NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a biopharmaceutical company developing innovative therapeutics for rare gastrointestinal and endocrine disorders, today reported data related to the seven patients who achieved complete independence from parenteral nutrition (PN) and/or intravenous fluids (IV) while on long-term Gattex® (teduglutide) therapy in STEPS 2, a 24-month, open-label study in adult short bowel syndrome (SBS). The data suggest that patients achieving independence from PN/IV tend to have colon in continuity and lower baseline PN/IV needs than typical patients studied, as all seven patients had baseline PN/IV requirements of less than 13 liters a week. A poster (P1267) titled “Long-Term Therapy With Teduglutide: Characteristics of Patients With Intestinal Failure Associated With Short Bowel Syndrome Who Achieved Complete Independence From Parenteral Nutrition and/or Intravenous Fluid Support” was presented by lead study investigator Ken Fujioka, MD and colleagues at the American College of Gastroenterology Annual Scientific Meeting in Las Vegas, NV. “Patients with short bowel syndrome face numerous medical risks and lifestyle challenges,” said Ken Fujioka, MD, Nutrition and Metabolic Research Center, Scripps Clinic, Del Mar. “They are often tethered to a PN/IV line, have to make frequent trips to the bathroom, suffer from disrupted sleep, and face serious complications including infections, blood clots, or liver damage. The fact that long-term treatment with Gattex has shown to help reduce PN/IV volume or even completely eliminate the need for it is exciting for patients and physicians. Gattex has the potential to be a truly life-changing treatment for adult short bowel syndrome.” Gattex is a novel, recombinant analog of human glucagon-like peptide 2, a peptide involved in normal intestinal function and fluid and nutrient absorption. A New Drug Application (NDA) for Gattex is being reviewed by the U.S. Food and Drug Administration (FDA). On October 16, 2012, the FDA’s Gastrointestinal Drugs Advisory Committee voted unanimously to recommend approval of Gattex® (teduglutide) for adults with short bowel syndrome (SBS). The committee’s recommendation will be considered by the FDA in its review of the company’s NDA. The Prescription Drug User Fee Act (PDUFA) date for completion of the review is December 30, 2012.
Characteristics of Gattex-Treated Patients Who Achieved Complete Independence from Parenteral SupportOf the seven patients who successfully weaned off PN/IV with Gattex treatment, two were male and five were female. Ages of the seven patients ranged from 39 to 69. Reasons for resection of the bowel included: ulcerative colitis, Crohn’s disease, injury, enteritis radiotherapy, mesenteric arterial infarction, and micro-vascular of unknown process. Five of the seven patients had colon in continuity. The number of years the patients had depended on PN/IV at the time they were weaned off varied from 2 to 14 years. All seven patients had a baseline PN/IV requirement of less than 13 liters a week. Four of the seven patients had a baseline PN/IV requirement of three days a week, one patient had four days a week, and two patients had six days a week. The duration of time the patients had been treated with Gattex at the time their PN/IV was discontinued ranged from 28 to 101 weeks. Adverse events among the seven patients were similar to those of the overall study population, with gastrointestinal events being the most common, including diarrhea, nausea, vomiting, and abdominal distension. About STEPS 2 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. To date, 12 patients or 14 percent have achieved independence from PN/IV fluids while on Gattex therapy in the ongoing STEPS 2 study. NPS expects the STEPS 2 study to complete in early 2013 and will report full results thereafter.
About Short Bowel SyndromeShort 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 adults with short bowel syndrome (SBS) and a Phase 3 registration study has been completed for Natpara® (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, a Takeda company. “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.