FBS0701 is a once-daily oral capsule in development for the treatment of iron overload due to chronic blood transfusions in adults and children. FBS0701 has received Orphan Product designation from both the U.S. Food and Drug Administration and the European Medicines Agency. FerroKin BioSciences has completed four Phase 1 clinical trials to evaluate the safety, tolerability, pharmacokinetics and iron clearing activity of FBS0701 in healthy volunteers and patients. Phase 2 dose-ranging studies in adults and children are ongoing. Initial data from the adult study, presented at the American Society of Hematology in December, supports the potential for FBS0701 as an effective iron chelator with a favorable safety profile.
About Iron Overload
Although iron is an element essential for life - the typical adult human contains 1,000-4,000 mg of iron - the body has no means to eliminate excess iron. Repeated transfusions can result in the accumulation of iron in key organs, compromising their normal function. Progressive iron overload can eventually lead to organ failure, particularly the heart and liver.
The need for repeated transfusions arises in the setting of both hereditary and acquired anemias. The most common forms of congenital anemia are the hemoglobinopathies, a group of related anemias including alpha and beta thalassemia and sickle cell disease affecting hemoglobin production. Acquired anemias tend to occur later in life and represent a diverse collection of conditions characterized by impaired blood cell production collectively called Myelodysplastic Syndrome (MDS).Seaview Securities LLC acted as financial adviser to FerroKin Biosciences, Inc. (1) EvaluatePharma ® consensus analyst estimates (2) N Engl J Med 2011; 364:146-156 January 13, 2011 Notes to editors SHIRE PLC Shire's strategic goal is to become the leading specialty biopharmaceutical company that focuses on meeting the needs of the specialist physician. Shire focuses its business on attention deficit hyperactivity disorder, human genetic therapies, gastrointestinal diseases and regenerative medicine as well as opportunities in other therapeutic areas to the extent they arise through acquisitions. Shire's in-licensing, merger and acquisition efforts are focused on products in specialist markets with strong intellectual property protection and global rights. Shire believes that a carefully selected and balanced portfolio of products with strategically aligned and relatively small-scale sales forces will deliver strong results.