Kyverna Therapeutics, Inc. · NASDAQ:KYTX
Kyverna Therapeutics — Investor Day (August 2025)
97 slides from Kyverna Therapeutics's investor day presentation, Aug 28, 2025
Slide 1: A New Era for Neuroimmunology: Unleashing the Power of CAR T
A New Era for Neuroimmunology: Unleashing the Power of CAR T August 28, 2025 ©2025 Kyverna Therapeutics, Inc.
Slide 2: Disclaimer and Forward-Looking Statements
Disclaimer and Forward-Looking Statements This presentation contains forward-looking statements that are based on management’s beliefs and assumptions and information currently available to management of Kyverna Therapeutics, Inc. (“Kyverna”, “we”, “our,” or the “Company”). All statements other than
Slide 3: Welcome and Intro
Welcome and Intro Warner Biddle Chief Executive Officer ©2025 Kyverna Therapeutics, Inc.
Slide 4: Key Takeaways from Today's Event
Key Takeaways from Today’s Event Kyverna is uniquely positioned to fundamentally change the treatment 1 paradigm in MG and SPS: KYV-101 has demonstrated durable, drug-free and disease-free remission with a single dose 2 Valuable commercial opportunity in SPS given significant unmet need in a rare di
Slide 5: Today's Agenda
Today’s Agenda Setting the Stage Speakers Welcome and intro Why CAR T in Autoimmune Warner Biddle Chief Executive Officer SPS Overview Sham Dholakia M.D., Ph.D., Chief Product Officer Establishing a New Treatment Standard in SPS Naji Gehchan M.D., MSc, MBA, Chief Medical & Development Officer MG Ove
Slide 6: Our Experts for Today's Event
Our Experts for Today’s Event Ricardo Grieshaber-Bouyer, M.D., Ph.D. Aiden Haghikia, M.D. Sri Muppidi, M.D. FAU Erlangen-Nümberg Hanover Medical School Stanford Medicine 6 ©2025 Kyverna Therapeutics, Inc.
Slide 7: Liberating Autoimmune Patients through the Curative Potential of CAR T-Cell Therapy
LIBERATING AUTOIMMUNE PATIENTS through the CURATIVE POTENTIAL OF CAR T-CELL THERAPY 7 ©2025 Kyverna Therapeutics, Inc.
Slide 8: Kyverna Is Poised to Deliver on the Curative Potential of CAR T for Autoimmune Patients
Kyverna Is Poised to Deliver on the Curative Potential of CAR T for Autoimmune Patients Derisked Opportunity with Near-Term 100 patients Catalysts Potential to be treated First-in-Class with KYV-101 with Clear Unique Experienced Cell Path to BLA CAR Construct Therapy Optimal for Leadership & Autoimm
Slide 9: KYV-101: Ideal Modality for Treating Autoimmune Diseases
KYV-101: Ideal Modality for Treating Autoimmune Diseases Alternate Modalities TARGETED ATTRIBUTES KYV-101 Antibodies T-Cell In-Vivo Allogeneic Engagers Single dose ? ? Deep B-cell depletion ? ? in tissue On-target specificity ? Drug-free, durable remission ? ? 9 ©2025 Kyverna Therapeutics, Inc.
