Full Press Release Details
(KYV-101) in Stiff Person Syndrome (SPS) December 15, 2025 2025 Kyverna Therapeutics, Inc.
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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 Miv-cel 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 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. 2025 Kyverna Therapeutics, Inc. 2
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. Sham Dholakia, M.D., Ph.D. Amanda Piquet, M.D., FAAN Chief Financial Officer Chief Product Officer University of Colorado Anschutz C line Dion Foundation Endowed Chair 2025 Kyverna Therapeutics, Inc. 3
CAR, chimeric antigen receptor.
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
endpoints, reversing disability scores; patients remained free of immunotherapies as of last follow up; data supports BLA submission expected in 1H 2026 FIRST completed registration-enabling CAR T trial for autoimmune disease could pave the way for
miv-cel to become the FIRST and ONLY approved therapy in SPS and CAR T-cell therapy for autoimmune disease Valuable commercial opportunity in SPS, supported by attractive market dynamics and focused commercialization strategy First-to-market
opportunity in SPS lays foundation for expansion into broader indications 2025 Kyverna Therapeutics, Inc. 4 CAR, chimeric antigen receptor.
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
Sensitivity Scale Data cutoff: 26Nov2025 2025 Kyverna Therapeutics, Inc. 5
Patient Video KYSA-8 Clinical Trial
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 walking
aid assistance or wheelchair Substantial disease burden; symptoms "Freezing attacks" and sudden falls characterized by muscle stiffness and 1,2 requiring ER care 1-3 painful muscle spasms, impacting mobility Risk of permanent disability
and Significant unmet medical need; no 3 increased mortality 2 approved therapies ER, emergency room; GABA, gamma-aminobutyric acid. 2025 Kyverna Therapeutics, Inc. 7 1. Rakocevic G, et al. BMC Neurol. 2019;19:1. 2. Dalakas MC. Nat Rev Neurol.
2024;20(10):587-601. 3. Duddy ME, Baker MR. Front Neurol Neurosci. 2009;26:147-165.
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, rituximab, and IVIg
3-5 Therapy and Supportive Care Physical, speech, and occupational therapy 1-4 Psychiatric Therapy Anti-depressants and non-pharmacologic approaches Time 1. Dalakas MC. Nat Rev Neurol. 2024;20(10):587-601.2. Alexopoulos H, Dalakas MC. Eur J Clin
Invest. 2010;40(11):1018-25. 3. Dalakas MC. Neurol Neuroimmunol Neuroinflamm. 2023;10(3):e200109. 2025 Kyverna Therapeutics, Inc. 8 4. Yale Medicine. Stiff Person Syndrome (SPS). Accessed 21 Aug 2025. 5. National Institute of Neurological
Disorders and Stroke. Stiff-Person Syndrome. Accessed 21 Aug 2025. Physical Mobility
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- and tissue-resident B cells Anti-CD19 scFv First SPS and gMG patients treated with a CD8 Hinge single dose of miv-cel achieved durable efficacy beyond 24 months without the CD8 TM 6 need for background therapies CD28
Costim No high-grade CRS or ICANS have been 3 observed CD3 CAR, chimeric antigen receptor; CRS, cytokine release syndrome; gMG, generalized myasthenia gravis; ICANS, immune effector cell-associated neurotoxicity syndrome; TM,
transmembrane. 2025 Kyverna Therapeutics, Inc. 9 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. 6. Named patient access data, Kyverna Therapeutics.
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 disability
safety profile 2025 Kyverna Therapeutics, Inc. 10 Immunotherapy defined as off-label immunosuppressants (e.g., prednisone), rituximab and/or IVIg.
Registrational Topline Results Naji Gehchan, M.D., MSc, MBA -
Chief Medical and Development Officer
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 One- Secondary endpoints: change from Inadequate response + year baseline at 16 weeks to immunomodulatory Single infusion of 8 therapy Follow 1 10 CAR T cells Modified Rankin Scale (mRS) Up Distribution
of Stiffness Index (DSI) Stiffness index 2 Hauser Ambulation Index (HAI) Heightened Sensitivity Scale (HSS) Rapid clinical enrollment underscores significant unmet need and Kyverna's ability to execute RMAT,
Regenerative Medicine Advanced Therapy; ODD, Orphan Drug Designation; 2025 Kyverna Therapeutics, Inc. 12 BLA, biologics license application; CAR, chimeric antigen receptor; cy, cyclophosphamide; flu, fludarabine; T25FW, timed 25-foot walk
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) 11.2
(7.8-89.5) Distribution-of-stiffness index, median (range) 3 (2-6) Anti-GAD65 or anti-GlyR (%) GAD65-positive 23 (88%) GlyR-positive 3 (12%) Prior immunomodulatory therapy 26 (100%) (IVIg/SCIg, PLEX, rituximab), n (%) Data cutoff:
26Nov2025 2025 Kyverna Therapeutics, Inc. 13 GAD, glutamic acid decarboxylase; GlyR, glycine receptor; IVIg, intravenous immunoglobulin; PLEX, plasmapheresis; SCIg, subcutaneous immunoglobulin.
