FoldRx Technology Enables Discovery of Novel Therapeutic Approach for Treating Cystic Fibrosis
News Jul 11, 2008
FoldRx Pharmaceuticals, Inc. has announced that it has discovered a series of novel drug prototypes that have shown potential in vitro to correct the protein-folding defect associated with cystic fibrosis (CF).
The discovery, made through a research collaboration announced last year with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the nonprofit affiliate of the Cystic Fibrosis Foundation, may pave the way to novel drugs capable of repairing the protein misfolding associated with cystic fibrosis, thus helping treat the underlying cause of the disease. The discovery was made using FoldRx’s proprietary yeast-based, high throughput screening platform.
“We are very encouraged by the rapid progress we have made in identifying novel drug prototypes that appear to restore proper functioning of the CFTR protein in tissue samples from CF patients,” said Richard Labaudiniere, Ph.D., president and CEO of FoldRx.
“In addition to the promise this discovery holds for the treatment of CF, it is also further validation of the potential of our yeast-based discovery platform in the development of drugs to treat a wide array of diseases caused by protein trafficking and misfolding.”
Scientists believe that many diseases such as cystic fibrosis, Parkinson's and Alzheimer's are caused by protein misfolding. An imperfectly folded protein, sometimes resulting from major gene mutations, can be rendered ineffective because its trafficking pathway is disrupted, leaving it unable to reach its target destination and leading to disease. Recent studies suggest that the ability to repair this defect may offer a therapeutic strategy for treating a variety of protein-misfolding diseases, including CF.
FoldRx’s drug prototypes for CF are the result of a collaboration with CFFT for which the company will receive a milestone payment based on this achievement. Robert J. Beall, Ph.D., president and CEO of the CF Foundation, noted “The Foundation's goal is to take advantage of every promising technology in the marketplace that may offer advanced treatment options for patients with CF. FoldRx brings a unique approach and cutting edge technology platform with potential to generate new compounds that treat the basic defect in CF, rather than just the symptoms. Their ability to deliver this key milestone in under a year shows the strength of their program and commitment to the disease.”
FoldRx has a number of additional ongoing programs designed to explore treatment for diseases caused by protein trafficking and misfolding, including a pivotal Phase II/III trial with its lead drug candidate, Fx-1006A, for the treatment of patients suffering from Transthyretin (TTR)-associated Amyloidoses with Polyneuropathy (ATTR-PN), a rare genetic disease that affects approximately 10,000 people worldwide, for which enrollment was completed last year.
Efficacy data are expected in July 2009. Furthermore, the yeast platform has been successfully applied to the discovery of a lead prototype series for treatment of Parkinson disease which have been successfully tested in an in vivo model of neurodegeneration.