Altus cystic fibrosis trial meets goal but results not as hoped

By Jennifer Robin Raj

BANGALORE, Aug 11 (Reuters) - Altus Pharmaceuticals Inc ALTU.O said its late-stage trial of a treatment for cystic fibrosis met its main goal, but shares fell 21 percent as the results failed to meet expectations and displayed some geographical disparity.

"We believe there is disappointment on the part of some investors who were expecting the data to show an even greater degree of efficacy," said Piper Jaffray analyst Andrew Fein, who believes the results are positive and sufficient to file for marketing approval.

The biopharmaceutical company said its overall top-line results were affected by a "marked difference" in patients within the United States and those outside of it.

The company was testing Trizytek, its enzyme replacement therapy, and the trial evaluated the efficacy and safety of pancreatic enzyme replacement therapy in cystic fibrosis patients.

Cystic fibrosis (CF) is a genetic disease that affects the internal organs, especially the lungs and digestive system, by clogging them with thick sticky mucus, making it hard to breathe and digest food.

The trial met its main goal of improvement in fat absorption and was statistically significant compared to placebo.

However, patients from three out of six non-U.S. countries did not appear to show a difference between Trizytek and placebo groups, the company said, adding that it was investigating possible factors that may have affected the results outside the United States.

The strength of the CF foundation in the U.S. has kept a considerable amount of control over the quality of the CF centers in the United States, Fein told Reuters.

"Alternatively, no such comparable organization exists outside the U.S. and we believe the data may have suffered a little from the lack of such an infrastructure," Fein said.

Shares of Altus Pharmaceuticals were trading down $1.05 at $3.89 after the bell. They closed at $4.94 Monday on Nasdaq. (Editing by Amitha Rajan)