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Verve Therapeutics (VERV) investor relations material

Verve Therapeutics Goldman Sachs 46th Annual Global Healthcare Conference summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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Goldman Sachs 46th Annual Global Healthcare Conference summary9 Jun, 2025

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.

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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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