Cequent Pharmaceuticals said last week that it has filed an investigational new drug application to begin human trials of CEQ508, the company’s orally delivered, RNAi-based treatment for advanced familial adenomatous polyposis.
FAP is an inherited, colorectal cancer syndrome characterized by the growth of colorectal polyps. Though the polyps are initially benign, they become malignant in nearly all cases in the absence of colectomy, according to the company.
CEQ508, which is based on Cequent’s transkingdom RNAi technology, is designed to inhibit the oncogene beta-catenin, which is expected to prevent new polyp formation and possibly slow the progression to malignancy of existing ones.
Cequent said it expects to begin dosing patients in the study, which will take place at the Fred Hutchinson Cancer Research Center in Seattle, during the first quarter of 2010.
In the company’s proposed phase I trial, CEQ508 will be administered in escalating doses to a total of 18 adult FAP patients. “A key readout and secondary objective of the proposed trial includes analysis of … beta-catenin expression changes in the gastrointestinal tract of patients determined from biopsy samples taken prior to taking the drug, and following a daily, 28-day dosing regimen,” Cequent said.