One Year of GATTEX™ Therapy Safely Reduces Parenteral Nutrition Dependence for Patients with Short Bowel Syndrome

Business Wire, Oct 8, 2008

-- Results Presented at American College of Gastroenterology Annual Meeting Show Majority of Responders Continued to Benefit from GATTEX in Phase 3-Extension Study --

ORLANDO, Fla. -- NPS Pharmaceuticals, Inc. (NASDAQ: NPSP) today presented positive one-year data from a Phase 3-extension study of GATTEX[TM] (teduglutide), an investigational product for patients dependent upon parenteral nutrition due to short bowel syndrome (SBS). SBS is primarily caused by intestinal resection and the subsequent inability to maintain oral fluid, electrolyte, and nutrient balances. The results showed GATTEX was well tolerated out to one year and provided the ability to safely reduce parenteral nutrition (PN) dependence. In addition, 75 percent of patients who responded to GATTEX during the first 24 weeks of therapy maintained their response, with many having further reductions in their PN volume. Three patients who gained independence from PN during the first 24 weeks of therapy remained off PN at week 52 and one additional patient was weaned from PN during the 28-week extension phase. These patients had so far remained PN-independent for periods ranging from 0.5 to 3.5 years.

The data were presented during an oral presentation on "Treatment Out to 1 Year with a GLP-2 Analog, Teduglutide, Safely Reduces Parenteral Nutrition (PN) Needs in PN-Dependent Short Bowel Syndrome (SBS) Patients" at the American College of Gastroenterology (ACG) Annual Scientific Meeting by Richard Gilroy, M.D., Assistant Professor of Medicine, Division of Gastroenterology/Hepatology, The University of Kansas Medical Center.

"After one year of therapy with GATTEX, patients with short bowel syndrome were able to dramatically reduce, and in some cases eliminate, their dependence on parenteral nutrition," said Dr. Gilroy. "Beyond the inconvenience of parenteral nutrition dependence, this patient population has many morbidities and even mortality associated with their therapy. GATTEX data to date suggests that patients could reduce their reliance on PN therapy, and likely thereby lessening PN-associated complications. Improvements in quality of life I suspect will follow and this should free them from parenteral nutrition's inconvenience. I believe this will lead to a reduction in days from PN per week. The growing body of data regarding GATTEX continues to support its development as a first-in-class drug."

GATTEX Phase 3-Extension Study Design

The GATTEX 28-week Phase 3 blinded, placebo-controlled-extension study enrolled 65 of the 71 patients (91 percent) who had completed a 24-week randomized Phase 3 study that evaluated low-dose GATTEX (0.05 mg/kg/day) and high-dose GATTEX (0.10 mg/kg/day) versus placebo. Patients who were already receiving GATTEX in the Phase 3 study continued on their dose for an additional 28 weeks for a total of 52 weeks of therapy. Patients who were receiving placebo in the Phase 3 study were randomized to receive one of the two doses for a total of 28 weeks of therapy.

The primary objective of the Phase 3 extension study was to evaluate the long-term safety and tolerability of daily GATTEX dosing for up to 52 weeks. A number of efficacy measures were included as secondary endpoints with response defined as maintaining, improving upon or developing a 20 percent or greater reduction in weekly PN volume at week 28 of the extension phase.

Key findings from the study:

* GATTEX demonstrated an excellent safety and tolerability profile for up to one year, which was the primary endpoint of the extension study. There were no relevant differences in the incidence rate of adverse events or serious adverse events among the treatment groups when compared to placebo, with the exception of injection site reactions, which were higher for the high-dose GATTEX group.

* Twelve of the 16 patients (75 percent) who responded to low-dose GATTEX during the initial 24-week phase maintained their response during the 28-week extension phase, with 10 of the 16 (62 percent) achieving further reductions in PN volumes during the extension phase.

* Six of the eight (75 percent) patients who responded to high-dose GATTEX during the initial 24-week phase maintained their response during the 28-week extension phase, with two of the eight (25 percent) achieving further reductions in PN volumes during the extension phase.

* Two low-dose patients and one high-dose patient gained independence from and discontinued PN by the end of treatment during the initial 24-week phase. All three remained off PN. One additional low-dose patient was weaned from PN during the extension study.

* Patients treated with low-dose GATTEX achieved mean weekly reductions from baseline in PN volume of 4.9L (57 percent) and those treated with high-dose GATTEX experienced mean weekly reductions from baseline of 3.3L (27 percent).

* Six out of six patients who had previously received placebo in the initial 24-week phase and were randomized to low-dose and two out of seven (29 percent) patients who had previously received placebo in the Phase 3 study and were randomized to high-dose GATTEX therapy achieved a 20 percent or greater reduction in PN after 28 weeks of therapy in the extension study.