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Myelodysplastic Syndrome - Pipeline Review, H2

2015Now Available at iData Insights


Myelodysplastic Syndrome - Pipeline Review, H2 2015 Summary Global Markets Direct s,
Myelodysplastic Syndrome - Pipeline Review, H2 2015, provides an overview of the Myelodysplastic
Syndromes therapeutic pipeline. This report provides comprehensive information on the therapeutic
development for Myelodysplastic Syndrome, 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 Myelodysplastic Syndrome 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 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 Myelodysplastic Syndrome - 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 Myelodysplastic Syndrome and enlists all their major and
minor projects - The report summarizes all the dormant and discontinued pipeline projects - A review of
the Myelodysplastic Syndrome 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 Myelodysplastic Syndrome 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 Myelodysplastic Syndrome - Plan mergers and acquisitions
effectively by identifying key players of the most promising pipeline - Devise corrective measures for
pipeline projects by understanding Myelodysplastic Syndrome 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
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the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove
them from pipeline.
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Table of Contents

Introduction 12
Global Markets Direct Report Coverage 12
Myelodysplastic Syndrome Overview 13
Therapeutics Development 14
Pipeline Products for Myelodysplastic Syndrome - Overview 14
Pipeline Products for Myelodysplastic Syndrome - Comparative Analysis 15
Myelodysplastic Syndrome - Therapeutics under Development by Companies 16
Myelodysplastic Syndrome - Therapeutics under Investigation by Universities/Institutes 23
Myelodysplastic Syndrome - Pipeline Products Glance 24
Late Stage Products 24
Clinical Stage Products 25
Early Stage Products 26
Myelodysplastic Syndrome - Products under Development by Companies 27
Myelodysplastic Syndrome - Products under Investigation by Universities/Institutes 35
Myelodysplastic Syndrome - Companies Involved in Therapeutics Development 36
4SC AG 36
Acceleron Pharma, Inc. 37
Actinium Pharmaceuticals, Inc. 38
Advancell 39
Agios Pharmaceuticals, Inc. 40
Alnylam Pharmaceuticals, Inc. 41
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Altor BioScience Corporation 42


Amgen Inc. 43
Apogenix GmbH 44
Arno Therapeutics, Inc. 45
Arog Pharmaceuticals, Inc. 46
Array BioPharma Inc. 47
Astex Pharmaceuticals, Inc. 48
Atara Biotherapeutics, Inc. 49
Bellicum Pharmaceuticals, Inc. 50
Bio-Path Holdings, Inc. 51
BioLineRx, Ltd. 52
Boehringer Ingelheim GmbH 53
Boryung Pharmaceutical Co., Ltd. 54
Boston Biomedical, Inc. 55
Bristol-Myers Squibb Company 56
Celator Pharmaceuticals, Inc. 57
Celgene Corporation 58
Celldex Therapeutics, Inc. 59
Cellerant Therapeutics, Inc. 60
Celyad SA 61
Clevexel Pharma SAS 62
Concert Pharmaceuticals, Inc. 63
Constellation Pharmaceuticals, Inc. 64
Cornerstone Pharmaceuticals, Inc. 65
CrystalGenomics, Inc. 66
CTI BioPharma Corp. 67

