Cabaletta Bio (NASDAQ:CABA)plummets 34.5% premarket after announcing top-line data on biologic activity from the two lowest dose cohorts in the DesCAARTes Phase 1 trial of DSG3-CAART for the treatment of patients with mucosal Pemphigus Vulgaris (mPV).
In cohort A1, participants received 20 million DSG3-CAART cells:
Two of three participants had DSG3 autoantibody levels that rose ≥20% along with disease activity scores that worsened within six months after DSG3-CAART infusion. Both participants reduced or discontinued selected systemic therapies prior to DSG3-CAART infusion, as required by the protocol.
One of three participants had modest DSG3 autoantibody levels and mild disease activity and had a negative DSG3 level at six months along with disease activity scores of zero on both scales with no systemic medications for mPV since DSG3-CAART infusion.
In cohort A2, participants received 100 million DSG3-CAART cells:
Two of three participants maintained stable DSG3 autoantibody levels that have not persistently changed +/- 20% of pre-infusion levels. Through the six month follow-up period, one of these patients maintained stable disease activity scores, while the other maintained stable scores initially before subsequently worsening.
One of three participants had DSG3 autoantibody levels that rose ≥20% from pre-infusion levels despite stable disease activity scores. This participant subsequently received systemic medication to improve disease activity after DSG3-CAART infusion.
mPV is a rare autoimmune blistering disease that is characterized by the loss of adhesion between cells of the skin or mucous membranes.