Phase 2 trial of neoadjuvant toripalimab plus axitinib in resectable mucosal melanoma

The outcomes of patients with resectable mucosal melanoma is poor. The combination of toripalimab (anti-PD-1) and axitinib has shown impressive results in metastatic mucosal melanoma in a phase 1b trial. A phase 2 trial at Peking University Cancer Hospital and Institute (Beijing, China) evaluated the combination of toripalimab and axitinib for resectable mucosal melanoma in the neoadjuvant setting, followed by surgery and adjuvant toripalimab. Pathologic response was seen in 33.3% of patients. Median event-free survival was 11.1 months, and median overall survival was not reached after median follow-up of 34.2 months. Neoadjuvant therapy was tolerable with grade 3 of 4 treatment-related adverse events in 27.5% and no treatment-related deaths. IHC analysis of tissue samples collected at baseline and after surgery showed a significant increase of tumor-infiltrating lymphocytes, particularly in pathological responders. The authors conclude that neoadjuvant toripalimab plus axitinib had promising activity among patients with resectable mucosal melanoma.