Kyverna Therapeutics : AAN Conference Call Presentation

Published: 2026-04-22 07:16:10 ET KYTX

Published on 04/22/2026 at 07:16 am EDT

April 22, 2026

Speakers

Warner Biddle

Chief Executive Officer

Naji Gehchan, M.D., MSc, MBA

Chief Medical & Development Officer

Amanda Piquet, M.D., FAAN

University of Colorado Anschutz Céline Dion Foundation Endowed Chair

Sri Muppidi M.D.

Stanford Medicine

Solidifying our Leadership in Autoimmune CAR T

Stiff Person Syndrome (SPS) -

Primary Analysis Review

Generalized Myasthenia Gravis (gMG) -

Phase 2 Data Update

Advancing Valuable Market Opportunity in SPS

Q&A

Lead indications, SPS and gMG, addressing significant unmet medical need

Transformative clinical results reinforce potential to change the treatment paradigm by delivering drug-free, disease-free remission with a single dose

BLA submission preparations underway for SPS, a valuable commercial opportunity Phase 3 gMG trial paves way to significant market opportunity underpinned by

miv-cel's differentiated clinical profile

SPS Registrational Primary Analysis

Further supports path to approval & confidence in launch

gMG Phase 2 Longer-Term Follow-Up

Further supports confidence in Phase 3 Trial

Statistically significant, durable clinical benefit across all endpoints, with reversal of disability scores

New secondary endpoint results and translational data demonstrate full spectrum of miv-cel clinical benefit

Even deeper responses as data mature, with

durability out to 52 weeks

100% of patients achieved clinically meaningful response across MG-ADL, QMG, and MGC

Majority of patients achieved MSE

100% free of immunotherapies and a well-tolerated safety profile

SPS - 100% free of immunotherapies for SPS as of Week 16 and through last follow-up. gMG - 100% free of immunotherapies, including NSISTs, high-dose steroids (>10 mg), and FcRn and complement inhibitors up to 24 weeks.

MG-ADL, myasthenia gravis Activities of Daily Living; MGC, Myasthenia Gravis Composite; MSE, minimal symptom expression; QMG, Quantitative Myasthenia Gravis.

Mivocabtagene Autoleucel (miv-cel)1,2

Fully Human Autologous CD19 CAR T With CD28 Costim

Anti-CD19 scFv CD8α Hinge

CD8α TM

CD28 Costim

CD3ζ

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

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.

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.

Patient Perspective from KYSA-8: Before and After Treatment with Miv-cel

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Naji Gehchan, M.D., MSc, MBA - Chief Medical and Development Officer

Amanda Piquet, M.D., FAAN - University of Colorado Anschutz, Céline Dion Foundation Endowed Chair

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

Inadequate response with off-label symptomatic and immunomodulatory therapies1,2,5

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.

Large, multicenter, retrospective study assessing T25FW in patients with SPS (n=153)

Key Takeaways

Majority of patients had no or limited (<20%) improvement in T25FW

Disability (mRS) did not improve over time

Walking aid use increased over time

All patients treated with off-label immunomodulators or symptomatic medication

Study Contextualizes Transformative Miv-cel Data and Supports the T25FW as a Valid Longitudinal Measure of Mobility in SPS

Newsome SD, et al. Presented at the 2026 AAN Annual Meeting [abstract 966].

Received Both ODD and RMAT Designations

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

Cy/Flu, cyclophosphamide and fludarabine; ODD, Orphan Drug Designation; RMAT,

Regenerative Medicine Advanced Therapy; T25FW, timed 25-foot walk test.

20% improvement considered clinically meaningful1

Timed 25-Foot Walk (T25FW)

Validated tool to assess walking ability1

Used to evaluate stiffness and loss of mobility in SPS2

Healthy adults can perform the T25FW in ~4-5 seconds3

T25FW = Average of 2 Walks

FIRST WALK

SECOND WALK

TURN AROUND

25 FEET

START/STOP START/STOP

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.

