INOVIO announced that its lead DNA medicine candidate, INO-3107, has been designated an innovative medicine as part of the U.K.'s Innovative Licensing and Access Pathway (ILAP). The designation, called an Innovation Passport, was granted by the ILAP Steering Group to INO-3107 for the treatment of patients with Recurrent Respiratory Papillomatosis (RRP), a debilitating, chronic rare disease of the respiratory tract caused by HPV-6 and/or HPV-11. The Innovation Passport is the entry point to the ILAP, which aims to accelerate time to market and facilitate patient access to medicines in the United Kingdom.

The Innovation Passport provides a single, streamlined roadmap for regulatory approval and development milestones. Recipients of the Innovation Passport are granted access to a range of development tools to support the design, development and approvals process in the U.K., as well as opportunities for enhanced regulatory and other stakeholder input. Specific benefits of ILAP include the potential for a 150-day accelerated Marketing Authorization Application (MAA) assessment, rolling review and a continuous benefit risk assessment.

The ILAP is delivered in partnership by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), the All Wales Therapeutics and Toxicology Centre, the National Institute for Health and Care Excellence and the Scottish Medicines Consortium, part of Healthcare Improvement Scotland. The MHRA launched ILAP at the start of 2021 to accelerate the development and access to promising medicines in the early stages of development. The pathway is part of the UK's plan to attract life sciences development in the post-Brexit era.

INO-3107 is designed to elicit an antigen-specific T cell response against both HPV-6 and HPV-11 proteins. These targeted T cells are designed to seek out and kill HPV-6 and HPV-11 infected cells, with the aim of potentially preventing or slowing the growth of new papillomas. In a Phase 1/2 clinical trial conducted with INO-3107, 81.3% (26/32) of patients 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). At the outset of the study (Day 0), patients had a clinically warranted procedure to have RRP tissue surgically removed, but any surgery performed after Day 0 during the dosing window was counted against the efficacy endpoint.

Treatment with INO-3107 generated a strong immune response in the trial, inducing activated CD4 T cells and activated CD8 T cells with lytic potential. T-cell responses were also observed at Week 52, indicating a persistent cellular memory response. INO-3107 was well tolerated by participants in the trial, resulting in mostly low-grade (Grade 1) treatment-emergent adverse effects such as injection site pain and fatigue.

Like other DNA medicines, INO-3107 has the ability to generate antigen-specific T cells that is not affected by anti-vector immunity impacting immunogenicity, either before administration or after the first dose unlike other T-cell generating platforms such as viral vectors. This feature of DNA medicines should allow INO-3107 to maintain T cell response and overall efficacy, which would make it an important therapeutic option for a majority of RRP patients. The FDA granted INO-3107 Orphan Drug designation and Breakthrough Therapy designation, and advised INOVIO that it could submit its BLA under the accelerated approval program using data from its already completed Phase 1/2 trial.

The European Commission has also granted INO-3107 Orphan Drug designation. INOVIO's delivery device, CELLECTRA®, received CE marking, a regulatory standard that certifies that a product has met European Union's safety, health, and environmental standards.