Verve Therapeutics (VERV) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Strategic focus and technology overview

• Developing one-time gene editing therapies for cardiovascular disease using base editing to permanently lower cholesterol by targeting the PCSK9 gene in the liver.

• Base editing approach makes a single DNA change, aiming for a functional cure for patients with high cholesterol, especially those with genetic conditions like HeFH.

• Intravenous infusion delivers the therapy, with preclinical data showing durable cholesterol reduction for at least three years in non-human primates.

Lead programs and clinical development

• Two lead programs, VERVE-101 and VERVE-102, both target PCSK9 but use different lipid nanoparticle (LNP) delivery systems; VERVE-101 is paused due to reversible lab abnormalities, while VERVE-102 uses a new, better-tolerated LNP with GalNAc for liver targeting.

• VERVE-102 is in a phase 1 single ascending-dose trial (Heart-2), enrolling both HeFH patients and those with early heart attacks, with interim data expected in the first half of 2025.

• Heart-2 trial now recruits patients with less advanced disease, using non-invasive screening to reduce confounding safety events.

• Dose escalation will inform phase 2 design, with plans to select a dose and initiate phase 2 in the second half of 2025; phase 2 may be placebo-controlled with 50–100 patients.

Market opportunity and unmet need

• HeFH affects about 3 million people in the US and Europe, with only 2% at cholesterol goal; current therapies have poor adherence and limited effectiveness.

• A one-time therapy could address the significant unmet need for lifelong cholesterol lowering, especially in patients with poor adherence to existing treatments.