ICG318 / iCell Gene Therap - LARVOL DELTA

Long-Term Remission Achieved with ICG318, a BCMA-CD19 Armored Compound CAR T-cell Therapy, in Refractory Systemic Lupus Erythematosus/Lupus Nephritis with Follow-Up Approaching 6 years (EULAR 2026) - No abstract available

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

Landmark Long-Term Remission Achieved with ICG318, a BCMA-CD19 Armored Compound CAR T-cell Therapy, in Refractory Systemic Lupus Erythematosus/Lupus Nephritis with Follow-Up Approaching 6 years (LUPUS 2026) - "P3-13 (excluding P11) received 3×10^6 autologous ICG318 cells/kg following non-fludarabine conditioning with single-agent cyclophosphamide (0.3 g/m2)...These findings support targeting both CD19 and BCMA in SLE/LN to achieve long-term sCR. With P1 sCR approaching 6 years, this may offer a first glimpse into the curative potential of CAR T-cell therapy in autoimmune diseases."

CAR T-Cell Therapy • Clinical • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Systemic Lupus Erythematosus

Landmark Long-Term Remission Achieved with ICG318, a BCMA-CD19 Armored Compound CAR T-cell Therapy, in Refractory Systemic Lupus Erythematosus/Lupus Nephritis with Follow-up Approaching 6 Years (TCT-ASTCT-CIBMTR 2026) - "P3-13 (excluding P11) received 3×10 6 autologous ICG318 cells/kg following non-fludarabine conditioning with single-agent cyclophosphamide (0.3 g/m 2 )... In this landmark long-term follow-up, ICG318 exhibited remarkable safety and efficacy achieving durable sCR in 10 of 12 patients and reconstituted humoral immunity in all patients. Despite serological absence of disease, P6 failed to satisfy DORIS remission and CRR criteria due to persistent proteinuria from chronic, not active, renal disease evidenced by no IgG complexes on biopsy. Thus, 11 of 12 patients achieved medication-free serological remission."

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

BCMA-CD19 armored compound CAR T cells in systemic lupus erythematosus: extended follow-up of a phase 1 clinical trial. (PubMed, J Hematol Oncol) - "Notably, successful spontaneous pregnancies and births of healthy full-term infants occurred post-treatment, warranting future study of fertility outcomes. sCR approaching 6 years raises the possibility of a long-term durable response to CAR T-cell therapy in humoral autoimmune disease."

Journal • P1 data • Glomerulonephritis • Immunology • Inflammation • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Systemic Lupus Erythematosus

BCMA-CD19 cCAR T for the Treatment of Refractory Lupus (clinicaltrials.gov) - P1/2 | N=20 | Not yet recruiting | Sponsor: iCell Gene Therapeutics

New P1/2 trial • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Systemic Lupus Erythematosus

First-in-Human Trial of BCMA-CD19 Compound CAR (cCAR) in Desensitization of Donor-Specific Anti-HLA Antibodies (DSAs) in Haploidentical Stem Cell Transplant Recipients (ASH 2024) - "All patients received conditioning with fludarabine (30 mg/m2/d) and cyclophosphamide (300 mg/m2/d). Remarkable depletion of antibody-producing "roots cause", B cells and plasma/long-lived plasma cells was observed in all patients. Our results suggest cCAR has the potential to benefit patients receiving solid organ transplants or those with other antibody-mediated diseases."

Clinical • First-in-human • P1 data • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Complement-mediated Rare Disorders • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Solid Organ Transplantation • Transplantation

BCMA-CD19 cCAR in Multiple Myeloma and Plasmacytoid Lymphoma (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: iCell Gene Therapeutics | Trial completion date: Sep 2021 ➔ Jul 2026 | Trial primary completion date: Sep 2021 ➔ Jul 2026

Trial completion date • Trial primary completion date • Hematological Malignancies • Lymphoma • Multiple Myeloma • Oncology

Validation of BCMA-CD19 Compound CAR-T Therapy in SLE Overlap Syndrome: Over 1.5-Year Follow-Up. (PubMed, Stem Cell Rev Rep) - "cCAR therapy effectively eliminates pathogenic cell populations, representing a promising translational strategy for treating refractory SLE and related AIDs."

