Tislelizumab shows favourable safety profile compared to sorafenib among liver cancer patients

Novartis has announced new data from the phase 3 RATIONALE 301 trial that shows how tislelizumab demonstrated non-inferior overall survival (OS) compared to sorafenib in patients with previously untreated, unresectable hepatocellular carcinoma (HCC).

RATIONALE 301 is a multi-regional, open-label, randomised phase 3 study of tislelizumab versus sorafenib in previously untreated patients with unresectable HCC. During the trial, 674 participants received either tislelizumab or sorafenib. The primary objective has been to compare OS between the two treatment groups.

The trial met its primary objective of non-inferiority for OS, while superiority was subsequently tested, which was not met. This data was presented at a late-breaking oral session at the 2022 European Society for Medical Oncology Congress.

OS results were consistent across pre-specified subgroups, including regions. Tislelizumab was associated with higher objective response rate and more durable responses than sorafenib. The safety profiles of both agents were consistent with previous reports and no new safety signals were identified.

"People living with advanced liver cancer face poor survival outcomes and frequently suffer from cirrhosis, which further complicates their treatment,” explained Richard Finn, Professor of Medicine at David Geffen School of Medicine. “These positive results show that tislelizumab has the potential to deliver a meaningful clinical benefit for patients with HCC who need more safe and effective therapeutic options.”

“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,” added Jeff Legos, global head of oncology and haematology development at Novartis. “We are excited to see the positive efficacy results and favourable safety profile of tislelizumab monotherapy in this setting and will begin discussing the data with regulatory authorities.”

HCC is the most common type of liver cancer globally and constitutes 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.

Novartis has the rights to develop, manufacture and commercialise tislelizumab in North America, Europe and Japan through a collaboration and licence agreement with BeiGene.