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Vertex's cystic fibrosis combination less effective than previously thought

Shares in US pharma company drop after it issues revised study data for VX-809 and Kalydeco combination

Vertex Pharmaceuticals has been forced to issue corrected phase II data for its combination of VX-809 and Kalydeco that now suggests the cystic fibrosis drug is not as effective as previously thought.

Shares in the US pharma firm fell 14.6 per cent following the announcement, having previously jumped over 50 per cent when the original data was published on May 7, 2012.

Vertex had initially said that 46 per cent of cystic fibrosis patients with two copies of the F508del mutation taking the drug combination experienced a 5 per cent or more lung function improvement, and that 30 per cent experienced at least a 10 per cent improvement following 56 days of treatment.

However, the actual results turned out to be less impressive, with only 35 per cent of patients achieving an improvement of 5 per cent or more and 19 per cent experience a minimum improvement of 10 per cent.

The error was down to a misinterpretation of data provided by a third-party vendor, Vertex's CEO Jeffrey Leiden said in a conference call.

"This mistake is very disappointing, unacceptable to us,” he added.

In addition, Leiden commented on further data released by Vertex involving the VX-809 and Kalydeco, which he said was more positive.

For patients with two copies of the F508del mutation, a mean absolute improvement in lung function of 8.5 percentage points was observed in patients treated with the combination compared to placebo from baseline after 56 days of treatment.

"The improvements in lung function seen to date in this study exceeded our expectations,” said Chris Wright, senior VP, global medicines development and medical affairs at Vertex.

“We're continuing to move forward as quickly as possible toward a pivotal study of VX-809 and KALYDECO in people with two copies of the F508del mutation."

Evaluation of patients with one copy of the F508del mutation, which is one of the most common mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, is ongoing.

Earlier this week, Kalydeco was recommended by the European Medicines Agency (EMA) for approval as the first drug to be used to treat the cause of cystic fibrosis rather than its effects, which include a build-up of sticky mucus in the body.

30th May 2012


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