Efficacy and safety of first line Cemiplimab in advanced BCC: A phase 2 trial

Basal cell carcinoma (BCC) is the most common type of skin cancer worldwide. BCC tumours are generally slow growing and rarely spread to other parts of the body and outcomes for patients treated with appropriate therapy are very good. Treatment can include surgery, radiation therapy, topical treatments and photodynamic therapy.However, in a small number of patients BCCs can get worse, progressing to an advanced stage (aBCC). These advanced tumours can become large, aggressive and penetrate through the skin into the underlying tissue. They are much more difficult to treat. Radiation can be ineffective and surgery may not be an option without causing significant morbidity, loss of function or disfigurement. The tumours also have a high risk of coming back. Newer treatments include treatment with inhibitors of the hedgehog pathway e.g. vismodegib and sonidegib. These have been shown to reduce the size of aBCC tumours in 45% of patients treated, with an average duration of 9.5 months. Although licensed for the treatment of aBCC, NICE have not approved them for use as a treatment in the UK. Therefore currently there is a lack of viable treatment options for these patients. These tumours have a high mutation burden suggesting treatment with immunotherapy drugs would be successful. Indeed the immunotherapy drug cemiplimab has been licensed for use in this patient group as second line treatment for locally advanced or metastatic BCC after treatment with hedgehog inhibitors has failed. Again due to the fact hedgehog inhibitors are not being used in the NHS this treatment is not an option for patients in the UK. Therefore in this study we propose to evaluate the safety and efficacy of cemiplimab as first line therapy for these patients with locally advanced BCC.