News

BIRMINGHAM, Ala. (Feb. 8, 2010) - A study conducted at Children's Hospital of Alabama and about 20 international research sites investigating the impact of a new compound on the defective protein responsible for most cases of cystic fibrosis (CF) has shown promising results, according to J.P. Clancy, MD, principal investigator of the study and director of the UAB Pediatric Pulmonary Center at the Children's Hospital of Alabama.

Results from a preliminary analysis of data from a 28-day Phase 2a clinical trial of VX-809 in CF patients age 18 years and older conducted in part at Children's showed that the drug was not only well-tolerated across the four dose groups studied. VX-809 treatment also reduced levels of chloride in sweat of treated subjects in a does dependent manner, pointing towards restoration of F508del CFTR activity. CFTR normally regulates salt and water movement in several tissues of the body and the most common mutation found in CF patients is F508del CFTR. CF patients have higher sweat chloride levels than the average population. Lowering those levels by restoring activity to CFTR is expected to reduce the severity of symptoms in CF patients, particularly the viscosity of mucus that they have difficulty expelling. Thickened mucus puts patients at risk for repeated lung infections and impairs other organ systems. Cystic fibrosis is a genetic, fatal disease; half of CF patients die by age 37.

The encouraging outcome of the VX-809 trial opens the door to future trials of VX 809 combined with VX-770, another CFTR-active compound recently shown to reduce sweat chloride levels in other CF patients. Clancy believes that the combined therapy could decrease salt levels even further. The approach is among the first to target and treat the underlying cause of the disease, rather than the symptoms. "There is high interest in the CF community in new approaches to CF therapy and we look forward to the future exploration of VX-809 and VX-770 as part of a novel combination regimen aimed at treating the majority of CF patients," Clancy said.

While the data is encouraging, Clancy is quick to note that the results of the studies do not represent a cure at this point. "These changes are an important step, a stepping stone," he said.

About the Children's Hospital of Alabama
The Children's Hospital of Alabama is the 10th busiest pediatric medical center in the United States. Located in Birmingham, Children's is one of only about 45 freestanding acute care hospitals of its kind in the nation and the only hospital in Alabama dedicated solely to the care and treatment of children. It is licensed for 275 beds.

Children's is home to the state's only Level I pediatric trauma center, the only pediatric renal care center, the largest pediatric burn center in the Southeast and is one of the largest pediatric outpatient centers in the nation. Children's treats 95-percent of the children in Alabama diagnosed with childhood cancers and other blood disorders, and offers the only pediatric bone marrow transplant program in the state. The UAB Pediatric Pulmonary Center at Children's employs a multi-disciplinary approach to improving the health of children with respiratory conditions. The center provides patient care in a family-centered environment, patient education and conducts research to develop new treatment methods.

Children's is a private, not-for-profit hospital governed by a volunteer board of trustees. The hospital was recently recognized for the excellence of its nursing care by achieving Magnet designation from the American Nurses Credential Center. Children's will celebrate its centennial anniversary in 2011. For more information, visit www.chsys.org.

About Vertex
Vertex Pharmaceuticals Incorporated, based in Cambridge, Mass., is a global biotechnology company committed to the discovery and development of breakthrough small molecule drugs for serious diseases. Vertex's product pipeline is focused on viral diseases, cystic fibrosis, inflammation, autoimmune diseases, epilepsy, cancer and pain. It initiated its CF research program in 1998 as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, Inc., the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation.

About Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease.

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Contact: Kathy Bowers, 205/939-9002 (Kathy.bowers@chsys.org)