Theriva Biologics Reaches Agreement with FDA on VCN-01 Phase 3 Trial Design

Theriva Biologics announced the outcomes of a recent Type B End-of-Phase 2 meeting with the FDA regarding the design of a Phase 3 clinical study of lead clinical candidate VCN-01 in combination with standard-of-care chemotherapy for the treatment of metastatic pancreatic adenocarcinoma. The FDA provided general agreement with Theriva's proposed design for a Phase 3 clinical trial, which closely tracks the design of the successful VIRAGE Phase 2 trial. As announced in 2025, the VIRAGE trial met its primary endpoints, with metastatic PDAC patients receiving VCN-01 with SoC chemotherapy having improved overall survival, progression free survival and Duration of Response compared to SoC chemotherapy alone. Greater improvements in OS and PFS were observed in patients who received two doses of VCN-01, leading Theriva to plan the Phase 3 trial to include repeat dosing and an adaptive design aimed to optimize the trial's timelines and outcomes. Consistent with scientific advice previously received from the Committee for Medicinal Products for Human Use of the European Medicines Agency, the FDA advised that a potential biologics licensing application for VCN-01 in metastatic PDAC could be supported by Theriva's proposed Phase 3 clinical trial comprising a single, high-quality, randomized, double-blinded, study comparing VCN-01 plus gemcitabine/nab-paclitaxel SoC to gemcitabine/nab-paclitaxel SoC plus placebo. The FDA further agreed on the proposed dosing of VCN-01 and gemcitabine/nab-paclitaxel in repeated "macrocycles", the proposed inclusion/exclusion criteria, the primary endpoint (overall survival), key secondary endpoints, and the use of an adaptive design. The FDA also clarified statistical expectations regarding the proposed interim analyses and the quality of data required for potential sample size re-estimation or a demonstration of early efficacy.