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

Strategic transformation, vision, and pipeline

• Pivoting to focus on autoimmune B-cell mediated diseases, with a major shift planned for mid-2025 and a rebranding as "Vor 2.0."

• Aims to transform treatment of B cell-driven autoimmune diseases with telitacicept, a selective BAFF/APRIL inhibitor, in-licensed from RemeGen and validated in over eight autoimmune diseases, treating more than 10,000 patients in China.

• Focus on global expansion, targeting large, underserved patient populations in myasthenia gravis (MG) and Sjögren's disease, both with blockbuster potential and high unmet medical need.

• Plans to leverage broad applicability across B cell-mediated autoimmune diseases, with next wave of indications under consideration.

• Strong financial position with $450 million in cash, supporting operations and milestones through mid-2028.

Clinical development, milestones, and data highlights

• Global Phase 3 trial for MG underway, with topline data expected in 1H27; global Phase 3 for Sjögren's to initiate in 1H26.

• Telitacicept already approved in China for SLE, RA, and MG, with BLA submissions for Sjögren's and IgA nephritis.

• MG Phase 3 trial in China demonstrated significant and durable symptom improvement; Sjögren's Phase 3 data in China showed robust efficacy in "pure" patients with sustained benefits and a best-in-disease profile.

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

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

Mechanism of action, efficacy, and safety

• Telitacicept is a TACI-Fc fusion protein that inhibits both BAFF and APRIL, targeting autoreactive B cells and plasma cells, remodulating the immune system without broad B-cell depletion.

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

• Favorable and predictable safety profile observed in tens of thousands of patients, with no burdensome vaccination requirements or signature B cell depletion-related serious adverse events; most adverse events are mild to moderate.