About Sickle Cell Disease
Sickle cell disease (SCD) is one of the most prevalent genetic disorders, affecting approximately 100,000 people in the U.S. alone. SCD is a chronic condition causing substantial death and illness, with life expectancy for adults remaining stubbornly stagnant in the fifth decade of life. One of the most debilitating effects of SCD 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 blood flow blockages and results in excruciating pain and potential organ damage, which often requires urgent medical care and inpatient hospital stays. In the U.S., the estimated yearly average of emergency department visits and hospitalizations by patients with SCD is nearly 200,000 and over 100,000, respectively, with VOC cited as the reason in more than 75% of cases. Other medical problems can also occur during VOC, which often complicates treatment and leads to prolonged hospital stays.
Rivipansel, our first compound to enter the clinic, is no longer part of our development pipeline. However, a comprehensive analysis of the Phase 3 data sets revealed the importance of hitting E-selectin as a target as well as the necessity of treating patients early in their experience of crisis. Specifically, in an oral presentation at the 62nd ASH Annual Meeting, newly disclosed data reinforced earlier findings on the improvement with rivipansel when administered within 26.3 hours of crisis. The new data presented at ASH included 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.
With input from the FDA, and clinicians who now prefer treating sickle cell patients in outpatient settings, we have shifted our focus to GMI-1687, and our goal is an investigational new drug filing in 2022. We believe GMI-1687 would be the only compound in development for acute crisis. We also believe that on-demand treatment of acute pain may be more attractive to patients than chronic prophylactic therapies. This molecule is far more potent than rivipansel and designed for subcutaneous administration, positioning GlycoMimetics to address a broader opportunity than with a drug such as rivipansel that requires intravenous administration for patients admitted to the hospital.