A Phase IB/II Study of AG-221 Alone and with Chemotherapy in Older Patients Newly Diagnosed with Acute Myeloid Leukemia

Full Title

BAML-16-001-S3: A Phase II Study to Assess the Efficacy of the Treatment of IDH2 Mutant AML with Targeted IDH2 Inhibition and Subsequent Response-Driven Addition of Hypomethylating Agent Therapy(WIRB)


Acute myeloid leukemia (AML) that contains an IDH2 genetic mutation without another mutation called NPM1 is challenging to treat successfully. The standard treatment has side effects that may be difficult for older patients to tolerate. In this study, researchers are evaluating the safety and effectiveness of the investigational drug AG-221 in patients age 60 and older with newly diagnosed AML containing the DIH2 mutation without the NPM1 mutation. AG-221 works by blocking the mutated IDH2 protein. Abnormal IDH2 causes too much of a substance called 2-HG to be produced, which may make leukemia cells grow.

Patients will initially receive AG-221 alone. If they achieve complete or partial remission, they will continue to receive this medication as long as they continue to benefit. If they do not achieve complete remission after five cycles of AG-221, they will also receive the drug azacitidine, which is already used to treat leukemia. AG-221 is taken orally (by mouth), while azacitidine is given by injection.


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have newly diagnosed, previously untreated AML containing an IDH2 mutation without an NPM1 mutation.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for patients age 60 and older.

For more information and to inquire about eligibility for this study, please contact Dr. Eytan Stein at 212-639-3314.