Full Press Release Details
Interim Data for KYSA-6 Phase 2
Clinical Trial of KYV-101 in Generalized Myasthenia Gravis Conference Call October 29, 2025 Exhibit 99.1
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from named-patient basis access. Similar to expanded access or compassionate use in the United States, "IH" or "Individueller Heilversuch," also known as "named-patient basis access," is a regulatory scheme in
Germany that allows for the supply of a treatment that has not received marketing authorization for an individual patient in response to a request by the treating physician on behalf of the named patient. This option can be pursued for the expected
benefit of a patient who has exhausted all available treatment options, under the discretion of the treating physician, with the patient's consent. The use of KYV-101 in the IH settings is not a substitute for, or intended to replace, our
clinical trials. The goal is not to assess the effectiveness of a potential therapy, but rather to provide an individual patient with a possible efficacious approach when all other treatment options have failed, as determined by the patient's
physician. While we do not expect to be able to use the results from these activities as the basis for approval in our applications for marketing approval to the U.S. Food and Drug Administration (FDA) or other foreign regulatory agencies, we
believe such activities may provide additional clinical insights beyond highly focused clinical trials in specific geographies. Disclaimer and Forward-Looking Statements 2025 Kyverna Therapeutics, Inc.
Introduction Warner Biddle -
Chief Executive Officer
Today's Agenda Warner Biddle
Chief Executive Officer Sri Muppidi M.D. Stanford Medicine Naji Gehchan M.D., MSc, MBA Chief Medical & Development Officer Marc Grasso M.D. Chief Financial Officer Introduction Interim Phase 2 gMG Data Results Neuroimmunology Franchise and
Future Growth Opportunities Q&A Speakers Q&A
Despite Available Treatment Options,
High Disease Burden Remains in Generalized Myasthenia Gravis (gMG) Novel therapies are needed that minimize or eliminate symptoms of disease while reducing risks associated with chronic immunosuppression MG is a B-cell and antibody-mediated
neuromuscular autoimmune disease that causes fluctuating muscle weakness and fatigue1,2 Current State of Treatment for Patients With gMG Inadequate symptom control3,4 Few reach minimal symptom expression (MSE)1,5-6 Majority require ongoing
immunosuppressant therapy1-4 Costly and chronic treatment options1,7 1. Howard Jr JF, et al. Lancet Neurol. 2021;20(7):526-536. 2. Vu T, et al. NEJM Evid. 2022;1(5):EVIDoa2100066. 3. DeHart-McCoyle M, et al. BMJ Med. 2023;2(1):e000241. 4. Dewilde S,
et al. BMJ Open. 2023;13(1):e066445. 5. AstraZeneca. ULTOMIRIS efficacy data from CHAMPION-MG. https://ultomirishcp.com/gmg/efficacy. Accessed 20 Aug 2025. 6. Vu T, et al. AAN 2025. S34.002. 7. Data on File, Kyverna Therapeutics.
KYV-101: Upstream Targeting at the
Disease Source - A Fundamentally Different Approach to Treating gMG FcRn, neonatal fragment crystallizable receptor; mAb, monoclonal antibody. 1. Tur C, et al. Ann Rheum Dis. 2025;84(1):106-114. 2. DeHart-McCoyle M, et al. BMJ Med.
