Vor Biopharma (VOR) Citigroup’s Biopharma Back to School Summit 2026 summary

Strategic focus and pipeline progress

• Main asset, Telitacicept, is a BAFF-APRIL inhibitor targeting B-cell-mediated autoimmune diseases, in-licensed in June 2025, with approvals in six indications in China and ongoing development in the West for myasthenia gravis (MG) and Sjögren's disease.

• Completed enrollment of phase III MG trial, with top-line data expected in the first half of 2027; ocular MG phase III planned for first half of 2027, leveraging operational momentum and KOL engagement.

• Sjögren's phase III trial began in March, with strong early enrollment and positive KOL feedback, aiming to address a large unmet need in a market potentially exceeding $10 billion.

• Additional indications are under consideration, with prioritization based on scientific rationale, commercial relevance, and competitive landscape; further expansion depends on MG trial outcomes.

Differentiation and clinical rationale

• Telitacicept offers a unique 2-to-1 BAFF to APRIL mechanism, enabling effective upstream B-cell modulation and potential for deeper, more durable clinical efficacy compared to FcRn and CD19-targeted therapies.

• In MG, the focus is on achieving minimal symptom expression and demonstrating both breadth and durability of response, with data to be presented at AANEM and further updates expected at 24 weeks post last patient dosed.

• For Sjögren's, Telitacicept targets both antibody-dependent and antibody-independent B-cell pathology, aiming to overcome disease heterogeneity and improve on challenging outcome measures like ESSDAI and ESSPRI.

Market opportunity and commercial strategy

• MG market in the U.S. is projected to grow from $3 billion to over $10 billion by decade's end, with significant unmet need for durable therapies; ocular MG represents a $1 billion opportunity and may prevent progression to generalized MG.

• Early and proactive engagement with KOLs and investigators has driven strong trial enrollment and positive market anticipation, with a dedicated medical affairs team in place to support future launches.

• Regulatory and operational readiness is emphasized, with ongoing interactions with FDA and EMA, and a focus on data quality and BLA preparation for MG.