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Amyloidosis - Pipeline Review, H2 2011

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Amyloidosis - Pipeline Review, H2 2011 Summary Global Markets Direct’s, 'Amyloidosis - Pipeline Review, H2 2011', provides an overview of the Amyloidosis therapeutic pipeline. This report provides information on the therapeutic development for Amyloidosis, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Amyloidosis. 'Amyloidosis - Pipeline Review, H2 2011' 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. Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease. Scope - A snapshot of the global therapeutic scenario for Amyloidosis. - A review of the Amyloidosis products under development by companies and universities/research institutes based on information derived from company and industry-specific sources. - Coverage of products based on various stages of development ranging from discovery till registration stages. - A feature on pipeline projects on the basis of monotherapy and combined therapeutics. - Coverage of the Amyloidosis pipeline on the basis of route of administration and molecule type. - Profiles of late-stage pipeline products featuring sections on product description, mechanism of action and research & development progress. - Key discontinued pipeline projects. - Latest news and deals relating to the products. Reasons to buy - Identify and understand important and diverse types of therapeutics under development for Amyloidosis. - Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage. - Plan

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									Amyloidosis – Pipeline Review, H2 2011




                         Amyloidosis - Pipeline Review, H2 2011
                                                                                          Reference Code: GMDHC1482IDB

                                                                                          Publication Date: October 2011




Amyloidosis – Pipeline Review, H2 2011                                                                           GMDHC1482IDB / Pub October 2011
© Global Markets Direct. This report is a licensed product and is not to be photocopied                                                  Page(1)
Amyloidosis – Pipeline Review, H2 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
Amyloidosis Overview .............................................................................................................................................................................................10
Therapeutics Development ......................................................................................................................................................................................11
      An Overview of Pipeline Products for Amyloidosis.............................................................................................................................................11
Amyloidosis Therapeutics under Development by Companies .................................................................................................................................13
Amyloidosis 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
Amyloidosis Therapeutics - Products under Development by Companies ................................................................................................................22
Amyloidosis Therapeutics - Products under Investigation by Universities/Institutes .................................................................................................23
Companies Involved in Amyloidosis Therapeutics Development..............................................................................................................................26
      GlaxoSmithKline plc ..........................................................................................................................................................................................26
      Takeda Pharmaceutical Company Limited ........................................................................................................................................................26
      Millennium Pharmaceuticals, Inc. ......................................................................................................................................................................27
      Alnylam Pharmaceuticals, Inc ...........................................................................................................................................................................27
      ProteoTech, Inc. ................................................................................................................................................................................................28
      BIOALVO S.A. ..................................................................................................................................................................................................28
      Immune System Therapeutics Ltd. ....................................................................................................................................................................29
      Celtic Therapeutics Holdings L.P.......................................................................................................................................................................29
Amyloidosis - Therapeutics Assessment..................................................................................................................................................................30
      Assessment by Monotherapy Products .............................................................................................................................................................30
      Assessment by Combination Products ..............................................................................................................................................................31
      Assessment by Route of Administration ............................................................................................................................................................32
      Assessment by Molecule Type ..........................................................................................................................................................................34
Drug Profiles ...........................................................................................................................................................................................................37
      MLN9708 - Drug Profile.....................................................................................................................................................................................37
            Product Description ....................................................................................................................................................................................37
            Mechanism of Action...................................................................................................................................................................................37
            R&D Progress .............................................................................................................................................................................................37
      SDGI-TT200801 - Drug Profile ..........................................................................................................................................................................39
            Product Description ....................................................................................................................................................................................39
            Mechanism of Action...................................................................................................................................................................................39
            R&D Progress .............................................................................................................................................................................................39
      Velcade - Drug Profile .......................................................................................................................................................................................40
            Product Description ....................................................................................................................................................................................40
            Mechanism of Action...................................................................................................................................................................................40
            R&D Progress .............................................................................................................................................................................................40


Amyloidosis – Pipeline Review, H2 2011                                                                                                                        GMDHC1482IDB / Pub October 2011
© Global Markets Direct. This report is a licensed product and is not to be photocopied                                                                                                                                Page(2)
Amyloidosis – Pipeline Review, H2 2011


