Spinal Muscular Atrophy (SMA) – Pipeline Review, H2 2012
出 版 商：Global Markets Direct
出版日期：2012/08/31
頁 數：70頁
文件格式：PDF
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Spinal Muscular Atrophy (SMA) – Pipeline Review, H2 2012
Summary
Global Markets Direct’s, 'Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2012', provides an overview of the Spinal Muscular Atrophy (SMA) therapeutic pipeline. This report provides information on the therapeutic development for Spinal Muscular Atrophy (SMA), complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Spinal Muscular Atrophy (SMA). 'Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2012' 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.
Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.
Scope
- A snapshot of the global therapeutic scenario for Spinal Muscular Atrophy (SMA).
- 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.
- Coverage of products based on various stages of development ranging from discovery till registration stages.
- A feature on pipeline projects on the basis of monotherapy and combined therapeutics.
- Coverage of the Spinal Muscular Atrophy (SMA) pipeline on the basis of route of administration and molecule type.
- Profiles of late-stage pipeline products featuring sections on product description, mechanism of action and research & development progress.
- Key discontinued pipeline projects.
- Latest news and deals relating to the products.
Reasons to buy
- Identify and understand important and diverse types of therapeutics under development for Spinal Muscular Atrophy (SMA).
- Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.
- Plan mergers and acquisitions effectively by identifying players with the most promising pipeline.
- Devise corrective measures for pipeline projects by understanding Spinal Muscular Atrophy (SMA) pipeline depth and focus of Spinal Muscular Atrophy (SMA) 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.
Summary
Global Markets Direct’s, 'Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2012', provides an overview of the Spinal Muscular Atrophy (SMA) therapeutic pipeline. This report provides information on the therapeutic development for Spinal Muscular Atrophy (SMA), complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Spinal Muscular Atrophy (SMA). 'Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2012' 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.
Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.
Scope
- A snapshot of the global therapeutic scenario for Spinal Muscular Atrophy (SMA).
- 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.
- Coverage of products based on various stages of development ranging from discovery till registration stages.
- A feature on pipeline projects on the basis of monotherapy and combined therapeutics.
- Coverage of the Spinal Muscular Atrophy (SMA) pipeline on the basis of route of administration and molecule type.
- Profiles of late-stage pipeline products featuring sections on product description, mechanism of action and research & development progress.
- Key discontinued pipeline projects.
- Latest news and deals relating to the products.
Reasons to buy
- Identify and understand important and diverse types of therapeutics under development for Spinal Muscular Atrophy (SMA).
- Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.
- Plan mergers and acquisitions effectively by identifying players with the most promising pipeline.
- Devise corrective measures for pipeline projects by understanding Spinal Muscular Atrophy (SMA) pipeline depth and focus of Spinal Muscular Atrophy (SMA) 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 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Spinal Muscular Atrophy (SMA) Overview 7
Therapeutics Development 8
An Overview of Pipeline Products for Spinal Muscular Atrophy (SMA) 8
Spinal Muscular Atrophy (SMA) Therapeutics under Development by Companies 10
Spinal Muscular Atrophy (SMA) Therapeutics under Investigation by Universities/Institutes 12
