HQK-1001 belongs to a class of compounds originally discovered at Boston University School of Medicine and licensed to the Company. These compounds, designated as Short Chain Fatty Acid Derivatives (SCFADs), have been shown to stimulate fetal globin 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. HQK-1001 is an orally administered SCFAD, which has shown an excellent safety profile and biologic effects on fetal globin induction and red blood cell production in the laboratory, relevant animal models, and in healthy human subjects in Phase 1 clinical trials. 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 THALASSEMIA
Sickle cell disease is a genetic disorder affecting the beta globin 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 in the U.S., where there are approximately 75-80,000 patients.
Beta thalassemias are among the most common genetic blood disorders worldwide, in which patients are unable to produce normal amounts of the beta globin chain of adult hemoglobin, with consequent rapid destruction of red blood cells and their progenitors, and moderate to severe, transfusion-dependent anemia. The standard therapies are transplantation and red blood cell transfusions, which cause iron overload an
|SOURCE HemaQuest Pharmaceuticals|
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