Inovio Pharmaceuticals' INO-3107 Receives FDA Support for Rolling BLA Submission for Rare Lung Disease Drug

Inovio Pharmaceuticals (INO) shares jumped 20.6% after the FDA agreed to a rolling BLA submission for INO-3107, a drug for recurrent respiratory papillomatosis (RRP). The FDA plans to review the application under the Priority Review pathway, aiming for acceptance by year-end 2025. INO-3107 is a DNA medicine candidate designed to elicit a targeted T cell response against HPV-6 and HPV-11. The drug enjoys breakthrough therapy and orphan drug designations in the US.

Inovio Pharmaceuticals (INO) has received a significant boost as the FDA agreed to a rolling Biologics License Application (BLA) submission for INO-3107, a drug designed to treat recurrent respiratory papillomatosis (RRP). The FDA has granted Inovio's request for a rolling submission plan, with the company projecting a completion of the submission within the next few months. The company aims to seek priority review, which could expedite the review process and potentially lead to FDA acceptance by the end of 2025 [1][2].

INO-3107 is a DNA-based immunotherapy that targets HPV-6 and HPV-11, the viruses responsible for RRP. The drug is designed to elicit a targeted T cell response, offering a transformative approach to treating this rare and debilitating disease. Inovio's investigational drug enjoys both breakthrough therapy and orphan drug designations in the United States, further underscoring its potential to address an unmet medical need [1][2].

The FDA's decision to accept the rolling submission plan is a major milestone for Inovio. The company has been working diligently to gather clinical data that demonstrates the efficacy of INO-3107. A completed phase I/II study showed a significant reduction in surgeries for RRP patients, with a 72% reduction after one year and an 86% reduction after two years. These results have been well-received by the FDA, which has advised Inovio against conducting a pivotal phase III study, instead supporting the BLA filing under the accelerated approval pathway [3].

The RRP market, although niche, represents a substantial opportunity. The U.S. market alone is valued at approximately $1.2 billion, with around 1,500 adult patients annually undergoing costly and invasive procedures. INO-3107's potential to reduce hospitalizations and surgical costs could attract payor support, particularly under value-based care models [3].

Inovio's financial position is also robust, with the company holding $68.4 million in cash reserves as of March 2025. This financial stability, combined with the company's strategic focus on the RRP indication, positions Inovio for a potential 2026 launch of INO-3107, pending FDA approval [3].

Investors should closely monitor Inovio's progress in the coming months as the company works towards completing its BLA submission and seeking priority review. The potential for INO-3107 to transform the treatment landscape for RRP patients makes it a compelling investment opportunity, particularly given the high unmet need and the drug's unique mechanism of action.

References:
[1] https://seekingalpha.com/news/4489218-inovio-says-fda-agrees-rolling-bla-submission-rare-disease-asset
[2] https://www.nasdaq.com/articles/ino-stock-soars-fda-backs-rolling-bla-rare-lung-disease-drug
[3] https://www.ainvest.com/news/inovio-pharmaceuticals-strategic-resurgence-driven-ino-3107-rrp-market-opportunity-2508/