THE POWER OFx RARE DISEASE IN CNS AND NEUROMUSCULAR CONDITIONS Experts. Experience. Execution. 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. Highly relevant operational know-how and subject matter expertise in managing and executing rare disease studies OUR INTEGRATED APPROACH End-to-end suite of global imaging services seamlessly integrated into the overall structure of the trial Experience Core Imaging A well-profiled network of sites and access to patient advocacy groups, KOL relationships and timely enrollment and high-quality conduct of trials. Recruitment & Site Relationships Global Labs A wide range of relevant biomarker assays through our wholly-owned central & bioanalytical lab network
Page 2 of 6 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: Facioscapulohumeral Muscular Dystrophy (FSHD) Pompe Disease Duchenne Muscular Dystrophy Mucopolysaccharidosis Sleep Wake Disorder Huntington s Disease Multiple System Atrophy Friedreich s Ataxia Charcot-Marie-Tooth Disease (CMT) Smith-Magenis Syndrome Medpace also has the expertise and processes in place to conduct trials in disorders that have similar endpoints including all types of muscular dystrophy, spinal muscular atrophy, myasthenia gravis, Fabry, ALS, etc. Recruitment and Global Site Relationships Due to the unique challenges associated with rare disease studies, partnering with a CRO with expertise in 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 retain members of specific patient populations. Medpace has also established a productive network of key clinical sites and large academic institutions who specialize in CNS rare disease disorders ensuring study timelines and key milestones are achieved. Our physicians and professional staff have in-depth knowledge of screening tools and rater scales and will provide oversight for rater services including subjective assessments, inter-rater reliability, and scale validation. Scientifically-Driven Clinical Research Medpace is unique in its scientifically-driven approach to clinical research. The Medpace model gives you the advantage of early and ongoing insight and guidance from therapeutic experts throughout trial design and execution. Our highly experienced medical doctors provide strategic direction for study design and planning, train operational staff, work with Investigators, provide medical monitoring, and meet with regulatory agencies. In addition, our medical monitors work collaboratively with our global regulatory affairs experts to provide strategic guidance into the best pathways to accelerate approval. Relationships with Patient Advocacy Groups Medpace recognizes the importance of collaborating with advocacy groups. Although advocacy groups vary greatly in size, scope, and purpose, they regularly offer insight into who is treating patients, what is important to 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. Medpace is well versed in working with sponsors and advocacy groups of all different sizes globally in order 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.
Page 3 of 6 Meet our Medical Experts Terence Eagleton, MBBS, BSc Senior Medical Director, Rare Diseases Sarah DeRossett, MD, PhD Senior Medical Director, Neurology Richard Scheyer, MD Vice President, Medical Affairs, Neurology, Pharmacology James Vornov, MD, PhD Vice President, Medical Affairs, Neurology, Analgesia, Psychiatry Thomas Thompson, MD Vice President, Medical Affairs, Psychiatry, Neurology Rare Disease Terence Eagleton, MBBS, BSc Senior Medical Director, Rare Diseases Dr. Terence Eagleton is an accomplished pharmaceutical physician who has worked in the biopharma industry for over 17 years. He has worked in all phases of international clinical research and global medical affairs in a wide variety of therapeutic areas but with a particular focus on rare metabolic and endocrine disorders. During his career, he has contributed significantly to the clinical development and commercialization of numerous innovative therapies for rare and ultra-rare indications that include, but are not limited to, Morquio Syndrome Type A, Hunter Syndrome, Hurler Syndrome, Gaucher Disease, Anderson-Fabry Disease, Lysosomal Acid Lipase Deficiency, Pompe Disease, Hereditary Angioedema, and X-linked Hypophosphatemia. Dr. Eagleton received his Bachelor of Science Degree (Honours) in Physiology and Medical Degree at University College London and following qualification, trained as a surgeon with a particular interest in Trauma Management and Patient Critical Care. Neurology Sarah DeRossett, MD, PhD Senior Medical Director, Neurology Dr. DeRossett is a board certified neurologist with more than 25 years of academic, clinical, and pharmaceutical research experience. Prior to joining Medpace, she served as VP of Clinical Research and Medical Affairs at a small, specialty pharmaceutical company and was a Senior Medical Director and Project Physician Leader at a large, global pharmaceutical company, where she served as the single point of accountability for the global clinical development of new medicines across a variety of neuroscience therapy areas. Her drug development experience spans Phases I-IV and includes neurodegenerative and rare diseases. She has led or directly supported drug development programs resulting in two European Medicines Agency and four FDA approvals. Dr. DeRossett earned her medical degree from the Emory University School of Medicine and completed her Neurology training at Johns Hopkins. She also holds a PhD in Neural and Behavioral Studies and was formerly on the faculty at Emory. Richard Scheyer, MD Vice President, Medical Affairs, Neurology, Pharmacology Dr. Richard Scheyer is a board certified neurologist with over 30 years of professional medical experience which includes 20 years dedicated to clinical drug development. He is a pioneer in translational medicine and Phase I/II drug development, with special interest in early demonstration of clinical efficacy. Dr. Scheyer received his B.S. in Physics from Stanford University, his M.D. from The State University of New York, Upstate Medical University, and completed residency training in Neurology and fellowship training in Epilepsy and Clinical Pharmacology at Yale University before joining the Yale faculty, serving as Associate Professor of Neurology. Prior to joining Medpace, Dr. Scheyer held a number of leadership roles at biopharmaceutical firms where he was responsible for clinical, biomarker, and pharmacogenomic strategy and execution for more than 70 development candidates across therapeutic areas. Experience includes small molecule, macromolecular, and genetic therapies for common and rare neurologic disease.
