Rafael Pharmaceuticals, Inc. announced the ongoing successful enrollment rate of its Phase 2 clinical trial for CPI-613® (devimistat) in combination with gemcitabine and cisplatin in patients with biliary tract cancer. The multicenter trial is for patients with locally advanced unresectable or metastatic biliary tract cancer who have had no prior treatment. CPI-613® (devimistat) is a first-in-class clinical lead compound of Rafael, which targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells.

Devimistat is designed to target the mitochondrial tricarboxylic acid (TCA) cycle, a process essential to tumor cell multiplication and survival, selectively in cancer cells. Devimistat substantially increases cellular stress and the sensitivity of cancer cells to a diverse range of chemotherapeutic agents. This synergy allows for potential combinations of devimistat with lower doses of these generally toxic drugs to be more effective with lower patient side effects.

Combination with devimistat represents a diverse range of opportunities to substantially improve patients' benefit in many different cancers. The U.S. Food and Drug Administration (FDA) has designated devimistat as an orphan drug for the treatment of pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, peripheral T-cell lymphoma, soft tissue sarcoma, Burkitt's lymphoma and biliary tract cancer. The European Medicines Agency (EMA) has granted orphan drug designation to devimistat for pancreatic cancer, acute myeloid leukemia and Burkitt's lymphoma.