Asia PR News

HONOLULU, Oct. 31 /PRNewswire-AsiaNet/ –

- Study Results Presented at American College of Gastroenterology
Annual Meeting -

Sargramostim therapy was associated with improved and maintained
quality-of-life (QOL) in patients with moderately to severely active Crohn’s
disease, according to data presented at the 70th annual American College of
Gastroenterology (ACG) Meeting.

These data from N.O.V.E.L. 1, a multi-center, randomized, double-blind,
placebo-controlled Phase 2 trial, demonstrated treatment with sargramostim
resulted in significant improvements in quality-of-life from baseline to Day
57 versus placebo, as measured by the Inflammatory Bowel Disease Questionnaire
(IBDQ) and the Short Form 36 (SF-36) health surveys. Improvements in QOL
ranging from moderate to large occurred early and were maintained throughout
the treatment.

"Crohn’s disease severely impacts both physical and emotional quality-of-
life, and current treatments are often as debilitating as the disease itself,"
said Brian K. Dieckgraefe, M,D., Ph.D., co-investigator and assistant
professor of medicine, Division of Gastroenterology, Washington University
School of Medicine, St. Louis. "This study demonstrates that sargramostim
offers significant benefits to Crohn’s disease patients in terms of improving
patient-reported outcomes and facilitating a more active and productive
quality-of-life."

Evolving research suggests a primary defect in Crohn’s disease may arise
from a breakdown in intestinal barrier function composed of several layers of
cells that protect the gastrointestinal (GI) tract. This breakdown may precede
secondary inflammatory processes associated with Crohn’s disease. Sargramostim
may address a primary defect of Crohn’s disease by helping to improve immune
cell function within the intestinal barrier, unlike other therapies that
broadly suppress immune response and inflammatory symptoms.

In the United States, sargramostim is marketed as LEUKINE(R) by Berlex,
Inc., a U.S. affiliate of Schering AG, Germany.

About the Study
This study was a randomized, placebo-controlled Phase 2 trial evaluating
the efficacy and safety of sargramostim 6 µg/kg/day for 8 weeks in patients
(N=124) with moderately to severely active Crohn’s disease.

The IBDQ, the standard instrument for assessment of health-related
quality-of-life (HRQOL) in patients with inflammatory bowel diseases, and the
SF-36, a multi-purpose, short-form health survey that is often used as a
generic instrument, were used to measure physical, emotional and social
outcomes from the patients’ point-of-view.

Patients who received sargramostim had a 20% to 22% improvement from
baseline total IBDQ Day 29 and thereafter versus 7% to 13% in the placebo
group (P = 0.0059 to 0.0397). This was supported by 12% to 20% improvements in
the Physical Component Summary score from Day 29 and thereafter, 35%
improvements in the General Health subscore of the SF-36 on Day 29 and follow
up Day 30 (P = 0.0366 and 0.0133), and 71% to 73% improvements in the Vitality
subscore of the SF-36 on Day 57 and follow-up Day 30 (P = 0.0131 and 0.0121,
respectively) for patients who received sargramostim. Improvements ranging
from 24% to 34% were also observed in the Bowel and Systemic subscores of the
IBDQ on Day 29 and thereafter (P = 0.0001 to 0.0384). Bodily Pain was also
improved by 39% to 46% on Day 57 and follow-up Day 30 (P = 0.0272 and 0.0077,
respectively). The Social subscore of the IBDQ was improved by 17% on follow-
up Day 30 versus 4% in the placebo group (P = 0.0288), while the Social score
of the SF-36 was improved by 20% to 36% from Day 29 and thereafter (P = 0.0102
to 0.0471). These are considered moderate to large improvements in QOL as
indicated by the effect size that ranged from 0.45 to 1.21.
The IBDQ and the SF-36 were completed at baseline; Days 15, 29, 43, and 57
of treatment; and 30 days after treatment. Differences between groups were
analyzed by the Van Elteren Test.

About Crohn’s Disease
There are approximately 400,000-500,000 cases of diagnosed Crohn’s disease
in the United States and an estimated one million diagnosed cases worldwide.
Crohn’s disease affects men and women equally and seems to be more common
within families. About 20 percent of people with Crohn’s disease have a blood
relative, most often a brother or sister, with some form of inflammatory bowel
disease (IBD).

The most common symptoms of Crohn’s disease are abdominal pain and
diarrhea. Other symptoms include fatigue, rectal bleeding, fever, loss of
appetite and weight loss.

Crohn’s disease can be difficult to diagnose because its symptoms are
similar to other intestinal disorders such as irritable bowel syndrome (IBS)
and ulcerative colitis. Crohn’s disease causes inflammation in the GI tract
and usually occurs as an inflammation of the small bowel or colon; however, it
can affect any part of the digestive tract from the mouth to the anus. The
inflammation from Crohn’s disease can extend deeply into the lining of the
affected organ, causing pain, diarrhea and frequent bowel movements.

Contact: Marybeth Quane
212-299-8972
Marybeth.Quane@zenogroup.com

SOURCE: Dr. Brian K. Dieckgraefe

CONTACT: Marybeth Quane, +1-212-299-8972, Marybeth.Quane@zenogroup.com/