Number of Patients Alive at 41 Months Doubled for Biliary Tract Cancer During Phase III Trial

Biliary tract cancer, or cholangiocarcinoma, involves bile ducts that are outside and inside the liver. Since its approval in 2017, over 220,000 patients have been treated with Imfinzi which is now being tested on a wide range of gastrointestinal cancers.

The Survival Rate for BTC

As noted in PharmaBiz, when BTC has been diagnosed early, the 5-year survival rate is 24%. The survival rate is reduced to 9% if the cancer reaches the regional lymph nodes and even lower if it has spread to distant parts of the body.

Topaz-1 and Survival Rates

Phase III Results of the TOPAZ-1 Trial at the three-year mark found that overall survival for AstraZeneca’s Imfinzi (durvalumab), had doubled for BTC when combined with a standard chemotherapy regimen. The TOPAZ-1 follow-up survival results were presented this year on April 18th at the cholangiocarcinoma conference in Utah.

GI cancers, in 2020, collectively accounted for about 5.1 million newly diagnosed cases and about 3.6 million deaths. TOPAZ-1 is a multicenter global phase III trial of Imfinzi combined with chemotherapy (cisplatin plus gemcitabine). The trial was conducted in 105 centers across 17 countries. It is being compared to placebo combined with first line treatment for 685 adult patients diagnosed with unresectable (inoperable) or metastatic BTC, gallbladder, and extrahepatic cholangiocarcinoma.

BTC are rare, aggressive gastrointestinal (GI) cancers that form in bile duct cells, or the gallbladder, or at the pancreatic duct that connects to the small intestine. Twice the number of patients treated with the Imfinzi-based regimen were alive after 41.3 months (14.6%) compared to patients treated with chemotherapy alone (6.9%).  

The primary goal of the trial pertained to overall survival. That goal was met in October 2021 establishing a 20% drop in the risk of death resulting from treatment with Imfinzi.
The Imfinzi combination was well tolerated. The longer follow-up did not produce any new safety signals. Results of the trial found that 15.4% of participants had adverse events related to the Imfinzi treatment compared to 17.3% chemotherapy alone.
Imfinzi plus chemotherapy continued to be well-tolerated, with no new safety signals observed with longer follow-up.

GI cancers are difficult to diagnose. These complications often result in delayed diagnosis at a stage in the cancer that has few treatment options. GI cancers represented about 5.1 million new cancers in 2020. This led to about 3.6 million deaths.

The TOPAZ-1 trial exhibited a remarkable overall survival with Imfinzi plus chemotherapy that was well tolerated. At over three years, this has been the longest survival follow-up for immunotherapy.

bile duct cancer biliary tract cancer cholangiocarcinoma clinical trial research treatment

Last modified: May 21, 2024