TOVX News & Events

Theriva Biologics reported the out-licensing of SYN-020, to Rasayana Therapeutics, a privately-held company targeting the Gut-Organ axis biology to discover and deliver transformative gut-targeted medicines for patients with chronic inflammatory diseases. "We are extremely pleased to complete the license of SYN-020 to Rasayana, which will enable the advancement of this important GI asset from our portfolio while we focus on our lead pancreatic cancer program, VCN-01," said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "We believe SYN-020 is a highly versatile asset with therapeutic opportunities in multiple high-value indications arising from GI dysfunction. The Rasayana team and advisors are world leaders in IAP and its clinical applications, and they are well positioned to advance SYN-020 development, ideally delivering benefits for patients and value for Theriva shareholders."

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.

"Recent presentations of clinical data for VCN-01 and preclinical data for VCN-12 at medical congresses have highlighted the progress made across our oncology pipeline," said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "Having successully completed the VIRAGE Phase 2b clinical trial, we are now pursuing interactions with the European Medicines Agency and the US FDA to seek alignment on a protocol for a proposed Phase 3 study of VCN-01 plus gemcitabine/nab-paclitaxel standard-of-care chemotherapy as first-line treatment for metastatic PDAC. In addition, we have made significant progress on the design of a potential Phase 2/3 clinical trial in the rare pediatric disease retinoblastoma for potential discussion with regulators in the first half of 2026. With capital raised from our recent warrant inducement and sales made pursuant to our at-the-market facility, we are well positioned to advance our regulatory and clinical strategies and explore potential partnerships for the innovative assets in our pipeline."

Theriva Biologics announced that the company had become aware of unusual trading activity in its common stock on the NYSE American on October 24. "The company has made inquiries and does not believe corrective actions are appropriate. The company is further announcing that there has been no material development in its business and affairs not previously disclosed or, to its knowledge, any other reason to account for the unusual market action. However, on October 13, 2025, the company announced the presentation of expanded data from its VIRAGE trial investigating VCN-01 at a mini oral session at the European Society for Medical Oncology Annual Congress," the company stated.