Agios Pharmaceuticals, a biotechnology company based in Cambridge, MA, has received Orphan Drug Designation from the U.S. FDA for its investigational drug, AG-120 for the treatment of IDH1-mutant positive acute myelogenous leukemial (AML). AML is a rare blood cancer originated from the myeloid line of undifferentiated blast cells. It is characterized by rapid disease progression if untreated. AG-120 is an orally available, selective inhibitor of the mutated IDH1 (isocitrate dehydrogenase 1). Wild type IDH1 encodes an enzyme responsible for regeneration of reducing power required for cellular anabolic reactions. However, mutant form of IDH1 protein produces aberrant metabolite, which may contribute to the formation and progression of certain types of cancers including AML. Agios has initiated two Phase I trials of AG-120 in patients with advanced hematologic malignancies and advanced solid tumors. Preliminary data analysis of these studies has shown a favorable safety and tolerability profile.