Cystic fibrosis treatment could prolong lives

DURHAM -- After years of research and regulatory setbacks, Inspire Pharmaceuticals finally has what looks like a winner: a cystic fibrosis drug that could change the course of the disease.

Denufosol tetrasodium does not cure cystic fibrosis, a genetic disease that kills half the people born with it before they turn 30. But results from a late-stage study called Tiger-1 showed that denufosol attacks the underlying defect of the disease, promising to prolong cystic fibrosis patients' lives.

Inspire announced the good news Friday morning. In the afternoon, employees partied with Tiger balloons and Tiger beer at the Durham corporate offices.

"It's an exciting day for us," company spokeswoman Jenny Kobin said.

Investors rejoiced, too. Inspire stock closed at $5.19, up 35 percent, or $1.34 per share.

Inspire was founded in 1995 with technology to develop a cystic fibrosis treatment.

The technology also looked promising to treat retinal disease, dry eye and bleeding complications during heart surgery, but the drugs produced a string of disappointing test results.

In 2006, work on two of the drugs stopped.

Prolacria, the dry eye drug, also has a spotty record and has been in regulatory limbo for more than four years.

To buy time, Inspire revamped its drug development pipeline and added a nasal spray for seasonal allergies, an allergy pill and a pinkeye treatment discovered by other companies.

Both allergy medicines tanked. And sales of the pinkeye treatment, which has been on the market for about a year, have been lower than expected.

The setbacks prolonged Inspire's financial losses and drubbed the stock. Shares that sold for more than $21 in October 2003 were down to $3 last month.

Wall Street had little to no expectations for denufosol either, analyst Ian Sanderson, a Cowen and Co. wrote in a research note.

Sanderson was still skeptical Friday, calling denufosol's Tiger-1 test results OK but not great.

Patients got results

But Dr. Michael Knowles, a director of the cystic fibrosis center at UNC-Chapel Hill, said the drug "could be a paradigm shift in the way cystic fibrosis is treated."

Knowles, who participated in a conference call with analysts Friday, had patients who took part in the Tiger-1 study.

Patients who inhaled denufosol breathed easier after 24 weeks and continued to improve as they stayed on the drug another 24 weeks, according to the study results. In comparison, patients who did not receive the drug showed improvement only once they switched to denufosol.

Cystic fibrosis causes a number of problems, including thick mucus that blocks airways and leads to inflammation and infection.

"The progressive improvement was striking," Knowles said.

Inspire is still testing denufosol in a safety study and a second Phase III clinical trial called Tiger-2. The drug is at least two years from becoming available. But to investors it finally looks as if the company's on the right track.