3-year survival update of phase 1b study of toripalimab plus axitinib for metastatic mucosal melanoma

A phase 1b study in China evaluated the combination of the VEGFR TKI axitinib and the anti-PD-1 antibody toripalimab for metastatic mucosal melanoma. In 2019 a promising activity of the combination was published (ORR 48.3%; median PFS 7.5 months). An update of the study has now been published. With 42.5 months median follow-up, the median DoR was 13.4 months, the median PFS was 7.5 months, and the median OS was 20.7 months. PD-L1 expression and tumor mutational burden were not associated with survival benefits, but a 12-gene expression profile signature correlated with improved PFS (17.7 versus 5.7 months; p=0,0083) and OS (35.6 versus 17.6 months; p=0,039).