Cabaletta Bio, Inc. · NASDAQ:CABA
Cabaletta Bio — Investor Presentation (September 2022)
30 slides from Cabaletta Bio's investor presentation, Sep 12, 2022
Slide 1: Corporate Presentation September 2022
Corporate Presentation SEPTEMBER 2022 Exhibit 99.1
Slide 2: Disclaimer
Disclaimer The following 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 or material distributed at or in connection wi
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: Cabaletta overview
Cabaletta® overview CRS – Cytokine release syndrome; SAEs – Serious adverse events One subject in cohort A5 developed Grade 1 CRS several hours after each of the 2 infusions which resolved within 2 days (related SAEs). The events were not considered to be DLTs and did not delay study progression Pre
Slide 5: Our scientific platform leverages clinically validated CAR T technology
Our scientific platform leverages clinically validated CAR T technology CAR T Therapy Chimeric Antigen Receptor T cell Kymriah CAAR T product candidates are designed for selective and specific elimination of the pathogenic B cells CAAR T Therapy Chimeric AutoAntibody Receptor T cell CABA CAAR T CD13
Slide 6: Foundational CAR T technology clinically validated in B cell-mediated cancers
Foundational CAR T technology clinically validated in treating B cell-mediated cancers Cabaletta: Advancing targeted cell therapy to autoimmunity Marketed Pivotal Clinical Preclinical / Discovery Oncology B Cell-Mediated Allo/Autoimmune Diseases CAR-T / CAAR-T T-reg, NK, TCR, RBC, macrophage, TIL (7
Slide 7: CABA platform designed to enable a portfolio of programs targeting B cell-mediated diseases
CABA™ platform designed to enable a portfolio of programs targeting B cell-mediated diseases Modular platform with “plug-and-play” architecture Clinically validated engineered T cell platform is the foundational technology PLA2R-CAART PLA2R+ membranous nephropathy DSG3-CAART Mucosal pemphigus vulgar
Slide 8: Pipeline by therapeutic area and indication
Therapeutic Area Indication Program Discovery2 Preclinical Phase 1 Phase 2/3 Dermatology Mucosal Pemphigus Vulgaris DSG3-CAART Mucocutaneous Pemphigus Vulgaris DSG3/1-CAART Neurology MuSK Myasthenia Gravis MuSK-CAART Nephrology PLA2R Membranous Nephropathy PLA2R-CAART Hematology Hemophilia A w/ FVII
Slide 9: DSG3-CAART for patients with mucosal pemphigus vulgaris
DSG3-CAART for patients with mucosal pemphigus vulgaris
Slide 10: Current treatments require broad immunosuppression with safety risks and transient efficacy
Current treatments require broad immunosuppression associated with safety risks and transient efficacy Overview of pemphigus vulgaris Image credit: D@nderm. http://www.vgrd.org/archive/cases/2004/pv/DSCN4996%20copy.JPG Joly, Pascal, et al. "First-line rituximab combined with short-term prednisone ve
Slide 11: Inclusion of all disease-relevant epitopes enables a one-size-fits-all approach for mPV
Inclusion of all disease-relevant epitopes enables a ‘one-size-fits-all’ approach for patients with mPV DSG3-CAART is designed to bind all known pathogenic autoantibodies Ohyama, Bungo, et al. "Epitope spreading is rarely found in pemphigus vulgaris by large-scale longitudinal study using desmoglein
Slide 12: DesCAARTes Phase 1 study of DSG3-CAART
White blood cells (including T cells) are collected T cells lentivirally transduced with DSG3 CAAR DSG3 CAAR T cell expansion DSG3 CAAR T cells are infused into the patient Adjunctive immunosuppressants are stopped; prednisone tapered to low dose prior to infusion Evaluating DSG3-CAART as monotherap
Slide 13: Safety assessed acutely, at days and at months, with biologic activity data within 6 months
Safety assessed acutely, at 28 days & at 3 months, with data on biologic activity within 6 months** DesCAARTes™ clinical trial assessments & current timeframes * For the combination cohort, patients will be pre-treated with IVIG and cyclophosphamide prior to DSG3-CAART infusion. ** Clearance of 28-d
Slide 14: Favorable safety profile at all reported doses of DSG3-CAART
Favorable safety profile at all reported doses of DSG3-CAART, including up to 7.5B cell dose cohort No DLTs observed in first 5 cohorts of DesCAARTes™ trial * 20M, 100M, 500M, 2.5B, 5.0B to 7.5B and 10B to 15B refers to the number of DSG3-CAART cells infused for patients in dosing cohorts A1 to A6m
Slide 15: Dose dependent persistence through cohort A4, leveling off with cohort A5
Dose dependent persistence observed through cohort A4, but leveling off with cohort A5 DSG3-CAART persistence through day 29 in cohorts A1 to A5 Mueller, Karen Thudium, et al. "Cellular kinetics of CTL019 in relapsed/refractory B-cell acute lymphoblastic leukemia and chronic lymphocytic leukemia." B
