Antisense Technology Shows Promising Results in Crohn's Disease Treatment


Significant number of patients taking once daily oral therapy achieve clinical remission during a recent trial.

Significant number of patients taking once daily oral therapy achieve clinical remission during a recent trial.

An experimental treatment utilizing a new approach to Crohn’s disease achieved promising results in a recent clinical trial.

Published in the March 19 issue of The New England Journal of Medicine, the study evaluated the Celgene Corporation’s treatment GED-0301 (mongersen) in patients with active Crohn's disease. The treatment provides a targeted therapy through the utilization of antisense technology.

"GED-0301 offers a unique 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," said Professor Giovanni Monteleone of the University of Rome Tor Vergata in a press release. "This orally administered therapy is designed to act locally with its novel mechanism of action. The results from the phase 2 trial suggest that GED-0301 should be studied further in phase 3 trials for Crohn's disease."

The phase 2, double-blind placebo controlled trial enrolled 166 adult patients with moderate-to-severe Crohn's disease, defined as Crohn's Disease Activity Index (CDAI) ranging from 220 to 400 at least a week before enrollment. The patients had documented inflammatory lesions in the terminal ileum and/or right colon.

The researchers found a significantly greater proportion of patients with active Crohn's disease were able to achieve the primary endpoint of clinical remission after 15 days and 28 days with once daily GED-0301 at 40 mg (55%) and 160 mg (65%) respectively, compared with GED-0301 10 mg (12%) or placebo (10%; P < 0.001). In addition, 67% of patients achieved glucocorticoid-free remission after 84 days through once daily 160 mg GED-0301 compared with 11% of patients with placebo (P=0.04).

In patients treated with once daily GED-0301 at 160 mg, 67% achieved clinical remission on day 15, 72% achieved clinical remission at day 28, and 67% achieved clinical remission at day 84, compared with 21%, 14%, and 21%, respectively, on placebo (P < 0.0001 vs. placebo, for each time point).

Similar results were found in the once daily GED-0301 40 mg cohort, with 58% achieving clinical remission on day 15, 70% achieving clinical remission on day 28, and 63% achieving clinical remission on day 84.

In patients treated with once daily GED-0301 10 mg, clinical remission was achieved by 15% on day 15, 29% on day 28, and 29% on day 84 (P=n.s. vs. placebo).

There were at least 1 adverse event reported in the GED-0301 groups at rates of 49%, 62%, and 49% in the 10 mg, 40 mg, and 160 mg once daily cohorts, respectively, compared with 67% in the placebo cohort.

The most common adverse events reported in the GED-0301 cohorts were abdominal pain (10-12%), worsening of Crohn's disease (10-15%), urinary tract infection (5-15%), and C-reactive protein increase (5-9%).

"A significant number of Crohn's disease patients don't reach remission with current therapies and are looking for additional options," Scott Smith, president of Celgene Inflammation and Immunology, said in a press release. "GED-0301 offers a completely different mechanism of action that has the potential to transform the Crohn's treatment landscape. We are encouraged by the phase II data and are committed to bringing innovative medicine to patients with Crohn's disease, starting with advancing the phase III trial for GED-0301."

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