resecabtagene autoleucel (CABA-201) / Cabaletta Bio, IASO BIO - LARVOL DELTA

RESET-Myositis TM : Clinical Trial Evaluating Rese-cel (Resecabtagene Autoleucel), A Fully Human, Autologous 4-1BB Anti-CD19 CAR T Cell Therapy in Idiopathic Inflammatory Myopathies: Correlative Findings (ASGCT 2025) - P1/2 | "Leukocyte and lymphocyte counts dropped quickly and transiently after a standard preconditioning regimen of fludarabine (25mg/m 2 /day x 3) and cyclophosphamide (1000mg/m 2 ) which inversely corresponded with a transient elevations in serum IL-15 levels in all patients. These emerging data detail the pharmacokinetics and pharmacodynamics of rese-cel in adult patients with IIM, supporting the potential for rese-cel to induce durable, drug-free remissions. Evaluation of the safety and efficacy of rese-cel continues in the ongoing Phase 1/2 RESET-Myositis TM trial. Disease Focus of Abstract:Autoimmune Disorders"

CAR T-Cell Therapy • Clinical • Dermatomyositis • Immunology • Infectious Disease • Myositis • Rare Diseases • CD20 • CD24 • CD8 • IFNG • IL15 • SAE1 • SRP54

RESET-SLE TM : Clinical Trial Evaluating Rese-cel (Resecabtagene Autoleucel), A Fully Human, Autologous 4-1BB Anti-CD19 CAR T Cell Therapy in Non-Renal SLE and Lupus Nephritis: Correlative Findings (ASGCT 2025) - P1/2 | "Leukocyte and lymphocyte counts reduced quickly and transiently after a standard preconditioning regimen of fludarabine (25mg/m 2 /day x 3) and cyclophosphamide (1000mg/m 2 ) that inversely corresponded with a transient elevation in serum IL-15 levels. These emerging data detail the pharmacokinetics and pharmacodynamics of rese-cel in patients with SLE and LN and support the potential for rese-cel to lead to durable, drug-free remissions. Evaluation of safety and efficacy of rese-cel continues in the ongoing Phase 1/2 RESET-SLE TM trial. Disease Focus of Abstract:Autoimmune Disorders"

CAR T-Cell Therapy • Clinical • Glomerulonephritis • Immunology • Infectious Disease • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • CD20 • CD24 • CD8 • IFNG • IL15

RESET-SSc TM : Clinical Trial Evaluating Rese-cel (Resecabtagene Autoleucel), A Fully Human, Autologous 4-1BB Anti-CD19 CAR T Cell Therapy in Systemic Sclerosis: Correlative Findings (ASGCT 2025) - P1/2 | "The patient experienced grade 2 cytokine release syndrome (CRS), manifesting as fever and hypotension which resolved following intravenous fluids without requiring tocilizumab...Leukocyte and lymphocyte counts dropped quickly and transiently after a standard preconditioning regimen of fludarabine (25 mg/m 2 /day x 3) and cyclophosphamide (1000 mg/m 2 ) which inversely corresponded with an elevation in serum IL-15 levels... These emerging data detail the pharmacokinetics and pharmacodynamics of rese-cel in one SSc patient . Rese-cel deeply and transiently depleted B cells in circulation and in lymphoid tissue and support the potential for rese-cel to induce durable, drug-free remission. Evaluation of the safety and efficacy of rese-cel continues in the ongoing Phase 1/2 RESET-SSc TM trial."

CAR T-Cell Therapy • Clinical • Hypotension • Immunology • Inflammation • Scleroderma • Systemic Sclerosis • CD20 • CD24 • CD8 • IFNG • IL15 • IL6

Clinical & Translational Findings Following Resecabtagene Autoleucel Anti-CD19 CAR T Cell Therapy in Autoimmune Disease (ASGCT 2025) - "Supported by the Hematologic and Immunologic Cell and Gene Therapy Committee Info Description: An overview of clinical and translational data in subjects dosed with rese-cel (Resecabtagene Autoleucel) autologous anti-CD19 CAR T cell therapy in autoimmune disease."

