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Published: Jan, 2015 | Pages:
132 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2015 Summary Global Markets Direct's, 'Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2015', provides an overview of the Spinal Muscular Atrophy (SMA)'s therapeutic pipeline. This report provides comprehensive information on the therapeutic development for Spinal Muscular Atrophy (SMA), complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Spinal Muscular Atrophy (SMA) and special features on late-stage and discontinued projects. Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct's proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis. The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products. Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease. Scope - The report provides a snapshot of the global therapeutic landscape of Spinal Muscular Atrophy (SMA) - The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities - The report reviews key players involved in the therapeutics development for Spinal Muscular Atrophy (SMA) and enlists all their major and minor projects - The report summarizes all the dormant and discontinued pipeline projects - A review of the Spinal Muscular Atrophy (SMA) products under development by companies and universities/research institutes based on information derived from company and industry-specific sources - Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages - A detailed assessment of monotherapy and combination therapy pipeline projects - Coverage of the Spinal Muscular Atrophy (SMA) pipeline on the basis of target, MoA, route of administration and molecule type - Latest news and deals relating related to pipeline products Reasons to buy - Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies - Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage - Develop strategic initiatives by understanding the focus areas of leading companies - Identify and understand important and diverse types of therapeutics under development for Spinal Muscular Atrophy (SMA) - Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline - Devise corrective measures for pipeline projects by understanding Spinal Muscular Atrophy (SMA) 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 - Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline
Table of Contents Table of Contents 2 List of Tables 6 List of Figures 7 Introduction 8 Global Markets Direct Report Coverage 8 Spinal Muscular Atrophy (SMA) Overview 9 Therapeutics Development 10 Pipeline Products for Spinal Muscular Atrophy (SMA) - Overview 10 Pipeline Products for Spinal Muscular Atrophy (SMA) - Comparative Analysis 11 Spinal Muscular Atrophy (SMA) - Therapeutics under Development by Companies 12 Spinal Muscular Atrophy (SMA) - Therapeutics under Investigation by Universities/Institutes 15 Spinal Muscular Atrophy (SMA) - Pipeline Products Glance 16 Late Stage Products 16 Clinical Stage Products 17 Early Stage Products 18 Unknown Stage Products 19 Spinal Muscular Atrophy (SMA) - Products under Development by Companies 20 Spinal Muscular Atrophy (SMA) - Products under Investigation by Universities/Institutes 22 Spinal Muscular Atrophy (SMA) - Companies Involved in Therapeutics Development 23 Bioblast Pharma Ltd. 23 Cellular Biomedicine Group, Inc. 24 Cytokinetics, Inc. 25 F. Hoffmann-La Roche Ltd. 26 Genethon 27 Genzyme Corporation 28 GMP-Orphan SAS 29 Isis Pharmaceuticals, Inc. 30 Longevity Biotech, Inc 31 Neurodyn Inc. 32 Neurotune AG 33 Nexgenix Pharmaceuticals, LLC 34 Novartis AG 35 OrphageniX, Inc. 36 Paratek Pharmaceuticals, Inc. 37 Pfizer Inc. 38 PTC Therapeutics, Inc. 39 Retrophin Inc. 40 Sarepta Therapeutics, Inc. 41 Trophos SA 42 Vybion, Inc. 43 Zambon Company S.p.A. 44 Spinal Muscular Atrophy (SMA) - Therapeutics Assessment 45 Assessment by Monotherapy Products 45 Assessment by