March 29, 2011 / 1:14 PM / 9 years ago

UPDATE 3-Vertex cystic fibrosis drug works in child study

* Follows positive phase 3 data last month

* Says on track to seek approval in second half of year

* Stock rises 3 pct (Adds analyst comment, updates stock movement)

By Lewis Krauskopf

NEW YORK, March 29 (Reuters) - Vertex Pharmaceuticals Inc (VRTX.O) released positive data for its promising cystic fibrosis treatment in children, showing the experimental medicine improved lung function in a late-stage study.

The data in children ages 6 to 11 for the oral drug, known as VX-770, comes after Vertex last month reported encouraging results from another late-stage trial in older patients that sent the biotechnology company’s stock soaring. [ID:nN23128966]

Its shares were up 3.1 percent at $48.79 in early afternoon on Tuesday, following the release of the children’s study.

The data underscores to Wall Street that Vertex is likely to be more than just a one-drug company. It is awaiting approval for an eagerly anticipated hepatitis C drug that stands to become the standard of care for treating the liver disease.

VX-770 targets a defective protein that causes cystic fibrosis, an inherited disease that impairs the lungs and digestive system and affects about 30,000 people in the United States.

The latest study involved 52 children who have the genetic mutation that Vertex says affects only 4 percent of cystic fibrosis sufferers.

At 24 weeks of use, patients on VX-770 had improved their lung function 12.5 percent compared with their baseline levels, meeting the main goal of the study. The mean relative improvement compared with those taking a placebo was 17.4 percent.

Thomas Russo, an analyst with Robert W. Baird, said the drug was not expected to have as pronounced an effect in children because they tend to begin with less severe disease than adults, making it more difficult to show significant improvement.

“Results were, in fact, highly significant and very impressive,” Russo said in a research note.

The company said it was on track to submit regulatory applications for approval in the United States and Europe in the second half of 2011.

Vertex is currently testing VX-770 in combination with another drug it is developing, VX-809, on cystic fibrosis patients with a far more common gene mutation. (Reporting by Lewis Krauskopf, additional reporting by Anand Basu in Bangalore; Editing by Gopakumar Warrier, Maureen Bavdek and Matthew Lewis)

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