9 March 2018 - - The European Medicines Agency has granted orphan medicinal product designation to US-based clinical-stage, cancer-selective gene therapy company Tocagen Inc. (NASDAQ: TOCA) for Toca 511 (vocimagene amiretrorepvec) and Toca FC (flucytosine) for the treatment of patients with glioma, a type of brain tumor, the company said.
Toca 511 and Toca FC, a two-part cancer-selective immunotherapy, is currently under evaluation in a global Phase 3 trial, called Toca 5, involving patients with first or second recurrence of high-grade glioma, namely glioblastoma or anaplastic astrocytoma, who are undergoing resection.
Tocagen's lead product candidate is comprised of an investigational biologic, Toca 511, and an investigational small molecule, Toca FC. Toca 511 is a retroviral replicating vector that selectively infects cancer cells and delivers a gene for the enzyme, cytosine deaminase. Through this targeted delivery, only infected cancer cells carry the CD gene and produce CD.
Toca FC is an orally administered prodrug, 5-fluorocytosine, which is converted into an anti-cancer drug, 5-fluorouracil, when it encounters CD. 5-FU kills cancer cells and immune-suppressive myeloid cells resulting in anti-cancer immune activation and subsequent tumor killing.
Tocagen is developing product candidates designed to activate a patient's immune system against their cancer. Its lead investigational product candidate, Toca 511 and Toca FC, is being developed initially for the treatment of recurrent high-grade glioma.