biotron limited (asx:bit) biotech showcase 2017...biotron – leader in viroporin-targeng drug...

BIOTRONLIMITED(ASX:BIT)BiotechShowcase2017

Forward Looking Statements

This presenta,onmay contain forward-looking statementswith respect to the financial condi,on, results andbusinessachievements/performanceofBiotronLimited(ACN086399144)andcertainoftheplansandobjec,vesof its management. These statements are statements that are not historical facts. Words such as “should”,“expects”, “an,cipates”, “es,mates”, “believes” or similar expressions, as they relate to Biotron Limited, areintended to iden,fy forward-looking statements. By their nature, forward-looking statements involve risk anduncertainty because they reflect Biotron’s current expecta,ons and assump,ons as to future events andcircumstancesthatmaynotproveaccurate.Thereisnoguaranteethattheexpectedevents,trendsorresultswillactuallyoccur.Any changes in suchassump,onsorexpecta,ons could causeactual results todiffermateriallyfromcurrentexpecta,ons.

InvestmentHighlights

•  Developingnewclassofan,viraldrugs

•  Leadprograms:BIT225targe,ngHIV-1eradica,onandHepa,,sCvirus(HCV)–mul,-billiondollarmarkets

•  Phase1/2atrialscompletedinover200subjects–safetyandefficacyvalidated

•  Severalnearterm,value-addingmilestonesdrivenbytwokeyHIV-1studiesin2017:

•  BIT225treatmentinterrup,onstudyandBIT225Phase2HIV-1humantrial

BoardMichaelHoy Non-execu,veChairman

MichelleMiller ManagingDirector

SusanPond Non-execu,veDirector

RobThomas Non-execu,veDirector

DenisWade Non-execu,veDirector

CorporateSnapshot

KeyFinancialMetricsTickerCode ASX:BIT

SharePrice(09Jan17) A$0.04

Marketcapitalisa,on A$12.24million

12MonthTradingRange A$0.040–0.105

SharesOutstanding 313million

CashPosi,on(09/2016) A$2.29million

•  SpunoutfromJohnCur,nSchoolofMedicalResearchattheAustralianNa,onalUniversityin1999

•  ListedonASXinJan2001(ASX:BIT)

•  HeadquarteredinSydney,Australia

BriefBiotronOverview

Biotron–LeaderinViroporin-TargePngDrugDevelopment

•  Coreexper,seisdesignanddevelopmentofanewclassofan,viraldrugstarge,ngviral-encodedviroporinproteins

•  Viroporinsarepresentinbroadrangeofviruses:Influenza(M2),HIV-1(Vpu),HCV(p7),DengueandWestNile(Mprotein),SARS(Eprotein)andothers

•  Broadplalorm:

•  Rapid,proprietaryprimarybacterialcell-basedscreeningassaysfortargetproteins

•  Focusedlibraryofcompoundsthattargettheseviralproteins

•  Pipelineofinternally-generated,first-in-classsmallmoleculeviroporininhibitorsforkeymarkets

Viroporins

•  Smallhydrophobicproteinswithion

channelac,vity•  Formhydrophilicporesinhostcell

membranes•  Keystagesoftheviralcyclesuchasvirus

uncoa,ng,transportandmatura,onareion-influencedprocessesinmanyviralspecies

•  Crucialforviralpathogenicityduetoinvolvementinvariousstepsofviruslifecycles

•  IdealtherapeuPctargetsNatureReviewsMicrobiology10,563-574

Compound Discovery Process

Designofcompoundstoexplore3DspaceanddetermineSARoftargetproteins;expansionofcompoundlibrarytoincreaseac,vityagainsttarget

Compoundsscreenedinproprietaryassaysetupforeachvirustargete.g.HIV-1Vpu;HCVp7;InfluenzaM2;DengueM;CoronavirusE.

Hitstestedagainstvirusincellcultures;Secondaryscreeningofhitsagainstotherkeyvirusese.g.HepB,influenza,Zika

Leadop,misa,onandselec,on.Currentlead:BIT225(HIV-1andHCV).BIT314(HCV)isnextgeneraPon

1

2

3

4

Addi,onalchemistrytorefinehits

Core Technology Drives Rich Compound Library

Libraryofcompoundsdesignedtotargetviroporins:Ini,ally>250compoundsdesignedandsynthesised;librarynow~350OTHER“HITS”INLIBRARYinclude:•  InfluenzaAandB•  Coronaviruses(Including

SARS)•  Epstein-Barrvirus(EBV)•  Hepa,,sBvirus(HBV)•  Zikavirus•  others

X-axis:compoundIDY-axis:virusZ-axis:strengthofhit

Biotron-AdvancedPipeline

INDICATION

COMPOUND

DISCOVERY PRECLINICAL PHASE1 PHASE2 PHASE3

HCV BIT225

HIV-1 BIT225

NextgeneraPon-HCV

BIT314

Dengue Leads

HIV-1Reservoirs

•  HIV-1remainshiddeninreservoirs,leadingtochronic,life-longinfec,on

–  Invisibletobody’simmunedefenses

–  Notsensi,vetoan,-HIV-1drugs

•  Eradica,onwillrequiremul,pleapproaches;approachesinclude:

