Theriva Biologics Receives Scientific Advice from EMA on VCN-01 Phase 3 Trial

Theriva Biologics announced the receipt of Scientific Advice from the Committee for Medicinal Products for Human Use of the European Medicines Agency on the design of a Phase 3 clinical trial of lead clinical candidate VCN-01 in combination with gemcitabine/nab-paclitaxel standard-of-care chemotherapy for the first-line treatment of metastatic PDAC. The Company has previously reported the results of the VIRAGE randomized, controlled Phase 2b clinical trial, demonstrating that PDAC patients in the VCN-01 plus gemcitabine/nab-paclitaxel SoC treatment arm demonstrated increased overall survival, progression free survival, and duration of response compared to patients in the control arm treated with gemcitabine/nab-paclitaxel SoC alone. Even greater increases in these parameters were observed in patients who received 2 doses of VCN-01 administered 3 months apart. VCN-01 has been granted Orphan Drug designation for the treatment of metastatic PDAC in Europe and the USA and Fast Track designation in the USA. CHMP advised that a potential future marketing authorization application for VCN-01 in metastatic PDAC could be supported by Theriva's proposed clinical development strategy comprising a single, high-quality, double-blinded, randomized, placebo-controlled Phase 3 trial if it demonstrates a compelling benefit-risk ratio with VCN-01 plus gemcitabine/nab-paclitaxel SoC compared to gemcitabine/nab-paclitaxel SoC alone. The CHMP scientific advice included agreement on the proposed inclusion/exclusion criteria, primary endpoint, secondary endpoints, sample size, and the use of an adaptive design to potentially optimize trial timelines and outcomes. Importantly, CHMP recognized the increased improvement in overall survival of patients receiving 2 doses of VCN-01 in the VIRAGE study, and agreed with the proposed dosing of VCN-01 and gemcitabine/nab-paclitaxel in repeated "macrocycles", enabling more than 2 doses of VCN-01 to be administered in the Phase 3 trial. They further suggested that more frequent dosing of VCN-01 could be considered. As previously reported, at November 10, 2025, Theriva had $15.5M in cash and equivalents, providing runway into Q1 2027 as the Company completes interactions with regulatory agencies regarding the PDAC and retinoblastoma programs and pursues partnerships to support VCN-01 manufacturing scale-up and conduct of the proposed pivotal clinical trial.