Quizartinib + decitabine + venetoclax in FLT3-ITD AML: Phase I/II trial results

Results from a phase I/II trial (NCT03661307) evaluating quizartinib + decitabine + venetoclax for the treatment of FLT3-internal tandem duplication (FLT3-ITD) acute myeloid leukemia (AML) were presented by Musa Yilmaz at the European Hematology Association (EHA) 2026 Congress, June 11–14, 2026, Stockholm, SE. The primary objective was to determine the recommended phase II dose (RP2D) and optimal dosing schedule in patients with newly diagnosed (ND; n = 42) or relapsed/refractory (R/R; n = 46) FLT3-ITD AML.

Key data: The RP2D for quizartinib was 26.5 mg, or 17.7 mg with a strong CYP3A4 inhibitor. Modified composite complete remission (mCRc) rates were 91% in the ND cohort and 61% in the R/R cohort, with complete remission (CR) achieved in 72% and 10% of patients, respectively. In the ND cohort, median relapse-free survival (RFS) was 25 months and median overall survival (OS) was 36 months. Median OS was not reached in patients who underwent stem cell transplantation (SCT) in first complete remission (CR1), compared with 36 months in those who did not. In the R/R cohort, the median OS was 6.3 months overall: 8.1 months among responders, and 3.4 months among non-responders. The most common Grade ≥3 non-hematologic adverse events (AEs) were infections, including febrile neutropenia (38% and 65%), other infections (29% and 39%), and lung infections (26% and 80%) in the ND and R/R cohorts, respectively. Grade ≥3 corrected QT interval (QTc) prolongation occurred in 5% and 13% of patients, respectively.

Key learning: Quizartinib + decitabine + venetoclax was associated with clinical activity in FLT3-ITD AML, both in ND and heavily pretreated patient populations, supporting continued evaluation of this regimen.