Full Press Release Details
The Future of Cancer Therapy
33rd Annual Healthcare Conference
Forward_Looking Statements
_ forward contains certain looking information about ZIOPHARM Oncology that is intended to be covered
by the safe harbor for forward Forward looking statements provided by the Private Securities Litigation
Reform Act of 1995, as amended. looking statements are statements that are not historical facts. Words such as expect(s), feel(s), believe(s),
will, may, anticipate(s) and similar expressions are intended _ forward to identify looking statements. These statements include,
but are not limited to, statements regarding our ability to successfully _ long develop and commercialize our therapeutic products,
our ability to expand our term business opportunities; financial projections and estimates and their underlying assumptions; and future performance. 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 _ forward from those expressed in, or implied or projected by, the looking information and statements.
These risks and uncertainties include, but are not limited to: whether any of our therapeutic candidates will advance further in the 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 any of our therapeutic candidates will be successfully marketed if approved; whether our DNA based
biotherapeutics discovery and development efforts will be successful; our ability to achieve the results contemplated
by our collaboration agreements; the strength and enforceability of our intellectual property rights; competition from pharmaceutical and biotechnology companies; the DNA
development of and our ability to take advantage of the market for based biotherapeutics;
our ability to raise additional capital to fund our operations on terms acceptable to us; general economic conditions; and theotherriskfactors contained inour periodic
andinterimreportsfiledwiththeSEC including, butnotlimitedto,ourannualreporton Form 10 K for the fiscal year ended December
31, 2013 and Quarterly 10 Report on Form Q for the period ended September
30, 2014, and other filings with the SEC, which are available at www.sec.gov. Our audience is 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 non
date hereof, or to reflect the occurrence of or occurrence of any events.
Lewis, M.D., Ph.D., CEO, ZIOPHARM Oncology
Ad_RTS IL 12 Overview
Adenoviral Interleukin _ mediated controlled delivery of 12
Oral Activator Activator
Intratumoral Injection
has leading expertise in clinical translation of designer cytokines
Oral Veledimex Precisely Controls the Expression _IL of 12
Baseline Maximum D1 D7 Post
100 mg 160 mg n=7 Veledimex Dose
Next _ IL steps for 12
Moving to active immunotherapy
ZIOPHARM hasleading expertise inclinical translation of designer cytokines
RheoSwitch Therapeutic System platform for conditional expression of
ZIOPHARM is partnering with MDACC in Boston and Houston
Greg Frost, Ph.D., SVP Health Sector, Intrexon Corporation
Non_Viral Adoptive Cellular Therapies
[Graphic Appears Here]
INTREXON and ZIOPHARM Announce Exclusive Licensing Agreement with MD Anderson Cancer Center for Chimeric Antigen Receptor (CAR) T Cell
and Associated Technologies for the Development of Non_Viral Adoptive Cellular Therapies
Resulting in Point of Care andOfftheShelf Cellular Solutions
PointofCareProduct OfftheShelfProduct
Integration with RheoSwitch Genetically modify and edit T cells control of T cell propagation and to express CAR and eliminate TCR specificity in patients
AttSite landing pads for precision
Facilitates a no _ culture solution to genetic engineering manufacturing
iPSC processing technologies combined with LEAP to engineer _ HLA an matched product
Recombinases and Transposases
Key Technology Synergies
/ ZIOPHARM / MD Anderson
RheoSwitch Most advanced family of ligands and switches
available Therapeutic System for dynamic range, safety, and spatial/temporal control
Non _ Viral Integration Firstin
human testing of Sleeping Beauty system in Platform hematopoietic cells
Industrialized assembly and screening of multigenic
for synthetic biology
Expertise in development and implementation of novel
Adoptive Cell Therapy immunotherapy trials
Established clinical pipeline for adoptive cellular therapies
&With Traditional Oncology Reimbursement for Cellular Therapies
Avoids Acute PBM Pressures Driven by Cost Density
Reimbursement Sche $$ Failure Rate ents Pay $
Cell and Ligand Reimbursement Years
Laurence J.N. Cooper,
M.D., Ph.D., Professor of Pediatrics at MD Anderson
The technology being discussed has been licensed by MD Anderson to Ziopharm and Intrexon and MD Anderson, which is my employer, will have an equity interest in both companies as a result
of the transaction. As an inventor of the licensed technology, I will share in the proceeds of the consideration received by MD Anderson under the license in accordance with UT System Rules and MD Anderson policies and to that extent, I have a
financial interest in both companies.
Increase in Patient Number Disrupts Manufacturing Scalability
Point of Care and Off the Shelf Manufacturing
Dual CAR _ Distribution Approaches for T
Genetically modified T cells
Onepatient Multiple Patients
Specific Autologous T cells Allogeneic T cells the
Patien Real time In advance of need helf
Disease cure Disease control
Point of _ care(POC) _ Centralized
Registration Trials & Global Distribution with Multiple Pharma Partners
Steps Toward Point of Care Distribution
T cell Acquisition T cell Manufacturing Time
Genetic Modification Infusion
Steps Toward Off the Shelf Distribution
T cell Acquisition Autologous
Infusion Off the Shelf Allogeneic
Portfolio of Effector Cells
Antigen Receptor (CAR+) T cells:
Target cell surface tumor
associated antigens (TAAs) independent of HLA
2. T Cell Receptor (TCR+) cells:
Target intracellular TAAs dependent on HLA
3. Natural Killer (NK) cells:
Target tumor with loss of HLA
Improving Therapeutic Potential of CART Cells
CAR T Cells for improved potency and persistence
Understanding ofthe effector cellbiology Co _ stimulation CAR with cytokine
Signals 1 & 2 Signal 3
RTS Cell for Gene and based Therapies
+ Addition of activator ligand or control solvent