Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) - Pipeline Review, H2 2017

Published: Oct, 2017 | Pages: 95 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) - Pipeline Review, H2 2017

Summary

Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) pipeline Target constitutes close to 28 molecules. Out of which approximately 26 molecules are developed by companies and remaining by the universities/institutes. The latest report Proprotein Convertase SubtilisinKexin Type 9 - Pipeline Review, H2 2017, outlays comprehensive information on the Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type.

Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) - Proprotein convertase subtilisin/kexin type 9 (PCSK9) is an enzyme encoded by the PCSK9 gene. It is a crucial player in the regulation of plasma cholesterol homeostasis. It binds to low-density lipid receptor family members. It acts via a non-proteolytic mechanism to enhance the degradation of the hepatic LDLR through a clathrin LDLRAP1/ARH-mediated pathway. It prevents the recycling of LDLR from endosomes to the cell surface or directs it to lysosomes for degradation. It induces ubiquitination of LDLR leading to its subsequent degradation. It inhibits intracellular degradation of APOB via the autophagosome/lysosome pathway in a LDLR-independent manner.  The molecules developed by companies in Phase III, Phase II, Phase I, IND/CTA Filed, Preclinical, Discovery and Unknown stages are 1, 3, 4, 3, 8, 6 and 1 respectively. Similarly, the universities portfolio in Preclinical and Discovery stages comprises 1 and 1 molecules, respectively. Report covers products from therapy areas Metabolic Disorders, Cardiovascular, Gastrointestinal, Immunology, Infectious Disease and Oncology which include indications Hypercholesterolemia, Hyperlipidemia, Atherosclerosis, Cardiovascular Disease, Homozygous Familial Hypercholesterolemia (HoFH), Non-Alcoholic Steatohepatitis (NASH), Type 2 Diabetes, Bacterial Sepsis, Hypertriglyceridemia, Inflammation, Liver Diseases, Metabolic Syndrome, Non Alcoholic Fatty Liver Disease (NAFLD) and Peripheral Arterial Disease (PAD)/ Peripheral Vascular Disease (PVD). 

Furthermore, this report also reviews key players involved in Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) targeted therapeutics development with respective active and dormant or discontinued projects. Driven by data and information sourced from 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.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The report provides a snapshot of the global therapeutic landscape for Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.)
- The report reviews Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) targeted therapeutics under development 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 in Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) targeted therapeutics and enlists all their major and minor projects 
- The report assesses Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) targeted therapeutics based on 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 and deals related to Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) targeted therapeutics

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 the targeted therapy areas and indications for Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.)
- Identify the use of drugs for target identification and drug repurposing
- 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 Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) development landscape 
- 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.

