RARE DISEASE IN CNS AND NEUROMUSCULAR … · and executing rare disease studies ... including rare...

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Experience

Global Labs

Core Imaging

Recruitment &Site Relationships

OURINTEGRATEDAPPROACH

Highly relevant operational know-how and subject matter expertise in managing and executing rare disease studies

A well-profiled network of sites and access to patient advocacy groups, KOL relationships and timely enrollment and high-quality conduct of trials

A wide range of relevant biomarker assays through our

wholly-owned central & bioanalytical lab network

End-to-end suite of global imaging services seamlessly

integrated into the overall structure of the trial

Deep Dive: Rare Disease in CNS and Neuromuscular Conditions

Accelerate your next study with Medpace’s noted medical and regulatory experts, highly experienced clinical trial management teams, central labs, and core imaging labs.

As a therapeutically-focused CRO, Medpace specializes in the design and conduct of global trials in neurology and psychiatry, including rare neurological disorders. We bring a global footprint, strategic medical, regulatory and operational leadership as well as fully integrated Central Labs and Core Imaging Services to enhance and expedite development.

RARE DISEASE IN CNS AND NEUROMUSCULAR

CONDITIONSExperts. Experience. Execution.

THE POWER OFxxx®

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CNS Rare Disease Experience

Medpace combines its team of neurologists with their cross-functional team of rare disease experts to support Sponsors developing therapies for rare neurological disorders. Medpace has conducted more than 80 global rare disease trials covering Phase I-IV, involving 1,702 sites and 5,768 patients. We have amassed experience in various rare neuromuscular indications such as:

• FacioscapulohumeralMuscularDystrophy(FSHD)• PompeDisease• DuchenneMuscularDystrophy• Mucopolysaccharidosis• SleepWakeDisorder• Huntington’sDisease• MultipleSystemAtrophy• Friedreich’sAtaxia• Charcot-Marie-ToothDisease(CMT)• Smith-MagenisSyndrome

Medpace also has the expertise and processes in place to conduct trials in disorders that have similar endpoints including alltypesofmusculardystrophy,spinalmuscularatrophy,myastheniagravis,Fabry,ALS,etc.

Recruitment and Global Site RelationshipsDue to the unique challenges associatedwith rare disease studies, partneringwith aCROwith expertisein enrolling rare disease patients and maximizing site selection to create realistic study feasibility is crucial. Medpace understands this challenge. Our multi-dimensional recruitment model enables us to implement innovative, comprehensive, and customized recruitment and retention strategies that identify, recruit, and

retainmembersofspecificpatientpopulations.

MedpacehasalsoestablishedaproductivenetworkofkeyclinicalsitesandlargeacademicinstitutionswhospecializeinCNSrarediseasedisordersensuringstudytimelinesandkeymilestonesareachieved.Ourphysiciansandprofessionalstaffhavein-depthknowledgeofscreeningtoolsandraterscalesandwillprovideoversightforraterservicesincludingsubjective assessments, inter-rater reliability, and scale validation.

Scientifically-Driven Clinical ResearchMedpaceisuniqueinitsscientifically-drivenapproachtoclinicalresearch.TheMedpacemodelgivesyoutheadvantageof early and ongoing insight and guidance from therapeutic experts throughout trial design and execution. Our highly experiencedmedicaldoctorsprovidestrategicdirectionforstudydesignandplanning,trainoperationalstaff,workwithInvestigators,providemedicalmonitoring,andmeetwith regulatoryagencies. Inaddition,ourmedicalmonitorsworkcollaborativelywithourglobalregulatoryaffairsexpertstoprovidestrategicguidanceintothebestpathwaystoaccelerateapproval.

Relationships with Patient Advocacy Groups Medpace recognizes the importance of collaborating with advocacy groups. Although advocacy groups vary greatlyinsize,scope,andpurpose,theyregularlyofferinsightintowhoistreatingpatients,whatisimportantto patients, and lessons learned from past clinical trials. Advocacy group collaboration raises awareness for your clinical trial and can be an invaluable partnership for the life of your drug.

Medpaceiswellversedinworkingwithsponsorsandadvocacygroupsofalldifferentsizesgloballyinorder to ensure clinical trials are designed and operated with the patients in mind. Medpace can help sponsors forge new relationships with advocacy groups or support existing relationships seemlessly.

