Paroxysmal Nocturnal Hemoglobinuria – Pipeline Review, H1 2017

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

The report provides comprehensive information on the therapeutics under development for Paroxysmal Nocturnal Hemoglobinuria, 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 Paroxysmal Nocturnal Hemoglobinuria 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 Paroxysmal Nocturnal Hemoglobinuria

– The report reviews pipeline therapeutics for Paroxysmal Nocturnal Hemoglobinuria 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 Paroxysmal Nocturnal Hemoglobinuria therapeutics and enlists all their major and minor projects

– The report assesses Paroxysmal Nocturnal Hemoglobinuria 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 Paroxysmal Nocturnal Hemoglobinuria

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 Paroxysmal Nocturnal Hemoglobinuria

- 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 Paroxysmal Nocturnal Hemoglobinuria 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

Achillion Pharmaceuticals Inc

Akari Therapeutics Plc

Alexion Pharmaceuticals Inc

Alnylam Pharmaceuticals Inc

Amgen Inc

Apellis Pharmaceuticals Inc

F. Hoffmann-La Roche Ltd

ISU ABXIS Co Ltd

Novartis AG

NovelMed Therapeutics Inc

Omeros Corp

Ra Pharmaceuticals Inc

Regeneron Pharmaceuticals Inc

Regenesance BV

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Paroxysmal Nocturnal Hemoglobinuria - Overview

Paroxysmal Nocturnal Hemoglobinuria - Therapeutics Development

Pipeline Overview

Pipeline by Companies

Products under Development by Companies

Paroxysmal Nocturnal Hemoglobinuria - Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Paroxysmal Nocturnal Hemoglobinuria - Companies Involved in Therapeutics Development

Achillion Pharmaceuticals Inc

Akari Therapeutics Plc

Alexion Pharmaceuticals Inc

Alnylam Pharmaceuticals Inc

Amgen Inc

Apellis Pharmaceuticals Inc

F. Hoffmann-La Roche Ltd

ISU ABXIS Co Ltd

Novartis AG

NovelMed Therapeutics Inc

Omeros Corp

Ra Pharmaceuticals Inc

Regeneron Pharmaceuticals Inc

Regenesance BV

Paroxysmal Nocturnal Hemoglobinuria - Drug Profiles

ACH-4471 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ALN-CC5 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ALXN-1210 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AMY-101 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

APL-2 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

APL-9 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Coversin - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

eculizumab biosimilar - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

eculizumab biosimilar - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

GNR-045 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Monoclonal Antibody for Paroxysmal Nocturnal Hemoglobinuria - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

NM-9405 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

OMS-721 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

OMS-906 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

RA-101348 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

RA-101495 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Regenemab - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

REGN-3918 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

RG-6107 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecule to Inhibit Factor D for PNH and Dense Deposit Disease - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules to Inhibit Factor D for Paroxysmal Nocturnal Hemoglobinuria and Membranoproliferative Glomerulonephritis Type II - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Synthetic Peptide to Inhibit Complement C5 for Autoimmune Disorders, Atypical Hemolytic Uremic Syndrome, Central Nervous System and Paroxysmal Nocturnal Hemoglobinuria - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

tesidolumab - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Paroxysmal Nocturnal Hemoglobinuria - Dormant Projects

Paroxysmal Nocturnal Hemoglobinuria - Discontinued Products

Paroxysmal Nocturnal Hemoglobinuria - Product Development Milestones

Featured News & Press Releases

Apr 24, 2017: Akari Therapeutics Demonstrates Positive Response with Coversin in Ongoing Phase 2 PNH Trial and in Additional Clinical Targets

Apr 06, 2017: Achillion Announces Initiation of Patient Dosing in Phase 2 Study of ACH-4471 for Paroxysmal Nocturnal Hemoglobinuria

Mar 30, 2017: Akari Therapeutics Announces FDA Fast Track Designation For Coversin

Mar 21, 2017: USPTO Grants Composition of Matter Patent to Achillion for Small Molecule Complement Alternative Pathway Factor D Inhibitors

Jan 06, 2017: FDA Grants Orphan Drug Designation To ALXN1210 For The Treatment Of Patients With Paroxysmal Nocturnal Hemoglobinuria

Jan 03, 2017: Akari Therapeutics Announces FDA Allowance of IND for Clinical Development of Coversin in PNH

Dec 20, 2016: Apellis Announces FDA Fast Track Designation for APL-2 in PNH

Dec 05, 2016: Data Presented at American Society of Hematology Meeting Demonstrate Potential Advantages of Factor D Inhibition for the Treatment of Complement Alternative Pathway-Mediated Diseases

Dec 05, 2016: Alnylam Presents Updated Results from Phase 1/2 Study of ALN-CC5 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Dec 05, 2016: Akari Therapeutics Announces Corporate Update with New Positive Clinical Data and a New Pipeline of Tick Derived and Engineered Proteins

Dec 04, 2016: New Data From ALXN1210 Dose-Escalation Study Presented At ASH Show Rapid And Sustained Reductions In LDH In Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Dec 02, 2016: Apellis Reports Positive Interim Results from Phase 1b Clinical Trials of APL-2 in PNH

Nov 30, 2016: Ra Pharmaceuticals to Present Overview of RA101495 and Oral C5 Inhibitor at the International PNH Interest Group Annual Scientific Assembly

Nov 21, 2016: Apellis to Present Safety and Efficacy Data from Clinical Trials of APL-2 in PNH at December Scientific Meetings

Nov 17, 2016: Ra Pharmaceuticals Receives Orphan Drug Designation in the European Union for RA101495 for Treatment of Paroxysmal Nocturnal Hemoglobinuria

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, H1 2017

Number of Products under Development by Companies, H1 2017

Products under Development by Companies, H1 2017

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

Number of Products by Stage and Target, H1 2017

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

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

Number of Products by Stage and Molecule Type, H1 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Achillion Pharmaceuticals Inc, H1 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Akari Therapeutics Plc, H1 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Alexion Pharmaceuticals Inc, H1 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Alnylam Pharmaceuticals Inc, H1 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Amgen Inc, H1 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Apellis Pharmaceuticals Inc, H1 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by F. Hoffmann-La Roche Ltd, H1 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by ISU ABXIS Co Ltd, H1 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Novartis AG, H1 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by NovelMed Therapeutics Inc, H1 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Omeros Corp, H1 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Ra Pharmaceuticals Inc, H1 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Regeneron Pharmaceuticals Inc, H1 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Regenesance BV, H1 2017

Paroxysmal Nocturnal Hemoglobinuria – Dormant Projects, H1 2017

Paroxysmal Nocturnal Hemoglobinuria – Discontinued Products, H1 2017

List of Figures

List of Figures

Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products by Targets, H1 2017

Number of Products by Stage and Targets, H1 2017

Number of Products by Mechanism of Actions, H1 2017

Number of Products by Stage and Mechanism of Actions, H1 2017

Number of Products by Routes of Administration, H1 2017

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

Number of Products by Molecule Types, H1 2017

Number of Products by Stage and Molecule Types, H1 2017

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