FDA approves Drug Trial for new drug for Recurrent Respiratory Papillomatosis

PLYMOUTH: The U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application to evaluate its DNA medicine INO-3107 in a Phase 1/2 trial for treatment of Recurrent Respiratory Papillomatosis or RRP. RRP is a rare disease caused by the human papillomavirus (HPV) types 6 and 11 infections, a condition that causes noncancerous tumour growths leading to life-threatening airway obstructions, and occasionally can progress to cancer. Currently, the disease is incurable and is mostly treated by surgery, which temporarily restores the airway. The tumour almost always recurs and the surgery must be repeated, often multiple times a year. RRP can severely impact the quality of life for those living with the disease.

Recurrent respiratory papillomatosis (RRP) is a rare disease (estimated at 15,000 active cases in the U.S.) that is characterized by the growth of tumours in the respiratory tract caused by the human papillomavirus.

We believe this DNA medicine has the potential to provide people living with RRP a long-term, if not life-long, improvement in their disease, especially as an alternative to often successive and debilitating surgeries that may temporarily remove HPV growths from the airways but do not address the underlying recurring virus," said Jeffrey Skolnik, MD, vice president of clinical development at Inovio, in a statement.

The open-label, multicenter Phase 1/2 trial will enroll approximately 63 subjects in the U.S. and will evaluate the efficacy, safety, tolerability, and immunogenicity of INO-3107 in subjects with HPV 6 and/or 11-associated RRP who have required at least two surgical interventions per year for the past three years for the removal of associated papilloma(s). For this study, adult subjects will first undergo surgical removal of their papilloma(s) and then receive four doses of INO-3107, one every three weeks. The primary efficacy endpoint will be a doubling or more in the time between surgical interventions following the first dose of INO-3107 relative to the frequency prior to study therapy. Upon obtaining sufficient safety and potential efficacy data in adults, Inovio plans to expand the trial to include pediatric patients as well as a potential booster regimen.

"Inovio's investigational DNA medicine INO-3107 is designed to destroy and clear tumors caused by HPV 6 and 11 infections from the body exactly where they are hiding," said Jeffrey Skolnik, M.D., Inovio's Vice President of Clinical Development. "We believe this DNA medicine has the potential to provide people living with RRP a long-term, if not life-long, improvement in their disease, especially as an alternative to often successive and debilitating surgeries that may temporarily remove HPV growths from the airways but do not address the underlying recurring virus."

J. Joseph Kim, Ph.D., Inovio's President and Chief Executive Officer, said "Our mission at Inovio is to rapidly provide patients with urgent health needs access to our novel DNA medicines. We are pleased the FDA has authorized our INO-3107 clinical trial, and look forward to working closely with the RRP patient and medical community to drive recruitment as quickly as possible."

In addition to initiating this efficacy trial, Inovio also plans to attain Orphan Disease designation with the FDA's Office of Orphan Products Development (OOPD). The FDA grants orphan status to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. OOPD provides a drug developer with certain benefits and incentives, including a period of marketing exclusivity if regulatory approval is ultimately received for the designated indication.

Inovio recently published data from its pilot clinical study of INO-3106 (DNA medicine candidate targeting HPV6 caused RRP) in the scientific journal Vaccines (MDPI). Study results demonstrated that INO-3106 generated immunogenicity and engagement and expansion of an HPV 6-specific cellular response, including cytotoxic T cells. The paper also showed that Inovio's immunotherapy allowed two out of two patients who previously required approximately two surgeries per year for several years to manage this disease to delay the need for surgery to a robust degree; with one patient able to delay surgery for over a year and a half (584 days surgery-free) and a second that remained surgery-free for over two and a half years (over 915 days surgery-free).