GlycoMimetics, Inc.

GlycoMimetics, Inc. is developing novel small molecule drugs
to treat orphan diseases.

 
fOR IMMEDIATE RELEASE
 

GlycoMimetics, Inc. Completes Phase 1 Clinical  Studies of Lead Drug Candidate
GMI-1070 Is Granted Orphan Drug Designation for Sickle Cell Disease

GAITHERSBURG, Md. -- March 26, 2009 -- GlycoMimetics, Inc. (GMI), a clinical stage pharmaceutical company that is developing a new class of glycobiology-based therapies for a broad range of indications, today announced it has successfully completed two Phase 1 safety studies of its lead drug candidate GMI-1070 for vaso-occlusive crisis of sickle cell disease. Clinical investigators report no serious adverse events in either of the two studies.

In addition, the Company announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GMI-1070  for the treatment of vaso-occlusive crisis in patients with sickle cell disease. The designation is granted to therapies for diseases that affect fewer than 200,000 patients in the United States. Sponsors of orphan drugs qualify for marketing exclusivity and other incentives created by the Orphan Drug Act. GMI plans to apply to the FDA for Fast Track designation of GMI-1070 later this year.

The Phase 1 studies were comprised of single- and multiple-ascending dose cohorts in healthy volunteers, and were designed to evaluate the safety and pharmacokinetics of GMI-1070.

“Demonstrating the safety of the drug candidate is a critical first milestone, and we’re encouraged to learn that the initial safety profile is quite benign,” said GMI Vice President of Clinical Development Helen Thackray, MD. “No serious adverse events were reported in either study. GMI-1070 also displayed excellent pharmacokinetic properties in both studies. Based on these results, we intend to begin dosing sickle cell patients with GMI-1070 in Phase 2 clinical trials later this year,” said Thackray.

About GlycoMimetics, Inc.
GMI is a privately held biopharmaceutical company that capitalizes on advances in the field of glycobiology. The Company uses rational design of small molecule drugs that mimic the functions of bioactive carbohydrates to develop new drug candidates. The company's initial focus is on therapeutics to treat inflammation, cancer, and infectious diseases. More information is available at the company's web site: http://www.glycomimetics.com.

About GMI-1070
GMI's lead compound, GMI-1070, is a rationally designed glycomimetic inhibitor of E-, P- and L-selectins, and inhibits a key early step in the inflammatory process leading to leukocyte adhesion and recruitment to inflamed tissue. GMI-1070 has been shown to be active in several models of diseases in which leukocyte adhesion and activation play a key role, including vaso-occlusive crisis of sickle cell disease. By inhibiting selectin interactions, GMI-1070 may be able to decrease the enhanced cell adhesion that results in vaso-occlusive crisis. In pre-clinical studies, GMI-1070 restored blood flow to affected vessels of sickle cell animals experiencing vaso-occlusive crisis. The pre-clinical data in sickle cell disease was presented at the annual meeting of the American Society of Hematology in December. GMI-1070 is also being evaluated in preclinical studies for the treatment of certain hematologic cancers, where selectin-mediated cell adhesion and migration is known to play a key role in the disease process. Initiation of Phase 2 clinical trials of GMI-1070 in sickle cell disease is planned in 2009.

About Vaso-Occlusive Crisis
Vaso-occlusive crisis is the main clinical feature of sickle cell disease, often resulting in significant clinical complications, and sometimes death. Currently, there are no mechanism-based therapies for treatment of vaso-occlusive crisis. Treatment consists primarily of supportive therapy in the form of hydration and pain control, typically requiring hospitalization for five to six days. There are over 75,000 hospitalizations per year associated with vaso-occlusive crisis in the US.

 
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