The document provides an overview of the global therapeutic pipeline for Paroxysmal Nocturnal Hemoglobinuria (PNH) as of the first half of 2015. It details over 20 drugs in development for PNH across multiple companies and stages of development. The pipeline is evaluated based on drug targets, mechanisms of action, routes of administration, molecule types, and comparative analyses. Recent clinical trial results and developments are also summarized.

1. Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H1 2015
Global Markets Directs, Paroxysmal Nocturnal Hemoglobinuria Pipeline Review, H1 2015, provides an overview of the Paroxysmal Nocturnal Hemoglobinurias
therapeutic pipeline.
This report provides comprehensive information on the therapeutic development for Paroxysmal Nocturnal Hemoglobinuria, complete with comparative analysis
at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest
updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Paroxysmal Nocturnal Hemoglobinuria and
special features on late-stage and discontinued projects.
Global Markets Directs 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 Directs proprietary databases, Company/University websites, SEC filings, investor presentations and
featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Directs team. Drug
profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the
latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that
the most recent developments are captured on a real time basis.
The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by
identifying new targets and MOAs to produce first-in-class and best-in-class products.
Scope
The report provides a snapshot of the global therapeutic landscape of Paroxysmal Nocturnal Hemoglobinuria The report reviews key pipeline products under
drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities The report
reviews key players involved in the therapeutics development for Paroxysmal Nocturnal Hemoglobinuria and enlists all their major and minor projects The report
summarizes all the dormant and discontinued pipeline projects A review of the Paroxysmal Nocturnal Hemoglobinuria products under development by
companies and universities/research institutes based on information derived from company and industry-specific sources Pipeline products coverage based on
various stages of development ranging from pre-registration till discovery and undisclosed stages A detailed assessment of monotherapy and combination therapy
pipeline projects Coverage of the Paroxysmal Nocturnal Hemoglobinuria pipeline on the basis of target, MoA, route of administration and molecule type Latest
news and deals relating related to pipeline products
Reasons to buy
Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies Identify emerging players
with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage Develop strategic initiatives by understanding the
focus areas of leading companies Identify and understand important and diverse types of therapeutics under development for Paroxysmal Nocturnal
Hemoglobinuria Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline 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
table Of Contents
table Of Contents 2
list Of Tables 4
list Of Figures 4
introduction 5
global Markets Direct Report Coverage 5
paroxysmal Nocturnal Hemoglobinuria Overview 6
therapeutics Development 7
pipeline Products For Paroxysmal Nocturnal Hemoglobinuria - Overview 7
pipeline Products For Paroxysmal Nocturnal Hemoglobinuria - Comparative Analysis 8
paroxysmal Nocturnal Hemoglobinuria - Therapeutics Under Development By Companies 9
paroxysmal Nocturnal Hemoglobinuria - Pipeline Products Glance 10
clinical Stage Products 10
early Stage Products 11
paroxysmal Nocturnal Hemoglobinuria - Products Under Development By Companies 12
paroxysmal Nocturnal Hemoglobinuria - Companies Involved In Therapeutics Development 13
achillion Pharmaceuticals, Inc. 13
alnylam Pharmaceuticals, Inc. 14
amyndas Pharmaceuticals Llc 15
apellis Pharmaceuticals, Inc. 16
omeros Corporation 17
ra Pharmaceuticals, Inc. 18
shire Plc 19
paroxysmal Nocturnal Hemoglobinuria - Therapeutics Assessment 20
assessment By Monotherapy Products 20
assessment By Target 21
assessment By Mechanism Of Action 23
assessment By Route Of Administration 25
Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H1 2015

2. assessment By Molecule Type 27
drug Profiles 29
ach-cfdis - Drug Profile 29
product Description 29
mechanism Of Action 29
r&d Progress 29
aln-cc5 - Drug Profile 30
product Description 30
mechanism Of Action 30
r&d Progress 30
amy-101 - Drug Profile 31
product Description 31
mechanism Of Action 31
r&d Progress 31
apl-2 - Drug Profile 32
product Description 32
mechanism Of Action 32
r&d Progress 32
c1 Esterase Inhibitor (human) - Drug Profile 34
product Description 34
mechanism Of Action 34
r&d Progress 34
coversin - Drug Profile 36
product Description 36
mechanism Of Action 36
r&d Progress 36
monoclonal Antibody For Paroxysmal Nocturnal Hemoglobinuria - Drug Profile 37
product Description 37
mechanism Of Action 37
r&d Progress 37
nm-9405 - Drug Profile 38
product Description 38
mechanism Of Action 38
r&d Progress 38
oms-721 - Drug Profile 39
product Description 39
mechanism Of Action 39
r&d Progress 39
ra-101348 - Drug Profile 41
product Description 41
mechanism Of Action 41
r&d Progress 41
paroxysmal Nocturnal Hemoglobinuria - Recent Pipeline Updates 42
paroxysmal Nocturnal Hemoglobinuria - Dormant Projects 51
paroxysmal Nocturnal Hemoglobinuria - Product Development Milestones 52
featured News & Press Releases 52
dec 04, 2014: Ra Pharmaceuticals Announces Presentation Of Data Supporting Clinical Development Of Novel Complement C5 Inhibitor At 2014 Ash Annual
Meeting 52
mar 28, 2013: Omeros Announces Toxicology Data That Support Advancing Masp-2 Inhibitor Into Clinical Trials 53
feb 07, 2013: Omeros Announces Toxicology Study Data From Masp-2 Inhibitor 54
appendix 56
methodology 56
coverage 56
secondary Research 56
primary Research 56
expert Panel Validation 56
contact Us 56
disclaimer 57
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Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H1 2015