The article, entitled "Randomized Phase 2 Study of GMI-1070 in SCD: Reduction In Time To Resolution Of Vaso-Occlusive Crisis and Decreased Opioid Use," highlights rivipansel's potential to improve clinical outcomes in sickle cell patients experiencing VOC. Results from the study were previously highlighted in two oral presentations and one poster presentation at the December 2013 ASH Annual Meeting and Exposition. The two oral presentations were selected as "Best of ASH."
"We are encouraged by the results obtained in the Phase 2 rivipansel
study and by the opportunity we've been afforded to publish the
promising clinical and research findings in Blood, an esteemed
peer-reviewed journal," said
Rivipansel has previously received both Orphan Drug and Fast Track
status for the treatment of VOC from the U.S. Food & Drug
Administration (FDA), and Orphan Product status in the
"If rivipansel continues to demonstrate efficacy in ongoing studies, it
would be the first drug to interrupt the mechanism of VOC in sickle cell
disease," said corresponding study author
About the Study
This prospective multi-national, multi-center, randomized double-blind, placebo-control study enrolled 76 patients 12 to 60 years in age with sickle cell (types SS or S-beta-zero-thalassemia) presenting with VOC to evaluate the efficacy, safety and pharmacokinetics in patients receiving rivipansel. People who took part in the study were evaluated and then randomly assigned to receive either rivipansel or a placebo by IV, in addition to all other usual treatments for their pain crisis.
The primary efficacy endpoint was reduction in time to resolution of VOC, defined as 1.5 cm sustained decrease in visual analog scale (VAS) pain score from baseline and transition to oral pain medications; or feeling ready to leave the hospital; or actual time of discharge from the hospital. In the primary efficacy analysis, the median time to resolution of VOC was reduced by greater than 2.5 days following treatment with rivipansel (time to resolution was 69.6 hours for the rivipansel group compared to 132.9 hours for the placebo group; p=0.187.) Of note, treatment with rivipansel improved clinical outcomes on a number of key secondary endpoints including an 83 percent reduction in mean cumulative IV opioid analgesic use, relative to placebo (p=0.010).
Adverse events (AEs) were evaluated as a secondary endpoint of the phase 2 study. The total AE rates, serious AEs, and AEs considered related to treatment were comparable between patients treated with rivipansel and placebo. The most common treatment emergent AEs (TEAEs) were nausea, constipation, headache and acute chest syndrome. The serious AE rate was 30 percent in both placebo and rivipansel groups, with the most common serious AE being re-hospitalization for VOC. Eighteen participants discontinued the drug, nine in each treatment group, for AEs, no improvement at day 5, or other reasons. All participants were followed until meeting a study endpoint.
About Rivipansel (GMI-1070)
Rivipansel is an investigational selectin inhibitor, inhibiting
E-selectin in particular. Selectins are a family of molecules which are
believed to play a key role in regulating cellular interactions within
the vasculature (blood vessels) and thereby mediating intravascular
blood flow. The intense pain associated with VOC is believed to be
caused primarily by microvascular blockage in the post-capillary
venules, from adhesion of leukocytes as well as clogging of
sickle-shaped red blood cells (which become rigid and inflexible when
sickled), both of which restrict blood flow causing local tissue
ischemia and pain.
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.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements regarding the
clinical development of rivipansel. Actual results may differ materially
from those in these forward-looking statements. For a further
description of the risks associated with these statements, as well as
other risks facing
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