Hematopoietic Stem Cell Transplantation - Pipeline Review, H2 2016

Global Markets Direct’s, ‘Hematopoietic Stem Cell Transplantation - Pipeline Review, H2 2016’, provides an overview of the Hematopoietic Stem Cell Transplantation pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Hematopoietic Stem Cell Transplantation, 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 Hematopoietic Stem Cell Transplantation 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 Hematopoietic Stem Cell Transplantation

The report reviews pipeline therapeutics for Hematopoietic Stem Cell Transplantation 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 Hematopoietic Stem Cell Transplantation therapeutics and enlists all their major and minor projects

The report assesses Hematopoietic Stem Cell Transplantation 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 Hematopoietic Stem Cell Transplantation

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 Hematopoietic Stem Cell Transplantation

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 Hematopoietic Stem Cell Transplantation 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

Actinium Pharmaceuticals, Inc.

Athersys, Inc.

Bellicum Pharmaceuticals, Inc.

Cell2B S.A.

Celldex Therapeutics, Inc.

Kiadis Pharma N.V.

Novartis AG

P2D Bioscience

Taiga Biotechnologies, Inc.

Targazyme, Inc.

Actinium Pharmaceuticals, Inc.

Athersys, Inc.

Bellicum Pharmaceuticals, Inc.

Cell2B S.A.

Celldex Therapeutics, Inc.

Kiadis Pharma N.V.

Novartis AG

P2D Bioscience

Taiga Biotechnologies, Inc.

Targazyme, Inc.

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 5

List of Figures 5

Introduction 6

Global Markets Direct Report Coverage 6

Hematopoietic Stem ...

Table of Contents

Table of Contents 2

List of Tables 5

List of Figures 5

Introduction 6

Global Markets Direct Report Coverage 6

Hematopoietic Stem Cell Transplantation Overview 7

Therapeutics Development 8

Pipeline Products for Hematopoietic Stem Cell Transplantation - Overview 8

Hematopoietic Stem Cell Transplantation - Therapeutics under Development by Companies 9

Hematopoietic Stem Cell Transplantation - Pipeline Products Glance 10

Clinical Stage Products 10

Early Stage Products 11

Hematopoietic Stem Cell Transplantation - Products under Development by Companies 12

Hematopoietic Stem Cell Transplantation - Companies Involved in Therapeutics Development 13

Actinium Pharmaceuticals, Inc. 13

Athersys, Inc. 14

Bellicum Pharmaceuticals, Inc. 15

Cell2B S.A. 16

Celldex Therapeutics, Inc. 17

Kiadis Pharma N.V. 18

Novartis AG 19

P2D Bioscience 20

Taiga Biotechnologies, Inc. 21

Targazyme, Inc. 22

Hematopoietic Stem Cell Transplantation - Therapeutics Assessment 23

Assessment by Monotherapy Products 23

Assessment by Target 24

Assessment by Mechanism of Action 26

Assessment by Route of Administration 28

Assessment by Molecule Type 30

Drug Profiles 32

ATIR-101 - Drug Profile 32

Product Description 32

Mechanism Of Action 32

R&D Progress 32

BPX-401 - Drug Profile 36

Product Description 36

Mechanism Of Action 36

R&D Progress 36

Casin - Drug Profile 38

Product Description 38

Mechanism Of Action 38

R&D Progress 38

CDX-301 - Drug Profile 39

Product Description 39

Mechanism Of Action 39

R&D Progress 39

CordSafe - Drug Profile 43

Product Description 43

Mechanism Of Action 43

R&D Progress 43

HSC-835 - Drug Profile 44

Product Description 44

Mechanism Of Action 44

R&D Progress 44

Iomab-B - Drug Profile 45

Product Description 45

Mechanism Of Action 45

R&D Progress 45

PF-05285401 - Drug Profile 50

Product Description 50

Mechanism Of Action 50

R&D Progress 50

TBX-1400 - Drug Profile 61

Product Description 61

Mechanism Of Action 61

R&D Progress 61

TXA-127 - Drug Profile 62

Product Description 62

Mechanism Of Action 62

R&D Progress 62

TZ-101 - Drug Profile 65

Product Description 65

Mechanism Of Action 65

R&D Progress 65

Hematopoietic Stem Cell Transplantation - Dormant Projects 68

Hematopoietic Stem Cell Transplantation - Discontinued Products 69

Hematopoietic Stem Cell Transplantation - Product Development Milestones 70

Featured News & Press Releases 70

Jun 30, 2016: Kiadis Pharma Orphan Drug Designation for ATIR101 further expanded to include treatment in a hematopoietic stem cell transplantation 70

May 18, 2016: Kiadis Pharma presents multiple sets of data at the International Society for Cellular Therapy 2016 Annual Meeting 70

Apr 25, 2016: Actinium to Host Webinar to Discuss SIERRA Trial -- Upcoming Pivotal, Phase 3 Clinical Study for Iomab-B 72

Apr 06, 2016: Actinium Announces Webinar to Discuss SIERRA Trial -- Upcoming Pivotal, Phase 3 Clinical Study for Iomab-B 72

Apr 04, 2016: Kiadis Pharma presents positive data on the primary endpoint of its single dose Phase II trial with ATIR101 74

