Multinational phase 2 trial of second-line cemiplimab after failure of hedgehog pathway inhibitor for metastatic basal cell carcinoma

There is no standard second-line treatment after failure of hedgehog pathway inhibitors (HHIs) for metastatic basal cell carcinoma (mBCC). A multinational phase 2 trial evaluated second-line cemiplimab (anti-PD-1) after HHI for mBCC. The objective response rate was 22% (95% CI 12-36), with complete response in 2 of 54 patients. Among responders, median time to response was 3 months (IQR 2-7) and median duration of response was not reached during median 8 months of follow-up. Disease control rate was 63% (95%-bti 49-76). Median progression-free survival was 10 months (95% CI 4-16) and median overall survival was 50 months (28-NE). The most common treatment-emergent adverse events were fatigue (43% of patients) and diarrhoea (37%). There were no treatment-related deaths. The authors conclude that cemiplimab demonstrated clinically meaningful antitumor activity and acceptable safety in patients with mBCC after failure of HHIs.