Slide 1: 48-week Phase 3 Clinical Trial Data from China for Telitacicept in Primary Sjögren's Disease
Global Science. One Purpose. 48-week Phase 3 Clinical Trial Data from China for Telitacicept in Primary Sjögren’s Disease October 28, 2025
Slide 2: Forward Looking Statement
Forward Looking Statement This presentation (the “Presentation”) contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 about Vor Biopharma Inc. (“Vor,” “Vor Bio” or the “Company”). The words “aim,” “anticipate,” “believe,” “can,” “could,” “des
Slide 3: Agenda
Agenda Opening Remarks & Data Highlights Jean-Paul Kress, M.D., Chairman & Chief Executive Officer Sjögren’s Disease – A Major Unmet Need Ronald van Vollenhoven, M.D., Ph.D., Professor of Rheumatology at Amsterdam University Medical Center 1. 2. Primary Sjögren’s Disease China Phase 3 Results Qing Z
Slide 4: Redefining Durability in gMG with Telitacicept
AANEM | Redefining Durability in gMG with Telitacicept gMG, generalized myasthenia gravis; *MGFA Scientific Session/AANEM 2025 Abstract; †Not placebo adjusted 48-week OLE results oral presentation on October 29, 2025 at 10:50AM PT MG-ADL Change From Baseline† -5.74 Week 24 -7.5 Week 48* -8.66 Week 2
Slide 5: A True Signal, No Noise: Potential to Redefine Treatment in pSD
A True Signal, No Noise No DMARDs, no steroids Clinically Meaningful, Statistically Clear Robust, dose-dependent improvements across physician- and patient-assessed outcomes Depth and Durability Across Domains Improvement of systemic activity, symptoms, and function Consistent Safety Profile No new
Slide 6: Early, Broad Symptom Improvements Observed with Telitacicept
Early, Broad Symptom Improvements Observed with Telitacicept Nearly 90% of patients report improvement as physicians confirm disease control in 3 out of 4 patients Placebo*: Participants randomized to the placebo group. ⁜The analysis of change from baseline in ESSDAI and ESSPRI score over Weeks 0-24
Slide 7: Potential Disease Modification Through Upstream and Downstream Control
Potential Disease Modification Through Upstream and Downstream Control BAFF, B-cell activating factor; BLyS, B Lymphocyte Stimulator; APRIL, a proliferation inducing ligand Dual BAFF/APRIL blockade is designed to stop B cell survival and plasma cell antibody production Regulated by APRIL Regulated b
Slide 8: Harnessing Native TACI Biology for Optimal Binding
Harnessing Native TACI Biology for Optimal Binding 1. DOI 10.1074/jbc.M411714200; 2. DOI 10.3389/fimmu.2018.02125; 3. WO2002094852A2; 4. Internal Telitaicept IP Preserving receptor integrity enhances binding strength and supports a favorable safety profile Natural Design Advantage Mimics full native
Slide 9: Telitacicept: Redefining What Success Looks Like In Autoimmune Disease
Telitacicept: Redefining What Success Looks Like In Autoimmune Disease 2021 - Systemic Lupus Erythematosus (SLE)† 2024 - Rheumatoid Arthritis (RA) 2025 – Generalized Myasthenia Gravis (gMG) †Conditional Approval, Full Approval in 2023; *Accelerated Approval in China Clinically meaningful results in
Slide 10: Primary Sjögren's Disease China Phase 3 Results
Primary Sjögren’s Disease China Phase 3 Results Qing Zuraw, M.D., M.P.H., M.B.A., Chief Development Officer
Slide 11: Phase 3 Trial in Primary Sjögren's Disease Completed in China
Phase 3 Trial in Primary Sjögren's Disease Completed in China Potential best-in-disease profile in China; randomized, double-blind, placebo-controlled study *Estimated. ESSDAI, EULAR Sjögren’s syndrome disease activity index; ESSPRI, EULAR Sjögren’s Syndrome Patient Reported Index; MFI, multidimensi
Slide 12: Patient Disposition
Patient Disposition Strong study execution across 79 sites in China *Participants randomized to the placebo group; †Participants who did not switch treatment throughout the trial; Teli, telitacicept RemeGen-sponsored trial Screening (n=480) Randomized (n=381) Received Treatment in Stage A (n=380) Pl
Slide 13: Baseline Characteristics
Baseline Characteristics Well-balanced, representative patient population across treatment arms SD: standard deviation; BMI: body mass index; Mon, months; ESSDAI, EULAR Sjögren’s syndrome disease activity index; ESSPRI, EULAR Sjögren’s Syndrome Patient Reported Index; MFI-20, multidimensional fatigu
Slide 14: Deep, Consistent ESSDAI Reduction Through Weeks
Deep, Consistent ESSDAI Reduction Through 48 Weeks 7x greater improvement means fewer active symptoms and broader systemic relief for patients Placebo*: Participants randomized to the placebo group. ⁜The analysis of change from baseline in ESSDAI score over Weeks 0-24 was based on the estimate popul
Slide 15: Deep, Consistent ClinESSDAI Reduction Through Weeks
