Vertex admits blunder over drug data
pharmafile | May 31, 2012 | News story | Sales and Marketing | Kalydeco, Kalydeco., Vertex, cystic fibrosis
Vertex has been embarrassed after it released claims for its pipeline drug VX-809 which turned out to be incorrect.
The US company has been riding high on the success of its new cystic fibrosis treatment Kalydeco, launched in the US earlier this year. Kalydeco is targeted to treat a subset of patients with a rare gene variant, who make up just 4% of all cystic fibrosis patients.
However, Kalydeco is in now in a Phase II trial combining it with VX-809, which treats patients with two copies of the most common CF mutation.
Vertex released very promising interim data on the drug combination on 7 May, which claimed that 46% of patients on the trial experienced an improvement in lung function of 5%, while 30% saw improvements of 10% in lung function.
Unfortunately, the figures were too good to be true – nearly two weeks later, Vertex was forced to admit in had got its numbers wrong, and released corrected statistics.
Instead of producing a 5% improvement in 46% of patients, the true figure was 35%, while the second figure was not 30% but 19 per cent.
The company says a ‘misinterpretation’ of the data it received from an outside data analysis firm was to blame for the error.
The correction went down badly with investors and the firm’s share price dived when the true figures were released.
Despite its blushes, industry analysts say the figures are still impressive.
In a research note Evaluate Pharma said: “The fact remains that the company has made a big step forward in this incredibly challenging illness.
“Emerging signs that its agents might modify the course of the progressive lung disease, where in the past only symptom management could be offered, have rightfully raised hopes that real progress is finally being made.”
The firm said that despite the data not being as good as it first thought, it was still on track to reach Phase III.
Chris Wright, Vertex’s senior VP of global medicines development and medical affairs, said: “The improvements in lung function seen to date in this study exceeded our expectations.
“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.”
Kalydeco received FDA approval in January to treat a small sub-set of CF patients – those who have the G551D mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene.
The drug is groundbreaking, as no other drugs are available to treat the cause of the disease, rather than just the symptoms.
Ben Adams
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