Vertex announces positive results from cystic fibrosis trial

The interim analysis showed that dosing of VX-770, an investigational CF potentiator, as an oral agent for 14 days resulted in improved lung function and in 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).

Results from the interim analysis support the hypothesis that improving chloride ion transport in cystic fibrosis (CF) patients may correlate to improvements in lung function and provide benefit to patients. Results are being shared with regulatory authorities and leading CF investigators in order to identify the most rapid path forward for the compound.

John Alam, executive vice president, medicines development, and chief medical officer of Vertex, said: “These early data provide clinical proof-of-concept that a CFTR modulator such as VX-770 can have a direct effect on CFTR activity in patients with CF and potentially improve lung function.

“While additional data and evaluation are needed to fully understand VX-770’s clinical profile and its disease-modifying potential in CF, we are pleased to see these early results. We look forward to moving into the second part of this Phase II trial for VX-770, which will enroll approximately 16 patients for dosing of VX-770 or placebo for 28 days, beginning in the second quarter of 2008.”