Precigen, Inc. · NASDAQ:PGEN
Precigen — Intrexon — 34th Annual J.P. Morgan Healthcare Conference (January 2016)
31 slides from Precigen's conference presentation, Jan 13, 2016
Slide 1: Enabling Next-Generation Gene and Cell Therapies with Better DNA
Enabling Next-Generation Gene and Cell Therapies with Better DNA® 34th Annual J.P. Morgan Healthcare Conference Samuel Broder, M.D. Senior Vice President, Health Sector Exhibit 99.1
Slide 2: Forward-Looking Statements
Forward-Looking Statements Safe Harbor Statement Some of the statements made in this presentation are forward-looking statements that involve a number of risks and uncertainties and are made pursuant to the Safe harbor Provisions of the Private Securities Litigation Reform Act of 1995. These forward
Slide 3: A Single Molecule at the Center of Biology
A Single Molecule at the Center of Biology
Slide 4: Powering the Bioindustrial Revolution
Powering the Bioindustrial Revolution™ Markets Human Plant Animal Bacteria Fungi HEALTH Gene and cellular therapies, API, biologics ENERGY Fuels, chemicals ENVIRONMENT Insect control, bioremediation FOOD Livestock, agriculture, aquaculture CONSUMER Personal care products UltraVector® Platform Gene E
Slide 5: Extensive Expertise in DNA Delivery
Extensive Expertise in DNA Delivery Electroporation Chemical Transduction Direct Injection Autologous Allogeneic Adeno- virus (Ad) Adeno-associated Virus (AAV) Retrovirus / Lentivirus AttSite™ Recombinases attR attL 5’ Cap Poly AAA mRNA Stem Cells Viral Delivery Utilize many viruses and also generat
Slide 6: Intrexon's Better DNA Engine
Intrexon’s Better DNA® Engine Cellular Biofactories* Fibroblasts T Cells * Not complete list. Active health sector programs utilize cartilage cells, iPSCs, cardiomyocytes, EggPC™ cells, among others. Retinal L. lactis After Intrexon Bioengineering T cells engineered with CARs or TCRs to better recog
Slide 7: Business Model Built to Capture Large Market Opportunity
Business Model Built to Capture Large Market Opportunity Business model initiated in 2011 enabling broad application of our technology across many diverse end markets includes Exclusive Channel Collaborations (ECCs) and Research Collaborations Through our ECC model, Intrexon’s cumulative TAF and mil
Slide 8: Broad Engagement in Health Sector
* Post split of all economics of deal with biopharmaceutical business of Merck KGaA with ZIOPHARM Oncology including upfront and potential milestones. Including those economics would result in $245.5 million in TAF/milestones to date and >$1.1 Billion in potential milestones for the health sector. $
Slide 9: Our Health Collaborations Across Various Conditions
Our Health Collaborations Across Various Conditions Oncology: CAR-T, TCR, Gene/Cell Therapy Infectious Diseases: C. difficile, Pertussis Ocular Disease: Wet AMD Rare Skin Disorders: RDEB, Linear Scleroderma Rare Diseases: Friedreich’s Ataxia Cardiac Diseases: Heart Failure Cartilage Repair: Off-The-
Slide 10: Synthetic Immunology: Applications to Cancer
Synthetic Immunology: Applications to Cancer Merck KGaA ZIOPHARM Oncology Inc. MD Anderson Cancer Center National Cancer Institute If Adoptive Cellular Therapy Cannot Be Controlled…its application will be severely limited Without Enduring Efficacy...this promising approach utilizing the immune syste
Slide 11: CAR-T: Intrexon / Merck KGaA
CAR-T: Intrexon / Merck KGaA, Darmstadt, Germany *Note that timelines are event-driven and may change Potential differentiation and timelines: Potential to address limitations of 1st / 2nd generation gene and cell-based therapy May improve safety profile of CAR-Ts through platform technologies inclu
Slide 12: Intrexon & ZIOPHARM: Delivering Multimodal Gene Therapies
Intrexon & ZIOPHARM: Delivering Multimodal Gene Therapies Controlled Gene Therapy Adoptive Cell Therapies Viral & Non-Viral Gene Integration Autologous and Allogeneic Approach Sleeping Beauty Lentivirus & other viral delivery T Cells NK Cells (CAR T & TCR)
Slide 13: The Importance of Sleeping Beauty
The Importance of Sleeping Beauty “The Sleeping Beauty transposon-transposase system represents a unique non-viral system for introducing genes encoding T-cell receptors and chimeric antigen receptors into lymphocytes that can be of great value in the development of personalized immunotherapies for
Slide 14: CAR T cells for tumors with unmet needs
In vivo model for myeloid malignancies CAR-T target Lentiviral CAR+ T cells Platform Target CAR T cells for tumors with unmet needs T cells Lentivirus Intrexon and ZIOPHARM are utilizing lentiviral delivery for tumors with unmet needs outside of the crowded viral CD19+ CAR T treatment landscape. We
Slide 15: Collaboration with MD Anderson Cancer Center
Collaboration with MD Anderson Cancer Center 2015 Development: Fully leveraging our clinical collaboration with MDACC to rapidly evaluate various strategies not limited to competitive CARs or TCRs Trial initiated in Q4’2015 with next-gen CD19 CAR design utilizing Sleeping Beauty 2016 and Beyond: Exp
Slide 16: Next-gen RTS Controlled Gene Therapy Approach to Wet AMD
