Cabaletta Bio, Inc. · NASDAQ:CABA
Cabaletta Bio — Investor Presentation (September 2025)
42 slides from Cabaletta Bio's investor presentation, Sep 3, 2025
Slide 1: Corporate Presentation September 2025
Corporate Presentation September 2025
Slide 2: Disclaimer
Disclaimer This presentation, including any printed or electronic copy of these slides, the talks given by the presenters, the information communicated during any delivery of the presentation and any question and answer session and any document distributed at or in connection with the presentation (
Slide 3: Develop and launch the first curative targeted cellular therapies for autoimmune diseases
Develop and launch the first curative targeted cellular therapies for patients with autoimmune diseases
Slide 4: Rese-cel: Delivering on the promise of CD19 targeting for autoimmunity
Over 70 clinical sites recruiting across 6 indications with myositis BLA submission expected in 2027 Rese-cel1: Delivering on the promise of CD19 targeting for autoimmunity SLE – systemic lupus erythematosus; LN – lupus nephritis; SSc – systemic sclerosis; BLA – biologics license application; PV – p
Slide 5: Disease-specific cohorts in RESET program designed to evolve into registrational studies
Disease-specific cohorts in RESET clinical program are designed to evolve directly into registrational studies Innovative clinical strategy to support accelerated regulatory path Program1 Trial Preclinical Phase 1/2 Registrational Rese-cel (CABA-201) 4-1BB CD19-CAR T RESET-Myositis™ CARTA Chimeric A
Slide 6: Rapidly completing enrollment in Phase 1/2 RESET trial cohorts
Enrollment continues in expansion cohorts until pivotal cohorts start enrolling Rapidly completing enrollment in Phase 1/2 RESET™ trial cohorts As compared to companies with actively recruiting U.S. clinical sites for autoimmune cell therapy trials under company-sponsored INDs per clinictrials.gov.
Slide 7: FDA alignment achieved in Type C meeting for rese-cel
FDA alignment achieved in Type C meeting for rese-cel; two open-label, single-arm cohorts to evaluate outcomes FDA aligned on key design elements of myositis registrational cohorts Registrational Cohorts2 ASyS DM & ASyS (n ~15) Initial Phase 1/2 Cohorts1 DM IMNM (n ~6) IMNM (n ~15) (combined n ~6) T
Slide 8: Anticipated rese-cel milestones with 2027 BLA submission planned
Planning to leverage myositis FDA alignment and execution across the portfolio of indications Anticipated Rese-cel Milestones with 2027 BLA Submission Planned 1H25 2H25 1H26 2H26 Align with FDA on registrational cohort designs Present complete Phase 1/2 data Initiate enrollment in registrational coh
Slide 9: Rese-cel: Autologous CD19-CAR T for Autoimmunity
Rese-cel: Autologus CD19-CAR T for Autoimmunity
Slide 10: Rese-cel: CD19-CAR T specifically designed for autoimmunity
Rese-cel binder with similar in vitro & in vivo activity to construct used in academic studies in autoimmunity1,3 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 f
Slide 11: Broad portfolio with six RESET trials addressing high unmet need
Broad portfolio with six RESET trials designed to address high unmet need and realize the potential of rese-cel RESET™ program advancing trials in a broad portfolio of diseases SLE – Systemic lupus erythematosus; DM – Dermatomyositis; SSc – Systemic sclerosis; gMG – Generalized myasthenia gravis; MS
Slide 12: RESET clinical trials have consistent design principles
Individual trials in myositis, SLE, and SSc share common elements of preconditioning, dose, and study design RESETTM clinical trials have consistent design principles1 †Follow up period encompasses 15 years in total, aligned to regulatory guidance for CAR T cell therapies. AE, adverse event; CABA, C
Slide 13: Key inclusion and exclusion criteria in RESET Phase 1/2 trials
Designed to evaluate the safety and tolerability of rese-cel in subjects with active, refractory disease Key inclusion and exclusion criteria in RESETTM Phase 1/2 trials ASyS, antisynthetase syndrome; CAR, chimeric antigen receptor; DM, dermatomyositis; HSCT, hematopoietic stem cell transplantation
Slide 14: Rese-cel expansion and B cell kinetics across indications
Peak rese-cel expansion and transient peripheral B cell depletion occurred within ~2 weeks post infusion Rese-cel expansion & B cell kinetics across indications *LN-1 had prolonged rese-cel detection due to TCR activation that corresponded to longer time to B cell repopulation. LN-4: follow up ong
