Cabaletta Bio, Inc. · NASDAQ:CABA
Cabaletta Bio — Investor Presentation (January 2022)
28 slides from Cabaletta Bio's investor presentation, Jan 10, 2022
Slide 1: Corporate Presentation January 2022
Corporate Presentation JANUARY 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 * 20M, 100M, and 500M refers to the number of DSG3-CAART cells infused for patients in dosing cohorts 1 to 3 (M – millions). As of January 8, 2022. Includes five disclosed product candidates and two undisclosed pipeline disease targets in our pipeline. Developing highly specific C
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 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: Phase 1 clinical trial in mPV evaluating up to 375x dose range
Phase 1 clinical trial in patients with mPV evaluating up to 375x dose range (20M up to 7.5B cells) DesCAARTes™ study of DSG3-CAART 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
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 & timeframes * Clearance of 28-day observation window without DLTs required to initiate next dosing cohort. This information represents data that we believe can be
Slide 14: Dose-dependent increase in DSG3-CAART persistence observed in Cohort A3
Dose-dependent increase in DSG3-CAART persistence observed in Cohort A3 vs. Cohorts A1 and A2 No DLTs observed to date in first 3 cohorts of DesCAARTes™ trial As of January 8, 2022. No clinically meaningful adverse events in any subject to date1, enabling progression to cohort A4 with dose of 2.5B c
Slide 15: Range of strategies for targeted cell therapy in autoimmune diseases
Dose of CAART cells 2 CAART cell expansion post-infusion 3 Range of strategies to consider for targeted cell therapy approaches in patients with autoimmune diseases Optimizing product and patient profiles – many options to consider Many options exist to optimize product and patient profiles Target c
Slide 16: MuSK-CAART for patients with MuSK myasthenia gravis
MuSK-CAART for patients with MuSK myasthenia gravis
Slide 17: High unmet need in MuSK myasthenia gravis; a valuable CAAR target
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 18: 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. Target cells represent a B cell tumor line (CD19 positive) that has been m
Slide 19: PLA2R-CAART for patients with PLA2R-associated membranous nephropathy
PLA2R-CAART for patients with PLA2R-associated membranous nephropathy
Slide 20: 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 21: PLA2R CAARs demonstrated continued activity in presence of anti-PLA2R autoantibodies
PLA2R CAARs demonstrated continued activity in the presence of anti-PLA2R autoantibodies PLA2R-CAART showed in vitro antigen-specific cytotoxicity1 As presented at the ASN Kidney Week 2021. NTD = non transduced T cell control against the same target cells. PLA2R-CAAR Cytotoxicity preserved despite p
Slide 22: Manufacturing
Manufacturing Cabaletta Bio’
Slide 23: 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. Stage 3: Cabaletta Facility Commercialization
Slide 24: Corporate Summary
Corporate Summary Cabaletta Bio’
Slide 25: 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 26: Multiple potential data catalysts with possible pipeline read-through
Multiple potential data catalysts with possible pipeline read-through * 20M, 100M, 500M, 2.5B and 5.0B to 7.5B refers to the number of DSG3-CAART cells infused for patients in dosing cohorts 1 to 5 (M – millions; B – billions). Assumes no dose-limiting toxicities are observed during cohort, uninterr
Slide 27: 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 28: Corporate Presentation JANUARY 2022
Corporate Presentation JANUARY 2022