Precigen, Inc. · NASDAQ:PGEN
Precigen — Intrexon — JP Morgan Healthcare Conference 2015
31 slides from Precigen's conference presentation, Jan 14, 2015
Slide 1: Enabling Next-Generation Gene and Cell Therapies
Enabling Next-Generation Gene and Cell Therapies JP Morgan Healthcare Conference Investor Presentation January 2015 Exhibit 99.1
Slide 2: Forward-Looking Statements
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 statem
Slide 3: New Standard in Non-Viral Adoptive Cellular Therapies
New Standard in Non-Viral Adoptive Cellular Therapies Laurence J.N. Cooper, M.D., Ph.D., Professor of Pediatrics at MD Anderson Gregory Frost, Ph.D., Senior Vice President, Head of Intrexon’s Health Sector Jonathan Lewis, M.D., Ph.D., Chief Executive Officer of ZIOPHARM Oncology Development of Non-V
Slide 4: What would it take for a disruptive new platform to eclipse $20B/year in sales
What would it take for a disruptive new platform to eclipse >$20B/year in sales of anti-cancer monoclonal antibodies? immunologically tolerated scalable manufacturing target specificity efficacy ADC’s ADCC bi-specific modular framework 2013 Worldwide mAb anti-cancer sales: FiercePharma
Slide 5: Unprecedented Efficacy
Unprecedented Efficacy Pre-Treatment Diagnosis CLL Estimated Tumor Burden >2 kg Post-Treatment No evidence of CLL (by histology, FACS, cytogenetics FISH and deep sequencing) NEJM. (2011) 365(8): 725–733
Slide 6: That Is Durable
That Is Durable No Evidence of Disease > 24mos NEJM. (2014) 371: 1507-1517
Slide 7: Adoptive Cell Therapy Has Shown Impressive Early Signs of Efficacy
Adoptive Cell Therapy Has Shown Impressive Early Signs of Efficacy NEJM. (2014) 371: 1507-1517
Slide 8: Yet ON Target Toxicity Is A MAJOR Issue For MANY Targets
Yet ON Target Toxicity Is A MAJOR Issue For MANY Targets J Clin Oncol. (2006) 24:e20–2
Slide 9: If Adoptive Cellular Therapy Cannot Be Controlled
If Adoptive Cellular Therapy Cannot Be Controlled… …its application will be severely limited Reassessing target antigens for adoptive T-cell therapy Nature Biotechnology. (2013) 31:999-1008
Slide 10: And If CARs Cannot Be Manufactured In A Scalable Fashion
And If CARs Cannot Be Manufactured In A Scalable Fashion… …they will remain as local ‘therapies’ instead of global solutions Blood. (2011) 118(18):4761-4762
Slide 11: Intrexon's Vision of Synthetic Immunology
Intrexon’s Vision of Synthetic Immunology Nature 487: 64-69. Molecular rewiring of immunologic circuitry in cellular products through synthetic multigenic programs to generate safe and highly-effective oncolytic effectors
Slide 12: Intrexon Is Building The Solutions For CAR-T Cell Therapies
Intrexon Is Building The Solutions For CAR-T Cell Therapies Intrexon has the integrated technology platforms to actualize the potential of CAR-T Cell therapies. Utilization of our RheoSwitch ® facilitates exquisite regulation of multiple bioeffectors in CAR-T Cells enabling physicians to control sys
Slide 13: Intrexon – ZIOPHARM - MDACC Collaboration
Intrexon – ZIOPHARM - MDACC Collaboration
Slide 14: Dual Approach to Identify Most Effective CAR-T Products
Dual Approach to Identify Most Effective CAR-T Products Registration Trials & Global Distribution with Multiple Pharma Partners • Candidate Generation • Product Refinement • Clinical POC • Single Center Translational Trials of Novel Prototypes • Point of Care Controlled pDNA CAR-T/TCR Design Univers
Slide 15: Key Technology Synergies
Technology Intrexon / ZIOPHARM / MD Anderson Key Technology Synergies RheoSwitch Therapeutic System® Most advanced family of ligands and switches available for dynamic range, safety, and spatial/temporal control Non-Viral Integration Platform First in-human testing of Sleeping Beauty system in hemat
Slide 16: Controlling Gene Expression with RheoSwitch (RTS) Platform
Controlling Gene Expression with RheoSwitch® (RTS®) Platform • RTS® platform provides transcriptional control of a broad, diverse range of therapeutic genes enabling application across many indications • RTS® modulates protein expression and allows for repeated inducibility • Clinically validated wi
Slide 17: RTS Conditional Delivery Of Distinct Immune Effectors
