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Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H2 2018

Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H2 2018

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H2 2018, 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, H2 2018, 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 Pre-Registration, Phase III, Phase II, Phase I, IND/CTA Filed, Preclinical and Discovery stages are 1, 3, 6, 4, 1, 8 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.


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,
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
Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, H2 2018
Number of Products under Development by Companies, H2 2018
Products under Development by Companies, H2 2018
Products under Development by Companies, H2 2018 (Contd..1), H2 2018
Number of Products by Stage and Target, H2 2018
Number of Products by Stage and Mechanism of Action, H2 2018
Number of Products by Stage and Route of Administration, H2 2018
Number of Products by Stage and Molecule Type, H2 2018
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Achillion Pharmaceuticals Inc, H2 2018
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Akari Therapeutics Plc, H2 2018
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Alexion Pharmaceuticals Inc, H2 2018
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Alnylam Pharmaceuticals Inc, H2 2018
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Amgen Inc, H2 2018
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Apellis Pharmaceuticals Inc, H2 2018
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Chugai Pharmaceutical Co Ltd, H2 2018
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by ISU ABXIS Co Ltd, H2 2018
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Novartis AG, H2 2018
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by NovelMed Therapeutics Inc, H2 2018
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Omeros Corp, H2 2018
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Ra Pharmaceuticals Inc, H2 2018
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Regeneron Pharmaceuticals Inc, H2 2018
Paroxysmal Nocturnal Hemoglobinuria - Dormant Projects, H2 2018
Paroxysmal Nocturnal Hemoglobinuria - Discontinued Products, H2 2018
List of Figures
Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, H2 2018
Number of Products under Development by Companies, H2 2018
Number of Products by Targets, H2 2018
Number of Products by Stage and Targets, H2 2018
Number of Products by Mechanism of Actions, H2 2018
Number of Products by Stage and Mechanism of Actions, H2 2018
Number of Products by Routes of Administration, H2 2018
Number of Products by Stage and Routes of Administration, H2 2018
Number of Products by Molecule Types, H2 2018
Number of Products by Stage and Molecule Types, H2 2018

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