INO-3107: Potential Breakthrough Treatment for RRP in Adults

Overview

• INO-3107 is an investigational DNA immunotherapy that is designed to teach the body to mount an effective immune response to HPV-6 and HPV-11, the strains of HPV that cause Recurrent Respiratory Papillomatosis (RRP).
• In a Phase 1/2 trial (RRP-001), 72% of patients saw a 50-to-100% reduction in the number of surgeries in the first year after starting treatment with INO-3107. INO-3107 was well tolerated, with most patients experiencing mostly low-grade (Grade 1) treatment-emergent Adverse Events such as injection site pain and fatigue.
• In a retrospective trial (RRP-002) involving 28 of the participants in the original RRP-001 trial, 86% of patients experienced a 50-to-100% reduction in surgeries in the second year with no additional dosing. Half of those patients required no surgeries at all.
• Published immunological data showed that INO-3107 induced new populations of antigen specific cytotoxic T cells in the blood that traveled to airway tissue and were associated with clinical benefit as measured by reduced need for surgeries.

Trial Details and Regulatory Designation

• The FDA accepted for review the Biologics License Application (BLA) for INO-3107 in December 2025 under the accelerated approval program, setting a Prescription Drug User Fee Act (PDUFA) target action date for October 30, 2026.
• INO-3107 has been granted Breakthrough Therapy and Orphan Drug designations from the FDA, as well as Orphan Drug designation in Europe and the Innovation Passport in the UK.
• Phase 1/2 trial details can be found on clinicaltrials.gov: NCT04398433

Estimated Market Opportunity

• According to most recent available data, RRP is believed to be prevalent in approximately 14,000 people in the U.S. with an estimated 1.8 new cases a year for every 100,000 people.

Morrow, M.P., Gillespie, E., Sylvester, A. et al. DNA immunotherapy for recurrent respiratory papillomatosis (RRP): phase 1/2 study assessing efficacy, safety, and immunogenicity of INO-3107. Nat Commun 16, 1518 (2025). https://doi.org/10.1038/s41467-025-56729-6

M. R. Amin, P. C. Belafsky, S. R. Best, et al., “ DNA Immunotherapy (INO-3107) Results in Long-Term Surgery Reduction in RRP,” The Laryngoscope (2025): 1–9, https://doi.org/10.1002/lary.32416.