INOVIO Advances DNA Medicine Pipeline with Focus on INO-3107 BLA Submission for RRP
核心洞察
INOVIO plans to submit a Biologics License Application (BLA) to the FDA for INO-3107 by mid-2025, seeking priority review for recurrent respiratory papillomatosis (搜索).
Retrospective data showed that 50% of RRP patients treated with INO-3107 achieved complete response, requiring no surgery two to three years post-treatment.
INOVIO aims to initiate a confirmatory trial for INO-3107 at approximately 20 U.S. academic centers, enrolling around 100 patients.
INOVIO Pharmaceuticals is gearing up for a potentially transformative year in 2025, with a key focus on advancing its DNA medicine pipeline. The company's lead candidate, INO-3107, is on track for a Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) by mid-2025, marking a significant step toward potential commercialization. INO-3107 is being developed as a non-surgical therapeutic option for recurrent respiratory papillomatosis (搜索) (RRP). If approved, it would be the first DNA medicine approved for any indication in the United States.
INO-3107: Targeting Recurrent Respiratory Papillomatosis
INO-3107 is designed to treat recurrent respiratory papillomatosis (搜索) (RRP), a rare disease characterized by the growth of papillomas in the respiratory tract, often requiring multiple surgeries. The planned confirmatory trial will enroll approximately 100 patients at around 20 leading U.S. academic centers, randomized 2:1 to receive either INO-3107 or placebo. Patients eligible for the trial will have a history of two or more surgeries per year to manage their RRP, representing a broad spectrum of the disease. INOVIO anticipates that the trial design could support expansion into global markets, based on feedback from European and UK regulators.
Durability and Immunogenicity Data
Recent durability data supports the rationale for redosing patients with INO-3107, with the goal of maintaining or improving clinical benefit. INOVIO plans to submit a redosing study protocol to the FDA. The company also intends to present and publish recently announced durability and immunology data, along with full efficacy and tolerability data from the completed Phase 1/2 clinical trial, in a peer-reviewed scientific journal.
Data from a retrospective trial indicated that half of the RRP patients treated with INO-3107 achieved a complete response (CR) and required no surgery when evaluated at the end of year two and into year three after the initial Phase 1/2 trial, increasing from the initial CR rate of 28% at the end of the first year. Of those patients who had an initial CR, 88% maintained their CR by end of the second year and 63% into the third year. 95% of patients maintained or enhanced their original Overall Response Rate (“ORR,” defined as those patients who achieved either a CR or a partial response) after two years, while 86% of patients maintained or enhanced their ORR into year three.
New immunology data demonstrated INO-3107's ability to induce antigen-specific T cell responses against HPV (搜索)-6 and HPV-11 and drive recruitment of those T cells into airway tissues and papilloma of RRP patients, which could potentially slow or eliminate papilloma regrowth. The Phase 1/2 trial data showed that INO-3107 was well-tolerated, with no treatment-related serious adverse events. The most commonly reported adverse events were injection site pain (31%) and fatigue (9%). Of the 32 patients in the trial, 26 patients, or 81%, experienced a decrease in the number of surgical interventions in the year after treatment when compared to the year before treatment.
Advancing Other Pipeline Programs
In addition to INO-3107, INOVIO is also advancing other pipeline programs. For INO-3112, the company aims to gain alignment on the planned Phase 3 trial design with European Union regulators. This trial, conducted in collaboration with Coherus BioSciences, will evaluate the combination of INO-3112 and LOQTORZI® (toripalimab-tpzi) in patients with locoregionally advanced, high-risk, HPV16/18-positive oropharyngeal squamous cell carcinoma (搜索) (OPSCC).
INOVIO is also working on INO-4201, a potential booster to ERVEBO®, and plans to finalize Phase 2 trial protocols and seek funding to support trial activities. Furthermore, the company expects to report first clinical data from its ongoing Phase 1 trial of a DNA-encoded monoclonal antibody (DMAb) targeting SARS-CoV-2 (搜索) in the first quarter of 2025. INOVIO's partner, ApolloBio (搜索), continues recruitment into its Phase 3 trial evaluating INO-3100 (搜索) as a potential treatment for HPV 16/18 (搜索) positive cervical dysplasia (搜索) in China.
Corporate Updates
INOVIO has strengthened its balance sheet with total net proceeds of more than $60 million from two offerings of equity securities in April and December 2024. The company also announced the appointment of Steven Egge as Chief Commercial Officer in July 2024, bringing extensive experience in HPV (搜索)-related diseases, cancers, vaccines, and rare diseases.