2022 fightmnd drug development grants

2022 FightMND Drug Development Grants

03 Acronyms and Definitions

04 Introduction04 FightMNDcallforProposals

05 Drug Development Grant Outline05 ScopeforDrugDevelopmentGrants06 DrugDevelopmentGrantsCategoriesSupported06 NewTherapeuticDevelopmentGrants06 RepurposingDevelopmentGrants07 AdditionalDetailsforDrugDevelopmentGrants07 Collaboration07 BiotechnologyandPharmaceuticalCompanies07 MNDResearchExperience07 Budget07 PhaseIClinicalTrials-Milestones07 TimelineforFutureDevelopment

08 Funding – Scope and Criteria

10 Reporting

11 Privacy, Conflict of Interest11 PrivacyandConfidentiality11 ConflictofInterest–ReviewersandGRP11 AcknowledgementofSupport

12 Submission Guidelines12 LetterofIntent14 FullApplications14 CoverSheet(seepage18)14 ResearchProposalforProject(8pagesmax.)15 Timeline(1Pagemax.)15 References(2pagesmax.)15 ImpactStatement(1pagemax.)15 TransitionPlan(3pagesmax.)16 Budget(1pagemax.)16 Milestones(1/2pagemax.-PhaseIClinicalTrialsOnly)17 DeclarationofResearchFundingfromOtherSources17 CurriculumVitaeOfAllInvestigators(3pagesmax.perInvestigator)17 CollaborationPlan(2pagesmax.)17 LetterofSupportfromtheAdministeringInstitution(s)(1pagemax.)17 HowtoSubmit18 2022FightMNDDrugDevelopmentGrantCoverSheet

19 Terms and Conditions19 FightMNDDrugDevelopmentGrants

25 Appendix 125 IntellectualPropertyRightsandCommercialActivities

02

Table of Contents

Administering Institution:Organisationresponsibleforadministrationoftheresearchproject,andthereceiptanddistributionofgrantfunds.AgrantcanonlyhaveoneAdministeringOrganisation.

Applicant:Researcherleadingtheresearch–thePrimaryInvestigator(PI),whoisresponsiblefortheoveralldirectionoftheproject,leadingandsupportingco-Investigators(CIs),completionandlodgementoftheapplication,andprogressandreportingontheproject.TheApplicantmustobtainwrittencommitmentfromtheirlaboratoryHeadandHeadsofDepartments/AdministeringInstitution,andmustassumeresponsibilityforundertakingandcompletingtheactivitiesoutlinedintheapplication.Wheretheprojectinvolvesmulti-siteresearch,theApplicantmustobtainwrittencommitmentfromallHeadsofDepartments/AdministeringInstitutionofcollaborativepartnersandco-InvestigatorsnotwithintheAdministeringOrganisation.

Co-Investigator(s): ResponsibleforcarryingoutsomeaspectsoftheresearchundertheguidanceandleadershipoftheApplicant/PrimaryInvestigator.

Collaboration and Collaborator/Collaborative Partner(s):Allpeople,InvestigatorsandOrganisationsinvolvedintheDrugDevelopmentProjectareconsideredtobecollaborators.Collaborationmaybebetweenacombinationofdisciplines,Departmentsand/orOrganisations.ItincludesOrganisationsorindividualsthatprovidespecificresourcesthatcontributetotheresearch.Co-Investigatorsrepresentaspecificsub-groupofcollaboratorswhoaredirectlyinvolvedintheconductoftheprojectbutarenotresponsibleforthedirectionandprogressoftheproject.Byencouragingcollaborativeagreements,FightMNDisaskingresearcherstoconsiderlookingbeyondtheirdiscreteDepartmentsandOrganisations,andtoseekoutpeoplewhomaybedoingsimilarresearch.Thismayallowforstrongerandhigherqualityresearchproposalsandreduceresearchduplication.

Goods and Services Tax:GoodsandServicesTax(GST)imposedinaccordancewiththeA New Tax System (Goods and Services Tax) Act 1999,andrelatedActsandRegulations.GSTwillbepaidontopofgrantamountswhereappropriate.ThiswillbedeterminedbytheAdministeringInstitution’sGSTstatus.

Motor Neurone Disease (MND):ForthepurposesoftheseDrugDevelopmentGrants,thedefinitionofMNDincludesthefollowingprogressiveneurologicaldisordersthatdestroymotorneurons:AmyotrophicLateralSclerosis(ALS);PrimaryLateralSclerosis(PLS);ProgressiveMuscularAtrophy(PMA);ProgressiveBulbarPalsy;andPseudobulbarPalsy.

Translational Research:Researchfacilitatingthetransferortranslationofnewbasicknowledgeofdiseasemechanismsgainedinthelaboratoryintothedevelopmentofnewmethodsforthetreatmentand/orpreventionofMNDinhumans.

Withinthisscope,researchprojectsmayinclude:

•thepreclinicaldevelopmentofnewtreatmentsandinterventionsforMND;or

•testingtheeffectivenessoftreatmentsforMND.

Acronyms and Definitions

03

FightMND Call for ProposalsDrugDevelopmentGrantapplicationsinsupportofpreclinicalresearch,developmentandassessmentoftherapeuticsforMND/ALSthroughto(andincluding)completionofPhaseIclinicaltrials.

APPLICATIONS OPEN: 01 November 2021

LETTER OF INTENT DUE: 24 January 2022, at 18:00 AEDT

SUBMIT LETTER OF INTENT TO: researchgrants@fightmnd.org.au

INVITATION TO SUBMIT FULL APPLICATION:04 February 2022

FULL APPLICATION DUE:01 April 2022, at 18:00 AEDT

RECIPIENTS NOTIFIED:July 2022

FightMNDispleasedtoannounceacallforDrugDevelopmentGrantapplicationstosupportpreclinicaltranslationalresearchthroughtoandincludingPhaseIclinicaltrials.

Grantapplicationswillbeconsideredfor:

•New Therapeutic Development Grants:Supportingpost-discovery,preclinicaldevelopmentoftherapeuticsforMNDthroughsupportofawiderangeofdevelopmentactivitiesrangingfromvalidationoftherapeuticleadstothesubmissionofinvestigationalnewdrugapplicationstoregulatorybodies.

Applications supported by these grants must begin with identified lead compounds in hand.

•Repurposing:Supportinghypothesis-drivendrugrepurposingeffortsfocusedonnewtherapeuticsforMND.

DrugDevelopmentGrantsareeachawardedwithanofferofuptoAUD $1,000,000insupportavailablefor3-yearprojects.(Projectswithaperiodofperformanceoflessthan3-yearswillalsobeconsidered).Supportwillnotnormallyexceed3yearsandapplicantsshouldsubmitproposalsthatarefocusedandcompatiblewitha3-yeartimeperiod.

ThecontinuationofaDrugDevelopmentGrantwithinthisperiodwillbesubjecttoperiodicreviewafterthesubmissionofsatisfactoryprogressreports,whicharerequiredatsix-monthlyintervals.Industrypartnershipapplicationsarestronglyencouragedwithsharedfundingproposals.

Note: it is NOT an Australian Regulatory requirement for drugs to have an IND submission prior to commencement of projects in Australia.

Introduction

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Scope for Drug Development GrantsThescopeforDrugDevelopmentGrantsisasoutlinedbelow:

•Grantssupportthepreclinicalresearch,development,andassessmentoftherapeuticsforMNDthroughto(andincluding)completionofPhaseIclinicaltrials.Theproposedprojectsareexpectedtobeproduct-drivenandfocusedclearlyontherapeutics,anditisanticipatedthattheagentsand/ordatageneratedfromtheseprojectswillleaddirectlytotheadvancementofnewtherapiesforMNDtorolloutintohumanclinicaltrials.

•ThereisapreferenceforDrugDevelopmentprojectsaimedatsporadicMNDwiththeuseofappropriatemodelsofdiseasetojustifysuchapplication.

•Standards for Preclinical Study Design:Allprojectsshouldadheretoacoresetofstandardsforrigorousstudydesignandreportingtomaximisethereproducibilityandtranslationalpotentialofthepreclinicalresearch.AnexampleofsuchstandardsisdescribedinLandis, S.C., et al. (2012), A Call for transparent reporting to optimize the predictive value of preclinical research, Nature 490:187-191.

