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.