Vor Biopharma (VOR) investor relations material

Strategic asset acquisition and pipeline focus

• In-licensed telitacicept, a BAFF/APRIL inhibitor, for global rights and all indications, targeting autoimmune diseases with initial focus on MG and Sjögren's.

• Asset supported by extensive late-stage data from China, including phase 3 trials in multiple indications and thousands of patients.

• Rapidly assembled capital and team to execute on the program, aiming to generate significant midterm value.

Clinical data insights and competitive positioning

• MG data from China showed a strong efficacy signal with a large delta versus placebo, attributed partly to low placebo rates in the Chinese population.

• Expectation that global trials may see higher placebo rates, but confident in maintaining a competitive efficacy delta.

• MG market projected to reach $10 billion by 2030, with current therapies showing limitations and demand for more durable, disease-modifying treatments.

• BAFF/APRIL inhibition offers broader efficacy by targeting both antibody-producing cells and upstream B-cell activation, potentially addressing non-responders to FcRn therapies.

• 48-week data in MG showed continued improvement beyond 24 weeks, suggesting durable and possibly disease-modifying effects.

Sjögren's opportunity and trial design

• Phase 3 data in Sjögren's from China demonstrated strong results, providing proof of concept and a foundation for global studies.

• Competitor trials faced high placebo responses due to site variability and SDI score inflation; plans to mitigate this with investigator training and central adjudication.

• Global phase 3 for Sjögren's to start next year, leveraging learnings from competitor experiences and aiming for a best-in-disease profile.

• Market for Sjögren's considered large and underserved, with potential for significant impact.