CEQ508 and Familial Adenomatous Polyposis (FAP)
CEQ508 is the first drug candidate in a novel class of therapeutic agents employing our Transkingdom RNA™ interference (tkRNAi) platform. CEQ508 is being developed for the treatment of Familial Adenomatous Polyposis (FAP), a hereditary condition that occurs in approximately 1:10,000 persons worldwide. FAP is caused by mutations in the Adenomatous Polyposis Coli (APC) gene. As a result of these mutations, epithelial cells lining the intestinal tract have increased levels of the protein β-catenin, which in turn, results in uncontrolled cell growth. Proliferation of the epithelial cells results in the formation of numerous (hundreds to thousands) non-cancerous growths (polyps) throughout the large intestine. By age 35, 95% of individuals with FAP have developed polyps and most will experience adverse effects including increased risk of bleeding and the potential for anemia. In more severe cases, obstruction of the intestines, abdominal pain, and severe bouts of diarrhea or constipation can occur. FAP patients are also at an increased risk of various cancers, the most concerning of which is a nearly 100% occurrence of colon cancer if measures are not taken to prevent the formation of polyps.
CEQ508 is being developed as an orally administered treatment to specifically target and reduce the levels of β-catenin in the epithelial cells of the small and large intestine, and is expected to represent a significant breakthrough in treatment of FAP. Currently the only approved pharmaceutical therapy for the treatment of FAP is nonsteroidal anti-inflammatory drug in combination with standard of care practices. For many patients, complete colectomy (surgical removal of the entire large intestine), which is usually performed in the late teenage years or early twenties, remains as the primary treatment. However, surgical intervention is not curative as the risk of polyps forming in the remaining portions of the intestinal tract and in the small intestine remains after colectomy.