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Vertex Reports Positive Results From Part Two Phase IIa Cystic Fibrosis Trial

October 21, 2008

Vertex Pharmaceuticals, a global biotechnology company, has announced positive results from a preliminary analysis of data from part two of the Phase IIa clinical trial of the investigational oral drug VX-770 in cystic fibrosis patients who carry the G551D CFTR mutation.

VX-770, an investigational cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, was well-tolerated when dosed orally as 150mg or 250mg twice daily for 28 days. In this analysis, no patients discontinued treatment and no serious adverse events were reported.

At both the 150mg and 250mg doses, significant improvements in lung function, as measured by an increase in FEV1, and significant improvements in the function of the CFTR protein, as measured by changes from baseline in sweat chloride levels and changes in nasal potential difference (NPD), were observed.

In patients receiving placebo, a smaller increase in FEV1 was observed at 28 days that was not statistically significant, and no significant changes from baseline in sweat chloride levels or NPD were observed.

Based on these results, Vertex intends to work with global regulatory authorities to finalize the design of a registration program for VX-770 targeted to begin in 2009. VX-770 was developed with support from Cystic Fibrosis Foundation Therapeutics, the nonprofit affiliate of the Cystic Fibrosis Foundation.

Robert Beall, president and CEO of the Cystic Fibrosis Foundation, said: “Data from the Phase II trial of VX-770 provides evidence that a small molecule can address the basic defect in cystic fibrosis and suggests that modulation of CFTR may play an important role in cystic fibrosis therapy.”




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