Additional Relevant Details The results, presented at the European Hematology Association 21st Congress in Copenhagen, Denmark, showed an overall response rate (combined complete remission (CR) and remission with incomplete recovery (CRi)) of 47 percent among 19 patients, including those who were older than 60 years of age, with primary refractory or relapsed disease, poor cytogenetic risk factors including FLT-3 ITDs, and/or extramedullary disease. Eight of the 19 patients achieved a best clinical response of CR, one patient achieved CRi, and one patient achieved morphologic leukemia-free state (MLFS). Five of the 19 patients went on to receive a hematopoietic stem cell transplant. There was no mortality reported during the treatment phase of 44 days, and no dose-limiting toxic reactions were observed among participants. Pharmacokinetic (PK) data showed a dose-dependent increase in plasma concentrations of GMI-1271, above levels associated with anti-leukemic activity in animal models of AML. In addition, biomarker analysis confirmed on-target activity for GMI-1271 at all dose levels. During dose escalation, patients received only one cycle of treatment with GMI-1271. As part of the Phase 2 expansion, certain patients will be eligible to receive more than one cycle of treatment.