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

Published: October 2015
No. of Pages: 113
   

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

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

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

Published By: Global Markets Direct
Product Code: Global Markets Direct13305


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