Inovio's INO-3107 Clears FDA Review Gate as Papillomatosis Treatment Advances Toward Approval

Inovio Pharmaceuticals has achieved a significant regulatory milestone with the FDA’s formal acceptance of its Biologics License Application for INO-3107, a novel therapeutic approach to recurrent respiratory papillomatosis—a rare disease characterized by recurring benign growths in the airway. The regulatory agency has set an October 30, 2026 target date for its decision and signaled it does not intend to hold an advisory committee hearing at this stage.

The move arrives with a notable caveat: the FDA indicated in its acceptance letter that Inovio may need to provide supplementary evidence to justify the accelerated approval pathway the company is pursuing. This conditional stance immediately pressured the stock, which dropped 19.21% in pre-market trading to $1.85, reflecting investor concerns about potential roadblocks ahead. Over the past year, INO shares have ranged between $1.30 and $2.98.

The Clinical Case: Why This Matters for Patients

Recurrent respiratory papillomatosis stems primarily from HPV-6 and HPV-11 infection, creating a cycle of airway obstruction and repeated surgical interventions. Current treatment relies almost entirely on surgery—the standard of care—but the condition’s defining characteristic is precisely its recurrence. Patients endure multiple procedures annually, each carrying risks to vocal cord integrity and overall quality of life.

INO-3107 represents a departure from this surgical treadmill. The therapy is engineered to trigger an antigen-specific T-cell immune response directed against HPV-6 and HPV-11-infected cells, potentially halting or delaying papilloma regrowth at the immunological level.

Phase 1/2 Data: The Proof Point

Clinical evidence supporting the BLA submission centered on patients with documented surgical histories—specifically, those requiring at least two procedures in the preceding year. The results are noteworthy:

• Year one: 72% of treated patients saw a 50-100% reduction in required surgeries
• Year two: 86% of evaluable patients sustained these clinical gains without additional dosing; notably, half underwent no surgeries at all

These outcomes were published in peer-reviewed journals, including Nature Communications and The Laryngoscope, lending third-party validation to the findings.

Safety data proved reassuring, with adverse events predominantly low-grade—injection-site discomfort and fatigue being the most commonly reported—establishing a manageable tolerability profile.

Regulatory Tailwinds and Financial Realities

INO-3107 carries both Orphan Drug and Breakthrough Therapy designations in the U.S., designations intended to expedite development and review of treatments for rare conditions. The accelerated approval pathway represents another advantage, though the FDA’s caveat suggests this status is not guaranteed to survive the formal review.

The company’s balance sheet adds temporal urgency to the approval timeline. As of September 30, 2025, Inovio held $50.8 million in cash and short-term investments—sufficient to sustain operations into Q2 2026, roughly aligned with the FDA’s target decision date.

Inovio management intends to meet with FDA officials to clarify the agency’s concerns and chart the remaining regulatory course. Investor confidence appears conditional: the market is now pricing in execution risk alongside the therapeutic opportunity.