 Table of Contents

Table of Contents 2
List of Tables 6
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) - Overview 8
Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) - Therapeutics Development 9
Products under Development by Stage of Development 9
Products under Development by Therapy Area 10
Products under Development by Indication 11
Products under Development by Companies 12
Products under Development by Universities/Institutes 17
Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) - Therapeutics Assessment 19
Assessment by Mechanism of Action 19
Assessment by Route of Administration 20
Assessment by Molecule Type 22
Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) - Companies Involved in Therapeutics Development 24
AFFiRiS AG 24
Betagenon AB 24
Bioleaders Corp 25
BioLingus AG 25
Catabasis Pharmaceuticals Inc 25
Dicerna Pharmaceuticals Inc 26
Eli Lilly and Co 26
Ensemble Therapeutics Corp 27
Kowa Co Ltd 27
Novartis AG 27
Pfizer Inc 28
Regeneron Pharmaceuticals Inc 28
Serometrix LLC 29
Shanghai Fudan-Zhangjiang Bio-Pharmaceutical Co Ltd 29
The Medicines Company 30
Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) - Drug Profiles 31
AK-102 - Drug Profile 31
Product Description 31
Mechanism Of Action 31
R&D Progress 31
alirocumab - Drug Profile 32
Product Description 32
Mechanism Of Action 32
R&D Progress 32
Antisense Oligonucleotide to Inhibit PCSK9 for Hypercholesterolemia - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
Antisense RNAi Oligonucleotide to Inhibit PCSK-9 for Hypercholesterolemia - Drug Profile 45
Product Description 45
Mechanism Of Action 45
R&D Progress 45
AT-04A - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
AT-06A - Drug Profile 47
Product Description 47
Mechanism Of Action 47
R&D Progress 47
BLSM-201 - Drug Profile 48
Product Description 48
Mechanism Of Action 48
R&D Progress 48
CAT-2000 Series - Drug Profile 49
Product Description 49
Mechanism Of Action 49
R&D Progress 49
CVI-LM001 - Drug Profile 50
Product Description 50
Mechanism Of Action 50
R&D Progress 50
DCRPCSK-9 - Drug Profile 51
Product Description 51
Mechanism Of Action 51
R&D Progress 51
Gene Therapy to Inhibit PCSK9 for Hypercholesterolemia - Drug Profile 52
Product Description 52
Mechanism Of Action 52
R&D Progress 52
inclisiran - Drug Profile 53
Product Description 53
Mechanism Of Action 53
R&D Progress 53
JS-002 - Drug Profile 59
Product Description 59
Mechanism Of Action 59
R&D Progress 59
K-312 - Drug Profile 60
Product Description 60
Mechanism Of Action 60
R&D Progress 60
lodelcizumab - Drug Profile 61
Product Description 61
Mechanism Of Action 61
R&D Progress 61
LY-3015014 - Drug Profile 62
Product Description 62
Mechanism Of Action 62
R&D Progress 62
Monoclonal Antibodies to Inhibit PCSK9 for Hypercholesterolemia - Drug Profile 63
Product Description 63
Mechanism Of Action 63
R&D Progress 63
Monoclonal Antibody to Inhibit PCSK9 for Hypercholesterolemia - Drug Profile 64
Product Description 64
Mechanism Of Action 64
R&D Progress 64
O-304 - Drug Profile 65
Product Description 65
Mechanism Of Action 65
R&D Progress 65
PF-06446846 - Drug Profile 67
Product Description 67
Mechanism Of Action 67
R&D Progress 67
Recombinant Protein to Inhibit PCSK9 for Hypercholesterolemia - Drug Profile 68
Product Description 68
Mechanism Of Action 68
R&D Progress 68
Small Molecule to Inhibit PCSK9 for Hypercholesterolemia - Drug Profile 70
Product Description 70
Mechanism Of Action 70
R&D Progress 70
Small Molecule to Inhibit PCSK9 for Hypercholesterolemia - Drug Profile 71
Product Description 71
Mechanism Of Action 71
R&D Progress 71
Small Molecules to Inhibit PCSK9 for Cardiovascular Disease - Drug Profile 72
Product Description 72
Mechanism Of Action 72
R&D Progress 72
Small Molecules to Inhibit PCSK9 for Hypercholesterolemia - Drug Profile 73
Product Description 73
Mechanism Of Action 73
R&D Progress 73
SX-PCK9 - Drug Profile 74
Product Description 74
Mechanism Of Action 74
R&D Progress 74
Synthetic Peptide to Inhibit PCSK9 for Hypercholesterolemia - Drug Profile 75
Product Description 75
Mechanism Of Action 75
R&D Progress 75
Vaccine to Target PCSK9 for Hypercholesterolemia - Drug Profile 76
Product Description 76
Mechanism Of Action 76
R&D Progress 76
Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) - Dormant Products 77
Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) - Discontinued Products 79
Proprotein Convertase Subtilisin/Kexin Type 9 (Proprotein Convertase 9 or Neural Apoptosis Regulated Convertase 1 or Subtilisin/Kexin Like Protease PC9 or PCSK9 or EC 3.4.21.) - Product Development Milestones 80
Featured News & Press Releases 80
Aug 28, 2017: New, One-Year Data from ORION-1 Phase II Study of Inclisiran Extends Excellent Long-Term Efficacy and Safety Profile, Affirming Dose for Phase III Trials 80
Aug 25, 2017: Regeneron and Sanofi to Present New Analyses from Praluent Injection Trials at the ESC Congress 2017 82
Jun 11, 2017: Medication That Inhibits PCSK9 Safely Reduces Cardiovascular Risk in Patients with Type 2 Diabetes 82
Jun 11, 2017: Sanofi and Regeneron Announce Positive Results from First Dedicated Studies Evaluating Praluent (alirocumab) in Individuals with Diabetes and Hypercholesterolemia 84
Apr 26, 2017: The Medicines Company and Alnylam Pharmaceuticals Announce Agreement with FDA on Phase III Clinical Program for Inclisiran 86
Apr 25, 2017: Regeneron And Sanofi Announce FDA Approval Of A New Once-Monthly Dosing Option For Praluent (Alirocumab) Injection 87
Mar 17, 2017: The Medicines Company and Alnylam Pharmaceuticals Report Positive Final Results from ORION-1 Phase II Study of Inclisiran 88
Mar 14, 2017: The Medicines Company to Webcast Presentation of ORION-1 Phase II Study of Inclisiran at ACC.17 89
Mar 07, 2017: Regeneron and Sanofi to Present New Phase 3 Praluent (alirocumab) Injection Clinical Trial Analyses at ACC.17 Scientific Sessions 89
Feb 08, 2017: Appeals Court Grants Stay of Permanent Injunction for Praluent (alirocumab) During Appeals Process 91
Jan 25, 2017: The Medicines Company to Present Results from ORION-1 Phase 2 Study of Inclisiran at the American College of Cardiology's 66th Annual Scientific Session 91
Jan 13, 2017: Sanofi and Regeneron File Notice of Appeal and Motion to Stay (Suspend) Injunction with Federal Circuit Court of Appeals in Patent Case Regarding Praluent (alirocumab) Injection 92
Jan 09, 2017: Amgen Statement on January 9, 2017, U.S. District Court Decision 92
Jan 08, 2017: The Medicines Company Announces Positive Top-Line Results from Day 180 Interim Analysis in Ongoing ORION-1 Phase 2 Study of Inclisiran 92
Jan 05, 2017: Regeneron and Sanofi to Appeal U.S. District Court Rulings in Ongoing Patent Litigation Regarding Praluent (alirocumab) Injection 93
Appendix 94
Methodology 94
Coverage 94
Secondary Research 94
Primary Research 94
Expert Panel Validation 94
Contact Us 94
Disclaimer 95

