KYTX
Published on 05/12/2026 at 05:49 pm EDT
May 2026
CAR T Leadership
Potentially first approved autoimmune CAR T with most patients treated to date
Valuable Commercial
Opportunity in SPS Immediately addressable market and premium pricing potential
Best-in-Class Profile
Demonstrated durable drug-free, disease-free remission with single dose
Pipeline-in-a-Product
Clinical data supports expansion into broader indications (e.g., gMG, PMS)
Focused Strategy
Neuroimmunology-led franchise
Strong Financial Position
Supporting anticipated SPS commercial launch and gMG Phase 3 trial
gMG, generalized myasthenia gravis; PMS, progressive multiple sclerosis; SPS, stiff person syndrome.
3 ©2026 Kyverna Therapeutics, Inc.
Validate miv-cel differentiation with transformative clinical data in SPS and gMG
First mover advantage with SPS; establish commercial foundation; efficient gMG launch to follow
Broaden patient access and expand our reach to additional indications
Focused indication expansion
(e.g., PMS)
Develop no LD or alternative LD regimen
Reduce COGS and drive greater capacity and scalability
COGs, Cost of Goods Sold; LD, lymphodepletion; PMS, progressive multiple sclerosis.
4 ©2026 Kyverna Therapeutics, Inc.
Tolerability
Miv-cel: Potential First-in-Class and Best-in-Class CAR T Designed for Potency &
More than 100 patients dosed with miv-cel across multiple indications3
Deep and broad depletion of peripheral-and tissue-resident B cells to support broad immune reset and durable remission4,5
No high-grade CRS or ICANS3
First SPS and gMG patients treated with a single dose of miv-cel achieved durable efficacy beyond 24 months without the need for chronic immunotherapies6
Mivocabtagene Autoleucel (miv-cel)1,2
Only Fully Human Autologous CD19 CAR T With CD28 Costim
Anti-CD19 scFv CD8α Hinge
CD8α TM
CD28 Costim
CD3ζ
CRS, cytokine release syndrome; Costim, co-stimulation; gMG, generalized myasthenia gravis; ICANS, immune effector cell-associated neurotoxicity syndrome; scFv, single-chain fragment variable; TM, transmembrane.
©2026 Kyverna Therapeutics, Inc.
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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.
Autoimmune Disease: Clinical Stage Modalities
Single
Dose
Miv-cel
(Autologous CD-19 CAR T)
T-cell
engager
Chronic
Low
Deep B-cell depletion in tissue
Complete
Antibody
mRNA
CAR T
Antibodies require chronic dosing and provide inadequate depth of response
Drug-free Duration
mRNA CAR T has insufficient B-cell
depletion and requires redosing
T cell engagers require chronic dosing and lack complete B-cell depletion
mRNA, messenger ribonucleic acid.
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©2026 Kyverna Therapeutics, Inc.
In-vivo technologies are pre-clinical stage for autoimmune and several years from commercial, if successful
Stiff Person Syndrome
RMAT, ODD
Report primary analysis at AAN 2026
Generalized Myasthenia Gravis
RMAT, ODD*, FTD†
Report updated data on Phase 2 portion of KYSA-6 trial at AAN 2026
Additional Pipeline Opportunities
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BLA, Biologics License Application; FTD, Fast Track Designation; IIT, Investigator-initiated trial; IND,
Investigational New Drug; ODD, Orphan Drug Designation; RMAT, Regenerative Medicine Advanced Therapy.
*EU & US. †Fast Track Designation does not assure that we will experience a faster development process,
regulatory review or regulatory approval process compared to conventional FDA procedures.
© 2026 Kyverna Therapeutics, Inc.
Program
Anticipated Milestones
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Broad Immune Reset
Naïve B Cells in SPS
Regulatory T Cells in SPS
Resets B cells and upregulates regulatory T cells,
important suppressors of CNS disease inflammation2-4
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aAbsolute numbers of CAR T cells in CSF assumed a volume of 140 mL and 150 mL for female and male, respectively.
CAR, chimeric antigen receptor; CNS, central nervous system; CSF, cerebrospinal fluid; miv-cel, mivocabtagene autoleucel; SPS, stiff person syndrome.
