2015 medicines in development -- leukemia & lymphoma
TRANSCRIPT
2015 Medicines in Development -- Leukemia & LymphomaA Report on Cancers of the Blood
Hematological Malignancies
Product Name Sponsor Indication Development Status
ABT-199/GDC-0199 AbbVie hematological malignancies Phase I
(Bcl-2 inhibitor) North Chicago, IL (see also leukemia, lymphoma) www.abbvie.com
Genentech www.gene.com
South San Francisco, CA
AG-120 Agios Pharmaceuticals advanced hematological malignancies Phase I
(IDH1 inhibitor) Cambridge, MA www.agios.com
AGS67E Agensys relapsed/refractory lymphoid malignancies Phase I
(antibody drug conjugate) Santa Monica, CA www.agensys.com
ALT-803 Altor BioScience hematological malignancies Phase I/II
(IL-15 superagonist complex) Miramar, FL (see also myeloma) www.altorbioscience.com
AMG 319 Amgen hematological malignancies Phase I
(PI3K-delta inhibitor) Thousand Oaks, CA www.amgen.com
AMG 900 Amgen hematological malignancies Phase I
(aurora kinase inhibitor) Thousand Oaks, CA www.amgen.com
anti-LAG3 Bristol-Myers Squibb hematological malignancies Phase I
Princeton, NJ www.bms.com
APTO-253 Aptose Biosciences relapsed/refractory hematological malignancies Phase I
(MTF-1 inhibitor) San Diego, CA www.aptose.com
Hematological Malignancies
Product Name Sponsor Indication Development Status
AR-42 Arno Therapeutics relapsed/refractory hematological malignancies Phase I/II
(pan-DAC inhibitor) Flemington, NJ www.arnothera.com
AZD9150 AstraZeneca hematological malignancies Phase I
(STAT3 inhibitor) Wilmington, DE www.astrazeneca.com
Isis Pharmaceuticals www.isispharm.com
Carlsbad, CA
BGJ398 Novartis Pharmaceuticals hematological malignancies Phase II
(FGF-R kinase inhibitor) East Hanover, NJ www.novartis.com
BKM120 Novartis Pharmaceuticals hematological malignancies Phase II
(PI3K inhibitor) East Hanover, NJ www.novartis.com
BPX-501 Bellicum Pharmaceuticals bone marrow transplantation in Phase I/II
(T cell replacement therapy) Houston, TX hematological malignancies www.bellicum.com
CB-839 Calithera Biosciences hematological malignancies Phase I
(glutaminase inhibitor) South San Francisco, CA www.calithera.com
duvelisib AbbVie advanced hematological malignancies Phase I
(PI3K-delta/gamma inhibitor) North Chicago, IL (see also leukemia, lymphoma) www.abbvie.com
Infinity Pharmaceuticals www.infi.com
Cambridge, MA
entopletinib Gilead Sciences hematological malignancies Phase II
(Syk inhibitor) Foster City, CA www.gilead.com
Hematological Malignancies
Product Name Sponsor Indication Development Status
FF-10501 Boston Strategics hematological malignancies Phase I/II
(cell differentiation stimulant) Boston, MA www.bostonstrategics.com
FUJIFILM Pharmaceuticals USA
Boston, MA
GS-4059 Gilead Sciences B-cell malignancies Phase II
(Btk inhibitor) Foster City, CA www.gilead.com
GS-9901 Gilead Sciences hematological malignancies Phase I
(PI3K-delta inhibitor) Foster City, CA www.gilead.com
HDM201 Novartis Pharmaceuticals hematological malignancies Phase I
(tumor suppressor protein East Hanover, NJ www.novartis.com
p53 modulator)
HSC835 Novartis Pharmaceuticals hematological malignancies Phase I/II
(stem cell therapy) East Hanover, NJ www.novartis.com
INCB39110+INCB40093 Incyte B-lymphoid malignancies Phase II
(JAK1+PI3K-delta inhibitor) Wilmington, DE www.incyte.com
INCB52793 Incyte advanced hematological malignancies Phase I
(JAK1 inhibitor) Wilmington, DE www.incyte.com
JCAR014 Juno Therapeutics adult B-cell malignancies Phase I/II
(T lymphocyte immunotherapy) Seattle, WA www.junotherapeutics.com
Keytruda® Merck hematological malignancies Phase I
pembrolizumab Whitehouse Station, NJ (see also myeloma) www.merck.com
Hematological Malignancies
Product Name Sponsor Indication Development Status
LDE225 Novartis Pharmaceuticals PTCH1 or SMO-activated hematological tumors Phase II
(Smo antagonist) East Hanover, NJ (see also leukemia, myeloproliferative) www.novartis.com
LEE011 Novartis Pharmaceuticals hematological malignancies Phase II
(CDK4/6 inhibitor) East Hanover, NJ (CDK4/6 pathway activation) www.novartis.com
MEDI-551+rituximab MedImmune hematological malignancies Phase I
(anti-CD19 mAb+anti-CD20 mAb) Gaithersburg, MD (see also leukemia, lymphoma) www.medimmune.com
MRX34 Mirna Therapeutics hematological malignancies Phase I
(liposome-encapsulated Austin, TX www.mirnarx.com
miR-34 mimic)
NiCord® Gamida Cell hematological malignancies Phase I/II
cord blood stem cell therapy Jerusalem, Israel www.gamida-cell.com
OMP-52M51 GlaxoSmithKline hematological malignancies Phase I
(NOTCH-1 inhibitor) Research Triangle Park, NC www.oncomed.com
OncoMed Pharmaceuticals
Redwood City, CA
Opdivo™ Bristol-Myers Squibb hematological malignancies Phase I
nivolumab Princeton, NJ (see also leukemia, lymphoma, myeloma) www.bms.com
oprozomib Onyx Pharmaceuticals relapsed/refractory hematological malignancies Phase I/II
(proteasome inhibitor) South San Francisco, CA (see also myeloma) www.onyx.com
Orphan Drug
Hematological Malignancies
Product Name Sponsor Indication Development Status
RG7388 Genentech hematological malignancies Phase I
(MDM2 antagonist) South San Francisco, CA www.gene.com
SAR3419 Sanofi US B-cell relapsed/refractory malignancies Phase II
(maytansin-loaded anti-CD19 mAb) Bridgewater, NJ www.sanofi.com
SL-401 Stemline Therapeutics blastic plasmacytoid dendritic cell Phase I/II
(IL-3R inhibitor) New York, NY neoplasms www.stemline.com
Leukemia & Lymphoma Society (see also leukemia, myeloproliferative) www.lls.org
White Plains, NY
SNX-5422 Esanex refractory hematological malignancies Phase I
(Hsp90 inhibitor) Indianapolis, IN
StemEx® Gamida Cell-Teva Joint Venture hematological malignancies Phase II/III completed
carlecortemcel-L Jerusalem, Israel (Fast Track) www.gamida-cell.com
Orphan Drug
TAK-659 Millennium Pharmaceuticals hematological malignancies Phase I
(SYK kinase inhibitor) Cambridge, MA www.millennium.com
TGR-1202 TG Therapeutics hematological malignancies Phase I
(PI3K-delta inhibitor) New York, NY www.tgtherapeutics.com
TKI258 Novartis Pharmaceuticals hematological malignancies (pathway activated) Phase II
(dovitinib) East Hanover, NJ www.novartis.com
Hematological Malignancies
Product Name Sponsor Indication Development Status
TZ101 Targazyme cord blood stem cell transplantation Phase I/II
(human recombinant enzyme) San Antonio, TX for hematological malignancies www.targazyme.com
Orphan Drug
ulocuplumab Bristol-Myers Squibb hematological malignancies Phase I
(anti-CXCR4) Princeton, NJ www.bms.com
urelumab Bristol-Myers Squibb hematological malignancies Phase I
Princeton, NJ (see also myeloma) www.bms.com
urelumab+Opdivo™ Bristol-Myers Squibb hematological malignancies Phase I
