Imfinzi plus chemotherapy significantly improved overall survival in 1st-line advanced biliary tract cancer.
At a predefined interim analysis, the Independent Data Monitoring Committee concluded that the trial met the primary endpoint by demonstrating an improvement in OS in patients treated with Imfinzi plus chemotherapy versus chemotherapy alone. The combination also demonstrated an improvement in progression-free survival (PFS) and overall response rate, key secondary endpoints. Imfinzi plus chemotherapy was well tolerated, had a similar safety profile versus the comparator arm and did not increase the discontinuation rate due to adverse events compared to chemotherapy alone.
BTC is a group of rare and aggressive cancers that occur in the bile ducts and gallbladder. Incidence of BTC often depends on the prevalence of common risk factors for each type within a geographical region. Approximately 50,000 people in the US, Europe and Japan and about 210,000 people worldwide are diagnosed with BTC each year. These patients have a poor prognosis, with approximately only 5% to 15% of all patients with BTC surviving five years. In December 2020, Imfinzi was granted Orphan Drug Designation in the US for the treatment of BTC.
TOPAZ-1 :TOPAZ-1 is a randomised, double-blind, placebo controlled, multicentre, global Phase III trial of Imfinzi in combination with chemotherapy (gemcitabine plus cisplatin) versus placebo in combination with chemotherapy as a 1st-line treatment in 685 patients with unresectable advanced or metastatic BTC including intrahepatic and extrahepatic cholangiocarcinoma, and gallbladder cancer (ampullary carcinoma was excluded). The trial is being conducted in more than 145 centres across 17 countries including in the US, Europe, South America and several countries in Asia including South Korea, Thailand, Japan, Taiwan and China. The primary endpoint is OS and key secondary endpoints include progression-free survival, objective response rate and safety.
Do-Youn Oh, MD, PhD, Professor, Division of Medical Oncology, Department of Internal Medicine at Seoul National University Hospital and Seoul National University College of Medicine, and principal investigator in the TOPAZ-1 Phase III trial, said: “Patients with advanced biliary tract cancer are in dire need of new treatments as progress in the 1st-line setting has remained largely stagnant for more than 10 years. TOPAZ-1 is the first Phase III trial to show that adding an immunotherapy to standard chemotherapy delivers a meaningful overall survival benefit for patients in this setting. Today’s exciting results are a major step forward in treating this disease and represent new hope for our patients.”