A Phase I Study of AG-881 to Treat Acute Myeloid Leukemia or Myelodysplastic Syndromes that Contain Mutated IDH1 or IDH2

Full Title

A Phase 1, Multicenter, Open-Label, Dose-Escalation and Expansion, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-881 in Patients with Advanced Hematologic Malignancies with an IDH1 and/or IDH2 Mutation


The purpose of this study is to find the highest dose of an investigational drug called AG-881 that can be given safely in patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) that contain a mutated form of a protein called IDH. There are two types of IDH, called IDH1 and IDH2. Abnormal IDH1 and IDH2 cause too much of a substance called 2-HG to be produced, which may make these cancers grow. Some people with AML or MDS have mutated forms of both IDH1 and IDH2.

AG-881 blocks mutated forms of the IDH1 and IDH2 proteins and may reduce 2-HG levels in diseased cells to normal levels. It is taken orally (by mouth).


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

  • Patients with AML must have IDH1 and/or IDH2 mutations and experienced continued disease growth despite prior IDH inhibitor therapy (such as AG-221 or AG-120).
  • Patients with MDS must have IDH1 and/or IDH2 mutations and disease that came back or continued to grow despite prior therapy.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for people age 18 and older.

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