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Rett Syndrome - Pipeline Review, H1 2016 Report / Search Code: WGR495860
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Publish Date: 30 April, 2016
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Enterprise P DF : $ 6000.0
Rett Syndrome - Pipeline Review, H1 2016 Summary Global Markets Direct’s , ‘Rett Syndrome - Pipeline Review, H1 2016’, provides an overview of the Rett Syndrome pipeline lands cape. The report provides comprehens ive information on the therapeutics under development for Rett Syndrome, complete with analys is by s tage of development, drug target, mechanis m of action (MoA), route of adminis tration (RoA) and molecule type. The report als o covers the des criptive pharmacological action of the therapeutics , its complete res earch and development his tory and lates t news and pres s releas es . Additionally, the report provides an overview of key players involved in therapeutic development for Rett Syndrome and features dormant and dis continued projects . Global Markets Direct’s report features inves tigational drugs from acros s globe covering over 20 therapy areas and nearly 3,000 indications . The report is built us ing data and information s ourced from Global Markets Direct’s proprietary databas es , company/univers ity webs ites , clinical trial regis tries , conferences , SEC filings , inves tor pres entations and featured pres s releas es from company/univers ity s ites and indus try-s pecific third party s ources . Drug profiles featured in the report undergoes periodic review following a s tringent s et of proces s es to ens ure that all the profiles are updated with the lates t s et of information. Additionally, various dynamic tracking proces s es ens ure that the mos t recent developments are captured on a real time bas is . The report helps in identifying and tracking emerging players in the market and their portfolios , enhances decis ion making capabilities and helps to create effective counter s trategies to gain competitive advantage. Note*: Certain s ections in the report may be removed or altered bas ed on the availability and relevance of data. Scope - The report provides a s naps hot of the global therapeutic lands cape of Rett Syndrome - The report reviews pipeline therapeutics for Rett Syndrome by companies and univers ities /res earch ins titutes bas ed on information derived from company and indus try-s pecific s ources - The report covers pipeline products bas ed on various s tages of development ranging from pre-regis tration till dis covery and undis clos ed s tages - The report features des criptive drug profiles for the pipeline products which includes , product des cription, des criptive MoA, R& D brief, licens ing and collaboration details & other developmental activities - The report reviews key players involved Rett Syndrome therapeutics and enlis ts all their major and minor projects - The report as s es s es Rett Syndrome therapeutics bas ed on drug target, mechanis m of action (MoA), route of adminis tration (RoA) and molecule type - The report s ummarizes all the dormant and dis continued pipeline projects - The report reviews lates t news related to pipeline therapeutics for Rett Syndrome Reas ons to buy - Gain s trategically s ignificant competitor information, analys is , and ins ights to formulate effective R& D s trategies - Identify emerging players with potentially s trong product portfolio and create effective counter-s trategies to gain competitive advantage - Identify and unders tand important and divers e types of therapeutics under development for Rett Syndrome - Identify potential new clients or partners in the target demographic - Develop s trategic initiatives by unders tanding the focus areas of leading companies - Plan mergers and acquis itions effectively by identifying key players and it’s mos t promis ing pipeline therapeutics - Devis e corrective meas ures for pipeline projects by unders tanding Rett Syndrome
pipeline depth and focus of Indication therapeutics - Develop and des ign in-licens ing and out-licens ing s trategies by identifying pros pective partners with the mos t attractive projects to enhance and expand bus ines s potential and s cope - Modify the therapeutic portfolio by identifying dis continued projects and unders tanding the factors that drove them from pipeline Contents:
