Vor Biopharma (VOR) investor relations material

Strategic ambition and platform

• Aims to transform treatment of B cell-driven autoimmune diseases through selective BAFF/APRIL inhibition, targeting both upstream B cell survival and downstream antibody production.

• Telitacicept is a dual BAFF/APRIL inhibitor with clinical validation in over eight autoimmune indications in China and a favorable safety profile in tens of thousands of patients.

• Strong cash position of $450M, providing runway into mid-2028 and funding all key clinical milestones.

Myasthenia gravis (MG) program

• MG represents a large and growing global opportunity, with ~260,000 diagnosed patients across key markets and a projected $10.8B market size by 2030.

• Current therapies mainly target symptoms and IgG, leaving unmet needs for disease-modifying treatments that address IgA and IgM autoantibodies.

• Telitacicept demonstrated the largest placebo-adjusted improvement in MG-ADL among leading mechanisms, with sustained and deepening benefit over 48 weeks.

• Phase 3 trial in China met primary and secondary endpoints, showing significant improvements in MG-ADL and QMG scores, with durable efficacy and consistent reduction in IgG, IgA, IgM, and B cells.

• Favorable safety profile observed, with no new safety signals and manageable adverse events.

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

Sjögren's disease (SD) program

• SD is a large, underserved autoimmune disease with ~870,000 diagnosed patients across key markets and no approved disease-modifying systemic therapies.

• Telitacicept showed statistically significant and clinically meaningful improvements in ESSDAI and ESSPRI scores, with robust, dose-dependent, and durable effects across systemic activity and symptoms.

• Nearly 90% of patients reported improvement, and 3 in 4 achieved disease control as confirmed by physicians.

• Consistent reduction in IgG, IgA, IgM, and B cells observed, supporting the mechanism of action.

• Favorable safety profile consistent with other indications, with no new safety signals.

• Global Phase 3 trial initiated in February 2026, with primary endpoint at 48 weeks.