     MDX-1097 - Drug Profile ...................................................................................................................................................................................41
          Product Description ....................................................................................................................................................................................41
          Mechanism of Action...................................................................................................................................................................................41
          R&D Progress .............................................................................................................................................................................................41
     Kiacta - Drug Profile ..........................................................................................................................................................................................42
          Product Description ....................................................................................................................................................................................42
          Mechanism of Action...................................................................................................................................................................................42
          R&D Progress .............................................................................................................................................................................................42
     Systebryl - Drug Profile .....................................................................................................................................................................................44
          Product Description ....................................................................................................................................................................................44
          Mechanism of Action...................................................................................................................................................................................44
          R&D Progress .............................................................................................................................................................................................44
     Revlimid + Dexamethasone + Endoxan - Drug Profile .......................................................................................................................................45
          Product Description ....................................................................................................................................................................................45
          Mechanism of Action...................................................................................................................................................................................45
          R&D Progress .............................................................................................................................................................................................45
     ALN-TTR01 - Drug Profile .................................................................................................................................................................................46
          Product Description ....................................................................................................................................................................................46
          Mechanism of Action...................................................................................................................................................................................46
          R&D Progress .............................................................................................................................................................................................46
     ALN-TTR02 - Drug Profile .................................................................................................................................................................................47
          Product Description ....................................................................................................................................................................................47
          Mechanism of Action...................................................................................................................................................................................47
          R&D Progress .............................................................................................................................................................................................47
     Iododoxorubicin - Drug Profile ...........................................................................................................................................................................48
          Product Description ....................................................................................................................................................................................48
          Mechanism of Action...................................................................................................................................................................................48
          R&D Progress .............................................................................................................................................................................................48
     Velcade + Dexamethasone + Melphalan - Drug Profile .....................................................................................................................................49
          Product Description ....................................................................................................................................................................................49
          Mechanism of Action...................................................................................................................................................................................49
          R&D Progress .............................................................................................................................................................................................49
     Velcade + Dexamethasone + Melphalan + Stem Cell Transplantation - Drug Profile .........................................................................................50
          Product Description ....................................................................................................................................................................................50
          Mechanism of Action...................................................................................................................................................................................50
          R&D Progress .............................................................................................................................................................................................50
     Revlimid + Dexamethasone + Cyclophosphamide - Drug Profile .......................................................................................................................52
          Product Description ....................................................................................................................................................................................52
          Mechanism of Action...................................................................................................................................................................................52
          R&D Progress .............................................................................................................................................................................................52
     Revlimid + Cyclophosphamide + Dexamethasone - Drug Profile .......................................................................................................................53
          Product Description ....................................................................................................................................................................................53
          Mechanism of Action...................................................................................................................................................................................53
          R&D Progress .............................................................................................................................................................................................53
     Dexamethasone + Pomalidomide - Drug Profile ................................................................................................................................................55
          Product Description ....................................................................................................................................................................................55
          Mechanism of Action...................................................................................................................................................................................55
          R&D Progress .............................................................................................................................................................................................55




Amyloidosis – Pipeline Review, H2 2011                                                                                                                    GMDHC1482IDB / Pub October 2011
© Global Markets Direct. This report is a licensed product and is not to be photocopied                                                                                                                          Page(3)
Amyloidosis – Pipeline Review, H2 2011


     Melphalan + Lenalidomide + Dexamethasone - Drug Profile .............................................................................................................................56
          Product Description ....................................................................................................................................................................................56
          Mechanism of Action...................................................................................................................................................................................56
          R&D Progress .............................................................................................................................................................................................56
     Bortezomib + Dexamethasone + Melphalan - Drug Profile ................................................................................................................................57
          Product Description ....................................................................................................................................................................................57
          Mechanism of Action...................................................................................................................................................................................57
          R&D Progress .............................................................................................................................................................................................57
     Bortezomib + Cyclophosphamide + Dexamethasone - Drug Profile...................................................................................................................58
          Product Description ....................................................................................................................................................................................58
          Mechanism of Action...................................................................................................................................................................................58
          R&D Progress .............................................................................................................................................................................................58
     Filgrastim + Dexamethasone + Melphalan + Autologous Hematopoietic Stem Cell Transplantation - Drug Profile.............................................60
          Product Description ....................................................................................................................................................................................60
          Mechanism of Action...................................................................................................................................................................................60
          R&D Progress .............................................................................................................................................................................................60
     Dexamethasone + Melphalan + Thalidomide + Filgrastim - Drug Profile ............................................................................................................62
          Product Description ....................................................................................................................................................................................62
          Mechanism of Action...................................................................................................................................................................................62
          R&D Progress .............................................................................................................................................................................................62
     Revlimid - Drug Profile ......................................................................................................................................................................................63
          Product Description ....................................................................................................................................................................................63
          Mechanism of Action...................................................................................................................................................................................63
          R&D Progress .............................................................................................................................................................................................63
     Melphalan + Autologous Stem Cell Transplantation + Bortezomib + Dexamethasone - Drug Profile .................................................................64
          Product Description ....................................................................................................................................................................................64
          Mechanism of Action...................................................................................................................................................................................64
          R&D Progress .............................................................................................................................................................................................64
     Filgrastim + Melphalan + Cyclophosphamide + Dexamethasone + Thalidomide + Peripheral Blood Stem Cell Transplantation - Drug Profile...66
          Product Description ....................................................................................................................................................................................66
          Mechanism of Action...................................................................................................................................................................................66
          R&D Progress ....................................
								
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