Amyloidosis – Pipeline Review, H1 2017

Global Markets Direct’s, ‘Amyloidosis – Pipeline Review, H1 2017’, provides an overview of the Amyloidosis pipeline landscape.

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

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

The report assesses Amyloidosis 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 Amyloidosis

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 Amyloidosis

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 Amyloidosis 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

Alnylam Pharmaceuticals Inc

Amgen Inc

Arcturus Therapeutics Inc

Bsim2

Celgene Corp

GlaxoSmithKline Plc

Ionis Pharmaceuticals Inc

Johnson & Johnson

Millennium Pharmaceuticals Inc

Neurimmune Holding AG

Novartis AG

Oncopeptides AB

Pfizer Inc

Proclara Biosciences Inc

Prothena Corp Plc

R Pharm

Regeneron Pharmaceuticals Inc

SOM Biotech SL

Spectrum Pharmaceuticals Inc

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 6

List of Figures 7

Introduction 8

Global Markets Direct Report Coverage 8

Amyloidosis - Overview 9

Amyloidosis - Therapeutics Development 10

Pipeline Overview 10

Pipeline by Companies 11

Pipeline by Universities/Institutes 14

Products under Development by Companies 15

Products under Development by Universities/Institutes 17

Amyloidosis - Therapeutics Assessment 18

Assessment by Target 18

Assessment by Mechanism of Action 20

Assessment by Route of Administration 22

Assessment by Molecule Type 24

Amyloidosis - Companies Involved in Therapeutics Development 26

Alnylam Pharmaceuticals Inc 26

Amgen Inc 26

Arcturus Therapeutics Inc 27

Bsim2 28

Celgene Corp 28

GlaxoSmithKline Plc 29

Ionis Pharmaceuticals Inc 29

Johnson & Johnson 30

Millennium Pharmaceuticals Inc 30

Neurimmune Holding AG 31

Novartis AG 31

Oncopeptides AB 32

Pfizer Inc 32

Proclara Biosciences Inc 33

Prothena Corp Plc 33

R Pharm 34

Regeneron Pharmaceuticals Inc 34

SOM Biotech SL 35

Spectrum Pharmaceuticals Inc 35

Amyloidosis - Drug Profiles 36

ALN-ANG - Drug Profile 36

Product Description 36

Mechanism Of Action 36

R&D Progress 36

ALN-TTRsc02 - Drug Profile 37

Product Description 37

Mechanism Of Action 37

R&D Progress 37

Antisense RNAi Oligonucleotides to Inhibit Amyloid Precursor Protein for Amyloidosis - Drug Profile 39

Product Description 39

Mechanism Of Action 39

R&D Progress 39

CAEL-101 - Drug Profile 40

Product Description 40

Mechanism Of Action 40

R&D Progress 40

canakinumab - Drug Profile 41

Product Description 41

Mechanism Of Action 41

R&D Progress 41

carfilzomib - Drug Profile 47

Product Description 47

Mechanism Of Action 47

R&D Progress 47

CLR-01 - Drug Profile 60

Product Description 60

Mechanism Of Action 60

R&D Progress 60

daratumumab - Drug Profile 62

Product Description 62

Mechanism Of Action 62

R&D Progress 62

dezamizumab - Drug Profile 76

Product Description 76

Mechanism Of Action 76

R&D Progress 76

dezamizumab + GSK-2315698 - Drug Profile 77

Product Description 77

Mechanism Of Action 77

R&D Progress 77

doxycycline hyclate - Drug Profile 78

Product Description 78

Mechanism Of Action 78

R&D Progress 78

EDE-1307 - Drug Profile 79

Product Description 79

Mechanism Of Action 79

R&D Progress 79

GSK-2315698 - Drug Profile 80

Product Description 80

Mechanism Of Action 80

R&D Progress 80

GSK-3039294 - Drug Profile 81

Product Description 81

Mechanism Of Action 81

R&D Progress 81

inotersen sodium - Drug Profile 82

Product Description 82

Mechanism Of Action 82

R&D Progress 82

ixazomib citrate - Drug Profile 86

Product Description 86

Mechanism Of Action 86

R&D Progress 86

LUNAR-TTR - Drug Profile 96

Product Description 96

Mechanism Of Action 96

R&D Progress 96

melphalan - Drug Profile 97

Product Description 97

Mechanism Of Action 97

R&D Progress 97

melphalan flufenamide - Drug Profile 101

Product Description 101

Mechanism Of Action 101

R&D Progress 101

Monoclonal Antibody to Inhibit ATTR for Familial Amyloid Polyneuropathy - Drug Profile 103

