San-Diego based biopharmaceutical company Tocagen has secured orphan drug status for its investigational gene therapy treatment for glioblastoma.
It follows hard on the heels of the combination treatment Toca 511 & Toca FC winning fast track designation just last month assisting the company in its quest to further advance the drug’s development.
Glioblastoma is the most common type of brain cancer, and most aggressive, with more than 10,000 new cases every year in the United States. However once diagnosed, patients have a five-year survival rate of less than 5%.
Harry Gruber, CEO of Tocagen, said: “There’s an extraordinary need for new treatment options for patients with this devastating disease.
“We believe the FDA’s granting of both orphan drug and fast track designations to Toca 511 & Toca FC will enable us to more efficiently advance our programme, which we hope will ultimately offer physicians and patients a new option in the fight against brain cancer.”
Both components of the combination treatment are designed to programme cancer cells to convert prodrug 5-FC into the FDA-approved anticancer drug, 5-flurorouracil (5-FU), which kills tumour cells and additionally significantly boosts immune responses.
Toca 511 is an injectable retroviral replicator vector that delivers a gene, responsible for the enzyme cytosine deaminase, to the tumour.
This is followed with oral doses of Toca FC which is converted within infected cancer cells into 5-FU, killing cancer cells while preserving healthy host cells.
The treatment will enter phase II and III trials later this year and so far interim results have shown median survival rates of 13.8 months compared with historical benchmarks of around 7 months.
This programme will be supported by companion diagnostic tests that Tocagen is working on in partnership with Siemens Healthcare Diagnostics under an agreement signed last year.
