GMI-1070 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.  As inhibition of all three selectin types (E-selectin, L-selectin and P-selectin) may be required for adequate therapeutic activity in certain inflammatory disorders, we believe that our compound’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 selected vaso-occlusive crisis of sickle cell disease as the first indication to explore with GMI-1070. Inflammation is a key mediator of vaso-occlusive crisis, a condition which represents a significant unmet medical need.  Sickle cell disease is one of the most prevalent genetic disorders in the US, affecting over 80,000 people.  It is a chronic condition with substantial morbidity and mortality, responsible for more than 75,000 hospitalizations per year in the US with an average stay of approximately 6 days. GMI-1070 has received both Orphan Drug and Fast Track status for vaso-occlusive crisis from the FDA. 

The main clinical feature of sickle cell disease is periodic painful vaso-occlusive episodes, known as vaso-occlusive crises or pain crises, which result in significant clinical complications.   Treatment for vaso-occlusive crisis consists primarily of supportive therapy, in the form of hydration and pain control, typically requiring extended hospitalization. No other therapies have been effective in aborting a vaso-occlusive crisis once it has begun. GMI-1070 is intended to treat vaso-occlusive crisis by inhibiting the cell activation and enhanced cell adhesion which causes the ischemia and pain.

In 2010, GlycoMimetics, Inc. announced the initiation of a Phase 2 clinical trial of GMI-1070 to study use of the compound to treat vaso-occlusive crisis of sickle cell disease. In late September 2010, GlycoMimetics announced the publication in Blood of preclinical data showing effects of GMI-1070 in an animal model of sickle cell crisis. These preclinical studies demonstrate significant promise for the compound in sickle cell crisis.  Use of GMI-1070 in the mouse sickle cell model resulted in normalized blood flow, reduced inflammation and increased survival.