SAN DIEGO, CA, USA I June 10, 2013 I 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.
ABOUT HQK-1001
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.
ABOUT SICKLE CELL DISEASE
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.
ABOUT HEMAQUEST PHARMACEUTICALS, INC.
HemaQuest (www.HemaQuest.com), 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.
SOURCE: HemaQuest Pharmaceuticals
Post Views: 163
SAN DIEGO, CA, USA I June 10, 2013 I 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.
ABOUT HQK-1001
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.
ABOUT SICKLE CELL DISEASE
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.
ABOUT HEMAQUEST PHARMACEUTICALS, INC.
HemaQuest (www.HemaQuest.com), 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.
SOURCE: HemaQuest Pharmaceuticals
Post Views: 163
