Rivipansel: In August 2019, Pfizer reported disappointing results from the Phase 3 RESET trial (NCT02187003) after it failed to meet the primary endpoint of the study. A post hoc analysis of the Phase 3 RESET study evaluating the efficacy of rivipansel in acute vaso-occlusive crisis (VOC) showed that patients treated with rivipansel within approximately 26 hours of the onset of pain in their crisis experienced statistically significant improvements in the primary efficacy endpoint of time to readiness for discharge compared to placebo. In addition, biomarker data showed reductions in soluble E-selectin confirming that rivipansel hit its intended biological target. GycoMimetics believes that these findings confirm the critical role of E-selectin in acute vaso-occlusion, as well as the importance of treating individuals early in the course of their acute painful crisis. These data are contained in abstracts published online (see page 26) in June 2020 and will be presented at the September 23-25 meeting of the Foundation for Sickle Cell Disease Research (FSCDR). We are committed to discussing these data with the U.S. Food and Drug Administration (FDA) to determine what, if any, next steps could be taken to carry this GlycoMimetics wholly-owned development program forward in acute VOC, either in pediatrics or in the overall population.
