Slide 1: CABA-201 Initial Clinical Data from the RESET-Myositis & RESET-SLE Phase 1/2 Trials
CABA-201 Initial Clinical Data from the RESET-Myositis™ & RESET-SLE™ Phase 1/2 Trials JUNE 2024 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: Today's Agenda
Today’s Agenda AGENDA TOPIC SPEAKER CABA-201 Overview Steven Nichtberger, MD Chief Executive Officer Current & Investigational Treatments for Patients with Autoimmune Disease Iain McInnes, MD, FRCP, PhD, FRSE, FMedSci Vice Principal and Head of the College of Medical, Veterinary and Life Sciences, M
Slide 4: CABA-201: CD19-CAR T specifically designed for autoimmunity
4 Designed to replicate and expand on the academic clinical data that generated interest in the field CABA-201: CD19-CAR T specifically designed for autoimmunity PK, pharmacokinetics; PD, pharmacodynamics, SAEs: serious adverse events Peng, BinghaoJ, et al. Presented at: American Society Gene and C
Slide 5: CABA-201 pipeline targeting a broad range of autoimmune diseases
Innovative and scalable clinical strategy with potential for accelerated development path CABA-201 pipeline targeting a broad range of autoimmune diseases RESET™ – REstoring SElf-Tolerance; IMNM – Immune-mediated necrotizing myopathy; SLE – Systemic lupus erythematosus; Ab – Antibody; AChR – Acetylc
Slide 6: Acceleration in enrollment anticipated in 2H24
Acceleration in enrollment anticipated in 2H24 with initial CABA-201 data & engaged clinical investigators Sites actively recruiting patients in the RESETTM clinical program1 5 patients enrolled across RESET-SLETM & RESET-MyositisTM, with 3 patients enrolled over the last 2 months 18 actively recrui
Slide 7: Current & Investigational Treatments for Patients with Autoimmune Diseases
Current & Investigational Treatments for Patients with Autoimmune Diseases
Slide 8: High unmet clinical need in SLE & myositis
Broad immunosuppression and chronic administration often required to achieve partial, transient responses Current therapies for autoimmunity do not achieve drug-free remission (Hematopoietic stem cell transplant has been shown to be curative in systemic sclerosis but has increased mortality in the f
Slide 9: Potential for treatment paradigm to evolve in autoimmunity
Potential for treatment paradigm to evolve in autoimmunity CAR T therapy has the potential to provide drug-free, durable & reliable responses Promising clinical responses reported in 15 patients with an academic 4-1BB CD19-CAR T1-3 100% Clinical responses in SLE, myositis, SSc off immunosuppressive
Slide 10: Initial CABA-201 Data in Myositis & Lupus
Initial CABA-201 Data in Myositis & Lupus
Slide 11: Phase 1/2 Myositis Study for CABA-201
Phase 1/2 Myositis Study for CABA-201 CY, cyclophosphamide; EULAR/ACR, European Alliance of Associations for Rheumatology/America College of Rheumatology; FLU, fludarabine; HSCT, hematopoietic stem cell transplantation. TIS, Total Improvement Score. Juvenile idiopathic inflammatory myopathy (JIIM, j
Slide 12: Phase 1/2 Lupus Study for CABA-201
Phase 1/2 Lupus Study for CABA-201 ANA, antinuclear antibody; SLEDAI-2K, Safety of Estrogens in Lupus Erythematosus National Assessment Systemic Lupus Erythematosus Disease Activity Index-2K. Key inclusion criteria Age ≥18 to ≤65 with an SLE diagnosis (2019 EULAR/ACR classification criteria) ANA+ or
Slide 13: Metrics to assess outcomes of B cell depletion in autoimmunity
Time to B cell repopulation B cell phenotype3 Autoantibody changes Durability of clinical activity Rate & severity of infection Chronic maintenance / concomitant medications, if any Up to 12+ months Translational & clinical parameters inform framework to evaluate advanced modalities in autoimmunity
Slide 14: Baseline characteristics of first two patients in RESET trials
