Alaunos Therapeutics, Inc. · NASDAQ:TCRT
Alaunos Therapeutics — ZIOPHARM Oncology — J.P. Morgan 32nd Annual Healthcare Conference (January 2014)
22 slides from Alaunos Therapeutics's conference presentation, Jan 15, 2014
Slide 1: J.P. Morgan 32nd Annual Healthcare Conference
www.ziopharm.com J.P. Morgan 32nd Annual Healthcare Conference January 2014 Jonathan Lewis, MD, PhD • Chief Executive Officer The Future of Cancer Therapy Exhibit 99.1
Slide 2: 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. Forward-looking statements are statements that are n
Slide 3: ZIOPHARM Today
Clinical-stage, synthetic-biology oncology company Gene based therapies regulating protein expression and controlling cellular function Clear leader in in vivo control of gene regulation and expression Technology channel partner Intrexon Corp. (NYSE: XON) Phase 2 program: Ad-RTS-IL-12 High intratumo
Slide 4: Why Focus on Synthetic DNA-Based Medicine?
Synthetic DNA enables: creation of new therapies with targeted biofunction precise control of biologic concentration and dosing better therapeutic index through controlled protein delivery and cellular targeting economically feasible approach to combination biologic therapies Why Focus on Synthetic
Slide 5: Molecular and Cellular Oncology DNA-Coded Toolset
Molecular and Cellular Oncology DNA-Coded Toolset
Slide 6: The Power of Intrexon's RheoSwitch Technology
Controlled Expression and Delivery of Therapeutic Proteins with RheoSwitch®: This is the most advanced clinical method to turn genes on and off The Power of Intrexon’s RheoSwitch® Technology Local High Potency Monogenic/Multigenic Dose-control Orally activated biologic on/off switch
Slide 7: IL-12 Production Is Modulated by Veledimex
IL-12 Production is Modulated by Veledimex (Activator Ligand) in HT 1080 Cells
Slide 8: Veledimex Tightly Controls IL-12 mRNA Expression in the Tumor
Veledimex Tightly and Precisely Controls the Expression of IL-12 ß mRNA in the Tumor
Slide 9: CD8+ and Memory T Cells Increase Following Ad-RTS-IL-12 Treatment
CD8+ CD45RO+ 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.
Slide 10: Phase 1/2 Melanoma Study
Subjects with unresectable stage III/IV melanoma, n=13 (Phase 1) + n= 8 (Phase 2, ongoing) Intratumoral injection of Ad-RTS-hIL-12 on Day 1 of each cycle Oral administration of veledimex to activate gene expression of hIL-12 Phase 1/2 Melanoma Study Primary Objective Evaluate the safety and tolerabi
Slide 11: Prominent Tumor Infiltrating Lymphocyte Response Correlates with Biologic Activity
C1D1 C1D16 C2D1 Initial increase in lesion size due to immune response seen at Cycle 1 Day 16 Lesion was undetectable at Cycle 2 Day 1 Subject ultimately progressed and was taken off study Prominent Tumor Infiltrating Lymphocyte Response Correlates with Biologic Activity
Slide 12: Glioblastoma Multiforme: IL-12 Preclinical Activity
Kaplan Meier Survival in GL261 Orthotopic Syngeneic Mouse Glioma Model Glioblastoma Multiforme: IL-12 Preclinical Activity 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
Slide 13: Tight control of expression and biologic activity
Tight control of expression and biologic activity High expression of IL-12 mRNA in tumors, tightly controlled by veledimex dose Increased tumor infiltrating lymphocytes (TILs) observed in the tumor microenvironment, suggesting multiple favorable biologic effects of IL-12 expression On-mechanism and
Slide 14: Significant Market Potential for Ad-RTS-IL-12
Significant Market Potential for Ad-RTS-IL-12
Slide 15: IL-12 Next Study Timelines
GBM Phase 1 dose-escalation study IL-12 Next Study Timelines Melanoma Phase 2 combination study with SOC Breast cancer Phase 2 combination study with SOC FPI 1H 2014 Preliminary data YE 2014 FPI 1H 2014 Preliminary data YE 2014 FPI 1H 2014 Preliminary data 1H 2015
Slide 16: A Growing Oncology Portfolio
Pre Clinical Phase 1 Phase 2 A Growing Oncology Portfolio Compound Ad-RTS-IL-12 IND Melanoma Breast GBM Cell-based programs Cell signal targeting Multigenic platforms Immunotherapy Programs
Slide 17: We can intelligently and intentionally construct multi-gene therapies
We can intelligently and intentionally construct multi-gene therapies
Slide 18: Combination Therapies Targeting Multiple Cancers: Potential INDs
Human mesenchymal stem cells (hMSCs) genetically modified with multigenic hIL-12, hIFN a , and CTLA4 decoy Potential use of hMSCs for tumor-targeted delivery of multiple RTS® regulated cancer immunotherapies Integration of modularized protein engineering technology and the RTS® platform to develop h
Slide 19: Upcoming Milestones
Program Milestone Timing Upcoming Milestones IL-12 Phase 2 breast cancer study data Phase 2 advanced melanoma study data Initiate Phase 1/2 glioblastoma multiforme study 1H 2014 Initiate Phase 2 melanoma combo study 1H 2014 Initiate Phase 2 breast cancer combo study 1H 2014 New Indications Report di
Slide 20: Financial Highlights
Approx. 100 million shares outstanding (pro forma) Approx. $77.4 million in cash and investments (pro forma) No debt Channel partner/top shareholder Intrexon Corp. (NYSE: XON) Financial Highlights
Slide 21: Ad-RTS-IL-12 advancing through Phase 2 studies in breast cancer and melanoma
Ad-RTS-IL-12 advancing through Phase 2 studies in breast cancer and melanoma On-mechanism and on-target response and toxicity observed Expanded data anticipated in late 2014 and in 2015 Pre-clinical/clinical data on glioblastoma in 2014 Poised to launch multiple new studies in 2014 and beyond INDs t
Slide 22: Jonathan Lewis, MD, PhD
www.ziopharm.com NASDAQ: ZIOP Jonathan Lewis, MD, PhD Chief Executive Officer The Future of Cancer Therapy
ZIOPHARM Oncology — J.P. Morgan 32nd Annual Healthcare Conference (January 2014) on 6ix