Verve Therapeutics (VERV) Jefferies 2024 Global Healthcare Conference summary

Key product updates and clinical data

• Developing once-and-done gene-editing therapies for heart disease, aiming for permanent LDL cholesterol reduction.

• VERVE-101 showed up to 73% LDL reduction and durable effect for 9+ months, but had transient lab abnormalities linked to its delivery system.

• VERVE-102 uses a new ionizable lipid and GalNAc ligand for improved safety, with preclinical and third-party data supporting better tolerability.

• HEART-2 trial targets patients with HeFH and premature coronary artery disease, using a single ascending dose design.

• Next data update and Lilly opt-in decision expected in 2025, based on dose escalation results.

Strategic partnerships and development plans

• Lilly holds an option for minority product rights, with a decision post-phase one based on dose escalation data.

• If Lilly opts in, it will fund a third of global development costs for 50% of US profits; Verve retains development control and ex-US rights.

• Phase two will be a randomized controlled trial to select doses for phase three, modeled after siRNA precedents.

• Registration trial benchmark is about 500 patients, with LDL cholesterol as the primary endpoint.

Market positioning and future outlook

• One-time gene-editing therapy aims to address poor adherence to chronic cholesterol-lowering regimens.

• Surveys show about a third of patients would prefer a one-and-done treatment.

• PCSK9 therapies currently reach only 2% of eligible patients, representing a large untapped market.

• Initial focus is on genetic high-cholesterol patients, with potential expansion to broader heart attack populations.