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This presentation contains certain forward-looking information about ZIOPHARM Oncology, Inc. that is intended to be covered by the safe harbor for
forward-looking statements provided by the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as
may, will, could, expects, plans, anticipates, and believes. These statements include, but are not limited to, statements regarding the progress, timing and results
of preclinical and clinical trials involving the Company s drug candidates, and the progress of the Company s research and development programs. All of such statements are subject to certain risks and uncertainties, many of which are
difficult to predict and generally beyond the control of the Company, that could cause actual results to differ materially from those expressed in, or implied by, the forward-looking statements. These risks and uncertainties include, but are not
limited to: whether chimeric antigen receptor T cell (CAR T) approaches, Ad-RTS-IL-12, TCR and NK cell-based therapies, or any of
our other therapeutic candidates will advance further in the pre-clinical or clinical trials process and whether and when, if at all, they will receive final approval from the U.S. Food and Drug Administration
or equivalent foreign regulatory agencies and for which indications; whether chimeric antigen receptor T cell (CAR T) approaches,
Ad-RTS-IL-12, TCR and NK cell-based therapies, and our other therapeutic products will be successfully marketed if approved; the
strength and enforceability of our intellectual property rights; competition from other pharmaceutical and biotechnology companies; and the other risk factors contained in our periodic and interim SEC reports filed from time to time with the
Securities and Exchange Commission, including but not limited to, our Annual Report on Form 10-K for the fiscal year ended December 31, 2015, and our Quarterly Report on Form
10-Q for the quarter ended September 30, 2016. Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date hereof, and we do not undertake any
obligation to revise and disseminate forward-looking statements to reflect events or circumstances after the date hereof, or to reflect the occurrence of or non-occurrence of any events.
The next generation of DNA-based immunotherapies will need to master cost and control
Bloomberg Markets Tech Pursuits Politics Opinion Businessweek
Deaths in Cancer Trials Remain
Mystery for Promising Therapy
Opinion: Balancing Risks and Rewards
of CAR T-Cell Therapy
New approaches to treating cancer have shown great promise, but they also come with serious risks that give us
By David Harris | December 15, 2016
Analys Questions High
Cost of T Cell Therapies as Potential Frontline Treatment for Blood Cancers
Experimental cancer therapy holds great promise
do the CAR-T Patient Deaths Mean for the Future of the Field?
others face higher manufacturing costs with CAR-T cell treatments
Clinically-Validated Genetic Engineering Tools to Increase Control, Reduce Complexity, and Manage Costs
The RheoSwitch Therapeutic System gene switch enables
precise control over gene expression in vivo
Non-viral Sleeping Beauty system has the potential to eliminate many GMP steps in cell manufacturing ex vivo, thereby reducing complexity of
Pediatric Brain Tumor
GBM & Checkpoint (PD-1)
Combination with OTS NK
CD19 2nd Gen. shortened
CD19 3rd Gen. CAR with mbIL15 (POC)
Off the shelf (OTS) primary NK cells
Genetically-engineered
Sleeping Beauty neoantigen
Sleeping Beauty neoantigen and cytokine
Modified Bacteria (microbiome)
Ad-RTS-hIL-12 + Veledimex
RheoSwitch Therapeutic System (RTS )
Intratumoral injection of Ad-RTS-hIL-12
Veledimex taken by mouth
IL-12 production calls in cellular immune
Cellular immune response shows durability beyond initial IL-12 signaling
A Study of Ad-RTS-hIL-12 + Veledimex in Subjects With
Recurrent or Progressive Glioblastoma
Subjects scheduled for resection
veledimex 3 2 hr before resection
Craniotomy Tumor Resection
Days 1 through 14 (QD)
20mg V + Ad 2x1011 vp
40mg V + Ad 2x1011 vp
30mg V + Ad 2x1011 vp
DANA-FARBER/BRIGHAM AND WOMEN S
PRITZKER SCHOOL OF MEDICINE
Northwestern Memorial Hospital Ability to control IL-12 gene expression in multicenter study
Clinicaltrials.gov: NCT02026271
Responds to the Presence/Absence and Dose of Veledimex in Recurrent or Progressive Glioblastoma Patients
Proportional output and effects*
*As of October 14, 2016
Ad-RTS-hIL-12 + Veledimex in Subjects With Breast Cancer: Positive Biomarker Data*
Baseline: Cold Tumor
6 Weeks: Inflamed Tumor
*European Society for Medical Oncology 2016
Interim Study Results
Based on tolerability and survival benefit (median OS=12.7 months, n=15), 20 mg was selected for an expansion cohort and we are following patients overall
Ad-RTS-hIL-12 + veledimex is
well tolerated and suggests a survival benefit over historical controls at 6, 9, and 12 months (median OS=9.6 months, n=25)
Toxicities were tolerable, predictable
and reversible upon discontinuing veledimex
There is a strong correlation between veledimex dose, BBB penetration, and
These data demonstrate that the RTS gene switch works in humans toggling not only as a switch to turn
on and off the production of IL-12, but also as a rheostat to control the level of IL-12
* As of January 6, 2017
Interim Median Overall
Survival (mOS) Relative to Historical Controls
Studies in recurrent GBM
Ad-RTS-hIL-12 + 20V n=15
Optune Randomized Phase III study, EF-11iv Physician s choice n=117
Polymer placebo n=112
Randomized Multi-institutional PhII Study
Carmustine wafer n=110
Multi-institutional PhII Study Temozolomide n=68
Randomized PhII Study (BELOB) Lomustine n=46
* As of January 6, 2017
Registration Pathway
for Recurrent Glioblastoma
face-to-face meeting with FDA in early Q1 2017
Determine design of pivotal trial to
be initiated in 2017
74,000 new cases annually worldwide
12,120 in the U.S. in 2016
Recurrence rates are ~90%
Less than 10% of patients are alive at 5 years
Source: World Health Organization, American
Cancer Society, Central Brain Tumor Registry of the United States
Clinical Studies with Ad-RTS-hIL-12 + Veledimex in 2017