Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2014

Published: Jul, 2014 | Pages: 114 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)

Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2014

Summary

Global Markets Direct's, 'Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2014', 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
Genzyme Corporation 20
F. Hoffmann-La Roche Ltd. 21
Isis Pharmaceuticals, Inc. 22
Pfizer Inc. 23
Zambon Company S.p.A. 24
Paratek Pharmaceuticals, Inc. 25
Cytokinetics, Inc. 26
Neurotune AG 27
Trophos SA 28
Nexgenix Pharmaceuticals, LLC 29
Genethon 30
Vybion, Inc. 31
OrphageniX, Inc. 32
Retrophin, LLC 33
Neurodyn Inc. 34
GMP-Orphan SAS 35
Cellular Biomedicine Group, Inc. 36
Spinal Muscular Atrophy (SMA) - Therapeutics Assessment 37
Assessment by Monotherapy Products 37
Assessment by Target 38
Assessment by Mechanism of Action 41
Assessment by Route of Administration 44
Assessment by Molecule Type 46
Drug Profiles 48
olesoxime - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
ISIS-SMNRx - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
PF-06687859 - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
RG-7800 - 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 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
AAV9-SMN Gene Therapy - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
tirasemtiv - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
PTK-SMA-2 - Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
NXD-30001 - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
DNA Oligonucleotides for Spinal Muscular Atrophy - Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
RE-003 - Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
Gene Therapy for Spinal Muscular Atrophy - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
ND-602 - Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
Antisense Oligonucleotide for Spinal Muscular Atrophy and Myotonic Dystrophy - Drug Profile 68
Product Description 68
Mechanism of Action 68
R&D Progress 68
Motor Neuron Precursor Cells - Drug Profile 69
Product Description 69
Mechanism of Action 69
R&D Progress 69
Small Molecule to Activate SMN Protein for Spinal Muscular Atrophy - Drug Profile 70
Product Description 70
Mechanism of Action 70
R&D Progress 70
Agrin Biologic For SMA - Drug Profile 72
Product Description 72
Mechanism of Action 72
R&D Progress 72
sodium phenylbutyrate - Drug Profile 73
Product Description 73
Mechanism of Action 73
R&D Progress 73
Small Molecules for CNS Disorders - Drug Profile 74
Product Description 74
Mechanism of Action 74
R&D Progress 74
PMO-25 - Drug Profile 75
Product Description 75
Mechanism of Action 75
R&D Progress 75
Synthetic Peptides for Central Nervous System Disorders - Drug Profile 76
Product Description 76
Mechanism of Action 76
R&D Progress 76
BBrm-01 - Drug Profile 77
Product Description 77
Mechanism of Action 77
R&D Progress 77
Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 78
Product Description 78
Mechanism of Action 78
R&D Progress 78
Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 80
Product Description 80
Mechanism of Action 80
R&D Progress 80
INT-41 - Drug Profile 81
Product Description 81
Mechanism of Action 81
R&D Progress 81
Antisense Oligonucleotides for Spinal Muscular Atrophy - Drug Profile 83
Product Description 83
Mechanism of Action 83
R&D Progress 83
ZP-037 - Drug Profile 84
Product Description 84
Mechanism of Action 84
R&D Progress 84
Spinal Muscular Atrophy (SMA) - Recent Pipeline Updates 85
Spinal Muscular Atrophy (SMA) - Dormant Projects 100
Spinal Muscular Atrophy (SMA) - Product Development Milestones 101
Featured News & Press Releases 101
May 22, 2014: Gene therapy extends survival in an animal model of spinal muscular atrophy 101
Apr 30, 2014: Isis Pharmaceuticals Reports Updated Results From ISIS-SMN Rx Clinical Studies In Infants And Children With Spinal Muscular Atrophy 101
Mar 10, 2014: Trophos announces top-line results of pivotal trial of olesoxime in spinal muscular atrophy 104
Feb 21, 2014: Isis Pharmaceuticals Reports Interim Results from ISIS-SMN Rx Multiple Dose Study in Children with Spinal Muscular Atrophy 105
Feb 13, 2014: Isis Pharmaceuticals Advances ISIS-SMN Rx in Infants and Children with Spinal Muscular Atrophy 106
Jan 22, 2014: Joint program in spinal muscular atrophy enters first stage of clinical development 107
Nov 22, 2013: Isis Pharmaceuticals to Investigate a Higher Dose of ISIS-SMN Rx in Children with Spinal Muscular Atrophy 108
Nov 12, 2013: Isis Pharmaceuticals Earns $1.5M from the Advancement of the Phase 2 Study of ISIS-SMN Rx in Infants with Spinal Muscular Atrophy 108
Nov 05, 2013: Trophos completes pivotal phase II/III study of olesoxime in Spinal Muscular Atrophy 109
Sep 19, 2013: Isis Reports Follow-Up Data From ISIS-SMN Rx Phase 1 Study In Children With Spinal Muscular Atrophy 110
Appendix 113
Methodology 113
Coverage 113
Secondary Research 113
Primary Research 113
Expert Panel Validation 113
Contact Us 114
Disclaimer 114

