2020 medicines in development ꟷ cancer - phrma...(anti-pd1 mab) north chicago, il solid tumors...
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2020 Medicines in Development ꟷ Cancer
Product Name Sponsor Indication Development Status
177-Lu-edotretide ITM Solucin neuroendocrine tumors Phase III
(peptide receptor radionucleotide Munich, Germany www.itm-radiopharm.com
therapy)
177-Lu-NeoB Advanced Accelerator Applications solid tumors Phase I
(radioligand therapy target GRPR) Millburn, NJ www.adacap.com
177-Lu-PSMA-617 Advanced Accelerator Applications metastatic castration-resistant Phase III
(targeted radioligand therapy) Millburn, NJ prostate cancer www.adacap.com
177-Lu-PSMA-R2 Advanced Accelerator Applications prostate cancer Phase I
(radioligand therapy target PSMA) Millburn, NJ www.adacap.com
2X-121 Oncology Venture US metastatic breast cancer, Phase II
(PARP inhibitor) Scottsdale, AZ advanced ovarian cancer www.oncologyventure.com
609A 3SBio solid tumors Phase I
(anti-PD1 antibody) Shenyang, China www.3sbio.com
67Cu-SARTATE™ Clarity Pharmaceuticals neuroblastoma Phase I/II
(peptide receptor radionuclide Sydney, Australia www.claritypharmaceuticals.com
therapy)
Medicines in Development: Cancer ǀ 2020 1
Product Name Sponsor Indication Development Status
7HP349 7 Hills Pharma solid tumors Phase I
(VLA-4/LFA-1 activator) Houston, TX www.7hillspharma.com
9-ING-41 Actuate Therapeutics myelofibrosis Phase II
(GSK-3β inhibitor) Fort Worth, TX www.actuatetherapeutics.com
ORPHAN DRUG
advanced cancers, Phase I
refractory malignancies (pediatric) www.actuatetherapeutics.com
A166 KLUS Pharma HER2-expressing solid tumors Phase I
(antibody-drug conjugate) Cranbury, NJ www.kluspharma.com
AB-106 AnHeart Therapeutics solid tumors Phase I
(ROS1/NTRK inhibitor) Hangzhou, China www.anhearttherapeutics.com
AB-110 Angiocrine hematologic malignancies Phase I
(cell replacement) San Diego, CA www.angiocrinebiosciences.com
AB122 (zimberelimab) Arcus Biosciences advanced solid tumors Phase I
(PD-1 inhibitor) Hayward, CA (monotherapy and combination www.gilead.com
Gilead Sciences therapy)
Foster City, CA
Medicines in Development: Cancer ǀ 2020 2
Product Name Sponsor Indication Development Status
AB154 (domvanalimab) Arcus Biosciences 1L non-small cell lung cancer Phase II
(TIGIT inhibitor) Hayward, CA (NSCLC) (combination therapy) www.gilead.cpom
Gilead Sciences
Foster City, CA
2L+ solid tumors (combination Phase I
therapy) www.gilead.com
AB-205 Angiocrine Bioscience relapsed/refractory lymphoma Phase I/II
(genetically-engineered cell therapy San Diego, CA (+high dose chemotherapy and www.angiocrinebioscience.com
derived from human umbilical vein autologous stem cell transplantation),
tissue) leukemia, myelodysplastic syndromes
(MDS) (+myeloablative allogeneic
double unit umbilical cord blood
transplantation)
AB680 Arcus Biosciences 1L pancreatic ductal adenocarcinoma Phase I
(CD73 inhibitor) Hayward, CA (combination therapy) www.gilead.com
Gilead Sciences
Foster City, CA
AB928 (etrumadenant) Arcus Biosciences 3L colorectal cancer, 1L NSCLC, Phase II
(A2A/A2B receptor antagonist) Hayward, CA pancreatic ductal adenocarcinoma www.gilead.com
Gilead Sciences
Foster City, CA
colorectal cancer, castration-resistant Phase I
prostate cancer, triple negative www.gilead.com
breast cancer, NSCLC
Medicines in Development: Cancer ǀ 2020 3
Product Name Sponsor Indication Development Status
ABBV-011 AbbVie small cell lung cancer (SCLC) Phase I
(antibody-drug conjugate) North Chicago, IL www.abbvie.com
ABBV-151 AbbVie solid tumors Phase I
(GARP- TGF-β1 inhibitor) North Chicago, IL www.abbvie.com
ABBV-155 AbbVie hematologic malignancies, Phase I
(antibody-drug conjugate) North Chicago, IL solid tumors www.abbvie.com
ABBV-181 (budigalimab) AbbVie hematologic malignancies, Phase I
(anti-PD1 mAb) North Chicago, IL solid tumors www.abbvie.com
ABBV-184 AbbVie acute myeloid leukemia (AML), Phase I
(surviving TCR/CD3 T cell engager) North Chicago, IL NSCLC www.abbvie.com
ABBV-368 AbbVie solid tumors (combination therapy) Phase I
(anti-OX40 mAb) North Chicago, IL www.abbvie.com
ABBV-467 AbbVie hematologic malignancies Phase I
(MCL1 protein inhibitor) North Chicago, IL www.abbvie.com
ABBV-621 AbbVie hematologic malignancies, Phase I
(TRAIL receptor agonist) North Chicago, IL solid tumors www.abbvie.com
Medicines in Development: Cancer ǀ 2020 4
Product Name Sponsor Indication Development Status
ABBV-744 AbbVie AML Phase I
(BDII-selective BET inhibitor) North Chicago, IL www.abbvie.com
inhibitor)
ABBV-927 AbbVie solid tumors Phase I
(anti-CD40 mAb) North Chicago, IL www.abbvie.com
ABBV-CLS-579 AbbVie solid tumors Phase I
North Chicago, IL www.abbvie.com
Calico Life Sciences www.calicolabs.com
South San Francisco, CA
ABBV-CX-2029 AbbVie diffuse large B-cell lymphoma (DLBCL), Phase II
(antibody-drug conjugate) North Chicago, IL esophageal cancer, head and neck www.abbvie.com
CytomX Therapeutics cancer, NSCLC www.cytomx.com
South San Francisco, CA
abexinostat Xynomic Pharmaceuticals kidney cancer Phase III
(HDAC inhibitor) Dover, DE www.xynomicpharma.com
follicular lymphoma Phase II
www.xynomicpharma.com
AbGn-107 AltruBio gastrointestinal cancer Phase I
(antibody-drug conjugate) Redwood City, CA www.altrubio.com
Medicines in Development: Cancer ǀ 2020 5
Product Name Sponsor Indication Development Status
abivertinib Sorrento Therapeutics NSCLC Phase III
(EGFR tyrosine kinase inhibitor) San Diego, CA www.sorrentotherapeutics.com
ABL001 (asciminib) Novartis 3L chronic myeloid leukemia (CML) Phase III
(BCR-ABL inhibitor) East Hanover, NJ (Fast Track) www.novartis.com
ABM-1310 ABM Therapeutics solid tumors Phase I
(BRAF inhibitor) San Diego, CA www.abmtx.com
ABP 798 AbbVie chronic lymphocytic leukemia (CLL), application submitted
(rituximab biosimilar) North Chicago, IL non-Hodgkin lymphoma (NHL) www.abbvie.com
Amgen www.amgen.com
Thousand Oaks, CA
ABSK021 Abbisko Therapeutics solid tumors Phase I
(CSF-1R inhibitor) Shanghai, China www.abbisko.com
ABT-165 AbbVie solid tumors Phase I
(DLL4xVEGF bispecific antibody) North Chicago, IL www.abbviecom
ACE1702 Acepodia solid tumors Phase I
(anti-HER2 antibody conjugated Burlingame, CA www.acepodia.com
human natural killer cells)
Medicines in Development: Cancer ǀ 2020 6
Product Name Sponsor Indication Development Status
Actimab-A (CD33) Actinium Pharmaceuticals newly-diagnosed AML (over age 60) Phase II
(antibody warhead enabling [AWE] New York, NY www.actiniumpharma.com
radioimmunoconjugate)
ORPHAN DRUGrelapsed/refractory AML Phase I
(combination therapy) www.actinumpharma.com
Actimab-M (CD33) Actinium Pharmaceuticals multiple myeloma Phase I
(AWE radioimmunoconjugate) New York, NY www.actiniumpharma.com
Actimab-MDS Actinium Pharmaceuticals MDS Phase II
(AWE radioimmunoconjugate) New York, NY www.actiniumpharma.com
Ad/MG1-E6E7 Turnstone Biologics HPV-associated cancers Phase I
(adenovirus vaccine) New York, NY www.turnstonebio.com
Ad/MG1-MAGEA3 Turnstone Biologics NSCLC (combination therapy) Phase I/II
(adenovirus vaccine) New York, NY www.turnstonebio.com
Ad/PNP PNP Therapeutics recurrent head and neck cancer Phase I/II
(purine nucleoside phosphorylase Birmingham, AL www.pnptherapeutics.com
gene therapy)
adagloxad simolenin OBI Pharma triple negative breast cancer Phase III
(cancer immunotherapy) San Diego, CA www.obipharma.com
Medicines in Development: Cancer ǀ 2020 7
Product Name Sponsor Indication Development Status
adavosertib AstraZeneca ovarian cancer, solid tumors Phase II
(WEE1 inhibitor) Wilmington, DE www.astrazeneca.com
Adcetris® Seagen DLBCL (combination therapy) Phase III
brentuximab vedotin Bothell, WA www.seagen.com
1L Hodgkin lymphoma (combination Phase II
therapy), 1L Hodgkin lymphoma, www.seagen.com
peripheral T-cell lymphoma (PTCL),
Hodgkin lymphoma (retreatment),
PTCL (retreatment), Hodgkin lymphoma
(pediatric), melanoma, NSCLC
ADG106 Adagene NHL, solid tumors Phase I
(anti-CD137 mAb) Suzhou, China www.adagene.com
ADG116 Adagene advanced solid tumors Phase I
(anti-CTLA 4 mAb)) Suzhou, China www.adagene.com
ADNIC Sorrento Therapeutics hematologic malignancies, Phase II
(paclitaxel) San Diego, CA solid tumors www.sorrentotherapeutics.com
Ad-p53 MultiVir lymphoma, solid tumors Phase II
(adenoviral p53 gene therapy) Houston, TX www.multivir.com
Medicines in Development: Cancer ǀ 2020 8
Product Name Sponsor Indication Development Status
Ad-p53 DCV MultiVir SCLC (combination therapy) Phase II
(dendritic cell vaccine) Houston, TX www.multivir.com
ADP-A2M4 Adaptimmune head and neck cancer, myxoid Phase II
(MAGE A4 T cell therapy) Philadelphia, PA liposarcoma, synovial sarcoma www.adaptimmune.com
ORPHAN DRUG
ADP-A2M4CD8 Adaptimmune MAGE-A4 positive tumors Phase I
(MAGE A4 T cell therapy) Philadelphia, PA www.adaptimmune.com
ADPT01 Novartis colorectal cancer (combination Phase I
East Hanover, NJ therapy) triple negative www.novartis.com
breast cancer (combination therapy)
Ad-RTS-hIL-12 + veledimex Ziopharm Oncology glioblastoma (+cemiplimab-rwlc) Phase II
(DNA-based in vivo gene therapy) Boston, MA (Fast Track) www.ziopharm.com
ORPHAN DRUG
glioblastoma (monotherapy), Phase I
glioblastoma (+nivolumab), www.ziopharm.com
pediatric brain tumors, diffuse
intrinsic pontine glioma (DIPG)
ADX-2191 Aldeyra Therapeutics intraocular lymphoma Phase II
(DHFR inhibitor) Lexington, MA www.aldeyra.com
Medicines in Development: Cancer ǀ 2020 9
Product Name Sponsor Indication Development Status
ADXS-503 Advaxis NSCLC Phase I/II
(live attenuated Listeria Princeton, NJ www.advaxis.com
monocytogenes (Lm)-based
immunotherapy)
ADXS-504 Advaxis prostate cancer Phase I
(live attenuated Listeria Princeton, NJ www.advaxis.com
monocytogenes (Lm)-based
immunotherapy)
ADXS-PSA Advaxis prostate cancer Phase I/II
(live attenuated Listeria Princeton, NJ www.advaxis.com
monocytogenes (Lm)-based
immunotherapy)
AE37 NuGenerex Immuno-Oncology triple negative breast cancer Phase II
(Ii-Key immunotherapeutic vaccine) Miramar, FL (+pembrolizumab) www.generex.com
AFM13 Affimed Therapeutics PTCL, transformed mycosis Phase II
(bispecific-antibody) Heidelberg, Germany fungoides www.affimed.com
ORPHAN DRUG
AFM24 Affimed Therapeutics EGFR-expressing solid tumors Phase I/II
(bispecific antibody) Heidelberg, Germany www.affimed.com
Medicines in Development: Cancer ǀ 2020 10
Product Name Sponsor Indication Development Status
afuresertib Laekna platinum-resistant ovarian cancer Phase II
(proto-oncogene c-akt+ inhibitor) Shanghai, China www.laeknatp.com
AG-270 Agios Pharmaceuticals MTAP-deleted lymphoma, Phase I
(MAT2A inhibitor) Cambridge, MA solid tumors www.agios.com
AGEN1181 Agenus solid tumors Phase I
(anti-CTLA-4 mAb) Lexington, MA www.agenusbio.com
AGEN1223 Agenus solid tumors Phase I
(bispecific antibody) Lexington, MA www.agenusbio.com
Gilead Sciences
Foster City, CA
AGEN2373 Agenus solid tumors Phase I
(anti-CD137 mAb) Lexington, MA www.agenusbio.com
Gilead Sciences www.gilead.com
Foster City, CA
aglatimagene besadenovec Advantagene intermediate/high risk prostate Phase III
(cancer suicide gene therapy) Auburndale, MA cancer (Fast Track) www.advantagene.com
ORPHAN DRUG
1L glioblastoma, NSCLC, localized Phase II
prostate cancer, pancreatic cancer www.advantagene.com
resectable NSCLC, Phase I
1L glioblastoma (+nivolumab) www.advantagene.com
Medicines in Development: Cancer ǀ 2020 11
Product Name Sponsor Indication Development Status
AIC100 AffyImmune Therapeutics advanced refractory thyroid cancer Phase I
(CAR-T cell therapy) Natick, MA www.affyimmune.com
ORPHAN DRUG
AIM-001 EpicentRx cancer Phase I
(oncolytic virus TGF-β trap transgene) La Jolla, CA www.epicentrx.com
AIP-303 Advanced Innovative Partners breast cancer Phase I
(peptide receptor radionuclide Surfside, Florida www.advancedinovativepartners.com
therapy)
AIV001 AiViva BioPharma basal cell carcinoma Phase I/II
(multi kinase inhibitor) Newport Beach, CA www.aiviva.com
AK104 Akeso Biopharma cervical cancer (Fast Track) Phase II
(PD-1/CTLA-4 bispecific antibody) Fremont, CA www.akesobio.com
AL101 Ayala Pharmaceuticals recurrent metastatic adenoid cystic Phase II
(gamma secretase inhibitor) Wilmington, DE carcinoma (Fast Track), triple www.ayalapharma.com
ORPHAN DRUG negative breast cancer
relapsed/refractory T-cell Phase I
lymphoblastic leukemia www.ayalapharma.com
AL102 Ayala Pharmaceuticals desmoid tumor, multiple myeloma Phase I
(gamma secretase inhibitor) Wilmington, DE www.ayalapharma.com
Medicines in Development: Cancer ǀ 2020 12
Product Name Sponsor Indication Development Status
AL3818 Advenchen Laboratories alveolar soft part sarcoma, Phase III
(PTK inhibitor) Moorpark, CA leiomyosarcoma, synovial sarcoma www.advenchen.com
ORPHAN DRUG
cervical cancer, endometrial cancer, Phase I/II
fallopian cancer, ovarian cancer, www.advenchen.com
peritoneal cancer
Alecensa® Roche/Genentech ALK-positive NSCLC (adjuvant) Phase III
alectinib South San Francisco, CA www.roche.com
Aliqopa™ Bayer Pharmaceuticals NHL (combination therapy) Phase III
copanlisib Whippany, NJ www.pharma.bayer.com
