Theriva Biologics Receives FDA Rare Pediatric Drug Designation for VCN-01 for Retinoblastoma Treatment

Theriva™ Biologics, Inc., a diversified clinical-stage company listed on the NYSE American under the ticker TOVX, made a significant announcement on July 31, 2024. The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Drug Designation (RPDD) for VCN-01, a treatment targeting retinoblastoma. VCN-01 is Theriva's leading product candidate and is a systemic, selective, stroma-degrading oncolytic adenovirus. Earlier, the FDA had also granted orphan drug designation to VCN-01 for retinoblastoma treatment.

Steven A. Shallcross, Chief Executive Officer of Theriva Biologics, expressed optimism about the FDA’s decision, emphasizing the critical need for new treatment avenues for pediatric patients suffering from retinoblastoma. He pointed out that this regulatory milestone is a crucial step forward and noted ongoing collaborations with top physicians and regulatory bodies to refine the clinical strategy for VCN-01. The aim is to use VCN-01 as an adjunct to chemotherapy for advanced cases of pediatric retinoblastoma. Recent results from an investigator-sponsored Phase 1 trial, which assessed the safety and activity of intravitreal VCN-01 in pediatric patients with refractory retinoblastoma, were positively evaluated by the study Monitoring Committee. These findings are expected to guide further clinical development in this area, which is recognized for its high unmet medical need.

The FDA awards RPDD for rare diseases, defined as conditions affecting fewer than 200,000 people in the U.S., that are serious and life-threatening and primarily affect individuals aged 18 or younger. Should the Biologics License Application (BLA) for VCN-01 receive FDA approval for treating retinoblastoma, Theriva could become eligible for a Priority Review Voucher. This voucher can be redeemed to expedite the review of any future marketing applications or can be sold or transferred.

Retinoblastoma is a type of tumor that develops in the retina and constitutes the most common form of eye cancer in children. It affects approximately 1 in 14,000 to 1 in 18,000 live births and accounts for 15% of pediatric ocular tumors.

VCN-01 is designed to be administered systemically and works by selectively replicating within tumor cells and breaking down the tumor stroma, a significant barrier to effective cancer treatment. This multi-faceted mechanism allows VCN-01 to exert several antitumor effects, including selective tumor cell lysis, enhanced chemotherapy penetration, and increased tumor immunogenicity, which exposes the tumor to the patient's immune system and co-administered immunotherapy products. The systemic administration of VCN-01 ensures its action not just on the primary tumor but also on metastatic sites. To date, VCN-01 has been administered to over 80 patients in various Phase 1 and investigator-sponsored clinical trials covering different types of cancers, including pancreatic ductal adenocarcinoma (PDAC), head and neck squamous cell carcinoma, ovarian cancer, colorectal cancer, and retinoblastoma.

Theriva Biologics is advancing an innovative oncolytic adenovirus platform intended for intravenous, intravitreal, and antitumoral delivery to induce tumor cell death, enhance the effectiveness of co-administered cancer therapies, and promote a sustained anti-tumor response by the patient’s immune system. Apart from VCN-01, Theriva’s other leading candidates include SYN-004 (ribaxamase), which aims to protect the gastrointestinal microbiome from damage caused by certain IV beta-lactam antibiotics, and SYN-020, a recombinant oral formulation of intestinal alkaline phosphatase (IAP) intended to treat both local gastrointestinal and systemic diseases.

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