Vor Biopharma Inc. · NASDAQ:VOR
Vor Biopharma — Investor Presentation (September 2025)
39 slides from Vor Biopharma's investor presentation, Sep 2, 2025
Slide 1: Global Science. One Purpose. Corporate Presentation
Global Science. One Purpose. Corporate Presentation September 2025 Exhibit 99.1
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: China Is Now the Epicenter of Drug Innovation
China Is Now the Epicenter of Drug Innovation 50%+ of new global molecules originate in China Out-licensing deals are surging ($58B+ in 2025) ICH, International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use; Source: Bloomberg and Bernstein analysis; 1. Bloombe
Slide 4: The Medicine Meets the Moment
01 The Medicine Meets the Moment 02 03 04 Telitacicept Dual BAFF/APRIL Inhibitor Optimal approach for B cell driven autoimmune diseases Addresses upstream and downstream signaling Myasthenia Gravis The Beachhead Indication Best-in-disease foundation from China Phase 3 clinical study Market shifting
Slide 5: Telitacicept Leads the BAFF/APRIL Field with Broad Approval Momentum
Telitacicept Leads the BAFF/APRIL Field with Broad Approval Momentum Strong cash position of $190M* with runway into 1Q27 covers critical milestones Indications Preclinical Phase 1 Phase 2 Phase 3 Marketing Approval Milestones Systemic Lupus Erythematosus (SLE) Rheumatoid Arthritis (RA) Myasthenia G
Slide 6: Disease Modification Through Upstream and Downstream Control
Disease Modification Through Upstream and Downstream Control Ab, antibody Dual BAFF/APRIL blockade stops B cell survival and plasma cell antibody production Drives B cell development and survival Promotes proliferation of mature B cells BAFF Upstream BAFF APRIL Telitacicept TACI-Fc fusion protein: d
Slide 7: Unprecedented Efficacy in China Across Autoimmune Diseases
Commercial Approvals †Conditional Approval, Full Approval in 2023; *Accelerated Approval in China; Est., estimated Unprecedented Efficacy in China Across Autoimmune Diseases BLA Submissions Best-In-Disease in China 3 2 3 2021 - Systemic Lupus Erythematosus (SLE)† 2024 - Rheumatoid Arthritis (RA) 202
Slide 8: Favorable Safety At Scale
*From pooled safety analysis across all telitacicept trials. AEs, adverse events; SAEs, serious adverse events. Favorable Safety At Scale Favorable and Predictable Safety Profile Observed Among ~1,800* Patients Studied in Clinical Trials No Burdensome Vaccination Requirements No Signature B Cell Dep
Slide 9: A Pipeline of Autoantibody-Driven Diseases & Blockbuster Opportunities
A Pipeline of Autoantibody-Driven Diseases & Blockbuster Opportunities ANCA-AAV, ANCA-associated Vasculitis; ITP, Immune Thrombocytopenic Purpura; CIDP, Chronic Inflammatory Demyelinating Polyradiculoneuropathy; NMOSD, Neuromyelitis Optica Spectrum Disorder; IgG4-RD, IgG4-related disease. Telitacice
Slide 10: Myasthenia Gravis: Moving Beyond IgG Therapies
Myasthenia Gravis Moving Beyond IgG Therapies
Slide 11: A Large and Growing Global Opportunity in Myasthenia Gravis
A Large and Growing Global Opportunity in Myasthenia Gravis ~260,000 diagnosed MG patients across key markets1 Significant Burden A large, diagnosed patient population across key markets establishes a substantial initial opportunity Favorable Growth Drivers Prevalence is growing due to increased awa
Slide 12: MG Market Rapidly Expanding But Lacks Disease-Modifying Treatments
MG Market Rapidly Expanding But Lacks Disease-Modifying Treatments MG, myasthenia gravis; gMG: generalized myasthenia gravis; 1. EvaluatePharma and Global Data sales and consensus High growth market with underserved patient segments gMG US Biologic Sales ~90,000 Diagnosed MG patients in the US 60% P
Slide 13: Current Myasthenia Gravis Therapies Target Symptoms, Not Disease
Biologic use rising (~35% of gMG patients1) with choices driven by efficacy, convenience, phenotype, and coverage FcRn inhibitors dominate but ~20-50% of patients experience insufficient treatment responses2 Complement inhibitors limited by safety, black box warning, and vaccination requirements3 B
