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EPIX Pharmaceuticals, Inc. (EPIX) Achieves Milestone from Collaboration with Cystic Fibrosis Foundation Therapeutics, Inc.

3/11/2009 9:15:35 AM

LEXINGTON, Mass.--(BUSINESS WIRE)--EPIX Pharmaceuticals, Inc. (NASDAQ:EPIX - News), a biopharmaceutical company focused on discovering and developing novel therapeutics through the use of its proprietary and highly efficient in silico drug discovery platform, today announced that it has achieved another milestone in its collaboration with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the nonprofit affiliate of the Cystic Fibrosis Foundation. Under the terms of the collaboration EPIX has earned an additional $500,000, bringing the total amount of milestone payments achieved under this collaboration to $5 million. The milestone payment is part of a research, development and commercialization agreement between EPIX and CFFT that focuses on discovering potential drug therapies targeting the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) ion channel. Under the terms of the agreement, EPIX will own all worldwide rights to any compound that results from the collaboration.

To earn the milestone, EPIX successfully completed in silico high throughput screening at four distinct sites in the delta F508 mutational form of CFTR for small molecules that may correct the defects in the mutation’s cellular processing and chloride channel gating. The delta F508 is the most common mutation of the key protein associated with cystic fibrosis. EPIX and CFFT believe that ligands to CFTR may act as molecular chaperones, stabilizing the folded structure and modulating CFTR activity.

“We continue to make progress in our collaboration with CFFT and believe that our research may eventually lead to significant advances in the treatment of cystic fibrosis,” said Elkan Gamzu, Ph.D., president and chief executive officer of EPIX. “This achievement, coupled with our announcement in September 2008 regarding the identification of dual-acting compounds that act as both potentiators and correctors, moves us one important step closer to lead optimization and the identification of clinical candidates.”

Early in the collaboration, EPIX became the first company to generate a 3-D model of the CFTR, a considerable step forward in the CF research field. All of these milestones resulted from EPIX’s highly efficient discovery platform which integrates proprietary in silico modeling techniques with hypothesis-driven approaches for the design and synthesis of compounds.

“Our collaboration with EPIX continues to yield important scientific advances in the field of cystic fibrosis research,” added Robert J. Beall, Ph.D., president and chief executive officer of Cystic Fibrosis Foundation. “This is the first time that a 3-D model has been used to test for compounds that may bind to multiple sites on the CFTR and play a key role in a future treatment for CF. We are pleased with the tangible results stemming from our relationship with EPIX and we look forward to continuing our work together as we focus on our goal of creating new therapies to treat CF.”

Cystic fibrosis (CF) is a life-threatening genetic disease that affects approximately 30,000 children and adults in the United States and nearly 70,000 people worldwide. It causes life-threatening lung infections and serious digestive complications. 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.

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation, the leading organization focused on curing and controlling cystic fibrosis, has invested more than $300 million in drug research with biotech companies since 1998 to develop therapies to fight CF. As a result, the Foundation has built a drug pipeline with more than 30 promising therapies in development. Virtually all of the approved cystic fibrosis therapies available today were made possible because of the support of the Foundation. Based in Bethesda, Md., the Foundation has 80 chapters and branch offices and supports and accredits a nationwide network of 115 CF care centers that provide treatment and vital resources to patients and families. For more information, visit

About EPIX

EPIX Pharmaceuticals is a biopharmaceutical company focused on discovering and developing novel therapeutics through the use of its proprietary and highly efficient in silico drug discovery platform. The company has a pipeline of internally-discovered drug candidates currently in clinical development to treat diseases of the central nervous system (see and lung conditions. EPIX also has collaborations with leading organizations, including GlaxoSmithKline, Amgen and Cystic Fibrosis Foundation Therapeutics. For more information, please visit the company’s website at

This news release contains express or implied forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are based on current expectations of management. These statements relate to, among other things, our expectations regarding our drug discovery efforts, the future development of our in-silico methodologies and the benefits of our collaboration with Cystic Fibrosis Foundation Therapeutics, Inc. These statements are neither promises nor guarantees, but are subject to a variety of risks and uncertainties, many of which are beyond our control, and which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, among other things: risks that product candidates may fail in the clinic or may not be successfully marketed or manufactured; risks relating to our ability to advance the development of product candidates currently in the pipeline or in clinical trials; failure to obtain the financial resources to complete development of product candidates; our inability to further identify, develop and achieve commercial success for new products and technologies; our inability to achieve commercial success for our products and technologies; our failure to comply with regulations relating to our products and product candidates, including FDA requirements; the risk that the FDA may interpret the results of our studies differently than we have; and risks of new, changing and competitive technologies and regulations in the U.S. and internationally. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. We undertake no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise. For additional information regarding these and other risks that we face, see the disclosure contained in our filings with the Securities and Exchange Commission, including our most recent Annual Report on Form 10-K.


EPIX Kim C. Drapkin, 781-761-7602 Chief Financial Officer or Pure Communications Jennifer Beugelmans, 646-596-7473

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