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August9,2017
SecondQuarter2017FinancialResultsandBusinessUpdate
©2017IntrexonCorp.Allrightsreserved.2
SafeHarborStatementSomeofthestatementsmadeinthispresentaFonareforward-lookingstatementsthatinvolveanumberofrisksanduncertainFesandaremadepursuanttotheSafeharborProvisionsofthePrivateSecuriFesLiFgaFonReformActof1995.Theseforward-lookingstatementsarebaseduponIntrexon’scurrentexpectaFonsandprojecFonsaboutfutureeventsandgenerallyrelatetoIntrexon’splans,objecFvesandexpectaFonsforthedevelopmentofIntrexon’sbusiness. AlthoughmanagementbelievesthattheplansandobjecFvesreflectedinorsuggestedbytheseforward-lookingstatementsarereasonable,allforward-lookingstatementsinvolverisksanduncertainFesandactualfutureresultsmaybemateriallydifferentfromtheplans,objecFvesandexpectaFonsexpressedinthispresentaFon.TheserisksanduncertainFesinclude,butarenotlimitedto,(i)Intrexon’scurrentandfutureECCsandjointventures;(ii)Intrexon’sabilitytosuccessfullyenternewmarketsordevelopaddiFonalproducts,whetherwithitscollaboratorsorindependently;(iii)actualoranFcipatedvariaFonsinIntrexon’soperaFngresults;(iv)actualoranFcipatedfluctuaFonsinIntrexon’scompeFtors’oritscollaborators’operaFngresultsorchangesintheirrespecFvegrowthrates;(v)Intrexon’scashposiFon;(vi)marketcondiFonsinIntrexon’sindustry;(vii)thevolaFlityofIntrexon’sstockprice;(viii)Intrexon’sability,andtheabilityofitscollaborators,toprotect Intrexon’s intellectual property and other proprietary rights and technologies; (ix) Intrexon’s ability, and the ability of its collaborators, to adapt tochanges in lawsor regulaFonsandpolicies; (x) theoutcomesofpendingand future liFgaFon; (xi) therateanddegreeofmarketacceptanceofanyproductsdevelopedbya collaboratorunderanECCor througha jointventure; (xii) Intrexon’sability to retainand recruit keypersonnel; (xiii) Intrexon’sexpectaFonsrelated to the use of proceeds from its public offerings and other financing efforts; (xiv) Intrexon’s esFmates regarding expenses, future revenue, capitalrequirementsandneedsforaddiFonalfinancing;and(xv)Intrexon’sexpectaFonsrelaFngtoitssubsidiariesandotheraffiliates.Foradiscussionofotherrisksand uncertainFes, and other important factors, any of which could cause Intrexon’s actual results to differ from those contained in the forward-lookingstatements,seethesecFonenFtled”RiskFactors“inIntrexon’sAnnualReportonForm10-K,aswellasdiscussionsofpotenFalrisks,uncertainFes,andotherimportant factors in Intrexon’s subsequent filingswith the SecuriFes and ExchangeCommission.All informaFon in this presentaFon is as of thedateof therelease,andIntrexonundertakesnodutytoupdatethisinformaFonunlessrequiredbylaw.Non-GAAPFinancialMeasuresThispresentaFonpresentsAdjustedEBITDA,whicharenon-GAAPfinancialmeasureswithinthemeaningofapplicablerulesandregulaFonsoftheSecuriFesandExchange Commission (SEC). For a reconciliaFon of Adjusted EBITDA to net loss a`ributable to Intrexon in accordance with generally accepted accounFngprinciplesandforadiscussionofthereasonswhythecompanybelievesthatthesenon-GAAPfinancialmeasuresprovideinformaFonthatisusefultoinvestorsseethetablesbelowunder“ReconciliaFonofGAAPtoNon-GAAPMeasures.” SuchinformaFonisprovidedasaddiFonalinformaFon,notasanalternaFvetoIntrexon’sconsolidatedfinancialstatementspresentedinaccordancewithGAAP,andisintendedtoenhanceanoverallunderstandingoftheCompany’scurrentfinancialperformance.
