BOSTON, Sept. 26, 2023 (GLOBE NEWSWIRE) -- Verve Therapeutics, a clinical-stage biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, today announced that the company will present interim data from its ongoing heart-1 Phase 1b clinical trial of VERVE-101 for patients with high-risk heterozygous familial hypercholesterolemia (HeFH) in a late-breaking science presentation at the American Heart Association's (AHA) Scientific Sessions 2023 being held in Philadelphia from November 11-13.
VERVE-101 is an investigational, in vivo base editing medicine designed to be a single-course treatment that inactivates the PCSK9 gene in the liver to durably reduce disease-driving low-density lipoprotein cholesterol (LDL-C). VERVE-101 is being developed initially as a treatment for patients with HeFH, a prevalent and potentially life-threatening genetic cardiovascular disorder that causes life-long severely elevated LDL-C, leading to increased risk of early-onset atherosclerotic cardiovascular disease (ASCVD).
heart-1 is a Phase 1b clinical trial designed to evaluate the safety and tolerability of VERVE-101 in single ascending dose (SAD) cohorts of the highest risk patients with HeFH, established ASCVD, and uncontrolled LDL-C levels on oral standard-of-care therapy. In addition, the clinical trial is designed to measure PCSK9 protein and LDL-C changes in patients and assess the potential for early proof-of-concept of the ability to base edit in the liver.
At AHA, Verve expects to report initial safety and pharmacodynamic data, as well as blood PCSK9 and blood LDL-C levels, from patients across four SAD cohorts.
Details of the late-breaking science session are as follows:
- Title: Safety and Pharmacodynamic Effects of VERVE-101, an Investigational DNA Base Editing Medicine Designed to Durably Inactivate the PCSK9 Gene and Lower LDL Cholesterol - Interim Results of the Phase 1b heart-1 Trial
- Session: Future of Lipid Lowering Therapy – Novel Mechanisms and Approaches
- Date and Time: Sunday, ...