Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) - Pipeline Review, H2 2017

Published: Sep, 2017 | Pages: 66 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) - Pipeline Review, H2 2017

Summary

Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) - Myostatin also known as growth differentiation factor 8 or GDF-8 is a myokine a protein produced and released by myocytes. This protein is part of the transforming growth factor beta (TGF?) superfamily. Myostatin is found almost in muscles used for movement, where it is active both before and after birth. This protein normally restrains muscle growth, ensuring that muscles do not grow too large. 

Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) pipeline Target constitutes close to 13 molecules. Out of which approximately 13 molecules are developed by Companies. The molecules developed by companies in Phase III, Phase II, Phase I and Preclinical stages are 1, 4, 1 and 7 respectively. Report covers products from therapy areas Musculoskeletal Disorders, Genetic Disorders, Central Nervous System, Gastrointestinal, Hematological Disorders, Metabolic Disorders and Oncology which include indications Duchenne Muscular Dystrophy, Amyotrophic Lateral Sclerosis, Muscular Dystrophy, Sarcopenia, Anemia, Becker Muscular Dystrophy, Charcot-Marie-Tooth Disease Type I, Crohn's Disease (Regional Enteritis), Fibrodysplasia Ossificans Progressiva (Myositis Ossificans Progressiva), Fibrosis, Inclusion Body Myositis (IBM), Insulin Resistance, Muscle Atrophy, Myelodysplastic Syndrome, Myelofibrosis, Sickle Cell Disease, Spinal Muscular Atrophy (SMA) and Thalassemia. 

The latest report GrowthDifferentiation Factor 8 - Pipeline Review, H2 2017, outlays comprehensive information on the Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. It also reviews key players involved in Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) targeted therapeutics development with respective active and dormant or discontinued projects.

The report is built using data and information sourced from 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.

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

Scope

- The report provides a snapshot of the global therapeutic landscape for Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN)
- The report reviews Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) targeted therapeutics under development 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 in Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) targeted therapeutics and enlists all their major and minor projects 
- The report assesses Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) targeted therapeutics based on 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 and deals related to Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) targeted therapeutics

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 the targeted therapy areas and indications for Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN)
- Identify the use of drugs for target identification and drug repurposing
- 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 Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) development landscape 
- 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