–  An,-latencyagentsforlatently-infectedTcells

–  Drugstomodifyimmuneresponse

–  Drugstarge,ngHIV-1inmacrophagelineagecells

MarioStevensonScien6ficAmerican299,78-83(2008)

HIV-1:TowardsaCure

•  Over1.1millionpeoplelivingwithHIV-1intheUSA,with1in6unawareofdiagnosis

•  US$11.9bnsalesinUS,EuropeandJapanin2013;expectedtogrowtoUS$16.8bnby2020

•  HIV-1pa,entsneedtostayonan,retroviraldrugs(ART)tokeepviruslevelsundercontrol

•  Long-termhealthimplica,onseveninpa,entsonan,retroviraldrugse.g.HAND,immuneac,va,on,etc

•  Newmodeofac,onsdrugsareneededtoeradicateorcureHIV-1infec,on

•  BIT225inhibitsassemblyandbuddingofnewvirusinmacrophages•  Phase2atrial(004)demonstratedthatBIT225canreduceHIV-1levelsinmacrophagecellsinvivo,

parallelinginvitrostudies(Wilkinsonetal,JAn,microbChemother.2015Nov29.pii:dkv389.[Epubaheadofprint])

•  Poten,albenefitsonimmuneagingandHIV-associateddemen,a•  Poten,alforuseinfutureviruseradica,ontreatment

BIT225TargetsHIV-1inReservoirCells

Time(days)+HIV-1

50100150200

16 17 19 21 22 23 24 25 26 27 28

+BIT225

BIT225StopsHIV-1Replica7oninHumanMacrophageCells

A B

(A)UntreatedControls (B)BIT225treatedcells

BIT225–ProvenClinicalAcPvityAgainstHIV-1•  BIT225-004:Phase1b/2arandomised,placebocontrolled,double-

blindtrial–  21pa,ents,HIV-1posi,ve,treatment-naïve;10daysdosing

withBIT225(monotherapy)•  ResultsdemonstratedthatBIT225:

1.   SignificantlyreducesHIV-1levelsinthemacrophage(reservoir)cells

2.   Crossestheblood-brainbarrier,openingupthepossibilityoftreatmentofAIDS-relateddemenPa

2.   Reducedmyeloid-specificimmuneacPvaPonmarkersduringtrial

2 4 6 8

10 12 14 16

5 10 15 20 25

HIV-1Re

plica,

on(p

g/20

0uL)

TimeinCo-culture(days)

BIT225Placebo

PotenPalroleforBIT225:-  AddiPontocurrentARTtoeradicatekeyreservoirs,impacPngimmuneacPvaPon-  Keycomponentofcure/eradicaPonstrategies

HIV-1ViralDynamics

BIT225-009 Hu-MouseATI

BIT225 HIV-1 Eradication – Next Steps

Crea,ngvalueinflec,onpointswithposi,vetrialdata•  Ini,atePhase2HIV-1ClinicalTrial-3monthtrialincombina,onwithART(BIT225-009);Expecttrial

commencementearly2017–Dataexpectedin3Q17•  Expectedoutcome(s)–Impactonkine7csofviralloaddecayincombina7onwithARTindica7ng

impactonunderlyingviralreservoir,alsoimpactonimmuneac7va7on

•  Analy,calTreatmentInterrup,on(ATI)Study–Currentlyunderwayevalua,ngBIT225inHIV-1InfectedHumanisedMice-DataexpectedQ12017

•  Expectedoutcome(s)–impactonviralkine7csincombina7onwithART,pluspoten7alimpactonreboundonceARTisstopped

•  Accumulatedsignificantquan,tyofclinicaldataforBIT225fromhealthyvolunteer,HCV&HIV-1pa,enttrials.

BIT225–FirstofaNewClassofHCVDAADrugs

•  Novel,oral,smallmoleculecompound

•  Onlyoneofitsclass(p7inhibitor)inclinicaltrials

•  Inhibitsviralassemblyandinfec,vity

•  Pan-genotypeac,vity:

•  Ac,veinvitroagainstallmaingenotypes

•  Clinicallyac,veagainstHCVGT1(1aand1b)andGT3

•  Seekingpartnershipsforfurtherdevelopment,inpar,cular,inAsia

POLYMERASE/PROTEASEINHIBITORSe.g.Sofosbuvir/Simeprevir

BIT225-ASSEMBLY/BUDDINGINHIBITOR

HCVBIT225ProgramSnapshot

-  PreparedbasedonaboveazertheAugustBoardmee,ng

-  Guidedthewordingoftheprospectusdrazandtheuseofproceeds

-  Whilecapitalrequirementsaredeterminedbasedonproposedplan,thefinalschedule

ofworkwillbelargelydictatedbyavailablecapital

-  FourclinicaltrialsIHCV-infectedsubjectscompleted(includingoneHIV-1/HCVcoinfectedtrial)

-  PromisingclinicalefficacyagainstHCV

-  HCVGT1(BIT225-005)–100%receiving400mgBIDfor28daysincombina,onwith48weeksIFN/RBV(perprotocol)werevirus-freeat48weeks

