Can-Fite BioPharma announced that the FDA has granted its request for orphan drug designation for CF102 for the treatment of hepatocellular carcinoma.

Earlier this year, Can-Fite announced results of the Phase 1/2 study of CF102 for the treatment of hepatocellular carcinoma. The study data demonstrate that the trial objectives were successfully achieved, demonstrating a favorable safety profile for CF102 in a patient population with hepatocellular carcinoma and Child-Pugh cirrhosis classes A and B. The median overall survival time was favorable. Most patients were treated in the second-line setting and some were Child-Pugh class B. Another finding indicated that the A3 adenosine receptor, which is the target of CF102, can serve as a biomarker to predict the patients’ reaction to treatment with CF102.

CF102 is a small orally bioavailable drug which bind with high affinity and selectivity to the A3 adenosine receptor. The latter is highly expressed in tumor cells whereas low expression is found in normal cells. The drug induces a robust anti-tumor effect via de-regulation of the Wnt signaling pathway, resulting in apoptosis of liver-cancer cells.

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