Initial Findings from PARADOX, Largest and Most Comprehensive Research Analyzing the Burden of Hypop

Initial Findings from PARADOX, Largest and Most Comprehensive Research Analyzing the Burden of Hypoparathyroidism, Reveals Collection of Nearly 40 Physical, Cognitive, Emotional Symptoms

-- 99% of patients experience multiple symptoms despite taking medications, such as calcium and vitamin D --

-- Patients experience an average of 16 symptoms -

-- Nearly 70% of patients experienced comorbidities including heart arrhythmias and kidney stones -

BEDMINSTER, N.J.--(BUSINESS WIRE)-- NPS Pharmaceuticals, Inc. (NAS: NPSP) , a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, yesterday presented initial findings from PARADOX, which is believed to be the largest and most comprehensive collection of research to date analyzing the burden of illness for hypoparathyroidism. Findings from 374 patients in PARADOX, which NPS conducted in partnership with the Mayo Clinic and the Hypoparathyroidism Association, reveal that patients with hypoparathyroidism experience an average of 16 of the 38 physical, emotional and cognitive symptoms cited, some of which were reported for the first time in this landmark assessment. Ninety-nine percent of patients with this rare, complex endocrine disorder experience multiple symptoms despite taking medications, such as calcium and vitamin D. Further, 69% of patients with hypoparathyroidism experience serious comorbid conditions, including heart arrhythmias and kidney stones. The findings were presented at ENDO 2013, The Endocrine Society's 95th Annual Meeting in San Francisco, CA.

"PARADOX provides much-needed evidence to elucidate the burden of hypoparathyroidism and the clinical, social and economic impact of this rare endocrine disorder," said Bart Clarke, MD, Associate Professor of Medicine at the Mayo Clinic in Rochester, MN and lead researcher for PARADOX. "Beyond the quantity, frequency, and pervasive nature of symptoms reported in PARADOX, what should not be overlooked is that these patients continued to be burdened by hypoparathyroidism despite attempts to manage the disorder through calcium and vitamin D supplementation. Hypoparathyroidism is the only classic endocrine disorder without an approved replacement therapy and these data reinforce the need for improved treatment options targeting the underlying cause of the disorder."

Key findings from this analysis of PARADOX suggest that patients with hypoparathyroidism have a high burden of illness, as 99% continue to experience multiple symptoms despite the use of calcium and vitamin D supplements and other medications. On average, patients reported experiencing a collection of 16 of the 38 symptoms reported in the analysis. The most common physical symptoms reported included fatigue (82%), muscle pain or cramping (78%), paresthesia (76%), tetany (70%), and joint or bone pain (67%), and pain/heaviness/weakness in extremities (53%). Cognitive and emotional symptoms were also prevalent. Brain fog/mental lethargy (72%), inability to focus or concentrate (65%), and memory loss or forgetfulness (61%), and sleep disturbances (57%) were common, while anxiety (59%) and depression (53%) were also reported. The findings also showed that symptoms persist for an average of 13 hours over the course of a day.

While patients indicated that they were managing their symptoms with calcium and vitamin D, they continued to develop comorbidities of hypoparathyroidism and suffer from acute episodes requiring emergency care and/or hospitalization. Sixty-nine percent of patients experienced comorbidities since diagnosis. Common comorbid conditions that were most frequently reported by patients in PARADOX included heart arrhythmias (66%) and kidney stones (36%). Bone fractures (16%) were also reported. Seventy-nine percent of patients reported hospital stays or emergency department visits, with the annualized rate for patients who classified their condition as severe exceeding those of patients with mild or moderate condition. Patients exceeded the national average for the general population for emergency department visits and hospital stays.

"I have been living with hypoparathyroidism for more than 50 years and have had to live with a litany of debilitating symptoms, including many of those described in PARADOX," said James Sanders, president of the Hypoparathyroidism Association. "This research legitimizes the symptoms that many patients in the hypoparathyroidism community have been experiencing for years, and provides validation that we deal with a significant burden each day, which is important to a rare disease patient."

PARADOX identified 38 symptoms associated with hypoparathyroidism, organized into three categories: physical (25 symptoms); cognitive (7 symptoms); and emotional (6 symptoms). Physical symptoms reported by more than 50% of patients were fatigue (82%), muscle pain or cramping (78%), paresthesia (76%), tetany (70%), joint or bone pain (67%), and pain/heaviness/weakness in extremities (53%). Clinical complications experienced by more than 10% of patients included neuropathy (27%), edema (23%), respiratory (22%), hypercalcemia (18%), nephrocalcinosis (17%), chronic kidney failure (13%), cataract formation (13%), and soft tissue calcifications (11%). Cognitive symptoms reported by more than 50% of patients were mental lethargy (72%), inability to focus or concentrate (65%), memory loss or forgetfulness (61%), and sleep disturbances (57%). Emotional symptoms reported by more than 50% of patients were anxiety (59%) and depression (53%). Patients reported suffering from multiple symptoms (mean=16) with daily impact (mean=13 hour/day). Females were significantly more likely to experience more symptoms than males.

About PARADOX

The PARADOX research assessed the clinical, social and economic implications of hypoparathyroidism in 374 patients in the U.S. aged 18 years or older who were diagnosed with the disorder for at least six months ago. The mean age of respondents was 49 years and a mean time with hypoparathyroidism of 13 years. Eight-five percent of respondents were female and 78% suffered with postsurgical hypoparathyroidism.

PARADOX was approved by an institutional review board, led by researchers from the Mayo Clinic, and conducted in conjunction with the Hypoparathyroidism Association. Data were collected through a 30-minute, web-based instrument which was developed with input from clinical experts, the Hypoparathyroidism Association, and patients. This instrument was primarily disseminated via email to Hypoparathyroidism Association members. Until now, there has been limited research exploring the impact of hypoparathyroidism on the lives of patients, and the nature, type and quantity of hypoparathyroidism symptoms have not been previously documented to this extent. NPS plans to present/publish additional findings from PARADOX at future medical meetings and in peer-reviewed publications.

About Hypoparathyroidism

Hypoparathyroidism is a rare endocrine disorder in which the body produces insufficient levels of parathyroid hormone, the principal regulator of calcium and phosphorus. When the body has too little parathyroid hormone, blood calcium levels drop and phosphorus levels increase, which can cause a number of physical and mental symptoms, including uncontrollable muscle spasms and cramps, tetany, seizures, fatigue, anxiety, and depression. There is currently no FDA-approved replacement therapy for hypoparathyroidism, which is currently managed with large doses of calcium supplementation and active vitamin D therapy to raise the calcium levels in the blood and reduce the severity of symptoms. Over time, calcium may build up in the body and result in serious health risks, including calcifications in the kidneys, heart or brain. Hypoparathyroidism is believed to affect as many as 100,000 Americans.

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 U.S. for adult Short Bowel Syndrome (SBS) patients who are dependent on parenteral support. Teduglutide is also approved for adult SBS in the European Union under the brand name Revestive®. NPS is also developing Natpara® (recombinant full-length human parathyroid hormone or rhPTH [1-84]) for the treatment of adult hypoparathyroidism and, subject to the resolution of certain manufacturing issues, expects to submit its Biologic License Application (BLA) to the FDA in 2013.

NPS's 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 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 the company's 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 (teduglutide [rDNA origin])for injection, 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 risk that the company may be unable to resolve the manufacturing issue in order to submit its BLA for Natpara, 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

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