May 22 2012
NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a specialty pharmaceutical
company developing innovative therapeutics for rare gastrointestinal and
endocrine disorders, today reported the presentation of data at the
Digestive Disease Week® (DDW®) meeting held May 19-22 in San Diego. In a
poster session, investigators presented data that continue to support
Gattex® (teduglutide) as a potential first-in-class treatment for short
bowel syndrome (SBS), a rare and debilitating condition in which the
body is unable to absorb enough nutrients and/or fluids through the
gastrointestinal tract. Patients with SBS depend on chronic parenteral
nutrition (PN) and/or intravenous (IV) fluids to survive.
Investigators reported the baseline characteristics of seven patients
with short bowel syndrome who successfully achieved complete
independence from PN and IV fluids while on Gattex therapy. In addition,
new findings demonstrated that the pharmacological properties of Gattex
are associated with significant increases in citrulline, an endogenous
non-protein amino acid, that is an indirect measure of enterocyte
intestinal mass. In a third presentation, results from a randomized
study showed that Gattex does not affect gastric emptying in healthy
"Gattex may represent an entirely new approach for treating adult short
bowel syndrome and these presentations from multiple clinical studies
reinforce our belief in the drug's therapeutic potential," said Roger
Garceau, MD, senior vice president and chief medical officer of NPS
Pharmaceuticals, Inc. "These data show continued reductions, and for
some complete independence from parenteral nutrition (PN) with long-term
Gattex therapy, which is encouraging given the serious complications
associated with PN."
The following summarizes the three posters presented at DDW:
Abstract Sa1962: "Short Bowel Syndrome Patients with
Intestinal Failure (SBS-IF) Successfully Achieved Complete Independence
from Parenteral Nutrition" by Jeppesen et al.
In Phase 3 studies, Gattex was shown to significantly reduce PN/IV fluid
dependence in patients with SBS with some patients achieving complete
independence. In this research, Dr. Jeppesen and colleagues described
the characteristics of seven patients who achieved complete independence
with Gattex therapy during two Phase 3 placebo-controlled studies and
the open-label extension phase of those trials.
The data showed that of 173 patients who received Gattex:
Three patients achieved independence during the randomized,
double-blind phase of Study CL0600-004, of which two patients received
0.05 mg/kg/day Gattex and one patient received 0.10 mg/kg/day Gattex.
One patient achieved independence during the extension phase of Study
Three patients achieved independence during the extension phase of
STEPS, the company's 24-week, placebo-controlled Phase 3 registration
Patients were weaned from PN/IV fluid as early as 12 weeks and as late
as 52 weeks after initiation of Gattex, suggesting that long-term
Gattex use is associated with continued reduction of PN/IV fluid
Baseline demographics and disease characteristics were highly variable
and the adverse event profile was similar to the overall study
population with the most frequently reported adverse events being
Abstract Sa1961: "Teduglutide, a Human Recombinant Analog of
Glucagon-Like Peptide-2 (GLP-2), Increases Plasma Citrulline Levels in
Patients with Short Bowel Syndrome" by Messing et al.
In this research, Dr. Messing and colleagues assessed changes in plasma
citrulline at Week 24 in two double-blind, randomized,
placebo-controlled Phase 3 studies of Gattex in short bowel syndrome and
showed that in both studies, the mean increase in plasma citrulline at
Week 24 vs. baseline was significantly greater in patients receiving
Gattex compared with those receiving placebo. Plasma citrulline, an
amino acid produced by enterocytes has been considered an indirect
measure of remnant enterocyte mass. Gattex has been show to promote
expansion of normal intestinal epithelium and increase enterocyte mass
by increasing villus height and crypt depth in the small bowel mucosa,
leading to increased absorptive area. Mean changes in plasma citrulline
levels from baseline occurred early in the course of treatment, with
increases as early as Week 4. Patients receiving placebo had minimal
changes from baseline in plasma citrulline levels.
Abstract Sa1959: "A Randomized, Double-Blind,
Placebo-Controlled, Multiple-Dose, Parallel-Group Study to Assess the
Effects of Teduglutide on Gastric Emptying in Healthy Subjects" by
Berg et al.
Dr. Berg and colleagues reported results from a double-blind,
single-center study that assessed the effect of Gattex on gastric
emptying in healthy subjects, and showed that Gattex did not affect
gastric emptying in healthy subjects as measured by acetaminophen
pharmacokinetics. Gattex did not exert any clinical meaningful effects
on serum insulin, glucagon or glucose and there were no clinically
significant differences with Gattex between fasted and fed states.
NPS Pharmaceuticals, Inc.