NEW YORK – Cabaletta Bio on Monday said the US Food and Drug Administration has cleared its investigational new drug (IND) application for CABA-201, opening the way for a clinical trial of the autologous CAR T-cell therapy in generalized myasthenia gravis (gMG).
CABA-201 is designed to target CD19 and eliminate B cells in patients with gMG. The rare and chronic autoimmune disease causes the body's autoantibodies to interfere with protein signaling at the neuromuscular junction and disrupt nerve-muscle communication. This, in turn, leads to muscle weakness and loss of function.
With IND clearance from the FDA, Philadelphia-based Cabaletta will proceed with an open-label Phase I/II study, which will test the cell therapy's activity in two cohorts based on patients' autoantibody status. In one cohort, researchers will enroll six patients with acetylcholine receptor (AChR) antibody-positive gMG and, in another, they will recruit six patients with AChR antibody-negative gMG.
The latest decision from the FDA marks the fourth IND clearance Cabaletta has gained for CABA-201. The firm is also studying the cell therapy as a treatment for systemic lupus erythematosus, myositis, and systemic sclerosis and plans to release initial clinical data from the lupus or myositis trials in the first half of next year.
"We are continuing to expand our CABA-201 portfolio beyond rheumatology and into neurology," Cabaletta CEO and Cofounder Steven Nichtberger said in a statement. "We believe there is an unmet need for a treatment option like CABA-201 that may provide a deep and durable, perhaps curative, outcome with a single dose in patients with gMG."