Cabaletta Bio, Inc. · NASDAQ:CABA
Cabaletta Bio — Investor Presentation (February 2025)
27 slides from Cabaletta Bio's investor presentation, Feb 18, 2025
Slide 1: Corporate Presentation February 2025
Corporate Presentation FEBRUary 2025 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: Leveraging increasing clinical data and unique development program for rese-cel
Planning to leverage increasing clinical data and unique development program for rese-cel1 2025: Pursuing an efficient path to approval CRS, cytokine release syndrome; DM – dermatomyositis; DORIS – definition of remission in SLE; ICANS, immune effector cell-associated neurotoxicity syndrome; LN – lu
Slide 5: RESET clinical program has disease-specific cohorts designed to evolve into registrational studies
RESET clinical program has disease-specific cohorts designed to evolve directly into registrational studies Innovative clinical strategy with potential for accelerated regulatory path Program1 Trial Preclinical Phase 1/2 Pivotal Rese-cel (CABA-201) 4-1BB CD19-CAR T RESET-Myositis™ CARTA Chimeric Ant
Slide 6: Chimeric Antigen Receptor T Cells for Autoimmunity (rese-cel)
Chimeric Antigen Receptor T Cells for Autoimmunity (rese-cel)
Slide 7: ~150 autoimmune patients have been dosed with autologous CAR T constructs
~150 autoimmune patients have been dosed with a range of autologous CAR T constructs (industry & academia)1 Autologous CAR T is potentially transformational for autoimmunity IIM – idiopathic inflammatory myopathy; MG – Myasthenia gravis; MS – Multiple sclerosis; SPS – Stiff person syndrome; SSc – Sy
Slide 8: Rese-cel: CD19-CAR T specifically designed for autoimmunity
Cabaletta rese-cel binder with similar in vitro & in vivo activity to construct used in academic studies1,3 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 for tre
Slide 9: 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 addressing several autoimmune markets SLE – Systemic lupus erythematosus; DM – Dermatomyositis; SSc – Systemic sclerosis; gMG – Generalized myasthenia gravis; MS – multiple
Slide 10: Rese-cel Clinical & Translational Data
Rese-cel Clinical & Translational Data
Slide 11: Key inclusion and exclusion criteria in RESET clinical program
Designed to evaluate the safety and tolerability of rese-cel in subjects with active, refractory disease Key inclusion and exclusion criteria in RESETTM clinical program ASyS, antisynthetase syndrome; CAR, chimeric antigen receptor; DM, dermatomyositis; HSCT, hematopoietic stem cell transplantation
Slide 12: RESET clinical trials have consistent design principles
Individual trials in myositis, SLE, and SSc 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, C
Slide 13: Baseline characteristics of first 10 patients in the RESET program
All patients had active, refractory disease and most had failed B cell-targeting therapies Baseline characteristics of first 10 patients in the RESET™ program *Baseline disease activity = activity before preconditioning. #Prednisone/prednisone equivalent dose †SLE-1 had class V LN; inclusion criteri
Slide 14: B cell repopulation with transitional naïve cells started as early as 2 months
B cell repopulation with transitional naïve cells started as early as 2 months post-infusion Consistent and deep B cell depletion by Day 221 PK, pharmacokinetic; PD, pharmacodynamic * Pre-infusion B-cell levels were measured at pre-preconditioning for all subjects other than IMNM-2 where apheresis w
Slide 15: Tissue resident depletion observed via lymph node biopsy
Tissue resident depletion, consistent with the deep B cell depletion in circulation, observed via lymph node biopsy Lymph node B cell depletion 21 days post infusion in first SSc patient LN biopsies were from the left inguinal area using USG at U. Michigan by Dr. Khanna All Images are 20X magnificat
Slide 16: Incidence and severity of adverse events in the first patients
Incidence and severity of adverse events in the first 10 patients* *As of Jan 8, 2025; Primary endpoint is incidence and severity of adverse events through Day 29. †Graded per ASTCT Consensus Grading Criteria. Of these patients, DM-1, SLE-2, SLE-3, SLE-4, LN-2, and SSc-Skin-1 received medication for
Slide 17: First adult DM patient dosed with CAR T demonstrated early response off immunosuppressants
1st known adult DM patient dosed with CAR T demonstrated compelling early response off immunosuppressants* RESET-Myositis™: Efficacy data following rese-cel infusion *As of Jan 8, 2025. CDASI-A, Cutaneous Dermatomyositis Disease Area and Severity Index – Activity; CK, creatinine kinase; HCQ, hydroxy
Slide 18: First IMNM patient demonstrated continuing clinical response off immunosuppressants
1st IMNM patient with longer follow up demonstrated continuing clinical response off immunosuppressants* RESET-Myositis™: Efficacy data following rese-cel infusion1 *As of Jan 8, 2025. †IMNM-1 Day 29 CK measurement was unavailable; Day 22 used. ‡IMNM-2 developed a PE at Day 38 with a prolonged hospi
Slide 19: SLE patients achieved DORIS remission and first LN patient achieved CRR
3 out of 4 SLE patients have achieved DORIS remission and 1st LN patient achieved CRR off all IS and steroids RESET-SLE™: Efficacy data following rese-cel infusion* *As of Jan 8, 2025 † Enrollment in the LN cohort requires class III/IV +/- V LN. SLE-1 had isolated class V LN and extra-renal SLE dise
Slide 20: Improvements in skin and lung function after discontinuing immunosuppressants
Improvements in skin across multiple body areas and in lung function, after discontinuing immunosuppressants* RESET-SSc™: efficacy data in first SSc patient post rese-cel infusion1 *As of Jan 8, 2025 patient is not taking immunosuppressants or steroids. DLCO, % predicted diffusing capacity for carbo
Slide 21: Rese-cel Product/Process Innovations & Indication Expansion
Rese-cel Product/Process Innovations & Indication Expansion
Slide 22: Several innovations to enhance patient experience and expand access
Several innovations to enhance patient experience, expand access and address scale in autoimmune disease Securing & expanding our leadership in autoimmune cell therapy Cohen, Adam D., et al. "B cell maturation antigen–specific CAR T cells are clinically active in multiple myeloma." The Journal of Cl
Slide 23: Staged approach allows efficient capital allocation while leveraging experienced partners
Staged approach allows for efficient allocation of capital while leveraging experienced partners Manufacturing strategy – securing reliable supply then innovating Innovative Manufacturing: Scale-Up, Reduce COGs & Improve Patient Experience Clinical & Commercial Supply: Penn, CDMOs & CABA Process Cli
Slide 24: Corporate Summary
Corporate Summary Cabaletta Bio”
Slide 25: 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 26: Cabaletta's Anticipated Key Milestones for 2025
Cabaletta’s Anticipated Key Milestones for 2025 Present rese-cel clinical & translational data at medical and scientific meetings 2025 Enroll and complete dosing in multiple disease-specific RESETTM cohorts 2025 Present clinical data evaluating rese-cel with no preconditioning from the RESET-PVTM tr
Slide 27: Corporate Presentation february 2025
Corporate Presentation february 2025
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