Alicaforsen
Alicaforsen is an antisense oligonucleotide targeted to down regulate the production of ICAM-1, a cell surface receptor which is involved in the process of inflammation.
In inflammatory bowel disease (IBD), the immune system incorrectly activates inflammatory processes. Part of this activation involves increasing the levels of gene messengers (mRNA) for the production of ICAM-1; there is a correlation between the levels of ICAM-1 and the severity of inflammation.
ICAM-1 facilitates the transport of white cells from the circulation to the site of inflammation, thereby propagating the inflammatory condition. When alicaforsen binds to ICAM-1 mRNA, ICAM-1 expression is reduced. In clinical trials this has been shown to reduce levels of inflammation, resulting in an improvement of clinical signs and symptoms (e.g. reduced stool frequency), and an improvement in the health of the underlying tissue (recorded by endoscopy).
The Company’s experienced Leadership Team is working to develop and deepen the pipeline for alicaforsen with treatments for ulcerative colitis, Crohn’s disease and other inflammatory diseases.
Alicaforsen enema formulation is appropriate for treating distal ulcerative colitis (disease which has not spread beyond the descending colon, adjacent to the rectum). We are in the process of preparing a pivotal Phase 3 trial for this formulation in ulcerative colitis, having discussed the protocol with the U.S. Food and Drug Administration and European Medicines Agency.
We are also progressing oral tablet formulations of the drug for the treatment of Crohn’s disease and ulcerative colitis. These are both medical conditions where there are limited treatment options.
