Clinical improvement observed early, with
highest clinical response and remission rates in the 12-week
treatment group
Endoscopic improvement seen across all
treatment groups at 12 weeks
Safety and tolerability were consistent with
previous studies
Celgene International Sàrl, a wholly-owned subsidiary
of Celgene Corporation (NASDAQ: CELG), today announced
that data from a randomized, double-blind, multicenter, exploratory
phase 1b study evaluating the effects of investigational oral
GED-0301 (mongersen) 160 mg on both endoscopic response and
clinical remission in patients with active Crohn’s disease will be
presented in Vienna, Austria at the United European
Gastroenterology Week (UEGW).
Patients with active Crohn’s disease [Crohn’s disease activity
index (CDAI) score 220-450], a total simple endoscopic score for
Crohn’s disease (SES-CD) ≥7, or an ileal disease SES-CD ≥4, were
randomized to three different active treatment regimens of four,
eight or 12 weeks of GED-0301 160 mg daily, followed by an
observation period off treatment. Endoscopic and clinical
assessments were reported through week 12. A total of 63 patients
were enrolled in the study.
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.
Clinical improvement was seen by week 2, and clinical response
(CDAI decrease ≥100) and remission (CDAI <150) rates were
highest in the 12-week treatment group at 67 and 48 percent
respectively, at week 12. The mean CDAI reduction from baseline at
week 12 in the 12-week treatment group was 133 points. Of the
patients with evaluable endoscopies at week 12 (n=52), 37 percent
had an endoscopic response (≥25 percent reduction in SES-CD score
from baseline), with no meaningful difference across treatment
groups. In addition, of those patients with greater endoscopic
disease activity at baseline (SES-CD score of >12; n=16), 63
percent exhibited a reduction ≥25 percent in SES-CD score and 31
percent had a reduction of ≥50 percent.
The rates of adverse events and serious adverse events were low
and similar across treatment groups. There were no new safety
signals for oral GED-0301 160 mg daily reported in this study.
“A significant number of Crohn’s disease patients don’t reach
remission with current therapies or will suffer relapses over time
and are in need of new treatment options,” said Brian Feagan, MD,
Director of Robarts Clinical Trials at Robarts Research Institute,
Western University, London, Ontario, Canada. “Based on these
findings, oral GED-0301 has the potential to provide a new, oral
option with a novel mechanism of action designed to act
locally.”
“We are encouraged that oral GED-0301 showed both meaningful
endoscopic improvement and clinical remission at an early time
point in this study," said Scott Smith, President, Celgene
Inflammation & Immunology. "The fact that this study included
nearly 50 percent biologic-experienced patients further reflects
the potential of GED-0301 as a novel approach for patients with
Crohn’s disease searching for alternatives."
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 active 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 100 weeks.
About GED-0301
The investigational oral antisense therapy GED-0301 is an
oligonucleotide 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 to reduce Smad7
levels with negligible systemic exposure.
GED-0301 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 International Sàrl, located in Boudry, Switzerland, is a
wholly owned subsidiary and international headquarters of Celgene
Corporation. Celgene Corporation, headquartered in Summit, New
Jersey, is an integrated global pharmaceutical company engaged
primarily in the discovery, development and commercialization of
innovative therapies for the treatment of cancer and inflammatory
diseases through gene and protein regulation. 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. Celgene undertakes 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 Celgene’s
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 Celgene’s Annual Report on Form 10-K and our other
reports filed with the U.S. Securities and Exchange Commission.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20161016005052/en/
CelgeneInvestors:Patrick E. Flanigan III, 908-673-9969Corporate
Vice President, Investor RelationsorMedia:Catherine Cantone,
732-564-3592Senior Director, Corporate Communications
Celgene (NASDAQ:CELG)
Historical Stock Chart
From Aug 2024 to Sep 2024
Celgene (NASDAQ:CELG)
Historical Stock Chart
From Sep 2023 to Sep 2024