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Clinical stage synthetic biology cancer company 2. We believe we are the farthest advanced at regulating gene function in vivo in humans, regulating gene expression, and titrating levels of expression 3. Lead clinical program is Ad RTS IL 12 4. We have multiple novel programs driving towards IND, and expect at least 8 INDs through 2015 5. These are built on a similar platform to Ad RTS IL 12, and in place of IL 12 will include different genes of interest for immunotherapy, cell signal targeting and novel multigenic approaches 6.
ASCO 2013 Screening C1D15 Subject 111 (160 mg) 17 September 2013 Prominent Inflammatory Response Correlates with High levels of IFN Initial increase in lesion size due to inflammatory response seen at Cycle 1 Day 16 Lesion was undetectable at Cycle 2 Day 1 Subject ultimately progressed and was taken off study C2D1 18 September 2013 C1D1 C1D16 Dose dependent Anti Tumor Activity of Ad RTS mIL 12 + Activator Ligand (AL) in Murine 4T1 Breast Cancer Model 0 2 4 6 8 10 12 14 16 0 4 8 12 16 20 24 28 32 Time (Days) Vehicle Ad RTS mIL 12 INXN 1001 (AL) 15 mg/m2 Ad RTS mIL 12 + AL 15 mg/m2 Ad RTS mIL 12 + AL 30 mg/m2 Ad RTS mIL 12 + AL 75 mg/m2 Ad RTS mIL 12 + AL 150 mg/m2 Tumor volume reached 100 200 mm 3 19 September 2013 Glioblastoma Multiforme: IL 12 Preclinical Activity 100% survival observed with Ad RTS IL 12 + AL or DC RTS IL 12 + AL INXN 1001 dosing Day 4 to EOS at ~ 675 mg/m ² /day in chow; DC RTS IL12 or Ad RTS IL12 on Day 5 Kaplan Meier Survival in GL261 Orthotopic Syngeneic Mouse Glioma Model 0 10 20 30 40 50 60 70 80 0 20 40 60 80 100 No Treatment 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) 20 September 2013 Highlights of IL 12 Program Currently demonstrating safety and efficacy in Phase 2 POC studies Preparing for combination studies in melanoma, breast cancer Expanding clinical program to GBM IL 12 program reaching key milestones in 2013/2014 21 September 2013 Incidence Melanoma 76,690 Breast Cancer 234,580 Glioblastoma 18,000 Significant Market Potential for IL 12 Source: American Cancer Society 2013/Schwartzbaum 2006 22 September 2013 1.
Genomic DNA and total RNA were extracted and analyzed as described. 1 Livak KJ and TD Schmittgen. 2001. Methods 25(4):402 8 . ASCO 2013 Off Off Off Off On 16 September 2013 5604 5.4 5778 0.5 0 1000 2000 3000 4000 5000 6000 7000 Screening C1D2 C1D15 Screening C1D2 C1D15 Subject 107 (100 mg) Subject 111 (160 mg) IL 12 Fold Change Relative to Screening Visit On 1 1 1 Cytotoxic T Cells Memory T Cells Increase in Tumor following Treatment with Ad RTS hIL 12 Images were obtained using an Aperio ScanScope XT whole slide imager and digitized at 20x.
