AML is a fast-growing cancer in which too many abnormal and immature white blood cells are rapidly made in the bone marrow and interfere with the production of normal blood cells. FLT3 is a validated target for AML. Mutation of the FLT3 protein, which is responsible for the proliferation of normal blood cells, is the most common genetic abnormality related to AML, present in approximately one-third of all AML patients and associated with adverse prognoses. The most common FLT3 mutation, the so-called internal tandem duplication, is linked to a particularly poor disease prognosis and is expected to be particularly susceptible to FLT3 inhibition. There are no FLT3 inhibitors currently approved for the treatment of AML.
Ponatinib has been shown to be active in FLT3 mutations—in 2011, ARIAD expects to present data in a small cohort of AML patients from the Phase 1 study of ponatinib.