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

Mechanistic basis of the acute safety profile of rese-cel, an autologous CD19-CAR T, in patients with autoimmune disease treated in four ongoing phase 1/2 clinical trials (ASH 2025) - P1/2 | "Here, we provide novel insight into the mechanism underlying the safetyprofile of resecabtagene autoleucel (rese-cel) an autologous CD19 41BBz CART therapy across fourseparate Phase I/II clinical trials in SLE, IIM, SSc, & MG.Objectives: The primary objective of each trial is safety & tolerability of rese-cel at Day 29...Patients were not given prophylactic tocilizumab pre-infusion... Rese-cel appears to be well tolerated in autoimmune patients with rates of CRS & ICANS of37% & 11% across all autoimmune indications, respectively (SLE patients had a 25% CRS rate & 12.5%ICANS rate, IIM patients had a 50% CRS rate & 0% ICANS rate, SSC patients had a 50% CRS & 50% ICANSrate, & MG patients had a 0% CRS & ICANS rate). Notably, the frequency & severity of CRS appears to below, with no CRS in 63% of patients (12/19) & most CRS events (6/7) being Grade 1. The lower frequency& severity of both CRS & ICANS events are likely..."

Clinical • P1/2 data • CNS Disorders • Immunology • Inflammatory Arthritis • Lupus • Myasthenia Gravis • Myositis • Scleroderma • Systemic Lupus Erythematosus • Systemic Sclerosis • CD24 • CD38 • CD8 • CXCL8 • IFNG • IL6

RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus (clinicaltrials.gov) - P1/2 | N=28 | Recruiting | Sponsor: Cabaletta Bio | N=12 ➔ 28 | Trial completion date: Dec 2027 ➔ Dec 2029 | Trial primary completion date: Dec 2027 ➔ Dec 2029

Enrollment change • Trial completion date • Trial primary completion date • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Systemic Lupus Erythematosus

Cabaletta Bio's (CABA) innovative therapy for myasthenia gravis, known as resecabtagene autoleucel, has secured orphan drug designation from the U.S. Food and Drug Administration (FDA) (Gurufocus)

Orphan drug • Myasthenia Gravis

Additional clinical data from RESET trials expected throughout 2026 (GlobeNewswire) - "On December 6, 2025, Cabaletta will present a poster...In addition, Cabaletta plans to present complete Phase 1/2 data from the RESET-SSc and RESET-SLE trials in the first half of 2026 and from the RESET-MG trial in the second half of 2026. The Company also anticipates clinical data on rese-cel without preconditioning from the initial RESET-SLE dose-escalation cohort and additional dosing from the RESET-PV trial in 2026."

P1/2 data • Myasthenia Gravis • Pemphigus Vulgaris • Systemic Lupus Erythematosus • Systemic Sclerosis

FDA alignment on additional registrational cohort designs expected through 2026 (GlobeNewswire) - "Cabaletta expects to align with the FDA on key registrational design elements for the RESET-SSc and RESET-SLE trials by year-end 2025 and for the RESET-MG trial in the first half of 2026. Subject to clinical data and regulatory alignment on registrational cohort design elements for these trials, the Company expects to initiate enrollment in registrational cohorts in 2026."

Trial status • Myasthenia Gravis • Systemic Lupus Erythematosus • Systemic Sclerosis

Recent Operational Highlights and Upcoming Anticipated Milestones (GlobeNewswire) - "In the Phase 1/2 DM/ASyS cohort, all patients with sufficient follow-up who would have met key criteria for the registrational cohort met the registrational primary endpoint. If successful, data from the DM/ASyS cohort will be used in part to support the Company’s first projected Biologics License Application (BLA) submission for rese-cel in myositis in 2027."

FDA filing • Myositis

CAB-201-005: RESET-MS: A Study to Evaluate the Safety and Efficacy of CABA-201 in Participants With Multiple Sclerosis (clinicaltrials.gov) - P1/2 | N=12 | Not yet recruiting | Sponsor: Cabaletta Bio | Initiation date: Aug 2025 ➔ Jun 2026

Trial initiation date • CNS Disorders • Multiple Sclerosis

RESET-Myositis: Complete Adult Phase 1/2 Data and Registrational Cohort Update (GlobeNewswire) - "Regarding safety, 4 of 13 patients experienced fever, or grade 1 cytokine release syndrome (CRS), and no immune effector cell-associated neurotoxicity syndrome (ICANS) was observed...All 4 DM/ASyS patients who met the key inclusion criteria for the registrational cohort with sufficient follow-up achieved immunomodulatory-free total improvement score (TIS) responses of moderate or major improvement at week 16. Based on these clinical data, Cabaletta is initiating a DM/ASyS registrational cohort within the RESET-Myositis trial...Cabaletta remains on track to initiate enrollment in the registrational DM/ASyS cohort this quarter....Prior to the potential initiation of a registrational IMNM cohort, additional patients will be enrolled in the Phase 1/2 cohort..."

