About Sickle Cell Disease
Sickle cell disease is one of the most prevalent genetic disorders, affecting between 90,000 and 100,000 people in the U.S. alone. It is a chronic condition with substantial illness and death, responsible for more than 70,000 hospitalizations per year in the U.S. One of the most debilitating effects of sickle cell disease is vaso-occlusive crisis (VOC), where changes in a blood protein cause red blood cells to become rigid and stick inside small blood vessels. This causes blockages in blood flow and results in pain, which can require medical care and hospital stays for pain medication. Other medical problems can also occur during vaso-occlusive crisis, which can complicate treatment and lead to longer hospital stays.
Rivipansel (GMI-1070) for Treatment of Vaso-occlusive Crisis of Sickle Cell Disease
GlycoMimetics conducted the initial clinical trials to test an investigational drug, rivipansel (GMI-1070) for sickle cell disease VOC. Rivipansel is intended to treat VOC by reducing the cell adhesion and inflammation that is believed to contribute to blocking blood flow. GlycoMimetics conducted clinical studies to evaluate if rivipansel may reduce the pain experienced by individuals during sickle cell crisis and shorten their time in the hospital. In January 2013, GlycoMimetics, Inc. completed enrolling patients in a Phase 2 sickle cell disease trial of rivipansel, GMI-1070, the final results of which were reported at the ASH meeting in December 2013. GlycoMimetics enrolled 76 individuals ages 12 to 60 at 22 trial sites in the United States and Canada. Results from the trial showed that participants receiving rivipansel, as compared to participants receiving placebo (an inactive treatment), experienced reductions in time to reach resolution of VOC, length of hospital stay and use of opioid analgesics for pain management.
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. Rivipansel has not been approved for use by any worldwide health authority.
Ongoing Clinical Trial for Rivipansel
In 2011, 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 and commercialization 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/. Topline results for this trial are expected in the second quarter of 2019.