Page 4 of 6 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 drug development. He has worked in multiple CNS therapeutic areas having directed programs in depression, suicidal ideation, Parkinson s disease, stroke, neuropathic pain, diabetic and chemotherapy-induced peripheral neuropathy, anesthesia and brain tumors. He has particular expertise in the rapid transition of compounds from the laboratory to clinical proof of concept through the use of technologies such as biomarkers, PK/PD modeling, adaptive design and clinical trial simulation. Dr. Vornov received his B.A. in Biology from Columbia University and his M.D. and Ph.D. from Emory University School of Medicine. He trained in Neurology at the Johns Hopkins Medical School where he served on the faculty for 10 years prior to transitioning to industry. Psychiatry Thomas Thompson, MD Vice President, Medical Affairs, Psychiatry, Neurology Dr. Thompson is Board Certified in Psychiatry and Geriatric Psychiatry with clinical development experience in numerous indications including Parkinson s disease, bipolar disorder, stroke, epilepsy, depression, schizophrenia, anxiety disorders, and pain. He has co-authored a book chapter on the genetics of bipolar disorder and has been published in numerous peer reviewed journals. Dr. Thompson has held leadership roles in the pharmaceutical industry and has a depth and breadth of experiences including medical affairs, global clinical development, regulatory submissions, medical governance, and medical monitoring. He received his B.S. in biology from the University of Central Florida, his M.D from Temple University School of Medicine, and his Psychiatry and Geriatric Psychiatry certificate from Emory University School of Medicine.
Page 5 of 6 Medpace Global Labs Provide Safety and Biomarker Analysis Medpace s central laboratory has supported over 45 studies from Phase I through Phase IV in the following CNS indications: CNS and Neuromuscular Diseases Acute Ischemic Stroke Alzheimer s Charcot-Marie-Tooth Disease Duchenne Muscular Dystrophy Epilepsy Familial Amyloid Neuropathy Familial Amyloid Polyneuropathy Huntington s Disease Jet Lag Lennox-Gastaut Syndrome Major Depressive Disorder Migraine Multiple Sclerosis Mytonic Dystrophy Neurological Disorders Non-24 Hour Sleep-wake Disorder Opioid Dependence Pain Postpartum Depression Sleep Disorders Stroke Medpace Labs provide consistency in methods and instrumentation across wholly-owned and purpose-built laboratories located in the US, Europe, China and Singapore. We offer a wide range of relevant safety and biomarker assays and have the ability to rapidly establish and validate novel assays as needed. Laboratory Testing Safety Testing Chemistry Serology Hematology Urinalysis Drugs of Abuse Screening Thyroid Testing Coagulation Cystatin 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) Validated Medpace Lab Partner Network *There may be some tests / biomarkers requested that are not on the central lab test menu. Although those biomarkers may not currently be on Medpace s validated menu, they are available via Medpace s laboratory partners. These assays may be candidates as new validations to the Medpace in house test menu. Each year Medpace validates over 70 new assays based on guidelines from the Clinical and Laboratory Standards Institute (CLSI) and in accordance with CAP and CLIA regulations.
Page 6 of 6 Core Imaging Expertise for Neuromuscular Studies High-quality image acquisition and interpretation is crucial for the success of trials relying on neuroimaging for 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 biomarkers for DMD trials. Capabilities include: MR Spectroscopy T2 Mapping Muscle Fat Fraction PDFF Complex MR Elastography ClinTrak EDC epro/ediary ClinTrak EDC, Medpace s proprietary clinical trial management system, has an epro/ediary component that allows for the safe and secure collection of PRO data directly from patients through multiple platforms. This component not only offers a flexible, highly customized patient interface but the patient-entered data is immediately integrated in the ClinTrak EDC database allowing Sponsors, research sites, Medpace teams and other permitted users web-based access to study data at all times. A Full-Service Approach to Clinical Research Driven by a full-service CRO model that coordinates and integrates all services for our clients, Medpace provides an accountable, seamless, integrated and efficient platform for executing clinical research increasing quality and speed while significantly reducing the need for duplicate management oversight. Our disciplined processes, site relationships, and technologies enable us to execute even the most complex global studies, from first-in-human through post-approval. Medical Affairs Regulatory Affairs and Medical Writing Clinical Monitoring Clinical Trial Management Biometrics and Data Sciences Safety and Pharmacovigilance Quality Assurance Global Labs, Imaging & Cardiovascular Core Labs medpace.com info@medpace.com North America Latin America Europe Asia Pacific Middle East Africa