Cystic Fibrosis - Pipeline Review, H2 2015

Published: December 2015
No. of Pages: 368
   

Global Markets Direct’s, ‘Cystic Fibrosis - Pipeline Review, H2 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.

Scope

  • 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

Cystic Fibrosis - Pipeline Review, H2 2015

Table of Contents

Table of Contents 2
List of Tables 8
List of Figures 10
Introduction 11
Global Markets Direct Report Coverage 11
Cystic Fibrosis Overview 12
Therapeutics Development 13
Pipeline Products for Cystic Fibrosis - Overview 13
Pipeline Products for Cystic Fibrosis - Comparative Analysis 14
Cystic Fibrosis - Therapeutics under Development by Companies 15
Cystic Fibrosis - Therapeutics under Investigation by Universities/Institutes 21
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 33
Cystic Fibrosis - Companies Involved in Therapeutics Development 34
Actelion Ltd 34
Akari Therapeutics, Plc 35
Alaxia SAS 36
AlgiPharma AS 37
AmpliPhi Biosciences Corporation 38
Arcturus Therapeutics, Inc 39
Aridis Pharmaceuticals LLC 40
Bayer AG 41
Boehringer Ingelheim GmbH 42
Carolus Therapeutics, Inc. 43
Celtaxsys, Inc. 44
Chiesi Farmaceutici SpA 45
Cilian AG 46
Concert Pharmaceuticals, Inc. 47
Corbus pharmaceuticals, Inc. 48
Cyclacel Pharmaceuticals, Inc. 49
DiscoveryBiomed, Inc. 50
Errant Gene Therapeutics, LLC 51
Galapagos NV 52
GlaxoSmithKline Plc 53
Grifols, S.A. 54
Grupo Praxis Pharmaceutical SA 55
Horizon Pharma Plc 56
Insmed Incorporated 57
Invion Limited 58
JHL Biotech, Inc. 59
Kamada Ltd. 60
La Jolla Pharmaceutical Company 61
Lamellar Biomedical Ltd 62
Laurent Pharmaceuticals Inc. 63
Merck & Co., Inc. 64
Microbion Corporation 65
Mucokinetica Ltd. 66
NanoBio Corporation 67
Nivalis Therapeutics, Inc. 68
Nostrum Pharmaceuticals, LLC 69
Novabiotics Limited 70
Novartis AG 71
OSE Pharma SA 72
Parion Sciences, Inc. 73
Pfizer Inc. 74
Pharmaxis Limited 75
PhaseBio Pharmaceuticals, Inc. 76
Polyphor Ltd. 77
Progenra, Inc. 78
ProQR Therapeutics N.V. 79
Protalix BioTherapeutics, Inc. 80
Proteostasis Therapeutics, Inc. 81
PTC Therapeutics, Inc. 82
Pulmatrix, Inc. 83
ReveraGen BioPharma, Inc. 84
Savara Inc. 85
Serendex Pharmaceuticals A/S 86
Shionogi & Co., Ltd. 87
Shire Plc 88
Synovo GmbH 89
TGV-Laboratories 90
The International Biotechnology Center (IBC) Generium 91
Verona Pharma Plc 92
Vertex Pharmaceuticals Incorporated 93
Xenetic Biosciences plc 94
Zambon Company S.p.A. 95
Cystic Fibrosis - Therapeutics Assessment 96
Assessment by Monotherapy Products 96
Assessment by Combination Products 97
Assessment by Target 98
Assessment by Mechanism of Action 102
Assessment by Route of Administration 105
