs related to VERVE-102 included low-grade infusion-related reactions and fatigue. All participants received the full planned dose, and no participant withdrew from the study.

Eli Lilly & Co. plans to initiate the Phase 2 clinical study of VERVE-102 before the end of the year, and will include U.S. research sites for the first time. If successful, the narrative logic of cardiovascular treatment is expected to change once again…