Gaucher Disease-Pipeline Review, H1 2016

Gaucher Disease-Pipeline Review, H1 2016


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Executive Summary

Gaucher Disease-Pipeline Review, H1 2016

Summary

Global Markets Direct's, 'Gaucher Disease-Pipeline Review, H1 2016', provides an overview of the Gaucher Disease pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Gaucher Disease, 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 Gaucher Disease 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 Gaucher Disease

The report reviews pipeline therapeutics for Gaucher Disease 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 Gaucher Disease therapeutics and enlists all their major and minor projects

The report assesses Gaucher Disease 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 Gaucher Disease

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 Gaucher Disease

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 Gaucher Disease 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



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Table of Contents

Table of Contents 2

List of Tables 6

List of Figures 7

Introduction 8

Global Markets Direct Report Coverage 8

Gaucher Disease Overview 9

Therapeutics Development 10

Pipeline Products for Gaucher Disease-Overview 10

Pipeline Products for Gaucher Disease-Comparative Analysis 11

Gaucher Disease-Therapeutics under Development by Companies 12

Gaucher Disease-Therapeutics under Investigation by Universities/Institutes 13

Gaucher Disease-Pipeline Products Glance 14

Clinical Stage Products 14

Early Stage Products 15

Gaucher Disease-Products under Development by Companies 16

Gaucher Disease-Products under Investigation by Universities/Institutes 17

Gaucher Disease-Companies Involved in Therapeutics Development 18

Actelion Ltd 18

Bioorganic Research and Services S.A. 19

Genzyme Corporation 20

greenovation Biotech GmbH 21

JCR Pharmaceuticals Co., Ltd. 22

Lixte Biotechnology Holdings, Inc. 23

Neuraltus Pharmaceuticals, Inc. 24

Okklo Life Sciences BV 25

Pharming Group N.V. 26

Protalix BioTherapeutics, Inc. 27

Sangamo BioSciences, Inc. 28

Shire Plc 29

The International Biotechnology Center (IBC) Generium 30

Gaucher Disease-Therapeutics Assessment 31

Assessment by Monotherapy Products 31

Assessment by Target 32

Assessment by Mechanism of Action 34

Assessment by Route of Administration 36

Assessment by Molecule Type 38

Drug Profiles 40

ambroxol-Drug Profile 40

Product Description 40

Mechanism Of Action 40

R&D Progress 40

BNT-001-Drug Profile 42

Product Description 42

Mechanism Of Action 42

R&D Progress 42

ibiglustat-Drug Profile 43

Product Description 43

Mechanism Of Action 43

R&D Progress 43

imiglucerase biosimilar-Drug Profile 45

Product Description 45

Mechanism Of Action 45

R&D Progress 45

LB-201-Drug Profile 46

Product Description 46

Mechanism Of Action 46

R&D Progress 46

LB-205-Drug Profile 47

Product Description 47

Mechanism Of Action 47

R&D Progress 47

miglustat-Drug Profile 48

Product Description 48

Mechanism Of Action 48

R&D Progress 48

MOSS-GBA-Drug Profile 50

Product Description 50

Mechanism Of Action 50

R&D Progress 50

NP-003-Drug Profile 51

Product Description 51

Mechanism Of Action 51

R&D Progress 51

OKL-1014-Drug Profile 52

Product Description 52

Mechanism Of Action 52

R&D Progress 52

PRX-112-Drug Profile 53

Product Description 53

Mechanism Of Action 53

R&D Progress 53

Recombinant Beta Glucosidase 2 Replacement for Gaucher's Disease and Male Infertility-Drug Profile 55

Product Description 55

Mechanism Of Action 55

R&D Progress 55

Recombinant Beta-Cerebrosidase Replacement for Gaucher's Disease-Drug Profile 56

Product Description 56

Mechanism Of Action 56

R&D Progress 56

SBLSD-3-Drug Profile 57

Product Description 57

Mechanism Of Action 57

R&D Progress 57

Small Molecule to Activate Beta-Galactosidase for GM1 Gangliosidosis and Gaucher's disease-Drug Profile 58

Product Description 58

Mechanism Of Action 58

R&D Progress 58

Small Molecule to Activate Glucocerebrosidase for Parkinson's, Gaucher's and Neurodegenerative Diseases-Drug Profile 59

Product Description 59

Mechanism Of Action 59

R&D Progress 59

Small Molecules to Activate Glucocerebrosidase for Gaucher and Parkinson Disease-Drug Profile 60

Product Description 60

Mechanism Of Action 60

R&D Progress 60

Small Molecules to Inhibit Glucosidase for Diabetes and Gaucher's Disease-Drug Profile 61

Product Description 61

Mechanism Of Action 61

R&D Progress 61

taliglucerase alfa-Drug Profile 62

Product Description 62

Mechanism Of Action 62

R&D Progress 62

velaglucerase alfa-Drug Profile 63

Product Description 63

Mechanism Of Action 63

R&D Progress 63

Gaucher Disease-Dormant Projects 64

Gaucher Disease-Discontinued Products 66

Gaucher Disease-Product Development Milestones 67

Featured News & Press Releases 67

Sep 02, 2014: Shire's VPRIV400 units/vial launched in Japan for the improvement of symptoms of Gaucher disease 67

Jul 04, 2014: Shire's VPRIV approved in Japan for the improvement of symptoms of Gaucher disease 67

Jun 18, 2014: Protalix BioTherapeutics Initiates Phase II Study With PRX-112, an Orally-Administered Enzyme Replacement Therapy for the Treatment of Gaucher Disease 68