nivolumab Princeton, NJ www.bms.com
varlilumab Celldex Therapeutics hematological malignancies Phase I
(CD27 antigen inhibitor) Hampton, NJ www.celldex.com
WT2725 Sunovion Pharmaceuticals hematological malignancies Phase I
(cancer immunotherapy) Marlborough, MA www.sunovion.com
Leukemia
Product Name Sponsor Indication Development Status
Å6 peptide Angstrom Pharmaceuticals chronic lymphocytic leukemia (CLL) (1st-line), Phase II
(CD44 antigen stimulant) Solana Beach, CA small lymphocytic leukemia (1st-line) www.angstrominc.com
ABL001 Novartis Pharmaceuticals chronic myeloid leukemia (CML), Philadelphia- Phase I
(Bcr-Abl kinase inhibitor) East Hanover, NJ positive acute lymphoblastic leukemia (Ph+ ALL) www.novartis.com
Leukemia
Product Name Sponsor Indication Development Status
ABT-199/GDC-0199 AbbVie relapsed/refractory CLL (combination therapy), Phase III
(Bcl-2 inhibitor) North Chicago, IL CLL (1st-line) (combination therapy) www.abbvie.com
Genentech (see also hematological, lymphoma) www.gene.com
South San Francisco, CA
relapsed/refractory CLL (17pdel) Phase II
www.abbvie.com
www.gene.com
CLL (combination therapy) Phase I
www.abbvie.com
www.gene.com
ACP-196 Acerta Pharma relapsed/refractory B-cell CLL, Phase II
(Btk inhibitor) San Carlos, CA small lymphocytic leukemia www.acerta-pharma.com
National Institutes of Health (see also lymphoma)
Rockville, MD
ACP-319 Acerta Pharma B-cell ALL (combination therapy) Phase I/II
San Carlos, CA (see also lymphoma, myeloma) www.acerta-pharma.com
relapsed/refractory CLL (combination therapy) Phase I
www.acerta-pharma.com
ADI-PEG 20 Polaris Pharmaceuticals acute myeloid leukemia (AML) Phase II
(PEG arginine deiminase) San Diego, CA (see also lymphoma) www.polarispharma.com
Leukemia
Product Name Sponsor Indication Development Status
AG-221 Agios Pharmaceuticals AML Phase I
(IDH2 inhibitor) Cambridge, MA www.agios.com
Orphan Drug Celgene www.celgene.com
Summit, NJ
alvocidib Tolero Pharmaceuticals AML (1st-line) Phase II
(CDK inhibitor) Lehi, UT www.toleropharma.com
Orphan Drug
relapsed/refractory AML Phase I
www.toleropharma.com
AMG 232 Amgen AML Phase I
(MDM2 inhibitor) Thousand Oaks, CA www.amgen.com
anti-CD22-CAR transduced T-cells Opus Bio ALL Phase I
Gaithersburg, MD (see also lymphoma) www.opusbio.com
National Cancer Institute
Bethesda, MD
Arzerra® GlaxoSmithKline relapsed CLL Phase III
ofatumumab Research Triangle Park, NC (see also lymphoma) www.gsk.com
Orphan Drug
ASP2215 Astellas Pharma US relapsed/refractory AML Phase I/II
(gilteritinib) Northbrook, IL www.astellas.com
AST-VAC1 Asterias Biotherapeutics AML Phase II completed
(telomerase-based cancer vaccine) Menlo Park, CA www.asteriasbiotherapeutics.com
Leukemia
Product Name Sponsor Indication Development Status
ATTCK20 Unum Therapeutics CLL Phase I
(viral-ACTR+anti-CD20) Cambridge, MA (see also lymphoma) www.unumrx.com
bendamustine ready-to-dilute Eagle Pharmaceuticals CLL application submitted
liquid formulation Woodcliff Lake, NJ (see also lymphoma) www.eagleus.com
Orphan Drug
BI 811283 Boehringer Ingelheim Pharmaceuticals AML Phase II completed
(aurora B kinase inhibitor) Ridgefield, CT www.boehringer-ingelheim.com
BI 836826 Boehringer Ingelheim Pharmaceuticals CLL Phase I
Ridgefield, CT www.boehringer-ingelheim.com
BI 836858 Boehringer Ingelheim Pharmaceuticals AML Phase I
Ridgefield, CT (see also myelodysplastic) www.boehringer-ingelheim.com
BL-8040 BioLineRx AML (combination therapy) Phase II
(CXCR4 receptor antagonist) Jerusalem, Israel www.biolinerx.com
Orphan Drug
Bosulif® Pfizer CML (1st-line) Phase III
bosutinib New York, NY www.pfizer.com
Orphan Drug
BP-100-1.01 Bio-Path Holdings ALL, AML, CML Phase I
(liposomal Grb-2) Houston, TX (see also myelodysplastic) www.biopathholdings.com
Orphan Drug
Leukemia
Product Name Sponsor Indication Development Status
BVD-523 BioMed Valley Discoveries AML Phase I/II
(ERK 1/2 kinase inhibitor) Kansas City, MO (see also myelodysplastic) www.biomed-valley.com
CC-486 Celgene post-induction AML maintenance Phase III
(azacitidine) Summit, NJ (see also myelodysplastic) www.celgene.com
cerdulatinib Portola Pharmaceuticals CLL Phase I
(dual Syk/JAK inhibitor) South San Francisco, CA (see also lymphoma) www.portola.com
CNDO-109 Coronado Biosciences AML Phase I/II
(natural killer cell stimulant) Burlington, MA www.coronadobiosciences.com
Orphan Drug
CPI-0610 Constellation Pharmaceuticals acute leukemia Phase I
(BET protein inhibitor) Cambridge, MA (see also lymphoma, myelodysplastic, myeloma, www.constellationpharma.com
Leukemia & Lymphoma Society myeloproliferative) www.lls.org
White Plains, NY
CPX-351 Celator Pharmaceuticals secondary AML (Fast Track) Phase III
(cytarabine/daunorubicin liposomal) Ewing, NJ www.celatorpharma.com
Orphan Drug Leukemia & Lymphoma Society www.lls.org
White Plains, NY
Leukemia
Product Name Sponsor Indication Development Status
crenolanib Arog Pharmaceuticals relapsed/refractory AML Phase III
(PDGFR kinase inhibitor) Dallas, TX www.arogpharma.com
newly diagnosed AML, post bone marrow Phase II
transplant in AML (maintenance therapy) www.arogpharma.com
AML (combination therapy) Phase I
www.arogpharma.com
CSL362 CSL AML Phase I
(anti-CD123 mAb) Victoria, Australia www.csl.com.au
Janssen Research & Development www.janssenrnd.com
Raritan, NJ
CTL019 Novartis Pharmaceuticals relapsed/refractory ALL (Breakthrough Therapy) Phase II
(chimeric antigen receptor T-cell East Hanover, NJ (see also lymphoma) www.novartis.com
therapy)
CWP232291 JW Pharmaceutical AML Phase I
(Wnt signaling pathway inhibitor) Seoul, Korea
DFP-10917 Delta-Fly Pharma ALL, AML Phase I/II
(cell cycle inhibitor) Tokushima, Japan
Leukemia
Product Name Sponsor Indication Development Status
duvelisib AbbVie relapsed/refractory CLL Phase III
(PI3K-delta/gamma inhibitor) North Chicago, IL (see also hematological, lymphoma) www.abbvie.com
Orphan Drug Infinity Pharmaceuticals www.infi.com
Cambridge, MA
relapsed/refractory CLL (combination therapy) Phase I
www.abbvie.com
www.infi.com
EPZ-5676 Celgene AML (altered MLL gene) Phase I
(DOT-1L inhibitor) Summit, NJ www.celgene.com
Orphan Drug Epizyme www.epizyme.com
Cambridge, MA
Erwinaze® Jazz Pharmaceuticals ALL in young adults Phase II
asparaginase erwinia Palo Alto, CA www.jazzpharm.com
chrysanthemi intravenous
Orphan Drug
ERY-ASP ERYTECH Pharmaceuticals ALL (adults) Phase I/II
(asparaginase modulator) Lyon, France www.erytech.com
Orphan Drug
EZN-2285 Sigma-Tau Pharmaceuticals ALL Phase III completed
(calaspargase pegol) Gaithersburg, MD www.sigmatau.com
ganetespib Synta Pharmaceuticals AML Phase II/III
(Hsp-90 inhibitor) Lexington, MA www.syntapharma.com
Leukemia
Product Name Sponsor Indication Development Status
