New Drug Disappoints for Pancreatic Cancer and AML Treatment


Study results showed that CPI-613 did not improve survival in patients with metastatic adenocarcinoma of the pancreas.

A new treatment method for patients with metastatic adenocarcinoma of the pancreas fell flat, as findings from a phase 3 study showed that CPI-613 (devimstat) plus the chemotherapy regimen FOLFIRINOX did not improve overall survival compared to standard of care chemotherapy.

“These cancers are incredibly difficult to treat, with few to no effective treatments available, but Rafael took the risk because we will always fight for our patients,” said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals, the manufacturer of CPI-613.

The AVENGER 500 study included 528 patients with metastatic adenocarcinoma of the pancreas who received no prior treatment. Participants received either CPI-613 plus modified FOLFIRINOX or FOLFIRINOX. There was no statistically significant difference in overall survival between the two groups, with average survival being 11.1 months and 11.7 months for the investigative and standard-of-care groups, respectively.

“While we are disappointed by the outcomes of these well-designed and well-executed studies, we remain committed to furthering our research and development in cancer metabolism for the treatment of hard-to-treat cancers, as our other studies continue,” Luther said. “I personally want to express my heartfelt appreciation to the patients, their loved ones, the researchers and principal investigators for their trust and support. I am also incredibly grateful to my team, who works tirelessly for the patients we treat.”

CPI-613 targets the mitochondrial tricarboxylic acid (TCA) cycle, which promotes the growth and multiplication cycle of cancer cells.

“Pancreatic cancer carries a high mortality rate and is extremely difficult to treat, but promising earlier clinical data encouraged us to move into this advanced phase trial with devimistat. While these are not the results we all hoped for, we will not give up, and we are hopeful that devimistat with its novel mechanism of action will demonstrate efficacy in other studies,” said Dr. Philip Philip, principal investigator and medical oncologist at the Karmanos Cancer Institute at Wayne State University. “We are working diligently to further analyze the data and determine the best plan of action to further assess the drug’s capabilities in the clinic.”

The drug was also being studied for the treatment of acute myeloid leukemia, though after analysis, the independent data monitoring committee has recommended that the trial should stop because the drug is not proving to be efficacious in this patient population.

“Earlier clinical trial data demonstrated positive results, despite the challenges we face in treating AML,” said Dr. Jorge Cortes, director of the Georgia Cancer Center at Augusta University. “We were all hoping for positive results, and while this trial did not meet our expectations, we will continue to test devimistat in other studies.”

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