The GlycoMimetics pipeline is focused on applying our specialized platform technology to the development of drugs to treat rare or orphan diseases. The drug candidates are first-in-class proprietary small molecules.  All of GlycoMimetics' compounds have been generated internally, leveraging the company’s in-depth understanding of glycobiology, or the study of carbohydrate biology.

GlycoMimetics has designed and synthesized a novel class of patented anti-inflammatory compounds that are selectin antagonists. Selectins play a critical role in a broad range of chronic and acute diseases and conditions.  From this class, GlycoMimetics selected GMI-1070. Phase 1 clinical trials are complete, as is a pilot study in patients with sickle cell disease.  A Phase 2 study is now underway in the U.S. and Canada to evaluate use of GMI-1070 to treat vaso-occlusive crisis of sickle cell disease. The compound has received Orphan Drug and Fast Track status from the FDA.  Based on preclinical studies, GMI-1070 may also have application in a number of other conditions, including certain blood cancers. A preclinical study combining GMI-1070 with Velcade® was chosen for an oral presentation at the 2010 annual meeting of the American Society of Hematology (“ASH”).

GlycoMimetics has designed and synthesized a family of compounds that are antagonists of both E-selectin and CXCR4.  These overlapping pathways have been shown in the scientific literature to play important roles in trafficking and metastasis of cancer cells.  They are also synergistically involved in certain inflammatory conditions.  GMI believes that this family of compounds holds promise as ‘next generation’ versions of drugs now on the market targeting CXCR4.

GlycoMimetics has discovered a second drug candidate, GMI-1051, which may be useful in treating pseudomonas infections in cystic fibrosis patients and others. The compound has been shown to inhibit multiple virulence functions of the bacterial lectins of Pseudomonas aeruginosa in a series of preclinical studies.  GlycoMimetics plans to evaluate this drug for use in conjunction with antibiotics in patients suffering from pseudomonas infections.

For more information about the science of glycobiology, please see our Overview of Glycobiology.