Navigation Links
Cystic Fibrosis Foundation announces positive early results for phase 2 clinical trial of VX-770
Date:3/27/2008

Bethesda, MD, March 27, 2008 The Cystic Fibrosis Foundation announced today that VX-770, an oral drug in development that targets a basic defect in CF, showed promising results in an ongoing Phase 2a clinical trial for patients who carry the G551D mutation of CF. The drug is being developed by Vertex Pharmaceuticals Incorporated.

Patients who took the drug for 14 days showed significant improvements in several key indicators of cystic fibrosis, including lung function, nasal potential difference measurements and sweat chloride levels. The findings suggest that VX-770 improves function of what is known as the faulty CFTR protein. This early data is promising and could have important implications for studies of other drugs in development.

This is the first time that any potential therapy has improved the abnormal sweat chloride (salt) levels in a person with CF. Excessive sweat chloride is a key clinical indicator of cystic fibrosis. The sweat test is the traditional diagnostic test for CF.

These early results are an extraordinary endorsement of our hypothesisthat small molecules can correct the basic defect and affect the clinical indicators of cystic fibrosis, said Robert J. Beall, Ph.D., president and CEO of the Foundation. The emerging data for VX-770 represents the most exciting results weve seen from a Phase 2 trial and increase our confidence that were on the right track.

The compound VX-770 resulted from a collaboration between the Foundation and Vertex. In 1998, the Foundation approached Aurora Biosciences (acquired by Vertex in 2001) and made an initial investment to use cutting-edge technologyknown as high throughput screeningto find compounds to attack the core defect in CF. Since that time, the Foundation has invested $79 million in the project, and the two organizations have closely collaborated to advance VX-770 and a second CF compound, known as VX-809, through research and into development.

At a time when the model of venture philanthropy was wholly untested, the Cystic Fibrosis Foundation was willing to take a chance that Vertexs CF program would yield meaningful results for people with cystic fibrosis, said Joshua Boger, Ph.D., president and CEO of Vertex Pharmaceuticals. The project carried significant risk that would have dissuaded many companies and investors from moving forward. Today, however, with the Foundations financial, scientific and clinical network support, we have been able to bring two novel CF compounds into development and are encouraged by these early results for VX-770.

Overall, the Foundation has invested nearly $300 million in CF drug research with dozens of biotech collaborators nationwide. These collaborations with for-profit companies are part of the Foundations unique venture philanthropy business model. The model, which is being emulated by other orphan disease organizations, has spawned a drug pipeline of more than 30 therapies in development.

Part One of the Phase 2a trial of VX-770 studied 20 cystic fibrosis patients over a 14-day period. Part Two of the study is expected to begin in the second quarter of 2008. Additional studies will evaluate the longer-term safety and efficacy of the compound.

Cystic fibrosis is a life-threatening genetic disease that affects about 30,000 people in the United States and 70,000 worldwide. CF is caused by a genetic mutation that results in a malfunctioning (or missing) protein that results in an imbalance of salt and water. This imbalance causes a cascade of mucus plugging, infection and inflammation in the lungs and other organs. The primary cause of death in CF is lung disease.

The predicted median age of survival for people with CF is age 37 more than double what it was 25 years ago.


'/>"/>

Contact: Laurie Fink
lfink@cff.org
301-841-2602
Cystic Fibrosis Foundation
Source:Eurekalert

Related medicine news :

1. Ibuprofen can slow lung disease in children with cystic fibrosis, Canadian study shows
2. Anaheim to Host Worlds Largest Cystic Fibrosis Medical Meeting
3. AMRI and Cystic Fibrosis Foundation Launch Major Research Collaboration
4. Cystic Fibrosis Foundation Named Charitable Organization of the Year by USA Track & Field
5. Cystic Fibrosis foundation lays out new treatment guidelines
6. Experts Release New Cystic Fibrosis Guidelines
7. Lung Transplants May Not Help Most Cystic Fibrosis Children
8. Ibuprofen associated with slower lung function decline in children with cystic fibrosis
9. Ibuprofen Preserves Lung Function in Kids With Cystic Fibrosis
10. Montana Passes Newborn Screening Law for Cystic Fibrosis
11. Cystic Fibrosis Foundation reports upward trend for key health outcomes
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:2/5/2016)... ... ... The American public tends to feel uncomfortable about drinking recycled waste water ... well water. The recent experience with lead contaminated water in Flint, Michigan, according to ... increasing public acceptance of recycled waste water as drinking water. , The Flint ...
(Date:2/5/2016)... ... ... Boar’s Head Brand®, one of the nation’s leading providers of premium delicatessen foods, ... out of your party preparation – follow these easy, yet delicious recipes with the ... , “The key to hosting a successful game-day party is creating a flavorful menu ...
(Date:2/5/2016)... (PRWEB) , ... February 05, 2016 , ... Dr. Justin ... announce their 2nd Annual No Cost Dental Day to individuals in need. The event ... purpose of this No Cost Dental Day is to provide dental care to community ...
(Date:2/5/2016)... ... ... Colorize is a web theme package created exclusively for Final ... dynamic moving camera. Colorize is perfect for personal and web related videos and its ... to 5 focus points per scene, stage floor scene presets that are great for ...
(Date:2/5/2016)... ... February 05, 2016 , ... California Mobile Kitchens ... debut of their latest mobile kitchen model, featuring customizable stainless steel interiors and ... commercial kitchens for use anywhere in the U.S. Many of their units can ...
Breaking Medicine News(10 mins):
(Date:2/5/2016)... -- Site Profile: --> ... Speech Recognition People, announced their latest primary healthcare case study where ... turnaround times and to save the practice money. Site ... Challenge: --> ,- Wirral CCG ,- VoicePower ... doctors ,- Wirral CCG ,- VoicePower client since 2013 ...
(Date:2/4/2016)... 2016  Edwards Lifesciences Corporation (NYSE: EW ), ... disease and critical care monitoring, announced today that it ... with Morgan Stanley & Co. LLC to repurchase $325 ... of the Company,s previously authorized program to repurchase up ... --> --> Under ...
(Date:2/4/2016)... , Feb. 4, 2016  SciClone Pharmaceuticals, Inc. ... it has entered into a settlement agreement with ... fully resolving the SEC,s investigation into possible violations ... the terms of the settlement agreement, SciClone has ... including disgorgement, pre-judgment interest and a penalty.  This ...
Breaking Medicine Technology: