Celgene Corporation has reported positive interim results from a Phase 1b clinical trial assessing the effects of oral GED-0301 (mongersen) on endoscopic and clinical outcomes in patients with active Crohn’s disease.
The trial aims to provide further data of GED-0301 activity in a difficult-to-treat, moderate-to-severe and very diverse patient population, including patients with endoscopically confirmed mucosal damage at entry, those who had previous surgeries, those who have taken biologics or are biologic naïve, and patients with a diagnosis of ileitis, ileocolitis or colitis.
Titled “A Randomized, Double-blind, Study to Explore the Effect of GED-0301 in Subjects With Active Crohn’s Disease” (NCT02367183), it is evaluating GED-0301 in 63 patients assigned to one of three different treatment regimens: 160 mg GED-0301 once daily for 12 weeks; 160 mg once daily for eight weeks, then four weeks of placebo; and 160 mg once daily for four weeks, followed by eight weeks on placebo.
Topline interim data at week 12 found that a proportion of treated patients showed endoscopic improvements (defined as 25 percent improvement from baseline). Clinical response and remission was also seen across all treatment groups, with a tolerability consistent with previous studies, and no new safety signals.
“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,” Scott Smith, president of Celgene Inflammation and Immunology, said in a press release. “These data are particularly encouraging for several reasons, including the difficult-to-treat patient population evaluated in the trial.”
After the 12 weeks, all patients will move to an off-treatment observation phase of up to 52 weeks, after which a 24-week on-treatment extension phase is possible.
“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, MD, a professor of Medicine and Chief, Division of Gastroenterology, and director of the 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.”
GED-0301 takes an unusual approach to treating Crohn’s, using antisense technology to target a key intracellular signaling protein thought to be involved in intestinal inflammation and the pathogenesis of the disease. It is orally administered and designed to act locally.
Fuller 12-week results will be presented at a future scientific conference later in the year, Celgene said in the release. The trial will continue until all patients complete the observation phase, with observation period results expected for next year. Preparations are underway for Phase 3 study.