Modified Rankin

Scale (mRS)

Hauser Ambulation

Degree of disability1

Time and degree of assistance to

No symptoms Nonsignificant

Disability

Slight Disability

Moderate Disability

Moderately Severe Disability

Severe Disability

Death

Index (HAI)

complete T25FW2

No symptoms Fatigue Abnormal gait

T25FW ≤10s

No Support

T25FW ≤20s

Unilateral support T25FW ≤20s

Unilateral support T25FW >20s*

Bilateral support T25FW >20s†

Cannot walk 25ft Frequent WC use

Restricted to WC

Can self transfer

Restricted to WC

No self transfer

Distribution-of-Stiffness

Index (DSI)

Muscle stiffness across body regions3,4

1 point for each stiff body region (0-6)

Heightened Sensitivity Scale (HSS)

Number of triggers of muscle spasms3,4

1 point for each trigger/stimulus (1-7)

T25FW, timed 25-foot walk; WC, wheelchair.

1. van Swietin JC, et al. Stroke. 1988; 19(5): 604-607. 2. Hauser SL, et al. New Engl J Med. 1983; 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.

46% Median Improvement at Week 16

Significant T25FW Improvement and Reduced Walking Aid Use

P = .0003

Baseline

n=26

Week 16

n=26

None

Unilateral

Patients, %

100

Bilateral

*Includes Includes IVIg/SCIg, PLEX, rituximab and/or prednisone (≥20 mg/day) for SPS symptoms.

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 followup, all 26 (100%) patients remained free of immunomodulatory or immunosuppressant therapies for SPS*

14 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.

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

96% of patients (25/26) had improvement in ≥1 primary or secondary efficacy endpoint

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

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

GAD65-IgG by RIA, nmol/L

750

P < .0001

56%

median reduction

500

250

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

Efficacy was maintained with B-cell reconstitution

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.

GAD65-IgG was reduced in 19/20 patients with

≥20 nM GAD65 at baseline

Naïve

Non-Class Switched

Memory

Class Switched

Memory

B-Cell Phenotypes

Regulatory T Cells

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

Data cutoff: 26Nov2025. Box and whisker plots showing median line (dark line), and interquartile range (box), and min/max (bars).

IgD, immunoglobulin D.

Treatment-Related Adverse

Events, n (%)

N=26

No high-grade CRS or ICANS observed

Most common treatment-related AEs were

CRS (any Grade)

24 (92)

Grade 1

10 (38)

Grade 2

14 (54)

ICANS (any Grade)

3 (12)

Grade 1

3 (12)

Grade 3/4 neutropenia

4 (15)

Any treatment-related serious AE

3 (12)

CRS (92%), fatigue (54%), diarrhea (38%), and

headache (31%)

4 patients had Grade 3/4 neutropenia, an expected AE with lymphodepletion and CAR T-cell therapies

− All events were manageable with treatment including G-CSF, fully resolved in 3 patients (median duration of 85 days), and was ongoing in 1 patient

− No serious infections associated with neutropenia

Treatment-related serious AEs occurred in 3 patients;

all fully resolved without sequalae

Data cutoff: 26Nov2025

CRS and ICANS graded using ASTCT criteria; other AEs graded using CTCAE criteria.

AE, adverse event; ASTCT, American Society for Transplantation and Cellular Therapy; CAR, chimeric antigen receptor; CTCAE, Common Terminology Criteria for Adverse Events; CRS, cytokine release syndrome; ICANS, immune effector cell-associated neurotoxicity syndrome; LD, lymphodepletion.

In KYSA-8 trial, a single dose of miv-cel resulted in:

Significant, robust, rapid improvements in mobility, disability, stiffness, and hypersensitivity

100% free of immunomodulatory or immunosuppressive therapies for SPS as of last follow-up

A consistent, well-tolerated, and manageable safety profile, with the potential for outpatient administration

Sustained clinical benefit following deep B-cell depletion and broad immune reset

Disclaimer

Kyverna Therapeutics Inc. published this content on April 22, 2026, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on April 22, 2026 at 11:15 UTC.