Early data from a new drug to treat Crohn's disease is impressive and explains why Celgene purchased the drug from a small Irish biotech company last spring.  The oral pill, GED-031 (also known as mongersen) has the potential to become a preferred Crohn's therapy potentially replacing injectable drugs. The results of a recently completed study were presented at the United European Gastroenterology Week in Vienna, Austria,

Inflammatory Bowel Disease (IBD) is an immune-mediated chronic inflammation of the gastrointestinal tract.  There are two types of IBD, Crohn’s disease and ulcerative colitis; both manifest as chronic immune-mediated inflammation of the gastrointestinal system. While they both cause similar symptoms, they are managed differently. Crohn’s disease may affect any part of the gastrointestinal system, from the mouth to the anus. Ulcerative colitis, however, is limited to the colon, otherwise known as the large intestine.  It is estimated that 1.4 million Americans have IBD, which tends to run in families and affects males and females equally.

Normally, the cells and proteins that make up the immune system protect individuals from infection. In people with IBD, however, the immune system mistakes food, bacteria, and other materials in the intestine for foreign or invading substances. When this happens, the body sends white blood cells into the lining of the intestines, where they produce chronic inflammation and ulcerations—called an autoimmune response.

The results of a recent phase II study evaluating 160 patients with active Crohn's disease, with a high dose of mongersen were recently published. The study demonstrated a statistically significant remission rate of 65% compared to 9.5% for a placebo, measured after two weeks of treatment. Remission was defined as a Crohn's Disease Activity Index (CDAI) score of less than 150, maintained for at least two weeks. The response rate for the high dose of mongersen (CDAI reduction of at least 100 points after 28 days) was 72% compared to 17% for placebo, also statistically significant.

Mongersen, targets the messenger RNA for Smad7, abnormally high levels of which lead to increased inflammation. Celgene says the distinct formulation of the tablet is designed to release mongersen into the far end of the small intestine and near the end of the colon, where it reduces Smad7 levels.  It is reported that Celgene is planning to start a broad phase 3 program to be implemented by the end of 2014 to confirm this encouraging data.

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