HemaQuest Pharmaceuticals Announces Clinical Progress On New Sickle Cell Anemia Drug

Main Category: Blood / Hematology
Also Included In: Clinical Trials / Drug Trials
Article Date: 10 Dec 2008 - 9:00 PDT

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HemaQuest Pharmaceuticals announced today the successful completion of Phase 1 clinical trials of HQK-1001, an orally-administered therapeutic which the company is developing to treat hemoglobin disorders, including sickle cell anemia and beta thalassemia. The company presented the results at the annual meeting of the American Society of Hematology in San Francisco.

HemaQuest performed two different trials. In the first placebo-controlled clinical study, 32 healthy volunteers were given single doses of HQK-1001, ranging from 2 to 20 mg/kg. This trial was followed by a second placebo-controlled study, in which 41 healthy volunteers received two weeks of daily doses of HQK-1001 ranging from 5 to 15 mg/kg.

In both studies, there were no clinically significant adverse effects, and the incidence of mild side effects was similar in subjects receiving placebo or HQK-1001, the company said. Pharmacokinetic studies showed that single doses at, or above, 10 mg/kg reached the targeted plasma drug levels. Significantly, subjects treated with HQK-1001 in the multiple dose Phase 1 clinical trial provided preliminary evidence of its therapeutic potential, as demonstrated by statistically significant increases in young red blood cells, known as reticulocytes.

HemaQuest Chief Scientific Officer and Vice President, Clinical Affairs, Susan Perrine, MD, said, "We are pleased to report these results demonstrating biological activity and favorable pharmacology of HQK-1001, and show that the drug was well-tolerated. We are very encouraged that a brief two-week treatment with HQK-1001 generated significant increases in reticulocytes, indicating that it may offer therapeutic benefit in hemoglobin disorders and other anemias."

HemaQuest President and CEO Ronald Berenson, MD, said, "These initial clinical studies provide the foundation for subsequent testing of HQK-1001 in patients with hemoglobin disorders, including sickle cell anemia and thalassemia in early 2009. If results of our Phase 1 clinical trials are confirmed in these upcoming studies, HQK-1001 may also be beneficial for treating other, common types of anemia characterized by reduced production of red blood cells."

Sickle cell anemia and beta thalassemia afflict millions of people, and are the most common genetic diseases. In the United States, there are nearly 80,000 patients suffering from one of these two inherited blood diseases. Few worldwide therapeutic alternatives exist for these serious and life-threatening diseases, which are associated with significant morbidity and reduced survival, creating a strong and pressing need for new treatments.

About Hemaquest Pharmaceuticals

HemaQuest Pharmaceuticals, Inc., established in late 2007, is a biopharmaceutical company focused on developing oral, small molecule therapeutics to treat hematological diseases including hemoglobin disorders. These therapeutics are based on short chain fatty acid derivative (SCFAD) technologies, which were discovered by Susan Perrine, MD, and her colleagues at Boston University. The company's first therapeutic, HQK-1001, has received orphan status drug designation in the U.S. for both sickle cell anemia and beta thalassemia. HemaQuest's investors include De Novo Ventures, based in Palo Alto, Calif.; Forward Ventures, based in San Diego; and Lilly Ventures of Indianapolis, Ind., the venture capital arm of Eli Lilly and Company.

Hemaquest Pharmaceuticals

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