View:
Download DOC
Download PDF
Download XLS
8-K

 

 

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

 

 

FORM 8-K

 

 

CURRENT REPORT

PURSUANT TO SECTION 13 OR 15(d)

OF THE SECURITIES EXCHANGE ACT OF 1934

Date of report (Date of earliest event reported): March 28, 2014

 

 

ZIOPHARM Oncology, Inc.

(Exact Name of Registrant as Specified in Charter)

 

 

 

Delaware   001-33038   84-1475672

(State or Other Jurisdiction

of Incorporation)

  

(Commission

File Number)

  

(IRS Employer

Identification No.)

 

One First Avenue, Parris Building 34, Navy Yard Plaza

Boston, Massachusetts

   02129
(Address of Principal Executive Offices)   (Zip Code)

(617) 259-1970

(Registrant’s telephone number, including area code)

Not applicable

(Former Name or Former Address, if Changed Since Last Report)

 

 

Check the appropriate box below if the Form 8-K is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

 

¨ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425).

 

¨ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12).

 

¨ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)).

 

¨ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)).

 

 

 

Item 8.01 Other Events

On March 28, 2014, ZIOPHARM Oncology, Inc., or the Company, will present the attached discussion of the Company’s synthetic-biology development strategy and milestones at the 21st Annual Future Leaders in the Biotech Industry Conference in New York, New York being held on Friday March 28, 2014.

A copy of the above referenced presentation is filed as Exhibit 99.1 to this Current Report on Form 8-K.

 

Item 9.01 Financial Statements and Exhibits

 

  (d) Exhibits

 

Exhibit

No.

   

Description

99.1    Presentation of the Company dated March 28, 2014

 

2

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

 

    ZIOPHARM Oncology, Inc.
    By:  

/s/ Kevin G. Lafond

Date: March 28, 2014     Name:   Kevin G. Lafond
    Title:   Vice President Finance, Chief Accounting Officer and Treasurer

 

3

INDEX OF EXHIBITS

 

Exhibit

No.

   

Description

99.1    Presentation of the Company dated March 28, 2014

 

4

EX-99.1
March 2014
The Future of Cancer Therapy
www.ziopharm.com
Exhibit 99.1

This
presentation
contains
certain
forward-looking
information
about
ZIOPHARM
Oncology
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. Words such as "expect(s)," "feel(s)," "believe(s)," "will," "may," "anticipate(s)"
and similar expressions are intended to identify forward-looking statements. These statements include, but are not limited to,
statements regarding our ability to successfully develop and commercialize our therapeutic products, our ability to expand our long-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 from those expressed in, or implied or projected by, the forward-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 development of and our ability to take advantage of the
market for DNA-based biotherapeutics; our ability to raise additional capital to fund our operations on terms acceptable to us; general
economic conditions; and the other risk factors contained in our
periodic and interim reports filed with the SEC including, but not limited
to, our annual report on Form 10-K for the fiscal year ended December 31, 2013.  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 date hereof, or to reflect the occurrence of or non-
occurrence of any events.
Forward-Looking Statements
1

Clinical-stage Immuno-Oncology company
Focus on treatment through DNA expression and cell control
Phase 1/2 program
targeting melanoma, breast cancer and
glioma
Intrexon partnership enabling potential paradigm shift
New INDs
through 2015 exploring multigenic approach
to cancer
treatment
ZIOPHARM Today
2

DNA synthesis and delivery enable:
creation of new therapies which
target cancer cells
precise control
of biologic
concentration and dosing
better therapeutic index
through controlled protein delivery
and cellular targeting
economically feasible approach
biologic therapies and Immuno-
Oncology
Why Focus on DNA-Based Medicine?
3

ZIOP: translational medicine and oncology drug development
XON: synthetic biology platform enabling DNA delivery and 
control
Exclusive channel partner agreement for the treatment of all
human cancer:
ZIOP responsible for product development and commercialization
XON responsible for manufacturing, process-improvement R&D, patents
50:50 revenue/net-profit split
Current targets: melanoma, breast cancer, glioma, other cancers
Intrexon Collaboration: Leveraging Our Assets
Page 4

We Have the Tools to Treat Cancer Better
5
Cytokines
mAb
scFv
scFv
toxin
Systemic
decoy
Intracellular
decoy
Metabolic
Enzyme
RNA
Protein
Ligand
DC
T Cell
B Cell
Macrophage
NK cell
Treg
Other immune
cells
Immune Cells
Direct tumor lysis
ADCC
Complement cytotoxicity
Innate immunity stimulation
Adaptive immunity stimulation
Immune evasion inhibition
Pro-apoptosis
Necrosis
Anti-angiogenesis
Growth inhibition
Anti-tumor metabolism
EMT blockade
Non-immune Cells
Effectors
Cells
Anti-tumor
function
Tumor
and microenvironment
MSC
Epithelial
Endothelial
Tumor
Cell
Skeletal
Muscle

Controlled Expression and Delivery of Therapeutic Proteins with Intrexon’s RheoSwitch®: 
This is the most advanced clinical method to turn genes on and off
The Power of RheoSwitch®
Technology
High Potency
Monogenic/Multigenic
Dose-control
Orally activated biologic on/off switch
Gene Expression
RheoSwitch®
6