solid tumors (+nivolumab), Phase I/II
pediatric solid tumors www.pharma.bayer.com
ALKS 4230 Alkermes head and neck cancer Phase II
(interleukin-2 receptor agonist) Waltham, MA (+pembrolizumab) www.alkermes.com
advanced solid tumors Phase I/II
www.alkermes.com
ALLO-501 Allogene Therapeutics NHL (combination therapy) Phase I
(CD19 CAR-T cell therapy) South San Francisco, CA www.allogene.com
Medicines in Development: Cancer ǀ 2020 13
Product Name Sponsor Indication Development Status
ALLO-501A Allogene Therapeutics NHL (combination therapy) Phase I
(CD19 CAR-T cell therapy) South San Francisco, CA www.allogene.com
ALLO-715 Allogene Therapeutics multiple myeloma Phase I
(CAR-T cell therapy) South San Francisco, CA (combination therapy) www.allogene.com
AlloStim Immunovative Therapies metastatic colorectal cancer Phase II
T cell therapy-device combination Jerusalem, Israel www.imunovative.com
therapy
almonertinib (HS-10296) EQRx NSCLC Phase I
(EGFR inhibitor) Cambridge, MA www.eqrx.com
AlphaMedix™ Orano Med neuroendocrine tumors Phase I
(Pb212-labeled somatostatin Plano, TX www.oranomed.com
analogue targeted alpha-emitter RadioMedix www.radiomedix.com
therapy) Houston, TX
ORPHAN DRUG
ALPN-202 Alpine Immune Sciences advanced malignancies Phase I
(conditional CD28 costimulator and Seattle, WA www.alpineimmunesciences.com
dual checkpoint inhibitor)
Medicines in Development: Cancer ǀ 2020 14
Product Name Sponsor Indication Development Status
alvocidib Sumitomo Dainippon acute myeloid leukemia (AML) Phase II
(CDK9 inhibitor) Pharma Oncology www.sdponcology.com
ORPHAN DRUG Cambridge, MA
AML (combination therapy), MDS Phase I
www.sdponcology.com
ALX148 ALX Oncology high-risk MDS (combination therapy) Phase I/II
(CD47 antigen inhibitor) Burlingame, CA www.alxoncology.com
lymphoma, solid tumors Phase I
www.alxoncology.com
amatuximab (MORAb-009) Eisai mesothelioma Phase I/II
(anti-mesothelin mAb) Woodcliff Lake, NJ www.eisai.com
ORPHAN DRUG
AMG 119 Amgen SCLC Phase I
(CAR-T cell therapy) Thousand Oaks, CA www.amgen.com
AMG 160 Amgen prostate cancer Phase I
(PSMAxCD3 bispecific T cell engager) Thousand Oaks, CA www.amgen.com
AMG 176 Amgen hematologic malignancies Phase I
(MCL-1 inhibitor) Thousand Oaks, CA www.amgen.com
Medicines in Development: Cancer ǀ 2020 15
Product Name Sponsor Indication Development Status
AMG 199 Amgen gastric/gastroesophageal junction Phase I
(MUC17xCD3 bispecific T cell engager) Thousand Oaks, CA cancer www.amgen.com
AMG 256 Amgen solid tumors Phase I
(anti-PD1/IL-21 mutein) Thousand Oaks, CA www.amgen.com
AMG 330 Amgen AML Phase I
(CD33xCD3 bispecific T cell engager) Thousand Oaks, CA www.amgen.com
AMG 337 NantPharma clear cell sarcoma Phase II
(c-Met inhibitor) Culver City, CA www.nantpharma.com
AMG 397 Amgen hematologic malignancies Phase I
(MCL-1 inhibitor) Thousand Oaks, CA www.amgen.com
AMG 404 Amgen solid tumors Phase I
(anti-PD-1 antibody) Thousand Oaks, CA www.amgen.com
AMG 427 Amgen AML Phase I
(CD3xFLT3 bispecific T cell engager) Thousand Oaks, CA www.amgen.com
AMG 506 Amgen solid tumors Phase I
(multi-specific FAPx4-1BB) Thousand Oaks, CA www.amgen.com
Medicines in Development: Cancer ǀ 2020 16
Product Name Sponsor Indication Development Status
AMG 509 Amgen prostate cancer Phase I
(bivalent T cell engager) Thousand Oaks, CA www.amgen.com
AMG 596 Amgen glioblastoma Phase I
(CD3xEGFR bispecific T cell engager) Thousand Oaks, CA www.amgen.com
AMG 650 Amgen solid tumors Phase I
(oral small molecule) Thousand Oaks, CA www.amgen.com
AMG 673 Amgen AML Phase I
(CD33xCD3 bispecific T cell engager) Thousand Oaks, CA www.amgen.com
AMG 701 Amgen multiple myeloma Phase I
(BCMAxCD3 bispecific T cell engager) Thousand Oaks, CA www.amgen.com
AMG 757 Amgen SCLC Phase I
(DLL3xCD3 bispecific T cell engager) Thousand Oaks, CA www.amgen.com
AMG 910 Amgen gastric/gastroesophageal junction Phase I
(CLDNxCD3 bispecific T cell engager) Thousand Oaks, CA cancer www.amgen.com
amivantamab Janssen Research & Development NSCLC (combination therapy) Phase III
(EGFR/cMET inhibitor) Raritan, NJ (Breakthrough Therapy) www.janssen.com
NSCLC Phase I
www.janssen.com
Medicines in Development: Cancer ǀ 2020 17
Product Name Sponsor Indication Development Status
Ampligen® AIM ImmunoTech breast cancer, colorectal cancer, Phase I/II
rintatolimod Ocala, FL melanoma, ovarian cancer, renal www.aimimmuno.com
ORPHAN DRUG cell carcinoma, pancreatic cancer
AMV564 Amphivena Therapeutics AML, MDS, solid tumors Phase I
(bispecific T cell engager) South San Francisco, CA www.amphivena.com
ORPHAN DRUG
AMXI 5001 AtlasMedx advanced malignancies Phase I/II
(PARP/microtubule polymerization San Francisco, CA www.atlassmedx.com
bifunctional inhibitor)
AMXT1501 Aminex Therapeutics solid tumors (+DFMO) Phase I
(polyamine uptake inhibitor) Seattle, WA www.aminextx.com
AN0025 Adlai Nortye Biopharma solid tumors Phase I
(EP4 antagonist) North Brunswick, NJ www.adlainortye.com
anetumab ravtansine Bayer Pharmaceuticals mesothelioma Phase II
(antibody drug conjugate) Whippany, NJ www.pharma.bayer.com
ORPHAN DRUG
NSCLC, breast cancer, Phase I
gastric/gastroesophageal junction www.pharma.bayer.com
cancer, gynecologic cancer,
advanced solid tumors
Medicines in Development: Cancer ǀ 2020 18
Product Name Sponsor Indication Development Status
annamycin Moleculin Biotech AML (Fast Track) Phase I/II
(second generation anthracycline) Houston, TX www.moleculin.com
ORPHAN DRUG
anti-HA1 T cell therapy HighPassBio leukemia Phase I
Cambridge, MA www.highpassbio.com
antroquinonol Golden Biotechnology NSCLC Phase II
ORPHAN DRUG Jersey City, NJ www.goldenbiotech.com
pancreatic cancer Phase I/II
www.goldenbiotech.com
AO-176 Arch Oncology multiple myeloma, Phase I/II
(anti-CD47 mAb) Brisbane, CA solid tumors www.archoncology.com
AP-002 Altum Pharmaceuticals solid tumors Phase I/II
(RANK ligand inhibitor) Vancouver, Canada www.altumpharma.com
Apealea® Elevar Therapeutics ovarian cancer Phase III
paclitaxel micellar Salt Lake City, UT www.elevartherapeutics.com
ORPHAN DRUG Oasmia Pharmaceutical
Uppsala, Sweden
Medicines in Development: Cancer ǀ 2020 19
Product Name Sponsor Indication Development Status
APG-115 Ascentage Pharma solid tumors (+pembrolizumab), Phase I/II
(MDM2-p53 inhibitor) Rockville, MD salivary gland carcinoma www.ascentagepharma.com
ORPHAN DRUG
AML Phase I
www.ascentagepharma.com
APG-1252 Ascentage Pharma myelofibrosis, SCLC Phase I/II
(Bcl-2/NclxL inhibitor) Rockville, MD www.ascentagepharma.com
ORPHAN DRUG
APG-1387 Ascentage Pharma hematologic malignancies, Phase I
(IAP antagonist) Rockville, MD solid tumors www.ascentagepharma.com
APG-2575 Ascentage Pharma CLL, small lymphocytic lymphoma (SLL), Phase II
(Bcl-2 selective inhibitor) Rockville, MD Waldenstrom's macroglobulinemia www.ascentagepharma.com
APL-101 Apollomics solid tumors Phase I
(c-Met inhibitor) Foster City, CA www.apollomicsinc.com
APL-501 Apollomics solid tumors Phase I
(PD-1 antagonist) Foster City, CA www.apollomicsinc.com
APN401 Apeiron Biologics solid tumors Phase I
(siRNA-based cell therapy) Vienna, Austria www.apeiron-biologics.com
Medicines in Development: Cancer ǀ 2020 20
Product Name Sponsor Indication Development Status
APS001F Anaeropharma Science solid tumors Phase I/II
(cytosine deaminase gene therapy) Tokyo, Japan www.anaeropharma.co.jp
APTO-253 Aptose Biosciences AML, MDS Phase I
(MTF-1 inhibitor) San Diego, CA www.aptose.com
ORPHAN DRUG
APVO436 Aptevo Therapeutics AML, MDS Phase I
(CD3xCD123 bispecific antibody) Seattle, WA www.aptevotherapeutics.com
ORPHAN DRUG
APX005M Apexigen esophageal cancer, Phase II
(IgG1 CD40 agonistic antibody) San Carlos, CA gastroesophageal junction cancer, www.apexigen.com
ORPHAN DRUG metastatic melanoma
NSCLC (+nivolumab), Phase I/II
metastatic melanoma (+nivolumab) www.apexigen.com
APX3330 Apexian Pharmaceuticals solid tumors Phase I
(APE1 inhibitor) Indianapolis, IN www.apexianpharma.com
AR23 Arbor Pharmaceuticals cancer Phase II
Atlanta, GA www.arborpharma.com
arfolitixorin Isofol Medical colorectal cancer Phase III
(thymidylate synthase inhibitor) Gothenburg, Sweden www.isofolmedical.com
Medicines in Development: Cancer ǀ 2020 21
Product Name Sponsor Indication Development Status
ARO-HIF2 Arrowhead Pharmaceuticals renal cell carcinoma Phase I
(siRNA therapeutic) Pasadena, CA www.arrowheadpharma.com
ARQ 751 Merck solid tumors Phase I
(c-akt inhibitor) Kenilworth, NJ www.merck.com
ARV-110 Arvinas metastatic castration-resistant Phase I
(androgen receptor New Haven, CT prostate cancer (Fast Track) www.arvinas.com
degradation enhancer)
ARV-471 Arvinas ER-positive HER2-negative Phase I
(estrogen receptor degrader) New Haven, CT breast cancer www.arvinas.com
ARX788 Ambrx HER2-expressing cancers Phase I
(antibody-drug conjugate) La Jolla, CA www.ambrx.com
ASN007 Asana BioSciences solid tumors Phase I
(ERK 1/2 inhibitor) Trenton, NJ www.asanabiosciences.com
ASP-1929 Rakuten Medical head and neck cancer (Fast Track), Phase III
(cetuximab sarotalocan) San Diego, CA www.rakuten-med.com
EGFR-expressing solid tumors Phase I/II
www.rakuten-med.com
Medicines in Development: Cancer ǀ 2020 22
Product Name Sponsor Indication Development Status
ASP1948/PTZ-329 Astellas Pharma US solid tumors Phase I
(anti-NRP1 antibody) Northbrook, IL www.astellas.com
Potenza Therapeutics www.potenzatherapeutics.com
Cambridge, MA
ASP1951/PTZ-522 Astellas Pharma US solid tumors Phase I
(GITR agnostic antibody) Northbrook, IL www.astellas.com
Potenza Therapeutics www.potenzatherapeutics.com
Cambridge, MA
ASP7517 Astellas Pharma AML, MDS Phase I
(WT1 cell therapy) Northbrook, IL www.astellas.com
ASP9801 Astellas Pharma US solid tumors Phase I
(oncolytic virus therapy) Northbrook, IL www.astellas.com
ASTX029 Astex Pharmaceuticals solid tumors Phase I/II
(ERK 1/2 inhibitor) Pleasanton, CA www.astx.com
ASTX030 Astex Pharmaceuticals MDS Phase I
(azacitidine/cedazuridine) Pleasanton, CA www.astx.com
ASTX295 Astex Pharmaceuticals solid tumors Phase I/II
(MDM2 antagonist) Pleasanton, CA www.astx.com
Medicines in Development: Cancer ǀ 2020 23
Product Name Sponsor Indication Development Status
ASTX660 (tolinapant) Astex Pharmaceuticals lymphoma, solid tumors Phase II
(IAP inhibitor) Pleasanton, CA www.astx.com
ORPHAN DRUG
AML (+ASTX727) Phase I
www.astx.com
AT-101 Ascentage Pharma CLL Phase II
(Bcl-2 inhibitor) Rockville, MD www.ascentagepharma.com
ATA2271 Atara Biotherapeutics mesothelioma Phase I
(anti-mesothelin CAR-T cell therapy) South San Francisco, CA www.atarabio.com
ATP128 Amal Therapeutics colorectal cancer Phase I
(chimeric recombinant Geneva, Switzerland www.amaltherapeurtics.com
protein cancer vaccine) Boehringer Ingelheim
Ridgefield, CT
ATRC-101 Atreca solid tumors Phase I
(mAb) South San Francisco, CA www.atreca.com
AU-011 Aura Biosciences uveal melanoma (Fast Track) Phase II
(light-activated therapeutic) Cambridge, MA www.aurabiosciences.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 24
Product Name Sponsor Indication Development Status
AUTO1 Autolus Therapeutics acute lymphoblastic leukemia (ALL) Phase I/II
(anti-CD19 CAR-T cell therapy) London, United Kingdom www.autolus.com
ORPHAN DRUG
AUTO3 Autolus Therapeutics DLBCL Phase I/II
(anti-CD19/CD22 CAR-T cell therapy) London, United Kingdom www.autolus.com
ORPHAN DRUG
AutoSynVax™ Agenus solid tumors Phase I
autologous cancer vaccine Lexington, MA www.agenusbio.com
AVB-500 Aravive clear cell kidney cancer, Phase I
(GAS6-AXL pathway inhibitor) Houston, TX ovarian cancer (Fast Track), www.aravive.com
urothelial cancer
AV-GBM-1 AIVITA Biomedical newly-diagnosed glioblastoma Phase II
(autologous dendritic cell Irvine, CA www.aivitabiomedical.com
immunotherapy)
AVID100 Bristol-Myers Squibb triple negative breast cancer Phase I/II
(anti-EGFR antibody-drug conjugate) Princeton, NJ head and neck cancer, NSCLC www.bms.com
AVID200 Bristol-Myers Squibb solid tumors Phase I
(TGFβ inhibitor) Princeton, NJ www.bms.com
AV-MEL-1 AIVITA Biomedical melanoma Phase I
(autologous dendritic cell vaccine) Irvine, CA www.aivitabiomedical.com
Medicines in Development: Cancer ǀ 2020 25
Product Name Sponsor Indication Development Status
axalimogene filolisbac (AXAL) Advaxis cervical cancer (Fast Track) Phase III
(cancer immunotherapy) Princeton, NJ www.advaxis.com
ORPHAN DRUG
axatilamab (SNDX-6352) Syndax Pharmaceuticals solid tumors Phase I
(CSF-1R mAb) Waltham, MA www.syndax.com
Ayvakit™ Blueprint Medicines systemic mastocytosis associated Phase II
avapritinib Cambridge, MA with hematologic malignancies www.blueprintmedicines.com
ORPHAN DRUG
AZD0466 AstraZeneca hematologic malignancies, Phase I
(Bcl2/xL inhibitor) Wilmington, DE solid tumors www.astrazeneca.com
AZD1390 AstraZeneca glioblastoma Phase I
(ATM kinase inhibitor) Wilmington, DE www.astrazeneca.com
AZD2811 AstraZeneca hematologic malignancies, Phase II
(aurora kinase B inhibitor) Wilmington, DE solid tumors www.astrazeneca.com
AZD4573 AstraZeneca hematologic malignancies Phase I
(CDK9 inhibitor) Wilmington, DE www.astrazeneca.com
AZD4635 AstraZeneca prostate cancer Phase II
