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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