Paroxysmal Nocturnal Hemoglobinuria – Pipeline Review, H2 2019

“Global Markets Direct’s, ‘Paroxysmal Nocturnal Hemoglobinuria – Pipeline Review, H2 2019’, 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

Aevitas Therapeutics Inc

Akari Therapeutics Plc

Alexion Pharmaceuticals Inc

Alnylam Pharmaceuticals Inc

Amgen Inc

Amyndas Pharmaceuticals LLC

Apellis Pharmaceuticals Inc

Attune Pharmaceuticals

Biocad

BioCryst Pharmaceuticals Inc

Chugai Pharmaceutical Co Ltd

ISU ABXIS Co Ltd

Novartis AG

NovelMed Therapeutics Inc

Omeros Corp

Ra Pharmaceuticals Inc

Regeneron Pharmaceuticals Inc

Samsung Bioepis Co Ltd

Turgut Ilaclari AS”

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

cemdisiran 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 Age-Related Macular Degeneration, 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

nomacopan 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 Molecule 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

Small Molecules for 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

Sep 05, 2018: Ra Pharma Announces Completion of End-of-Phase 2 Interactions with FDA and Design of Phase 3 PNH Program

Sep 04, 2018: Apellis pharmaceuticals announces that all four severely anemic Soliris-treated patients with paroxysmal nocturnal hemoglobinuria in ongoing phase 1b trial have been transitioned to APL-2 monotherapy

Aug 20, 2018: FDA accepts Priority Review of ALXN1210 as a treatment for patients with paroxysmal nocturnal hemoglobinuria in the US

Aug 08, 2018: Ra Pharmaceuticals announces early completion of enrollment in gMG Phase 2 program

Jul 10, 2018: Achillion Initiates Phase I First-in-Human Study of ACH-5548, a Third Oral Small Molecule Inhibitor of Complement Factor D

Jun 28, 2018: Alexion Submits Application For Approval Of ALXN1210 as a Treatment For Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) In The European Union (EU)

Jun 26, 2018: Apellis Pharmaceuticals Announces First Patient Enrolled in Phase 3 APL-2 Head-to-Head Study in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Soliris

Jun 26, 2018: Apellis Pharmaceuticals Provides Update on Two Phase Ib Trials for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Jun 19, 2018: Apellis Pharmaceuticals to Host R&D Day on June 26, 2018

Jun 19, 2018: Alexion Submits Application for Priority Review and Approval of ALXN1210 as a Treatment for Patients with Paroxysmal Nocturnal Hemoglobinuria in the U.S.

Jun 15, 2018: Akari Therapeutics to Present Poster at the 23rd Congress of the European Hematology Association

Jun 14, 2018: Alexion Announces Selection Of Positive Phase 3 Clinical Data For ALXN1210 In Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) For Late-Breaking Oral Presentation At Annual Conference Of The European Hematology Association (EHA)

May 17, 2018: Ra Pharmaceuticals to Present at the 23rd Congress of the European Hematology Association

Apr 16, 2018: Apellis Pharmaceuticals Provides Update on Phase 1b Open Label Study of APL-2 in PNH Patients Not Previously Treated with Eculizumab

Mar 15, 2018: Alexion Announces Positive Top-Line Results Showing Successful Phase 3 Clinical Study Of ALXN1210 In Complement Inhibitor Treatment-Naïve Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

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, H2 2019

Number of Products under Development by Companies, H2 2019

Number of Products under Development by Companies, H2 2019 (Contd..1), H2 2019

Products under Development by Companies, H2 2019

Products under Development by Companies, H2 2019 (Contd..1), H2 2019

Number of Products by Stage and Target, H2 2019

Number of Products by Stage and Mechanism of Action, H2 2019

Number of Products by Stage and Route of Administration, H2 2019

Number of Products by Stage and Molecule Type, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Achillion Pharmaceuticals Inc, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Aevitas Therapeutics Inc, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Akari Therapeutics Plc, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Alexion Pharmaceuticals Inc, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Alnylam Pharmaceuticals Inc, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Amgen Inc, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Amyndas Pharmaceuticals LLC, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Apellis Pharmaceuticals Inc, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Attune Pharmaceuticals, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Biocad, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by BioCryst Pharmaceuticals Inc, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Chugai Pharmaceutical Co Ltd, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by ISU ABXIS Co Ltd, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Novartis AG, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by NovelMed Therapeutics Inc, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Omeros Corp, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Ra Pharmaceuticals Inc, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Regeneron Pharmaceuticals Inc, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Samsung Bioepis Co Ltd, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Turgut Ilaclari AS, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Dormant Projects, H2 2019

Paroxysmal Nocturnal Hemoglobinuria – Discontinued Products, H2 2019”

List of Figures

“List of Figures

Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, H2 2019

Number of Products under Development by Companies, H2 2019

Number of Products by Targets, H2 2019

Number of Products by Stage and Targets, H2 2019

Number of Products by Mechanism of Actions, H2 2019

Number of Products by Stage and Mechanism of Actions, H2 2019

Number of Products by Routes of Administration, H2 2019

Number of Products by Stage and Routes of Administration, H2 2019

Number of Products by Molecule Types, H2 2019

Number of Products by Stage and Molecule Types, H2 2019”

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