Profile: NPS Pharmaceuticals, Inc. (NPSP.O)

NPS Pharmaceuticals, Inc. (NPS) is a biopharmaceutical company focused on the development of new treatment options for patients with gastrointestinal and endocrine disorders and medical needs. The Company’s clinical programs involve two therapeutic proteins: GATTEX (teduglutide) and NPSP558 (parathyroid hormone 1-84 [rDNA origin] injection). GATTEX is its analog of GLP-2, a protein involved in the regeneration and repair of the intestinal lining, and is in Phase 3 clinical development for parenteral dependent (PN) short bowel syndrome (SBS). NPSP558 is its recombinant human parathyroid hormone (PTH 1-84) that is in Phase 3 clinical development for hypoparathyroidism, a condition in which the body does not maintain normal calcium levels in the blood due to insufficient levels of parathyroid hormone. The Company’s subsidiaries include Allelix Biopharmaceuticals Inc., NPS Allelix Corp. (Allelix), Cinacalcet Royalty Sub LLC.

GATTEX (teduglutide)

The Company’s program for GATTEX is in Phase 3 clinical testing in patients with SBS who are PN-dependent. GATTEX is the brand name for teduglutide, its analog of human glucagon-like peptide 2 (GLP-2), a peptide secreted primarily in the distal intestine and involved in the regeneration and repair of the intestinal epithelium. Preclinical and clinical studies have demonstrated that GATTEX stimulates the repair and regeneration of cells lining the small intestine, expanding the surface area for absorption of nutrients. SBS is a disabling condition that can impair quality of life and lead to serious life-threatening complications. SBS arises after resection of the bowel due to Crohn's disease or other conditions. GATTEX treatment is to maintain fluid electrolyte, and nutrient balances through dietary management, including the use of PN. The double-blind, placebo-controlled safety and efficacy study of GATTEX is known as STEPS (Study of TEduglutide in PN-dependent Short-bowel syndrome).

The Company has also completed a single-center, double blind, and randomized, placebo-controlled ascending-dose study. Separate cohorts of healthy subjects were administered multiple doses of GATTEX or placebo in order to investigate the tolerability and pharmacokinetics of GATTEX. Teduglutide also treats other intestinal failure-related conditions, like PN-dependent pediatric SBS, pediatric feeding intolerance, and gastrointestinal mucositis. Pediatric SBS is caused by necrotizing enterocolitis (NEC). NEC is a gastrointestinal or GI disease that primarily affects premature infants. NEC involves infection and inflammation that causes destruction of the bowel or intestine or part of the bowel.

NPSP558 (parathyroid hormone 1-84 [rDNA origin] injection)

The Company’s NPSP558 is a recombinant, full-length (1-84), human parathyroid hormone (PTH 1-84) that it is developing in the United States as a potential treatment for hypoparathyroidism. Its previous clinical studies of this compound for the treatment of osteoporosis has established that daily subcutaneous dosing causes parathyroid hormone levels to rise rapidly and then return to normal levels over a period of hours. In September 2008, Phase 2 proof-of-concept study demonstrated that treatment with NPSP558 had an effect on abnormal bone skeletal properties in patients with hypoparathyroidism. Hypoparathyroidism is an endocrine disorder in which the body produces insufficient levels of parathyroid hormone. Parathyroid hormone is an 84-amino acid polypeptide that regulates the amount of calcium and phosphorus in bone and blood. NPSP558 is identical in structure to the 84-amino acid single-chain polypeptide human parathyroid hormone and mimics the action of natural parathyroid hormone, and has the mechanism of action to offer a physiological treatment.

In December 2008, the Company initiated a Phase 3 registration study, known as REPLACE, evaluating NPSP558 for the treatment of hypoparathyroidism. REPLACE is a double blind, placebo-controlled trial that will randomize approximately 110 patients at over a dozen sites in the U.S., Canada, and Europe.