Target 46 Assessment by Mechanism of Action 48 Assessment by Route of Administration 50 Assessment by Molecule Type 52 Drug Profiles 54 ALB-111 - Drug Profile 54 Product Description 54 Mechanism of Action 54 R&D Progress 54 Antisense Oligonucleotide for Spinal Muscular Atrophy and Myotonic Dystrophy - Drug Profile 56 Product Description 56 Mechanism of Action 56 R&D Progress 56 Antisense Oligonucleotide to Inhibit SMN2 gene for SMA - Drug Profile 57 Product Description 57 Mechanism of Action 57 R&D Progress 57 Antisense Oligonucleotides for Spinal Muscular Atrophy - Drug Profile 58 Product Description 58 Mechanism of Action 58 R&D Progress 58 ataluren - Drug Profile 59 Product Description 59 Mechanism of Action 59 R&D Progress 59 BBrm-01 - Drug Profile 62 Product Description 62 Mechanism of Action 62 R&D Progress 62 DNA Oligonucleotides for Spinal Muscular Atrophy - Drug Profile 63 Product Description 63 Mechanism of Action 63 R&D Progress 63 Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 64 Product Description 64 Mechanism of Action 64 R&D Progress 64 Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 65 Product Description 65 Mechanism of Action 65 R&D Progress 65 INT-41 - Drug Profile 66 Product Description 66 Mechanism of Action 66 R&D Progress 66 LMI-070 - Drug Profile 68 Product Description 68 Mechanism of Action 68 R&D Progress 68 ND-602 - Drug Profile 69 Product Description 69 Mechanism of Action 69 R&D Progress 69 NT-1654 - Drug Profile 70 Product Description 70 Mechanism of Action 70 R&D Progress 70 nusinersen - Drug Profile 71 Product Description 71 Mechanism of Action 71 R&D Progress 71 NXD-30001 - Drug Profile 73 Product Description 73 Mechanism of Action 73 R&D Progress 73 olesoxime - Drug Profile 75 Product Description 75 Mechanism of Action 75 R&D Progress 75 Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 78 Product Description 78 Mechanism of Action 78 R&D Progress 78 PF-06687859 - Drug Profile 79 Product Description 79 Mechanism of Action 79 R&D Progress 79 PMO-25 - Drug Profile 81 Product Description 81 Mechanism of Action 81 R&D Progress 81 PTKSMA-2 - Drug Profile 82 Product Description 82 Mechanism of Action 82 R&D Progress 82 RE-003 - Drug Profile 83 Product Description 83 Mechanism of Action 83 R&D Progress 83 RG-7800 - Drug Profile 84 Product Description 84 Mechanism of Action 84 R&D Progress 84 scAAV9.CB.SMN - Drug Profile 86 Product Description 86 Mechanism of Action 86 R&D Progress 86 Small Molecules for CNS Disorders - Drug Profile 88 Product Description 88 Mechanism of Action 88 R&D Progress 88 sodium phenylbutyrate - Drug Profile 89 Product Description 89 Mechanism of Action 89 R&D Progress 89 Stem Cell Therapy for Amyotrophic Lateral Sclerosis and Spinal Muscular Atrophy - Drug Profile 90 Product Description 90 Mechanism of Action 90 R&D Progress 90 Synthetic Peptides for Central Nervous System Disorders and Diabetic Neuropathy - Drug Profile 91 Product Description 91 Mechanism of Action 91 R&D Progress 91 tirasemtiv - Drug Profile 92 Product Description 92 Mechanism of Action 92 R&D Progress 92 ZP-037 - Drug Profile 99 Product Description 99 Mechanism of Action 99 R&D Progress 99 Spinal Muscular Atrophy (SMA) - Recent Pipeline Updates 100 Spinal Muscular Atrophy (SMA) - Dormant Projects 119 Spinal Muscular Atrophy (SMA) - Product Development Milestones 120 Featured News & Press Releases 120 Nov 25, 2014: Isis Pharmaceuticals Initiates Phase 3 Study of ISIS-SMN Rx in Children With Spinal Muscular Atrophy 120 Nov 17, 2014: Cure SMA Presents Isis Pharmaceuticals with SMA Breakthrough Award 121 Oct 10, 2014: Isis Pharmaceuticals Reports Data From ISIS-SMN Rx Phase 2 Studies in Infants and Children With Spinal Muscular Atrophy 122 Oct 02, 2014: Isis Pharmaceuticals to Host Webcast to Discuss ISIS-SMN Rx Results Presented at the World Muscle Society Congress 125 Oct 02, 2014: AveXis