Product Description 103

Mechanism Of Action 103

R&D Progress 103

Monoclonal Antibody to Target CD66b for Blood Cancer and Metabolic Disorders - Drug Profile 104

Product Description 104

Mechanism Of Action 104

R&D Progress 104

NEOD-001 - Drug Profile 105

Product Description 105

Mechanism Of Action 105

R&D Progress 105

NI-301 - Drug Profile 110

Product Description 110

Mechanism Of Action 110

R&D Progress 110

NPT-289 - Drug Profile 111

Product Description 111

Mechanism Of Action 111

R&D Progress 111

Oligonucleotides to Inhibit Transthyretin for Amyloidosis - Drug Profile 112

Product Description 112

Mechanism Of Action 112

R&D Progress 112

patisiran - Drug Profile 113

Product Description 113

Mechanism Of Action 113

R&D Progress 113

pomalidomide - Drug Profile 121

Product Description 121

Mechanism Of Action 121

R&D Progress 121

PRX-004 - Drug Profile 128

Product Description 128

Mechanism Of Action 128

R&D Progress 128

RPH-104 - Drug Profile 129

Product Description 129

Mechanism Of Action 129

R&D Progress 129

Small Molecule to Inhibit Amyloid Beta Peptide for Alzheimer's Disease and Amyloidosis - Drug Profile 130

Product Description 130

Mechanism Of Action 130

R&D Progress 130

Small Molecules to Inhibit Transthyretin for Familial Amyloid Polyneuropathy - Drug Profile 131

Product Description 131

Mechanism Of Action 131

R&D Progress 131

Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Cardiomyopathy - Drug Profile 132

Product Description 132

Mechanism Of Action 132

R&D Progress 132

Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Polyneuropathy - Drug Profile 133

Product Description 133

Mechanism Of Action 133

R&D Progress 133

tafamidis meglumine - Drug Profile 134

Product Description 134

Mechanism Of Action 134

R&D Progress 134

tolcapone - Drug Profile 137

Product Description 137

Mechanism Of Action 137

R&D Progress 137

Amyloidosis - Dormant Projects 139

Amyloidosis - Discontinued Products 140

Amyloidosis - Product Development Milestones 141

Featured News & Press Releases 141

Jan 04, 2017: Fortress Biotech Forms New Subsidiary, Caelum Biosciences to Develop Novel Treatment for AL Amyloidosis 141

Dec 16, 2016: Novartis receives positive CHMP recommendation for Ilaris to treat rare diseases in adults and children called Periodic Fever Syndromes 142

Nov 07, 2016: VYNDAQEL (tafamidis) Receives Approval in Brazil by ANVISA for the Treatment of Transthyretin Familial Amyloid Polyneuropathy 142

Nov 03, 2016: Data for Prothena NEOD001 to be Presented at the 58th Annual American Society of Hematology Meeting 143

Oct 10, 2016: Data Monitoring Committee Recommends Continuation of APOLLO Phase 3 Clinical Trial of Patisiran for Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN) 144

Sep 23, 2016: Novartis receives three new FDA approvals for the expanded use of Ilaris treating rare Periodic Fever Syndrome conditions 145

Aug 08, 2016: Pfizer Announces Publication of New Analysis Showing Long-Term Therapy with VYNDAQEL (tafamidis) Slowed Progression of Rare Neurodegenerative Disease 145

Jul 07, 2016: Ionis Pharmaceuticals Reports Positive Clinical Data on IONIS-TTR Rx at the XV International Symposium on Amyloidosis 147

Jul 07, 2016: Ionis Pharmaceuticals Reports Positive Clinical Data on IONIS-TTR Rx at the XV International Symposium on Amyloidosis 148

