Cystic Fibrosis - Pipeline Review - H1, 2015

Published: January 2015

Global Markets Direct’s, ‘Cystic Fibrosis - Pipeline Review, H1 2015’, provides an overview of the Cystic Fibrosis’s therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Cystic Fibrosis, 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 Cystic Fibrosis and special features on late-stage 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct’s 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.

Note: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease. 



  • The report provides a snapshot of the global therapeutic landscape of Cystic Fibrosis
  • 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 Cystic Fibrosis and enlists all their major and minor projects
  • The report summarizes all the dormant and discontinued pipeline projects
  • A review of the Cystic Fibrosis 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 Cystic Fibrosis 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 Cystic Fibrosis
  • Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
  • Devise corrective measures for pipeline projects by understanding Cystic Fibrosis 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 7
List of Figures 9
Introduction 10
Global Markets Direct Report Coverage 10
Cystic Fibrosis Overview 11
Therapeutics Development 12
Pipeline Products for Cystic Fibrosis - Overview 12
Pipeline Products for Cystic Fibrosis - Comparative Analysis 13
Cystic Fibrosis - Therapeutics under Development by Companies 14
Cystic Fibrosis - Therapeutics under Investigation by Universities/Institutes 20
Cystic Fibrosis - Pipeline Products Glance 22
Late Stage Products 22
Clinical Stage Products 23
Early Stage Products 24
Unknown Stage Products 25
Cystic Fibrosis - Products under Development by Companies 26
Cystic Fibrosis - Products under Investigation by Universities/Institutes 32
Cystic Fibrosis - Companies Involved in Therapeutics Development 33
Actelion Ltd 33
Alaxia SAS 34
AlgiPharma AS 35
Arcturus Therapeutics, Inc 36
Bayer AG 37
Beech Tree Labs, Inc. 38
Carolus Therapeutics, Inc. 39
Celsus Therapeutics Plc 40
Celtaxsys, Inc. 41
Chiesi Farmaceutici SpA 42
Cilian AG 43
Concert Pharmaceuticals, Inc. 44
Corbus pharmaceuticals, Inc. 45
Cubist Pharmaceuticals, Inc. 46
DiscoveryBiomed, Inc. 47
Errant Gene Therapeutics, LLC 48
Galapagos NV 49
GlaxoSmithKline plc 50
Grifols, S.A. 51
Invion Limited 52
InvivoGen Therapeutics 53
JHL Biotech, Inc. 54
Kamada Ltd. 55
Lamellar Biomedical Ltd 56
Laurent Pharmaceuticals Inc. 57
Microbion Corporation 58
Mucokinetica Ltd. 59
N30 Pharmaceuticals 60
NanoBio Corporation 61
Nostrum Pharmaceuticals, LLC 62
Novabiotics Ltd 63
Novartis AG 64
Ockham Biotech Limited 65
OSE Pharma SA 66
Parion Sciences, Inc. 67
Pfizer Inc. 68
Pharmaxis Limited 69
Polyphor Ltd. 70
Progenra, Inc. 71
ProQR Therapeutics N.V. 72
Protalix BioTherapeutics, Inc. 73
PTC Therapeutics, Inc. 74
Pulmatrix, Inc. 75
Reata Pharmaceuticals, Inc. 76
ReveraGen BioPharma, Inc. 77
Serendex Pharmaceuticals A/S 78
Shire Plc 79
Synovo GmbH 80
Verona Pharma Plc 81
Vertex Pharmaceuticals Incorporated 82
Xenetic Biosciences plc 83
Cystic Fibrosis - Therapeutics Assessment 84
Assessment by Monotherapy Products 84
Assessment by Combination Products 85
Assessment by Target 86
Assessment by Mechanism of Action 89
Assessment by Route of Administration 92
Assessment by Molecule Type 94
Drug Profiles 96
(fosfomycin + tobramycin) - Drug Profile 96
alpha-1 proteinase inhibitor (human) second generation - Drug Profile 97
ALX-009 - Drug Profile 100
Anti-Microbial Drug for Cystic Fibrosis - Drug Profile 101
ataluren - Drug Profile 102
BTL-na - Drug Profile 105
CFX-1 - Drug Profile 106
CSY-0073 - Drug Profile 107
CT-2009 - Drug Profile 108
CTX-4430 - Drug Profile 109
cysteamine - Drug Profile 110
D-Ivacaftor - Drug Profile 112
