Press Release

GlycoMimetics, Inc.

GlycoMimetics Completes Enrollment in Phase 2 Sickle Cell Disease Study with Lead Drug Candidate GMI-1070

GlycoMimetics Completes Enrollment in Phase 2 Sickle Cell Disease Study with Lead Drug Candidate GMI-1070GAITHERSBURG, MD, January 17, 2013GlycoMimetics, Inc. (GMI), a clinical-stage biotechnology company researching a new class of glycobiology-based therapies for a broad range of diseases, today announced that it has completed enrolling patients in its Phase 2 study of GMI-1070, its lead investigational drug candidate. This randomized, double-blinded study examined the efficacy, safety and pharmacokinetics of GMI-1070 in hospitalized sickle cell disease patients experiencing vaso-occlusive crisis (VOC). Since the trial was initiated GlycoMimetics has enrolled 76 patients ages 12 to 65 at 22 trial sites in the United States and Canada. The company anticipates reporting topline data in the second quarter of 2013.

“It is an important step for GlycoMimetics and the investigational sites to have enrolled the final patient in our study of GMI-1070 in VOC of sickle cell disease,” said Helen Thackray, M.D., Vice President of Clinical Development and Chief Medical Officer of GlycoMimetics. “Evaluating GMI-1070 in the treatment of individuals with sickle cell who have been hospitalized with vaso-occlusive crisis represents a critical next step towards potentially fulfilling an important medical need in this historically underserved patient population. The U.S. Food & Drug Administration underscored the importance of advancing new treatments for sickle cell when it granted GMI-1070 with Fast Track designation and Orphan Product status.”

In 2011, GlycoMimetics entered into a worldwide license agreement with Pfizer Inc. (NYSE: PFE) to develop and, if approved by applicable regulatory authorities, to commercialize GMI-1070 for all indications. The agreement provides potential value to GlycoMimetics of over $340 million upon achievement of certain regulatory and commercial milestones, plus tiered, double-digit royalties.

In December 2012 at the American Society of Hematology Annual Meeting, researchers shared data from a pilot study of GMI-1070 via an oral presentation, which was entitled, “Pan-selectin Antagonist GMI-1070 Affects Biomarkers of Adhesion, Activation and the Coagulation Cascade in Sickle Cell Patients at Steady State.” Data from the study demonstrated that GMI-1070 affects a number of biomarkers important in sickle cell disease, including some known to be involved in VOC.

About VOC

GlycoMimetics has selected vaso-occlusive crisis (VOC) of sickle cell disease as the first potential indication to explore with GMI-1070. Inflammation is a key mediator of VOC, a condition that represents a significant unmet medical need. Sickle cell disease is one of the most prevalent genetic disorders in the U.S., affecting over 80,000 people. It is a chronic condition causing substantial illness and death. For example, VOC is responsible for more than 75,000 hospitalizations per year in the U.S. with an average stay of approximately six days.

The main clinical feature of sickle cell disease is periodic painful VOC episodes, known as VOC or pain crises, which result in significant clinical complications. Treatment for VOC today consists primarily of supportive therapy, in the form of hydration and pain control, typically requiring extended hospitalization.

About GlycoMimetics, Inc.

GlycoMimetics is a privately held biotechnology company that capitalizes on advances in the field of glycobiology to treat inflammation, cancer, and infectious diseases. The company uses rational design of small molecule drugs that mimic the functions of bioactive carbohydrates to develop new drug candidates. For additional information, please visit the company’s web site:

Contact: Brian Hahn
Phone: 240-243-1207