To date, seven of 88 patients enrolled in STEPS 2 discontinued parenteral support
Complete STEPS 2 results expected in early 2013
NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a specialty pharmaceutical company developing innovative therapeutics for rare gastrointestinal and endoccrine disorders, today reported that four additional patients have successfully achieved independence from parenteral nutrition (PN) and intravenous (IV) fluids while on long-term Gattex® (teduglutide) therapy in STEPS 2, a 24-month open-label study in adult short bowel syndrome (SBS).
To date, seven patients have achieved independence from PN/IV fluids while on Gattex therapy in the ongoing STEPS 2 study. These patients had depended on PN/IV fluids for periods ranging from two to 14 years. SBS is a rare and debilitating condition in which the body is unable to absorb enough nutrients and/or fluids through the gastrointestinal tract and patients chronically depend on PN/IV fluids to survive.
"We have continued to see improvements in PN/IV fluid requirements for patients using Gattex in STEPS 2 and are very encouraged by these findings, especially since five patients achieved independence after one year or more of Gattex therapy," said Roger Garceau, MD, senior vice president and chief medical officer of NPS Pharmaceuticals. "Dependence on parenteral nutrition is associated with significant mortality and morbidities, such as central line infections, blood clots, and liver damage. The ability to completely eliminate parenteral support along with these life-threatening complications could significantly enhance the lives of patients with short bowel syndrome."
The primary objective of STEPS 2 is to study the long-term safety and durability of effect in patients who completed, participated in, or qualified for participation in STEPS, a 24-week, placebo-controlled, multi-center, international Phase 3 study of Gattex in adult patients with short bowel syndrome. Seventy-six out of the 78 patients who completed 24 weeks of treatment in STEPS elected to enroll in STEPS 2. In addition, 12 patients who successfully completed the optimization and stabilization phase of STEPS opted to enroll in STEPS 2 after STEPS was fully randomized.
The company expects the STEPS 2 study to complete in early 2013 and will report full results thereafter. There have been no new unexpected adverse events and the safety profile of Gattex remains consistent with previous reports.
About Gattex® (teduglutide)
Gattex (teduglutide) is a novel, recombinant analog of human glucagon-like peptide 2, a peptide involved in normal intestinal function and fluid and nutrient absorption. The lead indication for Gattex is the 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(R)) as a once-daily subcutaneous treatment for short bowel syndrome. In 2007, NPS granted Nycomed (recently acquired by Takeda Pharmaceutical Company Limited) 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 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) and a Phase 3 registration study has been completed for Natpara™ (recombinant human parathyroid hormone (rhPTH [1-84]) in adult hypoparathyroidism. 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.
"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.
Contacts:
NPS Pharmaceuticals, Inc.
Susan M. Mesco, 908-450-5516
smesco@npsp.com