RARE DISEASE IN CNS AND NEUROMUSCULAR CONDITIONS

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Meet our Medical Experts

Rare Disease

Terence Eagleton, MBBS, BScSenior Medical Director, Rare Diseases

Dr. Terence Eagleton is an accomplished pharmaceutical physician who has worked in the biopharma industry forover17years.Hehasworkedinallphasesofinternationalclinicalresearchandglobalmedicalaffairsinawidevarietyof therapeutic areasbutwith aparticular focuson raremetabolic andendocrinedisorders.Duringhis career, hehascontributedsignificantlytotheclinicaldevelopmentandcommercializationofnumerousinnovativetherapiesforrareandultra-rareindicationsthatinclude,butarenotlimitedto,MorquioSyndromeTypeA,HunterSyndrome,HurlerSyndrome,GaucherDisease,Anderson-FabryDisease,LysosomalAcidLipaseDeficiency,PompeDisease,HereditaryAngioedema,and X-linkedHypophosphatemia. Dr. Eagleton received his Bachelor of ScienceDegree (Honours) in Physiology andMedicalDegreeatUniversityCollegeLondonandfollowingqualification,trainedasasurgeonwithaparticularinterestinTraumaManagementandPatientCriticalCare.

Neurology

Sarah DeRossett, MD, PhDSenior Medical Director, Neurology

Dr. DeRossett is a board certified neurologist with more than 25 years of academic, clinical, and pharmaceuticalresearchexperience. Prior to joiningMedpace,sheservedasVPofClinicalResearchandMedicalAffairsatasmall,specialtypharmaceuticalcompanyandwasaSeniorMedicalDirectorandProjectPhysicianLeaderata large,globalpharmaceutical company, where she served as the single point of accountability for the global clinical development of newmedicinesacrossavarietyofneurosciencetherapyareas.HerdrugdevelopmentexperiencespansPhasesI-IVandincludes neurodegenerative and rare diseases. She has led or directly supported drug development programs resulting intwoEuropeanMedicinesAgencyandfourFDAapprovals.Dr.DeRossettearnedhermedicaldegreefromtheEmoryUniversitySchoolofMedicineandcompletedherNeurologytrainingatJohnsHopkins.ShealsoholdsaPhDinNeuralandBehavioralStudiesandwasformerlyonthefacultyatEmory.

Richard Scheyer, MDVice President, Medical Affairs, Neurology, Pharmacology

Dr.RichardScheyerisaboardcertifiedneurologistwithover30yearsofprofessionalmedicalexperiencewhichincludes20yearsdedicatedtoclinicaldrugdevelopment.HeisapioneerintranslationalmedicineandPhaseI/IIdrugdevelopment,withspecial interest inearlydemonstrationofclinicalefficacy.Dr.Scheyer receivedhisB.S. inPhysics fromStanfordUniversity,hisM.D.fromTheStateUniversityofNewYork,UpstateMedicalUniversity,andcompletedresidencytraininginNeurologyandfellowshiptraininginEpilepsyandClinicalPharmacologyatYaleUniversitybeforejoiningtheYalefaculty,servingasAssociateProfessorofNeurology.PriortojoiningMedpace,Dr.Scheyerheldanumberofleadershiprolesatbiopharmaceuticalfirmswherehewasresponsibleforclinical,biomarker,andpharmacogenomicstrategyandexecutionformorethan70developmentcandidatesacrosstherapeuticareas.Experienceincludessmallmolecule,macromolecular,and genetic therapies for common and rare neurologic disease.


Terence Eagleton, MBBS, BScSenior Medical Director, Rare Diseases

Richard Scheyer, MDVice President, Medical Affairs, Neurology, Pharmacology

Sarah DeRossett, MD, PhDSenior Medical Director, Neurology

James Vornov, MD, PhDVice President, Medical Affairs, Neurology, Analgesia, Psychiatry

Thomas Thompson, MDVice President, Medical Affairs, Psychiatry, Neurology

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James Vornov, MD, PhD Vice President, Medical Affairs, Neurology, Analgesia, Psychiatry

Dr. Vornov is an internationally known clinician-scientist with broad experience in both neuroscience and CNS drugdevelopment.HehasworkedinmultipleCNStherapeuticareashavingdirectedprogramsindepression,suicidalideation,Parkinson’sdisease,stroke,neuropathicpain,diabeticandchemotherapy-inducedperipheralneuropathy,anesthesiaandbrain tumors.Hehasparticularexpertise in the rapid transitionofcompounds from the laboratory toclinicalproofofconceptthroughtheuseoftechnologiessuchasbiomarkers,PK/PDmodeling,adaptivedesignandclinicaltrialsimulation.Dr.VornovreceivedhisB.A.inBiologyfromColumbiaUniversityandhisM.D.andPh.D.fromEmoryUniversitySchoolofMedicine.HetrainedinNeurologyattheJohnsHopkinsMedicalSchoolwhereheservedonthefacultyfor10yearspriorto transitioning to industry.

