Novartis and Bristol Myers Squibb have independently halted multiple clinical trials investigating their respective CAR-T cell therapies for autoimmune conditions. Novartis initiated holds on its rap-cel trials on August 24th after identifying three cases of immune effector cell-associated hemophagocytic syndrome (IEC-HS), a rare and potentially fatal immune reaction. These trials were evaluating rap-cel for lupus, myasthenia gravis, and multiple sclerosis. The company is actively collaborating with regulators and is conducting a thorough review of the accumulated safety and clinical data.
Bristol Myers Squibb also voluntarily paused enrollment in its zola-cel trials for autoimmune diseases. This decision was made as a precautionary measure to allow for a comprehensive review of clinical data, following the detection of transient and reversible inflammatory events during routine safety monitoring. Despite the pause, Bristol Myers Squibb maintains confidence in zola-cel, highlighting its potential for “transformational, treatment-free responses” in systemic lupus erythematosus and other autoimmune diseases. A prior Phase 1 study of zola-cel had also reported one case of IEC-HS, but the company asserted its safety profile remained consistent with known CAR-T therapies.
Both rap-cel and zola-cel utilize advanced CAR-T technology aimed at accelerating production compared to earlier generation products. Analyst Sami Corwin from William Blair suggested that this expedited production process might contribute to increased cell expansion and the observed toxicities, though other factors could also be involved. Both companies are prominent in cell therapy development, having brought cancer treatments to market and are now leading the effort to apply these therapies to autoimmune diseases. Other biotechs like Cabaletta Bio and Kyverna Therapeutics are also advancing their own CAR-T therapies for various autoimmune conditions.
