US FDA grants breakthrough therapy designation to Inovios INO-3107 to treat recurrent respiratory papillomatosis

Inovio, a biotechnology company focused on developing and commercializing DNA medicines to help treat and protect people from HPV-related diseases, cancer, and infectious diseases, announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for INO-3107 as a potential treatment for patients with Recurrent Respiratory Papillomatosis (RRP).

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).

"This is yet another important step for INO-3107 and recognition that this first-in-class DNA medicine candidate has the potential to improve the lives of patients with RRP," said Inovio's president and chief executive officer, Dr. Jacqueline Shea. "As we recently announced, we have been interacting with the FDA with the goal to launch a pivotal trial for INO-3107 in the near term. With this Breakthrough Therapy designation, we look forward to continuing to work with the agency so that we can generate the evidence needed to support approval of INO-3107 as quickly and efficiently as possible, with an ultimate aim to help RRP patients and deliver on the promise of DNA medicine."

The president of the Recurrent Respiratory Papillomatosis Foundation, Kim McClellan, said: "RRP patients will tell you that even one reduction in the number of disruptive, invasive surgeries they face would be life-changing. The potential impact of this treatment gives me great hope for the future and I'm happy to see that RRP is finally getting the attention it deserves."

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.

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.

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 (NCT:04398433). Overall, 81.3% (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% (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 (95% confidence interval -3, -2). 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.

Data from this phase 1/2 trial has been presented at scientific and medical conferences, including the 2023 Annual Meeting of the American Broncho-Esophageal Association (ABEA) in May and at the European Laryngological Association's Annual Meeting in June. Data from the trial was also published in May in the peer-reviewed journal, The Laryngoscope, under the title "Interim Results of a Phase 1/2 Open-Label Study of INO-3107 for HPV-6 and/or HPV-11–Associated RRP." The Laryngoscope is the official journal of the Triological Society (TRIO), the American Laryngological Association (ALA), and the ABEA.

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 US 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 US each year.

INO-3107 is Inovio's clinical-stage DNA medicine product candidate being developed as a potential treatment for RRP. INO-3107 is designed to elicit a targeted T cell response against HPV-6 and HPV-11, the HPV types responsible for causing RRP among other HPV-related diseases. These targeted T cells are designed to seek out and kill infected cells, with the aim of potentially preventing or slowing the growth of new papillomas. INO-3107 received Orphan Drug designation from the European Commission in May 2023 and from the US Food and Drug Administration in 2020.

Inovio is a biotechnology company focused on developing and commercializing DNA medicines to help treat and protect people from HPV-related diseases, cancer, and infectious diseases.

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