ir.npsp.com
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`http://ir.npsp.com/releasedetail.cfm?ReleaseID=829997
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`NPS Pharmaceuticals Announces Completion of Treatment Phase
`in STEPS Registration Study of GATTEX in Short Bowel
`Syndrome
`
`Jan 10, 2011
`
`BEDMINSTER, N.J., Jan 10, 2011 (BUSINESS WIRE) -- NPS Pharmaceuticals,
`Inc. (NASDAQ: NPSP) a specialty pharmaceutical company developing innovative
`therapeutics for rare gastrointestinal and endocrine disorders, today announced
`the completion of the 24-week treatment phase of the company's Phase 3
`registration study of GATTEX® (teduglutide). The double-blind, placebo-controlled
`safety and efficacy study known as STEPS randomized 86 patients with parenteral nutrition (PN) dependent short
`bowel syndrome (SBS) to receive drug or placebo over a 24-week treatment period.
`
`"With the last patient's final clinical visit, we remain on track with our development timelines for GATTEX in short
`bowel syndrome," said Francois Nader, MD, president and chief executive officer of NPS Pharmaceuticals. "We look
`forward to reporting top line results later this quarter and to filing for marketing authorization with the U.S. Food and
`Drug Administration if the results are positive."
`
`NPS is also advancing STEPS 2, an open-label continuation study in which all participants will receive up to an
`additional 24 months of GATTEX therapy. Ninety-seven percent of eligible patients who completed STEPS elected to
`enroll in STEPS 2.
`
`SBS is a rare disorder characterized by inadequate absorption of fluids and nutrients in people who have had a
`significant portion of their small intestine removed. Some SBS patients require the use of chronic PN or intravenous
`feeding to supplement and stabilize their nutritional needs. GATTEX is NPS' proprietary analog of human glucagon-
`like peptide 2 (GLP-2), a naturally-occurring peptide involved in the repair and maintenance of normal structure and
`function of the intestine. The goal of treatment with GATTEX is to restore the structural and functional integrity of the
`remaining intestine to reduce PN dependence.
`
`About the STEPS Studies
`
`STEPS is an international, double-blind, placebo-controlled Phase 3 registration study to confirm that GATTEX is well
`tolerated and reduces PN dependence in adults with SBS. The company believes positive results from STEPS will
`enable it to seek U.S. marketing approval for GATTEX.
`
`Eighty-six PN-dependent SBS patients were randomized in the STEPS study at approximately 30 sites in North
`America and Europe. The trial included an initial three- to eight-week optimization and stabilization period, after which
`patients were randomized 1:1 to compare daily subcutaneous dosing of 0.05 mg/kg of GATTEX to placebo over a 24-
`week treatment period.
`
`The primary efficacy endpoint is the percentage of patients who achieve a 20 percent or greater reduction in weekly
`PN volume at week 20 and maintain that response at week 24, when compared to baseline. The study's secondary
`endpoints will evaluate efficacy variables based on reductions in PN volume or the direct effects of improved intestinal
`absorption of fluid.
`
`NPS expects to report top-line results from STEPS in the first quarter of 2011 and if positive, subsequently seek U.S.
`marketing approval for GATTEX in SBS.
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`Patients who participated in STEPS had the option to enroll in STEPS 2, an open-label continuation study in which all
`participants will receive up to 24 months of GATTEX therapy.
`
`NPS is advancing STEPS and STEPS 2 with the support of its partner Nycomed and the two companies are sharing
`the external clinical costs for the studies.
`
`About Short Bowel Syndrome
`
`Short bowel syndrome, or 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 short bowel syndrome is nutritional support, including parenteral nutrition (PN) or intravenous
`feeding to supplement and stabilize nutritional needs.
`
`Although PN can provide nutritional support for short bowel syndrome patients, it does not improve the body's own
`ability to absorb nutrients. PN is also 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 a poor quality-of-life
`with difficulty sleeping, frequent urination and loss of independence.
`
`There are an estimated 10,000 to 15,000 SBS patients in North America who are dependent on PN, the direct cost of
`which can exceed $100,000 annually per patient.
`
`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. GATTEX is in Phase 3 development to reduce dependence on parenteral
`nutrition (PN) in patients with short bowel syndrome (SBS). NPS has reported findings from completed studies in
`which GATTEX demonstrated a favorable safety profile and reductions in mean PN volume from pretreatment
`baseline were observed. NPS is also advancing preclinical studies to evaluate teduglutide in additional intestinal
`failure related conditions.
`
`Teduglutide has received orphan drug designation for the treatment of SBS from the U.S. Food and Drug
`Administration and the European Medicines Agency.
`
`In 2007, NPS granted Nycomed the rights to develop and commercialize teduglutide outside the United States,
`Canada and Mexico. NPS retains all rights to teduglutide in North America.
`
`About NPS Pharmaceuticals
`
`NPS Pharmaceuticals is developing new treatment options for patients with rare gastrointestinal and endocrine
`disorders. The company is currently advancing two Phase 3 registration programs. Teduglutide, a proprietary analog
`of GLP-2, is being evaluated as GATTEX® in a Phase 3 registration study known as STEPS for intestinal failure
`associated with short bowel syndrome and is in preclinical development for chemotherapy-induced gastrointestinal
`mucositis and other pediatric indications. NPSP558 (parathyroid hormone 1-84 [rDNA origin] injection) is being
`evaluated in a Phase 3 registration study known as REPLACE as a hormone replacement therapy for
`hypoparathyroidism. NPS complements its proprietary programs with a royalty-based portfolio of products and
`product candidates that includes agreements with Amgen, Kyowa Hakko Kirin, Nycomed, and Ortho-McNeil
`Pharmaceutical.
`
`"NPS", "NPS Pharmaceuticals", and "GATTEX" are the company's registered trademarks. All other trademarks, trade
`names or service marks appearing in this press release are the property of their respective owners.
`
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`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, 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.
`
`SOURCE: NPS Pharmaceuticals, Inc.
`
`NPS Pharmaceuticals, Inc.
`Susan M. Mesco, 908-450-5516
`smesco@npsp.com
`
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