Assessment by Molecule Type 107
Drug Profiles 109
(ammonium chloride + citric acid + sodium citrate) - Drug Profile 109
(fosfomycin + tobramycin) - Drug Profile 110
(lumacaftor + ivacaftor) - Drug Profile 111
(tezacaftor + ivacaftor) - Drug Profile 114
ABPA-01 - Drug Profile 116
acebilustat - Drug Profile 118
Aerucin - Drug Profile 120
alpha-1 proteinase inhibitor (human) second generation - Drug Profile 121
ALX-009 - Drug Profile 124
amikacin sulphate - Drug Profile 126
amlexanox - Drug Profile 131
Anti-Pseudomonas IgY - Drug Profile 132
Antimicrobial Drug for Cystic Fibrosis - Drug Profile 134
AT-010 - Drug Profile 135
ataluren - Drug Profile 136
Biologics for Cystic Fibrosis Lung Infections - Drug Profile 140
budesonide - Drug Profile 141
Calcaftor - Drug Profile 142
CFX-1 - Drug Profile 143
CSY-0073 - Drug Profile 144
CT-2009 - Drug Profile 145
CTP-656 - Drug Profile 146
cysteamine IR - Drug Profile 147
dapsone - Drug Profile 149
Deoxyribonuclease II to Inhibit DNA Synthesis for Cystic Fibrosis - Drug Profile 150
doripenem - Drug Profile 151
dornase alfa biosimilar - Drug Profile 153
dornase alfa biosimilar - Drug Profile 154
Drug for Cystic Fibrosis - Drug Profile 155
FDL-169 - Drug Profile 156
fenretinide - Drug Profile 157
Gene Therapy to Activate CFTR for Cystic Fibrosis - Drug Profile 158
Gene Therapy to Activate CFTR for Cystic Fibrosis - Drug Profile 160
Gene Therapy to Activate CFTR Gene for Cystic Fibrosis - Drug Profile 161
GLPG-1837 - Drug Profile 162
GLPG-2222 - Drug Profile 164
GLPG-2665 - Drug Profile 165
glycerol phenylbutyrate - Drug Profile 166
GSK-2225745 - Drug Profile 169
JBT-101 - Drug Profile 170
kenpaullone - Drug Profile 172
levofloxacin - Drug Profile 173
LJPC-30Sa - Drug Profile 174
LJPC-30Sb - Drug Profile 175
LMS-611 - Drug Profile 176
LUNAR-CF - Drug Profile 177
mannitol - Drug Profile 178
MG-277 - Drug Profile 181
miglustat - Drug Profile 182
MKA-104 - Drug Profile 184
molgramostim - Drug Profile 185
Mul-1867 - Drug Profile 189
N-91115 - Drug Profile 190
N-91138 - Drug Profile 191
N-91169 - Drug Profile 192
nadolol - Drug Profile 193
NB-401 - Drug Profile 195
nitric oxide - Drug Profile 196
nitric oxide - Drug Profile 198
NVPQBE-170 - Drug Profile 199
Oligomer G for Cystic Fibrosis - Drug Profile 200
ORP-100 - Drug Profile 202
P-1055 - Drug Profile 203
Panaecin - Drug Profile 204
PB-01 - Drug Profile 206
PB-1046 - Drug Profile 208
PB-1120 - Drug Profile 210
Peptide to Block ENaC for Cystic Fibrosis and Hypertension - Drug Profile 211
POL-6014 - Drug Profile 212
PRX-110 - Drug Profile 214
Pseudomonas aeruginosa vaccine - Drug Profile 215
PTI-130 - Drug Profile 216
