Precigen, Inc. · NASDAQ:PGEN
Precigen — Intrexon — Corporate Update (January 2019)
53 slides from Precigen's corporate update, Jan 7, 2019
Slide 1: Precigen Company Update
Precigen Company Update Helen Sabzevari, PhD President, Precigen 7 January 2019
Slide 2: Forward-looking statements
Forward-looking statements Precigen, Inc. is a subsidiary of Intrexon Corporation (Nasdaq: XON). 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 Securit
Slide 3: Today's agenda
Today’s agenda o Company overview o Technology platforms o Therapeutic platform: UltraCAR-TTM o Preclinical and clinical portfolio o Therapeutic areas o Immuno-oncology o Infectious diseases o Autoimmune disorders o Summary o Q&A 3
Slide 4: Company overview
Company overview o Company highlights o Precigen’s evolution o State-of-the-art infrastructure
Slide 5: Company highlights – Precigen is a clinical stage company with a growing portfolio
Company highlights – Precigen is a clinical stage company with a growing portfolio of first-in-class therapies Precigen is a biopharmaceutical company advancing the next generation of gene and cellular therapies using precision technology to target immuno-oncology, autoimmune disorders, and infectio
Slide 6: Since our founding, Precigen has been evolving
Since our founding, Precigen has been evolving – setting the stage for rapid advancement of clinical programs Transition from ECC to Ziopharm agreement in-house model Merck KGaA agreement Focus on three core Oncology/Immuno-oncology therapeutic areas Autoimmune Disorders Infectious Diseases Build di
Slide 7: Precigen infrastructure includes state-of-the-art facilities and in-house R&D
Precigen infrastructure includes state-of-the-art facilities, growing manufacturing capabilities and in-house R&D functions Infrastructure State-of-the-art R&D facility in Germantown, MD Building GMP manufacturing facility. Inauguration in 2019 All R&D functions established in-house Research Vivariu
Slide 8: Technology platforms
Technology platforms o Technology platform advantages: CONSTRUCT, DELIVER, and CONTROL o UltraVector ® o Sleeping Beauty system and AttSiteTM recombinases o AdenoVerseTM o RheoSwitch ® o Kill switches and tissue specific promoters
Slide 9: Precigen's technology platforms
Precigen’s technology platforms CONSTRUCT powerful gene UltraVector® mbIL15 programs to drive efficacy DELIVER Sleeping Beauty AttSiteTM gene programs via viral AdenoVerseTM and non-viral based system recombinases approaches to drive lower costs CONTROL Tissue specific gene expression and RheoSwitch
Slide 10: UltraVector enables construction of powerful gene programs
UltraVector® enables construction of powerful gene programs UltraVector® enabled matrices facilitate rapid Significant improvement in identification of components and configurations for gene expression optimal expression of multiple genes Optimized 250 gene expression CONSTRUCT PF P 5 ORF(s) 3 PF 20
Slide 11: Sleeping Beauty system is the most clinically advanced non-viral delivery method
Sleeping Beauty system is the most clinically advanced non-viral delivery method for stable gene expression Non-viral Sleeping Beauty system offers a promising High efficiency of gene transfer alternative to expensive viral vectors for cell therapy CONSTRUCT(%) Expression Transposase Transgene(s) Tr
Slide 12: AttSite recombinases enable non-viral delivery for stable gene expression
AttSite™ recombinases enable non-viral delivery for stable gene expression for variety of cell types AttSite™ recombinases unidirectionally target genes to AttSite™ recombinases facilitate integration at low CONSTRUCT predictable pseudosites in a host cell genome copy number into a finite number of
Slide 13: AdenoVerse platform provides superior performance characteristics
