Cabaletta Bio Announces FDA Granted Orphan Drug Designation to CABA-201 for Treatment of Myositis

PHILADELPHIA, Feb. 01, 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 idiopathic inflammatory myopathies (IIM, or myositis). CABA-201 is in development as a potential treatment for autoimmune diseases driven by B cells. Four RESET (REstoring SElf-Tolerance) 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.”

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-Myositis Trial
The RESET-Myositis trial is a Phase 1/2 open-label study of CABA-201 in subjects with active idiopathic inflammatory myopathy (IIM, or myositis), including the subtypes of dermatomyositis (DM), anti-synthetase syndrome (ASyS) and immune-mediated necrotizing myopathy (IMNM). Subjects will receive a one-time infusion of CABA-201 at a dose of 1 x 10⁶ cells/kg, preceded by a standard preconditioning regimen of fludarabine and cyclophosphamide. Key inclusion criteria include patients between ages 18 to 65 (inclusive), evidence of active disease and disease activity despite prior or current treatment with standard of care treatments. Key exclusion criteria include cancer-associated myositis, significant lung or cardiac impairment, treatment with a B cell depleting agent within the prior approximately six months or treatment with a biologic agent within the prior approximately three months. As part of Cabaletta’s CARTA (Chimeric Antigen Receptor T cells for Autoimmunity) strategy, this trial is intended to evaluate the potential ability of CABA-201 to transiently, but fully, eliminate B cells, potentially enabling durable remissions via a “reset” of the immune system.