Digestive Disease Week Presentations Support Therapeutic Potential for Gattex (teduglutide) as Novel Treatment for Adult Short Bowel Syndrome

Update: Gattex (teduglutide) Now FDA Approved - December 21, 2012

-- Presentations include data on characteristics of patients who achieved independence from parenteral nutrition and intravenous fluids while on Gattex –

BEDMINSTER, N.J.--(BUSINESS WIRE)--May 21, 2012 - NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a specialty pharmaceutical company developing innovative therapeutics for rare gastrointestinal and endocrine disorders, today reported the presentation of data at the Digestive Disease Week® (DDW®) meeting held May 19-22 in San Diego. In a poster session, investigators presented data that continue to support Gattex® (teduglutide) as a potential first-in-class treatment for short bowel syndrome (SBS), a rare and debilitating condition in which the body is unable to absorb enough nutrients and/or fluids through the gastrointestinal tract. Patients with SBS depend on chronic parenteral nutrition (PN) and/or intravenous (IV) fluids to survive.

Investigators reported the baseline characteristics of seven patients with short bowel syndrome who successfully achieved complete independence from PN and IV fluids while on Gattex therapy. In addition, new findings demonstrated that the pharmacological properties of Gattex are associated with significant increases in citrulline, an endogenous non-protein amino acid, that is an indirect measure of enterocyte intestinal mass. In a third presentation, results from a randomized study showed that Gattex does not affect gastric emptying in healthy volunteers.

“Gattex may represent an entirely new approach for treating adult short bowel syndrome and these presentations from multiple clinical studies reinforce our belief in the drug's therapeutic potential,” said Roger Garceau, MD, senior vice president and chief medical officer of NPS Pharmaceuticals, Inc. “These data show continued reductions, and for some complete independence from parenteral nutrition (PN) with long-term Gattex therapy, which is encouraging given the serious complications associated with PN.”

The following summarizes the three posters presented at DDW:

Abstract Sa1962: “Short Bowel Syndrome Patients with Intestinal Failure (SBS-IF) Successfully Achieved Complete Independence from Parenteral Nutrition” by Jeppesen et al.

In Phase 3 studies, Gattex was shown to significantly reduce PN/IV fluid dependence in patients with SBS with some patients achieving complete independence. In this research, Dr. Jeppesen and colleagues described the characteristics of seven patients who achieved complete independence with Gattex therapy during two Phase 3 placebo-controlled studies and the open-label extension phase of those trials.

The data showed that of 173 patients who received Gattex:

 

  • Three patients achieved independence during the randomized, double-blind phase of Study CL0600-004, of which two patients received 0.05 mg/kg/day Gattex and one patient received 0.10 mg/kg/day Gattex.
  • One patient achieved independence during the extension phase of Study CL0600-004.
  • Three patients achieved independence during the extension phase of STEPS, the company's 24-week, placebo-controlled Phase 3 registration study.
  • Patients were weaned from PN/IV fluid as early as 12 weeks and as late as 52 weeks after initiation of Gattex, suggesting that long-term Gattex use is associated with continued reduction of PN/IV fluid requirements.
  • Baseline demographics and disease characteristics were highly variable and the adverse event profile was similar to the overall study population with the most frequently reported adverse events being gastrointestinal related.

Abstract Sa1961: “Teduglutide, a Human Recombinant Analog of Glucagon-Like Peptide-2 (GLP-2), Increases Plasma Citrulline Levels in Patients with Short Bowel Syndrome” by Messing et al.

In this research, Dr. Messing and colleagues assessed changes in plasma citrulline at Week 24 in two double-blind, randomized, placebo-controlled Phase 3 studies of Gattex in short bowel syndrome and showed that in both studies, the mean increase in plasma citrulline at Week 24 vs. baseline was significantly greater in patients receiving Gattex compared with those receiving placebo. Plasma citrulline, an amino acid produced by enterocytes has been considered an indirect measure of remnant enterocyte mass. Gattex has been show to promote expansion of normal intestinal epithelium and increase enterocyte mass by increasing villus height and crypt depth in the small bowel mucosa, leading to increased absorptive area. Mean changes in plasma citrulline levels from baseline occurred early in the course of treatment, with increases as early as Week 4. Patients receiving placebo had minimal changes from baseline in plasma citrulline levels.

Abstract Sa1959: “A Randomized, Double-Blind, Placebo-Controlled, Multiple-Dose, Parallel-Group Study to Assess the Effects of Teduglutide on Gastric Emptying in Healthy Subjects” by Berg et al.

Dr. Berg and colleagues reported results from a double-blind, single-center study that assessed the effect of Gattex on gastric emptying in healthy subjects, and showed that Gattex did not affect gastric emptying in healthy subjects as measured by acetaminophen pharmacokinetics. Gattex did not exert any clinical meaningful effects on serum insulin, glucagon or glucose and there were no clinically significant differences with Gattex between fasted and fed states.

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 is currently undergoing regulatory review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) as a once-daily subcutaneous treatment for 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 2007, NPS granted Nycomed (acquired by Takeda Pharmaceutical Company Limited) the rights to develop and commercialize teduglutide (Revestive®) 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 DDW

DDW is the largest international gathering of physicians, researchers and academics in the fields of gastroenterology, hepatology, endoscopy and gastrointestinal surgery. 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 19 - 22, 2012, at the San Diego Convention Center. The meeting showcases more than 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 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 (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.

Contact: NPS Pharmaceuticals
Susan Mesco, 908-450-5516
smesco@npsp.com

 

 

Posted: May 2012

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