Slide 14: Phase 3 Trial in Generalized Myasthenia Gravis Completed in China
Phase 3 Trial in Generalized Myasthenia Gravis Completed in China MG-ADL; Myasthenia Gravis Activities of Daily Living; QMG, Quantitative Myasthenia Gravis; QW, per week. ClinicalTrials.gov. NCT05737160. Updated May 5, 2025. Accessed August 19, 2025. https://clinicaltrials.gov/study/NCT05737160 Best
Slide 15: Well Balanced Baseline Characteristics
Well Balanced Baseline Characteristics Consistent with recent global Phase 3 populations AE, adverse event; AChR, acetylcholine receptor; EP, efficacy population; MG-ADL, Myasthenia Gravis-Activities of Daily Living; MGFA, Myasthenia Gravis Foundation of America; MuSK, muscle-specific tyrosine kinas
Slide 16: MG-ADL Responders - Primary Endpoint Met
MG-ADL Responders - Primary Endpoint Met MG-ADL, Myasthenia Gravis-Activities of Daily Living; MMRM, mixed models for repeated measures. Statistically significant and clinically meaningful improvement in activities of daily living -4.8 -5.74 -0.91 Change from Baseline in MG-ADL at Week 24 points Pla
Slide 17: QMG Responders - Secondary Endpoint Met
QMG Responders - Secondary Endpoint Met QMG, Quantitative Myasthenia Gravis; MMRM, mixed models for repeated measures. Statistically significant and clinically meaningful improvement in physician-assessed measure of muscle strength -6.4 -8.66 -2.27 Change from Baseline in QMG at Week 24 points Place
Slide 18: Telitacicept: Potential Best-In-Disease Efficacy Globally
Telitacicept: Potential Best-In-Disease Efficacy Globally Statistically significant and clinically meaningful improvement in MG-ADL score -4.8 FcRn Inhibitors Complement Inhibitors Anti-CD19 mAb PBO-adjusted LSM MG-ADL Change from Baseline gMG RemeGen-sponsored trial MG-ADL, Myasthenia Gravis-Activi
Slide 19: Telitacicept Significantly Improved MG-ADL and QMG Scores
Telitacicept Significantly Improved MG-ADL and QMG Scores gMG †Missing data imputed as non-response. MG-ADL, Myasthenia Gravis - Activities of Daily Living; QMG, Quantitative Myasthenia Gravis. Mean Change in QMG Score† -8.66 -2.27 Mean Change in MG-ADL Score† -5.74 -0.91 Proportion of patients with
Slide 20: Telitacicept Provides Sustained Disease Modification While FcRn Inhibitors Require Repeat Cycles
Telitacicept Provides Sustained Disease Modification While FcRn Inhibitors Require Repeat Cycles †Missing data imputed as non-response. MG-ADL, Myasthenia Gravis - Activities of Daily Living REMEGEN Phase 3 China Trial -5.74 -0.91 Argenx Phase 3 Adapt Trial Cross trial comparison = Mean Change in To
Slide 21: Consistent Reduction in IgG, IgA, IgM, and B Cells
Consistent Reduction in IgG, IgA, IgM, and B Cells gMG IgG -32.89% 0.69% IgA -59.57% 0.00% IGM -68.93% 0.53% B cell -24.37% 2.61% RemeGen-sponsored trial
Slide 22: Favorable Safety Profile
Favorable Safety Profile AE, adverse event; pSD, primary Sjogren’s disease; SLE, systemic lupus erythematosus; RA, rheumatoid arthritis; IgAN, IgA nephropathy Consistent with data from clinical trials in SLE, RA, pSD, and IgAN, and post-marketing data gMG Telitacicept Placebo (n=57) (n=57) n (%) Eve
Slide 23: Telitacicept Targets What Matters and Preserves What Protects
Telitacicept Targets What Matters and Preserves What Protects MoA, mechanism of action; MG-ADL, myasthenia gravis-activities of daily living; QMG, quantitative myasthenia gravis; PBO, placebo A balanced IgG and B cell reduction allows for superior outcomes MoA TACI-Fc Anti-FcRn Anti-CD19 IgG Reducti
Slide 24: Global Phase 3 in Generalized Myasthenia Gravis
Global Phase 3 in Generalized Myasthenia Gravis MG-ADL, Myasthenia Gravis-Activities of Daily Living; QMG, Quantitative Myasthenia Gravis; QW, per week. *Estimated. †For patients who discontinue treatment before the open-label extension (OLE) period, end of treatment (EOT) and end of study (EOS) tim
Slide 25: Sjögren's Disease: Moving Beyond Symptom Management
Sjögren's Disease Moving Beyond Symptom Management
Slide 26: Sjögren's: A Large, Vastly Underserved Autoimmune Disease
