2015 Medicines in Development -- Leukemia & Lymphoma A 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.