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Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2015

Published: Oct 21, 2015 | Pages: 113 | Publisher: Global Markets Direct | Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2015

Summary

Global Markets Direct's, 'Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 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 14 Spinal Muscular Atrophy (SMA) - Pipeline Products Glance 15 Late Stage Products 15 Clinical Stage Products 16 Early Stage Products 17 Spinal Muscular Atrophy (SMA) - Products under Development by Companies 18 Spinal Muscular Atrophy (SMA) - Products under Investigation by Universities/Institutes 19 Spinal Muscular Atrophy (SMA) - Companies Involved in Therapeutics Development 20 Astellas Pharma Inc. 20 Bioblast Pharma Ltd. 21 Cytokinetics, Inc. 22 F. Hoffmann-La Roche Ltd. 23 Genethon 24 Genzyme Corporation 25 GMP-Orphan SAS 26 Isis Pharmaceuticals, Inc. 27 Longevity Biotech, Inc 28 Neurodyn Inc. 29 Neurotune AG 30 Nexgenix Pharmaceuticals, LLC 31 Novartis AG 32 Sarepta Therapeutics, Inc. 33 Vybion, Inc. 34 Spinal Muscular Atrophy (SMA) - Therapeutics Assessment 35 Assessment by Monotherapy Products 35 Assessment by Target 36 Assessment by Mechanism of Action 38 Assessment by Route of Administration 40 Assessment by Molecule Type 42 Drug Profiles 44 ALB-111 - Drug Profile 44 Product Description 44 Mechanism of Action 44 R&D Progress 44 Antisense Oligonucleotide to Inhibit SMN2 gene for SMA - Drug Profile 46 Product Description 46 Mechanism of Action 46 R&D Progress 46 Antisense Oligonucleotides for Spinal Muscular Atrophy - Drug Profile 47 Product Description 47 Mechanism of Action 47 R&D Progress 47 ARM-210 - Drug Profile 48 Product Description 48 Mechanism of Action 48 R&D Progress 48 AVXS-101 - Drug Profile 49 Product Description 49 Mechanism of Action 49 R&D Progress 49 azithromycin - Drug Profile 51 Product Description 51 Mechanism of Action 51 R&D Progress 51 CK-2127107 - Drug Profile 52 Product Description 52 Mechanism of Action 52 R&D Progress 52 Gene Therapy to Activate SMN for Spinal Muscular Atrophy - Drug Profile 53 Product Description 53 Mechanism of Action 53 R&D Progress 53 Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 54 Product Description 54 Mechanism of Action 54 R&D Progress 54 Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 55 Product Description 55 Mechanism of Action 55 R&D Progress 55 INT-41 - Drug Profile 56 Product Description 56 Mechanism of Action 56 R&D Progress 56 LBT-3627 - Drug Profile 58 Product Description 58 Mechanism of Action 58 R&D Progress 58 LMI-070 - Drug Profile 59 Product Description 59 Mechanism of Action 59 R&D Progress 59 ND-602 - Drug Profile 60 Product Description 60 Mechanism of Action 60 R&D Progress 60 NT-1654 - Drug Profile 61 Product Description 61 Mechanism of Action 61 R&D Progress 61 nusinersen - Drug Profile 62 Product Description 62 Mechanism of Action 62 R&D Progress 62 NXD-30001 - Drug Profile 64 Product Description 64 Mechanism of Action 64 R&D Progress 64 olesoxime - Drug Profile 66 Product Description 66 Mechanism of Action 66 R&D Progress 66 Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 69 Product Description 69 Mechanism of Action 69 R&D Progress 69 PMO-25 - Drug Profile 70 Product Description 70 Mechanism of Action 70 R&D Progress 70 RG-7800 - Drug Profile 71 Product Description 71 Mechanism of Action 71 R&D Progress 71 Small Molecules for Central Nervous System Disorders - Drug Profile 73 Product Description 73 Mechanism of Action 73 R&D Progress 73 Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 74 Product Description 74 Mechanism of Action 74 R&D Progress 74 Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 75 Product Description 75 Mechanism of Action 75 R&D Progress 75 Small Molecules to Activate Troponin for Non-Neuromuscular and Neuromuscular Disorders - Drug Profile 76 Product Description 76 Mechanism of Action 76 R&D Progress 76 sodium phenylbutyrate - Drug Profile 77 Product Description 77 Mechanism of Action 77 R&D Progress 77 tirasemtiv - Drug Profile 78 Product Description 78 Mechanism of Action 78 R&D Progress 78 Spinal Muscular Atrophy (SMA) - Recent Pipeline Updates 85 Spinal Muscular Atrophy (SMA) - Dormant Projects 103 Spinal Muscular Atrophy (SMA) - Discontinued Products 104 Spinal Muscular Atrophy (SMA) - Product Development Milestones 105 Featured News & Press Releases 105 Oct 05, 2015: Data from Ongoing Study of AVXS-101 in Spinal Muscular Atrophy Type 1 Presented at World Muscle Congress 