Cytokine release syndrome • P1/2 data • Trial status • Dermatomyositis • Immunology

RESET-SLE: Preliminary Phase 1/2 Data and Expansion of No Preconditioning Strategy (GlobeNewswire) - "Three of 4 SLE patients with at least 3 months of follow-up achieved DORIS (definition of remission in SLE), and the fourth patient with pure class V LN achieved a complete renal response. Three of 4 LN patients with at least 3 months of follow-up showed renal response. All 9 patients were off all immunomodulators as of the data cut-off. Patients across both cohorts achieved a median 8-point reduction in SLEDAI-2K and a significant reduction in anti-dsDNA antibodies was observed...complete B cell depletion is possible with a single, weight-based dose of rese-cel without the use of a fludarabine and cyclophosphamide lymphodepleting regimen....Cabaletta is incorporating this new dose-escalation cohort into the RESET-SLE trial with initial clinical data anticipated in 2026."

P1/2 data • Trial status • Lupus Nephritis • Systemic Lupus Erythematosus

RESET-SSc: Preliminary Phase 1/2 Data (GlobeNewswire) - "Cabaletta is presenting preliminary Phase 1/2 clinical data from 6 RESET-SSc patients, including 3 in the severe skin (SSc-Skin) cohort and 3 in the organ (SSc-Organ) cohort. Three of these 6 patients experienced low-grade CRS (grade 1 or 2) and one ICANS event was observed (grade 3, previously reported in March 2025)....All 4 patients with at least 3 months of follow-up achieved an rCRISS-25 response off immunomodulators and steroids. These initial data suggest the potential for rese-cel to reset the immune system in systemic sclerosis, allowing patients to achieve transformative clinical responses off all immunomodulators and glucocorticoids. Cabaletta anticipates FDA alignment on the registrational cohort design this year."

P1/2 data • Trial status • Systemic Sclerosis

RESET-Myositis: Clinical Trial Evaluating Rese-cel (Resecabtagene Autoleucel), A Fully Human, Autologous 4-1BB CD19-CAR T Cell Therapy in Idiopathic Inflammatory Myopathies (ACR Convergence 2025) - P1/2 | "A weight-based infusion of 1x106 CAR T cells/kg was given following preconditioning with fludarabine and cyclophosphamide. Data from IIM patients infused with rese-cel show early drug-free clinical responses in most patients and acceptable safety in the setting of rese-cel expansion and peripheral B cell depletion. DM & ASyS patients achieved a higher threshold of response than IMNM which is not unexpected. These initial data suggest the potential for rese-cel to reset the immune system, allowing patients to achieve meaningful drug-free clinical responses."

CAR T-Cell Therapy • Clinical • Cardiovascular • Dermatomyositis • Immunology • Inflammation • Myositis • Pulmonary Embolism • Rare Diseases • Respiratory Diseases • CD20 • CD24 • IFNG

RESET-SSc: Clinical Trial Evaluating Rese-cel (Resecabtagene Autoleucel), A Fully Human, Autologous 4-1BB CD19-CAR T Cell Therapy in Systemic Sclerosis (ACR Convergence 2025) - P1/2 | "A weight-based infusion of 1x106 CAR T cells/kg was administered following preconditioning with fludarabine and cyclophosphamide. Data from the first SSc patient infused with rese-cel shows early IM-free clinical response and acceptable safety profile in the setting of CAR T cell expansion and complete peripheral and lymph node B cell depletion. These initial data suggest the potential for rese-cel to reset the immune system in SSc, allowing patients to achieve meaningful clinical responses off all IM therapies."

CAR T-Cell Therapy • Clinical • CNS Disorders • Epilepsy • Immunology • Inflammation • Scleroderma • Systemic Sclerosis • CD20 • CD24 • IFNG • PAX5

RESET-SLE: Clinical Trial Evaluating Rese-cel (Resecabtagene Autoleucel), a Fully Human, Autologous 4-1BB CD19-CAR T Cell Therapy in Non-Renal SLE and Lupus Nephritis (ACR Convergence 2025) - P1/2 | "A single infusion of 1x106 CAR T cells/kg was administered following preconditioning with fludarabine and cyclophosphamide. Data from SLE and LN patients infused with rese-cel shows early clinical responses, including meeting the DORIS remission criteria and complete renal response, and an acceptable safety profile, in the setting of CAR T cell expansion and peripheral B-cell reduction. These initial data suggest the potential for rese-cel to reset the immune system in SLE and LN, allowing patients to achieve meaningful clinical responses off all IM agents."

