In the STEPSTONE open-label study, ozanimod
demonstrated meaningful clinical and endoscopic improvements in
patients with moderately to severely active Crohn’s disease at week
12
In the TOUCHSTONE open-label extension study,
ozanimod continued to demonstrate clinically meaningful results in
moderately to severely active ulcerative colitis across multiple
measures of disease activity through week 92
The safety and tolerability profile of ozanimod
in both studies was generally consistent with previously reported
studies of ozanimod
Celgene Corporation (NASDAQ:CELG) today announced that
phase 2 data of investigational compound ozanimod in Crohn’s
disease and ulcerative colitis will be presented at the World
Congress of Gastroenterology at ACG2017 (WCOG at ACG2017)
in Orlando. The studies include new data from the STEPSTONE
study in patients with moderately to severely active Crohn’s
disease (CD) and long-term data from the open-label extension of
the TOUCHSTONE study in patients with moderately to severely active
ulcerative colitis (UC). Ozanimod is an investigational, selective
sphingosine 1-phosphate (S1P) 1 and 5 receptor modulator.
“The new STEPSTONE findings further validate our hypothesis of
ozanimod in Crohn’s disease and the latest data from the TOUCHSTONE
study continue to support the potential of ozanimod to provide
continued efficacy in ulcerative colitis at 92 weeks,” said Terrie
Curran, President, Celgene Inflammation and Immunology. “The
results in both studies are highly encouraging as we execute
pivotal studies of ozanimod in Crohn’s disease and ulcerative
colitis in hopes of advancing oral treatment options for these
patients.”
The STEPSTONE data presented at WCOG at ACG2017 will include the
first 12-week data from the phase 2, open-label study. STEPSTONE
evaluated endoscopic improvement and clinical outcomes following
treatment with ozanimod 1.0 mg daily for 12 weeks in 69 patients
with a mean age of 37.7 years with moderately to severely active
CD. Patients with available data at baseline and week 12 were
included in the analyses of the simple endoscopic score for Crohn’s
disease (SES-CD) matched intestinal segments (n=60) and the
analysis of Crohn’s disease activity index (CDAI) (n=59).
SES-CD score reductions from baseline of at least 50 percent and
25 percent were seen in 27 percent and 43 percent of patients,
respectively. Patients who had shorter disease duration (disease
duration ≤5 years) had a greater endoscopic response: 50 percent
and 25 percent reductions were seen in 36 percent and 50 percent of
patients, respectively. Patients who had lower baseline endoscopic
disease activity (SES-CD ≤12) also had a greater endoscopic
response: 50 percent and 25 percent reductions were seen in 30
percent and 49 percent of patients, respectively.
Reductions in mean CDAI scores from baseline were seen at week 4
(first post-baseline assessment), with further reductions through
week 12. At week 12, a CDAI response (CDAI decrease ≥100) was
observed in 66 percent of patients, and CDAI remission (CDAI
<150) was observed in 46 percent of patients. The mean CDAI
reduction at week 12 was 130 points.
The most frequently reported adverse events (AEs) and serious
AEs in patients appeared to be related to underlying CD. The most
common AEs (≥5 percent) were CD flare, abdominal pain, nausea,
lymphopenia, arthralgia, vomiting, increased gamma-glutamyl
transpeptidase (GGT), urinary tract infection, paresthesia, anal
abscess and increased alanine aminotransferase (ALT). The most
common SAEs in two or more patients were CD flare, fistulizing
disease, intestinal obstruction and abdominal abscess.
“Improvements in endoscopy are thought to correlate with
long-term benefits for patients with Crohn’s disease, a chronic
condition in which patients are regularly seeking out additional
options to manage their disease,” said Brian G. Feagan, M.D.,
Robarts Clinical Trials and the University of Western Ontario in
Canada. “The endoscopic, clinical improvement and the safety
profile of ozanimod reported in the STEPSTONE study are encouraging
and support its further development as a potential oral option for
patients with Crohn’s disease.”
A separate presentation at WCOG at ACG2017 will report 92-week
data on subjects who participated in the TOUCHSTONE open-label
extension. TOUCHSTONE evaluated the efficacy and safety of 0.5 mg
and 1.0 mg doses of ozanimod compared with placebo after eight
weeks of treatment (induction phase) in 197 patients with moderate
to severe UC. The primary endpoint was the proportion of patients
in remission at week 8. Patients who achieved a clinical response
at week 8 continued with their original treatment through week 32
in a maintenance phase. Previously reported results from TOUCHSTONE
demonstrated statistically significant improvements in both the
primary and secondary endpoints in patients who received 1.0 mg
dose of ozanimod versus placebo.