-  HIV-1/HCVGT3(BIT225-006)–100%receiving300mgBIDfor28daysincombina,onwith48weeksIFN/RBV(perprotocol)achievedSVR12i.e.curedofHCVinfecPon

-  BIT225increasestherateatwhichHCVisclearedincombinaPonwithotherdrugs

HCV–RemainsanOpportunity

•  EmergingevidencethatInterferonsparingtherapiesmaycausereac,va,onofHepa,,sB(HBV)

•  Evidenceofreac,va,onofhepa,,sBinco-infectedpa,ents(HBV&HCV)presentedatAASLD

•  30–50millionHCV-infectedsubjectsinChina

•  HighHCV/HBVco-infec,onrateinChina

•  Poten,alforanotherclassofDAAsuchasBIT225toshortentreatmentandreducecosts,inpar,cularinmarketsex-USA/Europe

UnlockingValueinCompoundLibrary

•  Renewedindustryinterestintarge,ngviraldiseasesincluding

•  Respiratorydiseasese.g.Respiratorysyncy,alvirus(RSV)&Influenza

•  Hepa,,sBvirus

•  TropicaldiseasesincludingDengue

•  Ebola,ZikaandMERS-CoVoutbreakshavecausedpublichealthissuesworldwide

•  BIT225hasdemonstratedtherobustnessofBiotron’sapproachwithtargePngviroporinproteins

•  Compoundswithac,vityagainstotherkeyviruseshavebeeniden,fied;secondaryscreeningisinprogress,withtheaimofiden,fyingpoten,alcandidatestoprogressintoIND-enablingstudies

•  MainfocusremainsoncommercialisingtheCompany’sHIV-1andHCVprograms,butessen,althatotheropportuni,esaredeveloped

DengueVirusProgram

•  2.5billionpeople(40%worldpopula,on)liveinareasatriskofDengue

•  ~100millionpeopleinfectedyearly

•  Aleadingcauseofillnessanddeathintropicsandsubtropics

•  Transmissionisbymosquito;mostpreven,onprogramstargetthevector

•  NoapprovedDengue-specifictherapeu,cdrug

•  Vaccinetrialshavehaddisappoin,ngresults

•  Biotronistarge,ngDengueMprotein–SimilartargettoHIV-1/VpuandHCV/p7

•  Severalcompoundswithpromisingac,vityhavebeengenerated;testsareon-going

•  Poten,alforpan-Flavivirustherapeu,c

www.sciencenews.org

HepaPPsBVirus

•  LimitedscreeningofBiotroncompoundlibraryhasgeneratedinteres,ngdata

•  Hitsiden,fied

•  Veryexperiencedscien,ficadvisoryandopera,onalteaminplaceforHBV

•  Seekingcollabora,ontoexplorehitsanddevelopprogram

•  Hepa,,sBremainsasignificantunmetneedwithamul,-billiondollarmarket

CommercialisaPonandPartnering

•  HIV-1Program-Significantvalueinflec,onpointsaroundHIV-1programdataexpectedin2017

•  HCVProgram-BIT225par,cularlywellsuitedtoAsia,withhighnumbersofHCV-infectedpa,entsincludingahighpropor,onofHCV/HBVco-infectedpa,ents

•  Earlystagecollabora,onopportuni,esforpre-clinicaltargets,suchas:

•  Dengue

•  Hepa,,sB

•  Addi,onaldevelopmentcollabora,onpoten,alfor“other”pharmatargets

•  Seekingpartnersforindividualtargetsoren,replalorm

KeyMilestonesfor2017

•  CompleteAnaly,calTreatmentInterrup,on(ATI)StudyinHIV-1InfectedHumanisedMice-DataexpectedQ22017

•  Expectedoutcome(s)–Impactonkine7csofviralloaddecayincombina7onwithARTindica7ngimpactonunderlyingviralreservoir

•  CompletePhase2HIV-1Trial-Dataexpectedin3Q17

•  Expectedoutcome(s)–impactonviralkine7csincombina7onwithART,pluspoten7alimpactonreboundonceARTisstopped

•  FinalisearegionalpartneringagreementforBIT225forHCV

InvestmentHighlights

PorfolioofpatentsandpatentapplicaPonsdirected to theCompany’s anP-viraldrugporfolio

STRONGINTELLECTUALPROPERTYPOSITION

TargePngviroporinproteinswitharapidscreeningproprietaryprimarybacterialcell-basedplaform-alibraryofover350compoundswithacPvityagainstarangeofviruses.

NOVELANTIVIRALPLATFORM

ClinicalandPreclinicalprogramsinindicaPonswithhighunmetclinicalneedorlargepaPentpopulaPonssuchasHIV-1,HCV&Dengue,HBV,Zika&Influenza

BROADANTIVIRALPIPELINE

Completed7humanClinicalTrialswithpromisingsafetyandefficacyoutcomes

ROBUSTCLINICALVALIDATION

15 20

REPORT ANNUAL

BIOTRON

41

DrMichelleMillerManagingDirector+61412313329mmiller@biotron.com.auwww.biotron.com.au