•Toimprovereproducibilityandpotentialtranslationofanimaldataintonewtreatmentsforpatients:

oPreclinicalanimalstudiesshouldfollowstandardisedprotocolssuchasthoseoutlinedintheLudolph et al. (2010), Guidelines for preclinical animal research in ALS/MND: A consensus meeting, Amyotrophic Lateral Sclerosis, 11:1-2,38-45.

oApplicationsincorporatingadditionalstudiesinhumantissuesand/oriPSClinestostrengthenanimaldataareencouragedandwillbelookeduponfavourably.iPSCorinducedneuronalmodelsshouldclearlydisplayhallmarkMNDpathology.

oProjectsusingmultiple preclinical diseasemodels/strains(e.g.SOD1,cytoplasmicmislocalisedTDP43,C9orf72,etc)tohelpdemonstratepossiblereplicationofeffectoftreatmentand/orimprovedapplicationofresultswillbelookeduponfavourably.

oProjectsincorporatingtheuseofbiomarkerstoassessandconfirmtargetengagementoftheleadcompoundandtohelpestablishpotentialdrugefficacysignalsarestronglyencouraged.

oRiluzoletherapyiswidelyusedwithinthehumanMND/ALSpatientpopulation.Therefore,allpreclinicalefficacy,PK,andtoxicologystudiesofnewcompoundssupportedbytheseDrugDevelopmentGrantsshould include experiments with and without riluzole as an add-on therapy, to assess for any possible drug interactions where appropriate.

oOver98%ofcompoundsintendedfortherapeuticuseintheCNSneverreachthemarketbecauseoftheirinherentinabilitytocrosstheBloodBrainBarrier(BBB).Therefore,itiscriticaltodeterminetheBBBpermeabilityofCNSdrugcandidatesearlyindrugdiscovery,sothatcandidateswithpoorCNSpenetrationcanbeexcludedorstructurallymodified,andpromisingcandidatescanbeacceleratedthroughthedevelopmentprocess.Applications should include details of BBB permeabilityassessmentandpreferablywillinclude2ormoreofthefollowingwhererelevant:

-in vitroBBBpermeabilityassessment;

-in vivoBBBpermeabilityassessment;and

-in silicoBBBpermeabilityassessment.

Drug Development Grant Outline

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Drug Development Grants Categories Supported

New Therapeutic Development Grants

New Therapeutic Development Grantssupportpost-discovery,preclinicaldevelopmentoftherapeuticsforMNDthroughawiderangeofdevelopmentactivities–fromvalidationoftherapeuticleadstothesubmissionofInvestigationalNewDrugapplicationstoregulatorybodies.

Applications supported by these grants must begin with identified lead compounds in hand.

•Applicationsmustincludeanysupportingpreliminarydatarelevanttothephase(s)ofthepreclinicaldevelopmentprocesscoveredbytheproposedresearch.

•Prioritiesforfundingconsiderationincludeprojectsincorporatingthefollowing:

oSecondaryvalidationofleadcompoundsobtainedfromscreeningorbyothermeanstodemonstratetargetselectivityandmechanismofaction;

oOptimisationofpotencyandpharmacologicalproperties;

oStudiesonformulationandstability;and

oIn vitroandin vivoefficacystudies,ADME,BBBpermeabilityassessment,toxicology,pharmacokineticsandpharmacodynamicsstudiesforthedevelopmentofpharmacologicalagentstotheUSInvestigationalNewDrug(IND)andAustralianTGACTNapplicationstage.

•Wherepossible,applicationsshouldincludedetailsforthedesignandimplementationoffull-scalecurrentGoodManufacturingPractice(cGMP)productionoftheleadcompoundandthedeliverysystemsforuseinadvancedpreclinicalandinitialclinicaltrials.

Repurposing Development Grants

RepurposingDevelopmentGrantssupporthypothesis-drivendrugrepurposingeffortsfocusedonnewtherapeuticsforMND.

•ProposalsshouldtestcompoundscurrentlyapprovedforotherindicationsinestablishedanimalmodelsofMND.

•Applicationsincorporatingadditionalstudiesinhumantissuesand/oriPSClinestostrengthenanimaldataareencouraged.iPSCorinducedneuronalmodelsshouldclearlydisplayhallmarkMNDpathology.

•Proposalsshouldbehypothesisdrivenanddrugschosenforrepurposingtestingshouldeither:

otargetamechanismofaction(s)commontobothdiseases;or

otargetanewornovelmechanismofaction(supportedbyappropriatepreliminarydataorevidence).

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Additional Details for Drug Development Grants

Collaboration

Thepreclinicaldrugdevelopmentprocessmayrequireresourcesbeyondthoseavailableatasingleorganisation.Therefore,DrugDevelopmentGrantsareopentoInvestigatorsparticipatinginsynergisticcollaborationsfocusedontestinganddevelopingleadagentsforthetreatmentofMND.Collaborationsshouldbededicatedtoasingle,synergisticpreclinicaldevelopmentprojectorstudyratherthananadditivesetofsubprojects(i.e.thecombinedeffortsofthecollaborationmustprovidegreaterbenefitthanthesumofindividualresearchinitiatives).

Ifacollaborationisproposed,lettersconfirming/supportingthecollaborationarerequired.Ifthecollaborationismulti-organisational,participatingorganisationswillensurethesuccessofthecollaborationbyresolvingpotentialintellectualandmaterialpropertyissuesandbyremovingorganisationalbarriersthatmightinterferewithachievinghighlevelsofcooperation.Detailsontheseprocessesmustbeincludedintheapplication.

Biotechnology and Pharmaceutical Companies

Biotechnologyorpharmaceuticalcompaniesareencouragedtoapply.Whetherabiotechnologyorpharmaceuticalcompanyappliesforthesegrantsasanindividualapplicantoraspartofacollaboration,thecompanyisexpectedtoleverageitsownresourcestocomplementthefundingprovidedbythesegrants.

MND Research Experience

ApplicantswithlimitedMNDresearchexperiencearestronglyencouragedtocollaboratewiththosehavingmoresubstantialexpertiseinMNDresearchand/orMNDmodelsystems.

Budget

Afulldetailedbudgetfortheproposedprojectistobeincludedwithayear-to-yearbreakdown.Thecontributions(ifapplicable)fromotherfundingsourcesshouldalsobeincluded.

Phase I Clinical Trials - Milestones

ApplicationsforaPhaseIclinicaltrialmustincludeProject/StudyMilestonesthatalignwiththeproposedbudget.Milestonescaninclude,butarenotlimitedto:

•HumanResearchEthicsCommittee(HREC)approvalforthestudy;

•GMPmanufacture/stabilitysetup;

•CompletionofGMPstability;

•ClinicalTrialcommencement;

•Recruitmentof10thtrialparticipant;

•Completionofallstudyvisitsanddatabaselock.

•FinalreportreceivedbyFightMND

Note:MeetingmilestonesisanimportantrequirementforPhaseIclinicaltrialssupportedbyFightMNDDrugDevelopmentGrants.Continuityoffundingisdependentonmilestoneachievement,andfuturepaymentinstalmentswillnotbemadeuntiltherelevantmilestonehasbeenachieved.Pleaseconsiderthiswhenplanningyourstudy,milestonesandbudget.

Timeline for Future Development

Whererelevant,applicationsforprojectsmustincludearealistictimelineoffuturedevelopmenttoaPhaseItrial.Projectsdemonstratingatruebench-to-bedsideapplicabilitybetween24–36monthswillbeconsideredfavourably.

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Thescopeandcriteriaforfundingisasoutlinedbelow:

•ThemaximumperiodofgrantfundingforDrugDevelopmentprojectsis3years.

•ThemaximumallowabledirectcostforDrugDevelopmentGrantsis$1,000,000AUD.FightMNDdoesnotprovidefundingforInstitutionalindirect,overheadoroncosts.Salaryoncostsareacceptableifitemised(e.g.superannuation,payrolltax,workcover).

•Morecost-effectivestudiesthatdonotrequestthefullavailablefundingamountareencouraged.

•Theapplicantmayrequesttheentiremaximumfundingamountforaprojectthatmayhaveaperiodofperformancelessthanthemaximumof3yearswithappropriatejustification.

•Timelinesmustbeclearlydescribed(Ganttchartformat)withcleargo/no-gomilestones.

