NEW YORK, NY, USA I April 8, 2014 I ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on the development and commercialization of new cancer therapies, announced today the presentation of data from a study demonstrating the anti-tumor effects and tolerability of Ad-RTS-mIL-12 in a glioblastoma (brain cancer) murine model. Ad-RTS-IL-12 is a novel DNA-based therapeutic candidate for the controlled, local expression of IL-12, an important protein for stimulating an anti-cancer T cell immune response. 

The study, titled “Treatment of glioblastoma through the controlled localized production of IL-12 by the RheoSwitch Therapeutic System® Platform” (abstract 3647), was presented at the American Association for Cancer Research 2014 Annual Meeting (AACR 2014) taking place April 5-9, 2014 in San Diego, California.  It was conducted jointly by ZIOPHARM and Intrexon Corporation (NYSE:XON), ZIOPHARM’s exclusive channel partner for the development of synthetic biology therapeutics in oncology.

For the study, intratumoral administration of Ad-RTS-mIL-12 was examined in an orthotopic murine glioma model, with production of IL-12 controlled using Intrexon’s RheoSwitch Therapeutic System® (RTS®) platform and oral administration of the activator ligand veledimex. The primary goals of this preclinical study were to examine how Veledimex crosses the blood brain barrier, and to compare dose-related effects on survival. Veledimex was found to effectively cross the blood brain barrier, with dose-related increases in plasma and brain tissue exposure, and no accumulation in brain tissue following repeat dosing.

The study data demonstrated that administration of Ad-RTS-mIL-12 + veledimex resulted in dose-related increases in survival of four- to five-fold, without exhibiting an adverse safety profile, when compared to median survival in vehicle control groups that included temozolomide, bevacizumab (Avastin®) and dexamethasone.

“The activation of a T-cell immune response by Ad-RTS-IL-12 is a promising approach to treating glioblastoma, which is a very challenging and aggressive form of brain cancer,” said Francois Lebel, M.D., senior vice president, clinical development and medical operations at ZIOPHARM. “We have now demonstrated in multiple preclinical studies that Ad-RTS-mIL-12 enables localized, controlled delivery of IL-12 in the brain and can produce a dose-dependent reduction in tumor growth with prolonged survival. We are very excited to start the clinical translation of Ad-RTS-IL-12 in glioblastoma.”

About ZIOPHARM Oncology, Inc.:

ZIOPHARM Oncology is a Boston, Massachusetts-based biotechnology company employing novel gene expression and control technology to deliver DNA for the treatment of cancer. ZIOPHARM’s technology platform employs Intrexon Corporation’s RheoSwitch Therapeutic System® technology to turn on and off, and precisely modulate, gene expression at the cancer site in order to improve the therapeutic index. This technology is currently being evaluated in Phase 2 clinical studies of the immune system cytokine interleukin-12 for the treatment of breast cancer and advanced melanoma. Multiple new Investigational New Drug applications for new targets using synthetic biology technology are expected through 2015. ZIOPHARM is also developing novel small molecules as potential cancer therapeutics.

SOURCE: Ziopharm