SAN FRANCISCO, Sept. 19, 2011 (GLOBE NEWSWIRE) -- FerroKin BioSciences today announced that the first dose has been administered in an international Phase 2 study of FBS0701, a novel once-daily oral iron chelator in development for the treatment of transfusional iron overload. This is an Open Label, Multi-Center, Single-Dose Pharmacokinetics, and Multiple Dose Study of the Safety, Efficacy and Tolerability of FBS0701 in a Pediatric Population with Transfusional Iron Overload.
"We remain enthusiastic about the progress this molecule has made in clinical development. Children represent a significant proportion of the population of patients with hereditary anemias who also have iron overload from transfusions. Our hope is to extend the clinical benefits of a new chelator to these patients. We are grateful to be working with such high quality clinical investigators who appreciate the need for new chelators," said Hugh Young Rienhoff, Jr., MD, founder and chief executive officer of FerroKin BioSciences. "Data from this important study will help us refine our understanding of the relative safety and efficacy of FBS0701 and place us in a solid position to succeed in our upcoming pivotal Phase 3 studies next year."
This Phase 2 open-label study will assess the pharmacokinetics, safety, efficacy and tolerability of FBS0701 in pediatric patients with transfusion-dependent anemias that may include: hereditary anemias such as sickle cell disease, β-thalassemia, and Diamond-Blackfan anemia. The study consists of two phases: the pharmacokinetic phase, using a single dose of FBS0701; and the chronic dosing phase, during which patients will receive an additional 48 weeks of FBS0701 dosing.
"I am pleased to see the FBS0701 development program progressing to this pediatric study. This is a significant step forward for a promising agent. We will be very interested to learn the pharmacokinetics, safety, tolerability and efficacy in children and adolescents requiring chronic transfusions." said Ellis Neufeld, M.D., Professor of Pediatrics, Associate Director of Hematology at the Children's Hospital Boston and the Lead Investigator on the study.
The study is being conducted at multiple sites in North America, the Middle East, and Europe. For a more detailed description of the clinical trial protocol, inclusion and exclusion criteria, and a list of participating sites, please visit clinicaltrials.gov and enter the study identifier - NCT01363908.
Additional Phase 2 and Phase 3 studies with FBS0701 are being planned.
About Iron Overload
Iron overload occurs in patients who are chronically transfused with red blood cells because of persistent anemia. The need for transfusion arises in the setting of both hereditary and acquired anemias — those anemias present at birth or occurring later in life – which include such conditions as thalassemia major, sickle cell anemia, and myelodyplasia. Anemia is a condition in which the number of red blood cells is too low. These conditions affect an estimated 200 million persons worldwide.
About FBS0701
FBS0701 is an orally available iron chelator currently in development for the treatment of transfusional iron overload. FBS0701 has received Orphan Drug Status from both the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA). FerroKin BioSciences has completed four clinical studies on the safety, pharmacokinetics, tolerability, and iron clearing activity of FBS0701: one in healthy volunteers and three in patients with iron overload resulting from transfusion therapy.
About FerroKin BioSciences
FerroKin BioSciences is a clinical-stage biotechnology company based in the San Francisco Bay Area focused on the development of chelation therapies for a broad range of clinical indications.
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