Inclusion Body Myositis (IBM) – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Inclusion Body Myositis (IBM) – Pipeline Review, H2 2016’, provides an overview of the Inclusion Body Myositis (IBM) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Inclusion Body Myositis (IBM), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Inclusion Body Myositis (IBM) and features dormant and discontinued projects.

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage.

Note: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

The report provides a snapshot of the global therapeutic landscape of Inclusion Body Myositis (IBM)

The report reviews pipeline therapeutics for Inclusion Body Myositis (IBM) by companies and universities/research institutes based on information derived from company and industry-specific sources

The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages

The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities

The report reviews key players involved Inclusion Body Myositis (IBM) therapeutics and enlists all their major and minor projects

The report assesses Inclusion Body Myositis (IBM) therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type

The report summarizes all the dormant and discontinued pipeline projects

The report reviews latest news related to pipeline therapeutics for Inclusion Body Myositis (IBM)

Reasons to buy

Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies

Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage

Identify and understand important and diverse types of therapeutics under development for Inclusion Body Myositis (IBM)

Identify potential new clients or partners in the target demographic

Develop strategic initiatives by understanding the focus areas of leading companies

Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics

Devise corrective measures for pipeline projects by understanding Inclusion Body Myositis (IBM) pipeline depth and focus of Indication therapeutics

Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope

Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Companies mentioned

Acceleron Pharma, Inc.

Escala Therapeutics, Inc.

Milo Biotechnology LLC

Nobelpharma Co., Ltd.

Novartis AG

Orphazyme ApS

Ultragenyx Pharmaceutical Inc.

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 4

List of Figures 5

Introduction 6

Global Markets Direct Report Coverage 6

Inclusion Body Myositis (IBM) Overview 7

Therapeutics Development 8

Pipeline Products for Inclusion Body Myositis (IBM) - Overview 8

Pipeline Products for Inclusion Body Myositis (IBM) - Comparative Analysis 9

Inclusion Body Myositis (IBM) - Therapeutics under Development by Companies 10

Inclusion Body Myositis (IBM) - Therapeutics under Investigation by Universities/Institutes 11

Inclusion Body Myositis (IBM) - Pipeline Products Glance 12

Late Stage Products 12

Clinical Stage Products 13

Early Stage Products 14

Inclusion Body Myositis (IBM) - Products under Development by Companies 15

Inclusion Body Myositis (IBM) - Products under Investigation by Universities/Institutes 16

Inclusion Body Myositis (IBM) - Companies Involved in Therapeutics Development 17

Acceleron Pharma, Inc. 17

Escala Therapeutics, Inc. 18

Milo Biotechnology LLC 19

Nobelpharma Co., Ltd. 20

Novartis AG 21

Orphazyme ApS 22

Ultragenyx Pharmaceutical Inc. 23

Inclusion Body Myositis (IBM) - Therapeutics Assessment 24

Assessment by Monotherapy Products 24

Assessment by Target 25

Assessment by Mechanism of Action 27

Assessment by Route of Administration 29

Assessment by Molecule Type 31

Drug Profiles 33

AAV1-Follistatin - Drug Profile 33

Product Description 33

Mechanism Of Action 33

R&D Progress 33

ACE-083 - Drug Profile 35

Product Description 35

Mechanism Of Action 35

R&D Progress 35

aceneuramic acid - Drug Profile 37

Product Description 37

Mechanism Of Action 37

R&D Progress 37

ALZ-801 - Drug Profile 42

Product Description 42

Mechanism Of Action 42

R&D Progress 42

arimoclomol - Drug Profile 45

Product Description 45

Mechanism Of Action 45

R&D Progress 45

bimagrumab - Drug Profile 47

Product Description 47

Mechanism Of Action 47

R&D Progress 47

DEXM-74 - Drug Profile 49

Product Description 49

Mechanism Of Action 49

R&D Progress 49

Gene Therapy for Hereditary Inclusion Body Myopathy - Drug Profile 51

Product Description 51

Mechanism Of Action 51

R&D Progress 51

UX-001P - Drug Profile 52

Product Description 52

Mechanism Of Action 52

R&D Progress 52

UX-007 - Drug Profile 53

Product Description 53

Mechanism Of Action 53

R&D Progress 53

Inclusion Body Myositis (IBM) - Dormant Projects 58

Inclusion Body Myositis (IBM) - Product Development Milestones 59

Featured News & Press Releases 59

Jul 27, 2016: Ultragenyx Announces Completion of Enrollment in Phase 3 Study of Aceneuramic Acid Extended Release (Ace-ER) in GNE Myopathy 59

Apr 21, 2016: MorphoSys : MorphoSys Provides Update on Results From Partner Phase 2b/3 RESILIENT Study of Bimagrumab 59

