Cystic Fibrosis Drug: Vertex Stock Drops Amid Unclear Study Results

Vertex’s cystic fibrosis drug study has been met with skepticism from stockholders, as the company’s shares fell by 13 percent following an announcement about final data from a midstage clinical study regarding a combination of its cystic fibrosis drugs. reports that the Cambridge-based company released finalized data about the study on Thursday, noting that the study “showed statistically significant improvements” in lung function among some adults with the disease.

Early in May, reports that the company issued an “interim analysis” about the Kalydeco therapy, which was used along with the experimental drug VX-809. The Kalydeco drug is the first of its kind to target the underlying cause of the disease, which is rare and often fatal. Vertex stock soared upon stockholders hearing the news.

A few weeks later, however, the company revised their analysis, saying that they had overstated the clinical data. In between announcements, several executives and company directors sold their stock shares, which were worth millions of dollars. The final announcement made on Thursday confirmed the company’s initial analysis, but despite this the company’s stock still plummeted.

Bloomberg Business Week reports that Mark Schoenebaum, an analyst with ISI Group in New York, explained the stock plummet by saying that:

“The company has declined to release the comparable data. This non apples-to-apples disclosure has created investor doubt that the data are ‘real.'”

Thursday’s Vertex press release included a note by Chris Wright, M.D., the company’s senior vice president of global medicines development and medical affairs, who said, according to that:

“We are focused on developing additional medicines to treat the underlying cause of cystic fibrosis, and these data represent exciting progress toward that goal. The data announced today show that the addition of Kalydeco to VX-809 resulted in improvements in lung function and support our plans to start a pivotal program in people with cystic fibrosis who have two copies of the most common CFTR mutation in early 2013.”

Do you think that Vertex’s cystic fibrosis study data is to be trusted, especially since the company has edited their findings about it twice?