Slide 10: Executing on Long-Term Vision to Be the Leader in CAR T Across Autoimmune Diseases
Executing on Long-Term Vision to Be the Leader in CAR T Across Autoimmune Diseases Expand Our Reach Broaden Patient Access • Additional studies across B-cell driven autoimmune diseases (MS, RA, LN, others) • Unlock additional patient value Establish First-Mover Advantage with KYV-102, rapid whole bl
Slide 11: Today's Focus: Building a First-in-Class Neuroimmunology Franchise
Today's Focus: Building a First-in-Class Neuroimmunology Franchise Stiff Person Syndrome Myasthenia Gravis Strategic Rationale ✓ Compelling initial clinical data in both indications ✓ First-mover advantage ✓ High unmet patient needs ✓ Fast to market ✓ SPS: highly debilitating and progressive disease
Slide 12: Multiple, Value-Creating Near-Term Catalysts
Multiple, Value-Creating Near-Term Catalysts Program Anticipated Milestones Complete Pivotal Phase 2 Enrollment mid-2025 Stiff Person Syndrome Report Topline Pivotal Phase 2 Data 1H 2026 RMAT, ODD BLA filing in 1H 2026 Confirm Registrational Path with Regulators 1H 2025 Myasthenia Gravis Report Inte
Slide 13: Helping Autoimmune Patients Achieve Drug-Free, Disease-Free Remission
Helping Autoimmune Patients Achieve Drug-Free, Disease-Free Remission Free of active > 23 Months First SPS patient disease and off immunosuppressants and glucocorticoids > 24 Months First MG patient 13 Named-patient basis access data. KYV-101 therapeutic dose is 1×108 CAR T cells. ©2025 Kyverna Ther
Slide 14: Ricardo Grieshaber-Bouyer, M.D., Ph.D. FAU Erlangen-Nümberg
Ricardo Grieshaber-Bouyer, M.D., Ph.D. FAU Erlangen-Nümberg
Slide 15: Breakthrough Immunotherapies: Clinical Experience and Molecular Mechanisms
Breakthrough Immunotherapies: Clinical Experience and Molecular Mechanisms Ricardo Grieshaber-Bouyer, M.D. Ph.D., MHBA Professor of Clinical Systems Immunology Head of the Clinical Trial Unit Department of Internal Medicine 3 – Rheumatology and Immunology www.rgb-lab.de
Slide 16: B Cells Are Essential Players in Immune-Mediated Neurological Diseases
B Cells Are Essential Players in Immune-Mediated Neurological Diseases Stathopoulos & Marinos. Neurotherapeutics 2022
Slide 17: Targeting CD19 Provides Broad Coverage Across B Cell Subsets
Targeting CD19 Provides Broad Coverage Across B Cell Subsets Heeger et al. Nature Reviews Nephrology 2024
Slide 18: CD19 CAR T-Cell Therapy Transformed Autoimmune Disease Treatment
CD19 CAR T-Cell Therapy Transformed Autoimmune Disease Treatment Acute cutaneous lupus Myositis Interstitial lung disease ILD (FAPI PET-CT)
Slide 19: Prior Rituximab Led to Incomplete B Cell Depletion in the CNS
Prior Rituximab Led to Incomplete B Cell Depletion in the CNS – Complete B Cell Depletion Accomplished via CD19 CAR T-Cell Therapies 20-year old male with active SLE with severe, progressive CNS involvement with subtotal paraplegia. Treatment refractory to HCQ, GLC, MTX, RTX, CYC, plasmapheresis Hag
Slide 20: KYV-101 Crosses the Blood Brain-Barrier and Expands Within the CNS
KYV-101 CD19 CAR T-Cell Therapy Crosses the Blood Brain-Barrier and Expands Within the CNS KYV-101 CAR T-Cell Expansion in CNS in 4 Patients with Progressive MS Excellent CAR-T Expansion in Blood and CSF 1 2 3 4 Total Total Total Total Fold Fold Fold Fold Counts Counts Counts Counts Increase Increas
Slide 21: CD19 CAR T-Cells Deplete B Cells in Lymphoid Tissues
CD19 CAR T-Cells Deplete B Cells in Lymphoid Tissues Significant Before number of CAR T B-cells are in the tissues After Deep Tissue CAR T Depletion of B-cells After Incomplete Tissue Rituximab Depletion of B-cells Tur C & Grieshaber-Bouyer R & Schett G & Ramondo MG. et al. Ann Rheum Dis 2024
Slide 22: Evidence of Immune RESET in the B Cell Compartment
Evidence of Immune RESET in the B Cell Compartment Expanded transitional and immature B cells Naïve B cell repertoire relative cell number Wilhelm A & Grieshaber-Bouyer R & Schett G & Krönke G. et al. JCI Insight 2024
Slide 23: CD19 CAR T-Cell Therapy Has Profound Effects Beyond B Cells
CD19 CAR T-Cell Therapy Has Profound Effects on Many Other Immune Cell Types Beyond B Cells Schematic workflow 24 immune cell subsets Myeloid Cells Monocytes Dendritic cells MoCl (Classical) coDC (Conventional) MoIn (Intermediate) plDC (Plasmacytoid) MoNC (Nonclassical) T cells CD4 T cells CD8 T cel
Slide 24: Profound Favorable Effects of CD19 CAR T-Cells on Regulatory T Cells
Profound Favorable Effects of CD19 CAR T-Cells on Regulatory T Cells Treg inhibitory proteins are significantly increased post treatment to a level comparable to healthy donors Upregulated HD-1 Healthy Donors PD-L1 CTLA-4 HD-2 HD-3 HD-4 Downregulated HD-5 -Log10P-value IIM-1 T cell AID Pre-CAR IIM-2
Slide 25: Improved Biomarker Profile with CD19 CAR T Cells
Improved Biomarker Profile with CD19 CAR T Cells Different molecular signature of remission after CD19 CAR-T cell therapy compared with conventional pharmacotherapy in SLE Garantziotis P & Grieshaber.Bouyer R & Schett G & Parodis I. et al. Ann Rheum Dis 2025
Slide 26: Immune Reset: from Cellular Mechanisms to Holistic Understanding
Immune Reset: from Cellular Mechanisms to Holistic Understanding Expanding understanding of mechanisms of immune reset Deep B-cell depletion leading to immune reset Enhanced tolerogenic cells Naïve B cell Downregulated type I IFN Decreased inflammatory myeloid cells Grieshaber-Bouyer R & Chou J & Sc
Slide 27: Summary
Summary New insights into CD19 CAR T-cell therapy in autoimmune diseases ➢ Broad and deep depletion of pathogenic B cells in tissues and circulation ➢ Positive impact beyond B cells on a broader set of immune cell types ➢ Specific impact on regulatory T cells ➢ Broad impact on inflammatory cytokines
Slide 28: Establishing a New Treatment Standard in SPS
Establishing a New Treatment Standard in SPS Sham Dholakia, M.D., Ph.D. Chief Product Officer ©2025 Kyverna Therapeutics, Inc.