CAR T-Cell Therapy • Clinical • Gene Therapies • Hematological Disorders • Immunology

Study of CAR T-cell therapy rese-cel in scleroderma enrolling in US (Scleroderma News) - "A Phase 1/2 trial testing Cabaletta Bio’s CAR T-cell therapy, rese-cel (resecabtagene autoleucel), in adults with hard-to-treat systemic sclerosis (SSc) is recruiting patients at several sites in the U.S. The open-label RESET-SSc trial (NCT06328777) is assessing the safety and efficacy of rese-cel as a single infusion into a vein, or intravenously, in patients ages 18-75. It plans to enroll two groups of patients, one with severe skin involvement and the other with heart, lung, and/or kidney involvement, with or without skin manifestations."

Enrollment status • Systemic Sclerosis

RESET-SScTM: Clinical Trial Evaluating Rese-cel (Resecabtagene Autoleucel), A Fully Human, Autologous 4-1BB Anti-CD19 CAR T Cell Therapy in Systemic Sclerosis (EULAR 2025) - No abstract available

CAR T-Cell Therapy • Clinical • Immunology • Scleroderma • Systemic Sclerosis

Cabaletta Bio Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Business Update (GlobeNewswire) - "In June 2025, Cabaletta plans to present new and updated clinical and translational data on rese-cel from the RESET-Myositis, RESET-SLE and RESET-SSc trials in three oral presentations at the upcoming EULAR 2025 Congress, which is being held at Fira de Barcelona in Barcelona, Spain from June 11-14, 2025."

P1/2 data • Myositis • Systemic Lupus Erythematosus • Systemic Sclerosis

RESET-MyositisTM: Clinical Trial Evaluating Rese-cel (Resecabtagene Autoleucel), A Fully Human, Autologous 4-1BB Anti-CD19 CAR T Cell Therapy in Idiopathic Inflammatory Myopathies (EULAR 2025) - No abstract available

CAR T-Cell Therapy • Clinical • Immunology • Myositis

RESET-SLE: Clinical Trial Evaluating Rese-cel (Resecabtagene Autoleucel), a Fully Human, Autologous 4-1BB Anti-CD19 CAR T Cell Therapy in Non-Renal SLE and Lupus Nephritis (EULAR 2025) - No abstract available

CAR T-Cell Therapy • Clinical • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology

Cellares and Cabaletta Bio Successfully Complete Manufacturing Technology Adoption Program for Rese-cel Using the Cell Shuttle Platform (Businesswire) - "Cellares...has concluded the Technology Adoption Program (TAP) on its automated cell therapy manufacturing Cell Shuttle for resecabtagene autoleucel...Rese-cel is the lead clinical candidate in development by Cabaletta Bio...This successful collaboration facilitates the opportunity to engage in a clinical and commercial manufacturing relationship. Cellares’ TAP assessed the feasibility of using Cellares' innovative Cell Shuttle platform to automate the manufacturing of Cabaletta's rese-cel drug product, a CD19-targeting CAR T cell therapy designed to treat patients with a broad range of autoimmune diseases."

Commercial • Immunology

Cabaletta Bio Announces Updated Clinical Data Demonstrating Deepening Clinical Responses across Multiple Indications with Rese-cel at February Scientific Meetings (GlobeNewswire) - P1/2 | N=24 | RESET-Myositis (NCT06154252) | P1/2 | N=12 | RESET-SLE (NCT06121297) | P1/2 | N=12 | RESET-SSc (NCT06328777) | Sponsor: Cabaletta Bio | "As of the data cut-off date of January 8, 2025, 10 patients had been dosed with rese-cel across the RESET-Myositis, RESET-SLE and RESET-SSc trials...In the RESET-Myositis trial, the first adult dermatomyositis patient maintained a major total improvement score (TIS) improvement at 3 months post-infusion; In the RESET-SLE trial, 3 out of 4 patients in the non-renal systemic lupus erythematosus (SLE) cohort achieved DORIS (definition of remission in SLE) remission; In the RESET-SSc trial, the first patient dosed with rese-cel in the severe skin cohort continued to demonstrate clinically meaningful skin improvements; Across the first 10 patients dosed with rese-cel with at least one month of follow-up, 90% experienced either no cytokine release syndrome (CRS) or grade 1 CRS (fever)..."

