Vaso-occlusive crisis is the main clinical feature of sickle cell disease and occurs when circulation is blocked by sickle-shaped blood cells, leading to ischemic injury. It results in severe pain and tissue damage, leading occasionally to patient complications and death, and as of yet, no mechanism-based therapies exist. Current treatment is limited to supportive therapy such as hydration and pain control. Vaso-occlusive crisis can last five to six days and results in over 75,000 hospitalizations a year in the United States. GMI-1070 is believed to inhibit selectin interactions, a significant step early on in the inflammatory process that leads to vaso-occlusive crisis, and in preclinical studies, the compound restored blood flow to affected blood vessels in animals with vaso-occlusive crisis.  

 

GMI-1070 is a rationally designed glcyomimetic inhibitor of E-, P- and L-selectins that interferes with the early step in the inflammatory process that leads to leukocyte adhesion and recruitment to inflamed tissue. Two Phase I trials were completed for GMI-1070 in 2009, with no serious adverse events reported. Preclinical studies are also underway to test GMI-1070 in other diseases such as hematologic malignancies, in which selectin-mediated cell adhesion and migration is known to play a significant part in the disease process.

 

 

"Pfizer is committed to helping improve the lives of patients with rare diseases, and we see potential for GlycoMimetics' GMI-1070 to be a significant advance in the treatment of vaso-occlusive crisis of sickle cell disease," said Yvonne Greenstreet, senior vice president and head of the Medicines Development Group within Pfizer's Specialty Care business unit, in a press release. "This experimental compound and partnership are emblematic of our strategy in rare disease, targeting areas of high unmet need to deliver improved patient outcomes."  

 

 

SOURCE: GlycoMimetics press release