Cabaletta Bio, Inc. · NASDAQ:CABA
Cabaletta Bio — Investor Presentation (August 2022)
28 slides from Cabaletta Bio's investor presentation, Aug 11, 2022
Slide 1: Corporate Presentation August 2022
Corporate Presentation AUGUST 2022 Exhibit 99.2
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; ICANS – Immune effector cell-associated neurotoxicity syndrome; SAEs – Serious adverse events Mueller, Karen Thudium, et al. "Cellular kinetics of CTL019 in relapsed/refractory B-cell acute lymphoblastic leukemia and chronic lymphocytic leukemia."
Slide 5: 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 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 enabling 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 includes multiple disease targets where cure is possible
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: Trial in patients with mPV evaluating up to 750x dose range
Trial in patients with mPV evaluating up to 750x dose range (20M up to 10-15B cells) DesCAARTes™ Phase 1 study of DSG3-CAART1 FDA has requested, and the Company has agreed, that we will share data from part A to inform a discussion on the optimal design of part C. According to FDA advice, the submis
Slide 13: Safety assessed acutely, at days and at months, with biologic activity data
Safety assessed acutely, at 28 days & at 3 months, with data on biologic activity within 6 months** DesCAARTes™ clinical trial assessments & current timeframes * Clearance of 28-day observation window without DLTs required to initiate next dosing cohort. DLTs include any grade 3 or 4 CRS or neurotox
Slide 14: Favorable safety profile at all reported doses of DSG3-CAART
Favorable safety profile at all reported doses of DSG3-CAART, including the 2.5B cell dose cohort No DLTs observed to date in first 4 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 t
Slide 15: DSG3-CAART persistence in cohort A4 approached levels seen in CART-19
DSG3-CAART persistence in cohort A4 approached levels seen in CART-19 with lymphodepletion in oncology Dose-dependent persistence of DSG3-CAART in cohorts A1 to A4 Mueller, Karen Thudium, et al. "Cellular kinetics of CTL019 in relapsed/refractory B-cell acute lymphoblastic leukemia and chronic lymph
Slide 16: 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 17: MuSK-CAART for patients with MuSK myasthenia gravis
MuSK-CAART for patients with MuSK myasthenia gravis
Slide 18: All known extracellular domains can be included in the CAAR design
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 19: 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 20: Strategy for upcoming MuSK-CAART trial informed by DesCAARTes study
Strategy for upcoming trial evaluating MuSK-CAART informed by progression of DesCAARTes™ study MusCAARTes™ study of MuSK-CAART * 100M, 500M, 2.5B and 5.0B to 7.5B refers to the number of MuSK-CAART cells infused for patients in dosing cohorts A1 to A4 (M – millions; B – billions). A total of 6 subje
Slide 21: PLA2R-CAART for patients with PLA2R-associated membranous nephropathy
PLA2R-CAART for patients with PLA2R-associated membranous nephropathy
Slide 22: Primary MN is an immune-mediated kidney disease with anti-PLA2R antibodies
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 23: Manufacturing
Manufacturing Cabaletta Bio’
Slide 24: 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 25: Corporate Summary
Corporate Summary Cabaletta Bio’
Slide 26: 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 27: Expanding network of partners; multiple potential data catalysts
Expanding network of academic & industry partners Multiple potential data catalysts with possible pipeline read-through Mueller, Karen Thudium, et al. "Cellular kinetics of CTL019 in relapsed/refractory B-cell acute lymphoblastic leukemia and chronic lymphocytic leukemia." Blood, The Journal of the
Slide 28: Corporate Presentation AUGUST 2022
Corporate Presentation AUGUST 2022