NPS Pharmaceuticals Announces Publication of Pivotal Phase 3 Study of
Gattex® (teduglutide) in Short Bowel Syndrome-Intestinal Failure Patients
-- Study available online in Gastroenterology --
BEDMINSTER, N.J. -- November 29, 2012
NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a biopharmaceutical company
developing innovative therapeutics for rare gastrointestinal and endocrine
disorders, announced today that its Phase 3 study of Gattex (teduglutide), a
novel analog of glucagon-like peptide 2, has been published online in the
peer-reviewed journal, Gastroenterology, the official journal of the American
Gastroenterological Association. The 24-week study, known as STEPS, shows that
Gattex is effective and well-tolerated in reducing parenteral support volume
and number of infusion days in short bowel syndrome-intestinal failure
“This crucial study demonstrates the potential of Gattex to be an important
treatment option for patients with short bowel syndrome,” said Palle B.
Jeppesen, M.D., lead author of the study and Associate Professor, Department
of Medical Gastroenterology, University Hospital of Copenhagen. “Short bowel
patients with intestinal failure rely on parenteral support to meet their
nutritional, fluid and electrolyte needs because their intestines aren’t able
to absorb enough nutrients, fluids and electrolytes. Being able to reduce or
even eliminate dependence on parenteral support should provide important
clinical benefits for patients with this debilitating condition.”
Methodology and Results
Researchers performed a 24-week study of patients with SBS-IF who were given
either daily subcutaneous dosing of 0.05 mg/kg teduglutide (n=43) or placebo
(n=43). Parenteral support (PS) was reduced if 48-hour urine volumes exceeded
baseline values by 10 percent. The primary efficacy endpoint was defined as
the number of patients who achieved a 20 to 100 percent reduction in weekly PS
volume, from baseline, at Weeks 20 and 24.
There were significantly more responders in the teduglutide group (27 of 43)
than the placebo group (13 of 43, p=.002). At Week 24, patients who received
teduglutide experienced an average 4.4 liter reduction in weekly PS volume
from a baseline of 12.9 liters. Patients who received placebo experienced an
average 2.3 liter reduction from a baseline of 13.2 liters (p<.001).
After completing 24 weeks of treatment, 54 percent (21 of 39) of
teduglutide-treated patients were able to reduce the number of infusion days
per week by one or more days, compared to 23 percent (9 of 39) of those
treated with placebo (p=0.005).
The distribution of treatment-emergent adverse events that led to study
discontinuation was similar between patients given teduglutide (n=2) or
placebo (n=3). The most commonly reported adverse events were
gastrointestinal-related and appear to be consistent with the pharmacological
effects of the drug.
The research was funded by NPS and Nycomed, a Takeda company. The study,
titled “Teduglutide Reduces Need for Parenteral Support Among Patients with
Short Bowel Syndrome with Intestinal Failure,” is now published online at
About Short Bowel Syndrome
Short bowel syndrome (SBS) is a highly disabling condition that can impair a
patient's quality of life and lead to serious life-threatening complications.
SBS typically arises after extensive resection of the bowel due to Crohn's
disease, ischemia or other conditions. SBS patients often suffer from
malnutrition, severe diarrhea, dehydration, fatigue, osteopenia, and weight
loss due to the reduced intestinal capacity to absorb nutrients, water, and
electrolytes. The usual treatment for SBS is nutritional support, including
parenteral nutrition (PN) and/or intravenous (IV) fluids to supplement and
stabilize nutritional needs.
Although PN can provide nutritional support for SBS patients, it does not
improve the body's own ability to absorb nutrients. PN is associated with
serious complications, such as infections, blood clots or liver damage, and
the risks increase the longer patients are on PN. Patients on PN often
experience poor quality of life with difficulty sleeping, and frequent
urination, and patients receiving chronic PN often experience a loss of
About Gattex® (teduglutide)
Gattex (teduglutide) is a novel, recombinant analog of human glucagon-like
peptide 2, a protein involved in the rehabilitation of the intestinal lining.
It has been developed to reduce dependence on parenteral nutrition (PN) in
adult patients with short bowel syndrome (SBS). Significant reductions in mean
PN/IV infusion volume from baseline to end of treatment were seen in the Phase
3 studies of teduglutide. In addition, some patients were able to be weaned
off PN during these trials. The most common treatment-emergent adverse events
with Gattex in the placebo-controlled studies that occurred at a higher
frequency with Gattex were abdominal pain, upper respiratory tract infections,
nausea, injection site reactions, abdominal distension, headaches, and
gastrointestinal stoma complications.
Gattex has received orphan drug designation for the treatment of SBS from the
European Medicines Agency (EMA) and the FDA.
In 2007, NPS granted Nycomed, a Takeda company, the rights to develop and
commercialize teduglutide outside the United States, Canada, Mexico and
Israel. NPS retains all rights to teduglutide in North America. The European
Commission granted European market authorization on August 30, 2012 for the
medicinal product teduglutide (trade name in Europe: Revestive®) as a
once-daily treatment for adult patients with short bowel syndrome.
About NPS Pharmaceuticals
NPS Pharmaceuticals is a biopharmaceutical company focused on bringing orphan
products to patients with rare disorders and few, if any, therapeutic options.
NPS is advancing two late-stage registration programs. A New Drug Application
is undergoing FDA review for Gattex® (teduglutide) as a treatment for adult
short bowel syndrome (SBS). NPS is also developing Natpara® (rhPTH[1-84]) for
the treatment of adult hypoparathyroidism and expects to submit its Biologic
License Application (BLA) to the FDA in mid-2013. NPS' earlier stage pipeline
includes two calcilytic compounds, NPSP790 and NPSP795, with potential
application in rare disorders involving increased calcium receptor activity,
such as autosomal dominant hypocalcemia with hypercalciuria (ADHH). NPS
complements its proprietary programs with a royalty-based portfolio of
products and product candidates that includes agreements with Amgen,
GlaxoSmithKline, Janssen Pharmaceuticals, Kyowa Hakko Kirin, and Nycomed
(acquired by Takeda Pharmaceutical Company Limited).
"NPS," "NPS Pharmaceuticals," "Gattex," and "Natpara" are the company's
trademarks. All other trademarks, trade names or service marks appearing in
this press release are the property of their respective owners.
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 the company's business include, but are not limited to, the
risks associated with any failure by the company to successfully complete its
preclinical and clinical studies within the projected time frames or not at
all, the risk of not gaining marketing approvals for Gattex and Natpara, the
risks associated with the company's strategy, as well as other risk factors
described in the company'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.
NPS Pharmaceuticals, Inc.
Susan M. Mesco, 908-450-5516
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