New study finds anti-CD19 CAR-T therapy, Zorpo-cel, safe and effective treatment for people with systemic lupus erythematosus
A new study exploring the investigational therapy, Zorpo-cel, met its primary safety and secondary efficacy endpoints in people with progressive and treatment-resistant systemic sclerosis (SSc), idiopathic inflammatory myopathies (IIM) and systemic lupus erythematosus (SLE). Zorpocabtagene autoleucel (Zorpo-cel, also known as MB-CART19.1) is an autologous CD19 CAR-T cell product designed to target B cells in people with autoimmune diseases.
The Phase 1/2 CASTLE (CAR-T cells in systemic B cell mediated autoimmune disease) basket trial consisted of 24 participants (10 with SLE, 9 with SSc and 5 with IIM) with highly active, severe and treatment-resistant autoimmune diseases. Participants had a median age of 39 years, a disease duration of four years, all having discontinued prior immunosuppressive therapy before receiving a single infusion of Zorpo-cel (following standard lymphodepleting chemotherapy). CASTLE’s primary safety outcomes included the rate of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), and secondary clinical efficacy outcomes were disease-specific clinical remission or response criteria at 24 weeks. The one-time treatment of Zorpo-cel led to drug-free remission within the 24-week study period. Additionally, all participants remained free of glucocorticoids and any other immunosuppressive treatment during the entire 24-week observation period.
The CASTLE study provides important new insights into CD19 CAR-T cell therapy in autoimmune disease and paves the way for future pivotal studies of Zorpo-cel. More research is needed to learn if T-cell engagers targeting B cells and plasma cells can reset the immune system and enable sustained drug-free remission, as seen with CAR-T cells. Continue to follow the Lupus Foundation of America for updates on lupus drug developments. Learn more about CAR-T cell therapy for treating lupus.
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