Inovio Pharmaceuticals Spotlights INO-3107 BLA Acceptance, FDA Talks Ahead of Oct. 30 PDUFA Date

Inovio Pharmaceuticals (NASDAQ:INO) used an appearance at Oppenheimer’s 36th Annual Life Science Conference to highlight recent regulatory progress for its lead program, INO-3107, and to outline key issues it is working through with the U.S. Food and Drug Administration (FDA) ahead of an October Prescription Drug User Fee Act (PDUFA) target date.

INO-3107 BLA accepted with October PDUFA date

Chief Executive Officer Jackie Shea said Inovio is a clinical-stage biotechnology company developing DNA medicines for HPV-related diseases, cancer, and infectious diseases. The company’s lead product candidate, INO-3107, is being developed as a treatment for recurrent respiratory papillomatosis (RRP), a rare disease caused by HPV 6 and 11.

Shea said the FDA has accepted Inovio’s biologics license application (BLA) for INO-3107 for review under the Accelerated Approval Program, with a PDUFA target date of October 30 this year and a standard review timeline. She added that INO-3107 has received Orphan Drug and Breakthrough Therapy designations from the FDA, as well as Orphan Drug designation in the European Union.

However, Shea also noted that the FDA’s acceptance letter included preliminary comments related to Inovio’s potential eligibility for the Accelerated Approval pathway. The company has requested a meeting with the agency to discuss those comments and said it is not currently planning to seek approval under the traditional pathway.

RRP market dynamics and INO-3107’s proposed differentiation

Shea described RRP as a serious condition characterized by wart-like growths (papillomas) in the airways, particularly the larynx, which can obstruct breathing, impair swallowing, and affect patients’ voices. She said the disease can spread throughout the respiratory tract, form pulmonary lesions, and in some cases become malignant.

Shea cited “most recent epidemiology estimates” of about 14,000 active RRP cases in the U.S., adding that the standard of care is repeated surgery and that severe cases may require hundreds of procedures over a lifetime. She emphasized that each surgery carries risk, including irreversible vocal cord damage, as well as quality-of-life and financial burdens. Shea said that in patients’ view, reducing surgeries is clinically meaningful, including a reduction of a single procedure.

INO-3107 is designed as an immunotherapy intended to generate antigen-specific cytotoxic T-cell responses against HPV6 and HPV11, targeting the underlying viral cause of RRP, Shea said.

Shea also referenced a competing product approved last year to treat RRP, stating that it does not work for all patients and that Inovio believes a significant unmet need remains. She said Inovio believes it has the potential for a preferred product profile based on company market research conducted by a third party with physicians who treat RRP.

In discussing clinical data shared during the event, Shea highlighted response measures described as reductions in surgeries after treatment compared with the year prior. She cited:

• Overall response rate (defined as a 50%–100% reduction in surgeries): 72% in year one, improving to 86% in year two (the second 12-month period).
• No-surgery outcomes: 28% in year one, improving to 50% in year two.

Shea said INO-3107 was “very well-tolerated,” with predominantly transient injection-site reactions, no discontinuations, and an office-based administration approach. She also said the product does not require ultra-cold chain logistics and that the delivery device can be used by trained healthcare professionals.

FDA pathway discussion: accelerated approval questions and precedent

During the question-and-answer portion, Shea said Inovio was pleased the FDA accepted the BLA for review under the Accelerated Approval Program, but was disappointed the agency did not grant the priority review designation Inovio had requested. Shea said many BLAs accepted under accelerated approval receive priority review, but characterized the FDA’s position as preliminary and “a potential file review issue.”

Shea added that the full approval of Precigen’s “gorilla adenovirus-based product” last year surprised Inovio, noting that it had been filed under the Accelerated Approval pathway. She said Inovio believes the statute and guidance for rare-disease product candidates reflect FDA’s current thinking and said the company looks forward to discussing the review pathway with the agency.

Chief Medical Officer Mike Sumner said Inovio believes the FDA’s comments are related to the prior full approval of the competing product, and said Inovio has been working to demonstrate “meaningful therapeutic benefit over available therapies.” Sumner outlined areas Inovio believes support accelerated approval eligibility, including comparable efficacy, an improved safety profile tied to not requiring certain surgeries during the dosing window, and the potential to treat patients not served by existing therapy.

Shea said the FDA requested an “Assessment Aid” for the upcoming meeting; Inovio submitted it using data from its BLA filings and reiterated the arguments it included in the original submission. The company said it is waiting for the FDA to schedule the meeting and expects it to occur in a timely fashion.

Trial design updates, redosing strategy, and pipeline priorities

Sumner said Inovio’s original confirmatory study design for INO-3107 was planned as placebo-controlled, but that following the competitor’s approval, Inovio believes the FDA has shifted to accept a single-arm study as adequate to support registration in RRP. He said the company submitted an update to its IND with a revised confirmatory trial design and expects feedback “fairly soon.” He added that additional phase III design features will depend in part on FDA feedback regarding the accelerated approval pathway and confirmatory trial expectations.

On Europe, Sumner said Inovio obtained clinical advice from CHMP in Europe and U.K. regulators indicating that approval would require data from two placebo-controlled trials to demonstrate efficacy and meet safety database expectations. He noted what he described as a “disconnect” between that feedback and Precigen’s decision to file a marketing authorization application based on single-arm data, while stating Inovio could only speak to its own interactions with regulators.

Shea also contrasted Inovio’s DNA plasmid approach with adenoviral vector approaches, saying INO-3107 does not include a viral vector that could generate anti-vector antibodies or face reduced efficacy in patients with pre-existing neutralizing antibodies. Sumner said Inovio is considering a redosing strategy and, “hopefully following approval,” would submit a protocol to begin annual redosing to maintain cytotoxic T-cell responses in a chronic, lifelong disease.

On commercial opportunity, Shea reiterated the 14,000–15,000 active case estimate in the U.S. and said Inovio believes claims database analysis suggests this could be an underestimate. She also cited the competitor’s pricing as about $115,000 per dose, or $460,000 per treatment regimen, describing the pricing environment as consistent with rare-disease commercialization.

Beyond INO-3107, Shea said Inovio is focused primarily on obtaining approval for the lead program, while seeking to advance other clinical candidates through partnerships. She mentioned INO-3112 (with plans to start a phase III trial), INO-5401 (with plans to start a randomized phase II trial in glioblastoma), and earlier-stage programs tied to the company’s dMAb platform. Shea said Inovio presented first preclinical data for a dMAb candidate producing factor VIII at the World Federation of Hemophilia Global Forum late last year, and expects to present additional preclinical data later this year.

In closing remarks, Shea said the company remains focused on the October PDUFA date and is responding to routine information requests from the FDA while the application is under active review.

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.