©2017IntrexonCorp.Allrightsreserved.IntrexonCorporaFonissharingthefollowingmaterialsforinformaFonalpurposesonly.SuchmaterialsdonotconsFtuteanoffertosellorthesolicitaFonofanoffertobuyanysecuriFesofIntrexon.AnyofferandsaleofIntrexon’ssecuriFeswillbemade,ifatall,onlyupontheregistraFonandqualificaFonofsuchsecuriFesunderallapplicablefederalandstatesecuriFeslawsorpursuanttoanexempFonfromsuchrequirements.Thea`achedinformaFonhasbeenpreparedingoodfaithbyIntrexon.However,IntrexonmakesnorepresentaFonsorwarranFesastothecompletenessoraccuracyofanysuchinformaFon.AnyrepresentaFonsorwarranFesastoIntrexonshallbelimitedexclusivelytoanyagreementsthatmaybeenteredintobyIntrexonandtosuchrepresentaFonsandwarranFesasmayariseunderlawupondistribuFonofanyprospectusorsimilarofferingdocumentbyIntrexon.
Forward-LookingStatements
Collabora;ons,MarketableProducts&SectorsUpdateAndrewLast,Ph.D.–ChiefOperaFngOfficer
©2017IntrexonCorp.Allrightsreserved.4
Precigen:Biotech2.0PlagormforHumanHealth
• Intrexonhasassembledtheworld’sleadingtechnologyplaJormforprogrammingandengineeringthemostpervasivecodeontheplanet
• Precigenwillu;lizeplaJormtobuildnextgenera;ontherapeu;cproductsfasterandcosteffec;vely
• Broadpipelineofclinicalandpre-clinicalprograms,bothpartneredandun-partnered
©2017IntrexonCorp.Allrightsreserved.5
NewCollaboraFons:MicrobialExpressionPlagorms
June2017-Enteredintoanexclusivecollabora;onwithJohnsonMaUhey(LSE:JMAT),agloballeaderinsciencethatenablescleanerair,improvedhealthandmoreefficientuseofnaturalresources,focusedonthedevelopmentofmicrobialstrainsforfermentaFveproducFonofpepFde-basedacFvepharmaceuFcalingredientsMay2017-Enteredintoaresearchcollabora;onagreementwithHuvepharmaEOOD,aglobalpharmaceuFcalcompanywithafocusondeveloping,manufacturing,andmarkeFnghumanandanimalhealthproducts,fortheuFlizaFonandfirstcommercialapplicaFonofIntrexon’sproprietaryfungalexpressionplagormtoproduceanewanimalfeedenzymedevelopedbyHuvepharma
©2017IntrexonCorp.Allrightsreserved.6
EnviroFlight:BSFLarvaeProducFonFacilityinUS
• EnviroFlight-aJointVenturebetweenIntrexon&DarlingIngredients(NYSE:DAR)o ConstrucFonoflargestcommercial-scale
blacksoldierfly(BSF)larvaeproducFonfacilityinUSbeganinMay2017
o SignificantlyexpandproducFonofadvancedingredientsforsustainablefeedandnutriFonderivedfromBSFlarvae
o Supportsdevelopmentofsustainablehigh-nutriFonBSFingredientsforthe$60Bglobalanimalfeedindustry
• Ini;alcapacityfromUSplantexpectedinQ1’2018o ProducFonwillbetargetedtowardlivestock,
petfoodandaquaculturemarkets
©2017IntrexonCorp.Allrightsreserved.7
AquAdvantage®Salmon
AquAdvantage®SalmonFirstengineeredfoodanimalapprovedby
USFDA/HealthCanada
• AquaBountyenteredintoanagreementtopurchasecertainassetsofBellFishCompanyo Land-basedfarmingfacilityinAlbany,Indiana
o Expectedannualcapacityof1200metrictonsà>$10millionayearinpotenFalsaleswithpotenFalforexpansion
o AquaBountyevaluaFngaddiFonalopportuniFesforlargerproducFonfaciliFesinUSandCanada
• AdvantagesofAquAdvantage®plaJormo LowerproducFoncostdrivenbyfastergrowthrateandless
feedrequirement
o NovaccinesoranFbioFcs
o Land-basedproducFonenables“produc(onanywhere”
o PotenFalforlowersupplyfluctuaFonsandsteadyprices
• Atlan;csalmonmarketinU.S.es;matedat>$2billiono AlmostallcommerciallyavailableAtlanFcsalmonisfarmedo USaloneimportsover200,000tonsoffarmedAtlanFc
salmonannually
©2017IntrexonCorp.Allrightsreserved.8
CommercialLaunchinFall2017