GMI-1271 GlycoMimetics AML Phase I
(E-selectin antagonist) Gaithersburg, MD www.glycomimetics.com
University of Michigan
Ann Arbor, MI
GO-203-2c Genus Oncology relapsed/refractory AML Phase I/II
(MUC1 inhibitor) Vernon Hills, IL www.genusoncology.com
Dana-Farber Cancer Institute
Boston, MA
GSK2879552 GlaxoSmithKline relapsed/refractory AML Phase I
(LSD1)/co-repressor protein inhibitor) Research Triangle Park, NC www.gsk.com
HSV-Tk MolMed high-risk acute leukemia Phase III
(thymidine kinase cell therapy) Milan, Italy www.molmed.com
Orphan Drug
Iclusig® ARIAD Pharmaceuticals FLT3-positive AML, Ph+ALL (combination therapy) Phase II
ponatinib Cambridge, MA www.ariad.com
Orphan Drug
IGN523 mAb Igenica Biotherapeutics AML Phase I
(anti-CD98 mAb) Burlingame, CA www.igenica.com
Imbruvica® Janssen Research & Development relapsed/refractory CLL (combination therapy), Phase III
ibrutinib Raritan, NJ treatment naïve CLL www.janssenrnd.com
Orphan Drug Pharmacyclics (see also lymphoma) www.pharmacyclics.com
Sunnyvale, CA
Leukemia
Product Name Sponsor Indication Development Status
IMMU-114 Immunomedics CLL Phase I
(anti-HLA-DR antibody) Morris Plains, NJ (see also lymphoma) www.immunomedics.com
Imprime PGG® Biothera high-risk CLL (combination therapy) Phase II
intravenous immunostimulant Eagan, MN (see also lymphoma) www.biothera.com
Mayo Clinic
Rochester, MN
inotuzumab ozogamicin Pfizer ALL Phase III
(CD22-targeted cytotoxic agent) New York, NY www.pfizer.com
Orphan Drug
Iomab™-B Actinium Pharmaceuticals hematopoietic stem cell transplantation (HSCT) Phase II
BC8-I-131 construct New York, NY in relapsed/refractory AML www.actiniumpharma.com
JCAR015 Juno Therapeutics adult ALL Phase I
(T lymphocyte immunotherapy) Seattle, WA (see also lymphoma) www.junotherapeutics.com
Orphan Drug
JCAR017 Juno Therapeutics pediatric ALL Phase I/II
(T lymphocyte immunotherapy) Seattle, WA www.junotherapeutics.com
JTCR016 Juno Therapeutics adult AML Phase I/II
(T lymphocyte immunotherapy) Seattle, WA www.junotherapeutics.com
JZP-416 Jazz Pharmaceuticals ALL in adolescents and children (Fast Track) Phase II
(recombinant L-asparaginase) Palo Alto, CA www.jazzpharm.com
Orphan Drug
Leukemia
Product Name Sponsor Indication Development Status
KB004 KaloBios Pharmaceuticals EphA3-positive AML Phase II
(EphA3 receptor antagonist) South San Francisco, CA (see also myelodysplastic, myeloproliferative) www.kalobios.com
KX01 Kinex Pharmaceuticals AML Phase I completed
(non-ATP competitive Sre kinase Buffalo, NY www.kinexpharma.com
inhibitor)
LDE225 Novartis Pharmaceuticals relapsed/refractory acute leukemia Phase II
(Smo antagonist) East Hanover, NJ (see also hematological, myeloproliferative) www.novartis.com
LGH447 Novartis Pharmaceuticals AML Phase I
(proto oncogene protein c East Hanover, NJ (see also myelodysplastic, myeloma) www.novartis.com
pim-1 inhibitor)
lintuzumab Ac-225 Actinium Pharmaceuticals AML in the elderly (1st-line) Phase I/II
(CD33-antigen inhibitor) New York, NY www.actiniumpharma.com
Orphan Drug
LOP628 Novartis Pharmaceuticals c-Kit-positive AML Phase I
(antibody-drug conjugate) East Hanover, NJ www.novartis.com
Marqibo® Spectrum Pharmaceuticals elderly Ph-negative ALL (1st-line) Phase III
vinCRIStine sulfate LIPOSOME Henderson, NV (Fast Track) www.sppirx.com
injection (see also lymphoma)
Orphan Drug
MEDI-551 MedImmune CLL Phase II
(anti-CD19 mAb) Gaithersburg, MD (see also hematological, lymphoma) www.medimmune.com
Leukemia
Product Name Sponsor Indication Development Status
Mekinist® GlaxoSmithKline hairy cell leukemia (BRAF-V600E-mutation) Phase II
trametinib Research Triangle Park, NC www.gsk.com
MGD006 MacroGenics AML Phase I
(CD123/CD3 bispecific antibody) Rockville, MD www.macrogenics.com
milatuzumab-doxorubicin Immunomedics CLL Phase I/II
(antibody-drug conjugate) Morris Plains, NJ (see also lymphoma) www.immunomedics.com
MLN4924 Millennium Pharmaceuticals AML Phase I
(Nedd8-activating enzyme inhibitor) Cambridge, MA www.millennium.com
mogamulizumab Kyowa Hakko Pharma adult T-cell leukemia Phase II
(anti-CCR4 humanized antibody) Princeton, NJ (see also lymphoma) www.kyowa-kirin.com
Orphan Drug
MOR208 MorphoSys ALL, CLL Phase II
(CD19 antigen inhibitor) Martinsried, Germany (see also lymphoma) www.morphosys.com
Orphan Drug Xencor www.xencor.com
Monrovia, VA
moxetumomab pasudotox MedImmune hairy cell leukemia Phase III
(anti-CD22 recombinant immunotoxin) Gaithersburg, MD www.medimmune.com
pediatric ALL Phase II
www.medimmune.com
Leukemia
Product Name Sponsor Indication Development Status
Notch inhibitor Bristol-Myers Squibb lymphoblastic leukemia Phase I
Princeton, NJ www.bms.com
ODSH Cantex Pharmaceuticals AML (1st-line) Phase II
(low anticoagulant heparin derivative) Weston, FL www.cantex.com
Oncoquest™-CLL XEME Biopharma CLL (1st-line) Phase I
personalized CLL vaccine Monmouth Junction, NJ www.xemebiopharma.com
Opdivo™ nivolumab+ Bristol-Myers Squibb CML Phase I
Sprycel® dasatinib Princeton, NJ www.bms.com
otlertuzumab Emergent BioSolutions CLL Phase II
(CD37 protein inhibitor) Rockville, MD www.emergentbiosolutions.com
Orphan Drug
OXi4503 OXiGENE AML Phase I
(vascular disrupting agent) South San Francisco, CA (see also myelodysplastic) www.oxigene.com
Orphan Drug
PF-04449913 Pfizer AML Phase II
(SMO antagonist) New York, NY www.pfizer.com
PKC412 Novartis Pharmaceuticals AML Phase III
(signal transduction inhibitor) East Hanover, NJ www.novartis.com
Orphan Drug
Leukemia
Product Name Sponsor Indication Development Status
PLX3397 Plexxikon relapsed/refractory AML Phase II
(Fms/Kit/Flt3-ITD inhibitor) Berkeley, CA www.plexxikon.com
PLX8394 Plexxikon hairy cell leukemia Phase I
(BRAF inhibitor) Berkeley, CA www.plexxikon.com
PM01183 PharmaMar acute leukemia Phase I
(marine-derived alkylating agent) Madrid, Spain www.pharmamar.com
pracinostat MEI Pharma AML (1st-line) Phase II
(HDAC inhibitor) San Diego, CA (see also myelodysplastic) www.meipharma.com
Orphan Drug
PRI-724 PRISM Pharma AML, CML Phase I/II
(CBP/beta�-catenin inhibitor) Tokyo, Japan www.prismbiolab.com
Promacta® GlaxoSmithKline AML Phase II
eltrombopag Research Triangle Park, NC (see also myelodysplastic) www.gsk.com
Qinprezo™ Sunesis Pharmaceuticals relapsed/refractory AML (combination therapy) Phase III
vosaroxin South San Francisco, CA (Fast Track) www.sunesis.com
(type II DNA topoisomerase inhibitor)
quizartinib Ambit Biosciences relapsed/refractory AML (Fast Track) Phase III
(FLT3 inhibitor) San Diego, CA www.ambitbio.com
Orphan Drug
Leukemia
Product Name Sponsor Indication Development Status
REGN1979 Regeneron Pharmaceuticals CLL Phase I
(anti-CD20/anti-CD3 mAb) Tarrytown, NY (see also lymphoma) www.regeneron.com
