Alaunos Therapeutics, Inc. · NASDAQ:TCRT
Alaunos Therapeutics — Ziopharm Oncology — Wells Fargo Healthcare Conference 2015
29 slides from Alaunos Therapeutics's conference presentation, Sep 10, 2015
Slide 1: Wells Fargo Healthcare Conference September 2015
Wells Fargo Healthcare Conference SEPTEMBER 2015 Exhibit 99.1
Slide 2: Forward-looking Statements
Forward-looking Statements 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 s
Slide 3: Highlights
• NASDAQ: ZIOP • Robust synthetic immunology pipeline Virotherapy & adoptive cell therapy Multiple partnerships • Differentiated technology platforms Gene switch, non-viral integration and viral-based delivery • Multiple trial launches in 2015/2016 • Well capitalized Cash and equivalents of $176.1 m
Slide 4: Delivering on the Premise and Promise of Gene and Immune-based Therapies
Delivering on the Premise and Promise of Gene and Immune-based Therapies Fully Aligned with research and development (Intrexon) Big pharma partnership (CARs) Bio-processing for next-generation products Aligned with academic centers for manufacturing of cells for investigator-initiated trials Contrac
Slide 5: Our Approach
Virotherapy RTS ® Gene Switches Viral & Non-viral Gene Integration Cell Therapy CAR-T, TCR and NK Cells Autologous and Allogeneic Our Approach
Slide 6: IL-12 Virotherapy
IL-12 Virotherapy Adenoviral vector engineered to express IL-12 utilizing RheoSwitch Therapeutic System ® (RTS ® ) gene switch Injected intra-tumor with IL-12 expression controlled via administration of oral activator ligand veledimex Regulated intra-tumoral expression of IL-12 promotes activation o
Slide 7: RTS Gene Switch Technology
Improved persistence and survival in tumor microenvironment Limits on-target, off-tumor toxicities Administer virus locally to achieve systemic anti-tumor effects (Breast) Administer virus locally to achieve local anti-tumor effects (GBM) RTS ® Gene Switch Technology
Slide 8: Ad-RTS-IL-12 Gene Therapy Clinical Response To Date
Expression of functional IL-12 in human subjects by direct intra-tumoral injection of Ad-RTS-hIL-12 + oral veledimex generates downstream IFNg production and rapid systemic elevation of IL-10 and IP-10 Documented systemic immune activation and clinical effect in advanced, heavily pretreated melanoma
Slide 9: Phase 1 Study of IL-12 Gene Therapy in Recurrent or Progressive Glioblastoma
Early Response Data Expected in 4Q 2015 Phase 1 Study of IL-12 Gene Therapy in Recurrent or Progressive Glioblastoma/Grade III Malignant Glioma A Multi-Center Gene Therapy Study A single cycle of Ad-RTS-hIL-12 + escalating veledimex 1O : Safety and tolerability 2O : Determine MTD and immune response
Slide 10: Phase 1b/2 Study of IL-12 Gene Therapy in Breast Cancer
Immunotherapy phase of treatment A single cycle of Ad-RTS-hIL-12 + veledimex goal of maintaining or improving pre- study response O : Safety and tolerability O : ORR, disease control and biomarkers N = 40 locally advanced or metastatic breast cancer of all subtypes up to 20% (8 subjects) with HER2+
Slide 11: Pipeline
Pipeline Demonstrated the ability of Veledimex to cross blood-brain barrier in animal models (AACR 2014) Proof-of-concept in humans being explored in ongoing multi-center gene therapy trial Combination therapies Veledimex for activation of genetically modified T cells in brain
Slide 12: Cell Therapy
CAR + T cells • Target cell surface tumor- associated antigens (TAAs) independent of HLA • “Public” cell surface antigens TCR + T cells • Target intracellular TAAs dependent on HLA • “Private” intra-cellular antigens NK cells • Target tumor with loss of HLA • No “antigens” Cell Therapy
Slide 13: Sleeping Beauty (SB) Non-viral Gene Integration
