Excited biotech analysts this morning heralded outstanding results for Vertex's Phase III study of the cystic fibrosis drug VX-770 as a breakthrough of historic proportions. Investigators say that the drug corrected a genetic defect that triggers CF, improving mean lung function in patients by 10.6 percent. That's better than twice the level needed to demonstrate a statistically significant outcome.
"The result is a big win for developer Vertex Pharmaceuticals and its partner, the Cystic Fibrosis Foundation, a patient group," commented Forbes' Matthew Herper. And he went on to note that Mark Schoenebaum, the biotech analyst at ISI Group, thought that any improvement over 10 percent would be considered a "home run," propelling the drug to $600 million in annual sales.
Investigators said that the drug triggered a "profound" improvement in lung function--forced expiratory volume in one second, or FEV1--through week 48 compared to placebo. Improvements in all key secondary endpoints were also observed through week 48 among those who received VX-770.
"The results from the phase III study represent a potential scientific breakthrough because VX-770, if eventually approved, would be the first and only drug to repair the underlying genetic cause of cystic fibrosis rather than merely treating the symptoms of the disease," writes TheStreet's Adam Feuerstein.
This particular genetic mutation-which was identified 22 years ago--is present in only a small group of CF patients. A second late-stage study is expected to wrap up later this year and Vertex says it will file for an approval in the second half of 2011.
For Vertex, the late-stage success marks yet another big R&D advance. Best known for its work on telaprevir, a blockbuster favorite among analysts predicting an approval for hepatitis C this year, Vertex has marched through the clinic with stellar results. Given the tough odds that any developer faces, the biotech has gained a reputation as one of the savviest developers in biotech.