1. Dunn J, et al. Presented at the ACTRIMS Forum 2026; February 5-7, 2026; San Diego, CA. Poster 112. 2. Piquet A, et al. Presented at the AAN Annual Meeting 2026; April 18-22, 2026; Chicago, IL. LBA Poster 8. 3. Goverman JM. N Engl J Med. 2021;384:578-580. 4. Harkins AL, et al. Crit Rev Immunol. 2022;42:1-27.
106
CNS Penetration and Expansion
CAR+ Miv-cel Expansion Detected in CSF at Day 14 in Progressive MSa
1000001005
104
103
102
101
10
1
Patient 1 Patient 2 Patient 3 Patient 4 Patient 5 Patient 6
Acts directly on cells in the CNS at the site of disease1
Cell Count by Flow Cytometry
Stiff Person Syndrome
First-to-market opportunity with highly efficient infrastructure
~6K U.S. diagnosed patients1
Severe, rare disease with no approved
therapies
High-cost burden (~$0.7 to $1.5M 3-yr cost per patient)2
Immediately addressable patients
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SOC, standard of care.
1. Crane PD, et al. Neurology. 2024;103(12):e210078. 2. Merative 2025 HCRU Analysis of Commercial Chronic Immunotherapy SPS patients.
3. Rodriguez E, et al. Muscle. Nerve. 2024;69(2):166-171. 4. Hendricks TM, et al. Am J Opthamol. 2019; 205:99-105. 5. ICER Report on MG 2021. 6. Global Data Pricing database.
Highly concentrated treatment network
Generalized Myasthenia Gravis
~80K U.S. diagnosed patients3,4; growing
market
Significant unmet need with current SOC
High-cost burden (~$2M 3-yr cost per patient)5,6
Opportunity to change the treatment paradigm
Strong commercial synergies with SPS
enables efficient scaling
Miv-cel: Potential for Significant Premium to Oncology CAR T Pricing; Biologics-like Margins
Neuroimmunology Franchise Synergies
Why Being 'First' Matters:
Develop relationships with neurologists and hospital staff
Activate commercial site network and establish processes with key autoimmune treatment centers
Establish price with payers
Build our end-to-end supply chain
Lay commercial foundation for a rapid gMG launch
Physicians
CMC
Approach
SPS
Treatment Centers
MG
Commercial and Medical Infrastructure
CMC, chemistry, manufacturing, and controls.
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©2026 Kyverna Therapeutics, Inc.
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80% of patients lose mobility, needing
walking aid assistance or wheelchair1-3
Only ~19% of patients remained able
to work after 4 years4
"Freezing attacks" and sudden falls
requiring ER care1,2
Risk of permanent disability and
increased mortality3
Devastating Impact on Patients
SPS impacts the inhibitory signaling pathways, which are the body's braking system and the target of autoantibodies produced by B
cells in SPS1,2
Symptoms characterized by muscle stiffness and painful muscle spasms, impacting mobility1-3
©2026 Kyverna Therapeutics, Inc.
Inadequate response with off-label symptomatic and immunomodulatory therapies1,2,5
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ER, emergency room.
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. 4.NCBI. https://www.ncbi.nlm.nih.gov/sites/books/NBK573078/
5. Dalakas MC. Neurol Neuroimmunol Neuroinflamm. 2023;10(3):e200109.
KYSA-8 Patient Perspectives:
Before and After Treatment with Miv-cel
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KYSA-8: Open-label, single-arm, multicenter study
Miv-cel Low-Dose Cy/Flu lymphodepletion
+
Single infusion of 1×108 CAR T cells
SPS immunotherapies are discontinued
Primary endpoints:
One-year Follow Up
N = 26
Age 18 to 75 years
Diagnosis of SPS
Inadequate response to immunomodulatory therapy
Stiffness index ≥2
Change from baseline in T25FW at 16 weeks
Safety
Secondary endpoints: change from baseline at 16 weeks
Modified Rankin Scale (mRS)
Distribution of Stiffness Index (DSI)
Hauser Ambulation Index (HAI)
Heightened Sensitivity Scale (HSS)
Rapid Clinical Enrollment Underscores Significant Unmet Need and Kyverna's Ability to Execute
Received Both ODD and RMAT Designations
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Cy/Flu, cyclophosphamide and fludarabine; ODD, Orphan Drug Designation; RMAT,
Regenerative Medicine Advanced Therapy; T25FW, Timed 25-Foot Walk.
