Cabaletta: FDA grants ODD to CABA-201 for myositis treatment

Cabaletta Bio announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, for the treatment of idiopathic inflammatory myopathies . CABA-201 is in development as a potential treatment for autoimmune diseases driven by B cells. Four RESET Phase 1/2 trials are advancing for the evaluation of CABA-201 across multiple autoimmune conditions, including the Phase 1/2 RESET-Myositis trial. “Myositis, believed to be driven by B cells, is a severe and potentially fatal autoimmune disease for which no curative therapy exists. Current treatment options provide modest efficacy, with a significant portion of diagnosed patients having an inadequate response to treatment, thus, there is a clear need for innovative medicines that can meaningfully change the treatment paradigm,” said David J. Chang, M.D., Chief Medical Officer of Cabaletta. “CABA-201 is designed to deeply and transiently deplete CD19-positive B cells, which may enable an immune system reset, and has the potential to deliver durable remission off therapy in patients diagnosed with myositis and other autoimmune diseases where B cells play a role. Orphan Drug Designation is an important recognition for investigational therapies for rare diseases and provides us with potentially valuable benefits as we seek to make a difference in the lives of patients and develop the first targeted, and potentially curative, cell therapy for patients with autoimmune diseases.”