HQK-1001 is an orally administered short chain fatty acid (SCFA) derivative, which has a unique combination of biological effects - induction of fetal globin and stimulation of red blood cell production - that addresses the underlying pathological mechanisms in sickle cell disease and beta thalassemia. Extensive studies in both the laboratory and relevant animal models carried out in transgenic mice and non-human primates have demonstrated the potential therapeutic effects of this compound in treating these serious, chronic illnesses. HemaQuest has obtained an exclusive worldwide license for a series of patents and patent applications from Boston University for these technologies that give it a strong proprietary position.

HQK-1001 completed short-term and chronic animal toxicology studies, which demonstrated that the drug candidate was safe and well-tolerated at and above doses planned for clinical studies. The compound has excellent oral bioavailability, making it an attractive medication for treating chronic disorders, such as sickle cell disease and beta thalassemia. HemaQuest has completed two placebo-controlled Phase 1 clinical trials in healthy volunteers, where orally administered HQK-1001 was demonstrated to be well-tolerated with no significant adverse events. These clinical studies also demonstrated that the compound had a favorable pharmacokinetic profile and evidence of pharmacodynamic effects as documented by significant increases in early red blood cells, known as reticulocytes.

In 2010, HemaQuest completed proof of concept trials of HQK-1001 in both beta thalassemia and sickle cell disease. These studies confirmed the safety of HQK-1001 in this patient population and provided evidence of pharmacodynamic effects, such as rises in fetal hemoglobin. In April 2011, HemaQuest initiated a multicenter Phase 2 trial to evaluate HQK-1001 given for longer duration and at higher doses than previously studied in patients with sickle cell disease. Enrollment in the trial was completed with 52 patients in September 2001. If positive, this Phase 2 trial will provide the basis for moving forward with advanced clinical development including pivotal studies beginning in 2012. In 2011, HemaQuest will also support two single-center trials of HQK-1001 given for longer duration than previously studied in patients with beta thalassemia.

HemaQuest has been granted Orphan Drug Designation in the US and the EU for HQK-1001 in both sickle cell disease and beta thalassemia.