HD Regulatory Science Consortium (HD-RSC) · HD Regulatory Science Consortium (HD-RSC) A consortium aimed at accelerating treatments for Huntington’s disease Kick-Off Meeting, Sheraton

HDRegulatoryScienceConsortium(HD-RSC)AconsortiumaimedatacceleratingtreatmentsforHuntington’sdisease

Kick-OffMeeting,SheratonSilverSpringHotel,November6-7,2017

Robi BlumensteinCHDIFoundation

“TheVision:CHDI’sDedicationtoSuccessfulHDTreatments”

2

MarthaBrumfieldCriticalPathInstitute

“Consortia-BasedStrategiesinNeurodegenerativeDiseases:CriticalPath

Institute’sTrackRecordinCollaborativeEfforts”

3

MikePanzara,WaveLifeSciencesScottSchobel,RocheLouiseVetter,HDSA

JulianaBronzova,UniQureMarkGordon,Teva

“TheNeed:WhataretheKeyChallengesforDrugDevelopmentinHD”

4

Moderator:JeffCarroll,WesternWashingtonUniversity

BillyDunnFDA

“RegulatoryImpactforHuntington’sDisease:FDAPerspectives”

5

10:35AM– 10:50AMBREAK

6

ManuelHaasEMA

“RegulatoryImpactforHuntington’sDisease:EMAPerspectives”

7

Issam ZinehFDA

“ModelInformedDrugDevelopment”

8

BrianCorriganPfizer

“FromModel-BasedClinicalTrialEnrichmenttoComprehensiveClinicalTrialSimulation”

9

11:45AM– 12:30PM

LUNCH

10

AmritaMohanCHDIFoundation

“SuccessinSharingDatafromHDNaturalHistoryStudies”

11

BrianCorrigan,PfizerBernhardLandwehrmeyer,UlmUniversity

KevinKrudys,FDAGeraldPodskalny,FDA

CharlesVenuto,UniversityofRochester

“HDDiseaseProgressionModeling:AligningDatawithContextofUseApplications”

12

Moderator:KlausRomero,CriticalPathInstitute

1:30PM– 1:45PM

BREAK

13

CristinaSampaioCHDIFoundation

“RationaleandImpactofBuildingaComprehensiveHDClinicalDatabase”

14

EricSiemersEliLilly

“BiomarkersasToolstoEnableDecision-MakinginHDDrugDevelopment”

15

GlennStebbinsRushUniversity

“ClinicalOutcomeMeasuresinHD:BeyondUHDRS”

16

SteveHersch,VoyagerBernardRavina,Voyager

BillyDunn,FDAEricBastings,FDA

“OutliningaRoadmapforClinicalTrialsinPre-ManifestHD”

17

Moderator:KarlKieburtz,UniversityofRochester

3:45PM– 4:00PMBREAK

18

CharlesSabinePatientAdvocate

“TheImpact:WhyThisMatterstoPatients”

19

6:00PMDINNERat:

CopperCanyonGrill928EllsworthDriveSilverSpring,MD

20

EmilyGantmanCHDIFoundation

“SummaryandHighlightsfromDay1ObjectivesforDay2”

21

DianeStephenson&DebraHannaCriticalPathInstitute

“TheOperations:HowHD-RSCWillWork”

22

Q&A23

DianeStephensonCriticalPathInstitute

IntroductiontoBreakoutSessions

24

10:00AM– 10:15AM

BREAK

25

10:15AM– 12:00PMBREAKOUTSESSIONS

1)Model-basedStrategiesforClinicalTrailEnrichment:FocusonBiomarkersRoom:Magnolia

2)ClinicalOutcomeAssessmentMeasuresinHDforUseinTrialsRoom:Persimmon1

3)TherapeuticDevelopmentinPre-manifestHD:OpportunitiesandChallengesRoom:Persimmon2

26

CristinaSampaioCHDIFoundation

“MeetingSummaryandConclusions”

27