Verve Therapeutics (VERV) Goldman Sachs 46th Annual Global Healthcare Conference summary

Strategic vision and clinical progress

• Focus on one-time gene editing therapy for lifelong cholesterol reduction to address atherosclerotic cardiovascular disease, aiming for enduring efficacy where current therapies fall short.

• HEART-2 trial of VERVE-102 showed up to 69% LDL reduction (mean 59%) at highest dose, with strong safety and tolerability across 14 patients.

• Durability data from earlier product (VERVE-101) demonstrated sustained LDL lowering for two years post-infusion, supporting the one-dose future vision.

• VERVE-102 uses a novel lipid nanoparticle delivery system with GalNAc ligand for liver targeting, improving safety and therapeutic index over VERVE-101.

• Dose escalation continues to identify the optimal fixed dose, with a transition to fixed dosing planned for phase II later in the year.

Safety, durability, and regulatory outlook

• No major safety issues observed with VERVE-102; no ALT or platelet elevations, and only one mild infusion reaction among 14 patients.

• Durability of effect is supported by both preclinical and human data, with edits persisting beyond typical liver cell turnover, and potential for redosing if needed.

• Long-term follow-up (15 years) is planned for all patients, with four to five years of data expected at registration.

• Regulatory agencies are increasingly supportive of in vivo gene editing, with recent high-profile cases and policy statements highlighting openness to transformative therapies.

Market research, patient and physician perspectives

• Surveys show strong openness among patients and cardiologists for a one-time gene editing therapy, especially among younger patients and those with genetic high cholesterol.

• About a third of patients and 40% of cardiologists (for HeFH) prefer a one-time gene edit over daily pills or injections; 20% preference among ASCVD patients.

• Payers may be more flexible on pricing due to large patient populations and low manufacturing costs, with potential for significant margin flexibility.