Rafael’s proprietary Altered Metabolism Directed (AMD) platform disrupts biochemical alterations in the conversion of glucose to energy that occur in many types of cancer cells. These essential “bioenergetic” differences are linked to pathways that control, among other things, cancer cell growth and development, as well as various forms of cell death, including apoptosis (programmed cell death) and necrosis. The platform is designed to produce drugs, such as the company’s lead drug CPI-613® (devimistat), that catastrophically disrupt energy-production pathways specific to cancer cells.
Rafael Pharmaceuticals has evaluated the activity of its AMD compounds against a wide array of established cancer cell lines in cell culture, including those representing some of the most difficult to treat cancer types as well as cancer cell lines characterized as multiple drug resistant. In all cases, the tested cell lines were 100% killed by the AMD compounds within the same AMD compound concentration range. Cells derived from non cancerous tissues were also tested and were apparently unaffected by the AMD compounds even at concentrations that are several fold higher than that at which cancer cells from the same tissue of origin were killed.
Some salient characteristics of AMD compounds observed in preclinical setting:
- A high degree of cancer cell selectivity.
- Broad spectrum activity against a wide variety of cancer cell lines representing different genetic mutations, including cancer cell lines characterized as being multiple drug resistant.
- A high degree of tolerability in animal models resulting in an unusually significant therapeutic index.