•Paymentstructurewillbebasedeither:

oOnachievementofmilestones,whichwillbefinalisedjointlywiththeInvestigatorsandtheFightMNDResearchTeamiftheprojectisapprovedforfunding.Aminimumof6-monthlyprogressreportswillberequired;or

oInsix(6)-monthlyinstalments.TheinitialsumawardedbyFightMNDwillbeforthefirstsix(6)monthsonly.Continuityoffunding,insix(6)-monthlyinstalments,willdependonthesubmission,bythePrimaryInvestigator,ofsatisfactoryprogressreports(every6months)andanitemisedfinancialreport(every12months),andtheirapprovalbyFightMND.

•Regardlessoftheperiodoffundingproposed,theapplicationmustnotexceedthemaximumallowablecosts.

•ForPhaseIclinicaltrials,continuityoffundingisdependentonmilestoneachievement.Futurepaymentinstalmentswillnotbemadeuntiltherelevantmilestonehasbeenachieved.

•TheresearchprojectcancommenceimmediatelyuponreceiptofasignedGrantAgreement,andmustcommencewithin3monthsoftheagreedcommencementdateoutlinedintheexecutedGrantAgreement.

•Travelcostsofupto$5,000AUDperyeartoattendand/orpresentatscientific/technicalmeetingsareallowedwithinthebudget.

•Investigatorsatallacademiclevelsareeligibletosubmitapplications.

•TheapplicantmustbethePrimaryInvestigator(PI)andhavetheleadroleindirectingtheproject.Inaddition:

oThelevelofcontributionandroleofthePIandotherco-Investigators(CIs)mustbeclearlydefinedontheapplicationcoversheet;

oThePImaybebasedinternationally;

oACVmustbeprovidedforallInvestigators(3pagesmax.foreachInvestigator);and

oApplicationsinvolvingcollaborationswithMNDresearcherswithinAustraliawillbelookeduponfavourably.

•SalariesforthePIorco-Investigatorswillnotbesupported(unlesstheyarewithin7-yearsofbeingawardedtheirPhD).Salaries,wherejustified,willbesupporteduptoalevelequivalenttothe‘PersonnelSupportPackage4’outlinedbytheNHMRCofAustralia.Seehttps://www.nhmrc.gov.au/funding/manage-your-funding/personnel-and-salary-support-packages

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Funding – Scope and Criteria

•Capitalequipment,depreciation,ormaintenanceofequipmentwillnotbefundedbyFightMNDDrugDevelopmentGrants.

•Cashandin-kindco-contributionsfromapplicantswillbeviewedfavourably.

•Anyotheractualorproposedsourcesoffundingtosupporttheprojectmustbedisclosed.

•FightMNDreservestherightofrefusalofanyprojectapplicationsthatitdeemsfalloutsidethesecriteria.

•SuccessfulapplicantsareencouragedtoprovideregularinformationorfeedbackontheirresearchtobecirculatedbyFightMNDforMNDpatientsandcarers.ThePrimaryInvestigator(orco-Investigator(s))is/arerequiredtopresentprogressoftheDrugDevelopmentProjectatresearchsymposiumsannually,andtoFightMNDdonorsandsupportersatFightMNDresearchsymposiums(datesandlocationstobeconfirmed).

•Itisanexpectationofsuccessfulapplicantsthatprojectfindingsaretobepublishedinappropriatepeer-reviewedacademicandprofessionaljournalswithdetailssenttoFightMND.Coststoenableopenaccessforpublicationsmaybeincludedinthebudget.

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Reporting

Fundingrecipientswillberequiredtosubmitreportsonaregularbasis.Thereportingscheduleisoutlinedinthefollowingtable.Fundingrecipientswillberequiredtosubmitreportsonaregularbasis.Thereportingscheduleisoutlinedinthefollowingtable.

REPORT REPORTING FREQUENCY DUE

Progressagainstpre-determined 6-monthly Every6monthsfromreceiptmilestonesand/ortargets* offunds

FinancialReports(tobe Annually Every12monthsfromthereceiptincludedinprogressreport)* offunds

FinalReport OnceOnly Atprojectcompletion,orwithin 12weeksafterprojectcompletion.

MilestoneNotificationfor AsagreedtoinGrantAgreement WhenMilestoneisachievedPhaseIClinicalTrials

Adhocreports* AsrequestedbyFightMND Onrequestwithanegotiabletime framenotgreaterthansixweeks

FightMNDResearch Biennially DuringFightMNDResearchSymposiumPresentation Symposium

*Thesereportswillbeusedtoassesswhethertheprojectisproceedingsatisfactorily,whetherfundsarebeingacquittedinaccordancewiththeoriginalapplicationgoals,andtoascertaintheongoingvalueofFightMNDfunding.

Fundingmaybesuspendedifprogressisconsideredunsatisfactory,oriffundshavenotbeenutilisedinaccordancewiththeDrugDevelopmentGrantAgreement.

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Privacy and ConfidentialityAllinformationcontainedinapplicationsforwardedtoFightMNDwillberegardedasconfidential.DocumentscontainingpersonalinformationwillbehandledandprotectedinaccordancewiththeprovisionsofthePrivacy and Data Protection Act 2014(Vic).Personalinformationwillonlybedisclosedwiththepermissionoftheindividualtowhomitrelates,orwheretheActallows.

Applicantsconsenttotheinformationsuppliedaspartoftheirapplicationbeingdisclosedforthepurposesoftheevaluationandadministrationoftheapplicationandgrant.Suchdisclosureincludesbutisnotlimitedtoindependentreviewers/assessors,theFightMNDGrantReviewPanel(GRP),theFightMNDBoard,andrelevantemployeesofFightMNDinvolvedintheresearchgrantprocess.

ApplicantsacknowledgethatannouncementoffundedDrugDevelopmentGrantapplicationswillinvolveadisseminationofinformationtothepublicabouttheirgeneralnature.

Conflict of Interest – Reviewers and GRPFightMNDrequiresitsindependentreviewersandtheGRPtoactinanethicalmanner,declareconflictsofinterest,andwithdrawfromconsideringapplicationswheresuchconflictdoesormayexist.

Acknowledgement of SupportSuccessfulapplicantsarerequiredtoacknowledgeFightMNDinanypublications,publicannouncements,media,andscientificmeetingpresentationsordiscussionforumspertainingtoresearchconducted.FightMNDmaterials,logos,andimagescanbesuppliedforthispurpose,ifrequired.

Privacy, Conflict of Interest

Letter of Intent

Pleaseusethetemplatebelowasanexampleoftheformatrequiredinsubmissionofyourletterofintent.Lettersofintentshouldbesubmittedwithaminimumsize12font(calibripreferred),one(1)cmmarginsandmustnotexceed3pages.AbriefCVofthePrimaryInvestigator(2pagesmax.)shouldalsobesubmittedwiththeletterofintentforconsideration.

Submission Guidelines

Project Title:

Primary Investigator:

Administering Institution:

Estimated budget:

Duration of project:

Briefly describe any relevant pre-clinical or clinical evidence that support the study rationale:

Project aims:

Project Key Words (4 minimum)

Briefly describe the study design:

Where applicable, list the MND models that will be utilised in the proposal:

Indicate how the aims of this project will help translate therapeutics for the treatment of MND/ALS to the clinic:

Number of subjects to be enrolled (if applicable):

Number of Australian Clinical Sites (if applicable):

Is the lead candidate a new or a re-purposed therapeutic?

Does the application include the use of a biomarker(s)to confirm target engagement of the lead compound? YES NO

Does the application address the blood-brain-barrierpermeability of the lead compound? YES NO

Please provide details of each collaborator involved in the project including:

• Name;

• Affiliations; and

• Specific role relevant to project aims.

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The total budget including indirect costs cannot exceed AUD $1,000,000 (unless additional funding sources are available outside of these grants). FightMND does not provide funding for indirect or overhead costs, or on-costs, of an Administering Institution.

Ifinvitedtosubmitafullapplication,wherethebudgetfortheoverallprojectexceedsthemaximumamountofAUD1,000,000awardedbyFightMND,thefullapplicationwillrequiredisclosureofbudgetdetailsoftheentirestudy,including:theTOTALfundingamountsofarsecuredfortheentirestudy;theapproachbeingmadebytheapplicantstosecuretheadditionalfunding(ifnotsecured);andtheanticipatedtimeframetosecuretheadditionalfunding.

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Full ApplicationsShould you be invited to submit a full application, submissions must include the following:

Cover Sheet (seepage21)

Coversheetswillincludethefollowingdetails.

•PrimaryInvestigatorandco-Investigators.