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 T25FW = Average of 2 Walks FIRST WALK TURN AROUND SECOND WALK 25 FEET START/STOP START/STOP 2025 Kyverna Therapeutics, Inc. 14 1. Hobart J, et al. Neurology. 2013; 80: 1509-1517. 2. Newsome SD and Johnson T J. Neuroimmunol.
2022;369:577915. 3. Motl RW, et al. Mult Scler J. 2017; 23(5): 704-710.
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% improvement
improvement at Week 16 Data cutoff: 26Nov2025 T25FW, timed 25-foot walk. 2025 Kyverna Therapeutics, Inc. 15 Immunotherapy defined as off-label immunosuppressants (e.g., prednisone), rituximab and/or IVIg.
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 None Unilateral
Bilateral Data cutoff: 26Nov2025 2025 Kyverna Therapeutics, Inc. 16
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 assistance to 0 1 2
3 4 5 6 7 8 9 Ambulation 2 No symptoms Fatigue Abnormal gait No Support Unilateral Unilateral Bilateral Cannot walk Restricted to Restricted to complete T25FW T25FW 10s T25FW 20s support support support 25ft WC WC * T25FW
20s T25FW >20s T25FW >20s Frequent WC Can self No self transfer Index (HAI) use transfer Distribution-of- Muscle stiffness across body 1 point for each stiff body region (0-6) Stiffness 3,4 regions Index (DSI) Number of Heightened
triggers of Sensitivity Scale 1 point for each trigger/stimulus (1-7) muscle 3,4 (HSS) spasms 1. van Swietin JC, et al. Stroke. 1988; 19(5): 604-607. 2. Hauser SL, et al. New Engl J Med. 1983; 2025 Kyverna Therapeutics, Inc. 17 308(4):
173-180. 3. Dalakas MC, et al. N Engl J Med. 2001; 345(26): 1870-1876. 4. Dalakas MC, et al. Ann Neurol. 2017; 82(2): 271-277.
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
Sensitivity Scale Data cutoff: 26Nov2025 2025 Kyverna Therapeutics, Inc. 18
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
the majority resolved within 28 Grade 1 3 (12) days of infusion Grade 3/4 neutropenia, n (%) 16 (62) Data cutoff: 26Nov2025 AE, adverse event; ASTCT, American Society for Transplantation and Cellular Therapy; CTCAE, Common Terminology Criteria for
Adverse Events; CRS, cytokine release syndrome; 2025 Kyverna Therapeutics, Inc. 19 ICANS, immune effector cell-associated neurotoxicity syndrome. CRS and ICANS graded using ASTCT criteria; other AEs graded using CTCAE criteria.
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+
Treatment: Over 8 years of IVIg plus valium, baclofen, and botulinum toxin 40 Symptoms: Back and neck pain, Patient T25FW headaches, stiffness, and spasms Baseline: 18.3 seconds 60 leading to decreased mobility Week 16: 5.0
seconds 73% Healthy Adult T25FW T25FW 1 ~4-5 seconds improvement 80 Miv-cel Safety Baseline Week 4 Week 8 Week 12 Week 16 Grade 2 CRS and Grade 3 neutropenia, resolved Improvement across all secondary efficacy endpoints: mRS, DSI,
HAI, and HSS CRS, cytokine release syndrome; DSI, distribution of stiffness index; GAD, GAD, glutamic acid decarboxylase; HAI, Hauser ambulation index; HSS, heightened sensitivity score; IVIg, intravenous 2025 Kyverna Therapeutics, Inc. 20
immunoglobulin; mRS, modified Rankin score; T25FW, timed 25-foot walk. 1. Motl RW, et al. Mult Scler J. 2017; 23(5): 704-710. Median Percent Change From Baseline in T25FW
Patient Video KYSA-8 Clinical Trial
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, durable
clinical improvement and reversed disability scores 100% elimination of off-label immunotherapies with a single dose Well-tolerated and manageable safety profile Data supports expected SPS BLA submission for miv-cel in 1H 2026 BLA, biologics license
application; CAR, chimeric antigen receptor. 2025 Kyverna Therapeutics, Inc. 22 Immunotherapy defined as off-label immunosuppressants (e.g., prednisone), rituximab and/or intravenous immunoglobulin.
Expanding Our Leadership in Autoimmune CAR T Warner Biddle -
Chief Executive Officer
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 groundwork for