Verve Therapeutics (VERV) Jefferies London Healthcare Conference 2024 summary

Strategic vision and clinical approach

• Aims to provide a one-time gene-editing therapy for lifelong cholesterol reduction, shifting away from daily pills or intermittent injections.

• Lead program targets PCSK9 using a base editor delivered via GalNAc lipid nanoparticles, with human proof of concept established.

• Pipeline includes additional programs targeting ANGPTL3 and Lp(a), all designed for single intravenous administration.

Clinical trial progress and milestones

• Seven patients have been dosed in phase one of the lead program, with no significant safety signals observed so far.

• Initial phase one data for VERVE-102 expected in the first half of next year, with full dose escalation and final data in the second half.

• Phase two initiation for VERVE-102 is planned for the second half of next year.

• ANGPTL3 program (VERVE-201) has dosed its first patient and will continue enrolling through 2025.

Efficacy benchmarks and safety considerations

• Target LDL reduction is 40% for HeFH and 50% for ASCVD patients, aiming for lifelong effect after a single treatment.

• Efficacy can be assessed within 28 days post-treatment, with safety signals typically emerging acutely and resolving quickly.

• Dose escalation is flexible, aiming to reach target product profile without adverse effects.