Basal Cell Nevus Syndrome & Locally Advanced Basal Cell Carcinoma

Basal Cell Nevus Syndrome is a rare, autosomal dominant (germline) condition. While rarely life-threatening, BCNS significantly diminishes patient quality of life due to the multiple cancerous skin tumors that appear predominantly on the head, neck and trunk. The biggest challenge for these patients is the appearance of numerous BCCs. A range of BCC severity has been reported for patients ranging from dozens and hundreds of tumors that can arise over a 12-month period. The majority of BCNS patients have a moderate phenotype developing around 20-40 BCCs annually which require frequent review and treatment.

The Clinical Need

A minimally invasive approach that is able to effectively, tolerably and durably treat multiple lesions as they present and also able to reduce the number of new lesions that arise.

The Disease

Basal cell carcinoma is a common skin cancer, with 3-4 million cases arising annually in the US. About 2% of these patients progress to advanced disease (about ~60,000 per yr. in the US). LA BCC comprise unresectable lesion, recurrent lesions (multiple recurrences) and lesions that would result in significant functional damage upon resection.

Current Management

The HHPI (Hedgehog pathway inhibitors, e.g., vismodegib and sonidegib) are FDA-approved agents for use in LA BCC when surgery and radiotherapy are not suitable. Cemiplimab has been approved by the FDA as second line treatment for patients previously treated with a hedgehog pathway inhibitor or for whom a HHPI is not appropriate. However, only a subset of LA BCC patients use HHPIs due to the modest CR rates.

The Clinical Need

The combination of SP-002 with an HHPI aims to improve clinical outcomes. Vismodegib as a mono therapy has an ORR of between 60-70% and the objective of SP-002, as an add-on to SOC HHPI, is to improve both CR rates and durability of response via the complementary and synergistic mechanisms of action.