Full Press Release Details
INVESTOR DAY | NOVEMBER 12, 2015
Forward Looking Statements Special Note
Regarding Forward-Looking Statements Some of the matters discussed in this presentation may constitute forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform
Act of 1995. Forward-looking statements relate to expectations, beliefs, projections, future plans and strategies, anticipated events or trends and similar expressions concerning matters that are not historical facts. The forward-looking statements
are based on Intrexon's beliefs, assumptions and expectations of its future performance, taking into account all information currently available to it. These beliefs, assumptions and expectations can change as a result of many possible events
or factors, not all of which are known to Intrexon or are within its control. The following factors, among others, could cause actual results to vary from the forward-looking statements: These risks and uncertainties include, but are not limited to,
(i) our ability to enter into new exclusive channel collaborations (ECCs), joint ventures, license agreements and other collaborations and agreements; (ii) developments concerning our collaborators and licensees; (iii) our ability to successfully
enter new markets or develop additional products, whether with our collaborators or independently; (iv) competition from existing technologies and products or new technologies and products that may emerge; (v) actual or anticipated variations in our
operating results; (vi) actual or anticipated fluctuations in our competitors' or our collaborators' and licensees' operating results or changes in their respective growth rates; (vii) our cash position; (viii) market conditions in our industry;
(ix) our ability, and the ability of our collaborators and licensees, to protect our intellectual property and other proprietary rights and technologies; (x) our ability, and the ability of our collaborators and licensees, to adapt to changes in
laws or regulations and policies; (xi) the ability of our collaborators and licensees to secure any necessary regulatory approvals to commercialize any products developed under the ECCs and license agreements; (xii) the rate and degree of market
acceptance of any products developed by a collaborator under an ECC or through a joint venture or license under a license agreement; (xiii) our ability to retain and recruit key personnel; (xiv) our expectations related to the use of proceeds from
Forward Looking Statements (xv) our
estimates regarding expenses, future revenue, capital requirements and needs for additional financing; and (xvi) our expectations relating to AquaBounty, Trans Ova Genetics and Oxitec Limited. Intrexon does not undertake any obligation to update any
forward-looking statements to reflect circumstances or events that occur after the date on which such statements were made except as required by law. Additional information about factors affecting Intrexon is available in Intrexon's Annual
Report on Form 10-K for the year ended December 31, 2014, and Quarterly Report on Form 10-Q for the period ended September 30, 2015, and other filings with the SEC, which are available at www.sec.gov. Use of Non-GAAP Financial
Measures This presentation presents Adjusted EBITDA and Basic Adjusted EBITDA per share, which are non-GAAP financial measures within the meaning of applicable rules and regulations of the Securities and Exchange Commission (SEC). For a
reconciliation of Adjusted EBITDA to net loss attributable to Intrexon in accordance with generally accepted accounting principles and for a discussion of the reasons why the company believes that these non-GAAP financial measures provide
information that is useful to investors please refer to Intrexon's earnings release dated November 9, 2015. Such information is provided as additional information, not as an alternative to our consolidated financial statements presented in
Company Introduction and Overview
Randal J. Kirk Chairman and Chief Executive Officer
Five Key Takeaways for Today Expansive
Engineering Biology Intrexon:
Differentiated Synthetic Biology
Platform Thomas D. Reed, Ph.D. Founder and Chief Science Officer
Programming the Most Pervasive Code on
Biological Framework At Center of
A Platform To Program Code of
