Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H1 2017

Published: Apr, 2017 | Pages: 90 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H1 2017, provides an overview of the Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders) pipeline landscape.

Paroxysmal nocturnal hemoglobinuria is a rare disease in which red blood cells break down earlier than normal. Symptoms include abdominal pain, back pain, dark urine, easy bruising or bleeding, headache and shortness of breath. The predisposing factors include age, obesity and hormone therapy. Treatment includes surgery, chemotherapy and radiation therapy.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Paroxysmal Nocturnal Hemoglobinuria and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 6, 2, 13 and 1 respectively.

Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders).
- The pipeline guide reviews pipeline therapeutics for Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders)

Reasons To Buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders) 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

 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

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