•A50wordlaysummaryoftheprojectsuitableformediareleaseiftheapplicationissuccessful.

•A250wordlaysummaryoftheprojectsuitableforpublicationontheFightMNDwebsiteandnewsletteriftheapplicationissuccessful.

oProvidebackgroundinformationnecessaryforreaderswithoutscientificormedicaltrainingtounderstandtherationaleandfeasibilityoftheproposedDrugDevelopmentproject.Itshouldalsoclearlydescribethescientificobjectivetheprojectisdesignedtoachieve.

oDescribetheultimateapplicabilityoftheresearch(inlayterms):

i.WhattypeofMNDpatients(e.g.Sporadicvs.Familial)willithelpandhowitwillhelpthem(e.g.symptomcontrolvs.diseaseprogression)?

ii.Whatarethepotentialclinicalapplications,benefits,andrisks?

iii.Whatistheprojectedtimetoachieveapatient-relatedoutcome?

iv.WhatarethelikelycontributionsofthisstudyinadvancingthedevelopmentofatherapyforMND?

•AStatementonthepotentialimpactoftheClinicalTrial(200wordsmax).

Research Proposal for Project (8PagesMax.)

Provideawell-developed,well-integrated,anddetailedresearchplanthatsupportsthetranslationalfeasibilityandpromiseoftheproject,including:

•Aims of the projectstatingclearlyandconciselywhichhypothesesarebeingtested,andtheapplicabilityofresultstothefurtherdevelopmentofapotentialtreatmentforMND;

•Background;

•Preliminary and supporting datarelevanttothephase(s)ofthepreclinicaldevelopmentasrequired;

•Research plan

i.Provideadetailedoutlineoftheresearchforthefullperiodofperformance,includingcleargo/no-gomilestoneswithjustifications.

ii.For animal studies:

-Explainhowandwhytheanimalspecies,strain,andmodel(s)beingusedcanaddressthescientificobjectives,andwhereappropriate,thestudy’srelevancetosporadichumanMNDbiology;

-Summarisetheprocedurestobeconductedanddescribehowthestudywillbecontrolled;

-Tofurthersupporttheadvancementofaparticulartherapeuticcandidate,studiesshouldaimtodemonstratebeneficialeffectsacrossarangeofoutcomes,includingmotororcognitivefunction,neurophysiology,histopathology,andsurvivalwhenusinginvivomodelsofMND;

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Submission Guidelines

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-Describetherandomisationandblindingproceduresforthestudy,andanyothermeasurestobetakentominimisetheeffectsofsubjectivebiasduringanimaltreatmentandassessmentofresults.Providejustificationifrandomisationand/orblindingwillnotbeutilised;

-Provideasamplesizeestimateforeacharmandthemethodbywhichitwasderived,includingpoweranalysiscalculations;and

-Describehowdatawillbehandled–includingrulesforstoppingdatacollection,criteriaforinclusionandexclusionofdata,howoutlierswillbedefinedandhandled,statisticalmethodsfordataanalysis,andidentificationoftheprimaryendpoint(s).

iii.In studies utilising iPSC lines,investigatorsshould:

-Incorporateuniformdifferentiationprotocolstoimprovereproducibility;

-Usecelllinesfromasufficientnumberofparticipantspergroupforstudiescomparingdiseaseandcontrol;

-Usegeneticallymatched(isogenic)mutation-correctedlineswhenapplicable(withrigorousqualitycontrolincludingkaryotyping)tocontrolforvariabilityduetointrinsicgeneticbackgroundofsubjects;

-Addressthelimitationsofandissuesrelatedtotheimmature/foetalnatureofthederivedexperimentaltissueandhow/ifthesewillbeaddressed;and

-EnsurethelinesclearlydisplayhallmarkMNDpathology.

iv.Addresspotentialpitfallsandproblemareaswithinthescopeoftheproposedprojectandpresentalternativemethodsandapproaches.

Timeline (1pagemax.)

IncludeadetailedtimelinefortheprojectinGanttchartformat,thatencompassesthedeliveryofresearchaims,proposedexperimentsandoutcomes.

References (2PagesMax.)

Impact Statement (1PageMax.)

Describe:

•HowtheprojectwillmakeanimportantcontributiontoMNDtherapeuticdevelopment;and

•Ingeneralterms,howtheoutcomesoftheproject,ifsuccessful,willbetranslatedtotheclinicandmadeavailabletoMNDpatients.

Transition Plan (3pagesmax.)

Theapplicant:

•Mustdemonstratethattheyhaveaccesstoallintellectualpropertyrightsnecessaryfordevelopmentandcommercialisation.

•Wherepossible,shoulddescribe/discussthemethodsandstrategiesproposedtomovetheleadcompound(s)intothenextphaseofdevelopment(e.g.clinicaltrials,commercialisation,and/ordeliverytothepatientpopulation)aftersuccessfulcompletionofthisproject.

Thetransitionplanshouldincludecomponentslistedbelow(whererelevant):

•Thedevelopmentand/orcommercialisationstrategy;

•Detailsofthefundingstrategytotransitiontothenextlevelofdevelopmentand/orcommercialisation(e.g.partners,pharma,internal/externalfundingopportunitiestobeappliedfor);

•Ascheduleandmilestonesfortransitioningtothenextphaseofdevelopment,includingaGanttchart;and

•Ariskanalysisforcost,schedule,manufacturability,andsustainabilitymovingforward.

Budget (1pagemax.)

Provideanitemisedbudgetwithjustificationandbreakdownofannualexpenditure.

WheretheapplicationistosupportabudgetthatexceedstheFightMNDgrant,applicantsshouldincludebudgetdetailsoftheentirestudy,including:

•Theoverallbudgetandcostofthestudy;

•Thecomponentofthestudythatthisapplicationwillfund;

•WhethertheTOTALamountoffundingrequiredfortheentirestudyhasbeensecured,andtheTOTALfundingamountsofarsecuredforthestudy;

•IftheTOTALamountoffundingrequiredforthestudyhasnotbeensecured(otherthantheamountrequestedinthisapplication),indicatethe:

-Approachthatisbeingmadebytheapplicantstosecuretheadditionalfunding;and

-Anticipatedtimeframetosecuretheadditionalfunding.

Budgetitemscaninclude:

•Salariesforteammembers.Salaries,wherejustified,willbesupporteduptoalevelequivalenttothe‘PersonnelSupportPackage4’outlinedbytheNHMRCofAustralia.Seehttps://www.nhmrc.gov.au/funding/manage-your-funding/personnel-and-salary-support-packages.;

•Salaryoncostsifitemised(e.g.superannuation,payrolltax,workcover);

•Directresearchcosts(e.g.reagentsandconsumablesetc.);

•TravelforattendanceatrelevantALS/MNDscientificmeetings,topresentthefindingsoftheprojecteachyearforthedurationofthegrant(max.$5,000peryear);and

•Coststoenableopenaccessforpublicationswithin6monthsofpublicationcanbeincludedintheprojectbudget(butnootherpublicationcosts).

Budgetitemscannotinclude:

•SalariesforPrimaryInvestigatorsorcoInvestigators(unlesstheyareanearly-careerresearcherwithin7yearsofPhDbeingawarded);

•Equipmentitems;

•Computers;and

•Indirect,overheadoroncostsoftheInstitution.

Milestones (1/2pagemax.-PhaseIClinicalTrialsOnly)

PhaseIclinicaltrialapplicationsmustincludeProject/StudyMilestonesthatalignwiththeproposedbudget(seeGrantOutlinesectiononpage7).

Milestonescaninclude,butarenotlimitedto:

•HumanResearchEthicsCommittee(HREC)approvalforthestudy;

•Atenrolmentofthefirststudyparticipant;

•Afterenrolmentof25%ofstudyparticipants;

•Afterenrolmentof50%ofstudyparticipants;

•Atcompletionofallstudyvisitsanddatabaselock.

•FinalreportreceivedbyFightMND.

Note:ContinuityoffundingforPhaseIclinicaltrialsisdependentonmilestoneachievement,andfuturepaymentinstalmentswillnotbemadeuntiltherelevantmilestonehasbeenachieved.Pleaseconsiderthiswhenplanningyourstudy,milestonesandbudget.

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Declaration of Research Funding from Other Sources

Declarationanddetailsofresearchfundingfromothersources(actualorproposed)thatrelatetotheDrugDevelopmentGrant’shypothesis,aimsandresearchplanmustbestatedandinclude:

•Investigator,andTitleofotherApplication;

•FundingSource/OrganisationandApplicationID;

•RoleofInvestigatoronotherApplication;

•Durationofotherfunding;and

•Totalamountrequested.