increasing number of host systems 1998 - 2009 Human / Mouse / CHO 2010 - 2015 Yeast / Fungi Plant Piscine Methanotroph Bovine / Porcine Equine / Canine L. lactis Fruit Insecta
BUILD LEARN Each stage of model
driven DBTL cycle integrates with bioinformatics platform TEST Exponential growth of cellular systems knowledge with each DBTL cycle Knowledge gained for different applications + different host systems as DNA code is universal Increasingly Valuable
Intrexon's proprietary
database maintains performance data on DNA parts, providing better designs and faster build times Data mining, modeling, and statistical analysis of the component library enable optimization and predictive biological experimentation Predictive
Bioengineering Across Multiple Hosts
Opens Large Markets Intrexon's Expression Host Expertise Health Gene and cellular therapies, API, biologics Food Agriculture, Aquaculture, Livestock Energy Fuels, Chemicals, Lubricants Environment Bioremediation, Sustainable Forestry Consumer
Enabling Collaborators with Custom
Biosolutions Intrexon Collaborator Domain Expertise End Market Strategy for Commercialization Biological Systems and Technology Leadership Alignment to design and commercialize better, faster, cheaper biosolutions 2015 Intrexon Corp. All
Accelerating Concept to Candidate
with Informatics Mike Kubal Senior Manager, Informatics
Power of Intrexon Informatics
- Accelerating Concept to Candidate KNOWLEDGE FRAMEWORK Knowledge Management System Learn Algorithms & Models External Data Robust Internal Systems Fueling the Product Candidate Invention Engine Flexible and Scalable Informatics Ecosystem
Predictive ability removes time,
cost and risk inherent with standard experimentation 1260 Vectors: Best performer will be identified but at 4x cost + 2x time relative to 307 Vectors 500 Vectors: High probability of identifying hits, at mid-range time/cost 307 Vectors: Calculated
BeyondBio : Our Centralized
Database Platform BeyondBio : A suite of tools for each stage of the Design-Build-Test-Learn workflow PathX Proprietary Learn Tool: Pathway Finding Pathway Generation Strain Design Target Pathway Non-target Pathway Non-target Pathway
Connecting Learn and Design with
Connecting Design with Commercial
Targets for Partners PathX allows Intrexon to quickly screen and prioritize vast numbers of potential targets for many industries from both a scientific and business perspective. Today PathX has identified potential biological routes for many target
Beyond the Borders of Health with
Better DNA Samuel Broder, M.D. Senior Vice President, Health Sector
A Single Molecule at the Center of
Better DNA at the Center of
Powering the Bioindustrial
Revolution Hosts Better DNA * Markets Human Plant Animal Bacteria Fungi Agriculture APIs Aquaculture Biologics CAR-T Cell Therapy Chemicals Consumer Products Fuels Gene Therapy Insect-Vector Control Livestock Genetic Programs Gene
Delivery Systems Gene Expression Control and Optimization (including RheoSwitch ) Self-Limiting Gene Paradigms for Insect-Vectors Multiplex Gene Systems Modular Parts AttSite Recombinase Bioinformatics *Partial List Intrexon's
Creating a Sizeable Market
Proprietary Approach for Gene
Expression and Regulation Peter Emtage, Ph.D. Vice President, Immunology
Evolution of Intrexon's
Platform Up to 20 gene programs in a network; each containing up to 19 variable components Enables the architecture and construction of complex, integrated bio-functional systems Highly scalable; built on rapid design-build-test framework with
intelligent software backbone Single gene program containing up to 5 variable components Early design of intelligent software backbone Our understanding of gene expression and regulation is commensurate with the evolution of Intrexon's
Our Extensive Expertise in DNA
Delivery Electroporation Chemical Transduction Direct Injection Autologous Allogeneic Adeno- virus (Ad) Adeno-associated Virus (AAV) Retrovirus / Lentivirus AttSite Recombinases Sleeping Beauty attR attL 5' Cap Poly AAA mRNA Stem Cells
Viral Delivery Utilize many viruses and also generate a variety of serotype vectors to provide optimal delivery Non-Viral DNA/mRNA Delivery Provides for low immunogenicity and also ability to accommodate large DNA constructs Cellular Delivery
Pseudo AttSite recombinase
