NPS Pharmaceuticals, a specialty pharmaceutical company developing innovative therapeutics for rare gastrointestinal and endocrine disorders, today announced the completion of patient randomization in its Phase 3 registration study of GATTEX® (teduglutide). The double-blind, placebo-controlled safety and efficacy study, which is known as STEPS, is being conducted in patients with parenteral nutrition (PN) dependent short bowel syndrome (SBS) and is now fully randomized with 86 patients.
"The timely completion of randomization in this Phase 3 study marks a significant milestone in our development program for GATTEX in short bowel syndrome," said Francois Nader, MD, president and chief executive officer of NPS Pharmaceuticals. "Short bowel syndrome can be highly debilitating and negatively impact quality-of-life. We believe GATTEX could represent an important advancement in patient care as a potential first-in-class therapy for reducing parenteral nutrition dependence by rehabilitating the intestine and enhancing nutrient absorption. We look forward to delivering our objective of reporting top line results early next year and filing a New Drug Application with the U.S. Food and Drug Administration as soon as possible thereafter."
SBS is a rare disorder characterized by the poor absorption of nutrients that typically occurs in people who have had more than 50% of their small intestine removed. A proportion of SBS patients require the use of chronic PN or intravenous feeding to supplement and stabilize their nutritional needs. Given GATTEX's mechanism of action, which is specific to promoting gastrointestinal rehabilitation, NPS believes it has the potential to treat PN-dependent SBS and other gastrointestinal conditions associated with intestinal failure.