Inovio Pharmaceuticals has commenced a phase 2 trial to evaluate the efficacy of VGX-3100 in patients with pre-cancerous lesions of the vulva, or vulvar intraepithelial neoplasia (VIN). VGX-3100 is an immunotherapy that targets human papillomavirus (HPV) 16 and 18 and is being studied for the treatment of HPV-related pre-cancerous lesions and persistent HPV infection that causes these lesions.

VIN has a very low rate of spontaneous, or natural, regression - below 5%. Currently there are no FDA approved non-surgical treatments for pre-cancerous lesions of the vulva. Surgery, the most common treatment, is associated with high rates of disease recurrence and can cause disfigurement, long-term pain, and psychological distress for the women who undergo the procedure. VIN recurs in approximately one of every two patients who undergo surgical treatment.

This randomized, open label phase 2 study will assess the efficacy of VGX-3100 in 36 women with high-grade HPV-related vulvar lesions. The immunotherapy will be administered with Inovio’s Cellectra intramuscular delivery device. The primary endpoint of the study is histologic clearance of high-grade lesions and virologic clearance of the HPV virus in vulvar tissue samples. The study will also evaluate safety and tolerability of VGX-3100.

Inovio’s immunotherapy aims to address the unmet medical need for VIN by providing a non-surgical option for women with this disease. In a previously conducted phase 2b randomized, placebo controlled study of 167 women with HPV-associated cervical pre-cancer, VGX-3100 led to a significantly higher rate of lesion regression and clearance of the underlying HPV viral infection. Inovio plans to initiate a phase 3 study of VGX-3100 as a treatment for high grade cervical dysplasia in 2017, and the treatment of VIN represent an important additional indication for its lead product.

Dr. Robert Edwards, chair of obstetrics and gynecology at the University of Pittsburgh School of Medicine at Magee-Women’s Hospital and Professor of Medicine in the Department of Obstetrics, Gynecology, and Reproductive Sciences at the University of Pittsburgh, School of Medicine, said, “HPV-induced VIN is one of the major causes of morbidity for young and middle-aged women with HPV-induced pre-cancer. It is associated with repetitive need for surgery, multiple biopsies, and a major cause of pain and sexual dysfunction."

Dr. J. Joseph Kim, Inovio's president and chief executive officer, said, “Inovio’s VGX-3100 may provide the first approved non-surgical treatment option for women with this HPV-related pre-cancer with a high recurrence rate. My optimism is based on our phase 2b efficacy results finding clearance of lesions and the HPV virus in many subjects in our evaluation of VGX-3100 in women with high grade cervical dysplasia.  Our ultimate goal is for VGX-3100 to become the “go-to” immunotherapy to treat all major HPV-related premalignant diseases.”