NPS Pharmaceuticals Announces Publication of Pivotal Phase 3 Study of Gattex (teduglutide) in Short Bowel Syndrome-Intestinal Failure Patients
-- Study available online in Gastroenterology --
BEDMINSTER, N.J.--(BUSINESS WIRE)--Nov 29, 2012 - NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a biopharmaceutical company developing innovative therapeutics for rare gastrointestinal and endocrine disorders, announced today that its Phase 3 study of Gattex (teduglutide), a novel analog of glucagon-like peptide 2, has been published online in the peer-reviewed journal, Gastroenterology, the official journal of the American Gastroenterological Association. The 24-week study, known as STEPS, shows that Gattex is effective and well-tolerated in reducing parenteral support volume and number of infusion days in short bowel syndrome-intestinal failure (SBS-IF) patients.
“This crucial study demonstrates the potential of Gattex to be an important treatment option for patients with short bowel syndrome,” said Palle B. Jeppesen, M.D., lead author of the study and Associate Professor, Department of Medical Gastroenterology, University Hospital of Copenhagen. “Short bowel patients with intestinal failure rely on parenteral support to meet their nutritional, fluid and electrolyte needs because their intestines aren't able to absorb enough nutrients, fluids and electrolytes. Being able to reduce or even eliminate dependence on parenteral support should provide important clinical benefits for patients with this debilitating condition.”
Methodology and Results
Researchers performed a 24-week study of patients with SBS-IF who were given either daily subcutaneous dosing of 0.05 mg/kg teduglutide (n=43) or placebo (n=43). Parenteral support (PS) was reduced if 48-hour urine volumes exceeded baseline values by 10 percent. The primary efficacy endpoint was defined as the number of patients who achieved a 20 to 100 percent reduction in weekly PS volume, from baseline, at Weeks 20 and 24.
There were significantly more responders in the teduglutide group (27 of 43) than the placebo group (13 of 43, p=.002). At Week 24, patients who received teduglutide experienced an average 4.4 liter reduction in weekly PS volume from a baseline of 12.9 liters. Patients who received placebo experienced an average 2.3 liter reduction from a baseline of 13.2 liters (p<.001).
After completing 24 weeks of treatment, 54 percent (21 of 39) of teduglutide-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).
The distribution of treatment-emergent adverse events that led to study discontinuation was similar between patients given teduglutide (n=2) or placebo (n=3). The most commonly reported adverse events were gastrointestinal-related and appear to be consistent with the pharmacological effects of the drug.
The research was funded by NPS and Nycomed, a Takeda company. The study, titled “Teduglutide Reduces Need for Parenteral Support Among Patients with Short Bowel Syndrome with Intestinal Failure,” is now published online at http://www.gastrojournal.org/current.
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 independence.
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 adult short bowel syndrome (SBS). NPS is also developing Natpara® (rhPTH[1-84]) for the treatment of adult hypoparathyroidism and expects to submit its Biologic License Application (BLA) to the FDA in mid-2013. 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 (acquired by Takeda Pharmaceutical Company Limited).
"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.
Posted: November 2012