NEW YORK – NPS Pharmaceuticals Inc.'s (NPSP) drug Gattex was successful in a late-stage study in treating patients with short bowel syndrome, reducing the amount of intravenous nutrition needed by patients with the condition.
The Bedminster, N.J., based drug maker plans to file for approval with the Food and Drug Administration in the second half of the year based on the results. NPS is partnered with privately held Swiss drug maker Nycomed to sell the drug outside of North America for gastrointestinal disorders. The companies managed and funded the study together.
Patients with short bowel syndrome have had much of their small intestine removed, usually because of infection or disease such as cancer. The removal makes it hard for the body to absorb the needed amount of nourishment, requiring the use of parenteral nutrition, which is a way of providing sustenance through intravenous means.
Gattex is an analog of a naturally occurring hormone in the intestine. It works by stimulating the repair and regeneration of cells that line the small intestine, allowing for increased nutrient absorption.
The 24-week trial included 86 patients that depended on parenteral nutrition.
In the study, 63 percent of GATTEX-treated patients showed a response to the drug, compared to 30 percent of those on placebo. Response was defined as achieving a 20 percent or greater reduction in weekly volume of parenteral nutrition at week 20 and week 24.
Four patients left the study due to side effects, with only one being in the GATTEX-treated arm.
NPS estimates that there are an estimated 10,000 to 15,000 patients with short bowel syndrome in North America who are dependent on parenteral nutrition, which it says can cost more than $100,000 a year per patient.
Canaccord Genuity analyst George Farmer recently projected that the company would price Gattex at $65,000 per patient per year. He expects the drug will come to the market in 2012 and bring in more than $250 million in revenue in 2016.
NPS received a $35 million up-front payment in 2007 from its partnership with Nycomed and has the potential to earn more than $180 million in milestone payments, according to regulatory filings. It will also receive a royalty on any eventual sales of Gattex by Nycomed.