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FDA Approves Updated Labeling for Gattex® for Injection to Include Long-Term Data

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Patients on Gattex treatment beyond two years continue to achieve days off parenteral support, with some achieving complete independence

June 30, 2014

NPS Pharmaceuticals, Inc., a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved updated labeling for Gattex® (teduglutide [rDNA origin]) for injection to include long-term data from the STEPS 2 study of adult patients with Short Bowel Syndrome (SBS). The data demonstrate that patients who continued Gattex treatment beyond two years achieved clinically meaningful reductions in parenteral support requirements, including volume and days off, with 13 out of 88 patients across all groups achieving complete independence.

In the U.S., Gattex is indicated for the treatment of adult patients with SBS who are dependent on parenteral support. Full prescribing information for Gattex is available by clicking here or visiting www.Gattex.com.

“We are pleased that the FDA has approved updated labeling for Gattex, as it provides important information for healthcare professionals and patients about long-term use of Gattex therapy,” said Roger Garceau, MD, FAAP, executive vice president and chief medical officer of NPS Pharma. “The STEPS 2 study demonstrated that there was an increased response to treatment over time in all groups receiving Gattex in terms of volume reductions, days off and complete weaning of parenteral support.”

About STEPS 2

The efficacy, safety, and tolerability of Gattex was evaluated in a randomized, double-blind, placebo-controlled, parallel-group, multi-national, multi-center clinical trial, known as STEPS, in adults with SBS who were dependent on parenteral nutrition/intravenous (PN/I.V.) support for at least 12 months and required PN at least 3 times per week. STEPS 2 was a 2-year open-label extension of STEPS in which 88 subjects received Gattex 0.05 mg/kg/day. Ninety-seven percent (76/78) of subjects who completed STEPS elected to enroll in STEPS 2 (37 received Gattex; 39 received Placebo). An additional 12 subjects entered STEPS 2, who had been optimized and stabilized but not randomized in STEPS because of closed enrollment.

30 months exposure

Thirty Gattex subjects completed a total duration of 30 months (STEPS followed by STEPS 2 treatment). Of these, 28 subjects (93%) achieved a 20% or greater reduction of parenteral support. Of responders in STEPS who had completed 2 additional years of continuous treatment with Gattex, 96% (21/22) sustained their response to Gattex. The mean reduction in PN/I.V. (n=30) was 7.55 L/week (a 65.6% reduction from baseline). Ten subjects were weaned off their PN/I.V. support while on Gattex treatment for 30 months. Subjects were maintained on Gattex even if no longer requiring PN/I.V. support. These 10 subjects had required PN/I.V. support for 1.2 to 15.5 years, and prior to Gattex had required between 3.5 L/week and 13.4 L/week of PN/I.V. support. At the end of study, 21 (70%), 18 (60%) and 18 (60%) of the 30 completers achieved a reduction of 1, 2, or 3 days per week in PN/I.V. support, respectively.

24 month exposure

Of the 39 placebo subjects from STEPS entering STEPS 2, 29 completed 24 months of treatment with Gattex. The mean reduction in PN/I.V. was 3.11 L/week (an additional 28.3% reduction) from the start of STEPS 2. Sixteen (55.2%) of the 29 completers achieved a 20% or greater reduction of parenteral support. At the end of study, 14 (48.3%), 7 (24.1%) and 5 (17.2%) achieved a reduction of 1, 2, or 3 days per week in PN/I.V. support, respectively. Two subjects were weaned off their PN/I.V. support while on Gattex. Of the 12 subjects entering STEPS 2 directly, 6 completed 24 months of treatment with Gattex. Similar effects were seen. One of the six subjects was weaned off their PN/I.V. support while on Gattex.

The rates of adverse events of special interest as described in the U.S. Prescribing Information remain consistent. The most common adverse events in STEPS 2 were abdominal pain, catheter sepsis, and episodes of decreased weight, nausea, gastrointestinal stoma complications, and abdominal distension.

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.

Teduglutide has orphan drug designation for the treatment of SBS from the European Medicines Agency (EMA) and the FDA.

About NPS Pharma

NPS Pharma is a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases. The company’s lead product, Gattex® (teduglutide [rDNA origin]) for injection is approved in the US for adult patients with Short Bowel Syndrome (SBS) who are dependent on parenteral support. In the EU, teduglutide (trade name: Revestive®) is approved for the treatment of adult patients with SBS; patients should be stable following a period of intestinal adaptation after surgery. Teduglutide is not approved for the treatment of pediatric SBS patients. The safety and efficacy of teduglutide in this population is currently being evaluated in a global registration trial.

A Biologics License Application is undergoing FDA review for Natpara® (rhPTH [1-84]) for the treatment of hypoparathyroidism, a rare endocrine disorder characterized by insufficient levels of parathyroid hormone. The Prescription Drug User Fee Act goal date for the Natpara application is October 24, 2014.

NPS Pharma’s earlier stage pipeline includes NPSP795, a calcilytic compound with potential application in rare disorders involving increased calcium sensing receptor activity, such as autosomal dominant hypocalcemia (ADH). NPS Pharma 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.

“NPS Pharma,” “NPS Pharmaceuticals,” “Gattex,” and “Revestive” are the company’s trademarks.

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