Cabaletta Bio Receives IND Clearance from FDA to Initiate First Clinical Trial of DSG3-CAART in Patients with Mucosal Pemphigus Vulgaris
Cabaletta Bio, Inc., a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies for the treatment of patients with B cell-mediated autoimmune diseases, announced this week that it has received clearance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) to initiate a first-in-human clinical trial of desmoglein 3 chimeric autoantibody receptor T cells (DSG3-CAART) in patients with mucosal pemphigus vulgaris (mPV) to assess the safety and tolerability of DSG3-CAART in these patients. The Company anticipates enrolling the first patient in 2020.
“The FDA’s clearance of our IND for DSG3-CAART is an important milestone for patients with mPV and the first IND clearance for a product candidate from our Cabaletta Approach to selective B cell Ablation (CABA™) platform,” said Steven Nichtberger, M.D., Chief Executive Officer and Co-Founder of Cabaletta Bio. “DSG3-CAART is the first of several CAAR T cell product candidates in our announced pipeline, which includes product candidates targeting patients with MuSK myasthenia gravis, the mucocutaneous form of pemphigus vulgaris (PV), and hemophilia A patients with inhibitors to factor VIII therapy.”
mPV is a potentially fatal, B cell-mediated chronic, rare autoimmune disease that causes painful blisters and sores on mucous membranes of affected patients, leading to severe and sometimes debilitating and life-altering effects. DSG3-CAART is designed to selectively target and eliminate B cells expressing autoantibodies specific for DSG3 that are the cause of mPV while preserving healthy B cell immune function. DSG3-CAART has the potential to generate persistent complete remission off therapy while avoiding the adverse effects of chronic and generalized immunosuppression. Currently available treatment options induce broad immunosuppression, which put the patient at risk of infection and often provide only transient complete remission with subsequent relapses for patients with moderate to severe mPV. Approximately 4,250 patients suffer from mPV in the United States and 6,250 patients in Europe, which accounts for approximately 25% of all PV cases.