Oct 02, 2015: Ultragenyx Announces Aceneuramic Acid Prolonged Release Marketing Authorization Application Filed and Accepted for Review by European Medicines Agency 60

May 28, 2015: Ultragenyx Announces First Patient Enrolled in Global Phase 3 Study of Aceneuramic Acid (Sialic Acid) Extended Release in GNE Myopathy 60

Jan 12, 2015: Ultragenyx Announces Intent to File for Conditional Approval in Europe for Sialic Acid Extended-Release Tablets in Hereditary Inclusion Body Myopathy 61

Oct 13, 2014: Ultragenyx Announces Interim Data From Phase 2 Extension Study of Sialic Acid Extended-Release at International Congress of the World Muscle Society 61

Apr 30, 2014: Ultragenyx Announces Positive Data From Phase 2 Study of Sialic Acid Extended-Release at Emerging Sciences Session of American Academy of Neurology Annual Meeting 62

Dec 20, 2013: Ultragenyx Announces Results from Phase 2 Study of Sialic Acid Extended-Release Treatment in Hereditary Inclusion Body Myopathy 63

Oct 02, 2013: MorphoSys Collaborator to Start Pivotal Study with Bimagrumab 64

Sep 26, 2013: Ultragenyx Announces Three Abstracts Accepted for Poster Presentation at 18th Annual World Muscle Society Congress 65

Aug 20, 2013: Novartis receives FDA breakthrough therapy designation for BYM338 (bimagrumab) for sporadic inclusion body myositis (sIBM) 65

Jul 03, 2013: Ultragenyx Announces a Positive Signal in Interim Data from Phase 2 Study of UX001 in Hereditary Inclusion Body Myopathy 66

Dec 15, 2011: New Zealand Pharma Obtains FDA Orphan Drug Designation For DEX-M74 To Treat Hereditary Inclusion Body Myopathy 66

Appendix 68

Methodology 68

Coverage 68

Secondary Research 68

Primary Research 68

Expert Panel Validation 68

Contact Us 68

Disclaimer 69

List of Tables

List of Tables

Number of Products under Development for Inclusion Body Myositis (IBM), H2 2016 8

Number of Products under Development for Inclusion Body Myositis (IBM) – Comparative Analysis, H2 2016 9

Number of Products under Development by Companies, H2 2016 10

Number of Products under Investigation by Universities/Institutes, H2 2016 11

Comparative Analysis by Late Stage Development, H2 2016 12

Comparative Analysis by Clinical Stage Development, H2 2016 13

Comparative Analysis by Early Stage Development, H2 2016 14

Products under Development by Companies, H2 2016 15

Products under Investigation by Universities/Institutes, H2 2016 16

Inclusion Body Myositis (IBM) – Pipeline by Acceleron Pharma, Inc., H2 2016 17

Inclusion Body Myositis (IBM) – Pipeline by Escala Therapeutics, Inc. , H2 2016 18

Inclusion Body Myositis (IBM) – Pipeline by Milo Biotechnology LLC, H2 2016 19

Inclusion Body Myositis (IBM) – Pipeline by Nobelpharma Co., Ltd., H2 2016 20

Inclusion Body Myositis (IBM) – Pipeline by Novartis AG, H2 2016 21

Inclusion Body Myositis (IBM) – Pipeline by Orphazyme ApS, H2 2016 22

Inclusion Body Myositis (IBM) – Pipeline by Ultragenyx Pharmaceutical Inc., H2 2016 23

Assessment by Monotherapy Products, H2 2016 24

Number of Products by Stage and Target, H2 2016 26

Number of Products by Stage and Mechanism of Action, H2 2016 28

Number of Products by Stage and Route of Administration, H2 2016 30

Number of Products by Stage and Molecule Type, H2 2016 32

Inclusion Body Myositis (IBM) – Dormant Projects, H2 2016 58

List of Figures

List of Figures

Number of Products under Development for Inclusion Body Myositis (IBM), H2 2016 8

Number of Products under Development for Inclusion Body Myositis (IBM) – Comparative Analysis, H2 2016 9

Number of Products under Development by Companies, H2 2016 10

Comparative Analysis by Late Stage Development, H2 2016 12

Comparative Analysis by Clinical Stage Development, H2 2016 13

Comparative Analysis by Early Stage Products, H2 2016 14

Assessment by Monotherapy Products, H2 2016 24

Number of Products by Targets, H2 2016 25

Number of Products by Stage and Targets, H2 2016 25

Number of Products by Mechanism of Actions, H2 2016 27

Number of Products by Stage and Mechanism of Actions, H2 2016 27

Number of Products by Routes of Administration, H2 2016 29

Number of Products by Stage and Routes of Administration, H2 2016 29

Number of Products by Molecule Types, H2 2016 31

Number of Products by Stage and Molecule Types, H2 2016 31

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