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HemaQuest Pharmaceuticals, Inc. Presents Promising Clinical Results at 52nd Annual Meeting of the American Society of Hematology (ASH)
Date:12/7/2010

evidence of the safety and potential therapeutic activity of HQK-1001 in patients with sickle cell disease and beta thalassemia intermedia. The fetal hemoglobin induction observed in both of the clinical trials is important, given its key role in ameliorating these diseases. Based on these promising clinical results, we plan to initiate a clinical trial testing higher dose levels and longer duration of therapy to more fully characterize the therapeutic potential of HQK-1001 in sickle cell disease."

ABOUT HQK-1001 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 (SCFADs), 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 was shown to ameliorate the outcome of patients with these diseases. 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. Additionally, the compound has received Orphan Drug Designation in the United States and Europe for both sickle cell disease and beta thalassemia.

ABOUT SICKLE CELL DISEASE AND BETA THALASSEMIASickle cell disease is a genetic blood disorder that affects approximately 80,000 patients in the U.S., and 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), a
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