Sjögren’s: A Large, Vastly Underserved Autoimmune Disease DMARDs, disease-modifying anti-rheumatic drugs; ESSDAI, EULAR Sjögren’s syndrome disease activity index Targeting ~100,0001 addressable patient US opportunity with a potential best-in-disease profile Symptom-Directed Local/topical therapies f
Slide 27: 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 28: Phase 2 Trial in Primary Sjögren's Disease Completed in China
Phase 2 Trial in Primary Sjögren's Disease Completed in China ESSDAI, EULAR Sjögren’s syndrome disease activity index; ESSPRI, EULAR Sjögren’s Syndrome Patient Reported Index; MFI, multidimensional fatigue inventory; MFI-20, multidimensional fatigue inventory; PGA, physician’s global assessment; PaG
Slide 29: Well Balanced Baseline Characteristics
Well Balanced Baseline Characteristics ESSDAI, EULAR Sjögren’s syndrome disease activity index; ESSPRI, EULAR Sjögren’s Syndrome Patient Reported Index; MFI, multidimensional fatigue inventory; MFI-20, multidimensional fatigue inventory; FAS, full analysis set; PPS, per-protocol set; SS, safety set;
Slide 30: ESSDAI Responders - Primary Endpoint Met
-3.8 +0.5 ESSDAI Responders - Primary Endpoint Met ESSDAI, EULAR Sjögren’s syndrome disease activity index Statistically significant and clinically meaningful improvement in ESSDAI at week 24 -4.3 Change from Baseline in ESSDAI at Week 24 points Placebo-adjusted decrease in ESSDAI: pSD -2.3 Xu D, et
Slide 31: Telitacicept: Potential Best-In-Disease Efficacy Globally
Telitacicept: Potential Best-In-Disease Efficacy Globally Statistically significant and clinically meaningful improvement in activities of daily living -4.3 FcRn Inhibitors Anti-BAFF-R mAb CD40L Antagonist PBO-adjusted LSM MG-ADL Change from Baseline pSD Efgartigimod - RHO; Nipocalimab - Bowman 2022
Slide 32: Consistent Reduction in IgG, IgA, IgM, and B Cells
Consistent Reduction in IgG, IgA, IgM, and B Cells pSD IgG IgA IGM B cell RemeGen-sponsored trial
Slide 33: Favorable Safety Profile
Favorable Safety Profile † AEs occurring in ≥ 2 subjects in any treatment group were listed. TEAE, treatment-emergent adverse event. Consistent with data from clinical trials in SLE, RA, gMG, and IgAN, and post-marketing data Placebo (N=14) Telitacicept 160 mg (N=14) Telitacicept 240 mg (N=14) TEAE,
Slide 34: Phase 3 Trial in Primary Sjögren's Disease
Phase 3 Trial in Primary Sjögren's Disease Completed in China Potential best-in-disease profile in China; randomized, double-blind, placebo-controlled study pSD *Estimated. ESSDAI, EULAR Sjögren’s syndrome disease activity index; ESSPRI, EULAR Sjögren’s Syndrome Patient Reported Index; MFI, multidim
Slide 35: Significant Near-Term Expansion Opportunities
2025 90k US patients Myasthenia Gravis 2026 380k US patients Myasthenia Gravis Primary Sjögren's Disease 2027+ 1M+ US patients Myasthenia Gravis Sjögren's Disease Systemic Lupus Erythematosus ANCA-associated Vasculitis Membraneous Nephropathy Immune Thrombocytopenic Purpura Bullous Pemphigoid Chroni
Slide 36: A Validated Therapy Poised For Rapid Global Expansion
gMG pSD Validating Biomarker Data Established Dose China Phase 3 Completed China Regulatory Status Global Phase 3 Enrolling Phase 3 Ready Launched BLA to be Submitted A Validated Therapy Poised For Rapid Global Expansion
Slide 37: Three Phase 3 China Readouts in Large Indications Before Year-End
Three Phase 3 China Readouts in Large Indications Before Year-End *Timing of readouts based on Remegen public statements; PBO, placebo; OLE, open-label extension; MG, myasthenia Gravis Each positive readout* strengthens the pathway to global commercialization Data: n=114, OLE 48-weeks Myasthenia Gra
Slide 38: Telitacicept Leads the BAFF/APRIL Field with Broad Approval Momentum
Telitacicept Leads the BAFF/APRIL Field with Broad Approval Momentum Strong cash position of $190M* with runway into 1Q27 covers critical milestones Indications Preclinical Phase 1 Phase 2 Phase 3 Marketing Approval Milestones Systemic Lupus Erythematosus (SLE) Rheumatoid Arthritis (RA) Myasthenia G
Slide 39: Thank You
Thank You. Investors@vorbio.com
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