Table of Contents Table of Contents 2 Lis t of Tables 6 Lis t of Figures 7 Introduction 8 Global Markets Direct Report Coverage 8 Rett Syndrome Overview 9 Therapeutics Development 10 Pipeline Products for Rett Syndrome - Overview 10 Pipeline Products for Rett Syndrome - Comparative Analys is 11 Rett Syndrome - Therapeutics under Development by Companies 12 Rett Syndrome - Therapeutics under Inves tigation by Univers ities /Ins titutes 13 Rett Syndrome - Pipeline Products Glance 14 Clinical Stage Products 14 Early Stage Products 15 Unknown Stage Products 16 Rett Syndrome - Products under Development by Companies 17 Rett Syndrome - Products under Inves tigation by Univers ities /Ins titutes 18 Rett Syndrome - Companies Involved in Therapeutics Development 19 Anavex Life Sciences Corp. 19 ArmaGen Inc. 20 Edis on Pharmaceuticals , Inc. 21 GW Pharmaceuticals Plc 22 Mits ubis hi Tanabe Pharma Corporation 23 Neuren Pharmaceuticals Limited 24 Neurolixis Inc. 25 Newron Pharmaceuticals S.p.A. 26 OPKO Health, Inc. 27 Oryzon Genomics S.A. 28 PharmatrophiX, Inc. 29 Raptor Pharmaceutical Corp. 30 Sage Therapeutics , Inc. 31 Ultragenyx Pharmaceutical Inc. 32 Rett Syndrome - Therapeutics As s es s ment 33 As s es s ment by Monotherapy Products 33 As s es s ment by Target 34 As s es s ment by Mechanis m of Action 36 As s es s ment by Route of Adminis tration 38 As s es s ment by Molecule Type 40 Drug Profiles 42 amlexanox - Drug Profile 42 Product Des cription 42 Mechanis m of Action 42 R& D Progres s 42 ANAVEX-273 - Drug Profile 43 Product Des cription 43 Mechanis m of Action 43 R& D Progres s 43 CPT-157633 - Drug Profile 45 Product Des cription 45 Mechanis m of Action 45 R& D Progres s 45 cys teamine DR - Drug Profile 46 Product Des cription 46 Mechanis m of Action 46 R& D Progres s 46 des ipramine hydrochloride - Drug Profile 49 Product Des cription 49 Mechanis m of Action 49 R& D Progres s 49 fingolimod hydrochloride - Drug Profile 50 Product Des cription 50 Mechanis m of Action 50 R& D Progres s 50 Gene therapy to Activate MECP2 for Rett Syndrome - Drug Profile 54 Product Des cription 54 Mechanis m of Action 54 R& D Progres s 54 LM-22A4 - Drug Profile 55 Product Des cription 55
Mechanis m of Action 55 R& D Progres s 55 NLX-101 - Drug Profile 57 Product Des cription 57 Mechanis m of Action 57 R& D Progres s 57 Oligonucleotide For Rett Syndrome - Drug Profile 58 Product Des cription 58 Mechanis m of Action 58 R& D Progres s 58 ORY-2001 - Drug Profile 59 Product Des cription 59 Mechanis m of Action 59 R& D Progres s 59 Recombinant Protein for Rett Syndrome - Drug Profile 60 Product Des cription 60 Mechanis m of Action 60 R& D Progres s 60 Recombinant Protein to Agonize BDNF for Rett Syndrome - Drug Profile 61 Product Des cription 61 Mechanis m of Action 61 R& D Progres s 61 REV-003 - Drug Profile 62 Product Des cription 62 Mechanis m of Action 62 R& D Progres s 62 SAGE-217 - Drug Profile 63 Product Des cription 63 Mechanis m of Action 63 R& D Progres s 63 s arizotan hydrochloride - Drug Profile 64 Product Des cription 64 Mechanis m of Action 64 R& D Progres s 64 Small Molecule to Activate TrkB Receptor for Central Nervous Sys tem, Metabolic Dis orders and ENT Dis orders - Drug Profile 66 Product Des cription 66 Mechanis m of Action 66 R& D Progres s 66 Small Molecules for Rett Syndrome - Drug Profile 68 Product Des cription 68 Mechanis m of Action 68 R& D Progres s 68 Small Molecules to Agonize Cannabinoid Receptor 2 for Fragile X Syndrome and Pervas ive Developmental Dis order - Drug Profile 69 Product Des cription 69 Mechanis m of Action 69 R& D Progres s 69 trofinetide - Drug Profile 70 Product Des cription 70 Mechanis m of Action 70 R& D Progres s 70 UX-007 - Drug Profile 73 Product Des cription 73 Mechanis m of Action 73 R& D Progres s 73 vatiquinone - Drug Profile 75 Product Des cription 75 Mechanis m of Action 75 R& D Progres s 75 Rett Syndrome - Recent Pipeline Updates 78 Rett Syndrome - Dormant Projects 108 Rett Syndrome - Product Development Miles tones 109 Featured News & Pres s Releas es 109 Feb 25, 2016: Anavex Pres ents Preclinical Res ults of ANAVEX 2-73 in Rett Syndrome 109 Oct 28, 2015: Newron Pharmaceuticals Announces Planned Pivotal Trial Des ign for Sarizotan for Patients with Rett Syndrome, a Rare Neurodevelopmental Dis eas e 110 Oct 13, 2015: Retts yndrome.org confirms financial s upport for Neuren’s clinical trials of trofinetide 111 Oct 12, 2015: Neuren receives Notice of Allowance for new US patent covering the us e of trofinetide to treat Rett s yndrome 111 Aug 14, 2015: “trofinetide” confirmed as the International Nonproprietary Name for Neuren’s NNZ -2566 112 Aug 13, 2015: Neuren confirms orphan drug des ignation in Europe for Rett s yndrome and Fragile X s yndrome 112 Jul 29, 2015: Neuren reports progres s in orphan drug programs 112 Jul 14, 2015: Newron Receives FDA Orphan Drug Des ignation for Sarizotan for the
Treatment of Rett Syndrome 113 Jun 25, 2015: Newron receives pos itive opinion for Orphan Medicinal Product Des ignation for Sarizotan to treat patients with Rett Syndrome from the Committee for Orphan Medicinal Products from the European Medicines Agency 114 Nov 12, 2014: Neuren’s NNZ -2566 s ucces s ful In Demons trating Clinical BeneďŹ t In Rett s yndrome Phas e 2 trial 115 Appendix 117 Methodology 117 Coverage 117 Secondary Res earch 117 Primary Res earch 117 Expert Panel Validation 117 Contact Us 117 Dis claimer 118 Lis t of Tables Number of Products under Development for Rett Syndrome, H1 2016 10 Number of Products under Development for Rett Syndrome - Comparative Analys is , H1 2016 11 Number of Products under Development by Companies , H1 2016 12 Number of Products under Inves tigation by Univers ities /Ins titutes , H1 2016 13 Comparative Analys is by Clinical Stage Development, H1 2016 14 Comparative Analys is by Early Stage Development, H1 2016 15 Comparative Analys is by Unknown Stage Development, H1 2016 16 Products under Development by Companies , H1 2016 17 Products under Inves tigation by Univers ities /Ins titutes , H1 2016 18 Rett Syndrome - Pipeline by Anavex Life Sciences Corp., H1 2016 19 Rett Syndrome - Pipeline by ArmaGen Inc., H1 2016 20 Rett Syndrome - Pipeline by Edis on Pharmaceuticals , Inc., H1 2016 21 Rett Syndrome - Pipeline by GW Pharmaceuticals Plc, H1 2016 22 Rett Syndrome - Pipeline by Mits ubis hi Tanabe Pharma Corporation, H1 2016 23 Rett Syndrome - Pipeline by Neuren Pharmaceuticals Limited, H1 2016 24 Rett Syndrome - Pipeline by Neurolixis Inc., H1 2016 25 Rett Syndrome - Pipeline by Newron Pharmaceuticals S.p.A., H1 2016 26 Rett Syndrome - Pipeline by OPKO Health, Inc., H1 2016 27 Rett Syndrome - Pipeline by Oryzon Genomics S.A., H1 2016 28 Rett Syndrome - Pipeline by PharmatrophiX, Inc., H1 2016 29 Rett Syndrome - Pipeline by Raptor Pharmaceutical Corp., H1 2016 30 Rett Syndrome - Pipeline by Sage Therapeutics , Inc., H1 2016 31 Rett Syndrome - Pipeline by Ultragenyx Pharmaceutical Inc., H1 2016 32 As s es s ment by Monotherapy Products , H1 2016 33 Number of Products by Stage and Target, H1 2016 35 Number of Products by Stage and Mechanis m of Action, H1 2016 37 Number of Products by Stage and Route of Adminis tration, H1 2016 39 Number of Products by Stage and Molecule Type, H1 2016 41 Rett Syndrome Therapeutics - Recent Pipeline Updates , H1 2016 78 Rett Syndrome - Dormant Projects , H1 2016 108 Lis t of Figures Number of Products under Development for Rett Syndrome, H1 2016 10 Number of Products under Development for Rett Syndrome - Comparative Analys is , H1 2016 11 Number of Products under Development by Companies , H1 2016 12 Comparative Analys is by Clinical Stage Development, H1 2016 14 Comparative Analys is by Early Stage Products , H1 2016 15 As s es s ment by Monotherapy Products , H1 2016 33 Number of Products by Top 10 Targets , H1 2016 34 Number of Products by Stage and Top 10 Targets , H1 2016 34 Number of Products by Top 10 Mechanis m of Actions , H1 2016 36 Number of Products by Stage and Top 10 Mechanis m of Actions , H1 2016 36 Number of Products by Routes of Adminis tration, H1 2016 38 Number of Products by Stage and Routes of Adminis tration, H1 2016 38 Number of Products by Molecule Types , H1 2016 40 Number of Products by Stage and Molecule Types , H1 2016 40
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