FerroKin Opens Phase 2 Trial of Oral Chelator

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Offline 7assan

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FerroKin Opens Phase 2 Trial of Oral Chelator
« on: September 13, 2010, 03:52:08 PM »
September 10, 2010 - FerroKin BioSciences has announced the initiation of an international Phase 2 study of FBS0701, a novel once-daily iron chelator in development for the treatment of transfusional iron overload. The Phase 2 study will assess the safety, tolerability, and pharmacodynamics of FBS0701.

 

"We are excited about the potential of FBS0701 in treating iron overloaded patients," said Hugh Young Rienhoff, Jr., MD, founder and chief executive officer of FerroKin BioSciences. "Data from this important study will help us further establish the relative safety and efficacy of FBS0701 in this patient population and we are pleased to have begun dosing patients."

The Phase 2 open-label, 24-week study will evaluate two dose levels of FBS0701 administered orally once daily. Patients in this study will have transfusion-dependent anemias which include: hereditary anemias such as sickle cell disease, B-thalassemia, and Diamond-Blackfan anemia and acquired anemias such as myelodysplastic syndrome and other forms of bone marrow failure.

"There remains a significant clinical need for an iron chelator that is safe and well tolerated by all patients," said Ellis Neufeld, M.D., Professor of Pediatrics at Children's Hospital Boston and lead clinical investigator of the Phase 2 study.

Currently, the study is being conducted at multiple sites in the US, Middle East, Asia, 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 -- NCT01186419. 

Additional studies including a study of FBS0701 in the pediatric population and a Phase 3 study are being planned.

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 three clinical studies on the safety, pharmacokinetics, tolerability, and iron clearing activity of FBS0701: one in healthy normal volunteers, and two in patients with iron overload resulting from transfusion therapy associated with the management of both hereditary and acquired chronic anemias, including sickle cell disease, thalassemia and myelodysplastic syndrome.

About Ferrokin Biosciences

FerroKin BioSciences is a clinical stage biotechnology company based in the San Francisco Bay Area and focused on developing chelation therapies for a broad range of clinical indications.



http://thalassemia.org/index.php?option=com_content&view=article&id=346:ferrokin-opens-phase-2-trial-of-oral-chelator&catid=1:latest-news

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Offline Scott

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Re: FerroKin Opens Phase 2 Trial of Oral Chelator
« Reply #1 on: September 14, 2010, 11:33:09 AM »
Excellent news!!

Re: FerroKin Opens Phase 2 Trial of Oral Chelator
« Reply #2 on: September 23, 2011, 07:29:10 AM »
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PRESS RELEASE
Sept. 19, 2011, 12:50 p.m. EDT
FerroKin BioSciences Announces Start of a Phase 2 Clinical Trial of FBS0701 as a Novel Oral Treatment for Transfusional Iron Overload in a Pediatric Population

SAN FRANCISCO, Sep 19, 2011 (GlobeNewswire via COMTEX) -- 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.

The FerroKin BioSciences logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=10580

This news release was distributed by GlobeNewswire, www.globenewswire.com

SOURCE: FerroKin BioSciences, Inc.



        CONTACT: FerroKin BioSciences
        info@ferrokin.com
       


(C) Copyright 2010 GlobeNewswire, Inc. All rights reserved.

http://www.marketwatch.com/story/ferrokin-biosciences-announces-start-of-a-phase-2-clinical-trial-of-fbs0701-as-a-novel-oral-treatment-for-transfusional-iron-overload-in-a-pediatric-population-2011-09-19

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Offline Sharmin

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Re: FerroKin Opens Phase 2 Trial of Oral Chelator
« Reply #3 on: September 24, 2011, 05:31:28 PM »
Thank you for posting this, it is always great to see more options available for thals.

Sharmin
Sharmin

 

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