Navigation Links
Cystic Fibrosis Foundation announces positive early results for phase 2 clinical trial of VX-770

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
Cystic Fibrosis Foundation

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:
(Date:6/27/2016)... ... June 27, 2016 , ... "FCPX editors can now ... of Final Cut Pro X," said Christina Austin - CEO of Pixel Film Studios. ... Final Cut Pro X users can now reveal the media of their split ...
(Date:6/27/2016)... ... 27, 2016 , ... A revolution is underway. Brooklyn-based ... experience for the millions of people who require these medical transport services annually. ... through the use of technology. Now, SmartEMS has put forth an industry-changing app ...
(Date:6/26/2016)... , ... June 26, 2016 , ... On June 10-11, ... of the 2016 Cereal Festival and World’s Longest Breakfast Table in Battle Creek, MI, ... the city’s history as home to some of the world’s leading providers of cereal ...
(Date:6/26/2016)... ... June 26, 2016 , ... Brent Kasmer, a legally blind and certified personal trainer ... through a fitness app. The fitness app plans to fix the two major problems leading ... a one size fits all type program , They don’t eliminate all the ...
(Date:6/25/2016)... ... , ... Austin residents seeking Mohs surgery services, can now turn to Dr. ... Russell Peckham for medical and surgical dermatology. , Dr. Dorsey brings specialization to include ... in Mohs Micrographic Surgery completed by Dr. Dorsey was under the direction of Glenn ...
Breaking Medicine News(10 mins):
(Date:6/24/2016)... Mass. , June 24, 2016   Pulmatrix, ... pharmaceutical company developing innovative inhaled drugs, announced today that ... Russell Investments reconstituted its comprehensive set of ... "This is an important milestone for Pulmatrix," ... will increase shareholder awareness of our progress in developing ...
(Date:6/23/2016)... Calif. , June 23, 2016 Any dentist ... many challenges of the current process. Many of them do ... of the technical difficulties and high laboratory costs involved. And ... to offer it at such a high cost that the ... it. Dr. Parsa Zadeh , founder of ...
(Date:6/23/2016)... ANGELES , June 23, 2016 ... CAPR ), a biotechnology company focused ... therapeutics, today announced that patient enrollment in its ... in Duchenne) has exceeded 50% of its 24-patient ... enrollment in the third quarter of 2016, and ...
Breaking Medicine Technology: