Phase 2 clinical trial of GMI-1070 for treatment of vaso-occlusive crisis of sickle cell disease initiated

GlycoMimetics, Inc., a clinical-stage biotechnology company developing a new class of glycobiology-based therapies for a broad range of indications, today announced the commencement of a Phase 2 clinical trial of GMI-1070 for treatment of vaso-occlusive crisis of sickle cell disease. The company also announced that GMI-1070 has received Fast Track designation from the U.S. Food and Drug Administration (FDA).

“GMI-1070 is a promising and versatile compound, with demonstrated activity in blocking leukocyte adhesion. We are encouraged by the initial data achieved in our Phase 1 and pilot clinical studies of GMI-1070, and we are pleased to advance our lead compound into Phase 2 development”

The Phase 2 trial is a randomized, double-blinded study of the efficacy, safety and pharmacokinetics of GMI-1070 in hospitalized sickle cell disease patients experiencing vaso-occlusive crisis. Vaso-occlusive crisis is a painful and life-threatening condition affecting individuals with sickle cell disease in which the flow of blood is blocked as sickle cells become stuck within a blood vessel. GMI-1070 is a pan-selectin inhibitor intended to treat vaso-occlusive crisis by blocking the inflammatory process underlying cell adhesion. The Phase 2 study is designed to evaluate the effects of GMI-1070 on the duration or intensity of vaso-occlusive episodes when it is administered following onset of the crisis. The study will be conducted simultaneously at multiple clinical sites in the US and Canada.

"GMI-1070 is a promising and versatile compound, with demonstrated activity in blocking leukocyte adhesion. We are encouraged by the initial data achieved in our Phase 1 and pilot clinical studies of GMI-1070, and we are pleased to advance our lead compound into Phase 2 development," said Helen Thackray, M.D., GlycoMimetics' Vice President of Drug Development. "Evaluating GMI-1070 in the treatment of sickle cell patients hospitalized with vaso-occlusive crisis represents a critical next step towards fulfilling an important medical need in this historically underserved patient population. The importance of advancing new treatments for sickle cell is underscored by the receipt of Fast Track designation and prior receipt of Orphan Product status for GMI-1070 from the FDA."

The Fast Track program of the FDA is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Fast Track designated drugs ordinarily qualify for priority review, thereby expediting the FDA review process.

In prior Phase 1 clinical evaluation in healthy volunteers and a pilot study in sickle cell patients not undergoing vaso-occlusive crisis at the time of dosing, GMI-1070 demonstrated encouraging safety and pharmacokinetic data, consistent with GlycoMimetics' preclinical studies of the compound.