Cabaletta Bio, Inc. · NASDAQ:CABA
Cabaletta Bio — Investor Presentation (October 2025)
49 slides from Cabaletta Bio's investor presentation, Oct 27, 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: Rese-cel: Delivering on the promise of CD19 for autoimmune patients
Myositis registrational cohort to initiate in 4Q25; enrollment complete in phase 1/2 trials for SLE/LN, SSc & MG Rese-cel1: Delivering on the promise of CD19 for autoimmune patients ASyS – antisynthetase syndrome; BLA – biologics license application; CRR – complete renal response; DM – dermatomyosit
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: FDA alignment achieved in Type C meeting on myositis registrational cohort
FDA alignment achieved in Type C meeting; single-arm evaluation of DM/ASyS sub-types at 16 weeks in a 14-patient cohort FDA aligned on key design elements of myositis registrational cohort Registrational Cohort2 ASyS DM & ASyS (n=14) Initial Phase 1/2 Cohorts1 DM (combined n ~6) TIS, total improveme
Slide 7: Anticipated rese-cel milestones with 2027 BLA submission
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 8: Rese-cel: Autologous CD19-CAR T for Autoimmunity
Rese-cel: Autologus CD19-CAR T for Autoimmunity
Slide 9: 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 BJ, et al. Mol Ther Methods Clin Dev. 2024;32(2):101267. Dai, Zhenyu, et al. "Development and functional characterization
Slide 10: 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 11: 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 12: 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 13: 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* *All data is as of 11 Sep, 2025, except DM-3 which includes Week 24 data as of 08 Oct 2025. **LN-1 had prolonged rese-cel detection due
Slide 14: Myositis: Unmet Need & Clinical Data
Myositis: Unmet Need & Clinical Data
Slide 15: 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 16: 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 17: Baseline characteristics: first 13 patients in RESET-Myositis
All patients had active, refractory disease despite multiple immunomodulatory agents, including IVIg Baseline Characteristics: First 13 Patients in RESET-Myositis* *As of 11 Sep, 2025. †Baseline disease activity = activity before preconditioning. ‡Reflects any exposure to RTX and IVIg prior or at ti
Slide 18: Incidence of relevant and related serious adverse events
Fever (Grade 1 CRS) in 4 of 13 patients and no ICANS in any patients Incidence of relevant and related serious adverse events* *As of 11 Sep, 2025; primary endpoint of the Phase 1/2 study is incidence and severity of adverse events through Day 29. Serious infections and related SAEs are reported to
Slide 19: DM: Efficacy data following rese-cel infusion
3 of 3 patients with DM with sufficient follow-up achieved major TIS responses at Week 16 DM: Efficacy data following rese-cel infusion* *As of 11 Sep, 2025. † Historical NXP-2 autoantibody, but none detected at Pre-preconditioning (Baseline) visit). ‡ At latest follow-up (Day 29). § Insufficient fo
Slide 20: ASyS: Efficacy data following rese-cel infusion
Patient who would meet key inclusion criteria in registrational cohort achieved a major TIS response at Week 16 ASyS: Efficacy data following rese-cel infusion* *As of 11 Sep, 2025. †Reflects trend from baseline to latest timepoint antibody results are available (Week 24 for both patients). In ASyS-
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 9 patients in RESET-SLE
All patients had active, refractory disease and had failed multiple B cell-targeted therapies Baseline characteristics: First 9 patients in RESET-SLE* *As of 11 Sep, 2025. †Baseline disease activity = activity before preconditioning. ‡SLE medications may include biologics, anti-malarials, and immuno
Slide 24: Incidence of relevant and related serious adverse events
Fever (Grade 1 CRS) reported in 3 of 9 patients & ICANS reported in 1 of 9 patients Incidence of relevant and related serious adverse events* *As of 11 Sep, 2025; primary endpoint is incidence and severity of adverse events through Day 29. Serious infections and related SAEs are reported to latest f
Slide 25: Non-renal SLE: Efficacy data following rese-cel infusion
