NPS Pharma Initiates Phase 2a Study of NPSP795 in Adult Patients with Autosomal Dominant Hypocalcemia (ADH)

Open-label, dose-escalating study to assess safety and tolerability of NPSP795, which is designed to directly address the underlying molecular defect associated with ADH

BEDMINSTER, N. J.--()--NPS Pharmaceuticals, Inc. (NASDAQ:NPSP), a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases, has initiated a Phase 2a study to evaluate the safety and tolerability of NPSP795 in adult patients with Autosomal Dominant Hypocalcemia (ADH), an ultra-rare genetic disorder of calcium homeostasis. ADH is caused by mutations of the calcium-sensing receptor (CaSR) gene that increase the sensitivity of the receptor to serum calcium. NPSP795 is a selective calcium receptor antagonist, which binds to the CaSR and decreases its sensitivity to serum calcium. It’s mechanism of action is believed to restore the normal physiological action of the CaSR and address the underlying molecular defect in ADH to return to normal calcium homeostasis. The company expects to report preliminary top-line data from the study in late 2014 or early 2015.

“NPS Pharma is committed to advancing the understanding of Autosomal Dominant Hypocalcemia (ADH), an ultra-rare genetic disease that can lead to serious renal complications,” said Roger Garceau, MD, FAAP, executive vice president and chief medical officer of NPS Pharma. “This new study is an important step in our exploration of the potential of NPSP795 for the treatment of patients with ADH, who do not have approved treatment options."

About the Phase 2a Study
The non-randomized, open-label, dose-escalating study will evaluate the safety and tolerability of an intravenous (IV) infusion of NPSP795. The study will also test the ability of various doses of NPSP795 to stimulate parathyroid hormone secretion, increase blood calcium and decrease renal calcium excretion. The study is expected to enroll up to 12 patients at the National Institutes of Health’s Clinical Center in Bethesda, MD. Additional information about the study is available here.

About Autosomal Dominant Hypocalcemia (ADH)
Autosomal Dominant Hypocalcemia, or ADH, is an ultra-rare, genetic disease caused by mutations of the calcium-sensing receptor (CaSR) gene that increases the sensitivity of the receptor to serum calcium. The underlying cause of symptoms and complications in patients are two-fold. First, absolute hypocalcemia can cause life-threatening seizures, laryngeal spasms, cardiac arrhythmias, tetany, paresthesias, muscle cramping, and decreased focus. In addition, the body’s perceived hypercalcemia results in inappropriately increased renal calcium excretion, decreased renal phosphate excretion, and hyperphosphatemia, putting patients at risk for renal complications, including renal stones, nephrocalcinosis, and impaired renal function, as well as soft tissue calcifications.

Patients with ADH can present immediately post-birth with severe hypocalcemia that can be life-threatening. Supportive approaches focus on managing the symptoms of hypocalcemia by carefully adjusting oral calcium and active vitamin D, but these efforts can worsen the condition by increasing calcium urine excretion and renal complications. Hypocalcemia may be asymptomatic in some patients with ADH; however, nervous system and kidney complications are often present in these patients.

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 current therapeutic areas of focus are gastrointestinal disease and endocrine disorders. These include Short Bowel Syndrome, a potentially fatal gastrointestinal disorder in which patients may have to rely on parenteral nutrition for their survival; Hypoparathyroidism, a complex endocrine disorder in which the parathyroid glands are either absent or damaged, and the body produces insufficient or no parathyroid hormone; and Autosomal Dominant Hypocalcemia, an ultra-rare, genetic disorder of calcium homeostasis caused by mutations of the calcium-sensing receptor gene. NPS Pharma continues to seek in-licensing opportunities to develop new therapies for a broad range of rare diseases, and 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 has operations in the U.S., Canada, Europe, Latin America and Japan. Learn more at: www.npsp.com

“NPS Pharma” and “NPS Pharmaceuticals” 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 and plans for the commercialization of its products, beliefs or expectations regarding our products in development, statements concerning the company’s plans for international expansion, beliefs or expectations regarding potential revenue and earnings from product sales, including beliefs regarding our ability to grow sales, expectations regarding the market size for our products, including those in development, and beliefs or expectations regarding our operating expenses. 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 (teduglutide [rDNA origin]) for injection, 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, risks related to regulatory approvals for recombinant human parathyroid hormone 1-84 (rhPTH [1-84]), 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 press release and NPS undertakes no duty to update this information, whether as a result of new information, future events or otherwise.

Contacts

NPS Pharma
Media:
Justine O’Malley, + 1 908 375-7665
JOmalley@npsp.com
or
Investors:
Susan Mesco, + 1 908 450-5516
SMesco@npsp.com

Contacts

NPS Pharma
Media:
Justine O’Malley, + 1 908 375-7665
JOmalley@npsp.com
or
Investors:
Susan Mesco, + 1 908 450-5516
SMesco@npsp.com