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Corporate Presentation FEBRUary 2025
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well as our ability to successfully complete research and further development and commercialization of our drug candidates in current or future indications, including the timing and results of our clinical trials and our ability to conduct and
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data read-out at upcoming medical or scientific meetings; our belief that rese-cel has the potential to provide drug-free, durable meaningful clinical responses, through an immune reset, including the potential for achieving drug-free remission in
patients with refractory myositis; the Company's advancement of separate Phase 1/2 clinical trials of rese-cel in patients with SLE, myositis, SSc and gMG and advancement of the RESET-PV and RESET-MS trials, including updates related to status,
safety data, efficiency of clinical trial design and timing of data read-outs or otherwise; our ability to leverage our experience in autoimmune cell therapy; our ability to enroll the requisite number of patients, dose each dosing cohort in the
intended manner and timing thereof, and advance the trial as planned in our Phase 1/2 clinical trials of rese-cel; the timing any planned regulatory filings for our development programs, including IND applications; the progress and results of our
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or applicable to, clinical responses in patients with mPV, risks related to clinical trial site activation or enrollment rates that are lower than expected, our ability to demonstrate sufficient evidence of safety, efficacy and tolerability in its
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Develop and launch the first curative
targeted cellular therapies for patients with autoimmune diseases
Planning to leverage increasing
clinical data and unique development program for rese-cel1 2025: Pursuing an efficient path to approval CRS, cytokine release syndrome; DM - dermatomyositis; DORIS - definition of remission in SLE; ICANS, immune effector cell-associated
neurotoxicity syndrome; LN - lupus nephritis; PK - pharmacokinetic; PD - pharmacodynamic; SLE - systemic lupus erythematosus; TIS - total improvement score. resecabtagene autoleucel; CABA-201 As of Jan 8, 2025. As of
Feb 13, 2025. Golder, et al. Lupus. 2018;27(3): 501-506 Unique development strategy designed to accelerate time to approval and launch Compelling clinical efficacy with favorable safety profile & deep B cell depletion Multiple near-term
catalysts including clarity on potential path to approval Multiple disease-specific trials with a common design allow for generation of disease-specific efficacy data with shared safety database Weight-based dose, single infusion; supported by
clinical & translational data2 Industry-leading clinical network: 50 active US and European sites 26 patients enrolled Enrolling ~1 pt/week since Nov. 20243 Deepening clinical efficacy data over-time with immunosuppressant and steroid-free
outcomes2: DORIS remission in 3 SLE patients Complete renal response in 1st LN pt Major TIS response in 1st DM patient Favorable safety profile in 1st 10 patients 90% either no CRS or Grade 1 CRS 90% no ICANS Deep systemic B cell depletion observed
in the periphery and confirmed in the tissue in scleroderma patient by lymph node biopsy Meeting scheduled with FDA to align on registrational trial designs in 1H25 for rese-cel Enroll and complete dosing in multiple disease-specific cohorts in 2025
Present clinical data on rese-cel at medical meetings throughout 2025, including data evaluating rese-cel without preconditioning Patients are seeking a drug-free, symptom-free life which is rarely achieved despite current therapies; physicians also
prioritize prevention of end-organ damage4
RESET clinical program has
disease-specific cohorts designed to evolve directly into registrational studies Innovative clinical strategy with potential for accelerated regulatory path Program1 Trial Preclinical Phase 1/2 Pivotal Rese-cel (CABA-201) 4-1BB CD19-CAR T
