Tocagen Receives EMA Priority Medicines (PRIME) Designation For Toca 511 In High Grade Glioma

SAN DIEGO, July 24, 2017 /PRNewswire/ -- Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, today announced the European Medicines Agency (EMA) has granted Toca 511 (vocimagene amiretrorepvec) PRIME (PRIority MEdicines) designation for the treatment of patients with high grade glioma (HGG), a type of brain tumor. Toca 511 & Toca FC is currently under evaluation in an international, randomized Phase 2/3 clinical trial, which is designed to serve as a potential registrational study. The trial involves patients with first or second recurrence of glioblastoma or anaplastic astrocytoma who are undergoing resection.

The PRIME designation application was based on clinical data from a Phase 1 resection study in patients with recurrent brain cancer and results from various preclinical models. Clinical data include results published in Science Translational Medicine, including safety data, patient survival data and durable, complete or partial tumor shrinkage determined by independent radiology review. Preclinical data include results published in Neuro-Oncology detailing proposed mechanisms of action for Toca 511 & Toca FC. These clinical and preclinical data were also reviewed by the U.S. Food and Drug Administration (FDA) and resulted in the granting of Breakthrough Therapy Designation for Toca 511 & Toca FC in recurrent high grade glioma in February 2017.

"The EMA's granting of PRIME designation for Toca 511 underscores the urgent need for new treatments for high grade glioma, one of the deadliest cancers," said Marty Duvall, chief executive officer of Tocagen. "We are committed to working closely with the EMA to expedite the advancement of our product candidate, and bringing a potentially transformative treatment option for high grade glioma to European patients and physicians as quickly as possible."

The PRIME program was launched by the EMA in 2016 to increase support for the development of medicines that target an unmet medical need. Under PRIME designation, drug developers receive enhanced interaction and early dialogue with the goal of optimizing development plans and accelerating evaluation so medicines can reach patients earlier. To be accepted, an investigational medicine must show the potential to benefit patients with unmet medical needs based on early clinical data. Medicines accepted into the PRIME program are considered priority medicines within the European Union (EU).

HGGs are among the most common and aggressive primary brain cancers. The total number of patients with HGG expected to be diagnosed in 2017 is approximately 160,000 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 cancer-selective immunotherapy comprised of an investigational biologic, Toca 511, and an investigational small molecule, Toca FC, that are designed to be used together. Toca 511 is an injectable retroviral replicating vector (RRV) that encodes a prodrug activator enzyme, cytosine deaminase (CD). CD is derived from yeast, and humans do not naturally have this gene. Its selective delivery to cancer cells means that the infected cancer cells selectively carry the CD gene and produce CD protein. Toca FC is an investigational orally administered prodrug, 5-fluorocytosine (5-FC) that is inactive as an anti-cancer drug. In animal models, Tocagen has shown that 5-FC is converted into the anticancer drug, 5-FU, at high concentrations in Toca 511-infected cancer cells that are producing CD protein. Together, the Toca 511 & Toca FC combination directly kills cancer cells and immune-suppressive myeloid cells resulting in activation of the immune system against cancer.

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 is developing its lead investigational product candidate, Toca 511 & Toca FC, initially for the treatment of 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. Tocagen has received grant support from leading brain cancer foundations, including Accelerate Brain Cancer Cure (ABC2), National Brain Tumor Society (NBTS), American Brain Tumor Association (ABTA), Musella Foundation and Voices Against Brain Cancer (VABC).

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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SOURCE Tocagen Inc.