About bile duct cancer
The bile duct drains bile from the liver into the small intestine. Bile duct cancer is a relatively rare cancer with an annual incidence of 1-2 cases per 100,000 in the Western world. The incidence rate has been rising worldwide over the past several decades. Survival is less than 25% at 2 years in patients with resectable tumour and less than 1% at 2 years in patients with unresectable tumour. Biliary tract sepsis, liver failure and/or malnutrition and cachexia due to locoregional effects of the disease are the most important causes of death.
Current treatment regime
Currently, surgery is the only curative option for these patients; yet the majority of the tumors are inoperable at presentation. Inoperable patients are treated with stenting to keep the bile duct open and with chemotherapy. There is no approved treatment, but the combination of gemcitabine and cisplatin has shown activity and has become standard treatment in some regions. There is a high need of better treatments to increase overall survival and quality of life.
fimaCHEM in the treatment of bile duct cancer
Bile duct cancer has been chosen as the first indication for fimaCHEM, as there is a clear medical need for better local treatment, access with light is easy by using standard endoscopic treatment procedures and the most used cytotoxic agents, gemcitabine, is one of the drugs that is significantly enhanced by PCI in preclinical studies. Fimaporfin has been granted Orphan Designation for this indication in EU and in USA.
A multi-centre Phase I dose-escalation study in 16 patients to assess the safety of fimaporfin (Amphinex®) induced PCI of gemcitabine, followed by systemic cisplatin/gemcitabine, has recently been completed in patients with inoperable bile duct cancer. The primary objective was to determine a tolerable dose for local bile duct treatment with fimaporfin induced PCI of gemcitabine, but the study also provided very promising early signs of tumour response. The safety of repeated treatment is currently being explored as a Phase I extension. PCI Biotech has opened an IND in the US and is now planning for a multi-site pivotal Phase II study in this patient population.