BOSTON, MA, USA I September 18, 2015 I ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on the development and commercialization of new cancer immunotherapies, today announced the presentation of clinical and preclinical data from the Company’s Ad-RTS-IL-12 program in various malignancies at the CRI-CIMT-EATI-AACR Inaugural International Cancer Immunotherapy Conference taking place September 16-19, 2015 in New York City. Ad-RTS-IL-12 is a novel gene therapy candidate for the controlled expression of IL-12, a critical protein for stimulating an anti-cancer T cell immune response.

The first poster presentation, titled, “Demonstration of Systemic Antitumor Immunity via Intratumoral Regulated Expression of IL-12 in Advanced Breast Cancer and Melanoma Patients,” highlights additional evidence of systemic immune activation with AD-RTS-hIL-12 and veledimex in advanced melanoma and breast cancer patients. The data were drawn from two previously completed open-label Phase II clinical studies: one which enrolled 12 patients with metastatic advanced stage breast cancer, and one which enrolled 26 patients with metastatic melanoma, both exploring the immune-mediated local and systemic anti-tumor effects of Ad-RTS-hIL-12 and veledimex. Among other findings, treatment with Ad-RTS-hIL-12 and veledimex in patients with melanoma was found to increase in the immune cytokine IL-12 and downstream cytokines, IFNg, IP-10 and IL-10, resulting in a significant increase in tumor infiltrating lymphocytes both locally, in injected lesions, and systemically, in non-injected lesions.

The second poster presentation, titled “Demonstration of Systemic Antitumor Immunity via Intratumoral Regulated Expression of IL-12 as a Gene Therapy Approach to Treatment of Cancer,” demonstrates the anti-tumor effects and tolerability of Ad-RTS-mIL-12 in murine models of glioblastoma (brain cancer), colon cancer and melanoma. The data demonstrated dose-related increases in veledimex in both plasma and brain tissue, leading to an increase in expression of IL-12 mRNA and in-tumor IL-12p70 expression with minimal increase in serum IL-12. Ad-RTS-mIL-12 + veledimex also demonstrated systemic memory upon rechallenge in multiple syngeneic mouse models, providing further evidence of systemic anti-tumor immunity elicited by Ad-RTS-mIL-12.

Both posters are available under “Presentations and Publications” on the Company’s website at www.ziopharm.com. In addition to the poster presentations, Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM, is scheduled to deliver an oral presentation titled “Non-viral gene transfer to redirect T cell specificity” on Saturday, September 19.

“Clinical and preclinical data from our Ad-RTS-IL-12 program continue to demonstrate the ability to achieve both local anti-tumor effect and systemic effects across a variety of malignancies,” said Francois Lebel, M.D., Executive Vice President, Research and Development, Chief Medical Officer at ZIOPHARM. “We look forward to understanding how these results translate to our ongoing, multicenter Phase 1 gene therapy study in malignant glioma as well as our Phase 1b/2 Study in locally advanced or metastatic breast cancer. These data also provide support for our integration of both the RTS® gene switch and cytokines into our broader adoptive cell therapy programs.”

About ZIOPHARM Oncology, Inc.:

ZIOPHARM Oncology is a Boston, Massachusetts-based biotechnology company employing novel gene expression, control and cell technologies to deliver safe, effective and scalable cell-based therapies for the treatment of cancer. The Company’s synthetic immuno-oncology programs, in collaboration with Intrexon Corporation (NYSE:XON) and the MD Anderson Cancer Center, include chimeric antigen receptor T cell (CAR-T) and other adoptive cell based approaches that use non-viral gene transfer methods for broad scalability. The Company is advancing programs in multiple stages of development together with Intrexon Corporation’s RheoSwitch Therapeutic System® (RTS®) technology, a switch to turn on and off, and precisely modulate, gene expression in order to improve therapeutic index. The Company’s pipeline includes a number of cell-based therapeutics in both clinical and preclinical testing which are focused on hematologic and solid tumor malignancies.

SOURCE: ZIOPHARM Oncology