Table of Contents
Table of Contents 2
List of Tables 8
List of Figures 9
Introduction 10
Global Markets Direct Report Coverage 10
Huntington's Disease Overview 11
Therapeutics Development 12
An Overview of Pipeline Products for Huntington's Disease 12
Huntington's Disease Therapeutics under Development by Companies 14
Huntington's Disease Therapeutics under Investigation by Universities/Institutes 17
Late Stage Products 19
Comparative Analysis 19
Mid Clinical Stage Products 20
Comparative Analysis 20
Early Clinical Stage Products 21
Comparative Analysis 21
Discovery and Pre-Clinical Stage Products 22
Comparative Analysis 22
Huntington's Disease Therapeutics – Products under Development by Companies 23
Huntington's Disease Therapeutics – Products under Investigation by Universities/Institutes 25
Companies Involved in Huntington's Disease Therapeutics Development 26
Genzyme Corporation 26
NsGene A/S 27
Pfizer Inc. 28
Hadasit Medical Research Services & Development Ltd 29
Living Cell Technologies Limited 30
NeuroSearch A/S 31
Cortex Pharmaceuticals, Inc. 32
Alnylam Pharmaceuticals, Inc 33
Phytopharm Plc 34
Prana Biotechnology Limited 35
Raptor Pharmaceuticals Corp. 36
Oryzon 37
The Avicena Group, Inc. 38
Ceregene, Inc. 39
Accera, Inc. 40
Wellstat Therapeutics Corporation 41
Aegera Therapeutics Inc. 42
GenKyoTex S.A. 43
Indus Biotech Private Limited 44
Siena Biotech S.p.A. 45
N-Gene Research Laboratories, Inc. 46
Vybion, Inc. 47
Varinel, Inc. 48
Prosensa Therapeutics B.V. 49
Huntington's Disease – Therapeutics Assessment 50
Assessment by Monotherapy Products 50
Assessment by Combination Products 51
Assessment by Route of Administration 52
Assessment by Molecule Type 54
Drug Profiles 57
Drug For Huntington's Disease - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
GKT-03 - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
ALN-HTT - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
CERE-120 - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
SEN196 - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
PBT2 - Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
RP103 - Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
HD-02 - Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
INDUS815C - Drug Profile 68
Product Description 68
Mechanism of Action 68
R&D Progress 68
AC-0523 - Drug Profile 69
Product Description 69
Mechanism of Action 69
R&D Progress 69
NSD-726 - Drug Profile 70
Product Description 70
Mechanism of Action 70
R&D Progress 70
Huntexil - Drug Profile 71
Product Description 71
Mechanism of Action 71
R&D Progress 71
Phenylbutyrate - Drug Profile 73
Product Description 73
Mechanism of Action 73
R&D Progress 73
Lithium + Depakote - Drug Profile 74
Product Description 74
Mechanism of Action 74
R&D Progress 74
CX929 - Drug Profile 75
Product Description 75
Mechanism of Action 75
R&D Progress 75
VAR10300 - Drug Profile 77
Product Description 77
Mechanism of Action 77
R&D Progress 77
NsG28 - Drug Profile 79
Product Description 79
Mechanism of Action 79
R&D Progress 79
NsG32 - Drug Profile 80
Product Description 80
Mechanism of Action 80
R&D Progress 80
NsG30 - Drug Profile 81
Product Description 81
Mechanism of Action 81
R&D Progress 81
NsG33 - Drug Profile 82
Product Description 82
Mechanism of Action 82
R&D Progress 82
NsG-33 - Drug Profile 84
Product Description 84
Mechanism of Action 84
R&D Progress 84
NsG35 - Drug Profile 85
Product Description 85
Mechanism of Action 85
R&D Progress 85
Cogane - Drug Profile 86
Product Description 86
Mechanism of Action 86
R&D Progress 86
Zyprexa Velotab - Drug Profile 88
Product Description 88
Mechanism of Action 88
R&D Progress 88
Sunphenon EGCg - Drug Profile 89
Product Description 89
Mechanism of Action 89
R&D Progress 89
Bendavia - Drug Profile 91
Product Description 91
Mechanism of Action 91
R&D Progress 91
Amantadine - Drug Profile 92
Product Description 92
Mechanism of Action 92
R&D Progress 92
Strattera - Drug Profile 93
Product Description 93
Mechanism of Action 93
R&D Progress 93
Citalopram - Drug Profile 94
Product Description 94
Mechanism of Action 94
R&D Progress 94
Coenzyme Q10 - Drug Profile 95
Product Description 95
Mechanism of Action 95
R&D Progress 95
UbiquinonE - Drug Profile 96
Product Description 96
Mechanism of Action 96
R&D Progress 96
PN-401 - Drug Profile 97
Product Description 97
Mechanism of Action 97
R&D Progress 97
Huntington's Disease Program - Drug Profile 99
Product Description 99
Mechanism of Action 99
R&D Progress 99
Cholesterol Absorption Inhibitor - Drug Profile 105
Product Description 105
Mechanism of Action 105
R&D Progress 105
NTCell - Drug Profile 106
Product Description 106
Mechanism of Action 106
R&D Progress 106
NG-094 - Drug Profile 107
Product Description 107
Mechanism of Action 107
R&D Progress 107
NP03 - Drug Profile 108
Product Description 108
Mechanism of Action 108
R&D Progress 108
