GlycoMimetics Initiates Phase 1/2 Clinical Trial of GMI-1271 as Potential Treatment for Acute Myeloid Leukemia in Combination with Chemotherapy

GlycoMimetics, Inc.

announced today that the first patient has been dosed in a Phase 1/2 clinical study designed to evaluate the safety, pharmacokinetics (PK) and efficacy of GMI-1271, a novel and proprietary E-selectin antagonist in the company's pipeline, when used in combination with chemotherapy in patients with acute myeloid leukemia (AML). GlycoMimetics is initially exploring the clinical use of the drug candidate in hematologic malignancies following the successful completion of a Phase 1 healthy volunteer study late last year. The Company announced last week that the U.S. Food & Drug Administration (FDA) has granted Orphan Drug designation to GMI-1271 for treatment of AML. "We have demonstrated a very attractive preclinical profile for GMI-1271, with research findings presented through four oral presentations and a poster at the 56th ASH Annual Meeting in December 2014. These data substantiate the focus on E-selectin as a potential target for blood-related malignancies and for solid tumors at risk of metastasis," said Helen Thackray, M.D., Vice President of Clinical Development and Chief Medical Officer at GlycoMimetics. "Based on our preclinical data and on a benign safety profile in Phase 1, we believe that GMI-1271 has the potential to be an important new therapy for people with certain blood cancers." This Phase 1/2, open-label multicenter study is designed to determine safety, PK and efficacy of GMI-1271 in combination with chemotherapy in male and female adult patients with AML. Study sites are located in the United States, Australia and Ireland. While the primary objective is to analyze safety, additional endpoints include: mobilization of AML blasts, effect on neutropenia, time to and duration of remission, evaluation of event-free survival and evaluation of the overall survival probability at six- and 12-months. Approximately 77 subjects will be enrolled. The study will include a dose escalation phase followed by expansion of the study once the dose for the Phase 2 portion has been selected.