Novartis and Bristol Myers Squibb have paused clinical trials of their respective CD19-directed CAR T therapies in autoimmune diseases while they review safety data.
Novartis halted eight studies of rapcabtagene autoleucel, or rap-cel, on August 24 after three patients developed serious immune effector cell-associated hemophagocytic syndrome (IEC-HS). Complications from the events led to the deaths of all three patients, the company confirmed to Reuters.
IEC-HS is a severe inflammatory reaction associated with immune effector cell therapies. Novartis described it as a known, potentially life-threatening risk of CAR T treatment and said it was working with independent safety boards and health authorities to review the cases and identify ways to detect dangerous reactions earlier.
The affected program includes phase II trials in systemic lupus erythematosus and lupus nephritis, systemic sclerosis, ANCA-associated vasculitis, and idiopathic inflammatory myopathies. Phase I/II studies in rheumatoid arthritis and Sjögren’s disease, generalized myasthenia gravis, relapsing multiple sclerosis, and non-active progressive multiple sclerosis have also been paused, according to Fierce Biotech.
Screening, randomization, and treatment administration have stopped while patients already treated in the trials will continue to be monitored. Novartis said oncology studies of rap-cel in leukemia and lymphoma were not affected.
Rap-cel is an autologous therapy manufactured using Novartis’ T-Charge platform, which is designed to reduce the time cells spend in culture before infusion.
Bristol Myers has separately paused enrollment in autoimmune disease trials of zolacabtagene autoleucel, or zola-cel. The company told BioPharma Dive that it had observed “transient and reversible inflammatory events” during routine safety monitoring and implemented the pause as a precaution while reviewing clinical data.
Zola-cel is another autologous CD19-directed therapy and uses Bristol Myers’ rapid-manufacturing NEX-T platform. A phase I report published in February included one case of IEC-HS, BioPharma Dive reported. Bristol Myers said the therapy’s overall safety profile remained consistent with the known risks of CAR T treatments and that it aimed to resume enrollment after completing its review.
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