Alaunos Therapeutics, Inc. · NASDAQ:TCRT
Alaunos Therapeutics — Ziopharm Oncology — Corporate Update (December 2015)
25 slides from Alaunos Therapeutics's corporate update, Dec 16, 2015
Slide 1: ZIOPHARM Review and Outlook
ZIOPHARM Review and Outlook December 16, 2015 EXHIBIT 99.1
Slide 2: 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 statements are statements that
Slide 3: Immuno-oncology: Introduction
Immuno-oncology: Introduction Tumor resistance is a hallmark of cancer and thus multiple modalities are needed with different mechanisms of action to overcome tumor escape Native immune response unable to treat cancer because the endogenous programming language in T cells and NK cells is muted by ca
Slide 4: Broad Approach Against Cancer
Broad Approach Against Cancer ZIOPHARM applying multiple modality and multi-delivery approach that encompasses viral & non-viral mechanisms, differentiating it from many other players in adoptive cellular therapy today Positioned to leap frog into leadership in hematological and solid tumor settings
Slide 5: Non-Viral Delivery: Sleeping Beauty
Non-Viral Delivery: Sleeping Beauty Sleeping Beauty (SB) non-viral DNA delivery system offers unique approach to adoptive cellular therapies including CARs & TCRs SB has potential to eclipse limitations associated with viral delivery including point of care, cost of goods, and customization of patie
Slide 6: Sleeping Beauty First-in-man Study
Sleeping Beauty First-in-man Study Long term follow-up data from 1st generation non-viral SB technology tested in two separate trials infusing CAR+ T cells after hematopoietic stem-cell transplantation (HSCT) T-cell survival Average # of days Maximum days Autologous 201* 360 Allogeneic 51 180 Patien
Slide 7: Sleeping Beauty: Next-generation CARs
Sleeping Beauty: Next-generation CARs CAR T NK cells Non-Viral Viral Autologous Allogeneic Established Disease Model Prior Sleeping Beauty CAR Design Modified-1 Sleeping Beauty CAR Design Established Disease Model No T cell Modified CAR + mIL-15 Tumor Burden Tumor Burden Combination of CAR signaling
Slide 8: Ad-RTS-IL-12 + Veledimex Clinical Update
Ad-RTS-IL-12 + Veledimex Clinical Update Early brain tumor data encouraging (N=7) Patients enrolled at multiple centers Biomarkers as expected Neurotoxicity minimal “On-target toxicities” as expected and promptly reversible upon stopping veledimex Early breast cancer data encouraging (N=6) First pat
Slide 9: Ad-RTS-IL-12 + Veledimex Control & Safety
Ad-RTS-IL-12 + Veledimex Control & Safety 52 patients treated to date in 4 different human studies SAEs and Grade 3 related toxicities are rapidly reversible upon discontinuation of veledimex Four cases of Cytokine Release Syndrome (drug related) Reversible with discontinuation of veledimex and supp
Slide 10: T Cell Receptors
T Cell Receptors TCRs NK cells Non-Viral Viral Autologous Allogeneic TCR+ T cells Target intracellular TAAs dependent on HLA “Private” intra-cellular antigens (neo-antigens)
Slide 11: Beyond T cells: Natural Killer Cells
NK cells Target tumors with loss of HLA Killing is independent of a target antigen Proof-of-principle trials ongoing at MDACC infusing autologous and allogeneic NK cells generated using feeder cells SNO 2015: Brain tumor ASH 2015: AML & MM Launching trials infusing off-the-shelf NK cells for AML and
Slide 12: Therapies for GvHD
| Therapies for GvHD ZIOPHARM Oncol INTREXON } - yf “— I Genetic modification Y mmoufcaton necpet IL2 Acute GVHD ee Chronic GVHD |
Slide 13: Peter Emtage, Ph.D., Vice President, Immunology, Intrexon Corporation
Peter Emtage, Ph.D. Vice President, Immunology Intrexon Corporation
Slide 14: Viral Delivery Approach in CAR T
Viral Delivery Approach in CAR T Ziopharm and Intrexon utilizing lenti-viral delivery in “white space” tumors outside of the very competitive landscape of CD19+ hematological malignancies Rapidly advancing clinical candidates for Myeloid malignancies and multiple myeloma (MM) and anticipate clinical
Slide 15: Targeting Myeloid Malignancies with Lenti CAR+ T cells
Target CAR T cells T cells Expansion Transduce T cells Lentivirus Targeting Myeloid Malignancies with Lenti CAR+ T cells CAR T NK cells Non-Viral Viral Autologous Allogeneic Cytotoxicity IFNγ production CAR expression Saline Treated Target CAR Treated In vivo Tumor Cell Model Myeloid malignancies CA
Slide 16: Autologous & Allogeneic Approach in Myeloid Malignancies
Autologous & Allogeneic Approach in Myeloid Malignancies Patient-derived (autologous) Off-the-shelf (allogeneic) CAR T NK cells Non-Viral Viral Autologous Allogeneic Shorten manufacturing time to produce T cells Match one donor with multiple recipients. Generate large numbers of T cells with capacit
Slide 17: Targeting Solid Tumor Malignancies
Targeting Solid Tumor Malignancies Outside of hematological malignant disease we have a number of solid tumor programs that target various tumor types: GBM Neuroblastoma Breast Prostate Colorectal Gastric Head and neck T Cells NK cells Non-Viral Viral Autologous Allogeneic
Slide 18: Addressing Off-target Effects
Addressing Off-target Effects We believe that all CAR and TCR companies will need to address off-target effects in solid tumors. To address these off target effects we are testing and implementing switch controllable gene expression modules as well as target discovery Considerable strides are being
Slide 19: "Off the Shelf" Therapies
“Off the Shelf” Therapies Developing a strategy for “off the shelf” which we believe will be an important advancement for patient and commercial success We believe that modulating both aspects of transplant biology is critical for success in the “off the shelf” space Prevention of Graft-versus-Host-
Slide 20: Exclusive strategic collaboration and license agreement with Merck KGaA
Exclusive strategic collaboration and license agreement to develop and commercialize CAR-T cancer therapies Biopharmaceutical business of Merck KGaA has nominated its 2 novel CAR T targets; they will lead IND filing and pre-IND interactions, clinical development and commercialization Intrexon and ZI
Slide 21: Collaboration with MDACC
Collaboration with MDACC Fully leveraging our clinical collaboration with MDACC to rapidly evaluate various strategies Development is not limited to competitive CARs or TCRs Exploring cutting edge immunology to harness the full capability of gene modified adoptive cell types, such as various immune-
Slide 22: ZIOPHARM's Partnerships
MDACC ZIOPHARM’s Partnerships DNA engine Switches CARs TCRs Cytokines CAR T cells TCR and IL-12 Rapid Clinical Development Intrexon ZIOPHARM Merck KGaA NCI
Slide 23: Pipeline
Preclinical Phase I Phase II Pipeline Ad-RTS-IL-12 NK Cells Primary NK cells Combination with Ad-RTS-IL-12 Genetically-engineered TCR Sleeping Beauty TCR Sleeping Beauty TCR and cytokine Other Regulatory T Cells Modified Bacteria GBM Breast Cancer DIPG GBM + Checkpoint leukemia/lymphoma Undisclosed
Slide 24: Summary
Summary R&D efforts provide us a competitive position in tumors outside of CD19 and an opportunity to take a leadership position as we leverage our immunological and technical expertise We are implementing a series of programming languages (operating systems) to control the immune system in humans R
Slide 25: ZIOPHARM Review and Outlook
ZIOPHARM Review and Outlook December 16, 2015