CHICAGO--(BUSINESS WIRE)-- NPS Pharmaceuticals, Inc. (NASDAQ:NPSP - News), a specialty pharmaceutical company developing innovative therapeutics for rare gastrointestinal and endocrine disorders, today announced that GATTEX® (teduglutide), a novel, recombinant analog of human glucagon-like peptide 2, was found to effectively and safely reduce parenteral support (PS) volume in adult short bowel syndrome-intestinal failure (SBS-IF) patients. Professor Palle Bekker Jeppesen, M.D., Department of Medical Gastroenterology, Rigshospitalet, University Hospital of Copenhagen, Denmark, presented results of the 24-week, placebo-controlled Phase 3 study known as STEPS today at the Late-Breaking Abstract Session at Digestive Disease Week® (DDW®) 2011 in Chicago, IL.
The study evaluated the ability of GATTEX to reduce the volume of PS in adult SBS-IF patients. The primary efficacy endpoint was defined as the percentage of patients who achieved a 20 to 100 percent reduction in weekly PS volume at Weeks 20 and 24, compared to baseline. In the intention-to-treat (ITT) population, 63 percent (27/43) of GATTEX-treated patients were responders versus 30 percent (13 of 43) of placebo-treated patients (p=0.002).
Patients treated with GATTEX for 24 weeks also achieved significantly greater reductions in weekly PS volume and infusion days versus placebo. At Week 24, patients who received GATTEX experienced an average 4.4 liter reduction in weekly PS volume from a pre-treatment baseline of 12.9 liters; patients 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 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). Despite the significant PS volume reductions, mean body weight remained unchanged for GATTEX-treated patients.
“SBS patients aren’t able to absorb enough nutrients or fluids and depend on parenteral support to meet their nutritional needs,” said Dr. Jeppesen. “These findings from the largest placebo-controlled SBS study conducted to date show that GATTEX has the potential to significantly reduce parenteral support in adult SBS patients, which could provide important clinical benefits. The potential to reduce dependence on parenteral support by one or more days a week is likely to be an exciting prospect for SBS patients, as it could help greatly improve their quality of life.”
“These results provide further evidence that GATTEX could be an important treatment option for patients suffering from the debilitating condition of SBS,” said Francois Nader, MD, president and chief executive officer of NPS Pharmaceuticals. “We expect to file for FDA approval of GATTEX in the second half of this year as a first-in-class treatment for SBS.”
The STEPS study showed that GATTEX was well tolerated. Liver blood tests showed statistically significant improvements in alanine transaminase (ALT), aspartate transaminase (AST) and bilirubin values in the GATTEX-treated group versus placebo. Alkaline phosphatase (ALP) and gamma glutamyl transpeptidase (GGT) values also improved in the GATTEX-treated group versus placebo; however, the difference was not statistically significant. Five of the 86 randomized patients discontinued the study due to adverse events, of which two were GATTEX-treated and three were placebo-treated. Adverse events appear to be consistent with the pharmacological effects of the drug.
The study abstract, titled “Teduglutide, a Novel Analogue of Glucagon-like Peptide 2 (GLP-2), Is Effective and Safe in Reducing Parenteral Support Volume in Short Bowel Syndrome–Intestinal Failure Subjects: Results From a 24-Week, Placebo-Controlled Phase 3 Trial (STEPS),” is available through MyDDW at http://ddw.apprisor.net/DDW_PRES.cfm?id=5613.
STEPS study design
STEPS was an international, double-blind, placebo-controlled Phase 3 pivotal study designed to provide additional evidence of safety and efficacy of GATTEX in reducing PS dependence in adult SBS patients.
Twenty-nine centers in North America and Europe enrolled patients in the STEPS study. Eighty-six patients were randomized and analyzed for efficacy and safety. The trial included an initial PS optimization and stabilization period, after which patients were randomized 1:1 to either daily subcutaneous dosing of 0.05 mg/kg of GATTEX or placebo, each for a 24-week treatment period. A total of 78 patients completed the study.
The primary efficacy endpoint was the percentage of patients who achieved a 20 to 100 percent reduction in weekly PS volume at Week 20 and maintained that response at Week 24, compared to baseline. The study's secondary endpoints included reductions in PS volume.
NPS conducted STEPS with the support of its partner, Nycomed, a global pharmaceutical company, headquartered in Switzerland, which holds the rights to develop and commercialize teduglutide outside of North America. Nycomed submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for teduglutide in March 2011. The two companies share certain external costs for the teduglutide development program.
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 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 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 for adult patients with short bowel syndrome (SBS). NPS has reported findings from completed studies in which GATTEX was found to effectively and safely reduce parenteral support (PS) volume in adult short bowel syndrome patients. Given GATTEX’s mechanism of action, NPS believes it has the potential to treat gastrointestinal conditions associated with intestinal failure.
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 DDW
Jointly sponsored by the American Association for the Study of Liver Diseases, the American Gastroenterological Association (AGA) Institute, the American Society for Gastrointestinal Endoscopy and the Society for Surgery of the Alimentary Tract, DDW takes place May 7 - 10, 2011, at McCormick Place, Chicago, IL. The meeting showcases approximately 5,000 abstracts and hundreds of lectures on the latest advances in GI research, medicine and technology. For more information, visit www.ddw.org.
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 adult 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.
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.
