Theriva™ Biologics Announces Upcoming Presentation of Data from VCN-01 Retinoblastoma Phase 1 Clinical Trial at APAO 2026

Core Insights - Theriva Biologics is presenting safety and clinical outcomes from a Phase 1 study of VCN-01 in refractory retinoblastoma patients at the APAO Congress in Hong Kong on February 7, 2026 [1] - Preclinical data indicates that the combination of topotecan and VCN-01 may enhance antitumor activity in difficult-to-treat cases of retinoblastoma [1] Company Overview - Theriva Biologics is a clinical-stage company focused on developing therapeutics for cancer and related diseases, addressing areas of high unmet medical need [8] - The company’s lead candidate, VCN-01, is an oncolytic adenovirus designed to selectively replicate within tumor cells and degrade tumor stroma, enhancing the efficacy of co-administered therapies [4][8] Clinical Study Details - The Phase 1 clinical study (NCT03284268) evaluates the safety and tolerability of two intravitreal injections of VCN-01 in pediatric patients with intraocular retinoblastoma [1] - The study aims to provide an alternative to enucleation for patients who have not responded to other forms of chemotherapy [3] Preclinical Findings - Preclinical data will be presented that shows the synergistic effects of combining topotecan with VCN-01, potentially improving treatment outcomes for patients with vitreous seeds [1][3] Regulatory Designations - VCN-01 has received Orphan Drug and Rare Pediatric Disease designations from the FDA for the treatment of retinoblastoma, indicating its potential significance in addressing this rare cancer [3][4] Retinoblastoma Overview - Retinoblastoma is the most common type of eye cancer in children, occurring in approximately 1 in 14,000 to 1 in 18,000 live newborns, with a significant incidence in the pediatric population [7] - The average age of diagnosis is around 2 years, and effective treatment remains a challenge, particularly in low-resource countries [7]