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Targeted Therapeutics for Hard-To-Treat Cancers

Investor PresentationDecember 2017

AboutUs

• Phase2oncologypipelineincludingaPARP/Tankyrase inhibitor(2X-121)positionedforQ12018mBC study– dataexpectedQ42018

• DrugResponsePredictor(DRP®)Dx technologyidentifieslikelyresponderstoeachcompoundbasedontheactionablepatternoftheindividualpatient’stumorgeneexpression

• Partneringconversationsforex-U.S.marketsinprogress

• Financingroundunderwayafter$3.5Mseedround- PotentiallyonlyprivatefinancingpriortoM&A/IPO

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ExecutiveTeam

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Peter Buhl Jensen, MDChairman

• FounderandCEOofTopoTarget A/S• SecuredEMAandFDAapprovalofSavene©/Totect©• DevelopedBelinostat,FDA-approvedin2014

George ElstonCEO

• 20+yearsveteranlifescienceexecutive• Oncology,ophthalmologyandwomen’shealth• BoardmemberDeutscheBankDBXTrustandpreviouslyCelldexTherapeutics

Marie Foegh, MDCMO

• 28yearsinpharmaceuticalandbiotech• ExecutiveatIPSENandBayerPharmaandAgile• 10+drugstakenthroughdevelopmentandFDAapproval

Jarne ElleholmCFO

• 20+yearsinpharmaceuticalandbiotech• VCpartnerandPharmaexecutiveexperience• ChairmanofScandinavianMicroBiodevices,Meta-IQ

ProgramDevelopmentTeam

Ulla H. BuhlFounderClinicalLiaison

Steen Knudsen, Ph.D. DRP Founder

Bruce Pratt, Ph.D. CMC

Dr. Joyce A. O’ShaughnessyBaylorUniversity,USOncology

Dr. Daniel D. Von HoffU.ofArizona,MayoClinic,USOncology

Dr. Mansoor Raza MirzaNSGO/DBCG Rigshospitalet,U.ofCopenhagen

Dr. Mary Lake Polan, YaleUniversity,Dept.ofOB/Gyn

Dr. Henry S. FriedmanDukeUniversityTischBrainTumorCenter

Oncology Venture team

Scientific Advisory BoardDr. Ursula A MatulonisDana-FarberCancerInstituteSmithCenterforWomen’sCancers

Mogens WinkelMadsen, Ph.D. Manufacturing

James G. Cullem J.D. FounderBDLiaison

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Phase2Pipeline

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Phase2DRP®Selected

Phase2Near-termMilestones

2X-121PARP1/2and

Tankyrase 1/2Inhibitor

•Metastaticbreastcancer

•Recurrentovariancancer•Prostatecancer(mCRPC)

•Pancreaticcancer

Phase2studyresults2H2018

Phase2studyresultsQ12019

2X-111Glutathione-enhancedPEGylatedLiposomal

Doxorubicin

•Brainmetastasesfrombreastcancer

•Recurrentglioblastomamultiforme

Phase2studyresults1H2019

2X-131Topoisomerase1

Inhibitor

•Recurrentovarian cancer Phase2studyresults1H2019

Big Data driven Pathway Detection:Systems biology (network) view

Knowledge Driven Pathway Detection:Traditional pathway view

VS

graph of 680 non-redundant proteins

ROOTED IN CONCEPTUALLY DIFFERENT VIEWS OF BIOLOGY

DrugResponsePredictor(DRP®)AssaybasedonrelevantgenesselectedfrommRNAinacancercelltranscriptome

DRP®CompanionDiagnostic

①Patternsindrugsensitivityfromhumancelllines(e.g.NCI60)reflectmechanismofactionofaspecificdrug.Thisidentifiesasubsetofgenesresponsibleforsensitivityandresistancetothatdrug.Thisnarrowsthegeneanalysisfrom20,000intothe“hundreds,”providingarawDRPscoreadrugforadditionalfilteringbasedonactualpatienttumordata.

② TherawDRPisfilteredforclinicalrelevanceagainstaproprietarydatabaseofover3,250humantumorsamplesfrom27differentcancers.Thismetadataanalysiseliminatesclinicallyirrelevantgeneexpressions(“backgroundnoise”),creatingthedrug-specificDRP.

