Gattex is NPS Pharmaceuticals’ recombinant glucagon-like peptide-2 (GLP-2) analog in development for the treatment of short bowel syndrome (SBS), a condition that occurs when the ability of the intestines to absorb fluids and nutrients is reduced, whether due to disease or surgical removal. NPS is currently conducting a second phase III trial. In the first trial, results for the low dose - but not the high dose - reached statistical significance for the primary endpoint. Because results for the high dose took precedence in the analysis, FDA viewed the trial as negative and requested a confirmatory trial. In this report, we review the science, the early results, and regulatory precedents as a means to anticipate Gattex’s chances of success in the ongoing phase III trial and in the marketplace.

Published: May 25th, 2010

Number of pages: 23

Authors: Dr. Ellen Brady and Dr. Ilana Fogelman

TABLE OF CONTENTS:
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INTRODUCTION
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1. Short bowel syndrome
− Clinical picture
− Current treatments

GATTEX (TEDUGLUTIDE, ALX-0600)
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1. Phase I and phase II kinetic data
2. Phase II pilot studies
3. Phase III

CLINICAL AND REGULATORY DISCUSSION
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1. Why the inverse dose response?
2. What other statistical issues are at play?
3. Is this endpoint clinically meaningful?
4. Are there general safety issues of concern?
5. More importantly, is there the potential to stimulate tumor growth?
6. How does the growth hormone experience inform the Gattex program?
7. What about use in other indications?
8. Are there other regulatory considerations?
9. What does all of this ultimately mean relative to Gattex approval?

MARKET ISSUES
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1. Overview
2. Competition

SUMMARY OPINION
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1. Likelihood of success in phase III and
commercial prospects.