RARE Daily

FDA Grants Breakthrough Therapy Designation for Inovio’s Treatment for Recurrent Respiratory Papillomatosis

September 7, 2023

Rare Daily Staff

The U.S. Food and Drug Administration has granted Breakthrough Therapy designation for Inovio’s INO-3107 as a potential treatment for patients with recurrent respiratory papillomatosis.

The FDA’s Breakthrough Therapy designation is a process designed to expedite the development and review of drug candidates that are intended to treat a serious or life-threatening condition and for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).

Recurrent respiratory papillomatosis (RRP) is a debilitating and rare disease caused primarily by HPV-6 and/or HPV-11. RRP is characterized by the development of small, wart-like growths, or papillomas, in the respiratory tract. While papillomas are generally benign, they can cause severe, life-threatening airway obstruction and respiratory complications. RRP can also significantly affect quality of life for patients by affecting the voice box, limiting the ability to speak effectively. Surgery to remove papillomas is the standard of care for RRP; however, the papillomas often grow back because the underlying HPV infection has not been eradicated. The most widely cited U.S. epidemiology data published in 1995 estimated that there were 14,000 active cases and about 1.8 per 100,000 new cases in adults each year. More recent pediatric epidemiology data cites a range of 0.5 – 0.7 per 100,000 new cases in children in the U.S. each year.

INO-3107 is an investigational DNA medicine candidate designed to elicit a targeted T cell response against HPV-6 and HPV-11, the HPV types that cause RRP and other HPV-related disease. This Breakthrough Therapy designation for INO-3107 follows receipt of Orphan Drug designation from the European Commission in May 2023 and from the FDA in 2020.

The Breakthrough Therapy designation is supported by data from Inovio’s completed phase 1/2 open-label, multicenter trial that assessed INO-3107’s safety, tolerability, immunogenicity, and efficacy in patients with HPV-6 and/or HPV-11-related RRP. Overall, 81.3 percent (26/32) patients in the trial had a decrease in surgical interventions in the year after INO-3107 administration compared to the prior year, including 28.1 percent (9/32) that required no surgical intervention during or after the dosing window. Patients in the trial had a median range of 4 surgeries (2-8) in the year prior to dosing. After dosing, there was a median decrease of 3 surgical interventions. Patients received four doses of INO-3107 on Day 0, and Weeks 3, 6, and 9. At the outset of the study (Day 0), patients could have RRP tissue surgically removed, but any surgery performed after Day 0 during the dosing window was counted against the efficacy endpoint. INO-3107 was well-tolerated by participants in the trial.

Inovio plans to initiate a pivotal trial of INO-3107 in the first quarter of 2024, subject to FDA clearance. As part of its development efforts, the company has engaged a leading clinical research organization to help run the pivotal trial, as well as key opinion leaders and investigators interested in developing a new treatment option for RRP patients.

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