Slide 29: KYV-101 Has Demonstrated Promising Efficacy in Stiff Person Syndrome
KYV-101 Has Demonstrated Promising Efficacy in Stiff Person Syndrome Pre-infusion 4-6 Months Post 8 Months Post Loss of Leg Control; Repeated Falls; Able to Walk and Able to Walk Unaided Drug-Free Remission Turn With Aids Without Fear of Falling Wheel-Chair Support for >23 months Reduced stiffness I
Slide 30: SPS Is Highly Debilitating Due to Lack of Mobility
SPS Is Highly Debilitating Due to Lack of Mobility SPS Pathophysiology1,2 Progressive rigidity and muscle spasms GABAergic inhibitory pathways and synaptic signaling targeted SPS commonly associated with GAD autoantibodies produced by B cells Source: Bose S, et al. Pract Neurol. 2025;25:6-17. 1. Bai
Slide 31: SPS Is a Progressive Disease with Significant Disease Burden
SPS is a Progressive Disease with Significant Disease Burden and Symptom Severity that Can Lead to Mortality SPS Symptoms1 80% of patients lose mobility, MUSCULAR needing walking aid assistance PSYCHIATRIC SYMPTOMS RIGIDITY or wheel-chair2-4 generalized anxiety disorder, depression, taut facial expr
Slide 32: No FDA-Approved Therapies for SPS; Off-Label Treatments Fail
No FDA-Approved Therapies for SPS; Off-Label Treatments Fail Majority of Patients Most SPS patients receive symptomatic therapies and many eventually advance to off-label immunotherapies Physical Mobility Symptomatic Treatments1-3 Muscle relaxants and anti-seizure Immunotherapy1-3 Off-Label immunosu
Slide 33: Significant Limitations of Off-Label Immunotherapies Drive Need for Novel Options
Significant Limitations of Off-Label Immunotherapies Drive Need for Novel Treatment Options in SPS IVIg (Intravenous Immunoglobulin) Rituximab (anti-CD20 mAb) • Only modest improvement in stiffness and • Limited and inconsistent efficacy as either a spasms for many patients1 monotherapy or combinati
Slide 34: KYV-101 in SPS: Longer Term Follow-Up Data
KYV-101 in SPS: Longer Term Follow-Up Data Demonstrates Strong Clinical Activity and Potential for Deep and Durable Responses Kyverna Experience at Therapeutic Dose in Initial 2 Compassionate Use Patients Drug-Free Remission: Improvement in Mobility All Patients off Background Therapies* No. of Immu
Slide 35: Focused Registrational Study Design Supports Rapid Path to Potential BLA in SPS
Focused Registrational Study Design Supports Rapid Path to Potential BLA Approval in SPS KYSA-8: Open-label, single-arm, multicenter study N = 25 Primary endpoints • Age 18 to 75 years • Change in T25-FW at 16 weeks • Safety • Diagnosis of SPS Cyc/Flu lymphodepletion • Inadequate response Key second
Slide 36: Rapid Clinical Progress Underscores the Significant Unmet Need in SPS
Rapid Clinical Progress Underscores the Significant Unmet Need in SPS and Kyverna’s Ability to Execute Recruited from Fully Enrolled Topline Data 3 Centers of in 7 Months and BLA Filing Excellence Anticipated 1H26 36 ©2025 Kyverna Therapeutics, Inc.
