Vertex' CF drug boosts FEV1 in G551D patients

The USA's Vertex Pharmaceuticals has reported positive results from a preliminary analysis of data from the second stage of the Phase IIa trial of its oral drug candidate VX-770 in cystic fibrosis patients with the G551D CFTR mutation. Vertex' investigational CFTR potentiator was well-tolerated at 150mg or 250mg twice daily for 28 days.

In this analysis, no patients discontinued treatment and no serious adverse events were reported. At both doses, significant improvements were observed in lung function, as measured by an increase in forced expiratory volume in one second, and significant improvements in the function of the Cystic Fibrosis Transmembrane Conductance Regulator protein, as measured by changes from baseline in sweat chloride levels and changes in nasal potential difference.

In the placebo group, a smaller, statistically non-significant rise in FEV1 was observed, and no significant changes from baseline in sweat chloride levels or NPD were seen. Based on these results, Vertex intends to work with regulatory authorities to finalize the design of a registration program for VX-770 scheduled to begin next year.