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PROGRAM BOOKASENT 20th Annual Meeting

March 7-10, 2018

Hilton Washington DC/Rockville Hotel & Executive Meeting Center in Rockville, MD

ASENT.ORG

Vision Statement: To advance the science of neurotherapeutics, and to improve and

accelerate the development of new treatments.

CHAMPION | $25,000 - $49,999

Supporters

LEADERSHIP | $10,000 - $24,999

DIRECTOR | $5,000 - $9,999

SUPPORTER | $1,000 - $2,499

FRIEND | up to $999

Individual Donors:Thomas Sutula, MD, PhDMartha Morrell, MD

Individual Donors:O’Neill D’Cruz, MD Robert W. Hamill, MDStewart Alan Factor, DO Sandra Pizzoferrato

Jessica Keenan Smith

ADVOCATE | $2,500 - $4,999

#ASENT18 asent.org �3

IMPORTANT DATES Abstract Submissions Open

May 2018

Abstract Submission DeadlineAugust 2018

Pipeline Invitation Period OpensSeptember 2018

Annual Meeting Registration Opens

October 2018

Pipeline Invitation Period ClosesNovember 2018

2019 Membership RenewalNovember 2018

2019 Annual MeetingMarch 5-8, 2019

TABLE OF CONTENTS Full Program - Page 4Pipeline Presentations - Page 6Posters - Page 14Awards - Page 18Business Meeting - Page 20Training in Neurotherapeutics Course Agenda - Page 21Supporters - Page 24Floor Plan - Page 32ASENT Leadership - Page 33Program Committee - Page 33Attendees - Page 34Save the Date - Back Cover

ASENT 20th Annual Meeting

Welcome to the American Society of Neurotherapeutics (ASENT) 20th Annual Meeting at the Hilton Washington DC/Rockville Hotel and Executive Meeting Center in Rockville, Maryland.

ASENT is an independent non-profit organization established in 1997 by leaders in academia, government, advocacy and industry to facilitate the process by which new therapies are made available to patients with neurological disorders.

On behalf of the Program Committee, Board of Directors, and faculty, we are delighted you are here to participate in a program that addresses the broad spectrum of neurotherapeutic topics.

We start on March 7 with our pre-meeting symposium, ASENT's Translational Research Course. Next up, we hope you will join us at the Pipeline Sessions on March 8. You will hear 24 presentations about the discovery and development of new CNS therapy candidates.

The symposia on March 9 and 10 include therapeutic advances in Epilepsy, Parkinson’s Disease, ALS, Alzheimer’s Disease, and ADHD. Technological breakthroughs will include the use of deep brain stimulation, wearable and implantable devices, genomic-based drug discovery in neurodegenerative disease, the growing emphasis and successes with adenoviruses, antisense, vaccines, and antibodies to treat neurodegenerative disease, and technological innovations in clinical trial research.

During this meeting we are pleased to honor Michael Okun, MD, with the ASENT Presidential Award. Hailed as ASENT’s highest honor to an individual, the 2018 ASENT Presidential Award recognizes extraordinary accomplishments in the field of clinical translational research. ASENT is also pleased to honor Eisai, Inc. with the Champion Award, the society’s highest honor to an organization or corporation.

We know you will find the ASENT meeting time well-spent. Take advantage of the many opportunities to interact and network with your colleagues who are participating in this year’s program. Please also consider joining ASENT if you plan on attending next year or want to become more involved in our planning activities.

Sincerely,

Martha Morrell, MDChief Medical Officer, NeuroPace, Inc.Clinical Professor of NeurologyStanford UniversityPresident, American Society for Experimental Neurotherapeutics

C. Anthony Altar, PhDVerge Genomics, Inc.Program Committee Chair

Bennett L. Lavenstein, MD Children’s Hospital Medical CenterASENT Board of Directors Program Committee Co-Chair

EXECUTIVE STAFF Jessica Keenan SmithExecutive DirectorSandra PizzoferratoDirector of OperationsTerrie Blalock Consultant

Program Schedule

Wednesday, March 7, 2018 TIME ACTIVITY

7:00 am - 7:30 am Breakfast/Registration

7:45 am - 8:15pm

Washington Theater/Presidential

Training in Neurotherapeutics Discovery and Development for Academic Scientists: Day 1Chairs: Michael Rogawski, MD, PhD, Professor, Neurology, University of California, DavisBarbara Slusher, PhD, MAS, Professor of Neurology, Psychiatry, Neuroscience, Medicine, and Oncology, Johns Hopkins School of MedicineKarl Scheidt, Professor, Northwestern Medicine, Founder, Third Coast Therapeutics

This NIH sponsored Course is open only to students selected through a competitive application process. This training program in neurotherapeutics discovery and development is an intensive 3½ -day course that provides trainees with the knowledge required to discover and advance a neurotherapeutic agent to Investigational New Drug (IND) Application. The course, which is designed to be applicable to diverse diseases of the nervous system, will equip students with a broad understanding of the various component steps in the neurotherapeutics drug discovery and development process. Participants must apply.

9:00 am - 3:35 pm

Plaza I

PRE-MEETING SYMPOSIUMASENT Translational Research CourseCo-Chair: Steven Schachter, MD, Professor, Neurology, Harvard Medical Schooll; Chief Academic Officer, Program Leader of Neuro-Technology at CIMITCo-Chair: Stewart Factor, DO, Professor of Neurology, Director of the Movement Disorders Program and Vance Lanier Chair of Neurology at Emory University School of Medicine

The goal of this full day course is to focus on healthcare providers and their interactions with industry and government to develop clinical translational research. Currently there is a gap in knowledge regarding the ability to move drugs and devices from bench to bedside including developing appropriate partnerships, understanding intellectual property, trial design and statistics, and regulatory paths and funding opportunities. This course will be a first step in filling those knowledge gaps.

9:00am – 9:10amPlaza I

Overview

Stewart Factor, DO, Professor of Neurology, Director of the Movement Disorders Program and Vance Lanier Chair of Neurology at Emory University School of Medicine

Forming and Sustaining Effective Collaborations between Clinicians and Engineers/Scientists

Steven Schachter, MD, Professor, Neurology, Harvard Medical Schooll; Chief Academic Officer, Program Leader of Neuro-Technology at CIMIT

Repurposing Drugs, including Regulatory Aspects

Freddie Ann Hoffman, MD, Founder, HeteroGeneity, LLC10:00am – 10:25am

Plaza IA FDA Staff Perspective on Neurological Medical Devices

Carlos Peña, PhD, MS, Director, Division of Neurological and Physical Medicine Devices (DNPMD), FDA

10:25am – 10:50amPlaza Foyer

Identifying Stakeholders in the Healthcare Ecosystem

Michael Gold, MD, Vice President, Development Neurosciences, AbbVie

Program Schedule

Wednesday, March 7, 2018 TIME ACTIVITY11:15am – 11:40am

Plaza ININDS Funding Opportunities and Resources for Translational Researchers Stephanie Fertig, MBA, Project Manager, SBIR, NINDS/NIH

11:40am – 12:30pm

Plaza I

Panel Discussion, overview of afternoon session

12:30pm – 1:50pm

Roosevelt

LUNCH BREAK Lunch and remainder of day with students of Training in Neurotherapeutics Discovery and Development for Academic Scientists

2:00pm - 4:00pm

Montrose

Publications MeetingChair: Michael Rogawski, MD, PhD, Professor, Neurology, University of California, Davis

4:00pm - 6:00pm

Montrose

Board MeetingChair: Martha Morrell, MD, Chief Medical Officer, NeuroPace, Inc.

1:55pm – 2:20pm

Plaza II/III

The Innovation Cycle and Defining Clinical Needs

Mike Dempsey, Entrepreneur in Residence and Director of the Accelerator program at the Center for the Integration of Medicine and Innovative Technology (CIMIT), Founder and CEO, Secora Care

2:20pm – 2:45pm

Plaza II/III

Putting Together an Action Plan to Patient: the Healthcare Innovation Impact and Tracking System (HIITS) Metrics

John Collins, PhD, COO, CIMIT2:45pm – 3:35pm

Plaza II/III

IP Relevant to Neurotherapeutics

Jeffrey Childers,PhD, Partner, Michael Best & Friedrich LLP (formerly Johns Hopkins Technology Transfer)

6:00 pm - 8:30 pm

Eisenhower

Careers in Neuroscience DinnerChair: Martha Morrell, MD Chief Medical Officer, NeuroPace, Inc.

Faculty: R. Jacob Vogelstein, PhD, Managing Partner | Nexus, Camden Partners Holdings, LLCTimothy Denison, PhD, VP of Research and Core Technology, Medtronic

Thursday, March 8, 2018 TIME ACTIVITY

7:30 am - 8:00 am

Plaza Foyer

Welcome Breakfast/RegistrationChair: Martha Morrell, MD, Chief Medical Officer, NeuroPace, Inc.

Meet ASENT committee members and board members

7:45 am - 8:30pm

Washington Theater/

Presidential Foyer

Training in Neurotherapeutics Discovery and Development for Academic Scientists: Day 2

8:30 am - 9:00 amPlaza II/III

President's Welcome RemarksChair: Martha Morrell, MD, Chief Medical Officer, NeuroPace, Inc.