③ Thedrug-specificDRPproducesascorefrom0-100basedonthespecificgenesrepresentedinapatient’stumor.A100scorewouldidentifyatumorasahighlylikelyresponder,withallgenesrepresentedforsensitivityandnoneforresistance.

④mRNAdatafrompatientbiopsiesarecomparedtothedrug-specificDRP,producinganindividualscore.ADRPcutoff(e.g. 70%)isselectedbasedonclinicalexperiencewithaparticularcancertypeanddrug.

APatient-unique“Fingerprint”ofGenesPredictsResponsivenesstoaDrug

Drug-specific DRP

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DRPAll genes shown in red are measured by DRP

(total 414 genes measured)

DNA Damage

PARP1BRCA

Apoptosis or successful repair

2X-121

Non-Homolend joining

Base excision

Homologousrepair

PARPtrapping

2X-121

Wnt GSK3B

AXIN1

Beta-Catenin Cell cycle

Tankyrase2X-121

DRP®GeneIdentification:2X-121PARP/Tankyrase Inhibitor

DRP®Validatedin40+ClinicalTrials

• Epirubicin inmetastaticbreastcancerhttp://abstracts.asco.org/199/AbstView_199_192238.html

• Epirubicin,Exemestan,Anastrozole,and Fulvestrant inadvancedbreastcancerhttp://www.medical-prognosis.com/investor-and-media/20170124-drp-successfully-predicts-effect-of-4-breast-cancer-drugs-for-personalized-medicine/?origin=announcements

• Cisplatin,Epirubicin and Capecitabine ingastroesophaeal cancerhttp://journals.plos.org/plosone/article?id=10.1371/journal.pone.0148070

• Adjuvant 5FUincoloncancer http://journals.plos.org/plosone/article?id=10.1371%2Fjournal.pone.0155123

• Belinostat inAMLhttp://www.oncologyventure.com/wp-content/uploads/2016/04/Bullinger_ESMO2012.pdf

• Adjuvant CisplatinandVinorelbine inNSCLChttp://oncologypro.esmo.org/Meeting-Resources/ESMO-2016/Multigene-expression-profile-for-predicting-efficacy-of-cisplatin-and-vinorelbine-in-non-small-cell-lung-cancer

• Fulvestrant inbreastcancerhttp://journals.plos.org/plosone/article?id=10.1371/journal.pone.0087415

• CHOPand CHOEPinlymphomas https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4333339/

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DRP®EnablesDynamicStudyDesigns

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NovelPrecisionMedicineTrialDesigns

JAMAOncology: doi:10.1001/jamaoncol.2106.5299

2X-121:PARP1/2andTankyrase 1/2Inhibitor

• Orallybioavailable,brainpenetrable,smallmoleculedrug

• Potentinhibitor of

• Dualinhibitoryactionof2X-121againstPARP1/2andTankyrase 1/2providesbroaderactivitythancurrentPARPinhibitors

• LackoftransportbyP-glycoproteinpotentiallyovercomesresistancetocurrentPARPinhibitors

• Establishedefficacy&safetyprofile;nomyelotoxicityobservedinPh1study11

PARP1Akeymoleculeinsensingandrepairingsingle-strandDNAbreaks

PARP2Anadditionalrepairmechanism

Tankyrase 1/2ImportantregulatorsofcanonicalWnt/β-catenin,acriticalcheckpointinmetastases,particularlyintriple-negativebreastcancer

