Contact Information: CONTACT: Jerome Lyons HemaQuest Pharmaceuticals T: 206.826.9900
HemaQuest Pharmaceuticals Receives European Orphan Medicinal Product Designations for Therapeutic to Treat Hemoglobin Disorders
| Source: HemaQuest Pharmaceuticals
BOSTON, MA--(Marketwire - March 26, 2009) - HemaQuest Pharmaceuticals today announced that
it has received two orphan medicinal product designations from the European
Commission for its product HQK-1001 for the treatment of sickle cell disease
and beta thalassemia. The new designations are in addition to orphan drug
designations for both diseases granted in the United States last year.
HemaQuest President and CEO Ronald Berenson, MD, said, "Receiving orphan
medicinal product designations for these hemoglobin disorders
is critical to our overall strategy for developing HQK-1001. In contrast to
the United States, where beta thalassemia is relatively rare, there are
several thousand patients in Europe who suffer from beta thalassemia, a
serious and life-threatening disease for which there are currently no
approved drugs to treat the underlying illness.
"For sickle cell anemia, there is a strong need for new drugs to treat this
disorder, which causes significant morbidity and mortality. The incentives
provided by orphan medicinal designation in Europe will enable HemaQuest to
aggressively pursue development of HQK-1001 in Europe as well as the United
States."
In the European Union (EU), products targeted to treat life-threatening or
very serious conditions that affect fewer than five in 10,000 people are
eligible for orphan medicinal product designation. Such status provides
significant advantages and assistance in obtaining marketing authorization
in the EU, including 10-year market exclusivity in the EU once the product
is approved, direct assistance from the EMEA in preparing the product
development plan, access to EMEA centralized licensing procedures, and
reduced fees.
Worldwide, sickle cell anemia and beta thalassemia afflict millions of
people. Few therapeutic alternatives exist for these serious and
life-threatening diseases, and they 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, an orally
administered therapeutic, 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.