Acute myeloid leukemia (AML) is a kind of cancer diseases, a cancer of the myeloid line of blood cells. AML seriously affects patients’ life as we previously mentioned in our blog. Thus, it is necessary to find out more anti-acute myeloid leukemia agents.

As we all konw, IDH (Isocitrate Dehydrogenase) is an enzyme, catalyzes decarboxylation of isocitrate to produce alpha-ketoglutarate (α-ketoglutarate) and CO₂. It also contains IDH1, IDH2 and IDH3. IDH1 reversely catalyzes the reaction of α-KG to isocitrate.

However, mutation of IDH1 causes loss of normal enzymatic function. What is worse, mutant IDH1 involves in cancer. IDH1 harbors mutations in human acute myeloid leukemia. IDH1 mutation occurs in the early phase of tumorigenesis.

Therefore, it is possible to discover some compounds as IDH1 mutation inhibitors. Fortunately, FDA has approved several agents to treat acute myeloid leukemia. Even so, we still need to explore more effective agents to deal with such complicated diseases.

Olutasidenib (FT-2102) is a highly potent, selective small molecule inhibitor of IDH1 mutations. Scientists use it in the research of human acute myeloid leukemia. To our delight, it has entered clinical research. In the phase 1/2 study, Olutasidenib is well tolerated both as a sing-treatment and in combination with azacitidine.

Overall, Olutasidenib exhibits favorable safety and PK/PD. It inhibits IDH1 mutations in AML patients. Further study need to be done to explore more potential in the treatment of acute myeloid leukemia.