HemaQuest Pharmaceuticals, a biotechnology company focused on developing small molecule therapeutics to treat hemoglobin disorders, has completed enrollment in a randomized, double-blind, placebo-controlled phase IIb study of HQK-1001 in patients with sickle cell disease.
The study, initiated in July 2012, enrolled a total of 77 patients in clinical sites in the U.S., Canada, Jamaica, Egypt and Lebanon. HemaQuest expects an interim analysis of the study in late 2013 and final results in mid-2014.
The phase IIb study was designed to evaluate the efficacy, safety and tolerability of HQK-1001, a small chain fatty acid derivative administered orally twice daily. The primary objective of the study is the induction of fetal hemoglobin measured as an increase from baseline over time. Secondary objectives include the effect on frequency of pain crises, frequency and intensity of daily pain, analgesic use and quality of life.
"We believe that HQK-1001 has the potential to reduce the frequency of serious pain crises, a significant unmet medical need worldwide for patients suffering from this devastating disease,” said Richard G. Ghalie, M.D., HemaQuest chief medical officer.