Vor Biopharma Inc. · NASDAQ:VOR
Vor Biopharma — American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting 2026
13 slides from Vor Biopharma's conference presentation, Oct 2, 2026
Slide 1: A Phase 3 Study of Telitacicept in Generalized Myasthenia Gravis: UPSTREAM MG Trial Design Update
A Phase 3 Study to Evaluate the Efficacy and Safety of Telitacicept in Patients With Generalized Myasthenia Gravis: UPSTREAM MG Trial Design Update Ali A. Habib,1 Mamatha Pasnoor,2 Tuan Vu,3 Bhupendra O. Khatri,4 Gil I. Wolfe,5 Amit Sachdev,6 Said Beydoun,7 George G. Li,8 Shaida Khan,9 Lawrence Mein
Slide 2: Disclosures
Disclosures • Ali A. Habib has received research support/honoraria from Alexion/AstraZeneca, Amgen, Arcellx, argenx, Cabaletta Bio, Cartesian, COUR Pharmaceuticals, GC Biopharma, Grifols, Immunovant, Janssen/Johnson & Johnson, Kyverna, Merck, MGNet (grant number: U54NS115054), Nkarta, NMD Pharma, No
Slide 3: Telitacicept Is a Fusion Protein Based on the Human TACI Receptor Designed to Target BAFF and APRIL
Telitacicept Is a Fusion Protein Based on the Human TACI Receptor Designed to Target BAFF and APRIL Mimics wild-type TACI, a natural binding receptor for Extracellular BAFF and APRIL with near-full preservation of TACI receptor endogenous sequence for stable BAFF/APRIL binding1,2 • Modified to avoid
Slide 4: Proposed Bifunctional Effect of Telitacicept: Modulates B-Cell Development and Autoantibody Production
Proposed Bifunctional Effect of Telitacicept: Modulates B-Cell Development and Autoantibody Production ONE MECHANISM, TWO EFFECTS BAFF Inhibition APRIL Inhibition Telitacicept may limit autoreactive B-cell Telitacicept may inhibit the activation and further development of differentiation and surviva
Slide 5: Telitacicept Helps Disrupt B-Cell–Mediated Autoantibody Production That Attacks the Neuromuscular Junction
Telitacicept Helps Disrupt B-Cell–Mediated Autoantibody Production That Attacks the Neuromuscular Junction in Myasthenia Gravis1-13 Autoreactive Autoreactive plasma cells plasmablasts BAFF BAFF Nerve terminal APRIL APRIL Anti-AChR Anti-MuSK antibodies antibodiesa Blockade of ACh ACh–AChR binding LRP
Slide 6: Telitacicept Helps Disrupt B-Cell–Mediated Autoantibody Production That Attacks the Neuromuscular Junction
Telitacicept Helps Disrupt B-Cell–Mediated Autoantibody Production That Attacks the Neuromuscular Junction in Myasthenia Gravis1-13 Autoreactive Autoreactive Telitacicept plasma cells plasmablasts Telitacicept BAFF BAFF Nerve terminal APRIL APRIL Anti-AChR Anti-MuSK antibodies antibodiesa Telitacice
Slide 7: Telitacicept Has Demonstrated Efficacy and Safety in a Phase 3 Randomized Double-Blind Study in China
Telitacicept Has Demonstrated Efficacy and Safety in a Phase 3, Randomized, Double-Blind Control Study in China • In a multicenter, phase 3 trial of Changes in MG-ADL Score From Baseline to Week 24a telitacicept in patients with gMG 1 conducted in China1,2 0 Least squares meanb change – Primary endp
Slide 8: UPSTREAM MG: A Phase 3, Randomized, Placebo-Controlled Study With an OLE Period
UPSTREAM MG: A Phase 3, Randomized, Placebo-Controlled Study With an OLE Period to Evaluate the Efficacy and Safety of Telitacicept in Patients With gMG • UPSTREAM MG consists of: Study Design: UPSTREAM MG – ≤4 weeks screening period Treatment period Primary and secondary OLE period OLE extension –
Slide 9: Primary and Secondary Endpoints
Primary and Secondary Endpoints • Primary endpoint – Change from baseline in MG-ADL score at week 24 • Secondary endpoints include: – Change from baseline in QMG score at week 24 – Change from baseline in MG-QOL15r at week 24 – Proportion of patients with a ≥2-point decrease in MG-ADL score at week
Slide 10: Participation Criteria for the Telitacicept gMG Global Phase 3 Clinical Trial
Participation Criteria for the Telitacicept gMG Global Phase 3 Clinical Trial Key Inclusion Criteria MG-ADL score ≥6 points at Positive antibodies Male or female aged ≥18 18+ years at time of screening screening and baseline with ocular-related score <50% of against AChR or MuSK at screening the tot
Slide 11: Participation Criteria for the Telitacicept gMG Global Phase 3 Clinical Trial (cont'd)
Participation Criteria for the Telitacicept gMG Global Phase 3 Clinical Trial (cont’d) Exclusion Criteria Diagnosed with any other Thymoma within 5 years or autoimmune disease, which Acute or chronic infection received thymectomy ≤6 can pose a safety or efficacy months prior to screeninga confoundin
Slide 12: Study Locations and Number of Sites for the Global Phase 3 Study
Study Locations and Number of Sites for the Global Phase 3 Study China Canada Poland Belgium France Georgia United States Spain Japan Italy Bulgaria Czechia Brazil Australia Argentina Study enrollment is completed across 15 countries, with top-line results anticipated in H1 2027 . As of September 8,
Slide 13: Conclusions
Conclusions • A global, phase 3, pivotal, multicenter, randomized, double-blind, placebo-controlled trial with a long- term OLE period is ongoing to evaluate the efficacy and safety of telitacicept in a global patient population1 • Results from this study will augment the established efficacy and sa