Slide 37: KYV-101: Potentially First-in-Class Therapy for SPS with Clear Path to BLA
KYV-101: Potentially First-in-Class Therapy for SPS Patients with Clear Path to BLA SPS is a progressive and highly debilitating disease with no FDA-approved therapies Longer-term follow-up data1 continues to demonstrate KYV-101’s potential to set a new treatment standard in SPS with first FDA-appro
Slide 38: MG Overview
MG Overview Dr. Aiden Haghikia, M.D. Sri Muppidi, M.D. Naji Gehchan, M.D., MSc, MBA M.D., Hanover Medical School Stanford Medicine Chief Medical & Development Officer ©2025 Kyverna Therapeutics, Inc.
Slide 39: Transformative Outcomes with KYV-101 in MG
Transformative Outcomes with KYV-101 in MG Aiden Haghikia, M.D. Professor and Chair Department of Neurology and Clinical Neurophysiology Medizinische Hochschule Hannover (MHH)
Slide 40: Severe, Refractory Anti-AChR Seropositive MG Patient
Severe, Refractory Anti-AChR Seropositive MG Patient Patient 1 33 year-old mother of 4, disability pension, initial MG diagnosis 2012 • Five myasthenic crises in the last two years, requiring ICU care and tracheostomy • Refractory to rituximab, bortezomib • Chronic treatment with MMF and steroids •
Slide 41: Denise's CAR T Journey
Denise’s CAR T Journey Pre CAR T-Cell 6 Days Post CAR 13 Days Post CAR Therapy T-Cell Therapy T-Cell Therapy For purposes of scientific exchange only. All therapies are investigational.
Slide 42: Reduction of Autoantibodies with Preservation of Humoral Immunity
Reduction of Autoantibodies with Preservation of Humoral Immunity Reduction of Maintenance of pathogenic autoantibodies humoral protection Patient 1 ab, antibody; AChR, acetylcholine receptor; IgG, immunoglobulin G; VZV, varicella zoster virus. Haghikia A, et al. Lancet Neurol. 2023; 22: 1104–05. Fo
Slide 43: Reduction in Nerve Signaling to Muscles in MG
Reduction in Nerve Signaling to Muscles in MG Complement-mediated degeneration of the neuromuscular junction Reduced nerve signaling during repetitive stimulation Normal MG Meriggiolo, Lancet Neurol. 2009
Slide 44: Immune Pathways and Therapeutic Approaches in MG
Immune Pathways and Therapeutic Approaches in MG 1 2 Initiation of the Autoantibodies autoimmunity by produced by antigen-presenting pathogenic B cells cells 3 4 Nerve signals to Autoantibody muscles blocked recycling by autoantibodies Iorio, Nat. Rev. Neurol. 2024
Slide 45: Antibody-Independent Mechanisms of B Cells in MG
Antibody-Independent Mechanisms of B Cells in MG
Slide 46: KYV-101 CD19 CAR T-Cell Therapy in Generalized MG
KYV-101 CD19 CAR T-Cell Therapy in Generalized MG Disease Disease Prior Lines CD20 Patient Diagnosis Age Duration Description of Treatment Failure Seropositive 5 myasthenic crises in 1 MG 11 years 33 years 2 years requiring ICU care and tracheostomy 7 ✓ Seropositive Rapid onset of severe disease; in
Slide 47: KYV-101 in Generalized MG - Efficacy
KYV-101 CD19 CAR T-Cell Therapy in Generalized MG - Efficacy Disease MG-ADL QMG Follow-up Patient Diagnosis Age Duration Baseline Follow up Baseline Follow up Duration Seropositive 1 11 years 33 years 7 0 15 2 24 months MG Seropositive 2 1 year 75 years 3 0 7 2 22 months MG Seropositive 3 10 years 3
Slide 48: KYV-101 in Generalized MG - Safety
KYV-101 CD19 CAR T-Cell Therapy in Generalized MG - Safety Disease Patient Diagnosis Age CRS Grade (1-4) ICANS Grade (1-4) Duration Seropositive 1 11 years 33 years None None MG Seropositive 2 1 year 75 years 2 None MG Seropositive 3 10 years 36 years None None MG CRS, cytokine release syndrome; ICA
Slide 49: For purposes of scientific exchange only
For purposes of scientific exchange only. All therapies are investigational.
