Multinational phase 1b-2 study of first-line enasidenib plus azacitidine for AML with IDH2 mutation

Enasidenib is an oral inhibitor of mutant isocitrate dehydrogenase-2 (IDH2) proteins. The multinational phase 1b-2 AG221-AML-005 trial evaluated safety and activity of enasidenib plus azacitidine versus azacitidine alone for newly diagnosed, mutant-IDH2 AML in patients ineligible for intensive chemotherapy. Addition of enasidenib to azacitidine was well tolerated and significantly improved overall response rate (odds ratio 4.9; p=0.0003), suggesting that this regimen can improve outcomes.