The Orphan Drug Act of 1983 transformed the rare disease treatment landscape. Prior to the Act’s passage, there was little commercial investment in drug development targeted towards rare conditions. The Act incentivized drug development through the creation of Orphan Drug status; this designation, granted to therapies intended to diagnose, treat, or prevent rare conditions, comes with benefits such as tax credits, fee waivers, and seven years of market exclusivity if/when the drug is approved. As shared by OncLive, the U.S. Food and Drug Administration recently granted Orphan Drug designation to VCN-01 for pancreatic cancer.
The Need for New Therapies
Right now, there are a number of issues that make pancreatic cancer notoriously difficult to treat. The pancreas sits in an anatomical spot that makes early diagnosis more challenging. Many people with this cancer remain undiagnosed until later stages of the disease. Between evasive pancreatic cancer cells and genetic mutations that don’t respond well to current available therapies, pancreatic cancer now comes with a 5-year survival rate of just 12%. Finding ways to improve this requires the advent of novel therapeutics.
In an article co-produced by VCN Biosciences and Nature Research, VCN-01 is a selective stroma-degrading oncolytic adenovirus that is:
designed to selectively target and replicate only in cancer cells, leading to the release of tumor neoantigens. Virus penetration within the tumor is improved through the expression of hyaluronidase by VCN-01, which simultaneously decreases intratu-moral fluid pressure and facilitates the uptake of the chemotherapy or biologic drug.
Outside of pancreatic cancer (and, specifically, pancreatic ductal adenocarcinoma), VCN-01 is also being developed for a range of solid tumors.
Studying VCN-01 for Pancreatic Cancer
So far, the safety, efficacy, and tolerability of VCN-01 has been evaluated in conjunction with other therapies. A Phase 1 study explored VCN-01 as a monotherapy, as well as alongside Abraxane, in people with solid tumors. VCN-01 was safe and contributed to an overall response rate of 50% in people receiving the combination treatment.
Now, an estimated 92 participants will enroll in the Phase 2b VIRAGE study which explores intravenous VCN-01 and chemotherapy for metastatic pancreatic ductal adenocarcinoma. In addition to furthering safety, efficacy, and tolerability data, researchers aim to identify:
- Overall response rate
- How long VCN-01 prevents the cancer from progressing
- Overall survival
- Response duration
- Biodistribution measures
- Immune response
- How well the cancer is controlled
- Whether (and how) VCN-01 is replicating