9:00 am - 12:00 pmPlaza II/III

Pipeline Presentations: Part 1Chair: Kathleen Clarence-Smith, MD, PhD, Co-Founder, Partner, KM Pharmaceuticals

9:00 - 9:10amPlaza II/III

IntroductionKathleen Clarence-Smith, MD, PhD, Co-Founder, Partner, KM Pharmaceuticals

A Neurometabolic Approach to Treating Alzheimer’s Disease: Hypothesis Testing in a Phase 2a Exploratory Clinical Trial with the New Chemical Entity T3D-959Stan Chamberlain, PhD, Chief Scientific Officer, T3D Therapeutics

CT1812 exhibits a synaptoprotective effect in Alzheimer’s disease patientsSusan M. Catalano, PhD, Founder and CSO, Cognition Therapeutics Inc

GM6- an endogenous multiple-target regulator that provides a novel therapeutic strategy for treatment of ALS and other neurodegenerative diseasesWinston Ko, CEO, Genervon Biopharmaceuticals LLC

Secretory Products of B Cells from Blood of Multiple Sclerosis Patients are Toxic to Oligodendrocytes and Neurons: Characterization of Toxic Factors and Use of Bioassay as a Screen of Potential Protective TherapiesRobert P. Lisak, MD, Wayne State University School of Medicine

Surviving Cell Death: The Difference between Tolerance and AutoimmunityLinda Kusner, PhD, George Washington School of Medicine and Health Sciences

Advancing targeted therapies for patient populations harboring ultra-rare mutations in a rare disease: Sarepta’s exon skipping platform for Duchenne muscular dystrophy.Guriqbal Basi, Sr. VP, Chief Scientific Officer, Sarepta Therapeutics

DMD Biglycan upregulates utrophineMichael Myers, Interim CEO, Tivorsan Pharmaceuticals

RNA Trans-splicing: Gene Editing for the BrainLloyd Mitchell, CEO & CSO RetroTherapy

10:30 - 10:45amPlaza Foyer

Lipid-shelled microbubbles for ultrasound-triggered release of Xenon to treat strokeChristy K. Holland, PhD, University of Cincinnati in Ohio

Edge Therapeutics: EG-1962. A novel treatment for aneurysmal Subarachnoid HemorrhageDan Brennan, Chief Operating Officer, Edge Therapeutics Inc.

Program Schedule

Discovery and development of NPT520-34, a novel therapeutic candidate for the treatment of Parkinson's disease and related neurodegenerative disordersDiana Price, PhD, Senior Director Neurosciences, Neuropore Therapies, Inc.

Glial cell Line-Derived Neurotrophic factor (GDNF) treatment of Parkinson’s disease: Emerging evidence of clinical benefitErich Mohr, PhD, R.Psych, CEO, MedGenesis Therapeutix

Lisuride-a drug with outstanding affinity for monoamine receptorsReinhard Horowski, MD, CEO, ARAMON Pharma UG

Retinal lesions predictors of progression for certain neurodegenerative disordersCedric O’Gorman, MD, MBA, Senior Vice President, Clinical Development and Medical Affairs, Axsome Therapeutics, Inc.

11:45 - 12:00pmPlaza II/III

CIMIT: A Unique Model for Accelerating Healthtech InnovationsSteven Schachter, MD, Professor of Neurology, Harvard Medical School; Chief Academic Officer, Program Leader of Neuro-Technology at CIMIT

12:00 pm - 2:00 pmRoosevelt

Lunch with Pipeline PresentersChair: Kathleen Clarence-Smith, MD, PhD, Co-Founder, Partner, KM Pharmaceuticals

12:00 pm - 1:00 pmMonroe

Advocacy Engagement Committee MeetingChair: Martha Morrell, MD, Chief Medical Officer, NeuroPace, Inc.

1:00 pm - 2:00 pmTwinbrooke

Industry Engagement Committee MeetingChair: Martha Morrell, MD, Chief Medical Officer, NeuroPace, Inc.

2:00 pm - 3:30 pmPlaza II/III

Pipeline Presentations: Part 2Chair: Kathleen Clarence-Smith, MD, PhD, Co-Founder, Partner, KM Pharmaceuticals

2:00 - 2:10 pmPlaza II/III

Identifying early clinical development priorities for disease-modifying therapies in neurodegenerative diseasesLudy Shih, MD, Associate Medical Director, Biogen, Inc.

Preclinical Through Early-Stage Clinical Development of a Novel NMDA Receptor Modulator, NYX-2925David Houck, PhD, VP, Drug Development, Aptinyx

2:20 - 2:30 pm

Plaza II/III

EpiWatch: seizure tracking with a consumer wearableNathan Crone, MD, Associate Professor of Neurology and director of the Cognitive Neurophysiology and Brain-Machine Interface Laboratory, Johns Hopkins University School of Medicine

2DG Update: Anticonvulsant Actions, Neuroprotection, and Prevention of Post-traumatic Epilepsy Thomas Sutula, MD, PhD, Founder, Chief Technical Officer and Director, NeuroGenomeX, Inc

GalR2-Preferring Galanin Analogs: Efficacy in Pain and Epilepsy ModelsCameron Metcalf, NeuroAdjuvants, Inc.

Propofol Prodrug for Acute Repetitive SeizuresMichael Rogawski, MD, PhD, Epalex Corporation

Program Schedule

Ganaxolone, an investigational neurosteroid treatment for children with CDKL5 deficiency disorder: Results from a Phase 2 TrialMichael Rogawski, MD, PhD, Marinus

Moving beyond peak-trough issues with spasticity drugsVikram Sudarsan, PhD, Head, Cipla Technologies

BPN14770 Clinical Development for Fragile X SyndromeMark Gurney, PhD, Chairman & CEO, Tetra Discovery Partners, Inc.

3:30 - 3:45 Break

3:45 pm - 5:30 pmRegency/Randolph

Poster Presentations and ReceptionChair: Kathleen Clarence-Smith, MD, PhD, Co-Founder, Partner, KM Pharmaceuticals

5:30 pm - 7:30 pmEisenhower

2018 ASENT Champion Award presented to Eisai, Inc.

Dinner Symposium: Patient Involvement in Drug Approval – debateChair: Robin Elliott, MA, President, Parkinson’s Disease Foundation

FacultyRoger Porter, MD, ConsultantJames Valentine, JD, MHS, Associate, Hyman, Phelps & McNamara, P.C.

Friday, March 9, 2018 TIME ACTIVITY

7:00 - 8:00 amMontrose Scientific Program Committee Meeting

7:00 - 8:00 amPlaza Foyer Breakfast/Registration

7:45 am - 8:30 pm Training in Neurotherapeutics Discovery and Development for Academic Scientists: Day 3

Symposium: Clinical Trials: Lessons Learned: From Patient Recruitment to Innovative DesignsCo-Chair: Dietrich Haubenberger, MD, Director, Clinical Trials Unit, NINDS/NIHCo-Chair: Erika F. Augustine, MD, MS, Associate Professor of Neurology and Pediatrics & Associate Director, Center for Health + Technology, University of Rochester

FacultyValue of subject registries in clinical development Stephen Peroutka, MD, PhD, Vice President, Global Therapeutic Area Head - NeurosciencePPD

The science of recruitmentJoshua Grill, PhD, Director Education, Institute for Memory Impairments and Neurological Disorders, University of California, Irvine

Lessons learned applying novel trial designs in industry development programs: What works, what doesn’t. Michael Gold, MD, Vice President, Development Neurosciences, AbbVie

Best practice & lessons learned for recruiting into Alzheimer’s trialsLawrence Friedhoff, MD, PhD, Business Development, Roivant Sciences, Inc.

Program Schedule

8:00 am - 10:00 amPlaza I

Emerging Therapies: ADHDCo-Chair: Enrique Carrazana, MD, Member, Board of Directors, Marinus PharmaceuticalsCo-Chair: Petra Kaufmann, MD, MSc, Director, NCATS Office of Rare Diseases Research

FacultyADHD and risk for substance use disorders: Can neuroimaging guide our understanding? Kevin M. Gray, MD, Professor, Department of Psychiatry and Behavioral Sciences, Medical University of South Carolina

Update on Supernus ADHD ProgramStefan Schwabe, MD, PhD, Executive Vice President, Head of R&D, Supernus Pharmaceuticals

Regulatory perspectives of drug development in psychostimulant abuseBeatriz Rocha, MD, PhD, Head Strategic Product Development, Covance

An abuse deterrent formulation of MethylphenidatePradeep Bhide, PhD, Rodgers Eminent Scholar Chair of Developmental Neuroscience, Florida State University and Co-Founder, Avekshan, LLC

The development of novel COMT inhibitors for the treatment of ADHDGregory V. Carr, PhD, Investigator, Lieber Institute for Brain Development, Assistant Professor of Pharmacology and Molecular Sciences, Johns Hopkins University School of Medicine.

10:00 am - 10:15 amPlaza Foyer

Symposium: Technological Innovations in Clinical Trials Research and Neurotherapeutic DevelopmentCo-Chair: Robert W. Hamill, MD, Professor, Neurological Sciences, Emeritus, University of VermontCo-Chair: E. Ray Dorsey, MD, MBA, David M. Levy Professor of Neurology & Director, Center for Health + Technology, University of Rochester

FacultyEvolution of neurology clinical trials including digital biomarkersE. Ray Dorsey, MD, MBA, David M. Levy Professor of Neurology & Director, Center for Health + Technology, University of Rochester

Novel applications of technology are changing the nature of trial recruitment, retention, and conductErika F. Augustine, MD, MS, Associate Professor of Neurology and Pediatrics & Associate Director, Center for Health + Technology, University of Rochester

Technology derived novel endpoints: Lessons from CTTI (Clinical Trials Transformation Initiative)Daniel Karlin, MD, Head of Clinical Informatics and Regulatory Strategy, Pfizer

Venture capital and neurotherapeutics: Lessons from EpilepsyWarren B. Lammert, Founder and Chief Investment Officer, Granite Point Capital Management, L.P.