PTI-428 - Drug Profile 217
PTIC-1811 - Drug Profile 218
PTIP-271 - Drug Profile 219
PulmoXen - Drug Profile 220
PUR-1900 - Drug Profile 221
PXS-4728A - Drug Profile 222
QBW-251 - Drug Profile 223
QBW-276 - Drug Profile 224
QR-010 - Drug Profile 225
QR-X4 - Drug Profile 226
Recombinant A1PI - Drug Profile 227
Recombinant Enzyme to Replace Acid Ceramidase for Farber Disease and Cystic Fibrosis - Drug Profile 229
Recombinant Enzyme to Target DNA for Cystic Fibrosis - Drug Profile 230
Recombinant Protein for Infectious Diseases and Respiratory Disorders - Drug Profile 231
riociguat - Drug Profile 232
RPL-554 - Drug Profile 234
S-1226 - Drug Profile 237
S-1229 - Drug Profile 239
S-18 - Drug Profile 240
seliciclib - Drug Profile 241
SHP-636 - Drug Profile 243
Small Molecule for Cystic Fibrosis - Drug Profile 244
Small Molecule for Pneumonia, COPD, Cystic Fibrosis and Pulmonary Tuberculosis - Drug Profile 245
Small Molecule to Activate CFTR for Cystic Fibrosis - Drug Profile 246
Small Molecule to Inhibit EnaC and CFTR for Cystic Fibrosis, Diarrhea and Autosomal Dominant Polycystic Kidney Disease - Drug Profile 247
Small Molecule to Inhibit HDAC for Cystic Fibrosis and COPD - Drug Profile 248
Small Molecules for Bacterial Infections - Drug Profile 249
Small Molecules for Bacterial Infections, Oncology and Cystic Fibrosis - Drug Profile 250
Small Molecules for Cystic Fibrosis - Drug Profile 251
Small Molecules to Activate CFTR for Cystic Fibrosis - Drug Profile 252
Small Molecules to Activate CFTR Protein for Cystic Fibrosis - Drug Profile 253
Small Molecules to Agonize PPAR-Gamma for COPD and Cystic Fibrosis - Drug Profile 254
Small Molecules to Inhibit Lethal Factor for Anthrax and Cystic Fibrosis - Drug Profile 255
Small Molecules to Inhibit Sodium Channels for Cystic Fibrosis - Drug Profile 256
Small Molecules to Inhibit USP8 for Cystic Fibrosis - Drug Profile 257
solithromycin - Drug Profile 258
SP-14 - Drug Profile 261
SP-9 - Drug Profile 262
Stem Cell Therapy for Cystic Fibrosis - Drug Profile 263
SYGN-113 - Drug Profile 264
SYGN-303 - Drug Profile 265
tritoqualine - Drug Profile 266
vancomycin hydrochloride - Drug Profile 267
Verolone - Drug Profile 269
VX-152 - Drug Profile 271
VX-371 - Drug Profile 272
VX-440 - Drug Profile 273
ZP-046 - Drug Profile 274
Cystic Fibrosis - Recent Pipeline Updates 275
Cystic Fibrosis - Dormant Projects 343
Cystic Fibrosis - Discontinued Products 351
Cystic Fibrosis - Product Development Milestones 352
Featured News & Press Releases 352
Appendix 359
Methodology 359
Coverage 359
Secondary Research 359
Primary Research 359
Expert Panel Validation 359
Contact Us 359
Disclaimer 360

