NPS Pharmaceuticals Reports Characteristics of Patients Who Achieved
Independence from Parenteral Support in STEPS 2 Study of Gattex®
(teduglutide) in Adult Short Bowel Syndrome
- Data presented at American College of Gastroenterology Annual Scientific
BEDMINSTER, N.J. -- October 22, 2012
NPS Pharmaceuticals, Inc.(NASDAQ: NPSP), a biopharmaceutical company
developing innovative therapeutics for rare gastrointestinal and endocrine
disorders, today reported data related to the seven patients who achieved
complete independence from parenteral nutrition (PN) and/or intravenous fluids
(IV) while on long-term Gattex® (teduglutide) therapy in STEPS 2, a 24-month,
open-label study in adult short bowel syndrome (SBS).
The data suggest that patients achieving independence from PN/IV tend to have
colon in continuity and lower baseline PN/IV needs than typical patients
studied, as all seven patients had baseline PN/IV requirements of less than 13
liters a week. A poster (P1267) titled “Long-Term Therapy With Teduglutide:
Characteristics of Patients With Intestinal Failure Associated With Short
Bowel Syndrome Who Achieved Complete Independence From Parenteral Nutrition
and/or Intravenous Fluid Support” was presented by lead study investigator Ken
Fujioka, MD and colleagues at the American College of Gastroenterology Annual
Scientific Meeting in Las Vegas, NV.
“Patients with short bowel syndrome face numerous medical risks and lifestyle
challenges,” said Ken Fujioka, MD, Nutrition and Metabolic Research Center,
Scripps Clinic, Del Mar. “They are often tethered to a PN/IV line, have to
make frequent trips to the bathroom, suffer from disrupted sleep, and face
serious complications including infections, blood clots, or liver damage. The
fact that long-term treatment with Gattex has shown to help reduce PN/IV
volume or even completely eliminate the need for it is exciting for patients
and physicians. Gattex has the potential to be a truly life-changing treatment
for adult short bowel syndrome.”
Gattex is a novel, recombinant analog of human glucagon-like peptide 2, a
peptide involved in normal intestinal function and fluid and nutrient
absorption. A New Drug Application (NDA) for Gattex is being reviewed by the
U.S. Food and Drug Administration (FDA). On October 16, 2012, the FDA’s
Gastrointestinal Drugs Advisory Committee voted unanimously to recommend
approval of Gattex® (teduglutide) for adults with short bowel syndrome (SBS).
The committee’s recommendation will be considered by the FDA in its review of
the company’s NDA. The Prescription Drug User Fee Act (PDUFA) date for
completion of the review is December 30, 2012.
Characteristics of Gattex-Treated Patients Who Achieved Complete Independence
from Parenteral Support
Of the seven patients who successfully weaned off PN/IV with Gattex treatment,
two were male and five were female. Ages of the seven patients ranged from 39
to 69. Reasons for resection of the bowel included: ulcerative colitis,
Crohn’s disease, injury, enteritis radiotherapy, mesenteric arterial
infarction, and micro-vascular of unknown process. Five of the seven patients
had colon in continuity.
The number of years the patients had depended on PN/IV at the time they were
weaned off varied from 2 to 14 years. All seven patients had a baseline PN/IV
requirement of less than 13 liters a week. Four of the seven patients had a
baseline PN/IV requirement of three days a week, one patient had four days a
week, and two patients had six days a week. The duration of time the patients
had been treated with Gattex at the time their PN/IV was discontinued ranged
from 28 to 101 weeks.
Adverse events among the seven patients were similar to those of the overall
study population, with gastrointestinal events being the most common,
including diarrhea, nausea, vomiting, and abdominal distension.
About STEPS 2
The primary objective of STEPS 2 is to study the long-term safety and
durability of effect in patients who completed, participated in, or qualified
for participation in STEPS, a 24-week, placebo-controlled, multi-center,
international Phase 3 study of Gattex in adult patients with short bowel
syndrome. Seventy-six out of the 78 patients who completed 24 weeks of
treatment in STEPS elected to enroll in STEPS 2. In addition, 12 patients who
successfully completed the optimization and stabilization phase of STEPS opted
to enroll in STEPS 2 after STEPS was fully randomized.
To date, 12 patients or 14 percent have achieved independence from PN/IV
fluids while on Gattex therapy in the ongoing STEPS 2 study. NPS expects the
STEPS 2 study to complete in early 2013 and will report full results
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 adults
with short bowel syndrome (SBS) and a Phase 3 registration study has been
completed for Natpara® (rhPTH [1-84]) in adult hypoparathyroidism. 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, a Takeda company.
“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 (office)
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