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

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.