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Vertex Pharma’s three-drug combinations improve breathing in some cystic fibrosis patients

Vertex Pharmaceuticals said Tuesday that its three-drug cocktails improved a measure of lung function in patients with cystic fibrosis by 9.6 percentage points or more, surpassing Wall Street's expectations and setting the company up to advance a medicine to treat thousands more patients with the disease.

The Boston-based biotech company is testing multiple combination regimens to find the best cocktail for a majority of patients with cystic fibrosis.

Vertex has brought to market medicines for genetic forms of the disease affecting about 30,000 of the 75,000 people with CF worldwide; the majority of patients, though, still have no approved treatment options that target the disease's underlying cause.

Cystic fibrosis is a rare, genetic disease that causes mucus to build up in the lungs and other organs, leading to infections, damage and respiratory failure. It's driven by mutations in a gene known as CFTR, and because different mutations manifest in slightly different ways, multiple drugs have been needed to effectively treat the disease.

The results released Tuesday are from studies aiming to expand treatment options to 90 percent of people with cystic fibrosis, those with one copy of a mutation known as F508del, and one minimal function mutation.

They were from two phase 2, or midstage, clinical trials, and one phase 1 study evaluating three different...


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