mediated stable integration Intact transgene integration at low copy number into pseudo AttSites in the genome 100% transgene integrity and 1-6 integrations per cell achieved Our Inducible Systems & Genome Engineering + recombinase
Irreversible integration Chromosome attB attR attL Chromosome attP Plasmid RheoSwitch controlled expression and delivery of therapeutic proteins Oral small molecule ligand- activated on/off/on gene switch Dose-dependent response for adjustable
RheoSwitch Platform Allow for
RheoSwitch Regulated
Expression of Bioactive RNA Dose-dependent ligand-induced expression results in accumulation of mature, processed miRNA Inducibly-expressed miRNAs are functional, demonstrating knock-down of target genes New Solutions on the Horizon Time Course with
Constant Enzyme Destabilized #1 Destabilized #2 WT Modulating Stability of Therapeutic Proteins Engineering of immunomodulator protein structure to improve clearance in the body Upon administration of destabilized enzyme, IFN levels decrease,
Control & Delivery Empower
Optimized Solutions Improved Safety, Efficacy, and Manufacturing Autologous & Allogenic Solutions Cytokines scFv constructs Chimeric antigen receptors TCRs or Chimeric TCRs Chimeric co-stimulatory receptors Chemokines miRNA Gene editing
techniques RheoSwitch regulation Transcription control Attsite Recombinases Multigenic approaches Viral and non-viral delivery Protein translation control Dendritic Cells T Cell NK cell Treg Other immune cells Cell Platforms Biological
Intrexon's Synthetic
Immunology Efforts Expanding Team of Scientists Viral & Non-viral gene delivery / Biological Effectors / Controlled Expression Knowledge Framework Driving Partner Solutions Through our Innovative DNA Engine 2015 Intrexon Corp. All rights
Leveraging Intrexon's Viral
DNA Delivery Tools for CAR-T Target CAR T cells T cells Expansion Transduce T cells Lentivirus Cytotoxicity IFN production CAR expression Saline Treated Target CAR Treated In vivo Tumor Cell Model 2015 Intrexon Corp. All rights
Our broad platform engineers
Harnessing the power of the immune
system to fight cancer Laurence Cooper M.D., Ph.D. Chief Executive Officer, ZIOPHARM Oncology, Inc.
Synthetic Biology Leads to Powerful
Engineering of Immune System Immune cells are capable of preventing and eliminating disease Yet the body's own programming language governing an immune response is unable to eliminate tumor cells Genes can be engineered ex vivo to achieve in
Intrexon & ZIOPHARM: Delivering
Viral Gene Therapy: Ad-RTS-IL12
Ongoing Trials A single cycle of Ad-RTS-hIL-12 & escalating veledimex To establish: - Efficiency of veledimex crossing human BBB - Safety and tolerability - Determine MTD and immune response - Anti-tumor activity A single cycle of Ad-RTS-hIL-12
+ veledimex Goal of maintaining or improving pre-study response To establish: - Safety and tolerability - Anti-tumor activity Multi-Center Phase I Study of IL-12 Gene Therapy in Recurrent or Progressive Glioblastoma / Grade III Malignant Glioma
Portfolio of Cell Therapies to
Address Oncology Landscape 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
Building on the Progress of MDACC
Trials for CAR+ T Therapy Infusion of autologous T cells in patients with advanced non-Hodgkin lymphoma (NHL) Infusion of allogeneic T cells in patients with advanced CD19+ acute lymphoblastic leukemia or NHL CD19-directed CAR+ T therapies utilizing
Sleeping Beauty (SB) non-viral gene integration after hematopoietic stem-cell transplantation (HSCT) HSCT CAR+ T cells HSCTCAR+ T cells 86% of patients (n=6) remain in complete remission (CR) 58% of patients (n=11/19) remain in CR 75% of patients
Portfolio of Next-generation Cell
Therapies CAR designs Cytokines Off-the-shelf Personalizing by targeting neo-antigens CAR+ T cells TCR+ T cells NK cells Cytokines Off-the-shelf Bioprocessing Manage the cost of immunotherapy and targeting solid tumors 2015 Intrexon Corp. All
Engineering Off-the-Shelf CAR+
Strong Collaborations to Bring
Differentiated Platforms to Fight
Harnessing the future 2015 2016+
Viral Gene Therapy Ad-RTS-IL-12 Glioma and Breast Expand Indications Combinations 2015 2016+ Adoptive Cell Therapy Hematological (ALL/NHL/CLL) Hematological (AML/MDS) CAR Design CAR & Cytokine Solid Tumors NK cells TCR Off-the-shelf 2015
Therapeutic Approaches Beyond Cancer