AdenoVerse™ platform provides superior performance characteristics over Ad5 and other rare human and non-human primate adenovirus A library of adenoviral vectors with diverse and unique Durable neutralizing antibody response CONSTRUCT DELIVER Large payload capacity Robust antigen specific T cell res
Slide 14: Control of gene expression in vivo with RheoSwitch
Control of gene expression in vivo with RheoSwitch® CONSTRUCT Switch OFF Switch ON (+activator ligand) RheoSwitch® contolled cytokine expression DELIVER by UltraCAR-T cells • Turn in vivo using • Highly controllable, oral ligand dose dependent gene expression CONTROL • Clinically evaluated with exce
Slide 15: Kill switches and tissue specific promoters improve safety profile
Kill switches and tissue specific promoters improve safety profile Kill switches Tissue specific promoters CONSTRUCT 1 0 0 7 5 y t i c i x o t o 5 0 t y C % 2 5 0 Kill Switch Activator N e g a t i v e C o n t r o l tional local expression of effector genes 15
Slide 16: Therapeutic platform: UltraCAR-T
Therapeutic platform: UltraCAR-T™
Slide 17: UltraCAR-T platform advantages: enhanced potency, safety, and scalability
UltraCAR-T™ platform advantages: enhanced potency, safety, and scalability POTENCY SAFETY SCALABILITY Multigenic expression Kill switch Rapid manufacturing Optimized CAR design Controlled gene Quick turnaround for expression with patients Long-term persistence RheoSwitch® No ex vivo expansion Prefer
Slide 18: UltraCAR-T advantage: scalable manufacturing with rapid turnaround
UltraCAR-T™ advantage: scalable manufacturing process with rapid turnaround time for patients mbIL15 improves potency of UltraCAR-T Memory like phenotype of UltraCAR-T mbIL15 • Improved expansion and persistence of T cells • Less differentiated, memory like T cell population Preferred phenotype • Im
Slide 19: Disrupting the market: transformative UltraCAR-T platform advancements
Disrupting the market: Precigen’s transformative UltraCAR-T™ platform advancements TPrecigen’s -UltraCAR UltraCAR-T Platform enables patient reinfusion within two days following non-viral gene transfer 1 Leukapheresis 2 Electroporation 3 UltraCAR-T Infusion Less than two days Electroporatio Unmodifi
Slide 20: Preclinical and clinical portfolio
Preclinical and clinical portfolio o Portfolio designed for novel combinations o Current preclinical and clinical portfolio by phase
Slide 21: Portfolio is designed to deliver novel combinations
Precigen’s portfolio is designed to specifically deliver novel combinations 21
Slide 22: Pipeline offers rapid value creation and potential for novel combinations
Precigen’s pipeline offers rapid value creation and potential for novel combinations 22 TA Product Platform Indication Discovery Preclinical Phase I PRGN-3006 UltraCAR-T AML, MDS INXN-3004 Viral CAR-T AML PRGN-3005 UltraCAR-T Solid Tumors PRGN-3007 UltraCAR-T Undisclosed PRGN-3008 UltraCAR-T Undiscl
Slide 23: Portfolio designed to deliver novel combinations in immunooncology
Precigen’s portfolio is designed to deliver novel combinations in immunooncology 23 Multifunctional Therapeutic Cytokine Therapeutic UltraCAR-T™ AdenoVerse™ Cancer Vaccine PRGN-3005 PRGN-3006 PRGN-3007 PRGN-3008 PRGN-2009 PRGN-2010 PRGN-5001 PRGN-5002 PRGN-2011 Novel combinations using Precigen’s po
Slide 24: Immuno-oncology therapeutic area
Immuno-oncology therapeutic area o Current immunotherapy landscape o Control of immuno-oncology technologies o Asset highlight: PRGN-3006 UltraCAR-TTM o Asset highlight: PRGN-3005 UltraCAR-TTM o Asset highlight: AdenoVerseTM cancer vaccine o Asset highlight: Multifunctional therapeutic
Slide 25: Current immunotherapy landscape has promise, but many drawbacks
