Safety and Efficacy Evaluation of AIV001 in Nonmelanoma Skin Cancer of the Low Risk Basal Cell Carcinoma Subtype

AIV001 (axitinib) has been formulated to provide focal disease-modifying therapy for patients with nonmelanoma skin cancer (NMSC) of the basal cell carcinoma (BCC) subtype. AIV001 targets angiogenesis, inflammation, and fibrosis associated with various pathological skin conditions and was formulated as a simple intradermal/intratumoral injection demonstrating prolonged skin residence. Surgical excision is the standard treatment for NMSC of the basal cell carcinoma subtype for lesions of < 20 mm. Surgical removal of lesions is effective but for some patients unwilling or contraindicated for surgery a nonsurgical option is needed. A nonsurgical option will eliminate post-surgical complications and scarring experienced after lesion removal. Also, specific anatomical location of lesions present cosmesis (i.e., face) or healing challenges (i.e., lower limbs). An effective injectable will benefit patients who are averse to surgery, at risk of wound healing complications or concerned with cosmesis outcomes or fatigued from multiple surgeries. Patient populations (i.e., elderly or patients with diabetes) who are at risk of delayed wound healing would benefit from an injectable option. This study will evaluate injection methods, interval of treatment, four ascending doses, safety, histological clearance and clinical clearance of biopsy-confirmed "low-risk" BCC lesion of <20 mm located on non-facial skin.

Inclusion criteria

• Male or female, aged 18 to 80 years, inclusive
• No clinically relevant abnormalities identified by a detailed medical history and vital signs
• Presence of a histologically confirmed low risk BCC lesion, with well-defined borders, and with a largest diameter measure before biopsy of 5 mm to 20 mm, located on arms or trunk
• Histological diagnosis of the target lesion must have been conducted 5 to 30 days prior to Day 1
• No other dermatological disease within 50 mm of the target lesion at Day 1
• No prior or concurrent treatment of the target lesion (including radiation therapy)
• Willing to undergo surgical excision approximately 63 days after first treatment.

Exclusion criteria

• History or presence of systemic cancer
• Prior radiation treatment at the lesion site or anywhere else on the body within the past 20 years
• Concurrent disease or treatment that suppresses the immune system (eg, previous organ transplant history, etc.)
• Clinically relevant cardiovascular, endocrine, hepatic, neurologic, renal, or other major systemic disease that could complicate execution of the protocol or interpretation of the study results.
• History of thrombotic events, hemorrhagic events, and gastrointestinal perforation and fistula
• History of recurrence or presence of any other tumor subtype in the target lesion
• Concurrent presence of a malignant lesion within 100 mm of the target lesion that will require treatment during the study
• Current enrollment in any other investigational drug or device study within 60 days of Day 1 of this study
• Evidence of dermatological disease or confounding dermatological condition that would hinder carrying out the study or interpreting the results (eg, atopic dermatitis, eczema, psoriasis, xeroderma pigmentosa, etc.)