10:15 am - 12:15 pmPlaza I

Symposium: Non-Small molecule alternatives to pharmaceuticals for neurologyChair: C. Anthony Altar, PhD, Chief Scientific Officer and Senior Vice President, Verge Genomics, Inc.

FacultyAntisense oligonucleotides for CNS disordersToby Ferguson, MD, PhD, Medical Director, Biogen

Antibodies for CNS disordersRonald DeMattos, PhD, Chief Scientific Officer, Neurobiologics, Eli Lilly and Company

Adeno-associated viruses for CNS disorders: Initial proof of concept for Parkinson's DiseaseSteven Paul, MD, CEO, Voyager Therapeutics

Active immunotherapy vaccines for CNS disordersAjay Verma, MD, PhD, United Neuroscience

Program Schedule

12:15 pm - 1:00 pmRoosevelt Lunch

1:00 pm - 2:30 pm Plaza I

Emerging Therapies: Alzheimer’s DiseaseChair: Korie Handwerger, PhD, Science Communications Manager, RA Capital Management, LLC

FacultyDevelopment-Stage Pharmotherapies in Alzheimer’s: Beyond Aβ Korie Handwerger, PhD, Science Communications Manager, RA Capital Management, LLC

Designing GAIM fusions to target multiple protein aggregates Rajaraman Krishnan, PhD, MS, Senior Director Research, Proclara Biosciences

The clinical development and therapeutic potential of AXS-05 for the neuropsychiatric symptoms of Alzheimer’s disease Cedric O’Gorman, SVP of Clinical Development and Medical Affairs, Axsome Therapeutics

Targeting hippocampal overreactivity in prodromal Alzheimer's diseaseMichela Gallagher, PhD, Founder and Scientific Advisor, AgeneBio, Kreiger-Eisenhower Professor of Psychological Brain Science, Johns Hopkins University

Stimulating protein clearance from the CNS for the treatment of Alzheimer's diseasePhillip Haydon, PhD, Co-Founder, GliaCure, the Annetta and Gustav Grisard Professor and Chair, Department of Neuroscience, Tufts University

SYMPOSIUM EXCLUSIVELY SPONSORED BY Janssen: Pharmaceutical Companies of Johnson and Johnson

Symposium: Investing in NeurotherapeuticsChairs: Mitchell Brin, MD, Sr. VP, Global Development & Chief Scientific Officer, Allergan

FacultyInvestment facts and metrics: Sources of funds for CNS Therapy development Jamie Munro PhD, Head of CMR and Global Practice Leader, Claivate Analytics

Where are life sciences investors making their investments, and why?Shawn Cross, President and COO, GT Biopharma

Panel: Improving the Outlook for CNS Investments Frank Fischer, President and CEO, NeuroPaceR. Jacob Vogelstein, PhD, Managing Partner | Nexus, Camden Partners Holdings, LLC Jamie Munro PhD, Head of CMR and Global Practice Leader, Claivate AnalyticsShawn Cross, President and COO, GT Biopharma

2:30 pm - 2:45 pmPlaza Foyer Break

Emerging Therapies: Epilepsy DevicesChair: Steven Schachter, MD, Professor, Neurology, Harvard Medical Schooll; Chief Academic Officer, Program Leader of Neuro-Technology at CIMIT

FacultyEpilog: Wearable Seizure diary Mark Lehmkuhle, PhD, Epitel, Inc.

Empatica. Embrace: Autonomic and seizure events - personalized monitoring and alerts Rosalind W. Picard, ScD, Chief Scientist, Empatica, Inc., Co-Founder, Affective Computing Research Group, Professor, Massachusetts Institute of Technology Media Lab

Brain and multi-modal physiological monitoring with NINscan Vladimir Ivkovic, PhD, Director Laboratory for Neuroimaging and Integrative Physiology, Harvard Medical School

New horizons in long-term EEG monitoring Knud Stampe, Marketing Manager, UNEEG medical

2:45 pm - 4:15 pmPlaza I

Emerging Therapies: ALSChair: C. Anthony Altar, PhD, Chief Scientific Officer and Senior Vice President, Verge Genomics, Inc.

FacultyNuclear Export inhibitor KPT-350 for ALS Sharon Tamir, Director, Strategic Product Development, Head of Neurodegenerative and Infectious Diseases, Karyopharm Therapeutics, Inc.

Genomic-based drug discovery for neurodegenerative diseasesAlice Zhang, CEO and Co-Founder,Verge Genomics, Inc.

Gene therapy for ALS using plasmid DNA expressing human hepatocyte growth factor: Scientific basis and results from a Phase I study.Marie-Laure Nevoret, MD, Clinical Director, VM BioPharma, ViroMed Co., Ltd.

An Endogenous Multiple-Target Regulator Provides a Novel Regulatory Peptide Therapeutic Strategy for Treatment of ALS and Other Neurodegenerative Diseases, as Confirmed by Consistent Safety and Efficacy Data from Pre-Clinical, Phase 2A Placebo-Controlled Clinical Trials, and from Functional, Biomarkers and Bioinformatic DataWinston Ko, Chairman and CEO of Genervon Biopharmaceuticals

4:15 pm - 4:30 pm Break

Emerging Therapies: Epilepsy DrugsChair: Steven Schachter, MD, Professor, Neurology, Harvard Medical Schooll; Chief Academic Officer, Program Leader of Neuro-Technology at CIMIT

FacultyZX008 for the treatment of Dravet Syndrome Gail Farfel, PhD, EVP, Chief Development Officer, Zogenix

ANAVEX 2‐73 as a potential treatment for Rett Syndrome and other pediatric or infantile disorders with seizure pathology Christopher Missling, PhD, President and CEO, Anavex Life Sciences Corp

CBD/CBDVKen Sommerville, Vice President, Clinical Research, Greenwich Biosciences (retired)

Adenosine for epilepsy preventionHai-Ying Shen, MD, PhD, Legacy Research Institute

Program Schedule

Saturday, March 10, 2018 TIME ACTIVITY

7:00 am - 7:30 amPlaza Foyer Breakfast/Registration

7:45 am - 12:15pm Training in Neurotherapeutics: Closing Day

ASENT Business MeetingChair: Martha Morrell, MD, Chief Medical Officer, NeuroPace, Inc.All members welcome to attend

Symposium: Technology enabled measurements: Emerging role in NeurotherapeuticsCo-Chair: William J. Marks, Jr., MD, MS, Head of Clinical Neurology, Verily Life SciencesCo-Chair: Martha Morrell, MD, Chief Medical Officer, NeuroPace, Inc.

FacultyPhysiological biomarkers in the brain: Revelations from direct brain sensing in epilepsyMartha Morrell, MD, Chief Medical Officer, NeuroPace, Inc.

Wearable technologies: Contributions to the understanding of neurological disordersWilliam J. Marks, Jr., MD, MS, Head of Clinical Neurology, Verily Life Sciences

App based systems to support neurotherapeutic development: a virtual clinical trial in MigraineGabriel Vargas, MD, PhD, Executive Medical Director, Digital Health & Neuroscience Therapeutic Area Head, Early Development, Amgen

Regulatory considerations for use of technology-derived data: the CDRH perspectiveCarlos Peña, PhD, MS, Director, Division of Neurological and Physical MedicineDevices (DNPMD), FDA

Moderated DiscussionWilliam J. Marks, Jr., MD, MS, Head of Clinical Neurology, Verily Life Sciences

10:00 am - 10:15 amPlaza Foyer Break

Poster Presentations and Reception: 20th Anniversary CelebrationChair: Martha Morrell, MD, Chief Medical Officer, NeuroPace, Inc.

All attendees invited to celebrate our 20th Anniversary Celebration.

7:00 pm - 8:00 pmThe Library

Investor ReceptionChair: Martha Morrell, MD, Chief Medical Officer, NeuroPace, Inc.

Program Schedule

Saturday, March 10, 2018 TIME ACTIVITY

2018 ASENT Presidential Award Presented to Michael Okun, MD

Emerging Therapies: Parkinson's DiseaseChair: Michael Okun, MD, Professor and Chair, Department of Neurology, Administrative Director & Co-Director, Center for Movement Disorders and Neurorestoration, Center for Translational Research in Neurodegenerative Diseases, the McKnight Brain Institute, University of Florida College of Medicine

FacultyIntroduction Michael Okun, MD, Professor and Chair, Department of Neurology, Administrative Director & Co-Director, Center for Movement Disorders and Neurorestoration, Center for Translational Research in Neurodegenerative Diseases, the McKnight Brain Institute, University of Florida College of Medicine

Current steering technologiesChris Butson, PhD, Associate Profesor, Scientific Computing & Imaging (SCI) Institute, Department of Bioengineering, University of Utah

Adaptive deep brain stimulationTimothy Denison, PhD, Vice President of Research and Core Technology, Medtronic

Repurposing Isradipine for neuroprotection in Parkinson’s DiseaseTanya Simuni, MD, Chief of Movement Disorders in the Department of Neurology. Northwestern University

Targeting Tumor Necrosis Factor-dependent inflammation in Parkinson’s DiseaseMalu Tansey, PhD, Associate Professor, Department of Physiology, Emory University

12:15 pm Adjourn

PIPELINE POSTERS

1 A NEUROMETABOLIC APPROACH TO TREATING ALZHEIMER'S DISEASE: HYPOTHESIS TESTING IN A PHASE 2A EXPLORATORY CLINICAL TRIAL WITH THE NEW CHEMICAL ENTITY T3D-959Stan Chamberlain, PhD, John Didsbury, Hoda Gabriel, PMP, Warren Strittmatter, M.D., Pipeline Presenter: Stan Chamberlain, Ph.D.