Slide 50: MG Treatment Landscape: Opportunity for CAR T-Cell Therapies
MG Treatment Landscape: Opportunity for CAR T-Cell Therapies Sri Muppidi, M.D. Stanford Medicine
Slide 51: Disclosures
Disclosures Advisory Board Meetings: • Alexion Pharma • Argenx Pharma • UCB Pharma • Amgen Pharma • J & J Pharma • Kyverna Therapeutics • Arcellx Pharma DSMB committee: • Dianthus Pharma • I will discuss some therapeutics in pipeline that are not FDA approved.
Slide 52: MG Disease Severity Over 10 Years
MG Disease Severity Over 10 Years • 19% remained symptomatic after 2 years • Remained treatment resistant Matthias Tomschik et al. Neurology 2020;95:e1426-e1436
Slide 53: MG-Activities of Daily Living (MG-ADL)
MG-Activities of Daily living (MG-ADL) Grade 0 1 2 3 Difficult to Intermittent slurring of Constant slurring or nasal, 1. Talking Normal understand • MG-ADL – To measure the nasal speech but can be understood speech impact of MG severity on Fatigue with solid 2. Chewing Normal Fatigue with soft food
Slide 54: Quantitative MG (QMG) Scale
Quantitative MG (QMG) Scale Test Item None Mild Moderate Severe Grade 0 1 2 3 • QMG requirements Double Vision on lateral gaze or left (circle one), seconds >60 11-60 1-10 Spontaneous − Spirometer Ptosis (upward gaze), seconds >60 11-60 1-10 Spontaneous Normal lid Complete, weak, Complete, without −
Slide 55: MG Composite (MGC) Scale
MG Composite (MGC) Scale Test Item None Mild Moderate Severe Ptosis, upward gaze (physician > 45 seconds = 0 11-45 seconds = 1 1-10 seconds = 2 Immediate = 3 • Mixed outcome measure examination) Double vision on lateral gaze, left > 45 seconds = 0 11-45 seconds = 1 1-10 seconds = 3 Immediate =4 or r
Slide 56: Complement Inhibitors Improve MG-ADL; However, Ongoing Treatment Is Needed
Complement Inhibitors Improve MG-ADL; However, Ongoing Treatment Is Needed Eculizumab1 Ravulizumab2 Zilucoplan3 0 1 0 Mean change from baseline in Placebo (n=63) -1 LSM change from baseline in -1 MG-ADL total score, 95%CI LSM change from baseline in MG-ADL total score, 95% CI MG-ADL total score (95%
Slide 57: FcRn Inhibitors Rapidly and Transiently Decrease MG-ADL
FcRn Inhibitors Rapidly and Transiently Decrease MG-ADL but Repeat Cycles Needed Efgartigimod1 Rozanolixizumab2 Nipocalimab3 0 0 1 Mean change from baseline in Mean change from baseline in Placebo (n=64) LSM change from baseline in 0 Placebo -1 MG-ADL total score, SE -1 MG-ADL total score, SE MG-ADL
Slide 58: Complement and FcRn Inhibitors
Complement and FcRn Inhibitors • Predominantly approved for AChR+ve MG patients • Increases the likelihood of achieving minimal symptom state • Likely helps reduce prednisone doses • Expensive therapies • Living with underlying disease; requires ongoing treatments • Continuous therapy • Ongoing back
Slide 59: Upstream Targeting with CD19 Monoclonal Antibody Improves MG-ADL
Upstream Targeting with CD19 Monoclonal Antibody Improves MG-ADL but Does not Eliminate Symptoms Inebilizumab • Investigational CD19 monoclonal antibody • Infusions every 6 months • Reduction in prednisone dose • Overall, B cell directed monoclonal antibodies have not resulted in dramatic outcomes •
Slide 60: Transient Transduction with Investigational mRNA CAR T-Cell Therapy Targeting BCMA
Transient Transduction with Investigational mRNA CAR T-Cell Therapy Targeting BCMA MG-ADL MG Composite QMG Chahin N, et al. Ann Clin Transl Neurol. 2025; Aug.