Sleeping Beauty (SB) Non - viral Gene Integration Step 1 (safety) Step 2 (efficacy) Step 3 (scale)
Slide 14: Pre-emptive Donor Lymphocyte Infusion with CD19-directed CAR+ T cells after Autologous HSCT
Pre-emptive donor lymphocyte Infusion with CD19- directed CAR + T cells infused after autologous hematopoietic stem-cell transplantation (HSCT) Population 7 patients with advanced non-Hodgkin lymphoma (NHL) Safety Patients have not demonstrated any acute or late toxicity to CAR + T-cell infusions Ef
Slide 15: Pre-emptive Donor Lymphocyte Infusion with CD19-directed CAR+ T cells after Allogeneic HSCT
Pre-emptive donor lymphocyte infusion with CD19- directed, CAR + T cells infused after allogeneic HSCT *Wilhelm et al. J Oncol Pharm Practice, 2014, 20:257 Population patients with advanced CD19 + ALL (n=17) or NHL (n=2) Safety 3 patients developed GVHD which is lower than the expected range after a
Slide 16: Persistence of Infused CAR+ T Cells
Persistence of Infused CAR + T Cells Trial at MDACC, Updated from EHA 2015; Will be submitted for publication
Slide 17: Improving Therapeutic Potential of CAR+ T Cells
Improving Therapeutic Potential of CAR + T Cells Signals 1 & 2 Signal 3
Slide 18: Next-generation Adoptive Cell-based Clinical Trials
1. New CAR designs for patients with hematologic malignancies and solid tumors 2. Combining CAR with cytokines – IL-12 – IL-15 • TCR-based T-cell therapy • NK-cell immunotherapy Next-generation Adoptive Cell-based Clinical Trials
Slide 19: How do we achieve on-target anti-cancer activity?
How do we achieve on-target anti-cancer activity? The Promise and Challenges of DNA-based Immuno-Oncology Avoid
Slide 20: Strategies to widen therapeutic index to Target Solid Tumors
Strategies to widen therapeutic index to Target Solid Tumors
Slide 21: Off-the-shelf (OTS) CAR T-cells
oO. ZIOPHARM Oncology Current manufacturing Off-the-shelf (OTS) CAR* T-cells One product for single patient +<«——+One product for many patients Lower Quality/quantity of T-cells¢——+T-cells from healthy donor Time to generate product +—P re-prepared for infusion Heterogeneity of product +—Limited het
Slide 22: Elimination of Endogenous TCR
Elimination of Endogenous TCR
Slide 23: Fitting Together the Pieces of the Puzzle
NK cells T cells TCRs CARs Virotherapy Cytokines Remote Controlled Fitting Together the Pieces of the Puzzle
Slide 24: Clinical Programs 2015
Ziopharm 2015 Ad-RTS-IL12 Multicenter CARs MDACC Clinical Programs
Slide 25: Clinical Programs 2016+
Ziopharm 2016+ Ad-RTS-IL12 CARs and interleukin Multicenter NK cells TCRs CARs Multicenter MDACC MDACC Multicenter Clinical Programs
Slide 26: Abstracts
Abstracts • European Hematology Association (June 2015) – Donor-derived, CD19-directed, CAR-modified T cells infused after allogeneic hematopoietic stem-cell transplantation as pre- emptive donor lymphocyte infusion in patients with CD19+ malignances • CRI-CIMT-EATI-AACR ( September 2015) – Demonstr
Slide 27: Upcoming Conferences: With More to Come for 2015
CRI-CIMT-EATI-AACR Immunotherapy Conference September 16 - 19, NY, NY SITC November 4 - 8, National Harbor , MD SNO November 19-23, San Antonio, TX CAR-T Summit November 12 - 13, Boston, MA ASH December 5-8, Orlando FL SABCS December 9-12, San Antonio TX Additional papers …With More to Come for 2015
Slide 28: ZIOPHARM Oncology Pipeline
ZIOPHARM Oncology Pipeline Solid Tumors 2016+ Heme 2015+ Virotherapy Multi- center trials 2015+ Multi Modal 2016+ Expanded Indications 2016+ Single Agent 2015+ CAR Cytokine 2016+ Adoptive Cell Therapy TCR 2016+ CAR 2015+ NK cells 2016+ T cells 2015+ Allogeneic 2016+ Autologous 2015+
Slide 29: Wells Fargo Healthcare Conference September 2015
Wells Fargo Healthcare Conference SEPTEMBER 2015
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