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Phase 3 GATTEX results presented at DDW highlight potential new treatment in short bowel syndrome
Date:5/19/2008

San Diego, California, May 19, 2008 NPS Pharmaceuticals, Inc. (NASDAQ: NPSP) today reported the presentation of Phase 3 data at the annual Digestive Disease Week (DDW) Congress on GATTEX (teduglutide), a novel investigational compound that may reduce dependence upon parenteral nutrition (PN) in patients with intestinal failure associated with short bowel syndrome (SBS).

The Phase 3 study evaluated two doses of GATTEX versus placebo. Results indicate that GATTEX was generally well-tolerated and effective in reducing the PN requirements of SBS patients, with the lower dose findings showing statistical significance versus placebo. The company is finalizing a protocol for a Phase 3 confirmatory study, which it expects will begin in the third quarter of 2008. Stephen OKeefe, M.D., professor of medicine, University of Pittsburgh, presented the Phase 3 results today at an American Gastroenterological Association (AGA) Institute clinical science plenary session.

Data from this study show that GATTEX has the potential to reduce or eliminate the need for parenteral nutrition, thereby reducing the complications associated with PN therapy and improving quality of life for patients with intestinal failure due to surgical resection (SBS), said Dr. OKeefe. These latest findings provide further support for the development of GATTEX as a potential first-in-class compound to reduce the dependency of SBS patients on IV fluids and nutrition without adverse side effects.

The pivotal Phase 3 study evaluated 83 SBS patients who are dependent on PN. After a PN stabilization and optimization period, patients were randomized to receive low-dose of GATTEX (0.05 mg/kg/day), high-dose GATTEX (0.10 mg/kg/day) or placebo for 24 weeks. A clinically significant PN response was defined as a weekly reduction of PN requirements of more than 20% at dosing week 20 and maintained through dosing week 24.

Key findings were as follows:

  • Forty-six percent (46%) of patients who received low-dose GATTEX (n=35) responded and achieved a highly statistically significant reduction in PN compared to placebo (p=0.007). Twenty-five percent (25%) of patients who received high-dose GATTEX (n=32) responded and showed a trend in the difference between the treatment group and placebo, but this did not reach statistical significance (p=0.161).
  • GATTEX was generally well-tolerated with no statistical differences in the incidence rates of adverse events or serious adverse events among the treatment groups when compared to placebo.
  • Two low-dose patients gained independence from and discontinued PN by week 16, and a third high-dose patient discontinued PN at the end of treatment.
  • The studys statistical analysis plan required that the results for the high-dose group show statistical significance before considering the results for the low-dose group; however, given GATTEXs orphan designation for SBS and the statistically strong and clinically meaningful findings in the low-dose group, NPS is in ongoing discussion with the U.S. Food and Drug Administration (FDA) related to the regulatory pathway for GATTEX. The company is preparing to initiate a Phase 3 confirmatory study in the third quarter of 2008.
  • Sixty-five of the 71 patients who completed the Phase 3 study enrolled in a 28-week Phase 3-extension study. 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 initial Phase 3 clinical study were randomized in the blinded extension study to receive either a low dose of GATTEX (0.05 mg/kg/day) or a high dose of GATTEX (0.10 mg/kg/day) for 28 weeks of therapy. GATTEX demonstrated a favorable safety profile. Highly statistically significant reductions in mean PN volume from pretreatment baseline were observed after 52 weeks of GATTEX therapy (24-week Phase 3 study and 28-week extension study). The company expects full results from the extension study will be presented at a future medical meeting.

AGA Clinical Science Plenary Session

Abstract 212: Teduglutide, a Novel GLP-2 Analog, in the Management of Short Bowel Syndrome (SBS) Patients Dependent On Parenteral Nutrition: a Multicenter, Multinational Placebo-Controlled Clinical Trial by O'Keefe et al.

The clinical benefit of GATTEX presented in the DDW plenary session was demonstrated in a 24-week multicenter Phase 3 study involving 83 patients with SBS. In this study, two different doses of GATTEX (0.05 and 0.1 mg/kg/day) were more effective than placebo in reducing PN requirements. In an intent-to-treat analysis, forty-six percent (46%) of patients receiving the lower dose of GATTEX (n=35) responded and achieved a significant reduction in PN compared to placebo (p=0.007). Twenty-five percent (25%) of patients receiving the higher dose of GATTEX (n=32) responded and showed a trend in the difference between the treatment group and placebo, but this did not reach statistical significance (p=0.161).

Two patients from the low-dose and one from the high-dose group discontinued PN by week 24. Both GATTEX doses were also associated with statistically significant increases in trunk lean body mass (p < 0.05 vs. placebo) and increases in plasma citrulline (p=0.01 vs. placebo), a potential biomarker of intestinal function in SBS.

These findings are consistent with results from earlier human studies suggesting that GATTEX can enhance physiological adaptation to SBS by amplifying intestinal trophism (influence of nutrition) and absorption. These effects were not associated with an increase in serious adverse events.

Additional Data Being Presented at DDW

Two additional presentations related to GATTEX and SBS will be presented at DDW:

Session: AGA Institute Short Bowel Syndrome
Abstract title: 757: Teduglutide, a Glucagon-Like Peptide-2 (GLP-2) Analog, Improves Fluid Balance in Short Bowel Syndrome (SBS) Patients Depending On Parenteral Support (PN) by Palle B Jeppesen, Bernard Messing, Marek Pertkiewicz, Jane Cyran, Lidia L Demchyshyn, Ronald Kershner.
Type: Research Forum
Date/Time: 5/20/2008 2:15 PM - 3:45 PM
Location: Room 30D San Diego Convention Center

Session: AGA Institute: Novel Diagnostics and Therapeutics in Intestinal Disease
Abstract title: W1291: Plasma citrulline acts as a biological marker of intestinal adaptation in a monkey model of short bowel syndrome by Lidia L. Demchyshyn, Kerri L. Trendler, Clynn Wilker.
Type: Poster Session
Date/Time: 5/21/2008 8:00 AM - 5:00 PM
Location: Sails Pavilion San Diego Convention Center


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Contact: Stephen Bingham
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Source:Eurekalert

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