Receives Orphan Drug Status from the US Food and Drug Administration for chariSMA for the Treatment of Spinal Muscular Atrophy 125 Aug 15, 2014: Isis Pharmaceuticals Earns $2M from the Advancement of ISIS-SMN Rx in Children with Spinal Muscular Atrophy 126 Aug 01, 2014: Isis Pharmaceuticals Initiates Phase 3 Study of ISIS-SMN Rx in Infants with Spinal Muscular Atrophy 127 Jun 13, 2014: Cytokinetics Announces Data Relating To Tirasemtiv Presented At The 2014 Annual Spinal Muscular Atrophy Conference 127 Jun 06, 2014: Cytokinetics Announces Two Presentations Relating To Tirasemtiv To Be Presented At The 2014 Annual Spinal Muscular Atrophy Conference 129 May 22, 2014: Gene therapy extends survival in an animal model of spinal muscular atrophy 129 Appendix 131 Methodology 131 Coverage 131 Secondary Research 131 Primary Research 131 Expert Panel Validation 131 Contact Us 132 Disclaimer 132
List of Tables Number of Products under Development for Spinal Muscular Atrophy (SMA), H1 2015 10 Number of Products under Development for Spinal Muscular Atrophy (SMA) - Comparative Analysis, H1 2015 11 Number of Products under Development by Companies, H1 2015 13 Number of Products under Development by Companies, H1 2015 (Contd..1) 14 Number of Products under Investigation by Universities/Institutes, H1 2015 15 Comparative Analysis by Late Stage Development, H1 2015 16 Comparative Analysis by Clinical Stage Development, H1 2015 17 Comparative Analysis by Early Stage Development, H1 2015 18 Comparative Analysis by Unknown Stage Development, H1 2015 19 Products under Development by Companies, H1 2015 20 Products under Development by Companies, H1 2015 (Contd..1) 21 Products under Investigation by Universities/Institutes, H1 2015 22 Spinal Muscular Atrophy (SMA) - Pipeline by Bioblast Pharma Ltd., H1 2015 23 Spinal Muscular Atrophy (SMA) - Pipeline by Cellular Biomedicine Group, Inc., H1 2015 24 Spinal Muscular Atrophy (SMA) - Pipeline by Cytokinetics, Inc., H1 2015 25 Spinal Muscular Atrophy (SMA) - Pipeline by F. Hoffmann-La Roche Ltd., H1 2015 26 Spinal Muscular Atrophy (SMA) - Pipeline by Genethon, H1 2015 27 Spinal Muscular Atrophy (SMA) - Pipeline by Genzyme Corporation, H1 2015 28 Spinal Muscular Atrophy (SMA) - Pipeline by GMP-Orphan SAS, H1 2015 29 Spinal Muscular Atrophy (SMA) - Pipeline by Isis Pharmaceuticals, Inc., H1 2015 30 Spinal Muscular Atrophy (SMA) - Pipeline by Longevity Biotech, Inc, H1 2015 31 Spinal Muscular Atrophy (SMA) - Pipeline by Neurodyn Inc., H1 2015 32 Spinal Muscular Atrophy (SMA) - Pipeline by Neurotune AG, H1 2015 33 Spinal Muscular Atrophy (SMA) - Pipeline by Nexgenix Pharmaceuticals, LLC, H1 2015 34 Spinal Muscular Atrophy (SMA) - Pipeline by Novartis AG, H1 2015 35 Spinal Muscular Atrophy (SMA) - Pipeline by OrphageniX, Inc., H1 2015 36 Spinal Muscular Atrophy (SMA) - Pipeline by Paratek Pharmaceuticals, Inc., H1 2015 37 Spinal Muscular Atrophy (SMA) - Pipeline by Pfizer Inc., H1 2015 38 Spinal Muscular Atrophy (SMA) - Pipeline by PTC Therapeutics, Inc., H1 2015 39 Spinal Muscular Atrophy (SMA) - Pipeline by Retrophin Inc., H1 2015 40 Spinal Muscular Atrophy (SMA) - Pipeline by Sarepta Therapeutics, Inc., H1 2015 41 Spinal Muscular Atrophy (SMA) - Pipeline by Trophos SA, H1 2015 42 Spinal Muscular Atrophy (SMA) - Pipeline by Vybion, Inc., H1 2015 43 Spinal Muscular Atrophy (SMA) - Pipeline by Zambon Company S.p.A., H1 2015 44 Assessment by Monotherapy Products, H1 2015 45 Number of Products by Stage and Target, H1 2015 47 Number of Products by Stage and Mechanism of Action, H1 2015 49 Number of Products by Stage and Route of Administration, H1 2015 51 Number of Products by Stage and Molecule Type, H1 2015 53 Spinal Muscular Atrophy (SMA) Therapeutics - Recent Pipeline Updates, H1 2015 100 Spinal Muscular Atrophy (SMA) - Dormant Projects, H1 2015 119
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