First Patient Enrolled in Toca 6, Tocagen's Trial for Metastatic Cancers Evaluating Intravenously Delivered Toca 511 Virus

SAN DIEGO, Sept. 22, 2016 /PRNewswire/ -- Tocagen, Inc., a clinical-stage, cancer-selective immunotherapy company, today announced the first patient has been enrolled in Toca 6, a Phase 1b study evaluating intravenously delivered Toca 511 (vocimagene amiretrorepvec) in combination with Toca FC (extended-release 5-fluorocytosine) in patients with metastatic cancers. The study is planned to include patients with colorectal cancer with liver metastases; metastatic renal cell carcinoma; locally advanced or metastatic pancreatic cancer or breast cancer; brain metastases from non-small cell lung cancer or breast cancers; and locally advanced or recurrent melanoma.

"We must strive for breakthroughs in the treatment of cancer and identifying therapies that work through novel mechanisms will be vital to achieve this goal," said Jaime Merchan, M.D., oncologist at Sylvester Comprehensive Cancer Center, part of UHealth, the University of Miami Health System; associate professor of medicine at the University of Miami Miller School of Medicine; and the principal investigator of the study who also enrolled the first patient. "Based on preclinical data, a unique immune activating mechanism of action is proposed for Toca 511 and Toca FC whereby the treatment targets and destroys cancer cells wherever they are located in the body while leaving healthy cells unharmed. I look forward to continuing the investigation of this promising approach in our fight against cancer."

The trial will evaluate if Toca 511-delivered genes and resulting proteins are detected in tumor samples, following intravenous administration of Toca 511. Safety, tumor response and survival results will also be assessed.

In this first patient, who has metastatic colorectal cancer, intravenous Toca 511 and oral Toca FC was well tolerated. Toca 511 viral genes and cytosine deaminase (CD) were present in tumor samples.  Additional results from this trial will be presented at a future scientific conference.

"With the initiation of this trial we have moved one step closer to evaluating our cancer-selective gene therapy in broad indications using intravenous Toca 511 administration," said Harry Gruber, M.D., chief executive officer of Tocagen. "We are particularly encouraged by the observation of viral transduction in this first case and we look forward to enrolling additional cancer patients with high unmet needs."

More information about the Toca 6 clinical trial can be found by searching clinicaltrials.gov using the clinical trial identifier NCT02576665.

In three Phase 1 studies in patients with recurrent brain cancer, Toca 511 was administered in one of three methods: intravenously followed by injection into the resection cavity wall at the time of surgery; into the resection cavity wall at the time of surgery; or intratumorally, and was followed in each case by cycles of orally administered Toca FC. These studies provided support for the ongoing Phase 2/3 study of Toca 511 & Toca FC in patients with recurrent glioblastoma and anaplastic astrocytoma, which is designed to serve as a potential registration study. A summary of Tocagen's Phase 1 clinical trials can be found on Tocagen's website.

Tocagen's cancer-selective gene therapy platform is built on retroviral replicating vectors (RRVs) which are designed to selectively integrate into the DNA of cancer cells which then serve as factories for these RRVs to replicate and infect neighboring cancer cells, providing long-term presence of the therapeutic gene(s). Tocagen's lead product candidate is a combination of an investigational biologic, Toca 511, and an investigational small molecule drug, Toca FC. Toca 511 is a proprietary injectable RRV that encodes a prodrug activator enzyme, CD. 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 orally administered, proprietary extended-release version of 5-fluorocytosine (5-FC), a prodrug that is inactive as an anti-cancer drug. The treatment is designed to convert Toca FC into the active anti-cancer drug, 5-fluorouracil (5-FU), at local high concentrations in Toca-511-infected cancer cells that are producing CD protein. 5-FU is a well-established anti-cancer agent used effectively in many conventional chemotherapy settings. In addition to the direct killing of Toca 511-infected cancer cells, 5-FU kills neighboring uninfected cancer cells and immunosuppressive myeloid cells including myeloid derived suppressor cells (MDSCs) in the tumor.

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. The company is developing its lead investigational product candidate, Toca 511 (vocimagene amiretrorepvec) & Toca FC (extended-release 5-fluorocytosine), initially for the treatment of recurrent high grade glioma (HGG), a disease with significant unmet medical need. Tocagen has initiated the Phase 2 portion of a randomized, controlled Phase 2/3 clinical trial of Toca 511 & Toca FC in patients with recurrent HGG, which is designed to serve as a potential registrational trial. More information about the clinical trial can be found at www.tocagen.com/toca5. Tocagen has initiated a clinical trial of Toca 511 & Toca FC in metastatic cancers, including colorectal, pancreatic, lung, breast, renal and melanoma. Tocagen obtained Fast Track designation from the U.S. Food and Drug Administration for Toca 511 & Toca FC as a treatment of recurrent HGG and Orphan drug designation for the treatment of glioblastoma multiforme (GBM), a subset 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). For more information, visit www.tocagen.com or follow @Tocagen.

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SOURCE Tocagen, Inc.

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