Summary
Global Markets Direct’s, ‘Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 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
Companies Mentioned
Astellas Pharma Inc.
AveXis, Inc.
Bio Blast Pharma Ltd.
Cytokinetics, Inc.
F. Hoffmann-La Roche Ltd.
Genethon
Genzyme Corporation
Ionis Pharmaceuticals, Inc.
Neurodyn Inc.
Neurotune AG
Novartis AG
Paratek Pharmaceuticals, Inc.
RaNA Therapeutics, Inc.
Sarepta Therapeutics, Inc.
Voyager Therapeutics, Inc.
WAVE Life Sciences Ltd.
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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
Bio Blast Pharma Ltd. 23
Cytokinetics, Inc. 24
F. Hoffmann-La Roche Ltd. 25
Genethon 26
Genzyme Corporation 27
Ionis Pharmaceuticals, Inc. 28
Neurodyn Inc. 29
Neurotune AG 30
Novartis AG 31
Paratek Pharmaceuticals, Inc. 32
RaNA Therapeutics, Inc. 33
Sarepta Therapeutics, Inc. 34
Voyager Therapeutics, Inc. 35
WAVE Life Sciences Ltd. 36
Spinal Muscular Atrophy (SMA) - Therapeutics Assessment 37
Assessment by Monotherapy Products 37
Assessment by Target 38
Assessment by Mechanism of Action 40
Assessment by Route of Administration 42
Assessment by Molecule Type 44
Drug Profiles 46
AAD-2004 - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
ALB-111 - Drug Profile 47
Product Description 47
Mechanism Of Action 47
R&D Progress 47
Antisense Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 48
Product Description 48
Mechanism Of Action 48
R&D Progress 48
Antisense Oligonucleotides for Spinal Muscular Atrophy - Drug Profile 49
Product Description 49
Mechanism Of Action 49
R&D Progress 49
AVXS-101 - Drug Profile 50
Product Description 50
Mechanism Of Action 50
R&D Progress 50
azithromycin - Drug Profile 53
Product Description 53
Mechanism Of Action 53
R&D Progress 53
CK-2127107 - Drug Profile 54
Product Description 54
Mechanism Of Action 54
R&D Progress 54
Gene Therapy to Activate SMN for Spinal Muscular Atrophy - Drug Profile 58
Product Description 58
Mechanism Of Action 58
R&D Progress 58
Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 59
Product Description 59
Mechanism Of Action 59
R&D Progress 59
Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 60
Product Description 60
Mechanism Of Action 60
R&D Progress 60
LMI-070 - Drug Profile 61
Product Description 61
Mechanism Of Action 61
R&D Progress 61
ND-602 - Drug Profile 62
Product Description 62
Mechanism Of Action 62
R&D Progress 62
NT-1654 - Drug Profile 63
Product Description 63
Mechanism Of Action 63
R&D Progress 63
nusinersen - Drug Profile 64
Product Description 64
Mechanism Of Action 64
R&D Progress 64
olesoxime - Drug Profile 70
Product Description 70
Mechanism Of Action 70
R&D Progress 70
Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 75
Product Description 75
Mechanism Of Action 75
R&D Progress 75
Oligonucleotides to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 76
Product Description 76
Mechanism Of Action 76
R&D Progress 76
PMO-25 - Drug Profile 77
Product Description 77
Mechanism Of Action 77
R&D Progress 77
PTKSMA-1 - Drug Profile 78
Product Description 78
Mechanism Of Action 78
R&D Progress 78
RG-7800 - Drug Profile 79
Product Description 79
Mechanism Of Action 79
R&D Progress 79
RG-7916 - Drug Profile 81
Product Description 81
Mechanism Of Action 81
R&D Progress 81
