Paroxysmal Nocturnal Hemoglobinuria – Pipeline Review, H1 2018

Global Markets Direct’s, ‘Paroxysmal Nocturnal Hemoglobinuria – Pipeline Review, H1 2018’, 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

Chugai Pharmaceutical Co Ltd

ISU ABXIS Co Ltd

Novartis AG

NovelMed Therapeutics Inc

Omeros Corp

Ra Pharmaceuticals Inc

Regeneron Pharmaceuticals Inc

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

Chugai Pharmaceutical Co Ltd

ISU ABXIS Co Ltd

Novartis AG

NovelMed Therapeutics Inc

Omeros Corp

Ra Pharmaceuticals Inc

Regeneron Pharmaceuticals Inc

Paroxysmal Nocturnal Hemoglobinuria Drug Profiles

ACH-4471 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ACH-5228 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ACH-5548 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

cemdisiran 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

Gene Therapy for Atypical Hemolytic Uremic Syndrome and Paroxysmal Nocturnal Hemoglobinuria Drug Profile

Product Description

Mechanism Of Action

R&D Progress

GNR-045 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

LNP-023 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-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

ravulizumab Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ravulizumab next generation 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 Molecules for Paroxysmal Nocturnal Hemoglobinuria 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

Feb 12, 2018: Ra Pharmaceuticals Announces Completion of Dosing in Phase 2 Program of RA101495 SC in Paroxysmal Nocturnal Hemoglobinuria

Feb 06, 2018: Akari Therapeutics Announces Completion of Phase II COBALT Trial of Coversin in Patients with PNH and Further Progress of Clinical Trials

Jan 08, 2018: Ra Pharmaceuticals Initiates Dosing in Phase 1b Pharmacokinetic Study Evaluating RA101495 SC in Patients with Renal Impairment

Dec 21, 2017: Ra Pharmaceuticals Initiates Dosing in Phase 2 Clinical Trial Evaluating RA101495 SC in Generalized Myasthenia Gravis Patients

Dec 20, 2017: Achillion Initiates Phase I First-In-Human Study of ACH-5228, a Next-Generation Oral Small Molecule Inhibitor of Complement Factor D

Dec 11, 2017: Results Of Phase 1b/2 Dose Regimen Optimization Studies For ALXN1210 In Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Presented At American Society Of Hematology (ASH) Meeting

Dec 08, 2017: Akari Therapeutics Announces Phase II COBALT trial of Coversin in Patients with PNH Met the Primary Endpoint

Dec 04, 2017: Ra Pharmaceuticals Announces Positive Interim Results from Phase 2 Study of RA101495 SC in Paroxysmal Nocturnal Hemoglobinuria

Nov 06, 2017: Achillion Announces ACH-4471 Granted Orphan Drug Designation by the FDA and Positive Opinion for Orphan Drug Designation in the European Union for the Treatment of Paroxysmal Nocturnal Hemoglobinuria

Nov 01, 2017: Achillion Provides Update on ACH-4471

Nov 01, 2017: Achillion Provides Update on ACH-5228

Nov 01, 2017: New Phase 1/2 Study Data for ALXN1210 as a Potential Future Treatment for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) to Be Presented at American Society of Hematology (ASH) Meeting

Oct 24, 2017: USPTO Grants Composition of Matter Patent to Achillion for ACH-4471, First-in-Class Small Molecule Complement Alternative Pathway Factor D Inhibitor

Oct 11, 2017: Akari Therapeutics Announces Further Clinical Progress

Sep 21, 2017: Akari Therapeutics Announces Regulatory Progress Following FDA Meeting

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 2018

Number of Products under Development by Companies, H1 2018

Products under Development by Companies, H1 2018

Products under Development by Companies, H1 2018 (Contd..1), H1 2018

Number of Products by Stage and Target, H1 2018

Number of Products by Stage and Mechanism of Action, H1 2018

Number of Products by Stage and Route of Administration, H1 2018

Number of Products by Stage and Molecule Type, H1 2018

Paroxysmal Nocturnal Hemoglobinuria Pipeline by Achillion Pharmaceuticals Inc, H1 2018

Paroxysmal Nocturnal Hemoglobinuria Pipeline by Akari Therapeutics Plc, H1 2018

Paroxysmal Nocturnal Hemoglobinuria Pipeline by Alexion Pharmaceuticals Inc, H1 2018

Paroxysmal Nocturnal Hemoglobinuria Pipeline by Alnylam Pharmaceuticals Inc, H1 2018

Paroxysmal Nocturnal Hemoglobinuria Pipeline by Amgen Inc, H1 2018

Paroxysmal Nocturnal Hemoglobinuria Pipeline by Apellis Pharmaceuticals Inc, H1 2018

Paroxysmal Nocturnal Hemoglobinuria Pipeline by Chugai Pharmaceutical Co Ltd, H1 2018

Paroxysmal Nocturnal Hemoglobinuria Pipeline by ISU ABXIS Co Ltd, H1 2018

Paroxysmal Nocturnal Hemoglobinuria Pipeline by Novartis AG, H1 2018

Paroxysmal Nocturnal Hemoglobinuria Pipeline by NovelMed Therapeutics Inc, H1 2018

Paroxysmal Nocturnal Hemoglobinuria Pipeline by Omeros Corp, H1 2018

Paroxysmal Nocturnal Hemoglobinuria Pipeline by Ra Pharmaceuticals Inc, H1 2018

Paroxysmal Nocturnal Hemoglobinuria Pipeline by Regeneron Pharmaceuticals Inc, H1 2018

Paroxysmal Nocturnal Hemoglobinuria Dormant Projects, H1 2018

Paroxysmal Nocturnal Hemoglobinuria Discontinued Products, H1 2018

List of Figures

List of Figures

Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products by Targets, H1 2018

Number of Products by Stage and Targets, H1 2018

Number of Products by Mechanism of Actions, H1 2018

Number of Products by Stage and Mechanism of Actions, H1 2018

Number of Products by Routes of Administration, H1 2018

Number of Products by Stage and Routes of Administration, H1 2018

Number of Products by Molecule Types, H1 2018

Number of Products by Stage and Molecule Types, H1 2018

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