GlycoMimetics, Inc. announced dosing of the first cohort of healthy volunteers in a Phase 1a study of GMI-1687 to evaluate safety, tolerability, and pharmacokinetics. This Phase 1a study is a double-blind, single-center, randomized, placebo-controlled, sequential, single ascending dose trial in healthy adult volunteers. It is expected to enroll approximately 40 subjects.

Eligible subjects will receive a single dose of GMI-1687 or placebo (6:2 ratio) via subcutaneous injection. Safety, tolerability, and pharmacokinetics of up to five dose levels (3.3, 10, 20, 40, and 80 mg) will be evaluated. About SCD: SCD is the most common inherited blood disorder in the United States, impacting approximately 100,000 people.

Worldwide, approximately 100 million people carry the SCD trait and an estimated five million people live with the disease. While the majority are of African descent, the disease can affect all ethnic groups, especially those from areas where malaria is or was endemic, such as the Middle East, India and the Southern Mediterranean. Acute pain crises, or vaso-occlusive crises (VOCs), are the most common clinical manifestation of SCD.

A VOC occurs when sickled red blood cells irritate the lining of blood vessels and cause an inflammatory response leading to vascular occlusion, tissue ischemia and pain. About GMI-1687: Discovered and developed by GlycoMimetics, GMI-1687 is a highly potent E-selectin antagonist that has been shown in animal models to be bioavailable after subcutaneous administration. This second-generation compound has potential application in inflammatory diseases, and the initial development focus will be on SCD.

E-selectin is believed to play a major role in VOCs, the vascular clots and blockages that cause pain crises in people living with SCD. Administration of GMI-1687 by subcutaneous injection, if successfully developed in the clinic, may enable this study drug to be approved as a point-of-care treatment option at the onset of a VOC.