Clinical and Immunological Results from Phase 1/2 Study of INO-3107 as a Treatment for Recurrent Respiratory Papillomatosis Published in Nature Communications

Core Insights - INOVIO's INO-3107 shows promise as a treatment for recurrent respiratory papillomatosis (RRP), with clinical trial data indicating significant reductions in the need for surgeries among patients [1][2][3] - The Phase 1/2 trial results were published in Nature Communications, highlighting the immunological impact and efficacy of INO-3107 [1][2] - INOVIO plans to submit a biologics license application (BLA) for INO-3107 in mid-2025, aiming for accelerated approval from the FDA [1][6] Clinical Trial Results - 81.3% of patients (26 out of 32) experienced a decrease in surgical interventions post-treatment, with 28.1% (9 out of 32) requiring no surgeries at all [3][5] - The Overall Response Rate, including both Complete and Partial Responses, was 72% [3] - Patients had a median of 4 surgeries in the year prior to treatment, with a median decrease of 3 surgical interventions after dosing [5] Safety and Tolerability - INO-3107 was well tolerated, with 41% of patients reporting treatment-related adverse events, primarily low-grade [3][5] - The most common adverse events included injection site pain (31%) and fatigue (9%), with no severe adverse events reported [3][5] Mechanism of Action - INO-3107 induced T cell responses specific to HPV-6 and HPV-11, including cytotoxic CD8+ T cells, which persisted at week 52, indicating a memory response [3][5] - The treatment engaged both innate and adaptive immune responses, leading to the emergence of new T cell populations that targeted infected tissues [2][3] Regulatory Designations - INO-3107 has received Orphan Drug and Breakthrough Therapy designations from the FDA, facilitating its path to market [6] - The European Commission also granted Orphan Drug designation, and INOVIO has CE-marked its CELLECTRA® delivery device for commercialization in the EU [6] Market Context - RRP is a rare disease primarily caused by HPV-6 and HPV-11, leading to significant quality of life issues and risks associated with repeated surgeries [4] - The standard treatment involves surgical removal of papillomas, but recurrence is common, highlighting the need for effective alternatives like INO-3107 [4][5]