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NPS Pharmaceuticals Initiates Phase 3 STEPS Study to Confirm GATTEX " Reduces Parenteral Nutrition Dependence in Short B

^{Published on 2008-12-02 06:48:57 - Market Wire}
 

BEDMINSTER, N.J.--([ BUSINESS WIRE ])--NPS Pharmaceuticals (NASDAQ: NPSP), a specialty pharmaceutical company focused on developing therapeutics for rare gastrointestinal and endocrine disorders, today announced that patient enrollment has begun in a Phase 3 registrational study to confirm previously reported data that demonstrated GATTEX™ (teduglutide) was well tolerated and reduced parenteral nutrition (PN) dependence in short-bowl syndrome (SBS) patients. The international, double-blind, placebo-controlled safety and efficacy study of GATTEX is known as STEPS (Study of TEduglutide in PN-dependent Short-bowel syndrome). SBS is primarily caused by intestinal resection and the subsequent inability to maintain oral fluid, electrolyte, and nutrient balances.

"Launching STEPS is an important milestone for NPS, bringing us closer to our goal of introducing a new standard of care to reduce parenteral nutrition dependence for SBS patients," said Francois Nader, M.D., president and chief executive officer of NPS Pharmaceuticals. "We are advancing STEPS with the support of our partner Nycomed and expect to complete patient enrollment in approximately 12 to 15 months."

STEPS Design

STEPS will enroll approximately 86 PN-dependent SBS patients in North America and Europe. The trial includes an initial three to eight week PN optimization and stabilization period, after which patients will be 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 objectives will evaluate efficacy variables based on reductions in PN volume or the direct effects of improved intestinal absorption of fluid. These variables include: duration of response (total number of weeks at greater than or equal to 20 percent reduction from baseline); the proportion of patients with a 20 percent or greater reduction or a two liter or greater reduction from baseline in weekly PN at week 20 and maintained through week 24; the number of patients who discontinue PN, including the time of discontinuation; and the absolute and percentage change in PN.

STEPS was designed with input from the U.S. Food and Drug Administration (FDA) and NPS believes positive results will enable it to seek U.S. marketing approval for GATTEX for patients with PN-dependent SBS.

In 2007, NPS granted Nycomed the rights to develop and commercialize teduglutide outside the United States, Canada and Mexico. NPS retains all North American rights.

Results of Phase 3 Studies

Initiation of the STEPS Phase 3 registrational study follows positive previously reported Phase 3 data.

In October 2007, NPS reported positive top-line results from a Phase 3 study of GATTEX in which 83 patients with SBS received either a low dose of GATTEX (0.05 mg/kg/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 percent. In an intent-to-treat analysis, forty-six percent (46 percent) 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 percent) 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 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.

Earlier this year, NPS reported positive one-year data from a Phase 3-extension study that demonstrated GATTEX was well tolerated out to one year and provided the ability to safely reduce PN dependence. Three patients who gained independence from PN during the first 24 weeks of therapy remained off PN at week 52 and one additional patient was weaned from PN during the 28-week extension phase. These patients remained PN-independent for periods ranging from 0.5 to 3.5 years.

Given the drug's orphan designation in SBS and the statistically strong and clinically meaningful findings in the low-dose group, NPS met with FDA to discuss the regulatory requirements for the development of GATTEX for SBS and FDA recommended that NPS conduct a confirmatory Phase 3 study before submitting a new drug application.

About Short Bowel Syndrome

Short bowel syndrome or 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 bowel. There are an estimated 10,000 to 15,000 SBS patients in North America who are dependent on parenteral nutrition (PN), 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 an inability to absorb adequate amounts of nutrients and water. The goals of current treatment are to maintain fluid, electrolytes 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 websites [ http://www.shortbowel.com ] and [ http://www.glucagon.com ].

About GATTEX™ (teduglutide)

GATTEX (teduglutide) is a proprietary analog of human glucagon-like peptide 2 that stimulates the repair and regeneration of cells lining the intestine. GATTEX is in Phase 3 clinical development to reduce dependence on parenteral nutrition (PN) in patients with short bowel syndrome (SBS). NPS has reported positive findings from recently completed studies in which GATTEX demonstrated a favorable safety profile and significant 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.

About NPS Pharmaceuticals

NPS Pharmaceuticals is developing therapeutics for rare gastrointestinal and endocrine disorders with high-unmet medical needs. The company is currently advancing two late-stage programs. Teduglutide, a proprietary analog of glucagon-like peptide-2, is in Phase 3 clinical development for intestinal failure associated with short bowel syndrome as GATTEX™ and in preclinical development for gastrointestinal mucositis and necrotizing enterocolitis. NPSP558 (parathyroid hormone 1-84 [rDNA origin] injection) is in Phase 3 clinical development as a hormone therapy for hypoparathyroidism. NPS complements its proprietary programs with a royalty-based portfolio of products and product candidates that includes strategic partnerships with Amgen, GlaxoSmithKline, Kyowa Kirin, and Nycomed. Additional information is available at [ http://www.npsp.com ].

"NPS" and "NPS Pharmaceuticals" 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 NPS's business include, but are not limited to, the risk of not successfully executing its preclinical and clinical studies and not gaining marketing approvals for GATTEX and NPSP558, the risks associated with the implementation of a new business strategy, the risks associated with the company's auction-rate securities, 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/A 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.