Mar 14, 2014: Shire Pharmaceuticals Initiates Voluntary Nationwide Recall Of One Batch, Packaged into Three Lots, of VPRIV (velaglucerase alfa for injection) 69

Feb 14, 2014: Shire Announces FDA Approval of Manufacturing Facility for VPRIV (Velaglucerase Alfa for Injection) Drug Substance 69

Feb 12, 2014: Protalix BioTherapeutics Announces Oral GCD Data to be Presented at WORLD Symposium 2014 70

Oct 14, 2013: Protalix BioTherapeutics Announces Positive Phase I Clinical Trial Results for Oral GCD in Gaucher Disease Patients 71

Apr 02, 2013: Protalix BioTherapeutics Treats First Gaucher Patient In Phase I Study With PRX-112 72

Mar 07, 2013: Protalix BioTherapeutics Receives Approval From Israeli Ministry Of Health To Initiate Phase I Clinical Trial Of PRX-112 For Treatment Of Gaucher Disease 72

Jun 28, 2012: Shire's Vpriv Shows Significant Improvement In Gaucher-Related Bone Disease 73

Jun 28, 2012: Shire's Vpriv Shows Significant Improvement In Gaucher-Related Bone Disease 74

Feb 22, 2012: Shire Announces European Approval Of Manufacturing Facility For VPRIV 75

Dec 19, 2011: Lixte's LB-205 Shows Therapeutic Potential For Gaucher Disease 75

Nov 22, 2011: Shire Announces Regulatory Filings In US And EU For New Manufacturing Facility 76

Mar 21, 2011: Shire Presents Data For VPRIV In Gaucher Disease At 2011 ACMG Annual Meeting 76

Appendix 77

Methodology 77

Coverage 77

Secondary Research 77

Primary Research 77

Expert Panel Validation 77

Contact Us 77

Disclaimer 78

List of Figures

Number of Products under Development for Gaucher Disease, H1 2016 10

Number of Products under Development for Gaucher Disease-Comparative Analysis, H1 2016 11

Number of Products under Development by Companies, H1 2016 12

Number of Products under Investigation by Universities/Institutes, H1 2016 13

Comparative Analysis by Clinical Stage Development, H1 2016 14

Comparative Analysis by Early Stage Products, H1 2016 15

Assessment by Monotherapy Products, H1 2016 31

Number of Products by Targets, H1 2016 32

Number of Products by Stage and Targets, H1 2016 32

Number of Products by Mechanism of Actions, H1 2016 34

Number of Products by Stage and Mechanism of Actions, H1 2016 34

Number of Products by Routes of Administration, H1 2016 36

Number of Products by Stage and Routes of Administration, H1 2016 36

Number of Products by Molecule Types, H1 2016 38

Number of Products by Stage and Molecule Types, H1 2016 38

List of Tables

Number of Products under Development for Gaucher Disease, H1 2016 10

Number of Products under Development for Gaucher Disease-Comparative Analysis, H1 2016 11

Number of Products under Development by Companies, H1 2016 12

Number of Products under Investigation by Universities/Institutes, H1 2016 13

Comparative Analysis by Clinical Stage Development, H1 2016 14

Comparative Analysis by Early Stage Development, H1 2016 15

Products under Development by Companies, H1 2016 16

Products under Investigation by Universities/Institutes, H1 2016 17

Gaucher Disease-Pipeline by Actelion Ltd, H1 2016 18

Gaucher Disease-Pipeline by Bioorganic Research and Services S.A., H1 2016 19

Gaucher Disease-Pipeline by Genzyme Corporation, H1 2016 20

Gaucher Disease-Pipeline by greenovation Biotech GmbH, H1 2016 21

Gaucher Disease-Pipeline by JCR Pharmaceuticals Co., Ltd., H1 2016 22

Gaucher Disease-Pipeline by Lixte Biotechnology Holdings, Inc., H1 2016 23

Gaucher Disease-Pipeline by Neuraltus Pharmaceuticals, Inc., H1 2016 24

Gaucher Disease-Pipeline by Okklo Life Sciences BV, H1 2016 25

Gaucher Disease-Pipeline by Pharming Group N.V., H1 2016 26

Gaucher Disease-Pipeline by Protalix BioTherapeutics, Inc., H1 2016 27

Gaucher Disease-Pipeline by Sangamo BioSciences, Inc., H1 2016 28

Gaucher Disease-Pipeline by Shire Plc, H1 2016 29

Gaucher Disease-Pipeline by The International Biotechnology Center (IBC) Generium, H1 2016 30

Assessment by Monotherapy Products, H1 2016 31

Number of Products by Stage and Target, H1 2016 33

Number of Products by Stage and Mechanism of Action, H1 2016 35

Number of Products by Stage and Route of Administration, H1 2016 37

Number of Products by Stage and Molecule Type, H1 2016 39

Gaucher Disease-Dormant Projects, H1 2016 64

Gaucher Disease-Dormant Projects (Contd..1), H1 2016 65

Gaucher Disease-Discontinued Products, H1 2016 66

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Actelion Ltd

Bioorganic Research and Services S.A.

Genzyme Corporation

greenovation Biotech GmbH

JCR Pharmaceuticals Co., Ltd.

Lixte Biotechnology Holdings, Inc.

Neuraltus Pharmaceuticals, Inc.

Okklo Life Sciences BV

Pharming Group N.V.

Protalix BioTherapeutics, Inc.

Sangamo BioSciences, Inc.

Shire Plc

The International Biotechnology Center (IBC) Generium

Gaucher Disease Therapeutic Products under Development, Key Players in Gaucher Disease Therapeutics, Gaucher Disease Pipeline Overview, Gaucher Disease Pipeline, Gaucher Disease Pipeline Assessment

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