Vaso-occlusive crisis is the main clinical feature of sicklecell disease and occurs when circulation is blocked by sickle-shaped bloodcells, 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-basedtherapies exist. Current treatment is limited to supportive therapy such ashydration and pain control. Vaso-occlusive crisis can last five to six days andresults in over 75,000 hospitalizations a year in the United States. GMI-1070is believed to inhibit selectin interactions, a significant step early on inthe inflammatory process that leads to vaso-occlusive crisis, and inpreclinical studies, the compound restored blood flow to affected blood vesselsin animals with vaso-occlusive crisis.
GMI-1070 is a rationally designed glcyomimetic inhibitor ofE-, P- and L-selectins that interferes with the early step in the inflammatoryprocess that leads to leukocyte adhesion and recruitment to inflamed tissue.Two Phase I trials were completed for GMI-1070 in 2009, with no serious adverseevents reported. Preclinical studies are also underway to test GMI-1070 inother diseases such as hematologic malignancies, in which selectin-mediatedcell adhesion and migration is known to play a significant part in the diseaseprocess.
"Pfizer is committed to helping improve the lives ofpatients with rare diseases, and we see potential for GlycoMimetics' GMI-1070to be a significant advance in the treatment of vaso-occlusive crisis of sicklecell disease," said Yvonne Greenstreet, senior vice president and head of theMedicines Development Group within Pfizer's Specialty Care business unit, in apress release. "This experimental compound and partnership are emblematic ofour strategy in rare disease, targeting areas of high unmet need to deliverimproved patient outcomes."
SOURCE: GlycoMimetics press release