List of Tables

Number of Products under Development by Stage of Development, H2 2017 9
Number of Products under Development by Therapy Areas, H2 2017 10
Number of Products under Development by Indication, H2 2017 11
Number of Products under Development by Companies, H2 2017 12
Number of Products under Development by Companies, H2 2017 (Contd..1) 13
Products under Development by Companies, H2 2017 14
Products under Development by Companies, H2 2017 (Contd..1), H2 2017 15
Products under Development by Companies, H2 2017 (Contd..2), H2 2017 16
Number of Products under Investigation by Universities/Institutes, H2 2017 17
Products under Investigation by Universities/Institutes, H2 2017 18
Number of Products by Stage and Mechanism of Actions, H2 2017 19
Number of Products by Stage and Route of Administration, H2 2017 21
Number of Products by Stage and Molecule Type, H2 2017 23
Pipeline by AFFiRiS AG, H2 2017 24
Pipeline by Betagenon AB, H2 2017 24
Pipeline by Bioleaders Corp, H2 2017 25
Pipeline by BioLingus AG, H2 2017 25
Pipeline by Catabasis Pharmaceuticals Inc, H2 2017 26
Pipeline by Dicerna Pharmaceuticals Inc, H2 2017 26
Pipeline by Eli Lilly and Co, H2 2017 27
Pipeline by Ensemble Therapeutics Corp, H2 2017 27
Pipeline by Kowa Co Ltd, H2 2017 27
Pipeline by Novartis AG, H2 2017 28
Pipeline by Pfizer Inc, H2 2017 28
Pipeline by Regeneron Pharmaceuticals Inc, H2 2017 29
Pipeline by Serometrix LLC, H2 2017 29
Pipeline by Shanghai Fudan-Zhangjiang Bio-Pharmaceutical Co Ltd, H2 2017 30
Pipeline by The Medicines Company, H2 2017 30
Dormant Products, H2 2017 77
Dormant Products, H2 2017 (Contd..1), H2 2017 78
Discontinued Products, H2 2017 79