Duchenne Muscular Dystrophy - Pipeline Review, H2 2011 by GlobalMarketsDirect

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									Duchenne Muscular Dystrophy – Pipeline Review, H2 2011




   Duchenne Muscular Dystrophy - Pipeline Review, H2
                        2011
                                                                                          Reference Code: GMDHC1347IDB

                                                                                          Publication Date: September 2011




Duchenne Muscular Dystrophy – Pipeline Review, H2 2011                                                        GMDHC1347IDB / Pub September 2011
© Global Markets Direct. This report is a licensed product and is not to be photocopied                                                 Page(1)
Duchenne Muscular Dystrophy – Pipeline Review, H2 2011



Ta b le o f Co n te n ts
Table of Contents                                                                                                                2
List of Tables                                                                                                                   5
List of Figures                                                                                                                  6
Introduction                                                                                                                     7
     Global Markets Direct Report Coverage                                                                                       7
Duchenne Muscular Dystrophy Overview                                                                                             8
Therapeutics Development                                                                                                         9
     An Overview of Pipeline Products for Duchenne Muscular Dystrophy                                                            9
Duchenne Muscular Dystrophy Therapeutics under Development by Companies                                                         11
Duchenne Muscular Dystrophy Therapeutics under Investigation by Universities/Institutes                                         13
Late Stage Products                                                                                                             14
     Comparative Analysis                                                                                                       14
Mid Clinical Stage Products                                                                                                     15
     Comparative Analysis                                                                                                       15
Early Clinical Stage Products                                                                                                   16
     Comparative Analysis                                                                                                       16
Discovery and Pre-Clinical Stage Products                                                                                       17
     Comparative Analysis                                                                                                       17
Duchenne Muscular Dystrophy Therapeutics - Products under Development by Companies                                              18
Duchenne Muscular Dystrophy Therapeutics - Products under Investigation by Universities/Institutes                              20
Companies Involved in Duchenne Muscular Dystrophy Therapeutics Development                                                      21
     GlaxoSmithKline plc                                                                                                        21
     AVI BioPharma Inc.                                                                                                         21
     Galapagos NV                                                                                                               22
     Summit Corporation plc                                                                                                     22
     Santhera Pharmaceuticals Holding AG                                                                                        23
     Amsterdam Molecular Therapeutics BV                                                                                        23
     The Avicena Group, Inc.                                                                                                    24
     Acceleron Pharma, Inc.                                                                                                     24
     PTC Therapeutics, Inc.                                                                                                     25
     UMN Pharma Inc.                                                                                                            25
     Genethon                                                                                                                   26
     Prosensa Therapeutics B.V.                                                                                                 26
Duchenne Muscular Dystrophy - Therapeutics Assessment                                                                           27
     Assessment by Monotherapy Products                                                                                         27
     Assessment by Route of Administration                                                                                      28
     Assessment by Molecule Type                                                                                                30
Drug Profiles                                                                                                                   32
     Ataluren - Drug Profile                                                                                                    32
          Product Description                                                                                                   32
          Mechanism of Action                                                                                                   32
          R&D Progress                                                                                                          32
     SMT C1100 - Drug Profile                                                                                                   34
          Product Description                                                                                                   34
          Mechanism of Action                                                                                                   34
          R&D Progress                                                                                                          34
     ACE-031 - Drug Profile                                                                                                     35


Duchenne Muscular Dystrophy – Pipeline Review, H2 2011                                               GMDHC1347IDB / Pub September 2011
© Global Markets Direct. This report is a licensed product and is not to be photocopied                                         Page(2)
Duchenne Muscular Dystrophy – Pipeline Review, H2 2011


