17
1 Optimizing Immunotherapies CORPORATE OVERVIEW (Please contact us to request confidential version of presentation with supporting data)

Vycellix Corporate Presentation … · Oncolytic Virus Within Tumor ... Pipeline Review. 9 ... Vycellix Corporate Presentation (NONCONFIDENTIAL WEBSITE)

Embed Size (px)

Citation preview

1

OptimizingImmunotherapies

CORPORATEOVERVIEW

(Pleasecontactustorequestconfidentialversionofpresentationwithsupportingdata)

www.Vycellix.com 2

CertainstatementscontainedinthispresentationorinotherdocumentsofVycellix,Inc.(the“Company”),alongwithcertainstatementsthatmaybemadebymanagementoftheCompanyorallyinpresentingthismaterial,maycontain“forward-lookingstatements”asdefinedinthePrivateSecuritiesLitigationReformActof1995.Thesestatementscanbeidentifiedbythefactthattheydonotrelatestrictlytohistoricorcurrentfacts.Theyusewordssuchas"estimate,""expect,""intend,""believe,""plan,""anticipate,"“projected”andotherwordsandtermsofsimilarmeaninginconnectionwithanydiscussionoffutureoperatingorfinancialperformanceorcondition.ThesestatementsarebaseduponthecurrentbeliefsandexpectationsoftheCompany'smanagementandaresubjecttosignificantrisksanduncertainties.

ForwardLookingStatements:

www.Vycellix.com 3

§ Closely-held biotech advancing innovative small moleculesthat optimize and enhance human immune responseagainst disease with a focus on cancers

§ Founded in 2015 on IP developed by medical researchers atSweden’s Karolinska Institutet (KI)

§ Pipeline of novel, targeted small molecules for ex vivo andin vivomodulation of cells

§ Lead products have demonstrated unrivaled enhancementof cytotoxic lymphocytes

§ Scientific founders include KI’s current Head of Gene & CellTherapy Group and KI’s former Dean of Research

CorporateOverview

4

EvrenAlici,M.D.,Ph.D.:CEO&Chairman•Head,GeneandCellTherapyGroup,DivisionofHematology,DepartmentofMedicine,Karolinska Institutet,Karolinska UniversityHospital,Stockholm

Hans-Gustaf Ljunggren,M.D.,Ph.D.:ChiefMedicalOfficer• FormerDeanofResearch,Karolinska Institutet andFounderoftheCenterforInfectiousMedicine,DepartmentofMedicine,Karolinska Institutet,Karolinska UniversityHospital,Stockholm

DouglasW.Calder:President• StrategicAdvisortotheNSUCellTherapyInstitute;formerExecutiveVP&OfficerrolesattheVaccine&GeneTherapyInstitute(VGTI),AccentiaBiopharmaceuticals,Viragen andBiovest International

SamuelDuffey,Esq.:ExecVP,GeneralCounsel•CEOofViraCell,ahollow-fibermanufacturingVLPvaccinecompanybasedatTexasA&M;formerrolesincludePresident&CEOofAccentiaBiopharmaceuticalsandBiovest International,andattorneyatSEC

MichaelJ.Keller,J.D.:ExecVP,IntellectualPropertyOfficer•CEO,KellerLifeScienceLaw;formerrolesincludeChiefPatentCounselforIVAX,partneratleadingIPlawfirms,andtechnologytransferrolesattheNationalCancerInstitute(NCI)

Vycellix FoundingBoardofDirectors:

www.Vycellix.com 5

§ Optimizinggenemodification

UnrivaledEnhancementofNKcells&TcellsLeadproductcandidates:VY-OZ&VY-X

§ Rapidlyincreasingexvivoand invivoimmuneresponse

ExVivoApplications

InVivoApplications

ExVivoApplications

Small Molecule Immunomodulator

Small Molecule Immunomodulator

InVivoApplications

Pre-INDCandidates:APipelineinEachMolecule

GeneticModificationEnhancement

Platform

OptimizesCRISPR-Cas9Gene

EditingRates

UnrivaledTransductionRatesforLymphocytesandStemCells

PlatformtoBoostSerialKillingfor

CytotoxicLymphocytes

UnrivaledIncreasedLevelsofPerforin &GranzymeB

SUPRESSINGCERTAINRNAPATHWAYS

OPTIMIZINGADOPTIVECELLTHERAPIES

IMPROVINGGENEEDITINGEFFICACY

DecreasesgRNARecognition&

IncreasesEditingEfficacy

OPTIMIZINGADOPTIVECELLTHERAPIES

Myositis,Myocarditis,Encephalitis,Meningitis

AdjuvantTherapyforDampening

ViralInflammation

HIGHLYSPECIFICANTI-TUMORAGENT

AdjuvantTherapytoOncolyticViralVectorTherapies

SignificantlyInhibitsTumorDevelopmentinMyelomaModels

EnhancesDistributionofOncolyticVirusWithinTumor

TargetedExpressionofPerforinandGranzymeB

www.Vycellix.com 7

ProductDevelopmentStrategy:

