INOVIO Completes Rolling BLA Submission Seeking Accelerated Approval for INO-3107 as a Treatment for RRP in Adults

Rhea-AI Summary

INOVIO (NASDAQ: INO) completed the rolling submission of a Biologics License Application (BLA) for INO-3107, a DNA immunotherapy for recurrent respiratory papillomatosis (RRP) in adults, submitted under the FDA's Accelerated Approval program.

The program includes prior Orphan Drug and Breakthrough Therapy designations. The company expects FDA file acceptance by year-end 2025 and, if priority review is granted, a potential PDUFA date in mid-2026. If approved, INO-3107 would be INOVIO's first commercial product and the first DNA medicine available in the United States.

Positive

• Completed rolling BLA submission for INO-3107
• INO-3107 has Orphan Drug designation
• INO-3107 has Breakthrough Therapy designation
• If approved, would be company's first commercial product

Negative

• FDA acceptance expected by year-end 2025, not yet confirmed
• Potential PDUFA mid-2026 is conditional on priority review being granted
• Approval and commercial launch remain subject to FDA review

Insights

Regulatory/Commercial Expert

Completion of a rolling BLA for INO-3107 advances INOVIO toward a potential first U.S. approval and its first commercial product.

INO-3107 is a DNA immunotherapy submitted under the FDA’s Accelerated Approval pathway with a priority review request; acceptance is expected by year-end 2025 and a possible PDUFA in mid-2026. If approved under Accelerated Approval, this would enable earlier market access for a therapy designated as both Orphan Drug and Breakthrough Therapy, positioning the product as a novel option for adults with recurrent respiratory papillomatosis.

Key dependencies include formal FDA file acceptance, the grant (or denial) of priority review, and the Agency’s assessment of the Accelerated Approval requirements. The submission’s impact hinges on regulatory timelines and any remaining review issues; these are the principal execution risks stated. Watch for the FDA’s 60-day filing decision by year-end 2025 and any announcement of a PDUFA date in mid-2026, as those milestones will materially change near-term regulatory visibility and commercialization planning.

• Recurrent respiratory papillomatosis (RRP) is a rare HPV-related disease of the respiratory tract with significant unmet need
• INO-3107 previously received Orphan Drug and Breakthrough Therapy designations; BLA submitted under FDA's Accelerated Approval program
• Expect to receive file acceptance by year end 2025 with potential PDUFA date in mid-2026 if request for priority review granted

PLYMOUTH MEETING, Pa., Nov. 3, 2025 /PRNewswire/ -- INOVIO (NASDAQ: INO), a biotechnology company focused on developing and commercializing DNA medicines to help treat and protect people from HPV-related diseases, cancer, and infectious diseases, today announced that it has completed the rolling submission of its Biologics License Application (BLA) for its DNA immunotherapy candidate INO-3107 for the treatment of RRP in adults.

INOVIO submitted the BLA under the FDA's Accelerated Approval program and has requested a priority review, which if granted, is expected to be completed within six months following the 60-day filing period. If approved, INO-3107 would be INOVIO's first commercial product and the first DNA medicine available in the United States.

"The potential to have a meaningful new treatment for RRP brings me so much hope for the RRP community, which has been desperate for relief from the risks and costs of repeated surgery," said Kim McClellan, President of the RRP Foundation. "Every patient deserves a therapy that works for them and I believe we are now one step closer to surgery being a last resort for the treatment of this disease."

"This is a pivotal moment in our efforts to deliver on the promise of INO-3107, an innovative DNA immunotherapy candidate that has the potential to become a paradigm-shifting treatment option for RRP," said Dr. Michael Sumner, Chief Medical Officer of INOVIO. "I'd like to thank the patients and physicians who participated in the INO-3107 clinical trial, as well as our internal team for their tremendous effort in completing INOVIO's first BLA submission. We look forward to continued communication and collaboration with the FDA during the review process and will be focused on finalizing our preparations for a potential commercial launch in 2026."

About RRP
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. INOVIO's market research to date with patients and healthcare professionals indicates that a reduction of even one surgery matters, because every surgery poses a significant risk of causing permanent damage to the vocal cords and comes with potential costs to the patient, including adverse impacts to both quality of life and finances. The most widely cited U.S. epidemiology data published in 1995 estimated that there were 14,000 active cases and about 1.8 per 100,000 new cases of RRP in adults each year.  

About INO-3107
INO-3107 is an investigational DNA medicine designed to elicit an antigen-specific T cell response against both HPV-6 and HPV-11 proteins. These targeted T cells 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 trial of 32 participants (RRP-001), 72% of patients saw a 50-to-100% reduction in the number of surgeries after starting treatment with INO-3107 at the end of the first year. A retrospective study involving 28 of the original trial participants (RRP-002) showed this number increasing to 86% at the end of the second 12-month period with no additional dosing. Half of those patients required no surgeries at all. Patients in RRP-001 had a median of 4 surgeries (range: 2-8) in the year prior to dosing. At the outset of the trial (Day 0), patients had a clinically warranted procedure to have papillomas surgically removed, but any surgery performed after Day 0 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, with trial participants experiencing mostly low-grade (Grade 1) treatment-emergent adverse effects such as injection site pain and fatigue. Like other DNA medicines, INO-3107 has shown 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 is anticipated to allow INO-3107 to maintain T cell response and overall efficacy, which could make it an important therapeutic option for a majority of RRP patients.

