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

Global Markets Direct’s, ‘Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) – Pipeline Review, H2 2017’, provides in depth analysis on Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) targeted pipeline therapeutics. The report provides comprehensive information complete with analysis by indications, stage of development, 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 Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN) targeted therapeutics development and features dormant and discontinued projects. The report analyses the pipeline products from therapy areas Musculoskeletal Disorders, Genetic Disorders, Central Nervous System, Gastrointestinal, Hematological Disorders, Metabolic Disorders and Oncology under development targeting Growth/Differentiation Factor 8 (Myostatin or GDF8 or MSTN).

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.

*Updated report will be delivered in 48 hours of order confirmation.

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

Companies mentioned

Acceleron Pharma Inc

Amgen Inc

Bioleaders Corp

F. Hoffmann-La Roche Ltd

Pfizer Inc

Regeneron Pharmaceuticals Inc

Sarepta Therapeutics Inc

Scholar Rock Inc

Table of Contents

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

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

List of Figures

List of Figures

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 Top 10 Indications, H2 2017 10

Number of Products by Stage and Mechanism of Actions, H2 2017 14

Number of Products by Routes of Administration, H2 2017 15

Number of Products by Stage and Routes of Administration, H2 2017 15

Number of Products by Molecule Types, H2 2017 17

Number of Products by Stage and Molecule Types, H2 2017 17

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