 Table of Contents

Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) - Overview 7
Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) - Therapeutics Development 8
Products under Development by Stage of Development 8
Products under Development by Therapy Area 9
Products under Development by Indication 10
Products under Development by Companies 11
Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) - Therapeutics Assessment 14
Assessment by Mechanism of Action 14
Assessment by Route of Administration 15
Assessment by Molecule Type 17
Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) - Companies Involved in Therapeutics Development 19
Acceleron Pharma Inc 19
Amgen Inc 19
Bioleaders Corp 20
F. Hoffmann-La Roche Ltd 20
Pfizer Inc 21
Regeneron Pharmaceuticals Inc 21
Sarepta Therapeutics Inc 22
Scholar Rock Inc 22
Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) - Drug Profiles 23
ACE-083 - Drug Profile 23
Product Description 23
Mechanism Of Action 23
R&D Progress 23
ACE-2494 - Drug Profile 26
Product Description 26
Mechanism Of Action 26
R&D Progress 26
ACE-2798 - Drug Profile 27
Product Description 27
Mechanism Of Action 27
R&D Progress 27
Antisense Oligonucleotide to Inhibit Myostatin for DMD and Becker MD - Drug Profile 28
Product Description 28
Mechanism Of Action 28
R&D Progress 28
ATA-842 - Drug Profile 29
Product Description 29
Mechanism Of Action 29
R&D Progress 29
BLS-ILB-MD1 - Drug Profile 30
Product Description 30
Mechanism Of Action 30
R&D Progress 30
domagrozumab - Drug Profile 31
Product Description 31
Mechanism Of Action 31
R&D Progress 31
luspatercept - Drug Profile 33
Product Description 33
Mechanism Of Action 33
R&D Progress 33
OSX-200 - Drug Profile 43
Product Description 43
Mechanism Of Action 43
R&D Progress 43
REGN-2477 + trevogrumab - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
RG-6206 - Drug Profile 45
Product Description 45
Mechanism Of Action 45
R&D Progress 45
SRK-015 - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
trevogrumab - Drug Profile 48
Product Description 48
Mechanism Of Action 48
R&D Progress 48
Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) - Dormant Products 50
Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) - Discontinued Products 51
Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) - Product Development Milestones 52
Featured News & Press Releases 52
Aug 01, 2017: Acceleron Announces First Patient Treated in Phase 2 Clinical Trial of ACE-083 in Charcot-Marie-Tooth Disease 52
Jul 10, 2017: Scholar Rock and the Miami Project to Cure Paralysis Present Preclinical Data Demonstrating Therapeutic Potential of SRK-015 in Spinal Cord Injury at 35th Annual National Neurotrauma Symposium 52
Jun 28, 2017: Scholar Rock to Develop SRK-015 to Improve Muscle Function in Patients with Spinal Muscular Atrophy 53
Jun 23, 2017: Acceleron Provides Updated Results from Ongoing Phase 2 Study of Luspatercept in Myelodysplastic Syndromes at the 22nd Congress of the European Hematology Association 54
Jun 23, 2017: Acceleron Provides Updated Results from Phase 2 Studies of Luspatercept in Beta-Thalassemia at the 22nd Congress of the European Hematology Association 55
Jun 01, 2017: Celgene and Acceleron Complete Target Enrollment in the MEDALIST and BELIEVE Phase 3 Studies of Luspatercept in Myelodysplastic Syndromes and Beta-Thalassemia 57
May 30, 2017: Pfizer Completes Enrollment of Phase 2 Study of Domagrozumab (PF- 06252616) in Duchenne Muscular Dystrophy 58
May 18, 2017: Acceleron Announces Clinical Presentations on Luspatercept at the 22nd Congress of the European Hematology Association 58
May 06, 2017: Luspatercept Phase 2 Data Presented at the 14th International Symposium on Myelodysplastic Syndromes 59
Apr 27, 2017: Acceleron Provides Clinical Development Updates on Luspatercept Program 60
Apr 06, 2017: Acceleron Announces Clinical Presentations on Luspatercept at the 14th International Symposium on Myelodysplastic Syndromes 61
Mar 16, 2017: Acceleron Announces Plans to Initiate a Phase 2 Trial of ACE-083 in Charcot-Marie-Tooth Neuromuscular Disease and Host Educational Webinar 61
Dec 22, 2016: Acceleron Announces First Patient Treated in Phase 2 Trial of ACE-083 in Facioscapulohumeral Muscular Dystrophy 62
Dec 05, 2016: Acceleron and Celgene Announce Updated Results from Phase 2 Studies of Luspatercept in Beta-Thalassemia Presented at the 58th Annual Meeting of the American Society of Hematology 62
Dec 04, 2016: Acceleron and Celgene Announce Updated Results from Ongoing Phase 2 Studies of Luspatercept in Myelodysplastic Syndromes at the 58th Annual Meeting of the American Society of Hematology 63
Appendix 65
Methodology 65
Coverage 65
Secondary Research 65
Primary Research 65
Expert Panel Validation 65
Contact Us 65
Disclaimer 66

List of Tables

Number of Products under Development by Stage of Development, H2 2017 8
Number of Products under Development by Therapy Areas, H2 2017 9
Number of Products under Development by Indication, H2 2017 10
Number of Products under Development by Companies, H2 2017 11
Products under Development by Companies, H2 2017 12
Products under Development by Companies, H2 2017 (Contd..1), H2 2017 13
Number of Products by Stage and Mechanism of Actions, H2 2017 14
Number of Products by Stage and Route of Administration, H2 2017 16
Number of Products by Stage and Molecule Type, H2 2017 18
Pipeline by Acceleron Pharma Inc, H2 2017 19
Pipeline by Amgen Inc, H2 2017 20
Pipeline by Bioleaders Corp, H2 2017 20
Pipeline by F. Hoffmann-La Roche Ltd, H2 2017 20
Pipeline by Pfizer Inc, H2 2017 21
Pipeline by Regeneron Pharmaceuticals Inc, H2 2017 21
Pipeline by Sarepta Therapeutics Inc, H2 2017 22
Pipeline by Scholar Rock Inc, H2 2017 22
Dormant Projects, H2 2017 50
Discontinued Products, H2 2017 51