GlycoMimetics, Inc. Inks Licensing Deal Worth Up to $340 Million With Pfizer

GlycoMimetics, Inc. recently announced it has entered into an exclusive worldwide licensing agreement with Pfizer Inc. for the GlycoMimetics investigational compound GMI-1070. GMI-1070 is a pan-selectin antagonist currently in Phase II development for the treatment of vaso-occlusive crisis associated with sickle cell disease. GMI-1070 has received Orphan Drug and Fast Track status from the US FDA.

Vaso-occlusive crisis, which can last 5 to 6 days on average, results in over 75,000 hospitalizations each year in the US. These crises cause pain and tissue damage leading to multiple organ damage, a requirement for life-long narcotic pain medications, and eventually to significantly shorter life spans. While the genetic and molecular cause of sickle cell disease has been known for more than 50 years, therapy for painful crises has not significantly advanced. GMI-1070 is thought to inhibit selectin interactions, a key early step in the inflammatory process leading to vaso-occlusive crisis. In preclinical studies, GMI-1070 restored blood flow to affected vessels of sickle cell animals experiencing vaso-occlusive crisis.

“We are very pleased to partner with Pfizer for the advancement of GlycoMimetics’ lead drug candidate, GMI-1070, which is initially being evaluated in patients with sickle cell disease experiencing vaso-occlusive crisis. This is a major unmet medical need,” said Rachel King, CEO of GlycoMimetics. “We value the resources and experience that Pfizer brings to the program, and recognize that the agreement is an important validation of GlycoMimetics’ unique chemistry expertise in discovery of proprietary drug candidates.”

Under the terms of the agreement, Pfizer will receive an exclusive worldwide license to GMI-1070 for vaso-occlusive crisis associated with sickle cell disease and for other diseases for which the drug candidate may be developed. GlycoMimetics will remain responsible for completion of the ongoing Phase II trial under Pfizer’s oversight, and Pfizer will then assume all further development and commercialization responsibilities. The potential value of the agreement for GlycoMimetics is approximately $340 million, including an up-front payment as well as development, regulatory, and commercial milestones. GlycoMimetics is also eligible for royalties on any sales.

“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. “This experimental compound and partnership are emblematic of our strategy in rare disease, targeting areas of high unmet need to deliver improved patient outcomes.”

“This partnership is an important milestone for GlycoMimetics as the company advances its clinical development program,” added Jim Barrett, PhD, Chairman of the Board of GlycoMimetics and General Partner, New Enterprise Associates. “It’s a testament to the progress made to date with GMI-1070, and will enhance continued development of this potential treatment for patients suffering from vaso-occlusive crisis.”

GMI-1070 is a rationally designed glycomimetic inhibitor of E-, P-, and L-selectins that interferes in a key early step in the inflammatory process leading to leukocyte adhesion and recruitment to inflamed tissue. GMI-1070 has shown activity in several models of diseases in which leukocyte adhesion and activation play a key role.

GMI-1070 is initially being developed for the treatment of vaso-occlusive crisis associated with sickle cell disease. By inhibiting selectin interactions, GMI-1070 may be able to decrease the enhanced cell adhesion that results in vaso-occlusive crisis. In preclinical studies, GMI-1070 restored blood flow to affected vessels of sickle cell animals experiencing vaso-occlusive crisis.

Two Phase I trials of GMI-1070 were successfully completed in the first quarter of 2009, with no serious adverse events reported. The program is currently in Phase II clinical testing. GMI-1070 is also being evaluated in preclinical studies for the treatment of other diseases, including hematologic malignancies, where selectin-mediated cell adhesion and migration is known to play a key role in the disease process. Issued US patents cover GMI-1070 with additional intellectual property issued and pending outside the US.