Promising Results For VX-770, An Oral Investigational Agent That Targets A Defective Protein Responsible For Cystic Fibrosis

Main Category: Cystic Fibrosis
Also Included In: Clinical Trials / Drug Trials;  Respiratory / Asthma
Article Date: 14 Jun 2008 - 4:00 PDT

email to a friend   printer friendly   view / write opinions   rate article

At the 31st European Cystic Fibrosis Society (ECFS) Annual Meeting in Prague, Czech Republic, researchers presented interim results from the first clinical trial of the investigational oral agent VX-770 in cystic fibrosis patients.

Data from the interim analysis suggested that dosing of VX-770, an investigational CF potentiator, as an oral agent for 14 days resulted in improved lung function and in an improved function of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein as measured by changes in sweat chloride levels and changes in nasal potential difference (NPD).

The data was presented in an oral session at the ECFS on Friday June 13 at 5:30 p.m. CEST p.m. by Frank Accurso, M.D., Director of the Cystic Fibrosis Center and Professor of Pediatrics at the University of Colorado School of Medicine, USA.

"While these are early data, it is unprecedented for an investigational oral compound for the treatment of CF to have such a marked effect on multiple measures of CF disease activity. We saw an average improvement in lung function of 10 percent in patients receiving the highest dose, compared to no observed improvement in patients who received placebo," said Dr. Accurso.

"These data suggest that VX-770 may be able to improve lung function by targeting an underlying defect in CFTR that causes the disease. In patients with CF, inadequately functioning or missing CFTR is believed to result in abnormal balance of fluid and salt in the airways.

These are early clinical data in a subset of patients with malfunctioning CFTR, but an important proof-of-concept, and we look forward to evaluating the longer-term safety and efficacy of VX-770 in additional studies."

Vertex Pharmaceuticals
www.vpharm.com