TOUCHSTONE participants in all three treatment arms entered the
ongoing open-label extension if they did not respond to treatment
after the induction phase, relapsed during the maintenance phase or
completed the maintenance phase (170 of the 197 patients). The
objective of the extension phase was to evaluate the long-term
efficacy and safety of daily ozanimod 1.0 mg. Efficacy data are
reported as observed through week 92 and safety data includes all
events through the data cut-off of March 2017.
At week 92, of the 100 patients who underwent efficacy
evaluations, 91 percent had little or no active disease based on
the physician global assessment (PGA 0 or 1), 97 percent had little
or no blood in their stools (rectal bleeding subscore [RBS] 0 or 1)
and 86 percent had no blood in the stools (RBS 0).
The safety profile at week 92 was similar to that reported in
the placebo-controlled portion of the study. The most common AEs
were UC flare, anemia, upper respiratory tract infection and back
pain. The only SAEs in two or more patients were anemia and UC
flare.
“People with ulcerative colitis need additional treatment
options that can provide long-term benefit,” said William Sandborn,
M.D., Professor of Medicine and Chief, Division of
Gastroenterology and Director, University of California
San Diego Inflammatory Bowel Disease Center. “These results further
support the potential of longer-term treatment with ozanimod.”
About STEPSTONE
STEPSTONE is a phase 2, open-label study in patients with
moderately to severely active Crohn’s disease (CD). Data from the
first 12 weeks examined endoscopic and clinical outcomes following
treatment with ozanimod 1.0 mg daily for 12 weeks. Active Crohn’s
disease was defined as CDAI score 220-450 and total SES-CD of 6 or
greater (or in isolated ileum disease SES-CD of 4 or greater). For
the 69 enrolled patients, baseline mean age was 37.7 years, mean
SES-CD was 13.3 and mean CDAI was 320. Mean CD duration was 10
years, with 54 percent of patients having had prior exposure to
biologic therapy. All endoscopic assessments were read in a blinded
manner by an imaging core lab. Daily electronic diary records were
used to collect CD symptoms (including abdominal pain and
soft/loose stool frequency). SES-CD was evaluated at baseline and
week 12, and CDAI was assessed at baseline and weeks 4, 8 and
12.
About TOUCHSTONE
TOUCHSTONE is a phase 2, randomized, double-blind,
placebo-controlled trial comparing the efficacy and safety of
ozanimod (also known as RPC1063) with placebo in patients with
moderately to severely active ulcerative colitis. A total of 197
patients were randomized and treated once daily with 1.0 mg
ozanimod (n=67), 0.5 mg ozanimod (n=65) or placebo (n=65) for eight
weeks (the induction phase). The primary endpoint was the
proportion of patients in remission (Mayo score ≤2, no subscore
> 1) at week 8. Secondary endpoints were the proportion of
patients achieving clinical response (reduction in Mayo score of ≥3
and ≥30 percent with a decrease in the rectal bleeding score of ≥1
or a rectal bleeding score ≤1), proportion of patients with mucosal
improvement (endoscopy score ≤1) and the change in Mayo score.
Safety assessments included ECG testing, pulmonary function
testing, optical coherence tomography and adverse events.
For the maintenance phase, patients who achieved a clinical
response at week 8 continued with their original treatment through
week 32. In the open-label extension phase, all patients (n=170)
were treated with ozanimod 1.0 mg. The week 44 visit was completed
by 131 patients.
About Ozanimod
Ozanimod is a novel, oral, selective, sphingosine 1-phosphate 1
and 5 receptor modulator in development for immune-inflammatory
indications including relapsing multiple sclerosis, ulcerative
colitis and Crohn's disease. Selective binding with S1PR1 receptors
is believed to inhibit a specific subset of activated lymphocytes
from migrating to sites of inflammation. The result is a reduction
of circulating lymphocyte count that leads to anti-inflammatory
activity. Importantly, immune surveillance is maintained.
Ozanimod 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 Ulcerative Colitis
Ulcerative colitis is a chronic, relapsing condition triggered
by an abnormal, prolonged immune response that creates long-lasting
inflammation and ulcers (sores) in the mucosa (lining) of the large
intestine (colon). Symptoms usually develop over time, rather than
suddenly. The disease can be debilitating and can sometimes lead to
life-threatening complications. Ulcerative colitis is the most
common form of inflammatory bowel disease worldwide. About one in
every 198 people in Europe, and one in every 402 people
in North America, have ulcerative colitis. In 2004, 2.1
million prescriptions were written to treat ulcerative colitis, and
716,000 ambulatory care visits were related to the disease. In
2010, there were 107,000 hospitalizations due to ulcerative
colitis.
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.
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