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Published: Feb, 2016 | Pages:
121 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2016 Summary Global Markets Direct's, 'Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2016', provides an overview of the Spinal Muscular Atrophy (SMA) pipeline landscape. The report provides comprehensive information on the therapeutics under development for Spinal Muscular Atrophy (SMA), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Spinal Muscular Atrophy (SMA) and features dormant 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, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis. The report 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. Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data. Scope - The report provides a snapshot of the global therapeutic landscape of Spinal Muscular Atrophy (SMA) - The report reviews pipeline therapeutics for Spinal Muscular Atrophy (SMA) by companies and universities/research institutes based on information derived from company and industry-specific sources - The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages - The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities - The report reviews key players involved Spinal Muscular Atrophy (SMA) therapeutics and enlists all their major and minor projects - The report assesses Spinal Muscular Atrophy (SMA) therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type - The report summarizes all the dormant and discontinued pipeline projects - The report reviews latest news related to pipeline therapeutics for Spinal Muscular Atrophy (SMA) Reasons to Buy - Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies - Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage - Identify and understand important and diverse types of therapeutics under development for Spinal Muscular Atrophy (SMA) - Identify potential new clients or partners in the target demographic - Develop strategic initiatives by understanding the focus areas of leading companies - Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics - 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 7 List of Figures 8 Introduction 9 Global Markets Direct Report Coverage 9 Spinal Muscular Atrophy (SMA) Overview 10 Therapeutics Development 11 Pipeline Products for Spinal Muscular Atrophy (SMA) - Overview 11 Pipeline Products for Spinal Muscular Atrophy (SMA) - Comparative Analysis 12 Spinal Muscular Atrophy (SMA) - Therapeutics under Development by Companies 13 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 Spinal Muscular Atrophy (SMA) - Products under Development by Companies 19 Spinal Muscular Atrophy (SMA) - Products under Investigation by Universities/Institutes 20 Spinal Muscular Atrophy (SMA) - Companies Involved in Therapeutics Development 21 Astellas Pharma Inc. 21 AveXis, Inc. 22 Bioblast Pharma Ltd. 23 Cytokinetics, Inc. 24 F. Hoffmann-La Roche Ltd. 25 Genethon 26 Genzyme Corporation 27 GMP-Orphan SAS 28 Ionis Pharmaceuticals, Inc. 29 Longevity Biotech, Inc 30 Neurodyn Inc. 31 Neurotune AG 32 Novartis AG 33 Sarepta Therapeutics, Inc. 34 Voyager Therapeutics, Inc. 35 Vybion, Inc. 36 WAVE Life Sciences Ltd. 37 Spinal Muscular Atrophy (SMA) - Therapeutics Assessment 38 Assessment by Monotherapy Products 38 Assessment by Target 39 Assessment by Mechanism of Action 41 Assessment by Route of Administration 43 Assessment by Molecule Type 45 Drug Profiles 47 ALB-111 - Drug Profile 47 Product Description 47 Mechanism of Action 47 R&D Progress 47 Antisense Oligonucleotide to Inhibit SMN2 gene for SMA - Drug Profile 49 Product Description 49 Mechanism of Action 49 R&D Progress 49 Antisense Oligonucleotides for Spinal Muscular Atrophy - Drug Profile 50 Product Description 50 Mechanism of Action 50 R&D Progress 50 ARM-210 - Drug Profile 51 Product Description 51 Mechanism of Action 51 R&D Progress 51 AVXS-101 - Drug Profile 52 Product Description 52 Mechanism of Action 52 R&D Progress 52 azithromycin - Drug Profile 54 Product Description 54 Mechanism of Action 54 R&D Progress 54 CK-2127107 - Drug Profile 55 Product Description 55 Mechanism of Action 55 R&D Progress 55 Gene Therapy to Activate SMN for Spinal Muscular Atrophy - Drug Profile 56 Product Description 56 Mechanism of Action 56 R&D Progress 56 Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 57 Product Description 57 Mechanism of Action 57 R&D Progress 57 Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 58 Product Description 58 Mechanism of Action 58 R&D Progress 58 INT-41 - Drug Profile 59 Product Description 59 Mechanism of Action 59 R&D Progress 59 LBT-3627 - Drug Profile 61 Product Description 61 Mechanism of Action 61 R&D Progress 61 LMI-070 - Drug Profile 62 Product Description 62 Mechanism of Action 62 R&D Progress 62 ND-602 - Drug Profile 63 Product Description 63 Mechanism of Action 63 R&D Progress 63 NT-1654 - Drug Profile 64 Product Description 64 Mechanism of Action 64 R&D Progress 64 nusinersen - Drug Profile 65 Product Description 65 Mechanism of Action 65 R&D Progress 65 NXD-30001 - Drug Profile 67 Product Description 67 Mechanism of Action 67 R&D Progress 67 olesoxime - Drug Profile 69 Product Description 69 Mechanism of Action 69 R&D Progress 69 Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 72 Product Description 72 Mechanism of Action 72 R&D Progress 72 Oligonucleotides