Small Molecules for Central Nervous System Disorders - Drug Profile 82
Product Description 82
Mechanism Of Action 82
R&D Progress 82
Small Molecules for Spinal Muscular Atrophy - Drug Profile 83
Product Description 83
Mechanism Of Action 83
R&D Progress 83
Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 84
Product Description 84
Mechanism Of Action 84
R&D Progress 84
Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 85
Product Description 85
Mechanism Of Action 85
R&D Progress 85
Small Molecules to Activate Survival Motor Neuron Protein for Spinal Muscular Atrophy - Drug Profile 86
Product Description 86
Mechanism Of Action 86
R&D Progress 86
Small Molecules to Activate Troponin for Non-Neuromuscular and Neuromuscular Disorders - Drug Profile 87
Product Description 87
Mechanism Of Action 87
R&D Progress 87
Small Molecules to Inhibit GSK-3 for Spinal Muscular Atrophy - Drug Profile 88
Product Description 88
Mechanism Of Action 88
R&D Progress 88
tirasemtiv - Drug Profile 89
Product Description 89
Mechanism Of Action 89
R&D Progress 89
VYSMN-101 - Drug Profile 100
Product Description 100
Mechanism Of Action 100
R&D Progress 100
Spinal Muscular Atrophy (SMA) - Dormant Projects 101
Spinal Muscular Atrophy (SMA) - Discontinued Products 103
Spinal Muscular Atrophy (SMA) - Product Development Milestones 104
Featured News & Press Releases 104
Apr 21, 2016: Ionis Pharmaceuticals Reports Data Update from Nusinersen Phase 2 Study in Infants with Spinal Muscular Atrophy and Reviews Neurological Disease Franchise 104
Mar 31, 2016: Ionis Pharmaceuticals Advances Nusinersen in the Ongoing SHINE Study in Patients with SMA 105
Mar 23, 2016: RaNA Therapeutics Presents Data on Advances in RNA Therapy Targeting Spinal Muscular Atrophy 105
Jan 12, 2016: Ionis Pharmaceuticals Completes Target Enrollment for Nusinersen Phase 3 Study, CHERISH, in Children with Spinal Muscular Atrophy 106
Jan 07, 2016: Spinal Muscular Atrophy Program Advances with Additional Product Candidate Entering Clinical Development 106
Jan 04, 2016: Cytokinetics Announces Start Of Phase 2 Clinical Trial Of Ck-2127107 In Patients With Spinal Muscular Atrophy 107
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 108
Oct 04, 2015: Phase 2 Data from Spinal Muscular Atrophy Program Presented at the 20th International Annual Congress of the World Muscle Society 109
Jul 14, 2015: Isis Pharmaceuticals Earns $2.15M for Advancing ISIS-SMN in Children With Spinal Muscular Atrophy 110
Jun 22, 2015: BioBlast Announces Positive Preclinical Proof-Of-Concept Results Of Its Novel Read- through Platform in Spinal Muscular Atrophy 110
Jun 22, 2015: Isis Pharmaceuticals Reports Data from ISIS-SMN Rx in Children with Spinal Muscular Atrophy 111
Jun 19, 2015: Cytokinetics Presents Results From Phase I Studies Of Ck-2127107 112
Jun 18, 2015: BioBlast Pharma to Present Preclinical Proofs of Concept in Spinal Muscular Atrophy at Two Leading Scientific Meetings in June 112
Jun 11, 2015: Isis Pharmaceuticals Reports Data From ISIS-SMN Rx Phase 2 Study in Infants With Spinal Muscular Atrophy 113
Apr 20, 2015: Phase 1 Data from Spinal Muscular Atrophy Program to be Presented at the 2015 American Academy of Neurology Annual Meeting 114
Appendix 115
Methodology 115
Coverage 115
Secondary Research 115
Primary Research 115
Expert Panel Validation 115
Contact Us 115