RESET-Myositis CARTA Chimeric Antigen Receptor T cells for Autoimmunity RESET-SLE RESET-SSc RESET-MG RESET-MS RESET-PV Dermatomyositis Antisynthetase syndrome Immune-mediated necrotizing myopathy Lupus
Nephritis Non-Renal SLE Skin + Organ Cohort AChR-Ab neg. gMG AChR-Ab pos. gMG Skin Cohort Rheumatology Neurology Dermatology FTD Mucocutaneous & mucosal pemphigus vulgaris Contains cohort(s) without preconditioning Juvenile Myositis Pediatric
Indication RESET - REstoring SElf-Tolerance; Ab - Antibody; AChR - Acetylcholine receptor; gMG - Generalized myasthenia gravis; MS - Multiple sclerosis; SLE - Systemic lupus erythematosus Additional
pipeline candidate includes MuSK-CAART for MuSK-Ab positive MG, currently being evaluated in a Phase 1 trial. FDA Fast Track Designation received in dermatomyositis, SLE and lupus nephritis, systemic sclerosis, mucosal pemphigus vulgaris, MuSK-Ab
positive MG, and multiple sclerosis. Progressive MS Relapsing MS
Chimeric Antigen Receptor T Cells for
Autoimmunity (rese-cel)
~150 autoimmune patients have been
dosed with a range of autologous CAR T constructs (industry & academia)1 Autologous CAR T is potentially transformational for autoimmunity IIM - idiopathic inflammatory myopathy; MG - Myasthenia gravis; MS - Multiple sclerosis;
SPS - Stiff person syndrome; SSc - Systemic sclerosis. Note: Other' indications includes CIDP, IgG4-related disease, ANCA-associated vasculitis, NMOSD, Lambert Eaton myasthenic syndrome, autoimmune encephalitis. 1. Data as
of November 2024 (ACR Convergence 2024) based on Cabaletta Bio literature review across industry and academia. 2. Abstract 1749: Safety and Long-term Efficacy of CD19-CAR T-cell Therapy in 30 Patients with Autoimmune Disease. ACR Convergence 2024.
Compelling immunosuppressant-free clinical responses observed across many autoimmune diseases, durable for up to 4 years Clinical recurrences observed in <5% of ~150 treated patients IIM patient relapsed after 12+ months of drug-free remission No
evidence of CD19 escape or failure Patient treated with BCMA-CAR T & now in remission2 ~150 Patients Treated (by Disease) Type of CAR T Construct Efficacy Observations1 Safety Observations1 Among ~150 autoimmune patients dosed with cell therapy,
frequency and severity of CRS/ICANS events were considerably lower than those observed in cancer patients <2% Gr 3+ CRS & Gr 3+ ICANS events observed Vaccine titers stable with CD19-CAR T mono-targeting therapy insufficient data with
CD19xBCMA-CAR T 31% 60% 8%
Cabaletta rese-cel binder with similar
in vitro & in vivo activity to construct used in academic studies1,3 Rese-cel: CD19-CAR T specifically designed for autoimmunity Peng, Binghao J, et al. "Preclinical specificity and activity of CABA-201, a fully human 4-1BB containing CD19
CAR T therapy for treatment-resistant autoimmune disease." Poster presented at: American Society Gene and Cell Therapy 26th Annual Meeting; 2023 May 19; Los Angeles, CA. Dai, Zhenyu, et al. "Development and functional characterization of novel
fully human anti CD19 chimeric antigen receptors for T cell therapy." Journal of Cellular Physiology 236.8 (2021): 5832-5847. M ller, Fabian, et al. "CD19 CAR T-Cell Therapy in Autoimmune Disease-A Case Series with Follow-up."
New England Journal of Medicine 390.8 (2024): 687-700. Maschan, Michael, et al. "Multiple site place-of-care manufactured anti-CD19 CAR-T cells induce high remission rates in B-cell malignancy patients." Nature Communications 12, 7200
(2021) Transmembrane domain in rese-cel is CD8 vs. TNFRSF19 (Troy) utilized in the academic construct. The two transmembrane domains have not been shown to have a significant difference in function or IFN- production in preclinical
studies. The CD8 transmembrane domain is employed in tisagenlecleucel. Volkov, Jenell, et al. "Case study of CD19 CAR T therapy in a subject with immune-mediate necrotizing myopathy treated in the RESET-Myositis phase I/II trial."
