Benzinga | Aug 25, 2021 10:01AM EDT

Rafael Pharma Reports Successful Completion Of Phase 1b Trial, Begins Phase 2 Trial For CPI-613 In Combo With Gemcitabine, Cisplatin In Patients With Biliary Tract Cancer

Rafael Pharmaceuticals, Inc. ("Rafael" or the "Company"), a leader in the growing field of cancer metabolism-based therapeutics, today announced the successful completion of a Phase 1b clinical trial of CPI-613(r) (devimistat) in combination with gemcitabine and cisplatin in patients with 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 who have had no prior treatment.

"We are extremely excited and gratified to have completed Phase 1b and to be able to advance to the next phase (Phase 2)," said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. "Advanced biliary tract cancer is a rare and aggressive cancer with a 5-year survival rate of less than 5%, and the continuation of the trial aligns with our mission to help patients with significant unmet medical needs."

"The Phase 1b part of the trial has successfully identified the recommended Phase 2 dose of CPI-613 when given in combination with gemcitabine and cisplatin in this patient population. The 2:1 randomized Phase 2 part of the trial has already started enrolling patients and will now determine the efficacy of devimistat at this maximum tolerated dose in combination with gemcitabine and cisplatin as compared with the combination chemotherapy alone" said Dr. Vaibhav Sahai, MBBS, M.S., primary investigator and medical oncologist at the University of Michigan Rogel Cancer Center in Ann Arbor, Michigan. An estimated 68 to 78 patients will be enrolled in the study (NCT04203160).