NEW YORK – The US Food and Drug Administration has granted Verve Therapeutics permission to begin a clinical trial to assess the activity of VERVE-102 in patients with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD), the company said Monday.
Verve already has begun testing the in vivo base-editing cardiovascular medicine within the Phase Ib Heart-2 trial outside the US, including in Canada and the UK. With the FDA's clearance of its investigational new drug (IND) application, the Boston-based biotech will be able to launch the clinical trial at sites in the US.
"With our ongoing Heart-2 clinical trial for VERVE-102 progressing internationally, we are excited to begin activating trial sites in the US as we expect it to play a key role in our continued clinical development," Verve Cofounder and CEO Sekar Kathiresan said in a statement.
VERVE-102 is designed to reduce low-density lipoprotein cholesterol (LDL-C) by inactivating the PCSK9 gene in the liver, using mRNA expressing an adenine base editor and a guide RNA targeting the PCSK9 gene.
In its IND application, Verve submitted interim data from the first three cohorts in the dose-escalation portion of the Heart-2 trial, in which Verve is evaluating VERVE-102's impact on blood PCSK9 and LDL-C levels, as well as its safety, tolerability, and pharmacokinetics, in adult patients with HeFH or premature CAD.
As of a data cutoff in January, patients appear to be tolerating VERVE-102 well in the clinical trial and haven't had any serious treatment-related adverse events. Verve expects to report demographic and initial safety and efficacy data from the Heart-2 trial in the second quarter of this year. The company said it is on track to report final data from the dose-escalation portion of the study in the second half of the year and initiate a Phase II trial.