Hemaquest Pharmaceuticals' HQK-1001 holds promise for sickle cell disease and beta thalassemia

Dec 8 2009

HemaQuest Pharmaceuticals presented data from new preclinical studies and early results from clinical trials of its lead drug candidate, HQK-1001, in sickle cell disease and beta thalassemia at the annual meeting of the American Society of Hematology in New Orleans. The preclinical studies demonstrated that HQK-1001 could augment the activity of a range of therapeutic agents that have been used in the past to treat hemoglobin disorders. In addition, the first clinical trials have begun testing HQK-1001 in patients with sickle cell disease or beta thalassemia. In these two HemaQuest-sponsored clinical trials, patients are being treated with increasing doses to determine its safety and pharmacodynamic effects. The studies are ongoing, and have demonstrated that the drug has a strong safety profile with early evidence of fetal globin induction, the key pharmacodynamic marker of therapeutic activity for this agent.

HemaQuest Chief Scientific Officer and Vice President, Clinical Affairs, Susan Perrine, MD, said, "We are pleased with the early clinical findings in patients with sickle cell disease and beta thalassemia. The fetal globin induction observed in both of the clinical trials is important, given its key role in ameliorating these diseases. Furthermore, the new evidence of additive or synergistic effects of HQK-1001 with other potential therapeutics suggests the potential for HQK-1001 to become an important drug in the treatment of these serious anemias."

HemaQuest President and CEO Ronald Berenson, MD, said, "These clinical studies provide early evidence of the safety and potential therapeutic activity of HQK-1001 in patients with sickle cell disease and beta thalassemia. We look forward to the successful completion of these trials and future clinical studies to document the therapeutic effects of HQK-1001 in patients with these serious and life-threatening diseases."