NPS Pharmaceuticals, Inc. (NASDAQ:NPSP), a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, today reported that results from the company’s STEPS 2 study of GATTEX ® (teduglutide) in patients with Short Bowel Syndrome (SBS) were accepted for presentation at the American College of Gastroenterology (ACG) Annual Scientific Meeting and Postgraduate Course in San Diego, California, October 11-16, 2013. STEPS 2 was a two-year open-label extension study that involved 88 adult SBS patients with short bowel syndrome. The abstract entitled, “Long-term Safety and Efficacy of Teduglutide for the Treatment of Intestinal Failure Associated with Short Bowel Syndrome: Final Results of the Steps-2 Study, a 2-year, Multicenter, Open-label Clinical Trial” by Schwartz et al. is also being recognized as an ACG Presidential Poster Award recipient. The ACG Educational Affairs Committee identifies the most highly ranked abstracts selected for the poster sessions in each category and acknowledges their achievement with this award. The company also reported that it has submitted a Supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for Gattex revising the product label to include long-term data from STEPS 2. “The data from STEPS 2 show the sustained effects of Gattex, as evidenced by parenteral support volume reduction, days off parenteral support or for some patients complete independence from parenteral support,” said Roger Garceau, MD, FAAP, executive vice president and chief medical officer of NPS Pharmaceuticals. “We’re especially pleased that 13 of the 88 patients in the study fall into the latter category, having been completely weaned off parenteral support while on Gattex. We look forward to the FDA’s response to our sNDA.” About Gattex ® (teduglutide [rDNA origin]) for Injection Gattex ® (teduglutide [rDNA origin]) for injection, for subcutaneous use is a novel, recombinant analog of human glucagon-like peptide 2, a protein involved in the rehabilitation of the intestinal lining. Gattex is indicated for the treatment of adult patients with short bowel syndrome (SBS) who are dependent on parenteral support. Significant reductions in mean PN/IV infusion volume from baseline to end of treatment were seen in the Phase 3 studies of Gattex. In addition, some patients were able to achieve independence from PN/IV support during these trials. The most common side effects of Gattex include stomach area (abdomen) pain or swelling, skin reaction where the injection was given, nausea, headache, cold or flu like symptoms, vomiting, and holding too much fluid in the body (swelling of face, ankles, hands or feet). 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 SBS. NPS is preparing to engage in the pricing and reimbursement processes in key European countries with the goal of commercially launching Revestive in Europe in the first half of 2014.
Teduglutide has orphan drug designation for the treatment of SBS from the European Medicines Agency (EMA) and the FDA.Important Safety Information (ISI) Gattex has been associated with serious risks including:
- Neoplastic growth. There is a risk for acceleration of neoplastic growth. Colonoscopy of the entire colon with removal of polyps should be done before initiating treatment with Gattex and is recommended after 1 year. Subsequent colonoscopies should be done as needed, but no less frequently than every 5 years. In case of intestinal malignancy discontinue Gattex. The clinical decision to continue Gattex in patients with active non-gastrointestinal malignancy should be made based on risk and benefit considerations.
- Intestinal obstruction. In patients who develop obstruction, Gattex should be temporarily discontinued pending further clinical evaluation and management.
- Biliary and pancreatic disease. Patients should undergo laboratory assessment (bilirubin, alkaline phosphatase, lipase, amylase) before starting Gattex. Subsequent laboratory tests should be done every 6 months. If clinically meaningful changes are seen, further evaluation is recommended including imaging, and continued treatment with Gattex should be reassessed.
- Fluid overload. There is a potential for fluid overload while on Gattex. If fluid overload occurs, especially in patients with cardiovascular disease, parenteral support should be appropriately adjusted, and Gattex treatment reassessed.
NPS's earlier stage pipeline includes NPSP795, a calcilytic compound with potential application in rare disorders involving increased calcium receptor activity, such as autosomal dominant hypocalcemia (ADH). NPS complements its proprietary programs with a royalty-based portfolio of products and product candidates that includes agreements with Amgen, GlaxoSmithKline, Janssen Pharmaceuticals, and Kyowa Hakko Kirin. Additional information about NPS is available through its corporate website, http://www.npsp.com.“NPS,” “NPS Pharmaceuticals,” “Gattex,” “Natpara,” “Preotact,” and “Revestive” are the company's trademarks. Disclosure notice 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. Forward looking statements include, but are not limited to, statements concerning our future financial performance. Risks associated to the company's business include, but are not limited to, the risks associated with any failure by the company to successfully commercialize Gattex, including the risk that physicians and patients may not see the advantages of Gattex and may therefore be reluctant to utilize the product, the risk that private and public payers may be reluctant to cover or provide reimbursement for Gattex, the risks associated with the company's strategy, global macroeconomic conditions, the impact of changes in management or staff levels, the effect of legislation effecting healthcare reform in the United States, 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, whether as a result of new information, future events or otherwise.