Verve’s Cholesterol-Lowering Treatment Shows No Safety Issues in Trial

Verve Therapeutics has won FDA clearance of its Investigational New Drug (IND) application for VERVE-102 for the treatment of patients with 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).

Verve Therapeutics is a clinical-stage company developing a new class of genetic medicines for cardiovascular disease with the potential to transform treatment from chronic therapies to single-course gene editing medicines. The company’s lead programs target the three cholesterol drivers of atherosclerosis. “The IND clearance from the U.S. FDA represents an important step in our journey to advance a new class of in vivo gene editing medicines for people worldwide living with cardiovascular disease,” said Sekar Kathiresan, M.D., Verve co-founder and CEO.

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 clinical trial. The Heart-2 clinical trial is evaluating the safety and tolerability of VERVE-102. Results show VERVE-102 has been well-tolerated, with no treatment-related serious adverse events and no clinically significant laboratory abnormalities observed.

In the second quarter of 2025, Verve expects to announce demographic and initial safety and efficacy data from the Heart-2 clinical trial. In addition, in the second half of 2025, Verve remains on track to report the final data for the dose escalation portion of the Heart-2 clinical trial, deliver the opt-in data package for the PCSK9 program to Eli Lilly, and initiate the Phase 2 clinical trial for the PCSK9 program. (Source: Verve Therapeutics Website, 24 March, 2025)