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

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


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Executive Summary

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

Summary

Global Markets Direct's, '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 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 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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Bioblast Pharma Ltd.

Cellular Biomedicine Group, Inc.

Cytokinetics, Inc.

F. Hoffmann-La Roche Ltd.

Genethon

Genzyme Corporation

GMP-Orphan SAS

Isis Pharmaceuticals, Inc.

Longevity Biotech, Inc

Neurodyn Inc.

Neurotune AG

Nexgenix Pharmaceuticals, LLC

Novartis AG

OrphageniX, Inc.

Paratek Pharmaceuticals, Inc.

Pfizer Inc.

PTC Therapeutics, Inc.

Retrophin Inc.

Sarepta Therapeutics, Inc.

Trophos SA

Vybion, Inc.

Zambon Company S.p.A.

Spinal Muscular Atrophy (SMA) Therapeutic Products under Development, Key Players in Spinal Muscular Atrophy (SMA) Therapeutics, Spinal Muscular Atrophy (SMA) Pipeline Overview, Spinal Muscular Atrophy (SMA) Pipeline, Spinal Muscular Atrophy (SMA) Pipeline Assessment


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