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

Strategic vision and clinical progress

• Focus on gene editing for cardiovascular disease aims for a one-time therapy with lifelong LDL cholesterol reduction, addressing unmet needs in enduring efficacy.

• HEART-2 trial for 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 reduction for two years post-infusion, supporting the one-dose concept.

• 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 linear relationship between RNA dose and LDL reduction observed so far.

Regulatory and safety landscape

• Regulatory agencies are increasingly supportive of in vivo gene editing, with recent high-profile endorsements and openness to root-cause therapies.

• Long-term safety is monitored through mandated 15-year follow-up, with risk-benefit analysis favoring intervention for high-risk patients.

• Off-target editing risk is minimized by design, and LNP-based therapies can be redosed if needed, unlike viral vectors.

• Two-year durability and safety data are considered strong indicators for longer-term outcomes by external bodies like ICER.

Market adoption and competitive landscape

• Surveys show strong openness among patients and physicians for a one-time gene editing therapy, especially among younger and genetically predisposed groups.

• About a third of patients and 40% of cardiologists (for HeFH) prefer a one-time therapy over chronic pills or injections.

• Manufacturing costs are expected to be low, enabling flexible pricing and broad market access, distinct from rare disease gene therapies.

• Oral PCSK9 inhibitors add to chronic care options but are not expected to significantly shift preference away from one-time therapies.