Verve Therapeutics (VERV) Goldman Sachs 45th Annual Global Healthcare Conference summary

Strategic vision and market opportunity

• Focus on one-time gene-editing therapies for cardiovascular disease, aiming for permanent LDL cholesterol reduction and functional cures.

• Large unmet need exists as current treatments have low patient adherence and limited long-term effectiveness.

• Market potential is significant, with current PCSK9 therapies representing only 2% of eligible patients and $3.6B in annualized sales.

• Cost of goods is expected to be manageable due to similarities with scalable mRNA vaccine technology.

• Patient and physician interest is high, with a third of surveyed patients preferring a one-time gene-editing therapy.

Clinical progress and product pipeline

• First-in-human data for VERVE-101 showed up to 70% LDL reduction and durable effects out to 9+ months, but transient safety signals led to a study pause.

• VERVE-102, a second-generation product with a new lipid nanoparticle and GalNAc targeting, aims to mitigate prior safety issues; Heart-2 trial has begun.

• Preclinical and third-party clinical data support improved safety and efficacy profile for VERVE-102.

• Heart-2 trial includes both HeFH and premature coronary artery disease patients, with first data expected in 2025.

• Strategy remains to select the best candidate for phase 2 in 2025, with mitigation strategies for VERVE-101 under evaluation.

Technology and delivery platform

• Delivery system is a key differentiator; VERVE-102 uses a proprietary GalNAc LNP with components licensed from Novartis and commercial suppliers.

• Modular platform allows rapid switching of gene targets once delivery is de-risked, supporting a robust pipeline.

• Base editing is the primary technology, but the platform is flexible to incorporate new editing and delivery methods.