Ad-RTS-IL-12: Solving the Problem of Serum
Concentration
Page 7
rIL-12 = recombinant
IL-12
hIL-12 = Ad-
RTS-
IL-12
*Atkins, MB et al Clin Cancer Res 1997;3:409-417
11137 ±
3530
27 ±
14
0
2000
4000
6000
8000
10000
12000
rIL-12*
hIL-12

IL-12 Production is Modulated by Veledimex
(Activator Ligand) in HT 1080 Cells
8
OFF
OFF
ON
-1
9
19
29
39
49
59
0
6
12
18
24
30
36
42
48
54
60
66
72
78
84
90
Time after viral transduction (hours)

Oral Veledimex Tightly and Precisely Controls
the Expression of IL-12ß
mRNA in the Tumor
9
Veledimex Dose
n=7
OFF
OFF
ON
0.1
1
10
100
1000
10000
100000
1000000
Baseline
Maximum D1-D7
7 Days Post-treatment
100 mg
160 mg

Cytotoxic T Cells & Memory T Cells (TILs)
Significantly Increase in Tumors Following
Ad-RTS-IL-12 Treatment
Images were obtained using an Aperio ScanScope XT whole-slide imager and digitized at 20x.
10
On
Off
CD8+
CD45RO+

Systemic Immune Activity:
Patterns of Anti-Tumor Response
Patient#
1
3
2
Page 11
-75
-50
-25
0
25
50

High expression of IL-12 mRNA in tumors, tightly controlled by veledimex dose
Clinical Observations to Date
12
We can control gene expression to achieve a desirable immune response
We have seen potent systemic biologic activity and reversible toxicity
Melanoma: potent biologic activity in injected and non-injected lesions
Breast: on-mechanism and on-target toxicity demonstrates powerful immune
response controlled by dose-dependent expression of IL-12
Adverse events consistent with immunotherapy use and immune response;
serious adverse events reversed after veledimex dosing stopped
Increased tumor infiltrating lymphocytes observed in the tumor
microenvironment,
suggesting
multiple
favorable
biologic
effects
of
IL-12
expression

Kaplan Meier Survival in GL261 Orthotopic Syngeneic Mouse Glioma
Model
Glioblastoma Multiforme:
IL-12 Preclinical Activity Measuring Survival
13
Veledimex (AL) dosing Day 4 to EOS at ~ 675 mg/m2/day in chow; DC-RTS-IL-12 or Ad-RTS-IL-12 on Day 5
100% survival observed with Ad-RTS-IL-12 + AL or DC-RTS-IL-12 + veledimex
0
10
20
30
40
50
60
70
80
0
20
40
60
80
100
DC-no vector
DC-RTS-IL12 (MOI 10000)
Ad-RTS-IL12
(5x10
9
)
AL chow
Ad-RTS-IL12
(5x10
9
) + AL
DC-RTS-IL12 (MOI 10000) + AL
Time (Days)
No Treatment

Significant Market Potential for Ad-RTS-IL-12
14
Melanoma
76,690
Breast Cancer
234,580
Incidence
Glioma
18,000

A Growing Oncology Portfolio
15
Compound
Pre Clinical
Phase 1
Phase 2
Ad-RTS-IL-12
IND
Melanoma
Breast
GBM
Cell-based programs
Immuno-Oncology
programs
Cell signal targeting
Multigenic platforms

Human allogeneic mesenchymal stem cells
genetically modified with genes that activate the
immune system to destroy cancer
Multigenic therapeutic antibodies such as single
chain versions of Herceptin®
and Erbitux®
Embedded cellular bioreactors to deliver multiple
proteins systemically using RheoSwitch®
platform
DNA Combination Therapies:
Potential Future INDs
16
*
AACR-NCI-EORTC 2013

Upcoming Clinical Milestones
17
Program
Milestone
Timing
Breast cancer
Initiate Phase 2 combination study with SOC
1H 2014
-
Report preliminary data
1H 2015
Report results from ongoing Phase 2 study
2014
Melanoma
Initiate Phase 2 combination study with SOC
1H 2014
-
Report preliminary data
YE 2014
Report results from ongoing Phase 2 advanced
melanoma study
2014
Glioblastoma
Initiate Phase 1/2 dose escalation study
1H 2014
-
Report preliminary data
YE 2014

Approx. 100 million shares outstanding
Approx. $68 million in cash and investments
No debt
Development partner/top shareholder:
Intrexon Corp. (NYSE: XON)
Financial Highlights
18

We understand the biologic basis of cancer-
and how to 
engineer
new products
We have the ability to deliver DNA and stimulate a controlled
therapeutic
immune response
Clinical testing is validating our vision
Intrexon
partnership is enabling expansion of our pipeline
Big pharma
is very
interested
in our approach
We have the potential to radically change the economics
of
cancer treatment
Our financial success
will be driven by multiple products and 
collaborations,
not one or two
Why Invest in ZIOPHARM?
Page 19

NASDAQ: ZIOP
The Future of Cancer Therapy
www.ziopharm.com