CommerciallaunchoffreshslicedArc;c®applesinUSinFallof2017
Non-chemical,preserva(ve-freeapproachtonon-browning
Nochangetoappletaste/textureFreshslicedapplemarketinUSes(matedat~$500millioninsales
©2017IntrexonCorp.Allrightsreserved.9
ArcFc®Non-browningPlagorm
Arc;c®AppleFirstUSDAapprovednon-browningapplewithout
useofchemicaloranFoxidanttreatments
• Plan;ngprogressandoutlook:o AnFcipateplanFngover250,000ArcFc®apple
treesduring2017andover500,000in2018
o Planningfor4millionArcFc®appletreestobeplantedbyyearend2020
• Commitmenttodevelopment:o Completed2017staffingandlabfacilitybuild-out
planstosupportFssuecultureandmolecularbiologyacFviFes
• Expandingapplica;onsofnon-browningplaJorm:o ProducFoncostadvantagegivennoneedfor
preservaFvecoaFngso AddiFonalfruits(cherries,pearsandavocados)o Vegetables(leGuce)
©2017IntrexonCorp.Allrightsreserved.10
BroadApplicaFonofInnovaFveArcFc®SoluFon
• UniqueArc;cAdvantage™solu;oncreatesbenefitsforcommercialtreefruitvarie;esacrosstheen;resupplychain,fromgrowerstoconsumers
• Conveniencedrivesconsumerpurchaseselec;on,especiallyinhealthysnackmarket
• Posi;velyimpactfoodwaste,lesseningthecurrent40%ofappleswasted
©2017IntrexonCorp.Allrightsreserved.11
Friendly™Aedes
Friendly™AedesScalableenviro-friendlymosquitosoluFontosuppressthedisease-carryingAedesaegyp(
• Receivedposi;veevalua;onforEuropeanStandardinrela;ontohumanhealthandtheenvironmento TheNaFonalInsFtuteofPublicHealthandthe
Environment,Netherlands(RIVM)publishedreportconcludingOxitec'sFriendly™mosquitoeswouldposenegligibleriskstohumanhealthandtheenvironment
o France’sHighCouncilforBiotechnologypublishedsupporFveposiFon
• Ongoinggeographicexpansionin2017o Brazil–SignedmulF-yearcontractinJuizdeFora,the
secondBrazilianmunicipality&firstcityinMinasGeraiso Colombia-AnnouncedMOUinComuna16region,anarea
of>104,000residentsinMunicipalityofSanFagodeCalio India–IniFatedoutdoorcagedtrialstodemonstratethe
efficacyofFriendly™mosquitoesincollaboraFonwithGangabishanBhikulalInvestmentandTradingLimited
o ExpecttoiniFatenewdeploymentsinexisFng&newregions
©2017IntrexonCorp.Allrightsreserved.12
DurableSuppressionwithOxitec’sSoluFon
Over80%suppressionofAedesaegyp(inCECAP/EldoradoPiracicaba,Brazilmaintainedinsecondyearwithreleaseofnearly60%lessOxitecmosquitoesascomparedtoyearone
Levelofcontrolillustra(veofstrengthanddurabilityofOxitec’ssolu(on
Favorablepublicopinionwith93%ofci(zenssuppor(ngOxitec’ssolu(ons*
*CW7MarketResearchInsFtutesurvey
Aedesaegyp*wildlarvae(Larvae/trap;4weekaverage)
©2017IntrexonCorp.Allrightsreserved.13
IntrexonMethaneBioconversionPlagorm
Intrexonhasdevelopeddisrup;veMBPtechnologythatenablestheprofitableuseoflowcostnaturalgastoreplaceoil
asthefeedstockforseveralhighvalueindustrialproducts.
NeedforcleanerburningfuelsinautomoFveandotherindustries
Gas-to-liquidsconversioncurrentlyreliesoncostly,energy-intensiveprocesses
OilsuppliesdesFnedfordepleFon
NegaFveeco-impactfromgasflaring
HighCapex&HighOpex
©2017IntrexonCorp.Allrightsreserved.14
SignificantGas-to-LiquidsOpportunitythroughMBP
Intrexon’sEngineeredMethanotrophsChemicalsachieved:• 2,3Butanediol*• Isobutyraldehyde*• 1,4Butanediol*• Isoprene
NaturalGasFeedstock
Fuelsachieved:• Isobutanol*• Farnesene
MBPplagormhasachievedsixdifferentmoleculestodate.ThefourmoleculesacFvelyunderdevelopment(*)withIntrexon’sMBPtechnology
haveacumulaFveTotalAddressableMarketofover$100billion.
