Exelixis Inc has initiated the Phase 1 first-in-man safety trial for its proprietary small molecule anticancer compound XL784. The Phase 1 trial will be conducted at a single center and is expected to enroll approximately 70 healthy subjects in a dose-escalating study intended to explore the safety, pharmacokinetic and pharmacodynamic merits of the compound following oral administration. The trial should be completed within a year. Concurrent with the Phase 1 trial, Exelixis plans to explore the therapeutic utility of the compound in various animal models of disease outside of cancer, including renal and cardiovascular disease.
"XL784 is of significant interest to us because of its preclinical safety profile and activity, and its potential utility and commercial value in treating cancer and other diseases," said George A. Scangos, president and chief executive officer.
The target against which XL784 is directed is a cell surface protease involved in cleavage of growth factors that promote cell growth and differentiation. The target was originally discovered in Exelixis' anti-angiogenesis research program, and shows both anti-angiogenic and anti-proliferative effects. The compound appears appropriate for testing in healthy volunteers and to date has not shown the toxicities associated with traditional anticancer compounds that act through a cytotoxic mechanism. A third-party manufacturer is supplying the drug product to be used in the clinical trial.