2 CT1812 EXHIBITS A SYNAPTOPROTECTIVE EFFECT IN ALZHEIMER'S DISEASE PATIENTS Susan Catalano, Lon S Schneider, MD, MS, Steven DeKosky, MD, Roger Morgan, MD, Courtney Rehak, Colleen Silky, Kelsie Mozzoni, Nicholas J Izzo, PhD, Michael Grundman, MD, MPH, Michael Schirm, PhD, Rudolf Guilbaud, MSc, Mark Watson, PhD, Daniel Chelsky, PhD, Charles Davis PhD, Henrik Zetterberg, MD, PhD, Kaj Blennow, MD, PhDPipeline Presenter: Susan M. Catalano, PhD

3 no poster GM6- AN ENDOGENOUS MULTIPLE-TARGET REGULATOR THAT PROVIDES A NOVEL THERAPEUTIC STRATEGY FOR TREATMENT OF ALS AND OTHER NEURODEGENERATIVE DISEASESPipeline Presenter: Winston Ko, MBA

4 no poster SECRETORY PRODUCTS OF B CELLS FROM BLOOD OF MULTIPLE SCLEROSIS PATIENTS ARE TOXIC TO OLIGODENDROCYTES AND NEURONS: CHARACTERIZATION OF TOXIC FACTORS AND USE OF BIOASSAY AS A SCREEN OF POTENTIAL PROTECTIVE THERAPIES Pipeline Presenter: Robert Lisak, MD

5 SURVIVING CELL DEATH: THE DIFFERENCE BETWEEN TOLERANCE AND AUTOIMMUNITYLinda Kusner, Henry J Kaminski, Robert Fenstermaker, Michael Ciesielski,Pipeline Presenter: Linda Kusner, PhD

6 ADVANCING TARGETED THERAPIES FOR PATIENT POPULATIONS HARBORING ULTRA-RARE MUTATIONS IN A RARE DISEASE: SAREPTA’S EXON SKIPPING PLATFORM FOR DUCHENNE MUSCULAR DYSTROPHY.Basi Guriqbal, Diane BerryPipeline Presenter: Basi Guriqbal

7 RNA TRANS-SPLICING: GENE EDITING FOR THE BRAINMitchell Lloyd, MDPipeline Presenter: Mitchell Lloyd, MD

8 no poster LIPID-SHELLED MICROBUBBLES FOR ULTRASOUND-TRIGGERED RELEASE OF XENON TO TREAT STROKEChristy K. Holland, PhDPipeline Presenter: Christy Holland, PhD

9 EG-1962 FOR THE TREATMENT OF ANEURYSMAL SUBARACHNOID HEMORRHAGEDaniel BrennanPipeline Presenter: Daniel Brennan

10 DISCOVERY AND DEVELOPMENT OF NPT520-34, A NOVEL THERAPEUTIC CANDIDATE FOR THE TREATMENT OF PARKINSON’S DISEASE AND RELATED NEURODEGENERATIVE DISORDERSDiana Price, Asma Khan, Wolfgang Wrasidlo, Douglas Bonhaus Diana Price, PhD

Posters

PIPELINE POSTERS

11 GLIAL CELL LINE-DERIVED NEUROTROPHIC FACTOR (GDNF) IN PARKINSON'S: EMERGING EVIDENCE OF CLINICAL BENEFIT Erich Mohr, PhD, R.Psych Drich M ohr, GDNF Study Group: Alan Whone, Matthias Luz, Mihaela Boca, Max Woolley, Lucy Mooney, Sonali Dharia, Jack Broadfoot, David Cronin, Christian Schroers, Neil U. Barua, Lara Longpre,, C. Lynn Barclay, Chris Boiko, Greg A. Johnson, H. Christian Fibiger, Rob Harrison, Owen Lewis, Gemma Pritchard, Mike Howell, Charlie Irving, David Johnson, Suk Kinch, Christopher Marshall, Andrew D. Lawrence, Stephan Blinder, Vesna Sossi, A. Jon Stoessl, Paul Skinner, and Steven S. GillPipeline Presenter: Erich Mohr, PhD, R.Psych

12 LISURIDE-A DRUG WITH OUTSTANDING AFFINITY FOR MONOAMINE RECEPTORSReinhard Horowski, MD, PhDPipeline Presenter: Reinhard Horowski, MD, PhD

13 THE CLINICAL DEVELOPMENT AND THERAPEUTIC POTENTIAL OF AXS-05 FOR NEUROPSYCHIATRIC DISORDERSCedric O'Gorman, Amanda Jones, Kellie Kennon, Herriot TabuteauPipeline Presenter: Cedric O'Gorman, MD

14 IDENTIFYING EARLY CLINICAL DEVELOPMENT PRIORITIES FOR DISEASE-MODIFYING THERAPIES IN NEURODEGENERATIVE DISEASESLudy Shih, Miroslaw Brys; Laura Fanning; Tara Fox; Beth Hirschhorn; Shavy Makh; Minhua Yang; James Xiao; Karl Evans; Natasha Penner; Danielle Graham; George Nomikos; Jesse CedarbaumPipeline Presenter: Ludy Shih, MD

15 PRECLINICAL THROUGH EARLY-STAGE CLINICAL DEVELOPMENT OF A NOVEL NMDA RECEPTOR MODULATOR, NYX-2925David Houck, PhDPipeline Presenter: David Houch, PhD

16 2DG UPDATE: ANTICONVULSANT ACTIONS, NEUROPROTECTION, AND PREVENTION OF POST-TRAUMATIC EPILEPSY Thomas Sutula, MD, PhDPipeline Presenter: Thomas Sutula, MD, PhD

17 GALR2-PREFERRING GALANIN ANALOGS: EFFICACY IN PAIN AND EPILEPSY MODELSCameron Metcalf, Grzegorz Bulaj, H. Steve WhitePipeline Presenter: Cameron Metcalf, PhD

18 GANAXOLONE, AN INVESTIGATIONAL NEUROSTEROID TREATMENT FOR CHILDREN WITH CDKL5 DEFICIENCY DISORDER: RESULTS FROM A PHASE 2 TRIAL.Michael Rogawski, MD, PhDPipeline Presenter: Michael Rogawski, MD, PhD

19 BPN14770 CLINICAL DEVELOPMENT FOR FRAGILE X SYNDROMEMark Gurney, Michael Tranfaglia MD, Patrica Cogram PhD, Scott Reines MD PhD, Elizabeth Berry-Kravis MD PhDPipeline Presenter: Mark Gurney, PhD

20 CIMIT: A UNIQUE MODEL FOR ACCELERATING HEALTHTECH INNOVATIONSSteven Schachter, John A. Parrish, Michael K. Dempsey, Diane Spiliotis, John CollinsPipeline Presenter: Steven Schachter, MD

Posters

SCIENTIFIC RESEARCH POSTERS21 A NOVEL FORMULATION OF HUPERZINE A, A NEW ANTICONVULSANT

Steven Schachter, Stephen Collins, Joshua Johnstone

22 IS NEUROLOGICAL DISEASE DUE TO DYSFUNCTIONAL BRAIN OSCILLATORS?Robert Black, PhD

23 PHARMACODYNAMIC PHMRI WITH JOINT ESTIMATION OF THE ARTERIAL DRUG INPUT CURVE Molly Charney, Jonathan M. Koller; Brad Miller; Lauren A. Marks; Kevin J. Black

24 NEUROPROTECTIVE METHLTHIAZOLE DERIVATIVES-MECHANISMS AND COMPARATIVE EFFICACY Doug Cowart, Sue H. Lee, Manel BenAissa, Jia Luo, Gregory Thatcher

25 USE OF IPSC-DERIVED HUMAN MOTOR NEURONS IN HIGH-THROUGHPUT PHENOTYPIC SCREENING Shouming Du, Michael Hendrickson, Zhong-Wei Du, Jennifer Kouznetsova, Wei Zheng

26 HOW THE EPILEPSY FOUNDATION SUPPORTS RESEARCH Sonya Dumanis, PhD, Brandy E. Fureman, PhD

27 PHARMACOKINETICS OF CLOBAZAM ORAL SOLUBLE FILM Allen H. Heller, Stephen Wargacki, Cassie Jung, David J. Wyatt, A. Mark Schobel

28 POPULATION PHARMACOKINETIC MODELING OF DIAZEPAM BUCCAL SOLUBLE FILM Allen H. Heller, Stephen Wargacki, Cassie Jung, David J. Wyatt, Guillaume Bonnefois, Olivier Barrière, Pierre-Olivier Tremblay, A. Mark Schobel

