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Verve Therapeutics
Verve Therapeutics (VERV) investor relations material
Verve Therapeutics has been acquired by Eli Lilly and Company
Verve Therapeutics Goldman Sachs 46th Annual Global Healthcare Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Goldman Sachs 46th Annual Global Healthcare Conference summary9 Jun, 2025
Strategic vision and clinical progress
Focus on one-time gene editing therapy for lifelong cholesterol reduction to address atherosclerotic cardiovascular disease, aiming for enduring efficacy where current therapies fall short.
HEART-2 trial of 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 lowering for two years post-infusion, supporting the one-dose future vision.
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 transition to fixed dosing planned for phase II later in the year.
Safety, durability, and regulatory outlook
No major safety issues observed with VERVE-102; no ALT or platelet elevations, and only one mild infusion reaction among 14 patients.
Durability of effect is supported by both preclinical and human data, with edits persisting beyond typical liver cell turnover, and potential for redosing if needed.
Long-term follow-up (15 years) is planned for all patients, with four to five years of data expected at registration.
Regulatory agencies are increasingly supportive of in vivo gene editing, with recent high-profile cases and policy statements highlighting openness to transformative therapies.
Market research, patient and physician perspectives
Surveys show strong openness among patients and cardiologists for a one-time gene editing therapy, especially among younger patients and those with genetic high cholesterol.
About a third of patients and 40% of cardiologists (for HeFH) prefer a one-time gene edit over daily pills or injections; 20% preference among ASCVD patients.
Payers may be more flexible on pricing due to large patient populations and low manufacturing costs, with potential for significant margin flexibility.
How does Verve's cost structure enable market access?
What is the rationale for fixed dosing in Phase II?
Compare Lilly's PCSK9 and LPA partnership models.
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Verve Therapeutics
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Frequently asked questions
Verve Therapeutics Inc., founded in 2018, is a genetic medicines company focused on developing gene editing treatments for cardiovascular diseases. The company's flagship product, VERVE-101, is a single-course gene editing treatment designed to permanently inactivate the PCSK9 gene in the liver, with an aim to address cardiovascular conditions effectively. The company is headquartered in Cambridge, Massachusetts, and its shares are listed on the NASDAQ.
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