Verve Therapeutics announces FDA clearance of IND application for VERVE-102

Verve Therapeutics (VERV) announced the clearance of its Investigational New Drug application by the U.S. Food and Drug Administration for VERVE-102 for the treatment of patients living with heterozygous familial hypercholesterolemia and/or premature coronary artery disease. 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. 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 for VERVE-102. The Heart-2 clinical trial is 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 and is currently being conducted at sites outside of the U.S. Data submitted to the FDA had a cut-off date of January 10, 2025, and included participants across the first three dose cohorts. As of the data cut-off date, 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. Enrollment in the Heart-2 clinical trial is progressing well, and the initial data set is expected to include participants across the first three dose cohorts with at least 28 days of follow-up for each participant.