Cabaletta Bio, Inc. has recently made a significant announcement regarding its product CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy. The U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to CABA-201 for the treatment of systemic sclerosis (SSC), a rare and life-threatening chronic autoimmune disease characterized by progressive skin and internal organ fibrosis.
The ODD qualification enables Cabaletta for certain incentives, including partial tax credit for clinical trial expenditures, waived user fees, and potential eligibility for seven years of marketing exclusivity. This designation reflects the recognition of investigational therapies for rare diseases and highlights the potential valuable benefits as CABA-201 is developed for patients with systemic sclerosis.
The RESET-SSC™ trial, a phase 1/2 open-label study of CABA-201 in subjects with SSC, consists of two parallel cohorts: the severe skin cohort and the organ cohort. Key inclusion criteria include patients between ages 18 and 70 with evidence of significant skin, pulmonary, renal, or cardiac involvement, whereas key exclusion criteria involve the primary diagnosis of another rheumatic autoimmune disease or recent treatment with certain agents.
CABA-201 is designed to deeply and transiently deplete CD19-positive B cells following a one-time infusion, potentially enabling an "immune system reset" with the potential for durable remission off therapy in patients with autoimmune diseases. The drug has received clearance from the FDA for investigational new drug (IND) applications in multiple autoimmune conditions, including systemic lupus erythematosus (SLE), myositis, systemic sclerosis (SSC), and generalized myasthenia gravis (GMG).
Systemic sclerosis affects approximately 88,000 patients in the U.S. and typically affects middle-aged individuals, particularly women. The standard treatment options for this disease, which have modest effects, include generalized immunosuppressive agents or drugs targeted to specific symptomatic manifestations. The risk of mortality in systemic sclerosis remains high, with an average survival of approximately 12 years following diagnosis.
Cabaletta Bio's focus on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases has brought attention to its CARTA (chimeric antigen receptor T cells for autoimmunity) strategy, with CABA-201 as the lead product candidate being evaluated in the RESET™ clinical trials in various autoimmune diseases.
The company's expanding platform is designed to develop potentially curative therapies that offer deep and durable responses for patients with a broad range of autoimmune diseases. Cabaletta Bio's headquarters and labs are located in Philadelphia, PA. Today the company's shares have moved -0.8% to a price of $17.85. For more information, read the company's full 8-K submission here.
