Multicenter phase 1-2 study of axatilimab for cGVHD after failure of at least two prior systemic therapies

Chronic graft-versus-host disease (cGVHD) is the major cause of late morbidity after allogeneic hematopoietic cell transplantation. CSF-1R-dependent macrophages promote cGHVD fibrosis, and their elimination in preclinical studies ameliorated cGVHD. Axatilimab is a humanized monoclonal antibody that inhibits CSF-1R signaling and restrains macrophage development. A multicenter phase 1-2 study evaluated axatilimab for cGVHD after at least two systemic therapy lines. The study provided proof of concept that blocking pathologic macrophage development in cGVHD is safe and can lead to therapeutic benefit in heavily pretreated patients.