105 Oct 04, 2015: Phase 2 Data from Spinal Muscular Atrophy Program Presented at the 20th International Annual Congress of the World Muscle Society 105 Jul 14, 2015: Isis Pharmaceuticals Earns $2.15M for Advancing ISIS-SMN in Children With Spinal Muscular Atrophy 106 Jun 23, 2015: AveXis Gains Orphan Drug Designation From the European Medicines Agency for Gene Therapy Treatment in Spinal Muscular Atrophy 106 Jun 22, 2015: BioBlast Announces Positive Preclinical Proof-Of-Concept Results Of Its Novel Read- through Platform in Spinal Muscular Atrophy 107 Jun 22, 2015: Isis Pharmaceuticals Reports Data from ISIS-SMN Rx in Children with Spinal Muscular Atrophy 108 Jun 19, 2015: Cytokinetics Presents Results From Phase I Studies Of Ck-2127107 109 Jun 18, 2015: BioBlast Pharma to Present Preclinical Proofs of Concept in Spinal Muscular Atrophy at Two Leading Scientific Meetings in June 109 Jun 11, 2015: Isis Pharmaceuticals Reports Data From ISIS-SMN Rx Phase 2 Study in Infants With Spinal Muscular Atrophy 110 Apr 20, 2015: Phase 1 Data from Spinal Muscular Atrophy Program to be Presented at the 2015 American Academy of Neurology Annual Meeting 111 Appendix 112 Methodology 112 Coverage 112 Secondary Research 112 Primary Research 112 Expert Panel Validation 112 Contact Us 112 Disclaimer 113
List of Tables
Number of Products under Development for Spinal Muscular Atrophy (SMA), H2 2015 10 Number of Products under Development for Spinal Muscular Atrophy (SMA) - Comparative Analysis, H2 2015 11 Number of Products under Development by Companies, H2 2015 13 Number of Products under Investigation by Universities/Institutes, H2 2015 14 Comparative Analysis by Late Stage Development, H2 2015 15 Comparative Analysis by Clinical Stage Development, H2 2015 16 Comparative Analysis by Early Stage Development, H2 2015 17 Products under Development by Companies, H2 2015 18 Products under Investigation by Universities/Institutes, H2 2015 19 Spinal Muscular Atrophy (SMA) - Pipeline by Astellas Pharma Inc., H2 2015 20 Spinal Muscular Atrophy (SMA) - Pipeline by Bioblast Pharma Ltd., H2 2015 21 Spinal Muscular Atrophy (SMA) - Pipeline by Cytokinetics, Inc., H2 2015 22 Spinal Muscular Atrophy (SMA) - Pipeline by F. Hoffmann-La Roche Ltd., H2 2015 23 Spinal Muscular Atrophy (SMA) - Pipeline by Genethon, H2 2015 24 Spinal Muscular Atrophy (SMA) - Pipeline by Genzyme Corporation, H2 2015 25 Spinal Muscular Atrophy (SMA) - Pipeline by GMP-Orphan SAS, H2 2015 26 Spinal Muscular Atrophy (SMA) - Pipeline by Isis Pharmaceuticals, Inc., H2 2015 27 Spinal Muscular Atrophy (SMA) - Pipeline by Longevity Biotech, Inc, H2 2015 28 Spinal Muscular Atrophy (SMA) - Pipeline by Neurodyn Inc., H2 2015 29 Spinal Muscular Atrophy (SMA) - Pipeline by Neurotune AG, H2 2015 30 Spinal Muscular Atrophy (SMA) - Pipeline by Nexgenix Pharmaceuticals, LLC, H2 2015 31 Spinal Muscular Atrophy (SMA) - Pipeline by Novartis AG, H2 2015 32 Spinal Muscular Atrophy (SMA) - Pipeline by Sarepta Therapeutics, Inc., H2 2015 33 Spinal Muscular Atrophy (SMA) - Pipeline by Vybion, Inc., H2 2015 34 Assessment by Monotherapy Products, H2 2015 35 Number of Products by Stage and Target, H2 2015 37 Number of Products by Stage and Mechanism of Action, H2 2015 39 Number of Products by Stage and Route of Administration, H2 2015 41 Number of Products by Stage and Molecule Type, H2 2015 43 Spinal Muscular Atrophy (SMA) Therapeutics - Recent Pipeline Updates, H2 2015 85 Spinal Muscular Atrophy (SMA) - Dormant Projects, H2 2015 103 Spinal Muscular Atrophy (SMA) - Discontinued Products, H2 2015 104


List of Figures
Number of Products under Development for Spinal Muscular Atrophy (SMA), H2 2015 10 Number of Products under Development for Spinal Muscular Atrophy (SMA) - Comparative Analysis, H2 2015 11 Number of Products under Development by Companies, H2 2015 12 Comparative Analysis by Clinical Stage Development, H2 2015 16 Comparative Analysis by Early Stage Products, H2 2015 17 Assessment by Monotherapy Products, H2 2015 35 Number of Products by Top 10 Targets, H2 2015 36 Number of Products by Stage and Top 10 Targets, H2 2015 36 Number of Products by Top 10 Mechanism of Actions, H2 2015 38 Number of Products by Stage and Top 10 Mechanism of Actions, H2 2015 38 Number of Products by Top 10 Routes of Administration, H2 2015 40 Number of Products by Stage and Top 10 Routes of Administration, H2 2015 40 Number of Products by Top 10 Molecule Types, H2 2015 42 Number of Products by Stage and Top 10 Molecule Types, H2 2015 42

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