(adenosine A2A receptor antagonist) Wilmington, DE www.astrazeneca.com
Medicines in Development: Cancer ǀ 2020 26
Product Name Sponsor Indication Development Status
AZD5153 AstraZeneca hematologic malignancies, Phase I
(BRD4 protein inhibitor) Wilmington, DE solid tumors www.astrazeneca.com
AZD5991 AstraZeneca hematologic malignancies Phase I
(MCL-1 inhibitor) Wilmington, DE www.astrazeneca.com
AZD7648 AstraZeneca hematologic malignancies, Phase I
(DNA-PK inhibitor) Wilmington, DE solid tumors www.astrazeneca.com
AZD8701 (ION736) AstraZeneca solid tumors Phase I
(antisense) Wilmington, DE www.astrazeneca.com
Ionis Pharmaceuticals
Carlsbad, CA
AZD9496 AstraZeneca ER-positive breast cancer Phase I
(SERD) Wilmington, DE www.astrazeneca.com
AZD9833 AstraZeneca ER-positive breast cancer Phase II
(SERD) Wilmington, DE www.astrazeneca.com
balixafortide Polyphor metastatic breast cancer Phase III
(CXCR4 inhibitor) Allschwil, Switzerland (Fast Track) www.polyphor.com
Medicines in Development: Cancer ǀ 2020 27
Product Name Sponsor Indication Development Status
balstilimab (AGEN2034) Agenus cervical cancer (monotherapy) application submitted
(PD-1 mAb) Lexington, MA (Fast Track) www.agenusbio.com
cervical cancer (+AGEN1884) Phase II
(Fast Track) www.agenusbio.com
Balversa™ Janssen Research & Development non-muscle invasive bladder cancer, Phase II
erdafitinib Raritan, NJ tumor agnostic (FGFR gene alteration) www.janssen.com
Bavencio® EMD Serono 1L NSCLC Phase III
avelumab Rockland, MA www.emdserono.com
Pfizer www.pfizer.com
New York, NY
1L Merkel cell carcinoma, head and Phase II
neck cancer (combination therapy), www.emdserono.com
NSCLC (combination therapy), www.pfizer.com
urothelial cancer (combination
therapy)
solid tumors (combination therapy) Phase I
www.emdserono.com
www.pfizer.com
bavituximab OncXerna Therapeutics gastric/gastroesophageal junction Phase II
(phosphatidylserine inhibitor) Waltham, MA cancer www.oncxerna.com
Medicines in Development: Cancer ǀ 2020 28
Product Name Sponsor Indication Development Status
BAY 1251152 Bayer Pharmaceuticals cancer Phase I
(PTEFb inhibitor) Whippany, NJ www.pharma.bayer.com
BAY 1834942 (tinurilimab) Bayer Pharmaceuticals solid tumors Phase I
(CEACAM6 fb antibody) Whippany, NJ www.pharma.bayer.com
BAY 1895344 Bayer Pharmaceuticals lymphoma, ovarian cancer, Phase I
(ATR inhibitor) Whippany, NJ solid tumors www.pharma.bayer.com
BAY 1905254 Bayer Pharmaceuticals solid tumors Phase I
(ILDR2 fb antibody) Whippany, NJ www.pharma.bayer.com
BAY 2287411 (anetumab corixetan) Bayer Pharmaceuticals solid tumors Phase I
(mesothelin-targeted thorium Whippany, NJ www.pharma.bayer.com
conjugate)
BAY 2315497 (PSMA-TTC) Bayer Pharmaceuticals prostate cancer Phase I
(PSMA-targeted throrium conjugate) Whippany, NJ www.pharma.bayer.com
BAY 2402234 Bayer Pharmaceuticals myeloid leukemia Phase I
(DHODH inhibitor) Whippany, NJ www.pharma.bayer.com
BAY 2416964 Bayer Pharmaceuticals solid tumors Phase I
(AhR inhibitor) Whippany, NJ www.pharma.bayer.com
Medicines in Development: Cancer ǀ 2020 29
Product Name Sponsor Indication Development Status
BAY 2701439 (HER2-TTC) Bayer Pharmaceuticals HER2-expressing solid tumors Phase I
(HER2-targeted thorium conjugate) Whippany, NJ www.pharma.bayer.com
BB-1701 Bliss Biopharmaceutical solid tumors Phase I
(antibody-drug conjugate) Hangzhou, China
bb21217 bluebird bio multiple myeloma Phase I
(CAR-T cell therapy) Cambridge, MA www.bluebirdbio.com
ORPHAN DRUG Bristol-Myers Squibb www.bms.com
Princeton, NJ
BBP-398 NaVire Pharma solid tumors Phase I
(SHP2 inhibitor) San Francisco, CA www.navirepharma.com
BCA101 Bicara Therapeutics solid tumors Phase I
(EGFRxTGFß bispecific antibody) Cambridge, MA
BCMA-CD19 cCAR iCell Gene Therapeutics multiple myeloma, plasmacytoid Phase I
(BCMA-CS1 cCAR-T cell therapy) Stony Brook, NY lymphoma www.icellgene.com
BCMA-CS1 cCAR iCell Gene Therapeutics multiple myeloma Phase I
(BCMA-CS1 cCAR-T cell therapy) Stony Brook, NY www.icellgene.com
BDB001 Seven and Eight Biopharmaceuticals lymphoma, solid tumors Phase I
(TLR 7/8 agonist) Edison, NJ www.7and8biopharma.com
Medicines in Development: Cancer ǀ 2020 30
Product Name Sponsor Indication Development Status
BDC-1001 Bolt Biotherapeutics solid tumors Phase I/II
(immune-stimulating antibody Redwood City, CA www.boltbio.com
conjugate)
BDTX-189 Black Diamond Therapeutics solid tumors (Fast Track) Phase I/II
(ErbB kinase inhibitor) Cambridge, MA www.blackdiamondtherapeutics.com
Beleodaq® Acrotech Biopharma hematologic malignancies Phase I
belinostat for injection East Windsor, NJ (wild-type UGT1A1*28 genotype), www.arcotechbiopharma.com
solid tumors (wild-type UGT1A1*28
genotype)
bemarituzumab (FPA-144) Five Prime Therapeutics 1L gastric/gastroesophageal junction Phase II
(FGFR2B antibody) South San Francisco, CA cancer www.fiveprime.com
ORPHAN DRUG
bemcentinib BerGenBio NSCLC (+pembrolizumab) Phase II
(RTK inhibitor) Bergen, Norway www.bergenbio.com
bempegaldesleukin Nektar Therapeutics head and neck cancer (+VB10-NEO), Phase I/II
(natural killer cell stimulant) San Francisco, CA NSCLC (+pembrolizumab) www.nektar.com
berubicin CNS Pharmaceuticals glioblastoma Phase I
(type II DNA topoisomerase inhibitor) Houston, TX www.cnspharma.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 31
Product Name Sponsor Indication Development Status
berzosertib (M6620) EMD Serono solid tumors Phase I
(ATR inhibitor) Rockland, MA www.emdserono.com
Betalutin® Nordic Nanovector DLBCL, follicular lymphoma (Fast Phase I
lutetium (177lu) lilotomab Oslo, Norway Track), NHL www.nordicnanovector.com
satetraxetan
ORPHAN DRUG
BGB-10188 BeiGene RAF-mutant solid tumors Phase I
(PI3K) Cambridge, MA www.beigene.com
BGB-3245 MapKure RAF-mutant solid tumors Phase I
(BRAF inhibitor) (BeiGene/SpringWorks JV) www.beigene.com
Cambridge, MA www.springworkstx.com
BGB-A1217 BeiGene solid tumors Phase I
(TIGIT) Cambridge, MA www.beigene.com
BGB-A425 BeiGene solid tumors Phase I
(TIM-1) Cambridge, MA www.beigene.com
BI-1206 BioInvent International non-Hodgkin lymphoma, Phase I/II
(CD32b antagonist) Lund, Sweden solid tumors www.bioinvent.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 32
Product Name Sponsor Indication Development Status
BI 1361849 (CV9202) Boehringer Ingelheim NSCLC Phase I
(mRNA vaccine) Ridgefield, CT www.boehringer-ingelheim.com
BI 1701963 Boehringer Ingelheim solid tumors Phase I
(SOS1/KRAS inhibitor) Ridgefield, CT www.boehringer-ingelheim.com
BI 3011441 Boehringer Ingelheim solid tumors Phase I
Ridgefield, CT www.boehringer-ingelheim.com
BI 754091 Boehringer Ingelheim solid tumors Phase I
(PD-1 antibody) Ridgefield, CT www.boehringer-ingelheim.com
BI 754111 Boehringer Ingelheim solid tumors Phase I
(LAG-3 antibody) Ridgefield, CT www.boehringer-ingelheim.com
BI 765063 Boehringer Ingelheim solid tumors Phase I
(SIRP1α antagonist) Ridgefield, CT www.boehringer-ingelheim.com
BI 836880 Boehringer Ingelheim NSCLC Phase I
(VEGFxAng-2 bispecific antibody) Ridgefield, CT www.boehringer-ingelheim.com
BI 891065 Boehringer Ingelheim solid tumors Phase I
(SMAC-mimetic) Ridgefield, CT www.boehringer-ingelheim.com
Medicines in Development: Cancer ǀ 2020 33
Product Name Sponsor Indication Development Status
BI 894999 Boehringer Ingelheim solid tumors Phase I
(BET inhibitor) Ridgefield, CT www.boehringer-ingelheim.com
BI 905677 Boehringer Ingelheim solid tumors Phase I
(LRP5/6 inhibitor) Ridgefield, CT www.boehringer-ingelheim.com
BI 905681 Boehringer Ingelheim solid tumors Phase I
(LRP5 antagonist) Ridgefield, CT www.boehringer-ingelheim.com
BI 905711 Boehringer Ingelheim solid tumors Phase I
(TRAIL2/CDH17 antibody) Ridgefield, CT www.boehringer-ingelheim.com
BI 907828 Boehringer Ingelheim solid tumors Phase I
(MDM2-p53 antagonist) Ridgefield, CT www.boehringer-ingelheim.com
bicalutamide implant Alessa Therapeutics prostate cancer Phase I
(androgen receptor antagonist) San Carlos, CA www.alessatherapeutics.com
bimiralisib (PQR309) PIQUR Therapeutics head and neck squamous cell Phase II
(pan-PI3K/mTOR inhibitor) Basel, Switzerland carcinoma www.piqur.com
Medicines in Development: Cancer ǀ 2020 34
Product Name Sponsor Indication Development Status
bintrafusp alfa EMD Serono 1L NSCLC Phase III
(TGFß trap/anti-PD-L1) Rockland, MA www.emdserono.com
GlaxoSmithKline
Research Triangle Park, NC
2L NSCLC, locally advanced NSCLC, Phase II
1L/2L biliary tract cancer, www.emdserono.com
2L cervical cancer
1L cervical cancer, solid tumors Phase I
www.emdserono.com
BIO-11006 BioMarck Pharmaceuticals NSCLC Phase II
(myristoylated alanine rich C Durham, NC www.biomarck.com
kinase substrate inhibitor)
biropepimut-S Gliknik squamous cell carcinoma Phase II
(peptide immunomodulator) Baltimore, MD of the oral cavity www.gliknik.com
ORPHAN DRUG
bispecific antibody Eli Lilly cancer Phase I
(Azymetric™ technology platform) Indianapolis, IN www.lilly.com
Zymeworks Biopharmaceuticals www.zymeworks.com
Seattle, WA
BJ-001 BJ Bioscience solid tumors Phase I
(recombinant fusion protein) Hangzhou, China www.bjbioscience.com
Medicines in Development: Cancer ǀ 2020 35
Product Name Sponsor Indication Development Status
Blenrep GlaxoSmithKline 3L+ multiple myeloma Phase III
belantamab mafodotin Research Triangle Park, NC www.gsk.com
Blincyto® Amgen ALL (pediatric) Phase III
blinatumomab Thousand Oaks, CA www.amgen.com
DLBCL (+pembrolizumab) Phase I
www.amgen.com
BLZ945 Novartis solid tumors (+PD-1 inhibitor) Phase II
(CSF-1 inhibitor) East Hanover, NJ www.novartis.com
BMS-813160 Bristol-Myers Squibb solid tumors Phase II
(CCR2/5 dual antagonist) Princeton, NJ www.bms.com
BMS-986012 Bristol-Myers Squibb solid tumors Phase I
(fucosyl GM1 inhibitor) Princeton, NJ www.bms.com
BMS-986158 Bristol-Myers Squibb hematologic malignancies, Phase I
(BET inhibitor) Princeton, NJ solid tumors www.bms.com
BMS-986178 Bristol-Myers Squibb solid tumors Phase I
(anti-OX40 antibody) Princeton, NJ www.bms.com
BMS-986179 Bristol-Myers Squibb solid tumors Phase I
(anti-CD73 mAb) Princeton, NJ www.bms.com
Medicines in Development: Cancer ǀ 2020 36
Product Name Sponsor Indication Development Status
BMS-986207 Bristol-Myers Squibb solid tumors Phase I
(anti-TIGIT mAb) Princeton, NJ www.bms.com
BMS-986218 Bristol-Myers Squibb solid tumors Phase I
(anti-CTLA-4 NF mAb) Princeton, NJ www.bms.com
BMS-986249 Bristol-Myers Squibb solid tumors Phase I
(anti-CTLA-4 probody) Princeton, NJ www.bms.com
BMS-986253 Bristol-Myers Squibb solid tumors Phase I
(anti-IL8) Princeton, NJ www.bms.com
BMS-986258 Bristol-Myers Squibb solid tumors Phase I
(anti-TIM-3 mAb) Princeton, NJ www.bms.com
Five Prime Therapeutics
South San Francisco, CA
BMS-986288 Bristol-Myers Squibb solid tumors Phase I
(anti-CTLA-4 NF probody) Princeton, NJ www.bms.com
BMS-986299 Bristol-Myers Squibb solid tumors Phase I
(NLRP3 agonist) Princeton, NJ www.bms.com
BMS-986301 Bristol-Myers Squibb solid tumors Phase I
(STING agonist) Princeton, NJ www.bms.com
Medicines in Development: Cancer ǀ 2020 37
Product Name Sponsor Indication Development Status
BMS-986310 Bristol-Myers Squibb solid tumors Phase I
(EP4 receptor antagonist) Princeton, NJ www.bms.com
BMS-986315 Bristol-Myers Squibb solid tumors Phase I
(anti-NKG2A mAb) Princeton, NJ www.bms.com
BMX-001 BioMimetix brain metastases, high-grade glioma, Phase II
(mangano porphyrin antioxidant Greenwood Village, CO head and neck cancer www.biomimetixpharma.com
mimetic)
BN-Brachyury vaccine Bavarian Nordic chordoma Phase II
(immunotherapy vaccine) Morrisville, NC www.bavarian-nordic.com
ORPHAN DRUG
BNT321 (MVT-5873) BioNTech pancreatic cancer Phase I
(IgG1 mAb) Mainz, Germany www.biontech.com
BNT411 BioNTech solid tumors, including SCLC Phase I/II
(TLR7 agonist) Mainz, Germany www.biontech.de
BNZ-1 Bioniz Therapeutics cutaneous T-cell lymphoma, Phase I/II
(IL-2/IL-9/IL-15 receptor antagonist) Irvine, CA large granular lymphocytic leukemia www.bioniz.xom
BOS172722 Boston Pharmaceuticals solid tumors Phase I
(MPS1 inhibitor) Cambridge, MA www.bostonpharmaceuticals.com
Medicines in Development: Cancer ǀ 2020 38
Product Name Sponsor Indication Development Status
BOS172738 Boston Pharmaceuticals solid tumors Phase I
(RET inhibitor) Cambridge, MA www.bostonpharmaceuticals.com
BP1002 Bio-Path Holdings advanced lymphoid malignancies Phase I
(RNAi nanoparticle targeting Bcl2) Bellaire, TX www.biopathholdings.com
BPM31510 (ubidecarenone) Berg pancreatic cancer Phase II
(cancer cell metabolism corrector) Framingham, MA (combination therapy) www.berg.com
ORPHAN DRUG
solid tumors (monotherapy and Phase I
combination therapy) www.berg.com
BPX-601 Bellicum Pharmaceuticals pancreatic cancer Phase I/II
(GoCAR-T cell therapy) Houston, TX www.bellicum.com
Braftovi® + Mektovi Pfizer 1L BRAF-mutant colorectal cancer Phase II
encorafinib + binimetinib New York, NY (Breakthrough Therapy), www.pfizer.com
ORPHAN DRUG 1L/2L BRAF-mutant NSCLC,
BRAF-mutant metastatic melanoma
brain metastases
brequinar Clear Creek Bio AML Phase I/II
(DHODH inhibitor) Cambridge, MA www.clearcreekbio.com
briciclib (intravenous) Onconova Therapeutics solid tumors Phase I
