Porphyria (Erythropoietic Protoporphyri) - Pipeline Review, H1 2016

Published: May, 2016 | Pages: 63 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Porphyria (Erythropoietic Protoporphyri) - Pipeline Review, H1 2016

Summary

Global Markets Direct's, 'Porphyria (Erythropoietic Protoporphyri) - Pipeline Review, H1 2016', provides an overview of the Porphyria (Erythropoietic Protoporphyri) pipeline landscape. 

The report provides comprehensive information on the therapeutics under development for Porphyria (Erythropoietic Protoporphyri), 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 Porphyria (Erythropoietic Protoporphyri) 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 Porphyria (Erythropoietic Protoporphyri)
- The report reviews pipeline therapeutics for Porphyria (Erythropoietic Protoporphyri) 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 Porphyria (Erythropoietic Protoporphyri) therapeutics and enlists all their major and minor projects
- The report assesses Porphyria (Erythropoietic Protoporphyri) 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 Porphyria (Erythropoietic Protoporphyri)

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 Porphyria (Erythropoietic Protoporphyri)
- 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 Porphyria (Erythropoietic Protoporphyri) 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

 Table of Contents

Table of Contents 2
List of Tables 4
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Porphyria (Erythropoietic Protoporphyri) Overview 7
Therapeutics Development 8
Pipeline Products for Porphyria (Erythropoietic Protoporphyri) - Overview 8
Pipeline Products for Porphyria (Erythropoietic Protoporphyri) - Comparative Analysis 9
Porphyria (Erythropoietic Protoporphyri) - Therapeutics under Development by Companies 10
Porphyria (Erythropoietic Protoporphyri) - Therapeutics under Investigation by Universities/Institutes 11
Porphyria (Erythropoietic Protoporphyri) - Pipeline Products Glance 12
Late Stage Products 12
Clinical Stage Products 13
Early Stage Products 14
Porphyria (Erythropoietic Protoporphyri) - Products under Development by Companies 15
Porphyria (Erythropoietic Protoporphyri) - Products under Investigation by Universities/Institutes 16
Porphyria (Erythropoietic Protoporphyri) - Companies Involved in Therapeutics Development 17
Alnylam Pharmaceuticals, Inc. 17
Clinuvel Pharmaceuticals Limited 18
Digna Biotech, S.L. 19
UniQure N.V. 20
Porphyria (Erythropoietic Protoporphyri) - Therapeutics Assessment 21
Assessment by Monotherapy Products 21
Assessment by Target 22
Assessment by Mechanism of Action 24
Assessment by Route of Administration 26
Assessment by Molecule Type 28
Drug Profiles 30
afamelanotide - Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
ALN-AS1 - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
AMT-021 - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
DB-027 - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
Enzyme Replacement Therapy for Acute Intermittent Porphyria - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
Porphyria (Erythropoietic Protoporphyri) - Recent Pipeline Updates 40
Porphyria (Erythropoietic Protoporphyri) - Discontinued Products 48
Porphyria (Erythropoietic Protoporphyri) - Product Development Milestones 49
Featured News & Press Releases 49
Mar 24, 2016: NICE's technical team invited stakeholders with an interest in the treatment of erythropoietic protoporphyria 49
Feb 16, 2016: Clinuvel brief on European commercialisation 49
Feb 12, 2016: SCENESSE receives US FDA orphan designation for cutaneous porphyrias 50
Sep 15, 2015: Alnylam Reports Positive Initial Clinical Results for ALN-AS1, an Investigational RNAi Therapeutic Targeting Aminolevulinic Acid Synthase 1 for the Treatment of Acute Hepatic Porphyrias 51
Aug 27, 2015: FDA grants Clinuvel a Type C Meeting 54
Jul 02, 2015: New England Journal of Medicine publishes pivotal Phase III SCENESSE studies 55
Jul 01, 2015: Treatment Reduces Symptoms in Syndrome that Causes Extreme Light Sensitivity 56
Jul 01, 2015: Treatment Reduces Symptoms in Syndrome that Causes Extreme Light Sensitivity 57
Jun 05, 2015: SCENESSE Commercialisation Update 58
May 26, 2015: Alnylam Initiates Phase 1 Clinical Trial for ALN-AS1, an Investigational RNAi Therapeutic Targeting Aminolevulinic Acid Synthase 1 (ALAS1) for the Treatment of Acute Hepatic Porphyrias, Including Acute Intermittent Porphyria (AIP) 59
Appendix 62
Methodology 62
Coverage 62
Secondary Research 62
Primary Research 62
Expert Panel Validation 62
Contact Us 62
Disclaimer 63

List of Tables

Number of Products under Development for Porphyria (Erythropoietic Protoporphyri), H1 2016 8
Number of Products under Development for Porphyria (Erythropoietic Protoporphyri) - Comparative Analysis, H1 2016 9
Number of Products under Development by Companies, H1 2016 10
Number of Products under Investigation by Universities/Institutes, H1 2016 11
Comparative Analysis by Late Stage Development, H1 2016 12
Comparative Analysis by Clinical Stage Development, H1 2016 13
Comparative Analysis by Early Stage Development, H1 2016 14
Products under Development by Companies, H1 2016 15
Products under Investigation by Universities/Institutes, H1 2016 16
Porphyria (Erythropoietic Protoporphyri) - Pipeline by Alnylam Pharmaceuticals, Inc., H1 2016 17
Porphyria (Erythropoietic Protoporphyri) - Pipeline by Clinuvel Pharmaceuticals Limited, H1 2016 18
Porphyria (Erythropoietic Protoporphyri) - Pipeline by Digna Biotech, S.L., H1 2016 19
Porphyria (Erythropoietic Protoporphyri) - Pipeline by UniQure N.V., H1 2016 20
Assessment by Monotherapy Products, H1 2016 21
Number of Products by Stage and Target, H1 2016 23
Number of Products by Stage and Mechanism of Action, H1 2016 25
Number of Products by Stage and Route of Administration, H1 2016 27
Number of Products by Stage and Molecule Type, H1 2016 29
Porphyria (Erythropoietic Protoporphyri) Therapeutics - Recent Pipeline Updates, H1 2016 40
Porphyria (Erythropoietic Protoporphyri) - Discontinued Products, H1 2016 48