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

Published: Oct, 2016 | Pages: 69 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

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

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Inclusion Body Myositis (IBM) - Pipeline Review, H2 2016, provides an overview of the Inclusion Body Myositis (IBM) (Musculoskeletal Disorders) pipeline landscape.

Inclusion Body Myositis (IBM) is one of the inflammatory myopathies that involve inflammation of the muscles or associated tissues, such as the blood vessels that supply the muscles. IBM symptoms include progressive weakness of the muscles of the wrists and fingers, the muscles of the front of the thigh, and the muscles that lift the front of the foot. 

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Inclusion Body Myositis (IBM) - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Inclusion Body Myositis (IBM) (Musculoskeletal Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Inclusion Body Myositis (IBM) (Musculoskeletal Disorders) pipeline guide also reviews of key players involved in therapeutic development for Inclusion Body Myositis (IBM) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I and Preclinical stages are 1, 2, 2, 2 and 2 respectively for Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively for Inclusion Body Myositis (IBM).

Inclusion Body Myositis (IBM) (Musculoskeletal Disorders) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Inclusion Body Myositis (IBM) (Musculoskeletal Disorders).
- The pipeline guide reviews pipeline therapeutics for Inclusion Body Myositis (IBM) (Musculoskeletal Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources. 
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Inclusion Body Myositis (IBM) (Musculoskeletal Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Inclusion Body Myositis (IBM) (Musculoskeletal Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects. 
- The pipeline guide reviews latest news related to pipeline therapeutics for Inclusion Body Myositis (IBM) (Musculoskeletal Disorders)

Reasons To Buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Inclusion Body Myositis (IBM) (Musculoskeletal Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Inclusion Body Myositis (IBM) (Musculoskeletal Disorders) 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

 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

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