©2017IntrexonCorp.Allrightsreserved.15
2,3Butanediol(BDO)ProgressUpdate
2,3butanediol
Methane bioconversion
Catalytic conversion
Naturalgas Butadiene
• Observed30%increasein2,3BDOyieldsontopof30%increaseachievedduringthefirstquarter
• 2,3BDOtestproducFonrunscompletedinpilotplant
• Providing2,3BDOproducedtochemicalcatalystcompaniesforconversiontobutadieneandqualitytesFng
• AnFcipatesiteselecFonforsmall-scalefacilitybyyearendandsubsequentlyiniFaFngdesignofplantwithprojectedgroundbreakingin2018
Intrexon’s500LPilotPlant
Intrexon’son-purposebutadieneprocessan(cipatedtohaveCOGSsub$1,000permetricton
©2017IntrexonCorp.Allrightsreserved.16
HarvestIntrexonEnterpriseFund
→ADSkincare,Inc.Advancedtopicaldeliverysystemtoimproveskinelas(city,firmnessandreduc(onoffinelinesandwrinkles
→CRSBio,Inc.Targeteddeliveryofan(bodiesfortreatmentofchronicrhinosinusi(s
→ExotechBio,Inc.Innova(veexosomepla`ormfordeliveringtherapeu(cRNAusingselecttargets
→GentenTherapeu;cs,Inc.ActoBio(cs®expressionofglutenpep(destotreatceliacdisease
→RelieveGene;cs,Inc.Non-opioidgenetherapyapproachforneuropathicpainresultantfromcancer
→ThriveAgrobio;cs,Inc.ActoBio(cs®toexpressnutri(veproteinstosupportintes(nalmatura(onandfunc(onandenhancenutrientabsorp(oninearlyweanedpigs
©2017IntrexonCorp.Allrightsreserved.17
ThriveAgrobioFcs:NutriFonforWeanlingPigs
TheProblem:• By2050globalconsumpFonof
animalproteinwillbetwo-thirdshigherthancurrentlevels
TheOpportunity:• Porkisthemostwidelyeatenmeatin
theworldtodayaccounFngfor36%ofglobalmeatconsumpFon
• WorldwideproducFonofindigenouspigmeatin2010was$168billion
• HealthyproducFonwillbevitaltomeetgrowingdemandinasustainablemanner
OurSolu;on:UFlizeIntrexon’sActoBioFcs®plagormtodevelopacost-effecFvebiologicthateasilyintegratesintocurrentcommercialporkproducFonsystemsenabling:
• Increasedgrowthperformance• Improvedfeedefficacy• Amoresustainablefoodsupply
Sources:h`p://www.fao.org/news/story/en/item/116937/icode/;h`p://www.fao.org/ag/againfo/themes/en/meat/backgr_sources.html;h`p://www.fao.org/docrep/019/i3440e/i3440e.pdf
©2017IntrexonCorp.Allrightsreserved.18
LeveragingActoBioFcs®toImproveFeedEfficiency
Goal:TodevelopanadvancedfeedaddiFveforimprovedguthealthandpigletweaningoutcomesApproach:UFlizeActoBioFcs®plagormtoexpressnutriFveproteinstargetedforthegastrointesFnaltract2017Milestones:o PosiFvedatafromaninvivoanimalstudy;9.6%to12.4%
moretotalweightgainover3weekdosingperiodinpigso Completeddevelopmentofcommerciallyrelevant
formulaFonwithfavorablecostofgoods
Nextsteps:o Largepigstudyusingcost-effecFveformulaFonasafeed
addiFve
HealthSectorAndrewLast,Ph.D.–ChiefOperaFngOfficer
©2017IntrexonCorp.Allrightsreserved.20
ClinicalDevelopmentOutlookfor2017
PaFentpopulaFon#basedonUnitedStatesonly;*EsFmatednewcasesperyear;**ZIOPHARMplanstoiniFatepivotaltrialin2017
Indica;on Gene/CellTherapy Pa;ent# Phase XONCost XONEconomicsRecurrentGlioblastoma Ad-RTS-IL12 >10,000* Phase3** 0 20%OpProfits
RecessiveDystrophicEB FCX-007 2,500 Phase1/2 0 14%NetSales
Adv.LymphoidMalignancies Non-viralSBCD19+CART >80,000* Phase1 0 20%OpProfits
Relapsed/RefractoryAML ViralCD33+CART <8,000* Phase1 0 20%OpProfits
PediatricBrainTumors Ad-RTS-IL12 >3,000 Phase1 0 20%OpProfits