Mid Clinical Stage Products 13
Comparative Analysis 13
Early Clinical Stage Products 14
Comparative Analysis 14
Discovery and Pre-Clinical Stage Products 15
Comparative Analysis 15
Spinal Muscular Atrophy (SMA) Therapeutics – Products under Development by Companies 16
Spinal Muscular Atrophy (SMA) Therapeutics – Products under Investigation by Universities/Institutes 17
Companies Involved in Spinal Muscular Atrophy (SMA) Therapeutics Development 18
F. Hoffmann-La Roche Ltd. 18
Isis Pharmaceuticals, Inc. 19
Oxford BioMedica plc 20
Paratek Pharmaceuticals, Inc. 21
Repligen Corporation 22
Trophos SA 23
Nexgenix Pharmaceuticals, LLC 24
Prosensa Therapeutics B.V. 25
Spinal Muscular Atrophy (SMA) – Therapeutics Assessment 26
Assessment by Monotherapy Products 26
Assessment by Combination Products 27
Assessment by Route of Administration 28
Assessment by Molecule Type 30
Drug Profiles 32
RG3039 - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
TRO-19622 - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
SMN1-G - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
Tetracyclines For SMA - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
NXD30001 - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
Sodium Phenylbutyrate (NaPB) - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
Valproic Acid - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
Valproic acid + Levocarnitine - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 43
Somatotropin - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
ISIS-SMN - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
PRO105 - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
Depakote + Carnitor - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
Drug For Spinal Muscular Atrophy - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
Drug For Spinal Muscular Atrophy - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
Spinal Muscular Atrophy Programme - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
RE-003 - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
Fasudil - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
ND-602 - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
Spinal Muscular Atrophy (SMA) Therapeutics – Drug Profile Updates 55
Spinal Muscular Atrophy (SMA) Therapeutics – Discontinued Products 59
Spinal Muscular Atrophy (SMA) Therapeutics - Dormant Products 60
Spinal Muscular Atrophy (SMA) – Product Development Milestones 61
Featured News & Press Releases 61
Apr 25, 2012: Repligen Reports Positive Results From Phase I Clinical Trial Of RG3039 For Spinal Muscular Atrophy 61
Mar 06, 2012: New Drug Shows Promise In Treating Spinal Muscular Atrophy, Researchers Discover 62
Dec 19, 2011: Isis Initiates Phase I Clinical Study Of ISIS-SMNRx In Patients With Spinal Muscular Atrophy 63
Oct 28, 2011: Repligen Provides Update On Phase I Trial For Families Of Spinal Muscular Atrophy Drug RG3039 64
Sep 08, 2011: Trophos Completes Patient Enrolment In Pivotal Efficacy Study Of Olesoxime In Spinal Muscular Atrophy 64
Jun 23, 2011: Repligen Receives US Fast Track Designation And European Orphan Medicinal Product Recommendation For RG3039 In Spinal Muscular Atrophy 65
May 19, 2011: Repligen Receives FDA Approval To Initiate Phase I Clinical Trial Of RG3039 For Treatment Of Spinal Muscular Atrophy 66
Apr 15, 2011: Trophos Presents Data At AAN On Olesoxime In Spinal Muscular Atrophy And MS 66
Oct 15, 2010: Trophos Initiates Pivotal Efficacy Study Of Olesoxime In Spinal Muscular Atrophy 67
Mar 12, 2009: Isis Pharmaceuticals Adds New Drug ISIS-SMNRx To Development Pipeline For The Treatment Of Spinal Muscular Atrophy 68
Appendix 69
Methodology 69
Coverage 69
Secondary Research 69
Primary Research 69
Expert Panel Validation 69
Contact Us 70
Disclaimer 70
List of Tables
Number of Products Under Development for Spinal Muscular Atrophy (SMA), H2 2012 8
Products under Development for Spinal Muscular Atrophy (SMA) – Comparative Analysis, H2 2012 9
Number of Products under Development by Companies, H2 2012 11
