Positive Early Safety and PK Results for GMI-1687 in Healthy Volunteers

In a recent clinical trial, GMI-1687, a potent E-selectin antagonist, demonstrated promising results in its initial human study. The Phase 1a study, which involved healthy volunteers, showed that the drug was well-tolerated and had a favorable safety profile, with no dose-limiting toxicities or safety concerns reported. The subcutaneous administration of GMI-1687 resulted in linear pharmacokinetics, achieving the desired plasma concentrations across various dosages.

GlycoMimetics, Inc., the company behind the development of GMI-1687, is focusing on sickle cell disease (SCD) as the initial target for this potential point-of-care treatment. SCD is a prevalent inherited blood disorder in the United States, affecting approximately 100,000 individuals. Globally, around 100 million people carry the trait, with an estimated five million living with the disease. The disorder is common among people of African descent and in regions where malaria is endemic, such as the Southern Mediterranean, Middle East, and India.

The clinical trial was a double-blind, randomized, and placebo-controlled study that enrolled 40 healthy adult participants. They received a single subcutaneous injection of GMI-1687 or a placebo. The trial evaluated five different dosages and successfully met its primary and secondary endpoints regarding safety, tolerability, and pharmacokinetics. The drug showed rapid renal clearance and maintained therapeutic plasma concentrations.

GMI-1687 is a second-generation E-selectin antagonist that is bioavailable when administered subcutaneously. E-selectin is thought to play a significant role in vaso-occlusive crises, which are the primary cause of pain in SCD patients. If further developed successfully, GMI-1687 could be a patient-controlled treatment option for SCD patients during the onset of pain crises. Currently, there is no FDA-approved treatment for the acute onset of painful vaso-occlusive crises.

GlycoMimetics is a late clinical-stage biotechnology firm that is pioneering the development of therapies based on glycobiology for cancers and inflammatory diseases. The company's approach is grounded in the understanding of how carbohydrates are involved in cell recognition. GlycoMimetics is leveraging its unique chemistry platform to discover small molecule drugs, known as glycomimetics, that can alter carbohydrate-mediated recognition in various disease states. The company aims to develop transformative therapies for diseases with significant unmet medical needs.

The full results of the Phase 1a study are still being analyzed, and they are expected to be presented at an upcoming medical conference. This development is a significant step forward in the quest to find effective treatments for SCD and other inflammatory conditions.