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.
Locally Advanced Basal Cell Carcinoma Patients
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.
H-Zone nodular BCC
Nodular Basal Cell Carcinomas in the H-zone
SP-002 is being developed as a novel intralesional therapy for patients with nodular BCCs located in the H zone. These are lesions that have a high-risk of recurrence and/or require major reconstruction or result in significant morbidity/dysfunction with surgery. SP-002 may also be suitable for patients that are considered not good candidates for surgery, due to medical reasons or patient preferences.
It is estimated that 3-4 million cases1 of Basal Cell Carcinomas arise annually in the US, of these an estimated 60-80% are Nodular Basal Cell Carcinomas2, of which ~20-40% are located on the H-zone3 or other high-risk anatomical sites or occur in patients that are not good candidates for surgery, due to medical reasons or patient preferences. It is estimated that an annual population of up to 857,000 patients annually in the United States may be addressable with SP-002 for this patient subset.