Pharma Focus America

Theriva Biologics Secures Orphan Drug Designation from U.S. FDA for VCN-01 in Pancreatic Cancer Treatment

Wednesday, June 28, 2023

Theriva Biologics, a clinical-stage company focused on developing cancer therapeutics, has announced that their lead clinical candidate, VCN-01, has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of pancreatic cancer. VCN-01 is a systemic, selective, stroma-degrading oncolytic adenovirus being evaluated in combination with standard-of-care chemotherapy as a first-line therapy for patients with pancreatic ductal adenocarcinoma (PDAC).

Pancreatic cancer has one of the lowest survival rates among all cancers, and there is a significant need for new treatment options. The orphan drug designation highlights the urgency of finding novel therapies for patients with PDAC. Theriva Biologics believes that VCN-01, with its multiple modes of action and promising clinical outcomes observed in Phase 1 studies, has the potential to address this unmet medical need.

Orphan drug designation is granted by the FDA to drugs being developed to treat rare diseases or conditions affecting fewer than 200,000 people in the United States. It provides several benefits to drug developers, including market exclusivity, tax credits, fee waivers, and assistance with clinical protocols.

VCN-01 is a systemically administered oncolytic adenovirus that selectively replicates within tumor cells and degrades the tumor stroma, which is a physical and immunosuppressive barrier to cancer treatment. By doing so, VCN-01 exerts multiple antitumor effects, including selectively infecting and lysing tumor cells, enhancing access to co-administered chemotherapy, and increasing tumor immunogenicity.

The ongoing multinational Phase 2b clinical study, VIRAGE, is evaluating intravenous VCN-01 in combination with standard-of-care chemotherapy for newly-diagnosed metastatic PDAC. The trial aims to enroll up to 92 participants and assess various endpoints, including overall survival, safety/tolerability of VCN-01, progression-free survival, objective response rate, biodistribution, VCN-01 replication, and immune response.

Theriva Biologics is dedicated to advancing the development of VCN-01 and closely monitoring the trial's progress. They may consider implementing steps to accelerate the clinical program based on emerging data.

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