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.
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 and total hemoglobin. In March 2012, HemaQuest completed an open-label, multi-dose Phase 2a study of HQK-1001 given for up to 6 months in 52 patients with sickle cell disease. Based on favorable study results, a multicenter, placebo-controlled Phase 2b study was initiated in 2012 to evaluate HQK-1001 given twice a day for 48 weeks in 74 patients with sickle cell disease.
Two-investigator-sponsored studies were initiated in 2012 to study the safety and pharmacodynamics of HQK-1001 given daily for 6 months 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.