Our most advanced product candidates are GATTEX™ (teduglutide) and NPSP558 (parathyroid hormone 1-84 [rDNA origin] injection).  These are both therapeutic proteins to restore or replace biological function that we are developing for specialty gastrointestinal and endocrine disorders with high-unmet medical needs. 

Our proprietary portfolio also includes a number of compounds that are available for out-licensing for areas outside of our proprietary therapeutic and/or geographic focus.  These include PREOS® (parathyroid hormone 1-84 [rDNA origin] injection) for osteoporosis, teduglutide for Crohn’s disease, and glycine reuptake inhibitors and NPSP156 for central nervous system disorders.  For a more complete discussion, please visit our partnering opportunities.

GATTEX™ (teduglutide)

GATTEX™ (teduglutide) is our proprietary analog of naturally occurring 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 teduglutide stimulates the repair and regeneration of cells lining the small intestine, expanding the surface area for absorption of nutrients.  Our lead indication for teduglutide is short bowel syndrome or SBS, a complex disorder primarily caused by intestinal resection.  SBS patients suffer from malnutrition, severe diarrhea, dehydration, fatigue, osteopenia, and weight loss due to a loss in the ability to absorb adequate amounts of nutrients and water.  The goals of current treatment are to maintain fluid electrolyte, and nutrient balances through dietary management, including the use of parenteral nutrition (PN).  

We are currently advancing a Phase 3 registration study to confirm previously reported data that demonstrated GATTEX was well tolerated and reduced PN dependence in SBS patients.  The international, double-blind, placebo-controlled safety and efficacy study of GATTEX is known as STEPS (Study of TEduglutide in PN-dependent Short-bowel syndrome).  Initiation of our STEPS study follows positive findings from completed studies in which GATTEX demonstrated a favorable safety profile and significant reductions in mean PN volume from pretreatment baseline were observed. We believe positive results from STEPS will enable us to seek U.S. marketing approval for GATTEX for the treatment of SBS.  For more information on STEPS, including enrollment criteria, please click here.

We are also advancing preclinical studies to evaluate teduglutide in additional intestinal failure related conditions, such as pediatric indications and gastrointestinal mucositis.

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

NPSP558 is our proprietary recombinant, full-length (1-84), human parathyroid hormone (PTH 1-84) that we are developing in the U.S. as a potential treatment for hypoparathyroidism, a rare condition in which the body produces insufficient levels of parathyroid hormone.  Because the primary role of parathyroid hormone is to maintain normal calcium levels in the blood, the consequences of its absence is low circulating calcium levels or hypocalcemia, which can cause tingling of the hands, fingers, and mouth, serious muscle cramping, tetany or convulsions.   The consequences of hypoparathyroidism include vitamin D deficiency, hypercalciuria, and bones of poor material quality.   Because NPSP558 is identical in structure to the 84 amino acid single-chain polypeptide human parathyroid hormone and mimics the action of natural parathyroid hormone, we believe it has the potential to be the first true hormone therapy for hypoparathyroidism and return the body to a physiological or “eucalcemic” state. 

We are currently advancing 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.  The primary objective is to demonstrate, over a 24-week treatment period, that once-daily subcutaneous dosing with NPSP558 at doses of 50µg, 75µg or 100µg is a safe and effective hormone therapy for the treatment of patients with hypoparathyroidism.  We believe positive results from REPLACE will enable us to seek U.S. marketing approval for NPSP558 for the treatment of hypoparathyroidism.

For more information on REPLACE, including enrollment criteria, please click here.