(cyclin D1 inhibitor) Newtown, PA www.onconova.com
Medicines in Development: Cancer ǀ 2020 39
Product Name Sponsor Indication Development Status
Brukinsa™ BeiGene 1L CLL/SLL, 1L mantle cell lymphoma Phase III
zanubrutinib Cambridge, MA (Breakthrough Therapy), www.beigene.com
ORPHAN DRUG Waldenstrom's macroglobulinemia
(Fast Track)
previously-treated CLL/SLL, Phase II
marginal zone lymphoma, www.beigene.com
follicular lymphoma (combination
therapy)
BST-236 BioSight newly-diagnosed AML (Fast Track) Phase II
(aspacytarabine drug conjugate) Lod, Israel www.biosight-pharma.com
ORPHAN DRUG
BT1718 Bicycle Therapeutics solid tumors Phase I/II
(MT1 targeting conjugate) Lexington, MA www.bicycletherapeutics.com
BT5528 Bicycle Therapeutics solid tumors Phase I/II
(EphA2 targeting conjugate) Lexington, MA www.bicycletherapeutics.com
BT8009 Bicycle Therapeutics solid tumors Phase I/II
(Nectin-4 targeting conjugate) Lexington, MA www.bicycletherapeutics.com
BTX-A51 BioTheryX AML, MDS Phase I
(dual CKIα/CDK7-9 inhibitor) San Diego, CA www.biotheryx.com
Medicines in Development: Cancer ǀ 2020 40
Product Name Sponsor Indication Development Status
BVD-523 (ulixertinib) BioMed Valley Discoveries advanced solid tumors Phase II
(ERK inhibitor) Kansas City, MO www.biomed-valley.com
BXQ-350 Bexion Pharmaceuticals solid tumors Phase I
(apoptosis stimulant) Covington, KY www.bexionpharma.com
CA102N Holy Stone Healthcare solid tumors Phase I
(hyaluronic acid/H-Nim conjugate) Taipei, Taiwan www.hshc.tx
CA-170 Curis lymphoma, solid tumors Phase I
(VISTA/PD-L1 antagonist) Lexington, MA (VISTA expressing) www.curis.com
CA-4948 Curis hematologic malignancies, MDS Phase I
(IRAK4 kinase inhibitor) Lexington, MA www.curis.com
CA4P Mateo/Oncotelic cancer Phase II completed
(vascular disrupting agent) Agoura Hills, CA www.oncotelic.com
CAB-AXL-ADC BioAtla solid tumors Phase I/II
(antibody-drug conjugate) San Diego, CA www.bioatla.com
CAB-ROR2-ADC BioAtla solid tumors Phase I/II
(antibody-drug conjugate) San Diego, CA www.bioatla.com
Medicines in Development: Cancer ǀ 2020 41
Product Name Sponsor Indication Development Status
Cabometyx® Exelixis metastatic castration-resistant Phase III
cabozantinib Alameda, CA prostate cancer (combination therapy), www.exelixis.com
ORPHAN DRUG 1L renal cell carcinoma (combination
therapy) (Breakthrough Therapy),
hepatocellular carcinoma
(combination therapy),
differentiated thyroid cancer
castration-resistant prostate Phase I
cancer, renal cell carcinoma, www.exelixis.com
hepatocellular carcinoma,
colorectal cancer, differentiated
thyroid cancer, endometrial cancer,
NSCLC, gastric/gastroesophageal
junction cancer, head and neck
cancer, breast cancer, ovarian cancer,
urothelial cancer
Calquence® AstraZeneca 1L CLL, relapsed/refractory CLL, Phase III
acalabrutinib Wilmington, DE 1L mantle cell lymphoma, 1L DLBCL www.astrazeneca.com
non-Hodgkin lymphoma, Phase I
hematologic malignancies www.astrazeneca.com
(+ceralasertib)
Camcevi™ Foresee Pharmaceuticals prostate cancer application submitted
leuprorelin depot Taipei, Taiwan www.foreseepharma.com
(6-month formulation)
Medicines in Development: Cancer ǀ 2020 42
Product Name Sponsor Indication Development Status
camidanlumab tesirine (ADCT-301) ADC Therapeutics Hodgkin lymphoma Phase II
(antibody-drug conjugate) Lausanne, Switzerland www.adctherapeutics.com
Genmab
Copenhagen, Denmark
solid tumors Phase I
www.adctherapeutics.com
camsirubicin (MNPR-201) Monopar Therapeutics soft tissue sarcoma Phase II
(doxorubicin analog) Wilmette, IL www.monopartx.com
ORPHAN DRUG
CAN04 Cantargia solid tumors Phase I
(anti-IL1 TRAP antibody) Lund, Sweden www.cantargia.com
cantrixil (TRE-E-002-1) Kazia Therapeutics ovarian cancer Phase I
(third-generation benzopyran) Sydney, Australia www.kaziatherapeutics.com
ORPHAN DRUG
capivasertib AstraZeneca 1L triple negative breast cancer, Phase III
(Akt inhibitor) Wilmington, DE inoperable or metastatic breast www.astrazeneca.com
cancer, PTEN-deficient hormone
sensitive prostate cancer
prostate cancer Phase II
www.astrazeneca.com
Medicines in Development: Cancer ǀ 2020 43
Product Name Sponsor Indication Development Status
CAR-CIK immunotherapy CoImmune ALL Phase I/II
(genetically-modified cytokine Durham, NC www.coimmune.com
induced killer cells)
CART-ddBCMA Arcellx multiple myeloma (Fast Track) Phase I
(anti-BCMA T cell therapy) Gaithersburg, MD www.arcellx.com
ORPHAN DRUG
cavrotolimod Exicure solid tumors Phase I
(TLR9 agonist) Chicago, IL www.exicuretx.com
CB-5339 Cleave Therapeutics AML, MDS Phase I
(CDC48/p97-ATPase inhibitor) San Francisco, CA www.cleavetherapeutics.com
CBL0137 Incuron solid tumors Phase I
(curaxin) Buffalo, NY www.incuron.com
CBP501 CanBas solid tumors Phase I
(peptide mimetic) Shizuoka, Japan www.canbas.co.jp
CC-90009 (CELMoD) Bristol-Myers Squibb AML Phase I
(cereblon E3 ubiquitin ligase Princeton, NJ www.bms.com
modulating drug)
CC-90010 Bristol-Myers Squibb astrocytoma, glioblastoma, Phase I
(BET inhibitor) Princeton, NJ hematologic malignancies www.bms.com
Medicines in Development: Cancer ǀ 2020 44
Product Name Sponsor Indication Development Status
CC-90011 Bristol-Myers Squibb extensive-stage SCLC, NHL Phase II
(LSD1 inhibitor) Princeton, NJ www.bms.com
CC-92480 (CELMoD) Bristol-Myers Squibb multiple myeloma Phase I
(cereblon E3 ubiquitin ligase Princeton, NJ www.bms.com
modulating drug)
CC-93269 Bristol-Myers Squibb multiple myeloma Phase I
(BCMA TCE) Princeton, NJ www.bms.com
CC-94676 Bristol-Myers Squibb colorectal cancer, prostate cancer, Phase I
(androgen receptor ligand Princeton, NJ solid tumors www.bms.com
directed degrader)
CC-95251 Bristol-Myers Squibb NHL, solid tumors Phase I
(anti-SIRPα) Princeton, NJ www.bms.com
CC-95775 Bristol-Myers Squibb NHL, solid tumors Phase I
(BET inhibitor) Princeton, NJ www.bms.com
CC-97540 Bristol-Myers Squibb NHL Phase I
(CD19-NEX T CAR-T cell therapy) Princeton, NJ www.junotherapeutics.com
CC-98633 Bristol-Myers Squibb multiple myeloma Phase I
(BCMA-NEX T CAR-T cell therapy) Princeton, NJ www.junotherapeutics.com
Medicines in Development: Cancer ǀ 2020 45
Product Name Sponsor Indication Development Status
CC-99282 (CELMoD) Bristol-Myers Squibb NHL Phase I
(cereblon E3 ubiquitin ligase Princeton, NJ www.junotherapeutics.com
modulating drug)
CC-99712 Bristol-Myers Squibb multiple myeloma Phase I
(BCMA antibody-drug conjugate) Princeton, NJ www.bms.com
CCW702 AbbVie castration-resistant prostate cancer Phase I
(cancer immunotherapy) North Chicago, IL www.abbvie.com
Calibr
La Jolla, CA
CCX872 ChemoCentryx pancreatic cancer Phase II
(CCR2 receptor antagonist) Mountain View, CA www.chemocentryx.com
CD11301 Galderma cutaneous T-cell lymphoma Phase II
(immune response modifier) Ft. Worth, TX www.galderma.com
CD123-CD33 cCAR iCell Gene Therapeutics hematologic malignancies Phase I
(cCAR-T cell therapy) Stony Brook, NY www.icellgene.com
CD19 CARvac iCell Gene Therapeutics B-cell malignancies Phase I
(CARvac T cell vaccine) Stony Brook, NY www.icellgene.com
CD19 t-haNK NantKwest ALL, DLBCL Phase I
(CAR-NK cell therapy) Culver City, CA www.nantkwest.com
Medicines in Development: Cancer ǀ 2020 46
Product Name Sponsor Indication Development Status
CD20-CD19 cCAR iCell Gene Therapeutics B-cell malignancies Phase I
(cCAR-T cell therapy) Stony Brook, NY www.icellgene.com
CD38 CAR-T Sorrento Therapeutics multiple myeloma Phase I
(CD38 CAR-T cell therapy) San Diego, CA www.sorrentotherapeutics.com
CD4CAR iCell Gene Therapeutics T-cell malignancies Phase I
(CAR-T cell therapy) Stony Brook, NY www.icellgene.com
ORPHAN DRUG
CDK-002 Codiak BioSciences solid tumors Phase I/II
(exosome therapeutic) Cambridge, MA www.codialbio.com
CDX-1140 Celldex Therapeutics solid tumors Phase I
(CD40 antigen stimulant) Hampton, NJ www.celldex.com
CDX-527 Celldex Therapeutics solid tumors Phase I
(bispecific antibody) Hampton, NJ www.celldex.com
Cell in a Box PharmaCyte Biotech pancreatic cancer Phase II
(microencapsulated CYP-2B1- Laguna Hills, CA www.pharmacyte.com
expressing cells + ifosfamide)
ORPHAN DRUG
CEND-1 Cend Therapeutics pancreatic cancer Phase I
ORPHAN DRUG San Diego, CA (combination therapy) www.cendrx.com
Medicines in Development: Cancer ǀ 2020 47
Product Name Sponsor Indication Development Status
ceramide nanoliposome Keystone Nano solid tumors Phase I
State College, PA www.keystonenano.com
cerdulatinib Alexion relapsed/refractory CLL, NHL Phase I
(SYK/JAK inhibitor) Boston, MA www.alexion.com
ORPHAN DRUG
cetrelimab (JNJ-63723283) Janssen Research & Development castration-resistant prostate cancer Phase I
(PD-1 Inhibitor) Raritan, NJ (+apalutamide) www.janssen.com
CFI-400945 Treadwell Therapeutics castration-resistant prostate Phase II
(PLK4 inhibitor) New York, NY cancer www.treadwelltx.com
CFI-402257 Treadwell Therapeutics solid tumors Phase I
(TTK inhibitor) New York, NY www.treadwelltx.com
CFI-402411 Treadwell Therapeutics solid tumors Phase I/I
(HPK1 inhibitor) New York, NY www.treadwelltx.com
CG-0070 CG Oncology non-muscle invasive bladder cancer Phase III
(oncolytic immunotherapy) Irvine, CA www.cgoncology.com
non-muscle invasive bladder cancer Phase II
(combination therapy) www.cgoncology.com
Medicines in Development: Cancer ǀ 2020 48
Product Name Sponsor Indication Development Status
CG-806 Aptose Biosciences AML, CLL/SLL, NHL Phase I
(BTK/FLT3 inhibitor) San Diego, CA www.aptose.com
CI-8993 Curis advanced solid tumors Phase I
(VISTA inhibitor) Lexington, MA www.curis.com
ciforadenant Corvus Pharmaceuticals multiple myeloma (+daratumumab), Phase I
(A2AR inhibitor) Burlingame, CA renal cell carcinoma (+atezolizumab), www.corvuspharma.com
castration-resistant prostate cancer
(+atezolizumab)
ciltacabtagene autoleucel Janssen Research & Development multiple myeloma Phase III
(BCMA CAR-T cell therapy) Raritan, NJ (Breakthrough Therapy) www.janssen.com
ORPHAN DRUG
multiple myeloma (+1-3PL) Phase II
www.janssen.com
cirmtuzumab Oncternal Therapeutics CLL, mantle cell lymphoma Phase II
(ROR1 antagonist) San Diego, CA www.oncternal.com
ORPHAN DRUG
breast cancer Phase I
www.oncternal.com
CISH inactivated tumor infiltrating Intima Bioscience gastrointestinal cancer Phase I/II
lymphocyte cell therapy New York, NY www.intimabioscience.com
Medicines in Development: Cancer ǀ 2020 49
Product Name Sponsor Indication Development Status
CK-101 Checkpoint Therapeutics NSCLC Phase I
(EGFR inhibitor) New York, NY www.checkpointtx.com
ORPHAN DRUG
CLBR001+SWI019 AbbVie B-cell malignancies (Fast Track) Phase I
(CAR-T + sCAR-T cell therapy) North Chicago, IL www.abbvie.com
Calibr
La Jolla, CA
CLL1-CD33 cCART iCell Gene Therapeutics hematologic malignancies Phase I
(compound CAR-T cell therapy) Stony Brook, NY www.icellgene.com
CLN-081 Cullinan Oncology NSCLC Phase I
(EGFR Exon 20 mutation inhibitor) Cambridge, MA www.cullinanoncology.com
Taiho Pharmaceutical
Tokyo, Japan
CLR 131 Cellectar Biosciences multiple myeloma (Fast Track), Phase II
(cancer-targeted Florham Park, NJ B-cell lymphoma www.cellectar.com
radiopharmaceutical)
ORPHAN DRUGhead and neck cancer, Phase I
pediatric relapsed or refractory www.cellectar.com
malignant tumors
CLT030 Cellerant Therapeutics AML Phase I
(antibody-drug conjugate) San Carlos, CA www.cellerant.com
Medicines in Development: Cancer ǀ 2020 50
Product Name Sponsor Indication Development Status
CMN-001 CoImmune renal cell carcinoma Phase II
(dendritic cell-based immunotherapy) Durham, NC www.coimmune.com
CMP-001 Checkmate Pharmaceuticals melanoma (monotherapy and Phase I
(TLR9 agonist) Cambridge, MA combination therapy) www.checkmatepharma.com
ORPHAN DRUG
CNV-NY BioMed Valley Discoveries solid tumors Phase I
(clostridium novyi-NT tumor Kansas City, MO www.biomed-valley.com
fighting bacteria)
cobomarsen miRagen Therapeutics cutaneous T-cell lymphoma Phase II
(microRNA inhibitor) Boulder, CO www.miragen.com
ORPHAN DRUG
T-cell lymphoma/leukemia (adults) Phase I
www.miragen.com
cofetuzumab pelidotin AbbVie solid tumors Phase I
(ABBV-647/PF-06647020) North Chicago, IL www.abbvie.com
(PTK7 inhibitor) Pfizer www.pfizer.com
New York, NY
COM701 Compugen solid tumors (monotherapy and Phase I
(PVRIG inhibitor) South San Francisco, CA combination therapy) www.cgen.com
Medicines in Development: Cancer ǀ 2020 51
Product Name Sponsor Indication Development Status
COM902 Compugen advanced malignancies Phase I
(TIGIT inhibitor) South San Francisco, CA www.cgen.com
Copiktra® Secura Bio indolent NHL, PCTL (Fast Track) Phase II
duvelisib Las Vegas, NV www.securabio.com
ORPHAN DRUG
head and neck cancer Phase I/II
(combination therapy) www.securabio.com
cosibelimab (CK-301/TG-1501) Checkpoint Therapeutics B-cell malignancies Phase I
(anti-PD-L1 inhibitor) New York, NY www.checkpointtx.com
TG Therapeutics
New York, NY
COTI-2 Cotinga Pharmaceuticals solid tumors Phase I
(p53 gene modulator) Boston, MA
CPI-006 Corvus Pharmaceuticals advanced cancers Phase I
(anti-CD73 immunomodulator) Burlingame, CA www.corvuspharma.com
CPI-0209 Constellation Pharmaceuticals solid tumors Phase I
