===Verve Therapeutics (VERV) reported earnings on Mon 7 Nov 22 (b/o)

• reports VERVE-101 Investigational New Drug Application placed on hold by FDA 

Verve Therapeutics reports VERVE-101 Investigational New Drug Application placed on hold by U.S. Food and Drug Administration 

• Verve announced today that the U.S. Food and Drug Administration (FDA) has placed a hold on its IND application to conduct a clinical trial evaluating VERVE-101 in patients with heterozygous familial hypercholesterolemia (HeFH), a prevalent and potentially life-threatening subtype of atherosclerotic cardiovascular disease (ASCVD), in the United States. VERVE-101 is a novel, investigational gene editing medicine designed to be a single-course treatment to permanently turn off the PCSK9 gene in the liver to reduce disease-driving low-density lipoprotein cholesterol (LDL-C). VERVE-101 is currently being evaluated in the heart-1 Phase 1 clinical trial in New Zealand and the United Kingdom (U.K.). Verve submitted its IND application for VERVE-101 to the FDA in October and received notification of a hold from the FDA on Friday, November 4, 2022. The company expects to receive an official letter with the FDA's questions within 30 days. Verve plans to provide updates pending engagement with the FDA and intends to work closely with the FDA to resolve the hold as promptly as possible in order to initiate dosing in the U.S.
• heart-1 Trial Update: Clinical data from the ongoing heart-1 study in New Zealand and the U.K. were not included in the IND package submitted to the FDA. Verve has completed dosing of VERVE-101 in the first dose cohort of the dose-escalation portion of the heart-1 trial, which was well tolerated in all three patients. There have been no treatment-related adverse events reported to date, and all adverse events observed have been Grade 1 in nature. The independent Data Safety Monitoring Board (DSMB) has reviewed safety data from the first cohort and recommended dose escalation to the planned second dose level, which is expected to begin soon. Enrollment efforts are ongoing in New Zealand and the U.K. The company plans to report initial safety and pharmacodynamic data for all dose cohorts of the dose-escalation portion of the heart-1 study at a medical meeting in the second half of 2023.