Inovio OKd on Phase 1/2 Trial for DNA Medicine for Rare Disease

• Respiratory Papillomatosis (RRP) is a rare, potentially fatal orphan disease caused by Human Papillomavirus (HPV) 6 and 11
• Inovio’s DNA medicine pipeline includes 15 clinical programs focused on HPV-associated diseases, cancer, and infectious diseases, including the novel coronavirus (2019-nCoV)

Inovio Pharmaceuticals, Inc. (NASDAQ: INO) today announced 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 tumor 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 tumor 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.
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.”
https://www.biospace.com/article/releases/inovio-receives-authorization-from-the-u-s-fda-to-begin-phase-1-2-clinical-trial-for-ino-3107-a-dna-medicine-to-treat-a-rare-disease-recurrent-respiratory-papillomatosis-rrp-/