29 SCUBA: SUBGROUP BASED BAYESIAN ADAPTIVE DESIGNS FOR PRECISION CLINICAL TRIALS

30 NNI-351, A FIRST-IN-CLASS CANDIDATE FOR DISEASE-MODIFYING ADHD TREATMENT Judith Kelleher-Andersson, Matthew Wells

31 THERAPEUTIC EXPOSURES OF CX-8998, A POTENT, SELECTIVE AND STATE DEPENDENT CAV3 CHANNEL ANTAGONIST WITH DOSE DEPENDENT EFFICACY IN CAV3 DRIVEN NEUROLOGICAL MODELS Margaret Lee, Spyros Papapetropoulos MD, PhD Evan Newbold PhD

32 THE KEY ROLES OF NIGELLA SATIVA-OIL IN REGULATING MICROGLIA AND MITOCHONDRIAL ACTIVITY IN PARKINSONISM Tafheem Malik, Tasneem Fatima, Sheem Hasan, Shahid Pervez, Darakhshan Jabeen Haleem

33 ANAVEX2-73, A SIGMA-1 RECEPTOR AGONIST, TAKING AIM AT PRECISION MEDICINE TO TREAT NEUROLOGICAL DISORDERS Christopher Missling, Emmanuel O Fadiran, Daniel Klamer; Nell Rebowe

34 A PHASE I/II TRIAL OF A HEPATOCYTE GROWTH FACTOR (HGF) DNA PLASMID FOR THE TREATMENT FOR ALS

35 CX-8998: A NOVEL, STATE-DEPENDENT CAV3 CHANNEL ANTAGONIST IN PHASE 2 DEVELOPMENT FOR TREMOR AND EPILEPSY Spyr Papapetropoulos , Stacey Boyer, PhD Margaret Lee, PhD

SCIENTIFIC RESEARCH POSTERS36 NETWORK FOR EXCELLENCE IN NEUROSCIENCE CLINICAL TRIALS,

NEURONEXT: UPDATE ON SUCCESS AND FIVE-YEAR RENEWAL APPLICATION Julie Qidwai, Marianne Kearney Chase, Dixie Ecklund, Mike Bosch, Brenda Thornell, Christopher Coffey, Merit Cudkowicz

37 MULTI-MODAL RETENTION IN A PHASE III CLINICAL TRIAL IN EARLY PARKINSON DISEASE (STEADY-PD III) Tanya Simuni, Robert Holloway, Karen Hodgeman, Brittany Greco, Susan Henderson, Jillian Lowell, Saloni Sharma, Christopher G. Tarolli, and Kevin Biglan on behalf of the STEADY-PD III Investigators of the Parkinson Study Group

38 TH1 CYTOKINES, SECRETION IN DEMENTIA: CORRELATION WITH TYPE AND SEVERITY OF DISEASE. Benhamin Sredni, Kenigsbuch-Sredni D., Huberman M.

39 A PHASE IIA, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, 3-ARM PARALLEL-GROUP, MULTICENTER STUDY WITH ACTIVE IMMUNOTHERAPEUTIC VACCINE UB-311 IN PATIENTS WITH MILD ALZHEIMER'S DISEASE A. Verma, H. J. Yu, P. N. Wang, M. J. Chiu, C. C. Huang, C. C. Chang, H.C. Chen, C. Y. Wang

40 INTRAVENOUS ALLOPREGNANOLONE FOR TREATMENT OF STATUS EPILEPTICUS: SAFETY AND PHARMACOKINETICS IN DOGS Vuu, I, Coles LD, Leppik IE, Rogawski MA, Zolkowska D, Wu C-Y, Patterson EE, Cloyd JC

41 DETECTION OF MEMORY AND COGNITIVE IMPAIRMENT IN TYPE II DIABETIC PATIENTS: A COMPARISON OF MEMTRAX, AD8 AND MMSE IN AN OLD CHINESE POPULATION Guoqiang Xing, Liang Zhou, Qi Zhang, Mengjie Cai, Xi Bai, Zhiwei Guo, Qiwen Mu, Han Zhang, Yuzhang Zhao, Xuezhu Huang, Mingming Ding, John S. Lin, Xianbo Zhou, J. Wesson Ashford

42 VALPROIC ACID DERIVATIVES VALNOCTAMIDE AND SEC-BUTYLPROPYLACETAMIDE TERMINATE TETRAMETHYLENEDISULFOTETRAMINE-INDUCED STATUS EPILEPTICUS MORE RAPIDLY THAN DIAZEPAM Dorota Zolkowski, Meir Bialer, Carson W. Flamm, Nancy Garibay, David Bibi, Michael A. Rogawski, Ein Karem

Posters

Recognition

ASENT 2018 PRESIDENTIAL AWARDMICHAEL OKUN, MD

Michael S. Okun, M.D., Adelaide Lackner Professor and Chair of Neurology, and Co-director of the Fixel Center for Neurological Diseases at the University of Florida Health College of Medicine, will be honored with the 2018 Presidential Award at the ASENT 20th Annual Meeting, in Rockville, MD, on Saturday, March 10, 2018 at 10:15 am.

Hailed as ASENT’s highest honor to an individual, the 2018 ASENT Presidential Award recognizes extraordinary accomplishments in the field of clinical translational research. Michael S. Okun received his M.D. with honors from the University of Florida. He was fellowship trained by Mahlon DeLong, Jerrold Vitek and Ray Watts at Emory University in Atlanta GA before moving to found the movement disorders program at the University of Florida. He is currently Chair of Neurology, Professor and Co-director of the Fixel Center for Neurological Diseases at the University of Florida Health College of Medicine. The UF center he co-founded with Kelly D. Foote, M.D. is unique in that it is comprised of over 50 interdisciplinary faculty members from diverse areas of campus, all of whom are dedicated to care, outreach, education and research. Dr. Okun was instrumental in the construction of a one-stop patient-centered clinical-research experience for national and international patients seeking care at the University of Florida. This change in care and research delivery has since been named the service and science hub model of care. The UF based center draws national and international visitors interested in deploying this innovative clinical-research model. Dr. Okun has served as the National Medical Director for the Parkinson’s Foundation since 2006 and as the Medical Advisor for Tyler’s Hope for a Dystonia Cure. He has been supported by grants from the National Institutes of Health, the Smallwood Foundation, the Tourette Association of America, the Parkinson Alliance, the Bachmann-Strauss Foundation, the Parkinson’s Foundation, and the Michael J. Fox Foundation. Dr. Okun has an active research career exploring non-motor basal ganglia brain features and currently holds two NIH R01 grants and several foundation grants exploring various aspects of deep brain stimulation and neuromodulation. Dr. Okun has been an integral part of some of the pioneering studies exploring the cognitive, behavioral, and mood effects of brain stimulation and since 2005 his laboratory has been working to uncover the electrical brain signals associated with human tic. He has also partnered with Dr. Ayse Gunduz and Kelly Foote to develop a first generation of closed loop adaptive deep brain stimulation approaches. Dr. Okun was the founding PI for the International Database and Public Registry for Tourette Deep Brain Stimulation. He and his group have contributed data to support the FDA approval of several device related approaches now used to treat human disease. Dr. Okun holds the Adelaide Lackner Professorship in Neurology and has published over 400 peer-reviewed articles. He is a poet (Lessons From the Bedside, 1995) and his book, Parkinson's Treatment: 10 Secrets to a Happier Life, was translated into over 20 languages. Dr. Okun was recognized in a 2015 White House ceremony by the Obama administration as a Champion of Change for Parkinson’s Disease.A formal presentation of the award will take place at the ASENT 20th Annual Meeting just prior to Dr. Okun’s symposium on Saturday, March 10, 2018 at 10:15 am, at the Hilton Washington DC/Rockville Hotel and Executive Center in Rockville, MD.

ASENT 2018 CHAMPION AWARDEISAI, Inc.

EISAI, Inc., will be honored with the 2018 Champion Award at the ASENT 20th Annual Meeting, in Rockville, MD, on Thursday, March 8, 2018 at 6:00 pm at the start of the Dinner Symposium.

Hailed as ASENT’s highest honor to a company or organization, the 2018 ASENT Champion Award recognizes extraordinary dedication to the field of neurotherapeutics.

Eisai is a fully integrated pharmaceutical business that operates in two global business groups: oncology and neurology (dementia-related diseases and neurodegenerative diseases). Each group functions as an end-to-end global business with discovery, development, and marketing capabilities. Our U.S. headquarters, commercial and clinical development organizations are located in New Jersey; our discovery labs are in Massachusetts and Pennsylvania; and our global demand chain organization resides in Maryland and North Carolina. To learn more about Eisai Inc., please visit us at www.eisai.com/US.

A formal presentation of the award will take place at the ASENT 20th Annual Meeting just prior to the Dinner Symposium on Thursday, March 8, 2018 at 6:00 pm, at the Hilton Washington DC/Rockville Hotel and Executive Center in Rockville, MD.

Recognition

Annual Business Meeting

SaturdayMarch 10, 2018

Hilton Washington DC/Rockville Hotel & Executive Meeting Center

Plaza II/II7:30 - 8:00 AM

1. Call to Order2. Support Recognition3. Financial Report4. Executive and Board Member

Recognition5. Neurotherapeutics Journal Report6. Membership Report7. Important Dates for 2019 Annual

Meeting

Corporate Partners

At Janssen, we seek answers to some of the toughest questions in medicine. We believe nothing is more powerful than collaboration. There should not be barriers in the pursuit of groundbreaking treatments.

