The first potential treatment use for rivipansel, the lead investigational drug candidate in the GlycoMimetics pipeline, may be in “vaso-occlusive crisis (VOC) of sickle cell disease.” Rivipansel is intended to treat VOC by reducing the cell adhesion, activation and inflammation that are believed to contribute to reducing blood flow through the microvasculature. GlycoMimetics has evaluated if this may reduce the pain experienced by individuals during sickle cell crisis and shorten their time in the hospital. Pfizer, GlycoMimetics’ development partner for rivipansel, is also studying whether this may reduce other medical problems that result from cell adhesion and activation, and vascular occlusion.
Rivipansel has received Orphan Drug status and Fast Track status from the U.S. Food and Drug Administration (FDA), which may lead to speedier delivery to people living with sickle cell disease, if the treatment is found to be effective and approved by the regulatory agency. GMI-1070 has not been approved for use by any health authority anywhere in the world.
About the Rivipansel (GMI-1070) Trial
In January 2013, GlycoMimetics, Inc. completed enrollment in a Phase 2 sickle cell disease study with rivipansel. The randomized, double-blinded study examined the efficacy, safety and pharmacokinetics of rivipansel in hospitalized sickle cell disease patients experiencing VOC. GlycoMimetics enrolled 76 individuals ages 12 to 60 at 22 trial sites in the United States and Canada. The company reported topline data from the trial in April 2013 and presented full data from the clinical trial in two oral presentations and one poster presentation at the December 2013 meeting of the American Society of Hematology (ASH.) One of the oral presentations was selected a “Best of ASH.”
In the Phase 2 trial, patients treated with rivipansel experienced reductions in time to reach resolution of VOC, length of hospital stay and use of opioid analgesics for pain management, in each case as compared to patients receiving placebo.
Future Clinical Trials for Rivipansel
A few years ago, GlycoMimetics entered into a worldwide license agreement with Pfizer for the development and, if approved by applicable regulatory authorities, commercialization of rivipansel. GlycoMimetics was responsible for development through the Phase 2 clinical trial and Pfizer is now responsible for all future clinical development of rivipansel. Pfizer is currently conducting the RESET (Rivipansel: Evaluating Safety, Efficacy and Time to Discharge) study—a Phase 3 clinical trial assessing the efficacy and safety of rivipansel for the treatment of VOC in individuals hospitalized with sickle cell disease who are six years of age or older. For more information on the RESET study, visit: http://www.resetsicklecell.com/.