Verve Therapeutics to present Phase 1b data on HeFH candidate

Verve Therapeutics 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 Scientific Sessions 2023 in Philadelphia November 11-13. VERVE-101 is a base editing medicine designed as 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. heart-1 is a Phase 1b clinical trial designed to evaluate the safety and tolerability of VERVE-101 in single ascending dose 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.