Revlimid® Celgene CLL (2nd-line maintenance therapy) Phase III
lenalidomide Summit, NJ (see also lymphoma, myelodysplastic, myeloma) www.celgene.com
Orphan Drug
rexlemestrocel-L Mesoblast ALL or AML in patients undergoing transplantation Phase III
(mesenchymal precursor cells) New York, NY with myeloablative conditioning www.mesoblast.com
Orphan Drug (see also lymphoma)
RG7775 Genentech AML Phase I
(MDM2 IV prodrug) South San Francisco, CA www.gene.com
RP-323 Rich Pharmaceuticals AML Phase II
(tetradecanoylphorbol acetate) Beverly Hills, CA www.richpharmaceuticals.com
sapacitabine Cyclacel Pharmaceuticals AML in elderly (1st-line) Phase III
(DNA-directed DNA polymerase Berkeley Heights, NJ (see also myelodysplastic) www.cyclacel.com
inhibitor)
Orphan Drug
selinexor Karyopharm Therapeutics AML, CLL (Richter's transformation) Phase II
(exportin-1 protein inhibitor/nuclear Newton, MA (see also lymphoma, myeloma) www.karyopharm.com
nuclear inhibitor)
Orphan Drug
SGI110 Astex Pharmaceuticals AML Phase II
(DNMT1 inhibitor) Dublin, CA (see also myelodysplastic) www.astx.com
Leukemia
Product Name Sponsor Indication Development Status
SGN-CD19A Seattle Genetics ALL Phase I
(denintuzumab mafodotin) Bothell, WA (see also lymphoma) www.seattlegenetics.com
SGN-CD33A Seattle Genetics AML Phase I
(antibody-drug conjugate) Bothell, WA www.seattlegenetics.com
SL-401 Stemline Therapeutics relapsed/refractory AML, AML in first complete Phase I/II
(IL-3R inhibitor) New York, NY remission www.stemline.com
Orphan Drug (see also hematological, myeloproliferative)
Sprycel® Bristol-Myers Squibb refractory CML (pediatric) Phase II
dasatinib Princeton, NJ www.bms.com
Synribo® Teva Pharmaceuticals AML in the elderly Phase II
omacetaxine mepesuccinate North Wales, PA (see also myelodysplastic) www.tevapharm.com
Tasigna® Novartis Pharmaceuticals CML (treatment free remission) Phase III
nilotinib East Hanover, NJ www.novartis.com
Orphan Drug
TEN-010 Tensha Therapeutics relapsed/refractory AML Phase I
(BET inhibitor) Cambridge, MA (see also myelodysplastic) www.tenshatherapeutics.com
TG-02 Tragara Pharmaceuticals refractory AML, relapsed CLL Phase I
(CDK inhibitor) Carlsbad, CA (see also myeloma) www.tragarapharma.com
Leukemia
Product Name Sponsor Indication Development Status
TG-1101 TG Therapeutics CLL (combination therapy) Phase III
(anti-CD20 mAb) New York, NY (see also lymphoma) www.tgtherapeutics.com
Orphan Drug
TH-302 EMD Serono advanced leukemia Phase I
(hypoxia-activated prodrug) Rockland, MA (see also myeloma) www.emdserono.com
Threshold Pharmaceuticals www.thresholdpharm.com
South San Francisco, CA
tocedostat CTI BioPharma AML Phase I/II
(aminopeptidase inhibitor) Seattle, WA www.ctibiopharma.com
Orphan Drug Fred Hutchinson Cancer Research Center
Seattle, WA
M.D. Anderson Cancer Center
Houston, TX
volasertib Boehringer Ingelheim Pharmaceuticals AML (Breakthrough Therapy) Phase III
(PLK-1 antagonist) Ridgefield, CT www.boehringer-ingelheim.com
Orphan Drug
WT1 cancer vaccine SELLAS Life Sciences AML Phase I completed
Zurich, Switzerland (see also myeloma) www.sellaslifesciences.com
Memorial Sloan Kettering
Cancer Center
New York, NY
Zydelig® Gilead Sciences CLL (1st-line), relapsed/refractory CLL Phase III
idelalisib Foster City, CA (see also lymphoma) www.gilead.com
Lymphoma
Product Name Sponsor Indication Development Status
abemaciclib Eli Lilly mantle cell lymphoma Phase II
(CDK4/6 inhibitor) Indianapolis, IN www.lilly.com
abexinostat Pharmacyclics follicular lymphoma, Phase II
(HDAC inhibitor) Sunnyvale, CA mantle cell lymphoma www.pharmacyclics.com
ABT-199/GDC-0199 AbbVie diffuse large B-cell lymphoma (DLBCL), Phase II
(Bcl-2 inhibitor) North Chicago, IL relapsed/refractory follicular lymphoma www.abbvie.com
Genentech (combination therapy) www.gene.com
South San Francisco, CA (see also hematological, leukemia)
ACP-196 Acerta Pharma mantle cell lymphoma Phase II
(Btk inhibitor) San Carlos, CA (see also leukemia) www.acerta-pharma.com
DLBCL, follicular lymphoma, Phase I
Waldenström macroglobulinemia www.acerta-pharma.com
ACP-319 Acerta Pharma non-Hodgkin lymphoma (NHL) (combination therapy) Phase I/II
San Carlos, CA (see also leukemia, myeloma) www.acerta-pharma.com
Adcetris® Seattle Genetics post-transplant Hodgkin lymphoma relapse Phase III
brentuximab vedotin Bothell, WA prevention, relapsed CD30-positive cutaneous www.seattlegenetics.com
Orphan Drug T-cell lymphoma (CTCL) , Hodgkin lymphoma (1st-line
with chemotherapy), CD30-positive mature T-cell
lymphoma (1st-line with chemotherapy) (Fast Track)
relapsed/refractory CD30-positive NHL, Phase II
Hodgkin lymphoma (age 60 and over), www.seattlegenetics.com
DLBCL (1st-line), Hodgkin lymphoma (2nd-line)
Lymphoma
Product Name Sponsor Indication Development Status
ADI-PEG 20 Polaris Pharmaceuticals NHL Phase II
(PEG arginine deiminase) San Diego, CA (see also leukemia) www.polarispharma.com
AEB071 Novartis Pharmaceuticals CD79-mutant or ABC-subtype DLBCL Phase I/II
(Pkc inhibitor) East Hanover, NJ (combination therapy) www.novartis.com
Afinitor® Novartis Pharmaceuticals DLBCL Phase III
everolimus East Hanover, NJ www.novartis.com
AFM 13 Affimed Therapeutics Hodgkin lymphoma Phase I
(CD30 antigen modulator) Heidelberg, Germany www.affimed.com
Orphan Drug Leukemia & Lymphoma Society www.lls.org
White Plains, NY
alisertib Millennium Pharmaceuticals relapsed/refractory peripheral T-cell lymphoma Phase III
(aurora A kinase inhibitor) Cambridge, MA (PTCL) www.millennium.com
anti-CD22-CAR transduced T-cells Opus Bio NHL Phase I
Gaithersburg, MD (see also leukemia) www.opusbio.com
National Cancer Institute
Bethesda, MD
AR-12 Arno Therapeutics lymphoma Phase I
(PI3K/Akt inhibitor) Flemington, NJ www.arnothera.com
ARGX-110 arGEN-X Waldenström macroglobulinemia Phase I
(anti-CD70 antibody) Breda, Netherlands www.argen-x.com
Leukemia & Lymphoma Society www.lls.org
White Plains, NY
Lymphoma
Product Name Sponsor Indication Development Status
Arzerra® GlaxoSmithKline relapsed DLBCL, relapsed/refractory follicular Phase III
ofatumumab Research Triangle Park, NC lymphoma www.gsk.com
(see also leukemia)
ATTCK20 Unum Therapeutics NHL Phase I
(viral-ACTR+anti-CD20) Cambridge, MA (see also leukemia) www.unumrx.com
bendamustine ready-to-dilute Eagle Pharmaceuticals NHL application submitted
liquid formulation Woodcliff Lake, NJ (see also leukemia) www.eagleus.com
Orphan Drug
BiovaxID® Biovest International follicular NHL (Fast Track), Phase III
dasiprotimut T Minneapolis, MN mantle cell lymphoma www.biovest.com
(personalized cancer vaccine)
Orphan Drug
Blincyto™ Amgen NHL Phase II
blinatumomab Thousand Oaks, CA www.amgen.com
Orphan Drug
