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Cabozantinib Demonstrates Evidence of Anti-Tumor Effects in Heavily Pretreated Patients with Hepatocellular Carcinoma


Exelixis (NASDAQ: EXEL) reported Saturday positive updated interim data from the cohort of hepatocellular carcinoma patients participating in the ongoing phase 2 randomized discontinuation trial of cabozantinib. Chris Verslype, M.D., Ph.D., professor of medicine at the Departments of Digestive Oncology/Hepatology at the University Hospitals Gasthuisberg, Leuven, Belgium, and an investigator on the trial, presented the data today in an oral session at the American Society of Clinical Oncology 2012 Annual Meeting (Abstract #4007). The meeting is taking place in Chicago, Illinois.

The results comprise data from 41 patients with advanced hepatocellular carcinoma with measurable disease at baseline and documented progressive disease per RECIST criteria. Patients in the open label 12-week Lead-In Stage of the trial received a daily dose of 100 mg cabozantinib. Eligible patients had Child-Pugh Score A. Eighty percent had received 1-2 prior lines of systemic therapy: 56% had prior tyrosine kinase inhibitor therapy, including 51% previously treated with sorafenib. At baseline, extrahepatic spread of disease (which is associated with a poorer prognosis) was present in 73% of subjects, 39% had hemoglobin < 11 g/dL, 34% had thrombocytopenia, and median alpha-fetoprotein level was 368 ng/ml. Tumor assessments per original RECIST 1.0 were conducted using conventional CT/MRI at baseline and every 6 weeks thereafter.

Progression-Free survival and Overall Survival. Median PFS was 4.4 months, and was similar for sorafenib-pretreated and sorafenib-naïve patients. Median OS in the 41 patients was 15.1 months.

Response Rate. The week 12 disease control rate (partial response and stable disease at week 12) was 66%. Evidence of objective tumor regression was observed in 78% of patients, including those with or without prior sorafenib therapy. The best radiologic response per RECIST in the Lead-In stage of the study for 36 patients with at least one post-baseline measurement was PR in 2 patients (5%) and SD in 32 patients (78%).

“The progression-free survival and overall survival results observed to date are encouraging,” said Eric Van Cutsem, M.D., Ph.D., professor of internal medicine and digestive oncology at the University Hospitals Gasthuisberg, Leuven, Belgium, and senior investigator on the presentation. “It is also interesting that the activity of cabozantinib in this population was irrespective of sorafenib pre-treatment status. The data suggest that dual inhibition of MET and VEGF with cabozantinib may provide clinical benefit beyond what can be achieved through inhibition of either pathway alone.”

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