Exelixis reported positive preliminary data from a cohort of hepatocellular carcinoma (HCC) patients participating in the ongoing Phase 2 randomized discontinuation trial (RDT) of cabozantinib. The data are from a trial evaluating the activity of cabozantinib in multiple tumor types. The results include data from 41 patients with advanced HCC.

Patients initially received open-label cabozantinib at 100mg daily during a 12-week Lead-In phase. Patients achieving a PR at week 12 were eligible for continued open-label treatment with cabozantinib, and patients with progressive disease discontinued treatment. Patients with SD entered the randomized discontinuation phase. Patients progressing on placebo had the option of receiving salvage therapy with cabozantinib.

The week 12 disease control rate (PR and SD at week 12) was 68%. Evidence of objective tumor regression was observed in 78% of patients, including those with or without prior sorafenib therapy. The best radiologic response in the lead-in stage of the study for 36 patients with at least one post-baseline measurement was confirmed partial response (cPR) in 2 patients and stable disease (SD) in 32 patients. One additional patient had a partial response that was confirmed after the patient completed the lead-in stage and proceeded to the randomized component of the trial. Median progression-free survival (PFS) was 4.2 months, and was similar for sorafenib-pretreated and sorafenib-naïve patients.

Cabozantinib is a potent, dual inhibitor of MET and VEGFR2. Cabozantinib is an investigational agent that provides coordinated inhibition of metastasis and angiogenesis to kill tumor cells while blocking their escape pathways.

The therapeutic role of cabozantinib is currently being investigated across several tumor types.

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