CAR T-Cell Therapy • Clinical • Glomerulonephritis • Immunology • Infectious Disease • Inflammation • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Systemic Lupus Erythematosus • CD20 • CD24 • IFNG

Cabaletta Bio Presents First Rese-cel Data with No Preconditioning Demonstrating Biologic Activity and Early Clinical Responses at the 2025 ESGCT Annual Congress (GlobeNewswire) - "Rese-cel exhibited similar CAR T cell expansion and contraction kinetics relative to translational data reported from other RESET trials with preconditioning. All three patients experienced substantial depletion of B cells within the first month post-infusion, with patients 2 and 3 achieving complete peripheral B cell depletion...Rese-cel was generally well tolerated with no immune effector cell-associated neurotoxicity syndrome (ICANS) reported...Meaningful early clinical responses were observed in all three patients starting in the first month post-infusion based on Pemphigus Disease Area Index (PDAI) score for skin, scalp and mucosal surfaces. From baseline to latest follow-up, PDAI activity scores improved as follows: Patient 1 (4 mo): 24 to 10; Patient 2 (3 mo): 83 to 3; Patient 3 (1 mo): 22 to 2."

Late-breaking abstract • P1/2 data • Pemphigus Vulgaris

All adult Phase 1/2 cohorts within the myositis, lupus, scleroderma and myasthenia gravis RESET trials are fully enrolled as of September 30, 2025 (GlobeNewswire)

Enrollment closed • Lupus • Myasthenia Gravis • Myositis • Systemic Sclerosis

RESET-PV: Initial clinical and translational data evaluating rese-cel (resecabtagene autoleucel), an autologous 4-1BBz CD19-CAR T cell therapy, without preconditioning, in pemphigus vulgaris (ESGCT 2025) - No abstract available

CAR T-Cell Therapy • Clinical • Late-breaking abstract • Dermatology • Immunology • Pemphigus Vulgaris

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) - P1/2 | "A single weight-based infusion of 1x10 6 CAR T cells/kg is administered following a preconditioning regimen (fludarabine 25 mg/m 2 /d on Days -5, -4 and -3, and cyclophosphamide 1,000 mg/m 2 on Day -3)...The patient experienced grade 2 cytokine release syndrome (CRS), manifesting as fever and hypotension which resolved following intravenous fluids without requiring tocilizumab... Data from the first SSc patient with significant skin involvement and disability dosed with rese-cel demonstrates early immunosuppressive-free clinical response and favorable safety profile in the setting of CAR T cell expansion and complete peripheral and lymph node B-cell depletion. These initial data suggest the potential for rese-cel to reset the immune system in SSc, allowing patients to achieve meaningful clinical responses while off all immunosuppressive therapies. Additional patients undergoing rese-cel infusion in early 2025 will be presented at the EULAR meeting."

CAR T-Cell Therapy • Clinical • Cardiovascular • Hypotension • Immunology • Inflammation • Rheumatology • Scleroderma • Systemic Sclerosis • CD20 • CD24 • CD38 • CD8 • IFNG • IL6

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) - P1/2 | "A single weight-based infusion of 1x10 6 CAR T cells/kg is administered following a preconditioning regimen (fludarabine 25 mg/m 2 /d on Days -5, -4 and -3, and cyclophosphamide 1,000 mg/m 2 on Day -3). Data from IIM patients dosed with rese-cel demonstrate early immunomodulatory-free clinical responses and a favorable safety profile in the setting of CAR T cell expansion and peripheral B cell depletion. These initial data suggest the potential for rese-cel to reset the immune system in IIM, allowing patients to achieve meaningful clinical responses while off all immunomodulatory therapies and tapering glucocorticoids."