CBL0137 Cleveland BioLabs refractory lymphoma Phase I
(apoptosis stimulant) Buffalo, NY www.cbiolabs.com
CC-122 Celgene DLBCL Phase I
(signal transduction pathway modulator) Summit, NJ www.celgene.com
cerdulatinib Portola Pharmaceuticals B-cell NHL, small lymphocytic lymphoma Phase I
(dual Syk/JAK inhibitor) South San Francisco, CA (see also leukemia) www.portola.com
Lymphoma
Product Name Sponsor Indication Development Status
CMD-003 Cell Medica Epstein-Barr virus-positive extranodal Phase II
(cell therapy) London, England NK/T-cell lymphoma www.cellmedica.co.uk
copanlisib Bayer HealthCare Pharmaceuticals NHL Phase II
(PI3K inhibitor) Whippany, NJ www.bayerpharma.com
CPI-0610 Constellation Pharmaceuticals lymphoma Phase I
(BET inhibitor) Cambridge, MA (see also leukemia, myelodysplastic, myeloma, www.constellationpharma.com
Leukemia & Lymphoma Society myeloproliferative) www.lls.org
White Plains, NY
CPI-613 Cornerstone Pharmaceuticals advanced lymphoma Phase I/II
(altered Energy metabolism-derived Cranbury, NJ www.cornerstonepharma.com
[AEMD] compound)
CTL019 Novartis Pharmaceuticals DLBCL Phase I
(chimeric antigen receptor T-cell East Hanover, NJ (see also leukemia) www.novartis.com
therapy)
CUDC-427 Curis late-stage lymphoma Phase I
(IAP inhibitor) Lexington, MA www.curis.com
CUDC-907 Curis relapsed/refractory lymphoma Phase I
(HDAC/pan-PI3K inhibitor) Lexington, MA (see also myeloma) www.curis.com
Leukemia & Lymphoma Society www.lls.org
White Plains, NY
DCR-MYC Dicerna Pharmaceuticals NHL Phase I
(c-myc inhibitor) Watertown, MA (see also myeloma) www.dicerna.com
Lymphoma
Product Name Sponsor Indication Development Status
Debio 1143 Debiopharma lymphoma Phase I
(IAP inhibitor) Lausanne, Switzerland www.debiopharm.com
DI-Leu16-IL2 ALOPEXX Oncology NHL Phase I/II
(CD20 antigen inhibitor) Concord, MA www.alopexx.com
DS-3032 Daiichi Sankyo lymphoma Phase I
(MDM2 inhibitor) Parsippany, NJ www.dsi.com
DS-3078 Daiichi Sankyo lymphoma Phase I
(mTOR inhibitor) Parsippany, NJ www.dsi.com
DS-8273 Daiichi Sankyo lymphoma Phase I
(anti-DR5 antibody) Parsippany, NJ www.dsi.com
duvelisib AbbVie previously-treated follicular lymphoma Phase III
(PI3K-delta/gamma inhibitor) North Chicago, IL (see also hematological, leukemia) www.abbvie.com
Orphan Drug Infinity Pharmaceuticals www.infi.com
Cambridge, MA
refractory indolent NHL Phase II
www.abbvie.com
www.infi.com
treatment naïve follicular lymphoma Phase I
(combination therapy) www.abbvie.com
www.infi.com
Lymphoma
Product Name Sponsor Indication Development Status
E7438/EPZ-6438 Eisai DLBCL Phase I/II
(EZH2 enzyme inhibitor) Woodcliff Lake, NJ www.eisai.com
Epizyme www.epizyme.com
Cambridge, MA
Folotyn® Spectrum Pharmaceuticals PTCL (1st-line) Phase III
pralatrexate injection Henderson, NV www.spprix.com
Orphan Drug
Gazyva® Genentech DLBCL (1st-line), refractory indolent NHL, Phase III
obinutuzumab South San Francisco, CA follicular lymphoma (1st-line) www.gene.com
GDC-0575 Genentech lymphoma Phase I
(ChK1 inhibitor) South San Francisco, CA www.gene.com
GSK2816126 GlaxoSmithKline relapsed/refractory DLBCL, transformed follicular Phase I
(EZH2 enzyme inhibitor/histone Research Triangle Park, NC lymphoma www.gsk.com
modulator)
Imbruvica® Janssen Research & Development relapsed/refractory mantle cell lymphoma, Phase III
ibrutinib Raritan, NJ treatment naïve mantle cell lymphoma, www.janssenrnd.com
Pharmacyclics (combination therapy), relapsed/refractory www.pharmacyclics.com
Sunnyvale, CA indolent NHL (combination therapy), newly-
diagnosed DLBCL (combination therapy), previously
treated Waldenström macroglobulinemia
(combination therapy)
(see also leukemia)
IMGN529 ImmunoGen NHL Phase I
(CD37 protein inhibitor) Waltham, MA www.immunogen.com
Lymphoma
Product Name Sponsor Indication Development Status
IMMU-114 Immunomedics NHL Phase I
(anti-HLA-DR antibody) Morris Plains, NJ (see also leukemia) www.immunomedics.com
IMO-8400 Idera Pharmaceuticals DLBCL (MYD88 L265P oncogenic mutation), Phase I/II
(TLR antagonist) Cambridge, MA Waldenström macroglobulinemia www.iderapharma.com
Orphan Drug
Imprime PGG® Biothera advanced indolent NHL Phase II
intravenous immunostimulant Eagan, MN (see also leukemia) www.biothera.com
Dana-Farber Cancer Institute
Boston, MA
interleukin-12 gene therapy OncoSec Medical CTCL Phase II
San Diego, CA www.oncosec.com
Istodax® Celgene PTCL (1st-line) Phase III
romidepsin Summit, NJ www.celgene.com
Orphan Drug
JCAR015 Juno Therapeutics relapsed/refractory NHL (post autologous treatment) Phase I
(T lymphocyte immunotherapy) Seattle, WA (see also leukemia) www.junotherapeutics.com
JNJ-26481585 Janssen Research & Development CTCL Phase II
(HDAC inhibitor) Raritan, NJ www.janssenrnd.com
JNJ-42756493 Janssen Research & Development late-stage lymphoma Phase I
(FGFR inhibitor) Raritan, NJ www.janssenrnd.com
Lymphoma
Product Name Sponsor Indication Development Status
KTE-C19 CAR Kite Pharma refractory aggressive NHL Phase I/II
(chimeric antigen receptor) Santa Monica, CA www.kitepharma.com
Orphan Drug National Cancer Institute
Bethesda, MD
LY2780301 Eli Lilly NHL Phase I
(p70S6/AKT dual inhibitor) Indianapolis, IN www.lilly.com
LY3039478 Eli Lilly lymphoma Phase I
(NOTCH inhibitor) Indianapolis, IN www.lilly.com
Marqibo® Spectrum Pharmaceuticals aggressive NHL (1st-line) Phase III
vinCRIStine sulfate LIPOSOME Henderson, NV (see also leukemia) www.sppirx.com
injection
MEDI-551 MedImmune DLBCL Phase II
(anti-CD19 mAb) Gaithersburg, MD (see also hematological, leukemia) www.medimmune.com
MEDI-551+MEDI0680 MedImmune DLBCL Phase I
(anti-CD19 mAb+anti-CD20 mAb) Gaithersburg, MD (see also hematological, leukemia) www.medimmune.com
milatuzumab-doxorubicin Immunomedics NHL Phase I/II
(antibody-drug conjugate) Morris Plains, NJ (see also leukemia) www.immunomedics.com
mocetinostat Mirati Therapeutics DLBCL Phase II
(HDAC inhibitor) San Diego, CA (see also myelodysplastic) www.mirati.com
Orphan Drug
Lymphoma
Product Name Sponsor Indication Development Status
mogamulizumab Kyowa Hakko Pharma CTCL Phase III
(anti-CCR4 humanized antibody) Princeton, NJ (see also leukemia) www.kyowa-kirin.com
Orphan Drug
adult T-cell lymphoma Phase II
www.kyowa-kirin.com
MOR208 MorphoSys NHL Phase II
(CD19 antigen inhibitor) Martinsried, Germany (see also leukemia) www.morphosys.com
Orphan Drug Xencor www.xencor.com
Monrovia, CA
Oncoquest™-L XEME Biopharma follicular NHL Phase II
personalized lymphoma vaccine Monmouth Junction, NJ www.xemebiopharma.com
Opdivo™ Bristol-Myers Squibb DLBCL, follicular NHL, Phase II
nivolumab Princeton, NJ Hodgkin lymphoma (Breakthrough Therapy) www.bms.com
(see also hematological, leukemia, myeloma)
Pixuvri® CTI BioPharma DLBCL (combination therapy) Phase III
