Six patients achieved composite complete remission (complete remission [CR] + CR with incomplete hematologic recovery + CR with incomplete platelet recovery), 2 of whom proceeded to hematopoietic cell transplantation with long-term leukemia-free survival. This trial was registered at www.clinicaltrials.gov as #NCT02864290.
Similarly, the combination of ASP1235, venetoclax and azacitidine showed a superior anti-tumor effect in a THP-1 xenograft model without obvious body weight loss. These findings provide supportive evidence that the triple combination of ASP1235, venetoclax and azacitidine would improve the clinical outcome of ASP1235 monotherapy and venetoclax plus azacitidine regimen in AML patients.