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:1/23/2017)... (PRWEB) , ... January 23, 2017 , ... "ProRandom is a set of camera tools ... create dynamic looks in Final Cut Pro X," said Christina Austin - CEO of Pixel ... to two layers of text with video footage. ProRandom works by using a virtual ...
(Date:1/23/2017)... ... January 23, 2017 , ... “Crossing the Bar”: a moving and eloquent ... life. “Crossing the Bar” is the creation of published author, Charlotte Hotte, a North ... who credits the inspiration of the book to her sister, Denise, wishes to acknowledge ...
(Date:1/22/2017)... ... , ... Phytocéane invites clients to take an exotic journey deep into the ... MILKY CREAM. Inspired by the beauty of Zanzibar, a Tanzanian archipelago off the coast ... coral to create this gentle, velvety body cream to envelop the skin in moisture ...
(Date:1/21/2017)... (PRWEB) , ... January 21, 2017 , ... Salveo for ... it is bringing its product to the United States as part of its presence ... last 25 years, Alcovit aims to reduce the productions of nasty toxins as a ...
(Date:1/21/2017)... ... January 21, 2017 , ... ... Dermatology has recently joined their multi-specialty medical group. The dermatology practice provides ... services. , “We’re excited to add this excellent dermatology practice to our group’s ...
Breaking Medicine News(10 mins):
(Date:1/23/2017)... remain until legal experts and senior industry leaders involved in supply chain, ... to discuss the flow of pharmaceutical products post Brexit at the only ... In the run up to the show, SMi Group have ... the event download centre . ... Some of those confirmed include: EMA, Aeterna Zentaris, ...
(Date:1/21/2017)... Research and Markets has announced the addition of the ... (Tumor, Clinical Chemistry, Microbiology, Esoteric), By Type of Lab (Hospital, ... HIV/AIDS etc.), Forecast to 2022" report to their offering. ... US ... clinical lab testing, which has evolved as a major source ...
(Date:1/21/2017)... , January 20, 2017 According to ... (Descriptive, Prescriptive), Application (Marketing, R&D, Compliance, SCM), Component (Software, Service), Delivery ... Forecasts to 2021" published by MarketsandMarkets, the market is expected to ... 2016, at a CAGR of 13.3% during the forecast period. ... ...
Breaking Medicine Technology: