sydney nsw 2000 tel: (61-2) 9300 3344 fax: (61-2) 9221 ...jan 13, 2017  · presentation to the...

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Level 2, 66 Hunter Street Sydney NSW 2000 Tel: (61-2) 9300 3344 Fax: (61-2) 9221 6333 E-mail: [email protected] Website: www.biotron.com.au 13 January 2017 The Manager Companies ASX Limited 20 Bridge Street Sydney NSW 2000 (22 pages by email) Dear Madam BIOTRON LIMITED PRESENTS AT BIOTECH SHOWCASE TM 2017 Biotron Limited advises that Dr Michelle Miller, Managing Director, will be giving the attached presentation to the Biotech Showcase TM 2017 Conference being held this week in San Francisco, California, USA. In addition, Dr Miller will give briefings to USA institutional investors and pharmaceutical company representatives as part of activities surrounding the annual JP Morgan Healthcare Conference. This is one of the most important annual healthcare investor conferences, attracting thousands of healthcare and life science business executives, as well as investors and analysts, to San Francisco. The Biotech Showcase TM features corporate presentations by innovative life science companies to an audience of public and private investors, business development executives and professional advisors who are interested in investment opportunities and collaborations. Now in its ninth year, it expects to attract upwards of 2,800 attendees. Yours sincerely Peter J. Nightingale Company Secretary pjn8740 For personal use only

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Level 2, 66 Hunter Street

Sydney NSW 2000

Tel: (61-2) 9300 3344

Fax: (61-2) 9221 6333

E-mail: [email protected]

Website: www.biotron.com.au

13 January 2017

The Manager Companies

ASX Limited

20 Bridge Street

Sydney NSW 2000 (22 pages by email)

Dear Madam

BIOTRON LIMITED PRESENTS AT BIOTECH SHOWCASETM

2017

Biotron Limited advises that Dr Michelle Miller, Managing Director, will be giving the attached

presentation to the Biotech ShowcaseTM

2017 Conference being held this week in San Francisco,

California, USA.

In addition, Dr Miller will give briefings to USA institutional investors and pharmaceutical company

representatives as part of activities surrounding the annual JP Morgan Healthcare Conference. This

is one of the most important annual healthcare investor conferences, attracting thousands of

healthcare and life science business executives, as well as investors and analysts, to San Francisco.

The Biotech ShowcaseTM

features corporate presentations by innovative life science companies to an

audience of public and private investors, business development executives and professional advisors

who are interested in investment opportunities and collaborations. Now in its ninth year, it expects

to attract upwards of 2,800 attendees.

Yours sincerely

Peter J. Nightingale

Company Secretary

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BIOTRONLIMITED(ASX:BIT)Update–January2017

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Forward Looking Statements

Thispresenta,onmaycontainforward-lookingstatementswithrespecttothefinancialcondi,on,resultsandbusinessachievements/performanceofBiotronLimited(ACN086399144)andcertainof the plans and objec,ves of its management. These statements are statements that are nothistoricalfacts.Wordssuchas“should”,“expects”,“an,cipates”,“es,mates”,“believes”orsimilarexpressions,astheyrelatetoBiotronLimited,areintendedtoiden,fyforward-lookingstatements.By their nature, forward-looking statements involve risk and uncertainty because they reflectBiotron’scurrentexpecta,onsandassump,onsastofutureeventsandcircumstancesthatmaynotprove accurate. There is no guarantee that the expected events, trends or results will actuallyoccur. Any changes in such assump,ons or expecta,ons could cause actual results to differmateriallyfromcurrentexpecta,ons.

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BiotronLimited

•  SpunoutfromJohnCur,nSchoolofMedicalResearchattheAustralianNa,onalUniversityin1999

•  ListedonASXinJan2001(ASX:BIT)

•  HeadquarteredinSydney,Australia

•  Directors•  MichaelHoy Chairman

•  MichelleMillerCEO&ManagingDirector;ex-Johnson&JohnsonResearch;ex-Start-upAustraliabiotechfund

•  DenisWade Independentnon-execu,vedirector;ex-J&J(ChairmanandMDJohnson&JohnsonResearch); DirectorofHeartwareInc(NASDAQ:HTWR)

•  SusanPond Independentnon-execu,vedirector;ex-J&J(ChairmanandMDJohnson&JohnsonResearch)

•  RobThomas Independentnon-execu,vedirector;DirectorofHeartwareInc(NASDAQ:HTWR);Starpharma (ASX:SPL),REVAMedicalLimited(ASX:RVA);VirginAustraliaLimited(ASX:VAH)

Slide2

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Biotron–LeaderinViroporin-TargeHngDrugDevelopment

•  Biotron’scoreexper,seisbasedondesignanddevelopmentofanewclassofan,viraldrugstarge,ngviralionchannelproteins(viroporins)

•  Viroporinsarepresentinbroadrangeofviruses:

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

•  Broadplakorm:

•  Rapid,proprietaryprimarybacterialcell-basedscreeningassaysfortargetproteins

•  Targetedlibraryofcompoundsthattargettheseviralproteins

•  Pipelineofinternally-generated,first-in-classsmallmoleculeviroporininhibitorsforkeymarkets

Slide3

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Viroporins

Slide4

•  Smallhydrophobicproteinswithionchannelac,vity

•  Formhydrophilicporesinhostcellmembranes

•  Keystagesoftheviralcyclesuchasvirusuncoa,ng,transportandmatura,onareion-influencedprocessesinmanyviralspecies

•  Crucialforviralpathogenicityduetoinvolvementinvariousstepsofviruslifecycles

•  Idealtherapeu,ctargetsNatureReviewsMicrobiology10,563-574

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Biotron’sCoreTechnology

Slide5

Designedlibraryofcompoundstotargetviroporins:

IniHally>250compoundsdesignedandsynthesised;librarynow~350

BIT225(HIV-1andHCV)

BIT314(HCV)

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

Hitstestedagainstvirusincellcultures

Leadop,misa,onandselec,on

OTHER“HITS”INLIBRARYinclude:-  InfluenzaAandB-  Coronaviruses

-  IncludingSARS-  Epstein-Barrvirus(EBV)-  HepaHHsBvirus(HBV)-  Zikavirus-  others

BIT225:-  RepresentaHonofthevalue

thatresideswithinBiotron’scoreexperHse;

-  Valuable,Phase2clinicalasset

DENGUE–SeveralcompoundswithpromisinganHviralacHvity

OTHERVIRUSES–Secondaryscreeningofhitsagainstkeyvirusese.g.HepB,influenza,Zika

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BIT225Snapshot

-  PreparedbasedonaboveaoertheAugustBoardmee,ng

-  Guidedthewordingoftheprospectusdraoandtheuseofproceeds

-  Whilecapitalrequirementsaredeterminedbasedonproposedplan,thefinalschedule

ofworkwillbelargelydictatedbyavailablecapital

-  FirstinclassdrugandnewdrugtargetfortreatmentofHIV-1andHepa,,sCvirus(HCV)

-  Sevenclinicaltrialscompleted;anotherisfullyrecruitedwithdataexpected1Q16

-  Over200subjectsdosedintrialstodate

-  PromisingclinicalefficacyagainstHIV-1andHCV

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

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

-  BIT225increasestherateatwhichHCViscleared

-  BIT225efficientlyinhibitsHIV-1replica,oninmacrophagereservoircellsinvitroandinvivo(BIT225-004)

Slide7

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BIT225–FirstofaNewClassofHCVDAADrugs

ü  Novel,oral,smallmoleculecompound

ü  Onlyoneofitsclass(p7inhibitor)inclinicaltrials

ü  Inhibitsviralassemblyandinfec,vity

ü  Pan-genotypeac,vity:

ü  Ac,veinvitroagainstallmaingenotypes

ü  Clinicallyac,veagainsthard-to-treatHCVGT1(1aand1b)andGT3

ü  Seekingpartnershipsforfurtherdevelopment,inpar,cularinAsia

POLYMERASE/PROTEASEINHIBITORSe.g.Sofosbuvir/Simeprevir

BIT225-ASSEMBLY/BUDDINGINHIBITOR

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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

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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

Slide10

MarioStevensonScien@ficAmerican299,78-83(2008)

