Eli Lilly and Company (NYSE: LLY) reported that its experimental gene editing treatment VERVE-102 reduced cholesterol levels by up to 62% in a phase 1b clinical trial, with effects lasting up to 18 months after a single intravenous dose.

The Heart-2 trial evaluated VERVE-102 in 35 participants with heterozygous familial hypercholesterolemia or premature coronary artery disease. The treatment reduced PCSK9 protein levels by 51% to 88% across different dose levels, with corresponding LDL cholesterol reductions ranging from 9% at the lowest dose to 62% at the highest dose of 1.0 mg/kg.

VERVE-102 uses base editing technology to permanently turn off the PCSK9 gene in liver cells. The treatment was well tolerated with no serious adverse events related to the drug and no dose-limiting toxicities reported. Side effects included mild infusion reactions and fatigue.

"Twenty years ago, genetics showed us that people born with PCSK9 naturally turned off have low LDL-C for life and are remarkably protected from heart attack, yet today's chronic therapies struggle to deliver this lifelong lowering," said Sekar Kathiresan, Lilly senior vice president and co-founder of Verve Therapeutics.

The FDA has granted Fast Track designation for VERVE-102 to reduce LDL cholesterol in participants with hyperlipidemia and high cardiovascular risk. Lilly plans to begin enrolling a phase 2 study by the end of this year.

The trial results were presented at the European Atherosclerosis Society Congress and published in The New England Journal of Medicine. Participants will be followed for up to 15 years to assess long-term safety and efficacy.