Swiss pharmaceutical giant Novartis AG (NVS.US) is experiencing a stark contrast in its neuro-immunology pipeline, as progress in one area is overshadowed by a serious setback in another. The company has been forced to halt eight clinical trials investigating CAR-T therapies following the deaths of three patients, while simultaneously announcing encouraging results for its experimental oral multiple sclerosis medication.
The suspension of the trials was confirmed in late August, affecting studies focused on autoimmune and neurological conditions. The investigational approach, known as chimeric antigen receptor T-cell therapy (CAR-T), involves engineering a patient's own immune cells to recognize and attack specific disease targets.
A spokesperson for the company attributed the pause to the fatalities, which were caused by severe immune reactions. The exact timing of when these adverse events occurred remains undisclosed. The spokesperson indicated that a comprehensive review of the safety incidents is underway, acknowledging the fatal immune response as a known risk associated with CAR-T therapy.
In collaboration with independent safety committees overseeing the suspended studies, the company is also sharing information with regulatory authorities. In a related development, rival Bristol-Myers Squibb (BMY.US) has also paused its own similar CAR-T trials in autoimmune diseases as a precautionary measure.
On a more positive note, the company reported that its investigational oral MS medication achieved success in two late-stage clinical trials. This development positions the drug as a potential new oral treatment option for multiple sclerosis, a chronic autoimmune disease affecting the central nervous system.
Data from the trials showed that patients receiving the treatment, known as remibrutinib, experienced fewer relapses and brain lesions compared to those taking teriflunomide. Importantly, the study reported no signals of liver toxicity. The drug is seen as a strategic successor to its blockbuster MS injection, Kesimpta, providing a more convenient oral alternative.
This positive outcome strengthens the case for BTK inhibitors, a class of drugs designed to suppress harmful immune responses in the disease. The success is notable given that similar drugs developed by Sanofi and Merck failed to outperform teriflunomide on relapse reduction in their late-stage studies. The development also intensifies competition with Roche Holding AG, another major player in the MS market.
The company plans to file for global regulatory approval for remibrutinib in multiple sclerosis, with detailed results to be presented at a medical conference in Toronto later this year. The drug is already approved in the U.S. and Europe for the treatment of chronic spontaneous urticaria, a skin condition characterized by recurrent hives.