Next-gen RTS® Controlled Gene Therapy Approach to Wet AMD Intrexon and Sun Pharmaceuticals formed joint venture to develop gene therapies for treatment of blinding ocular diseases 1st target is Wet Age-related Macular Degeneration (AMD); market est’d at $6B Adeno-associated viral (AAV) delivery tran
Slide 17: On-Off-On Expression with RheoSwitch Platform
+AL -AL +AL Eyes harvested Day 0, 14, 24, 34 On-Off-On Expression with RheoSwitch® Platform AAV-RTS-EPO AAV2-RTS-IFNα In vivo pre-clinical data demonstrated ‘On-Off-On’ effect via RTS® platform and tight control with multiple proteins
Slide 18: RTS Regulates Lead Therapeutic Candidate
RTS® Regulates Lead Therapeutic Candidate Efficacy observed only with lead therapeutic candidate in presence of RTS® activator ligand veledimex AAV-RTS-XON AAV-RTS-XON AAV-CMV-FLuc No Treatment
Slide 19: Re-Engineering Fibroblasts into Biotherapeutics
Re-Engineering Fibroblasts into Biotherapeutics Intrexon’s Gene Therapy Product Engine for FCX-007, FCX-013… Human Autologous Fibroblast Product (azficel-T BLA) Collection Culture Local Administration Vector Preparation Gene Packaging Gene Transduction FCX-007 – Orphan drug candidate for Recessive D
Slide 20: ActoBiotics Platform: New Frontier in Cellular Therapeutics
ActoBiotics® Classic Biologics Oral administration Intravenous administration No toxicity or immunogenicity Systemic toxicity, immunogenicity Targeted delivery; optimal tissue availability Limited tissue penetration, notably in the gut Multiple proteins Single protein Low cost of goods Relatively hi
Slide 21: Unique Opportunities in Diabetes with Oral ActoBiotics
Oral ActoBiotics® Stomach Gastric emptying Pancreas Insulin secretion Glucagon secretion Liver Brain Appetite Glucose production Insulin sensitivity Muscle Adipose tissue Glucose uptake and storage Glucose uptake, Storage & utilization Adapted from Gastroenterology 2007 132, 2131-2157 Intestinal Del
Slide 22: Advancement of Porcine to Human Organ Xenotransplantation
Advancement of Porcine to Human Organ Xenotransplantation Organ Waiting List Transplant Kidney 108,238 14,879 Liver 15,275 5,651 Pancreas 1,040 187 Heart 4,198 2,331 Lung 1,552 1,694 Heart / Lung 49 12 Intrexon’s integrated technologies and proprietary platforms, including our genetically diverse Lo
Slide 23: Attacking Global Health Epidemics at their Source
Attacking Global Health Epidemics at their Source * http://www.idcostcalc.org/contents/dengue/index.html Mandacaru Itaberaba Panama 96% 92% 99% 94% 93% Pedra Branca Cayman Islands Aedes aegypti Oxitec’s lead health program centers on the primary vector for dengue, chikungunya, yellow fever and Zika
Slide 24: New Zika Virus Epidemic
New Zika Virus Epidemic Brazilian cases of microcephaly (a birth defect that may be linked to Zika virus infection) in thousands December 1st, 2015 – World Health Organization (WHO) and Pan American Health Organization (PAHO) issued epidemiological alert given “increase of congenital anomalies, Guil
Slide 25: Brazil: A Case Study
Brazil: A Case Study Early 1970s No Aedes aegypti No dengue Recent years 1.5 million cases of dengue in 2015, up 176% y-o-y with 811 dengue-related deaths First local transmission of chikungunya in 2014 and Zika virus entered in 2015 Over $1 billion spent on the dengue vector control program annuall
Slide 26: Health Sector: 2016 Outlook & Milestones
Health Sector: 2016 Outlook & XON/Collaborator Milestones Anticipate up to three INDs filed in oncology including lentiviral CAR T myeloid malignancies treatment, primary NK cells for AML, and gene therapy combining checkpoint inhibitor with Ad-RTS-IL-12 for GBM Anticipate IND filing for next-gen RT
Slide 27: Appendix
APPENDIX 22 Owibinreancop. Arsene INTREXON’
Slide 28: Appendix: Diversified XON/Collaborator Health Pipeline
Appendix: Diversified XON/Collaborator Health Pipeline Field / Collaborators Indications Pre-Clinical Phase I Phase II Oncology (ZIOPHARM, Merck Serono, National Cancer Institute) Ad-RTS-IL-12: Breast cancer Malignant Glioma CAR/Cytokine Products: B-Cell Malignancies Myeloid Malignancies RTS-Control
Slide 29: Appendix: Third Quarter and Nine Month Results
Appendix: Third Quarter and Nine Month Results Fiscal Year 2015 Financials Third Quarter YTD Revenues $53.4 M $132.1 M Net loss attributable to Intrexon $38.2 M $51.8 M Basic EPS $(0.34) $(0.47) Adjusted EBITDA $3.8 M* $43.6 M* Basic adjusted EBITDA per share $0.03* $0.40* Total Cash and Investments
Slide 30: Appendix: Reconciliation of GAAP to Non-GAAP Measures
Appendix Intrexon Corporation and Subsidiaries Reconciliation of GAAP to Non-GAAP Measures (Unaudited) Adjusted EBITDA and Adjusted EBITDA per share. To supplement Intrexon’s financial information presented in accordance with U.S. generally accepted accounting principles (“GAAP”), Intrexon presents
Slide 31: Appendix (continued)
Appendix (continued) The following table presents a reconciliation of net income attributable to Intrexon to EBITDA and also to Adjusted EBITDA, as well as the calculation of Adjusted EBITDA per share, for each of the periods indicated: Three months endedNine months endedSeptember 30,September 30,20
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