Cambridge, Mass. – February 26, 2009 – FoldRx announced today that it will host a program at its company headquarters on Friday, February 27 to raise awareness of rare diseases, including Transthyretin Amyloidosis (ATTR), a rare and fatal condition for which FoldRx is working to develop a treatment. The event is being held in conjunction with the National Organization for Rare Disorders (NORD) Rare Disease Day 2009 (www.rarediseaseday.org), of which FoldRx is one of the sponsors.
In ATTR, amyloid fibrils are formed as a result of the ‘misfolding’ of a protein called transthyretin (TTR). These fibrils accumulate in various tissues including the heart and peripheral nerve tissues, leading to cardiomyopathy and neuropathy. Symptoms include shortness of breath and ankle swelling (cardiomyopathy) and progressive loss of sensation and motor function, pain and weakness (neuropathy).
“FoldRx is committed to advancing disease-modifying therapeutics to meet the needs of patients with ATTR, as well as Cystic Fibrosis and other rare diseases,” noted Richard Labaudinière, president and CEO of FoldRx Pharmaceuticals, Inc. “This event is an opportunity to raise public awareness of the needs of patients affected by ATTR and their physicians and to reinforce our commitment to advance a potential therapy as rapidly as possible.”
FoldRx’s lead compound, Fx-1006A, a novel compound designed to stop the progression of this disease by preventing the misfolding of TTR and accumulation of amyloid fibrils, is currently being evaluated in a number of clinical trials, including a pivotal Phase II/III study in patients with TTR amyloid polyneuropathy. Results of this study are anticipated in Summer 2009.
In addition to conducting the clinical studies with Fx-1006A, FoldRx is dedicated to improving the understanding of this disease. The TRACS study (Transthyretin Amyloid Cardiac Study) was an observational study that evaluated the course of TTR amyloid cardiomyopathy for up to two years in 29 patients. A more ambitious project is the Transthyretin Amyloidosis Outcome Survey (THAOS), a world-wide, web-based patient registry which is designed to collect health information on patients affected by ATTR. The data will help doctors to better understand and improve the treatment and management of patients with this disease.
As part of FoldRx’s ongoing commitment to finding therapies for this and other rare diseases such as Cystic Fibrosis, the company is supporting NORD in its sponsorship of Rare Disease Day. Rare Disease Day, held on the last day of February each year, is designed to raise awareness with policy makers and the public of rare diseases and of their impact on patients’ lives.
“People with rare diseases remain a medically underserved population in every country,” said Peter Saltonstall, president of the National Organization for Rare Disorders (NORD), which is sponsoring Rare Disease Day in the U.S. “This day is intended to bring together the patients and families with rare diseases to discuss the need for greater awareness, more research, and better access to diagnosis and treatment.”
FoldRx also has a novel yeast-based discovery platform, which has been applied to the discovery of a lead prototype series for treatment of Parkinson disease and tested in an in vivo model of neurodegeneration. In addition, FoldRx is working on the development of a series of novel drug prototypes that have shown potential in vitro to correct the protein-folding defect associated with cystic fibrosis.
About FoldRx Pharmaceuticals, Inc.
FoldRx Pharmaceuticals is a development and discovery company focusing on first-in-class, disease-modifying, small molecule therapeutics to treat diseases of protein misfolding and aggregation (amyloidosis). Protein misfolding is increasingly being recognized as an underlying cause of many chronic degenerative diseases. By applying FoldRx’s proprietary expertise in protein folding and its platform for drug and target discovery, the company is building a pipeline. FoldRx’s initial pipeline includes a program in clinical development to treat genetic neurologic and cardiovascular disorders, TTR Amyloidosis, including Polyneuropathy and Cardiomyopathy, and discovery programs in Cystic Fibrosis and neurodegenerative diseases, including Parkinson’s disease, based on its broad, proprietary, yeast-based drug discovery platform. For more information on FoldRx, please visit the company’s web site at www.foldrx.com.
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For information contact: Download a PDF copy here.
Krystle Ficco
Feinstein Kean Healthcare
617-761-6702
krystle.ficco@fkhealth.com