News | September 5, 2007

EPIX Pharmaceuticals Achieves Milestone In Collaboration With Cystic Fibrosis Foundation Therapeutics

Lexington, MA - EPIX Pharmaceuticals, Inc. , a biopharmaceutical company focused on discovering and developing drug discovery platform,through the use of its proprietary and highly efficient in silico drug discovery platform, recently 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.

"This milestone marks further progress made in our collaboration with CFFT and, following our recent milestone achievement under our agreement with GlaxoSmithKline, provides another example of the efficiency and productivity of the EPIX discovery engine and development approach," stated Michael G. Kauffman, M.D., Ph.D., chief executive officer of EPIX. "So far we have focused mainly on GPCRs as drug targets, and we are very happy to see the benefits of our in-silico methodologies expand to ion channels, which are also proteins embedded in the cell membrane. This hit is a first step in applying our computational-medicinal chemistry approach to the discovery of a drug candidate that could increase the levels of the CFTR protein and thereby improve the activity of CFTR protein for patients with cystic fibrosis. We look forward to continuing our research efforts with Cystic Fibrosis Foundation Therapeutics."

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.

SOURCE: EPIX Pharmaceuticals, Inc.