CPI-613® (devimistat)

Devimistat is designed to selectively target cancer cell mitochondria to treat some of the most intractable forms of cancer.

Cornerstone’s lead compound, CPI-613® (devimistat), is designed to target the mitochondria of cancer cells in order to disrupt their energy production, cutting off the fuel for disease growth.

Cancer progresses because it utilizes the mitochondria – the energy machinery of the cells – to generate the building blocks of the next tumor in order to fuel cancer growth and proliferation.

Because it is designed to be highly specific, minimally toxic and effective against a wide variety of cancers, devimistat can be used for recurrent and hard-to-treat cancers, including on a chronic basis; it may also enhance the sensitivity of cancer cells to other treatment modalities, including chemotherapy.

The pioneering discovery in the middle of the 20th century that metabolic activity in tumor tissues is different from that of normal tissues generated significant interest in developing cancer therapies that alter metabolic pathways as a means of limiting the supply of crucial nutrients in cancer cells.

Though considered by many to be a highly promising approach, progress in cancer metabolism-based therapeutics has been slow due to the complex nature of these pathways; in recent years, however, progress in the understanding of cancer metabolism has led to a renaissance of interest in this area.

Devimistat is undergoing multiple clinical trials and has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the following indications: Burkitt’s lymphoma, biliary tract cancer, soft tissue sarcoma, myelodysplastic syndrome (MDS) and peripheral T-cell lymphoma. In addition, the European Medicines Agency (EMA) has granted orphan drug designation to devimistat for Burkitt’s lymphoma.