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Vor Biopharma (VOR) investor relations material

Vor Biopharma Stifel Virtual Immunology and Inflammation Forum summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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Stifel Virtual Immunology and Inflammation Forum summary15 Sep, 2025

Strategic repositioning and asset acquisition

  • Company restructured as 'Vor 2.0' around telitacicept, a late-stage immunology asset licensed from RemeGen with global rights ex-China.

  • Telitacicept is a dual BAFF/APRIL inhibitor, targeting both upstream and downstream B-cell pathways for durable efficacy and disease modification.

  • Over 70,000 patients treated in China, with 3,000 in clinical trials and proven efficacy in three indications; two more PLAs being filed.

  • Focused on large, underserved indications such as myasthenia gravis and Sjögren’s disease.

  • Assembled a world-class leadership team to execute on these opportunities.

Differentiation and clinical data

  • Telitacicept closely mimics the wild type TACI receptor, providing optimal inhibition of BAFF and APRIL and balanced, safe therapy.

  • Demonstrates consistent efficacy across immunoglobulin types (IgG, IgA, IgM) and normalizes B cells, addressing disease root causes.

  • Offers potential advantages over FCRN antagonists and complement inhibitors by targeting broader disease mechanisms.

Myasthenia gravis program

  • Myasthenia gravis is a key focus, with 90,000 U.S. patients and high unmet need for durable, disease-modifying treatments.

  • Phase III China trial showed unprecedented, durable activity on MGADL endpoint, with benefits enduring through 24 and 48 weeks.

  • Long-term data to be presented at AANEM, highlighting continued improvement and disease trajectory modification.

  • Global phase III trial is enrolling, leveraging seamless transition from RemeGen and high-quality China data.

  • Study design accounts for potential higher placebo response in global settings, aiming to replicate best-in-disease results.

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Frequently asked questions

Vor Biopharma Inc. is a clinical-stage biotechnology company focused on developing innovative treatments for blood cancers. The company's approach combines cell and genome engineering to create therapies that target and treat hematological malignancies. Vor's proprietary platform primarily involves engineering hematopoietic stem cells (HSCs) to enhance their compatibility with targeted therapies. The lead product candidate, an engineered HSC known as tremtelectogene empogeditemcel (trem-cel), is currently in clinical trials for the treatment of acute myeloid leukemia (AML). Vor is also working on additional therapies that involve chimeric antigen receptor T-cells (CAR-T) and antibody-drug conjugates to further expand its cancer treatment portfolio. The company is headquartered in Cambridge, Massachusetts, and its shares are listed on the Nasdaq.

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