Curriculum Vitae of all Investigators (3pagesmax.perInvestigator)

Include:

•Academicbackground;

•Presentandpastemploymentpositions;

•AwardsandPrizes;

•Researchgrantssupport(past5years);and

•Peerreviewedpublications(donotincludepublications“inpreparation”or“underreview”).PleaseprovideaDOInumberforpapersrecentlyaccepted.

Collaboration Plan (2pagesmax.)

•Name(s)oftheDepartment(s)andInstitution(s)wheretheDrugDevelopmentprojectwillbecarriedout,andnominationofthePrimaryDepartment/Institution.

•SuitabilityoftheDepartmentandInstitutionforsupportingtheDrugDevelopmentGrantandoutlinedprojectsshouldbeclearlyoutlined.

•Describethespecificrole(s)ofeachcollaboratorintheproposedprojectandevidencethattheyareequippedtofulfiltherole.

•Iftheprojectinvolvesamulti-organisationalcollaboration,participatingorganisationswillensurethesuccessofthecollaborationbyresolvingpotentialintellectualandmaterialpropertyissuesandbyremovingorganisationalbarriersthatmightinterferewithachievinghighlevelsofcooperation.Detailsontheseprocessesshouldbeincludedintheapplication,includingalldetailsofintellectualpropertyownership,andadescriptionofanyappropriateintellectualandmaterialpropertyplansamongstcollaboratingorganisations.

Letter of Support from the Administering Institution(s)(1pagemax.)

IncludealetterofsupportfromtheAdministering/SponsoringInstitutionstatingtheirsuitabilityandroleinadvancingtheDrugDevelopmentProject’sprogressandsuccess,andconfirmingthatappropriateinfrastructure,equipment,consumablesandlaboratoryspacewillbeprovidedforthedurationoftheproject.

How to Submit

TheapplicationdeadlinesaredescribedintheIntroductionsectionofthisguideline.

•Allapplicantsareaskedtosubmittheirapplicationselectronically(filesizenottoexceed5MB)asaPDF(minimumsize12font–calibripreferred,minimum1cmmargins).

•DrugDevelopmentGrantapplicationsshouldbesubmittedtoFightMNDbyemailtoDrDavorStanic,atresearchgrants@fightmnd.org.au.

2022 FightMND Drug Development Grant Cover Sheet

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1. Project Title

2. Title, name and qualifications of Primary Investigator (PI)

3. Key Words (4 minimum)

4. Email address of PI

5. Mobile phone no. of PI

6a. Name, institution, % contribution to project and role of PI

6b. Name, institution, % contribution to project and role of co-investigator

6c. Name, institution, % contribution to project and role of co-investigator (add rows if more than 2 co-investigators)

7. Administering Organisation / Sponsoring institution administering the grant

8. Name of research grant administrator for (7)

9. Contact details for (8)

10. Total Budget Estimates for each year Year1$ Year2$ Year3$ TOTAL-$

11. A 50 word max. lay summary of the project suitable for media release if the application is successful.

12. Summary/Lay description (250 words max. – use separate page if required).

Describeintermsandlanguageapplicable tothegeneralpublic,theoverallaimsand expectedoutcomesofthisproject.

13. Statement on the potential impact of the project (200 words max. – use separate page if required).

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Terms and Conditions

FightMND Drug Development Grants

Terms and Conditions

AllcommunicationconcerningDrugDevelopmentGrantapplicationsandadministrationshouldbeaddressedtoDrDavorStanicatFightMND,byemailtoresearchgrants@fightmnd.org.au.

1. Funding Arrangements

1.1.FightMNDDrugDevelopmentGrantsaretime-limited,andapplicantsshouldensurethatproperconsiderationisgiventothisintheproposal.Whentheprojectisapprovedinprinciple,theinitialsumawardedbyFightMNDwillbeforthefirstsix(6)monthsonly.Approvaloffundingforsubsequentinvoicesatsix(6)monthlyintervalswillbesubjecttoavailabilityoffundsandthereceipt,fromthegranteeand/orAdministeringInstitution,ofsatisfactoryprojectprogressandfinancialreports,andachievementofagreedmilestonesiftheDrugDevelopmentGrantawardedisforaPhaseIclinicaltrial.MembersoftheFightMNDteamandBoardwillreviewprogressreportstodecideoutcomes.

1.2.Iftheapplicantunder-spendsinanyyear,FightMNDcan,atitsdiscretion,giveapprovalforthebalancetobecarriedintothefollowingyear.ExpenditurebeyondtheenddatewillonlybepermittedifauthorisedbyFightMNDinadvance.RequestsmustbemadebycontactingFightMNDatleasteightweekspriortotheoriginalcompletiondate,byemailtoDrDavorStanicatresearchgrants@fightmnd.org.au.

1.3.ThevalueoftheDrugDevelopmentGrantisuptoatotalof$1,000,000AUDforprojectperformanceofuptothree(3)years.

1.4.FightMNDwillnotmeetindirectoroverheadcostsoron-costsoftheAdministeringInstitution,suchas:generaltravel,financeservices,stafffacilities,staffdevelopment,publicrelations,institutionallibraries,routinesecretarialwork,personnelservices,stationeryorcontributionstogeneraldepartmentalcosts,andpublicationcosts(exceptforthosenecessarytoenableopenaccessforpublications).

1.5.Conferenceattendance:FightMNDwillallowupto$5,000perannumtowardsthecostofrelevantconferenceattendanceandparticipationbythePrimaryInvestigatortobedrawnfromthetotalsumawarded.Thismaybeusedduringthelifeoftheprojecttowardsthecostsofregistrationfeesandtravel,butnottocoverseparatehotelaccommodationorothersubsistencecosts.Invoices,receiptsorotherevidenceofspendingmustbeprovided.Theinvestigatorsareencouragedtopresenttheirwork.ThePrimaryInvestigatorisexpectedtoattendatleastonerelevantmeetingperyear.

1.6.Paymentofinstalmentsisconditionalonreceiptandapprovalofsatisfactoryprojectprogressandfinancialreports,andachievementofagreedmilestonesiftheDrugDevelopmentGrantawardedisforaPhaseIclinicaltrial(seecondition1.1and15.1).

1.7.Fundingfromothersources:financialsupportforclearlydefinedaspectsofaprojectfromseparatefundingsourcesispermittedunderFightMNDgrants.Suchsupplementaryfundingmustbedisclosedatthetimeofthegrantapplicationoratthetimesuchfundingisreceived.

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2. Equipment

2.1.FightMNDwillnotfundanyequipmentpurchasedaspartofaDrugDevelopmentGrant.

3. Ethical Considerations

3.1.ItistheresponsibilityoftheApplicantstohaveethicalcommitteeapprovalforallorpartoftheplannedresearch.Thisshouldideallybeinplaceatthetimeofapplyingforfunding.

3.2.Approvalsmustbereceived,andcopiesprovidedtoFightMNDuponrequest,priortotheDrugDevelopmentGrantcommencing.

4. Personal Direction of the Project

4.1.ItisexpectedthatthePrimaryInvestigatorwillbeactivelyengagedindirectingtheproject.ContinueduseofFightMNDfundsduringaprolongedabsenceofthePrimaryInvestigatorrequireswrittenagreementtocontinuetheresearchunderthedirectionofanotherqualifiedInvestigator,ideallyobtainedpriortotheabsence.ThegranteeoranapprovedrepresentativeoftheAdministeringInstitutionmustapplytoandnotifyFightMNDbyemailtoDrDavorStanicatresearchgrants@fightmnd.org.au,withanexplanationofthesituation,providingdetailsofthearrangementsforconductingtheresearchduringtheirabsence(seeTermsandConditions11.2).

5. Recruitment and Employment of Staff

5.1.FightMNDdoesnotactasanemployerand,therefore,inallcaseswherefinancialsupportisprovidedfortheemploymentofstaff,theAdministeringInstitutionundertakestoissueacontractofemploymentinaccordancewithanyotherrelevantActrelatingtotheconditionsofemployment.

5.2.FightMNDwillnotberesponsibleforclaimsunderstatuteoratcommonlaw,norwilltheyindemnifytheAdministeringInstitutionagainstaclaimforcompensationoragainstanyclaimsforwhichtheInstitutionmaybeliableasanemployerorotherwise.