CAR T-Cell Therapy • Clinical • Cardiovascular • Dermatomyositis • Immunology • Myositis • Pulmonary Embolism • Rare Diseases • Respiratory Diseases • CD8 • IFNG

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) - P1/2 | "A single weight-based infusion of 1x10 6 CAR T cells/kg is administered following a preconditioning regimen (fludarabine 25 mg/m 2 /d on Days -5, -4 and -3, and cyclophosphamide 1,000 mg/m 2 on Day -3)...Rese-cel has been well-tolerated with Grade 1 cytokine release syndrome (CRS) (fever) reported in 2 patients, SLE-2 and LN-1, neither of whom received tocilizumab...All patients remain off all SLE-related immunosuppression, and SLE-1 and SLE-2 have completed their glucocorticoid taper, while LN-1 has tapered to prednisone 6 mg/day as of the most recent follow-up visit... Data from SLE patients dosed with rese-cel demonstrate early immunosuppressive-free clinical remission and/or responses and favorable safety profile, including CRS events, in the setting of CAR T cell expansion and peripheral B-cell depletion. These initial data suggest the potential for rese-cel to reset the immune system in SLE, allowing patients to achieve meaningful clinical responses while..."

CAR T-Cell Therapy • Clinical • Glomerulonephritis • Immunology • Infectious Disease • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • CD4 • CD8 • IFNG

Cabaletta Bio Announces New Rese-cel Safety and Efficacy Data in Patients with…Lupus…to Be Presented at the EULAR 2025 Congress (GlobeNewswire) - P1/2 | N=12 | RESET-SLE (NCT06121297) | Sponsor: Cabaletta Bio | "7 out of 7 patients achieved a clinical response off all immunomodulators and glucocorticoids. All non-renal systemic lupus erythematosus (SLE) patients without nephropathy achieved DORIS as of the latest follow-up. The first lupus nephritis (LN) patient achieved DORIS and a complete renal response, while follow-up is continuing on the other two LN patients who were treated more recently. In the 8 patients with safety follow-up, 2 of 8 experienced grade 1 CRS (fever) and 1 ICANS event was observed (grade 4, previously reported)."

P1/2 data • Systemic Lupus Erythematosus

Cabaletta Bio Announces New Rese-cel Safety and Efficacy Data in Patients with…Scleroderma to Be Presented at the EULAR 2025 Congress (GlobeNewswire) - P1/2 | N=12 | RESET-SSc (NCT06328777) | Sponsor: Cabaletta Bio | "Both of the patients reported in the severe skin cohort had meaningful modified Rodnan Skin Score (mRSS) improvements after discontinuing immunomodulatory drugs and steroids, which was sustained out to 6 months in the first patient. The first patient also met the revised Composite Response Index in Systemic Sclerosis (CRISS) criteria starting at 3 months, highlighting the potential of rese-cel to provide a drug-free, clinical response in patients with systemic sclerosis (SSc). One patient experienced transient grade 2 CRS and one ICANS event was observed (grade 3, previously reported)."

P1/2 data • Scleroderma • Systemic Sclerosis

Cabaletta Bio Announces New Rese-cel Safety and Efficacy Data in Patients with Myositis…to Be Presented at the EULAR 2025 Congress (GlobeNewswire) - P1/2 | N=24 | RESET-Myositis (NCT06154252) | Sponsor: Cabaletta Bio | "7 out of 8 patients achieved a clinical response off all immunomodulators, while off or actively tapering steroids. All 4 antisynthetase syndrome (ASyS) and dermatomyositis (DM) patients achieved a clinically meaningful total improvement score (TIS) response, with 3 of these patients having achieved a major TIS response as of the data cut-off date. Clinically meaningful drug-free responses were observed in 3 of 4 refractory patients with immune-mediated necrotizing myopathy (IMNM), which are consistent with published data. These findings continue to reflect the more modest TIS responses in IMNM compared to other myositis subtypes. Regarding safety, 4 of 8 patients experienced grade 1 CRS (fever), and no ICANS was observed."

P1/2 data • Immunology • Myositis

Rese-cel Translational Profile (GlobeNewswire) - P=NA| N=NA | "Peak rese-cel expansion was observed within approximately two weeks. B cells were rapidly and transiently reduced in peripheral blood within the first month and their repopulation was observed beginning approximately two months after infusion, generally expressing a transitional, naïve phenotype. Tissue-resident B cell depletion was confirmed via a lymph node biopsy in the first systemic sclerosis patient."

Clinical data • Systemic Sclerosis

RESET-MS: A Study to Evaluate the Safety and Efficacy of CABA-201 in Participants With Multiple Sclerosis (clinicaltrials.gov) - P1/2 | N=12 | Not yet recruiting | Sponsor: Cabaletta Bio

New P1/2 trial • CNS Disorders • Multiple 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: Correlative Findings (ASGCT 2025) - P1/2 | "Leukocyte and lymphocyte counts dropped quickly and transiently after a standard preconditioning regimen of fludarabine (25mg/m2/day x 3) and cyclophosphamide (1000mg/m2) 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-MyositisTM 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