FDA green-lights Verve’s IND for base editing cholesterol-lowering treatment

Verve Therapeutics announced that the U.S. FDA has cleared its investigational new drug (IND) application for VERVE-102 for the treatment of patients living with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD).

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).

With the Heart-2 clinical trial for VERVE-102 progressing internationally, the IND clearance allows Verve to begin activating trial sites in the U.S. As part of the IND submission, Verve provided the FDA with interim clinical data from the dose-escalation portion of the ongoing Heart-2 phase 1b trial, evaluating the safety and tolerability of VERVE-102 in patients living with HeFH and/or premature CAD, with additional analyses for pharmacokinetics and changes in blood PCSK9 protein and LDL-C levels.

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.

The IND clearance is good news for Verve, who revealed last month that Vertex had bowed out of a four-year research collaboration focused on an in vivo gene editing program for liver disease.