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New Drug to Treat Sickle Cell Crisis Begins Pilot Study in Patients

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GlycoMimetics, Inc. has announced that a pilot study of the company’s lead candidate, GMI-1070, is underway in sickle cell patients. The first patients in this pilot study were treated at Children’s Hospital & Research Center Oakland in California.

GMI-1070 is a first-in-class pan-selectin inhibitor intended to treat vaso-occlusive crisis by inhibiting the inflammatory processes underlying cell adhesion.

The pilot study will evaluate the safety and pharmacokinetics of GMI-1070, and is also designed to measure the effect of GMI-1070 on blood flow and biomarkers of inflammation in patients with sickle cell disease. The trial will enroll up to twenty sickle cell disease patients not experiencing vaso-occlusive crisis.

“The dosing of patients with this novel agent marks an important milestone in the advancement of an exciting and much-needed approach to addressing sickle cell crisis,” said Helen Thackray, M.D., GlycoMimetics’ Vice President of Drug Development. “The data we obtain from this pilot study will enable additional studies in sickle cell patients in the midst of vaso-occlusive crisis.”

“GMI-1070 appears to have interesting potential as a new treatment for vaso-occlusive crisis in sickle cell patients. Vaso-occlusive crisis is a painful and life-threatening complication of sickle cell disease that truly lacks effective treatment options,” said Lori Styles, M.D., Hematologist/Oncologist and the lead clinical investigator at Children’s for this program. “We are pleased to have the opportunity to be a leading clinical investigation site in the evaluation of GMI-1070.”

GlycoMimetics recently announced the completion of two Phase 1 clinical studies of GMI-1070’s safety in healthy volunteers. Clinical investigators reported no serious adverse events in either of the two studies.