Theriva Biologics Gets Positive EMA Scientific Advice For Phase 3 Trial Design Of VCN-01 In Metastatic Pancreatic Ductal Adenocarcinoma

Theriva Biologics, a diversified clinical-stage company developing treatments for cancer and related diseases where significant unmet medical need remains, announced that it has received Scientific Advice from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency. This guidance relates to the design of a Phase 3 clinical trial for the company’s lead candidate, VCN-01, which will be evaluated in combination with standard-of-care gemcitabine and nab-paclitaxel chemotherapy for first-line treatment of metastatic pancreatic ductal adenocarcinoma (PDAC).

Theriva previously reported results from its VIRAGE Phase 2b clinical trial, a randomized and controlled study that evaluated VCN-01 together with gemcitabine and nab-paclitaxel. In that study, patients who received VCN-01 along with the standard chemotherapy regimen showed improvements in overall survival, progression-free survival, and duration of response compared to those who received chemotherapy alone. These outcomes were even more pronounced in patients who were administered two doses of VCN-01 given three months apart. VCN-01 has been granted Orphan Drug designation for metastatic PDAC in both Europe and the United States, as well as Fast Track designation in the United States.

According to the Scientific Advice, CHMP stated that a future marketing authorization application for VCN-01 in metastatic PDAC could be supported by Theriva’s clinical development plan. This includes a single, high-quality, double-blind, randomized, placebo-controlled Phase 3 trial, provided the study demonstrates a strong benefit-risk profile for the combination of VCN-01 and chemotherapy compared to chemotherapy alone. 

CHMP agreed with the company’s proposed trial design, including the inclusion and exclusion criteria, the primary endpoint of overall survival, key secondary endpoints such as progression-free survival, duration of response, and patient-reported outcomes, and the proposed sample size. They also supported the use of an adaptive trial design to help optimize study timelines and outcomes. 

Importantly, CHMP acknowledged the improved survival benefit observed with two doses of VCN-01 in the VIRAGE study and agreed with the plan to incorporate repeated “macrocycles” of dosing in the Phase 3 trial so that patients can receive more than two doses. They also suggested that more frequent dosing could be considered.

Steven A. Shallcross, Chief Executive Officer of Theriva Biologics, stated that the company is greatly encouraged by the guidance received from the EMA. He noted that the regulatory agency’s support for the macrocycle dosing strategy aligns with the VIRAGE study findings, where two doses of VCN-01 resulted in better patient outcomes. Based on this, the company expects that administering three or more doses of VCN-01 could potentially offer even stronger survival benefits. 

Theriva plans to hold an End-of-Phase 2 meeting with the U.S. Food and Drug Administration in the first half of 2026, after which it intends to finalize the protocol for a multinational Phase 3 trial designed to provide a new treatment option for a rapidly fatal cancer. Shallcross emphasized that clear regulatory guidance is crucial for advancing the company’s partnering efforts for the VCN-01 program. In addition to discussions related to PDAC, Theriva intends to meet with both the EMA and FDA in 2026 to seek input on a potential Phase 2/3 study of VCN-01 in retinoblastoma, a rare childhood cancer for which the therapy has already received Rare Pediatric Disease designation.

As previously disclosed, Theriva reported cash and equivalents of 15.5 million dollars as of November 10, 2025. This funding is expected to support operations through the first quarter of 2027 as the company continues its regulatory interactions for the PDAC and retinoblastoma programs and pursues partnerships to aid in scaling up VCN-01 manufacturing and preparing for future pivotal clinical trials.