The oral FLT3 inhibitor emavusertib is safe and effective in patients with pretreated acute myeloid leukemia (AML) with FLT3 mutations, according to data from the TakeAim Leukemia trial.
Eric Winer, MD, of the Dana-Farber Cancer Institute, presented preliminary data from the ongoing, open-label, phase I/II trial at the 2024 ASCO® Annual Meeting.
Of 11 patients who received emavusertib at the recommended phase II dose of 300 mg twice daily, eight patients had prior treatment with FLT3 inhibitors.
To evaluate the safety, clinical activity, and molecular characterization of emavusertib, Dr. Winer and colleagues performed next-generation sequencing of 68 genes in bone marrow or peripheral blood mononuclear cells at baseline and on treatment.
Patients who responded to treatment achieved more than 90% bone marrow blast reduction compared with baseline, as well as decreased or undetectable FLT3 internal tandem duplication levels. Emavusertib also decreased the variant allele frequency of RUNX1, NRAS, and TET2, which were identified as common co-mutations in the patient population. Two (18%) patients experienced grade 3 or higher treatment-related adverse events.
“AML patients with FLT3 [mutations] demonstrated increased bone marrow blast reductions (P ≤ 0.05) on emavusertib treatment when compared to FLT3 [wild type] AML patients,” wrote Dr. Winer and colleagues. “Mutational profiles are suggestive of disease-modifying activity of emavusertib.”
Funding for this study was provided by Curis, Inc.
Reference
Winer ES, Verma A, Groepper S, et al. Preliminary safety, efficacy and molecular characterization of emavusertib (CA-4948) in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) with FLT3 mutation (FLT3m). Abstract #6539. Presented at the 2024 ASCO® Annual Meeting; May 31-June 4, 2024; Chicago, Illinois