In the CHOICE-01 research, toripalimab and chemotherapy were used as the first-line therapy for advanced non-small-cell lung cancer (NSCLC), and their effectiveness and safety were examined.

Advanced NSCLC without EGFR/ALK mutations patients (N = 465) were randomly assigned 2:1 to receive toripalimab 240 mg (n = 309) or a placebo (n = 156) once every 3 weeks in combination with chemotherapy for 4-6 cycles, then to receive toripalimab or a placebo once every 3 weeks in addition to standard care going forward. Histology, programmed death ligand-1 expression status, and smoking status were stratification variables. According to RECIST v1.1, the investigator’s progression-free survival (PFS) was the major end objective. Safety and general survival were secondary endpoints.

When the PFS was finally analyzed, the toripalimab arm’s PFS was shown to be considerably longer than the placebo arm’s (median PFS 8.4 v 5.6 months, hazard ratio = 0.49; 95% CI, 0.39 to 0.61; two-sided P< .0001). The median OS not achieved v 17.1 months, hazard ratio = 0.69; 95% CI, 0.53 to 0.92; two-sided P =.0099; showed that the toripalimab arm had a considerably longer OS than the placebo arm in the interim OS analysis. The frequency of grade ≥3 negative occurrences was comparable in both groups. Regardless of the presence of programmed death ligand-1, treatment outcomes were comparable. Patients with high tumor mutational load were linked with significantly superior PFS in the toripalimab arm (median PFS 13.1 v 5.5 months, interaction P =.026), according to genomic analysis employing whole-exome sequencing from 394 available tumor samples. Notably, toripalimab significantly improved PFS and OS in patients with mutations in the focal adhesion-PI3K-Akt signaling pathway (interaction P values ≤.001).

With a tolerable safety profile, toripalimab with chemotherapy significantly increased PFS and OS in patients with advanced NSCLC who had not received treatment previously. The nonsquamous subpopulation was primarily responsible for the OS advantage, according to subgroup analysis.