NPS Pharma Says Data from STEPS 2 Trial of Gattex Supports Long-Term Use

NPS Pharmaceuticals, Inc. NPSP, a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, today announced new findings from its STEPS 2 study supporting the long-term use of Gattex^® (teduglutide [rDNA origin]) for injection in adult patients with Short Bowel Syndrome (SBS). These data were presented at the American College of Gastroenterology (ACG) Annual Scientific Meeting and Postgraduate Course in San Diego, CA. In the U.S., Gattex is indicated for the treatment of adult patients with Short Bowel Syndrome who are dependent on parenteral support. STEPS 2 is a two-year open-label extension study of 88 adult patients with SBS. Investigators reported that the long-term use of Gattex in patients with SBS resulted in additional, clinically meaningful reductions in the volume and days per week of parenteral support requirements in this extension study. In addition, 10 of the 13 patients who achieved complete independence from parenteral support were those who received 30 months of Gattex in the 6-month STEPS pivotal study and the 24-month STEPS 2 study. Two patients who received placebo in STEPS and 24 months of Gattex in STEPS 2 and one patient who bypassed STEPS and was enrolled directly into STEPS 2 also achieved independence from parenteral support. No new unexpected safety concerns were observed with long-term Gattex treatment and the product's safety profile remains consistent with the product's label. The poster, entitled “Long-term Safety and Efficacy of Teduglutide for the Treatment of Intestinal Failure Associated with Short Bowel Syndrome: Final Results of the Steps-2 Study, a 2-year, Multicenter, Open-label Clinical Trial,” was recognized as an ACG Presidential Poster Award recipient, which identifies the most highly-ranked abstracts in poster sessions. “We are pleased to see that long-term treatment with Gattex resulted in clinically meaningful reductions in parenteral support as seen in the previous STEPS study,” said Lauren Schwartz, MD, assistant professor of medicine, Mount Sinai School of Medicine, and study investigator. “Further, it is particularly encouraging that 13 patients achieved complete independence from parenteral support with long-term Gattex therapy. The ability to reduce or even completely eliminate the need for parenteral support could meaningfully impact the lives of patients with this rare and debilitating condition.” The primary objective of STEPS 2 was to study long-term safety and durability of effect in patients who completed, participated in, or qualified for participation in STEPS, a 24-week, placebo-controlled, multi-center, international Phase 3 study of Gattex in adult subjects with SBS. STEPS 2 enrolled a total of 88 patients in the following three treatment groups: * Gattex/Gattex group: 37 subjects who completed 6 months of Gattex therapy in the STEPS study * Placebo/Gattex group: 39 subjects who received placebo for 6 months in STEPS * Not-treated/Gattex group: 12 subjects who successfully completed the optimization and stabilization phase of STEPS and were enrolled directly in STEPS 2 after STEPS was fully randomized Sixty-five of the 88 patients enrolled in STEPS 2 (74 percent) completed the study. Seven of the 37 patients in the Gattex/Gattex group withdrew from the study, four of whom were due to adverse events. Sixteen of the 51 patients in the Placebo/Gattex and the Not-treated/Gattex groups withdrew from the study, 12 of whom were due to adverse events. The majority of patients in STEPS 2 achieved and maintained clinically meaningful responses after Gattex treatment. A responder was defined as a subject who achieved a 20 to 100 percent reduction in the volume of parenteral support from baseline. A higher percentage of patients who received Gattex for 30 months (the Gattex/Gattex group) responded and achieved greater reductions in parenteral support volume and days per week compared with those who received Gattex for 24 months (Placebo/Gattex group and Not-treated/Gattex group). In addition, 33 percent of patients (10/30) who received 30 months of Gattex achieved complete independence from parenteral support.   Key efficacy data are summarized below for the patients who completed STEPS 2:                   Gattex/Gattex   Placebo/Gattex   Not- treated/Gattex Number of patients who achieved complete   10/30 (33%)   2/29 (7%)   1/6 (17%) independence from parenteral support Responder rate (20-100 percent reduction in   28/30 (93%)   16/29 (55%)   4/6 (67%) parenteral support volume from baseline) Average percentage reduction in parenteral   66%   28%   39% support volume from baseline Number of patients who reduced infusion days   21/30 (70%)   14/29 (48%)   3/6 (50%) by at least one day per week Number of patients who reduced infusion days   18/30 (60%)   5/29 (17%)   2/6 (33%) by at least three days per week       The rates of adverse events of special interest as described in the U.S. Prescribing Information remain consistent. The most common adverse events were abdominal pain, catheter sepsis, and episodes of decreased weight, nausea, gastrointestinal stoma complications, and abdominal distension. NPS has submitted a Supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for Gattex to ^ revise the product label to include long-term data from STEPS 2.