GAITHERSBURG, Md.—GlycoMimetics, Inc. and Pfizer Inc. haveannounced the signing of an exclusive worldwide licensing agreement forGlycoMimetics' investigation compound GMI-1070, a pan-selectin antagonist thatis currently in Phase II development as a treatment for vaso-occlusive crisisassociated with sickle cell disease. The compound has received both Fast-Trackdesignation and Orphan Drug status from the U.S. Food and Drug Administration.
"We are very pleased to partner with Pfizer for theadvancement of GlycoMimetics' lead drug candidate, GMI-1070, which is initiallybeing evaluated in patients with sickle cell disease experiencingvaso-occlusive crisis. This is a major unmet medical need," Rachel King, CEO ofGlycoMimetics, said in a press release. "We value the resources and experiencethat Pfizer brings to the program, and recognize that the agreement is animportant validation of GlycoMimetics' unique chemistry expertise in discoveryof proprietary drug candidates."
Per the terms of the agreement, Pfizer will gain anexclusive worldwide license to GMI-1070 for vaso-occlusive crisis associatedwith sickle cell disease as well as for other diseases for which the compoundmight be developed. GlycoMimetics is responsible for completing the currentPhase II trial under Pfizer's oversight, after which Pfizer will assumedevelopment and commercialization duties. GlycoMimetics stands to receiveapproximately $340 million total, including an upfront payment and development,regulatory and commercial milestones, as well as royalties on any sales.
"This partnership is an important milestone forGlycoMimetics as the company advances its clinical development program," JimBarrett, Ph.D., Chairman of the Board of GlycoMimetics and General Partner, NewEnterprise Associates, said in a press release. "It's a testament to theprogress made to-date with GMI-1070, and will enhance continued development ofthis potential treatment for patients suffering from vaso-occlusive crisis."
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
