Novartis and BeiGene have announced new data from a phase 3 trial that shows tislelizumab demonstrated non-inferior overall survival (OS) compared to sorafenib in patients with previously untreated, unresectable hepatocellular carcinoma (HCC).
Results from the phase 3 RATIONALE 301 trial of tislelizumab versus sorafenib as first-line treatment in patients with unresectable HCC were presented at a late-breaking session at the 2022 European Society for Medical Oncology (ESMO) Congress on 10 September.
The study met its primary objective of non-inferiority for OS. Superiority was subsequently tested, which was not met.
Moreover, tislelizumab was associated with higher objective response rate – 14.3% versus 5.4% – and more durable responses – 36.1 months versus 11 months – compared with sorafenib.
Median progression-free survival was 2.1 months with tislelizumab compared to 3.4 months with sorafenib, while median treatment duration was longer with tislelizumab – 4.1 months versus 2.7 months.
Jeff Legos, executive vice president, global head of oncology and haematology development, Novartis said: “HCC is an aggressive disease with poor survival outcomes, and there is an urgent need for additional treatment options with improved tolerability in the first line setting.
“We are excited to see the positive efficacy results and favourable safety profile of tislelizumab monotherapy in this setting and will begin discussing this data with regulatory authorities.”
HCC is the most common type of liver cancer globally, accounting for 75-85% of liver cancer diagnoses. In 2022, it is estimated that there will be more than 800,000 new liver cancer diagnoses and 700,000 deaths worldwide.
Over 70% of HCC patients in the US are diagnosed with unresectable cancers, and US patients with metastasised cancers face a five-year survival rate of 12% or less, Novartis reported.
Through a collaboration and licence agreement with BeiGene in January 2021, Novartis has the rights to develop, manufacture and commercialise tislelizumab in Europe, North America and Japan.