RecurrentGlioblastoma Ad-RTS-IL12&Checkpoint >10,000* Phase1 0 20%OpProfits
Indica;on Gene/CellTherapy Pa;ent# IND XONCost XONEconomicsAML Off-the-ShelfNK ~20,000* 2017 0 20%OpProfits
LinearScleroderma FCX-013 ~40,000 2017 0 14%NetSales
OralMucosiFs ActoBioFcsAG013 ~500,000 2017 0 12%NetSales
Clostridiumdifficile LanFbioFcsOG716 ~500,000* 2017 0 25%OpProfits
SolidTumors SleepingBeautyTCR n/a 2017 n/a n/a
WetAMD AAV-RTS >1million 2017 50%(JV) >50%OpProfits
T1Diabetes ActoBioFcs >1.25million 2017 50%(JV) 50%OpProfits
CardiacDisease pXoXMulF-Gene >20million 2017 75%(Sub) 75%OpProfits
Upto8addi*onalINDsfiledbyyearend2017
©2017IntrexonCorp.Allrightsreserved.21
UFlizingSleepingBeautySysteminT-CellTherapiesTarge;ngNeoan;gensinSolidTumorsWithTCR-
ModifiedTCellsUsingNon-ViralSleepingBeautySystem CARs:Intrexon,ZIOPHARM,&MerckKGaA• DisFncFvemethodcentersonproprietaryRTS®
plagormtoregulateexpressionofmembrane-boundinterleukin-15co-expressedwithCARsandSleepingBeautynon-viralgeneintegraFon
• IL-15iskeydriveroftherapeuFceffectinCAR+Ttherapy*
• CARtargetsareexpressedonawiderangeofcancers,includinghematologicandsolidtumors
TCRs:Intrexon,ZIOPHARM,&NCI
• CRADAwithNCIforthedevelopmentofACT-basedimmunotherapiesgeneFcallymodifiedusingtheSleepingBeautysystemtoexpressTCRsforthetreatmentofsolidtumors
• SleepingBeautyusedtoexpressneoanFgen-specificTCRstodevelopindividualizedimmunotherapiesforpaFentswithcancer
*JournalofClinicalOncology.2017Mar14:JCO2016713024:LymphomaremissionscausedbyCD19-specificCAR+Tcellsareassociatedwithelevatedseruminterleukin-15levels;NatureMedicine22,26–36(2016;MolecularTherapeuFcs.2016Jun;24(6):1078-89
©2017IntrexonCorp.Allrightsreserved.22
Q2ProgressinOncologyGene&CellTherapies
0 10 20 30 40 50 60 70 80 900
20
40
60
80
100Vehicle/VehicleAd 5e9+V 10 mg/m2
Days
Perc
ent S
urviv
al
0 20 40 60 800
20
40
60
80
100Vehicle/VehicleAd+V 30mg/m2 qdx14antiPD1 15mg/m2 q4dx5Ad+V 30mg/m2 +antiPD1 15mg/m2
Days
Perc
ent S
urvi
val
Pon;neGliomaPre-clinicalStudy
0 10 20 30 40 50 60 70 80 900
20
40
60
80
100Vehicle/VehicleAd 5e9+V 10 mg/m2
Days
Pe
rce
nt
Su
rviv
al
An;-PD-1Combina;onPre-clinicalStudyZIOPHARMPhase1studyforrecurrentglioblastoma(rGBM)• UpdateatASCOreportedmedianoverall
survivalof12.5months,withmeanfollow-upof9.2months(20mgcohort,N=15)
• Comparesfavorablytohistoricalcontrols
Enrollmentinstereotac;carmhasbegun• PaFentsnotscheduledforresecFon• Runwaytopediatric&anF-PD-1
combinaFonstudies
ZIOPHARMannouncedacceptanceofINDforCD-33specificCARTcelltherapy• Expectstotreat1stpaFentinQ3’2017
0 20 40 60 800
20
40
60
80
100Vehicle/VehicleAd+V 30mg/m2 qdx14antiPD1 15mg/m2 q4dx5Ad+V 30mg/m2 +antiPD1 15mg/m2
Days
Per
cen
t Su
rviv
al
Vehicle/VehicleAd+V30mg/m2qdx14anFPD115mg/m2q4dx5Ad+V30mg/m2+anFPD115mg/m2
Vehicle/VehicleAd5e9+V10mg/m2
©2017IntrexonCorp.Allrightsreserved.23
RareDiseases
FCX-007RareSkinDiseaseGeneTherapy(FibrocellScience):• DataSafetyMonitoringBoardrecommendedconFnuaFonofPhase1/2clinicaltrialfortreatment
ofrecessiveepidermolysisbullosa(RDEB)followingplannedreviewofsafetydatafromfirstadultpaFentdosed
• CompleteddosingoffirstcohortofthreeadultpaFentsinPhase1porFonoftheclinicaltrial