Current immunotherapy landscape has promise, but many drawbacks Despite the success, only a minority of patients respond; in some indications there is no response Relapse rates are high among responders to checkpoint inhibitors Combination trials with checkpoint inhibitors have yielded only incremen
Slide 26: Focus on immuno-oncology with full control of technologies
Focus on immuno-oncology with full control of technologies Full developmental control across targets Royalty bearing license to Ziopharm for CD19 and right to negotiate for second undisclosed target Exclusive rights to non-viral Sleeping Beauty system UltraCAR-T™ platform CAR-T cells Full developmen
Slide 27: Asset highlight: PRGN-3006 UltraCAR-T
Immuno-oncology therapeutic area Asset highlight: PRGN-3006 UltraCAR-TTM
Slide 28: PRGN-3006 received FDA clearance for IND to initiate Phase 1/1b study
PRGN-3006 UltraCAR-T™, a first-in-class therapy in AML, received FDA clearance for IND to initiate Phase 1/1b study 28 Autologous T cell investigational therapy Rapid manufacturing No ex vivo propagation step Infusion within 2 days after gene transfer Next generation Sleeping Beauty design for optim
Slide 29: PRGN-3006 exhibits enhanced functional activity and safety in vitro
PRGN-3006 UltraCAR-T™ exhibits enhanced functional activity and safety in vitro Enhanced cytotoxicity Enhanced antigen specific 6 0 expansion y t i c 4 0 i N e g a t i v e C o n t r o l C A R - T x n o n e g o n e g t C A R - T ( m b I L 1 5 ) i C A R - T ( m b I L 1 5 ) o s t P R G N - 3 0 0 6 n P
Slide 30: PRGN-3006 cells eliminated tumor burden and improved survival in vivo
PRGN-3006 UltraCAR-T™ cells eliminated tumor burden and improved survival in an in vivo model of AML 30 Treatment Median OS (days) Saline 17 Negative Control CAR-T 17 CAR-T (mbIL15neg) 26 PRGN-3006 53 Anti-tumor activity Overall survival (OS) PRGN-3006 UltraCAR-T™ cells administered in less than 2 d
Slide 31: mbIL15 improves expansion and persistence of PRGN-3006 cells
mbIL15 improves expansion and persistence of PRGN-3006 UltraCAR-T™ cells Superior in vivo expansion and persistence of PRGN-3006 UltraCAR-T™ cells in tumor bearing mice Expression of mbIL15 supports survival and promotes extended persistence in absence of cytokines 31 Enhanced survival In vivo – AML
Slide 32: Asset highlight: PRGN-3005 UltraCAR-T
Immuno-oncology therapeutic area Asset highlight: PRGN-3005 UltraCAR-TTM
Slide 33: PRGN-3005 UltraCAR-T, a first-in-class therapy in solid tumors
PRGN-3005 UltraCAR-T™, a first-in-class therapy in solid tumors 33 Highlights and Differentiation Non-viral Sleeping Beauty system to coexpress CAR, mbIL15 and kill switch Autologous T cell product Specific targeting of tumor cells via CAR, improved UltraCAR-T™ persistence due to mbIL15 and built-in
Slide 34: PRGN-3005 specifically lyse tumor cells in dose dependent manner in vitro
PRGN-3005 UltraCAR-T™ specifically lyse tumor cells in dose dependent manner in vitro PRGN-3005 exhibits robust target specific cytotoxicity of tumor cells in dose dependent manner 34 PRGN-3005 Negative Control CAR-T 0 20 40 60 80 100 Targetneg Cells 4:1 2:1 1:1 0.5:1
Slide 35: PRGN-3005 effectively eliminated tumor burden in mice
PRGN-3005 UltraCAR-T™ effectively eliminated tumor burden in mice 35 Enhanced anti-tumor effect Enhanced expansion PRGN-3005 UltraCAR-T™ cells administered in less than 2 days post gene transfer eliminated tumor in all treated mice PRGN-3005 UltraCAR-T™ cells show enhanced expansion and persistence
Slide 36: PRGN-3005 demonstrates enhanced anti-tumor effect
PRGN-3005 UltraCAR-T™ demonstrates enhanced anti-tumor effect PRGN-3005 Negative control CAR-T Tumor cells – Green; Apoptosis = Red 36
Slide 37: Plans to rapidly advance PRGN-3005 UltraCAR-T
Plans to rapidly advance PRGN-3005 UltraCAR-T™ 37 First in class UltraCAR-T molecule for solid tumors Non-viral gene transfer Rapid manufacturing enables quick turnaround for patients PRGN-3005 demonstrated enhanced anti-tumor activity in mouse model Rapidly advancing PRGN-3005 development
