Theriva Biologics Granted Orphan Drug Designation for Potential Pancreatic Cancer Treatment


Theriva Biologics (NYSE American: TOVX) has been granted Orphan Drug Designation by the FDA for VCN-01, a potential treatment for pancreatic cancer. VCN-01 is currently being evaluated in a multinational Phase 2b clinical trial as a first-line therapy for patients with pancreatic ductal adenocarcinoma (PDAC).

The FDA previously granted Orphan Drug Designation to VCN-01 for the treatment of retinoblastoma.


Compelling clinical outcomes observed in Phase 1 studies of VCN-01 in combination with chemotherapy or immunotherapy in patients with PDAC and other solid tumors.

VCN-01 is an oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment.

VCN-01 is administered systemically and enables the drug to act on both the primary tumor and metastases.

There is an urgent need for new treatment options for patients with PDAC, which has one of the lowest survival rates of all cancers.

About Theriva Biologics

Theriva Biologics (NYSE American: TOVX), is a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need. The Company’s wholly-owned Spanish subsidiary Theriva Biologics, S.L., has been developing a new oncolytic adenovirus platform designed for intravenous (IV), intravitreal and antitumoral delivery to trigger tumor cell death, improve access of co-administered cancer therapies to the tumor, and promote a robust and sustained anti-tumor response by the patient’s immune system. In addition to VCN-01, the Company’s clinical-stage candidates include (1) SYN-004 (ribaxamase) which is designed to degrade certain commonly used IV beta-lactam antibiotics within the gastrointestinal (GI) tract to prevent microbiome damage, thereby limiting overgrowth of pathogenic organisms such as VRE (vancomycin resistant Enterococci) and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients); and (2) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases.

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Theriva Biologics Granted Orphan Drug Designation for Potential Pancreatic Cancer Treatment

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