RTS® Conditional Delivery Of Distinct Immune Effectors IFN IL-12 p70 CTLA- 4 DCY IFN IL-12 CTLA-4 DCY RTS ® has shown conditional delivery of immune effectors in a single vector pg/mL Uninduced Induced Uninduced Induced Uninduced Induced
Slide 18: RTS Spatial and Temporal Expression
RTS® Spatial and Temporal Expression Tissue Specific Cells Ubiquitous Promoters Tissue Specific Promoters Non-Tissue Specific Cells Ubiquitous Promoters Tissue Specific Promoters No Expression RTS ® system can be tailored for cell type and state specific spatial regulation
Slide 19: Features of the AttSite Recombinase System
Features of the AttSite® Recombinase System AttSite® Recombinase Technology Precision Control Safety • Library of AttSite® characterized serine recombinases • Unidirectional stable integration technology (unlike cre/lox) • Targeted gene delivery to pre- positioned AttSites in the genome • Allows lon
Slide 20: Non-Viral Integration Platform for Large Payloads
Non-Viral Integration Platform for Large Payloads Pseudo AttSite® Recombinase Mediated Stable Integration AttSite® recombinases facilitate integration of intact transgenes at low copy number into a finite number of pseudo AttSites® in the genome. One hundred percent transgene integrity and 1-6 integ
Slide 21: Intrexon's Multi-Horizon Strategy of Synthetic Immunology
Intrexon’s Multi-Horizon Strategy of Synthetic Immunology Build Test Learn Design Build Test Learn Design Build Test Learn Design Autologous Allograft Immortalized Universal Donor CAR/TCR-T CAR/TCR-T GM-DC GM-DC UltraCART UltraCART GM-ERC/NK GM-ERC/NK
Slide 22: Intrexon Empowering Broad Gene and Cell Therapy Applications
Intrexon Empowering Broad Gene and Cell Therapy Applications Applications Utilizing Intrexon Technology Rare Disease: Friedreich’s Ataxia Oncology: Autologous Approach Oncology: Allogeneic Platforms Ocular Disease: Age-related Macular Degeneration Cartilage Repair: Allogeneic Chondrocyte Cells Rare
Slide 23: Gene-based Therapies for Ocular Diseases Utilizing RTS
Gene-based Therapies for Ocular Diseases Utilizing RTS® Activator Ligand Delivery via Pill or Eye Drops Gene of Interest Activator Ligand Protein of Interest RTS ® Platform For Controlled Ocular Expression • Current approaches to chronic ocular diseases require intraocular delivery with repeated inj
Slide 24: Cell-based Treatments for Rare Skin Diseases
Cell-based Treatments for Rare Skin Diseases • Collaboration with Fibrocell Science to develop genetically-modified human dermal fibroblasts expressing collagen VII for treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) • RDEB has no approved treatment and is often lethal before the age
Slide 25: Intrexon in 2015
25 © 2015 Intrexon Corp. All rights reserved. 25 Intrexon in 2015
Slide 26: Continued Expansion Into New Fields
Continued Expansion Into New Fields Intrexon Fields of Collaboration Energy Environment Consumer Food Health
Slide 27: Integrating through Collaboration into Industrial Leadership
Cell Engineering Molecular Engineering Organ & Organism Engineering Integrating through Collaboration into Industrial Leadership Energy Consumer Health Environment Food Gene & Cell Therapies Animal Cloning Bovine Embryos Research Animal Models GM Food Animals Synthetic Biology
Slide 28: Increasing Capital Efficiency to Unburden Product Economics
Increasing Capital Efficiency to Unburden Product Economics $0 $5 $10 $15 $20 $25 Q1 2013 Q2 2013 Q3 2013 Q4 2013 Q1 2014 Q2 2014 Q3 2014 Revenues (in millions) 0% 20% 40% 60% 80% FY 2013 Q3 2013 Q4 2013 Q1 2014 Q2 2014 Q3 2014 Cost Recovery as % of Cash OpEx FY2013 & Past 4 Quarters Lead the Second
Slide 29: Thank You!
THANK YOU!
Slide 30: Appendix A
Appendix A Intrexon Corporation and Subsidiaries Reconciliation of GAAP to Non-GAAP Measures (Unaudited) Adjusted EBITDA . reconciliation of Adjusted EBITDA to Intrexon's net income or loss attributable to Intrexon under GAAP appears below. Adjusted EBITDA is a non-GAAP financial measure that Intrex
Slide 31: Appendix A continued
Appendix A continued (1) Pro forma adjusted EBITDA per share, basic and diluted have been calculated for the three and nine month periods ended September 30, 2013 after giving effect to (i) the conversion of 112,906,464 shares of Intrexon's preferred stock outstanding on January 1, 2013 into 64,517,