(EZHZ inhibitor) Cambridge, MA www.constellationpharma.com
Medicines in Development: Cancer ǀ 2020 52
Product Name Sponsor Indication Development Status
CPI-0610 Constellation Pharmaceuticals 1L myelofibrosis (Fast Track) Phase III
(BET inhibitor) Cambridge, MA www.constellationpharma.com
ORPHAN DRUG
2L myelofibrosis (Fast Track) Phase II
www.constellationpharma.com
CPI-100 Coordination Pharmaceuticals solid tumors Phase I
(nanotechnology-based Elk Grove Village, IL www.coordinationpharma.com
immunotherapy)
CPI-1205 Constellation Pharmaceuticals castration-resistant prostate cancer, Phase I
(EZH2 enzyme inhibitor) Cambridge, MA solid tumors (immuno-oncology www.constellationpharma.com
combinations)
CPI-200 Coordination Pharmaceuticals solid tumors Phase I
(nanotechnology-based Elk Grove Village, IL www.coordinationpharma.com
immunotherapy)
CPI-818 Corvus Pharmaceuticals T-cell lymphoma Phase I
(ITK inhibitor) Burlingame, CA www.corvuspharma.com
CPP-1X Cancer Prevention Pharmaceuticals gastric cancer Phase II
(ornithine decarboxylase inhibitor) Tucson, AZ www.canprevent.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 53
Product Name Sponsor Indication Development Status
CPP-1X/sul Cancer Prevention Pharmaceuticals familial adenomatous polyposis application submitted
(ornithine decarboxylase inhibitor/ Tucson, AZ (Fast Track) www.canprevent.com
sulindac)
ORPHAN DRUGcolon cancer risk reduction Phase III
www.canprevent.com
CPX-POM CicloMed solid tumors Phase I
(mitosis inhibitor) Kansas City, MO www.ciclomed.com
crenolanib AROG Pharmaceuticals 2L/3L relapsed/refractory AML Phase III
(FLT3/PDGFRα/β inhibitor) Plano, TX (Fast Track), 1L newly-diagnosed www.arogpharma.com
ORPHAN DRUG AML (Fast Track), gastrointestinal
stromal tumor (Fast Track)
post-transplant maintenance AML, Phase II
glioma www.arogpharma.com
gastric cancer Phase I
www.arogpharma.com
CS1001 CStone Pharmaceuticals T-cell lymphoma (Breakthrough Phase II
(anti-PD-L1 mAb) Suzhou, China Therapy) www.cstonepharma.com
ORPHAN DRUG
advanced solid tumors Phase I
www.cstonepharma.com
Medicines in Development: Cancer ǀ 2020 54
Product Name Sponsor Indication Development Status
CT053 CARsgen Therapeutics multiple myeloma Phase I
(CAR-T cell therapy) Bellaire, TX www.carsgen.com
ORPHAN DRUG
CTT1403 Cancer Targeted Technology prostate cancer Phase I
(PSMA-targeted radiopharmaceutical) Woodinville, WA www.cancertargetedtechnology.com
CTX110 CRISPR Therapeutics CD19-positive malignancies Phase I/II
(allogeneic CRISPR/Cas9 Cambridge, MA www.crisprtx.com
gene-edited CAR-T cell therapy)
CTX120 CRISPR Therapeutics multiple myeloma Phase I
(allogeneic CRISPR/Cas9 Cambridge, MA www.crisprtx.com
gene-edited CAR-T cell therapy)
ORPHAN DRUG
CTX130 CRISPR Therapeutics hematologic malignancies, Phase I
(allogeneic CRISPR/Cas9 Cambridge, MA solid tumors www.crisprtx.com
gene-edited CAR-T cell therapy)
CTX-471 Compass Therapeutics solid tumors Phase I
(CD137 agonist) Cambridge, MA www.compasstherapeutics.com
CUE-101 Cue Biopharma HPV-positive head and neck Phase I
(IL-1/pMHC fusion protein) Cambridge, MA squamous cell carcinoma www.cuebiopharma.com
Medicines in Development: Cancer ǀ 2020 55
Product Name Sponsor Indication Development Status
CVM-1118 TaiRx hepatocellular carcinoma Phase II
(angiogenesis inhibitor) Taipei, Taiwan www.trx.com.tw
CWP232291 JW Pharmaceutical AML, multiple myeloma Phase I
(WNT inhibitor) Seoul, South Korea www.jw-pharmaceutical.co.kr
CX072+CX2009 CytomX Therapeutics triple negative breast cancer Phase II
(PD-L1 probody immunotherapy + South San Francisco, CA www.cytomx.com
CD166 probody drug conjugate)
CX2009 CytomX Therapeutics ER-positive HER2-negative Phase II
(CD166 probody drug conjugate) South San Francisco, CA breast cancer www.cytomx.com
CX2029 CytomX Therapeutics lymphoma, solid tumors Phase I/II
(antibody-drug conjugate) South San Francisco, CA www.cytomx.com
CYAD-01 Celyad Oncology AML, MDS Phase I
(NKG2D CAR-T cell therapy) New York, NY www.celyad.com
CYAD-02 Celyad Oncology AML, MDS Phase I
(NKG2D CAR-T cell therapy) New York, NY www.celyad.com
CYAD-101 Celyad Oncology colorectal cancer Phase I
(NKG2D CAR-T cell therapy) New York, NY www.celyad.com
Medicines in Development: Cancer ǀ 2020 56
Product Name Sponsor Indication Development Status
CYC140 Cyclacel Pharmaceuticals AML, MDS Phase I
(PLK1 inhibitor) Berkeley Heights, NJ www.cyclacel.com
CYNK-001 Celularity multiple myeloma Phase I/II
(placental-derived natural killer Warren, NJ www.celularity.com
cell therapy)
AML, recurrent glioblastoma Phase I
www.celularity.com
Cyramza® Eli Lilly subcutaneous solid tumors, Phase I/II
ramucirumab Indianapolis, IN desmoplastic small round cell www.lilly.com
tumor (1-29 years), synovial
sarcoma (1-29 years)
CYT-0851 Cyteir Therapeutics B-cell malignancies, solid tumors Phase I/II
(RAD51 Inhibitor) Lexington, MA www.cyteir.com
CYTO-401 Cytocom pancreatic cancer Phase II
(proenkephalin analog) Winter Park, FL www.cytocom.com
CYTO-402 Cytocom hepatocellular carcinoma Phase I
(proenkephalin analog) Winter Park, FL www.cytocom.com
CYTO-403 Cytocom thyroid cancer Phase I
(proenkephalin analog) Winter Park, FL www.cytocom.com
Medicines in Development: Cancer ǀ 2020 57
Product Name Sponsor Indication Development Status
D-0502 InventisBio ER-positive HER2-negative Phase I
(SERD) Shanghai, China breast cancer www.inventisbio.com
D2C7 Istari Oncology glioblastoma (monotherapy) Phase II
(antibody-toxin conjugate) Research Triangle Park, NC www.istarioncology.com
ORPHAN DRUG
glioblastoma (combination therapy) Phase I
www.istarioncology.com
Danyelza® Y-mAbs Therapeutics 1L neuroblastoma (pediatric), Phase II
naxitamab New York, NY chemoimmunotherapy for www.ymabs.com
ORPHAN DRUG high-risk neuroblastoma,
2L relapsed osteosarcoma
daromun Philogen melanoma Phase III
(L19-IL2 and L19-TNF fusion protein) Siena, Italy www.philogen.com
Darzalex® Janssen Research & Development relapsed refractory multiple myeloma application submitted
daratumumab Raritan, NJ (1+ prior lines with PomDex) www.janssen.com
smoldering multiple myeloma, Phase III
1L multiple myeloma transplant www.janssen.com
eligible and ineligible (combination
therapy)
Medicines in Development: Cancer ǀ 2020 58
Product Name Sponsor Indication Development Status
DAY101 (TAK-580) Day One Biopharmaceuticals low grade glioma (pediatric) Phase I
(Raf kinase inhibitor) South San Francisco, CA (Breakthrough Therapy) www.dayonebio.com
ORPHAN DRUG
DB102 (enzastaurin) Denovo Biopharma 1L DLBCL Phase III
(serine-threonine kinase inhibitor) San Diego, CA www.denovobiopharma.com
ORPHAN DRUG
DB106 (vosaroxin) Denovo Biopharma 2L AML (Fast Track) Phase III
(DNA intercalator/type II DNA San Diego, CA www.denovobiopharma.com
topoisomerase inhibitor)
ORPHAN DRUG
DCC-3014 Deciphera Pharmaceuticals solid tumors Phase I
(CSF1R inhibitor) Waltham, MA www.deciphera.com
DCVAC/PCa SOTIO castration-resistant prostate cancer Phase III
(active cellular immunotherapy) Boston, MA www.sotio.com
DCVax®-Direct Northwest Biotherapeutics solid tumors Phase I/II
autologous dendritic cell vaccine Bethesda, MD www.nwbio.com
DCVax®-L Northwest Biotherapeutics newly-diagnosed glioblastoma Phase III
autologous dendritic cell vaccine Bethesda, MD www.nwbio.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 59
Product Name Sponsor Indication Development Status
DCVax®-Prostate Northwest Biotherapeutics prostate cancer Phase II
autologous dendritic cell vaccine Bethesda, MD www.nwbio.com
Debio 1143 Debiopharm head and neck cancer (combination Phase III
(apoptosis protein inhibitor) Lausanne, Switzerland therapy) (Breakthrough Therapy) www.debiopharm.com
solid tumors Phase I/II
www.debiopharm.com
Debio 1347 Debiopharm solid tumors (Fast Track) Phase II
(panFGFR inhibitor) Lausanne, Switzerland (FGFR1-3 fusion/rearrangement) www.debiopharm.com
defactinib Verastem Oncology cancer Phase II
(FAK inhibitor) Needham, MA www.verastem.com
ORPHAN DRUG
delolimogene mupadenorepvec Lokon Pharma pancreatic cancer, Phase I/II
(immunostimulatory gene therapy Uppsala, Sweden malignant melanoma www.lokonpharma.com
with oncolytic adenovirus therapy)
ORPHAN DRUG
derazantinib Basilea Pharmaceutica cholangiocarcinoma Phase II
(FGFR kinase inhibitor) Basel, Switzerland www.basilea.com
ORPHAN DRUG
urothelial cancer Phase I/II
www.basilea.com
Medicines in Development: Cancer ǀ 2020 60
Product Name Sponsor Indication Development Status
Descartes-08 Cartesian Therapeutics multiple myeloma Phase I/II
(anti-BCMA CAR-T cell therapy) Rockville, MD www.cartesiantherapeutics.com
Descartes-11 Cartesian Therapeutics multiple myeloma Phase II
(anti-BCMA CAR-T cell therapy) Rockville, MD www.cartesiantherapeutics.com
devimistat Rafael Pharmaceuticals 1L metastatic pancreatic cancer Phase III
(lipoic acid analogue/ Cranbury, NJ (combination therapy) (Fast Track), www.rafaelpharma.com
AEMD compound) AML (combination therapy)
ORPHAN DRUG
Burkitt's leukemia, Burkitt's lymphoma Phase II
high grade B-cell lymphoma, www.rafaelpharma.com
1L locally advanced pancreatic cancer
(combination therapy)
metastatic colorectal cancer Phase I/II
(combination therapy), www.rafaelpharma.com
myelodysplastic syndromes
(combination therapy)
1L advanced or metastatic Phase I
pancreatic cancer (combination www.rafaelpharma.com
therapy), T-cell lymphoma
(combination therapy)
DF1001 Dragonfly Therapeutics solid tumors Phase I/II
(NK cell engager immunotherapy) Waltham, MA www.dragongflytx.com
Medicines in Development: Cancer ǀ 2020 61
Product Name Sponsor Indication Development Status
DF6002 Bristol-Myers Squibb solid tumors Phase I/II
(IL12 Fc immunotherapy) Princeton, NJ www.dragongflytx.com
Dragonfly Therapeutics
Waltham, MA
DFP-10917 Delta-Fly Pharma AML Phase III
(DNA polymerase inhibitor) Tokushima, Japan www.delta-flypharma.co.jp
DFP-11207 Delta-Fly Pharma solid tumors Phase I
(thymidylate synthase Tokushima, Japan www.delta-flypharma.co.jp
expression inhibitor)
DFP-14927 Delta-Fly Pharma solid tumors Phase I
(DNA polymerase inhibitor) Tokushima, Japan www.delta-flypharma.co.jp
DHP107 Daehwa Pharmaceutical recurrent breast cancer Phase II
(oral paclitaxel) Seoul, South Korea www.dhpharm.co.kr
DICOPP® Cantex Pharmaceuticals pancreatic cancer Phase II
disulfiram + copper Westin, FL www.cantex.com
DI-Leu16-IL2 Alopexx Oncology non-Hodgkin lymphoma Phase II
(CD20 antigen inhibitor) Concord, MA www.alopexx.com
Medicines in Development: Cancer ǀ 2020 62
Product Name Sponsor Indication Development Status
DKN-01 Leap Therapeutics gynecologic cancer (combination Phase II
(DKK1 protein inhibitor) Cambridge, MA therapy), hepatobiliary cancer www.leaptx.com
ORPHAN DRUG (combination therapy),
gastroesophageal cancer (combination
therapy) (Fast Track)
prostate cancer (combination Phase I/II
therapy), hepatobiliary cancer www.leaptx.com
(combination therapy)
DKY709 Novartis solid tumors (combination therapy) Phase I
(IKZF2 protein degrader) East Hanover, NJ www.novartis.com
DM3001 DeMelle OncoPharma pancreatic cancer Phase I
(cytoplasmic/nuclear receptor Tarpon Springs, FL www.demelle-oncopharma.com
modulator)
DM-CHOC-PEN Dekk-Tec brain tumors Phase II
(DNA alkylating agent) New Orleans, LA www.dekk-tec.com
DN1508052 Shanghai De Novo Pharmatech solid tumors Phase I
(TLR8 agonist) Shanghai, China www.denovo-sh.com
Medicines in Development: Cancer ǀ 2020 63
Product Name Sponsor Indication Development Status
DNX-2401 (tasadenoturev) DNAtrix recurrent glioblastoma (Fast Track) Phase II
ORPHAN DRUG Houston, TX (+pembrolizumab), www.dnatrix.com
DIPG (pediatric) Phase I
(combination therapy) www.dnatrix.com
DNX-2440 DNAtrix recurrent glioblastoma Phase I
(OX40L-armed virus) Houston, TX www.dnatrix.com
DocePLUS™ Plus Therapeutics SCLC, solid tumors Phase I
albumin-stabilized PEGylated Austin, TX www.plustherapeutics.com
liposomal docetaxel
ORPHAN DRUG
doranidazole Shuttle Pharmaceuticals pancreatic cancer Phase I
(cell wall inhibitor) Rockville, MD www.shuttlepharma.com
dostarlimab AnaptysBio dMMR/MSI-H recurrent endometrial application submitted
(anti-PD-1 antibody) San Diego, CA cancer www.gsk.com
GlaxoSmithKline
Research Triangle Park, NC
recurrent or primary advanced Phase III
endometrial cancer, epithelial www.gsk.com
ovarian cancer
solid tumors Phase I
www.gsk.com
Medicines in Development: Cancer ǀ 2020 64
Product Name Sponsor Indication Development Status
dovitinib Allarity Therapeutics renal cell carcinoma Phase III
(pan-tyrosine kinase inhibitor) Cambridge, MA www.allarity.com
DPV-001 UbiVac adjuvant NSCLC Phase II
(cancer vaccine) Portland, OR www.ubivac.com
triple negative breast cancer, Phase I
head and neck cancer, www.ubivac.com
prostate cancer
DPX-Survivac/CPA IMV ovarian cancer (monotherapy and Phase II
(peptide cancer vaccine) Quebec, Canada combination therapy) (Fast Track), www.imv-inc.com
ORPHAN DRUG solid tumors (+pembrolizumab)
DRP-104 Dracen Pharmaceuticals advanced solid tumors Phase I/II
(glutamine inhibitor) New York, NY www.dracenpharma.com
DS-1055 Daiichi Sankyo solid tumors Phase I