Psychiatry

Thomas Thompson, MDVice President, Medical Affairs, Psychiatry, Neurology

Dr.ThompsonisBoardCertifiedinPsychiatryandGeriatricPsychiatrywithclinicaldevelopmentexperienceinnumerousindicationsincludingParkinson’sdisease,bipolardisorder,stroke,epilepsy,depression,schizophrenia,anxietydisorders,andpain.Hehasco-authoredabookchapteronthegeneticsofbipolardisorderandhasbeenpublishedinnumerouspeerreviewedjournals.Dr.Thompsonhasheldleadershiprolesinthepharmaceuticalindustryandhasadepthandbreadthofexperiencesincludingmedicalaffairs,globalclinicaldevelopment,regulatorysubmissions,medicalgovernance,andmedicalmonitoring.HereceivedhisB.S.inbiologyfromtheUniversityofCentralFlorida,hisM.DfromTempleUniversitySchoolofMedicine,andhisPsychiatryandGeriatricPsychiatrycertificatefromEmoryUniversitySchoolofMedicine.


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Medpace Global Labs Provide Safety and Biomarker AnalysisMedpace’scentrallaboratoryhassupportedover45studiesfromPhaseIthroughPhaseIVinthefollowingCNS indications:

Medpace Labs provide consistency in methods and instrumentation across wholly-owned and purpose-built laboratories locatedintheUS,Europe,ChinaandSingapore.Weofferawiderangeofrelevantsafetyandbiomarkerassaysandhavethe ability to rapidly establish and validate novel assays as needed.

*Theremaybesometests/biomarkersrequestedthatarenotonthecentrallabtestmenu.AlthoughthosebiomarkersmaynotcurrentlybeonMedpace’svalidatedmenu,theyareavailableviaMedpace’slaboratorypartners.TheseassaysmaybecandidatesasnewvalidationstotheMedpaceinhousetestmenu.EachyearMedpacevalidatesover70newassaysbasedonguidelinesfromtheClinicalandLaboratoryStandardsInstitute(CLSI)andinaccordancewithCAPandCLIA regulations.


CNS and Neuromuscular Diseases Acute Ischemic StrokeAlzheimer’sCharcot-Marie-Tooth DiseaseDuchenne Muscular DystrophyEpilepsyFamilial Amyloid NeuropathyFamilial Amyloid PolyneuropathyHuntington’s DiseaseJet LagLennox-Gastaut SyndromeMajor Depressive Disorder

MigraineMultiple SclerosisMytonic Dystrophy Neurological DisordersNon-24 Hour Sleep-wake DisorderOpioid DependencePainPostpartum DepressionSleep DisordersStroke

Laboratory Testing Medpace

Validated Assays

Medpace Lab Partner

Network

Safety Testing Chemistry Serology Hematology Urinalysis Drugs of Abuse Screening Thyroid Testing CoagulationCystatin C (Serum)IL-6 (Serum){NSE) Neuron-specific enolase (Serum)S100 (Serum)Inflammatory Biomarkers (Serum)Genotyping Cerebral Spinal Fluid Testing (CSF) Total Tau (CSF) Neurofilament Light Chain (CSF) Neurogranin (CSF) YKL-40 (CSF)

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Core Imaging Expertise for Neuromuscular StudiesHigh-qualityimageacquisitionandinterpretationiscrucialforthesuccessoftrialsrelyingonneuroimagingfor patient selection or as a primary endpoint. Medpace provides centralized neuroimaging expertise, led by radiologists and neurologists with decades of clinical and trial experience.

Medpace Imaging Core Lab provides an end-to-end suite of imaging services to enhance and expedite biopharmaceutical and imaging contrast agent development, including a broad spectrum of imaging

biomarkersforDMDtrials.Capabilitiesinclude:

· MR Spectroscopy· T2Mapping· MuscleFatFraction· PDFFComplex· MRElastography

ClinTrak® EDC ePRO/eDiaryClinTrak EDC, Medpace’s proprietary clinical trial management system, has an ePRO/eDiary componentthat allows for the safe and secure collection of PRO data directly from patients through multiple platforms. Thiscomponentnotonlyoffersaflexible,highlycustomizedpatientinterfacebutthepatient-entereddataisimmediatelyintegratedintheClinTrakEDCdatabase–allowingSponsors,researchsites,Medpaceteamsand other permitted users web-based access to study data at all times.

A Full-Service Approach to Clinical ResearchDrivenby a full-serviceCROmodel that coordinates and integrates all services for our clients,Medpaceprovidesanaccountable,seamless,integratedandefficientplatformforexecutingclinicalresearch–increasingqualityandspeedwhilesignificantlyreducingtheneedforduplicatemanagementoversight.Ourdisciplinedprocesses, site relationships, and technologies enable us to execute even the most complex global studies, fromfirst-in-humanthroughpost-approval.

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