Kyverna Therapeutics, Inc. · NASDAQ:KYTX
Kyverna Therapeutics — Technical Presentation (December 2025)
30 slides from Kyverna Therapeutics's technical presentation, Dec 15, 2025
Slide 1: Topline Data: KYSA-8 Registrational Trial of Miv-cel (KYV-101) in Stiff Person Syndrome
Exhibit 99.1 Topline Data: KYSA-8 Registrational Trial of Miv-cel (KYV-101) in Stiff Person Syndrome (SPS) December 15, 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: Today's Agenda
Today’s Agenda Speakers • Setting the Stage for Miv-cel’s Transformative Impact in SPS Warner Biddle Naji Gehchan, M.D., MSc, MBA Chief Executive Officer Chief Medical • Registrational Topline & Development Officer Results Q&A • Expanding Our Leadership in Autoimmune CAR T • Q&A Marc Grasso, M.D. Sh
Slide 4: Kyverna's Leadership in Autoimmune CAR T: Miv-cel's Transformative Impact in SPS
Kyverna’s Leadership in Autoimmune CAR T: Miv-cel’s Transformative Impact in SPS SPS is a debilitating, progressive autoimmune disease with no FDA-approved therapies and significant unmet medical need Miv-cel achieved statistically significant clinical benefit across all primary and secondary endpoi
Slide 5: Miv-cel Achieved Statistically Significant Benefit on Primary and All Secondary Efficacy Endpoints
Miv-cel Achieved Statistically Significant Benefit on Primary and All Secondary Efficacy Endpoints Primary Endpoint P Value 0.0002 Timed 25-Foot Walk Secondary Endpoints P Value Modified Rankin Scale <0.0001 Distribution of Stiffness Index <0.0001 Hauser Ambulation Index <0.0001 <0.0001 Heightened S
Slide 6: Patient Video KYSA-8 Clinical Trial
Patient Video KYSA-8 Clinical Trial
Slide 7: SPS is a Debilitating, Progressive Autoimmune Disease with No FDA-Approved Therapies
SPS is a Debilitating, Progressive Autoimmune Disease with No FDA-Approved Therapies SPS impacts the GABA signaling Devastating Impact on Patients pathway which is the body’s braking system and the target of autoantibodies 80% of patients lose mobility, needing 1,2 produced by B cells in SPS 1-3 wal
Slide 8: Off-Label Treatments Fail Majority of Patients and Have Serious Safety Risks
Off-Label Treatments Fail Majority of Patients and Have Serious Safety Risks Most patients with SPS receive symptomatic treatments and many try off-label immunotherapies 1-3 Symptomatic Treatments Muscle relaxants, benzodiazepines, and anti-seizures 1-3 Immunotherapy Off-label immunosuppressants, ri
Slide 9: Miv-cel: Unique CAR Designed for Potency & Tolerability in Autoimmune Diseases
Miv-cel: Unique CAR Designed for Potency & Tolerability in Autoimmune Diseases 1,2 Mivocabtagene Autoleucel (miv-cel) • More than 100 patients dosed with Fully Human Autologous CD19 CAR T 3 miv-cel across multiple indications With CD28 Costim • Observed deep and broad depletion of 4,5 peripheral- an
Slide 10: Topline Data Positions Miv-cel to Potentially be FIRST and ONLY Approved Therapy for SPS
Topline Data Positions Miv-cel to Potentially be FIRST and ONLY Approved Therapy for SPS Miv-cel Has the Potential to Deliver Transformative Outcomes for SPS Patients 3. 2. 4. 1. Reversing Well-tolerated Eliminating chronic Durable, single- progressive and manageable immunotherapies dose treatment d
Slide 11: Registrational Topline Results
Registrational Topline Results Naji Gehchan, M.D., MSc, MBA – Chief Medical and Development Officer
Slide 12: Registrational Trial Designed to Support Rapid Path to BLA
Registrational Trial Designed to Support Rapid Path to BLA Received Both ODD and RMAT Designation KYSA-8: Open-label, single-arm, multicenter study N = 26 Primary endpoints: • Change from baseline in T25FW at 16 weeks Miv-cel • Age 18 to 75 years • Safety • Diagnosis of SPS Cy/Flu lymphodepletion On
Slide 13: Baseline Characteristics Representative of a Real-World Spectrum of SPS Patients
Baseline Characteristics Representative of a Real-World Spectrum of SPS Patients Baseline Characteristics N=26 Age, median years (range) 56 (32–73) Sex, female | male, n (%) 16 (62%) | 10 (38%) Time since SPS diagnosis, median years (range) 3.2 (0.6–25.0) Timed 25-foot walk, median seconds (range) 1
Slide 14: Primary Endpoint Assesses Impact of SPS on Walking Ability
Primary Endpoint Assesses Impact of SPS on Walking Ability Timed 25-Foot Walk (T25FW) 20% improvement 1 • Validated tool to assess walking ability considered clinically 2 • Used to evaluate stiffness and loss of mobility in SPS 1 meaningful 3 • Healthy adults can perform the T25FW in ~4-5 seconds T2
Slide 15: Primary Endpoint Met: Robust and Sustained Improvement in Mobility
Primary Endpoint Met: Robust and Sustained Improvement in Mobility All Patients Remained Free of Immunotherapies Through Last Follow Up Significant Improvement in Timed 25-Foot Walk • Statistically significant improvement in T25FW (P=0.0002) • 81% of patients achieved clinically meaningful 46% impro