List of Tables

Number of Products under Development for Cystic Fibrosis, H2 2015 21
Number of Products under Development for Cystic Fibrosis - Comparative Analysis, H2 2015 22
Number of Products under Development by Companies, H2 2015 24
Number of Products under Development by Companies, H2 2015 (Contd..1) 25
Number of Products under Development by Companies, H2 2015 (Contd..2) 26
Number of Products under Development by Companies, H2 2015 (Contd..3) 27
Number of Products under Development by Companies, H2 2015 (Contd..4) 28
Number of Products under Investigation by Universities/Institutes, H2 2015 29
Comparative Analysis by Late Stage Development, H2 2015 30
Comparative Analysis by Clinical Stage Development, H2 2015 31
Comparative Analysis by Early Stage Development, H2 2015 32
Comparative Analysis by Unknown Stage Development, H2 2015 33
Products under Development by Companies, H2 2015 34
Products under Development by Companies, H2 2015 (Contd..1) 35
Products under Development by Companies, H2 2015 (Contd..2) 36
Products under Development by Companies, H2 2015 (Contd..3) 37
Products under Development by Companies, H2 2015 (Contd..4) 38
Products under Development by Companies, H2 2015 (Contd..5) 39
Products under Development by Companies, H2 2015 (Contd..6) 40
Products under Investigation by Universities/Institutes, H2 2015 41
Cystic Fibrosis - Pipeline by Actelion Ltd, H2 2015 42
Cystic Fibrosis - Pipeline by Akari Therapeutics, Plc, H2 2015 43
Cystic Fibrosis - Pipeline by Alaxia SAS, H2 2015 44
Cystic Fibrosis - Pipeline by AlgiPharma AS, H2 2015 45
Cystic Fibrosis - Pipeline by AmpliPhi Biosciences Corporation, H2 2015 46
Cystic Fibrosis - Pipeline by Arcturus Therapeutics, Inc, H2 2015 47
Cystic Fibrosis - Pipeline by Aridis Pharmaceuticals LLC, H2 2015 48
Cystic Fibrosis - Pipeline by Bayer AG, H2 2015 49
Cystic Fibrosis - Pipeline by Boehringer Ingelheim GmbH, H2 2015 50
Cystic Fibrosis - Pipeline by Carolus Therapeutics, Inc., H2 2015 51
Cystic Fibrosis - Pipeline by Celtaxsys, Inc., H2 2015 52
Cystic Fibrosis - Pipeline by Chiesi Farmaceutici SpA, H2 2015 53
Cystic Fibrosis - Pipeline by Cilian AG, H2 2015 54
Cystic Fibrosis - Pipeline by Concert Pharmaceuticals, Inc., H2 2015 55
Cystic Fibrosis - Pipeline by Corbus pharmaceuticals, Inc., H2 2015 56
Cystic Fibrosis - Pipeline by Cyclacel Pharmaceuticals, Inc., H2 2015 57
Cystic Fibrosis - Pipeline by DiscoveryBiomed, Inc., H2 2015 58
Cystic Fibrosis - Pipeline by Errant Gene Therapeutics, LLC, H2 2015 59
Cystic Fibrosis - Pipeline by Galapagos NV, H2 2015 60
Cystic Fibrosis - Pipeline by GlaxoSmithKline Plc, H2 2015 61
Cystic Fibrosis - Pipeline by Grifols, S.A., H2 2015 62
Cystic Fibrosis - Pipeline by Grupo Praxis Pharmaceutical SA, H2 2015 63
Cystic Fibrosis - Pipeline by Horizon Pharma Plc, H2 2015 64
Cystic Fibrosis - Pipeline by Insmed Incorporated, H2 2015 65
Cystic Fibrosis - Pipeline by Invion Limited, H2 2015 66
Cystic Fibrosis - Pipeline by JHL Biotech, Inc., H2 2015 67
Cystic Fibrosis - Pipeline by Kamada Ltd., H2 2015 68
Cystic Fibrosis - Pipeline by La Jolla Pharmaceutical Company, H2 2015 69
Cystic Fibrosis - Pipeline by Lamellar Biomedical Ltd, H2 2015 70
Cystic Fibrosis - Pipeline by Laurent Pharmaceuticals Inc., H2 2015 71
Cystic Fibrosis - Pipeline by Merck & Co., Inc., H2 2015 72
Cystic Fibrosis - Pipeline by Microbion Corporation, H2 2015 73
Cystic Fibrosis - Pipeline by Mucokinetica Ltd., H2 2015 74
Cystic Fibrosis - Pipeline by NanoBio Corporation, H2 2015 75
Cystic Fibrosis - Pipeline by Nivalis Therapeutics, Inc., H2 2015 76
Cystic Fibrosis - Pipeline by Nostrum Pharmaceuticals, LLC, H2 2015 77
Cystic Fibrosis - Pipeline by Novabiotics Limited, H2 2015 78
