Cabaletta Bio, Inc. (Nasdaq: CABA) has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for CABA-201, an investigational therapy developed to treat idiopathic inflammatory myopathies (IIM), commonly referred to as myositis. CABA-201 is a fully human CD19-CAR T cell therapy currently under development for autoimmune diseases associated with B cells.

Myositis is a severe autoimmune disease lacking a curative therapy, often with fatal outcomes. Existing treatment options have limited efficacy, leaving a substantial number of patients with inadequate responses. CABA-201 aims to address this gap by targeting CD19-positive B cells, potentially enabling an immune system reset and providing durable remission off therapy.

Dr. David J. Chang, Chief Medical Officer of Cabaletta, emphasized the critical need for innovative treatments in myositis, highlighting the potential of CABA-201 to transform the treatment landscape. Orphan Drug Designation is a significant recognition for rare disease therapies, providing certain incentives to the developer, such as partial tax credits, waived user fees, and potential eligibility for seven years of marketing exclusivity.

The RESET-Myositis™ trial, a Phase 1/2 study, is assessing CABA-201 in individuals with active myositis, including subtypes like dermatomyositis and immune-mediated necrotizing myopathy. The trial involves a one-time infusion of CABA-201 preceded by a standard preconditioning regimen. The CARTA (Chimeric Antigen Receptor T cells for Autoimmunity) strategy guides the trial, aiming to evaluate CABA-201's potential to fully eliminate B cells and achieve durable remissions through an immune system "reset."

CABA-201 has also received FDA clearance for Investigational New Drug applications in other autoimmune conditions, including systemic lupus erythematosus, systemic sclerosis, and generalized myasthenia gravis. The therapy is part of Cabaletta's broader approach, involving four Phase 1/2 trials across multiple autoimmune conditions, employing a parallel cohort design with a consistent starting dose.

Myositis encompasses a group of autoimmune diseases characterized by muscle inflammation and weakness, with potential impacts on other organs. Three subtypes, dermatomyositis, anti-synthetase syndrome, and immune-mediated necrotizing myopathy, affect around 66,000 patients in the U.S., often leading to severe functional impairment and life-threatening situations. Current treatments involve immune suppression medications and intensive therapies, but a significant portion of patients remains refractory to existing treatments.

 

Source: globenewswire.com

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