Collaboration goes beyond new treatments. From early discovery to access and distribution, we seek partners who want the same things we do: better outcomes for our patients. Our mission drives us. Our patients inspire us. We collaborate with the world for the health of everyone in it.

Learn more at www.janssen.com

Collaboration.Now that’s what we call a medical breakthrough.

Corporate Partners

For nearly 100 years, Upsher-Smith Laboratories, LLC has been a valued, dedicated partner in healthcare. Now backed by the resources of our new Japanese parent company Sawai Pharmaceutical Co., Ltd., we’ll continue striving to be a trusted source for quality, affordable generic and branded medications that bring confidence to patients and families, create better health outcomes, and lower the cost of care.

Our two companies share a strikingly similar family history and hold many of the same cherished goals and values—most importantly, the philosophy of always putting patients first. Together we hope to leverage each other for growth worldwide, and embark on an exciting new chapter.

Learn more about us at www.upsher-smith.com

YOUR PARTNERIN HEALTHCARE

110848-03-CO-General-ASENT-Mar2018.indd 1 2/22/18 10:04 AM

Corporate Partners

KJob Number: 22307Revision Nm: 0Date: 02/21/18

YMC199-54467 Pg 1

YESTERDAY A legacy rooted in unlocking the potential of cannabinoid medicines to address rare conditions with limitedtreatment options.

TODAY One of the largest clinical trial programs to date researching cannabinoids to treat rare forms of pediatric-onset epilepsy.

TOMORROW A promise to continue advancing cannabinoid science and providing medications that have the potential to address the unmet needs of patients.

GREENWICH Biosciences is a biopharmaceutical company developing novel medications in a broad range of disease states, with a focus on Dravet syndrome, Lennox-Gastaut syndrome, tuberous sclerosis complex, and infantile spasms.

To learn more, visit www.greenwichbiosciences.com.

Lilly W.Living with Epilepsy

Unmatched long-term patient satisfaction.*

*Survey of 7,000 commercially treated HF10 therapy patients. 2018013 Rev. A

Corporate Partners

ACADIA is a biopharmaceutical company focused on the development and commercialization of innovative medicines that address unmet medical needs in central nervous system disorders. ACADIA maintains a website at www.acadia-pharm.com to which we regularly post copies of our press releases as well as additional information and through which interested parties can subscribe to receive e-mail alerts.

Biocodex is an independent, family-owned pharmaceutical company founded in Paris, France, in 1953. The company’s initial focus was gastroenterology, and specifically, a promising probiotic discovery, Saccharomyces boulardii (marketed under the brand name Florastor® in the United States). Biocodex has partnered with healthcare professionals for 60 years, taking on the mission of developing meaningful solutions to today’s challenging healthcare problems. Biocodex has grown into a multinational research and development, manufacturing and commercial enterprise. The company currently operates in over 100 countries with subsidiaries in Belgium, Russia, Turkey, Morocco, the United States, and will be opening the Canadian subsidiary in July, 2013. Today, Biocodex specializes internationally in the therapeutic areas of gastroenterology, neuropsychiatry and pain treatment.

At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases. Founded in 1978 as one of the world’s first global biotechnology companies by Charles Weissman, Heinz Schaller, Kenneth Murray and Nobel Prize winners Walter Gilbert and Phillip Sharp, today Biogen has the leading portfolio of medicines to treat multiple sclerosis; has introduced the first and only approved treatment for spinal muscular atrophy; and is focused on advancing neuroscience research programs in Alzheimer’s disease and dementia, multiple sclerosis and neuroimmunology, movement disorders, neuromuscular disorders, pain, ophthalmology, neuropsychiatry, and acute neurology. Biogen also manufactures and commercializes biosimilars of advanced biologics.

Corporate Partners

Eisai Co., Ltd. is a leading global research and development-based pharmaceutical company headquartered in Japan. We define our corporate mission as “giving first thought to patients and their families and to increasing the benefits health care provides,” which we call our human health care (hhc) philosophy. With approximately 10,000 employees working across our global network of R&D facilities, manufacturing sites and marketing subsidiaries, we strive to realize our hhc philosophy by delivering innovative products to address unmet medical needs, with a particular focus in our strategic areas of Neurology and Oncology.

Greenwich Biosciences was established in 2013 as the US subsidiary of GW Pharmaceuticals plc. We are advancing an orphan drug program in the field of epilepsy with a focus on Epidiolex®(cannabidiol), which is in Phase 3 clinical development for the treatment of Dravet syndrome, Lennox-Gastaut syndrome, Tuberous Sclerosis Complex and expects to commence a Phase 3 program in Infantile Spasms.

GT Biopharma is a clinical stage biopharmaceutical company focused on immuno-oncology and central nervous system products

At the Janssen Pharmaceutical Companies of Johnson & Johnson, we are working to create a world without disease. Transforming lives by finding new and better ways to prevent, intercept, treat and cure disease inspires us. We bring together the best minds and pursue the most promising science.

KM Pharmaceutical Consulting provides Project Management services to Biotechnolgy and Pharmaceutical companies.

Medtronic plc (www.medtronic.com), headquartered in Dublin, Ireland, is among the world's largest medical technology, services and solutions companies - alleviating pain, restoring health and extending life for millions of people around the world. Medtronic employs more than 84,000 people worldwide, serving physicians, hospitals and patients in more than 160 countries. The company is focused on collaborating with stakeholders around the world to take healthcare Further, Together.

Corporate Partners

Neurelis, Inc. is a privately-held San Diego-based specialty pharmaceutical company organized to license, develop, and commercialize product candidates for epilepsy and the broader central nervous system (CNS) market. The lead product for Neurelis is NRL-1, a treatment of acute repetitive or cluster seizures. NRL-1 (intranasal diazepam) is a proprietary formulation of diazepam, delivered via a nasal formulation in a spray, being developed for the management of pediatric and adult patients who require intermittent use of diazepam to control bouts of acute repetitive seizure activity, also known as cluster seizures. NRL-1 has been granted Orphan Drug and Fast Track Designations by the FDA. In clinical trials, NRL-1 has demonstrated high bioavailability, low variability from dose to dose, and was well-tolerated – with reliable dosing, regardless of when administered.

Neurocrine Biosciences is a San Diego based biotechnology company focused on neurologic, psychiatric and endocrine related disorders. The Company markets INGREZZA® (valbenazine) capsules in the United States for the treatment of adults with tardive dyskinesia. INGREZZA is a novel, selective vesicular monoamine transporter 2 (VMAT2) inhibitor, and is the first FDA approved product indicated for the treatment of adults with tardive dyskinesia. The Company's three late-stage clinical programs are: elagolix, a gonadotropin-releasing hormone antagonist for women's health that is partnered with AbbVie Inc.; opicapone, a novel, once-daily, peripherally-acting, highly-selective catechol-o-methyltransferase inhibitor under investigation as adjunct therapy to levodopa in Parkinson's patients; and INGREZZA, a novel, once-daily, selective VMAT2 inhibitor under investigation for the treatment of Tourette syndrome.

NeuroPace is an innovative medical device company dedicated to improving quality of life for individuals with medically refractory partial onset seizures. In addition to treating epilepsy, responsive neurostimulation holds the promise of treating other disabling neurological, psychiatric, and chronic disorders that negatively impact quality of life for millions of patients throughout the world.

Corporate Partners

Headquartered in Redwood City, California, Nevro is a global medical device company focused on providing innovative products that improve the quality of life of patients suffering from debilitating chronic pain. Nevro has developed and commercialized the SENZA® spinal cord stimulation (SCS) system, an evidence-based, non-pharmacologic neuromodulation platform for the treatment of chronic pain. The SENZA® system is the only SCS system that delivers Nevro's proprietary HF10™ therapy. Senza, Senza II, HF10, Nevro and the Nevro logo are trademarks of Nevro.

Riverside is a specialty pharmaceutical company dedicated to improving the treatment of Parkinson's disease and related disorders

From our inception in 1957 to the present, we have focused solely on intellectual property law. We handle every aspect of IP law, and we have kept this area as our exclusive focus as a way to ensure that we provide the highest possible expertise to our clients. We have obtained more U.S. patents than any other law firm in the world, and we have successfully litigated IP disputes in every popular forum, including U.S. District Courts across the country, the United States International Trade Commission, (USITC), the Patent Trial and Appeal Board (PTAB), and the Trademark Trial and Appeal Board (TTAB).

Upsher-Smith Laboratories, LLC is a trusted U.S. pharmaceutical company that has strived to deliver quality, affordable generic medications for nearly a century. In June 2017, Upsher-Smith was acquired by Sawai Pharmaceutical Co., Ltd., a large publicly traded generics company in Japan that shares a similar philosophy of always putting patients first.

Zogenix is committed to developing and commercializing therapies for central nervous system disorders that address specific clinical needs for people living with rare diseases and other CNS disorders who need innovative treatment alternatives to improve their daily functioning.

Floor Plan

Leadership

Officers

PresidentMartha Morrell, MDNeuroPace, Inc.

President ElectThomas P. Sutula, MD, PhDUniversity of Wisconsin, Madison

Immediate Past PresidentHoward J. Federoff, MD, PhDUniversity of California, Irvine

SecretaryRobin A. Elliott, MAParkinson’s Disease Foundation & World Parkinson’s Congress

TreasurerRobert W. Hamill, MDUniversity of VermontCollege of MedicineDepartment of Neurological Sciences

Directors

Amy S. Chappell, M.D. Naples, FL

Timothy Coetzee, PhD National Multiple SclerosisSociety

O’Neill D’Cruz, MD, MBA,FAAN OD Consulting and Neurological Services, PLLC

Stewart A. Factor, DOEmory University School of Medicine

Lynn D. Kramer, MD Eisai, Inc.

