US pharmaceutical company Eli Lilly and Company (NYSE:LLY) on Monday reported 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). The 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.

The company said that 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. 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 and no dose-limiting toxicities reported.

The US Food and Drug Administration (FDA) has granted Fast Track designation for VERVE-102 to reduce LDL-C in participants with hyperlipidaemia and high lifetime cardiovascular risk.

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