Thalassemia - Pipeline Review, H2 2016

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Date: 31-Aug-2016
No. of pages: 144
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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

Thalassemia - Pipeline Review, H2 2016

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 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

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
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