Can-Fite BioPharma Ltd., a biotechnology company with a pipeline of proprietary small molecule drugs that address inflammatory and cancer diseases, announced the company's oncology drug candidate, CF102, has been granted Orphan Drug designation by the European Medicines Agency (EMA) for the indication of hepatocellular carcinoma (HCC), the most common form of liver cancer.
CF102 will benefit from protocol assistance and a 10-year market exclusivity following market authorisation in the 28 European Union (EU) member states, as well as 3 additional European Economic Area (EEA) countries.
"The EMA's Orphan Drug designation for CF102 is the latest in a series of catalysts that we believe are accelerating the clinical development path of our liver cancer drug towards market approval. As we actively recruit patients in our phase II study of CF102 in Europe, we are pleased the EMA will support CF102 through protocol assistance and post-authorization market exclusivity," stated Can-Fite CEO Dr. Pnina Fishman.
In the US, CF102 has already received Fast Track designation as a second line for the treatment of HCC of patients who have previously received Nexavar (sorafenib) and Orphan Drug designation for the treatment of HCC. Israel's Ministry of Health has also approved CF102 for compassionate use for HCC.
Can-Fite is conducting a phase II study with CF102 in patients with advanced HCC in the US, Europe and Israel. The randomized, double blind, placebo controlled study is expected to complete enrollment by the end of the first half of 2016 in 78 patients with Child-Pugh class B cirrhosis who failed the only FDA approved drug on the market, Nexavar (sorafenib). Patients are treated twice daily with 25 mg of oral CF102, which has been found to be the most efficacious dose in Can-Fite's earlier phase I/II study resulting in the longest overall survival time, with excellent safety results.
According to Global Industry Analysts, the global market for liver cancer drugs is projected to exceed $2 billion in 2015. Nexavar annual sales, as reported by Bayer, were €773 million in 2014.
CF102 is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). A3AR is highly expressed in tumour cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug. In Can-Fite's pre-clinical and clinical studies, CF102 has demonstrated a robust anti-tumour effect via deregulation of the Wnt signaling pathway, resulting in apoptosis of liver cancer cells.