Verve Therapeutics Announces Updates On PCSK9 Program, Company To Pause Enrollment In Heart-1 Clinical Trial Following Asymptomatic Grade 3 Transient Alt Elevation And Thrombocytopenia Seen In Sixth Participant Enrolled In 0.45 Mg/Kg Dose Cohort

Author: Benzinga Newsdesk | April 02, 2024 07:08am

VERVE-101 is being evaluated in the Heart-1 Phase 1b clinical trial with trial endpoints of safety and tolerability as well as changes in blood PCSK9 protein and low-density lipoprotein cholesterol (LDL-C) levels in patients living with heterozygous familial hypercholesterolemia (HeFH), established atherosclerotic cardiovascular disease (ASCVD), and uncontrolled hypercholesterolemia. Six participants have been dosed at 0.45 mg/kg of VERVE-101, with a total of 13 participants dosed in the study. For the first five participants in the 0.45 mg/kg cohort with follow-up to at least 28 days, VERVE-101 demonstrated time-averaged LDL-C reductions ranging from 21% to 73%, and averaging 46% (as of a data cut-off date of March 18, 2024). In the two patients with the longest follow-up in the 0.45 mg/kg or 0.6 mg/kg cohorts, LDL-C lowering has been durable out to 270 days, with follow-up ongoing.

However, the sixth participant treated in the 0.45 mg/kg cohort experienced a Grade 3 drug-induced transient increase in serum alanine aminotransferase (ALT) as well as a serious adverse event of Grade 3 drug-induced thrombocytopenia within the first four days after dosing. The participant did not experience any bleeding or other symptoms related to the laboratory abnormalities, and the abnormalities resolved fully within a few days.

In light of such observed laboratory abnormalities associated with VERVE-101, Verve, in consultation with the study's independent data and safety monitoring board (DSMB), has decided to pause enrollment in the Heart-1 clinical trial. Verve is conducting an investigation into the laboratory abnormalities and based on those results, expects to work with regulatory authorities to define a path forward for VERVE-101. These safety events were reported to the U.S. Food and Drug Administration (FDA), MHRA, and the New Zealand Medicines and Medical Devices Safety Authority (Medsafe). The VERVE-101 Investigational New Drug Application (IND) and other CTAs remain active.

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