Rafael Expands CPI-613 Trial

Rafael Pharmaceuticals announced the expansion of its Phase 2 clinical trial of CPI-613® (devimistat) for patients with relapsed or refractory Burkitt’s lymphoma/leukemia. The clinical trial will begin enrolling patients at Massachusetts General Hospital in Boston starting this month. The trial is currently underway at Memorial Sloan Kettering Cancer Center in New York City and at City of Hope in Duarte, California.

"Burkitt’s lymphoma is a rare disease, with approximately 1,200 people in the United States diagnosed annually, so treatment options for these patients are very limited," said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. "The disease is highly aggressive and has a high rate of relapse. Expanding our clinical trial will increase access and create hope for patients and their loved ones across the country."

In June 2018, devimistat received orphan drug designation for the treatment of relapsed Burkitt’s lymphoma from the U.S. Food and Drug Administration (FDA). Orphan drug designation refers to a special status granted by The Orphan Drug Act (ODA) to a drug used to treat a rare disease or condition.

"Currently no definitive second-line therapy exists for patients with relapsed Burkitt’s lymphoma," said Ariela Noy, M.D., an oncologist at Memorial Sloan Kettering Cancer Center and principal investigator on the devimistat clinical trial for relapsed Burkitt’s lymphoma. "The expansion of the clinical trial across the country will help reach patients that have a clear unmet medical need for additional treatment options."

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 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’s side effects. Combination with devimistat represent a diverse range of opportunities to substantially improve patient’s benefit in many different cancers. The U.S. Food and Drug Administration (FDA) has given Rafael approval to initiate pivotal Phase 3 clinical trials in pancreatic cancer (AVENGER 500) and acute myeloid leukemia (ARMADA 2000), and has designated devimistat as an orphan drug for the treatment of pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, peripheral T-cell lymphoma and Burkitt’s lymphoma. The EMA has granted orphan drug designation to devimistat for pancreatic cancer and acute myeloid leukemia.

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