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ThePediatric PraziquantelConsortiumHelpingchildrenwithSchistosomiasis

December8th,2017

OnbehalfoftheConsortium,Dr.RemcodeVrueh(Lygature)Consortiumcoordinator

GHITFundR&DForum,Tokyo

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Anewpublic-privatepartnershiptoaddressahighunmetmedicalneedinyoungchildren

HighPrevalence

HighMedicalneed

Pre-schoolagedchildrenneglected

Estimated27MioyoungchildreninfectedoratriskofschistosomiasisinSub-SaharanAfrica

Currenttreatment(praziquantel 600mgtablets)cannotbeadministeredtoyoungchildren

à clear unmetmedicalneedforasuitablepediatric formulationforthechildrenaged3months

to6years

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ConsortiumBoard

ConsortiumTeam

Internationalnon-profitR&DConsortiumwithafocusonextendedpartnershipintoendemiccountries

InternationalExpertPanel(WorldHealthOrganization)

asobserver

Continuallyseekingfundingandadvicefromexternalexpertsandpartners

Grants:Bill&MelindaGatesFoundationGHITFund(3x)

Inkindand/orincashcontribution

Exitbegin ofOct 2017

Foundedin2012

ThePediatricPraziquantelConsortium

UniversitéFélixHouphouët-Boigny(Coted’Ivoire)

Tojoin endofDec 2017

KEMRI(Kenya)

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Cesol 600 mgCurrent commercial tablet

PZQ new ODT 150 mg tablet

Trueinnovation

L-PZQandrac-PZQoro-dispersibletablets(ODTs)

• Orallydisintegrating

• Improvedtaste• Conventional

manufacturingprocess

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Completed(2015)

TwoPhaseIBioavailabilitystudies(SouthAfrica)

Completed(2015)TasteStudyofthenewODTsinAfricanchildren(Tanzania)

Ongoing(2016)

PhaseIIPK/PDdosefindingStudy(IvoryCoast)

Planned(2018)

PhaseIIIconfirmatorytrials(Kenya/IvoryCoast)

FullclinicaldevelopmentprogramforPZQ-ODTs

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Capacity building- Strengthening Health infrastructure

11.03.2016,pre-study visit

BEFORE

15.06.2016,initiation visit

AFTERClinical research facility annex refurbished (Ivory Coast - Man region)

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Capacity building- Strengthening R&Dinfrastructure

StudyteamsupervisedbythePI(Prof. N’Goran),membersoftheMerck&SwissTPHteam

BloodsamplingandDryBloodSpotmethod

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Nextsteps:Regulatory&Manufacturingplan

1stregistrationEMAarticle58

andWHOPre-

qualification

Clinicalprogram

WHOEOI

Safetydatabasebuildup-

ConductPharmacovigilancestudywithWHOinvolvement

RegistrationinAfricanendemiccountries(WHOcollaborativeprocedure)*

Manufacturinglaunchreadiness

(Farmanguinhos)

StartProcurementforindividualcase

management

LaunchinfirstAfricanendemiccountries

Largescaledistributionforprophylacticuse

2016-2019 End2019 End2020 End2020

2020

20232019

TechTransfertolocalAfricanmanufacturer

(tbd)

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OnekeyforsuccessCommunication&stakeholderengagement

ConsortiumTeamandBoardrepresentativestogetherwithAfricanregulatoryexperts(Geneva,2014)

AccessexpertmeetinginQ32018Keystakeholders:Consortiumpartners,ourfunders,NTDcontrolprogramfunders,WHO,UNICEF,KOLsandpossiblyendemiccountrydecisionmakers

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http://www.pediatricpraziquantelconsortium.orghttp://www.merckgroup.com/mghi.html