PresentationtointernationalporphyriapatientgroupMelbourne,AustraliaandLeatherhead,UK,February8,2016
ClinuvelPharmaceuticalsLtd(ASX:CUV;XETRA‐DAX:UR9;ADR:CLVLY)presentedtoaninternationalporphyriapatientmeeting,heldinRotterdam,theNetherlands,onFebruary6,2016.Thepresentationandspeakingnotesareappendedtothisannouncement.‐End‐AboutClinuvelPharmaceuticalsLimitedClinuvelPharmaceuticalsLtd(ASX:CUV;XETRA‐DAX:UR9;ADR:CLVLY)isaglobalbiopharmaceuticalcompanyfocused on developing drugs for the treatment of a range of severe disorders. With its unique expertise inunderstandingtheinteractionoflightandhumanskin,thecompanyhasidentifiedpatientpopulationswithaclinicalneedforphotoprotectionandforrepigmentation.Theworldwideprevalenceofthesepatientgroupsrangefrom5,000to45million.Clinuvel’sleadcompound,SCENESSE®(afamelanotide16mg),wasapprovedbytheEuropeanCommissionin2014forthepreventionofphototoxicityinadultpatientswitherythropoieticprotoporphyria(EPP).HeadquarteredinMelbourne,Australia,ClinuvelhasoperationsinEurope,Switzerland,theUSandSingapore.Formoreinformationgotohttp://www.clinuvel.com.SCENESSE®isaregisteredtrademarkofClinuvelPharmaceuticalsLtd.InvestorenquiriesInvestorRelations@clinuvel.com
Forward‐LookingStatementsThis release to theAustralianSecuritiesExchangeand topressmaycontain forward‐lookingstatements, includingstatements regardingfutureresults,performanceorachievements.Thesestatementsinvolveknownandunknownrisks,uncertaintiesandotherfactorswhichmaycauseClinuvel’sactualresults,performanceorachievementstobemateriallydifferentfromanyfutureresults,performancesorachievementsexpressedorimpliedbytheforward‐lookingstatements.Thesestatementsreflectourcurrentviewswithrespecttofutureeventsandarebasedonassumptionsandsubjecttorisksanduncertainties.Giventheseuncertainties,youshouldnotplaceunduerelianceontheseforward‐lookingstatements.Someofthefactorsthatcouldaffecttheforward‐lookingstatementscontainedhereininclude:thattheFDAmayrequireadditionalstudiesbeyondthestudiesplannedforproductcandidatesormaynotprovideregulatoryclearances,includingforSCENESSE®;thattheFDAmaynotprovideregulatoryapprovalforanyuseofSCENESSE®orthattheapprovalmaybelimited;thatClinuvelmayneverfileanNDAforSCENESSE®regulatoryapprovalintheUS;thattheCompanymaynotbeabletoaccessadequatecapitaltoadvanceitsvitiligoprograms;thattheCompanymaynotbeabletoretainitscurrentpharmaceuticalandbiotechnologykeypersonnelandknowhowforfurtherdevelopmentofitsproductcandidatesormaynotreachfavourableagreementswithpotentialpricingandreimbursementagenciesinEuropeandtheUS.Level5,160QueenStreet T+61396604900 www.clinuvel.comMelbourne,Victoria3000 F+61396604999Australia
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InlinewithEuropeanGuidelines onMedicalAdvertisingandUKAdvertisingStandardsforMedicinalProducts,ClinuvelwillnotdiscusstheprofileoftheproductSCENESSE®(afamelanotide16mg)duringthismeeting.Theproductisapprovedtopreventphototoxicityinadulterythropoieticprotoporphyria(EPP)patients.InformationontheproductcanbefoundonClinuvel’swebsiteatwww.Clinuvel.com.Today’sinvitationisappreciatedandisaheadof thelaunchofSCENESSE®inEurope.ThedistributionofSCENESSE®asafirst‐in‐classproduct.Thereareanumberofchallengesinyearstocome,oneoftheseistheperceptionthattheproductcouldbeusedoff‐label;Clinuvelwilldoitsutmosttoavoidthis.Regulatoryconcernscentrearoundthisaspect.
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Thepossibleforwardlookingstatementsinthisdiscussionare notaimedatinvitingorsolicitinginterestinthecompany.
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Today’sdiscussionpointsarerelevant tophysicians,expertsinthetreatmentofthedisease,andEPPpatientsandtheirfamilies.
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Thefirstdiscussionpoint isaimedatcharacterisationofthedisorder.
