Vertex Pharmaceutical (VRTX) shares soared 15% Wednesday as Wall Street cheered the results of a late-stage study of its new cystic fibrosis drug, an experimental treatment that targets the underlying cause of the disease rather than just its symptoms.
In patients with cystic fibrosis, a defective gene and the protein it creates cause the body to produce unusually thick, sticky mucus that builds up in the lungs and the pancreas, leading to life-threatening lung infections and an impairment of the body's ability to properly break down and absorb food.
The drug, VX-770, is a pill that targets the defective protein. It showed sustained improvements in lung function of over 10% in patients with a specific gene mutation. The caveat, though, is that the specific mutation being targeted by the drug, G551D, affects just 4% of the CF population. But it's possible that the results of this trial will provide a basis for the use of VX-770 to treat other mutations. VX-770 is currently being tested in combination with another drug, VX-809, in people with two copies of the more common Delta F508 mutation.
In the trial, patients also showed improvement across all key secondary goals of the study, Vertex said, including decreased pulmonary and respiratory symptoms and weight gain (seven pounds on average). In addition, average sweat chloride (salt) levels, a key clinical indicator of CF, dropped toward normal levels, indicating that the drug is having an effect on the underlying defect.
VX-770 helps "open the gate" at the cell surface -- enabling the protein to pass through, as it would in healthy cells. This restores a proper flow of salt and fluids on the surface of the lung, the company explained.
"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, executive VP of global research and development, and chief scientific officer for Vertex.
In patients with cystic fibrosis, a defective gene and the protein it creates cause the body to produce unusually thick, sticky mucus that builds up in the lungs and the pancreas, leading to life-threatening lung infections and an impairment of the body's ability to properly break down and absorb food.
The drug, VX-770, is a pill that targets the defective protein. It showed sustained improvements in lung function of over 10% in patients with a specific gene mutation. The caveat, though, is that the specific mutation being targeted by the drug, G551D, affects just 4% of the CF population. But it's possible that the results of this trial will provide a basis for the use of VX-770 to treat other mutations. VX-770 is currently being tested in combination with another drug, VX-809, in people with two copies of the more common Delta F508 mutation.
In the trial, patients also showed improvement across all key secondary goals of the study, Vertex said, including decreased pulmonary and respiratory symptoms and weight gain (seven pounds on average). In addition, average sweat chloride (salt) levels, a key clinical indicator of CF, dropped toward normal levels, indicating that the drug is having an effect on the underlying defect.
VX-770 helps "open the gate" at the cell surface -- enabling the protein to pass through, as it would in healthy cells. This restores a proper flow of salt and fluids on the surface of the lung, the company explained.
"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, executive VP of global research and development, and chief scientific officer for Vertex.
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