Summary
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Aplastic Anemia – Pipeline Review, H2 2016, provides an overview of the Aplastic Anemia (Hematological Disorders) pipeline landscape.
Aplastic anemia is a syndrome of bone marrow failure characterized by peripheral pancytopenia and marrow hypoplasia. Symptoms include fatigue, pale skin, rapid or irregular heart rate, shortness of breath with exertion, nosebleeds and bleeding gums, prolonged bleeding from cuts, skin rash, dizziness and headache. Risk factors include exposure to toxic chemicals, treatment with high-dose radiation or chemotherapy for cancer, certain blood diseases, autoimmune disorders and serious infections. Treatment includes immunosuppressants and stem cell transplant.
Report Highlights
Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Aplastic Anemia – Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Aplastic Anemia (Hematological Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.
The Aplastic Anemia (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Aplastic Anemia and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, IND/CTA Filed and Preclinical stages are 1, 2, 1, 1 and 5 respectively.Aplastic Anemia.
Aplastic Anemia (Hematological Disorders) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.
Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
Scope
- The pipeline guide provides a snapshot of the global therapeutic landscape of Aplastic Anemia (Hematological Disorders).
- The pipeline guide reviews pipeline therapeutics for Aplastic Anemia (Hematological Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Aplastic Anemia (Hematological Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Aplastic Anemia (Hematological Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Aplastic Anemia (Hematological Disorders)
Reasons to buy
- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Aplastic Anemia (Hematological Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Aplastic Anemia (Hematological Disorders) 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.
Companies Mentioned
3SBio Inc
Acceleron Pharma Inc
Amgen Inc
BioLineRx Ltd
F. Hoffmann-La Roche Ltd
Gamida Cell Ltd
Pluristem Therapeutics Inc
Regen BioPharma Inc
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Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Aplastic Anemia Overview 7
Therapeutics Development 8
Pipeline Products for Aplastic Anemia - Overview 8
Aplastic Anemia - Therapeutics under Development by Companies 9
Aplastic Anemia - Pipeline Products Glance 10
Late Stage Products 10
Clinical Stage Products 11
Early Stage Products 12
Aplastic Anemia - Products under Development by Companies 13
Aplastic Anemia - Companies Involved in Therapeutics Development 14
3SBio Inc 14
Acceleron Pharma Inc 15
Amgen Inc 16
BioLineRx Ltd 17
F. Hoffmann-La Roche Ltd 18
Gamida Cell Ltd 19
Pluristem Therapeutics Inc 20
Regen BioPharma Inc 21
Aplastic Anemia - Therapeutics Assessment 22
Assessment by Monotherapy Products 22
Assessment by Target 23
Assessment by Mechanism of Action 25
Assessment by Route of Administration 27
Assessment by Molecule Type 29
Drug Profiles 31
Anemir - Drug Profile 31
Product Description 31
Mechanism Of Action 31
R&D Progress 31
BL-8040 - Drug Profile 32
Product Description 32
Mechanism Of Action 32
R&D Progress 32
Cell Therapy for Aplastic Anemia - Drug Profile 42
Product Description 42
Mechanism Of Action 42
R&D Progress 42
CordIn - Drug Profile 43
Product Description 43
Mechanism Of Action 43
R&D Progress 43
Gene Therapy to Activate Telomerase for Aplastic Anemia - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
HemaXellerate - Drug Profile 45
Product Description 45
Mechanism Of Action 45
R&D Progress 45
PLXR-18 - Drug Profile 48
Product Description 48
Mechanism Of Action 48
R&D Progress 48
romiplostim - Drug Profile 54
Product Description 54
Mechanism Of Action 54
R&D Progress 54
sotatercept - Drug Profile 57
Product Description 57
Mechanism Of Action 57
R&D Progress 57
thrombopoietin - Drug Profile 61
Product Description 61
Mechanism Of Action 61
R&D Progress 61
Aplastic Anemia - Dormant Projects 62
Aplastic Anemia - Discontinued Products 63
