FDA Lifts Hold On Verve's Gene-Editing Therapy

(RTTNews) - Clinical-stage biotechnology company Verve Therapeutics, Inc. Monday announced the lifting of the clinical hold and clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) to conduct a clinical trial in the United States evaluating VERVE-101 for the treatment of heterozygous familial hypercholesterolemia (HeFH).

VERVE-101 is an 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). HeFH is a prevalent and life-threatening inherited disease characterized by lifelong elevations in blood LDL-C and accelerated atherosclerotic cardiovascular disease (ASCVD).

Verve submitted interim clinical data from the dose-escalation portion of the ongoing heart-1 Phase 1b clinical trial and addressed the FDA's preclinical questions in its response to the clinical hold. The heart-1 trial is evaluating the safety, tolerability, pharmacokinetic and pharmacodynamic profile of VERVE-101 in patients with HeFH, and is currently being conducted at sites in the United Kingdom and New Zealand. Verve is scheduled to report interim clinical data from the heart-1 trial in a late-breaking science presentation at the American Heart Association's (AHA) Scientific Sessions 2023 on November 12, 2023 at 3:30pm E.T. in Philadelphia.

"The clearance of our IND application by the FDA is a significant milestone in our effort to offer patients living with HeFH a transformative alternative to the chronic care model of disease management. This clearance, for the first time, enables clinical development of an in vivo base editing product candidate in the United States," said Andrew Bellinger, M.D., Ph.D., chief scientific officer of Verve. "Our interactions with the FDA have been valuable, and we plan to incorporate our learnings from this regulatory process to execute a global regulatory strategy across the rest of our pipeline. With the clearance of this IND, we plan to begin the process of activating U.S. clinical trial sites for the heart-1 clinical trial."