Verve Therapeutics has paused enrollment in its Heart-1 clinical trial for VERVE-101, a cardiovascular disease gene editing treatment, due to a safety concern after a participant experienced temporary health issues.
The Boston-based biotech revealed this week that the patient, who received a 0.45 mg/kg dose, experienced temporary elevated ALT (liver enzyme) levels and low platelet count, although they showed no symptoms.
VERVE-101 has been fraught with regulatory hurdles. In November 2022, the FDA placed a hold on its IND application for its single-course, in vivo liver gene editing treatment targeting heterozygous familial hypercholesterolemia (HeFH), a genetic disorder causing high low-density lipoprotein cholesterol levels.
A month later, Verve received a clinical hold letter from the FDA explaining the hold, in which the agency asked for more details on accidental germline editing risks, potency differences between human and non-human cells, and off-target effects in non-hepatocyte cell types. To address the FDA's concerns, Verve submitted interim clinical data from its ongoing Heart-1 trial and addressed preclinical questions. By October 2023, the FDA had lifted the clinical hold, allowing Verve to proceed with U.S. trials for VERVE-101.
Now, while Verve investigates the recent lab abnormalities in VERVE-101 and works with regulatory authorities to define a path forward, the company is turning its attention to VERVE-102, a different PCSK9 gene editing treatment, with regulatory approval for trials in the U.K. and Canada expected to come in the second quarter of 2024.
