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First-line treatment with tislelizumab was found to result in a noninferior overall survival benefit to that achieved with sorafenib in adult patients with unresectable hepatocellular carcinoma, meeting the primary end point of the phase 3 RATIONALE 301 trial.
First-line treatment with tislelizumab (BGB-A317) was found to result in a noninferior overall survival (OS) benefit to that achieved with sorafenib (Nexavar) in adult patients with unresectable hepatocellular carcinoma (HCC), meeting the primary end point of the phase 3 RATIONALE 301 trial (NCT03412773).1
Moreover, the human IgG4 anti–PD-1 monoclonal antibody demonstrated a toxicity profile that proved to be consistent with what has previously been reported, with no new signals observed.
“Patients with unresectable HCC face a devastating prognosis, with a median life expectancy of 1 year. Currently, there are few treatment options if patients cannot tolerate TKI therapy or if their condition progresses,” Mark Lanasa, MD, PhD, chief medical officer of Solid Tumors at BeiGene, stated in a press release. “We are encouraged by the outcome of the final analysis of RATIONALE-301 and look forward to sharing the full safety and efficacy results at an upcoming medical conference.”
The global, open-label, phase 3 RATIONALE-301 trial enrolled patients with a histologically confirmed diagnosis of HCC that was Barcelona Clinic Liver Cancer stage B or C and not amenable to or that had progressed following locoregional therapy; their disease could not be amenable to a curative therapeutic approach.2 Patients were required to have measurable disease, Child-Pugh class A disease, an ECOG performance status of 0 or 1, and acceptable organ function.
Patients could not have known fibrolamellar HCC, sarcomatoid HCC, or mixed cholangiocarcinoma and HCC histology, nor could they have tumor thrombus involving the main trunk of the portal vein or inferior vena cava. Other exclusion criteria included having received locoregional therapy to the liver within 28 days prior to randomization on study, clinical evidence of portal hypertension with bleeding esophageal or gastric varices, or a bleeding or thrombotic disorder, among others.
Study participants received either intravenous tislelizumab at 200 mg once every 3 weeks or oral sorafenib at a twice daily dose of 400 mg.
Beyond the primary end point of noninferiority of OS between the treatment arms, a key secondary end point is overall response rate per blinded independent review committee (BIRC) assessment and by RECIST v1.1 criteria. Other secondary end points include progression-free survival, duration of response, BIRC-assessed time to progression, health-related quality of life measures, and safety.