Dravet Syndrome (Severe Myoclonic Epilepsy of Infancy) - Pipeline Review, H2 2016

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1 Dravet Syndrome (Severe Myoclonic Epilepsy of Infancy) - Pipeline Review, H2 2016

2 Dravet Syndrome (Severe Myoclonic Epilepsy of Infancy) - Pipeline Review, H BioPortfolio has been marketing business and market research reports from selected publishers for over fifteen years. BioPortfolio offers a personal service to our customers with dedicated research managers who will work with you to source the best report for your needs. Based in the UK, BioPortfolio is well positioned to coordinate our customers' orders sourced from over 50 global report publishers. We are pleased to present details of this report to assist your buying decision and administrative process. You will find easy-to-use How To Buy information on the last page of this document. We look forward to being of service to you. If you have bulk and/or recurring requirements, please get in touch - we can liaise with publishers to obtain sample pages and negotiate discounts on your behalf. Phone: +44 (0) or reportstore@bioportfolio.com 2

3 Dravet Syndrome (Severe Myoclonic Epilepsy of Infancy) - Pipeline Review, H Dravet Syndrome (Severe Myoclonic Epilepsy of Infancy) - Pipeline Review, H Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Dravet Syndrome - Pipeline Review, H2 2016, provides an overview of the Dravet Syndrome (Central Nervous System) pipeline landscape. Dravet syndrome, also known as severe myoclonic epilepsy of infancy (SMEI), is a rare and catastrophic form of intractable epilepsy that begins in infancy. Symptoms include ataxia (unsteadiness), sleep disturbance, and behavior problem. Report Highlights Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Dravet Syndrome - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Dravet Syndrome (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. The Dravet Syndrome (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Dravet Syndrome (Severe Myoclonic Epilepsy of Infancy) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 2, 2, 2, 2 and 1 respectively.dravet Syndrome. Dravet Syndrome (Central Nervous System) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis. Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data. Scope - The pipeline guide provides a snapshot of the global therapeutic landscape of Dravet Syndrome (Central Nervous System). - The pipeline guide reviews pipeline therapeutics for Dravet Syndrome (Central Nervous System) by companies and universities/research institutes based on information derived from company and industry-specific sources. 3

4 - The pipeline guide covers pipeline products based on several stages of development ranging from preregistration till discovery and undisclosed stages. - The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities. - The pipeline guide reviews key companies involved in Dravet Syndrome (Central Nervous System) therapeutics and enlists all their major and minor projects. - The pipeline guide evaluates Dravet Syndrome (Central Nervous System) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. - The pipeline guide encapsulates all the dormant and discontinued pipeline projects. - The pipeline guide reviews latest news related to pipeline therapeutics for Dravet Syndrome (Central Nervous System) Reasons to buy - Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies. - Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage. - Find and recognize significant and varied types of therapeutics under development for Dravet Syndrome (Central Nervous System). - Classify potential new clients or partners in the target demographic. - Develop tactical initiatives by understanding the focus areas of leading companies. - Plan mergers and acquisitions meritoriously by identifying key players and it s most promising pipeline therapeutics. - Formulate corrective measures for pipeline projects by understanding Dravet Syndrome (Central Nervous System) pipeline depth and focus of Indication therapeutics. - Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope. - Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline. Additional Details Publisher : Global Markets Direct Reference : GMDHC8745IDB Number of Pages : 75 Report Format : PDF Publisher Information : 4