Data will be presented near term at AACR/NCI/EORTC, SABCS, and additional scientific meetings and publications ZIOP 3 September 2013 DNA Based Medicine Enables Therapies with desired biofunction to be purpose built The concentration/dose of biologic therapy to be precisely controlled Improvement in therapeutic index, because potent biologic modifiers can be turned on and off and targeted to specific tissues and cells Pursuit of combination biologic therapy in an economically feasible manner 4 September 2013 Powerful, scalable technology platform Potential for innovation in cancer drug development: ZIOP collaboration with Intrexon enables: better control of therapeutic index/frequency multigenic approaches pipeline expansion with multiple INDs attractive partnership opportunities Strategic focus on synthetic biology Foundation in Place for Significant Growth New cancer treatment paradigm Tumor targeting Statistical modeling Precision engineering 5 September 2013 Clinical stage synthetic biology platform (DNA and cell based therapy) driving development Our advantage is that we are the farthest advanced at precisely regulating gene function in vivo in humans Multiple value creating milestones in 2013/14 Pre clinical/clinical data Scientific publications and presentations Ad IL 12 advancing through Phase 2 POC studies Poised to deliver multiple new INDs in 2014/15/16 New IND submissions will expand into new genes of interest, and cancer indications On Track to Execute 6 September 2013 Cells DC T Cell B Cell Macrophage NK cell Treg Other 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 Anti tumor function MSC Epithelial Endothelial Fibroblast and ECM Tumor cell Tumor and microenvironment Effectors Immune cells Non immune Cytokines mAb scFv scFv toxin Systemic decoy Intracellular decoy Metabolic Enzyme Protein Ligand RNA Molecular and Cellular Oncology DNA Coded Toolset 7 September 2013 Intrexon Collaboration: Fueling the IND Engine Genetic control of cells Controlled delivery of therapeutic proteins cancer drug translational and development expertise Promising portfolio of cancer therapeutics gene engineering and cell control technology 8 September 2013 Upcoming Milestones Program Milestone Timing 2013 2014 IL 12 Phase 2 breast cancer study data 2H 2013/2014 Phase 2 advanced melanoma study data 2H 2013/2014 Initiate Phase 1/2 glioblastoma multiforme study Initiate Phase 2 melanoma combo study Initiate Phase 2 breast cancer combo study 1H 2014 1H 2014 1H 2014 Synthetic Biology Report discovery and preclinical data 2H 2013/1H 2014 Submit new INDs for monogenic/multigenic studies 2H 2014 Small Molecule Program Report interim SCLC data (MATISSE) 1H 2014 Publications and Presentations AACR NCI EORTC; SABCS; Scientific Publications 2H 2013 Corporate Seek partnering opportunities Pursue new cancer targets Divest non strategic assets Ongoing 9 September 2013 The Potential of DNA Based Therapeutics Improve treatment of all cancers, starting with breast cancer, melanoma, and GBM Treat currently incurable cancers Generate a multi billion dollar opportunity 10 September 2013 A Growing Oncology Portfolio IND Compound Pre Clinical Phase 1 Phase 2 Ad RTS IL 12 Melanoma Breast GBM Cell based programs Cell signal targeting Multigenic platforms Immunotherapy Programs 11 September 2013 Multiple Opportunities for External Partnering Aggressive IND Strategy Supporting Future Growth 12 September 2013 DNA Based Therapeutics Controlled Delivery of Therapeutic Proteins with RheoSwitch : This is the most advanced clinical method to turn genes on and off Dose control Orally activated biologic on/off switch Local High Potency Monogenic/Multigenic Gene Expression RheoSwitch 13 September 2013 Initial Target: Interleukin 12, A Potent Cancer Therapeutic Cytokine with anti tumor effects Potent Stimulation of T cells and gamma interferon Narrow therapeutic index/ toxicity has limited IL 12 utility Improved therapeutic index may be possible using RheoSwitch technology 14 September 2013 IL 12 Production is Modulated by Activator Ligand in HT 1080 Cells On Off On AL=75nM 15 September 2013 Regulated expression of IL 12 in the tumor by switch control Subjects received intratumoral injection of 1x10 12 viral particles (INXN 2001) on Day 1 of each cycle and INXN 1001 (subject 107, 100 mg; subject 111, 160 mg) on Days 1 7 of each cycle.
Clinical stage synthetic biology cancer company 2. We believe we are the farthest advanced at regulating gene function in vivo in humans, regulating gene expression, and titrating levels of expression 3. Lead clinical program is Ad RTS IL 12 4. We have multiple novel programs driving towards IND, and expect at least 8 INDs through 2015 5. These are built on a similar platform to Ad RTS IL 12, and in place of IL 12 will include different genes of interest for immunotherapy, cell signal targeting and novel multigenic approaches 6.
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 o or non occurrence of any events. 2 September 2013 1.
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, 2012, and our Quarterly Report on Form 10 Q for the fiscal quarter ended June 30, 2013.
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.
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.
BioCentury Newsmakers Jonathan Lewis, MD, PhD Chief Executive Officer The Future of Cancer Therapy Exhibit 99.1 September 2013 Forward Looking Statements 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.
Data will be presented near term at AACR/NCI/EORTC, SABCS, and additional scientific meetings and publications ZIOP 23 September 2013 BioCentury Newsmakers Jonathan Lewis, MD, PhD Chief Executive Officer The Future of Cancer Therapy September 2013