Bile duct cancer (cholangiocarcinoma) selected as next indication for PCI Biotech. Clinical proof of concept study to start by end of 1H 2013

Bile duct cancer (cholangiocarcinoma) selected as next indication for PCI Biotech. Clinical proof of concept study to start by end of 1H 2013

Oslo, 21 August 2012 - PCI Biotech (PCIB) the Norwegian biopharmaceutical company, reported today that it has chosen bile duct cancer as the next indication for the use of its PCI (photochemical internalisation) localised cancer therapy. In this indication PCI Biotech's proprietary photosensitiser Amphinex® will be used in combination with the generic cytotoxic agent gemcitabine. The aim is to start the clinical Proof of Concept study within end of 1H 2013.

CEO Per Walday explained: "Bile duct cancer has been chosen as the next indication for PCI for a number of compelling reasons. Bile duct cancer is highly drug resistant and there is a clear medical need for a better local treatment. Access with light is easy by using already established endoscopy-based treatment procedures and one of the most commonly used cytotoxic agents is gemcitabine which our preclinical studies have already shown is significantly enhanced by PCI."

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 patients are inoperable at presentation. Inoperable patients are treated with stenting to keep the bile duct open and with chemotherapy. The combination of gemcitabine and cisplatin has shown promising results and has become standard treatment in some regions, but there is still a need for better treatments to increase overall survival and quality of life.

Could PCI play a role in the treatment of bile duct cancer?
Improved local methods to slow tumour progression and keep the bile duct open are important to extend life span and quality. Bile duct cancer is characterized by a remarkable resistance to common chemotherapy, and new drug classes or alternative methods are needed. The most studied and used drug is gemcitabine, which is one of the identified drugs that are significantly enhanced by PCI in preclinical studies. Light access is easy through the endoscopic methods that are routinely used in the treatment of this disease.

Clinical study
The Proof of Concept study is planned to be an open-label, multi-centre Phase I/II study in up to 45 patients to assess the safety and efficacy of Amphinex® induced PCI of gemcitabine, followed by systemic cisplatin/gemcitabine in patients with inoperable bile duct cancer. The study will consist of a dose escalation/phase I part to assess the tolerance of local bile duct treatment and a randomized double-arm phase II part. In phase II patients will be randomized to either a control arm (stenting alone followed by gemcitabine/cisplatin chemotherapy) or the PCI arm (stenting followed by Amphinex induced PCI treatment of gemcitabine followed by gemcitabine/cisplatin chemotherapy). The randomization ratio for this study is 2.5:1 in favor of the PCI arm. The Phase I primary objective will be to determine a tolerable dose for local bile duct treatment with Amphinex® induced PCI of gemcitabine, while the Phase II primary objective will be to assess efficacy in terms of progression free survival.

Inclusion of patients is expected to be completed by 2014. Inclusion of patients is estimated to take approximately 6 months in Phase I and approximately 10 months in Phase II. Estimated costs for the study is approximately NOK 7 million for Phase I and approximately NOK 11 million for Phase II.