In 12 patients, the mean decrease at six months in a 60-point measure of myasthenia gravis disease activity — known as the Myasthenia Gravis Composite score — was greater than six points, the biotech said Tuesday morning. At the six-month mark, four out of 12 participants were observed to have minimum symptoms, according to Cartesian. Two responder patients from the primary analysis were lost to follow-up.
The company also reported that it retreated three patients, two of whom have minimum symptoms two years after first receiving treatment. The third patient was retreated more recently and had a six-point improvement on their MGC score two months after retreatment.
“Targeted therapy over the last five years, six years, while very gratifying and transformational, still require multi-dosing on an ongoing basis in order to maintain effect,” James Howard, the lead investigator from the University of North Carolina School of Medicine, said during an analyst call. “The ability to treat our patients so infrequently and still maintain quality of life and prolonged duration of effect is exceptionally gratifying.”
Back in July, Cartesian announced that the Phase 2b study hit its primary endpoint, with 10 of 14 patients achieving at least a five-point improvement in their Myasthenia Gravis Composite score. By comparison, only three of 12 in the placebo group achieved that improvement.
The biotech is now planning its pivotal Phase 3 study, which it calls AURORA. The study will enroll approximately 100 patients with myasthenia gravis who have blood test results showing the presence of certain autoantibodies. Cartesian expects to begin the trial in the first half of next year.
Other companies like Kyverna Therapeutics and Cabaletta Bio are developing CAR-T cell therapies for myasthenia gravis amid burgeoning interest in the autoimmune cell therapy field. Unlike Cartesian, their treatment regimens are more similar to conventional CAR-T therapies in that they require preconditioning chemotherapy and can potentially lead to immunotherapy-related side effects.
Outside of cell therapy approaches, the myasthenia gravis category has become competitive as well, with companies like argenx, UCB, Johnson & Johnson and Immunovant either marketing new treatments or developing drugs for the disease.

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