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Thalassemia-Pipeline Review, H2 2016

Thalassemia-Pipeline Review, H2 2016


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Executive Summary

Thalassemia-Pipeline Review, H2 2016

Summary

Global Markets Direct's, 'Thalassemia-Pipeline Review, H2 2016', provides an overview of the Thalassemia pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Thalassemia, 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 Thalassemia 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 Thalassemia

The report reviews pipeline therapeutics for Thalassemia 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 Thalassemia therapeutics and enlists all their major and minor projects

The report assesses Thalassemia 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 Thalassemia

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 Thalassemia

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 Thalassemia 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

Table of Contents

Table of Contents 2

List of Tables 8

List of Figures 9

Introduction 10

Global Markets Direct Report Coverage 10

Thalassemia Overview 11

Therapeutics Development 12

Pipeline Products for Thalassemia-Overview 12

Pipeline Products for Thalassemia-Comparative Analysis 13

Thalassemia-Therapeutics under Development by Companies 14

Thalassemia-Therapeutics under Investigation by Universities/Institutes 16

Thalassemia-Pipeline Products Glance 17

Late Stage Products 17

Clinical Stage Products 18

Early Stage Products 19

Thalassemia-Products under Development by Companies 20

Thalassemia-Products under Investigation by Universities/Institutes 22

Thalassemia-Companies Involved in Therapeutics Development 23

Acceleron Pharma, Inc. 23

Acetylon Pharmaceuticals, Inc. 24

Acino International AG 25

Agios Pharmaceuticals, Inc. 26

Alnylam Pharmaceuticals, Inc. 27

Bellicum Pharmaceuticals, Inc. 28

Bluebird bio, Inc. 29

Editas Medicine, Inc. 30

Emmaus Medical, Inc. 31

Errant Gene Therapeutics, LLC 32

Gamida Cell Ltd. 33

Gilead Sciences, Inc. 34

Incyte Corporation 35

Ionis Pharmaceuticals, Inc. 36

IRBM Science Park SpA 37

Johnson & Johnson 38

Kiadis Pharma N.V. 39

La Jolla Pharmaceutical Company 40

Merck & Co., Inc. 41

Pfizer Inc. 42

PharmaEssentia Corporation 43

Sangamo BioSciences, Inc. 44

Thalassemia-Therapeutics Assessment 45

Assessment by Monotherapy Products 45

Assessment by Target 46

Assessment by Mechanism of Action 48

Assessment by Route of Administration 50

Assessment by Molecule Type 52

Drug Profiles 54

ACY-957-Drug Profile 54

Product Description 54

Mechanism Of Action 54

R&D Progress 54

AG-348-Drug Profile 56

Product Description 56

Mechanism Of Action 56

R&D Progress 56

ALN-TMP-Drug Profile 59

Product Description 59

Mechanism Of Action 59

R&D Progress 59

ambrisentan-Drug Profile 61

Product Description 61

Mechanism Of Action 61

R&D Progress 61

Antisense Oligonucleotides to Inhibit BCL11A and KLF1 for Hematological Disorders-Drug Profile 63

