NPS Pharmaceuticals, Inc., a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, has started a global registration study of teduglutide in paediatric patients with Short Bowel Syndrome (SBS) who are dependent on parenteral support.

The 12-week, open-label study evaluating teduglutide will be conducted at sites throughout the US and Europe. The primary outcome measures are the number of reported adverse events and changes in parenteral and enteral support requirements, respectively. Additional information about the trial, including a detailed listing of study sites, is available at the National Institutes of Health’s clinical trial registry, which can be found here: http://clinicaltrials.gov/show/NCT01952080

“We are particularly proud to advance teduglutide in paediatric Short Bowel Syndrome, as it may bring us one step closer to fulfilling a significant unmet need among children across the globe who face a life burdened by this rare disease,” said Roger Garceau, MD, FAAP, executive vice president and chief medical officer of NPS Pharmaceuticals. “We look forward to reporting top line results following the completion of the study in late 2014 or early 2015.”

The four-cohort study is expected to enroll up to 36 patients. Non-randomized subjects will receive teduglutide in each of the three active cohorts for 12 weeks. The study will attempt to enroll a fourth cohort of patients in an observational arm that would receive a current standard of care with parenteral support and will be considered a safety control group. All subjects will be screened prior to the start of treatment to establish baseline characteristics including safety, eligibility and nutritional support parameters. Eligibility includes a current history of SBS for a minimum of 12 months prior to screening, which resulted from a major intestinal resection and SBS requiring stable administration of parenteral support for a minimum of three months prior to enrollment, based upon the opinion of the investigator.

Teduglutide is a novel, recombinant analog of human glucagon-like peptide 2, a protein involved in the rehabilitation of the intestinal lining. In the US, teduglutide (trade name: Gattex) is approved for the treatment of adult patients with Short Bowel Syndrome (SBS) who are dependent on parenteral support. Gattex is not approved for the treatment of pediatric SBS patients. For more information about Gattex, visit: www.Gattex.com. In the EU, teduglutide (trade name: Revestive) is approved for the treatment of adult patients with SBS; patients should be stable following a period of intestinal adaptation after surgery. The company’s goal is to commercially launch Revestive in select European countries in the first half of 2014. Teduglutide is not approved for the treatment of pediatric SBS patients. The safety and efficacy of teduglutide in this population is currently being evaluated in a global registration trial.

Teduglutide has orphan drug designation for the treatment of SBS from the US Food and Drug Administration and European Medicines Agency.