EPIX Pharmaceuticals, Inc. has announced that it has received a $500,000 milestone payment from Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation.
In this third milestone from its collaboration with CFFT, EPIX successfully identified a hit compound that corrects the functionality of the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) ion channel in a cell-based model system. A mutation in the CFTR gene is one of the key factors that ultimately leads to the symptoms, complications and premature mortality in people with CF.
The milestone payment is part of a research, development and commercialization agreement between EPIX and CFFT signed in 2005 that focuses on discovering potential drug therapies targeting the CFTR ion channel.
The most common mutations in the CFTR gene can impair the ability of the protein to make it into the cell membrane and, therefore, to transport chloride in and out of cells. In the lungs and gastrointestinal tract, the reduced levels of CFTR in the membrane lead to the development of the abnormally thick, sticky mucous that causes chronic, life-threatening lung infections and impairs digestion in people with cystic fibrosis.
Using its proprietary in silico drug discovery platform, EPIX has modeled several domains of the CFTR protein structure. The goal of this project is to utilize these models to identify compounds that help the CFTR move into the cell membrane more efficiently. EPIX is working with CFFT and using its computational drug discovery capabilities to discover drug candidates that can help restore proper functioning of the CFTR protein.