List of Tables
Number of Products under Development for Spinal Muscular Atrophy (SMA), H2 2014 10
Number of Products under Development for Spinal Muscular Atrophy (SMA) - Comparative Analysis, H2 2014 11
Number of Products under Development by Companies, H2 2014 13
Number of Products under Investigation by Universities/Institutes, H2 2014 14
Comparative Analysis by Late Stage Development, H2 2014 15
Comparative Analysis by Clinical Stage Development, H2 2014 16
Comparative Analysis by Early Stage Development, H2 2014 17
Products under Development by Companies, H2 2014 18
Products under Investigation by Universities/Institutes, H2 2014 19
Spinal Muscular Atrophy (SMA) - Pipeline by Genzyme Corporation, H2 2014 20
Spinal Muscular Atrophy (SMA) - Pipeline by F. Hoffmann-La Roche Ltd., H2 2014 21
Spinal Muscular Atrophy (SMA) - Pipeline by Isis Pharmaceuticals, Inc., H2 2014 22
Spinal Muscular Atrophy (SMA) - Pipeline by Pfizer Inc., H2 2014 23
Spinal Muscular Atrophy (SMA) - Pipeline by Zambon Company S.p.A., H2 2014 24
Spinal Muscular Atrophy (SMA) - Pipeline by Paratek Pharmaceuticals, Inc., H2 2014 25
Spinal Muscular Atrophy (SMA) - Pipeline by Cytokinetics, Inc., H2 2014 26
Spinal Muscular Atrophy (SMA) - Pipeline by Neurotune AG, H2 2014 27
Spinal Muscular Atrophy (SMA) - Pipeline by Trophos SA, H2 2014 28
Spinal Muscular Atrophy (SMA) - Pipeline by Nexgenix Pharmaceuticals, LLC, H2 2014 29
Spinal Muscular Atrophy (SMA) - Pipeline by Genethon, H2 2014 30
Spinal Muscular Atrophy (SMA) - Pipeline by Vybion, Inc., H2 2014 31
Spinal Muscular Atrophy (SMA) - Pipeline by OrphageniX, Inc., H2 2014 32
Spinal Muscular Atrophy (SMA) - Pipeline by Retrophin, LLC, H2 2014 33
Spinal Muscular Atrophy (SMA) - Pipeline by Neurodyn Inc., H2 2014 34
Spinal Muscular Atrophy (SMA) - Pipeline by GMP-Orphan SAS, H2 2014 35
Spinal Muscular Atrophy (SMA) - Pipeline by Cellular Biomedicine Group, Inc., H2 2014 36
Assessment by Monotherapy Products, H2 2014 37
Number of Products by Stage and Target, H2 2014 40
Number of Products by Stage and Mechanism of Action, H2 2014 43
Number of Products by Stage and Route of Administration, H2 2014 45
Number of Products by Stage and Molecule Type, H2 2014 47
Spinal Muscular Atrophy (SMA) Therapeutics - Recent Pipeline Updates, H2 2014 85
Spinal Muscular Atrophy (SMA) - Dormant Projects, H2 2014 100