The Phase III CELESTIAL trial by Ipsen (London, UK), investigating the use of cabozantinib as a liver cancer therapy, has been halted due to “compelling evidence of efficacy”, after the drug met its primary endpoint of overall survival (OS).
The data was presented at the American Society of Clinical Oncology Gastrointestinal Cancers Symposium (18th-20th January, CA, USA). Ewan McDowall, General Manager of Ipsen UK & Ireland, explained the significance of the results: “These data will form the basis of our licence submission, bringing us one step closer to making this positive new treatment available to patients in the UK & Ireland.”
Cabozantinib is a tyrosine kinase inhibitor, intended for use in renal cell carcinoma. The Ipsen study enrolled 760 patients with hepatocellular carcinoma (HCC) from 19 countries, examined at over 100 study sites.
Results released from the trial demonstrated that cabozantinib provided a statistically significant and clinically meaningful improvement versus placebo in OS, at the planned second interim analysis for the population of second- and third-line patients enrolled in this study.
Median OS was 10.2 months with cabozantinib versus 8 months with placebo. Median progression-free survival was more than doubled, at 5.2 months with cabozantinib and 1.9 months with placebo. Objective response rates per RECIST 1.1 were 4% with cabozantinib and 0.4% with placebo (p=0.0086). Disease control (partial response or stable disease) was achieved by 64% of the cabozantinib group compared with 33% of the placebo group.
The results come as a breakthrough in hepatocellular carcinoma therapy, which has historically exhibited poor rates of survival due to the limited number of drugs available, as summarized by Alexandre Lebeaut, Executive Vice-President & Chief Scientific Officer of Ipsen: “Liver cancer is one of the leading causes of cancer deaths worldwide and more effective treatment options are urgently needed…cabozantinib has been shown to provide a survival benefit and therefore has the potential to bring a new oral systemic treatment to previously treated patients with advanced liver cancer.”