BOSTON, MA, USA I May 22, 2014 I ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on the development and commercialization of new cancer therapies, announced today results demonstrating the potent anti-tumor and anti-cancer stem cell (CTC) effects of Ad-RTS-IL-12 in a glioma (brain cancer) model, and in other preclinical and clinical settings. Ad-RTS-IL-12 is a novel DNA-based therapeutic candidate for the controlled expression of IL-12, an important protein for stimulating an anti-cancer T cell immune response. The presentation, titled “Intratumoral Regulated Expression of IL-12 as a Gene Therapy Approach to Immunotherapy,” was presented in an oral session at the 17th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), held May 21-24 in Washington, DC.

Results from human and laboratory studies of Ad-RTS-IL-12 demonstrate that precise control of IL-12 gene expression levels can be achieved using Intrexon Corporation’s (NYSE:XON) RheoSwitch Therapeutic System® (RTS®). Rapid, tight modulation of in vivo expression of IL-12 using the activator ligand veledimex was demonstrated across these studies. When IL-12 expression is “switched on” it rapidly leads to expression and an immune response. This immune response is characterized by an increase in tumor infiltrating lymphocytes with system wide immune activation. This modulated immune response has resulted in anti-tumor effects in both injected and systemic non-injected legions in Phase 1 and Phase 2 studies of Ad-RTS-IL-12 in subjects with melanoma and breast cancer.

The data presented today further demonstrate that Ad-RTS-IL-12 has potent anti-cancer effects in a glioma model, showing both a reduction in tumor mass and prolonged survival when compared to existing treatment standards. The data also show a significant reduction in brain cancer stem cells, as measured by dramatically reduced nestin levels. Brain cancer stem cells are thought to play a critical role in recurrence and metastasis.

“Ad-RTS-IL-12 seems to offer precise control over a potent immuno-oncology weapon, the IL-12 cytokine,” said Antonio Chiocca, MD, PhD, Prof. and Chairman of Neurosurgery at Brigham and Women’s Hospital and Harvard Medical School. “With malignant glioma in particular, delivery of IL-12 is one of several promising experimental approaches being tested, but unlike other therapies the ability to control IL-12 expression in vivo may prove to be ground breaking. This is increasingly being demonstrated both in preclinical models, as well as in the clinic, where patients are showing reversal of immuno-toxicity, including cytokine storm, and then resume therapy.” He continued: “IL-12’s role in reducing cancer stem cells is also very encouraging, as the role of these cells in recurrence and metastasis is increasingly well understood. I look forward to further data from these clinical and preclinical programs, as well as to the initiation of a Phase 1 study of Ad-RTS-IL-12 in brain cancer.”

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 Oncology