The drug Cabaletta Bio lupus was approved for trial
CABA-201 is an investigative human CD19-CAR T cell therapy containing 4-1BB, designed to deplete CD19-positive B cells and enhance disease activity in patients with systemic lupus erythematosus (SLE) and lupus nephritis (LN).
Cabaletta has been cleared to begin a phase 1/2 clinical trial CABA-201 for the treatment of six SLE patients with active LN, and in a separate parallel cohort, six patients with active SLE without renal involvement, at starting doses equivalent to those used in Nature Medicine publications. September 2022 of 4-1BB containing the CD19-CAR T construct evaluated in SLE patients.
“Despite advances over the last few decades, treatment options for SLE remain inadequate. There are currently no curative options available to achieve long-lasting remission of the disease. Existing therapies usually result in generalized immunosuppression, require chronic administration, and are often co-administered with steroids and other immunosuppressive drugs to reduce disease burden, which can lead to continued disease activity, treatment-related side effects, and reduced quality of life. said David J. Chang, Cabaletta’s chief medical officer.
“We believe the FDA’s decision to grant fast-track designation to CABA-201 underscores the unmet need for a treatment that has the potential to provide a deep, long-lasting response for people living with lupus and possibly other autoimmune diseases in which B cells contribute to disease. We look forward to starting a phase 1/2 trial for CABA-201 and further evaluating its therapeutic potential for patients in need.”
CABA-201 was designed to be administered as a one-time infusion, to evaluate its potential to temporarily, but completely, eliminate B cells, enabling “immune system reset” with long-lasting remission in patients with SLE. The phase 1/2 clinical trial is an open label study designed to evaluate CABA-201 in SLE subjects with active LN or active SLE without renal involvement.
CABA-201 will be administered at a dose of 1.0 x 106 cells/kg, and this study will enroll 6 subjects in the active LN cohort and 6 subjects in the active SLE cohort without renal involvement, in parallel. Subjects will be treated with a standard preconditioning regimen consisting of fludarabine and cyclophosphamide prior to CABA-201 infusion. This is the first trial to assess Cabaletta’s CARTA (Chimeric Antigen Receptor T cells for Autoimmunity) strategy.
About systemic lupus erythematosus
SLE is a potentially severe chronic autoimmune disease in which the immune system attacks healthy tissue throughout the body, most commonly affecting young women between 15 and 40 years of age with higher frequency and greater severity in people of color. It is characterized by abnormal B cell function and autoantibody production resulting in a variety of clinical manifestations including end organ damage and increased risk of death.
SLE affects approximately 160,000-320,000 patients in the US, with LN being the most common end-organ manifestation, affecting approximately 40% of SLE patients. Among these patients, the risk of end-stage renal disease is approximately 17% and the risk of death is approximately 12%, respectively within 10 years after diagnosis.