Skin Cancer: Basal Cell

    Actionable Target Abnormality Prevalence (%) Clinical Experience with Targeted Agent
    PTCH1 Mutation 39 Vismodegib (Hedgehog inhibitor):
    • Phase 2: RR for metastatic 30%, RR for locally advanced 43%, including CR in 21% (1)
    Sonidegib (Hedgehog inhibitor):
    • Phase 2: RR for metastatic 15%, RR for locally advanced 43% (2 pts) (2)
    SMO Mutation 12
    PD-1/PD-L1 N/A N/A REGN2810 (PD-1 inhibitor):
    • Case report: Durable PR ongoing 12+ mo (3)
    Nivolumab (PD-1 inhibitor):
    • Case report: Ongoing near CR at 4+ mo; high mutational burden (>50 mut/MB); 19 functional alterations in tissue as well as
    Other topics in Targets by Organ Site