Spinal Muscular Atrophy (SMA) – Pipeline Review, H1 2012 by GlobalMarketsDirect

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									  Spinal Muscular Atrophy (SMA) – Pipeline Review, H1
                         2012


                                                                                          Reference Code: GMDHC1994IDB
                                                                                              Publication Date: April 2012




Spinal Muscular Atrophy (SMA) – Pipeline Review, H1 2012                                      GMDHC1994IDB / Pub April 2012
© Global Markets Direct. This report is a licensed product and is not to be photocopied                             Page(1)
Spinal Muscular Atrophy (SMA) – Pipeline Review, H1 2012




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 ........................................................... 11
Mid Clinical Stage Products.............................................................................................................................................................. 12
    Comparative Analysis .................................................................................................................................................................. 12
Early Clinical Stage Products ........................................................................................................................................................... 13
    Comparative Analysis .................................................................................................................................................................. 13
Discovery and Pre-Clinical Stage Products ...................................................................................................................................... 14
    Comparative Analysis .................................................................................................................................................................. 14
Spinal Muscular Atrophy (SMA) Therapeutics – Products under Development by Companies ........................................................ 15
Spinal Muscular Atrophy (SMA) Therapeutics – Products under Investigation by Universities/Institutes ......................................... 16
Companies Involved in Spinal Muscular Atrophy (SMA) Therapeutics Development....................................................................... 17
    F. Hoffmann-La Roche Ltd. ......................................................................................................................................................... 17
    Isis Pharmaceuticals, Inc. ............................................................................................................................................................ 18
    Oxford BioMedica plc .................................................................................................................................................................. 19
    Repligen Corporation................................................................................................................................................................... 20
    Trophos SA.................................................................................................................................................................................. 21
    Nexgenix Pharmaceuticals, LLC.................................................................................................................................................. 22
Spinal Muscular Atrophy (SMA) – Therapeutics Assessment .......................................................................................................... 23
    Assessment by Monotherapy Products ....................................................................................................................................... 23
    Assessment by Combination Products ........................................................................................................................................ 24
    Assessment by Route of Administration ...................................................................................................................................... 25
    Assessment by Molecule Type .................................................................................................................................................... 27
Drug Profiles..................................................................................................................................................................................... 29
    RG3039 - Drug Profile ................................................................................................................................................................. 29
         Product Description................................................................................................................................................................. 29
         Mechanism of Action ............................................................................................................................................................... 29
         R&D Progress ......................................................................................................................................................................... 29
    Olesoxime - Drug Profile ............................................................................................................................................................. 31
         Product Description................................................................................................................................................................. 31
         Mechanism of Action ............................................................................................................................................................... 31
         R&D Progress ......................................................................................................................................................................... 31
    SMN1-G - Drug Profile ................................................................................................................................................................ 33
         Product Description................................................................................................................................................................. 33
         Mechanism of Action ............................................................................................................................................................... 33



Spinal Muscular Atrophy (SMA) – Pipeline Review, H1 2012                                                                                           GMDHC1994IDB / Pub April 2012
© Global Markets Direct. This report is a licensed product and is not to be photocopied                                                                                                   Page(2)
Spinal Muscular Atrophy (SMA) – Pipeline Review, H1 2012




        R&D Progress ......................................................................................................................................................................... 33
    NXD30001 - Drug Profile ............................................................................................................................................................. 34
        Product Description................................................................................................................................................................. 34
        Mechanism of Action ............................................................................................................................................................... 34
        R&D Progress ......................................................................................................................................................................... 34
    Sodium Phenylbutyrate (NaPB) - Drug Profile ............................................................................................................................. 35
        Product Description................................................................................................................................................................. 35
        Mechanism of Action ............................................................................................................................................................... 35
        R&D Progress ......................................................................................................................................................................... 35
    Valproic Acid - Drug Profile.......................................................................................................................................................... 36
        Product Description................................................................................................................................................................. 36
        Mechanism of Action ............................................................................................................................................................... 36
        R&D Progress ......................................................................................................................................................................... 36
    Valproic acid + Levocarnitine - Drug Profile................................................................................................................................. 37
        Product Description................................................................................................................................................................. 37
        Mechanism of Action ............................................................................................................................................................... 37
        R&D Progress ......................................................................................................................................................................... 37
    Somatotropin - Drug Profile ......................................................................................................................................................... 39
        Product Description................................................................................................................................................................. 39
        Mechanism of Action ............................................................................................................................................................... 39
        R&D Progress ......................................................................................................................................................................... 39
    ISIS-SMNRx - Drug Profile .......................................................................................................................................................... 40
        Product Description................................................................................................................................................................. 40
        Mechanism of Action ............................................................................................................................................................... 40
        R&D Progress ......................................................................................................................................................................... 40
    Depakote + Carnitor - Drug Profile .............................................................................................................................................. 41
        Product Description................................................................................................................................................................. 41
        Mechanism of Action ............................................................................................................................................................... 41
        R&D Progress ......................................................................................................................................................................... 41
    Drug For Spinal Muscular Atrophy - Drug Profile.....................................................................................................................
								
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