Slide 15: Myositis: Unmet Need & Clinical Data
Myositis: Unmet Need & Clinical Data
Slide 16: Myositis: High rates of disability & increased risk of mortality
Highly concentrated treatment network in the US Myositis: High rates of disability & increased risk of mortality High disease burden: disability & mortality Typical patient is a middle-aged female who experiences muscle weakness, fatigue, pain, shortness of breath and difficulty swallowing Moderate
Slide 17: Myositis: Limited treatment options for ~80k U.S. patients
IVIg is the only approved therapy (only for patients with the adult dermatomyositis subtype) Myositis: Limited treatment options for ~80k U.S. patients Lundberg, Ingrid E., et al. "Idiopathic inflammatory myopathies." Nature Reviews Disease Primers 7.1 (2021): 86. Analysis from quantitative survey
Slide 18: Baseline characteristics: first 8 patients in RESET-Myositis
All patients had active, refractory disease and most had failed IVIg; an expensive & burdensome B cell therapy Baseline Characteristics: First 8 Patients in RESET-Myositis* *As of May 6, 2025. ^Patient(s) who had clinical data presented in February 2025 scientific meetings. †Baseline disease activit
Slide 19: Incidence of relevant and related serious adverse events
Fever (Grade 1 CRS) in 4 of 8 patients and no ICANS in any patients Incidence of relevant and related serious adverse events* *As of May 6, 2025; primary endpoint is incidence and severity of adverse events through Day 29. ^Patient(s) who had clinical data presented in February 2025 scientific meeti
Slide 20: DM & ASyS: Efficacy data following rese-cel infusion
Clinical responses have been observed off immunomodulators and glucocorticoids in all patients DM & ASyS: Efficacy data following rese-cel infusion* *As of May 6, 2025. †DM-1 Week 32 CK value not available; Week 28 CK value used for TIS calculation. ^Patient(s) who had clinical data presented in Feb
Slide 21: IMNM: Efficacy data following rese-cel infusion
Modest clinical responses have been observed off immunomodulators in 3 of 4 patients IMNM: Efficacy data following rese-cel infusion1* *As of May 6, 2025. ^Patient(s) who had clinical data presented in February 2025 scientific meetings. †Minimal to moderate improvement at all time points from Week 2
Slide 22: Systemic Sclerosis: Unmet Need & Clinical Data
Systemic Sclerosis: Unmet Need & Clinical Data
Slide 23: Systemic sclerosis: Profound unmet need & limited options
Associated with progressive morbidity and high mortality1,2 Systemic sclerosis: Profound unmet need & limited options AHSCT, autologous hematopoietic stem cell transplantation; SSc, systemic sclerosis. 1. Allanore Y, et al. Nat Rev Dis Primers. 2015;1:15002. 2. Denton CP, et al. Lancet. 2017;390(101
Slide 24: Baseline characteristics & serious adverse events in first SSc patients
Baseline characteristics & serious adverse events in first 2 SSc patients* *As of May 6, 2025; primary endpoint is incidence and severity of adverse events through Day 29. ^Patient(s) who had clinical data presented in February 2025 scientific meetings. **Baseline disease activity = activity before
Slide 25: RESET-SSc: Efficacy data following rese-cel infusion
Improvements in both SSc patients after discontinuing immunomodulatory drugs and steroids RESET-SSc™: Efficacy data following rese-cel infusion1 *As of May 6, 2025. †RNA Pol III IgG level performed locally at U Mich. ‡ Pulmonary evaluations not available for SSc-Skin-2 at Week 4. CRISS, Composite Re
Slide 26: Lymph node B cell depletion in SSc-Skin-1
Tissue resident depletion, consistent with the deep B cell depletion in circulation, observed via lymph node biopsy Lymph node B cell depletion in SSc-Skin-11 *Lymph node biopsies were from the left inguinal area using USG at U. Mich. by Dr. Khanna. CAR, chimeric antigen receptor; rese-cel, resecabt
Slide 27: Lupus: Unmet Need & Clinical Data
Lupus: Unmet Need & Clinical Data
Slide 28: SLE & LN: Represent a high unmet clinical need
Increased mortality risk & negative impact on quality of life for patients with SLE & LN SLE & LN: Represent a high unmet clinical need ESRD, end-stage renal disease; LN, lupus nephritis; SLE, systemic lupus erythematosus. 1. Zen M, et al. Eur J Intern Med. 2023;112:45–51. 2. Rahman P, et al. Lupus.
