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HemaQuest Pharmaceuticals Receives Orphan Drug Designations for Therapeutic to Treat Hemoglobin Disorders
| Source: HemaQuest Pharmaceuticals
BOSTON, MA--(Marketwire - November 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.