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
[Report Updated: 10-10-2017]

Introduction

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
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.) - Pipeline Review, H2 2017 [Report Updated: 10-10-2017]

• 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

Additional Details

Publisher

Global Markets Direct

Publisher Information

Reference

139648 | GMDHC1038TDB

Number of Pages

95

Report Format

PDF

Related Reports