San Diego and Seattle-based biopharmaceutical company HemaQuest Pharmaceuticals, Inc. which focuses its clinical research on developing small molecule therapeutics based on its proprietary Short Chain Fatty Acid Derivatives or SCFADs technologies to treat hemoglobin diseases, earlier today announced that clinical investigators have enrolled the first patient in a randomized multi-dose Phase II study of HQK-1001 in patients with sickle cell disease.

The study, which is being conducted by investigators in clinical sites in the US, Canada, Jamaica, Egypt and Lebanon, is expected to enroll approximately 50 patients. The primary objective is safety and tolerability of HQK-1001. There are several secondary objectives including an increase in fetal hemoglobin production. HemaQuest expects interim results from the study in late 2011 and final results in the first quarter of 2012.

“Based on the promising results from our prior Phase II clinical studies, we are excited to initiate this clinical study testing higher dose levels and longer duration of therapy to more fully characterize the therapeutic potential of HQK-1001 in sickle cell disease,” said HemaQuest Chief Medical Officer Richard G. Ghalie, MD. “This is an important next step toward potentially making HQK-1001 available as a treatment for this devastating disease in underserved populations around the world.”

Sickle cell disease
Sickle cell disease is a genetic blood disorder that affects approximately 110,000 patients in the U.S. and Europe and 250,000 in the Middle East. Sickle cell disease is characterized by production of an abnormal beta hemoglobin chain of adult hemoglobin, which results in distorted, rigid sickle red blood cells, which block blood vessels, causing lack of oxygen to tissues, acute episodes of pain (pain crises), lung injury (acute chest syndrome), and strokes. Infections are common, and chronic damage occurs in many organs, including the spleen, bones, kidneys, lungs, brain, and eyes. The sole drug which is approved to treat the disease is a cancer chemotherapy drug, hydroxyurea. The lifespan of sickle cell patients is markedly reduced.

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HQK-1001 belongs to a class of compounds originally discovered at Boston University School of Medicine. These compounds, designated as Short Chain Fatty Acid Derivatives, have been shown to stimulate fetal hemoglobin expression and red blood cell production in the laboratory and in small clinical trials in patients with hemoglobin disorders, including sickle cell disease and beta thalassemia. Increased fetal hemoglobin production in red blood cells has been shown to reduce the frequency of pain crises and hospitalizations of patients with sickle cell disease HQK-1001 is an orally administered SCFAD, which has shown an excellent safety profile and biologic effects on fetal hemoglobin induction and red blood cell production in the laboratory, relevant animal models, and in clinical trials carried out in healthy human subjects as well as patients with sickle cell disease and beta thalassemia. The compound has received Orphan Drug Designation in the United States and Europe for both sickle cell disease and beta thalassemia.

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