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NPS Pharmaceuticals, Inc. (NPSP)'s Says Data From STEPS 2 Trial of Gattex Supports Long-Term Use in Phase 3 Study


10/14/2013 6:49:37 AM

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New Data from STEPS 2 Support the Long Term Use of Gattex® (Teduglutide [rDNA origin]) for Injection in Adult Patients with Short Bowel Syndrome

BEDMINSTER, N.J.--(BUSINESS WIRE)-- NPS Pharmaceuticals, Inc. (NPSP), a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, today announced new findings from its STEPS 2 study supporting the long-term use of Gattex® (teduglutide [rDNA origin]) for injection in adult patients with Short Bowel Syndrome (SBS). These data were presented at the American College of Gastroenterology (ACG) Annual Scientific Meeting and Postgraduate Course in San Diego, CA.

In the U.S., Gattex is indicated for the treatment of adult patients with Short Bowel Syndrome who are dependent on parenteral support.

STEPS 2 is a two-year open-label extension study of 88 adult patients with SBS. Investigators reported that the long-term use of Gattex in patients with SBS resulted in additional, clinically meaningful reductions in the volume and days per week of parenteral support requirements in this extension study. In addition, 10 of the 13 patients who achieved complete independence from parenteral support were those who received 30 months of Gattex in the 6-month STEPS pivotal study and the 24-month STEPS 2 study. Two patients who received placebo in STEPS and 24 months of Gattex in STEPS 2 and one patient who bypassed STEPS and was enrolled directly into STEPS 2 also achieved independence from parenteral support. No new unexpected safety concerns were observed with long-term Gattex treatment and the product’s safety profile remains consistent with the product’s label.

The poster, 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,” was recognized as an ACG Presidential Poster Award recipient, which identifies the most highly-ranked abstracts in poster sessions.

“We are pleased to see that long-term treatment with Gattex resulted in clinically meaningful reductions in parenteral support as seen in the previous STEPS study,” said Lauren Schwartz, MD, assistant professor of medicine, Mount Sinai School of Medicine, and study investigator. “Further, it is particularly encouraging that 13 patients achieved complete independence from parenteral support with long-term Gattex therapy. The ability to reduce or even completely eliminate the need for parenteral support could meaningfully impact the lives of patients with this rare and debilitating condition.”

The primary objective of STEPS 2 was to study 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 subjects with SBS.

STEPS 2 enrolled a total of 88 patients in the following three treatment groups:

Gattex/Gattex group: 37 subjects who completed 6 months of Gattex therapy in the STEPS study

Placebo/Gattex group: 39 subjects who received placebo for 6 months in STEPS

Not-treated/Gattex group: 12 subjects who successfully completed the optimization and stabilization phase of STEPS and were enrolled directly in STEPS 2 after STEPS was fully randomized

Sixty-five of the 88 patients enrolled in STEPS 2 (74 percent) completed the study. Seven of the 37 patients in the Gattex/Gattex group withdrew from the study, four of whom were due to adverse events. Sixteen of the 51 patients in the Placebo/Gattex and the Not-treated/Gattex groups withdrew from the study, 12 of whom were due to adverse events.

The majority of patients in STEPS 2 achieved and maintained clinically meaningful responses after Gattex treatment. A responder was defined as a subject who achieved a 20 to 100 percent reduction in the volume of parenteral support from baseline. A higher percentage of patients who received Gattex for 30 months (the Gattex/Gattex group) responded and achieved greater reductions in parenteral support volume and days per week compared with those who received Gattex for 24 months (Placebo/Gattex group and Not-treated/Gattex group). In addition, 33 percent of patients (10/30) who received 30 months of Gattex achieved complete independence from parenteral support.

The rates of adverse events of special interest as described in the U.S. Prescribing Information remain consistent. The most common adverse events were abdominal pain, catheter sepsis, and episodes of decreased weight, nausea, gastrointestinal stoma complications, and abdominal distension.

NPS has submitted a Supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for Gattex torevise the product label to include long-term data from STEPS 2.

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.

Prescribers should select the appropriate patients to receive Gattex in accordance with the approved prescribing information, discuss the benefits and risks of Gattex with patients, and monitor patients as specified in the approved prescribing information and report adverse events to NPS' Gattex information line at 1-855-5GATTEX (1-855-542-8839) or event/product complaint line at 1-855-215-5550.

About NPS Pharmaceuticals

NPS Pharmaceuticals is a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide. The company’s lead product, Gattex® (U.S.)/Revestive® (EU) (teduglutide [rDNA origin]) for injection is approved for adult Short Bowel Syndrome (SBS) patients who are dependent on parenteral support. NPS has also developed Natpara® (rhPTH [1-84]) for the treatment of adult hypoparathyroidism and expects to submit its marketing application to the U.S. Food and Drug Administration in the fourth quarter of 2013.

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.

Contact:

NPS Pharmaceuticals, Inc.

Susan M. Mesco, 908-450-5516 (office)

smesco@npsp.com

www.npsp.com

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