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Verve Therapeutics Pauses Enrollment in Cardiovascular Base-Editing Drug After Safety Report

NEW YORK – Verve Therapeutics on Tuesday said it would pause enrollment in its Heart-1 Phase Ib trial of VERVE-101, the company's gene-editing therapy for cardiovascular conditions, as it investigates adverse events observed in one trial participant.

The company's stock price opened at $8.67 per share Tuesday, down 32.2 percent from $12.79 at the end of the day Monday, and dropped to a new 52-week low of $7.50 per share later Tuesday morning.

VERVE-101 is designed to use base editing to turn off the PCSK9 gene in the liver to reduce low-density lipoprotein cholesterol (LDL-C), which drives multiple cardiovascular diseases.

The Boston-based biotech said it will instead prioritize development of VERVE-102, another therapy designed to turn off the PCSK9 gene using base editing, but which uses a different lipid nanoparticle (LNP) delivery system than VERVE-101. The UK Medicines and Healthcare Products Regulatory Agency and Health Canada have cleared clinical trial applications for VERVE-102, and Verve plans to launch a Phase Ib trial to test the investigational therapy in patients with heterozygous familial hypercholesterolemia (HeFH) and premature coronary artery disease, dubbed Heart-2, in Q2.

Thirteen patients had received VERVE-101 as part of the Heart-1 trial, in which Verve was investigating safety, tolerability, and changes in blood PCSK9 protein and LDL-C levels in patients with HeFH, established atherosclerotic cardiovascular disease (ASCVD), and uncontrolled hypercholesterolemia.

LDL-C reductions were observed in most patients; however, one patient experienced an asymptomatic grade III drug-induced transient increase in serum alanine aminotransferase and a serious adverse event of grade III drug-induced thrombocytopenia within four days of treatment. These safety events, which may be attributable to the LNP delivery system, were reported to the US Food and Drug Administration, MHRA, and the New Zealand Medicines and Medical Devices Safety Authority. The patient did not experience bleeding or other symptoms related to laboratory abnormalities, and the issues resolved within a few days.

Verve, in consultation with the study's independent data and safety monitoring board, has decided to pause enrollment in the Heart-1 study as it investigates the laboratory abnormalities. The company said it expects to work with regulatory authorities to determine a path forward for VERVE-101. 

"The Heart-1 clinical trial continues to support proof of concept for in vivo base editing of the PCSK9 gene in the liver, with a meaningful and durable lowering of LDL-C," Verve Cofounder and CEO Sekar Kathiresan said in a statement. "We plan to further investigate the laboratory abnormalities observed in the Heart-1 clinical trial in order to inform the next steps for VERVE-101."

Verve's cash runway extends into late 2026, the company said.