FCX-013RareSkinDiseaseGeneTherapy(FibrocellScience):• GrantedRareDiseaseDesignaFonforFCX-013forthetreatmentofmoderatetoseverelinear
scleroderma• Highlightedpre-clinicaldataforFCX-013atthe20thAnnualMeeFngoftheAmericanSocietyof
Gene&CellTherapy
©2017IntrexonCorp.Allrightsreserved.24
CompleFonofGenVecAcquisiFon
→DevelopinganextgeneraFonviralplagormwithsignificantlyhigherpayloadcapacity(exceeding30kb)comparedtocurrentviraldeliverymethods(4.5kb–9kb)
→Willfurtheradvancethefieldofgene-basedmedicinebyenablingdeliveryofmulFpletherapeuFceffectorsnotpossiblewithcurrentviraldeliverysystems
→RTS®plagormcombinedwithGenVec’sAdV-basedtechnologyisprojectedtoacceleratecu�ng-edgegenetherapiesthatregulateinvivoexpression
• VectorswithnoorverylowseroprevalenceinthehumanpopulaFon• Non-integraFngtransgenelimitsprobabilityofdisturbanceofvitalcellulargenes• EfficienttransducFonindividingcellsandnon-dividingcells• Largepackagingcapability(upto12kb)withmulFpleexpressioncasse`es• ImprovedsafetywithmulFpledeleFonsinvectorgenomes• Administeredtoover3,000clinicalstudysubjects• Scalableplagormwithefficientmanufacturingprocessanda`racFvecostofgoods
AdenoVerse™Strengths
PrecigenHelenSabzevari,Ph.D.-SVP,HumanTherapeuFcsandHeadofR&DforPrecigen
©2017IntrexonCorp.Allrightsreserved.26
EvoluFonofPrecigen:NewModelforBioPharma
• Uniqueposi(onamongPharmaandBiotech
• Comprehensivesuiteoftechnologypla`orms,throughinven(onandacquisi(on,underoneroof
• Crea(onofuniqueinternalpor`olioforR&Dinvarious
therapeu(careasbasedoncombina(onsofpla`ormsincludingbiologics,gene(cengineering,immunology,microbiome,vaccines,amongothers
• Leadingposi(oninsingleandmul(geneprogrammingwith
limitedoff-targeteffects
• Wellposi(onedtocapitalizeonthesubstan(alpromiseofengineeringbiologytosolvecri(calissuesinhumanhealthandpersonalizedcare
PrecigenTechnologyPlaJorm
Precise&ControlledTherapy
©2017IntrexonCorp.Allrightsreserved.27
CAR-TTherapy:EarlyPromiseandChallenges
• Cu�ngedgeofcancerimmunotherapyo FirstFDAapprovalexpectedin2017for
hematologicalmalignancy
• CAR-TcellsexpandinpaFentsuponengagementwithanFgenexpressingtumor/normalcells
• Majorchallengeofcurrentapproachesissignificanttoxicityduetolackofcontrolo CytokineReleaseSyndrome(CRS)o TumorLysisSyndromeo Neurotoxicity
• LimitaFonsofcurrentapproacheso ExpressionofanFgenonnormalFssueso AnFgenlossandheterogeneous
expressionofanFgen
Kenderianetal.,CancerResearch,2014
©2017IntrexonCorp.Allrightsreserved.28
RegulaFngCAR-TCells:RheoSwitch®andKillSwitch
TransgeneexpressioncanbeturnedONandOFFindosedependentmanner
Intrexon’splagormenablingcontroloverefficacyandsafetyofCAR-Tcells
RTS®controlledco-expressionofcytokines
RTS®controlledexpressionofCAR
KillSwitchforselecFvedepleFonofCAR-Tcells
©2017IntrexonCorp.Allrightsreserved.29
KillSwitch
GeneSwitchControlofCARExpression
4,500
5,500
6,500
7,500
8,500
9,500
ON OFF ON
CARExpressionRheoSwitch®
VeledimexONVeledimexOFF
CAR
!CAR
!CARCAR
©2017IntrexonCorp.Allrightsreserved.30
ImprovedPersistencewithRTS®ControlledmbIL-15
VeledimexOFF
0
50,000
100,000
150,000
200,000
250,000
ON OFF ON
mbIL-15Expression
mbIL-15mRNALevels
FoldChangeinmbIL-15mRNAoverCARonlycontrol
CAR
!
