Vor Biopharma Inc. · NASDAQ:VOR
Vor Biopharma — American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) 2025
14 slides from Vor Biopharma's conference presentation, Oct 22, 2025
Slide 1: Efficacy and Safety of Telitacicept in Patients with Generalized Myasthenia Gravis: Results from a Phase 3 Study
2025 Efficacy and Safety of Telitacicept in Patients with Generalized Myasthenia Gravis: Results from a Phase 3 Study Presenter: Dr. George Li gli@medsolcrc.com
Slide 2: Financial Disclosure
2025 Financial Disclosure Dr. George Li serves as a scientific consultant for RemeGen Biosciences. Dr. Jian Yin is a consultant in a global scientific advisor committee for Alexion. Dr. Wenxiang Wang is an employee of RemeGen Co., Ltd, and owns company stock. Dr. Lin Li is an employee of RemeGen Co.
Slide 3: Introduction of Generalized Myasthenia Gravis (g)MG
2025 Introduction of Generalized Myasthenia Gravis (g)MG • MG is a rare autoimmune disease affecting about 700,000 people worldwide. In an autoimmune disease, the body’s immune system mistakenly attacks its own healthy tissues. The current therapy for MG focuses on treating symptoms caused by the di
Slide 4: Telitacicept Mechanism of Action
4 2025 Telitacicept Mechanism of Action Dual BAFF/APRIL blockade stops B cell survival and plasma cell antibody production Telitacicept TACI-Fc fusion protein: Blocking BAFF inhibits abnormal dual inhibition of BAFF/APRIL Blocking APRIL inhibits abnormal development and maturation of B cells product
Slide 5: Study Design
2025 Study Design • A Phase 3, multiple center, randomized, double-blinded, placebo-controlled study • Patient population: Positive AChR-Ab or MuSK-Ab; MGFA class II, III, or IVa; MG-ADL score ≥ 6; QMG score ≥ 8; and maintenance of a stable standard treatment regimen. Primary Endpoint: Change from B
Slide 6: Primary and Secondary Endpoints
2025 Primary and Secondary Endpoints Primary Endpoint: Change from baseline in MG-ADL score at Week 24 Secondary Endpoints: Change from baseline in MG-ADL score at Week 12, 36, and 48 Change from baseline in QMG score at Week 12, 24, 36, and 48 Proportion of patients achieved MG-ADL score reduction
Slide 7: Study Results: Patient Disposition and Baseline Characteristics
2025 Study Results: Patient Disposition and Baseline Characteristics Telitacicept Placebo (N=57) (N=57) Age (yr), mean ± SD 49.1 ± 14.69 49.6 ± 15.03 Sex Male, n (%) 30 (52.6) 21 (36.8) Female, n (%) 27 (47.4) 36 (63.2) Disease duration (month), mean± SD 83.09 ± 84.507 76.05 ± 87.817 MGFA classifica
Slide 8: Sustained MG-ADL Reduction Over 48 weeks
2025 Sustained MG-ADL Reduction Over 48 weeks 98.1% 96.2% 90.2%* -1.6 -6.3* -6.4 12.0% -7.5 Time (wk) Time (wk) MG-ADL Score Change from Baseline Percentage of Patients with MG-ADL Score ≥3 Points Reduction * Telitacicept arm continue with the same treatment, Placebo arm switched to Telitacicept dur
Slide 9: Sustained QMG Reduction Over 48 weeks
2025 Sustained QMG Reduction Over 48 weeks -1.9 87.0% 94.2% 90.2%* 16.0% -7.8 -9.3* -9.8 Time (wk) Time (wk) QMG Score Change from Baseline Percentage of Patients with QMG Score ≥5 Points Reduction * Telitacicept arm continue with the same treatment, Placebo arm switched to Telitacicept during OLE p
Slide 10: Changes in IgG, IgA, IgM, CD19+ B count
2025 Changes in IgG、 IgA、 IgM、 CD19+ B count 0.00% 0.69% -59.57% -58.10%* -32.89% -30.72%* -60.60% -35.81% Time (wk) Time (wk) -2.61% -9.09% -24.37% -40.14% Time (wk) Time (wk) 10
Slide 11: Incidence of Adverse Event (SS)
2025 Incidence of Adverse Event (SS) Open-label treatment period (Weeks 24-48) Weeks 0-48 Telitacicept 240 mg Placebo → Telitacicept 240 mg Telitacicept 240 mg Telitacicept at least one (N=54) (N=53) (N=57) dose* (N=110) TEAE n (%) 39 (72.2) 45 (84.9) 51 (89.5) 96 (87.3) ADR n (%) 25 (46.3) 36 (67.9
Slide 12: Incidence of Infections (SS)
2025 Incidence of Infections (SS) Open-label Treatment Period Weeks 0-48 SOC Telitacicept 240 mg Placebo Telitacicept 240 mg Telitacicept at Least One Dose Number of Number of Number of Number of PT Number of Participants Number of Participants Number of Participants Number of Participants Events Ev
Slide 13: Conclusion
2025 Conclusion • Telitacicept showed sustained efficacy for up to 48 weeks and was well tolerated in gMG patients in this phase 3 trial from China. • A global phase 3 pivotal, multiple center, randomized, double-blind, placebo control trial with long term open label extension study is ongoing to ev
Slide 14: Acknowledgement
2025 Acknowledgement • Thanks to the patients who participated in the study. • Thanks to all the investigators and their site staffs for their contribution to the study.