Cabaletta Bio, Inc. · NASDAQ:CABA
Cabaletta Bio — Investor Presentation (October 2025)
51 slides from Cabaletta Bio's investor presentation, Oct 9, 2025
Slide 1: Corporate Presentation October 2025
Corporate Presentation October 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: Delivering on the promise of CD19 for autoimmune patients
Over 70 clinical sites recruiting across 6 indications with myositis BLA submission expected in 2027 Rese-cel1: Delivering on the promise of CD19 for autoimmune patients SLE – systemic lupus erythematosus; LN – lupus nephritis; SSc – systemic sclerosis; BLA – biologics license application; PV – pemp
Slide 5: Disease-specific cohorts 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: Enrollment continues in expansion cohorts until pivotal cohorts start enrolling
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, SSc & MG 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,
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 and increased risk of mortality
Highly concentrated treatment network in the US; dermatomyositis represents ~75% of this market 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, shortne
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: Lupus: Unmet Need & Clinical Data
Lupus: Unmet Need & Clinical Data
Slide 22: 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 23: Baseline characteristics: first 8 patients in RESET-SLE
All patients had active, refractory disease and had failed multiple B cell-targeted 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 p
Slide 24: 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 25: Non-renal SLE: Efficacy data following rese-cel infusion
As of the data cut-off, 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 cla
Slide 26: LN: Efficacy data following rese-cel infusion
As of the data cut-off, 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): L
Slide 27: Systemic Sclerosis: Unmet Need & Clinical Data
Systemic Sclerosis: Unmet Need & Clinical Data
Slide 28: Systemic sclerosis: Profound unmet need and 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 29: Baseline characteristics and 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 30: 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 31: 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 32: Myasthenia Gravis: Unmet Need
Myasthenia Gravis: Unmet Need
Slide 33: Myasthenia gravis: Significant disease and 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 34: Rese-cel – Initial Dose Data without Preconditioning
Rese-cel – Initial Dose Data without Preconditioning
Slide 35: Evidence of biologic and clinical activity in all three PV patients
Clear evidence of biologic and clinical activity in all three PV patients in the initial dose cohort PDAI improvements were present in all three and were compelling in two of the three patients All patients remain off all immunomodulators while GCs are being tapered from low doses Complete B cell de
Slide 36: RESET-PV Phase 1/2 trial: key inclusion and exclusion criteria
Designed to evaluate the safety and tolerability of rese-cel in PV subjects with active, refractory disease RESET-PV™ phase 1/2 trial: key inclusion & exclusion criteria1 As of 11 September 2025. DSG3, desmoglein 3; PV, pemphigus vulgaris; RESET™, REstoring SElf-Tolerance. Cabaletta Bio: Data on fil
Slide 37: Baseline characteristics of first 3 patients in RESET-PV
All pts had moderate to severe, active, refractory disease & failed B cell-targeting therapies, including RTX Baseline characteristics of first 3 patients in RESET-PV™ As of 11 September 2025. Cabaletta Bio: Data on file. 1M, 1 million CAR T cells/kg; DSG1, desmoglein 1; DSG3, desmoglein 3; GC, gluc
Slide 38: Similar rese-cel expansion in PC and non-PC cohorts
Similar rese-cel expansion observed in PC and non-PC cohorts in the absence of elevated serum IL-15 Peak expansion occurred slightly later in patients without PC As of 11 September 2025. Cabaletta Bio: Data on file. Gray vertical dotted line indicates day of rese-cel infusion (study visit Day 1). Gr
Slide 39: IFN-γ induction occurred later in PV subjects without PC
IFN-γ induction occurred ~ 1 week later in PV subjects without PC than in rese-cel patients with PC IFN-γ induction observed ~ 2 weeks after infusion As of 11 September 2025. Cabaletta Bio: Data on file. Gray vertical dotted line indicates day of rese-cel infusion (study visit Day 1). Gray vertical
Slide 40: B cell depletion and serum BAFF induction observed in all subjects
PV-1M-1 had an ~ 84% reduction, PV-1M-2 & PV-1M-3 had 100% reduction of B cells at initial rese-cel dose B cell depletion & serum BAFF induction observed in all subjects As of 11 September 2025. Cabaletta Bio: data on file. Gray shading in BAFF plot is range of median serum BAFF induction observed i
Slide 41: Incidence of relevant and related serious adverse events
Incidence of relevant and related serious adverse events* *As of 11 September 2025. Cabaletta Bio: Data on file. Primary endpoint is incidence and severity of adverse events through Day 29. **Graded per ASTCT Consensus Grading Criteria. ‡Coded in System Organ Class of Infections and Infestations and
Slide 42: Patient 1: Early efficacy data following rese-cel infusion without PC
Patient 1: Early efficacy data following rese-cel infusion without PC* *As of 11 September 2025. Cabaletta Bio: Data on file. Disease severity intervals as defined Krain RL, et al. Br J Dermatol. 2021;184(5): 975–977. Gray vertical dotted line indicates day of rese-cel infusion (study visit Day 1).
Slide 43: Patient 2: Early efficacy data following rese-cel infusion without PC
Patient 2: Early efficacy data following rese-cel infusion without PC* *As of 11 September 2025. Cabaletta Bio: Data on file. Disease severity intervals as defined in Krain RL, et al. Br J Dermatol. 2021;184(5): 975–977. Gray vertical dotted line indicates day of rese-cel infusion (study visit Day 1
Slide 44: Patient 3: Early efficacy data following rese-cel infusion without PC
Patient 3: Early efficacy data following rese-cel infusion without PC* *As of 11 September 2025. Cabaletta Bio: Data on file. Disease severity intervals as defined in Krain RL, et al. Br J Dermatol. 2021;184(5): 975–977. Gray vertical dotted line indicates day of rese-cel infusion (study visit Day 1
Slide 45: Rese-cel Manufacturing
Rese-cel Manufacturing Cabaletta Bio’
Slide 46: 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 47: Corporate Summary
Corporate Summary Cabaletta Bio’
Slide 48: Track record of operational success in autoimmunity cell therapy
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 49: Appendix
Appendix Cabaletta Bio’
Slide 50: TIS is a composite tool measuring 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 51: Corporate Presentation October 2025
Corporate Presentation October 2025