Number of Products under Investigation by Universities/Institutes, H2 2012 12
Comparative Analysis by Mid Clinical Stage Development, H2 2012 13
Comparative Analysis by Early Clinical Stage Development, H2 2012 14
Comparative Analysis by Discovery and Pre-Clinical Stage Development, H2 2012 15
Products under Development by Companies, H2 2012 16
Products under Investigation by Universities/Institutes, H2 2012 17
F. Hoffmann-La Roche Ltd., H2 2012 18
Isis Pharmaceuticals, Inc., H2 2012 19
Oxford BioMedica plc, H2 2012 20
Paratek Pharmaceuticals, Inc., H2 2012 21
Repligen Corporation, H2 2012 22
Trophos SA, H2 2012 23
Nexgenix Pharmaceuticals, LLC, H2 2012 24
Prosensa Therapeutics B.V., H2 2012 25
Assessment by Monotherapy Products, H2 2012 26
Assessment by Combination Products, H2 2012 27
Assessment by Stage and Route of Administration, H2 2012 29
Assessment by Stage and Molecule Type, H2 2012 31
Spinal Muscular Atrophy (SMA) Therapeutics – Drug Profile Updates 55
Spinal Muscular Atrophy (SMA) Therapeutics – Discontinued Products 59
Spinal Muscular Atrophy (SMA) Therapeutics – Dormant Products 60
List of Figures
Number of Products under Development for Spinal Muscular Atrophy (SMA), H2 2012 8
Products under Development for Spinal Muscular Atrophy (SMA) – Comparative Analysis, H2 2012 9
Products under Development by Companies, H2 2012 10
Products under Investigation by Universities/Institutes, H2 2012 12
Mid Clinical Stage Products, H2 2012 13
Early Clinical Stage Products, H2 2012 14
Discovery and Pre-Clinical Stage Products, H2 2012 15
Assessment by Monotherapy Products, H2 2012 26
Assessment by Combination Products, H2 2012 27
Assessment by Route of Administration, H2 2012 28
Assessment by Stage and Route of Administration, H2 2012 29
Assessment by Molecule Type, H2 2012 30
Assessment by Stage and Molecule Type, H2 2012 31
Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Spinal Muscular Atrophy (SMA) Overview 7
Therapeutics Development 8
An Overview of Pipeline Products for Spinal Muscular Atrophy (SMA) 8
Spinal Muscular Atrophy (SMA) Therapeutics under Development by Companies 10
Spinal Muscular Atrophy (SMA) Therapeutics under Investigation by Universities/Institutes 12
Mid Clinical Stage Products 13
Comparative Analysis 13
Early Clinical Stage Products 14
Comparative Analysis 14
Discovery and Pre-Clinical Stage Products 15
Comparative Analysis 15
Spinal Muscular Atrophy (SMA) Therapeutics – Products under Development by Companies 16
Spinal Muscular Atrophy (SMA) Therapeutics – Products under Investigation by Universities/Institutes 17
Companies Involved in Spinal Muscular Atrophy (SMA) Therapeutics Development 18
F. Hoffmann-La Roche Ltd. 18
Isis Pharmaceuticals, Inc. 19
Oxford BioMedica plc 20
Paratek Pharmaceuticals, Inc. 21
Repligen Corporation 22
Trophos SA 23
Nexgenix Pharmaceuticals, LLC 24
Prosensa Therapeutics B.V. 25
Spinal Muscular Atrophy (SMA) – Therapeutics Assessment 26
Assessment by Monotherapy Products 26
Assessment by Combination Products 27
Assessment by Route of Administration 28
Assessment by Molecule Type 30
Drug Profiles 32
RG3039 - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
TRO-19622 - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
SMN1-G - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
Tetracyclines For SMA - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
NXD30001 - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
Sodium Phenylbutyrate (NaPB) - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
Valproic Acid - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
Valproic acid + Levocarnitine - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 43
Somatotropin - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
ISIS-SMN - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
PRO105 - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
Depakote + Carnitor - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
Drug For Spinal Muscular Atrophy - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
