Research Overview Jacqueline French, MD Chief Scientific Officer Epilepsy Foundation

Unmet Need 30% of people living with epilepsy do not have seizure control Number has not changed in decades despite 15+ drugs/devices entering the market since the 1990s We need to funnel new ideas into the clinical pipeline Epilepsy Foundation Research Overview

Epilepsy Foundation Response To develop an epilepsy research ecosystem for excellence, innovation, radical thinking and exciting discoveries to end epilepsy Epilepsy Foundation Research Overview

POST-APPROVAL RESEARCH & MONITORING Our Investments Along the Pipeline Basic Research DRUG DISCOVERY FDA REVIEW POST-APPROVAL RESEARCH & MONITORING PRE -CLINICAL CLINICAL TRIALS My Seizure Diary Trial Portal Registry Cohorts Epilepsy Foundation Research Overview

New Research Program Launched ENGAGE IMAGINE IMPLEMENT New Research Program Launched

Our Process Structure of Epilepsy Innovation Institute Epilepsy Foundation Research Overview

Unpredictability Community Survey 2016 What aspects of epilepsy impact people the most? Where should researchers focus? What is your biggest frustration with epilepsy? What is your biggest hope for epilepsy in next 5 years? Unpredictability Epilepsy Foundation Research Overview

Early Warning Seizure Detection System 2017 Topic My Seizure Gauge Early Warning Seizure Detection System Epilepsy Foundation Research Overview

Epilepsy Research AT The National Institutes of Health Brandy Fureman, PhD VP Research & New Therapies Epilepsy Foundation

NIH FUNDING COMPRISES THE MAJORITY OF FUNDING FOR EPILEPSY RESEARCH Source: Interagency Collaboration to Advance Research in Epilepsy (iCARE). National Institutes of Health Research

NIH Research Priorities for Grants 2014 Benchmarks for Epilepsy Research Understand the causes of the epilepsies and epilepsy-related neurologic, psychiatric and somatic conditions Prevent epilepsy and its progression Improve treatment options for controlling seizures and epilepsy-related conditions without side effects Limit or prevent adverse consequences of seizures and their treatment across the lifespan National Institutes of Health Research

NIH Epilepsy Research Programs Center Without Walls: Encouraging Global Collaborative Networks for Research 2010 – Established the Epi4K Consortia: Sequence at least 4000 people living with epilepsy 20 NEW genes discovered in 2016 alone Expanded to Epi25 (international effort) 2014 – Established Center for SUDEP Research Focused on preventing and risk-identification 2017 – Established EPIBios4RX: Epilepsy Bioinformatics Study to study epilepsy in patients with traumatic brain injuries National Institutes of Health Research

NIH Epilepsy Therapy Screening Program Encouraging Drug Discovery Mission: to encourage and facilitate the discovery of new therapeutic agents for the treatment of epilepsy disorders. Researchers & Industry can submit compounds for drug screening (for free) Compounds are tested for anti-seizure activity on a battery of well-established seizures animal models Several FDA approved drugs (i.e. Felbamate, Toprimate, Lacosamide, and Retigabine) went through this screening process National Institutes of Health Research

NIH Active Clinical Trials Studies to Improve Quality of Care Established Status Epilepticus Treatment Trial (ESETT) Comparing effectiveness of 3 anticonvulsant medications who have failed first line of therapy Maternal Outcomes and Neurodevelopmental Effects of Anti-epileptic Drugs (MONEAD) Following women with epilepsy during pregnancy to improve maternal and child outcomes Febrile Status Epilepticus (FEBSTAT) Following children with febrile studies to understand who is at increased risk for epilepsy National Institutes of Health Research

Other NIH Initiatives Other Research Which Can Impact Epilepsy Research Brain Research through Advancing Innovative Neurotechnologies (BRAIN) All of US Initiative (Precision Medicine) National Institutes of Health Research

Advancements in Research from the past 5 years Discussion: Advancements in Research from the past 5 years Jackie French, MD – Chief Scientific Officer Brandy Fureman, PhD – VP of Research & New Therapies Moderator: Sonya Dumanis, PhD – Director of Innovation Institute