Disclaimer 116
List of Tables
Number of Products under Development for Spinal Muscular Atrophy (SMA), H2 2016 11
Number of Products under Development for Spinal Muscular Atrophy (SMA) - Comparative Analysis, H2 2016 12
Number of Products under Development by Companies, H2 2016 14
Number of Products under Investigation by Universities/Institutes, H2 2016 15
Comparative Analysis by Late Stage Development, H2 2016 16
Comparative Analysis by Clinical Stage Development, H2 2016 17
Comparative Analysis by Early Stage Development, H2 2016 18
Products under Development by Companies, H2 2016 19
Products under Investigation by Universities/Institutes, H2 2016 20
Spinal Muscular Atrophy (SMA) - Pipeline by Astellas Pharma Inc., H2 2016 21
Spinal Muscular Atrophy (SMA) - Pipeline by AveXis, Inc., H2 2016 22
Spinal Muscular Atrophy (SMA) - Pipeline by Bio Blast Pharma Ltd., H2 2016 23
Spinal Muscular Atrophy (SMA) - Pipeline by Cytokinetics, Inc., H2 2016 24
Spinal Muscular Atrophy (SMA) - Pipeline by F. Hoffmann-La Roche Ltd., H2 2016 25
Spinal Muscular Atrophy (SMA) - Pipeline by Genethon, H2 2016 26
Spinal Muscular Atrophy (SMA) - Pipeline by Genzyme Corporation, H2 2016 27
Spinal Muscular Atrophy (SMA) - Pipeline by Ionis Pharmaceuticals, Inc., H2 2016 28
Spinal Muscular Atrophy (SMA) - Pipeline by Neurodyn Inc., H2 2016 29
Spinal Muscular Atrophy (SMA) - Pipeline by Neurotune AG, H2 2016 30
Spinal Muscular Atrophy (SMA) - Pipeline by Novartis AG, H2 2016 31
Spinal Muscular Atrophy (SMA) - Pipeline by Paratek Pharmaceuticals, Inc., H2 2016 32
Spinal Muscular Atrophy (SMA) - Pipeline by RaNA Therapeutics, Inc., H2 2016 33
Spinal Muscular Atrophy (SMA) - Pipeline by Sarepta Therapeutics, Inc., H2 2016 34
Spinal Muscular Atrophy (SMA) - Pipeline by Voyager Therapeutics, Inc., H2 2016 35
Spinal Muscular Atrophy (SMA) - Pipeline by WAVE Life Sciences Ltd., H2 2016 36
Assessment by Monotherapy Products, H2 2016 37
Number of Products by Stage and Target, H2 2016 39
Number of Products by Stage and Mechanism of Action, H2 2016 41
Number of Products by Stage and Route of Administration, H2 2016 43
Number of Products by Stage and Molecule Type, H2 2016 45
Spinal Muscular Atrophy (SMA) - Dormant Projects, H2 2016 101
Spinal Muscular Atrophy (SMA) - Dormant Projects (Contd..1), H2 2016 102
Spinal Muscular Atrophy (SMA) - Discontinued Products, H2 2016 103
List of Figures
Number of Products under Development for Spinal Muscular Atrophy (SMA), H2 2016 11
Number of Products under Development for Spinal Muscular Atrophy (SMA) - Comparative Analysis, H2 2016 12
Number of Products under Development by Companies, H2 2016 13
Number of Products under Investigation by Universities/Institutes, H2 2016 15
Comparative Analysis by Clinical Stage Development, H2 2016 17
Comparative Analysis by Early Stage Products, H2 2016 18
Assessment by Monotherapy Products, H2 2016 37
Number of Products by Targets, H2 2016 38
Number of Products by Stage and Targets, H2 2016 38
Number of Products by Mechanism of Actions, H2 2016 40
Number of Products by Stage and Mechanism of Actions, H2 2016 40
Number of Products by Routes of Administration, H2 2016 42
Number of Products by Stage and Routes of Administration, H2 2016 42
Number of Products by Molecule Types, H2 2016 44
Number of Products by Stage and Molecule Types, H2 2016 44
