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GlycoMimetics Announces Presentation of Rivipansel Data in Pediatric Patients at American Society of Pediatric Hematology Oncology 27th Annual Meeting

May 15, 2014

- Symposium presentation highlights potential role of rivipansel (GMI-1070), GlycoMimetics’ lead drug candidate, in addressing complications of vaso-occlusive crisis in pediatric patients with sickle cell disease -

GAITHERSBURG, Md.--(BUSINESS WIRE)-- GlycoMimetics, Inc. (NASDAQ: GLYC) announced today that data from a Phase 2 clinical trial of its lead drug candidate rivipansel (GMI-1070) in pediatric patients has been presented at the American Society of Pediatric Hematology Oncology (ASPHO) 27th Annual Meeting. As part of the symposium entitled, “Sickle Cell Disease: Saving the Brain and Treating the Pain,” Timothy McCavit, M.D., M.S., Assistant Professor of Pediatric Hematology-Oncology at UT Southwestern Medical Center, highlighted the results observed in a randomized, multi-center, double-blind, adaptive Phase 2 study of rivipansel in pediatric sickle cell disease patients with vaso-occlusive crisis (VOC). The ASPHO Meeting is taking place May 14 through May 17 at the Palmer House Hilton in Chicago.

“Because sickle cell disease is genetic with symptoms presenting in patients at very young ages, there is a huge unmet need for managing its symptoms in pediatric populations. Dr. McCavit’s presentation at the ASPHO meeting underscores the potential role of rivipansel in addressing VOC in pediatric patients,” said Helen Thackray, M.D., Vice President of Clinical Development and Chief Medical Officer at GlycoMimetics. “We remain committed to supporting the advancing study of this potential therapy in both children and adults and look forward to continued research through the Phase 3 study, which will be led by our collaborator Pfizer, Inc.”

Dr. McCavit’s presentation highlighted data from study participants aged 12 to 18 years, with findings similar to those observed among the adult population treated with rivipansel in terms of improving time to resolution of VOC, time to discharge, and time to reduction in pain. In these pediatric patients, researchers observed a greater than 50% reduction in time to transition to oral pain medications, as well as a reduction in time to hospital discharge, in each case compared to standard treatment for pain in pediatric subjects. Statistical significance was achieved for time to transition to oral pain medications (p=0.037), while the results for time to discharge and time to resolution of VOC, the primary endpoint, were just outside of the range for statistical significance (p=0.06). Safety results for the pediatric subjects were consistent with prior findings in treatment of adult patients.

Rivipansel has previously received both Orphan Drug and Fast Track status for the treatment of VOC from the U.S. Food & Drug Administration (FDA), and Orphan Product status in the European Union. GlycoMimetics is developing rivipansel in collaboration with Pfizer, Inc.

The ASPHO annual meeting symposium on Sickle Cell Disease was held on Wednesday, May 14 from 5:45-7:45 p.m. Central Time. Information is featured at:

About Sickle Cell Disease

There are more than 90,000 people in the U.S. with sickle cell disease, and many of them have multiple, acute VOCs annually. These painful crises result in more than 73,000 hospitalizations per year in the U.S., with an average stay of approximately six days.

About Rivipansel

Rivipansel is being developed in collaboration with Pfizer to treat VOC of sickle cell disease. The compound is a synthetic glycomimetic molecule, which was rationally designed to inhibit all three selectin types (a pan-selectin inhibitor). Selectins are glycoprotein cell adhesion molecules implicated in inflammatory processes. To achieve adequate therapeutic activity in certain inflammatory disorders, inhibition of all three selectin types (E-selectin, L-selectin and P-selectin) may be required. GlycoMimetics believes that rivipansel’s ability to inhibit all selectins will provide distinct advantages over other approaches that target only one selectin, or which are so broadly active as to be non-specific.

GlycoMimetics has conducted a Phase 2 randomized, double-blinded study examining the efficacy, safety and pharmacokinetics of rivipansel in hospitalized sickle cell disease patients experiencing VOC. GlycoMimetics enrolled 76 patients ages 12 to 60 at 22 trial sites in the United States and Canada. The company reported topline data from the trial in April 2013 and presented full data from the clinical trial in two oral presentations and one poster presentation at the December 2013 meeting of the American Society of Hematology (ASH). One of the oral presentations was selected to be among “Best of ASH.” In the Phase 2 trial, patients treated with GMI-1070 experienced reductions in time to reach resolution of VOC, length of hospital stay and use of opioid analgesics for pain management, in each case as compared to patients receiving placebo.

About GlycoMimetics, Inc.

GlycoMimetics is a clinical stage biotechnology company focused on the discovery and development of novel glycomimetic drugs to address unmet medical needs resulting from diseases in which carbohydrate biology plays a key role. Glycomimetics are molecules that mimic the structure of carbohydrates involved in important biological processes. Using its expertise in carbohydrate chemistry and knowledge of carbohydrate biology, GlycoMimetics is developing a pipeline of glycomimetic drug candidates that inhibit disease-related functions of carbohydrates, such as the roles they play in inflammation, cancer and infection.

This press release contains forward-looking statements regarding GlycoMimetics’ planned activities with respect to the clinical development of rivipansel and other matters. Actual results may differ materially from those in these forward-looking statements. For a further description of the risks associated with these statements, as well as other risks facing GlycoMimetics, please see the risk factors described in the Company’s quarterly report on form 10-Q that was filed with the U.S. Securities and Exchange Commission on May 9, 2014, and other filings the company makes with the SEC from time to time. Forward-looking statements speak only as of the date of this release, and GlycoMimetics undertakes no obligation to update or revise these statements, except as may be required by law.

GlycoMimetics, Inc.

Brian Hahn, 240-243-1207

Source: GlycoMimetics, Inc.

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