Thalassemia - Pipeline Review, H2 2012

Global Markets Direct, December 2012, Pages: 76

Thalassemia – Pipeline Review, H2 2012

Summary

Global Markets Direct’s, 'Thalassemia - Pipeline Review, H2 2012', provides an overview of the indication’s therapeutic pipeline. This report provides information on the therapeutic development for Thalassemia, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Thalassemia. Thalassemia - Pipeline Review, Half Year is built using data and information sourced from Global Markets Direct’s proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct’s team.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

- A snapshot of the global therapeutic scenario for Thalassemia.
- A review of the Thalassemia products under development by companies and universities/research institutes based on information derived from company and industry-specific sources.
- Coverage of products based on various stages of development ranging from discovery till registration stages.
- A feature on pipeline projects on the basis of monotherapy and combined therapeutics.
- Coverage of the Thalassemia pipeline on the basis of route of administration and molecule type.
- Key discontinued pipeline projects.
- Latest news and deals relating to the products.

Reasons to buy

- Identify and understand important and diverse types of therapeutics under development for Thalassemia.
- Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.
- Plan mergers and acquisitions effectively by identifying players of the most promising pipeline.
- 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.

2
List of Tables 5
List of Figures 6
Introduction 7
REPORT COVERAGE 7
Thalassemia Overview 8
Therapeutics Development 9
An Overview of Pipeline Products for Thalassemia 9
Thalassemia Therapeutics under Development by Companies 11
Thalassemia Therapeutics under Investigation by Universities/Institutes 12
Late Stage Products 13
Comparative Analysis 13
Mid Clinical Stage Products 14
Comparative Analysis 14
Early Clinical Stage Products 15
Comparative Analysis 15
Pre-Clinical Stage Products 16
Comparative Analysis 16
Thalassemia Therapeutics – Products under Development by Companies 17
Thalassemia Therapeutics – Products under Investigation by Universities/Institutes 18
Companies Involved in Thalassemia Therapeutics Development 19
Gamida Cell Ltd. 19
Alnylam Pharmaceuticals, Inc 20
Acceleron Pharma, Inc. 21
HemaQuest Pharmaceuticals, Inc. 22
GENETIX Pharmaceuticals, Inc. 23
Emmaus Medical, Inc. 24
Errant Gene Therapeutics, LLC 25
Thalassemia – Therapeutics Assessment 26
Assessment by Monotherapy Products 26
Assessment by Combination Products 27
Assessment by Route of Administration 28
Assessment by Molecule Type 30
Drug Profiles 32
sotatercept - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
ACE-536 - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
LentiGlobin - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
NiCord - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
Busulfan + Fludarabine+ Campath 1H + Cyclophosphamide + Mesna + Stem Cells Transplant - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 39
HQK-1001 - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
alemtuzumab + sirolimus + cyclophosphamide + Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation + Low Dose Irradiation - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 43
alemtuzumab + sirolimus + Non-Myeloablative Allogeneic Peripheral Hematopoietic Stem Cell Transplantation + Radiation Therapy - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
glutamine - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
fludarabine phosphate + busulfan + alemtuzumab + Allogeneic Stem Cell Transplantation - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
Deferoxamine + Exjade - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
decitabine - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
Thalagen - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
Hematopoietic Stem Cell Transplantation - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
Sodium 2,2 Dimethylbutyrate - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
Drug Targeting BCL11A - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
ALN-TMP - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
losartan potassium - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
Lentiviral-Mediated Gene Therapy - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Haematopoietic Stem Cells + Ex Vivo Modified CD38 Cells - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
Thalassemia Therapeutics – Drug Profile Updates 59
Thalassemia Therapeutics – Discontinued Products 64
Thalassemia Therapeutics - Dormant Products 65
Thalassemia – Product Development Milestones 67
Featured News & Press Releases 67
Oct 26, 2012: bluebird bio Receives $9.3m From California Institute For Regenerative Medicine To Further Gene Therapy Clinical Development 67
Oct 25, 2012: Acceleron Pharma's ACE-536 Corrects Anemia And Iron Overload In Preclinical Model Of Beta-Thalassemia 67
Sep 19, 2012: Gamida Cell Completes Enrollment For Phase I/II Study Of Second Pipeline Product Nicord For Hematological Malignancies 68
Dec 13, 2011: Novartis Pivotal Study Of Exjade Shows Significant Reduction Of Iron Overload In Patients With Non-Transfusion-Dependent Thalassemia 69
Oct 14, 2011: FDA Approves Ferriprox To Treat Patients With Excess Iron In Body 70
Sep 19, 2011: FerroKin BioSciences Starts Phase II Clinical Trial Of FBS0701 As New Oral Treatment For Transfusional Iron Overload In Pediatric Population 71
Sep 14, 2011: ApoPharma Announces FDA Advisory Committee Recommendation In Favor Of Ferriprox Approval 71
Mar 16, 2011: bluebird bio Receives Upto $4.2m From French Muscular Dystrophy Association For LentiGlobin 72
Dec 07, 2010: HemaQuest Presents Promising Clinical Results Of HQK-1001 At ASH Annual Meeting 72
Sep 15, 2010: bluebird bio Announces Publication Of Promising Phase I/II Study Results Of LentiGlobin In Nature 73
Appendix 75
Methodology 75
Coverage 75
Secondary Research 75
Primary Research 75
Expert Panel Validation 75
Contact Us 76
Disclaimer 76