          Product Description                                                                                        35
          Mechanism of Action                                                                                        35
          R&D Progress                                                                                               35
     AVI-4658 - Drug Profile                                                                                         37
          Product Description                                                                                        37
          Mechanism of Action                                                                                        37
          R&D Progress                                                                                               37
     AVI-5038 - Drug Profile                                                                                         39
          Product Description                                                                                        39
          Mechanism of Action                                                                                        39
          R&D Progress                                                                                               39
     GSK2402968 - Drug Profile                                                                                       40
          Product Description                                                                                        40
          Mechanism of Action                                                                                        40
          R&D Progress                                                                                               40
     DMD-02 - Drug Profile                                                                                           42
          Product Description                                                                                        42
          Mechanism of Action                                                                                        42
          R&D Progress                                                                                               42
     PRO044 - Drug Profile                                                                                           43
          Product Description                                                                                        43
          Mechanism of Action                                                                                        43
          R&D Progress                                                                                               43
     G100192 - Drug Profile                                                                                          44
          Product Description                                                                                        44
          Mechanism of Action                                                                                        44
          R&D Progress                                                                                               44
     AMT-080 - Drug Profile                                                                                          45
          Product Description                                                                                        45
          Mechanism of Action                                                                                        45
          R&D Progress                                                                                               45
     UMN-03 - Drug Profile                                                                                           46
          Product Description                                                                                        46
          Mechanism of Action                                                                                        46
          R&D Progress                                                                                               46
     GLPG0492 - Drug Profile                                                                                         47
          Product Description                                                                                        47
          Mechanism of Action                                                                                        47
          R&D Progress                                                                                               47
     Gentamicin - Drug Profile                                                                                       48
          Product Description                                                                                        48
          Mechanism of Action                                                                                        48
          R&D Progress                                                                                               48
     Catena - Drug Profile                                                                                           49
          Product Description                                                                                        49
          Mechanism of Action                                                                                        49
          R&D Progress                                                                                               49
     Sunphenon EGCg - Drug Profile                                                                                   51




Duchenne Muscular Dystrophy – Pipeline Review, H2 2011                                    GMDHC1347IDB / Pub September 2011
© Global Markets Direct. This report is a licensed product and is not to be photocopied                              Page(3)
Duchenne Muscular Dystrophy – Pipeline Review, H2 2011


          Product Description                                                                                                                  51
          Mechanism of Action                                                                                                                  51
          R&D Progress                                                                                                                         51
     Increlex - Drug Profile                                                                                                                   53
          Product Description                                                                                                                  53
          Mechanism of Action                                                                                                                  53
          R&D Progress                                                                                                                         53
     PRO045 - Drug Profile                                                                                                                     54
          Product Description                                                                                                                  54
          Mechanism of Action                                                                                                                  54
          R&D Progress                                                                                                                         54
     PRO053 - Drug Profile                                                                                                                     55
          Product Description                                                                                                                  55
          Mechanism of Action                                                                                                                  55
          R&D Progress                                                                                                                         55
     PRO052 - Drug Profile                                                                                                                     56
          Product Description                                                                                                                  56
          Mechanism of Action                                                                                                                  56
          R&D Progress                                                                                                                         56
     PRO055 - Drug Profile                                                                                                                     57
          Product Description                                                                                                                  57
          Mechanism of Action                                                                                                                  57
          R&D Progress                                                                                                                         57
     Flavocoxid - Drug Profile                                                                                                                 58
          Product Description                                                                                                                  58
          Mechanism of Action                                                                                                                  58
          R&D Progress                                                                                                                         58
     Duchenne Muscular Dystrophy Program - Drug Profile                                                                                        59
          Product Description                                                                                                                  59
          Mechanism of Action                                                                                                                  59
          R&D Progress                                                                                                                         59
Duchenne Muscular Dystrophy Therapeutics – Drug Profile Updates                                                                                60
Duchenne Muscular Dystrophy Therapeutics - Discontinued Products                                                                               65
     Duchenne Muscular Dystrophy - Featured News                                                                                               66
          Aug 24, 2011: Santhera Obtains US Patent For Use Of Catena In Treatment Of Muscular Dystrophies                                      66
          Aug 15, 2011: AVI BioPharma Initiates Dosing In Phase II Study Of Eteplirsen In Duchenne Muscular Dystrophy Patients                 66
          Jul 25, 2011: The Lancet Publishes Clinical Trial Data That Demonstrate Statistically Significant And Dose-Dependent Expression Of
          Dystrophin In Duchenne Muscular Dystrophy Patients Treated With AVI BioPharma's Eteplirsen                                           67
          Jun 09, 2011: AVI BioPharma Provides Update On Initiation Of Eteplirsen Phase II Clinical Trial                             
								
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