Deep, Consistent ClinESSDAI Reduction Through 48 Weeks ESSDAI and ClinESSDAI remain nearly identical at week 48 -4.6 vs -4.5 ClinESSDAI excludes biological domain (IgG, complement), representing a more sensitive measure of pure clinical disease activity Sustained improvement in clinical benefit beyo
Slide 16: More Than Half of Patients Achieve Low Disease Activity
More Than Half of Patients Achieve Low Disease Activity Nearly 5x more patients on 160mg vs placebo achieved this threshold Low Disease Activity (ESSDAI <5) Represents Minimal Systemic Involvement 55% vs 12% Consistent improvement sustained through 48 weeks, indicating durable immune stabilization P
Slide 17: Sustained Improvement in ESSPRI Through Weeks
Sustained Improvement in ESSPRI Through 48 Weeks Reduction in patient-reported fatigue, pain, and dryness by ~2.6 points at one year Placebo*: Participants randomized to the placebo group. ⁜The analysis of change from baseline in ESSPRI score over Weeks 0-24 was based on the estimate population (EP)
Slide 18: Telitacicept Produced Clinically Meaningful Improvement in Fatigue
Telitacicept Produced Clinically Meaningful Improvement in Fatigue 12-point improvement over 48 weeks to a critical daily symptom for patients Placebo*: Participants randomized to the placebo group. ⁜The analysis of change from baseline in MFI-20 score over Weeks 0-24 was based on the estimate popul
Slide 19: BAFF/APRIL MOA Associated With Improvement Across All ESSPRI Domains
BAFF/APRIL MOA Associated With Improvement Across All ESSPRI Domains MOA, mechanism of action Meaningful improvement across dryness, fatigue, and pain – symptoms that define daily patient life
Slide 20: STAR: Exploratory Endpoint Demonstrates Multi-Domain Improvement
STAR: Exploratory Endpoint Demonstrates Multi-Domain Improvement Nearly 3 in 4 patients achieved ≥5 point response Placebo*: Participants randomized to the placebo group. A STAR responder was defined as a participant with a total STAR score of ≥5 points. Participants with missing scores in any domai
Slide 21: Consistent Reduction in IgG, IgA, IgM, and B Cells
Consistent Reduction in IgG, IgA, IgM, and B Cells IgG IgA IGM B cell RemeGen-sponsored trial -24.81% 2.08% -16.33% -45.81% -0.18% -32.48% -58.07% -1.64% -46.46% -63.71% 20.8% -59.62% # # # # # # # # # # # # # # # # # # # # # # # # # # # # # # # # # # # # # # # # # # # # # * # # # # # # # # # # # #
Slide 22: Favorable Safety Profile
Favorable Safety Profile Consistent with data from clinical trials in SLE, RA, gMG, and IgAN, and post-marketing data TEAE, treatment emergent adverse event; TRAE, treatment related adverse event; TESAE, treatment emergent serious adverse event; TRSAE, treatment related serious adverse event; SLE, s
Slide 23: pSD Commercial Opportunity
pSD Commercial Opportunity Dallan Murray, Chief Commercial Officer
Slide 24: Telitacicept: Potential Best-In-Disease Efficacy Globally (MG-ADL)
Telitacicept: Potential Best-In-Disease Efficacy Globally Statistically significant and clinically meaningful improvement in MG-ADL score PBO-adjusted LSM MG-ADL Change from Baseline Based on historical clinical data; not a head-to-head trial Efgartigimod - ADAPT; Nipocalimab - Vivacity-MG3; Rozanol
Slide 25: Telitacicept: Potential Best-In-Disease Efficacy Globally (ESSDAI)
Telitacicept: Potential Best-In-Disease Efficacy Globally Statistically significant and clinically meaningful improvement in ESSDAI -4.3 PBO-adjusted LSM ESSDAI Change from Baseline Efgartigimod - RHO; Nipocalimab - Bowman 2022, Lancet; Ianalumab - St. Clair 2024, Nature and Grader-Beck 2025 ACR; Da
Slide 26: Sjögren's: One of the Most Common Systemic Autoimmune Diseases Globally
Sjögren’s: One of the Most Common Systemic Autoimmune Diseases Globally ~870,000 diagnosed Sjögren’s patients across key markets1 Favorable Growth Drivers Rising diagnosis rates from better awareness and testing Underpenetrated Market No approved disease-modifying systemic therapies Patients managed
Slide 27: Telitacicept Checks Every Box for Sjögren's Disease
Prove Efficacy on ESSDAI Show statistically significant ≥3-point reduction in ESSDAI Phase III primary ESSDAI endpoint met with >3-point reduction vs placebo. Must be Safe Deliver a clean, non-cytotoxic B-cell approach; avoid infection or PML risk Favorable safety profile — no PML, minimal infection
Slide 28: Telitacicept: Redefining Success In Autoimmune Disease
01 Telitacicept Redefining Success In Autoimmune Disease 02 03 Telitacicept The Most Advanced BAFF/APRIL Inhibitor Globally Validated mechanism of action Proven pipeline-in-a-product Proof of concept in 5+ indications Myasthenia Gravis The Beachhead Indication Significant MG-ADL and QMG score reduct
Slide 29: Thank You
Thank You. Investors@vorbio.com