Inovio Pharmaceuticals (NASDAQ:INO) said its biologics license application for INO-3107, an investigational DNA medicine for recurrent respiratory papillomatosis, or RRP, remains under U.S. Food and Drug Administration review, with a target action date of Oct. 30.
President and CEO Jacqueline Shea said the clinical-stage biotechnology company is in the late stages of the regulatory process for INO-3107 under the FDA’s Accelerated Approval Program. The company has completed its late-cycle review meeting and scheduled FDA inspections, she said. Inovio expects label negotiations with the agency to begin in September.
Repeated surgery remains the standard treatment because surgical procedures remove growths but do not address the underlying viral infection, Shea said. Some patients may require hundreds of surgeries over their lifetimes, and procedures can carry risks including irreversible vocal-cord damage.
Clinical Data and Regulatory Discussion
INO-3107 was designed to generate an antigen-specific T-cell response against HPV types 6 and 11. The company’s BLA is supported by its Phase I/II RRP-001 study and the RRP-002 follow-up study, which tracked patients after treatment without providing additional doses.
In RRP-001, enrolled patients had required at least two surgeries to manage their disease in the previous year. Patients received four doses of INO-3107 after entering the study following a clinically required surgery, and any surgery needed after the initial treatment date, including during the dosing period, was counted toward the study endpoint.
Shea said 72% of patients achieved a 50% to 100% reduction in surgeries during the first year after treatment compared with the prior year. That proportion rose to 86% in the second year. Meanwhile, 28% of patients required no surgeries in the first 12 months after treatment, increasing to 50% in the second 12 months. The extension study produced a median follow-up period of 2.8 years.
During a July informal clinical meeting with the FDA, Inovio presented its safety and efficacy data, its approach to RRP treatment and its rationale for accelerated approval eligibility, Shea said. The FDA did not discuss a preliminary comment in the file acceptance letter concerning accelerated approval eligibility, according to Shea, but indicated that feedback on the design of a confirmatory trial would be forthcoming.
The company said it believes INO-3107 could offer a differentiated profile because its clinical trial did not require surgeries during the dosing window to maintain minimal residual disease. Shea also said the DNA-based approach is not affected by pre-existing neutralizing antibodies and may avoid limitations associated with an immunosuppressive papilloma microenvironment.
Commercial Planning and Cash Runway
As it approaches the FDA decision date, Inovio is preparing for a potential commercial launch. Shea said the company plans to use experienced field teams alongside a contract sales organization, a targeted marketing campaign and a patient-support hub. The company also intends to pursue access among payers and hospital systems and educate patients and caregivers about INO-3107’s potential profile.
Inovio said it has sufficient cash runway into late in the first quarter of next year, including through a potential INO-3107 launch.
Pipeline Partnerships
Beyond INO-3107, Shea highlighted several partnered programs. In China, Inovio partner ApolloBio reported that its pivotal Phase III study of VGX-3100 for cervical dysplasia met its primary endpoint. ApolloBio plans to use the data to potentially seek approval in China and related territories. If approved, Inovio could receive regulatory and sales-based milestone payments as well as royalties, Shea said.
Inovio also recently partnered with Akeso to evaluate INO-5401 with Akeso’s bispecific checkpoint inhibitor in glioblastoma. The combination is planned for evaluation in the Dana-Farber Cancer Institute-sponsored INSIGhT trial. The companies aim to assess whether the regimen can improve survival and delay disease progression in patients with the brain cancer.
Shea additionally discussed Inovio’s earlier-stage dMAb and dProt platforms, which are intended to produce antibodies or therapeutic proteins directly within the body. In a Phase I trial involving SARS-CoV-2 antibodies, the company said it produced two monoclonal antibodies at potentially therapeutic levels for up to 96 weeks, with stable blood levels, no observed anti-drug antibodies and injection-site reactions as the most common side effect.
The company has also presented preclinical hemophilia A data and disclosed work in Fabry disease and hypophosphatasia. Inovio is seeking partners to advance those rare-disease programs and other platform opportunities.
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.