Both patients with refractory disease, including to B cell-targeting antibodies & other agents Baseline characteristics of first two patients in RESET™ trials dsDNA, double-stranded DNA; IMNM, immune-mediated necrotizing myopathy; MMF, mycophenolate mofetil; MMT-8, manual muscle testing of 8 muscles
Slide 15: CABA-201 was well-tolerated in initial patients
No CRS, ICANS or infections of any grade reported through follow-up period1 CABA-201 was well-tolerated in initial patients Data cut-off as of 28 May 2024. Protocol requires a minimum of 4-day hospitalization for monitoring. PI-directed taper from 10mg daily prednisone. Grade 4 leukopenia, neutropen
Slide 16: CABA-201 exhibited anticipated profile of expansion and contraction
CABA-201 exhibited anticipated profile of expansion and contraction1 CABA-201 demonstrated expansion & targeted B cell depletion Response appears to be consistent with published data of cryopreserved CAR T products as well as the expansion profile of BCMA-CAR T products in patients with multiple mye
Slide 17: Complete B cell depletion achieved and maintained
Complete B cell depletion achieved by day 15 on flow cytometry & maintained in context of WBC recovery Systemic B cell depletion with CABA-201 WBC, white blood cell. 1. Nadir of lymphocyte count following fludarabine and cyclophosphamide administration estimated based on respective product labels.2,
Slide 18: Antibody reduction & clinical improvement in disease activity
Antibody reduction & clinical improvement in disease activity as anticipated with follow-up of 12 weeks1 CK reduction & clinical improvement observed in SRP IMNM Data cut-off as of May 28, 2024. Luminex assay developed and performed by Cabaletta Labs. Qualitative commercial assay (Myositis Antigen P
Slide 19: Early efficacy signals in first patient in non-renal SLE cohort
0 0 Month 3 Month 6 SLEDAI-2K Score 25 20 30 10 5 15 Academic published data for illustrative purposes 19 Trend toward improvement in disease manifestations with follow up of 4 weeks2 Early efficacy signals in first patient in non-renal SLE cohort1 Patient in non-renal SLE cohort due to isolated Cla
Slide 20: IMNM patient data provides insights supporting tissue-level effects of CAR T
BAFF B cells Preconditioning CABA-201 infusion IMNM patient data provides insights supporting tissue-level effects of CAR T B cell repopulation occurred at 2 months in first IMNM patient Data cut-off as of May 28, 2024. IFNγ peak prior to peripheral CABA-201 peak suggests tissue-resident B cell cyto
Slide 21: Initial patient phenotyping data consistent with potential immune system reset
Initial patient phenotyping data consistent with potential immune system reset; confirmatory analyses ongoing B cell repopulation with naïve B cells BCR, B cell receptor; Note: Flow plot gating reflects CD19+ CD20+ live lymphocytes. 1. Patient received multiple courses of rituximab, with most recent
Slide 22: Conclusions
Conclusions Cabaletta Bio”
Slide 23: Key takeaways from initial CABA-201 data in first two patients
Key takeaways from initial CABA-201 data in first two patients1 Data cut-off as of 28 May 2024. Müller F, et al. N Engl J Med. 2024;390(8):687-700. Third-party CASTLE Phase I/II basket study. As of June 12, 2024. 18 clinical sites now enrolling patients in the CABA-201 RESET™ program across four tri
Slide 24: Realizing the vision to transform autoimmune disease treatment
24 Realizing the vision to transform autoimmune disease treatment 1H24: No reported CRS or ICANS in first myositis or SLE patients2 Clinical update on each patient at EULAR symposium2 Evaluating CABA-201 without preconditioning in PV study Advancing program to potentially eliminate apheresis Securin
Slide 25: Q&A
Q&A Cabaletta Bio”