P1/2 data • Myositis • Systemic Lupus Erythematosus • Systemic Sclerosis

Cabaletta Bio Announces Presentations Featuring Updated Clinical Data on Rese-cel at Upcoming Scientific Meetings in February (GlobeNewswire) - "Cabaletta Bio, Inc...announced that updated clinical data on resecabtagene autoleucel (rese-cel, formerly referred to as CABA-201) will be featured in presentations at the 2025 American Association for the Advancement of Science Annual Meeting, which is being held at the Hynes Convention Center in Boston, MA from February 13-15, 2025, and the 5th International Conference on Lymphocyte Engineering, which is being held at the Holiday Inn Munich – City Center in Munich, Germany from February 20-22, 2025. The presentations will include safety, translational data and clinical outcomes from the first 10 patients dosed with rese-cel across multiple ongoing clinical trials in the RESET clinical development program."

P1/2 data • Myositis • Systemic Lupus Erythematosus • Systemic Sclerosis

Cabaletta Bio Outlines Strategic Priorities and Anticipated Key Milestones for 2025 (GlobeNewswire) - "The first patient has been enrolled in the RESET-PV trial, evaluating rese-cel without preconditioning in patients with pemphigus vulgaris. The first juvenile myositis clinical site in the RESET-Myositis trial is now open and actively recruiting. The U.S. Food and Drug Administration (FDA) previously granted Rare Pediatric Disease designation for rese-cel in juvenile dermatomyositis. The first patient has been enrolled in the RESET-MG trial, evaluating rese-cel in patients with myasthenia gravis.The Investigational New Drug (IND) application for rese-cel has been allowed to proceed within the routine 30-day window by the FDA for the RESET-MS trial...The Company now plans to meet with the FDA regarding registrational trial designs for rese-cel in the first half of 2025 based on the emerging clinical and translational data and increased pace of enrollment...Present new and updated clinical and translational data on rese-cel throughout 2025."

Clinical data • Enrollment open • FDA event • IND • Trial status • Dermatomyositis • Multiple Sclerosis • Myasthenia Gravis • Pemphigus Vulgaris

RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy (clinicaltrials.gov) - P1/2 | N=24 | Recruiting | Sponsor: Cabaletta Bio | N=18 ➔ 24

Enrollment change • Dermatomyositis • Immunology • Myositis • Rare Diseases

220: Achieving an Immune System RESET: The Next Act in CAR T Therapy (ACR Convergence 2024) - "Sponsored by Cabaletta Bio...Speakers will also provide an overview of the distinguishing features of CABA-201, a CD19-CAR T cell therapy candidate, and the RESET™ Ph 1/2 clinical program in several autoimmune diseases, including lupus, myositis, and systemic sclerosis. Not an official program of the ACR. Non-CME"

Immunology • Inflammatory Arthritis • Lupus • Myositis • Scleroderma • Systemic Sclerosis

Correlative Studies of CABA-201, a Fully Human, Autologous 4-1BB Anti-CD19 CAR T Cell Therapy in Patients with Immune-Mediated Necrotizing Myopathy and Systemic Lupus Erythematosus from the RESET-MyositisTM and RESET-SLETM Clinical Trials (ACR Convergence 2024) - P1/2 | "Leukocyte and lymphocyte counts dropped quickly and transiently after a standard preconditioning regimen of fludarabine (25mg/m2/dy x 3) and cyclophosphamide (1000mg/m2) which inversely corresponded with a transient peak in serum IL-15 levels in both patients. These emerging data detail the pharmacokinetics and pharmacodynamics of CABA-201 in IIM and SLE. Evaluation of the selected CABA-201 dose continues in the ongoing RESET-Myositis and RESET-SLE trials."

