Drug intelligence / Profile preview

gilteritinib + idarubicin + cytarabine

Development stage
Phase 2
Lead developer
Astellas Pharma
Modality
Classical Binding Small Molecules → Small Molecules, Covalent Small Molecules → Small Molecules, Nucleic Acid-Directed Small Molecules → Small Molecules
Administration
Oral, Intravenous
01

Overview

**Gilteritinib + idarubicin + cytarabine** is a combination regimen studied for the treatment of acute myeloid leukemia (AML) with FLT3 mutations. Gilteritinib is an oral, small molecule inhibitor of Fms-like tyrosine kinase 3 (FLT3) and AXL, targeting FLT3-ITD and FLT3-TKD mutations, which are prevalent in AML and associated with poor prognosis. Idarubicin is an anthracycline chemotherapy agent inhibiting DNA and RNA polymerase, and cytarabine is a nucleoside analog inhibiting DNA synthesis. The combination has demonstrated potent pro-apoptotic and anti-leukemic efficacy in both preclinical studies and clinical trials, particularly in FLT3-mutated AML, showing higher remission rates compared to chemotherapy alone.

02

Targets

AXL (AXL receptor tyrosine kinase)DNADNA polymerase familyFLT3 (Fms related receptor tyrosine kinase 3)TOP2A (DNA topoisomerase II)

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