Cabaletta Bio, Inc. · NASDAQ:CABA
Cabaletta Bio — Corporate Update (November 2024)
28 slides from Cabaletta Bio's corporate update, Nov 18, 2024
Slide 1: CABA-201 Clinical and Translational Data from the RESET Phase 1/2 Trials
Exhibit 99.1 CABA-201 Clinical and Translational Data from the RESET Phase 1/2 Trials NOVEMBER 2024 © 2024 Cabaletta Bio. All rights reserved.
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 Steven Nichtberger, MD CABA-201 Overview Chief Executive Officer Lessons from Oncology: Expanding CAR T Carl H. June, MD Director of the Center for Cellular Immunotherapies, Penn Medicine Cell Therapies into Autoimmunity CABA-201 Clinical and Translational Data Da
Slide 4: CABA-201: CD19-CAR T specifically designed for autoimmunity
CABA-201: CD19-CAR T specifically designed for autoimmunity 1,3 Cabaletta’s CD19 binder with similar in vitro & in vivo activity to construct used in academic studies Clinical data reported by IASO using 4 licensed CD19 binder in oncology Fully human anti-CD19 binder Similar binding affinity & biolo
Slide 5: RESET clinical program for CABA-201, a CD19-directed CAR T
RESET clinical program for CABA-201, a CD19-directed CAR T Multiple autoimmune diseases evaluated in distinct disease cohorts across five clinical trials Phase 1/2 Pivotal Program Trial Preclinical Dermatomyositis Rheumatology Anti-synthetase syndrome Neurology RESET-Myositis IMNM Dermatology Contai
Slide 6: Expanding clinical site footprint across RESET program
1 Expanding clinical site footprint across RESET program TM 16 patients enrolled and 10 patients dosed across RESET studies, with 40 actively recruiting U.S. sites TM • Clinical development expanding to Europe in 2025 with EMA CTA authorization for CABA-201 received for RESET-SLE • Gerwin Winter app
Slide 7: Lessons from Oncology: Expanding CAR T Cell Therapies into Autoimmunity
Lessons from Oncology: Expanding CAR T Cell Therapies into Autoimmunity 7
Slide 8: Success of CAR T in oncology established over decades
Success of CAR T in oncology established over decades B cell cancer experience with CAR T informs use in autoimmune patients 1987 1994 2003 2017 1 First CAR design First report of CAR T cells First anti-CD19 First CAR T cell therapy approved by FDA 2 5 7 killing tumor cells in mice CAR reported (tis
Slide 9: Considerations for CAR T therapy in cancer and autoimmunity
Considerations for CAR T therapy in cancer and autoimmunity 1 Factors that predict adverse events and relapse differ in patients with autoimmune diseases Cancer Autoimmune disease Risk of side effects related to target B cell burden (including 1-5 CAR CRS and ICANS) T cell Healthy cells Risk of trea
Slide 10: Potential adverse events after CAR T cell therapy in cancer
Potential adverse events after CAR T cell therapy in cancer Physician experience in oncology has established algorithms for routine management of common AEs 1 CRS/ICANS assessment parameters Measures alterations in speech, Fever orientation, handwriting, attention, ICE score 2 & receptive aphasia Hy
Slide 11: CABA-201 Clinical and Translational Data from the RESET Clinical Program
CABA-201 Clinical and Translational Data TM from the RESET Clinical Program 11
Slide 12: Autoimmune disease patients face substantial unmet medical needs
Autoimmune disease patients face substantial unmet medical needs 1-4 Despite therapies with chronic broad immunosuppression, mortality is increased, and quality of life is reduced* Systemic lupus Myositis Systemic sclerosis erythematosus 6 • Chronic inflammation often leads to • Progressive, multi-o
Slide 13: Key inclusion and exclusion criteria in RESET clinical program
TM Key inclusion and exclusion criteria in RESET clinical program Designed to evaluate the safety and tolerability of CABA-201 in subjects with active, refractory disease 1–3 Key inclusion criteria Evidence of active disease despite prior or current treatment with standard of care RESET-Myositis RE
Slide 14: RESET clinical trials have consistent design principles
1 RESET clinical trials have consistent design principles Individual trials in myositis, SLE, and SSc share common elements of preconditioning, dose, and study design † Day 1 Day 29 Study follow-up through Year 3 Day -5 to Day -3 Leukapheresis Primary endpoint: Additional Endpoints Single infusion S
Slide 15: Baseline characteristics of first patients in the RESET program
