New Pediatric and Secondary Endpoint Data from Rivipansel Phase 3 RESET Trial Presented at The Annual Scientific Conference on Sickle Cell and Thalassemia (ASCAT) Meeting
- Data highlight the importance of early intervention with fast-acting E-selectin inhibitors to disrupt the underlying inflammatory mechanisms driving acute vaso-occlusive crisis (VOC)
- Abstracts for two of GlycoMimetics’ wholly-owned product candidates, rivipansel and GMI-1687, accepted for poster and oral presentations
“The important data published today provide further support for the potential benefits of treatment with rivipansel early in the course of VOC, as observed in sickle cell patients in the RESET trial – both in the total patient population as well as in the pediatric subgroup. In addition to highlighting the importance of treating individuals early in the course of their acute painful crisis, these new findings confirm the critical role of E-selectin in acute vaso-occlusion and the opportunity to resolve that occlusion and pain with effective intervention,” said
“The favorable safety profile of rivipansel observed in the Phase 3 RESET trial, as evaluated in a population with pediatric, adolescent, and adult patients, is highly encouraging to us. We are engaging in discussions with the FDA to determine what, if any, next steps could be taken to carry this program forward in acute VOC, either in pediatrics or in the overall population. This treatment setting remains an area of unmet medical need, as there are no drugs approved nor currently in late-stage development for acute intervention,” she added.
The rivipansel abstract includes data from a supportive analysis of the Phase 3 RESET trial of 345 patients (ranging in age from six years to adults, with a mean age of 22 years) who were experiencing acute VOC requiring hospitalization for treatment. The new data demonstrate that early rivipansel treatment conferred clinically meaningful improvements for two key secondary endpoints not previously reported: shortening IV opioid use and decreasing the hospital stay. Specifically, for the total treated patient population (n=320), rivipansel treatment within 26.4 hours of pain onset (earliest quartile of duration of VOC until treatment) reduced:
- median TTD by 41.5 hours (from 112.8 to 71.3 hours; p=0.02), and
- median TTDIVO by 50.5 hours (from 104.0 to 53.5 hours; p=0.03) vs. placebo.
In addition, newly scheduled for presentation at this meeting are data for the pediatric subgroup, who make up a large proportion (41%) of patients treated in the RESET trial. Namely, data from children 6-17 years old in the study who were treated within 30 hours of VOC onset show:
- a reduction in median TTRFD by 29.3 hours (p=0.02),
- a reduction in median TTD by 23.2 hours (p=0.02),
- a reduction in median TTDIVO by 15.4 hours (p=0.045), and
- more children ready for discharge by 24, 48 and 72 hours, compared to placebo.
The abstract also features key findings previously reported at the
The second abstract, accepted for oral presentation, discloses data from two different preclinical models of VOC using GlycoMimetics’ highly potent and specific E-selectin antagonist, GMI-1687. These data show the drug candidate’s efficacy as a subcutaneously administered treatment for VOC. Using both a human sickle cell transplantation model and the Townes mouse model, GMI-1687 was shown to prevent sickle red blood cell adherence to inflamed vasculature, inhibit vessel occlusion and restore normal blood flow. Preliminary data showing the activity of GMI-1687 for the treatment of VOC were disclosed in an oral presentation late September at the FSCDR virtual meeting.
Title: Early Initiation of Treatment with Rivipansel for Acute Vaso-Occlusive Crisis in Sickle Cell Disease (SCD) Achieves Earlier Discontinuation of IV Opioids and Shorter Hospital Stay: RESET Clinical Trial Analysis.
Title: Treatment of Acute Vaso-occlusion in Mouse Models of Sickle Cell Disease Following Intravenous or
Session: Sickle Cell Disease – Basic Science: Emerging Therapies
Date and Time:
About Sickle Cell Disease (SCD) and VOC
SCD is the most common inherited blood disorder in
Rivipansel, the company’s wholly-owned glycomimetic drug candidate that binds to all three members of the selectin family (E-, P- and L-selectin), was GlycoMimetics’ first candidate to enter clinical development. After the Phase 3 RESET trial conducted by Pfizer, GlycoMimetics’ former collaborator, produced disappointing results in 2019, new efficacy data from a post hoc analysis of rivipansel were published in
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