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CYSTIC FIBROSIS FOUNDATION AWARDS $21 MILLION TO VERTEX.


Vertex Pharmaceuticals Vertex Pharmaceuticals is a biotechnology company with activities spanning the length of the pharmaceutical product pipeline, from target identification through to clinical trials and marketing.  Incorporated (Nasdaq: VRTX An earlier real time operating system for embedded systems from Mentor Graphics, Wilsonville, OR (www.mentor.com). VRTX was the progenitor of the VxWorks OS from Wind River and was superseded by Nucleus OS.

VRTX - Virtual Real-Time Executive.
) has expanded its drug discovery and development collaboration with Cystic Fibrosis Foundation The Cystic Fibrosis Foundation (CFF) is a non-profit organization in the United States established to provide the means to cure and control cystic fibrosis. The Foundation provides information about cystic fibrosis (CF) and finances CF research that aims to improve the  Therapeutics, Inc. (CFFT CFFT Complex Fast Fourier Transform
CFFT Civitavecchia Fruit & Forest Terminal (Italy)
CFFT Central Florida Floor Tech, Inc (Florida) 
), the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. Under the expanded collaboration, CFFT will make approximately $21 million in contracted research payments to Vertex through 2004 and 2005, providing funding for Vertex's late-stage CF drug discovery effort. Recent progress at Vertex in the design of small molecule drugs targeting the basic defect in cystic fibrosis cystic fibrosis (sĭs`tĭk fībrō`sĭs), inherited disorder of the exocrine glands (see gland), affecting children and young people; median survival is 25 years in females and 30 years in males.  (CF) provides the foundation for the new agreement. Vertex will retain the right to develop and commercialize any compounds discovered.

Vertex Pharmaceuticals initiated its research in CF in May 2000 as part of collaboration with the CF Foundation. Using proprietary expertise in ion channels, including high-content cell assays and medicinal chemistry Medicinal or pharmaceutical chemistry is a scientific discipline at the intersection of chemistry and pharmacology involved with designing, synthesizing and developing pharmaceutical drugs. , Vertex has focused on designing selective ion channel modulators that could restore the function of the defective Cystic Fibrosis Transmembrane transmembrane /trans·mem·brane/ (trans-mem´bran) extending across a membrane, usually referring to a protein subunit that is exposed on both sides of a cell membrane.

trans·mem·brane
adj.
 conductance Regulator (CFTR) protein. Defective CFTR is one of the key factors that ultimately leads to the symptoms, complications and mortality in people with CF. This defective channel affects the transport of sodium and chloride in and out of the cells, which leads to the thick, sticky mucus in the lungs and the pancreas.

"Through its state-of-the-art technologies, Vertex has enabled the rapid design of small molecule compounds that, in laboratory studies, have corrected the defective ion transport in CF and therefore may address the root cause of disease," said Robert J. Beall, Ph.D., president and CEO (1) (Chief Executive Officer) The highest individual in command of an organization. Typically the president of the company, the CEO reports to the Chairman of the Board.  of the CF Foundation and CFFT. "By expanding our collaboration with Vertex, we are assuring that these compounds move forward quickly and prudently, in the hopes that at least one may become an effective basic defect therapy in CF."

Under the terms of the agreement, CFFT will issue a milestone payment to Vertex for advancement of the first compound into clinical development. Vertex expects to recognize approximately $7 million as revenue from this collaboration for the calendar year 2004.

"Our new agreement with CFFT is the first of a series of business and scientific advancements in 2004 that we believe will validate Vertex's leadership position in ion channel drug discovery," said Joshua Boger, Ph.D., Vertex's chairman and CEO. "In addition to providing near-term revenue to Vertex, this agreement brings forward potential breakthrough drug candidates for CF toward clinical development, while retaining for Vertex full commercialization rights. This underscores Vertex's commitment to developing breakthrough drugs for serious diseases and expands our ability to bring some of these breakthrough drugs to the marketplace ourselves."

CF Drug Discovery at Vertex Pharmaceuticals

Vertex has designed proprietary compounds for CF that are highly active in vitro in vitro /in vi·tro/ (in ve´tro) [L.] within a glass; observable in a test tube; in an artificial environment.

in vi·tro
adj.
In an artificial environment outside a living organism.
 and may reverse the effects of the defective ion channel via two separate mechanisms of action. One class of compounds acts as "potentiators", directly increasing the gating ability of the defective ion channel. Vertex has identified a second class of compounds that act as "correctors", enhancing the number of CFTR channels at the cell surface. Most recently, both classes of compounds have been shown to improve CFTR function in bronchial bronchial /bron·chi·al/ (brong´ke-al) pertaining to or affecting one or more bronchi.

bron·chi·al
adj.
Relating to the bronchi, the bronchial tubes, or the bronchioles.
 epithelial cells Epithelial cells
Cells that form a thin surface coating on the outside of a body structure.

Mentioned in: Corneal Transplantation
 that have been isolated from CF patients. Drug discovery efforts under the new collaboration will focus on optimizing and selecting drug candidates for clinical development from one or both of these compound classes.

Drug candidates identified in Vertex's CF drug discovery program may have important future application in CF, as well as more broadly in the treatment of pulmonary diseases.

About Vertex

Vertex Pharmaceuticals Incorporated, Cambridge Mass., is a global biotechnology company committed to the discovery and development of breakthrough small molecule drugs for serious diseases. The company's strategy is to commercialize its products both independently and in collaboration with major pharmaceutical partners. Vertex's product pipeline is principally focused on viral diseases, inflammation, autoimmune diseases Autoimmune diseases
A group of diseases, like rheumatoid arthritis and systemic lupus erythematosus, in which immune cells turn on the body, attacking various tissues and organs.

Mentioned in: Complement Deficiencies, Premature Menopause
 and cancer. Vertex co-promotes the new HIV HIV (Human Immunodeficiency Virus), either of two closely related retroviruses that invade T-helper lymphocytes and are responsible for AIDS. There are two types of HIV: HIV-1 and HIV-2. HIV-1 is responsible for the vast majority of AIDS in the United States.  protease inhibitor protease inhibitor (prō`tē-ās'), any of a class of drugs that interfere with replication of the AIDS virus (HIV), by blocking an enzyme (protease) necessary in the late stages of its reproduction.  Lexiva(tm) (fosamprenavir calcium) with GlaxoSmithKline.

About Cystic Fibrosis and the Cystic Fibrosis Foundation

Cystic fibrosis (CF) is a genetic disease affecting approximately 30,000 people in the United States. A defective gene causes the body to produce abnormally thick, sticky mucus that leads to chronic and life-threatening lung infections and impairs digestion. When the CF Foundation was created in 1955, few children lived to attend elementary school. Today, because of research and care supported by the CF Foundation with money raised through donations from individuals, corporations and foundations, the median age of survival for people with CF is in the early 30s.

The mission of the Cystic Fibrosis Foundation is to assure the development of the means to cure and control CF and to improve the quality of life for those with the disease. For information on CF and the programs of the CF Foundation, call 800/FIGHT CF or 301/841-2665 or visit http://www.cff.org.

For more informattion about Vertex, call 617/444-6108.
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Publication:Biotech Financial Reports
Geographic Code:1USA
Date:Jul 1, 2004
Words:803
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