Jul 05, 2016: Prothena Presents New Data from Phase 1/2 Study of NEOD001 Demonstrating Improvements in Three Organ Systems in Previously-Treated Patients with AL Amyloidosis 150

Jul 01, 2016: Alnylam Reports New Results on patisiran at the XV International Symposium on Amyloidosis 153

Jun 27, 2016: Alnylam to Report New Patisiran Results at the XV International Symposium on Amyloidosis 155

Jun 21, 2016: Prothena to Present at 15th International Symposium on Amyloidosis Showcasing Broad Commitment to Advancing New Therapies for Systemic Amyloidoses 156

Jun 09, 2016: Alnylam Initiates Phase 1 Clinical Trial for ALN-TTRsc02, an Investigational RNAi Therapeutic for the Treatment of TTR-Mediated Amyloidosis 158

Appendix 159

Methodology 159

Coverage 159

Secondary Research 159

Primary Research 159

Expert Panel Validation 159

Contact Us 159

Disclaimer 160

List of Tables

List of Tables

Number of Products under Development for Amyloidosis, H1 2017 10

Number of Products under Development by Companies, H1 2017 12

Number of Products under Development by Companies, H1 2017 (Contd..1), H1 2017 13

Number of Products under Development by Universities/Institutes, H1 2017 14

Products under Development by Companies, H1 2017 15

Products under Development by Companies, H1 2017 (Contd..1), H1 2017 16

Products under Development by Universities/Institutes, H1 2017 17

Number of Products by Stage and Target, H1 2017 19

Number of Products by Stage and Mechanism of Action, H1 2017 21

Number of Products by Stage and Route of Administration, H1 2017 23

Number of Products by Stage and Molecule Type, H1 2017 25

Amyloidosis – Pipeline by Alnylam Pharmaceuticals Inc, H1 2017 26

Amyloidosis – Pipeline by Amgen Inc, H1 2017 27

Amyloidosis – Pipeline by Arcturus Therapeutics Inc, H1 2017 27

Amyloidosis – Pipeline by Bsim2, H1 2017 28

Amyloidosis – Pipeline by Celgene Corp, H1 2017 28

Amyloidosis – Pipeline by GlaxoSmithKline Plc, H1 2017 29

Amyloidosis – Pipeline by Ionis Pharmaceuticals Inc, H1 2017 29

Amyloidosis – Pipeline by Johnson & Johnson, H1 2017 30

Amyloidosis – Pipeline by Millennium Pharmaceuticals Inc, H1 2017 30

Amyloidosis – Pipeline by Neurimmune Holding AG, H1 2017 31

Amyloidosis – Pipeline by Novartis AG, H1 2017 31

Amyloidosis – Pipeline by Oncopeptides AB, H1 2017 32

Amyloidosis – Pipeline by Pfizer Inc, H1 2017 32

Amyloidosis – Pipeline by Proclara Biosciences Inc, H1 2017 33

Amyloidosis – Pipeline by Prothena Corp Plc, H1 2017 33

Amyloidosis – Pipeline by R Pharm, H1 2017 34

Amyloidosis – Pipeline by Regeneron Pharmaceuticals Inc, H1 2017 34

Amyloidosis – Pipeline by SOM Biotech SL, H1 2017 35

Amyloidosis – Pipeline by Spectrum Pharmaceuticals Inc, H1 2017 35

Amyloidosis – Dormant Projects, H1 2017 139

Amyloidosis – Discontinued Products, H1 2017 140

List of Figures

List of Figures

Number of Products under Development for Amyloidosis, H1 2017 10

Number of Products under Development by Companies, H1 2017 11

Number of Products under Development by Universities/Institutes, H1 2017 14

Number of Products by Top 10 Targets, H1 2017 18

Number of Products by Stage and Top 10 Targets, H1 2017 18

Number of Products by Top 10 Mechanism of Actions, H1 2017 20

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2017 20

Number of Products by Routes of Administration, H1 2017 22

Number of Products by Stage and Routes of Administration, H1 2017 22

Number of Products by Molecule Types, H1 2017 24

Number of Products by Stage and Molecule Types, H1 2017 24

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