(anti-GAPP antibody) Basking Ridge, NJ www.dsi.com
DS-1062 Daiichi Sankyo triple negative breast cancer, NSCLC Phase I
(anti-TROP2-ADC) Basking Ridge, NJ www.dsi.com
DS-3201 (valemetostat) Daiichi Sankyo ALL, AML, PTCL Phase I
(EZH1/2 inhibitor) Basking Ridge, NJ www.dsi.com
Medicines in Development: Cancer ǀ 2020 65
Product Name Sponsor Indication Development Status
DS-6157 Daiichi Sankyo gastrointestinal stromal tumors Phase I
(anti-GPR20-ADC) Basking Ridge, NJ www.dsi.com
DS-7300 Daiichi Sankyo solid tumors Phase I
(anti-B7-H3-ADC) Basking Ridge, NJ www.dsi.com
DSP107 KAHR Medical solid tumors Phase I/II
(anti-CD47 fusion protein) Jerusalem, Israel www.kahrbio.com
DSP-0337 Sumitomo Dainippon advanced solid tumors Phase I
(napabucasin prodrug) Pharma Oncology www.sdponcology.com
Cambridge, MA
DSP-0509 Sumitomo Dainippon advanced solid tumors Phase I/II
(TLR7 agonist) Pharma Oncology www.sdponcology.com
Cambridge, MA
DSP-7888 Sumitomo Dainippon glioblastoma Phase II
(WT1 cancer vaccine) Pharma Oncology www.dsponcology.com
ORPHAN DRUG Cambridge, MA
advanced solid tumors Phase I/II
www.dsponcology.com
DST-2970 DisperSol Technologies prostate cancer Phase I
(CYP17A1 protein inhibitor) Georgetown, TX www.dispersoltech.com
Medicines in Development: Cancer ǀ 2020 66
Product Name Sponsor Indication Development Status
DSTAT Chimerix AML (Fast Track) Phase III
(dociparstat sodium) Durham, NC www.chimerix.com
ORPHAN DRUG
DTRM-555 Zhejiang DTRM Biopharma CLL, NHL Phase II
(BTK inhibitor) Hangzhou, China
DTRMWXHS-12 Zhejiang DTRM Biopharma CLL, B-cell lymphoma Phase I
(BTK inhibitor) Hangzhou, China
dubermatinib (TP-0903) Sumitomo Dainippon advanced solid tumors Phase I
(AXL inhibitor) Pharma Oncology www.sdponcology.com
Cambridge, MA
DV281 Dynavax Technologies NSCLC Phase I completed
(TLR9 agonist) Emeryville, CA www.dynavax.com
DZD1516 Dizal Pharmaceuticals HER2-positive breast cancer Phase I
(HER2 tyrosine kinase inhibitor) Shanghai, China www.dizalpharma.com
DZD9008 Dizal Pharmaceuticals NSCLC Phase I
(EGFR inhibitor) Shanghai, China www.dizalpharma.com
E6201 Spirita Oncology melanoma CNS metastases Phase I
(MEK inhibitor) Boston, MA www.spiritaoncology.com
Medicines in Development: Cancer ǀ 2020 67
Product Name Sponsor Indication Development Status
E7766 Eisai solid tumors Phase I
(STING agonist) Woodcliff Lake, NJ www.eisai.com
ECT-001 ExCellThera high-risk hematologic malignancies Phase II
(ex vivo expansion of umbilical Montreal, Canada www.excellthera.com
hematopoietic stem cell therapy)
EDP1503 Evelo Biosciences solid tumors Phase I/II
(monoclonal microbial therapeutic) Cambridge, MA www.evelobio.com
eflornithine Orbus Therapeutics anaplastic astrocytoma Phase III
(ornithine decarboxylase inhibitor) Palo Alto, CA (Breakthrough Therapy) www.orbustherapeutics.com
ORPHAN DRUG
eFT226 (zotatifin) eFFECTOR Therapeutics solid tumors Phase I/II
(eIF4A inhibitor) San Diego, CA www.effector.com
eftilagimod alpha (IMP321) Immutep head and neck squamous cell Phase II
(LAG3 fusion protein) Sydney, Australia carcinoma, NSCLC www.immutep.com
EGF816 (nazartinib) Novartis NSCLC (combination therapy) Phase I
(EGFR inhibitor) East Hanover, NJ www.novartis.com
EI1071 Elixiron Immunotherapeutics cancer Phase I
(CSF-1R inhibitor) Taipei, Taiwan www.elixiron.com
Medicines in Development: Cancer ǀ 2020 68
Product Name Sponsor Indication Development Status
elacestrant (RAD1901) Radius Health ER-positive breast cancer Phase III
(SERD) Waltham, MA (Fast Track) www.radiuspharm.com
eltanexor (KPT-8602) Karyopharm Therapeutics colorectal cancer, prostate cancer, Phase I
(SINE inhibitor) Newton, MA MDS www.karyopharm.com
Elzonris® Stemline Therapeutics chronic myelomonocytic leukemia Phase II
tagraxofusp-erzs New York, NY (CMML), myelofibrosis www.stemline.com
EMB-01 EpimAb Biotherapeutics solid tumors Phase I/II
(bispecific antibody) Shanghai, China www.epimab.com
enadenotucirev PsiOxus Therapeutics metastatic or advanced Phase I
(oncolytic virus therapeutic) Abingdon, United Kingdom epithelial tumors www.psioxus.com
enapotamab vedotin Genmab solid tumors Phase I/II
(HuMax-AXL-ADC) Plainsboro, NJ www.genmab.com
encequidar Athenex Oncology metastatic breast cancer Phase III
(P-gp inhibitor) Buffalo, NY (combination therapy) www.athenexoncology.com
ORPHAN DRUG
angiosarcoma of the skin, Phase I/II
gastric cancer, solid tumors www.athenexoncology.com
Medicines in Development: Cancer ǀ 2020 69
Product Name Sponsor Indication Development Status
endoxifen Atossa Therapeutics breast cancer Phase II
(tamoxifen metabolite) Seattle, WA www.atossatherapeutics.com
Enhertu® AstraZeneca 3L HER2-positive gastric cancer application submitted
fam-trastuzumab deruxtecan-nxki Wilmington, DE www.dsi.com
ORPHAN DRUG Daiichi Sankyo www.astrazeneca.com
Basking Ridge, NJ
2L/3L HER2-positive breast cancer, Phase III
3L HER2-low breast cancer, www.dsi.com
HER2-low breast cancer (chemo naïve) www.astrazeneca.com
triple negative breast cancer, Phase II
HER2-positive colorectal cancer, www.dsi.com
2L HER2-positive gastric cancer www.astrazeneca.com
(Breakthrough Therapy), NSCLC
(Breakthrough Therapy), NSCLC,
HER2-expressing tumors
breast cancer (combination therapy), Phase I
bladder cancer (combination therapy) www.dsi.com
1L/2L HER2-positive gastric cancer www.astrazeneca.com
Breakthrough Therapy),
NSCLC (combination therapy)
enoblituzumab MacroGenics head and neck squamous cell Phase II
Rockville, MD carcinoma www.macrogenics.com
Medicines in Development: Cancer ǀ 2020 70
Product Name Sponsor Indication Development Status
ensartinib Xcovery 1L NSCLC Phase III
(ALK inhibitor) Palm Beach Gardens, FL www.xcovery.com
2L NSCLC, ROS1 gene rearrangement Phase II
NSCLC, solid tumors www.xcovery.com
entinostat Syndax Pharmaceuticals HR-positive, HER2-negative breast Phase III
(HDAC inhibitor) Waltham, MA cancer (combination therapy) www.syndax.com
(Breakthrough Therapy)
entospletinib Kronos Bio AML Phase II
(Syk inhibitor) San Mateo, CA www.kronosbio.com
envafolimab (KN035) TRACON Pharmaceuticals sarcoma Phase II
(anti-PD-L1 antibody) San Diego, CA www.traconpharma.com
undifferentiated pleomorphin Phase I
sarcoma, myxofibrosarcoma, www.traconpharma.com
solid tumors
EO2401 Enterome progressive glioblastoma, Phase I/II
(multi-peptide therapeutic Cambridge, MA metastatic adrenocortical carcinoma, www.enterome.com
vaccine) malignant pheochromocytoma/
paraganglioma
EOS100850 iTeos Therapeutics solid tumors Phase I
(adenosine A2A antagonist) Cambridge, MA www.iteostherapeutics.com
Medicines in Development: Cancer ǀ 2020 71
Product Name Sponsor Indication Development Status
EP-100 Esperance Pharmaceuticals ovarian cancer Phase II
(peptide conjugate targeting LHRH) Houston, TX www.esperancepharma.com
solid tumors Phase I
www.esperancepharma.com
epacadostat Incyte solid tumors Phase I/II
(IDO1 inhibitor) Wilmington, DE www.incyte.com
epcoritamab AbbVie B-cell hematologic malignancies Phase II
(DuoBody® CD3xCd20) North Chicago, IL www.genmab.com
Genmab
Plainsboro, NJ
EPI-7386 ESSA Pharma metastatic castration-resistant Phase I
(androgen receptor antagonist) Houston, TX prostate cancer (Fast Track) www.essapharma.com
eprenetapopt (APR-246) Aprea Therapeutics 1L TP53-mutant MDS (Fast Track) Phase III
(tumor suppressor protein Boston, MA (Breakthrough Therapy) www.aprea.com
p53 stimulant)
ORPHAN DRUGpost-transplant MDS maintenance, Phase II
1L TP53-mutant AML (Fast Track) www.aprea.com
TP53-mutant CLL, TP53-mutant Phase I
mantle cell lymphoma, solid tumors www.aprea.com
Medicines in Development: Cancer ǀ 2020 72
Product Name Sponsor Indication Development Status
eRapa™ Emtora Biosciences low-grade prostate cancer Phase I
rapamycin micro-encapsulated San Antonio, TX www.emtorabio.com
Erleada® Janssen Research & Development high-risk prostate cancer, localized Phase III
apalutamide Raritan, NJ prostate cancer (androgen www.janssen.com
deprivation therapy), metastatic
castration-resistant chemotherapy
naïve prostate cancer (+abiraterone)
ERY974 Chugai Pharmaceutical solid tumors Phase I
(GPCC3xCD3 bispecific antibody) Tokyo, Japan www.chugai-pharm.co.jp
ET140202 Eureka Therapeutics hepatocellular carcinoma Phase I/II
(CAR-T cell therapy) Emeryville, CA www.eurekatherapeutics.com
ET140203 Eureka Therapeutics hepatocellular carcinoma Phase I/II
(CAR-T cell therapy) Emeryville, CA www.eurekatherapeutics.com
exicorilant Corcept Therapeutics castration-resistant prostate cancer Phase II
(GR II antagonist) Menlo Park, CA (+enzalutamide) www.corcept.com
fadraciclib Cyclacel Pharmaceuticals AML, CLL, MDS, solid tumors Phase I
(CDK inhibitor) Berkeley Heights, NJ www.cyclacel.com
FCN-437 Fochon Pharmaceuticals solid tumors Phase I
(CDK4/6 inhibitor) San Leandro, CA www.fochon.com
Medicines in Development: Cancer ǀ 2020 73
Product Name Sponsor Indication Development Status
fenretinide intravenous CerRx PTCL Phase II
Lubbock, TX www.cerrx.com
fexapotide (NX-1207) Nymox Pharmaceutical early-stage prostate cancer Phase II completed
(apoptosis stimulant) Hasbrouck Heights, NJ www.nymox.com
FF-10101-01 FUJIFILM Pharmaceuticals U.S.A. AML Phase I
(FLT3 inhibitor) Cambridge, MA www.fujifilmpharma.com
FF-10502-01 FUJIFILM Pharmaceuticals U.S.A. solid tumors Phase II
(DNA polymerase inhibitor) Cambridge, MA www.fujifilmpharma.com
FF-10832 FUJIFILM Pharmaceuticals U.S.A. solid tumors Phase I
(liposomal gemcitabine) Cambridge, MA www.fujifilmpharma.com
FF-10850 FUJIFILM Pharmaceuticals U.S.A. solid tumors Phase I
(DNA topoisomerase I inhibitor) Cambridge, MA www.fujifilmpharma.com
FF-21101 FUJIFILM Pharmaceuticals U.S.A. solid tumors Phase II
(anti-P-cadherin antibody, Cambridge, MA www.fujifilmpharma.com
90Y conjugate)
ficlatuzumab AVEO Oncology head and neck squamous cell Phase II
(HGF inhibitor) Boston, MA carcinoma, metastatic pancreatic www.aveooncology.com
ductal cancer
Medicines in Development: Cancer ǀ 2020 74
Product Name Sponsor Indication Development Status
FID-007 Fulgent Pharma solid tumors Phase I
(paclitaxel nanoencapsulated) Temple City, CA www.fulgentpharma.com
fimaporfin delivered gemcitabine PCI Biotech cholangiocarcinoma Phase II
ORPHAN DRUG Oslo, Norway www.pcibiotech.no
fimepinostat Curis HYC-altered lymphoma, including Phase II
(HDAC/PI3K Inhibitor) Lexington, MA DLBCL (Fast Track) www.curis.com
ORPHAN DRUG
fisogatinib Blueprint Medicines hepatocellular carcinoma Phase I
(FGFR4 inhibitor) Cambridge, MA www.blueprintmedicines.com
ORPHAN DRUG
FKB238 Centus Biotherapeutics NSCLC Phase III
(bevacizumab biosimilar) Cambridge, United Kingdom www.centusbiotherapeutics.com
flotetuzumab MacroGenics AML Phase III
(CD123xCD3 bispecific antibody) Rockville, MD www.macrogenics.com
ORPHAN DRUG
FLX475 RAPT Therapeutics cancer Phase I/II
(CCR4 receptor antagonist) South San Francisco, CA www.rapt.com
Medicines in Development: Cancer ǀ 2020 75
Product Name Sponsor Indication Development Status
FN-1501 Fosun Pharmaceutical solid tumors Phase I
(FLT3/CDK2/CDK4/CDK6 inhibitor) Shanghai, China www.fosunpharma.com
FOR46 Fortis Therapeutics multiple myeloma, castration- Phase I
(anti-CD46 antibody-drug conjugate) San Diego, CA resistant prostate cancer
FPA150 Five Prime Therapeutics solid tumors Phase I
(B7-H4 antibody) South San Francisco, CA www.fiveprime.com
FPI-1434 Fusion Pharmaceuticals solid tumors Phase I
(engineered alpha particles) Boston, MA www.fusionpharma.com
FPT155 Five Prime Therapeutics solid tumors Phase I
(CD80-FC fusion protein) South San Francisco, CA www.fiveprime.com
FS118 F-star Therapeutics advanced malignancies Phase I
(LAG-3xCD223 bispecific antibody) Cambridge, MA www.f-star.com
FSI-189 Gilead Sciences NHL Phase I
(SIRPα inhibitor) Foster City, CA www.gilead.com
FT-2102 FORMA Therapeutics AML, glioma, MDS, solid tumors Phase I/II
(IDH1 inhibitor) Watertown, MA www.formatherapeutics.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 76
Product Name Sponsor Indication Development Status
FT-7051 FORMA Therapeutics metastatic castration-resistant Phase I
(IDH1m inhibitor) Watertown, MA prostate cancer www.formatherapeutics.com
FT500 Fate Therapeutics advanced solid tumors Phase I
(NK cell immunotherapy) San Diego, CA www.fatetherapeutics.com
FT516 Fate Therapeutics AML, B-cell lymphoma, Phase I
(NK cell immunotherapy) San Diego, CA advanced solid tumors www.fatetherapeutics.com
FT538 Fate Therapeutics AML, multiple myeloma Phase I
(NK cell immunotherapy) San Diego, CA www.fatetherapeutics.com
FT596 Fate Therapeutics CLL, B-cell lymphoma Phase I
(NK cell immunotherapy) San Diego, CA www.fatetherapeutics.com
fulvestrant Eagle Pharmaceuticals breast cancer Phase I
(injectable formulation) Woodcliff Lake, NJ www.eagleus.com
G1T-48 G1 Therapeutics breast cancer Phase I/II
(SERD) Research Triangle Park, NC www.g1therapeutics.com
Medicines in Development: Cancer ǀ 2020 77
Product Name Sponsor Indication Development Status
galinpepimut-S Sellas Life Sciences AML (Fast Track) Phase III
(WT1 cancer vaccine) New York, NY www.sellaslifesciences.com
ORPHAN DRUG
multiple myeloma (Fast Track) Phase II
www.sellaslifesciences.com
