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.
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