The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to INO-3107 for the treatment of recurrent respiratory papillomatosis.

Recurrent respiratory papillomatosis (RRP) is caused primarily by human papillomavirus (HPV) types 6 and 11. It is characterized by the development of small, noncancerous papillomas in the respiratory tract that can lead to life-threatening airway obstructions. INO-3107 is a DNA plasmid immunotherapy designed to elicit a targeted T-cell response against HPV-6 and HPV-11.

The Breakthrough Therapy designation was based on data from a phase 1/2 open-label trial ( Identifier: NCT04398433) that evaluated the safety, tolerability, immunogenicity, and efficacy of INO-3107 in patients with HPV-6 and/or HPV-11-associated RRP (N=32). Study participants had a median range of 4 surgeries (range, 2-8) in the year before receiving INO-3107. Treatment was administered as an intramuscular injection followed by electroporation at day 0 and weeks 3, 6, and 9 (4 doses in total).

Findings showed that 81.3% of patients had a decrease in surgical interventions in the year after administration compared with the prior year, with 28.1% requiring no surgical intervention during or after the treatment period. Compared with the prior year, a median decrease of 3 surgical interventions (95% CI, -3, -2) was observed after dosing.  

Immunogenicity data showed that INO-3107 induced cellular responses against HPV 6 and HPV 11, activating both CD4+ and CD8+ T cells, including cytotoxic CD8 cells; these responses were still observed at week 52. The treatment was found to be well tolerated, with injection site pain, fatigue, and headache being the most commonly reported adverse events.

“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.”

INO-3107 previously received the FDA’s Orphan Drug designation for this indication.

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