On October 2, 2018 Tocagen Inc. (Nasdaq: TOCA) reported it has received Protocol Assistance from the European Medicines Agency (EMA) under the Company’s PRIME (PRIority MEdicines) designation for Toca 511 (vocimagene amiretrorepvec) & Toca FC (flucytosine, extended-release) for the treatment of patients with recurrent high grade glioma (HGG) (Press release, Tocagen, OCT 2, 2018, View Source;p=RssLanding&cat=news&id=2369807 [SID1234529957]). In particular, the EMA indicated that the statistical analyses, seamless design, and the use of overall survival as the primary endpoint in the ongoing Phase 3 Toca 5 clinical trial are appropriate for a potential marketing authorization application for Toca 511 & Toca FC.

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"We are pleased the global Toca 5 trial has the potential to serve as the basis for a regulatory application in Europe, which we believe may expedite the advancement of Toca 511 & Toca FC as a treatment for patients with brain cancer. If the pivotal trial data are positive, our gene therapy would be the first medicine in over 20 years to show a treatment benefit in a randomized trial in patients with this disease," said Marty Duvall, chief executive officer of Tocagen. "Our interactions with the European regulators under the PRIME pathway are conducted in the spirit of collaboration and urgency to bring forward a new treatment for brain cancer as quickly as possible."

Toca 5 is an international, randomized, multi-center study evaluating the safety and efficacy of Toca 511 & Toca FC compared to standard of care in patients undergoing resection for recurrent HGG. The trial recently completed the planned enrollment of 380 patients. The primary endpoint of the trial is overall survival. Secondary endpoints of the Toca 5 trial include durable response rate, defined as complete or partial responses lasting at least 24 weeks, which is being assessed as a novel endpoint in the post-surgical setting of recurrent HGG. Tocagen plans to conduct a second interim analysis of Toca 5 in the first half of 2019 and the final planned safety and efficacy analyses by the end of 2019. More information about Toca 5 can be found on ClinicalTrials.gov using the clinical trial identifier NCT02414165.

The Committee on Orphan Medicinal Products (COMP) of the EMA has designated both Toca 511 & Toca FC as orphan medicinal products indicated for the treatment of glioma. In addition, the U.S. Food and Drug Administration granted Breakthrough Therapy Designation for Toca 511 & Toca FC in recurrent HGG.

Recurrent HGG is among the most common and aggressive primary brain cancers and often strikes in the prime of life. The two most common forms of HGGs are glioblastoma and anaplastic astrocytoma. The total number of new diagnoses of HGG expected in 2018 is about 197,000 worldwide, with approximately 38,000 in Europe. Unfortunately, HGG recurs in most patients after frontline treatment, and standard of care treatment typically offers a median survival of only seven to nine months.

About Toca 511 & Toca FC
Tocagen’s lead product candidate is a two-part cancer-selective immunotherapy comprising an investigational biologic, Toca 511, and an investigational small molecule, Toca FC. Toca 511 is a retroviral replicating vector (RRV) that selectively infects cancer cells and delivers a gene for the enzyme, cytosine deaminase (CD). Through this targeted delivery, only infected cancer cells carry the CD gene and produce CD. Toca FC is an orally administered prodrug, 5-fluorocytosine (5-FC), which is converted into an anti-cancer drug, 5-fluorouracil (5-FU), when it encounters CD. 5-FU kills cancer cells and immune-suppressive myeloid cells resulting in anti-cancer immune activation and subsequent tumor killing.