Journal • ANCA Vasculitis • Glomerulonephritis • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Inflammation • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Sjogren's Syndrome • Systemic Lupus Erythematosus • Thrombocytopenic Purpura • Vasculitis

BCMA-CD19 CCAR T Cell Treatment of Refractory Immune Thrombocytopenia Associated with Autoimmune Diseases (clinicaltrials.gov) - P1 | N=20 | Recruiting | Sponsor: iCell Gene Therapeutics

New P1 trial • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

BCMA-CD19 Compound CAR-T (cCAR) Safely Provides a Complete Humoral Reset Eliminating All Autoantibodies Resulting in Long-term Medication-Free Complete Remission Among Systemic Lupus Erythematosus (SLE) and Lupus Nephritis (LN) Patients (ACR Convergence 2024) - "All LN patients at screening treated with HCQ, glucocorticoid, immunosuppressant; majority with belimumab. BCMA-CD19 cCAR can safely achieve long-term medication-free Complete Remission. Will update dataset at ACR. Further research needed."

Clinical • Glomerulonephritis • Hematological Malignancies • Immunology • Infectious Disease • Inflammatory Arthritis • Lupus • Lupus Nephritis • Lymphoma • Nephrology • Novel Coronavirus Disease • Oncology • Renal Disease • Systemic Lupus Erythematosus

iCell Presents Positive New Follow-up Clinical Data Confirming BCMA CD19 cCAR Safely Delivers Long-term Medication Free Complete Remission from Systemic Lupus Erythematosus & Lupus Nephritis at EULAR 2024 (Businesswire) - P1 | N=15 | NCT05474885 | Sponsor: iCell Gene Therapeutics | "iCell Gene Therapeutics...today announced new longer-term follow-up data from the Company’s BCMA CD19 compound chimeric antigen receptor (cCAR) T cell immunotherapy development program....'We are excited to progress this therapy in clinical development and plan to file INDs in the United States and China soon.'...Overall, the cCAR therapy was found to be generally safe and well-tolerated. The 10 LN patients receiving the target dose achieved depletion of B cells from peripheral blood within 10 days post-cCAR treatment and depletion of immunoglobulin within 42 days, and complete recovery of B cells and IgM was seen within 2-6 months post-cCAR, IgA within a year and IgG is >400 mg/dL in all patients....The data suggest that cCAR therapy was safe and effective in inducing MFR and depleting disease-causing autoantibodies in SLE and LN patients."

P1 data • Lupus Nephritis • Systemic Lupus Erythematosus

iCell Gene Therapeutics Announces Positive Clinical Data from Investigator Initiated Phase 1 Trial Evaluating BCMA-CD19 Compound CAR in Patients with Systemic Lupus Erythematosus/Lupus Nephritis Published in Annals of the Rheumatic Diseases (Businesswire) - P1 | N=15 | NCT05474885 | Sponsor: iCell Gene Therapeutics | "iCell Gene Therapeutics...today announced the publication in Annals of the Rheumatic Diseases of positive results of an investigator initiated clinical trial (IIT) for its BCMA-CD19 compound chimeric antigen receptor (cCAR) T cell immunotherapy. The clinical trial evaluated the safety and efficacy of a complete humoral reset of both long-lived plasma cells and B cells in 13 systemic lupus erythematosus (SLE) patients treated with iCell’s cCAR, including 11 patients with SLE and lupus nephritis (LN)....iCell considers this IIT to be a successful proof of concept; the Company plans to file an investigational new drug application (IND) for its BCMA-CD19 cCAR in the United States and China....'We look forward to receiving further follow up data confirming the promising results to date, and we plan to present this data on June 12th at the European Alliance of Associations for Rheumatology (EULAR) 2024 Congress.'"

P1 data • Systemic Lupus Erythematosus

BCMA-CD19 compound CAR T cells for systemic lupus erythematosus: a phase 1 open-label clinical trial. (PubMed, Ann Rheum Dis) - "Data suggest that cCAR therapy was safe and effective in inducing MFR and depleting disease-causing autoantibodies in patients with SLE."

CAR T-Cell Therapy • Journal • P1 data • Glomerulonephritis • Hematological Malignancies • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Lymphoma • Nephrology • Oncology • Systemic Lupus Erythematosus

Immune Reset in B Cell mediated autoimmune diseases using BCMA-CD19 Compound CAR (cCAR) in an open label phase I clinical trial (FOCIS 2023) - No abstract available

Clinical • P1 data • Immunology

BCMA-CD19 cCAR T Cell Treatment of Relapsed/Refractory Systemic Lupus Erythematosus (SLE) (clinicaltrials.gov) - P1 | N=15 | Recruiting | Sponsor: iCell Gene Therapeutics

CAR T-Cell Therapy • New P1 trial • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus • CD19

Treatment of Systemic Lupus Erythematosus using BCMA-CD19 Compound CAR. (PubMed, Stem Cell Rev Rep) - No abstract available

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