Product Description 63

Mechanism Of Action 63

R&D Progress 63

ATIR-201-Drug Profile 64

Product Description 64

Mechanism Of Action 64

R&D Progress 64

BB-305-Drug Profile 65

Product Description 65

Mechanism Of Action 65

R&D Progress 65

benserazide-Drug Profile 71

Product Description 71

Mechanism Of Action 71

R&D Progress 71

BPX-501-Drug Profile 73

Product Description 73

Mechanism Of Action 73

R&D Progress 73

BtX-13-Drug Profile 77

Product Description 77

Mechanism Of Action 77

R&D Progress 77

CNTO-530-Drug Profile 78

Product Description 78

Mechanism Of Action 78

R&D Progress 78

CordIn-Drug Profile 79

Product Description 79

Mechanism Of Action 79

R&D Progress 79

Drug for Thalassemia and Hemochromatosis-Drug Profile 80

Product Description 80

Mechanism Of Action 80

R&D Progress 80

Gene Therapy for Sickle Cell Disease and Thalassemia-Drug Profile 81

Product Description 81

Mechanism Of Action 81

R&D Progress 81

glutamine-Drug Profile 82

Product Description 82

Mechanism Of Action 82

R&D Progress 82

GSK-2696277-Drug Profile 85

Product Description 85

Mechanism Of Action 85

R&D Progress 85

IONISTMPRSS-6LRx-Drug Profile 86

Product Description 86

Mechanism Of Action 86

R&D Progress 86

LJPC-401-Drug Profile 87

Product Description 87

Mechanism Of Action 87

R&D Progress 87

luspatercept-Drug Profile 88

Product Description 88

Mechanism Of Action 88

R&D Progress 88

M-009-Drug Profile 94

Product Description 94

Mechanism Of Action 94

R&D Progress 94

M-012-Drug Profile 95

Product Description 95

Mechanism Of Action 95

R&D Progress 95

NiCord-Drug Profile 97

Product Description 97

Mechanism Of Action 97

R&D Progress 97

PF-04447943-Drug Profile 100

Product Description 100

Mechanism Of Action 100

R&D Progress 100

Piggybac-HBB-Drug Profile 101

Product Description 101

Mechanism Of Action 101

R&D Progress 101

PTG-300-Drug Profile 102

Product Description 102

Mechanism Of Action 102

R&D Progress 102

RNAi Gene Therapy for Sickle Cell Disease and Beta Thalassemia-Drug Profile 103

Product Description 103

Mechanism Of Action 103

R&D Progress 103

ruxolitinib phosphate-Drug Profile 104

Product Description 104

Mechanism Of Action 104

R&D Progress 104

SCD-101-Drug Profile 117

Product Description 117

Mechanism Of Action 117

R&D Progress 117

sirolimus-Drug Profile 118

Product Description 118

Mechanism Of Action 118

R&D Progress 118

Small Molecule for Beta Thalassemia and Sickle Cell Anemia-Drug Profile 119

Product Description 119

Mechanism Of Action 119

R&D Progress 119

Small Molecule to Inhibit Histone Deacetylase 1 for Beta-Thalassemia-Drug Profile 120

Product Description 120

Mechanism Of Action 120

R&D Progress 120

Small Molecules to Inhibit PRMT5 for Haemoglobinopathies and Solid Tumours-Drug Profile 121

Product Description 121

Mechanism Of Action 121

R&D Progress 121

sotatercept-Drug Profile 122

Product Description 122

Mechanism Of Action 122

R&D Progress 122

Stem Cell Therapy for Beta Thalassemia-Drug Profile 127

Product Description 127

Mechanism Of Action 127

R&D Progress 127

Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease-Drug Profile 128

Product Description 128

Mechanism Of Action 128

R&D Progress 128

Thalagen-Drug Profile 129

Product Description 129

Mechanism Of Action 129

R&D Progress 129

Thalassemia-Dormant Projects 130

Thalassemia-Product Development Milestones 131

Featured News & Press Releases 131

Jul 28, 2016: Protagonist Therapeutics Receives SBIR Funding for the Development of Injectable Hepcidin Mimetics for Treatment of Iron Overload Disorders 131

Jun 10, 2016: Acceleron and Celgene Announce Updated Results from an Ongoing Phase 2 Study of Luspatercept in Beta-Thalassemia Presented at the 21st Congress of the European Hematology Association 131

May 19, 2016: Agios to Present Clinical data from PKR Activator AG-348 at the 21st Congress of the European Hematology Association 132

May 19, 2016: Acceleron Announces Data Presentations with Luspatercept at the 21st Congress of the European Hematology Association 133

May 09, 2016: Merganser Biotech Announces Issuance of U.S. Patent 9,315,545 Covering Development Compound M012 134

May 06, 2016: Drug-like Compound Shows Promise in Treating Two Blood Diseases 134

May 04, 2016: Acetylon Pharmaceuticals Publishes New Mechanism for the Treatment of Sickle Cell Disease/Beta-Thalassemia with Selective HDAC1/2 Inhibition in PLoS One 135

Feb 24, 2016: Merganser Biotech Initiates First Clinical Trial 136

Dec 07, 2015: Acetylon Presents Data on the Pharmacokinetics and HbE/HbG Hemoglobin Induction Following Once Daily Dosing of an Oral HDAC1,2 Inhibitor in Animal Models 136

Dec 02, 2015: Emmaus Life Sciences to Present Additional New Data from its Phase 3 Sickle Cell Disease Trial at 57th Annual ASH Meeting 137

Nov 05, 2015: Acceleron Announces New Data on Luspatercept at the 2015 American Society of Hematology Annual Meeting 138

Oct 23, 2015: Acceleron Highlights Phase 3 Studies, New Clinical Results and Research Strategies at Research and Development Day Event 139

Oct 19, 2015: La Jolla Pharmaceutical Doses First Patient in Phase 1 Clinical Trial of LJPC-401 in Patients at Risk of Iron Overload 139

Aug 11, 2015: La Jolla Pharmaceutical Company Announces FDA Acceptance of IND for LJPC-401 140

Jun 12, 2015: Acceleron Announces New Luspatercept Phase 2 Clinical Results at the 20th Congress of the European Hematology Association 141

Appendix 143

Methodology 143

Coverage 143

Secondary Research 143

Primary Research 143

Expert Panel Validation 143

Contact Us 143

Disclaimer 144

List of Figures

Number of Products under Development for Thalassemia, H2 2016 12

Number of Products under Development for Thalassemia-Comparative Analysis, H2 2016 13

