Multicenter phase 3 trial of first-line chemotherapy with or without toripalimab for recurrent or metastatic nasopharyngeal carcinoma

Gemcitabine-cisplatin (GC) is the current standard first-line therapy for recurrent or metastatic nasopharyngeal carcinoma (RM-NPC). The phase 3 JUPITER-02 trial, at 35 centers in China, Taiwan, and Singapore, evaluated addition of toripalimab (anti-PD-1) to first line GC for RM-NPC. The trial included 289 patients, who were randomized 1:1 to toripalimab or placebo, added to GC for six cycles, followed by maintenance with toripalimab or placebo. At the final progression-free survival analysis, the toripalimab group had a significantly longer progression-free survival than the placebo group (median 21.4 versus 8.2 months; HR 0.52; 95% CI 0,37-0.73). With a median follow-up of 36.0 months, the median overall survival was not reached in the toripalimab group and was 33.7 months in the placebo group (HR 0.63; 95% CI 0.45-0.89). The incidence of all adverse events, grade 3 or higher adverse events, and fatal adverse events was similar between the groups. However, adverse events leading to discontinuation and immune-related adverse events were more frequent in the toripalimab group. The authors conclude that addition of toripalimab to first-line GC for RM-NPC resulted in statistically significant and clinically relevant progression-free and overall survival benefit, with manageable safety (visual abstract).