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Jun 12 2008, 7:01 PM EST

Clinical Researchers Present Promising Results for VX-770, an Oral Investigational Agent that Targets a Defective Protein Responsible for Cystic Fibrosis

News source: Business Wire

At the 31st European Cystic Fibrosis Society (ECFS) Annual Meeting in Prague, Czech Republic, researchers will today present 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 will be 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."

Interim Analysis Summary:

The results, which were first reported earlier this year and are being presented in a medical meeting for the first time today, are from 20 patients with the G551D mutation in CFTR who received either VX-770 or placebo in addition to standard therapies for 14 days as part of a blinded, randomized, two-period crossover study design. Four subjects received placebo during both 14-day dosing periods; 8 subjects received 25 mg of VX-770 twice-daily during one period and 75 mg in the other; and 8 subjects received 75 mg of VX-770 twice-daily during one period and 150 mg, the highest dose in the study, during the other dosing period. There was a one to four week wash-out between each dosing period.

-- Safety: In the 14-day trial, VX-770 appeared to be well-tolerated. Observed adverse events were similar between VX-770 and placebo treatment. Two serious adverse events were observed in one patient and were not attributed to VX-770.

-- Lung Function: In the Phase 2a trial, lung function in patients was assessed with FEV(1), a standard test that measures the amount of air that can be exhaled in one second. FEV(1) is the lung function test most commonly used to monitor progression of airway disease in CF patients. Patients with CF typically experience a decline in lung function of 1-2% per year during their life, as measured by FEV(1). Over the 14-day dosing period in the Phase 2a study, patients receiving the highest dose of VX-770 (150mg twice daily) showed a mean increase from baseline in FEV(1) of 10.1%, or 0.22L (p less than 0.008). In contrast, patients receiving placebo showed a slight decrease in FEV(1) (less than 1%; 0.03L) over the 14-day period.

-- Sweat Chloride: Elevated sweat chloride levels are a diagnostic hallmark that occur in all CF patients and result directly from defective CFTR activity in epithelial cells in the sweat duct. The amount of chloride in the sweat is measured using a standard skin test. Patients with CF typically have sweat chloride levels in excess of 60 mmol/L, while normal values are less than 40 mmol/L. In patients receiving the highest dose of VX-770 (150mg twice daily) in the Phase 2a study, s

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