Mar 30, 2016: Actinium Receives Orphan Drug Designation From FDA for Iomab-B in Treating Refractory and Relapsed Acute Myeloid Leukemia in Elderly Patients 76

Mar 29, 2016: Kiadis Pharma to host a webcast of the full dataset on the primary endpoint of its single-dose Phase II trial with ATIR101 76

Mar 08, 2016: Actinium Pharmaceuticals Announces Filing of Provisional Patent Application Related to Commercial Scale Labeling and Processes for Iomab-B 77

Feb 20, 2016: Celldex Presents Preliminary Cohort Data from Pilot Study of CDX-301 in Allogeneic Hematopoietic Stem Cell Harvest at the 2016 BMT Tandem Meeting 77

Feb 02, 2016: Actinium Pharmaceuticals Announces Participation at Upcoming Annual Meeting of Pre-Eminent Blood and Marrow Transplant Organization 78

Nov 30, 2015: Actinium to Present on Iomab-B at 8th Annual LD Micro Main Event Conference on December 2, 2015 79

Apr 23, 2015: Targazyme Announces FDA Concurrence On Targazyme’s Special Protocol Assessment For A Planned Phase 3 Registration Trial For The Treatment Of Patients With Hematologic Malignancie 79

Mar 17, 2015: Targazyme and M.D. Anderson Cancer Center Receive FDA IND Clearance for Phase I/II Study of TZ101-Treated Regulatory T Cells to Prevent Graft vs Host Disease in Patients Eligible for Hematologic Stem Cell Transplantation 80

Feb 10, 2015: Targazyme Receives Orphan Drug Designation to TZ101 for Use With Regulatory T Cells to Prevent & Reduce the Severity of Graft Versus Host Disease in Patients Eligible for Hematologic Stem Cell Transplantation 80

Sep 25, 2014: Celldex Therapeutics Initiates Pilot Study of CDX-301 in Allogeneic Hematopoietic Stem Cell Transplantation 81

Appendix 83

Methodology 83

Coverage 83

Secondary Research 83

Primary Research 83

Expert Panel Validation 83

Contact Us 83

Disclaimer 84

List of Tables

List of Tables

Number of Products under Development for Hematopoietic Stem Cell Transplantation, H2 2016 8

Number of Products under Development by Companies, H2 2016 ...

List of Tables

Number of Products under Development for Hematopoietic Stem Cell Transplantation, H2 2016 8

Number of Products under Development by Companies, H2 2016 9

Comparative Analysis by Clinical Stage Development, H2 2016 10

Comparative Analysis by Early Stage Development, H2 2016 11

Products under Development by Companies, H2 2016 12

Hematopoietic Stem Cell Transplantation - Pipeline by Actinium Pharmaceuticals, Inc., H2 2016 13

Hematopoietic Stem Cell Transplantation - Pipeline by Athersys, Inc., H2 2016 14

Hematopoietic Stem Cell Transplantation - Pipeline by Bellicum Pharmaceuticals, Inc., H2 2016 15

Hematopoietic Stem Cell Transplantation - Pipeline by Cell2B S.A. , H2 2016 16

Hematopoietic Stem Cell Transplantation - Pipeline by Celldex Therapeutics, Inc., H2 2016 17

Hematopoietic Stem Cell Transplantation - Pipeline by Kiadis Pharma N.V., H2 2016 18

Hematopoietic Stem Cell Transplantation - Pipeline by Novartis AG, H2 2016 19

Hematopoietic Stem Cell Transplantation - Pipeline by P2D Bioscience, H2 2016 20

Hematopoietic Stem Cell Transplantation - Pipeline by Taiga Biotechnologies, Inc., H2 2016 21

Hematopoietic Stem Cell Transplantation - Pipeline by Targazyme, Inc., H2 2016 22

Assessment by Monotherapy Products, H2 2016 23

Number of Products by Stage and Target, H2 2016 25

Number of Products by Stage and Mechanism of Action, H2 2016 27

Number of Products by Stage and Route of Administration, H2 2016 29

Number of Products by Stage and Molecule Type, H2 2016 31

Hematopoietic Stem Cell Transplantation - Dormant Projects, H2 2016 68

Hematopoietic Stem Cell Transplantation - Discontinued Products, H2 2016 69

List of Figures

List of Figures

Number of Products under Development for Hematopoietic Stem Cell Transplantation, H2 2016 8

Number of Products under Development by Companies, H2 2016 ...

List of Figures

Number of Products under Development for Hematopoietic Stem Cell Transplantation, H2 2016 8

Number of Products under Development by Companies, H2 2016 9

Comparative Analysis by Clinical Stage Development, H2 2016 10

Comparative Analysis by Early Stage Products, H2 2016 11

Assessment by Monotherapy Products, H2 2016 23

Number of Products by Targets, H2 2016 24

Number of Products by Stage and Targets, H2 2016 24

Number of Products by Mechanism of Actions, H2 2016 26

Number of Products by Stage and Mechanism of Actions, H2 2016 26

Number of Products by Routes of Administration, H2 2016 28

Number of Products by Stage and Routes of Administration, H2 2016 28

Number of Products by Molecule Types, H2 2016 30

Number of Products by Stage and Molecule Types, H2 2016 30

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