2023;2(1):E241. 3. Myasthenia Gravis. NIH - NINDS. https://www.ninds.nih.gov/health-information/disorders/myasthenia-gravis. 4 Sender R, et al. Proc. Natl. Acad. Sci. U.S.A. 2023;120 (44) e2308511120. Complement inhibitors transiently inhibit
autoantibody immune activity2 KYV-101 deeply depletes B cells including in tissues1 B-cell targeting mAbs cannot fully penetrate tissues and primarily target peripheral blood B cells1 FcRn inhibitors transiently reduce autoantibody accumulation2
Upstream Downstream Disease Source in Lymphoid Tissues B cells Lymph node Autoantibodies produced by B cells disrupt nerve signals at muscles2 <1% of B cells in peripheral blood4 Disease Symptoms at Muscles Nerve Muscle Acetylcholinesterase
inhibitors increase concentration of nerve signaling molecules3
KYV-101: Unique CAR Designed for
Potency & Tolerability in Autoimmune Diseases Fully Human Autologous CD19 CAR T With CD28 Costim 2025 Kyverna Therapeutics, Inc. | Confidential - Internal Use Only Anti-CD19 scFv CD8 TM CD28 Costim CD3 CD8
Hinge KYV-101 CAR Construct1,2 More than 100 patients dosed with KYV-101 across multiple indications; no high-grade CRS or ICANS have been observed3 KYV-101 has been shown to induce deep and broad depletion of blood- and tissue-resident B cells,4,5
which are an upstream therapeutic target in MG Initial 3 patients with gMG treated under expanded access pathway have achieved stable MSE with no background immunosuppressive therapies ongoing for 15 to 24 months3 CAR, chimeric antigen receptor;
CRS, cytokine release syndrome; gMG, generalized myasthenia gravis; ICANS, immune effector cell-associated neurotoxicity syndrome; MG-ADL, myasthenia gravis activities of daily living; MSE, minimal symptom expression; TM, transmembrane. 1. Brudno
JN, et al. Nat Med. 2020;26:270-280. 2. Alabanza L, et al. Mol Ther. 2017;25:2452-2465. 3. Data on File, Kyverna Therapeutics. 4. Minopoulou I, et al. Ann Rheum Dis.2025;84(3):e4-e7. 5. Albach FN, et al. Rheumatology. 2025;64(6):4075-4077.
Compelling Phase 2 Results Reinforce
KYV-101's Potential to Change the Treatment Paradigm in gMG MG-ADL, myasthenia gravis activities of daily living; QMG, quantitative myasthenia gravis score. More Patients to Minimal Symptom Expression (MSE) Single-Dose Treatment Unprecedented
Disease Control + Manageable Safety Profile Opportunity to Remove Background Therapies Only KYV-101 Has the Potential to Deliver ALL Four Components of a Paradigm-Shifting Therapy 1. 2. 3. 4.
Changing the Treatment Paradigm in gMG
Naji Gehchan, MD, MSc, MBA - Chief Medical and Development Officer
KYSA-6: Phase 2/3 Study of KYV-101
in gMG AchR, acetylcholine receptor; Cy/Flu, cyclophosphamide and fludarabine; gMG, generalized myasthenia gravis; LRP4, low-density lipoprotein receptor-related protein 4; MG-ADL, myasthenia gravis Activities of Daily Living; MGC, Myasthenia Gravis
Composite; MGFA, Myasthenia Gravis Foundation of America; MuSK, muscle-specific kinase; QMG, Quantitative Myasthenia Gravis. N = 6 Age 18 to 75 years Diagnosis of gMG, Class IIB-IV per MGFA criteria Autoantibodies to AChR, MuSK, or LRP4 MG-ADL
6 Failed 2 immunosuppressive/ immunomodulatory therapies OR failed 1 immunosuppressive therapy and required chronic plasmapheresis or IVIg to control symptoms Primary endpoints MG-ADL at 24 weeks Adverse events Key secondary
endpoints QMG and MGC scores PK/PD Phase 2 design: Open-label, single-arm, multicenter study KYV-101 Cy/Flu lymphodepletion + Single infusion of 1 108 CAR T cells 18-month follow up Interim analysis of ongoing phase 2 study with data cutoff of
Patients Treated With KYV-101 Had
Moderate to Severe gMG All patients had robust CAR T-cell expansion and B-cell depletion AChE-I, acetylcholinesterase inhibitor; AChR, acetylcholine receptor; FcRn, neonatal fragment crystallizable receptor; IVIG, Intravenous Immunoglobulin;
MG-ADL, myasthenia gravis activities of daily living; MGC, Myasthenia Gravis Composite; MGFA, MG Foundation of America; MuSK, muscle-specific kinase; NSIST, nonsteroidal immunosuppressive therapy; PLEX, plasma exchange; QMG, quantitative myasthenia
