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Spinal Muscular Atrophy (SMA)-Pipeline Review, H2 2016

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Spinal Muscular Atrophy (SMA)-Pipeline Review, H2 2016


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Spinal Muscular Atrophy (SMA)-Pipeline Review, H2 2016

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Spinal Muscular Atrophy (SMA)-Pipeline Review, H2 2016



Executive Summary

Spinal Muscular Atrophy (SMA)-Pipeline Review, H2 2016

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

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

To know more information on Purchase by Section, please send a mail to sales@kenresearch.com

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

Single User License:
Report can be used by individual purchaser only

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Report can be shared by unlimited users within one corporate location, e.g. a regional office

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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.

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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