PARP:Poly(ADPribose)polymerase

olaparibNiraparibE7449

Responsebiomarker

PgP mediatedresistance Myelotox Tankyrase WNT PARPtrapping BBB

penetration

Strongmaintenanceopportunity

olaparib BRCA Yes(1,2) Yes No(8) No Yes No(1) Yes

niraparib BRCA/Myriad(3)HRD YesSPC Yes No No Yes No Yes

veliparib BRCA Yes(4,5) Yes No No No(4,5)

rucaparib BRCA Yes(6) Yes Yes(8) Yes(7) Yes No(6) Yes

talazoparib BRCA Yes No(10) No Yes No(11) Yes

BGB-290(12) BRCAHRD Yes Yes Yes Yes

2X-121 DRP(9) No No Yes Yes Yes Yes Yes

a) in: Profile of veliparib and its potential in the treatment of solid tumors https://www.ncbi.nlm.nih.gov/pmc/articles/PMC45245911) http://www.nature.com/nm/journal/v19/n11/full/nm.3369.html?message-global=remove2) http://www.pnas.org/content/105/44/17079.long https://www.genomeweb.com/cancer/myriad-genetics-stock-drops-after-study-shows-niraparib-may-not-need-cdx3) http://dmd.aspetjournals.org/content/39/7/11614) https://www.ncbi.nlm.nih.gov/pubmed/246475725) https://www.ncbi.nlm.nih.gov/pubmed/24962512 ; http://www.ascopost.com/issues/may-1-2014/preliminary-study-suggests-veliparib-may-be-effective-in-resistant-brca-mutated-ovarian-cancers/6) https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4882768/pdf/bjc201641a.pdf7) http://www.nature.com/scibx/journal/v5/n13/pdf/scibx.2012.323.pdf8) https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4027629/pdf/ml400292s.pdf9) Novel strategies in biomarker discovery are determination of the genomic or expressional signatures of PARP inhibitor sensitive tumors. The rationale behind this approach is that one can define a specific profile of, for

example, HR-deficient tumors. RNA profiling or gene expression arrays have been used for this purpose and show potential in identifying PARP inhibitor sensitive tumors http://www.medscape.com/viewarticle/842072; http://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0064268&type=printable; ttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC4053087/pdf/bcr3625.pdf;

10) http://files.shareholder.com/downloads/MDV/2370126714x0x898732/BB3A5044-7FBC-4CDA-96CB-810A55269B7D/Talazoparib_IR_Presentation_2016-07-06_FINAL.pdf11) http://www.nature.com.ep.fjernadgang.kb.dk/nchembio/journal/v13/n2/pdf/nchembio.2248.pdf12) Source: Beigene filing with SECon19Jan2016

“Forthetranslationalscientist,theidentificationofreliablebiomarkerswillbecriticalforthesuccessofthistargetedagent”aPARPOverview

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2X-121: PARP1/2andTankyrase 1/2InhibitorEstablishedClinicalHistory

SingleAgent Patients Indication Results

Phase1 (UK) 41(28atRx dose)

SolidTumors(includingbreast,pancreaticand

ovarian)

• Welltolerated• 46%diseasecontrol• 7.1%partialresponsesinallcomers• 2durablepartialresponses,200+days

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Thisstudydidnotutilizethe2X-121DRP®

2X-121:PatientBiopsiesforDRP®Validation

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2X-121:StrongDRP®Prediction

• Blindedstudy,13patients

• 2X-121DRPpredictedpatientslikelytorespondandnotrespondtotreatment

• 2X-121DRPcorrectlypredictedresponsetotreatmentandOverallSurvival(p=0.07)- HazardratioonOverallSurvival=0.26

• Clearseparationbetweenresponders&non-responders

• IdentifiedrespondersirrespectiveofBRCAmutationstatus

Total PatientsinGroup OverallSurvivalPredictedresponders 7 5

Predictednon-responders 6 1

15 OverallSurvival:survivalat400daysfromcommencementoftreatmentwith2X-121

Phase1TrialResults:E7449/2X-121

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2X-121DRPcorrectlyidentified• The2patientswithpartialresponse• Non-responders

2X-121:StrongDRP®Prediction

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Prospective/RetrospectiveDRPValidation

0 200 400 600 800

0.00.2

0.40.6

0.81.0

Days

Overa

ll surv

ival

Predicted sens to E7449Predicted resistant to E7449

P=0.07 HR=0.26

Predicted sensitive to 2X-121Predicted resistant to 2X-121

2X-121:PARP1/2andTankyrase 1/2Inhibitor

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ClinicalOpportunitieswithDRP®CDx

