Aplastic Anemia – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Aplastic Anemia – Pipeline Review, H2 2017’, provides an overview of the Aplastic Anemia pipeline landscape.

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

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

The report assesses Aplastic Anemia 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 Aplastic Anemia

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 Aplastic Anemia

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 Aplastic Anemia 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

Companies mentioned

3SBio Inc

Acceleron Pharma Inc

Amgen Inc

BioLineRx Ltd

F. Hoffmann-La Roche Ltd

Gamida Cell Ltd

Novartis AG

Regen BioPharma Inc

Table of Contents

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

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

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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