Eli Lilly and Company has reported positive interim results from the ongoing Phase Ib Heart-2 clinical trial of VERVE-102, an investigational therapy intended as a one-time treatment for hypercholesterolaemia.

The single-ascending-dose, open-label trial showed notable reductions in LDL-cholesterol (LDL-C) and proprotein convertase subtilisin/kexin type 9 (PCSK9) protein after a single infusion.

It involved 35 participants with heterozygous familial hypercholesterolaemia (HeFH) or premature coronary artery disease (CAD).

A single dose of VERVE-102, administered intravenously, showed dose-dependent mean reductions in circulating PCSK9 ranging from 51% to 88% and corresponding reductions in LDL-C from 9% to 62% across six dose cohorts.

These results have been durable for up to 18 months post-treatment.

VERVE-102 was generally well tolerated, with no treatment-related serious adverse events or dose-limiting toxicities reported.

Adverse events included low-grade infusion reactions and fatigue, but all participants received their full doses, and none withdrew from the trial.

Lilly senior vice-president Sekar Kathiresan said: “20 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.

“The Heart-2 results provide early clinical evidence that a single dose of VERVE-102 may mimic the LDL-C lowering effects of PCSK9 cardioprotective variants, potentially transforming cardiovascular care from chronic management to a one-time treatment.”

The US Food and Drug Administration (FDA) has granted fast track designation to VERVE-102 for reducing LDL-C in people with hyperlipidaemia and elevated long-term cardiovascular risk.

Lilly plans to begin Phase II studies of VERVE-102 by this year's end.

Last month, Eli Lilly’s Jaypirca (pirtobrutinib) succeeded in its fourth Phase III trial in chronic lymphocytic leukaemia (CLL).