Bristol Myers Squibb and Cabaletta Bio are set to present promising Phase I data on CD19-directed CAR T therapies for severe, refractory lupus at the American College of Rheumatology 2024. BMS’s CC-97540 and Cabaletta Bio’s CABA-201 showed significant efficacy, offering new hope for severe, refractory lupus and other challenging autoimmune diseases. Both studies highlight CAR T’s potential to achieve lasting drug-free remission, marking a groundbreaking advancement in the future treatment of lupus
CC-97540 (BMS-986353) is an investigational CAR T therapy produced with a next-generation NEX-T manufacturing process, while CABA-201 is a fully human CAR T therapy targeting immune-mediated conditions like systemic lupus erythematosus (SLE) and immune-mediated necrotizing myopathy (IMNM).
Based on the abstract, Phase I results for CC-97540 reveal strong efficacy in severe lupus, especially difficult-to-treat renal cases, with all patients achieving notable improvements in Systemic Lupus Erythematosus Disease Activity Index (SLEDAI), Physician Global Assessment (PGA), and Health Assessment Questionnaire Disability Index (HAQ-DI) scores and sustained B cell depletion—without severe side effects. Similarly, Cabaletta Bio’s CABA-201 showed rapid B cell depletion and a 16-point SLEDAI-2K improvement in SLE, all without ongoing immunosuppressives.
Simi Badyal MSc, Senior Competitive Intelligence Immunology Analyst at GlobalData, comments: “CAR T cell therapies have gained immense traction in recent years within immunology and autoimmune diseases, driven by promising results from the Georg Schett study, first published in 2022.”
GlobalData’s latest Cell & Gene Therapies in Rheumatology report reveals that osteoarthritis and lupus have led the cell and gene therapy (CGT) pipeline in rheumatology, with over 70 and 50 assets in development, respectively.”
Lupus shows substantial activity in earlier phases, underscoring a growing focus on addressing the high unmet need in both SLE and lupus nephritis (LN). However, currently only three therapies are approved across these indications: GSK’s Benlysta (belimumab) for both SLE and LN, AstraZeneca’s Saphnelo (anifrolumab) for SLE, and Aurinia’s Lupkynis (voclosporin) for LN.
The early results from BMS and Cabaletta Bio highlight the transformative potential of CAR T cell therapies for severe autoimmune diseases, providing powerful remission pathways at lower doses with improved safety. There is robust CAR T cell expansion, complete B cell depletion, and an improvement in key lupus clinical endpoints such as SLEDAI scores—all without the need for ongoing immunosuppressive therapy.
Badyal concludes, “CAR T cell therapies have the potential to redefine the future treatment for lupus, offering hope for durable, drug-free remission in a disease with historically limited options. BMS’ and Cabaletta Bio’s progress in lupus suggests a paradigm shift: CAR T cells may soon enable tailored, single-infusion treatments that recalibrate the immune system and offer long-term disease remission for lupus patients, a vision once thought improbable.”
Edits made by EP News Bureau