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Aplastic Anemia - Pipeline Review, H2 2017

Published By :

Global Markets Direct

Published Date : Nov 2017

Category :

Pharmaceutical

No. of Pages : 75 Pages

Aplastic Anemia - Pipeline Review, H2 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Aplastic Anemia - Pipeline Review, H2 2017, 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 2017, 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, Preclinical and Discovery stages are 2, 3, 1, 1, 3 and 1 respectively.

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 Directs 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 its 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.
Table of Contents
Table of Contents 2
List of Tables 4
List of Figures 4
Introduction 5
Global Markets Direct Report Coverage 5
Aplastic Anemia - Overview 6
Aplastic Anemia - Therapeutics Development 7
Pipeline Overview 7
Pipeline by Companies 8
Products under Development by Companies 9
Aplastic Anemia - Therapeutics Assessment 10
Assessment by Target 10
Assessment by Mechanism of Action 12
Assessment by Route of Administration 14
Assessment by Molecule Type 16
Aplastic Anemia - Companies Involved in Therapeutics Development 18
3SBio Inc 18
Acceleron Pharma Inc 18
Amgen Inc 19
BioLineRx Ltd 19
F. Hoffmann-La Roche Ltd 20
Gamida Cell Ltd 20
Novartis AG 20
Regen BioPharma Inc 21
Aplastic Anemia - Drug Profiles 22
Anemir - Drug Profile 22
Product Description 22
Mechanism Of Action 22
R&D Progress 22
BL-8040 - Drug Profile 23
Product Description 23
Mechanism Of Action 23
R&D Progress 23
Cell Therapy for Aplastic Anemia - Drug Profile 36
Product Description 36
Mechanism Of Action 36
R&D Progress 36
CordIn - Drug Profile 37
Product Description 37
Mechanism Of Action 37
R&D Progress 37
eltrombopag olamine - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
EXG-34217 - Drug Profile 45
Product Description 45
Mechanism Of Action 45
R&D Progress 45
Gene Therapy to Activate Telomerase for Aplastic Anemia - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
HemaXellerate - Drug Profile 47
Product Description 47
Mechanism Of Action 47
R&D Progress 47
romiplostim - Drug Profile 51
Product Description 51
Mechanism Of Action 51
R&D Progress 51
sotatercept - Drug Profile 54
Product Description 54
Mechanism Of Action 54
R&D Progress 54
thrombopoietin - Drug Profile 59
Product Description 59
Mechanism Of Action 59
R&D Progress 59
Aplastic Anemia - Dormant Projects 60
Aplastic Anemia - Discontinued Products 61
Aplastic Anemia - Product Development Milestones 62
Featured News & Press Releases 62
Apr 20, 2017: NIH study in NEJM shows Novartis drug eltrombopag as first-line therapy with standard treatment improves responses in severe aplastic anemia 62
Oct 24, 2016: Pan Am Cancer Treatment Center Provides Final HemaXellerate Clinical Data to Regen BioPharma, for treatment of Bone Marrow Suppression Study 63
Sep 22, 2016: Regen BioPharma Responds to FDA Comments on Its Application for Orphan Drug Designation on its Aplastic Anemia Therapy 64
Aug 25, 2016: Health Canada issues advisory on REVOLADE citing risk of severe hepatotoxicity 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
Sep 25, 2015: Regen BioPharma Announces Positive Results from GLP Safety Study for HemaXellerate 69
Sep 10, 2015: BioLineRx Announces Regulatory Submission for Phase 2 Trial of BL-8040 as Novel Treatment for Hypoplastic Myelodysplastic Syndrome and Aplastic Anemia 70
Sep 02, 2015: Novartis receives EU approval for new Revolade use as first-in-class therapy for patients with severe aplastic anemia 70
Jul 24, 2015: Novartis drug Revolade recommended by CHMP for EU approval to treat patients with severe aplastic anemia, a serious blood disorder 71
Jul 21, 2015: Regen BioPharma Successfully Completes in Life Portion of Experiments Requested by FDA for HemaXellerate Pre-Clinical Study 72
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 2017 7
Number of Products under Development by Companies, H2 2017 8
Products under Development by Companies, H2 2017 9
Number of Products by Stage and Target, H2 2017 11
Number of Products by Stage and Mechanism of Action, H2 2017 13
Number of Products by Stage and Route of Administration, H2 2017 15
Number of Products by Stage and Molecule Type, H2 2017 17
Aplastic Anemia - Pipeline by 3SBio Inc, H2 2017 18
Aplastic Anemia - Pipeline by Acceleron Pharma Inc, H2 2017 18
Aplastic Anemia - Pipeline by Amgen Inc, H2 2017 19
Aplastic Anemia - Pipeline by BioLineRx Ltd, H2 2017 19
Aplastic Anemia - Pipeline by F. Hoffmann-La Roche Ltd, H2 2017 20
Aplastic Anemia - Pipeline by Gamida Cell Ltd, H2 2017 20
Aplastic Anemia - Pipeline by Novartis AG, H2 2017 21
Aplastic Anemia - Pipeline by Regen BioPharma Inc, H2 2017 21
Aplastic Anemia - Dormant Projects, H2 2017 60
Aplastic Anemia - Discontinued Products, H2 2017 61

List of Figures
Number of Products under Development for Aplastic Anemia, H2 2017 7
Number of Products under Development by Companies, H2 2017 8
Number of Products by Targets, H2 2017 10
Number of Products by Stage and Targets, H2 2017 10
Number of Products by Mechanism of Actions, H2 2017 12
Number of Products by Stage and Mechanism of Actions, H2 2017 12
Number of Products by Routes of Administration, H2 2017 14
Number of Products by Stage and Routes of Administration, H2 2017 14
Number of Products by Molecule Types, H2 2017 16
Number of Products by Stage and Molecule Types, H2 2017 16

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