Number of Products under Development by Companies, H2 2016 14

Number of Products under Investigation by Universities/Institutes, H2 2016 16

Comparative Analysis by Clinical Stage Development, H2 2016 18

Comparative Analysis by Early Stage Products, H2 2016 19

Assessment by Monotherapy Products, H2 2016 45

Number of Products by Top 10 Targets, H2 2016 46

Number of Products by Stage and Top 10 Targets, H2 2016 46

Number of Products by Top 10 Mechanism of Actions, H2 2016 48

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2016 48

Number of Products by Routes of Administration, H2 2016 50

Number of Products by Stage and Routes of Administration, H2 2016 50

Number of Products by Top 10 Molecule Types, H2 2016 52

Number of Products by Stage and Molecule Types, H2 2016 52

List of Tables

Number of Products under Development for Thalassemia, H2 2016 12

Number of Products under Development for Thalassemia-Comparative Analysis, H2 2016 13

Number of Products under Development by Companies, H2 2016 14

Number of Products under Development by Companies, H2 2016 (Contd..1) 15

Number of Products under Investigation by Universities/Institutes, H2 2016 16

Comparative Analysis by Late Stage Development, H2 2016 17

Comparative Analysis by Clinical Stage Development, H2 2016 18

Comparative Analysis by Early Stage Development, H2 2016 19

Products under Development by Companies, H2 2016 20

Products under Development by Companies, H2 2016 (Contd..1) 21

Products under Investigation by Universities/Institutes, H2 2016 22

Thalassemia-Pipeline by Acceleron Pharma, Inc., H2 2016 23

Thalassemia-Pipeline by Acetylon Pharmaceuticals, Inc., H2 2016 24

Thalassemia-Pipeline by Acino International AG, H2 2016 25

Thalassemia-Pipeline by Agios Pharmaceuticals, Inc., H2 2016 26

Thalassemia-Pipeline by Alnylam Pharmaceuticals, Inc., H2 2016 27

Thalassemia-Pipeline by Bellicum Pharmaceuticals, Inc., H2 2016 28

Thalassemia-Pipeline by bluebird bio, Inc., H2 2016 29

Thalassemia-Pipeline by Editas Medicine, Inc., H2 2016 30

Thalassemia-Pipeline by Emmaus Medical, Inc., H2 2016 31

Thalassemia-Pipeline by Errant Gene Therapeutics, LLC, H2 2016 32

Thalassemia-Pipeline by Gamida Cell Ltd., H2 2016 33

Thalassemia-Pipeline by Gilead Sciences, Inc., H2 2016 34

Thalassemia-Pipeline by Incyte Corporation, H2 2016 35

Thalassemia-Pipeline by Ionis Pharmaceuticals, Inc., H2 2016 36

Thalassemia-Pipeline by IRBM Science Park SpA, H2 2016 37

Thalassemia-Pipeline by Johnson & Johnson, H2 2016 38

Thalassemia-Pipeline by Kiadis Pharma N.V., H2 2016 39

Thalassemia-Pipeline by La Jolla Pharmaceutical Company, H2 2016 40

Thalassemia-Pipeline by Merck & Co., Inc., H2 2016 41

Thalassemia-Pipeline by Pfizer Inc., H2 2016 42

Thalassemia-Pipeline by PharmaEssentia Corporation, H2 2016 43

Thalassemia-Pipeline by Sangamo BioSciences, Inc., H2 2016 44

Assessment by Monotherapy Products, H2 2016 45

Number of Products by Stage and Target, H2 2016 47

Number of Products by Stage and Mechanism of Action, H2 2016 49

Number of Products by Stage and Route of Administration, H2 2016 51

Number of Products by Stage and Molecule Type, H2 2016 53

Thalassemia-Dormant Projects, H2 2016 130

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Acceleron Pharma, Inc.

Acetylon Pharmaceuticals, Inc.

Acino International AG

Agios Pharmaceuticals, Inc.

Alnylam Pharmaceuticals, Inc.

Bellicum Pharmaceuticals, Inc.

Bluebird bio, Inc.

Editas Medicine, Inc.

Emmaus Medical, Inc.

Errant Gene Therapeutics, LLC

Gamida Cell Ltd.

Gilead Sciences, Inc.

Incyte Corporation

Ionis Pharmaceuticals, Inc.

IRBM Science Park SpA

Johnson & Johnson

Kiadis Pharma N.V.

La Jolla Pharmaceutical Company

Merck & Co., Inc.

Pfizer Inc.

PharmaEssentia Corporation

Sangamo BioSciences, Inc.

Thalassemia Therapeutic Products under Development, Key Players in Thalassemia Therapeutics, Thalassemia Pipeline Overview, Thalassemia Pipeline, Thalassemia Pipeline Assessment


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