Summary
The purpose of this Phase I, multicenter study is to evaluate the safety,
pharmacokinetics, pharmacodynamics and clinical activity of AG-120 in advanced
hematologic malignancies that harbor an IDH1 mutation. The first portion of the study is
a dose escalation phase where cohorts of patients will receive ascending oral doses of
AG-120 to determine maximum tolerated dose (MTD) and/or the recommended Phase II dose.
The second portion of the study is a dose expansion phase where four cohorts of patients
will receive AG-120 to further evaluate the safety, tolerability, and clinical activity
of the recommended Phase II dose. Additionally, the study includes a substudy evaluating
the safety and tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of
AG-120 in subjects with relapsed or refractory myelodysplastic syndrome with an IDH1
mutation. Anticipated time on study treatment is until disease progression or
unacceptable toxicity occurs.