Verve Therapeutics (VERV) investor relations material

Strategic vision and product development

• Focus on one-time gene editing therapy for lifelong cholesterol reduction to address enduring efficacy in cardiovascular disease treatment.

• VERVE-102, a next-generation compound, showed up to 69% LDL reduction with a mean of 59% at the highest dose, with strong safety and tolerability in the HEART-2 trial.

• Durability of effect demonstrated, with LDL reduction sustained at 60% two years post-infusion.

• VERVE-102 features a new lipid nanoparticle delivery system and a GalNAc targeting ligand for improved safety and liver specificity.

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

Clinical data and safety profile

• HEART-2 trial data from 14 patients showed robust LDL lowering and a favorable safety profile, with minimal infusion reactions and no significant lab abnormalities.

• No read-through safety concerns from similar products, as late ALT elevations seen in other programs are not observed with VERVE-102.

• Durability data for VERVE-102 will be expanded in the second half of the year, with follow-up ranging from 3 to over 12 months.

• Long-term safety is being monitored, with regulatory-mandated 15-year follow-up for all patients.

• Off-target editing risk is minimized, and benefit-risk is considered favorable for high-risk cardiovascular patients.

Regulatory and market landscape

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

• Market research shows strong openness among patients and physicians, especially younger patients and those with genetic high cholesterol.

• About a third of surveyed patients and 40% of cardiologists for HeFH patients prefer a one-time gene editing therapy over chronic options.

• Pricing flexibility is anticipated due to manageable manufacturing costs and a large patient population, differentiating from rare disease gene therapies.

• Oral PCSK9 inhibitors are not expected to significantly shift preference away from one-time therapies for key patient segments.