NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a specialty pharmaceutical company developing innovative therapeutics for rare gastrointestinal and endocrine disorders, today announced that GATTEX® (teduglutide), a novel, recombinant analog of human glucagon-like peptide 2, was found to effectively and safely reduce parenteral support (PS) volume in adult short bowel syndrome-intestinal failure (SBS-IF) patients. Professor Palle Bekker Jeppesen, M.D., Department of Medical Gastroenterology, Rigshospitalet, University Hospital of Copenhagen, Denmark, presented results of the 24-week, placebo-controlled Phase 3 study known as STEPS today at the Late-Breaking Abstract Session at Digestive Disease Week® (DDW®) 2011 in Chicago, IL.
The study evaluated the ability of GATTEX to reduce the volume of PS in adult SBS-IF patients. The primary efficacy endpoint was defined as the percentage of patients who achieved a 20 to 100 percent reduction in weekly PS volume at Weeks 20 and 24, compared to baseline. In the intention-to-treat (ITT) population, 63 percent (27/43) of GATTEX-treated patients were responders versus 30 percent (13 of 43) of placebo-treated patients.
Patients treated with GATTEX for 24 weeks also achieved significantly greater reductions in weekly PS volume and infusion days versus placebo. At Week 24, patients who received GATTEX experienced an average 4.4 liter reduction in weekly PS volume from a pre-treatment baseline of 12.9 liters; patients who received placebo experienced an average 2.3 liter reduction from a pre-treatment baseline of 13.2 liters (p≤0.001). After completing 24 weeks of treatment, 54 percent (21 of 39) of GATTEX-treated patients were able to reduce the number of infusion days per week by one or more days, compared to 23 percent (9 of 39) of those treated with placebo.
"SBS patients aren't able to absorb enough nutrients or fluids and depend on parenteral support to meet their nutritional needs," said Dr. Jeppesen. "These findings from the largest placebo-controlled SBS study conducted to date show that GATTEX has the potential to significantly reduce parenteral support in adult SBS patients, which could provide important clinical benefits. The potential to reduce dependence on parenteral support by one or more days a week is likely to be an exciting prospect for SBS patients, as it could help greatly improve their quality of life."
"These results provide further evidence that GATTEX could be an important treatment option for patients suffering from the debilitating condition of SBS," said Francois Nader, MD, president and chief executive officer of NPS Pharmaceuticals. "We expect to file for FDA approval of GATTEX in the second half of this year as a first-in-class treatment for SBS."
The STEPS study showed that GATTEX was well tolerated. Liver blood tests showed statistically significant improvements in alanine transaminase (ALT), aspartate transaminase (AST) and bilirubin values in the GATTEX-treated group versus placebo. Alkaline phosphatase (ALP) and gamma glutamyl transpeptidase (GGT) values also improved in the GATTEX-treated group versus placebo; however, the difference was not statistically significant. Five of the 86 randomized patients discontinued the study due to adverse events, of which two were GATTEX-treated and three were placebo-treated. Adverse events appear to be consistent with the pharmacological effects of the drug.
Source: NPS Pharmaceuticals, Inc.