Vor Biopharma (VOR) investor relations material

Strategic focus and pipeline progress

• Main asset Telitacicept, a BAFF-APRIL inhibitor, targets B-cell-mediated autoimmune diseases and was in-licensed in June 2025.

• Telitacicept is approved in six indications in China, with Western development focused on myasthenia gravis (MG) and Sjögren's disease.

• Completed enrollment of phase III MG trial, with plans to present key data at AANEM and initiate ocular MG phase III in H1 2027.

• MG and ocular MG represent multi-billion dollar opportunities, with Sjögren's disease also seen as a potential $10B+ market.

• Early phase III enrollment in Sjögren's is exceeding expectations, reflecting strong execution and market interest.

Differentiation and mechanism of action

• Telitacicept's 2-to-1 BAFF to APRIL mechanism allows dosing flexibility and targets both upstream and downstream B-cell pathology.

• Unlike other BAFF-APRIL inhibitors, Telitacicept can modulate both autoreactive B cells and plasma cells, potentially offering broader efficacy.

• The drug's mechanism is positioned to address unmet needs in MG and Sjögren's by providing durable and deep clinical responses.

Clinical development and regulatory strategy

• MG phase III trial was optimized after acquisition, leveraging KOL engagement and operational improvements to accelerate enrollment.

• Top-line MG data expected in H1 2027, with focus on MG-ADL delta, breadth of response, and durability as key endpoints.

• Safety profile consistent with prior data: minor infections slightly increased, but no rise in serious or opportunistic infections.

• Regulatory engagement ongoing with FDA and EMA, with BLA preparation for MG as a top priority.