Cabaletta Bio Announces FDA Granted Orphan Drug Designation to CABA-201 for Treatment of Systemic Sclerosis
PHILADELPHIA, March 20, 2024 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, for the treatment of systemic sclerosis (SSc). CABA-201 is in development as a potential treatment for autoimmune diseases driven by B cells. The RESET™ (REstoring SElf-Tolerance) clinical trial program includes four Phase 1/2 trials advancing for the evaluation of CABA-201 across multiple autoimmune conditions, including the Phase 1/2 RESET-SSc™ trial.
“Patients diagnosed with systemic sclerosis, a rare and life-threatening chronic autoimmune disease characterized by progressive skin and internal organ fibrosis, face limited treatment options as current therapies provide only modest effects and focus on treating the complications associated with the disease. With an average patient survival of 12 years following diagnosis, there is a significant unmet need for new treatment options that focus on eliminating the root cause of the disease to prevent further organ damage for patients,” said David J. Chang, M.D., Chief Medical Officer of Cabaletta. “Based on the role of B cells and the recently published academic clinical data with CD19-CAR T therapy in systemic sclerosis, we believe CABA-201 may transform the treatment for systemic sclerosis. Orphan Drug Designation is an important recognition for investigational therapies for rare diseases and provides us with potentially valuable benefits as we develop CABA-201 for patients with systemic sclerosis.”
The FDA grants Orphan Drug Designation to drugs or biologics intended to treat or prevent rare diseases or conditions that affect fewer than 200,000 individuals in the United States. This designation qualifies Cabaletta for certain incentives, which may include partial tax credit for clinical trial expenditures, waived user fees and potential eligibility for seven years of marketing exclusivity.
About the RESET-SSc™ Trial
The RESET-SSc™ trial is a Phase 1/2 open-label study of CABA-201 in subjects with SSc across two parallel cohorts. The severe skin cohort will include six patients with severe skin involvement, and the organ cohort will include six patients who meet the pulmonary, cardiac, or renal involvement criteria regardless of skin involvement. Subjects will receive a one-time infusion of CABA-201 at a dose of 1 x 106 cells/kg, preceded by a standard