GlycoMimetics Announces Positive Initial Safety and Pharmacokinetic Results from Phase 1a Healthy Volunteer Study of GMI-1687

GlycoMimetics, Inc. (Nasdaq: GLYC), a late clinical-stage biotechnology company discovering and developing glycobiology-based therapies for cancers and inflammatory diseases, today announced positive initial safety, tolerability, and pharmacokinetic results from a Phase 1a study of GMI-1687 in healthy volunteers.

“These positive results represent an important milestone in the development of GMI-1687 as a potential point-of-care treatment option intended to help people living with sickle cell disease when they need it most, at the onset of pain crises,” said Harout Semerjian, Chief Executive Officer of GlycoMimetics. “Our Phase 1a data confirm this highly potent, second-generation E-selectin antagonist has an excellent profile for further development, with no dose-limiting toxicities or safety signals observed along with linear pharmacokinetics. We look forward to completing analysis of the study and advancing partner and financing discussions that support further development of this potentially important new therapeutic option for SCD.”

This double-blind, single-center, randomized, placebo-controlled, sequential, single ascending dose Phase 1a trial in healthy adult volunteers enrolled 40 subjects. Eligible subjects received a single subcutaneous injection of GMI-1687 or placebo (6:2 ratio). Five dose levels were evaluated, including 3.3, 10, 20, 40, and 80 mg. The study met its primary and secondary endpoints of safety/tolerability and pharmacokinetics. There were no observed dose limiting toxicities or safety signals. Subcutaneous dosing achieved target therapeutic plasma concentration and linear pharmacokinetics with rapid renal clearance across all dosing levels. Analysis of data is ongoing with full results expected to be presented at an upcoming medical conference.

About Sickle Cell Disease

SCD is the most common inherited blood disorder in the United States, afflicting approximately 100,000 people. Worldwide, about 100 million people carry the SCD trait, and an estimated five million live with the disease. While a majority are of African descent, SCD can affect all ethnic groups, in particular those from areas of endemic malaria, including the Southern Mediterranean, Middle East, and India. Acute pain crises, or vaso-occlusive crises (VOCs), are the most common clinical manifestation of SCD. VOCs occur when sickled red blood cells irritate the lining of blood vessels and cause an inflammatory response that leads to vascular occlusion, tissue ischemia and pain.