gravis. Characteristic N=6 Age, mean years (range) 45.5 (21-62) Sex, female | male, n (%) 5 (83%) | 1 (17%) Duration of MG, mean years (range) 5.3 (1.7-13.3) Total outcome score, mean (range) MG-ADL 11.2 (7-16) QMG 17.3 (9-28) MGC 21.8
(15-30) MGFA Class IIIb | Class IV at screening, n (%) 4 (67%) | 2 (33%) AChR | MuSK-positive at screening/historically, n (%) 5 (83%) | 1 (17%) Prior therapies, n (%) AChE inhibitors 6 (100%) NSISTs 6 (100%) FcRn and/or complement inhibitors 5
(83%) Steroids 6 (100%) IVIG and/or PLEX 4 (67%) Rituximab 3 (50%)
KYV-101 Demonstrated Rapid, Robust,
and Sustained Reductions in MG-ADL and QMG Data cutoff: October 3, 2025. BL, baseline; MG-ADL, myasthenia gravis activities of daily living; QMG, quantitative myasthenia gravis. MG-ADL score QMG score Baseline Change from BL Week 2 Week 24
Mean MG-ADL 11.2 -7.8 -8.0 Baseline Change from BL Week 2 Week 24 Mean QMG 17.3 -7.8 -7.7
After a Single Dose of KYV-101,
Patients Achieved Substantial and Clinically Meaningful Reductions in MG Outcome Scores and Treatment Burden Substantially Improved MG-ADL, QMG, and MGC 100% clinically meaningful response by MG-ADL & QMG (Reduction of 2 by MG-ADL,
3 by QMG) 100% responders by MG-ADL ( 3 point reduction by MG-ADL) 67% reached MSE (MG-ADL of 0 or 1) (n=3 with 24 weeks follow-up) 100% clinically meaningful response by MGC with -12.0 mean reduction at 24 weeks
(Reduction of 3 by MGC) Reduced Treatment Burden 100% free of nonsteroidal immunosuppressants (NSISTs), high-dose steroids (>10 mg), and FcRn and complement inhibitors up to 24 weeks Of the 6 patients, 5 remained free of these agents
as of their last follow-up Data cutoff: October 3, 2025. BL, baseline; FcRn, neonatal fragment crystallizable receptor; MG-ADL, myasthenia gravis activities of daily living; MGC, Myasthenia Gravis Composite; MSE, minimal symptom expression; QMG,
quantitative myasthenia gravis.
KYV-101 was Well-Tolerated with No
High-Grade CRS and No ICANS Observed Safety Patients (n=6) CRS (any Grade), n (%) 6 (100) Grade 1 4 (67) Grade 2 2 (33) ICANS (any Grade), n (%) 0 (0) Grade 3/4 TEAEs, n (%) 3 (50) Neutropenia 3 (50) Lymphopenia 1 (17) Lymphocyte count decreased 1
(17) Any treatment-related serious AE, n (%) 1 (17) No ICANS observed CRS was low-grade and manageable in all patients 4 of 6 patients only experienced fever (Grade 1 CRS) 3 patients with expected AEs associated with lymphodepletion and CAR T-cell
therapy 2 patients had transient Grade 3/4 neutropenia that resolved within 10 days of infusion 1 Grade 4 neutropenia SAE was manageable with G-CSF, not associated with infections, and had improved to Grade 1 by data cutoff Data cutoff: October
3, 2025 CRS and ICANS graded using ASTCT criteria; other AEs graded using CTCAE criteria. AE, adverse event; ASTCT, American Society for Transplantation and Cellular Therapy; CTCAE, Common Terminology Criteria for Adverse Events; CRS, cytokine
release syndrome; G-CSF, granulocyte colony-stimulating factor; ICANS, immune effector cell-associated neurotoxicity syndrome; TEAE, treatment-emergent adverse event.
Well-Positioned to Achieve
Best-in-Class Results in gMG with Phase 3 Trial In 6 patients with moderate to severe gMG, a single dose of KYV-101 resulted in: Robust, rapid, and sustained improvements regardless of prior biologic exposure Reduction of MG disease scores at 24
weeks: -8.0 for MG-ADL and -7.7 for QMG 100% responders by MG-ADL score ( 3-point reduction) Mean reductions at Week 2: -7.8 for MG-ADL and -7.8 for QMG At interim readout, most patients at or trending to MSE MSE achieved in 2/3 patients with
24 weeks of follow-up 100% free of NSISTs, high-dose steroids (>10mg), and FcRn and complement inhibitors up to 24 weeks Consistent, well-tolerated, and manageable safety profile FcRn, neonatal fragment crystallizable receptor; MG-ADL,
myasthenia gravis activities of daily living; MGC, Myasthenia Gravis Composite; MSE, minimal symptom expression; NSISTs, nonsteroidal immunosuppressants; QMG, quantitative myasthenia gravis.
