A phase I/II study of ARGX-110 in patients with relapsed/refractory acute myeloid leukemia

Blood Cancer
Adrian Ochsenbein, MD
University Hospital, Inselspital


Acute myeloid Leukemia (AML) is an aggressive and fatal blood cancer and while standard chemotherapy can induce a complete remission in 50 to 75% of younger patients, the majority of patients eventually relapse and die of recurrent disease. This is especially true for older patients who are not fit for the intense treatment or allogeneic stem cell transplant. In the face of highly toxic chemotherapies, on average less than 30% of AML patients survive long-term. This phase I study will assess the safety of an experimental new drug, ARGX-110, an immunotherapy, in combination with azacytidine, a chemotherapeutic, in patients with AML and high-risk myelodysplastic syndrome (MDS). In AML, two proteins called CD27 and CD70 bind to increase cell division and a malignant state. ARGX-110 targets CD70, preventing the interaction between CD27 and CD70, and thus preventing uncontrolled cell growth. Furthermore, azacytidine has been found to increase the amount of CD70 on AML cells, providing more targets for ARGX-110, and potentially making ARGX-110 even more effective.

Trial Registartion: ClinicalTrials.gov Identifier: NCT03030612