Amyotrophic Lateral Sclerosis – Pipeline Review, H1 2012

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					   Amyotrophic Lateral Sclerosis – Pipeline Review, H1
                          2012


                                                                                          Reference Code: GMDHC1751IDB
                                                                                           Publication Date: February 2012




Amyotrophic Lateral Sclerosis – Pipeline Review, H1 2012                                   GMDHC1751IDB / Pub February 2012
© Global Markets Direct. This report is a licensed product and is not to be photocopied                             Page(1)
Amyotrophic Lateral Sclerosis – Pipeline Review, H1 2012




Table of Contents
Table of Contents ............................................................................................................................................................................... 2
    List of Tables ............................................................................................................................................................................... 12
    List of Figures .............................................................................................................................................................................. 13
Introduction....................................................................................................................................................................................... 14
    Global Markets Direct Report Coverage ...................................................................................................................................... 14
Amyotrophic Lateral Sclerosis Overview .......................................................................................................................................... 15
Therapeutics Development............................................................................................................................................................... 16
    An Overview of Pipeline Products for Amyotrophic Lateral Sclerosis .......................................................................................... 16
Amyotrophic Lateral Sclerosis Therapeutics under Development by Companies ............................................................................ 18
Amyotrophic Lateral Sclerosis Therapeutics under Investigation by Universities/Institutes .............................................................. 23
Late Stage Products ......................................................................................................................................................................... 26
    Comparative Analysis .................................................................................................................................................................. 26
Mid Clinical Stage Products.............................................................................................................................................................. 27
    Comparative Analysis .................................................................................................................................................................. 27
Early Clinical Stage Products ........................................................................................................................................................... 28
    Comparative Analysis .................................................................................................................................................................. 28
Discovery and Pre-Clinical Stage Products ...................................................................................................................................... 29
    Comparative Analysis .................................................................................................................................................................. 29
Amyotrophic Lateral Sclerosis Therapeutics – Products under Development by Companies .......................................................... 30
Amyotrophic Lateral Sclerosis Therapeutics – Products under Investigation by Universities/Institutes............................................ 34
Companies Involved in Amyotrophic Lateral Sclerosis Therapeutics Development ......................................................................... 37
    Genzyme Corporation ................................................................................................................................................................. 37
    Biogen Idec Inc. ........................................................................................................................................................................... 38
    GlaxoSmithKline plc .................................................................................................................................................................... 39
    Isis Pharmaceuticals, Inc. ............................................................................................................................................................ 40
    Eisai Co., Ltd. .............................................................................................................................................................................. 41
    Mitsubishi Tanabe Pharma Corporation ...................................................................................................................................... 42
    Hadasit Medical Research Services & Development Ltd............................................................................................................. 43
    TCA Cellular Therapy .................................................................................................................................................................. 44
    BrainStorm Cell Therapeutics Inc. ............................................................................................................................................... 45
    Aeolus Pharmaceuticals, Inc. ...................................................................................................................................................... 46
    Neuralstem, Inc. .......................................................................................................................................................................... 47
    ReGenX Biosciences, LLC .......................................................................................................................................................... 48
    Nutra Pharma Corporation .......................................................................................................................................................... 49
    Cytokinetics, Inc .......................................................................................................................................................................... 50
    Phytopharm Plc ........................................................................................................................................................................... 51
    SYGNIS Pharma AG ................................................................................................................................................................... 52
    Allon Therapeutics Inc. ................................................................................................................................................................ 53
    The Avicena Group, Inc. .............................................................................................................................................................. 54
    Ceregene, Inc. ............................................................................................................................................................................. 55
    Snowdon Inc. ............................................................................................................................................................................... 56
    BioMAS Ltd.................................................................................................................................................................................. 57



Amyotrophic Lateral Sclerosis – Pipeline Review, H1 2012                                                                                   GMDHC1751IDB / Pub February 2012
© Global Markets Direct. This report is a licensed product and is not to be photocopied                                                                                                  Page(2)
Amyotrophic Lateral Sclerosis – Pipeline Review, H1 2012