pixantrone Seattle, WA www.ctibiopharma.com
PNT2258 ProNAi Therapeutics relapsed/refractory DLBCL, Phase II
(bcl-2 oncogene inhibitor) Plymouth, MI relapsed/refractory NHL www.pronai.com
polatuzumab vedotin Genentech DLBCL, NHL Phase II
(anti-CD79b antibody-drug conjugate) South San Francisco, CA www.gene.com
Lymphoma
Product Name Sponsor Indication Development Status
REGN1979 Regeneron Pharmaceuticals NHL Phase I
(anti-CD20/anti-CD3 mAb) Tarrytown, NY (see also leukemia) www.regeneron.com
Resimmune® Angimmune CTCL Phase II
anti-CD3 immunotoxin Rockville, MD www.angimmune.com
Revlimid® Celgene DLBCL (maintenance treatment), follicular lymphoma, Phase III
lenalidomide Summit, NJ relapsed/refractory indolent lymphoma www.celgene.com
(see also leukemia, myelodysplastic, myeloma)
DLBCL-ABC subtype (1st-line) Phase II
www.celgene.com
rexlemestrocel-L Mesoblast NHL in patients undergoing transplantation with Phase III
(mesenchymal precursor cells) New York, NY myeloablative conditioning www.mesoblast.com
Orphan Drug (see also leukemia)
RG7446 Genentech lymphoma (combination therapy) Phase I
(PD-L1 mAb) South San Francisco, CA www.gene.com
ricolinostat (ACY-1215) Acetylon Pharmaceuticals lymphoma Phase I/II
(HDAC inhibitor) Boston, MA (see also myeloma) www.acetylon.com
rituximab biosimilar Amgen NHL Phase III
(APB-798) Thousand Oaks, CA www.amgen.com
rituximab biosimilar Boehringer Ingelheim Pharmaceuticals follicular lymphoma (1st-line) Phase I
(BI 695500) Ridgefield, CT www.boehringer-ingelheim.com
Lymphoma
Product Name Sponsor Indication Development Status
rituximab biosimilar Merck follicular lymphoma (1st-line) Phase I completed
(MK-8808) Whitehouse Station, NJ www.merck.com
rituximab biosimilar Pfizer follicular lymphoma (1st-line) Phase III
(PF-05280586) New York, NY www.pfizer.com
RRx-001 EpicentRx late-stage lymphoma Phase I
(free radical stimulant) Mountain View, CA www.epicentrx.com
SD101 Dynavax low-grade recurrent B-cell lymphoma Phase I/II
(toll-like receptor 9 agonist) Berkeley, CA www.dynavax.com
selinexor Karyopharm Therapeutics DLBCL Phase II
(exportin-1 protein inhibitor/nuclear Newton, MA (see also leukemia, myeloma) www.karyopharm.com
nuclear inhibitor)
Orphan Drug
SGN-CD19A Seattle Genetics NHL Phase I
(denintuzumab mafodotin) Bothell, WA (see also leukemia) www.seattlegenetics.com
SGN-CD70A Seattle Genetics NHL Phase I
(antibody-drug conjugate) Bothell, WA www.seattlegenetics.com
SGX301 Soligenix CTCL (1st-line) Phase II
(photosensitizer) Princeton, NJ (Fast Track) www.soligenix.com
Orphan Drug
Lymphoma
Product Name Sponsor Indication Development Status
SHAPE TetraLogic Pharmaceuticals CTCL Phase II
(HDAC inhibitor) Malvern, PA www.tetralogicpharma.com
Orphan Drug
SNDX-275 Syndax Pharmaceuticals relapsed/refractory Hodgkin lymphoma Phase II
(HDAC inhibitor) Waltham, MA www.syndax.com
TG-1101 TG Therapeutics mantle cell lymphoma (combination therapy) Phase II
(anti-CD20 mAb) New York, NY (see also leukemia) www.tgtherapeutics.com
Orphan Drug
Treanda® Teva Pharmaceuticals advanced indolent NHL (1st-line) application submitted
bendamustine North Wales, PA www.tevapharm.com
Orphan Drug
relapsed/refractory mantle cell lymphoma Phase II
(combination therapy) www.tevapharm.com
TSR-011 TESARO lymphoma Phase I/II
(ALK/TRK inhibitor) Waltham, MA www.tesaro.com
veltuzumab Immunomedics NHL (combination therapy) Phase II
(humanized anti-CD20 mAb) Morris Plains, NJ www.immunomedics.com
VS-5584 Verastem late-stage lymphoma Phase I
(PI3K/mTORC inhibitor) Needham, MA www.verastem.com
Lymphoma
Product Name Sponsor Indication Development Status
Zydelig® Gilead Sciences indolent NHL Phase III
idelalisib Foster City, CA (see also leukemia) www.gilead.com
indolent NHL (1st-line) Phase II
www.gilead.com
Myelodysplastic Syndromes
Product Name Sponsor Indication Development Status
ASTX727 Astex Pharmaceuticals myelodysplastic syndromes Phase I/II
(DNA methylation inhibitor/nucleoside Dublin, CA www.astx.com
deaminase inhibitor fixed-dose
combination)
BI 836858 Boehringer Ingelheim Pharmaceuticals myelodysplastic syndromes Phase II
Ridgefield, CT (see also leukemia) www.boehringer-ingelheim.com
birinapant TetraLogic Pharmaceuticals myelodysplastic syndromes Phase II
(apoptosis stimulator) Malvern, PA www.tetralogicpharma.com
BP-100-1.01 Bio-Path Holdings myelodysplastic syndromes Phase I
(liposomal Grb-2) Houston, TX (see also leukemia) www.biopathholdings.com
BVD-523 BioMed Valley Discoveries myelodysplastic syndromes Phase I/II
(ERK 1/2 kinase inhibitor) Kansas City, MO (see also leukemia) www.biomed-valley.com
CC-486 Celgene myelodysplastic syndromes (lower-risk) (Fast Track) Phase III
(azacitidine) Summit, NJ (see also leukemia) www.celgene.com
Myelodysplastic Syndromes
Product Name Sponsor Indication Development Status
CPI-0610 Constellation Pharmaceuticals myelodysplastic syndromes Phase I
(BET protein inhibitor) Cambridge, MA (see also leukemia, lymphoma, myeloma, www.constellationpharma.com
Leukemia & Lymphoma Society myeloproliferative) www.lls.org
White Plains, NY
galunisertib Eli Lilly myelodysplastic syndromes Phase II/III
(TGF-beta inhibitor) Indianapolis, IN www.lilly.com
KB004 KaloBios Pharmaceuticals EphA3-positive myelodysplastic syndromes Phase II
(EphA3 receptor antagonist) South San Francisco, CA (see also leukemia, myeloproliferative) www.kalobios.com
LGH447 Novartis Pharmaceuticals myelodysplastic syndromes Phase I
(proto oncogene protein c East Hanover, NJ (see also leukemia, myeloma) www.novartis.com
pim-1 inhibitor)
luspatercept (ACE-536) Acceleron Pharma myelodysplastic syndromes Phase II
(TGF-ß protein super family inhibitor) Cambridge, MA www.acelleronpharma.com
Orphan Drug Celgene www.celgene.com
Summit, NJ
MEDI4736 MedImmune myelodysplastic syndromes Phase I
(anti-PD-L1 mAb) Gaithersburg, MD www.medimmune.com
mocetinostat Mirati Therapeutics myelodysplastic syndromes Phase II
(HDAC inhibitor) San Diego, CA (see also lymphoma) www.mirati.com
Orphan Drug
OPN-305 Opsona Therapeutics myelodysplastic syndromes Phase I/II
(toll-like receptor 2 antagonist) Dublin, Ireland www.opsona.com
Myelodysplastic Syndromes
Product Name Sponsor Indication Development Status
OXi4503 OXiGENE myelodysplastic syndromes Phase I
(vascular disrupting agent) South San Francisco, CA (see also leukemia) www.oxigene.com
pracinostat MEI Pharma myelodysplastic syndromes (1st-line), Phase II
(HDAC inhibitor) San Diego, CA refractory myelodysplastic syndromes www.meipharma.com
(see also leukemia)
Promacta® GlaxoSmithKline myelodysplastic syndromes Phase II
eltrombopag Research Triangle Park, NC (see also leukemia) www.gsk.com
Revlimid® Celgene myelodysplastic syndromes (non-deletion 5q) Phase III
