Vor Biopharma (VOR) investor relations material

Key clinical and mechanistic insights

• Telitacicept is a dual BAFF/APRIL inhibitor, targeting both upstream and downstream B-cell pathways, offering rapid and durable efficacy in myasthenia gravis (MG).

• The mechanism provides broader immunoglobulin coverage (IgG, IgA, IgM), potentially addressing non-responders to FcRn therapies and positioning the drug for strong second-line use.

• Sustained minimal symptom expression (MSE) is a focus, with upcoming data to show both attainment and durability of remission.

• Safety profile is well characterized, with over 3,000 patients in trials and tens of thousands treated in China.

• Reformulation efforts are underway to move from two weekly injections to a single prefilled syringe and auto-injector for improved patient convenience.

Market positioning and commercial strategy

• The MG market is rapidly expanding, with only about 20% of patients currently treated with biologics, leaving significant growth potential.

• First-mover advantage is emphasized, with a three-year lead over competitors and a strong safety database.

• The dual mechanism and broad immunoglobulin targeting differentiate telitacicept from other therapies, supporting potential best-in-disease data.

• Ocular MG is a key opportunity, with plans to be third to market and first with an upstream targeted mechanism.

• Lifecycle planning includes rapid transition to more convenient formulations and targeting both biologic-naive and treated segments.

Clinical development and future outlook

• Global phase III study in MG recently completed, with most patients enrolled under current leadership, ensuring consistency and optimal population.

• Differences between China and global trials include age, disease duration, and cultural reporting tendencies, but physician-administered outcomes are expected to be similar.

• Anticipated data readouts in the next six to nine months, including results on sustained MSE and ocular MG.

• Ongoing phase III in Sjögren's syndrome, with study design adjusted to account for higher placebo responses seen in global trials.

• BAFF/APRIL mechanism is believed to address the breadth of B-cell-driven pathology in Sjögren's, potentially offering superior efficacy.