Theriva Biologics' VCN-01 Given Orphan Medicinal Designation for Retinoblastoma by EU Commission

Theriva Biologics, a clinical-stage company focused on developing therapies for cancer and related diseases, announced that the European Commission has accepted the European Medicines Agency (EMA) recommendation to grant orphan medicinal product designation to its lead candidate VCN-01. VCN-01, a systemic, selective, stroma-degrading oncolytic adenovirus, is intended for the treatment of retinoblastoma. This designation follows similar recognition by the United States Food and Drug Administration (FDA), which has previously awarded orphan drug status and rare pediatric disease designation to the same candidate for the treatment of retinoblastoma.

Steven A. Shallcross, the CEO of Theriva Biologics, expressed satisfaction with this European Commission decision, highlighting the critical demand for new treatments for retinoblastoma. He noted the promising outcomes from an investigator-sponsored Phase 1 trial that evaluated the safety and activity of VCN-01 in pediatric patients with refractory retinoblastoma. Shallcross emphasized the company’s ongoing collaboration with leading physicians and regulatory bodies globally to improve the clinical strategy for VCN-01, aiming to use it as an adjunct to chemotherapy for children battling this severe disease.

The EMA’s orphan designation is given to treatments meant for life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union or are unlikely to be profitable enough to warrant the investment required for development. Furthermore, the condition must have no existing approved therapies, or the new treatment must offer significant benefit over existing options. The designation offers multiple benefits to drug developers, including ten years of market exclusivity after the therapy receives marketing authorization in the EU, alongside protocol assistance, administrative support, and reduced fees for regulatory activities.

Retinoblastoma, a tumor originating in the retina, is the most common type of eye cancer in children. It affects about 1 in 14,000 to 18,000 live newborns and constitutes 15% of pediatric tumors in children under one year old. The average age of diagnosis is two, and it rarely occurs in children older than six. In Europe, the estimated incidence of retinoblastoma is 1 per 13,844 live births, with around 300 new cases annually. Despite advancements, preserving life and preventing severe outcomes such as loss of vision remain significant challenges. Additionally, in low-resource countries, children are more likely to suffer from eye loss and die from metastatic disease.

VCN-01, designed to be administered systemically, selectively targets and destroys tumor cells while breaking down the tumor stroma, a barrier that impedes cancer treatment. This multifunctional approach allows VCN-01 to exert multiple antitumor effects: infecting and lysing tumor cells, improving the effectiveness of co-administered chemotherapy, and enhancing tumor immunogenicity. This approach helps the patient's immune system and co-administered immunotherapies to better combat the tumor. VCN-01 has been tested in over 140 patients in clinical trials for various cancers, including pancreatic ductal adenocarcinoma (PDAC), head and neck squamous cell carcinoma, ovarian cancer, colorectal cancer, and retinoblastoma.

Theriva Biologics, listed on the NYSE American under the ticker TOVX, is dedicated to developing therapeutic solutions for cancer and diseases with high unmet needs. The company's subsidiary, Theriva Biologics, S.L. in Spain, is advancing an oncolytic adenovirus platform for intravenous, intravitreal, and antitumoral delivery to induce tumor cell death, improve the delivery and efficacy of co-administered cancer therapies, and stimulate a robust immune response against tumors. Besides VCN-01, Theriva’s clinical-stage candidates include SYN-004 (ribaxamase) aimed at preventing microbiome damage from IV beta-lactam antibiotics and SYN-020, aimed at treating both local gastrointestinal and systemic diseases.

Theriva Biologics continues to innovate in the field of oncology therapeutics, striving to address significant unmet medical needs and improve outcomes for patients suffering from challenging diseases like retinoblastoma.