    Trophos SA.................................................................................................................................................................................. 58
    NeuroNova AB............................................................................................................................................................................. 59
    Q Therapeutics, Inc. .................................................................................................................................................................... 60
    GenKyoTex S.A. .......................................................................................................................................................................... 61
    KineMed, Inc................................................................................................................................................................................ 62
    Adeona Pharmaceuticals, Inc. ..................................................................................................................................................... 63
    Maas Biolab ................................................................................................................................................................................. 64
    Nexgenix Pharmaceuticals, LLC.................................................................................................................................................. 65
    miRagen Therapeutics, Inc. ......................................................................................................................................................... 66
    Pharnext SAS .............................................................................................................................................................................. 67
    Cellceutix Pharmaceuticals, Inc. .................................................................................................................................................. 68
    Neuraltus Pharmaceuticals, Inc. .................................................................................................................................................. 69
    Varinel, Inc................................................................................................................................................................................... 70
    SK Bio-Pharmaceuticals .............................................................................................................................................................. 71
Amyotrophic Lateral Sclerosis – Therapeutics Assessment ............................................................................................................. 72
    Assessment by Monotherapy Products ....................................................................................................................................... 72
    Assessment by Combination Products ........................................................................................................................................ 73
    Assessment by Route of Administration ...................................................................................................................................... 74
    Assessment by Molecule Type .................................................................................................................................................... 76
Drug Profiles..................................................................................................................................................................................... 79
    Radicut - Drug Profile .................................................................................................................................................................. 79
        Product Description................................................................................................................................................................. 79
        Mechanism of Action ............................................................................................................................................................... 79
        R&D Progress ......................................................................................................................................................................... 79
    ISIS-SOD1Rx - Drug Profile ........................................................................................................................................................ 80
        Product Description................................................................................................................................................................. 80
        Mechanism of Action ............................................................................................................................................................... 80
        R&D Progress ......................................................................................................................................................................... 80
    GKT-03 - Drug Profile .................................................................................................................................................................. 81
        Product Description................................................................................................................................................................. 81
        Mechanism of Action ............................................................................................................................................................... 81
        R&D Progress ......................................................................................................................................................................... 81
    E0302 - Drug Profile .................................................................................................................................................................... 82
        Product Description................................................................................................................................................................. 82
        Mechanism of Action ............................................................................................................................................................... 82
        R&D Progress ......................................................................................................................................................................... 82
    RPI-MN - Drug Profile .................................................................................................................................................................. 83
        Product Description................................................................................................................................................................. 83
        Mechanism of Action ............................................................................................................................................................... 83
        R&D Progress ......................................................................................................................................................................... 83
    AEOL 10150 - Drug Profile .......................................................................................................................................................... 85
        Product Description................................................................................................................................................................. 85
        Mechanism of Action ............................................................................................................................................................... 85
        R&D Progress ......................................................................................................................................................................... 85


Amyotrophic Lateral Sclerosis – Pipeline Review, H1 2012                                                                                   GMDHC1751IDB / Pub February 2012
© Global Markets Direct. This report is a licensed product and is not to be photocopied                                                                                                  Page(3)
Amyotrophic Lateral Sclerosis – Pipeline Review, H1 2012




    AX200 - Drug Profile.................................................................................................................................................................... 88
        Product Description................................................................................................................................................................. 88
        Mechanism of Action ............................................................................................................................................................... 88
        R&D Progress ......................................................................................................................................................................... 88
    CK-2017357 - Drug Profile .......................................................................................................................................................... 89
        Product Description................................................................................................................................................................. 89
        Mechanism of Action ............................................................................................................................................................... 89
        R&D Progress ......................................................................................................................................................................... 89
    ALS-02 - Drug Profile .................................................................................................................................................................. 92
        Product Description................................................................................................................................................................. 92
        Mechanism of Action ............................................................................................................................................................... 92
        R&D Progress ......................................................................................................................................................................... 92
    ALS-05 - Drug Profile .................................................................................................................................................................. 93
        Product Description................................................................................................................................................................. 93
        Mechanism of Action ............................................................................................................................................................... 93
        R&D Progress ......................................................................................................................................................................... 93
    Dexpramipexole - Drug Profile..................................................................................................................................................... 94
        Product Description................................................................................................................................................................. 94
        Mechanism of Action ............................................................................................................................................................... 94
        R&D Progress ......................................................................................................................................................................... 94
    CERE-135 - Drug Profile ............................................................................................................................................................. 96
        Product Description................................................................................................................................................................. 96
        Mechanism of Action ............................................................................................................................................................... 96
        R&D Progress ......................................................................................................................................................................... 96
    AAD2004 - Drug Profile ............................................................................................................................................................... 97
        Product Description................................................................................................................................................................. 97
        Mechanism of Action ............................................................................................................................................................... 97
        R&D Progress ......................................................................................................................................................................... 97
    Myogane - Drug Profile ................................................................................................................................................................ 98
        Product Description................................................................................................................................................................. 98
        Mechanism of Action ..............................
				
DOCUMENT INFO
Description: Amyotrophic Lateral Sclerosis - Pipeline Review, H1 2012', provides an overview of the Amyotrophic Lateral Sclerosis therapeutic pipeline. This report provides information on the therapeutic development for Amyotrophic Lateral Sclerosis, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Amyotrophic Lateral Sclerosis. 'Amyotrophic Lateral Sclerosis - Pipeline Review, H1 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.
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