Vor Biopharma
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Vor Biopharma (VOR) investor relations material

Vor Biopharma 44th Annual J.P. Morgan Healthcare Conference summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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44th Annual J.P. Morgan Healthcare Conference summary13 Jan, 2026

Strategic transformation and vision

  • Pivoting to an autoimmune-focused company, leveraging telitacicept as a lead asset, with a shift completed by mid-2025.

  • Aims to transform treatment of B cell-driven autoimmune diseases with a selective BAFF/APRIL inhibitor, telitacicept.

  • Focus on global expansion, targeting large, underserved patient populations in myasthenia gravis and Sjögren's disease.

  • Company is well-capitalized with $450 million, supporting operations and milestones through mid-2028.

  • Assembled a leadership team with deep expertise in autoimmune disease, commercial strategy, and clinical development.

Clinical development and milestones

  • Global Phase 3 trial for myasthenia gravis underway, with topline data expected in 1H27.

  • Global Phase 3 trial for Sjögren's disease to initiate in 1H26.

  • Telitacicept has demonstrated efficacy and safety in eight autoimmune diseases, with commercial approvals in China for lupus, RA, and MG.

  • MG Phase 3 data from China show significant and durable symptom improvement, with nearly symptom remission at week 48.

  • Expansion opportunities identified in additional B cell-mediated autoimmune diseases.

Mechanism of action and clinical efficacy

  • Telitacicept is a TACI-Fc fusion protein that inhibits both BAFF and APRIL, targeting autoreactive B cells and plasma cells.

  • Demonstrates disease modification by reducing pathogenic B cells and autoantibody production.

  • Clinical trials show largest placebo-adjusted improvement in MG-ADL for myasthenia gravis and robust, durable ESSDAI reduction in Sjögren's disease.

  • Over 10,000 patients treated in China, with robust safety and efficacy data across multiple autoimmune indications.

  • Telitacicept positioned as best- and first-in-class BAFF/APRIL inhibitor with potential for broad commercial success.

Mitigate global variability in China data?
Key protocol changes for Sjögren's trial?
Prioritize future indication selection?
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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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