©2026 Kyverna Therapeutics, Inc.
Significant T25FW Improvement and Reduced Walking Aid Use
P = .0003
81% of patients achieved clinically meaningful improvement (≥20% reduction from baseline)1
31% completed T25FW in <5 seconds;
typical time for healthy adults2
Of the 12 patients requiring a walking aid for T25FW at baseline, 67% (8/12) no longer needed assistance at
week 16
As of week 16 and through last follow-
Baseline
n=26
Week 16
n=26
54 12 35
85 4 12
0 20 40 60 80 100
Patients, %
None
Unilateral
Bilateral
up, all 26 (100%) patients remained free of immunomodulatory or immunosuppressant therapies for SPS*
*Includes Includes IVIg/SCIg, PLEX, rituximab and/or prednisone (≥20 mg/day) for SPS symptoms.
©2026 Kyverna Therapeutics, Inc.
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Data cutoff: 26Nov2025. Percentages may total more than 100% due to rounding. BL, baseline; T25FW, timed 25-foot walk.
1. Hobart J, et al. Neurology. 2013;80(16):1509-17. 2. Motl RW, et al. Mult Scler. 2017;23(5):704-710.
46% Median Improvement at Week 16
Modified Rankin Scale Hauser Ambulation Index
0 1 2 3 4 5 6 0 1 2 3 4 5 6 7 8 9
Significant (P< .0001) mean improvements in mRS and HAI of -0.8 (SD, 0.86) and -1.6 (1.13) and SPS-specific measures, DSI and HSS, of -1.5 (1.75) and -3.2 (2.01), respectively
©2026 Kyverna Therapeutics, Inc.
96% of patients (25/26) had improvement in ≥1 primary or secondary efficacy endpoint
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Data cutoff: 26Nov2025. Percentages may total more than 100% due to rounding.
BL, baseline; DSI, Distribution-of-Stiffness Index; HAI, Hauser Ambulation Scale; HSS, Heightened Sensitivity Scale; mRS, Modified Rankin Scale; wk, week.
6-Minute Walk Test (MWT): >4-fold improvement
over clinical minimally important change1
89-meter
median improvement at week 16
36-Item Short Form Health Survey (SF-36): Week 16 scores comparable to healthy adults for most domains2,3
©2026 Kyverna Therapeutics, Inc.
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Data cutoff: 26Nov2025.
1. Oosterveer DM, et al. Mult Scler Relat Disord. 2022;57:103438. 2. Maglinte GA, et al. J Clin Epidemiol. 2012;65(5):497-502. 3. Wu Q, et al. Medicine (Baltimore). 2023;102(24):e33979.
Robust CAR T-cell Expansion Deep B-cell Depletion Reduced GAD65-IgG
Median IQR
Median IQR
GAD65-IgG by RIA, nmol/L
750
P < .0001
56%
median reduction
500
250
0
Baseline Week 16
n=20 n=20
CAR-positive T cells peaked by day 14 • 54% of patients had B-cell
reconstitution by week 16
©2026 Kyverna Therapeutics, Inc.
Efficacy was maintained with B-cell reconstitution
GAD65-IgG was reduced in 19/20 patients with
≥20 nM GAD65 at baseline
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Data cutoff: 26Nov2025. Box and whisker plots showing median line (dark line), and interquartile range (box), and min/max (bars).
CAR, chimeric antigen receptor; IgG, immunoglobulin G; GAD65, glutamic acid decarboxylase 65.
Naïve
Non-Class Switched
Memory
Class Switched
Memory
B-Cell Phenotypes
Regulatory T Cells
Regulatory T Cells
©2026 Kyverna Therapeutics, Inc.
Newly emerging B-cell population showed significantly increased naïve phenotype with concomitant decrease in memory phenotype
Significant increase in
regulatory T cells at week 16
20
Data cutoff: 26Nov2025. Box and whisker plots showing median line (dark line), and interquartile range (box), and min/max (bars).
IgD, immunoglobulin D.
Disclaimer
Kyverna Therapeutics Inc. published this content on May 12, 2026, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on May 12, 2026 at 21:48 UTC.