Molecular Therapy 32.11 (2024): 3821-3828. Abstract 1733: Safety and Efficacy of CABA-201, a Fully Human, Autologous 4-1BB Anti-CD19 CAR T Cell Therapy in Patients with Immune-Mediated Necrotizing Myopathy and Systemic Lupus Erythematosus from the
RESET-MyositisTM and RESET-SLETM Clinical Trials. ACR 2024. Rese-cel product design & clinical / translational data 4-1BB costimulatory domain with fully human binder Binder with similar affinity & biologic activity to academic FMC63 binder
while binding to the same epitopes1,2 Same weight-based dose as in academic studies Potential to provide immune reset based on initial clinical and translational data5 Initial patients treated with rese-cel have shown compelling clinical responses
with safety data that supports autoimmune development6 Fully human anti-CD19 binder 4-1BB costimulatory domain CD3- signaling domain Rese-cel4
Broad portfolio with six RESET trials
designed to address high unmet need and realize the potential of rese-cel RESET program addressing several autoimmune markets SLE - Systemic lupus erythematosus; DM - Dermatomyositis; SSc - Systemic sclerosis; gMG -
Generalized myasthenia gravis; MS - multiple sclerosis; ESRD - End-stage renal disease; PV - pemphigus vulgaris SSc gMG PV ~90k ~55k ~15k Middle age onset common Progressive skin & organ fibrosis with lung, cardiac, renal
damage Average survival of 12y Bimodal age of onset Profound weakness that can be disabling Risk for myasthenic crises, with respiratory failure Phase 1/2 Trials Rheum Neuro Derm No Flu/Cy Pure autoantibody & B-cell mediated autoimmune disease
Characterized by painful blisters & erosions SLE ~160-320k Affects young women & people of color ~40% with lupus nephritis, which carries ~25% risk of death or ESRD within 10y > > > Myositis ~70k Typical onset middle age Only
FDA-approved therapy is IVIg in DM High mortality due to lung & cardiac involvement > MS ~750k Chronic inflammation, axon loss, cognitive impairment, and irreversible neurologic damage > ~85k ~150k ~60k ~100k ~550k ~20k U.S. Prevalence EU
Designed to evaluate the safety and
tolerability of rese-cel in subjects with active, refractory disease Key inclusion and exclusion criteria in RESETTM clinical program ASyS, antisynthetase syndrome; CAR, chimeric antigen receptor; DM, dermatomyositis; HSCT, hematopoietic stem
cell transplantation; IIM, idiopathic inflammatory myopathy; JIIM, juvenile idiopathic inflammatory myopathy; LN, lupus nephritis; MAA, myositis-associated antibody; SLEDAI-2k, SLE disease activity index 2000; SSc, systemic sclerosis. 1.
ClinicalTrials.gov. Available at: www.clinicaltrials.gov/study/NCT06121297 (accessed October 2024). 2. ClinicalTrials.gov. Available at: www.clinicaltrials.gov/study/NCT06328777 (accessed October 2024). 3. ClinicalTrials.gov. Available at:
www.clinicaltrials.gov/study/NCT06154252 (accessed October 2024). RESET-SLE Diagnosis of SLE (SLE or LN) Age 18 and 65 Positive ANA or anti-dsDNA at screening SLE (non-renal): active, moderate to severe SLE, SLEDAI-2K 8;
pure class V LN eligible LN: active, biopsy-proven LN class III or IV ( class V) RESET-Myositis Diagnosis of IIM (ASyS, DM, or IMNM) Age 18 and 75 Presence of at least one myositis antibody JIIM: Age 6 and 17
with presence of at least one MSA or MAA Diagnosis of SSc limited or diffuse Age 18 and 70 Evidence of significant skin, pulmonary, renal, or cardiac involvement RESET-SSc Evidence of active disease despite prior or current
treatment with standard of care Key inclusion criteria1-3 Key exclusion criteria1-3 B cell-depleting agent within prior 3-6 months; Previous CAR T therapy and/or HSCT Presence of kidney disease other than LN Current symptoms of severe,
progressive, or uncontrolled pulmonary or cardiac disease Cancer-associated myositis Significant lung or cardiac impairment Severe lung or cardiac impairment Anticipate enrolling and completing dosing in multiple disease-specific cohorts in 2025;
similarly designed RESET-MG Phase 1/2 trial enrolling
Individual trials in myositis, SLE,