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Spinal Muscular Atrophy (SMA) – Pipeline Review, H1 2016

$2,000

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 ….

Total Pages: 121

spinal-muscular-atrophy-sma-pipeline-review-h1-2016
600
#9e2f22

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

List of Figures
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 13
Comparative Analysis by Clinical Stage Development, H1 2016 17
Comparative Analysis by Early Stage Products, H1 2016 18
Assessment by Monotherapy Products, H1 2016 38
Number of Products by Targets, H1 2016 39
Number of Products by Stage and Targets, H1 2016 39
Number of Products by Mechanism of Actions, H1 2016 41
Number of Products by Stage and Mechanism of Actions, H1 2016 41
Number of Products by Routes of Administration, H1 2016 43
Number of Products by Stage and Routes of Administration, H1 2016 43
Number of Products by Molecule Types, H1 2016 45
Number of Products by Stage and Molecule Types, H1 2016 45


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