Verve Halts Trial for Lead Gene Editing Program Due to Safety Concerns

Verve Therapeutics has suspended enrollment in the Phase Ib Heart-1 study evaluating its lead gene editing program VERVE-101 following a serious adverse event, the company announced Tuesday.

A patient, who received a 0.45-mg/kg dose of VERVE-101, developed a grade 3 elevation in serum alanine aminotransferase levels, a key biomarker often used to assess liver damage. The patient also experienced grade 3 thrombocytopenia within four days after treatment. Both adverse events were deemed to be induced by VERVE-101.

The laboratory abnormalities did not translate into clinical symptoms, such as bleeding, and were fully resolved within a few days, according to Verve.

Still, an independent data and safety monitoring board recommended pausing enrollment in Heart-1 while Verve investigates the cause of the adverse events. The company has also reported these safety events to the FDA and other health regulatory authorities worldwide. VERVE-101’s Investigational New Drug application also remains active, according to the announcement.

Verve’s stock price dropped 31% in premarket trading on Tuesday in response to the news, reported Seeking Alpha.

Verve CEO Sekar Kathiresan in a statement said that the...