Slide 38: Asset highlight: AdenoVerse cancer vaccine
Immuno-oncology therapeutic area Asset highlight: AdenoVerseTM cancer vaccine
Slide 39: AdenoVerse cancer vaccines
AdenoVerse™ cancer vaccines 39 High-level and durable antigenspecific immune responses Large payload capacity and optimized gene expression Ability to repeat administration improves potency Novel antigen design improves immune response
Slide 40: Robust antigen-specific response and anti-tumor activity by AdenoVerse vaccine
Robust antigen-specific response and anti-tumor activity by AdenoVerse™ cancer vaccine candidate in vivo 40 Anti-tumor activity Novel antigen designs for cancer vaccine candidates produce robust antigen-specific response AdenoVerse™ vaccine effectively controls tumor in mice In vitro screening PRGN-
Slide 41: Asset highlight: Multifunctional therapeutic
Immuno-oncology therapeutic area Asset highlight: Multifunctional therapeutic
Slide 42: Multifunctional therapeutic provides better T cell activation vs. anti-PD-1
Precigen’s multifunctional therapeutic candidate provides better T cell activation & anti-tumor response vs. approved anti-PD-1 checkpoint inhibitor 42 Improved T cell activation Improved anti-tumor activity
Slide 43: Candidate induces faster T cell infiltration into tumor spheroid
Precigen’s candidate induces faster T cell infiltration into tumor spheroid compared to anti-PD1 PRGN-5001 Anti-PD1 Key: Tumor = Green PBMCs = Red Fibroblast = Unlabeled 43
Slide 44: Infectious diseases therapeutic area
Infectious diseases therapeutic area
Slide 45: AdenoVerse vaccine candidates generate superior immune responses
Precigen AdenoVerseTM vaccine candidates and technology platform generate superior immune responses 45 Antigen specific immune response Novel antigen designs for infectious disease vaccine candidates produce robust antigenspecific response In vivo screening AdenoVerse™ vaccine produces superior immu
Slide 46: Autoimmune disorders therapeutic area
Autoimmune disorders therapeutic area
Slide 47: Factors involved in the initiation of autoimmunity
Factors involved in the initiation of autoimmunity 47 HLA Genetic Cytokines/receptors Infection, microbiome, tissue injury Generate proinflammatory environment Activation of autoreactive lymphocytes Defective Tregs (either development, stability, or function) Environmental Graphic adapted from: J Cl
Slide 48: Autoimmunity asset demonstrated potent suppression of effector cell function
Precigen’s autoimmunity asset demonstrated potent suppression of effector cell function Suppression of effector T cell proliferation Suppression of inflammatory cytokine production + + A u t o l o g o u s C D 4 T C e l l s A u t o l o g o u s C D 8 T C e l l s 1 0 0 1 0 0 1 0 0 0 0 n n 8 0 8 0 o o i
Slide 49: Summary
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Slide 50: Looking ahead to 2019
Looking ahead to 2019 50 Initiate PRGN-3006 UltraCAR-TTM Phase 1 trial in AML and MDS Rapidly advance PRGN-3005 UltraCAR-TTM for solid tumor Rapidly advance PRGN-2009 AdenoVerseTM vaccine for solid tumor Rapidly advance one infectious disease candidate Rapidly advance preclinical candidates to go/no
Slide 51: Long-term vision: Value creation through patient focus
Long-term vision: Value creation through patient focus Foundation Rights to proprietary platforms and technologies Therapeutic area focus Enhanced scientific team and capabilities Execution Aggressively advance programs though clinical development Early go/no go decisions to de-risk portfolio Value
Slide 52: Q&A
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Slide 53: Advancing medicine with precision
ADVANCING MEDICINE WITH PRECISION TM