Data show endoscopic improvement and clinical
response and remission at week 12
Safety and tolerability consistent with
previous studies
Results expected to be presented at upcoming
medical meetings
Celgene Corporation (NASDAQ:CELG) today announced interim
topline data from a randomized, double-blind, multicenter,
exploratory phase 1b study evaluating the effects of oral GED-0301
(mongersen) on both endoscopic and clinical outcomes in patients
with active Crohn’s disease.
The trial, CD-001, is an ongoing study evaluating three
different treatment regimens of GED-0301 in a 12-week treatment
phase, followed by an observation phase up to 52 weeks (off
treatment). The primary objective of the study is to explore the
effect of GED-0301 on endoscopic outcomes. The trial enrolled a
total of 63 patients across multiple countries.
The study was designed to further enhance the understanding of
GED-0301 activity in a difficult-to-treat, moderate-to-severe
patient population. This population was more diverse than prior
GED-0301 studies and included patients with endoscopically
confirmed mucosal damage at entry and those who had previous
surgeries. The study also included both biologic exposed and
biologic naïve patients as well as patients with a diagnosis of
Ileitis, Ileocolitis or colitis.
Topline data from CD-001 show that in a proportion of patients
treated with oral GED-0301 there was endoscopic improvement
(defined as a 25 percent improvement from baseline) and clinical
response and remission across all treatment groups at week 12.
Findings to date reveal no new safety signals and tolerability is
consistent with earlier studies.
“Given the high unmet need in Crohn’s disease, we are pleased
that oral GED-0301 showed both endoscopic improvements and
clinically meaningful responses and remission at an early timepoint
in this study,” said Scott Smith, President of Celgene Inflammation
and Immunology. “These data are particularly encouraging for
several reasons, including the difficult-to-treat patient
population evaluated in the trial.”
“At this early 12-week timepoint, we’re looking at the
proportion of patients who had a 25 percent or greater endoscopic
improvement, suggesting mucosal healing is underway in these
patients,” said Dr. William Sandborn, M.D., Professor of Medicine
and Chief, Division of Gastroenterology and Director, University of
California San Diego Inflammatory Bowel Disease Center. “These data
support the notion that GED-0301, a potential first-in-class oral
antisense therapy, may target an underlying cause of Crohn’s
disease, rather than simply improving symptoms.”
Full data from the 12-week timepoint have been submitted for
presentation at an upcoming scientific meeting later this year. The
CD-001 study is ongoing until all patients complete the observation
phase. Data from the observation portion of the trial are expected
in 2017. The Phase III registration program is ongoing.
About CD-001
CD-001 is a phase 1b randomized, double-blind, multicenter,
exploratory study evaluating the effects of oral GED-0301 on
endoscopic and clinical outcomes in patients with active Crohn’s
disease. A total of 63 patients were randomized in a 1:1:1 ratio to
receive one of three treatment regimens in a 12-week treatment
phase: GED-0301 160 mg once daily for 12 weeks; GED-0301 160 mg
once daily for eight weeks followed by four weeks of placebo; or
GED-0301 160 mg once daily for four weeks followed by eight weeks
of placebo. This treatment phase was followed by an off-treatment
observation phase for up to 52 weeks. Eligible patients can also
enter an extension phase (on treatment) for an additional 24
weeks.
About GED-0301
The investigational oral antisense therapy GED-0301 is an
oligonucleotide that is designed to target the messenger RNA (mRNA)
for Smad7, thereby reducing Smad7 protein levels. In patients with
Crohn’s disease, abnormally high levels of Smad7 interfere with
TGF-β1 anti-inflammatory pathways in the gut, leading to increased
inflammation. GED-0301 is designed to act locally and is thought to
reduce Smad7 levels with negligible systemic exposure.
GED-0301 (mongersen) is an investigational compound that is not
approved for any use in any country.
About Crohn’s Disease
Crohn’s disease is an immune-mediated, chronic inflammatory
condition of the gastrointestinal tract. Estimated to affect as
many as three out of every 1,000 people in Europe and North
America, the disease is becoming more common for all ethnic groups.
Symptoms of Crohn’s disease — including abdominal pain, diarrhea,
fatigue, fever, weight loss and malnutrition — most commonly begin
to appear between the ages of 13 and 30, although the disease can
strike at any age. The disease may affect any part of the GI tract,
from the mouth to the anus, but most commonly affects the end of
the small bowel (the ileum) and the beginning of the colon. The
exact cause of Crohn’s disease is unknown, and there is no cure.
People with Crohn’s disease have a slightly reduced life
expectancy.
About Celgene
Celgene Corporation, headquartered in Summit, New Jersey, is an
integrated global biopharmaceutical company engaged primarily in
the discovery, development and commercialization of innovative
therapies for the treatment of cancer and inflammatory diseases
through next-generation solutions in protein homeostasis,
immuno-oncology, epigenetics, immunology and neuro-inflammation.
For more information, please visit www.celgene.com. Follow
Celgene on Social Media: @Celgene, Pinterest,
LinkedIn, Facebook and YouTube.
Forward-Looking Statements
This press release contains forward-looking statements, which
are generally statements that are not historical facts.
Forward-looking statements can be identified by the words
"expects," "anticipates," "believes," "intends," "estimates,"
"plans," "will," “outlook” and similar expressions. Forward-looking
statements are based on management’s current plans, estimates,
assumptions and projections, and speak only as of the date they are
made. We undertake no obligation to update any forward-looking
statement in light of new information or future events, except as
otherwise required by law. Forward-looking statements involve
inherent risks and uncertainties, most of which are difficult to
predict and are generally beyond our control. Actual results or
outcomes may differ materially from those implied by the
forward-looking statements as a result of the impact of a number of
factors, many of which are discussed in more detail in our Annual
Report on Form 10-K and our other reports filed with the Securities
and Exchange Commission.
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Celgene CorporationInvestors:Patrick E. Flanigan III,
908-673-9969Corporate Vice President, Investor
RelationsorMedia:Catherine Cantone, 908-897-4256Senior Director,
Corporate Communications
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