Verve shares positive data from base editing cholesterol-lowering treatment trial

Verve Therapeutics announced positive initial data from a phase 1b clinical trial of its in vivo base editing therapy in patients with heterozygous familial hypercholesterolemia and/or premature coronary artery disease, teeing up a possible opt-in from Eli Lilly.

VERVE-102 is a novel, investigational in vivo base editing medicine designed to be a single-course treatment that inactivates the PCSK9 gene in the liver to durably lower blood low-density lipoprotein cholesterol (LDL-C).

Among 14 participants across three dose levels in the Heart-2 trial, VERVE-102 was well-tolerated, with no treatment-related serious adverse events and no clinically significant laboratory abnormalities observed. A single infusion of VERVE-102 led to dose-dependent decreases in blood PCSK9 protein levels and LDL-C, with a mean reduction in blood LDL-C of 53% and a maximum LDL-C reduction of 69% observed among four participants in the 0.6 mg/kg dose cohort.

“These initial Heart-2 data are promising with respect to both safety and efficacy and suggest the potential for a new era of cardiovascular disease treatment where a single dose might lead to lifelong control of LDL-C,” said Eugene Braunwald, M.D., distinguished Hersey professor of medicine at Harvard Medical School and founding chairman, TIMI Study Group, Brigham and Women’s Hospital.

Verve expects to announce the final data from the dose escalation portion of the Heart-2 clinical trial, including durability data, in the second half of 2025.

The company plans to dose the first patient in the Phase 2 clinical trial of VERVE-102 in the second half of 2025. The U.S. FDA cleared Verve’s IND application for VERVE-102 last month, allowing Verve to begin enrolling patients at U.S. trial sites.

The VERVE-102 asset is linked to a deal with Eli Lilly. Through Lilly’s acquisition of Beam Therapeutics in October 2023, the drugmaker picked up certain product rights to Verve’s cardiovascular in vivo gene editing programs targeting PCSK9 and ANGPTL3, which included VERVE-102. Lilly has the right to opt-in to share 33% of worldwide development expenses and to jointly commercialize and share profits and expenses related to commercialization in the U.S. on a 50/50 basis. Verve plans to deliver the opt-in data package for VERVE-102 to Lilly in the second half of 2025.