ü Platformtechnologyfortheefficient,cost-effectivemodulationofcellandgenetherapies

ü Monotherapyand/oradjuvanttherapeuticstargetingcancersandinfectiousdisease

ü EnhancersofgeneeditingforCRISPR-Cas9systems

8

PipelineReview

www.Vycellix.com 9

DespiteNKcellsbeinginherentlyresistanttoretroviralinfections,VY-OZ:

§ Enablesretroviralandlentiviral genedeliverytoNKcells,targetingintracellularantiviraldefensemechanisms

§ Providesdosedependenteffectwithnoobservedtoxicity

VY-OZ:ExVivoGeneticModification

www.Vycellix.com 10

§ OptimizesgeneticmodificationofTcells&NKcells

§ Supportsdesignof:- NKcellsexpressingcytokines- Silencedinhibitoryreceptors- Activatingreceptors- CARs

§ IncreasesgeneeditingratesforCRISPRsystems

VY-OZExVivoPlatformSummary

11

PipelineReview

www.Vycellix.com 12

Standardpracticeforadoptivecelltransferislimitedbyprocesses,includinginvitrocytokineexposure,thatfailtoobtainsufficientserialkillingbyeachcytotoxiclymphocyte.

Incontrast,VY-X:§ IsanovelRNAconstructthatishighlyspecificandleadstoa

significantlyincreasedloadofperforin andgranzyme BinNKcellsandTcells

§ OvercomestheobstaclesoflimitedefficacyofadoptivetransferofNKcellsandTcellsbyincreasingcytotoxicactivity

VY-XExVivoPlatform:EnhancesSerialKilling

www.Vycellix.com 13

§ 1600-2000x increaseinPerforin§ 800-1100x increaseinGranzyme B§ FirstknownreportofaRNAconstructleading

tosuchadramaticdifferenceintheproteomicprofileofcytotoxiclymphocytes

VY-XExVivoPlatform:IncreasestheKillingPowerofCytotoxicCells

www.Vycellix.com 14

BecauseVY-X isanovelRNAconstructthatishighlyspecificandleadstoasignificantlyincreasedloadofperforin andgranzyme BinNKcellsandcytotoxicTcells,itisbeingadvancedasadirecttherapeutic.

§ Showssignificantdelayintumordevelopmentcomparedtoactivecontrols inexperimentaltumorsystems

§ Safewithnooff-targeteffectsorsevereadverseeventsobserved

§ Believedtoimproveclinicaloutcomeswhencombinedwithotherimmunotherapies

VY-XInVivo:DirectCancerTherapeutic

www.Vycellix.com 15

IPProtectedTechnologyPlatform§ Assets are not encumbered by any external ownership

rights, royalty obligations or other restrictions§ Market exclusivity strategy includes patents, trade

secrets and planned orphan drug protections§ IP filing: “Enhanced Gene Delivery to Natural Killer Cells,

Hematopoietic Stem Cells and Macrophages”§ IP filing: “Novel RNA Construct”

www.Vycellix.com 16

InvestmentHighlights:§ VY-OZ&VY-X (exvivo)representimmediatemarketopportunities

asplatformstooptimizeadoptiveimmunotherapyby:- enhancinggeneticmodification- improvingserialkillingcapacity

§ VY-X (in vivo) represents a highly-specific anti-cancer agentenhancing T cell/NK cell mediated immune response

§ VY-OZ (ex vivo) represents a genome editing optimization agent toincrease gene editing rates for CRISPR-Cas9 systems using viralvectors

§ Other market opportunities for pipeline: oncolytic viral vector uses,targeting viral inflammation and veterinary applications

§ IP provides long-term market exclusivity protections

§ Significant corporate partnering/licensing interest

§ World-class scientific leadership

17

OptimizingImmunotherapiesTorequestConfidentialslidedeck

withsupportingdata,pleasecontact:DouglasW.Calder,President

Phone:772-418-6302/Email:[email protected]