On January 21, 2020 Rafael Pharmaceuticals, Inc. ("Rafael" or the "Company"), a leader in the growing field of cancer metabolism-based therapeutics, reported its collaboration with Michigan Medicine to proceed with a Phase 1b/2 clinical trial of CPI-613 (devimistat) in combination with gemcitabine and cisplatin for patients with biliary tract cancer (Press release, Rafael Pharmaceuticals, JAN 21, 2020, View Source [SID1234553562]). The study will be a multicenter randomized trial of gemcitabine and cisplatin with or without devimistat as a first-line therapy for patients with locally advanced, unresectable or metastatic biliary tract cancer who have had no prior treatment.

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The goal of the Phase 1b part of the trial will be to identify the maximum tolerated dose (MTD), or the highest dose of devimistat that does not cause side effects, when given in combination with gemcitabine and cisplatin. This will determine the recommended dose for the randomized Phase 2 part of the trial. The goal of Phase 2 will be to then determine the efficacy of devimistat in combination with gemcitabine and cisplatin as compared with the combination chemotherapy alone. An estimated 68 to 78 patients will be enrolled in the study.

"Biliary tract cancer is a rare and aggressive cancer that affects approximately 15,000 people in the United States each year," said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. "Launching this trial aligns with our mission to help patients with significant unmet medical needs."

Vaibhav Sahai, MBBS, MS., Associate Professor of Medical Oncology at Michigan Medicine, is the Principal Investigator on the study.

"Devimistat has demonstrated promising results in pancreatic cancer," said Dr. Sahai. "Rare cancers require dedicated and innovative research, and I am hopeful that we will advance treatment options for patients with biliary tract cancer."

About CPI-613 (devimistat)

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.