Drug For Spinal Muscular Atrophy - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
Spinal Muscular Atrophy Programme - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
RE-003 - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
Fasudil - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
ND-602 - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
Spinal Muscular Atrophy (SMA) Therapeutics – Drug Profile Updates 55
Spinal Muscular Atrophy (SMA) Therapeutics – Discontinued Products 59
Spinal Muscular Atrophy (SMA) Therapeutics - Dormant Products 60
Spinal Muscular Atrophy (SMA) – Product Development Milestones 61
Featured News & Press Releases 61
Apr 25, 2012: Repligen Reports Positive Results From Phase I Clinical Trial Of RG3039 For Spinal Muscular Atrophy 61
Mar 06, 2012: New Drug Shows Promise In Treating Spinal Muscular Atrophy, Researchers Discover 62
Dec 19, 2011: Isis Initiates Phase I Clinical Study Of ISIS-SMNRx In Patients With Spinal Muscular Atrophy 63
Oct 28, 2011: Repligen Provides Update On Phase I Trial For Families Of Spinal Muscular Atrophy Drug RG3039 64
Sep 08, 2011: Trophos Completes Patient Enrolment In Pivotal Efficacy Study Of Olesoxime In Spinal Muscular Atrophy 64
Jun 23, 2011: Repligen Receives US Fast Track Designation And European Orphan Medicinal Product Recommendation For RG3039 In Spinal Muscular Atrophy 65
May 19, 2011: Repligen Receives FDA Approval To Initiate Phase I Clinical Trial Of RG3039 For Treatment Of Spinal Muscular Atrophy 66
Apr 15, 2011: Trophos Presents Data At AAN On Olesoxime In Spinal Muscular Atrophy And MS 66
Oct 15, 2010: Trophos Initiates Pivotal Efficacy Study Of Olesoxime In Spinal Muscular Atrophy 67
Mar 12, 2009: Isis Pharmaceuticals Adds New Drug ISIS-SMNRx To Development Pipeline For The Treatment Of Spinal Muscular Atrophy 68
Appendix 69
Methodology 69
Coverage 69
Secondary Research 69
Primary Research 69
Expert Panel Validation 69
Contact Us 70
Disclaimer 70
List of Tables
Number of Products Under Development for Spinal Muscular Atrophy (SMA), H2 2012 8
Products under Development for Spinal Muscular Atrophy (SMA) – Comparative Analysis, H2 2012 9
Number of Products under Development by Companies, H2 2012 11
Number of Products under Investigation by Universities/Institutes, H2 2012 12
Comparative Analysis by Mid Clinical Stage Development, H2 2012 13
Comparative Analysis by Early Clinical Stage Development, H2 2012 14
Comparative Analysis by Discovery and Pre-Clinical Stage Development, H2 2012 15
Products under Development by Companies, H2 2012 16
Products under Investigation by Universities/Institutes, H2 2012 17
F. Hoffmann-La Roche Ltd., H2 2012 18
Isis Pharmaceuticals, Inc., H2 2012 19
Oxford BioMedica plc, H2 2012 20
Paratek Pharmaceuticals, Inc., H2 2012 21
Repligen Corporation, H2 2012 22
Trophos SA, H2 2012 23
Nexgenix Pharmaceuticals, LLC, H2 2012 24
Prosensa Therapeutics B.V., H2 2012 25
Assessment by Monotherapy Products, H2 2012 26
Assessment by Combination Products, H2 2012 27
Assessment by Stage and Route of Administration, H2 2012 29
Assessment by Stage and Molecule Type, H2 2012 31
Spinal Muscular Atrophy (SMA) Therapeutics – Drug Profile Updates 55
Spinal Muscular Atrophy (SMA) Therapeutics – Discontinued Products 59
Spinal Muscular Atrophy (SMA) Therapeutics – Dormant Products 60
List of Figures
Number of Products under Development for Spinal Muscular Atrophy (SMA), H2 2012 8
Products under Development for Spinal Muscular Atrophy (SMA) – Comparative Analysis, H2 2012 9
Products under Development by Companies, H2 2012 10
Products under Investigation by Universities/Institutes, H2 2012 12
Mid Clinical Stage Products, H2 2012 13
Early Clinical Stage Products, H2 2012 14
Discovery and Pre-Clinical Stage Products, H2 2012 15
Assessment by Monotherapy Products, H2 2012 26
Assessment by Combination Products, H2 2012 27
Assessment by Route of Administration, H2 2012 28
Assessment by Stage and Route of Administration, H2 2012 29
Assessment by Molecule Type, H2 2012 30
Assessment by Stage and Molecule Type, H2 2012 31
