Muscular Dystrophy – Pipeline Review, Q2 2011
Muscular Dystrophy - Pipeline Review, Q2 2011
Reference Code: GMDHC630IDB
Publication Date: June 2011
Muscular Dystrophy – Pipeline Review, Q2 2011 GMDHC630IDB / Pub June 2011
© Global Markets Direct. This report is a licensed product and is not to be photocopied
Page(1)
Muscular Dystrophy – Pipeline Review, Q2 2011
Ta b le o f Co n te n ts
Table of Contents 2
List of Tables 7
List of Figures 8
Introduction 9
Global Markets Direct Report Coverage 9
Muscular Dystrophy Overview 10
Therapeutics Development 11
An Overview of Pipeline Products for Muscular Dystrophy 11
Muscular Dystrophy Therapeutics under Development by Companies 13
Muscular Dystrophy Therapeutics under Investigation by Universities/Institutes 15
Late Stage Products 18
Comparative Analysis 18
Mid Clinical Stage Products 19
Comparative Analysis 19
Early Clinical Stage Products 20
Comparative Analysis 20
Discovery and Pre-Clinical Stage Products 21
Comparative Analysis 21
Muscular Dystrophy Therapeutics - Products under Development by Companies 22
Muscular Dystrophy Therapeutics - Products under Investigation by Universities/Institutes 24
Companies Involved in Muscular Dystrophy Therapeutics Development 26
GlaxoSmithKline plc 26
Takeda Pharmaceutical Company Limited 26
AVI BioPharma Inc. 27
Insmed Incorporated 27
Galapagos NV 28
Summit Corporation plc 29
Santhera Pharmaceuticals Holding AG 30
Amsterdam Molecular Therapeutics BV 30
Shenzhen Beike Biotechnology Co., Ltd. 31
Acceleron Pharma, Inc. 31
PTC Therapeutics, Inc. 32
UMN Pharma Inc. 32
SanBio, Inc. 33
Asklepios BioPharmaceutical, Inc. 33
Prosensa Therapeutics B.V. 34
Muscular Dystrophy - Therapeutics Assessment 35
Assessment by Monotherapy Products 35
Assessment by Combination Products 36
Assessment by Route of Administration 37
Assessment by Molecule Type 39
Drug Profiles 41
Ataluren - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
Sovrima - Drug Profile 43
Muscular Dystrophy – Pipeline Review, Q2 2011 GMDHC630IDB / Pub June 2011
© Global Markets Direct. This report is a licensed product and is not to be photocopied Page(2)
Muscular Dystrophy – Pipeline Review, Q2 2011
Product Description 43
Mechanism of Action 43
R&D Progress 43
Iplex - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
SMT C1100 - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
ACE-031 - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
AVI-4658 - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
AVI-5038 - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
GSK2402968 - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
PRO044 - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
L-Glutamine - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
Gentamicin - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
G100192 - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
Tinset - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
AMT-080 - Drug Profile 58
Muscular Dystrophy – Pipeline Review, Q2 2011 GMDHC630IDB / Pub June 2011
© Global Markets Direct. This report is a licensed product and is not to be photocopied Page(3)
Muscular Dystrophy – Pipeline Review, Q2 2011
Product Description 58
Mechanism of Action 58
R&D Progress 58
SB308 - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
Anti-Sense Exon-Skipping Therapy - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
Biostrophin - Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
Drug for Myotonic Dystrophy Type 1 - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
Creatine Monohydrate + Glutamine - Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
Glutamine + Creatine Monohydrate - Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
UMN-03 - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
GLPG0492 - Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
Gentamicin - Drug Profile 68
Product Description 68
Mechanism of Action 68
R&D Progress 68
Catena - Drug Profile 69
Product Description 69
Mechanism of Action 69
R&D Progress 69
Deflazacort - Drug Profile 70
Product Description 70
Mechanism of Action 70
R&D Progress 70
Sunphenon EGCg - Drug Profile 71
Muscular Dystrophy – Pipeline Review, Q2 2011 GMDHC630IDB / Pub June 2011
© Global Markets Direct. This report is a licensed product and is not to be photocopied Page(4)
Muscular Dystrophy – Pipeline Review, Q2 2011
Product Description 71
Mechanism of Action 71
R&D Progress 71
Pentoxifylline - Drug Profile 73
Product Description 73
Mechanism of Action 73
R&D Progress 73
Prednisone - Drug Profile 74
Product Description 74
Mechanism of Action 74
R&D Progress 74
Coenzyme Q10 + Lisinopril - Drug Profile 75
Product Description 75
Mechanism of Action 75
R&D Progress 75
rAAV2.5-CMV - Drug Profile 76
Product Description 76
Mechanism of Action 76
R&D Progress 76
Revatio - Drug Profile 77
Product Description 77
Mechanism of Action 77
R&D Progress 77
Glutamine + Creatine - Drug Profile 78
Product Description 78
Mechanism of Action 78
R&D Progress 78
IGF-1 - Drug Profile 80
Product Description 80
Mechanism of Action 80
R&D Progress 80
Dehydroepiandrosterone - Drug Profile 81
Product Description 81
Mechanism of Action 81
R&D Progress 81
Epigallocatechin-Gallate - Drug Profile 82
Product Description 82
Mechanism of Action 82
R&D Progress 82
PRO045 - Drug Profile 83
Product Description 83
Mechanism of Action 83
R&D Progress 83
PRO053 - Drug Profile 84
Product Description 84
Mechanism of Action 84
R&D Progress 84
PRO135 - Drug Profile 85
Muscular Dystrophy – Pipeline Review, Q2 2011 GMDHC630IDB / Pub June 2011
© Global Markets Direct. This report is a licensed product and is not to be photocopied Page(5)
Muscular Dystrophy – Pipeline Review, Q2 2011
Product Description 85
Mechanism of Action 85
R&D Progress 85
PRO052 - Drug Profile 86
Product Description 86
Mechanism of Action 86
R&D Progress 86
PRO055 - Drug Profile 87
Product Description 87
Mechanism of Action 87
R&D Progress 87
rAAV1.tMCK.Human-Alpha-Sarcoglycan - Drug Profile 88
Product Description 88
Mechanism of Action 88
R&D Progress 88
Flavocoxid - Drug Profile 89
Product Description