FDA accepts ZIOPHARM's IND for ZIN ATI-001 to treat melanoma

ZIOPHARM Oncology, Inc. (Nasdaq: ZIOP), a drug development company employing small molecule and synthetic biology approaches to cancer therapy, announced today that the U.S. Food & Drug Administration (FDA) has accepted the Company's investigational new drug (IND) application to begin clinical study of ZIN ATI-001, a novel DNA-based therapeutic candidate also known as Ad-RTS-IL-12 + AL (INXN 2001/1001), in oncology. When initiated, the Phase I study will evaluate safety in addition to immunological and biological effects of the therapeutic candidate in patients with melanoma. ZIN ATI-001 is the second clinical oncology product candidate from the ZIOPHARM-Intrexon Corporation exclusive synthetic biology channel partnership.

ZIN ATI-001 employs an adenoviral vector to deliver, directly into the patient's own cells, a gene which expresses Interleukin-12 (IL-12), a potent, naturally occurring anticancer cytokine central to the initiation and regulation of cellular immune responses. Production of IL-12 within cells is then tightly regulated by the Intrexon RheoSwitch Therapeutic System™ (RTS^TM), a "gene switch" controlled by an orally administered activator ligand (AL). Preclinical studies have shown that the immunological mechanism of action of ZIN ATI-001 is similar to that of ZIN-CTI-001 (DC-RTS-IL-12 + AL), ZIOPHARM's most advanced DNA-based product candidate, currently in Phase Ib. Positive clinical data of ZIN-CTI-001, the first-ever to demonstrate small molecule-controlled production of an anticancer protein in humans, were recently presented at the 2011 Annual Meeting of the American Society of Clinical Oncology.

"The rapid acceptance of this IND underscores ZIOPHARM's strengths as our exclusive partner for the development of DNA-based therapeutics in oncology," stated RJ Kirk, Intrexon's Chairman and CEO, and a Director of the ZIOPHARM Board of Directors. "ZIN ATI-001, which offers an effective, yet simpler approach to introducing IL-12 therapy, is the first of many products we expect to introduce into the clinic as partners over the next two years. ZIOPHARM's understanding of the development spectrum, from preclinical work through large outcome studies, ensures that the great promise of this technology is delivered quickly and intelligently."