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HemaQuest Pharmaceuticals Announces Clinical Progress on New Sickle Cell Anemia Drug
HemaQuest Presents Results at American Society of Hematology Annual Meeting
| Source: HemaQuest Pharmaceuticals
SAN FRANCISCO, CA--(Marketwire - December 9, 2008) - 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. (www.HemaQuest.com), 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.