Vor Biopharma (VOR) TD Cowen 46th Annual Health Care Conference summary

Key presentations and strategic updates

• Highlighted telitacicept as a differentiated BAFF/APRIL inhibitor with strong phase III data in myasthenia gravis (MG) and Sjögren's disease, targeting both upstream and downstream B-cell pathways.

• Emphasized a robust safety profile, with tens of thousands of patients treated commercially in China and no deep B-cell depletion or severe immune suppression.

• Company re-centered its strategy in mid-2025 around telitacicept, assembling a new expert team and focusing on late-stage trials and launch preparation.

• Maintains a strong cash position of $450 million, providing runway through mid-2028 and funding for global phase III programs.

Clinical development and trial progress

• MG phase III global trial is enrolling well, with top-line data expected in the first half of 2027; protocol improvements include longer open-label extension.

• China phase III MG data showed a -4.8 placebo-adjusted MG-ADL improvement, nearly double other agents, with continued benefit over 48 weeks.

• No need for drug holidays with telitacicept, reducing risk of disease flare and supporting chronic treatment.

• Sjögren's phase III global trial to dose first patient by end of first half, enrolling 250 patients with 48-week treatment and extension period.

• Phase III in Sjögren's powered based on robust China data; company confident one global study will suffice for approval.

Market opportunity and competitive positioning

• MG market forecasted to exceed $10 billion in U.S. sales by decade's end, with significant unmet need for chronic, safe therapies.

• Sjögren's represents a large, underserved market with over 100,000 addressable U.S. patients and no approved modern biologics.

• Telitacicept demonstrated best-in-disease efficacy in both MG and Sjögren's, with clinically meaningful and statistically significant improvements.

• Differentiation from competitors includes chronic dosing without immune suppression, strong safety, and broad applicability across B-cell-mediated autoimmune diseases.

• Both MG and Sjögren's are viewed as multi-billion dollar opportunities, with potential for further expansion into additional indications.