Verve Therapeutics (VERV) Canaccord Genuity 44th Annual Growth Conference & Private Company Showcase 2024 summary

Strategic vision and disease focus

• Mission centers on addressing atherosclerotic cardiovascular disease by providing durable, lifelong LDL cholesterol reduction through one-time gene editing therapies.

• Current therapies require lifelong adherence, but up to 50% of patients discontinue within a year, highlighting unmet need.

• Pipeline targets genetically validated mechanisms, starting with high-risk genetic populations and expanding to broader groups.

Pipeline and technology development

• Lead programs target PCSK9 using base editing, with VERVE-102 in clinical trials and VERVE-101 as predecessor.

• VERVE-102 uses a novel GalNAc-LNP delivery system, expected to improve safety over VERVE-101.

• VERVE-101 showed 46% mean LDL reduction and durable effect, but had transient safety issues attributed to its LNP.

• VERVE-102 is enrolling in the Heart-2 trial, with interim phase I data expected in H1 2025 and phase II to start in H2 2025.

Clinical progress and safety

• Heart-1 trial demonstrated durable LDL lowering out to 9 months, with ongoing follow-up and potential for further updates.

• Preclinical and clinical data support that new LNP in VERVE-102 is safer, with acute safety issues expected to be detectable within days post-infusion.

• Heart-2 trial uses a single-ascending dose design to identify a safe, effective dose, with a focus on both efficacy and acute safety.