Slide 16: Anti-DSG3 antibody levels across cohorts A1-A4
Anti-DSG3 antibody levels across cohorts A1-A4 Antibody levels as measured via ELISA generally stable, though patient A4-2 had transient response Cohort A1 (2x107) Cohort A2 (1x108) Cohort A3 (5x108) Cohort A4 (2.5x109) Cohort A1 (2x107) Cohort A2 (1x108) Cohort A3 (5x108) Cohort A4 (2.5x109) * * Da
Slide 17: Transient improvement in disease activity and steroid taper
Transient improvement in disease activity by 3 months & steroid taper during that period Data on Subject A4-2 (2.5x109 DSG3-CAART Dose) Oral Disease Severity Score 1 DSG3-CAART persistence detected at 3 months (not observed in other subjects in Cohorts A1-A4) Anti-DSG3 antibody levels decreased >20%
Slide 18: Combo sub-study prioritized to potentially overcome cytokine and autoantibody effects
Combo sub-study prioritized to potentially overcome possible cytokine & autoantibody effects on CAART activity Strategy and rationale for planned cohorts in DesCAARTes™ trial Amagai, Masayuki, et al. "A randomized double-blind trial of intravenous immunoglobulin for pemphigus." Journal of the Americ
Slide 19: Strategies to increase in vivo activity of DSG3-CAART
Range of strategies being implemented and/or under consideration to increase in vivo activity of DSG3-CAART Optimizing DSG3-CAART product and patient profiles Many options exist to optimize product and patient profiles; ongoing evaluation to enhance DSG3-CAART exposure in DesCAARTes™ trial Healthy B
Slide 20: MuSK-CAART for patients with MuSK myasthenia gravis
MuSK-CAART for patients with MuSK myasthenia gravis
Slide 21: High unmet need in MuSK myasthenia gravis; all known extracellular domains can be included
6.0 - 7.5% All known extracellular domains can be included in the CAAR design High unmet need in MuSK myasthenia gravis; a valuable CAAR target Hain, Berit, et al. "Successful treatment of MuSK antibody–positive myasthenia gravis with rituximab." Muscle & Nerve: Official Journal of the American Asso
Slide 22: MuSK-CAART eliminated anti-MuSK target cells in an animal model
MuSK-CAART eliminated anti-MuSK target cells2 in an animal model where CART19 cells were a positive control MuSK-CAART demonstrated specific in vivo target engagement1 https://cabalettabio.com/technology/posters-publications; recently presented at AAI Immunology 2022, MGFA International and ASGCT 20
Slide 23: Strategy for upcoming MusCAARTes trial of MuSK-CAART
Strategy for upcoming trial evaluating MuSK-CAART informed by learnings from DesCAARTes™ study MusCAARTes™ study of MuSK-CAART * 500M, 2.5B, 7.5B refers to the number of MuSK-CAART cells infused for patients in dosing cohorts A1 to A3 (M – millions; B – billions). A total of 6 subjects will need to
Slide 24: PLA2R-CAART for patients with PLA2R-associated membranous nephropathy
PLA2R-CAART for patients with PLA2R-associated membranous nephropathy
Slide 25: Primary MN is an immune-mediated kidney disease with anti-PLA2R antibodies in ~75% of patients
Primary MN is an immune-mediated kidney disease with anti-PLA2R antibodies in ~75% of patients Preclinical stage CAART program in membranous nephropathy Accumulating evidence of a correlation between PLA2R antibodies and disease activity, remission and relapse in primary MN PLA2R autoantibody levels
Slide 26: Corporate Summary
Corporate Summary Cabaletta Bio’
Slide 27: Cabaletta Bio leadership
BOARD OF DIRECTORS Cabaletta Bio leadership LEADERSHIP TEAM Anup Marda Chief Financial Officer Arun Das, M.D. Chief Business Officer David J. Chang, M.D., M.P.H. Chief Medical Officer Martha O’Connor Chief HR Officer Michael Gerard General Counsel Steven Nichtberger, M.D. President, CEO & Chairman A
Slide 28: Three-stage manufacturing approach allows efficient allocation of capital
Three-stage approach allows for efficient allocation of capital while leveraging experienced partners Manufacturing strategy Penn-sponsored CD19 CAR IND CMC data has been cross-referenced in DSG3-CAART IND to provide additional data reflecting alignment; cross reference not required for MuSK-CAART I
Slide 29: Multiple potential data catalysts with possible pipeline read-through
Multiple potential data catalysts with possible pipeline read-through Assumes no dose-limiting toxicities are observed in the cohort, uninterrupted enrollment and no delays due to COVID-19 resurgence occur in the trial. Matthews, Ian, et al. "Muscle-specific receptor tyrosine kinase autoantibodies—a
Slide 30: Corporate Presentation SEPTEMBER 2022
Corporate Presentation SEPTEMBER 2022
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