Other DRP®andWnt pathways

Other homologousRecombination deficient

BRCA1+2mutated

Likely non-responders to2X-121

Highlikelihoodsensitivesubgroupsareidentifiedbythe2X-121DRP®

MetastaticbreastcancerOvariancancerPancreaticcancerBrainmetastasesfrombreastcancerEndometrial cancerProstate cancer

2X-121:Phase2ClinicalPlans- InitialFocusedStudies

MetastaticBreastCancer• Selecttop10%DRP®• <30heavilypre-treatedpatients• Utilize1,200+patientregistryinDenmark

• Brainmetastasistobeincluded

• 2H2018dataexpected• 50%+responserateanticipated• Potentialforacceleratedapproval

ProstateCancer

• Selecttop10%DRP®• <30heavilypre-treatedpatients• InitialEUstudywithUSsitestofollowunderIND

• 30%+responserateanticipated• Potentialforacceleratedapproval

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Phase2StudyPlan

PotentialOutcomePathways

2X-121:Phase2ClinicalPlans– U.S.Studies

RelapsedOvarianCancer

• Selecttop10%DRP®• Enroll<30heavilypre-treatedpatients(incl.priorPARPrefractory)

• 30%+responserateanticipated• Potentialacceleratedapproval

PancreaticCancer

• Selecttop20%DRP®• Enroll<30heavilypre-treatedpatients

• Endpoint:OS• Opportunityforacceleratedapprovalandorphandesignation

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Phase2StudyPlan

PotentialOutcomePathways

2X-121:Phase2ClinicalPlans- Basket

NCICollaboration– PediatricCancer

• CancertypeselectionbasedonknownPARPi activity- e.g.Neuroblastoma

• DRP®cutoffTBD

• Minimumofdoublingexistingresponseratevs.otherPARPi

• Opportunityforacceleratedapproval

PARPi TumorStudies

• SelectionbasedonknownPARPiactivityinspecifictumortypes

• DRP®cutoffTBD

• Minimumofdoublingexistingresponseratevs.otherPARPi

• Opportunityforacceleratedapproval

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Phase2StudyPlan

PotentialOutcomePathways

2X-121:CurrentStatus

• ProductobtainedfromEisai- 13Kcapsulesforinitialstudies- 14kgofAPI- 78kgofintermediateproduct

• 2X-121DRP®established&validated

• Phase2mBC studyinitiationexpectedQ12018- Registryof~1,200DRP-screenedbreastcancerpatientsinDenmark

• U.S.pre-INDmeetinginJanuary;INDfilingexpectedQ12018

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2X-111: Glutathione-enhancedPEGylatedLiposomalDoxorubicin

• Glutathione(GSH)– enhancementofPEGylatedliposomeexploitstheGSHtransportpumpintheBBBtoallowtransferof2X-111intothebrain

• PatentsincludeGSH-PEGylatedliposomedeliverysystemincombinationwithanthracyclines

NovelTrans-BBBDrugCandidate

glutathione

PEG

doxorubicin

liposome

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DRP®forAnthracyclinePredictsResponseinmBC

13months PFSwithDRP®at75%

7months PFSwithDRP®at25%

In135patientswithmBCtreatedwithepirubicin theDRPpredictedPFSwitha

HazardRatioof0.5(p=0.02)

PredictedResponse

0%

100%

Buhl et al. Retrospective-prospective blinded evaluation predicting efficacy of epirubicin by a multigene assay in advanced breast cancer within a Danish Breast Cancer Cooperative Group (DBCG) cohort. J Clin Oncol 35, 2017 (suppl; abstr 1071).24

2X-111:Glutathione-enhancedPEGylatedLiposomalDoxorubicin

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EstablishedClinicalHistorySingleAgent Patients Indication Results

Phase1 37 Safety Welltolerated

Phase2a 17 Brain metastasesfrombreastcancer

PR:2(12%)SD:9(52%)

Phase2a 20 GBM PR:1(5%)SD:7(35%)

