Vor Biopharma (VOR) investor relations material

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.