Aplastic Anemia - Pipeline Review, H1 2016 Summary Global Markets Direct's, 'Aplastic Anemia - Pipeline Review, H1 2016', 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
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 Clinical Stage Products 10 Early Stage Products 11 Aplastic Anemia - Products under Development by Companies 12 Aplastic Anemia - Companies Involved in Therapeutics Development 13 3SBio Inc. 13 Acceleron Pharma, Inc. 14 Amgen Inc. 15 BioLineRx, Ltd. 16 F. Hoffmann-La Roche Ltd. 17 Novartis AG 18 Pluristem Therapeutics Inc. 19 Regen BioPharma, Inc. 20 Aplastic Anemia - Therapeutics Assessment 21 Assessment by Monotherapy Products 21 Assessment by Target 22 Assessment by Mechanism of Action 24 Assessment by Route of Administration 26 Assessment by Molecule Type 28 Drug Profiles 30 Anemir - Drug Profile 30 Product Description 30 Mechanism of Action 30 R&D Progress 30 BL-8040 - Drug Profile 31 Product Description 31 Mechanism of Action 31 R&D Progress 31 Cell Therapy for Aplastic Anemia - Drug Profile 34 Product Description 34 Mechanism of Action 34 R&D Progress 34 eltrombopag olamine - Drug Profile 35 Product Description 35 Mechanism of Action 35 R&D Progress 35 Gene Therapy to Activate Telomerase for Aplastic Anemia - Drug Profile 38 Product Description 38 Mechanism of Action 38 R&D Progress 38 HemaXellerate - Drug Profile 39 Product Description 39 Mechanism of Action 39 R&D Progress 39 PLXR-18 - Drug Profile 40 Product Description 40 Mechanism of Action 40 R&D Progress 40 romiplostim - Drug Profile 43 Product Description 43 Mechanism of Action 43 R&D Progress 43 sotatercept - Drug Profile 45 Product Description 45 Mechanism of Action 45 R&D Progress 45 thrombopoietin - Drug Profile 47 Product Description 47 Mechanism of Action 47 R&D Progress 47 Aplastic Anemia - Recent Pipeline Updates 48 Aplastic Anemia - Dormant Projects 71 Aplastic Anemia - Discontinued Products 72 Aplastic Anemia - Product Development Milestones 73 Featured News & Press Releases 73 May 11, 2016: Regen BioPharma Discusses Recent Developments in Aplastic Anemia Therapy and Future Potential Expansion of HemaXellerate 73 Jan 26, 2016: Regen BioPharma Submits Application to FDA for Orphan Drug Designation for its HemaXellerate Product 73 Dec 15, 2015: Regen BioPharma Receives FDA Clearance to Initiate Clinical Trial of HemaXellerate 74 Nov 23, 2015: BioLineRx Announces Initiation of Phase 1/2 Trial for Novel Treatment in Two Bone Marrow Failure Conditions 75 Nov 17, 2015: Regen BioPharma Submits Response to FDA Questions on HemaXellerate Investigational New Drug Application 76 Nov 17, 2015: Pluristem to Present New Data on PLX-R18 at American Society of Hematology's Annual Meeting 76 Sep 25, 2015: Regen BioPharma Announces Positive Results from GLP Safety Study for HemaXellerate 77 Sep 10, 2015: BioLineRx Announces Regulatory Submission for Phase 2 Trial of BL-8040 as Novel Treatment for Hypoplastic Myelodysplastic Syndrome and Aplastic Anemia 77 Sep 02, 2015: Novartis receives EU approval for new Revolade use as first-in-class therapy for patients with severe aplastic anemia 78 Jul 24, 2015: Novartis drug Revolade recommended by CHMP for EU approval to treat patients with severe aplastic anemia, a serious blood disorder 79 Appendix 80 Methodology 80 Coverage 80 Secondary Research 80 Primary Research 80 Expert Panel Validation 80 Contact Us 80 Disclaimer 81
List of Tables
Number of Products under Development for Aplastic Anemia, H1 2016 8 Number of Products under Development by Companies, H1 2016 9 Comparative Analysis by Clinical Stage Development, H1 2016 10 Comparative Analysis by Early Stage Development, H1 2016 11 Products under Development by Companies, H1 2016 12 Aplastic Anemia - Pipeline by 3SBio Inc., H1 2016 13 Aplastic Anemia - Pipeline by Acceleron Pharma, Inc., H1 2016 14 Aplastic Anemia - Pipeline by Amgen Inc., H1 2016 15 Aplastic Anemia - Pipeline by BioLineRx, Ltd., H1 2016 16 Aplastic Anemia - Pipeline by F. Hoffmann-La Roche Ltd., H1 2016 17 Aplastic Anemia - Pipeline by Novartis AG, H1 2016 18 Aplastic Anemia - Pipeline by Pluristem Therapeutics Inc., H1 2016 19 Aplastic Anemia - Pipeline by Regen BioPharma, Inc., H1 2016 20 Assessment by Monotherapy Products, H1 2016 21 Number of Products by Stage and Target, H1 2016 23 Number of Products by Stage and Mechanism of Action, H1 2016 25 Number of Products by Stage and Route of Administration, H1 2016 27 Number of Products by Stage and Molecule Type, H1 2016 29 Aplastic Anemia Therapeutics - Recent Pipeline Updates, H1 2016 48 Aplastic Anemia - Dormant Projects, H1 2016 71 Aplastic Anemia - Discontinued Products, H1 2016 72
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