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
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.
"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
"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.
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.
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.
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
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Patrick E. Flanigan III, 908-673-9969
Corporate Vice President, Investor Relations
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