lenalidomide Summit, NJ (see also leukemia, lymphoma, myeloma) www.celgene.com
Orphan Drug
rigosertib Onconova Therapeutics high-risk and low-risk Phase III
(PI3K/Plk1 inhibitor) Newtown, PA myelodysplastic syndromes www.onconova.com
Orphan Drug
sapacitabine Cyclacel Pharmaceuticals myelodysplastic syndromes in the elderly Phase II
(DNA-directed DNA polymerase Berkeley Heights, NJ (see also leukemia) www.cyclacel.com
inhibitor)
Orphan Drug
SGI110 Astex Pharmaceuticals myelodysplastic syndromes Phase II
(DNMT1 inhibitor) Dublin, CA (see also leukemia) www.astx.com
Myelodysplastic Syndromes
Product Name Sponsor Indication Development Status
sotatercept (ACE-011) Acceleron Pharma myelodysplastic syndromes Phase II
(activin inhibitor) Cambridge, MA www.acceleronpharma.com
Celgene www.celgene.com
Summit, NJ
Synribo® Teva Pharmaceuticals myelodysplastic syndromes Phase II
omacetaxine mepesuccinate North Wales, PA (see also leukemia) www.tevapharm.com
Orphan Drug
TEN-010 Tensha Therapeutics refractory myelodysplastic syndromes Phase I
(BET inhibitor) Cambridge, MA (see also leukemia) www.tenshatherapeutics.com
Myeloma
Product Name Sponsor Indication Development Status
ACP-319 Acerta Pharma multiple myeloma (combination therapy) Phase I/II
San Carlos, CA (see also leukemia, lymphoma) www.acerta-pharma.com
afuresertib GlaxoSmithKline multiple myeloma Phase I
(AKT inhibitor) Research Triangle Park, NC www.gsk.com
ALT-803 Altor BioScience multiple myeloma Phase I/II
(IL-15 superagonist complex) Miramar, FL (see also hematological) www.altorbioscience.com
Aplidin® PharmaMar multiple myeloma Phase III
plitidepsin Madrid, Spain www.pharmamar.com
Orphan Drug
Myeloma
Product Name Sponsor Indication Development Status
AT7519 Astex Pharmaceuticals multiple myeloma Phase II
(CDK inhibitor) Dublin, CA www.astx.com
Novartis Pharmaceuticals www.novartis.com
East Hanover, NJ
BYL719 Novartis Pharmaceuticals relapsed/refractory multiple myeloma Phase I/II
(PI3K-alpha inhibitor) East Hanover, NJ (combination therapy) www.novartis.com
CB-5083 Cleave Biosciences multiple myeloma Phase I
(CDC48/p97 inhibitor) Burlingame, CA www.cleavebio.com
CPI-0610 Constellation Pharmaceuticals multiple myeloma Phase I
(BET protein inhibitor) Cambridge, MA (see also leukemia, lymphoma, myelodysplastic, www.constellationpharma.com
Leukemia & Lymphoma Society myeloproliferative) www.lls.org
White Plains, NY
CUDC-907 Curis multiple myeloma Phase I
(HDAC/pan-PI3K inhibitor) Lexington, MA (see also lymphoma) www.curis.com
Leukemia & Lymphoma Society www.lls.org
White Plains, NY
daratumumab Janssen Research & Development relapsed/refractory multiple myeloma (1st-line) Phase III
(IgG1K mAb) Raritan, NJ (combination therapy) www.janssenrnd.com
multiple myeloma (2nd-line) (Fast Track) Phase II
www.janssenrnd.com
DCR-MYC Dicerna Pharmaceuticals multiple myeloma Phase I
(c-myc inhibitor) Watertown, MA (see also lymphoma) www.dicerna.com
Myeloma
Product Name Sponsor Indication Development Status
DKN-01 HealthCare Pharmaceuticals multiple myeloma Phase I/II
(DKK1 protein inhibitor) Cambridge, MA www.healthcarepharmaceuticals.com
elotuzumab AbbVie multiple myeloma (1st-line) (combination therapy), Phase III
North Chicago, IL relapsed/refractory multiple myeloma www.abbvie.com
Bristol-Myers Squibb (combination therapy) (Breakthrough Therapy) www.bms.com
Princeton, NJ
multiple myeloma (2nd-line) Phase II
www.abbvie.com
www.bms.com
filanesib Array BioPharma multiple myeloma Phase II
(KSP inhibitor) Boulder, CO www.arraybiopharma.com
GSK2110183 GlaxoSmithKline multiple myeloma Phase I
(AKT inhibitor) Research Triangle Park, NC www.gsk.com
GSK2857916 GlaxoSmithKline relapsed/refractory multiple myeloma Phase I
(antibody-drug conjugate) Research Triangle Park, NC www.gsk.com
indatuximab ravtansine Biotest multiple myeloma Phase I/II
(BT-062) Dreieich, Germany www.biotest.de
Orphan Drug ImmunoGen www.immunogen.com
Waltham, MA
ixazomib Millennium Pharmaceuticals relapsed/refractory multiple myeloma, Phase III
(proteasome inhibitor) Cambridge, MA previously untreated multiple myeloma, www.millennium.com
Orphan Drug maintenance therapy in myeloma following
autologous stem cell transplant
Myeloma
Product Name Sponsor Indication Development Status
Keytruda® Merck multiple myeloma (combination therapy) Phase I
pembrolizumab Whitehouse Station, NJ (see also hematological) www.merck.com
Kyprolis® Onyx Pharmaceuticals multiple myeloma (1st-line), relapsed multiple Phase III
carfilzomib South San Francisco, CA myeloma (combination therapy) www.onyx.com
Orphan Drug
LGH447 Novartis Pharmaceuticals multiple myeloma (combination therapy) Phase I/II
(proto oncogene protein c East Hanover, NJ (see also leukemia, myelodysplastic) www.novartis.com
pim-1 inhibitor)
lirilumab+elotuzumab Bristol-Myers Squibb multiple myeloma Phase I
Princeton, NJ www.bms.com
lirilumab+Opdivo™ Bristol-Myers Squibb multiple myeloma Phase I
nivolumab Princeton, NJ www.bms.com
marizomib intravenous Triphase Accelerator relapsed/refractory multiple myeloma Phase II
(proteasome inhibitor) San Diego, CA www.triphaseco.com
Orphan Drug
melphalan intravenous Spectrum Pharmaceuticals multiple myeloma application submitted
(Captisol-Enabled™ melphalan) Henderson, NV www.spprix.com
Orphan Drug
MOR202 Celgene multiple myeloma Phase I
(anti-CD38 antibody) Summit, NJ www.celgene.com
MorphoSys
Martinsried, Germany
Myeloma
Product Name Sponsor Indication Development Status
NY-ESO-1/LAGE-1-specific T-cells Adpatimmune multiple myeloma Phase I/II
Oxon, United Kingdom www.adaptimmune.com
oprozomib Onyx Pharmaceuticals multiple myeloma (combination therapy) Phase I/II
(proteasome inhibitor) South San Francisco, CA (see also hematological) www.onyx.com
Orphan Drug
PVX-410 OncoPep smoldering multiple myeloma Phase I/II
(therapeutic vaccine) North Andover, MA www.oncopep.com
Orphan Drug Leukemia & Lymphoma Society www.lls.org
White Plains, NY
Revlimid® Celgene multiple myeloma (newly diagnosed) application submitted
lenalidomide Summit, NJ (see also leukemia, lymphoma, myelodysplastic) www.celgene.com
Orphan Drug
multiple myeloma (maintenance therapy) Phase III
www.celgene.com
ricolinostat (ACY-1215) Acetylon Pharmaceuticals relapsed/refractory multiple myeloma Phase II
(HDAC inhibitor) Boston, MA (see also lymphoma) www.acetylon.com
Leukemia & Lymphoma Society www.lls.org
White Plains, NY
SAR650984 Sanofi US multiple myeloma Phase II
(anti-CD38 naked mAb) Bridgewater, NJ www.sanofi.com
Myeloma
Product Name Sponsor Indication Development Status
selinexor Karyopharm Therapeutics multiple myeloma Phase II
(exportin-1 protein inhibitor/nuclear Newton, MA (see also leukemia, lymphoma) www.karyopharm.com