Cystic Fibrosis - Pipeline by Novartis AG, H2 2015 79
Cystic Fibrosis - Pipeline by OSE Pharma SA, H2 2015 80
Cystic Fibrosis - Pipeline by Parion Sciences, Inc., H2 2015 81
Cystic Fibrosis - Pipeline by Pfizer Inc., H2 2015 82
Cystic Fibrosis - Pipeline by Pharmaxis Limited, H2 2015 83
Cystic Fibrosis - Pipeline by PhaseBio Pharmaceuticals, Inc., H2 2015 84
Cystic Fibrosis - Pipeline by Polyphor Ltd., H2 2015 85
Cystic Fibrosis - Pipeline by Progenra, Inc., H2 2015 86
Cystic Fibrosis - Pipeline by ProQR Therapeutics N.V., H2 2015 87
Cystic Fibrosis - Pipeline by Protalix BioTherapeutics, Inc., H2 2015 88
Cystic Fibrosis - Pipeline by Proteostasis Therapeutics, Inc., H2 2015 89
Cystic Fibrosis - Pipeline by PTC Therapeutics, Inc., H2 2015 90
Cystic Fibrosis - Pipeline by Pulmatrix, Inc., H2 2015 91
Cystic Fibrosis - Pipeline by ReveraGen BioPharma, Inc., H2 2015 92
Cystic Fibrosis - Pipeline by Savara Inc., H2 2015 93
Cystic Fibrosis - Pipeline by Serendex Pharmaceuticals A/S, H2 2015 94
Cystic Fibrosis - Pipeline by Shionogi & Co., Ltd., H2 2015 95
Cystic Fibrosis - Pipeline by Shire Plc, H2 2015 96
Cystic Fibrosis - Pipeline by Synovo GmbH, H2 2015 97
Cystic Fibrosis - Pipeline by TGV-Laboratories, H2 2015 98
Cystic Fibrosis - Pipeline by The International Biotechnology Center (IBC) Generium, H2 2015 99
Cystic Fibrosis - Pipeline by Verona Pharma Plc, H2 2015 100
Cystic Fibrosis - Pipeline by Vertex Pharmaceuticals Incorporated, H2 2015 101
Cystic Fibrosis - Pipeline by Xenetic Biosciences plc, H2 2015 102
Cystic Fibrosis - Pipeline by Zambon Company S.p.A., H2 2015 103
Assessment by Monotherapy Products, H2 2015 104
Assessment by Combination Products, H2 2015 105
Number of Products by Stage and Target, H2 2015 107
Number of Products by Stage and Mechanism of Action, H2 2015 111
Number of Products by Stage and Route of Administration, H2 2015 114
Number of Products by Stage and Molecule Type, H2 2015 116
Cystic Fibrosis Therapeutics - Recent Pipeline Updates, H2 2015 283
Cystic Fibrosis - Dormant Projects, H2 2015 351
Cystic Fibrosis - Dormant Projects (Contd..1), H2 2015 352
Cystic Fibrosis - Dormant Projects (Contd..2), H2 2015 353
Cystic Fibrosis - Dormant Projects (Contd..3), H2 2015 354
Cystic Fibrosis - Dormant Projects (Contd..4), H2 2015 355
Cystic Fibrosis - Dormant Projects (Contd..5), H2 2015 356
Cystic Fibrosis - Dormant Projects (Contd..6), H2 2015 357
Cystic Fibrosis - Dormant Projects (Contd..7), H2 2015 358
Cystic Fibrosis - Discontinued Products, H2 2015 359

List of Figures

Number of Products under Development for Cystic Fibrosis, H2 2015 21
Number of Products under Development for Cystic Fibrosis - Comparative Analysis, H2 2015 22
Number of Products under Development by Companies, H2 2015 23
Number of Products under Investigation by Universities/Institutes, H2 2015 29
Comparative Analysis by Late Stage Development, H2 2015 30
Comparative Analysis by Clinical Stage Development, H2 2015 31
Comparative Analysis by Early Stage Products, H2 2015 32
Assessment by Monotherapy Products, H2 2015 104
Assessment by Combination Products, H2 2015 105
Number of Products by Top 10 Targets, H2 2015 106
Number of Products by Stage and Top 10 Targets, H2 2015 106
Number of Products by Top 10 Mechanism of Actions, H2 2015 110
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2015 110
Number of Products by Routes of Administration, H2 2015 113
Number of Products by Stage and Routes of Administration, H2 2015 113
Number of Products by Top 10 Molecule Types, H2 2015 115
Number of Products by Stage and Top 10 Molecule Types, H2 2015 115

Published By: Global Markets Direct
Product Code: Global Markets Direct14313


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