The company plans to conduct the Phase II study in Israel, Europe and the US and it will include 78 subjects that will be dosed with the drug as a second-line treatment of advanced hepatocellular carcinoma with Child-Pugh Class B cirrhosis.
The study will investigate the efficacy and safety of CF102 vs. placebo. The protocol has been approved by the IRB at the Rabin Medical Center, Petach Tikva, and the company intends to follow up with European and US submissions shortly.
The study protocol was developed with the assistance of Tufts University School of Medicine Department of Hematology chairman and Oncology Professor of Medicine Dr Keith Stuart. This center will also participate in the study.
The US Food and Drug Administration (FDA) has granted Orphan Drug designation for CF102, for the treatment of hepatocellular carcinoma.
FDA orphan drug status grants various incentives for developing these drugs, including shortened approval procedures, tax breaks on R&D costs, and financing assistance. If the drug is the first to reach market, it also receives seven years exclusivity.
The company reported earlier that data from the Phase I/II study were published recently in The Oncologist, one of the leading journals in this field, and was presented at the 18th World Congress on Advances in Oncology.
The company reported that the Phase I/II study data demonstrated that the trial objectives were successfully achieved, demonstrating a very favorable safety profile for CF102 in a patient population with hepatocellular carcinoma and Child-Pugh cirrhosis classes A and B.
In addition, the median overall survival time was very encouraging given that 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. Interestingly, one of the patients included in the Phase I/II study has been treated for 4 years with CF102 and is continuing to be treated, with CF102.