Verve Therapeutics (VERV) investor relations material

Key data and clinical progress

• HEART-2 trial of VRRB102 showed up to 60% LDL reduction at highest dose, with strong safety and tolerability across 14 patients.

• Durability data from earlier product (VRRB101) demonstrated sustained 60% LDL lowering two years post-infusion, supporting the one-time therapy vision.

• VRRB102 uses a new lipid nanoparticle delivery system with GalNAc ligand, improving safety and targeting compared to VRRB101.

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

• Full dose escalation and durability data will be presented in the second half of the year at a major medical meeting.

Differentiation, safety, and regulatory outlook

• VRRB102's delivery system and targeting reduce lab abnormalities seen with earlier versions and competitors.

• No read-through expected from recent Intellia liver safety events due to different timing and mechanisms of ALT elevation.

• Long-term safety is supported by minimal off-target edits and planned 15-year follow-up for all dosed patients.

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

• Two-year durability is seen as a strong indicator for long-term efficacy and safety, with payers and agencies likely to credit five years or more if early data remain flat.

Market, patient, and partnership insights

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

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

• Oral PCSK9 options add to chronic care but are not expected to diminish demand for one-time therapies due to persistent unmet need.

• Eli Lilly has an opt-in decision for VRRB102 in the second half of the year, potentially sharing US profits and development costs.

• Verve's pipeline targets three main cholesterol carriers (PCSK9, ANGPTL3, Lp(a)), with ANGPTL3 in clinic and Lp(a) partnered with Lilly; all leverage the same delivery platform.