Theriva Secures FDA Agreement On Phase 3 Trial Design For VCN-01 In Metastatic Pancreatic Cancer

Theriva Biologics has reported the outcomes of a Type B End-of-Phase 2 meeting with the U.S. Food and Drug Administration to discuss the Phase 3 development plan for its lead candidate, VCN-01. The investigational therapy is being studied in combination with standard chemotherapy for patients with metastatic pancreatic ductal adenocarcinoma (PDAC), a cancer with limited treatment options and poor survival outcomes.

The FDA broadly agreed with Theriva’s proposed Phase 3 trial design, which mirrors the structure of the previously completed VIRAGE Phase 2 study. Reported in 2025, the Phase 2 trial met its primary goals, showing that patients treated with VCN-01 alongside standard-of-care chemotherapy experienced improvements in overall survival, progression-free survival, and duration of response compared with chemotherapy alone. Notably, patients who received two doses of VCN-01 showed stronger survival benefits, prompting plans to incorporate repeat dosing and an adaptive framework in the late-stage study.

“We are very pleased to align with the FDA on the key elements of our proposed pivotal Phase 3 trial evaluating VCN-01 plus gemcitabine/nab-paclitaxel SoC in metastatic PDAC patients,” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “Data from our VIRAGE Phase 2b study demonstrated improved outcomes in PDAC patients treated with 2 doses of VCN-01, and we believe that administering multiple macrocycles of VCN-01 and gemcitabine/nab-paclitaxel may further improve patient outcomes. The combined feedback from the FDA and the EMA now enables us to finalise the protocol for a pivotal Phase 3 clinical trial and pursue development funding and/or partnerships, which, if successful, may deliver a novel and effective treatment option for patients with this difficult-to-treat solid tumour cancer.”

Regulatory alignment also extends internationally. Prior scientific advice from the European Medicines Agency’s Committee for Medicinal Products for Human Use is consistent with the FDA’s guidance, indicating that a single, well-controlled Phase 3 study could support a future biologics license application if successful. The planned randomised, double-blind trial will compare VCN-01 plus gemcitabine/nab-paclitaxel with placebo chemotherapy. Regulators agreed on dosing schedules using repeat macrocycles, study eligibility criteria, overall survival as the primary endpoint, key secondary endpoints such as progression-free survival, and the use of adaptive statistical methods, including interim analyses and potential sample-size adjustments.