6. Staff Management Responsibility

TheAdministeringInstitutionmustacceptfullresponsibilityfor:

6.1.Themanagement,monitoringandcontrolforallstaff(permanent,temporaryandstudents)employedorinvolvedinanyresearchfundedbyaFightMNDgrant;

6.2.Themanagement,monitoringandcontrolofallresearchworkfundedasaresultofaFightMNDgrant.

7. Termination Of Employment

7.1.IfthetenureoftheappointmentofstaffrecruitedtoworkontheFightMND-supportedprojectcontinuesbeyondthedefinedperiodoftheGrant,theAdministeringInstitutionwillbesolelyresponsibleforallcostsbeyondtheperiodoftheGrant.FightMNDacceptsnoliabilityforcontractsandcostsextendingbeyondthedefinedgrantperiod.

8. Employment Term Contracts

8.1.WheremembersofstaffhavebeenundercontracttotheAdministeringInstitutionpriortotheactivationoftheFightMNDDrugDevelopmentGrant,FightMNDwillnotreimbursecostsattributedtoanypriorcommitment.Thisincludesanyredundancypaymentsdueforservicepriortothegrantperiod.

8.2.ThecontractofemploymentofferedmustnotextendbeyondtheterminationoftheDrugDevelopmentGrant(unlesstheAdministeringInstitutionwishestoextendthecontractatitsownexpense).

9. Maternity and Other Long-Term Leave

9.1.TheAdministeringInstitutionwillmeetthecostofanylong-termleave,otherthanholiday,andwillensurethatallannualleaveentitlementistakenwithintheDrugDevelopmentGrantperiod.Long-termleavemayincludematernity,paternityorlong-termsickleave.

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9.2.MaternityorpaternityleaveistheresponsibilityoftheAdministeringInstitutionemployingstaffundertakingaFightMNDproject.LeavewillbeprovidedaccordingtotheAdministeringInstitution’slocaltermsandconditionsofemployment.ThecostsofsuchleavearetheresponsibilityoftheAdministeringInstitutionandarenotprovidedforbyFightMND.

9.3.IfaFightMNDfundedemployeeisduetotakeanyplannedlong-termleave,theDrugDevelopmentGrantPrimaryInvestigatorshouldinformFightMNDofthedatesinadvance.ThiswillenablediscussiontodecidewhethertheGrantshouldbesuspendedfortheperiodofabsenceuntilfull-timeemploymentcanberesumed(seeTermsandConditions4and11.2).Ifunplannedlong-termleaveoccurs,theDrugDevelopmentGrantPrimaryInvestigatororanapprovedrepresentativeoftheAdministeringInstitutionshouldcontactFightMNDbyemailtoresearchgrants@fightmnd.org.auassoonaspossibletodiscussthesituation.

10. Activation of an Awarded Drug Development Grant

10.1.DrugDevelopmentGrantsareactivatedonreceiptofasignedGrantAgreementandreceiptofthefirstinvoice.If,foranyreason,thestartdateoftheprojectisdelayedaftertheGrantAgreementhasbeenreturned,FightMNDmustbeinformedatonce,aGrantAgreementDeedofVariationformcompleted,andanewstartdateagreed(seeTermsandConditions11.2).Ifnecessary,arevisedGrantAgreementwillneedtobecompletedandreturned.

10.2.Iftheprojectdoesnotstartwithinthree(3)monthsoftheoriginalagreedstartdate,FightMNDmaywithdrawtheDrugDevelopmentGrantoffer.Thegranteeand/orAdministeringInstitutionwillhavetoreapplyforfundinginafuturegrantround,incompetitionwithotherapplicantsatthetime.

10.3.EthicalApproval:FightMNDmustreceiveevidencethatethicalapproval(ifrequired)isinplacepriortotheprojectstarting.Paymentofinvoiceswillbedelayeduntilevidencehasbeenprovided.ItistheresponsibilityofthePrimaryInvestigatortohaveethicalapprovalfortheproposedresearchandthisshouldideallybeinplaceatthetimeofapplyingforfunding.

11. Change of Terms of an Awarded Drug Development Grant

11.1.Reallocationoffundsfromoneexpenseheadingoftheapprovedbudgettoanother,asdetailedintheGrantAgreement,requireswrittenpermissionfromFightMND.

11.2.GranteeswillberequiredtosubmitalettertoFightMNDdetailinganyandallproposedchangestotheprojectandcompleteaGrantAgreementDeedofVariation.Letters/DeedsofVariationmustbesubmittedatleasteightweekspriortothechangestakingplaceandsubmittedforapprovaltoFightMNDbyemailtoDrDavorStanicatresearchgrants@fightmnd.org.au.FightMNDmustbekeptinformedatalltimesofanychangestotheoriginalgrantfundedandtheGrantAgreement.

11.3.AnyrequestformajorchangesinthetermsoftheDrugDevelopmentGrant,e.g.foradditionalstafforbudgetitems,mustbemadeintheformofanewandseparategrantapplication,whichwillbeconsideredincompetitionwithallothernewapplications.

12. Changes to Conditions of an Awarded Drug Development Grant

12.1.FightMNDreservestherighttochangetheTermsandConditionsofDrugDevelopmentGrantsatanytime.IfthisoccursduringthelifetimeofaDrugDevelopmentGrant,therevisedTermsandConditionsmaybeappliedinplaceofthoseissuedatthecommencementoftheGrant.

12.2.Successfulapplicantswillbegivenatleast8weeks’noticeofanychangetoconditionsofthegrant.

13. Early Termination of an Awarded Drug Development Grant

13.1.FightMNDreservestherighttoterminateanawardedDrugDevelopmentGrantatanytime.Circumstanceswhichmightleadtoterminationinclude:

•AnybreachintheTermsandConditionsunderwhichtheGrantwasawarded;

•Iftheprojecthasnotstartedwithinthreemonthsoftheagreedstartdate;

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•Theworkisdivergingmarkedlyfromtheoriginalapprovedproject.TheDrugDevelopmentGrantPrimaryInvestigatororanapprovedrepresentativeoftheAdministeringInstitutionmustinformFightMNDimmediatelywhentheyareawareofachangeofdirection(seeTermsandConditions11.2).Theremay,however,becircumstancesinwhichthechangeisacceptableonscientificgrounds;

•Failuretosubmitadequateprogressreports,orseriousandunresolvableproblemsidentifiedbyasitevisit;and/or

•WorkhasceasedontheGrant,orthePrimaryInvestigatorhasceasedtobeactivelyinvolvedintheproject.FightMNDmustbeinformedimmediatelyifthissituationarises(seeTermsandConditions11.2).

FightMNDwillendeavourtogive60dayspriornoticebeforeterminationofanawardedDrugDevelopmentGrant.

13.2.IfaDrugDevelopmentGrantisterminated,FightMNDwillmeetcostsproperlyandnecessarilyincurredundertheGrantAgreementuptotheterminationdate.However,paymentswillnot,inaggregate,exceedtheamountoftheDrugDevelopmentGrantremainingtobepaidatthetimeofitstermination.

13.3.IntheeventofworkbeingdiscontinuedbytheAdministeringInstitution,writtennotificationmustbesenttoFightMND,togetherwithareportontheworkcarriedouttodate,settingoutreasonsforthetermination.

14. Extension to an Awarded Drug Development Grant

14.1.ItistheresponsibilityofthePrimaryInvestigatortoapplyforfurthersupportbeforetheendoftheDrugDevelopmentGrantperiod,ifthisisrequired.Applicationsforanextensionofsupportmaybeconsideredinisolationorasanewapplicationincompetitionwithotherapplicationsatthetimeofapplying(seeTermsandConditions11.2).

14.2.Adequatetime(atleasteightweeks),shouldbeallowedforanapplicationtobeprocessedandFightMNDacceptsnoresponsibilityforanycostsincurredduetothefailureofagranteetomakesuchanapplicationingoodtime.