"
CAR
mbIL-15
CAR
VeledimexON
0.0
20.0
40.0
60.0
80.0
100.0
3 7 10 14%CellSurvial
Daysinculturefollowingcytokineremoval
CAR-TCellSurvival
• VeledimexdosedependentexpressionofmbIL-15canbeturnedONandOFF
• ImprovedpersistenceofCAR-Tcellsbyco-expressionofmbIL-15onlyinpresenceofveledimex
CARmbIL-15
!CAR
mbIL-15
CAR
"mbIL-15
©2017IntrexonCorp.Allrightsreserved.31
TregsPlayAMajorRoleInAutoimmuneDiseases
InflammatorybowlDisease(IBD) T1diabetes(T1D)
Mul;pleSclerosis(MS)
Lupus(SLE)
RheumatoidArthri;s(RA)
• RegulatoryTCells(Tregs)arecriFcalforcontrolofimmunesystem• TregshaveimpairedfuncFonalityinautoimmunediseaseswhere
paFent’simmunesystemisunleashed
Tregs
©2017IntrexonCorp.Allrightsreserved.32
Intrexon’seTregs:ChangeofParadigm
0
20
40
60
80
100
1:1 1:2 1:4 1:8 1:16 NoTregs
CD4Tcells CD8Tcells
Tcellsupp
ression[%
]
• OvercominglimitaFonsofnaturallyderivedTregs• InvitrostudiesdemonstratepotentsuppressionofproliferaFonofeffectorTcellsthroughengineeredTregs(eTregs)
• PotenFalfornewwaveoftherapeuFcsforvariousautoimmunediseases
Tregs
FinancialOverviewJoelLiffmann–SeniorVicePresident,Finance
©2017IntrexonCorp.Allrightsreserved.34
SecondQuarterFinancialPerformance
*Non-GAAPfinancialmeasure.
SelectFinancialHighlights 2Q2017 2Q2016
Revenues $54.4M $52.5M
CollaboraFonandLicensingRevenues $28.2M $27.5M
Product&ServicesandOtherRevenues $26.2M $25.0M
AdjustedEBITDA $(2.0)M* $(5.4)*
Impactofchangeindeferredrevenuerelatedtoupfrontandmilestonepayments $(9.4)M $116.1M
BasicEPS $(0.16) $(0.42)
TotalEquity&PreferredStockSecuriFesInConnecFonWithECCs $168.6M $159.0M
Cash,CashEquivalents,andShort-andLong-TermInvestments $157.2M $321.2M
©2017IntrexonCorp.Allrightsreserved.35
PorgolioExpansion&MaturaFon
June2017
1Over30
January2011
Collabora;ons
June2017
$17.5MM
$392Million
January2011
TAFandReceivedMilestones
June2017
$0 $233Million
January2011
CostRecovery
PorJolioofBackendEconomics
FutureMilestones&Royal;es
©2017IntrexonCorp.Allrightsreserved.36
Summaryü Over30collaboraFonsinplacecapitalizingon
ourleadershipposiFonintheengineeringofbiology
ü StrongcashposiFonwith$157millionincash,cashequivalents,andshort-terminvestments,andequitysecuriFesandpreferredstockvaluedat$168million
ü Developinghighvaluebio-soluFonswithourcollaboratorsinlargeestablishedmarketswithbuilt-indemand
ü PosiFonedtodelivermeaningfulreturnstoourshareholdersthroughourscalable,capitalefficientmodelandsuccessfulexecuFonofcurrent&futureopportuniFes
Q&ASession