Pharma Focus America

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

Thursday, June 29, 2023

Theriva Biologics (NYSE American: TOVX) has announced that the US Food and Drug Administration (FDA) granted orphan drug designation to their lead clinical candidate, VCN-01, for the treatment of pancreatic cancer. VCN-01 is a selective, stromal-degrading, systemic adenovirus oncolytic virus being developed by Theriva Biologics.

The orphan drug designation recognizes the urgent need for new treatment options for pancreatic cancer patients, as it has one of the lowest survival rates among all cancers. Theriva Biologics CEO, Steven A. Shallcross, expressed the importance of developing novel therapies for this indication, noting the limited progress made in improving standard treatments for pancreatic cancer despite its increasing incidence.

VCN-01 has shown promising clinical results in Phase 1 studies when combined with chemotherapy or immunotherapy for patients with pancreatic ductal adenocarcinoma (PDAC) and other solid tumors. The drug is designed to selectively replicate and aggressively target tumor cells, while also degrading the stromal barrier that hinders effective cancer treatment. These mechanisms allow VCN-01 to exert multiple anti-tumor effects, such as selectively infecting and lysing tumor cells, improving the access of co-administered chemotherapy, and enhancing the tumor's immunogenicity.

Orphan drug designation is granted by the FDA to drugs being developed for rare diseases or conditions affecting fewer than 200,000 people in the United States. It provides various benefits to drug developers, including potential market exclusivity for seven years after FDA approval, tax credits for qualifying clinical trials, waived application fees, reduced annual product fees, clinical protocol assistance, and potential qualification for accelerated development programs.

Theriva Biologics is currently conducting the multinational Phase 2b clinical study known as VIRAGE. This study evaluates the intravenous administration of VCN-01 in combination with standard-of-care chemotherapy (gemcitabine/nab-paclitaxel) as a first-line treatment for patients with PDAC. The study aims to assess overall survival, safety, tolerability, progression-free survival, objective response rate, biodistribution, VCN-01 replication, and immune response. The trial is open-label and closely monitored, with potential adjustments to the clinical program based on emerging data.

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