Exelixis, Inc. (NASDAQ:EXEL) today reported positive updated interim
data from the cohort of hepatocellular carcinoma (HCC) patients
participating in the ongoing phase 2 randomized discontinuation trial
(RDT) 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. Slides from
the presentation are available at http://www.exelixis.com/resources/events/asco-2012.
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 (TKI) 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 (Hb) < 11 g/dL, 34% had thrombocytopenia, and median
alpha-fetoprotein (AFP) 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 (PFS) and Overall
Survival (OS). 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 [PR] and stable disease [SD] 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%).