Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, today announced the European Medicines Agency (EMA) has granted orphan medicinal product (orphan drug) designation to Toca 511 (vocimagene amiretrorepvec) & Toca FC (flucytosine) for the treatment of patients with glioma, a type of brain tumor. Toca 511 & Toca FC is currently under evaluation in a global Phase 3 trial, called Toca 5, involving patients with first or second recurrence of high-grade glioma (HGG), namely glioblastoma or anaplastic astrocytoma, who are undergoing resection.

"The EMA's granting of orphan medicinal product designation to Toca 511 & Toca FC emphasizes the urgent need for new therapies that may benefit patients living with glioma," said John Wood, vice president, regulatory affairs and quality, at Tocagen. "We are committed to working closely with the EMA under this orphan designation and our previously announced PRIME (PRIority MEdicines) designation to bring our product candidate to European patients and physicians as quickly as possible."

The EMA's orphan medicinal product designation aims to facilitate the development of medicines for rare diseases. To qualify for the designation, a medicine must meet a number of criteria, including its ability to treat, prevent or diagnose a disease that is life-threatening or chronically debilitating; prevalent in less than 5 in 10,000 people in the European Union (EU); and provide a significant benefit over existing treatments. Orphan medicines are eligible for conditional marketing authorization; ten-year market exclusivity in the EU after product approval; and fee waivers, such as the marketing authorization application fee, among other incentives.

Gliomas, particularly HGGs, are among the most common and aggressive primary brain cancers. The total number of patients with HGG expected to be diagnosed in 2018 is approximately 190,000 worldwide and approximately 36,000 in Europe. The two most common forms of HGG are glioblastoma and anaplastic astrocytoma. Standard treatment for newly diagnosed HGG includes safe surgical removal of as much of the tumor as possible followed by radiation therapy and chemotherapy. However, HGG recurs in most patients even after maximal treatment. After recurrence, median survival is typically seven to nine months.

About Toca 511 & Toca FC

Tocagen's lead product candidate is a two-part cancer-selective immunotherapy comprised of 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.

About Tocagen

Tocagen is a clinical-stage, cancer-selective gene therapy company developing first-in-class, broadly applicable product candidates designed to activate a patient's immune system against their own cancer. Tocagen's lead investigational product candidate, Toca 511 & Toca FC, is under evaluation in a pivotal Phase 3 trial for recurrent high-grade glioma (HGG), a disease with significant unmet medical need. The U.S. Food and Drug Administration (FDA) has granted Toca 511 & Toca FC Breakthrough Therapy Designation for the treatment of recurrent HGG and the European Medicines Agency (EMA) has granted Toca 511 PRIME (PRIority MEdicines) designation for the treatment of HGG. 

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding our business plans and objectives, expectations regarding timing and success of our clinical trials and planned clinical trials, timing and substance of communications with the FDA and EMA, a commercialization of our product candidates. Risks that contribute to the uncertain nature of the forward-looking statements include: risks associated with our product candidate development activities including the risks of clinical trials; the risk that our product candidates will cause serious adverse events or otherwise fail to prove effective and safe; the risk that we are unable to execute on our strategy; and the risks associated with commercialization of our product candidates if approved for sale. These and other risks and uncertainties are described more fully under the caption "Risk Factors" and elsewhere in Tocagen's filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Tocagen undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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