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HemaQuest Pharmaceuticals Announces Clinical Progress on New Sickle Cell Anemia Drug
SAN FRANCISCO - Dec. 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.
HemaQuest Pharmaceuticals Receives Orphan Drug Designations for Therapeutic to Treat Hemoglobin Disorders
BOSTON - Nov. 4, 2008 - HemaQuest Pharmaceuticals today announced that the U.S. Food and Drug Administration (FDA) has awarded the company orphan drug designations for sickle cell anemia and beta thalassemia for HQK-1001, which is an orally administered therapeutic under clinical development for these two indications.
HemaQuest President and CEO Ronald Berenson, MD, said, "Orphan drug designations confirm the urgent medical need to develop new therapies to treat these two serious and life-threatening hemoglobin disorders. These designations by the FDA also provide us with strong incentives for our novel proprietary drug candidate, HQK-1001, which initially is being developed to treat both sickle cell anemia and beta thalassemia."
Orphan drug status provides certain tax benefits and confers market exclusivity for a minimum of seven years after drug approval by the FDA to encourage companies to develop medicines that affect less than 200,000 people in the United States. Both hemoglobin disorders, sickle cell anemia and beta thalassemia, fall into this category with a combined total of less than 80,000 patients suffering from these inherited blood diseases in the U.S.
Worldwide, sickle cell anemia and beta thalassemia afflict millions of people. Few therapeutic alternatives exist for these serious and life-threatening diseases, which are associated with significant morbidity and reduced patient survival, creating a strong and pressing need for new treatments.
HemaQuest recently completed Phase 1 clinical trials of HQK-1001 in healthy subjects and plans to begin clinical studies of this compound in both sickle cell anemia and beta thalassemia in the near future. The compound's therapeutic potential was discovered by Susan Perrine, MD, the company's chief scientific officer and vice president of clinical affairs, and her colleagues at Boston University.
ABOUT HEMAQUEST PHARMACEUTICALS
HemaQuest Pharmaceuticals (www.HemaQuest.com), which was established in late 2007, is a biopharmaceutical company focused on developing small molecule therapeutics based on its short chain fatty acid derivative (SCFAD) technologies to treat hemoglobin diseases. HemaQuest is also developing other SCFADs that could prove useful in treating other hematological disorders, such as other kinds of anemia and neutropenia. The company's investors include De Novo Ventures, a Palo Alto, Calif., life sciences venture capital partnership; Forward Ventures, a life sciences venture capital firm based in San Diego; and Lilly Ventures of Indianapolis, Ind., the venture capital arm of Eli Lilly and Company.
HemaQuest Announces Initiation of Phase 1 Trial of HQK-1001 to Treat Hemaglobin Disorders
NEWTON, Mass. - Jan. 17, 2008 - HemaQuest Pharmaceuticals, which received its first round of venture financing three months ago, today announced that the U.S. Food and Drug Administration has accepted its application for an investigational new drug (IND) and has started a Phase I clinical trial of its orally administered, patented therapeutic, HQK-1001. The product is being developed to treat the two most common inherited blood diseases, sickle cell anemia and thalassemia.
The Phase I clinical study is being carried out in healthy volunteers, who will receive increasing doses of HQK-1001 to evaluate its safety and pharmacokinetics in anticipation of future clinical trials in patients with these hemoglobin disorders.
HemaQuest President and CEO Ronald Berenson, MD, said, "We are pleased to reach this important milestone within three months of the founding of our company. We are committed to continuing the rapid development path for HQK-1001, which has the potential to be a major advance in treating serious and life-threatening blood disorders."
Susan Perrine, MD, the company's chief scientific officer and vice president of clinical affairs, whose discoveries led to HQK-1001, added, "This clinical trial is the culmination of many years of laboratory and animal studies of HQK-1001. It represents the first step in the clinical development of this exciting new therapeutic, which could make a significant impact on improving the lives of the many patients with sickle cell anemia and thalassemia."
HemaQuest Announces $20M Series A Financing For Anemia Drug Development
NEWTON, Mass. - Nov. 1, 2007 - HemaQuest Pharmaceuticals, a new company developing proprietary small molecule therapeutics to treat serious blood disorders, today announced that it received $20 million in Series A financing.
HemaQuest will use the funds primarily to support the clinical development of its lead drug candidate, an orally administered agent for treating sickle cell anemia and beta thalassemia.
Investors in the funding round include De Novo Ventures, a Palo Alto, Calif., life sciences venture capital partnership; Forward Ventures, a life sciences venture capital firm based in San Diego; and Lilly Ventures of Indianapolis, the venture capital arm of Eli Lilly and Company.
Ronald Berenson, MD, HemaQuest's president and CEO, said, "The HemaQuest technology platform is capable of producing the first safe, disease-modifying drugs for treating sickle cell anemia and beta thalassemia. These hemoglobin disorders afflict several million patients in the U.S., Europe, and worldwide. With few treatment options today, the therapeutic agents we are developing have the potential to make a real difference in many patients' lives."
HemaQuest intends to submit an IND for its lead molecule before the end of 2007. In addition, the company said it plans to develop additional programs in its pipeline, including novel treatments for anemia, neutropenia, and other hematological disorders.
"The company brings together world-class scientists, potential breakthrough drugs, and a proven development team, putting it in an ideal position to make major advances in treating patients with serious blood diseases," said Lilly Ventures' Bryan Dunnivant, who will serve as chairman of HemaQuest's board.
HemaQuest's Chief Scientific Officer and Vice President, Clinical Affairs, Susan Perrine, MD, said, "As a practicing physician, the lack of effective therapies to alleviate the tremendous suffering of patients with hemoglobin disorders is discouraging. I am excited to have the opportunity to develop targeted therapeutics based on patented discoveries made by our co-founder, Douglas Faller, MD, PhD, and my group at Boston University."
Co-founder George Stamatoyannopoulos, M.D., Dr.Sci., Professor of Medicine and Genome Sciences and Director of the Markey Molecular Medicine Center at the University of Washington, one of the world's experts on hemoglobin disorders, added, "The therapies we are developing address the major hemoglobin abnormalities related to sickle cell anemia and thalassemia, and have the potential to be a significant advance in treating these diseases."
Sickle cell anemia and beta thalassemia are hemoglobin disorders, known as hemoglobinopathies. Patients with sickle cell disease suffer from acute painful crises, strokes, lung disease, heart damage, infections, and anemia. In beta thalassemia, severe anemia requires frequent blood transfusions, contributing to iron overload, which damages the heart, liver, and other major organs. With few therapeutic options, death at an early age is common to both of these hemoglobinopathies.