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 were presented at the September 23-25 meeting of the Foundation for Sickle Cell Disease Research (FSCDR) and in greater detail at the ASCAT meeting in late October 2020. We are committed to discussing these data with the U.S. Food and Drug Administration (FDA) and with KOLs 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.
On December 7, 2020, in an oral presentation at the 62nd ASH Annual Meeting, newly disclosed data reinforced our earlier findings on the improvement with rivipansel when administered early during an acute vaso-occlusive crisis (VOC) in individuals with sickle cell disease (SCD). The new data were part of an analysis of an Open Label Extension (OLE) trial of rivipansel that ran in parallel to the Phase 3 RESET trial and provided an independent, contemporaneous dataset for comparison of clinically meaningful efficacy outcomes with rivipansel. Specifically, these included the achievement of statistically significant improvements in primary and key secondary endpoints, with early treatment in the pediatric subgroup as well as the all-ages treatment group and importantly, on several key secondary endpoints, including time to discharge and time to discontinuation of IV opioids.