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Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2015

Published: Jan-2015 | Format: PDF | Global Markets Direct | Number of pages: 132 | Code: MRS - 18404


Global Markets Directs, 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 Directs 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 Directs 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 Directs 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.

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 Content

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

List of Figures

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 12
Comparative Analysis by Clinical Stage Development, H1 2015 17
Comparative Analysis by Early Stage Products, H1 2015 18
Assessment by Monotherapy Products, H1 2015 45
Number of Products by Top 10 Targets, H1 2015 46
Number of Products by Stage and Top 10 Targets, H1 2015 46
Number of Products by Top 10 Mechanism of Actions, H1 2015 48
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015 48
Number of Products by Top 10 Routes of Administration, H1 2015 50
Number of Products by Stage and Top 10 Routes of Administration, H1 2015 51
Number of Products by Top 10 Molecule Types, H1 2015 52
Number of Products by Stage and Top 10 Molecule Types, H1 2015 52

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