Baseline characteristics of first 8 patients in the RESET program All patients had active, refractory disease and most had failed B cell-targeting therapies RESET-Myositis RESET-SLE RESET-SSc † SSc-Skin-1 SLE-1 Patient / Cohort IMNM-1 IMNM-2 DM-1 SLE-2 SLE-3 LN-1 Class V LN (severe skin cohort) Age,
Slide 16: Incidence and severity of adverse events in the first patients
Incidence and severity of adverse events in the first 8 patients* RESET-Myositis RESET-SLE RESET-SSc Cohort IMNM DM Non-renal SLE LN SSc – Severe Skin Patient IMNM-1 IMNM-2 DM-1 SLE-1 SLE-2 SLE-3 LN-1 SSc-Skin-1 † CRS None None None None Grade 1 None Grade 1 Grade 2 † ICANS None None None None None
Slide 17: Consistent and complete B cell depletion by Day 15
1 Consistent and complete B cell depletion by Day 22 In patients with >3-month follow-up, B cell repopulation with naïve cells started as early as 8 weeks B cell depletion/repopulation & CABA-201 expansion B cell depletion & CABA-201 expansion through Day 30 through Day 150 CABA-201 exhibited a PK/P
Slide 18: RESET-Myositis: Early efficacy data following CABA-201 infusion
RESET-Myositis : Early efficacy data following CABA-201 infusion st ‡ 1 known adult DM patient dosed with CAR T demonstrated compelling early response off immunosuppressants Disease activity & improvement measures DM patient demonstrated a major TIS response at Day 29 CK CDASI-A TIS 1000 25 Disconti
Slide 19: RESET-Myositis: Efficacy data following CABA-201 infusion
RESET-Myositis : Efficacy data following CABA-201 infusion st ‡ 1 IMNM patient with longer follow up demonstrated continuing response off immunosuppressants without flares st Disease activity & improvement measures 1 IMNM patient showed a moderate TIS response at Week 24 TIS (N=3) CK (N=3) 100 5000
Slide 20: RESET-SLE: Efficacy data in SLE following CABA-201 infusion
RESET-SLE : Efficacy data in SLE following CABA-201 infusion ‡ All 3 SLE patients demonstrated clinical responses off immunosuppressants; first patient completed steroid taper SLE-1 (Class V LN) SLE-2 SLE-3 30 30 30 26 25 25 25 20 20 20 Discontinued Discontinued Discontinued AZA, HCQ GC, MMF, HCQ HC
Slide 21: RESET-SLE: Efficacy data in LN following CABA-201 infusion
RESET-SLE : Efficacy data in LN following CABA-201 infusion ‡ LN-1 demonstrated marked improvement of proteinuria off all immunosuppressants, continuing steroid taper st UPCR decreased from 7.22 to 0.63 mg/mg at Week 16 1 LN patient SLEDAI reduced by 14 points at Week 16 Discontinued all Proteinuria
Slide 22: ICANS event timeline in first LN patient
ICANS event timeline in first LN patient Patient with recent fever in setting of acute and severe inflammation • 24-year-old female with SLE for ~2 years and with active class III LN • Active, severe (SLEDAI-2K = 22; UPCR = 7.22 mg/mg) refractory disease despite 5 systemic treatments* for SLE at scr
Slide 23: Outcomes in first LN patient months post-treatment
Outcomes in first LN patient 4 months post-treatment Compelling clinical response since discontinuation of all immunosuppressants, continuing steroid taper Patient Month 4 Follow-up* ‡ • Off all immunosuppressants • Prednisone 8mg/day; taper ongoing • SLEDAI-2K: 22 à 8 “Overall, I feel much better t
Slide 24: Emerging efficacy data in first SSc patient
Emerging efficacy data 42 days post infusion in first SSc patient Early disease improvements in face and hands after discontinuation of disease-specific medication 1 1 1 Baseline mRSS score by body area Overall mRSS score Day 42 mRSS score by body area Baseline Day 22 Day 42 mRSS 42 38 36 • Modified
Slide 25: Conclusions
Conclusions Cabaletta Bio”
Slide 26: Summary from clinical & translational data on the first patients
Summary from clinical & translational data on the first 8 patients • CABA-201 appears to have a favorable risk-benefit profile • In patients with recent fever or infections, delaying CAR T infusion should be considered • CABA-201 provided compelling efficacy in highly active and refractory autoimmun
Slide 27: Realizing the vision to transform autoimmune disease treatment
Realizing the vision to transform autoimmune disease treatment 2025: Meet with FDA on potential registrational CABA-201 trial designs 2H24: Additional 1H25: clinical data in Initial clinical Executing IND specific clinical myositis & SLE data in MG program to potentially accelerate at ACR 2024 path
Slide 28: Q&A
Q&A Cabaletta Bio”