Slide 61: My Current Approach to MG Care
My Current Approach to MG Care Generalized MG Treatment AChR positive MG MuSK MG Options Mild Moderate Severe PYD, Pred, PYD, Pred, FcRn PYD, FcRn B cell therapy 1st line Oral IS therapies therapies, Pred (Rituximab) Pred, FcRn FcRn therapies Oral IS Oral IS therapies, Oral IS 2nd line+ Complement C
Slide 62: CAR T-Cell Therapies Can Be Paradigm Shift in MG Care
CAR T-Cell Therapies Can Be Paradigm Shift in MG Care • There are a number of newer MG therapies already available; however, all newer therapies require continued treatment • MG-ADL and MSE capture symptom control and improvement at a point in time but do not account for intermittent worsening and d
Slide 63: Changing the Treatment Paradigm in MG
Changing the Treatment Paradigm in MG Naji Gehchan, M.D., MSc, MBA Chief Medical & Development Officer ©2025 Kyverna Therapeutics, Inc.
Slide 64: Changing the Treatment Paradigm in MG with KYV-101
Changing the Treatment Paradigm in MG with KYV-101 Today High Unmet Goals of Patient Need KYV-101 Suboptimal results with costly, chronic treatment options Drug-free, disease-free remission with single dose Continued reliance on background therapies Elimination of background therapies Improve qualit
Slide 65: KYV-101 for MG: Upstream Targeting at the Disease Source
KYV-101 for MG: Upstream Targeting at the Disease Source KYV-101 deeply depletes Disease Source in B cells including in tissues1 Lymphoid Tissues Lymph node Upstream B cells B-cell targeting mAbs cannot fully penetrate tissues and primarily target <1% of B cells in peripheral blood B cells1 peripher
Slide 66: Current Generalized MG Treatment Landscape Before KYV-101
Current Generalized MG Treatment Landscape Before KYV-101 Treatment Naïve Options Refractory Treatment Options ~50% of patients2 ~50% of patients2 Immunosuppressive therapies (ISTs; e.g., steroids, Immunosuppressive therapies mycophenolate mofetil, azathioprine) ±Acetylcholinesterase inhibitors (ACh
Slide 67: Leading Therapies in MG Have High Administration Burden
Leading Approved and Investigational Therapies in MG Have High Administration Burden, Requiring Frequent and Chronic Dosing Summary of Dosing Schedules in gMG Year 1 Year 2 Year 3 Total Doses 4 weekly doses and 4 weeks off† FcRn Inhibitor1,2 VYVGART® 72 Approved Complement Inhibitor3 First doses 2 w
Slide 68: They Also Leave Patients with Residual Disease Burden
They Also Leave Patients with Residual Disease Burden and Require Chronic Background Therapy Approved Investigational* FcRn Complement CD19 BCMA mRNA Inhibitor1 Inhibitor2,3 mAb4,5 CAR T6 VYVGART® ULTOMIRIS® UPLIZNA® Descartes-08 Primary Endpoint 4 weeks 6 months 6 months 3 months Depth of Response
Slide 69: KYV-101 in MG: Longer-term Follow-up Data
KYV-101 in MG: Longer-term Follow-up Data Demonstrate Durable Drug-Free, Disease-Free Remission with Single Dose Kyverna Experience at Therapeutic Dose in Initial 3 Compassionate Use Patients Disease-Free Remission: Drug-Free Remission: All Patients off MG Background Therapies* All Patients Reached
Slide 70: Goal for Interim Phase 2 Data
Goal for Interim Phase 2 Data: Demonstrate Positive Trend Towards Durable, Drug-Free, Disease-Free Remission with KYV-101 Trends we aim to see in patients with longer follow-up period: Meaningful Setting a new Elimination of Manageable and reduction in MG-ADL standard in MSE background therapy predi
Slide 71: Registrational MG Trial Designed to Highlight KYV-101's Differentiated Profile
Registrational MG Trial Is Designed to Highlight KYV-101’s Differentiated Clinical Profile While Supporting Rapid Path to BLA Innovative, FDA- Efficient and Differentiated to Aligned Trial Robust Design Show Durability Expanding KYSA-6 Phase 2 trial into Randomized, superiority trial with 24-week en
Slide 72: Significant Effect Size Allows for Efficient Registrational Trial Design
Significant Effect Size Allows for Efficient KYV-101 Registrational Trial Design ~60-patient, global, open-label, randomized controlled Phase 2/3 trial with crossover design Co-Primary Endpoints (MG-ADL & QMG) KYV-101 Screening, Cyc/Flu lymphodepletion + N = ~60 Single infusion of 1×108 CAR T cells
Slide 73: Key Criteria and Endpoints for KYV-101 Registrational Trial in MG
Key Criteria and Endpoints for KYV-101 Registrational Trial in MG Key Inclusion Criteria Co-Primary Endpoints • Age 18 to 75 years • Change from baseline in MG-ADL and QMG score at 24 weeks compared to SOC • Diagnosis of generalized MG, Class II-IV per MGFA criteria • Autoantibodies to AChR or MuSK
Slide 74: Changing the Treatment Paradigm in MG with KYV-101
Changing the Treatment Paradigm in MG with KYV-101 High unmet need; suboptimal treatments with costly, chronic treatment options and continued reliance on background therapies Longer-term follow-up data1 reinforce KYV-101’s potential to change the treatment paradigm in MG by delivering durable, drug
Slide 75: Path to Commercializing our Neuroimmunology Franchise
Path to Commercializing our Neuroimmunology Franchise Dan Maziasz, MBA Chief Business Officer ©2025 Kyverna Therapeutics, Inc.