Cyclacel Pharmaceuticals, Inc. 68


Daiichi Sankyo Company, Limited 69
DiNonA Inc. 70
Eisai Co., Ltd. 71
Eleos Inc. 72
Eli Lilly and Company 73
EpiZyme, Inc. 74
F. Hoffmann-La Roche Ltd. 75
FORMA Therapeutics, Inc. 76
Fujifilm Corporation 77
Gamida Cell Ltd. 78
IGF Oncology, LLC. 79
ImmunoGen, Inc. 80
Incyte Corporation 81
Juno Therapeutics Inc. 82
JW Pharmaceutical Corporation 83
Kainos Medicine, Inc. 84
KaloBios Pharmaceuticals, Inc. 85
Karyopharm Therapeutics, Inc. 86
Kiadis Pharma B.V. 87
Kyowa Hakko Kirin Co., Ltd. 88
Les Laboratoires Servier SAS 89
Lixte Biotechnology Holdings, Inc. 90
medac GmbH 91
MedImmune, LLC 92
MEI Pharma, Inc. 93
Merck & Co., Inc. 94
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Merus B.V. 95
Millennium Pharmaceuticals, Inc. 96
Mirati Therapeutics Inc. 97
Mirna Therapeutics, Inc. 98
Nemucore Medical Innovations, Inc. 99
Novartis AG 100
Onconova Therapeutics, Inc. 101
Opsona Therapeutics Limited 102
OXiGENE, Inc. 103
Pfizer Inc. 104
Pharma Mar, S.A. 105
Regen BioPharma, Inc. 106
Rich Pharmaceuticals, Inc. 107
Stemline Therapeutics, Inc. 108
Sumitomo Dainippon Pharma Co., Ltd. 109
Sunesis Pharmaceuticals, Inc. 110
Syndax Pharmaceuticals, Inc. 111
Synta Pharmaceuticals Corp. 112
Targazyme, Inc. 113
TetraLogic Pharmaceuticals 114
Teva Pharmaceutical Industries Limited 115
Threshold Pharmaceuticals, Inc. 116
Tragara Pharmaceuticals, Inc. 117
Myelodysplastic Syndrome - Therapeutics Assessment 118
Assessment by Monotherapy Products 118
Assessment by Combination Products 119

Assessment by Target 120


Assessment by Mechanism of Action 128
Assessment by Route of Administration 135
Assessment by Molecule Type 137
Drug Profiles 139
(cytarabine hydrochloride + daunorubicin hydrochloride) - Drug Profile 139
(decitabine + E-7727) - Drug Profile 142
4SC-202 - Drug Profile 144
765IGF-MTX - Drug Profile 146
acadesine - Drug Profile 147
AG-120 - Drug Profile 149
ALN-HPN - Drug Profile 151
alpelisib - Drug Profile 152
ALT-803 - Drug Profile 154
Antisense RNAi Oligonucleotide to Inhibit NR2F6 Protein for Oncology - Drug Profile 157
AR-42 - Drug Profile 158
asinercept - Drug Profile 160
atezolizumab - Drug Profile 162
ATIR-101 - Drug Profile 166
azacitidine - Drug Profile 168
BBMPI-03 - Drug Profile 172
BI-836858 - Drug Profile 173
binimetinib - Drug Profile 174
birinapant - Drug Profile 179
BL-8040 - Drug Profile 182
BLI-1301 - Drug Profile 184
bortezomib - Drug Profile 185
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BP-100101 - Drug Profile 191


BPX-501 - Drug Profile 193
BR-05001 - Drug Profile 195
CC-90007 - Drug Profile 196
CDX-1401 - Drug Profile 199
Cell Therapy to Target Tumor Associated Antigen for Hematopoietic Malignancies - Drug Profile 201
Cell Therapy to Target WT-1, NY-ESO-1, PRAME and Survivin for Oncology - Drug Profile 202
Cell Therapy to Target WT1 for Cancer - Drug Profile 203
cenersen sodium - Drug Profile 204
CG-200745 - Drug Profile 206
CIGB-300 - Drug Profile 207
CMCS-1 - Drug Profile 209
CPI-0610 - Drug Profile 211
CPI-613 - Drug Profile 212
crenolanib besylate - Drug Profile 214
CSC-012 - Drug Profile 216
CTP-221 - Drug Profile 217
CVXL-0056 - Drug Profile 218
CWP-291 - Drug Profile 219
darbepoetin alfa - Drug Profile 221
dasatinib - Drug Profile 223
decitabine - Drug Profile 226
Dendritic Cell Therapy for Hematological Malignancies - Drug Profile 229
Dendritic Cell Therapy for Oncology - Drug Profile 230
Dendritic Cell Therapy to Target WT1 for Oncology - Drug Profile 231
DNP-001 - Drug Profile 232

