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Nov 2, 2011

NPS On Track to File for FDA Approval of Short Bowel Syndrome Drug Later This Year

  • NPS Pharmaceuticals released new data from an open-label study with its lead short bowel syndrome (SBS) candidate, Gattex™ (teduglutide), and says it is on track to submit an NDA to FDA later this year. Data from the open-label Steps 2 trial showed that 91% of patients responded to treatment with the glucagon-like peptide 2 (GLP-2) analog, after 12 months of therapy. Response was defined as achieving a 20–100% reduction in the need for parenteral nutrition/intravenous fluid (PN/IV) volume from baseline. A further 24% of patients reduced their infusion days per week by three or more days after 12 months of Gattex therapy, and three patients were able to completely discontinue PN/IV fluids.

    Steps 2 was an open-label extension to the original 24-week Phase III Steps trial in patients with SBS, and included 76 of the 78 patients who completed 24 weeks of treatment in Steps. Another 12 patients who successfully completed the optimization and stabilization phases of Steps opted to enroll in Steps 2.

    Data from an interim analysis of 34 Steps 2 patients who had received at least 12 months of Gattex therapy also showed that 53% of subjects reduced their infusion days per week by one or more, and 38% reduced their infusion days per week by two or more.

    A total of 566 patients have been treated with teduglutide across 15 cincial studies, NPS notes, and there have been two deaths in Gattex therapy cohorts related to cancers that were discovered during the teduglutide study period. One was a metastatic adenocarcinoma of probable gastrointestinal origin, in a patient with a prior history of chemo- and radiotherapy treated Hodgkin disease. In this patient the neoplasm was discovered after 313 days of Gattex therapy. The second cancer death resulted from a non-small cell lung cancer in a 64-year-old smoker. In this case the tumor was discovered 85 days after initiation of treatment with Gattex. A third case of lung squamous cell carcinoma was diagnosed in a 74-year-old smoker 535 days after treatment with Gattex was started. The firm says the three cancer cases have been investigated by an independent safety review board, and no changes in study design or subject monitoring have been requested.

    NPS is focused on developing therapeutics for rare gastrointestinal and endocrine disorders. The firm’s marketed products include Sensipar/Regpara (cinacalcet HCL), which is indicated for the treatment of secondary hyperparathyroidism, and for hypercalcemia in parathyroid cancer, and is partnered with Amgen (worldwide except Asia) and Kyowa Kirin (for Asian territories). Cinacalcet is also in Phase II development with Amgen (worldwide except Asia) for the treatment of primary hyperparathyroidism.

    NPS’ lead pipeline candidates include Gattex and a second Phase III-stage candidate, NPSP558 (parathyroid hormone 1-84 [rDNA origin] inection), which is in development as a hormone therapy for hypoparathyroidism. The parathyroid hormone product is already marketed outside the U.S., in partnership with Nycomed, as a treatment for osteoporosis, and NPS is separately carrying out U.S. Phase III clinical trials with the recombinant hormone for the osteoporosis indication, and is looking to out-license or partner the product.

    Teduglutide, meanwhile, is separately in Phase II studies as a potential treatment for Crohn disease. The firm says the Crohn disease indication for teduglutide is outside its core development focus, however, and has been designated for out-licensing or partnering opportunities, along with a Phase I-stage glycine reuptake inhibitor program for CNS disorders. 


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