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Gattex

Generic name: teduglutide
Treatment for: Short Bowel Syndrome

Update: Gattex (teduglutide) Now FDA Approved - December 21, 2012

NPS Pharmaceuticals Submits Chemistry, Manufacturing and Controls (CMC) Section of New Drug Application for Gattex

BEDMINSTER, N.J.--(BUSINESS WIRE)--Aug 18, 2011 - NPS Pharmaceuticals, Inc., a specialty pharmaceutical company developing orphan therapeutics for rare gastrointestinal and endocrine disorders, today announced that it has submitted the chemistry, manufacturing and controls (CMC) section of its new drug application (NDA) for Gattex (teduglutide) to the U.S. Food and Drug Administration (FDA). Gattex is a novel, recombinant analog of human glucagon-like peptide 2 that the company is developing for the treatment of adults with short bowel syndrome or SBS. SBS is a highly disabling disorder in which the body is unable to absorb sufficient nutrients and/or fluids through the gastrointestinal tract. Patients with SBS often rely on parenteral nutrition (PN) or intravenous (IV) fluids to survive.

"This rolling submission is an important milestone for NPS and brings us one step closer to commercializing Gattex," said Francois Nader, MD, president and chief executive officer of NPS Pharmaceuticals. "We believe Gattex represents an important treatment advance for patients with short bowel syndrome, and we look forward to submitting the remainder of our U.S. marketing application later this year."

FDA regulations permit an applicant to submit the CMC section 90 to 120 days before the anticipated submission of the remainder of the application. FDA will review the early CMC submission as resources permit.

Earlier this year, NPS reported positive data from a 24-week Phase 3 pivotal study of Gattex. The study, known as STEPS, evaluated the ability of Gattex to reduce PN and IV fluid requirements of adult subjects with SBS. The primary efficacy endpoint was defined as the percentage of patients who achieved a 20 to 100 percent reduction in weekly PN/IV volume at Weeks 20 and 24, compared to baseline. In the intent-to-treat population, 63 percent (27/43) of Gattex-treated patients were responders versus 30 percent (13 of 43) of placebo-treated patients (p=0.002).

Subjects treated with Gattex for 24 weeks achieved significantly greater reductions in weekly PN and IV fluid volume and infusion days versus placebo. At Week 24, subjects who received Gattex experienced an average 4.4 liter reduction in weekly PN/IV volume from a pre-treatment baseline of 12.9 liters; subjects who received placebo experienced an average 2.3 liter reduction from a pre-treatment baseline of 13.2 liters (p ≤ 0.001). After completing 24 weeks of treatment, 54 percent (21 of 39) of Gattex-treated subjects 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 company is also advancing STEPS 2, an open-label continuation study of STEPS in which each subject receives Gattex. At an interim update in May 2011, NPS reported that three subjects participating in STEPS 2 were able to gain independence from and discontinue PN and IV fluids.

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. The lead indication for Gattex is treatment of adults with short bowel syndrome. NPS has reported findings from completed studies in which Gattex was well tolerated and effectively reduced parenteral nutrition and intravenous fluid volume requirements in subjects with adult short bowel syndrome.

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 March 2011, Nycomed submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for clearance to market teduglutide (Revestive) as a once-daily subcutaneous treatment for short bowel syndrome. 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 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 or intravenous fluids (parenteral support) to supplement and stabilize nutritional needs.

Although parenteral support can meet basic nutrition and fluid requirements by delivering them intravenously, it does not improve the body's own ability to absorb nutrients. Parenteral support is also associated with serious complications, such as infections, blood clots or liver damage, and the risks increase the longer patients are on it. Patients on parenteral support 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 the U.S. who are dependent on parenteral support, the direct cost of which can exceed $100,000 annually per patient.

About NPS Pharmaceuticals

NPS Pharmaceuticals is an outsourcing-based development company focused on bringing biopharmaceuticals to patients with rare disorders and few, if any, therapeutic options. The company is advancing two Phase 3 registration programs, Gattex (teduglutide) in short bowel syndrome (SBS) and NPSP558 (parathyroid hormone 1-84 [rDNA origin] injection) in 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.

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 NPSP558, 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.

Contact: NPS Pharmaceuticals, Inc.
Susan M. Mesco, 908-450-5516
smesco@npsp.com

Posted: August 2011

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