GAITHERSBURG, Md.--(BUSINESS WIRE)--GlycoMimetics, Inc. announced today that the company had presented at and participated in a workshop entitled, “Translating New Therapeutics for Sickle Cell Disease to the Marketplace.” The meeting, sponsored by the National Heart, Lung, and Blood Institute at the National Institutes of Health, was held August 19-21, 2013 at the Natcher Conference Center on the NIH campus.
“Translating New Therapeutics for Sickle Cell Disease to the Marketplace.”
The workshop included presentations from public and private sector experts on various factors involved in bringing new sickle cell disease therapies to market, including the pathophysiology of sickle cell disease, clinical trial design, and building strategic partnerships, among others.
Helen Thackray, M.D., Vice President of Clinical Development and Chief Medical Officer at GlycoMimetics, presented “Challenges to Translation: Including Trial Design,” which highlighted various aspects of clinical trial design and implementation, including unique issues faced by sickle cell disease drug development companies. The presentation focused on challenges in trial design, data and electronic records, trial feasibility, and compliance and documentation. Among other opportunities noted in the presentation, Dr. Thackray underscored the critical role of a multi-disciplinary research team in providing a coordinated research approach.
“This meeting provided a wonderful opportunity to foster discussions between communities that are integral in translating research ideas and bringing new discoveries to those affected by sickle cell disease,” said Dr. Thackray. “We were honored to share our perspective on challenges, as well as opportunities, related to the conduct of clinical studies like the Phase 2 trial we conducted of GMI-1070, a novel potential therapy for the treatment of vaso-occlusive crisis (VOC) in sickle cell disease.”
The randomized, double-blinded Phase 2 clinical trial, which formed the basis for the presentation, examined the safety, efficacy, and pharmacokinetics of GMI-1070 in hospitalized sickle cell patients experiencing VOC. The trial, which enrolled 76 patients ranging from 12 to 60 years old at 22 trial sites in the United States and Canada, enrolled its last patient in January 2013.
For more information on the research meeting, visit: http://www.cvent.com/events/annual-sickle-cell-disease-clinical-research-meetings/event-summary-b0cafc512d25422096ef3a6a28095937.aspx.
About GlycoMimetics, Inc.
GlycoMimetics is a clinical stage biotechnology company focused on the discovery and development of novel glycomimetic drugs to address unmet medical needs resulting from diseases in which carbohydrate biology plays a key role. Glycomimetics are molecules that mimic the structure of carbohydrates involved in important biological processes. Using its expertise in carbohydrate chemistry and knowledge of carbohydrate biology, the company is developing a pipeline of glycomimetic drug candidates that inhibit disease-related functions of carbohydrates, such as the roles they play in inflammation, cancer and infection. For additional information, please visit the company’s web site at http://www.glycomimetics.com.
About Sickle Cell Disease and VOC
Sickle cell disease is a genetic disease affecting 90,000 to 100,000 people in the United States. One of the most severe complications of sickle cell disease is vaso-occlusive crisis (VOC). VOC is typically characterized by excruciating, debilitating pain that occurs periodically throughout the life of a person with sickle cell disease. VOC is responsible for more than 73,000 hospitalizations per year in the United States with an average hospital stay of approximately six days. The current standard of care for VOC consists of supportive therapy, primarily in the form of hydration and pain management, typically requiring extended hospitalization.