The FDA has granted INO-3107 both Orphan Drug and Breakthrough Therapy designations and previously advised INOVIO that it could submit a BLA under the FDA's accelerated approval program using data from INOVIO's completed Phase 1/2 trial. The European Commission granted INO-3107 Orphan Drug designation. In addition, INOVIO has CE-marked its CELLECTRA^® delivery device in the EU, which allows INOVIO to commercialize the device in the EU and other geographies that recognize CE-marking. The United Kingdom awarded INO-3107 the Innovation Passport. This designation serves as the entry point to the Innovative Licensing and Access Pathway (ILAP), which aims to accelerate time to market and facilitate patient access to medicines.

About INOVIO's DNA Medicines Platform
INOVIO's DNA medicines platform has two innovative components: precisely designed DNA plasmids, delivered by INOVIO's proprietary investigational medical device, CELLECTRA. INOVIO uses proprietary technology to design its DNA plasmids, which are small circular DNA molecules that work like software the body's cells can download to produce specific proteins to target and fight disease. INOVIO's proprietary CELLECTRA delivery devices are designed to optimally deliver its DNA medicines to the body's cells without requiring chemical adjuvants or lipid nanoparticles and without the risk of the anti-vector response historically seen with viral vector platforms.

About INOVIO
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. INOVIO's technology optimizes the design and delivery of innovative DNA medicines that teach the body to manufacture its own disease-fighting tools. For more information, visit www.inovio.com.

Forward-Looking Statements
This press release contains certain forward-looking statements relating to our business, including the request for priority review for the BLA submission and goal of FDA's acceptance of the submission by the end of 2025, as well as the potential benefits of INO-3107. Actual events or results may differ from the expectations set forth herein as a result of a number of factors, including uncertainties inherent in pre-clinical studies, clinical trials, product development programs and commercialization activities and outcomes, the availability of funding to support continuing research and studies in an effort to prove safety and efficacy of electroporation technology as a delivery mechanism or develop viable DNA medicines, our ability to support our pipeline of DNA medicine products, the ability of our collaborators to attain development and commercial milestones for products we license and product sales that will enable us to receive future payments and royalties, the adequacy of our capital resources, the availability or potential availability of alternative therapies or treatments for the conditions targeted by us or collaborators, including alternatives that may be more efficacious or cost effective than any therapy or treatment that we and our collaborators hope to develop, issues involving product liability, issues involving patents and whether they or licenses to them will provide us with meaningful protection from others using the covered technologies, whether such proprietary rights are enforceable or defensible or infringe or allegedly infringe on rights of others or can withstand claims of invalidity and whether we can finance or devote other significant resources that may be necessary to prosecute, protect or defend them, the level of corporate expenditures, assessments of our technology by potential corporate or other partners or collaborators, capital market conditions, the impact of government healthcare proposals and other factors set forth in our Annual Report on Form 10-K for the year ended December 31, 2024, our Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, and other filings we make from time to time with the Securities and Exchange Commission. There can be no assurance that any product candidate in our pipeline will be successfully developed, manufactured, or commercialized, that the results of clinical trials will be supportive of regulatory approvals required to market products, or that any of the forward-looking information provided herein will be proven accurate. Forward-looking statements speak only as of the date of this release, and we undertake no obligation to update or revise these statements, except as may be required by law.

Contacts
Media: Jennie Willson, (267) 429-8567, communications@inovio.com
Investors: Peter Vozzo - ICR Healthcare, (443) 213-0505, investor.relations@inovio.com 

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SOURCE INOVIO Pharmaceuticals, Inc.

FAQ

What did INOVIO (INO) announce on November 3, 2025 about INO-3107?

INOVIO announced completion of a rolling BLA submission for INO-3107 for adult RRP under the FDA Accelerated Approval program.

Does INO-3107 have any FDA designations for RRP?

Yes. INO-3107 previously received both Orphan Drug and Breakthrough Therapy designations.

When does INOVIO expect FDA file acceptance and a possible PDUFA date for INO-3107?

INOVIO expects file acceptance by year-end 2025 and a potential PDUFA date in mid-2026 if priority review is granted.

Would INO-3107 be a notable product for INOVIO if approved?

If approved, INO-3107 would be INOVIO's first commercial product and the first DNA medicine available in the United States.

What regulatory pathway did INOVIO use for the INO-3107 BLA submission?

The BLA for INO-3107 was submitted under the FDA's Accelerated Approval program with a request for priority review.