Maryland biotech GlycoMimetics has granted Pfizer an exclusive global license to its investigational treatment for vaso-occlusive crisis associated with sickle cell disease, currently in mid-stage clinical trials. The compound, GMI-1070, is a pan-selectin antagonist that has received Orphan Drug and Fast Track status from the U.S. Food and Drug Administration.

Besides the vaso-occlusive crisis indication, Pfizer will have exclusive rights to develop the drug for other diseases. GlycoMimetics will remain responsible for completion of the ongoing proof-of-concept trial under Pfizer’s oversight, and Pfizer will then assume all further development and commercialization responsibilities.

GlycoMimetics stands to make up to $340 million, which includes an upfront payment as well as development, regulatory and commercial milestones. The biotech will also be eligible for royalties on sales of any commercialized products.

Vaso-occlusive crisis can last five to six days and results in more than 75,000 hospitalizations each year in the United States. It causes pain and tissue damage and leads to multiple organ damage, requiring a lifetime of pain medication. GlycoMimetics’ drug is thought to inhibit an interaction that is a key early step in the inflammatory process leading to a crisis event. Preclinical studies showed it restored blood flow to affected vessels in animals with sickle cell disease experiencing vaso-occlusive crisis.

GlycoMimetics sees the tie-up with Pfizer as an important validation of its chemistry drug discovery platform. For Pfizer, the compound and partnership “are emblematic of our strategy in rare disease, targeting areas of high unmet need to deliver improved patient outcomes,” says Yvonne Greenstreet, senior vice president and head of the Medicines Development Group within Pfizer’s Specialty Care business unit.