Thesestudiesdidnotutilizethe2X-111DRP®

2X-111:ClinicalDevelopmentPlans

GlioblastomaMultiforme• Selecttop20%DRP®• Enroll<20patients

a) 4+patientsPR/SDat6+months→ acceleratedapprovaldiscussionwithFDA

b) 2-3patientsPR/SD→enroll10additionalpatients

BrainMetastasesfromBreastCancer• Selecttop40%DRP®• Enroll<20patients

a) 30%+patientsPR→ repeatstudy;acceleratedapprovaldiscussionwithFDA

b) 4-5patientsPR→ pivotalPhase2withevaluationofothermetastases

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Phase2StudyPlan

PotentialOutcomePathways

2X-111:Glutathione-enhancedPEGylatedLiposomalDoxorubicin

• U.S.INDobtainedJune2017• DrugproductmanufacturingQ1(potentialTaiwanpartner)• 2X-111DRP®established&validated• Danishregistryof~1,200DRP-screenedbreastcancerpatientsavailableformBC Phase2study– umbrellastudy

• GlioblastomastudyatCopenhagenandDukeUniversityHospitals

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CurrentStatus

2X-131:Topoisomerase1Inhibitor

• Orallybioavailablesmallmoleculecamptothecin

• Bindstothetopoisomerase1-DNAcomplex

• Preventsre-ligationofsinglestrandbreaksthatareinducedbytopoisomerase1torelievetorsionalstraininDNA

• MOA:cytotoxicityfromdoublestrandDNAdamageproducedduringDNAsynthesis

• Favorable safetyprofile

• Clinically-demonstratedefficacyinwiderangeofcancers

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2X-131:EstablishedClinicalHistory

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CompletedPhase1&Phase2Studiesin>400PatientsConductedinEU,US,JapanandChina

Phase TreatedPatients Tumor types1 108 Solidtumors1 35 Solidtumors1 43 Glioma1 4 ADME1 157 Solidtumors1 26 Solidtumors

250 Breast69 Ovarian

ADME-AbsorptionDistributionMetabolismExcretion

2X-131: Topoisomerase1InhibitorEstablishedClinicalHistory

SingleAgent Patients Indication Results

Phase2 69 Ovariancancer • Welltoleratedwithimprovedefficacy &safety profilevs.topotecan &irinotecan

• 47%responserate in19patientswithPlatinumFreeInterval(PFI)≥6months

• 16%PRinpatientswithPFI<6months

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Thisstudydidnotutilizethe2X-131DRP®

2X-131:Phase2ClinicalDevelopmentPlan

OvarianCancer

• Selecttop30%DRP®• Enroll<20heavilypre-treatedpatientswithPFI<6months

a) 3+patients→ repeatstudy;discussacceleratedapprovalwithFDA

b) <3patients→ revisitDRP®cutoff

EndometrialCancer

• Selecttop20%DRP®• Enroll<20late-stagepre-treatedpatients

• Opportunityforacceleratedapprovalandorphandesignation

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Phase2StudyPlan

PotentialOutcomePathways

2X-131: CurrentStatus

• DrugproductavailablefromChinapartnerforPhase2studies

• PlanningDRP®-selectedPhase2studyinrecurrentovariancancer

• ExpandedcollaborationdiscussionswithChinapartner

• U.S.INDfiling/reopeningplannedforQ12018

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Q12018 Q22018 Q32018 Q42018 Q12019 Q22019

DevelopmentTimeline

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2X-121Metastaticbreastcancer- Phase2

2X-121 Recurrentovariancancer- Phase2

2X-121Pancreaticcancer- Phase2

2X-111 BCBM– Phase2

2X-111 Glioblastomamultiforme– Phase2

2X-131Recurrentovariancancer– Phase2

Potential1H’19CompanyProfile

• PositivereadoutsfromsevenDRP-focusedPhase2studies

• MultiplepivotalPhase2studies foracceleratedapprovalfilings

• Additionalindicationsincludingtumor-agnostic(basket)studies

• Multiplecollaborationsex-US.• Ex-U.S.partnershipsinplace

PositionedforM&A/IPOParallelPath

2X-121 Prostatecancer- Phase2

Contacts

GeorgeO.ElstonChiefExecutiveOfficer

george.elston@2xoncology.com

AmyRaskopfIR/CorporateCommunicationsamy.raskopf@2xoncology.com

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