List of Tables
Number of Products Under Development for Thalassemia, H2 2012 9
Products under Development for Thalassemia – Comparative Analysis, H2 2012 10
Number of Products under Development by Companies, H2 2012 11
Number of Products under Investigation by Universities/Institutes, H2 2012 12
Comparative Analysis by Late Stage Development, H2 2012 13
Comparative Analysis by Mid Clinical Stage Development, H2 2012 14
Comparative Analysis by Early Clinical Stage Development, H2 2012 15
Comparative Analysis by Pre-Clinical Stage Development, H2 2012 16
Products under Development by Companies, H2 2012 17
Products under Investigation by Universities/Institutes, H2 2012 18
Gamida Cell Ltd., H2 2012 19
Alnylam Pharmaceuticals, Inc, H2 2012 20
Acceleron Pharma, Inc., H2 2012 21
HemaQuest Pharmaceuticals, Inc., H2 2012 22
GENETIX Pharmaceuticals, Inc., H2 2012 23
Emmaus Medical, Inc., H2 2012 24
Errant Gene Therapeutics, LLC, H2 2012 25
Assessment by Monotherapy Products, H2 2012 26
Assessment by Combination Products, H2 2012 27
Assessment by Stage and Route of Administration, H2 2012 29
Assessment by Stage and Molecule Type, H2 2012 31
Thalassemia Therapeutics – Drug Profile Updates 59
Thalassemia Therapeutics – Discontinued Products 64
Thalassemia Therapeutics – Dormant Products 65
Thalassemia Therapeutics – Dormant Products (Contd..1) 66

List of Figures
Number of Products under Development for Thalassemia, H2 2012 9
Products under Development for Thalassemia – Comparative Analysis, H2 2012 10
Products under Development by Companies, H2 2012 11
Products under Investigation by Universities/Institutes, H2 2012 12
Late Stage Products, H2 2012 13
Mid Clinical Stage Products, H2 2012 14
Early Clinical Stage Products, H2 2012 15
Pre-Clinical Stage Products, H2 2012 16
Assessment by Monotherapy Products, H2 2012 26
Assessment by Combination Products, H2 2012 27
Assessment by Route of Administration, H2 2012 28
Assessment by Stage and Route of Administration, H2 2012 29
Assessment by Molecule Type, H2 2012 30
Assessment by Stage and Molecule Type, H2 2012 31

Customers who bought this item also bought

• Drug Addiction – Pipeline Review, H1 2013
• Substance (Drug) Abuse – Pipeline Review, H1 2013
• Nausea – Pipeline Review, H1 2013
• Bone Disorders – Pipeline Review, H1 2013
• Smoking Cessation – Pipeline Review, H1 2013
• Brain Tumor – Pipeline Review, H1 2013
• Pandemic Influenza – Pipeline Review, H1 2013
• Skin And Skin Structure Infections (SSSI) – Pipeline Review, H1 2013
• Ebola Viral Infections – Pipeline Review, H1 2013
• Premature Ejaculation – Pipeline Review, H1 2013