Ziopharm Oncology Presents Data on Very Rapid Production of CAR T Cells at Keystone Symposia Emerging Cellular Therapies: T Cells and Beyond

Human CAR+ T cells with membrane-bound IL-15 manufactured in less than two days exhibited anti-tumor activity and persistence in pre-clinical studies

BOSTON, MA, USA I February 12, 2018 I Ziopharm Oncology, Inc. (Nasdaq:ZIOP), a biotechnology company focused on development of next generation immunotherapies utilizing gene- and cell-based therapies to treat patients with cancer, today announced data demonstrating point-of-care (P-O-C) manufacturing of human T cells expressing chimeric antigen receptor (CAR) that persist and have an anti-tumor effect in preclinical models were presented at the Keystone Symposia Emerging Cellular Therapies: T Cells and Beyond in Keystone, Colorado.

The data presented showed T cells expressing CD19-specific CAR with membrane-bound IL-15 (mbIL15) were generated with the non-viral Sleeping Beauty system in less than two days and did not require ex vivo activation or propagation. T cells designed to express mbIL15 showed greater persistence and more potent antitumor activity than comparator T cells without mbIL15 in these studies.

Lenka V. Hurton, Ph.D., a researcher in the Division of Pediatrics at the University of Texas MD Anderson Cancer Center, presented the findings in a talk entitled, "Rapid production of T cells co-expressing CAR and membrane-bound IL-15 potentiates antitumor activity and promotes in vivo memory." She also presented a poster under the same title during the Keystone Symposia.

Ziopharm is advancing its non-viral Sleeping Beauty platform towards using its point-of-care, or P-O-C, technology, a very rapid manufacturing process of genetically modified CAR+ T cells co-expressing mbIL15, with the first in-human trial utilizing this approach expected to commence in 2018. Ziopharm believes that manufacturing under P-O-C has the potential to reduce the costs associated with T-cell therapies and the potential to broaden application based on avoiding the need for centralized manufacturing as is the case when using a virus to genetically modify T cells.

Dr. Hurton's poster and presentation slides are based on research conducted in collaboration with The University of Texas MD Anderson Cancer Center and Precigen Inc., a wholly-owned subsidiary of Intrexon Corporation (NYSE:XON). The poster is available in the Presentations and Publications section of the Company's website, www.ziopharm.com.

About Ziopharm Oncology, Inc.

Ziopharm Oncology is a Boston-based biotechnology company focused on development of next-generation immunotherapies utilizing gene- and cell-based therapies to treat patients with cancer. In partnership with Precigen Inc., a wholly-owned subsidiary of Intrexon Corporation (NYSE:XON), Ziopharm is focused on the development of two platform technologies designed to deliver safe, effective and scalable cell- and viral-based therapies for the treatment of multiple cancer types: Controlled IL-12 and Sleeping Beauty for genetically modifying T cells. The Company's lead asset, Ad-RTS-hIL-12 plus veledimex, has demonstrated in clinical trials the potential to control interleukin-12, leading to an infiltration of T cells that fight brain cancer. The Company also is advancing therapies using Sleeping Beauty, a non-viral approach to genetically modify chimeric antigen receptor (CAR+) and T-cell receptor (TCR+) T cells, which target specific antigens in blood cancers and neoantigens solid tumors. Sleeping Beauty is designed using the Company's point-of-care technology, a shortened manufacturing process which potentially can be developed as a decentralized manufacturing process based in hospitals. These programs are being advanced in collaboration with Precigen and with MD Anderson Cancer Center, the National Cancer Institute and Merck KGaA, Darmstadt, Germany.

SOURCE: ZIOPHARM Oncology

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