Slide 16: Many Patients Eliminated Ambulatory Aids After a Single Dose of Miv-cel
Many Patients Eliminated Ambulatory Aids After a Single Dose of Miv-cel Reduction in Use of Ambulatory Aids Baseline • Of 12 patients who n=26 required a walking aid-device prior to treatment, 67% Week 16 n=26 (8/12) no longer needed assistance 0 20 40 60 80 100 to walk Patients, % Ambulatory Aids N
Slide 17: Secondary Endpoints Assess Extent of Disability and SPS-Specific Symptoms
Secondary Endpoints Assess Extent of Disability and SPS-Specific Symptoms Degree of Modified Rankin 0 1 2 3 4 5 6 1 disability No symptoms Nonsignificant Slight Moderate Severe Death Moderately Scale (mRS) Disability Disability Disability Disability Severe Disability Hauser Time and degree of assist
Slide 18: Miv-cel Achieved Statistically Significant Benefit on Primary and All Secondary Efficacy Endpoints
Miv-cel Achieved Statistically Significant Benefit on Primary and All Secondary Efficacy Endpoints Primary Endpoint P Value 0.0002 Timed 25-Foot Walk Secondary Endpoints P Value Modified Rankin Scale <0.0001 Distribution of Stiffness Index <0.0001 Hauser Ambulation Index <0.0001 <0.0001 Heightened S
Slide 19: Well-Tolerated and Manageable Safety Profile
Well-Tolerated and Manageable Safety Profile Safety N=26 • No high-grade CRS or ICANS CRS (any Grade), n (%) 24 (92) • 16 patients had Grade 3/4 neutropenia, a known AE Grade 1 10 (38) associated with CAR T Grade 2 14 (54) treatments • All events were ICANS (any Grade), n (%) 3 (12) manageable with
Slide 20: Patient Case Study: Miv-cel in KYSA-8 Patient With Severe Disease
Patient Case Study: Miv-cel in KYSA-8 Patient With Severe Disease and Long-History of Other Symptomatic Treatments Patient History Miv-cel Efficacy • Age: 52-year-old man 0 • Time from diagnosis: 8.5 years Clinically Meaningful Improvement (20% Reduction) 20 • Antibody status: anti-GAD65+ • Treatmen
Slide 21: Patient Video KYSA-8 Clinical Trial
Patient Video KYSA-8 Clinical Trial
Slide 22: Today's Results Represent a Breakthrough in SPS
Today’s Results Represent a Breakthrough in SPS Potentially the First and Only Approved Therapy in SPS and CAR T for Autoimmune First completed registration-enabling trial in autoimmune CAR T Miv-cel achieved highly statistically significant benefit on primary and all secondary efficacy endpoints, d
Slide 23: Expanding Our Leadership in Autoimmune CAR T
Expanding Our Leadership in Autoimmune CAR T Warner Biddle – Chief Executive Officer
Slide 24: SPS Data Solidifies Kyverna's Leadership in Autoimmune CAR T
SPS Data Solidifies Kyverna’s Leadership in Autoimmune CAR T SPS Autoimmune Patients CAR T Could pave the way for Miv-cel to potentially FIRST mover potentially FIRST be the FIRST advantage for approved therapy in SPS, approved CAR T-cell Kyverna, laying designed to help patients therapy for groundw
Slide 25: Miv-cel: Potential to Be First and Only Approved Therapy in SPS with Addressable Patient Population
Miv-cel: Potential to Be First and Only Approved Therapy in SPS with Addressable Patient Population Ready for Better Options Miv-cel 2,3 Addressable Market Initial Total Miv-cel ~6k Priority Addressable Market U.S. Diagnosed 1,2 ~2.0 to 2.5k Patients ~5.5k Patients SPS Patients 30-40% of total diagn
Slide 26: Our Conviction in Delivering on a Valuable Commercial Opportunity in SPS
Our Conviction in Delivering on a Valuable Commercial Opportunity in SPS Attractive SPS Market Dynamics Focused Commercialization Strategy ✓ High unmet need ✓ First-mover advantage ✓ No approved therapies ✓ Scalable CDMO model 1,2 ✓ ~6,000 patients in U.S. - larger ✓ Targeting academic centers than
Slide 27: Promising Path to Achieving First-in-Class Neuroimmunology CAR T Franchise
Promising Path to Achieving First-in-Class Neuroimmunology CAR T Franchise INNOVATION & PIPELINE NEUROIMMUNOLOGY MS & RA: Early IIT data reinforce SPS: Positive topline data, valuable miv-cel’s potential across commercial opportunity; BLA on track autoimmune diseases for 1H 2026 MG: Positive Phase 2
Slide 28: Patient Video KYSA-8 Clinical Trial
Patient Video KYSA-8 Clinical Trial
Slide 29: Q&A
Warner Biddle Naji Gehchan, M.D., MSc, MBA Sham Dholakia, M.D., Ph.D. Chief Executive Officer Chief Medical Chief Product Officer & Development Officer Q&A Marc Grasso, M.D. Amanda Piquet, M.D., FAAN Chief Financial Officer University of Colorado Anschutz Céline Dione Foundation Endowed Chair
Slide 30: Kyverna's Leadership in Autoimmune CAR T: Miv-cel's Transformative Impact in SPS
Kyverna’s Leadership in Autoimmune CAR T: Miv-cel’s Transformative Impact in SPS SPS is a debilitating, progressive autoimmune disease with no FDA-approved therapies and significant unmet medical need Miv-cel achieved statistically significant clinical benefit across all primary and secondary endpoi
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