dapsone - Drug Profile 113
Deoxyribonuclease II to Inhibit DNA Synthesis for Cystic Fibrosis - Drug Profile 114
dornase alfa biosimilar - Drug Profile 115
Drug for Cystic Fibrosis - Drug Profile 116
Drug to Activate Kir 4.2 for Cystic Fibrosis - Drug Profile 117
Drugs for COPD and Cystic Fibrosis - Drug Profile 118
Drugs for Cystic Fibrosis Lung Infections - Drug Profile 119
EBX-001 - Drug Profile 120
FDL-169 - Drug Profile 121
fenretinide - Drug Profile 122
fosfomycin - Drug Profile 123
Gene Therapy to Activate CFTR for Cystic Fibrosis - Drug Profile 124
Gene Therapy to Activate CFTR for Cystic Fibrosis - Drug Profile 125
GLPG-1837 - Drug Profile 126
GSK-2225745 - Drug Profile 128
Heparin - Drug Profile 129
JBT-101 - Drug Profile 130
levofloxacin - Drug Profile 131
LMS-611 - Drug Profile 132
lumacaftor - Drug Profile 133
LUNAR-CF - Drug Profile 136
mannitol - Drug Profile 137
miglustat - Drug Profile 140
MKA-104 - Drug Profile 142
molgramostim - Drug Profile 143
N-1785 - Drug Profile 145
N-1861 - Drug Profile 146
N-6022 - Drug Profile 147
N-91115 - Drug Profile 148
N-91138 - Drug Profile 149
N-91169 - Drug Profile 150
nadolol - Drug Profile 151
NB-401 - Drug Profile 153
Next Generation Correctors - Drug Profile 154
nitric oxide - Drug Profile 155
nitric oxide - Drug Profile 156
Oligomer G for Cystic Fibrosis - Drug Profile 157
Oligonucleotide to Activate CFTR for Cystic Fibrosis - Drug Profile 159
ORP-100 - Drug Profile 160
P-1037 - Drug Profile 161
Peptide to Block ENaC for Cystic Fibrosis and Hypertension - Drug Profile 162
POL-6014 - Drug Profile 163
PRX-110 - Drug Profile 165
pseudomonas aeruginosa vaccine - Drug Profile 166
PulmoXen - Drug Profile 167
PUR-118 - Drug Profile 168
PUR-1900 - Drug Profile 169
PUR-2000 - Drug Profile 170
PXS-4728A - Drug Profile 171
QBW-251 - Drug Profile 172
QR-010 - Drug Profile 173
Recombinant A1PI - Drug Profile 174
Recombinant Enzyme for Cystic Fibrosis - Drug Profile 176
Recombinant Enzyme for Farber Disease and Cystic Fibrosis - Drug Profile 177
Recombinant Human Follistatin - Drug Profile 178
Recombinant Protein for Infectious Diseases and Respiratory Disorders - Drug Profile 179
riociguat - Drug Profile 180
RPL-554 - Drug Profile 182
Small Molecule for Cystic Fibrosis - Drug Profile 185
Small Molecule for Pneumonia, COPD, Cystic Fibrosis and Pulmonary Tuberculosis - Drug Profile 186
Small Molecule to Activate CFTR Protein for Cystic Fibrosis - Drug Profile 187
Small Molecule to Activate Nrf2 for Cystic Fibrosis - Drug Profile 188
Small Molecule to Inhibit EnaC and CFTR for Cystic Fibrosis, Diarrhea and Autosomal Dominant Polycystic Kidney Disease - Drug Profile 189
Small Molecule to Inhibit HDAC for Cystic Fibrosis and COPD - Drug Profile 190
Small Molecules for Bacterial Infections, Oncology and Cystic Fibrosis - Drug Profile 191
Small Molecules for Cystic Fibrosis - Drug Profile 192
Small Molecules to Activate CFTR for Cystic Fibrosis - Drug Profile 193
Small Molecules to Activate CFTR for Cystic Fibrosis - Drug Profile 194
Small Molecules to Agonize PPAR-Gamma for COPD and Cystic Fibrosis - Drug Profile 195
Small Molecules to Inhibit Lethal Factor for Anthrax and Cystic Fibrosis - Drug Profile 196
Small Molecules to Inhibit USP8 for Cystic Fibrosis - Drug Profile 197
solithromycin - Drug Profile 198
SP-14 - Drug Profile 200
SP-9 - Drug Profile 201
tritoqualine - Drug Profile 202
VBP-15 - Drug Profile 203
VRT-325 - Drug Profile 205
VX-661 - Drug Profile 206
Cystic Fibrosis - Recent Pipeline Updates 207
Cystic Fibrosis - Dormant Projects 246
Cystic Fibrosis - Discontinued Products 252
Cystic Fibrosis - Product Development Milestones 253
Featured News & Press Releases 253
Appendix 260
Methodology 260
Coverage 260
Secondary Research 260
Primary Research 260
Expert Panel Validation 260
Contact Us 260
Disclaimer 261