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6 Table Of Contents for Dravet Syndrome (Severe Myoclonic Epilepsy of Infancy) - Pipeline Review, H Table of ContentsTable of Contents 2List of Tables 4List of Figures 5Introduction 6Global Markets Direct Report Coverage 6Dravet Syndrome (Severe Myoclonic Epilepsy of Infancy) Overview 7Therapeutics Development 8Pipeline Products for Dravet Syndrome (Severe Myoclonic Epilepsy of Infancy) - Overview 8Dravet Syndrome (Severe Myoclonic Epilepsy of Infancy) - Therapeutics under Development by Companies 9Dravet Syndrome (Severe Myoclonic Epilepsy of Infancy) - Pipeline Products Glance 10Late Stage Products 10Clinical Stage Products 11Early Stage Products 12Dravet Syndrome (Severe Myoclonic Epilepsy of Infancy) - Products under Development by Companies 13Dravet Syndrome (Severe Myoclonic Epilepsy of Infancy) - Companies Involved in Therapeutics Development 14Biscayne Pharmaceuticals Inc 14GW Pharmaceuticals Plc 15INSYS Therapeutics Inc 16OPKO Health Inc 17PTC Therapeutics Inc 18Sage Therapeutics Inc 19Xenon Pharmaceuticals Inc 20Zogenix Inc 21Dravet Syndrome (Severe Myoclonic Epilepsy of Infancy) - Therapeutics Assessment 22Assessment by Monotherapy Products 22Assessment by Target 23Assessment by Mechanism of Action 25Assessment by Route of Administration 27Assessment by Molecule Type 29Drug Profiles 31Antisense Oligonucleotide for Dravet Syndrome - Drug Profile 31Product Description 31Mechanism Of Action 31R&D Progress 31ataluren - Drug Profile 32Product Description 32Mechanism Of Action 32R&D Progress 32BIS Drug Profile 40Product Description 40Mechanism Of Action 40R&D Progress 40cannabidiol - Drug Profile 42Product Description 42Mechanism Of Action 42R&D Progress 42cannabidiol - Drug Profile 48Product Description 48Mechanism Of Action 48R&D Progress 48CUR Drug Profile 51Product Description 51Mechanism Of Action 51R&D Progress 51fenfluramine hydrochloride - Drug Profile 52Product Description 52Mechanism Of Action 52R&D Progress 52SAGE Drug Profile 55Product Description 55Mechanism Of Action 55R&D Progress 55Small Molecule to Block Nav1.6 for Dravet Syndrome - Drug Profile 57Product Description 57Mechanism Of Action 57R&D Progress 57Dravet Syndrome (Severe Myoclonic Epilepsy of Infancy) - Dormant Projects 58Dravet Syndrome (Severe Myoclonic Epilepsy of Infancy) - Product Development Milestones 59Featured News & Press Releases 59Nov 22, 2016: New Data on Zogenixs ZX008 for Lennox Gastaut Syndrome & Dravet Syndrome to be Presented at 70th Annual American Epilepsy Society Meeting 59Oct 24, 2016: Newly Published Data Show Biscayne Pharmaceuticals' Novel Antiepileptic Drug Can Eliminate Seizures In Models Of Dravet Syndrome 60Jun 28, 2016: Biscayne Pharmaceuticals Presents Data On Novel Antiepileptic Agent BIS-001 AT EILAT Conference On New Antiepileptic Drugs 61Jun 27, 2016: GW Pharmaceuticals Announces Positive Phase 3 Pivotal Trial Results for Epidiolex (cannabidiol) in the Treatment of Lennox-Gastaut Syndrome 62Jun 08, 2016: Xenon Pharmaceuticals Presents Update on Nav1.6 Sodium Channel Inhibitor at the Jefferies 2016 Healthcare Conference 64Jun 07, 2016: Zogenix Announces Initiation of Multi-National Phase 3 Clinical Trial for ZX008 in Dravet Syndrome 64May 05, 2016: Zogenix Announces ZX008 New Efficacy and Safety Data for Treatment of Seizures in Dravet Syndrome 65Apr 21, 2016: New Data on Zogenix ZX008 for Dravet Syndrome to be Presented at 14th International Child Neurology Congress 66Mar 14, 2016: GW Pharmaceuticals Announces Positive Phase 3 Pivotal Study Results for Epidiolex (cannabidiol) 66Feb 25, 2016: Edison Issues ADR Update on GW Pharmaceuticals 68Jan 19, 2016: Zogenix Receives Fast Track Designation From FDA for Development of ZX008 in Dravet Syndrome 68Jan 11, 2016: Zogenix Announces Initiation of Phase 3 Program for ZX008 in Dravet Syndrome 69Dec 14, 2015: Zogenix Announces FDA Acceptance of Investigational New Drug Application for ZX008 Phase 3 Clinical Trial in Dravet Syndrome 69Dec 07, 2015: Biscayne Pharmaceuticals Presents Data At 2015 AES Meeting Confirming BIS-001 Is A Novel Mechanism, Highly Potent And Well-Tolerated Anti-Epileptic Drug 70Dec 07, 2015: GW Pharmaceuticals Announces New Physician Reports of Epidiolex Treatment Effect in Children and Young Adults With Treatment- Resistant Epilepsy 70Appendix 74Methodology 74Coverage 74Secondary Research 74Primary Research 74Expert Panel Validation 74Contact Us 74Disclaimer 75 6

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