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:3/24/2017)... ... March 24, 2017 , ... Vighter ... the same time by providing Prehospital Trauma Life Support (PHTLS) course scholarships to ... premier prehospital trauma education developed in cooperation with the American College of Surgeons ...
(Date:3/24/2017)... ... March 24, 2017 , ... Digital Scientists, a software innovation ... the opening of a Greenville, South Carolina location. The lab has set ... Greenville. , “We’ve been working with South Carolina clients for years from our office ...
(Date:3/24/2017)... (PRWEB) , ... March 24, 2017 , ... Texas ... which can be found at 9618 Huebner Road. The clinic is the group’s 7th ... Clinic Director, and Dr. Ali Higgins, PT, will provide care from the clinic, which ...
(Date:3/24/2017)... (PRWEB) , ... March 24, 2017 , ... ... Center, Sedona, Arizona’s Premier Center for Shamanic Healing and Spiritual Awakening, proudly ... Delgado, June 9--24, 2017. This sacred and spiritual journey during the Summer ...
(Date:3/24/2017)... ... 24, 2017 , ... Northridge dentists, Dr. Michel ... sleep apnea and TMJ at their office. TMJ, or temporomandibular joint disorder, has ... type, is increasingly being treated at dental offices with newly developed procedures and ...
Breaking Medicine News(10 mins):
(Date:3/24/2017)... -- Eli Lilly and Company (NYSE: LLY ... in its U.S. operations in 2017. The company,s ... research laboratories, manufacturing sites, and general and administrative ... for Lilly products, as well as its robust ... pain, diabetes and other unmet medical needs.  ...
(Date:3/24/2017)... Tenn. , March 23, 2017  Provectus ... or the "Company"), a clinical-stage oncology and dermatology ... announced Definitive Financing Commitment Term Sheet (the "Definitive ... a group of the Company,s stockholders, who are ... Group" in a Form 8-K filed with the ...
(Date:3/23/2017)... , Mar 23, 2017 Research and ... Analysis & Trends - Industry Forecast to 2025" report to ... ... of around 6.9% over the next decade to reach approximately $3.5 ... estimates and forecasts for all the given segments on global as ...
Breaking Medicine Technology: