Rationale for development

Biliary tract cancer, including gallbladder tumours, is the second most common primary hepatobiliary cancer, after hepatocellular cancer.1 Estimates suggest there are almost 140,000 deaths each year from biliary tract cancer, a 22% increase since 1990.2 However, despite the availability of surgery and chemotherapy options for early and locally advanced disease, patients are not able to access any indicated second line treatments.

1. Hennedige TP, Neo WT, Venkatesh SK. Imaging of malignancies of the biliary tract- an update. Cancer Imaging. 2014;14(1
2. GBD 2013 Mortality and Causes of Death Collaborators. Global, regional, and national age-sex specific all-cause and cause-specific mortality for 240 causes of death, 1990-2013: a systematic analysis for the Global Burden of Disease Study 2013. Lancet 2015; 385:117-71
3. Ulrich-Frank Pape, Stefan Kasper, Marianne Sinn, Karel Caca, Jan Kuhlmann, Ulrich Keilholz, Arndt Vogel, and Nalan Utku. Randomized, multicenter phase II trial of CAP7.1 in patients with advanced biliary tract cancers. Journal of Clinical Oncology 2016; 34:4_suppl, 441-441

Etoposide toniribate

Etoposide toniribate is a novel prodrug of anticancer agent, etoposide. The treatment is activated in cells expressing carboxylesterase 2 (CES2), an enzyme that is found in the gastrointestinal tract and is particularly active in tumour cells.3


Progress to date

In Phase II studies etoposide toniribate showed efficacy in patients with advanced disease who had already been treated with the current standard of care, with 56% of patients meeting the primary objective of disease control, including tumour shrinkages.3