GlycoMimetics, Inc. reported that Pfizer Inc. announced that the Phase 3 Rivipansel (GMI-1070): Evaluating Safety, Efficacy and Time to Discharge (RESET) pivotal study did not meet its primary or key secondary efficacy endpoints.
The objective of the trial was to evaluate the efficacy and safety of rivipansel in patients aged six and older with sickle cell disease (SCD) who were hospitalized for a vaso-occlusive crisis (VOC) and required treatment with intravenous (IV) opioids. The primary endpoint was time to readiness-for-discharge and the key secondary efficacy endpoints were time-to-discharge, cumulative IV opioid consumption, and time to discontinuation of IV opioids.
“We are both surprised and deeply disappointed by this outcome, as we had strongly hoped that rivipansel would have a positive benefit for people living with sickle cell disease,” said Rachel King, Chief Executive Officer of GlycoMimetics. “We are grateful to the many people who supported and advanced this program over the years of clinical study, especially to sickle cell patients and their families.”
Rivipansel is a glycomimetic drug candidate that acts as a pan-selectin antagonist, meaning it binds to all three members of the selectin family – E-, P- and L-selectin. Rivipansel is an investigational treatment for VOC in people with SCD and not approved for use. In 2011, GlycoMimetics and Pfizer Inc. entered into a worldwide license agreement for the development and, if approved by applicable regulatory authorities, commercialization of rivipansel. Since completion of the Phase 2 clinical trial, Pfizer has been responsible for clinical development of rivipansel, including the RESET clinical trial
About SCD and VOC
SCD is the most common inherited blood disorder in the United States, impacting approximately 100,000 people. Worldwide, approximately 100 million people carry the SCD trait and an estimated five million live with the disease. While the majority of people with SCD are of African descent, the disease can affect all ethnic groups, especially those from areas where malaria is or was endemic, such as the Middle East, India and the Southern Mediterranean. Acute pain crises or VOCs are the most common clinical manifestation of SCD. A VOC occurs when sickled red blood cells irritate the lining of blood vessels and cause an inflammatory response leading to vascular occlusion, tissue ischemia and pain.
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’ most advanced drug candidate, rivipansel, a pan-selectin antagonist, is an investigational treatment for VOC being evaluated by Pfizer. GlycoMimetics’ wholly owned drug candidate, uproleselan, an E-selectin antagonist, was evaluated in a Phase 1/2 clinical trial as a potential treatment for AML. It has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration and is being evaluated across a range of patient populations including a company-sponsored Phase 3 trial in relapsed/refractory AML. GlycoMimetics has also completed a Phase 1 clinical trial with a third drug candidate, GMI-1359, a combined CXCR4 and E-selectin antagonist. GlycoMimetics is located in Rockville, MD in the BioHealth Capital Region. Learn more at www.glycomimetics.com.