15. Reports

15.1.ThePrimaryInvestigatorisrequiredtosubmitthefollowingreports:

•Annual progress reports:dueevery12monthsfromtheDrugDevelopmentGrantstartdate,asstatedontheexecutedDrugDevelopmentGrantAgreement(seeTermsandConditions1.1).AshortsummaryinlanguageintelligibletothelayreadershouldalsobesubmittedforpossibleuseinFightMNDpublicationsandonourwebsite;

•Interim reports:briefsix-monthlyreportsofnomorethanthreepagesonprojectprogress;

•Final report:requiredwithintwelveweeksaftercompletionoftheDrugDevelopmentGrantproject.Adetailedfinalreportcoveringthewholeprojectwillbesubstitutedfortheannualreport.Inaddition,asummaryshouldalsobeprovidedinlanguageintelligibletothelayreader.Researchersmustavoidtheuseofjargonandtechnicallanguageandshouldpitchthesummaryatthelevelofasciencefeatureinabroadsheetnewspaper.ThesummarymaybeusedinFightMNDpublications;and

•FightMND Research Symposium:thePrimaryInvestigatorisrequiredtopresentprogressoftheDrugDevelopmentprojectannually,andattheFightMNDResearchSymposium.

•Notification of Milestone achievement(PhaseIClinicalTrialGrantsonly):FightMNDshouldbenotifiedandsupportingdocumentsprovidedwhentheClinicalTrialhasachievedagreedmilestones,byemailtoDrDavorStanicatresearchgrants@fightmnd.org.au.

15.2.InstalmentsfortheGrantwillbepaidonlyafterreceiptofprogressreportsandtheirapprovalbyFightMND.Paymentmaybedelayedifreportsarenotsubmittedontimeand/orifclarificationisrequired.ForClinicalTrials,instalmentsoftheGrantwillbepaidonlyafterreceiptofrelevantandagreedmilestoneachievement.

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15.3.FeedbacktopeoplelivingwithMNDand/orCarers.AllgranteesareencouragedtoprovideregularinformationontheirresearchtobecirculatedbyFightMNDforpatientsandcarers.Wherevolunteersareinvolvedinresearch,granteesarerequiredtoprovideregularfeedbacktotheparticipantsandFightMND,inadditiontoannualreportsandpublications.

16. Site Visits and Progress Meetings

16.1.FightMNDreservestherighttovisitthegrantee’slaboratoryduringtheperiodoftheDrugDevelopmentGranttodiscussprojectprogress,andwelcomesinvitationstodoso.

16.2.GranteesmaybeaskedtoattendsixmonthlyprogressmeetingstodiscussprogresswithFightMNDrepresentativesanddonors.Thesemaybearrangedinconjunctionwithsitevisits.

16.3.GranteesmaybeaskedtotakepartinFightMNDcommunicationprojectssuchasvideocontenttohelpfacilitatefeedbacktoFightMND’sdonorsonoutcomesrelatedtotheDrugDevelopmentGrant.

17. Publications, Presentations, Acknowledgments and Publicity

17.1.GranteesareexpectedtoseekpublicationoffindingsinrefereedjournalsduringandassoonaspossibleduringandafterconclusionoftheDrugDevelopmentGrantproject(subjecttoTermandCondition18).FightMNDandthegranteeand/orAdministeringInstitutionjointlyundertaketonotifyeachotherbeforepublishedreferenceismadetothefindingsoftheproject,andtodiscussandreachagreementontheformofpublicationwhereverpossible.

17.2.Granteesand/ortheAdministeringInstitutionmustinformFightMNDimmediatelywhenresultsfromFightMND-fundedresearchareacceptedforpublicationorpresentation.Thegranteeand/orAdministeringInstitutionmustprovideFightMNDwithreprints,photocopiesorelectroniccopiesofthefinalversionofanysuchpublications.

17.3.Open Access Policy:Granteesaremandatedtomaketheirpeer-reviewedpapers,directlyarisingfromtheDrugDevelopmentGrant,availablethroughopenaccess.TheseresearchpapersshouldbeavailablewithinthePubMedCentralrepositoryassoonaspossible,butdefinitelywithinsixmonthsofpublication.Coststoenableopenaccessforpublicationscanbeincludedintheprojectbudget.

17.4.Posters – costs and accessibility:IfFightMND-fundedresearchisacceptedforpresentationasaposter,thecostsofposterproductionmaybeclaimedaspartoftheconsumablesbudget(toamaximumof$250perDrugDevelopmentGrant).ThepostermustacknowledgeFightMNDasasourceoffundingandshouldincludeFightMND’slogo.FightMNDshouldbeprovidedwithanelectroniccopyoftheposterforuseonourwebsiteandsocialmedia.

17.5.Toensurethelong-termsustainabilityofincomeforresearchandtoreflectandmaintainourreputationforfundingresearchofthehighestscientificexcellenceandofgreatestrelevancetoMND,allopportunitiestopromoteFightMNDmustbepursued.TheDrugDevelopmentgranteeandtheAdministeringInstitutionareobligedtoco-operatewithFightMNDoveranypublicityorfundraisingactivityarisingfromresearchfundedbyFightMND.Whereitisthemainfunderoftheresearch,FightMNDreservestherighttoleadonpublicity.

17.6.Granteesand/ortheAdministeringInstitutionmustnotifytheFightMNDresearchteambyemailtoDrBecSheeanatresearch@fightmnd.org.auandDrDavorStanicatresearchgrants@fightmnd.org.au,andtheMarketingandCommunicationsDirectorAntheaHargreavesatanthea@fightmnd.org.au,atleastfiveworkingdaysinadvanceofanypublicityarisingfromresearchwhollyorco-fundedbyaFightMNDDrugDevelopmentGrant.FightMNDmustbegivenatleast24hours’noticeofanymediareleaseinconnectionwiththefundedproject.AnypressreleaseorothermaterialincludingreferencetoFightMND-fundedresearchmustbeapprovedbyourteambeforeitisreleasedtothemedia.

17.7.InanyoralorwrittenreportorposterpresentationrelatingtoFightMND-fundedresearch,thegranteeand/orauthormustacknowledgeFightMND’ssupportanddisplaytheFightMNDlogowherepractical.AllreferencestoFightMND-fundedworkplacedonwebsites,electronicbulletinboardsandsimilarplatformsmuststateclearlythattheworkisfundedby“FightMND”andideallyalinkshouldbeincludedtotheFightMNDwebsite:www.fightmnd.org.au.

17.8.GranteesmustensurethatFightMND’ssupportisacknowledgedinallpublications,presentationsandsimilarcommunications.ItisessentialforDrugDevelopmentgranteestoacknowledgethattheirresearchhasbeensupportedwhollyorinpartbyFightMND,eitherinthetextorinafootnote.TheDrugDevelopmentGrantreference/IDmustalsobeprovided.

17.9.WhenspeakingpubliclyandtorepresentativesofthemediaaboutFightMND-fundedresearch,granteesandresearchersshouldensuretheymakeitcleartothemediaandothersthattheyshouldbepresentedasa“FightMND-fundedscientist”.ResearchersshouldconsultwithFightMND’sResearchDirector,DrBecSheean,atresearch@fightmnd.org.auandMarketingandCommunicationsDirectorAntheaHargreavesatanthea@fightmnd.org.au,beforespeakingtothemedia.

17.10.Thereisasubtlebutimportantdifferencebetweenspeakingasa“FightMND-fundedscientist”andactingasaspokespersonforFightMND.Representativesofthemediamaynotalwaysbeawareofthisdifference.GranteesandResearcherswhospeaktothemediamustensurethattheirpersonalviewsarenotmisrepresentedasbeingattributabletoFightMND.

18. Patents, Copyright and Other Intellectual Property

18.1.Ifideas,processesorproductsofpotentialcommercialvaluearegeneratedasaresultoftheproject,theGranteeand/orAdministeringInstitutionmustobtainthewrittenconsentofFightMNDbeforetakinganystepstoexploittheresultscommercially.TheGranteeandAdministeringInstitutionacceptsthatFightMNDmayrequireashareoffinancialgaininreturnforitsconsent.ThisrestrictionshallcontinuetobindthepartiesnotwithstandinganyterminationoftheDrugDevelopmentGrant.Forfurtherdetail,pleaseseeAppendix1-IntellectualPropertyrightsandcommercialactivities.

19. FightMND Meetings and Events

19.1.GranteesareaskedtomakethemselvesorotherappropriateresearchteammembersavailabletoreportontheDrugDevelopmentGrantprojectatFightMNDmeetings,fundraisingeventsandoccasionallyatothertimesbyinvitation.

19.2.TheremaybeoccasionswherethegranteeorotherappropriateresearchteammemberswillbeaskedtopresenttheirworkrelatingtotheDrugDevelopmentGrantprojectatscientificand/orhealthcareprofessionals’meetings.

