Cornerstone Pharmaceuticals Announces Successful Completion of Phase 1b Clinical Trial

Cornerstone Pharmaceuticals, Inc. recently announced the successful completion of a Phase 1b clinical trial of CPI-613 (devimistat) in combination with gemcitabine and cisplatin for the treatment of advanced unresectable biliary tract cancer. In collaboration with Michigan Medicine, the Phase 1b study consisted of a multicenter trial of gemcitabine and cisplatin with devimistat as first-line therapy for patients with locally advanced unresectable or metastatic biliary tract cancer.

The Phase 1b portion of the trial enrolled 20 patients and concluded that the combination of gemcitabine and cisplatin with devimistat is well tolerated. The trial reported encouraging data with an overall response rate of 45% and a median progression-free survival of 14.9 months with seven patients still in treatment. After a median follow-up of 11.5 months, the overall survival is not yet estimable. The randomized Phase 2 portion of the trial is open and accruing patients across 10 cancer centers in the US, targeting an enrollment of 78 patients.

“We are very pleased with the results of the trial, as we advance to Phase 2,” said Sanjeev Luther, President and CEO of Cornerstone Pharmaceuticals. “Advanced biliary tract cancer is a rare and aggressive form of cancer with a 5-year survival rate of less than 5%. The continuation of the trial aligns with our mission to provide hope to patients and loved ones affected by biliary tract cancer and other rare cancers.”

“With strong and continued trial enrollment rates, the research community remains engaged in identifying the capabilities devimistat has in biliary cancer,” said Vaibhav Sahai, MD, MBBS, MS, principal investigator of the trial and medical oncologist at the University of Michigan Rogel Cancer Center in Ann Arbor, Michigan. “Devimistat is a potentially life-saving therapy, and we remain optimistic in its promise as the study continues to yield positive results, giving us great hope for the future of biliary cancer care.”

CPI-613 (devimistat) is a first-in-class clinical lead compound of Cornerstone, 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.

Cornerstone Pharmaceuticals is committed to the development of therapies for rare cancers that have few to no treatment options available. 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. Devimistat is undergoing multiple clinical trials and has been granted orphan drug designation by the US 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.

With science stemming from the world’s most renowned institutions and compassion that runs deep, Cornerstone strives to improve the lives of people with rare cancers, inaugurating a new era of hope. For more information, visit