Aplastic Anemia - Product Development Milestones 64
Featured News & Press Releases 64
Oct 24, 2016: Pan Am Cancer Treatment Center Provides Final HemaXellerate Clinical Data to Regen BioPharma, for treatment of Bone Marrow Suppression Study 64
Sep 22, 2016: Regen BioPharma Responds to FDA Comments on Its Application for Orphan Drug Designation on its Aplastic Anemia Therapy 64
May 11, 2016: Regen BioPharma Discusses Recent Developments in Aplastic Anemia Therapy and Future Potential Expansion of HemaXellerate 65
May 03, 2016: Regen BioPharma Receives Preliminary Clinical Data from Pan Am Cancer Center Regarding Their HemaXellerate Study in Cancer Patients 66
Jan 26, 2016: Regen BioPharma Submits Application to FDA for Orphan Drug Designation for its HemaXellerate Product 67
Dec 15, 2015: Regen BioPharma Receives FDA Clearance to Initiate Clinical Trial of HemaXellerate 67
Nov 23, 2015: BioLineRx Announces Initiation of Phase 1/2 Trial for Novel Treatment in Two Bone Marrow Failure Conditions 68
Nov 17, 2015: Regen BioPharma Submits Response to FDA Questions on HemaXellerate Investigational New Drug Application 69
Nov 17, 2015: Pluristem to Present New Data on PLX-R18 at American Society of Hematology’s Annual Meeting 69
Sep 25, 2015: Regen BioPharma Announces Positive Results from GLP Safety Study for HemaXellerate 70
Sep 10, 2015: BioLineRx Announces Regulatory Submission for Phase 2 Trial of BL-8040 as Novel Treatment for Hypoplastic Myelodysplastic Syndrome and Aplastic Anemia 71
Jul 21, 2015: Regen BioPharma Successfully Completes in Life Portion of Experiments Requested by FDA for HemaXellerate Pre-Clinical Study 71
Jul 09, 2015: Regen BioPharma Reports Successful Dosing of HemaXellerate in FDA-Requested GLP Animal Study 72
May 05, 2015: Regen BioPharma presents HemaXellerate at the 13th International Symposium on Myelodysplastic Syndromes Held April 29 to May 2, 2015 in Washington, DC 73
Apr 22, 2015: Regen BioPharma Engages Charles River Laboratories to Initiate FDA-Requested Study to Support Initiation of HemaXellerate Clinical Trial 73
Appendix 74
Methodology 74
Coverage 74
Secondary Research 74
Primary Research 74
Expert Panel Validation 74
Contact Us 74
Disclaimer 75
List of Tables
Number of Products under Development for Aplastic Anemia, H2 2016 8
Number of Products under Development by Companies, H2 2016 9
Comparative Analysis by Late Stage Development, H2 2016 10
Comparative Analysis by Clinical Stage Development, H2 2016 11
Comparative Analysis by Early Stage Development, H2 2016 12
Products under Development by Companies, H2 2016 13
Aplastic Anemia - Pipeline by 3SBio Inc, H2 2016 14
Aplastic Anemia - Pipeline by Acceleron Pharma Inc, H2 2016 15
Aplastic Anemia - Pipeline by Amgen Inc, H2 2016 16
Aplastic Anemia - Pipeline by BioLineRx Ltd, H2 2016 17
Aplastic Anemia - Pipeline by F. Hoffmann-La Roche Ltd, H2 2016 18
Aplastic Anemia - Pipeline by Gamida Cell Ltd, H2 2016 19
Aplastic Anemia - Pipeline by Pluristem Therapeutics Inc, H2 2016 20
Aplastic Anemia - Pipeline by Regen BioPharma Inc, H2 2016 21
Assessment by Monotherapy Products, H2 2016 22
Number of Products by Stage and Target, H2 2016 24
Number of Products by Stage and Mechanism of Action, H2 2016 26
Number of Products by Stage and Route of Administration, H2 2016 28
Number of Products by Stage and Molecule Type, H2 2016 30
Aplastic Anemia - Dormant Projects, H2 2016 62
Aplastic Anemia - Discontinued Products, H2 2016 63
List of Figures
Number of Products under Development for Aplastic Anemia, H2 2016 8
Number of Products under Development by Companies, H2 2016 9
Comparative Analysis by Clinical Stage Development, H2 2016 11
Comparative Analysis by Early Stage Products, H2 2016 12
Assessment by Monotherapy Products, H2 2016 22
Number of Products by Targets, H2 2016 23
Number of Products by Stage and Targets, H2 2016 23
Number of Products by Mechanism of Actions, H2 2016 25
Number of Products by Stage and Mechanism of Actions, H2 2016 25
Number of Products by Routes of Administration, H2 2016 27
Number of Products by Stage and Routes of Administration, H2 2016 27
Number of Products by Molecule Types, H2 2016 29
Number of Products by Stage and Molecule Types, H2 2016 29
