Inovio Pharmaceuticals Nears Oct. 30 FDA Decision for INO-3107 in RRP

Key Points

• Inovio faces an FDA decision on October 30 for INO-3107, its DNA medicine for recurrent respiratory papillomatosis (RRP), under the accelerated approval pathway.
• In a 32-patient Phase I/II study, patients saw substantial reductions in repeat surgeries: 72% had at least a 50% reduction in year one and 86% in year two; half required no surgeries in year two.
• The FDA has questioned accelerated-approval eligibility, and Inovio may need a confirmatory trial of roughly 100 patients. The company has prepared commercial partnerships and raised $18.3 million, extending its cash runway into early 2027.

Inovio Pharmaceuticals NASDAQ: INO is approaching an October 30 PDUFA date for INO-3107, its lead DNA medicine candidate for recurrent respiratory papillomatosis, or RRP, President and CEO Jacky Shea said during a Cantor Fitzgerald fireside chat.

Shea described Inovio as a clinical-stage company developing DNA medicines for HPV-related diseases, cancer and infectious diseases. The company’s platform uses plasmid DNA delivered through its proprietary CELLECTRA device, which applies brief electrical pulses to help the DNA enter skin or muscle cells. Once inside cells, the plasmids are transcribed into mRNA and translated into proteins intended to produce sustained protein levels or generate immune responses.

According to Shea, the platform is particularly suited to producing antigen-specific T-cell responses, which the company sees as important in cancer and virally mediated diseases. In the United States, CELLECTRA is regulated as part of a combination product rather than through a separate approval process. The device already has CE marking in the European Union, she said.

RRP Program Targets Repeat Surgery Burden

RRP is caused primarily by HPV types 6 and 11 and can lead to wart-like papilloma growths around the vocal cords, Shea said. The condition can make speaking, swallowing and breathing difficult, while in some cases disease can spread through the respiratory tract and become malignant.

Current care generally relies on surgical removal of papillomas, either through laser procedures or scalpels. However, surgery does not eliminate the underlying virus, Shea said, meaning papillomas can return and patients can face repeat procedures. Those procedures can also cause permanent scarring or damage to vocal cords and respiratory tissues.

Inovio’s Phase I/II study of INO-3107 enrolled 32 patients with confirmed HPV 6- or HPV 11-associated RRP who had undergone two to eight surgeries in the preceding year. Patients received four doses over nine weeks after a clinically required surgery.

• In the first year after treatment, 72% of patients experienced a 50% to 100% reduction in surgeries compared with the prior year, according to Shea.
• That figure increased to 86% in the second year.
• Twenty-eight percent of patients required no surgeries in year one, rising to 50% in year two.

Shea said the trial showed mainly grade 1 and grade 2 adverse events, with few systemic events. She also said patients have reported mild-to-moderate injection-site discomfort from the CELLECTRA administration process that generally resolves within five to 10 minutes.

FDA Review and Potential Confirmatory Trial

INO-3107’s biologics license application was submitted under the accelerated approval pathway. Shea said the FDA’s file acceptance letter included a preliminary conclusion that the application might not be eligible for review under that pathway.

Because an RRP product had previously received full approval, Inovio must demonstrate continuing unmet need and meaningful therapeutic benefit over available treatment to support accelerated approval, Shea said. The company subsequently submitted an assessment aid containing its clinical safety and efficacy data along with additional FDA-requested analyses.

Inovio held an informal clinical meeting with the FDA in July. Shea said company representatives were joined by patient advocacy organizations and a physician treating RRP patients, who discussed the continued unmet need and potential clinical benefit of INO-3107. The FDA did not comment on the review pathway at that time because the review was at a late stage, she said.

The company has completed pre-licensure inspections, receiving one observation that it believes has been resolved. Shea said Inovio expected to enter labeling discussions in September, while noting that the company would also need to confirm the design of any accelerated-approval confirmatory trial with the FDA.

Inovio had previously proposed a placebo-controlled study of about 100 patients, randomized two-to-one between active treatment and placebo. Shea said the study could include year-one and year-two readouts to confirm clinical benefit, although the FDA could request additional patients or endpoints.

Commercial Preparation and Pipeline Plans

Shea said Inovio has made key commercial preparations for a potential launch, including partnerships for specialty distribution, specialty pharmacy, patient support and third-party logistics. The company is also working with Syneos Health for contract sales and medical science liaison deployment.

RRP treatment is concentrated among an estimated 300 to 400 laryngologists at roughly 100 U.S. treatment centers, Shea said. Inovio estimates at least 14,000 potential RRP patients based on claims-database work, though Shea said the company believes the number could be higher.

Beyond INO-3107, Inovio’s clinical pipeline includes INO-3112 for HPV 16- and HPV 18-positive head and neck cancer, partnered with Coherus, and a glioblastoma program partnered with Akeso. The company also has earlier-stage protein-replacement work in hemophilia A, Fabry disease and hypophosphatasia.

At the end of the second quarter, Inovio had $36.7 million in cash. Shea said a subsequent offering generated $18.3 million in net proceeds, extending the company’s cash runway into the end of the first quarter of 2027 and through a potential INO-3107 launch if approved.

About Inovio Pharmaceuticals (NASDAQ:INO)

Inovio Pharmaceuticals is a biotechnology company focused on the discovery, development and commercialization of DNA-based immunotherapies and vaccines aimed at treating and preventing infectious diseases and cancers. The company leverages proprietary technologies to design synthetic DNA sequences that encode antigens capable of eliciting targeted immune responses. Inovio's business activities span early research through clinical development, with a primary emphasis on advancing candidates against viral pathogens such as SARS-CoV-2, human papillomavirus (HPV), HIV, Ebola, Zika and other emerging threats.

Central to Inovio's platform is its SynCon® technology, which constructs optimized DNA plasmids for broad antigen coverage, and the Cellectra® electroporation device, designed to enhance cellular uptake and expression of DNA vaccines.

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