Tocagen Doses 1st Patient in Cancer Therapy Clinical Trial

Tocagen Inc. today announced that the first patient has been dosed in its clinical trial investigating the intravenous administration of selective cancer therapy Toca 511 & Toca FC, in patients with recurrent high grade glioma (HGG) including glioblastoma multiforme (GBM, Grade 4 HGG), the most common and aggressive form of primary brain cancer.

The multicenter, open-label study is evaluating the safety, tolerability and pharmacokinetics of ascending doses of Toca 511 administered intravenously before scheduled tumor removal. Toca 511 is also administered into the wall of the resection cavity at the time of tumor removal, followed by cycles of oral Toca FC. Removed tumor will be tested for the presence of Toca 511. Patients will be monitored for changes in clinical status and clinical response data will be collected.

"Patients diagnosed with recurrent brain cancer have limited treatment options, with patients typically surviving less than eight months, so there is a high level of need for new therapies to fight this disease," said Steven Kalkanis M.D., vice chair, department of neurosurgery at the Henry Ford Hospital and principal investigator of this study. "Early data involving intra-tumoral delivery have shown Toca 511 & Toca FC can selectively kill cancer cells and not healthy brain cells. As the first center to enroll a patient in this new study, we look forward to evaluating the potential of this additional delivery approach." Ian Y. Lee, M.D., neurosurgeon at the Henry Ford Hospital conducted the brain tumor surgery for this first case.

Toca 511 & Toca FC is an investigational gene therapy that is designed to selectively infect and kill cancer cells via a proposed dual mechanism of action: first, the Toca 511 virus selectively infects and mediates direct cancer cell killing via local production of 5-FU, a potent anticancer agent, and second, the immune system becomes activated selectively against the tumor resulting in long-term, systemic anticancer activity.

Harry Gruber, M.D., CEO of Tocagen, added, "We are making exciting progress with our selective cancer immunotherapeutic programs. Exploring the intravenous delivery of Toca 511 is an important step in our fight against brain cancer and towards expanding the types of cancers we can target using Tocagen's technology, including metastatic cancers. We are also advancing our preclinical candidates that are designed to activate the immune system selectively to kill cancer cells: Toca gamma, which produces gamma interferon within cancer cells, as well as Toca RNAi, which inhibits immune checkpoints in cancer cells."

More information about the trial can be found at ClinicalTrials.gov using the identifier NCT01985256. If interested in participating in this trial, an inquiry can be submitted here. In addition to Henry Ford Hospital, clinical trials with Toca 511 & Toca FC in patients with recurrent high grade glioma, including GBM, are being conducted at other leading neurooncology centers in the United States.

About Toca 511 & Toca FCbr/> The combination of Toca 511 (vocimagene amiretrorepvec) for injection and Toca FC (flucytosine) extended-release tablets, is being investigated in Phase I/II studies at leading centers across the United States in patients with recurrent high grade glioma, including glioblastoma multiforme (GBM). Toca 511 is a retroviral replicating vector (RRV) that is designed to deliver a prodrug activator gene called cytosine deaminase (CD) selectively to cancer cells. After allowing time for Toca 511 to spread through the tumor, those cancer cells expressing the CD gene can convert the antibiotic drug flucytosine into the anticancer drug 5-fluorouracil (5-FU). By producing 5-FU locally with CD, this technology has the potential to produce much higher intra-tumoral concentrations of 5-FU than can be currently attained with systemic administration. In addition to this initial, direct killing of cancer cells, preclinical data suggests a selective, local and systemic anticancer immune response without off-target toxicity, such as autoimmunity. The Toca 511 & Toca FC combination product is being investigated in three ascending dose trials in patients with recurrent high grade glioma.

About Tocagen
Tocagen Inc. is a privately funded, clinical stage biopharmaceutical company pursuing the discovery, development and commercialization of gene therapy products for the selective treatment of cancer. Tocagen is initially focusing on treatments for patients with advanced cancer for whom no adequate treatments currently exist. Toca 511 & Toca FC, the company's lead investigational combination product, is being evaluated in three clinical trials in patients with recurrent high grade glioma, including glioblastoma multiforme (GBM). 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). For more information about Tocagen or Toca 511 please visit www.tocagen.com or www.clinicaltrials.gov using the identifier NCT01470794, NCT01156584 or NCT01985256.

SOURCE: Tocagen Inc.