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Thestigmaspatientshavereported toourteamsovertheyearsarefoundinanabundanceofletters,emailsandfromphoneconversations(left).Thelabellingofthediseasebybystanders,‘outsiders’,differsfromthedescriptionpatientsseemtogivethemselves(right).
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In2005therewere anumberofkeyquestionsindevelopingadrugforEPP,howeverthehypothesisofSCENESSE®wasansweredafterthefirststudyin2006.Theresponsesfrompatientsandphysicianshasbeenstrongerthanexpected.
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In generalonecannotfullyunderstandthesufferingofapatientuntiloneexperiencestheordeal,symptomsanddiseaseher/himself.Thiswillalwaysbethechasmbetweendrugcompaniesandpatients.Adiscussiononhuman’sdiscriminatorysensesandespeciallyEPP‐patients’abilitytodiscern“visiblelight”isled.
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OvertheyearseightcharacteristicsofEPPhavebeenidentifiedbyourteamsandexperts,andallofthemmakethediseasecomplextoevaluate.Inaddition,astherearenoappropriatescientifictoolstomeasurethecharacteristicsymptoms,theClinuvelprogramledtooptimumsynthesisofamolecule,formulation,environmentandvariableclinicalprogram.
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Aspartoftheregulatorydiscussionsandfilings,thisdiagramwas developedandisdiscussedtoday.EPPpatientsareself‐limitingtheirabilitytoexposeduetothecharacteristicsofthedisorder.AnumberofbehaviouralcomponentsmakeEPPdifferentfromotherlightinduceddisorders,rarediseasesororphandiseases.
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Thenexttopicisthepaediatric development.
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Theestimatedtime ofdevelopmentisapproximately36months,costingmillionsofEUROsincludingproductdevelopment,clinicaltrials,regulatoryfilings,followupofpatients.Theregulatoryviewswilllargelydeterminefurthertimelines.ThesuccessfulcompletionofthedevelopmentofthepaediatricproductdependsoncontinuedsafetyofSCENESSE®reportedinadultEPPpatients,capturedviatheEuropeanEPPDiseaseRegistry(EEDR).
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Thethirdtopicisriskmanagement.
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Themaineightpointsarepartoftheprotocolised treatmentofEPPinEurope,alsoexpectedinotherpartsoftheworld.TheoverallcommitmentforanydeveloperofnoveldrugsistoemphasisePHARMACOVIGILANCE,theabilitytobeextravigilantforitspatientslongterm;Clinuvelundertakesthis.
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Clinuvel’sinitialroll‐outfocusisonthose countrieswithknownpatientpopulations.Thecompany’sdistributioncentreisLeatherhead,UK.
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WithintheEUeachcountryisassessingtheproductonanumberofcriteria,including: benefit,costtosociety,numberofpatientsexpected and riskofoff‐labeluse.
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UnderEuropeanmarketingauthorisation,Clinuvelis establishingandmaintaininganEPP‐specificDiseaseRegistry(EEDR).Theownershipofthemedicaldatawillremainwiththeindividualpatientsandresideineachmedicalcentre,howeverthepseudonymisedmedicaldatarelatedtoEPPwillbemade availabletothe EEDR,andClinuvelwill haveunrestrictedaccesstothesedataforanalyticalpurposestofulfilregulatoryrequirementsoflongtermfollowupofpatients.
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SettimelineshavebeenestablishedbyEMAforClinuvelto reportdatafromitsEuropeanprogram.Thetimelinesgivenhereprovideasample12monthreportingrequirement.
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Thedataflowestablished forpharmacovigilanceiscomplex.Ateachpointoftheseprocesses,thereare controlsinplaceandallthesecontrolsformpartofapharmacovigilancemanagementsystem.Each‘station’onthechartrequiresindependentauditingofprocesses,propermanagementandadequateprotectionofmedicaldata.Anumberofadvisorybodies,governanceboards(GovernanceBoard‐ GOB;DataSafetyMonitoringBoard‐ DSMB;MelanocyteSignallingExpertGroup‐ MSEG)andthirdpartyauditorsworkaroundtheclockaspartofpharmacovigilancewithSCENESSE®.Thiscomplexsystemiscompulsoryandaimstoprotectsafetyofpatientslongterm.
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Asummaryis provided.
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Intherenaissanceartistsweretryingtocapturelightinshadowson canvas.ClinuvelhopestoprovidelightforEPPpatientswhohaveledalifeonthefringeandindarkness.
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Clinuvel hasdevotedmorethanadecadetoonedruginonedisorder,theresponseofpatientsandphysicianshasmadethisjourneyworthwhile.
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Thankyou fortheinvitation.
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