As of the data cut-off, 3 of 4 SLE patients with ≥6 months follow-up achieved DORIS (4th in CRR) Non-renal SLE: Efficacy data following rese-cel infusion* *As of 11 Sep, 2025. †SLE-1 had pure Class V LN and extra-renal SLE disease activity and SLE-5 had Class II LN with moderate-severe chronicity an
Slide 26: LN: Efficacy data following rese-cel infusion
As of the data cut-off, LN-1 in CRR, LN-2 in PRR and LN-3 with histologic response on repeat renal biopsy LN: Efficacy data following rese-cel infusion* *As of 11 Sep, 2025. †DORIS = Clinical SLEDAI-2K=0 (irrespective of serology); Physician Global Assessment <0.5; antimalarials; low-dose GCs (predn
Slide 27: Improvements in SLEDAI-2K and reduction in anti-dsDNA antibodies
Improvements in SLEDAI-2K over time and significant reduction in anti-dsDNA antibodies after discontinuing immunomodulators Efficacy data following rese-cel infusion* *As of 11 Sep, 2025. †Week 20 urinalysis components of the SLEDAI-2K (WBC, RBC and casts) imputed from Week 16 for total SLEDAI-2K sc
Slide 28: Systemic Sclerosis: Unmet Need & Clinical Data
Systemic Sclerosis: Unmet Need & Clinical Data
Slide 29: 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 30: Baseline characteristics: first 6 patients in RESET-SSc
All patients had active, refractory disease and were on 1 to 3 disease-specific therapies at screening Baseline characteristics: First 6 Patients in RESET-SSc* *As of 11 Sep, 2025; primary endpoint is incidence and severity of adverse events through Day 29 †Baseline disease activity = activity befor
Slide 31: Incidence of relevant and related serious adverse events
CRS reported in 3 of 6 patients and ICANS reported in 1 of 6 patients Incidence of relevant and related serious adverse events* *As of 11 Sep, 2025; primary endpoint of the Phase 1/2 study is incidence and severity of adverse events through Day 29. No patient experienced clinical sequelae from CRS,
Slide 32: SSc: Improvements in overall mRSS score
Improvements in overall mRSS score in SSc patients off all immunomodulators and off or tapering steroids SSc: Efficacy data following rese-cel infusion* *As of 11 Sep, 2025. †Assessed at Week 3. mRSS, modified Rodnan Skin Score (measure of skin thickness in SSc across 17 body areas, with a maximum s
Slide 33: SSc: Efficacy data following rese-cel infusion
As of the data cut-off, 4 of 4 SSc patients with at least 12 weeks follow-up achieved rCRISS-25 response SSc: Efficacy data following rese-cel infusion* *As of 11 Sep, 2025; primary endpoint is incidence and severity of adverse events through Day 29. †Reflects trend from baseline to latest available
Slide 34: Lymph node B cell depletion in SSc-Skin-11
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, resecab
Slide 35: Myasthenia Gravis: Unmet Need
Myasthenia Gravis: Unmet Need
Slide 36: 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 37: Rese-cel – Initial Dose Data without Preconditioning
Rese-cel – Initial Dose Data without Preconditioning
Slide 38: 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 39: 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 40: Similar rese-cel expansion and B cell depletion with and without PC
Similar magnitude of rese-cel expansion & B cell depletion kinetics in patients treated with and without PC Similar PK & B cell depletion in rese-cel treated patients without PC* *As of 11 September 2025. Gray vertical dotted line indicates day of rese-cel infusion (study visit Day 1). Gray shading
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: Early clinical activity of rese-cel without preconditioning
Near complete resolution of clinical symptoms and rapid reduction in autoantibodies in 2 of 3 patients Early clinical activity of rese-cel without preconditioning* *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)
Slide 43: Rese-cel Manufacturing
Rese-cel Manufacturing Cabaletta Bio’
Slide 44: 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 45: Corporate Summary
Corporate Summary Cabaletta Bio’
Slide 46: 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 47: Appendix
Appendix Cabaletta Bio’
Slide 48: TIS is a composite tool measuring patient 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 49: Corporate Presentation October 2025
Corporate Presentation October 2025