Victor Krauthamer, PhD Food and Drug Administration

Bennett L, Lavenstein, MD Co-ChairChildren’s Hospital Medical Center

Toby Ferguson, MD, PhD Biogen Idec, Inc

Steven Schachter, MD CIMIT/Harvard Medical School

John R. Richert, MD Richert Partners

Ex-Officio Board Members

NIH Blueprint Course Liaison Barbara Slusher, PhD, MASJohn Hopkins Brain Science Institute

Neurotherapeutics®Editor-in-ChiefM. Maral Mouradian, MD Rutgers University

Program Committee Co-ChairC. Anthony Altar, PhD Verge Genomics

CommunicationsTerrie Blalock, RN, MSTBlalock Consulting

Federal Liaison Amir Tamiz, PhD NIH, NINDS, OTR

David Shoup The Curry Rockefeller Group

Members

Co-ChairC. Anthony Altar, PhD Verge Genomics

Bennett L, Lavenstein, MD Co-ChairChildren’s Hospital Medical Center

Mitchell Brin, MDAllergan, Inc.

Enrique Carrazana, MDMarinus Pharmaceuticals

Amy Chappell, MDNaples, FL

Kathleen Clarence-Smith, MD, PhDKM Pharmaceuticals Consulting, LLC

Robin Elliott, MAParkinson’s Disease Foundation (retired)

Stewart A. Factor, DOEmory University School of Medicine

Howard J. Federoff, MD, PhDUniversity of California, Irvine

Robert Hamill, MDUniversity of Vermont

Korie Handwerger, PhDRA Capital Management

Petra Kaufmann, MD, MScNCATS

Peter KolchinskyRA Capital Management

Victor KrauthamerFDA (retired)

Patrick Mabray, MD, PhDBoston Medical Center

Martha Morrell, MDNeuroPace, Inc.

Jill G.C. Rasmussen, MBchB, FRCGP, FFPMPsi-Napse

Thomas Sutula, MD, PhDUniversity of Wisconsin, Madison

Dwain Tolbert, PhD, FcPLundbeck, Inc.

Program Committee

Board of Directors

Attendees

Reem Al Olaby, MDUC Davis MIND Instituteralolabi@ucdavis.edu

C. Anthony Altar, PhDVerge Genomics, Inc.tony@vergegenomics.com

Johnathon AndersonSomos Therapeuticsjohnathon.d.anderson@gmail.com

Erika Augustine, MDUniversity of Rochester Medical Centererika_augustine@urmc.rochester.edu

Gai Ayalongai_ayalon@yahoo.com

James BarrowJohns Hopkins Drug Discoveryjames.barrow@libd.org

Ron BartekFARA - Friedreich's Ataxia Research Alliancefara@curefa.org

Manel Ben-AissaCOLLEGE OF PHARMACY (UIC)benaissa@uic.edu

C. Frank Bennett, PhDIonis Pharmaceuticals, Inc.fbennett@ionisph.com

Bernard BerneFDABernard.Berne@Fda.Hhs.Gov

Pradeep Bhide, PhDFlorida State UniversityPradeep.Bhide@Med.Fsu.Edu

Robert Black, PhDScion NeuroStimrblack@scionneurostim.com

Kevin Black, MDWashington University in St. Louiskevin@wustl.edu

Terrie Blalock, RNTBlalock and Associatestblalock1@nc.rr.com

Bennett Blumenkopf, MDFDABENNETT.BLUMENKOPF@FDA.HHS.GOV

Mitchell Brin, MDAllergan, Inc.brin_mitchell@allergan.com

Carole BurnsJohns Hopkins Technology Venturescburns21@jhu.edu

Christopher Butson, PhDUniversity of Utahbutson@sci.utah.edu

Gregory CarrLieber Institute for Brain Developmentgreg.carr@libd.org

Enrique Carrazana, MDNeurelisecarrazana@aol.com

Susan CatalanoCognition Therapeuticsscatalano@cogrx.com

Johnny Cebak, PhD, OMS-IIILincoln Memorial University - Debusk College of Osteopathic Medicinejohn.cebak@gmail.com

Stanley Chamberlain, PhDT3D Therapeutics, Inc.stanchamberlain@t3dtherapeutics.com

Amy Chappell, MDaschappell5@gmail.com

Molly CharneyWashington University in St. Louismcharney@wustl.edu

Thomas N. Chase, MD Chase Pharmaceuticals, Inc.tchase@chasetherapeutics.com

Jeffrey ChildersMichael Bestjwchilders@michaelbest.com

Kathleen E. Clarence Smith, MD, PhDGT-Pharmaceuticalskcsmith@kmphc.com

Jim CloydCenter for Orphan Drug Research, University of Minnesotacloyd001@umn.edu

Robin Conwit, MDNIH /NINDSrc296d@nih.gov

Chuck CywinNIH, NINDS, Office of Translational Researchcharles.cywin@nih.gov

O'Neill D'CruzOD Consulting and Neurological Services, PLLConeilldcruz@gmail.com

Bhaskar DasThe Icahn School of Medicine at Mount SinaiBhaskar.das@mssm.edu

Ranjeet Dash Johns Hopkins Drug Discoveryrdash5@jhmi.edu

Ronald DeMattos, PhDEli Lilly & Companydemattosrb@lilly.com

Mike DempseyCIMITmdempsey1@partners.org

Timothy Denison, PhDMedtronictimothy.denison@medtronic.com

Suhayl Dhib-Jalbut, MDRutgers Universityjalbutsu@rutgers.edu

Ray Dorsey, MDUniversity of Rochesterray.dorsey@chet.rochester.edu

Shouming DuBRAINXELL, INCsdu@brainxell.com

Sonya DumanisEpilepsy Foundationsdumanis@efa.org

Debra Ehrlich, MDNational Institutes of Health/NINDS - CounterActdebra.ehrlich@nih.gov

Robin Elliott, MAParkinson's Disease Foundation & World Parkinson Congressrelliott@pdf.org

Salvatore Enna, PhDKansas Univeristy Medical Centersenna@kumc.edu

Stewart Factor, DOEmory University School of Medicinesfactor@emory.edu

Gail FarfelZogenix, Inc.gail@farfel.com

Howard Federoff, MD, PhDUniversity of California - Irvinekellylb@uci.edu

as of February 26, 2018

Daniel Felsing, MDUniversity of Texas Medical Branchdefelsin@utmb.edu

Toby Fergusontoby.ferguson@biogen.com

Stephanie FertigNIH, NINDS, Office of Translational Researchstephanie.fertig@nih.gov

Frank FischerNeuroPace, Inc.ffischer@neuropace.com

Carson FlammUC Davis Medical Centercw.flamm@gmail.com

Eric FrancaFDAERIC.FRANCA@FDA.HHS.GOV

Lawrence Friedhoff, MD, PhD, FACPRoivant Sciences, Inc.lawrence.friedhoff@roivant.com

Micheal GallagherAgeneBio, Incmichela.gallagher@agenebio.com

Michael Gold, MD, MSAbbVie, Inc.michael.gold@abbvie.com

Steven GoldmanUniversity of Rochestersteven_goldman@urmc.rochester.edu

Margaret Grabbmerriwetherr@mail.nih.gov

Patricia A. Grady, PhDNational Institute of Nursing Research (NINR)info@ninr.nih.gov

Kevin Gray, MDMedical University of South Carolinagraykm@musc.edu

Joel Greenspan, PhDUniv. of Maryland, Baltimorejgreenspan@umaryland.edu

Joshua Grilljgrill@uci.edu

Mark Gurney, PhDTetra Discovery Partnersmark@tetradiscovery.com

Stacie Gutowski, PhDFDASTACIE.GUTOWSKI@FDA.HHS.GOV

Yomayra Guzman Vazquez, PhDNew York School of Medicineyomy.guzman@nyumc.org

Mark Hallett, MDNational Institutes of Health/NINDS - CounterActhallettm@ninds.nih.gov

Robert W. Hamill, MDUniversity of Vermont College of Medicinerobert.hamill@med.uvm.edu

Korie HandwergerRA Capital Management, LLCkhandwerger@racap.com

Jessy Hartjessyhart187@gmail.com

Lauren Harte-Hargrove, PhDCure, Citizens United For Research In EpilepsyLauren.Harte@cureepilepsy.org

Dietrich Haubenberger National Institute of Neurological Disorders and Stroke/NIHhaubenbergerd@ninds.nih.gov

Philip HaydonGliaCure, Incphilip.haydon@gliacure.com

Allen H Heller, MD, MPHPharma Study Design, LLCahheller2013@gmail.com

Freddie Ann Hoffman, MDHeteroGeneity, LLCfhoffman@consulthg.com

Michael HoffmannFDAMICHAEL.HOFFMANN@FDA.HHS.GOV

David Houck, MDAptinyxdavidhouck@aptinyx.com

James Inglesejinglese@mail.nih.gov

William JanzenEpizyme, Incwjanzen@epizyme.com

Rasmus Stig Jensenrsj@uneeg.com

Rasmus Stig JensenUNEEG medicalks@uneeg.com

Yuan JiNorthShore University HealthSystmemkoaeraser@gmail.com

Velvet JourniganMarshall University journigan@marshall.edu

Michael Kaplitt, MD PhDWeill Cornell Medicinened2011@med.cornell.edu

Daniel Karlin, MDPfizer Incdaniel.karlin@pfizer.com

Barbara KarpNational Institute of Neurological Disorders and Stroke/NIHkarpb@ninds.nih.gov

