Theriva Biologics Inc saw its shares surge over 70% higher in after-hours US trading following news that its lead clinical candidate, VCN-01, has been granted orphan drug designation by the US Food and Drug Administration (FDA) for the treatment of pancreatic cancer.
VCN-01 is a 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) in the ongoing VIRAGE Phase 2b clinical study.
Pancreatic cancer has one of the lowest survival rates among all cancers, emphasizing the urgent need for new treatment options.
In a statement, Steven A. Shallcross, the CEO of Theriva Biologics, commented: "The growing clinical data that underscore VCN-01's multiple modes of action and the compelling clinical outcomes observed in Phase 1 studies give us confidence that VCN-01 has the potential to address this unmet medical need."
Orphan drug designation is granted by the FDA to drugs aimed at treating rare diseases affecting fewer than 200,000 people in the US. The designation provides several benefits to drug developers, including potential market exclusivity for seven years, tax credits for qualified clinical trials, reduced fees, clinical protocol assistance, and expedited development programs.
VCN-01 is a systemically administered oncolytic adenovirus designed to selectively replicate within tumor cells and degrade the tumor stroma, thereby overcoming barriers to cancer treatment. It exerts multiple antitumor effects and has shown promising results in Phase 1 studies when combined with chemotherapy or immunotherapy in patients with PDAC and other solid tumors.
The treatment's VIRAGE Phase 2b clinical trial aims to enroll up to 92 adult participants with newly-diagnosed metastatic PDAC. The trial's primary endpoints include overall survival and VCN-01 safety/tolerability. Other endpoints include progression-free survival, objective response rate, and measures of biodistribution, VCN-01 replication, and immune response.
Theriva Biologics is also developing other clinical-stage candidates, including SYN-004 (ribaxamase), designed to prevent microbiome damage, and SYN-020, an oral formulation intended to treat gastrointestinal and systemic diseases.
Contact the author at jon.hopkins@proactiveinvestors.com