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

  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 –

Business Wire

BEDMINSTER, N.J. -- June 17, 2013

NPS Pharmaceuticals, Inc. (NASDAQ: 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.


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,

“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
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