Robust Phase 2 Results Strengthen
Confidence in Phase 3 Powering Assumptions, Efficient Trial-Size, and Co-primary Endpoint Measurement MG-ADL, myasthenia gravis activities of daily living; QMG, quantitative myasthenia gravis score. Interim Phase 2 Results Increase Phase 3
Probability of Success Reductions in MG-ADL and QMG exceeded the magnitude of effect assumed for Phase 3 co-primary endpoints Deep, sustained treatment effect was observed at week 24 - the timepoint of assessment for Phase 3 co-primary
Innovative and FDA-Aligned
Registrational Phase 3 Trial Design ~60-patient, global, open-label, randomized controlled Phase 2/3 trial with crossover design KYV-101 Cy/Flu lymphodepletion + Single infusion of 1 108 CAR T cells Standard of Care Traditional agents or
complement pathway inhibitors N = ~60 Randomized 1:1 Baseline Measurement Screening, washout, apheresis, and resume standard of care 24 weeks 18 Months Co-Primary Endpoints (MG-ADL & QMG) KYV-101 Crossover Follow Up Follow Up Standard of care
may consist of traditional agents (e.g., prednisone, azathioprine, mycophenolate, methotrexate, chronic IVIG/PLEX) or complement pathway inhibitors (e.g., eculizumab, ravulizumab). Anti-CD20 or -CD19 monoclonal antibodies or FcRn inhibitors not
allowed as defined in inclusion criteria. Cy/Flu, cyclophosphamide and fludarabine; FcRn, neonatal fragment crystallizable receptor; MG-ADL, myasthenia gravis activities of daily living; PLEX, plasma exchange; QMG, quantitative myasthenia gravis
Unprecedented MG Clinical Outcome
Measures Achieved with a Single Dose of KYV-101 Approved Investigational* FcRn Inhibitor1 VYVGART Complement Inhibitor2,3 ULTOMIRIS CD19 mAb4,5 UPLIZNA BCMA mRNA CAR T6 Descartes-08 KYV-101 CD19 CAR T (KYSA-6, n=3) Primary Endpoint
4 weeks 6 months 6 months 3 months 6 months Depth of Response Mean reduction from baseline to primary endpoint (non-placebo adjusted) MG-ADL Reduction ~4.6 3.1 4.2 ~4.2 8.0 QMG Reduction ~6.2 2.8 4.8 ~3.9 7.7 % Responders Patients with
3-point MG-ADL improvement from baseline to primary endpoint (non-placebo adjusted) ~73% ~57% ~79% ~70% 100% Achieve Minimal Symptom Expression (MSE) % of patients achieving MG-ADL of 0 or 1 40% 43% Not reported 33% 67% BCMA, b-cell
maturation antigen; FcRn, neonatal fragment crystallizable receptor; mAb, monoclonal antibody; MG-ADL, myasthenia gravis activities of daily living; mRNA, messenger RNA; QMG, quantitative myasthenia gravis score. *Under investigation in MG. 1.
Howard Jr JF, et al. Lancet Neurol. 2021;20(7):526-536. 2. Vu T, et al. NEJM Evid. 2022;1(5):EVIDoa2100066. 3. AstraZeneca. ULTOMIRIS efficacy data from CHAMPION-MG. https://ultomirishcp.com/gmg/efficacy. Accessed 20 Aug 2025. 4. Nowak RJ, et
al. N Engl J Med. 2025;392(23):2309-2320. 5. Nowak RJ, et al. AAN 2025. LS2.002. 6. Vu T, et al. AAN 2025. S34.002. At any point before primary endpoint 6 months to 1 year At any point before primary endpoint Note: These observations are derived
from separate clinical settings; comparisons across trials are not based on head-to-head studies.
Accelerating Potential
First-in-Class CAR T Franchise with MG and SPS Warner Biddle - Chief Executive Officer
Potential to Change the Treatment
Paradigm in a Large and Growing Market with KYV-101 ~80k U.S. Diagnosed gMG Patients1,2 Addressable Market1,3 KYV-101 ~12k Patients 15% of total diagnosed Patients with inadequate response to > 1 biologic* ~40k Patients 50% of total diagnosed
Patients with inadequate response to immunosuppressants Initial Priority Total KYV-101 Addressable Market 2025 Kyverna Therapeutics, Inc. gMG, generalized myasthenia gravis. *Biologics defined as immunomodulatory therapies including FcRN
blockers, complement inhibitors, rituximab or chronic IVIg use. 1. Rodriguez E, et al. Muscle. Nerve. 2024;69(2):166-171. 2. Hendricks TM, et al. Am J Opthamol. 2019; 205:99-105. 3. Clarivate DRG Report (2024).