Top-Line Data Confirm Pivotal Study Findings
BEDMINSTER, N.J., March 17 /CNW/ -- NPS Pharmaceuticals, Inc. (Nasdaq:
NPSP) today reported positive top-line results from a 28-week blinded Phase
3-extension study of GATTEX(TM) (teduglutide) for short bowel syndrome (SBS)
patients who are dependent upon parenteral nutrition (PN). The extension
study enrolled 65 of the 71 patients (91%) 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.
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
(24-week Phase 3 study and 28-week extension study). Overall, both doses of
GATTEX were safe and generally well-tolerated, with no statistical differences
in the incidence rate of adverse events or serious adverse events among the
treatment groups when compared to placebo-treated patients in the Phase 3
study, with the exception of injection site reactions, which were higher in
the high-dose GATTEX dose. A number of efficacy measures were also included
as a secondary endpoint of the extension study. The company expects full
results from the Phase 3-extension study to be presented at a future medical
Key efficacy findings included:
-- Sixty-eight percent (68%) of the 25 patients who had received low-dose
GATTEX therapy and continued on low-dose GATTEX, and fifty-two percent
(52%) of the 27 patients who had received high-dose GATTEX therapy and
continued on high-dose GATTEX achieved a 20% or greater reduction in PN
after a total of 52 weeks of therapy.
-- Patients treated with low-dose GATTEX demonstrated a mean 51% reduction
in PN volume from pretreatment baseline to the end of 52 weeks of
therapy (p< 0.001) and those treated with high-dose GATTEX experienced
a mean 24% reduction (p< 0.001).
-- Six out of six (100%) patients who had previously received placebo in
the Phase 3 study and were randomized to low-dose GATTEX therapy, and
two out of seven (29%) patients who had previously received placebo in
the Phase 3 study and were randomized to high-dose GATTEX therapy,
achieved a 20% or greater reduction in PN after a total of 28 weeks of
therapy in the extension study.
-- The two low-dose patients and one high-dose patient who were able to
discontinue PN in the Phase 3 study remained off PN after 52 weeks of
Bernard Messing, M.D., Head of the Division of Gastroenterology and
Nutrition Support at Hopital Lariboisiere-St. Lazare, Paris, stated: "These
data support and expand upon previous clinical findings that showed low-dose
GATTEX to be a safe and effective potential therapy for short bowel syndrome
patients dependent on Parenteral Nutrition. This study provides interesting
and very promising results, as improvement of this debilitating chronic
disease associated with severe intestinal failure is so difficult to achieve
with current treatment options."
Francois Nader, M.D., chief executive officer-elect, stated: "We are
very pleased with these results, which reinforce our belief that GATTEX may be
a potential new standard of care for SBS. The consistency of these data with
the previously reported Phase 3 results is encouraging and we look forward to
presenting the full data sets for both studies at upcoming medical meetings.
We continue to communicate with FDA and work with our partner Nycomed to
complete the design of the confirmatory Phase 3 study, which we expect to
initiate in the third quarter of this year."
Phase 3-Extension Study Design
The extension study enrolled 65 of the 71 patients (91%) patients who had
previously completed a Phase 3 clinical trial that evaluated low-dose GATTEX
(0.05 mg/kg/day), high-dose GATTEX (0.10 mg/kg/day) and placebo for
PN-dependent SBS patients. 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.
Previously Reported Phase 3 Results
In October 2007, NPS reported positive top-line results from the
company's Phase 3 study of GATTEX in which 83 patients with short bowel
syndrome (SBS) received either a low dose of GATTEX (0.05
milligrams/kilogram/day), a higher dose (0.10 mg/kg/day) or placebo. The
clinical efficacy endpoint of the study was a reduction in PN of at least 20%
comparing baseline to weeks 20 to 24, measured as a graded response to capture
reductions up to 100%. 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 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 study's
criteria for conducting the statistical analysis of the primary endpoint
required that the results for the high-dose group show statistical
significance before the results of the low-dose group could be considered.
Given the drug's orphan designation in SBS and the statistically strong
(p=0.007) and clinically meaningful findings in the low-dose group, the
company met with the FDA to discuss the regulatory pathway for GATTEX.
About Short Bowel Syndrome (SBS)
SBS is a highly disabling condition that impairs quality of life and can
lead to serious life-threatening complications. SBS typically arises after
extensive resection of the small bowel. There are an estimated 10,000 to
15,000 SBS patients in North America who are PN-dependent, the cost of which
can exceed $100,000 annually per patient. SBS patients suffer from
malnutrition, severe diarrhea, dehydration, fatigue, osteopenia, and weight
loss due to a loss in the ability to absorb adequate amounts of nutrients and
water. The goals of current treatment are to maintain fluid electrolyte, and
nutrient balances through dietary management, including the use of PN.
Long-term complications of the condition may include an increased risk of
systemic infections due to the presence of an intravenous feeding line,
degenerative changes in the bones and nerves due to vitamin and mineral
deficiencies, and liver failure. Potential benefits derived from reduced
dependence on intravenous feeding may include improved nutrition, lower rates
of infections, and improved quality of life due to more time away from
intravenous feeding, which may provide greater mobility and improved sleep.
More information about SBS is available on the website,
http://www.shortbowel.com and http://www.glucagon.com.
A potential first-in-class drug, GATTEX (teduglutide) is a proprietary
analog of naturally occurring human glucagon-like peptide 2 (GLP-2), a peptide
secreted primarily in the distal intestine and involved in the regeneration
and repair of the intestinal epithelium. NPS is also pursuing development of
GATTEX as a possible treatment for chemotherapy-induced gastrointestinal
mucositis in cancer patients and necrotizing enterocolitis in preterm infants.
About NPS Pharmaceuticals
NPS Pharmaceuticals is developing specialty therapeutics for
gastrointestinal and endocrine disorders. The company is currently advancing
two late-stage programs: GATTEX(TM) (teduglutide) is in Phase 3 clinical
development for intestinal failure associated with short bowel syndrome and
NPSP558 (parathyroid hormone 1-84 [rDNA origin] injection) is in Phase 2
clinical development as a hormone therapy for hypoparathyroidism. NPS has
also completed a Phase 3 osteoporosis study for PREOS(R) (parathyroid hormone
1-84 [rDNA origin] injection). NPS complements its internal programs with
strategic partnerships with Amgen, GlaxoSmithKline, Kirin, and Nycomed.
Additional information is available at http://www.npsp.com.
Statements made in this press release, which are not historical in
nature, constitute forward-looking statements for purposes of the safe harbor
provided by the Private Securities Litigation Reform Act of 1995. These
statements are based on the company's current expectations and beliefs and are
subject to a number of factors and uncertainties that could cause actual
results to differ materially from those described in the forward-looking
statements. Risks associated to NPS's business include, but are not limited
to, the risk of obtaining the necessary regulatory approvals for GATTEX, as
well as other factors expressed in NPS's periodic filings with the U.S.
Securities and Exchange Commission, including its Annual Report on Form 10-K
and Form 10-Qs. All information in this press release is as of the date of
this release and NPS undertakes no duty to update this information.
For further information:
For further information: Susan Mesco of NPS Pharmaceuticals, Inc.,
+1-908-450-5516 Web Site: http://www.npsp.com