malignant pleural mesothelioma Phase I
(combination therapy) (Fast Track), www.sellaslifesciences.com
ovarian cancer (combination therapy),
advanced cancers (combination therapy)
Gavreto™ Blueprint Medicines RET-altered solid tumors Phase I/II
pralsetinib Cambridge, MA www.blueprintmedicines.com
Roche/Genentech
South San Francisco, CA
GB1275 Gossamer Bio (GB006) solid tumors, including pancreatic Phase I/II
(CD11b modulator) San Diego, CA cancer www.gossamerbio.com
ORPHAN DRUG
GC33 (codrituzumab) Chugai Pharmaceutical hepatocellular carcinoma Phase I
(glypican 3 inhibitor) Tokyo, Japan www.chugai-pharm.co.jp
GC4419 (avasopasem) Galera Therapeutics pancreatic cancer (+stereotactic Phase II
(superoxide dismutase modulator) Malvern, PA body radiation therapy) www.galeratx.com
Medicines in Development: Cancer ǀ 2020 78
Product Name Sponsor Indication Development Status
GC4711 Galera Therapeutics NSCLC (+stereotactic body radiation Phase II
(superoxide dismutase modulator) Malvern, PA therapy) www.galeratx.com
GD2-GD3 vaccine Y-mAbs Therapeutics high-risk neuroblastoma Phase II
ORPHAN DRUG New York, NY www.ymabs.com
GDC-0084 (paxalisib) Kazia Therapeutics newly-diagnosed glioblastoma Phase II
(PI3K inhibitor) Sydney, Australia (Fast Track) www.kaziatherapeutics.com
ORPHAN DRUG
GEM3PSCA Bristol-Myers Squibb solid tumors Phase I
(CD3xPSCA bispecific antibody) Princeton, NJ www.bms.com
GEMoaB
Dresden, Germany
GEN333 Bristol-Myers Squibb AML Phase I
(CD3xCD33 bispecific antibody) Princeton, NJ www.bms.com
GEMoaB
Dresden, Germany
GEN-009 Genocea Biosciences solid tumors Phase I
(neoantigen cancer vaccine) Cambridge, MA www.genocea.com
GEN-011 Genocea Biosciences solid tumors Phase I
(autologous cell therapy) Cambridge, MA www.genocea.com
Medicines in Development: Cancer ǀ 2020 79
Product Name Sponsor Indication Development Status
GEN-1 Celsion ovarian cancer Phase II
(IL-12 gene therapy nanoparticle) Lawrenceville, NJ www.celsion.com
ORPHAN DRUG
GEN1029 Genmab solid tumors Phase I
(HexaBody®-DR5/DR5) Plainsboro, NJ www.genmab.com
GEN1042 Genmab solid tumors Phase I
(DuoBody® CD40x4-1BB) Plainsboro, NJ www.genmab.com
GEN1044 AbbVie solid tumors Phase I
(DuoBody® CD3x5T4) North Chicago, IL www.genmab.com
Genmab
Plainsboro, NJ
GEN1046 Genmab solid tumors Phase I
(DuoBody® PD-L1x4-1BB) Plainsboro, NJ www.genmab.com
GEN2 GenVivo liver cancer Phase I
(cancer immunotherapeutic) San Marino, CA www.genvivoinc.com
GEN3009 AbbVie hematologic malignancies Phase I
(DuoBody® CD37xCD37) North Chicago, IL www.genmab.com
Genmab
Plainsboro, NJ
Medicines in Development: Cancer ǀ 2020 80
Product Name Sponsor Indication Development Status
Gilotrif® Boehringer Ingelheim NSCLC (+pembrolizumab) Phase II
afatinib Ridgefield, CT www.boeringer-ingelheim.com
neuroectodermal tumors, Phase I/II
rhabdomyosarcoma www.boeringer-ingelheim.com
GL-ONC1 Genelux ovarian cancer Phase I/II
(oncolytic immunotherapy) San Diego, CA www.genelux.com
GLR2007 Gan & Lee Pharmaceuticals solid tumors, including glioblastoma, Phase I/II
(CDK4/6 inhibitor) Bridgewater, NJ NSCLC www.ganlee.com
ORPHAN DRUG
glufosfamide Eleison Pharmaceuticals pancreatic cancer (Fast Track) Phase III
(glucose conjugate of ifosfamide) Princeton, NJ www.eleison-pharma.com
ORPHAN DRUG
GMI-1359 GlycoMimetics HR-positive metastatic breast cancer Phase I
(E-selectin/CXCR4 antagonist) Rockville, MD www.glycomimetics.com
GNX102 GlycoNex solid tumors Phase I
(anti-TACAs antibody) New Taipei City, Taiwan www.glyconex.com.tw
goserelin extended release Luye Pharma prostate cancer Phase III
Shanghai, China www.luye.cn
Medicines in Development: Cancer ǀ 2020 81
Product Name Sponsor Indication Development Status
GP2 Greenwich Life Sciences prevent breast cancer recurrences Phase II
(HER2/Neu GP2 immunotherapy) Stafford, TX following surgery www.greenwichlifesciences.com
GP-2250 Geistlich pancreatic cancer Phase I/II
(taurultam and an oxathiazine Wolhusen, Switzerland www.geistlich-pharma.com
derivative analogue)
GPRC5D CAR-T cell therapy Bristol-Myers Squibb multiple myeloma Phase I
Princeton, NJ www.bms.com
Eureka Therapeutics
Emeryville, CA
GQ1001 GeneQuantum Healthcare HER2-positive solid tumors Phase I
(HER2 antibody-drug conjugate) Beijing, China www.genequantum.com
GRANITE Gritstone Oncology microsatellite stable colorectal Phase I/II
(neoantigen-based immunotherapy) Emeryville, CA cancer (Fast Track), gastroesophageal www.gritstoneoncology.com
cancer
Graspa® Erytech Pharma pancreatic cancer (Fast Track) Phase III
eryaspase (asparaginase encapsulated) Cambridge, MA www.erytech.com
ORPHAN DRUG
2L ALL, 1L triple negative breast Phase II
cancer www.erytech.com
Medicines in Development: Cancer ǀ 2020 82
Product Name Sponsor Indication Development Status
GR-MD-02 (belapectin) Galectin Therapeutics melanoma(+pembrolizumab), Phase I
(galectin 3 inhibitor) Norcross, GA head and neck squamous cell www.galectintherapeutics.com
carcinoma (+pembrolizumab),
NSCLC (+pembrolizumab)
GRN-1201 BrightPath Biotherapeutics NSCLC (+pembrolizumab) Phase II
(peptide vaccine) Tokyo, Japan www.brightpathbio.com
GS-1423 Gilead Sciences solid tumors Phase I
(anti-CD73/TGFß trap) Foster City, CA www.gilead.com
GS-4224 Gilead Sciences NSCLC Phase I
(oral PD-L1 inhibitor) Foster City, CA www.gilead.com
GSK3174998 GlaxoSmithKline hematologic malignancies, Phase I
(OX40 agonist) Research Triangle Park, NC solid tumors www.gsk.com
GSK3326595 GlaxoSmithKline hematologic malignancies, Phase I/II
(PRMT5 inhibitor) Research Triangle Park, NC solid tumors www.gsk.com
GSK3359609 GlaxoSmithKline head and neck squamous cell Phase II/III
(ICOS receptor agonist) Research Triangle Park, NC carcinoma www.gsk.com
GSK3368715 GlaxoSmithKline cancer Phase I
(type 1 PRMT inhibitor) Research Triangle Park, NC www.gsk.com
Medicines in Development: Cancer ǀ 2020 83
Product Name Sponsor Indication Development Status
GSK3377794 GlaxoSmithKline synovial sarcoma Phase II
(letetresgene-autoleucel) Research Triangle Park, NC (Breakthrough Therapy) www.gsk.com
ORPHAN DRUG
GSK3537142 GlaxoSmithKline cancer Phase I
(anti-NY-ESO-1) Research Triangle Park, NC www.gsk.com
Immunocore
Conshohocken, PA
GSK3745417 GlaxoSmithKline cancer Phase I
(STING agonist) Research Triangle Park, NC www.gsk.com
GSK3845097 GlaxoSmithKline cancer Phase I
(engineered TCR T cells targeting Research Triangle Park, NC www.gsk.com
NY-ESO-1)
GSK3901961 GlaxoSmithKline cancer Phase I
(engineered TCR T cells targeting Research Triangle Park, NC www.gsk.com
NY-ESO-1)
GSK4069889 (TSR-022) AnaptysBio cancer Phase I/II
(anti-T cell immunoglobulin/ San Diego, CA www.gsk.com
TIM-1 antibody) GlaxoSmithKline
Research Triangle Park, NC
Medicines in Development: Cancer ǀ 2020 84
Product Name Sponsor Indication Development Status
GSK4074386 (TSR-033) AnaptysBio cancer Phase I
(LAG-3 antibody) San Diego, CA www.gsk.com
GlaxoSmithKline
Research Triangle Park, NC
GSK6097608 GlaxoSmithKline cancer Phase I
(CD96 antagonist) Research Triangle Park, NC www.gsk.com
GT103 Grid Therapeutics NSCLC Phase I/II
(complement factor H inhibitor) Durham, NC www.gridtherapeutics.com
GTB-3550 GT Biopharma hematologic malignancies Phase I/II
(tri-specific killer engager) Beverly Hills, CA www.gtbiopharma.com
GZ17-6.02 Genzada Pharmaceuticals USA lymphoma, solid tumors Phase I
(super enhancer inhibitor) Hutchinson, KS www.genzada.com
H3B-6527 H3 Biomedicine (Eisai) hepatocellular carcinoma Phase I
(FGFR4 inhibitor) Cambridge, MA www.h3biomedicine.com
ORPHAN DRUG
H3B-6545 H3 Biomedicine (Eisai) breast cancer Phase I/II
(estrogen receptor-alpha inhibitor) Cambridge, MA www.h3biomedicine.com
breast cancer (combination therapy) Phase I
www.h3biomedicine.com
Medicines in Development: Cancer ǀ 2020 85
Product Name Sponsor Indication Development Status
H3B-8800 H3 Biomedicine (Eisai) hematologic malignancies Phase I
(SF3B1 protein modulator) Cambridge, MA www.h3biomedicine.com
ORPHAN DRUG
Halaven® Eisai triple negative breast cancer Phase I/II
eribulin mesylate Woodcliff Lake, NJ www.eisai.com
haNK ImmunityBio 2L Merkel cell carcinoma Phase II
(activated natural killer cell therapy) Culver City, CA (+N-803 & avelumab) www.nantkwest.com
ORPHAN DRUG NantKwest
Culver City, CA
HB-201 Hookipa Pharma HPV-related squamous cell carcinoma Phase I/II
(antigenic E6/E7 fusion protein) New York, NY www.hookipapharma.com
HB-202/201 Hookipa Pharma HPV-related squamous cell carcinoma Phase I/II
(antigenic E6/E7 fusion protein) New York, NY www.hookipapharma.com
HC-1119 Hinova Pharmaceuticals USA metastatic castration-resistant Phase III
(androgen receptor antagonist) San Diego, CA prostate cancer www.hinovapharma.com
HDM201 Novartis hematologic malignancies Phase I
(MDM2 inhibitor) East Hanover, NJ www.novartis.com
HemaMax Neumedicines cutaneous T-cell lymphoma Phase II
recombinant human interleukin-12 Los Angeles, CA www.neumedicines.com
Medicines in Development: Cancer ǀ 2020 86
Product Name Sponsor Indication Development Status
HH2710 HaiHe Biopharma solid tumors Phase I
(ERK1/2 inhibitor) Shanghai, China www.en.haihepharma.com
HH2853 HaiHe Biopharma solid tumors Phase I
(EZH1/2 inhibitor) Shanghai, China www.en.haihepharm.com
HHCYH33 HaiHe Biopharma solid tumors Phase I
(PI3Kα inhibitor) Shanghai, China www.en.haihepharm.com
Hiltonol® Oncovir solid tumors Phase II
Poly ICLC vaccine Washington, DC www.oncovir.com
malignant pleural mesothelioma, Phase I
solid tumors (combination therapy) www.oncovir.com
HL-085 Kechow Pharma solid tumors Phase I
(MAP 1 kinase inhibitor) Shanghai, China
HM43239 Hanmi Pharmaceutical AML Phase I/II
(FLT-3 inhibitor) Seoul, South Korea www.hanmipharm.com
HMPL-012 (surufatinib) Hutchison MediPharma advanced solid tumors, including Phase I/II
(multi tyrosine kinase inhibitor) Hong Kong biliary tract cancer, neuroendocrine www.chi-med.com
ORPHAN DRUG tumors (Fast Track), soft tissue
sarcoma
Medicines in Development: Cancer ǀ 2020 87
Product Name Sponsor Indication Development Status
HMPL-013 (fruquintinib) Hutchison MediPharma metastatic colorectal cancer Phase III
(VEFGR antagonist) Hong Kong (Fast Track) www.chi-med.com
solid tumors Phase I
www.chi-med.com
HMPL-523 Hutchison MediPharma NHL Phase I
(SYK inhibitor) Hong Kong www.chi-med.com
HMPL-689 Hutchison MediPharma lymphoma Phase I
(PI3Kδ inhibitor) Hong Kong www.chi-med.com
HPN217 AbbVie multiple myeloma Phase I
(B-cell maturation antigen) North Chicago, IL www.harpoontx.com
Harpoon Therapeutics
South San Francisco, CA
HPN328 Harpoon Therapeutics SCLC Phase I/II
(DLL2 T cell engager) South San Francisco, CA www.harpoontx.com
HPN424 Harpoon Therapeutics prostate cancer Phase I
(PSMA targeting T-cell engager) South San Francisco, CA www.harpoontx.com
HPN536 Harpoon Therapeutics cancer with mesothelin Phase I/II
(T lymphocyte stimulant) South San Francisco, CA expression www.harpoontx.com
Medicines in Development: Cancer ǀ 2020 88
Product Name Sponsor Indication Development Status
HQP1351 Ascentage Pharma refractory CML (Fast Track) Phase II
(bcr-abl tyrosine kinase/KIT/ Rockville, MD www.ascentagepharma.com
FLT3 inhibitor)
ORPHAN DRUG
HS-130 Heat Biologics solid tumors Phase I
(OX40L cell therapy) Morrisville, NC www.heatbio.com
HSC100 Tacitus Therapeutics hematologic malignancies Phase I
(stem cell therapy) New York, NY www.tacitustherapeutics.com
huMNC2-CAR44 T cells Minerva Biotechnologies breast cancer Phase I
(anti-MUC1 CAR-T cell therapy) Waltham , MA www.minervabio.com
I-131-109 Progenics Pharmaceuticals prostate cancer Phase II
(PSMA-targeted radiotherapy) New York, NY www.progenics.com
iberdomide (CC-220) Bristol-Myers Squibb multiple myeloma Phase II
(immunomodulatory therapeutic) Princeton, NJ www.bms.com
non-Hodgkin lymphoma Phase I
www.bms.com
IBI188 Innovent Biologics MDS Phase I
(anti-CD47 mAb) San Mateo, CA www.innoventbio.com
Medicines in Development: Cancer ǀ 2020 89
Product Name Sponsor Indication Development Status
Ibrance® Pfizer ER-positive HER2-positive breast Phase III
palbociclib New York, NY cancer www.pfizer.com
ibudilast (MN-166) MediciNova newly-diagnosed glioblastoma, Phase I/II
(PDE4/10 inhibitor/MIF inhibitor) La Jolla, CA recurrent glioblastoma www.medicinova.com
ORPHAN DRUG
Iclusig® Takeda 1L Ph-positive ALL Phase III
ponatinib Deerfield, IL www.takeda.com
ICP-022 InnoCare Pharma r/r B-cell malignancies Phase I
(BTK inhibitor) Beijing, China www.innocarepharma.com
ICP-192 InnoCare Pharma solid tumors Phase I/II
(FGFR inhibitor) Beijing, China www.innocarepharma.com
IDE196 IDEAYA Biosciences solid tumors Phase I/II
(PKC inhibitor) South San Francisco, CA www.ideayabio.com
Medicines in Development: Cancer ǀ 2020 90
Product Name Sponsor Indication Development Status
ide-cel (bb2121) bluebirdbio multiple myeloma (after 3+ prior application submitted
(idecabtagene vicleucel) Cambridge, MA therapies) (Breakthrough Therapy) www.bms.com
ORPHAN DRUG Bristol-Myers Squibb
Princeton, NJ
3L, 5L multiple myeloma Phase III
www.bms.com
2L multiple myeloma, Phase II
4L+ multiple myeloma www.bms.com
high-risk multiple myeloma Phase I
