Cabaletta CAR-T eliminates B cells in autoimmune disease patients without preconditioning

Cabaletta Bio presented initial dose data from a phase 1/2 trial evaluating rese-cel, an autologous CAR-T cell therapy, without preconditioning in three evaluable patients with pemphigus vulgaris, a rare autoimmune disease that causes painful blisters on the body.

The RESET-PV trial is the first study within Cabaletta’s RESET clinical development program to evaluate rese-cel without the use of cyclophosphamide and fludarabine as preconditioning agents. All three patients experienced substantial depletion of B cells within the first month post-infusion, with patients 2 and 3 achieving complete peripheral B cell depletion. In these two patients, rapid reduction in autoantibodies to desmoglein was observed and the increase in peak B cell activating factor (BAFF) was within the range of patients dosed with rese-cel plus preconditioning from pre-infusion through the latest follow-up, suggestive of deep B cell depletion in the tissue.

Rese-cel was generally well tolerated with no immune effector cell-associated neurotoxicity syndrome (ICANS) reported.

“Based on the observed biologic activity and early clinical responses, we plan to first expand patient enrollment in the RESET-PV trial at the current dose and potentially evaluate higher doses of rese-cel in PV patients, as warranted. In addition, we are pursuing the incorporation of no preconditioning regimens in certain other RESET clinical trial program cohorts,” said David J. Chang, M.D., chief medical officer at Cabaletta.

>>>>Read more about the rise of cell and gene therapies in autoimmune diseases in our recent cover story.