Theriva Biologics (TOVX) Corporate presentation summary

Pipeline and clinical development

• Lead candidate VCN-01 is advancing toward a Phase 3 trial for first-line metastatic pancreatic cancer and retinoblastoma, with ongoing preparations and regulatory alignment in the US and EU.

• VCN-01 has demonstrated promising Phase 1 and 2b data in pancreatic, head and neck, colorectal, and ovarian cancers, as well as retinoblastoma.

• Additional candidates, including VCN-X and Albumin Shield OVs, are in earlier stages targeting solid tumors and prevention of aGVHD.

• The company is seeking financing and partnerships to support pivotal trial execution.

• Cash position as of March 2026 is $14.4M, with a projected runway into Q1 2027.

VCN-01 mechanism and clinical results

• VCN-01 is a systemically delivered, tumor-selective, stroma-degrading adenovirus expressing PH20 hyaluronidase, designed to enhance tumor penetration and immune response.

• Clinical experience includes 144 patients across multiple tumor types, with favorable safety and efficacy signals.

• In metastatic pancreatic cancer, VCN-01 plus standard chemotherapy improved overall survival (median 10.8 vs 8.6 months; HR 0.57) and progression-free survival (median 7.0 vs 4.6 months; HR 0.55) compared to standard care alone.

• Two doses of VCN-01 further increased survival (median OS 14.8 vs 11.6 months; HR 0.44) and response duration (median 11.2 vs 5.4 months; HR 0.22).

• Safety profile was acceptable, with most adverse events being transient and reversible.

Regulatory and trial strategy

• FDA and EMA have agreed on key Phase 3 trial design elements, including endpoints, sample size, and adaptive design.

• A single successful Phase 3 trial could support marketing authorization.

• Additional Phase 2a studies are exploring more frequent dosing to optimize outcomes.

• The retinoblastoma program leverages orphan and rare pediatric disease designations, with a Phase 2/3 trial planned and potential for a Priority Review Voucher.