19.3.WhenspeakingandpresentingatFightMNDevents,granteesorotherappropriateresearchteammembersareexpectedtomakeitclearinthepresentationtheirfundingconnectionwithFightMND.

20. FightMND Case Studies

20.1.GranteesareaskedtomakethemselvesavailableascasestudiesreflectingtheworkofFightMNDforitswide-rangingcommunicationsandfundraisingactivities.

21. Scientific Integrity

21.1.Intherareeventofscientificfraudoccurring,FightMNDwishestomakeitclearthatitistheresponsibilityoftheemployingauthoritytoinvestigateanysuspectedcaseoffraudulentactivity.FightMNDagreestoprovidefundingprovidingtheemployingauthoritycanproduceevidenceofaprocedurefordealingwithscientificfraud.IffraudshouldbeproventheDrugDevelopmentGrantmustberepaidinfulltoFightMNDforthwith.

22. Indemnity

22.1FightMNDdoesnotprovidecoverfornegligentornon-negligentharmforparticipantsinFightMND-fundedstudies.TheAdministeringInstitutionshouldensurethatlocalarrangementsareinplaceshouldclaimsarise.

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Appendix 1

Intellectual Property Rights and Commercial ActivitiesAsacharity,FightMNDisobligedtoensurethattheoutcomesofitsfundedresearchareappliedforthepublicbenefit.Insomecircumstances,thisobligationmaybebestachievedthroughtheprotectionofIntellectualPropertyresultingfromtheresearchandthefacilitationofcommercialexploitationofthisIntellectualProperty.

Theterm‘IntellectualProperty’(IP)describesanyworkorinventionthatresultsfromoriginalcreativethought.

IPfallsintodifferentcategories:

•Copyright:protectswritten,dramaticandartisticwork,software,films,soundrecordingsandbroadcasts.

•Patents:protectstechnicalinventions,novelproductsorprocesses.

•Trademarks:distinguishthegoodsandservicesofoneorganisationfromanother.

•Designrights:protectsthevisualappearanceofproducts.

Someoftheseprotectionsneedtoberegistered(trademarks,patents)andsomedonot(copyright,designrights).IftheIPisnotprotected,anotherindividualororganisationmaycopythedesignorcommercialiseandselltheinventionwithoutconsentorpayment.

Therefore,forgrantswhereFightMNDfundingmayleadtothegenerationofIntellectualProperty,thefollowingadditionalconditionsshallapply:

1.1.AnyIntellectualPropertydevelopedduringthecourseofconductingresearchsupportedbyFightMNDDrugDevelopmentGrantsunderthisagreement(ProjectIP)shallbeownedbytheAdministeringInstitution.

1.2.TheAdministeringInstitutionmustcomplywiththeNationalPrinciplesofIntellectualPropertyManagementforPubliclyFundedResearchbyhavinginplacestrategies,policies,andproceduresfortheidentification,protection,management,andexploitationofIntellectualProperty,includingthatresultingfromfundingbycharitiessuchasFightMND.http://www.arc.gov.au/national-principles-intellectual-property-management-publicly-funded-research

1.3.TheAdministeringInstitutionshouldensurethatallpersonsinreceiptoffundingfromFightMND,orworkingonfundedactivity(includingemployees,students,visitingstaffandsub-contractors),areemployedorretainedontermsthatvestintheInstitutionallIntellectualPropertyarisingfromfundingbyFightMND.

1.4.TheAdministeringInstitution,grantholdersandco-InvestigatorsshouldinformFightMNDofanypre-existingarrangementsofwhichtheyareaware,andwhichcouldleadtoabreachofFightMND-fundedstandardconditions.TheInstitutionshouldtakereasonableendeavourstoensurethatnoconsultancies,thirdpartyrestrictionsorarrangementswhichmightimpactonaFightMND-fundedgrantareenteredintoinrelationtoanyFightMND-fundedpersonoractivitywithoutprioragreementofFightMND.FightMND-fundedInvestigatorsorindividualsinvolvedinaFightMND-fundedprojectshouldnotusematerialsorcompounds(otherthanthoseobtainedcommercially),ontermswhichwouldplacerestrictionsonthepublicationoftheresults.Institutionsshouldtakereasonableendeavourstoensurethat“reach-throughclaims”havenotbeengrantedonanyFightMND-fundedIPinfavourofcommercialorganisationsprovidingmaterialsorcompoundstoFightMND-fundedindividualsforresearchpurposes.However,FightMND

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I.AllofthecostsassociatedwithcommercialisingoftheProjectIP(includingpatentandlegalcosts)willbepaidbytheAdministeringInstitution.OutofanynetproceedsreceivedbytheAdministeringInstitutionfromcommercialisingtheProjectIP(afteralloftheAdministeringInstitution’scostsassociatedwithcommercialisingtheProjectIPhavefirstbeendeducted),theAdministeringInstitutionwillpay10%ofallnetcommercialisationproceedstoFightMNDuntilsuchtimeasFightMNDhasreceivedanamountequaltotheamountoftheDrugDevelopmentGrantfundingprovidedunderthisagreementmultipliedbyfive(5).

II.Tenpercent(10%)ofthecostsassociatedwithcommercialisingtheProjectIP(includingpatentandlegalcosts)willbepaidbyFightMNDasandwhenthecostsfalldue,andtheremaining90%ofthecommercialisationcostswillbepaidbytheAdministeringInstitution.OutofanynetproceedsreceivedbytheAdministeringInstitutionfromcommercialisingtheProjectIP(afteralltheAdministeringInstitution’scostsassociatedwithcommercialisingtheProjectIPhavefirstbeendeductedandFightMND’scostshavebeenreimbursed),theAdministeringInstitutionwillpay10%ofallnetcommercialisationproceedstoFightMNDinperpetuity.

III.FightMNDwillnotseekanypaymentfromthenetcommercialisationproceedsarisingfromcommercialisationoftheProjectIP.

1.8.IftheAdministeringInstitutiondoesnotwishtoprotect,manageorexploittheIP,orfailstocomplywiththeagreedstrategy,FightMNDmaydirecttheAdministeringInstitutiontotakestepstoprotecttheIPattheAdministeringInstitution’sexpenseortotransfertheIPtoFightMND.

1.9.IftheAdministeringInstitutionwishestouseanythirdparty(otherthanitsrecognisedtechnologytransfercompany)tocarryoutitsobligationswithrespecttoIP,itmustprovidedetailsto,andobtainpriorwrittenapprovalfrom,FightMND.

recognisesthatcompaniesprovidingmaterialsmayoftenrequireexclusiverightstoanyIntellectualPropertyarisingfromuseofthatmaterial,andthatthisrequirementisoftennon-negotiable.WhereIntellectualPropertyarisesfromresearchlinkedindirectlytotheuseofmaterialprovidedundersuchagreement,theprovidershouldbeofferedatime-limitedopportunitytotakeoutarevenuegeneratinglicence.

1.5.TheAdministeringInstitutionandtheDrugDevelopmentGrantholdersareboundtonotifyFightMNDpromptlyinwritingwhennewProjectIParisesfromtheGrantandtakereasonablestepstoensurethatsuchIPisprotectedandnotpublishedorotherwisedisclosedpubliclypriortoprotection(whilstatthesametimeensuringthatpotentialdelaysinpublicationareminimised).

1.6.TheAdministeringInstitutionshouldseekFightMND’sconsenttocommerciallyexploittheresultsofanyresearchithasfunded.Consentwillnotbeunreasonablywithheld,andFightMNDwillonlyrefuseanAdministeringInstitution’srequestwhereitconsidersthattheproposedcommercialexploitationwouldruncountertoitsinterestsandcharitableobjectives.IntheeventthatFightMNDdoesnotprovidearesponsetotheAdministeringInstitution’srequestwithinthirty(30)businessdays,theInstitutionoritstechnologytransfersubsidiarywillautomaticallyhavetherighttoproceedwithsuchcommercialexploitation.TheAdministeringInstitutionisnotrequiredtoseekFightMND’sconsentinassigningIntellectualPropertytoitstechnologytransfercompany.

1.7.Withinthirty(30)businessdaysofreceivingthenotificationfromtheAdministeringInstitution,andpriortotheAdministeringInstitutionapplyingforregistrationofanyProjectIP,FightMNDwilladvisetheAdministeringInstitutioninwritingwhichoneofthefollowingfinancialarrangementswillapplyinrelationtocommercialisationoftheProjectIP: