NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, today announced that the company has initiated a global registration study of teduglutide in pediatric patients with Short Bowel Syndrome (SBS) who are dependent on parenteral support.

The 12-week, open-label study evaluating teduglutide will be conducted at sites throughout the US and Europe. The primary outcome measures are the number of reported adverse events and changes in parenteral and enteral support requirements, respectively. Additional information about the trial, including a detailed listing of study sites, is available at the National Institutes of Health's clinical trial registry, which can be found here: http://clinicaltrials.gov/show/NCT01952080

"We are particularly proud to advance teduglutide in pediatric Short Bowel Syndrome, as it may bring us one step closer to fulfilling a significant unmet need among children across the globe who face a life burdened by this rare disease," said Roger Garceau, MD, FAAP, executive vice president and chief medical officer of NPS Pharmaceuticals. "We look forward to reporting top line results following the completion of the study in late 2014 or early 2015."

The four-cohort study is expected to enroll up to 36 patients. Non-randomized subjects will receive teduglutide in each of the three active cohorts for 12 weeks. The study will attempt to enroll a fourth cohort of patients in an observational arm that would receive a current standard of care with parenteral support and will be considered a safety control group. All subjects will be screened prior to the start of treatment to establish baseline characteristics including safety, eligibility and nutritional support parameters. Eligibility includes a current history of SBS for a minimum of 12 months prior to screening, which resulted from a major intestinal resection and SBS requiring stable administration of parenteral support for a minimum of three months prior to enrollment, based upon the opinion of the investigator.

About Short Bowel Syndrome

Short Bowel Syndrome, or SBS, is a serious, complex disorder in which the body is unable to absorb enough nutrients and fluids through the gastrointestinal tract to sustain life. SBS typically occurs when a large portion of the intestine has been removed by surgery caused by disease or injury. In rare cases, it is congenital. Common symptoms of SBS can include diarrhea, dehydration, malnutrition, and weight loss, which are closely related to the functional issues of the bowel. For more information about SBS, visit: www.ShortBowelSupport.com.

About Teduglutide

Teduglutide is a novel, recombinant analog of human glucagon-like peptide 2, a protein involved in the rehabilitation of the intestinal lining. In the US, teduglutide (trade name: Gattex®) is approved for the treatment of adult patients with Short Bowel Syndrome (SBS) who are dependent on parenteral support. Gattex is not approved for the treatment of pediatric SBS patients. For more information about Gattex, visit: www.Gattex.com. 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. The company's goal is to commercially launch Revestive in select European countries in the first half of 2014. 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.

Teduglutide has orphan drug designation for the treatment of SBS from the US Food and Drug Administration and European Medicines Agency.

About NPS Pharmaceuticals

NPS Pharmaceuticals is a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide. 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. NPS has also developed Natpara® (rhPTH [1-84]) for the treatment of hypoparathyroidism and submitted a Biologics License Application to the US Food and Drug Administration in October 2013.

NPS' 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 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," "Revestive," and "Natpara" 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/Revestive, including the risk that physicians and patients may not see the advantages of Gattex/Revestive 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/Revestive, 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.

NPS Pharmaceuticals, Inc.
Susan M. Mesco, 908-450-5516
smesco@npsp.com
www.npsp.com