Vertex Cystic Fibrosis Drug Works in Study with Children

NEW YORK (Reuters) – Pharmaceuticals Vertex on Tuesday unveiled positive data for its promising treatment of cystic fibrosis in children by showing that experimental medicine improved lung function in a final-stage study.

Data from the 6-to-11-year study of the oral drug known as VX-770 are released after Vertex reported last month encouraging results for another late-stage trial in older patients who boosted the price of actions of the biotechnology firm.

Stocks rose 3.1 percent on Tuesday to $ 48.79, after the revelation of the results of the study in children.

The data highlight Wall Street’s perception that the firm has promising prospects. Vertex is awaiting approval for a hepatitis C drug that hopes to become the standard treatment for that liver disease.

VX-770 points to a defect in a protein that causes cystic fibrosis, a disease that damages 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 four percent of patients with cystic fibrosis.

At 24 weeks of use, patients who received VX-770 had improved lung function by 12.5 percent compared to their baseline levels, meeting the main objective of the study. This means that the relative improvement compared to those taking placebo was 17.4 percent.

Thomas Russo, an analyst with Robert W. Baird, said the drug’s characteristics make it difficult to demonstrate significant improvements.

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

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