EMA Endorses Theriva Biologics’ Phase 3 VCN-01 Trial Design for Metastatic Pancreatic Cancer—Path Cleared for Innovative Approach

EMA Agreement Sets Stage for Pivotal VCN-01 Study in PDAC

Theriva Biologics (NYSE:TOVX) has announced a critical step forward for its lead asset, VCN-01, as the European Medicines Agency (EMA) offered overall agreement with the company’s proposed Phase 3 clinical trial in metastatic pancreatic ductal adenocarcinoma (PDAC). The agency’s feedback supports the trial’s sample size, adaptive design, and repeated dosing strategy, all of which aim to maximize trial efficiency and patient outcomes.

Key Elements of EMA Endorsement: Adaptive Design and Dosing Flexibility

Unlike conventional static designs, EMA agreed to an adaptive trial format, allowing Theriva to optimize timelines and potentially pivot based on ongoing results. The EMA specifically recognized strong survival signals in a Phase 2b study where patients receiving two doses of VCN-01 saw improved outcomes versus standard chemotherapy alone. Notably, the Phase 3 protocol may allow administration of three or more doses per patient, a move the agency believes could further enhance benefit. The primary endpoint will be overall survival, accompanied by secondary metrics such as progression-free survival, duration of response, and patient-reported outcomes.

Clinical Evidence: Strong Survival Signals Fuel Optimism

The agency’s optimism is grounded in previous VIRAGE Phase 2b trial data, where the combination of VCN-01 with gemcitabine/nab-paclitaxel led to notably better patient outcomes—including increases in progression-free survival, overall survival, and duration of response. Additional dosing appeared to boost results further, underpinning the rationale for an expanded, repeated-dose framework in the upcoming study. VCN-01 has Orphan Drug designation in both Europe and the US, signaling regulatory priority for its development in rare and challenging oncology indications.

Financial Health Provides Stability for Trial Execution

Theriva reported $15.5 million in cash and equivalents as of November 2025, offering a projected runway into Q1 2027. This funding horizon is expected to cover key regulatory meetings—such as a pivotal End-of-Phase 2 FDA meeting slated for the first half of 2026—and support clinical protocol development, manufacturing scale-up, and ongoing partnership activities.

VCN-01: A Targeted Approach to Overcoming Pancreatic Cancer Barriers

VCN-01 represents a novel class of therapy—an engineered, oncolytic adenovirus designed to infect, replicate within, and destroy tumor cells directly. In PDAC, a disease notorious for late detection and low resectability, VCN-01’s mechanism also degrades the tumor’s protective stroma, potentially increasing the penetration and effectiveness of co-administered chemotherapy and harnessing the immune system. To date, 142 patients have received VCN-01 in trials targeting multiple cancers, further establishing a base of safety and exploratory efficacy data.

Broader Pipeline and Next Steps

With regulatory clarity now backing their strategy, Theriva plans to advance the VCN-01 program not just in PDAC, but also in rare pediatric cancers like retinoblastoma, with future FDA and EMA consultations ahead. The company’s diversified pipeline includes SYN-004 for microbiome protection and SYN-020 for GI and systemic diseases—showing a commitment to treating unmet needs across oncology and beyond.

Key Takeaway: Regulatory Support and Financial Readiness Strengthen Theriva’s Position

Investors and stakeholders may want to watch for the finalized Phase 3 protocol and the upcoming FDA meeting in 2026 as major milestones. With both scientific advice and cash reserves aligned, Theriva Biologics is positioned to execute a high-impact pivotal trial designed to address one of oncology’s toughest challenges: metastatic pancreatic cancer.

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