CAR T-Cell Therapy • Clinical • Dermatomyositis • Glomerulonephritis • Immunology • Infectious Disease • Inflammation • Inflammatory Arthritis • Lupus • Lupus Nephritis • Myositis • Nephrology • Rare Diseases • Systemic Lupus Erythematosus • IFNG • IL15

Safety and Efficacy of CABA-201, a Fully Human, Autologous 4-1BB Anti-CD19 CAR T Cell Therapy in Patients with Immune-Mediated Necrotizing Myopathy and Systemic Lupus Erythematosus from the RESET-MyositisTM and RESET-SLETM Clinical Trials (ACR Convergence 2024) - P1/2 | "For both trials, a single infusion of 1×106 CAR T cells/kg is administered following a preconditioning regimen of cyclophosphamide and fludarabine...At 3 months post infusion, the IMNM subject achieved a meaningful response in the total improvement score, associated with improvements in the CK to 308 and MMT-8 to 134, as well as reduction in anti-SRP and anti-Ro52 levels, while off methotrexate and glucocorticoids. At 1 month post infusion, the SLE subject achieved a SLEDAI-2K improvement of 16 points with resolution of vasculitis, arthritis, and hematuria and reduction in the anti-dsDNA titer, while off mycophenolate and hydroxychloroquine and on a prednisone taper.Safety outcomes are in Table 2. Both subjects tolerated CABA-201 treatment with no SAE, CRS, or ICANS... Both subjects tolerated CABA-201 treatment with no SAE, CRS, or ICANS. The administered dose of CABA-201 resulted in CAR T cell expansion, peripheral B cell depletion, and biomarker and..."

CAR T-Cell Therapy • Clinical • Dermatomyositis • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Myositis • Nephrology • Pediatrics • Rare Diseases • Rheumatology • Systemic Lupus Erythematosus • Vasculitis

Cabaletta Bio Presents Positive Clinical Safety and Efficacy Data on CABA-201 at ACR Convergence 2024 (GlobeNewswire) - P1/2| N=18 | RESET-Myositis (NCT06154252) | P1/2| N=18 | RESET-SLE (NCT06121297) | P1/2| N=18 | RESET-SSc (NCT06328777)| Sponsor: Cabaletta Bio | "Cabaletta Bio...today announced new and updated clinical data on CABA-201 demonstrating the potential to achieve drug-free, compelling clinical responses based on eight patients dosed across the ongoing Phase 1/2 RESET-Myositis, RESET-SLE and RESET-SSc clinical trials....As of the data cut-off date of November 1, 2024, eight patients had been dosed with CABA-201 with sufficient follow-up to be evaluable across the RESET clinical development program....Across these eight patients treated with CABA-201, patients were administered a one-time infusion of CABA-201 at 1 x 106 cells/kg, following a preconditioning regimen of fludarabine and cyclophosphamide. The primary endpoint of each trial is safety and tolerability within 28 days of infusion."

P1/2 data • Myositis • Systemic Lupus Erythematosus • Systemic Sclerosis

Cabaletta Bio Reports Third Quarter 2024 Financial Results and Provides Business Update (GlobeNewswire) - "Clinical data from the RESET-Myositis and RESET-SLE trials, along with initial clinical data from the RESET-SSc trial, to be presented this weekend in oral and poster presentations at ACR Convergence 2024....'We are encouraged by the accelerating pace of enrollment and, data permitting, anticipate meeting with the FDA next year regarding registrational program designs for CABA-201'....Generalized myasthenia gravis (gMG): Patient enrollment in the RESET-MG trial is ongoing and initial clinical data from the trial are expected in the first half of 2025....Pemphigus vulgaris (PV): Patient enrollment is ongoing in the RESET-PV trial, formerly referred to as the DesCAARTes trial."

Enrollment closed • FDA event • P1/2 data • Immunology • Myasthenia Gravis • Myositis • Pemphigus Vulgaris • Systemic Lupus Erythematosus • Systemic Sclerosis

Lupus Research Alliance Announces Lupus Research Highlights at ACR Convergence 2024 (PRNewswire) - "The Lupus Research Alliance (LRA), the largest private funder of lupus research worldwide, today announced that a total of 33 studies, funded by the organization or supported by its clinical research affiliate Lupus Therapeutics, will be presented at ACR Convergence 2024. The annual meeting of the American College of Rheumatology takes place November 14-19, at the Walter E. Washington Convention Center in Washington, D.C."