Slide 76: Kyverna Is Uniquely Positioned to Launch and Commercialize KYV-101
Kyverna Is Uniquely Positioned to Launch and Commercialize KYV-101 as the First CAR T in Autoimmune FIRST FAST EFFICIENT to launch in uptake given commercial model autoimmune unmet need and first given synergies CAR-T field mover advantage across SPS and MG 76 ©2025 Kyverna Therapeutics, Inc.
Slide 77: U.S. SPS Patient Population Significantly Larger than Previously Reported
Recent Epidemiology Study Indicates that U.S. SPS Patient Population Is Significantly Larger than Previously Reported U.S. Diagnosed SPS Patients SPS Market Overview • Recent epi study from University of Colorado ~6k indicates that SPS affects 1.4 to 2.1 per 100,000 individuals2 6x − Kyverna U.S. cl
Slide 78: SPS Is a Valuable Commercial Opportunity
SPS is a Valuable Commercial Opportunity with Potential for KYV-101 to Quickly Set a New Treatment Standard as First Approved Therapy KYV-101 Addressable Market2,3 ~6k Initial Total KYV-101 Priority Addressable Market U.S. Diagnosed SPS Patients1,2 ~2.0 to 2.5k Patients ~5.5k Patients 30-40% of tota
Slide 79: U.S. MG Market Is Significant and Continues to Grow
U.S. MG Market is Significant and Continues to Grow Driven by Increasing Awareness and Adoption of New Therapies U.S. Diagnosed MG Patients MG Market Overview • ~100K total diagnosed U.S. patients1 100K 20K − ~80% with seropositive generalized MG2,3 80K − Robust prevalence growth in MG1,5 40K − ~50%
Slide 80: KYV-101 Potential to Change the Treatment Paradigm in MG
KYV-101 Potential to Change the Treatment Paradigm in MG by Delivering Durable, Drug-free, Disease-free Remission with Single Dose KYV-101 Addressable Market1,3,4 ~80k Initial Total KYV-101 Priority Addressable Market U.S. Diagnosed gMG Patients1,2 ~12k Patients ~40k Patients 15% of total diagnosed
Slide 81: Current Treatment Options Have High-Cost Burden and Suboptimal Outcomes
Current Treatment Options in SPS and MG Have High-Cost Burden and Suboptimal Patient Outcomes Creating an Opportunity for KYV-101 High-Cost Burden Suboptimal Patient Outcomes4,5 Estimated 3-Year Total Cost per Patient* • Residual symptoms and disease burden ~$2 Million impacting patient quality of l
Slide 82: Commercial Strategy to Build an Efficient and Scalable Neuroimmunology Franchise
Commercial Strategy to Build an Efficient and Scalable Neuroimmunology Franchise Starting with SPS and Rapidly Expanding to MG Neuroimmunology Franchise Synergies Leverage SPS to set the foundation for efficient CAR T market entry and scalable growth in MG Treatment Physicians Centers First Mover Ad
Slide 83: SPS and MG Patients Concentrated at Key Academic Centers
SPS and MG Patients Are Concentrated at Key Academic Centers Enabling a Lean Commercial Model • SPS and MG patients are typically treated by Targeted U.S. Treatment Centers neurologists and same care teams at these academic centers • Focus on building relationships and establishing network of key ac
Slide 84: KYV-101 Is Set Up for Commercial Success
KYV-101 Is Set Up for Commercial Success Given Autologous CAR T Class Evolution and Differentiated Product Profile CAR T foundation is established Commercial strategy leverages CAR T and translates KYV-101 infrastructure and attributes unique to to autoimmune KYV-101 System capacity continues to Lev
Slide 85: KYV-101: Attractive Commercial Opportunity in Neuroimmunology
KYV-101: Attractive Commercial Opportunity in Neuroimmunology Significant KYV-101 addressable market in both SPS and MG SPS: Set a new treatment standard as first FDA-approved therapy MG: Change the treatment paradigm in large, growing market Opportunity for KYV-101 given current treatment options h
Slide 86: Conclusion and Q&A
Conclusion and Q&A Warner Biddle Chief Executive Officer ©2025 Kyverna Therapeutics, Inc.