Drug For Huntington's Disease - Drug Profile 110
Product Description 110
Mechanism of Action 110
R&D Progress 110
OXD-4 - Drug Profile 111
Product Description 111
Mechanism of Action 111
R&D Progress 111
Drug For Huntington's Disease - Drug Profile 112
Product Description 112
Mechanism of Action 112
R&D Progress 112
JM6 - Drug Profile 113
Product Description 113
Mechanism of Action 113
R&D Progress 113
Enzyme Inhibitor Program - Drug Profile 114
Product Description 114
Mechanism of Action 114
R&D Progress 114
Huntington's Disease Therapeutics – Drug Profile Updates 115
Huntington's Disease Therapeutics – Discontinued Products 127
Huntington's Disease Therapeutics - Dormant Products 128
Huntington's Disease – Product Development Milestones 129
Featured News & Press Releases 129
Feb 01, 2012: BrainStorm's NurOwn Stem Cell Technology Shows Promise For Treating Huntington's Disease 129
Jan 04, 2012: Prana Biotechnology Receives FDA Approval To Start Clinical Trial For Huntington's Disease Using PBT2 129
Dec 28, 2011: Alnylam Announces Publication Of Preclinical Results With ALN-HTT For Treatment Of Huntington's Disease 130
Dec 16, 2011: Journal Of Biological Chemistry Endorses Prana's Drug Pipeline Strategy For Treating Neurodegenerative Diseases 131
Oct 27, 2011: Australian Federal Government Funds Study Into Neuroprotective Benefits Of Prana's PBT2 132
Sep 27, 2011: NeuroSearch Provides Update On Huntexil Development Program And Plans Restructuring Of Company's Operations 132
Sep 19, 2011: Neurocrine Biosciences Initiates Phase II Clinical Trial Of NBI-98854 135
Sep 16, 2011: Prana’s Planned Huntington’s Trial Receives Positive Response From Patients And Researchers At World Congress On Huntington’s Disease, Melbourne 136
Sep 14, 2011: Prana Biotechnology Presents Research Data On PBT2 At World Congress On Huntington’s Disease, Melbourne 137
Sep 12, 2011: Prana Biotechnology Presents PBT2 Huntington's Disease Trial Data At World Congress On Huntington's Disease In Melbourne 137
Appendix 139
Methodology 139
Coverage 139
Secondary Research 139
Primary Research 139
Expert Panel Validation 140
Contact Us 140
Disclaimer 140
List of Tables
Number of Products Under Development for Huntington's Disease, H1 2012 12
Products under Development for Huntington's Disease – Comparative Analysis, H1 2012 13
Number of Products under Development by Companies, H1 2012 15
Number of Products under Development by Companies, H1 2012 (Contd..1) 16
Number of Products under Investigation by Universities/Institutes, H1 2012 18
Comparative Analysis by Late Stage Development, H1 2012 19
Comparative Analysis by Mid Clinical Stage Development, H1 2012 20
Comparative Analysis by Early Clinical Stage Development, H1 2012 21
Comparative Analysis by Discovery and Pre-Clinical Stage Development, H1 2012 22
Products under Development by Companies, H1 2012 23
Products under Investigation by Universities/Institutes, H1 2012 25
Genzyme Corporation, H1 2012 26
NsGene A/S, H1 2012 27
Pfizer Inc., H1 2012 28
Hadasit Medical Research Services & Development Ltd, H1 2012 29
Living Cell Technologies Limited, H1 2012 30
NeuroSearch A/S, H1 2012 31
Cortex Pharmaceuticals, Inc., H1 2012 32
Alnylam Pharmaceuticals, Inc, H1 2012 33
Phytopharm Plc, H1 2012 34
Prana Biotechnology Limited, H1 2012 35
Raptor Pharmaceuticals Corp., H1 2012 36
Oryzon, H1 2012 37
The Avicena Group, Inc., H1 2012 38
Ceregene, Inc., H1 2012 39
Accera, Inc., H1 2012 40
Wellstat Therapeutics Corporation, H1 2012 41
Aegera Therapeutics Inc., H1 2012 42
GenKyoTex S.A., H1 2012 43
Indus Biotech Private Limited, H1 2012 44
Siena Biotech S.p.A., H1 2012 45
N-Gene Research Laboratories, Inc., H1 2012 46
Vybion, Inc., H1 2012 47
Varinel, Inc., H1 2012 48
Prosensa Therapeutics B.V., H1 2012 49
Assessment by Monotherapy Products, H1 2012 50
Assessment by Combination Products, H1 2012 51
Assessment by Stage and Route of Administration, H1 2012 53
Assessment by Stage and Molecule Type, H1 2012 56
Huntington's Disease Therapeutics – Drug Profile Updates 115
Huntington's Disease Therapeutics – Discontinued Products 127
Huntington's Disease Therapeutics – Dormant Products 128
List of Figures
Number of Products under Development for Huntington's Disease, H1 2012 12
Products under Development for Huntington's Disease – Comparative Analysis, H1 2012 13
Products under Development by Companies, H1 2012 14
Products under Investigation by Universities/Institutes, H1 2012 17
Late Stage Products, H1 2012 19
Mid Clinical Stage Products, H1 2012 20
Early Clinical Stage Products, H1 2012 21
Discovery and Pre-Clinical Stage Products, H1 2012 22
Assessment by Monotherapy Products, H1 2012 50
Assessment by Combination Products, H1 2012 51
Assessment by Route of Administration, H1 2012 52
Assessment by Stage and Route of Administration, H1 2012 53
Assessment by Molecule Type, H1 2012 54
Assessment by Stage and Molecule Type, H1 2012 55