Slide 29: Baseline characteristics: first 8 patients in RESET-SLE
All patients had active, refractory disease and had failed multiple B cell-targeting therapies Baseline characteristics: First 8 patients in RESET-SLE* *As of June 2, 2025. ^Patient(s) who had clinical data presented in February 2025 scientific meetings. †Baseline disease activity = activity before
Slide 30: Incidence of relevant and related serious adverse events
Fever (Grade 1 CRS) reported in 2 of 8 patients & ICANS reported in 1 of 8 patients Incidence of relevant and related serious adverse events* *As of June 2, 2025; Primary endpoint is incidence and severity of adverse events through Day 29. ^Patient(s) who had clinical data presented in February 2025
Slide 31: Non-renal SLE: Efficacy data following rese-cel infusion
As of the latest follow up, 3 of 4 SLE patients§ achieved DORIS Non-renal SLE: Efficacy data following rese-cel infusion* *As of June 2, 2025. ^Patient(s) who had clinical data presented in February 2025 scientific meetings. †Enrollment in the LN cohort requires class III/IV +/- V LN. SLE-1 had pure
Slide 32: LN: Efficacy data following rese-cel infusion
As of the latest follow up 1st LN patient achieved CRR LN: Efficacy data following rese-cel infusion* *As of June 2, 2025. ^Patient(s) who had clinical data presented in February 2025 scientific meetings. ‡SLEDAI-2K components present at latest follow up (SLEDAI-2K contribution score in parenthesis)
Slide 33: Myasthenia Gravis: Unmet Need
Myasthenia Gravis: Unmet Need
Slide 34: Myasthenia gravis: Significant disease & treatment burden
High impact of disease due to patient symptoms & cost burden, particularly for refractory patients Myasthenia gravis: Significant disease & treatment burden Gilhus NE, et al. Eur J Neurol. 2024 2. Dresser L, et al. J Clin Med. May 2021. 3. Results from quantitative survey of U.S. MG-treating physici
Slide 35: Rese-cel Manufacturing & Product/Process Innovations
Rese-cel Manufacturing & Product/Process Innovations
Slide 36: Manufacturing strategy to BLA submission
Manufacturing strategy to BLA submission LVV – lentiviral vector Oxford is a commercial supplier for lentiviral vector utilized in approved CAR T products. Lonza has extensive experience manufacturing commercial cell and gene therapies. Innovative Manufacturing: Clinical Supply: Penn has reliably pr
Slide 37: Several innovations to prioritize patient experience and expand access
Several innovations to prioritize patient experience, expand access and address scale in autoimmune disease Securing & expanding our leadership in autoimmune cell therapy Cohen, Adam D., et al. "B cell maturation antigen–specific CAR T cells are clinically active in multiple myeloma." The Journal of
Slide 38: Corporate Summary
Corporate Summary Cabaletta Bio’
Slide 39: Cabaletta Bio leadership
Track record of operational success evaluating & developing novel cell therapy candidates in autoimmunity Cabaletta Bio leadership LEADERSHIP TEAM Anup Marda Chief Financial Officer Nicolette Sherman Chief HR Officer Michael Gerard General Counsel Steven Nichtberger, M.D. President, CEO & Chairman H
Slide 40: Appendix
Appendix Cabaletta Bio’
Slide 41: TIS is a composite tool measuring relative improvement from baseline
TIS is a composite tool measuring a patient’s relative improvement from their baseline Myositis outcomes captured through validated composite endpoint Major Total Improvement Score 100 80 0 Minimal Moderate 60 40 20 Week 24 Baseline Week 12 None Extramuscular Activity 20 max achievable score Manual
Slide 42: Corporate Presentation September 2025
Corporate Presentation September 2025
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