Slide 87: Key Takeaways from Today's Event
Key Takeaways from Today’s Event Kyverna is uniquely positioned to fundamentally change the treatment 1 paradigm in MG and SPS: KYV-101 has demonstrated durable, drug-free and disease-free remission with a single dose 2 Valuable commercial opportunity in SPS given significant unmet need in a rare di
Slide 88: Q&A Participants
Warner Biddle Sham Dholakia, M.D., Ph.D Naji Gehchan MD, MSc, MBA Chief Executive Officer Chief Product Officer Chief Medical & Development Officer Q&A Marc Grasso, M.D. Chief Financial Officer Dan Maziasz, MBA Chief Business Officer Ricardo Grieshaber-Bouyer, M.D., Ph.D Aiden Haghikia, M.D. Sri Mup
Slide 89: Liberating Autoimmune Patients Through the Curative Potential of CAR T-Cell Therapy
Liberating Autoimmune Patients Through the Curative Potential of CAR T-Cell Therapy KYV-101 has potential to deliver durable, disease-free, drug-free remission with single dose First-in-class potential with neuroimmunology strategy Attractive market opportunity supported by focused commercial approa
Slide 90: Appendix
Appendix ©2025 Kyverna Therapeutics, Inc.
Slide 91: KYV-101: Unique CAR Design Optimal for Autoimmune Diseases
KYV-101: Unique CAR Design Optimal for Autoimmune Diseases Fully Human Design Developed for POTENCY • CD19 targeting eliminates broadest range of B-cell subsets while sparing long-lived plasma cells1 Anti-CD19 scFv • CD28 costimulation is highly potent1,2 • Deep B-cell depletion in tissue and immune
Slide 92: KYV-102: Transforming the Next Generation of CAR T Patient Delivery
KYV-102: Transforming the Next Generation of CAR T Patient Delivery No Apheresis, No/Low Lymphodepletion, Reduces Costs, Improves Patient Access KYV-101 KYV-102 TARGETED BENEFITS CD19 CAR optimal Utilizes same CAR Improve patient experience for autoimmune construct as KYV-101 Unlock system capacity
Slide 93: KYSA-6 Phase 2 Study Design
KYSA-6 Phase 2 Study Design Foundational for Advancement Into Registrational Trial KYSA-6: Open-label, single-arm, multicenter study N=6 Primary endpoints • Age 18 to 75 years • MG-ADL at 24 weeks • Diagnosis of gMG, Class • Adverse events IIB-IV per MGFA criteria Cyc/Flu lymphodepletion Key seconda
Slide 94: Abbreviations
Abbreviations ab, antibody cMBc, class switched memory B cells AChEi, acetylcholinesterase inhibitor CMC, chemistry, manufacturing, and controls AChR, acetylcholine receptor CNS, central nervous system AID, autoimmune disease coDC, conventional lDC plasmacytoid dendritic cells aMBc, atypical memory
Slide 95: Abbreviations
Abbreviations FDA, US Food and Drug Administration ICD-10, International Classification of Diseases, 10th Revision fDC, follicular dendritic cell ICU, healthy donor FDR, false discovery rate IDL, interstitial lung disease Flu, fludarabine IFN, interferon FTD, Fast Track Designation Ig, immunoglobuli
Slide 96: Abbreviations
Abbreviations LTβ, lymphotoxin-β MSE, minimal symptom expression LTβR, lymphotoxin-β receptor MTX, methotrexate mAb, monoclonal antibody MuSK, muscle-specific tyrosine kinase Mbim, IgM+ IgD– memory B cells Nkhi, CD56hi NK cells MG, myasthenia gravis Nklo, CD56low NK cells MG-ADL, myasthenia gravis a
Slide 97: Abbreviations
Abbreviations RA, rheumatoid arthritis T4nv, naïve CD4 T cells REMS, risk evaluation and mitigation strategy T4ra, CD45RA+ effector CD4 T cells RMAT, Regenerative Medicine Advanced Therapy T8cm, central memory CD8 T cells RNA-seq, ribonucleic acid sequencing T8em, effector memory CD8 T cells RTX, ri