A Phase I Study of AG-221 in Patients with Advanced Acute Myeloid Leukemia or Myelodysplastic Syndromes with an IDH2 Mutation

Full Title

A Phase 1, Multicenter, Open-Label, Dose-Escalation and Expansion, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-221 in Subjects with Advanced Hematologic Malignancies with an IDH2 Mutation


The purpose of this study is to find the highest dose of an investigational drug called AG-221 that can be given safely in patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) that have continued to grow despite or have returned after receiving standard therapy, and which contain a mutated form of a protein called IDH2. Researchers believe that AG-221 may be more effective and have fewer side effects than additional chemotherapy in these patients.

AG-221 blocks a mutated form of the IDH2 protein. Abnormal IDH2 causes too much of a substance called 2-HG to be produced, which may make these cancers grow. Laboratory studies have shown that AG-221 can reduce the amount of leukemia.


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

  • Patients must have AML or MDS that has persisted or returned despite therapy, or they should have untreated AML and be age 60 or older and unable to receive standard therapy.
  • Patients’ cancers must contain mutated IDH2.
  • At least 2 weeks must have passed since completion of prior chemotherapy or radiation therapy and entry into the study, and patients must have recovered from any serious side effects.
  • Patients must be able to be ambulatory for more than half of their normal waking hours.
  • This study is open to patients age 18 and older.

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