to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 73 Product Description 73 Mechanism of Action 73 R&D Progress 73 PMO-25 - Drug Profile 74 Product Description 74 Mechanism of Action 74 R&D Progress 74 RG-7800 - Drug Profile 75 Product Description 75 Mechanism of Action 75 R&D Progress 75 RG-7916 - Drug Profile 76 Product Description 76 Mechanism of Action 76 R&D Progress 76 Small Molecules for Central Nervous System Disorders - Drug Profile 77 Product Description 77 Mechanism of Action 77 R&D Progress 77 Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 78 Product Description 78 Mechanism of Action 78 R&D Progress 78 Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 79 Product Description 79 Mechanism of Action 79 R&D Progress 79 Small Molecules to Activate Troponin for Non-Neuromuscular and Neuromuscular Disorders - Drug Profile 80 Product Description 80 Mechanism of Action 80 R&D Progress 80 sodium phenylbutyrate - Drug Profile 81 Product Description 81 Mechanism of Action 81 R&D Progress 81 tirasemtiv - Drug Profile 82 Product Description 82 Mechanism of Action 82 R&D Progress 82 VYSMN-101 - Drug Profile 89 Product Description 89 Mechanism of Action 89 R&D Progress 89 Spinal Muscular Atrophy (SMA) - Recent Pipeline Updates 90 Spinal Muscular Atrophy (SMA) - Dormant Projects 110 Spinal Muscular Atrophy (SMA) - Discontinued Products 111 Spinal Muscular Atrophy (SMA) - Product Development Milestones 112 Featured News & Press Releases 112 Jan 12, 2016: Ionis Pharmaceuticals Completes Target Enrollment for Nusinersen Phase 3 Study, CHERISH, in Children with Spinal Muscular Atrophy 112 Jan 07, 2016: Spinal Muscular Atrophy Program Advances with Additional Product Candidate Entering Clinical Development 112 Jan 04, 2016: Cytokinetics Announces Start Of Phase 2 Clinical Trial Of Ck-2127107 In Patients With Spinal Muscular Atrophy 113 Oct 15, 2015: Isis Pharmaceuticals Initiates SHINE Study to Provide ISIS-SMN for Patients With SMA Who Have Completed the Phase 3 ENDEAR and CHERISH Studies 113 Oct 04, 2015: Phase 2 Data from Spinal Muscular Atrophy Program Presented at the 20th International Annual Congress of the World Muscle Society 115 Jul 14, 2015: Isis Pharmaceuticals Earns $2.15M for Advancing ISIS-SMN in Children With Spinal Muscular Atrophy 115 Jun 22, 2015: BioBlast Announces Positive Preclinical Proof-Of-Concept Results Of Its Novel Read- through Platform in Spinal Muscular Atrophy 116 Jun 22, 2015: Isis Pharmaceuticals Reports Data from ISIS-SMN Rx in Children with Spinal Muscular Atrophy 117 Jun 19, 2015: Cytokinetics Presents Results From Phase I Studies Of Ck-2127107 118 Jun 18, 2015: BioBlast Pharma to Present Preclinical Proofs of Concept in Spinal Muscular Atrophy at Two Leading Scientific Meetings in June 118 Appendix 120 Methodology 120 Coverage 120 Secondary Research 120 Primary Research 120 Expert Panel Validation 120 Contact Us 120 Disclaimer 121
List of Tables Number of Products under Development for Spinal Muscular Atrophy (SMA), H1 2016 11 Number of Products under Development for Spinal Muscular Atrophy (SMA) - Comparative Analysis, H1 2016 12 Number of Products under Development by Companies, H1 2016 14 Number of Products under Investigation by Universities/Institutes, H1 2016 15 Comparative Analysis by Late Stage Development, H1 2016 16 Comparative Analysis by Clinical Stage Development, H1 2016 17 Comparative Analysis by Early Stage Development, H1 2016 18 Products under Development by Companies, H1 2016 19 Products under Investigation by Universities/Institutes, H1 2016 20 Spinal Muscular Atrophy (SMA) - Pipeline by Astellas Pharma Inc., H1 2016 21 Spinal Muscular Atrophy (SMA) - Pipeline by AveXis, Inc., H1 2016 22 Spinal Muscular Atrophy (SMA) - Pipeline by Bioblast Pharma Ltd., H1 2016 23 Spinal Muscular Atrophy (SMA) - Pipeline by Cytokinetics, Inc., H1 2016 24 Spinal Muscular Atrophy (SMA) - Pipeline by F. Hoffmann-La Roche Ltd., H1 2016 25 Spinal Muscular Atrophy (SMA) - Pipeline by Genethon, H1 2016 26 Spinal Muscular Atrophy (SMA) - Pipeline by Genzyme Corporation, H1 2016 27 Spinal Muscular Atrophy (SMA) - Pipeline by GMP-Orphan SAS, H1 2016 28 Spinal Muscular Atrophy (SMA) - Pipeline by Ionis Pharmaceuticals, Inc., H1 2016 29 Spinal Muscular Atrophy (SMA) - Pipeline by Longevity Biotech, Inc, H1 2016 30 Spinal Muscular Atrophy (SMA) - Pipeline by Neurodyn Inc., H1 2016 31 Spinal Muscular Atrophy (SMA) - Pipeline by Neurotune AG, H1 2016 32 Spinal Muscular Atrophy (SMA) - Pipeline by Novartis AG, H1 2016 33 Spinal Muscular Atrophy (SMA) - Pipeline by Sarepta Therapeutics, Inc., H1 2016 34 Spinal Muscular Atrophy (SMA) - Pipeline by Voyager Therapeutics, Inc., H1 2016 35 Spinal Muscular Atrophy (SMA) - Pipeline by Vybion, Inc., H1 2016 36 Spinal Muscular Atrophy (SMA) - Pipeline by WAVE Life Sciences Ltd., H1 2016 37 Assessment by Monotherapy Products, H1 2016 38 Number of Products by Stage and Target, H1 2016 40 Number of Products by Stage and Mechanism of Action, H1 2016 42 Number of Products by Stage and Route of Administration, H1 2016 44 Number of Products by Stage and Molecule Type, H1 2016 46 Spinal Muscular Atrophy (SMA) Therapeutics - Recent Pipeline Updates, H1 2016 90 Spinal Muscular Atrophy (SMA) - Dormant Projects, H1 2016 110 Spinal Muscular Atrophy (SMA) - Discontinued Products, H1 2016 111
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