Clinical data • Lupus • Lupus Nephritis • Systemic Lupus Erythematosus

Cabaletta Bio to Present New and Updated Clinical Data on CABA-201 in Oral and Poster Presentations at ACR Convergence 2024 (GlobeNewswire) - "Cabaletta Bio...announced two presentations at the upcoming American College of Rheumatology (ACR) Convergence 2024, which is being held at the Walter E. Washington Convention Center in Washington, D.C. from November 14-19, 2024. The presentations will feature new and updated clinical data on CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, which is being evaluated in multiple ongoing clinical trials in the RESET clinical development program."

Clinical data • Systemic Lupus Erythematosus

Case study of CD19-directed chimeric antigen receptor T-cell therapy in a subject with immune-mediate necrotizing myopathy treated in the RESET-Myositis™ phase I/II trial. (PubMed, Mol Ther) - P1/2 | "Post-infusion, CABA-201 expansion peaked at day 15 and was preceded by a serum IFN-γ peak on day 8 with peaks in serum IL-12p40 and IP-10 on day 15. These data detail the safety, efficacy, and pharmacodynamics of CABA-201 in the first IMNM subject."

CAR T-Cell Therapy • Journal • P1/2 data • Immunology • Inflammation • Myositis • CD4 • IFNG • SRP54 • SRP72

Preclinical specificity & activity of a fully human 41BB-expressing anti-CD19 CART- therapy for treatment-resistant autoimmune disease. (PubMed, Mol Ther Methods Clin Dev) - "Novel examination of CABA-201 generated from primary T cells from multiple patients with autoimmune disease displayed robust CAR surface expression and effective elimination of the intended target autologous CD19+ B cells in vitro. Together, these findings support the tolerability and activity of CABA-201 for clinical development in patients with autoimmune disease."

Journal • Preclinical • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus • CD19

Cabaletta Bio Reports Positive Initial Clinical Data from Phase 1/2 RESET-Myositis and RESET-SLE Trials of CABA-201 (GlobeNewswire) - P1/2 | N=18 | RESET-Myositis (NCT06154252) | P1/2 | N=12 | RESET-SLE (NCT06121297) | Sponsor: Cabaletta Bio | "Company to host live investor conference call and webcast today at 8:00 a.m. ET....Cabaletta Bio...today reported positive initial clinical data from each of the first two patients dosed with CABA-201 in the Phase 1/2 RESET-Myositis and RESET-SLE trials....As of May 28, 2024, the data cut-off date, one patient treated in the immune-mediated necrotizing myopathy (IMNM) cohort in the RESET-Myositis trial had completed three months of follow-up and one patient enrolled in the systemic lupus erythematosus (SLE) non-renal cohort in the RESET-SLE trial had completed one month of follow-up....The primary endpoint of each trial is safety and tolerability within 28 days of infusion....At week 12 of follow-up for the IMNM patient, the data show a decline in creatinine kinase from 617 at infusion to 308 and a total improvement score (TIS) of 30."

P1/2 data • CNS Disorders • Immunology • Inflammation • Lupus • Myasthenia Gravis • Myositis • Scleroderma • Systemic Lupus Erythematosus • Systemic Sclerosis

Cabaletta Bio Reports Positive Initial Clinical Data from Phase 1/2 RESET-Myositis and RESET-SLE Trials of CABA-201 (GlobeNewswire) - “With the RESET-SSc and RESET-MG trials recently opening for enrollment, an additional cohort evaluating patients with juvenile myositis incorporated into the RESET-Myositis trial and the momentum provided by the promising early clinical data, we are looking forward to accelerating clinical trial enrollment in the RESET clinical program. We continue to expect to report initial clinical data from the Phase 1/2 RESET-SSc and RESET-MG trials as well as additional data from the RESET-Myositis and RESET-SLE trials in the second half of this year."

P1/2 data • CNS Disorders • Immunology • Inflammation • Lupus • Myasthenia Gravis • Myositis • Scleroderma • Systemic Lupus Erythematosus • Systemic Sclerosis