Petra KaufmannNational Center for Advancing Translational Sciences/NIHpetra.kaufmann@nih.gov

Erin KeeganFDAERIN.KEEGAN@FDA.HHS.GOV

Matthew KennedyMerck Research Labsmatthew.kennedy@merck.com

Gaspar KitangeMayo Clinickitange.gaspar@mayo.edu

Winston KoGenervon Biopharmaceuticals LLCinfo@genervon.com

Dorothy KoGenervon Biopharmaceuticals LLCdko@genervon.com

Ivan Kotchetkovivan@jhmi.edu

Attendees

Rajaraman KrishnanProclara Biosciencesrkrishnan@proclarabio.com

Meredith KuoUniversity of California, San Franciscoszuyu.kuo@ucsf.edu

Linda KusnerGeorge Washington Universitylkusner@gwu.edu

Pascal Laeng, PhDNIH, NINDS, Division of Translational Researchpascal.laeng@nih.gov

Warren LammertGranite Point Capitalwarren@granitepoint.com

Nicholas LanghalsNIH/National Institute of Neurological Disorders and Strokenick.langhals@nih.gov

Nicholas LanghalsNIH/National Institute of Mental Healthnick.langhals@nih.gov

Bennett Lavenstein, MDChildren's Hospital Medical Centerblavenst@childrensnational.org

Robert LisakWayne State Universityrlisak@med.wayne.edu

Naomi LomeliUniversity of California - Irvinenrlomeli@uci.edu

Patrick Mabray, MD, PhDBoston Medical Centerpatrick.mabray@bmc.org

A. Hadi Maghzi, MDCedars Sinai Medical Centerhadi.maghzi@gmail.com

William Marks, Jr.Verily Life Scienceswjmarks@google.com

Kennan Marsh AbbVie, Inc.kennan.marsh@abbvie.com

Kennan MarshAbbVie, Inc.kennan.marsh@abbvie.com

David McMullen, MDNIH/NIMHdavid.mcmullen@nih.gov

Cynthia McMurrayLawrence Berkeley Labctmcmurray@lbl.gov

Carolina Mendoza-Puccini, MDcarolina.mendoza-puccini@nih.gov

Cameron Metcalf, PhDUniversity of Utahcameron.s.metcalf@utah.edu

Jeffrey Meteer, PhDAquestive Therapeuticsjmeteer@aquestive.com

Steven Miller, PhDGreenwich Biosciencesslmiller@greenwichbiosciences.com

Erich Mohr, PhD, R PsychMedGenesis Therapeutix, Inc.erichmohr@medgenesis.com

Martha Morrell, MDNeuroPace, Inc.mmorrell@neuropace.com

Matthew Moschitto, Northwestern Universitymatthew.moschitto@northwestern.edu

M. Maral MouradianRutgers-Robert Wood Johnson Medical Schoolm.mouradian@rutgers.edu

Frank NagyAquestive Therapeuticsnagyfr@gmail.com

Prabagaran NarayanasamyUNMCp.narayanasamy@unmc.edu

Avindra Nath, MDNational Institutes of Health (NIH)avindra.nath@nih.gov

Anoo NathanSmart Monitoranoo@smart-monitor.com

Marie-Laure Nevoret, MDVM BioPharmanevoret@viromed.co.kr

Attendees

Michael Okun, MDUniversity of Florida College of Medicineokun@neurology.ufl.edu

Quasar PadiathUniversity of Pittsburghqpadiath@pitt.edu

Mary Ann Pelleymounter, PhDNIH, NINDS, Division of Translational Researchtimothy.lyden@nih.gov

Stephen PeroutkaPPD, INC.drperoutka@gmail.com

Ronald Pfeiffer, MDOHSU Parkinson Center of Oregonpfeiffro@ohsu.edu

Rosalind PicardEmpaticarp@empatica.com

Sandra PizzoferratoASENTsandrapizzoferrato.asent@gmail.com

Roger J Porter, MDUniv of Pennsylvania; USUHSRJPorterMD@gmail.com

Diana Price, PhDNeuropore TherapiesdlpriceSD@gmail.com

Laszlo RadnaiThe Scripps Research Institutelradnai@scripps.edu

Beatriz Rocha, MDCovancebeatriz.rocha@covance.com

Nancy RogackiLundbeck LLCnrog@lundbeck.com

Michael Rogawski, MD PhDUniversity of California-Davisrogawski@yahoo.com

Laura RosenTakedalaura.rosen@takeda.com

Erika Ross, PhDCala Healtherika@calahealth.com

Cynthia Rothblum-Oviatt A-T Children's Projectcynthia@atcp.org

Lee RubinHarvard Universitylee_rubin@harvard.edu

David Rushworth, MDBaylor College of Medicinedavid.rushworth@bcm.com

Chris RyanRenovo Neural, Inc.cryan@renovoneural.com

Katie SaleAmerican Brain Coalitionksale@americanbraincoalition.org

Matt SalomonThe ALS Association - DC/MD/VA Chaptermsalomon@alsinfo.org

Nancy SantilliHuman Care Systemsnancysantilli01@gmail.com

Frank Sasinowski Hyman, Phelps & McNamara, P.C.fjs@hpm.com

Steven Schachter, MDCIMITsschacht@bidmc.harvard.edu

Karl ScheidtNorthwestern Universityscheidt@northwestern.edu

Ralf SchmidUniversity of Pennsylvania, School of Medicinersschmid@upenn.edu

Stefan SchwabeR W Johnson Research Institutesschwabe@supernus.com

David SegalUniversity of California, Davisdjsegal@ucdavis.edu

Joonil SeogFDAJOONIL.SEOG@FDA.HHS.GOV

Anjali Sharma, Postdoctoral ResearcherJohns Hopkins School of Medicineanjali.25sept@gmail.com

Sida ShenNorthwestern Universitysida.shen@northwestern.edu

Haiying Shenhshen@downeurobiology.org

Vivek ShenoyRenovo Neural, Inc.vshenoy@renovoneural.com

Ludy Shih, MDBiogen, Inc.ludyshih@gmail.com

Julia Shirvan, MD, DPhilBiogen Idec, Inc.jciampa@partners.org

Shai SilberbergNIH, NINDS, OTRsilberbs@ninds.nih.gov

Tanya SimuniNorthwestern University Feinberg School of Medicinetsimuni@nm.org

Barbara SlusherJohns Hopkins Drug Discoverybslusher@jhmi.edu

Adaline SmithIronwood Pharmaceuticalsasmith@ironwoodpharma.com

Kenneth Sommerville, MD, FAANSommerville Consultingkwsommerville@gmail.com

Edward Spack, PhDMedaRed Inc.egspack@comcast.net

TJ StalveyAquestive Therapeuticststalvey@aquestive.com

Knud StampeUNEEG medicalks@uneeg.com

Joseph Steiner, PhDNational Institutes of Health (NIH)joe.steiner@nih.gov

Ondrej StepanekJohn Hopkins Drug Discoveryostepan1@jhmi.edu

Laura StoppelRA Capital Management, LLClstoppel@racap.com

Thomas Sutula, MD, PhDUniversity of Wisconsin - Madisonsutula@neurology.wisc.edu

Carmen Tamayo, MDHeteroGeneity, LLCctamayo@consultHG.com

Amir TamizNational Institute of Neurological Disorders and Strokeamir.tamiz@nih.gov

Malu Tansey, PhDEmory University School of Medicinemalu.tansey@emory.edu

Susumu Tomita susumu.tomita@yale.edu

Richard TraubDept. Neural and Pain Sciences, Univ. MD School of Dentistryrtraub@umaryland.edu

Elizabeth Tucker, MDJohns Hopkins University School of Medicineetucker9@jhmi.edu

Christopher UnsworthYale University - Office of Cooperative Researchchristopher.unsworth@yale.edu

James Valentine, JD, MHSHyman, Phelps & McNamara, P.C.jvalentine@hpm.com

Gabriel VargasAmgen Incgavargas@amgen.com

Ajay VermaUnited Neuroscienceaverma@unitedneuroscience.com

Mark Versavel, MD, PhD, MBAvZenium LLCmark.versavel@vzenium.com

Jacob Vogelsteinjvogelstein@camdenpartners.com

Irene VuuUniversity of Minnesotavuuxx002@umn.edu

Attendees

Feng Wangfeng.wang@labiomed.org

Monica WeldonBridge the Gap - SYNGAP Education and Research Foundation Monicaw@bridgesyngap.org

Dhanya Williams, MSFDADHANYA.WILLIAMS@FDA.HHS.GOV

Clinton Wrightclinton.wright@nih.gov

Qingguo XuVirginia Commonwealth Universityqxu@vcu.edu

Haichen Yang, MDICON Plc.haichen.yang@iconplc.com

Sheila YiViroMed Co., Ltdsheila@viromed.co.kr

Jennifer Zimmer, MDEli Lilly and Companyzimmer_jennifer_ann@lilly.com

Save the Date

21st Annual Meeting March 5-8, 2019 Hilton Washington DC/Rockville Hotel & Executive Meeting Center

ASENT2019

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