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

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

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

BIT225TargetsHIV-1inReservoirCells

Slide11

Time(days)+HIV-1

50100150200

16 17 19 21 22 23 24 25 26 27 28

+BIT225

BIT225StopsHIV-1Replica7onA B

(A)UntreatedControls (B)BIT225treatedcells

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BIT225–ProvenClinicalAcHvityAgainstHIV-1

•  BIT225-004:Phase1b/2arandomised,placebocontrolled,double-blindtrial

–  21pa,ents,HIV-1posi,ve,treatment-naïve;10daysdosingwithBIT225(monotherapy)

•  Resultsdemonstratedthat:

1.   BIT225significantlyreducesHIV-1levelsinthemacrophage(reservoir)cellsBIT225cancrosstheblood-brainbarrier,openingupthepossibilityoftreatmentofAIDS-relateddemenHa

2.   BIT225reducedmyeloid-specificimmuneacHvaHonmarkersduringtrial

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

ResultssupportapotenHalroleforBIT225incure/eradicaHonstrategies

Slide12

Phase2HIV-1trialofBIT225incombinaHonwithcurrentanH-HIV-1drugsscheduledtocommenceearly2017

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HIV-1ViralDynamics

BIT225-009 Hu-MouseATI

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CurrentHIV-1ProgramTrials

Slide14

•  Phase2trialan,cipatedtocommenceearly2017

•  12weeksBIT225incombina,onwithcART

•  Expectedoutcome(s)–Impactonkine7csofviralloaddecayincombina7onwithARTindica7ngimpactonunderlyingviralreservoir,alsoimpactonimmuneac7va7on

•  Headlinedata3Q17

•  HumanisedMouseStudy

•  Modellingtreatmentinterrup,on(ATI)

•  Inprogress

•  Expectedoutcome(s)–impactonviralkine7csincombina7onwithART,pluspoten7alimpactonreboundonceARTisstopped

•  Data1Q17

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UnlockingValueinCompoundLibrary

Slide15

•  Renewedindustryinterestintarge,ngviraldiseasesincluding

•  Respiratorysyncy,alvirus(RSV)

•  Hepa,,sBvirus

•  TropicaldiseasesincludingDengue

•  Influenza(inpar,culardrugresistantstrains)

•  EbolaandMERS-CoVoutbreakshavecausedpublichealthissuesworldwide

•  BIT225hasdemonstratedtherobustnessofBiotron’sapproachwithtargeHngviroporinproteins

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

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

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X-axis: compound ID Y-axis: virus Z-axis: strength of hit

Compound Library is Rich Source of Hits

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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-Flavitherapeu,c

Slide17

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HepaHHsBVirus

•  LimitedscreeningofBiotroncompoundlibraryhasgeneratedinteres,ngdata

•  Hitsiden,fied

•  Veryexperiencedscien,ficadvisoryandopera,onalteaminplaceforHBV

•  Seekingcollabora,ontoexplorehitsanddevelopprogram

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SummaryI•  Uniquecoreexper,seagainstnovelviraltargets

•  Demonstratedproofofconceptofsuccessfultarge,ngofviroporinswithBIT225

•  BIT225isanovelapproachwithdemonstratedpromisingefficacyinPhase2a/2clinicaltrials

•  HCVandHIV-1arehighgrowth,mul,-billiondollarmarkets

•  Treatmentgapsremain

•  Representsanewclassofdirect-ac,ngHCVdrugs

•  Poten,altofillsignificantHCVtreatmentgaps,shortentreatmentandreducecosts

•  Poten,altoeradicateimportantHIV-1reservoirs,plusmayimpactonimmuneac,va,on

•  Robustdatapackagehasbeengeneratedtosupportcombina,onstudieswithpoten,alpartners

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SummaryII

•  Technologycoreisanan,viralplakormwithnewclassofsmallmoleculeswithbroadrangeofac,vi,es

•  Extendingearlierstageprogramsforotherkeyviruses:

•  DevelopingleadsforprogramsincludingDengueandHBV

•  Iden,fyinghitsforothervirusesincludingRSV,Zika,BK,andothers

•  Denguevirus–applyingfornon-equityfundingfromUSorganisa,ons

•  Poten,altoexpandtoareasofpoten,alpartnerinterest

•  Seekingcollabora,onsforindividualprogramsoren,replakorm

Slide20

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

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