Vertex’s cystic fibrosis drug shows huge promise
pharmafile | February 28, 2011 | News story | Research and Development, Sales and Marketing | VX-770, Vertex, cystic fibrosis
US pharma company Vertex has announced extremely promising results for a drug that could be a major advance in treating cystic fibrosis.
VX-770 is an oral medicine that targets the defective protein that causes cystic fibrosis, and was studied in a phase III trial in cystic fibrosis (CF) sufferers with the G551D mutation.
Data from the STRIVE study showed very significant improvements in lung function. Patients who received the drug were 55% less likely to have a pulmonary exacerbation than those on placebo.
The trial also saw patients gain an average of 3.1 kilograms (nearly 7lbs) over the 48 weeks of the trial, addressing the common problem of low weight in CF patients.
Another marker of CF patients is elevated levels of chloride ions in their sweat, because of defective chloride transporter proteins that cause the disease. Patients taking VX-770 showed a marked decrease (from 100 millimoles per litre to less than 60 mmol/L) in their concentration, suggesting the drug targets the disease mechanism effectively.
Only around 4% of CF patients have at least one copy of the G551D mutation, which means most CF patients won’t benefit from the drug, however its insights are likely to eventually extend to other patients.
“Treating the underlying cause of cystic fibrosis with VX-770 led to clinical improvements that were far beyond our expectations, providing support for an entirely new approach to the treatment of this disease,” said Peter Mueller, head of R&D and chief scientific officer for Vertex. “All primary and key secondary outcome measures in this study supported VX-770 over placebo.
“Patients’ lung function improved, they gained weight, experienced fewer respiratory symptoms and felt substantially better. Due to the significance of these data and the great need for new, more effective medicines, we will work with regulatory agencies to determine the fastest way to get VX-770 approved for people with this specific type of CF.”
If the drug proves to be a breakthrough and gains approval, analysts forecast peak sales of around $500 million.
The drug has been developed in collaboration with US charity the Cystic Fibrosis Foundation. Robert Beall, president and chief executive of the research charity, said: “We have much more to do to eliminate this disease, but these data are extremely exciting, especially for people with the G551D mutation and their families. They also offer significant hope that a similar approach to treatment may help others living with CF.”
The trial showed a relative mean improvement in lung function of approximately 17% from baseline compared to placebo achieved by people treated with VX-770. A mean absolute improvement from baseline of approximately 10.5% compared to placebo.
There are an estimated 70,000 people with CF worldwide. The median predicted age of survival for a person with the disease is currently around 37 years.
Most CF sufferers have at least one copy of the F508del mutation, which is thought to be the main mutation responsible for the disease.
Vertex has also published results from another trial, DISCOVER, which tested VX-770 in patients with two copies of the F508del mutation, but its action proved to be not statistically significant compared to placebo.
Results from a trial combining the drug with another Vertex candidates, VX-809, are expected later this year and Vertex believes these will yield more promising results in these patients.
Andrew McConaghie
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