Cornerstone Pharmaceuticals Announces Successful Completion Of Phase 1b Clinical Trial For CPI-613 In Combination With Gemcitabine And Cisplatin In Patients With Biliary Tract Cancer

Cornerstone Pharmaceuticals, Inc. (“Cornerstone” or the “Company”), a company focused on rare cancer therapeutics and formerly known as Rafael Pharmaceuticals, today 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 U.S., 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, M.D., MBBS, M.S., 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.”