FDA Places Hold on Verve’s IND for Gene-Editing Candidate
The FDA has placed a hold on Verve Therapeutics’ Investigational New Drug (IND) application to conduct a clinical trial evaluating the company’s VERVE-101 gene editing treatment to inactivate a specific gene in the liver and reduce low-density lipoprotein cholesterol.
The therapy candidate is targeted to treat patients with heterozygous familial hypercholesterolemia, an inherited genetic disorder that causes hardening of the arteries.
VERVE-101 is being evaluated in a phase 1 trial in the UK and New Zealand. So far, dosing has been completed in the first cohort of the dose-escalation portion of the clinical trial.
Verve said that it did not include the data from the ongoing study in its IND application to the FDA. The company is awaiting details from the agency on what’s needed for the study to go ahead.
The clinical hold is the first notable setback for Verve since it was co-founded by cardiologist and geneticist Sek Kathiresan in 2018. Kathiresan formed the company to develop a treatment that’s meant to prevent heart attacks for life with a single infusion. Since then, it raised $267 million in one of 2021’s larger biotech initial public offerings and became the first company to test “base” editing — a more precise form of CRISPR gene editing — in a human clinical trial.
That trial, known as heart-1, is underway in New Zealand and the U.K. and is testing VERVE-101 in patients with a rare and inherited form of high cholesterol. Three patients have been treated to date, and Verve hasn’t observed any treatment-related adverse events. All side effects have been mild, and a trial monitoring board has cleared the company to begin evaluating a higher dose, Verve said Monday. Initial results will be disclosed at a medical meeting next year.
“The safety profile observed in the first dose cohort with VERVE-101 is encouraging, and in-line with safety data generated with VERVE-101 in our preclinical studies,” said Andrew Bellinger, Verve’s chief medical and scientific officer, in a statement.
Enrollment will continue in New Zealand and the U.K. But Verve’s plans have now been slowed in the U.S., where regulators are closely scrutinizing gene editing medicines. Regulators previously paused applications of gene editing drugs from partners Vertex and CRISPR Therapeutics, as well as Editas Medicine, before allowing their initial trials to begin. Earlier this year, the FDA halted a request from Beam Therapeutics, Verve’s longtime partner, to start testing a gene editing cancer treatment and has since asked for more technical details.
Verve shares fell 25% in pre-market trading on Monday.
November 8, 2022