DS-3032 - Drug Profile 233


DSP-7888 - Drug Profile 234
durvalumab - Drug Profile 235
E-6201 - Drug Profile 239
eltrombopag olamine - Drug Profile 241
entinostat - Drug Profile 244
epacadostat - Drug Profile 248
erismodegib - Drug Profile 250
everolimus - Drug Profile 253
evofosfamide - Drug Profile 259
FF-10501 - Drug Profile 267
FT-1101 - Drug Profile 268
galunisertib - Drug Profile 269
ganetespib - Drug Profile 271
Gene Therapy for Oncology - Drug Profile 276
glasdegib - Drug Profile 277
guadecitabine - Drug Profile 279
HDM-201 - Drug Profile 283
Hu8F4 - Drug Profile 284
IMGN-779 - Drug Profile 285
indisulam - Drug Profile 286
Iomab-B - Drug Profile 288
ixazomib citrate - Drug Profile 290
JTCR-016 - Drug Profile 294
KB-004 - Drug Profile 295
KHK-2823 - Drug Profile 297
KM-101 - Drug Profile 298
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LB-100 - Drug Profile 299


lenalidomide - Drug Profile 301
lirilumab - Drug Profile 306
lurbinectedin - Drug Profile 308
luspatercept - Drug Profile 311
MCLA-?117 - Drug Profile 313
midostaurin - Drug Profile 314
mocetinostat - Drug Profile 316
Monoclonal Antibodies to Target CD16 and CD33 for Oncology - Drug Profile 319
Monoclonal Antibody Conjugate 2 to Target CD45 for Cancer - Drug Profile 320
Monoclonal Antibody Conjugate to Inhibit Protein S100-A9 for MDS - Drug Profile 321
Monoclonal Antibody Conjugate to Target CD45 for Oncology - Drug Profile 322
Monoclonal Antibody for Myelodysplastic Syndrome - Drug Profile 323
MRX-102 - Drug Profile 324
MRX-34 - Drug Profile 326
napabucasin - Drug Profile 328
NiCord - Drug Profile 331
NMI-900 - Drug Profile 332
omacetaxine mepesuccinate - Drug Profile 333
ONC-201 - Drug Profile 336
OPN-305 - Drug Profile 339
OXi-4503 - Drug Profile 341
pacritinib - Drug Profile 343
pembrolizumab - Drug Profile 346
pevonedistat hydrochloride - Drug Profile 355
pexmetinib - Drug Profile 357

PIM-447 - Drug Profile 358


pinometostat - Drug Profile 359
pracinostat - Drug Profile 362
quizartinib dihydrochloride - Drug Profile 364
rigosertib sodium - Drug Profile 367
RP-323 - Drug Profile 372
ruxolitinib phosphate - Drug Profile 373
S-055746 - Drug Profile 378
sapacitabine - Drug Profile 379
selinexor - Drug Profile 381
SL-401 - Drug Profile 387
Small Molecule to Inhibit KDM1A for Oncology - Drug Profile 389
Small Molecule to Inhibit SF3B1 for myelodysplastic syndrome - Drug Profile 390
Small Molecules for Oncology - Drug Profile 391
sotatercept - Drug Profile 392
TEN-010 - Drug Profile 394
TG-02 - Drug Profile 395
tosedostat - Drug Profile 398
trametinib dimethyl sulfoxide - Drug Profile 400
treosulfan - Drug Profile 403
TZ-101 - Drug Profile 405
ulixertinib - Drug Profile 407
Vaccine for Oncology - Drug Profile 408
Vaccine to Target Wilms Tumor Protein for Oncology - Drug Profile 409
Vaccine to Target WT1 for Oncology - Drug Profile 410
VAL-1000 - Drug Profile 411
volasertib - Drug Profile 412
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vorinostat - Drug Profile 415


vosaroxin - Drug Profile 419
WT-4869 - Drug Profile 421
Myelodysplastic Syndrome - Recent Pipeline Updates 422
Myelodysplastic Syndrome - Dormant Projects 662
Myelodysplastic Syndrome - Discontinued Products 667
Myelodysplastic Syndrome - Product Development Milestones 669
Featured News & Press Releases 669
Appendix 675
Methodology 675
Coverage 675
Secondary Research 675
Primary Research 675
Expert Panel Validation 675
Contact Us 675
Disclaimer 676

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