Health & Fitness
Health and FitnessHealth and Fitness 
Thu, February 24, 2011
BEDMINSTER, N.J.--([ BUSINESS WIRE ])--NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a specialty pharmaceutical company developing innovative therapeutics for rare gastrointestinal and endocrine disorders, will be joining the National Organization for Rare Disorders (NORD) and other rare disease organizations around the world in observing World Rare Disease Day on February 28 to draw attention to rare diseases as an important global public health concern.
"As a company dedicated to the development of new biopharmaceuticals to treat rare gastrointestinal and endocrine disorders, we are pleased to show our continued support for Rare Disease Day"
aAs a company dedicated to the development of new biopharmaceuticals to treat rare gastrointestinal and endocrine disorders, we are pleased to show our continued support for Rare Disease Day,a said Francois Nader, M.D., president and chief executive officer of NPS Pharmaceuticals. aThis annual observance celebrates the development of new treatments for rare diseases and highlights the continuing unmet needs of thousands of patients around the world with these conditions. We salute NORD, its members and its international affiliates for their commitment to finding new cures for these rare and often neglected diseases and disorders.a
aThere are nearly 30 million Americansa"and millions more around the worlda"affected by rare diseases,a said Peter L. Saltonstall, president and CEO of NORD. aEveryone knows someone with a rare disease. But, while many of these diseases are serious and lifelong, most have no treatment and many are not even being studied by researchers. This leaves patients and families without hope for a better future.a
A rare disease is one that affects fewer than 200,000 Americans. There are nearly 7,000 such diseases affecting nearly 30 million Americans.
A Commitment to Rare Disorders
NPS currently focuses on two serious rare disorders: short bowel syndrome, or SBS, and hypoparathyroidism, and is currently developing GATTEX® (teduglutide) and NPSP558 (parathyroid hormone 1-84 [rDNA origin] injection), respectively, to address the high unmet medical needs of these orphan indications. In Phase 3 clinical data released last month, GATTEX showed promise as a treatment for adult SBS. NPS expects to release top-line results from a Phase 3 study of NPSP558 in hypoparathyroidism later this year.
Patients with either SBS or hypoparathyroidism face significant challenges and current palliative treatments are associated with serious co-morbidities.
Short Bowel Syndrome or SBS
SBS is a serious, complex disorder in which the body is unable to absorb enough nutrients and/or fluids through the gastrointestinal track to sustain life when on a conventionally accepted normal diet. These patients often rely on intravenous or parenteral nutrition to survive; however, this can lead to serious life-threatening complications, including infections, blood clots, liver and kidney damage and failure. Moreover, patients on PN often experience a poor quality-of-life with difficulty sleeping, frequent bowel movements and urination and loss of independence.
GATTEX is NPSa™ proprietary analogue of human glucagon-like peptide 2 (GLP-2), a naturally-occurring peptide involved in the repair and maintenance of normal structure and function of the intestine. The goal of treatment with GATTEX is to improve the structural and functional integrity of the remaining intestine and enhance absorption of nutrients and fluids and minimize or eliminate the need for PN.
There are an estimated 10,000 to 15,000 adult SBS patients in the U.S. who are dependent on PN, the direct cost of which can exceed $100,000 annually per patient.
Hypoparathyroidism
Hypoparathyroidism is a rare disorder in which the body produces insufficient levels of parathyroid hormone, the bodya™s 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 muscular and neurological symptoms, as well as bone impairments. There is no approved treatment of hypoparthyroidism. It is one of the few remaining hormone deficiency syndromes in which replacement therapy using the native hormone is not clinically available. Hypoparathyroidism is currently managed with large doses of calcium and vitamin D supplementation 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.
NPSP558, a bioengineered replica of human parathyroid hormone, is being evaluated in a Phase 3 registration study, known as REPLACE, as the first hormone replacement therapy for the underlying cause of hypoparathyroidism. Because it mimics the action of natural parathyroid hormone, NPSP558 has the potential to treat hypoparathyroidism and offer a more physiological treatment outcome than what is available with existing treatments.
NPS has estimated that approximately 60,000 to 65,000 patients suffer from hypoparathyroidism in the U.S.
About Rare Disease Day
World Rare Disease Day was launched in Europe four years ago and last year was observed in 46 nations. It is always observed on the last day of February. On that day, patients and patient organizations will post stories, videos and blogs online and host events to raise awareness of these diseases, which are often called aorphansa.
In the U.S., the coalition supporting Rare Disease Day includes patient organizations and advocacy groups, medial professionals and associations, government agencies, researchers, and companies developing treatments for rare diseases.
Rare Disease Day 2011 activities in the U.S. will include creating an online library of two-minute videos about specific rare diseases and how they affect patientsa™ daily lives. Also, patients across the nation will help NORD create a database of physician experts. Patients will share their personal stories through a survey hosted by NORD and the Pew Research Center, and there will be a drive to enlist support for a new Rare and Neglected Diseases Congressional Caucus.
In 1983, the Orphan Drug Act was passed by Congress to create financial incentives for companies to develop treatments for rare diseases. Since then, more than 360 orphan drugs and biologics have been approved by the Food and Drug Administration (FDA). It is estimated that from 11 to 14 million Americans benefit from these products, but that still leaves more than 15 million Americans with diseases for which there is no approved treatment.
For more information about Rare Disease Day activities in the U.S., go to [ www.rarediseaseday.us ]. For information about global activities, go to [ www.rarediseaseday.org ]).
About NPS Pharmaceuticals
NPS Pharmaceuticals is an outsourcing-based development company focused on bringing biopharmaceuticals to patients with rare disorders and few, if any, therapeutic options. The company is advancing two Phase 3 registration programs, GATTEX® (teduglutide) in short bowel syndrome (SBS) and NPSP558 (parathyroid hormone 1-84 [rDNA origin] injection) in hypoparathyroidism. NPS complements its proprietary programs with a royalty-based portfolio of products and product candidates that includes agreements with Amgen, Kyowa Hakko Kirin, Nycomed, and Ortho-McNeil Pharmaceutical.
aNPSa, aNPS Pharmaceuticalsa, and aGATTEXa are the companya™s registered trademarks.
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. Risks associated to the companya™s business include, but are not limited to, the risks associated with any failure by the company to successfully complete its preclinical and clinical studies within the projected time frames or not at all, the risk of not gaining marketing approvals for GATTEX and NPSP558, the risks associated with the companya™s strategy, as well as other risk factors described in the companya™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.