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By Vinay Singh

Vertex Pharmaceuticals (NASDAQ:VRTX) saw its shares soar after it released interim mid-stage data on a combination drug that exceeded the company's expectations. The combination therapy, which consists of Vertex's already approved cystic fibrosis drug, Kalydeco, and its experimental VX-809 drug, may have the potential to become the best line of treatment for most cystic fibrosis patients worldwide.

Shares of Vertex jumped 55 percent on the day of the announcement to finish at $58.12.

Just four months ago, the company successfully gained U.S. Food and Drug Administration for Kalydeco, the first treatment of cystic fibrosis that targeted the underlining cause of the disease.

But Kalydeco only works on about 4 percent, or 1,200, of the 30,000 or so Americans who carry the specific gene mutation that Kalydeco treats. Vertex had been exploring combinations that could expand its treatments to the larger population of cystic fibrosis patients and may have found a hit with its Kalydeco/VX-809 combination.

Cystic fibrosis is a disease that causes the thin layer of mucus that lines the lungs to become thick, clogging airways and leading to infections that can damage the lungs. Often, patients afflicted with cystic fibrosis die prematurely due to worsening infections and complications breathing. The life expectancy for cystic fibrosis patients is 37 years.

Interim data from Vertex's mid-stage trial on the Kalydeco and VX-809 shows great promise. Of those who received the combination therapy, the lung function of approximately 46 percent patients improved 5 percentage points or more while the lung function of about 30 percent of patients improved by 10 percentage points or more. None of the patients given a placebo achieved a 5 percent improvement from baseline to day 56 of the study.

Joseph Pilewski, a cystic fibrosis physician at the University of Pittsburgh Medical Center, cautions that the data is very preliminary and by no means a definitive success, however, he believe it is "very exciting." He told The New York Times that "if this were to bear out in a Phase III pivotal trial it will dramatically impact the treatment options we have for patients with cystic fibrosis."

In a conference call to analysts, Vertex's CEO Jeffrey Leiden said the data exceeded the company's expectations, and that the success of the combo therapy will accelerate VX-809 and Kalydeco into pivotal trials.

Final results of the mid-stage trial will not be available until later this year and a final set of pivotal late-stage trials must be run before Vertex can apply for approval from the FDA.

Source: Vertex's Cystic Fibrosis Drug Impresses Investors