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HemaQuest Pharmaceuticals Completes Patient Enrollment in Phase 2b Clinical Study of HQK-1001 in Patients with Sickle Cell Disease

SAN DIEGO, June 10, 2013 /PRNewswire/ -- HemaQuest Pharmaceuticals, Inc. (HemaQuest), a biotechnology company focused on developing small molecule therapeutics to treat hemoglobin disorders, announced today that it has completed enrollment in a randomized, double-blind, placebo-controlled Phase 2b study of HQK-1001 in patients with sickle cell disease. The study, initiated in July 2012, enrolled a total of 77 patients in clinical sites in the US, Canada, Jamaica, Egypt and Lebanon. HemaQuest expects an interim analysis of the study in late 2013 and final results in mid-2014.

"The completion of patient enrollment in this trial is an exciting milestone for our company and an important one for the development path of HQK-1001," said HemaQuest Chief Medical Officer Richard G. Ghalie, MD. "We believe that HQK-1001 has the potential to reduce the frequency of serious pain crises, a significant unmet medical need worldwide for patients suffering from this devastating disease. We look forward to the initial data from the study later this year."

The Phase 2b study was designed to evaluate the efficacy, safety and tolerability of HQK-1001, a small chain fatty acid derivative administered orally twice daily. The primary objective of the study is the induction of fetal hemoglobin measured as an increase from baseline over time. Secondary objectives include the effect on frequency of pain crises, frequency and intensity of daily pain, analgesic use, and quality of life.


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. HQK-1001 is an orally administered SCFAD which has been assessed in a number of clinical trials in sickle cell disease and beta thalassemia. These trials have established a safe and well tolerated dose which results in the induction of fetal hemoglobin. Induction of fetal hemoglobin production in red blood cells has been shown to reduce the frequency of pain crises and hospitalizations of patients with sickle cell disease. The compound has received Orphan Drug Designation in the United States and Europe for both sickle cell disease and beta thalassemia.


Sickle cell disease is a genetic blood disorder that affects approximately 140,000 patients in the U.S. and Europe, 100,000 in Latin America and 250,000 in the Middle East. Sickle cell disease is characterized by production of an abnormal beta hemoglobin chain of adult hemoglobin, which results in distorted, rigid sickle red blood cells that 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 approved to treat the disease is a cancer chemotherapy drug, hydroxyurea. The lifespan of sickle cell patients is markedly reduced.


HemaQuest (, established in late 2007, is a San Diego and Seattle-based biopharmaceutical company focused on developing small molecule therapeutics based on its proprietary SCFAD technologies to treat hemoglobin diseases. The Company's investors include Aberdare Ventures, De Novo Ventures, Forward Ventures, Latterell Venture Partners and Lilly Ventures.

Under U.S. law, a new drug cannot be marketed until it has been investigated in clinical trials and approved by the FDA as being safe and effective for the intended use. Statements included in this press release that are not historical in nature are "forward-looking statements" within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. You should be aware that our actual results could differ materially from those contained in the forward-looking statements, which are based on management's current expectations and are subject to a number of risks and uncertainties, including, but not limited to, our failure to successfully commercialize our product candidates; costs and delays in the development and/or FDA approval, or the failure to obtain such approval, of our product candidates; uncertainties or differences in interpretation in clinical trial results; our inability to maintain or enter into, and the risks resulting from our dependence upon, collaboration or contractual arrangements necessary for the development, manufacture, commercialization, marketing, sales and distribution of any products; competitive factors; our inability to protect our patents or proprietary rights and obtain necessary rights to third party patents and intellectual property to operate our business; our inability to operate our business without infringing the patents and proprietary rights of others; general economic conditions; the failure of any products to gain market acceptance; our inability to obtain any additional required financing; technological changes; government regulation; changes in industry practice; and one-time events. We do not intend to update any of these factors or to publicly announce the results of any revisions to these forward-looking statements.


Tamara A. Seymour

T: 858.356.5582

Chief Financial Officer


SOURCE HemaQuest Pharmaceuticals, Inc.
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