nuclear inhibitor)
Orphan Drug
Sylvant® Janssen Research & Development smoldering multiple myeloma Phase II
siltuximab Raritan, NJ www.janssenrnd.com
TAS-120 Taiho Oncology multiple myeloma Phase I/II
(FGF/FGFR inhibitor) Princeton, NJ www.taihooncology.com
TG-02 Tragara Pharmaceuticals multiple myeloma Phase I
(CDK inhibitor) Carlsbad, CA (see also leukemia) www.tragarapharma.com
TH-302 EMD Serono multiple myeloma Phase I/II
(hypoxia-activated prodrug) Rockland, MA (see also leukemia) www.emdserono.com
Threshold Pharmaceuticals www.thresholdpharm.com
South San Francisco, CA
urelumab+elotuzumab Bristol-Myers Squibb multiple myeloma Phase I
Princeton, NJ www.bms.com
WT1 cancer vaccine SELLAS Life Sciences multiple myeloma Phase I completed
Zurich, Switzerland (see also leukemia) www.sellaslifesciences.com
Memorial Sloan Kettering
Cancer Center
New York, NY
Myeloma
Product Name Sponsor Indication Development Status
Zelboraf® Genentech multiple myeloma (BRAF V600 mutation-positive) Phase II
vemurafenib South San Francisco, CA www.gene.com
Zolinza® Merck multiple myeloma (combination therapy) Phase III
vorinostat Whitehouse Station, NJ www.merck.com
Orphan Drug
Myeloproliferative Neoplasms
Product Name Sponsor Indication Development Status
CPI-0610 Constellation Pharmaceuticals myeloproliferative neoplasms Phase I
(BET protein inhibitor) Cambridge, MA (see also leukemia, lymphoma, www.constellationpharma.com
myelodysplastic, myeloma)
imetelstat Geron myelofibrosis (1st-line) Phase II
(telomerase inhibitor) Menlo Park, CA www.geron.com
Janssen Research & Development www.janssenrnd.com
Raritan, NJ
KB004 KaloBios Pharmaceuticals EphA3-positive myelofibrosis Phase II
(EphA3 receptor antagonist) South San Francisco, CA (see also leukemia, myelodysplastic) www.kalobios.com
LCL161 Novartis Pharmaceuticals myelofibrosis Phase II
(IAP inhibitor) East Hanover, NJ www.novartis.com
M.D. Anderson Cancer Center
Houston, TX
LDE225 Novartis Pharmaceuticals myelofibrosis (combination therapy) Phase I/II
(Smo antagonist) East Hanover, NJ (see also hematological, leukemia) www.novartis.com
Myeloproliferative Neoplasms
Product Name Sponsor Indication Development Status
momelotinib Gilead Sciences myelofibrosis Phase III
(JAK inhibitor) Foster City, CA www.gilead.com
Orphan Drug
NS-018 NS Pharma myelofibrosis Phase I/II
(JAK2 inhibitor) Paramus, NJ www.nippon-shinyaku.co.jp
pacritinib Baxter International myelofibrosis (Fast Track) Phase III
(JAK2/FLT3 dual inhibitor) Deerfield, IL www.baxter.com
Orphan Drug CTI BioPharma www.ctibiopharma.com
Seattle, WA
PRM-151 Promedior myelofibrosis (Fast Track) Phase II
(rhPTX-2 for injection) Lexington, MA www.promedior.com
Orphan Drug
SL-401 Stemline Therapeutics advanced high-risk myeloproliferative neoplasms Phase I/II
(IL-3R inhibitor) New York, NY (see also hematological, leukemia) www.stemline.com
Definitions
Application Submitted—An application for marketing has been submitted by the company to the U.S. Food and Drug Administration (FDA).
Breakthrough Therapy—Upon request by a sponsor, the FDA can grant this designation to expedite the development and review of a drug or biologic intended, alone or in
combination with one or more other drugs, to treat a serious or life threatening disease or condition and preliminary clinical evidence indicates that it may demonstrate substantial
improvement over existing therapies on one or more clinically-significant endpoints, such as substantial treatment effects observed early in clinical development. If a drug or
biologic is designated as a breakthrough therapy, the FDA will expedite the development and review. With this designation, all Fast Track features convey to the medicine.
Fast Track—Upon request by a sponsor, the FDA can grant this designation to facilitate the development and expedite the review of a drug or biologic to treat a serious
condition and fill an unmet medical need. When considering a biopharmaceutical company’s request for Fast Track designation for an investigational drug or biologic, the
FDA evaluates whether it will affect factors such as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe
condition to a more serious one, and whether a condition can be adequately addressed by available therapy. With Fast Track designation, early and frequent
communication between the FDA and the biopharmaceutical company is encouraged throughout the entire drug development and review process to help to quickly resolve
any questions or issues that arise, potentially leading to an earlier approval and access by patients.
Orphan Designation—Upon request by a sponsor, the FDA can grant special status (“orphan status”) to a drug or biologic to treat a rare disease or condition. In order
to receive an orphan designation, a qualifying drug or biologic must be intended for the treatment, diagnosis, or prevention of a rare disease or condition that affects
fewer than 200,000 people in the United States, or that affects more than 200,000 persons but is not expected to recover the costs of its development and marketing.
Phase I—Researchers test the investigational drug or biologic in a small group of people, usually between 20 and 100 healthy adult volunteers, to evaluate its initial
safety and tolerability profile, determine a safe dosage range, and identify potential side effects.
Phase II—The investigational drug or biologic is given to volunteer patients, usually between 100 and 500, to determine whether it is effective, identify an optimal dose,
and to further evaluate its short-term safety.
Phase III—The investigational drug or biologic is given to a larger, more diverse patient population, often involving between 1,000 and 5,000 patients (but sometimes many more),
to generate statistically significant evidence to confirm its safety and effectiveness. Phase III studies are the longest studies and usually take place in multiple sites around the world.
The content of this report has been
obtained through public, government and
industry sources, and the Adis “R&D
Insight” database based on the latest
information. Report current as of February
24, 2015. The medicines in this listing
include medicines being developed by U.S.-
based companies conducting trials in the
United States and abroad, PhRMA-member
companies conducting trials in the United
States and abroad, and foreign companies
conducting clinical trials in the United
States. The information may not be
comprehensive. For more specific
information about a particular product,
contact the individual company directly or
go to www.clinicaltrials.gov. The entire
series of Medicines in Development is
available on PhRMA's website.