GlycoMimetics completes two Phase I sickle cell disease trials

GlycoMimetics, a clinical stage pharmaceutical company, has completed two Phase I safety studies of its lead drug candidate GMI-1070 for vaso-occlusive crisis of sickle cell disease.

The Phase I studies were comprised of single- and multiple-ascending dose cohorts in healthy volunteers, and were designed to evaluate the safety and pharmacokinetics of GMI-1070.

In addition, the company announced that the FDA has granted orphan drug designation to GMI-1070 for the treatment of vaso-occlusive crisis in patients with sickle cell disease. GlycoMimetics plans to apply to the FDA for fast track designation of GMI-1070 later in 2009.

GMI-1070 is also being evaluated in preclinical studies for the treatment of certain hematologic cancers, where selectin-mediated cell adhesion and migration is known to play a key role in the disease process. Initiation of Phase II clinical trials of GMI-1070 in sickle cell disease is planned in 2009.

Helen Thackray, vice president of clinical development at GlycoMimetics, said: Demonstrating the safety of the drug candidate is a critical first milestone, and we’re encouraged to learn that the initial safety profile is quite benign. No serious adverse events were reported in either study. GMI-1070 also displayed excellent pharmacokinetic properties in both studies. Based on these results, we intend to begin dosing sickle cell patients with GMI-1070 in Phase II clinical trials later in 2009.