www.bms.com
Idhifa® Agios Pharmaceuticals relapsed/refractory AML with Phase III
enasidenib Cambridge, MA IDH2 mutation www.bms.com
Bristol-Myers Squibb
Princeton, NJ
1L AML with IDH2 mutation Phase II
www.bms.com
ifabotuzumab Humanigen glioblastoma Phase I
(anti-EphA3 mAb) Burlingame, CA www.humanigen.com
Ifx-Hu2.0 (pDNA) Morphogenesis cutaneous squamous cell carcinoma, Phase I
(whole cell cancer vaccine) Tampa, FL Merkel cell carcinoma www.morphogenesis-inc.com
Medicines in Development: Cancer ǀ 2020 91
Product Name Sponsor Indication Development Status
IGF-MTX IGF Oncology myelodysplastic syndromes Phase I/II
(insulin-like-growth-factor St. Paul, MN www.igfoncology.com
methotrexate-conjugate)
IGM-2323 IGM Biosciences non-Hodgkin lymphoma Phase I
(CD20 x CD3 bispecific antibody) Mountain View, CA www.igmbio.com
IGM-8444 IGM Biosciences hematologic malignancies, Phase I
(TRAIL agonist) Mountain View, CA solid tumors www.igmbio.com
IGN002 Spectrum Pharmaceuticals non-Hodgkin lymphoma Phase I
(interferon/CD20 mAb fusion protein) Henderson, NV www.sppirx.com
IK-007 Ikena Oncology NSCLC (+pembrolizumab) Phase I/II
(EP4 antagonist) Boston, MA www.ikenaoncology.com
IK-175 Bristol-Myers Squibb solid tumors, including urothelial Phase I
(AHR antagonist) Princeton, NJ cancer www.ikenaoncology.com
Ikena Oncology
Boston, MA
Ilaris® Novartis 1L NSCLC, 2L NSCLC, adjuvant NSCLC Phase III
canakinumab East Hanover, NJ www.novartis.com
Medicines in Development: Cancer ǀ 2020 92
Product Name Sponsor Indication Development Status
ilixadencel Immunicum renal cell carcinoma Phase II completed
(allogeneic dendritic cell vaccine) Stockholm, Sweden (combination therapy) www.immunicum.se
advanced cancers Phase I/II
(combination therapy) www.immunicum.se
IM156 ImmunoMet Therapeutics solid tumors Phase I
(OXPHOS inhibitor/AMPK activator) Houston, TX www.immunomet.com
IMA201 Immatics US solid tumors Phase I
(T-cell receptor therapy) Houston, TX www.immatics.com
IMA202 Immatics US solid tumors Phase I
(MAGEA1 adoptive T-cell therapy) Houston, TX www.immatics.com
IMA203 Immatics US hematologic malignancies, Phase I
(PRAME adoptive T-cell therapy) Houston, TX solid tumors www.immatics.com
Medicines in Development: Cancer ǀ 2020 93
Product Name Sponsor Indication Development Status
Imbruvica® Janssen Research & Development treatment naïve mantle cell Phase III
ibrutinib Raritan, NJ lymphoma (combination therapy), www.janssen.com
Pharmacyclics indolent non-Hodgkin lymphoma
Sunnyvale, CA (combination therapy), follicular
lymphoma, mantle cell lymphoma
(combination therapy),
1L CML (combination therapy)
solid tumors Phase II
www.janssen.com
IMC-002 ImmuneOncia Therapeutics lymphoma, solid tumors Phase I
(anti-CD47 mAb) Gyeonggi-do, South Korea www.immuneoncia.com
IMC-F106C Immunocore solid tumors Phase I/II
(PRAME) Conshohocken, PA www.immunocore.com
IMCgp100 (tebentafusp) Immunocore uveal melanoma (Fast Track) Phase II
(bispecific fusion protein) Conshohocken, PA www.immunocore.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 94
Product Name Sponsor Indication Development Status
Imfinzi® AstraZeneca muscle invasive bladder cancer, Phase III
durvalumab Wilmington, DE non-muscle invasive bladder cancer, www.astrazeneca.com
ORPHAN DRUG 1L urothelial cancer (combination
therapy), 1L triple negative breast
cancer, locally-advanced cervical
cancer, MDS, 1L biliary tract cancer,
hepatocellular carcinoma,
1L hepatocellular carcinoma
(combination therapy)
locally-advanced NSCLC,
1L NSCLC, locally-advanced NSCLC
(combination therapy), 1L SCLC
1L NSCLC, 1L head and neck cancer
1L bladder cancer, Phase II
1L metastatic microsatellite-stable www.astrazeneca.com
colorectal cancer (combination
therapy), solid tumors (combination
therapy, head and neck cancer
(+MEDI0457) (Fast Track),
biliary tract cancer (combination
therapy), 1L NSCLC (combination
therapy), prostate cancer (+AZD4635),
esophageal cancer, gastric cancer
(combination therapy), extensive-
stage SCLC
Medicines in Development: Cancer ǀ 2020 95
Product Name Sponsor Indication Development Status
Imfinzi® (CONTINUED) AstraZeneca solid tumors (combination therapy) Phase I
durvalumab Wilmington, DE locally-advanced head and neck www.astrazeneca.com
ORPHAN DRUG (combination therapy), SCLC
(combination therapy),
1L pancreatic ductal adenocarcinoma
(combination therapy)
esophageal cancer
(combination therapy)
IMG-7289 Imago BioSciences essential thrombocythemia (Fast Track), Phase II
(LSD1 inhibitor) South San Francisco, CA myelofibrosis (Fast Track), www.imagobio.com
ORPHAN DRUG polycythemia vera
IMGN632 ImmunoGen ALL, AML, blastic plasmacytoid Phase I/II
(CD123 antibody drug conjugate) Waltham, MA dendritic cell neoplasm (BPDCN) www.immunogen.com
(Breakthrough Therapy),
CD123-positive hematologic
malignancies
IMGC936 ImmunoGen solid tumors Phase I
(ADAM9 inhibitor) Waltham, MA www.immunogen.com
MacroGenics
Rockville, MD
Imlygic® Amgen metastatic melanoma Phase III
talimogene laherparepvec Thousand Oaks, CA (+pembrolizumab) www.amgen.com
Imprime PGG HiberCell metastatic breast cancer, Phase II
cancer immunotherapy New York, NY triple negative breast cancer www.hibercell.com
Medicines in Development: Cancer ǀ 2020 96
Product Name Sponsor Indication Development Status
IMSA101 ImmuneSensor Therapeutics solid tumors Phase I/II
(MPYS protein stimulant) Dallas, TX www.immunesensor.com
IMU-201 (PD1-Vaxx) Imugene NSCLC Phase I
(PD-L1 immunotherapy) Sydney, Australia www.imugene.com
Imx-110 Immix Biopharma solid tumors Phase I/II
(curcumin/doxorubicin) Los Angeles, CA www.immixbio.com
IN10018 InxMed metastatic melanoma Phase I
(PTK2/FAK inhibitor) Shanghai, China www.en.inxmed.com
INB03 INmune Bio cancer Phase I
(TNFα inhibitor) La Jolla, CA www.inmunebio.com
INB-100 IN8bio hematologic malignancies Phase I
(allogeneic gamma-delta T-cell New York, NY www.in8bio.com
immunotherapy)
INB-200 IN8bio newly-diagnosed glioblastoma Phase I
(autologous gamma-delta T-cell New York, NY www.in8bio.com
immunotherapy)
INBRX-105 Inhibrx solid tumors Phase I
(PD-L1x41BB bispecific antibody) La Jolla, CA www.inhibrx.com
Medicines in Development: Cancer ǀ 2020 97
Product Name Sponsor Indication Development Status
INBRX-106 Inhibrx solid tumors Phase I
(hexavalent OX40 agonist) La Jolla, CA www.inhibrx.com
INBRX-109 Inhibrx solid tumors Phase I
(tetravalent DR5 agonist) La Jolla, CA www.inhibrx.com
INCAGN1876 Agenis solid tumors Phase I
(GITR inhibitor) Lexington, MA www.incyte.com
Incyte
Wilmington, DE
INCAGN1949 Agenis solid tumors Phase I
(OX40 inhibitor) Lexington, MA www.incyte.com
Incyte
Wilmington, DE
INCAGN2385 Agenis solid tumors Phase I
(LAG-3 inhibitor) Lexington, MA www.incyte.com
Incyte
Wilmington, DE
INCAGN2390 Agenis solid tumors Phase I
(TIM-3 inhibitor) Lexington, MA www.incyte.com
Incyte
Wilmington, DE
Medicines in Development: Cancer ǀ 2020 98
Product Name Sponsor Indication Development Status
INCB00928 Incyte myeloproliferative diseases Phase II
(ALK2 inhibitor) Wilmington, DE www.incyte.com
INCB01158 Incyte solid tumors Phase I
(ARG) Wilmington, DE www.incyte.com
INCB57643 Incyte myelofibrosis Phase I
(BET inhibitor) Wilmington, DE www.incyte.com
INCB81776 Incyte solid tumors Phase I
(AXL/MER) Wilmington, DE www.incyte.com
INCB86550 Incyte solid tumors Phase I
(PD-L1) Wilmington, DE www.incyte.com
INCB99280 Incyte solid tumors Phase I
Wilmington, DE www.incyte.com
infigratinib (BGJ398) QED Therapeutics cholangiocarcinoma (Fast Track) application submitted
(FGF-R inhibitor) San Francisco, CA www.qedtx.com
ORPHAN DRUG
urothelial cancer Phase III
www.qedtx.com
inhaled lipid-complexed cisplatin Eleison Pharmaceuticals osteosarcoma Phase II
Princeton, NJ www.eleison-pharma.com
Medicines in Development: Cancer ǀ 2020 99
Product Name Sponsor Indication Development Status
INO-5151 Inovio Pharmaceuticals prostate cancer Phase II
(DNA vaccine) Plymouth Meeting, PA www.inovio.com
INO-5401 Inovio Pharmaceuticals glioblastoma Phase II
(T-cell activating DNA immunotherapy) Plymouth Meeting, PA www.inovio.com
INT230-6 Intensity Therapeutics solid tumors Phase I/II
(triple fixed-ratio cancer vaccine) Westport, CT www.intensitytherapeutics.com
INV-1120 Ionova Life Sciences solid tumors Phase I
(immunomodulator) Shenzhen, China www.ionovabio.com
IO-202 Immune-Onc Therapeutics AML, CMML Phase I
(LILRB4 inhibitor) Palo Alto, CA www.immune-onc.com
ORPHAN DRUG
iobenguane I-131 (131I-MIBG) Jubilant DraxImage neuroblastoma Phase II
Montreal, Canada www.draximage.com
New Approaches to Neuroblastoma
Therapy Consortium
Los Angeles, CA
Iomab-ACT (CD45) Actinium Pharmaceuticals gene therapy and cell therapy Phase I
(apamistamab -I-131) New York, NY conditioning in HIV-related www.actiniumpharma.com
ORPHAN DRUG lymphoma
Medicines in Development: Cancer ǀ 2020 100
Product Name Sponsor Indication Development Status
IOMb-B (CD45) Actinium Pharmaceuticals BMT conditioning in AML Phase III
New York, NY www.actiniumpharma.com
IOV-2001 Iovance Biotherapeutics CLL/SLL Phase I
(adoptive cell therapy) San Carlos, CA www.iovance.com
IPH2201 (monalizumab) AstraZeneca head and neck cancer Phase I/II
(NKG2A) Wilmington, DE www.astrazeneca.com
Innate Pharma
Rockville, MD
IPH4102 (lacutamab) Innate Pharma peripheral T-cell lymphoma, Phase II
(anti-KIR3DL2 antibody) Rockville, MD cutaneous T-cell lymphoma, www.innate-pharma.com
ORPHAN DRUG mycosis fungoides,
Sezary syndrome (Fast Track)
IPH5201 AstraZeneca solid tumors Phase I
(CD39 antigen inhibitor) Wilmington, DE www.astrazeneca.com
Innate Pharma
Rockville, MD
IPH5401 (avdoralimab) Innate Pharma solid tumors Phase I
(C5a receptor antagonist) Rockville, MD www.innate-pharma.com
Medicines in Development: Cancer ǀ 2020 101
Product Name Sponsor Indication Development Status
IPI-549 (eganelisib) Infinity Pharmaceuticals 2L urothelial cancer (+nivolumab) Phase II
(PIK3γ inhibitor) Cambridge, MA (Fast Track), 1L triple negative www.infi.com
breast cancer (combination therapy)
(Fast Track), 1L renal cell carcinoma,
solid tumors Phase I
www.infi.com
IRX-2 Brooklyn ImmunoTherapeutics squamous cell carcinoma of Phase II
(cytokine immunostimulant) Brooklyn, NY the oral cavity (Fast Track) www.brooklynitx.com
ORPHAN DRUG
IRX4204 Io Therapeutics NSCLC, prostate cancer Phase II
(RXR agonist) Santa Ana, CA www.io-therapeutics.com
HER2-positive breast cancer Phase I
www.io-therapeutics.com
IRX5183 Io Therapeutics AML Phase I
(RARα agonist) Santa Ana, CA www.io-therapeutics.com
ISA101 ISA Pharmaceuticals HPV16-positive squamous cell Phase II
(cancer vaccine) Leiden, Netherlands carcinoma of the oropharynx www.isa-pharma.com
(+cemiplimab)
ISB 1302 Ichnos Sciences HER2-positive breast cancer Phase I
(HER2 x CD3 bispecific antibody) New York, NY www.ichnossciences.com
Medicines in Development: Cancer ǀ 2020 102
Product Name Sponsor Indication Development Status
ISB 1342 Ichnos Sciences multiple myeloma Phase I
(CD38 x CD3 bispecific antibody) New York, NY www.ichnossciences.com
ORPHAN DRUG
Istodax® Bristol=Myers Squibb PTCL Phase III
romidepsin Princeton, NJ www.bmas.com
isunakinra Buzzard Pharmaceuticals solid tumors Phase I/II
(IL-1 antagonist) Stockholm, Sweden www.buzzardpharma.com
ITI-1000 Immunomic Therapeutics newly-diagnosed glioblastoma Phase II
(LMAP-Vax-pp65 dendritic Rockville, MD www.immunomix.com
cell vaccine
JAB-3068 AbbVie solid tumors Phase I
(SHP2 inhibitor) North Chicago, IL www.abbvie.com
Jacobio Pharmaceuticals
Beijing, China
JAB-3312 AbbVie solid tumors Phase I
(SHP2 inhibitor) North Chicago, IL www.abbvie.com
Jacobio Pharmaceuticals
Beijing, China
Jakafi® Incyte myelofibrosis (+parsaclisib) Phase II
ruxolitinib Wilmington, DE www.incyte.com
Medicines in Development: Cancer ǀ 2020 103
Product Name Sponsor Indication Development Status
JBH492 Novartis hematologic malignancies Phase I
(antibody-drug conjugate) East Hanover, NJ www.novartis.com
Jelmyto® UroGen Pharma non-muscle invasive bladder cancer Phase II
mitomycin Princeton, NJ www.urogen.com
JEZ567 Novartis AML Phase I
(CD123 CAR-T cell therapy) East Hanover, NJ www.novartis.com
JNJ-63709178 Janssen Research & Development AML Phase I
(CD123xCD3 DuoBody) Raritan, NJ www.janssen.com
JNJ-63898081 Janssen Research & Development solid tumors Phase I
(PSMAxCD3 bispecific antibody) Raritan, NJ www.janssen.com
JNJ-64007957 (teclistamab) Janssen Research & Development multiple myeloma Phase II
(BCMAxCD3 bispecific antibody) Raritan, NJ www.janssen.com
JNJ-64264681 Janssen Research & Development CLL, NHL Phase I
(BTK inhibitor) Raritan, NJ www.janssen.com
JNJ-64619178 Janssen Research & Development MDS, NHL, solid tumors Phase I
(PRMT5 inhibitor) Raritan, NJ www.janssen.com
Medicines in Development: Cancer ǀ 2020 104
Product Name Sponsor Indication Development Status
JNJ-67571244 Janssen Research & Development AML, MDS Phase I
(CD33xCD3 bispecific antibody) Raritan, NJ www.janssen.com
JNJ-67856633 Janssen Research & Development CLL, NHL Phase I
(MALT1 inhibitor) Raritan, NJ www.janssen.com
JNJ-69086420 Janssen Research & Development prostate cancer Phase I
(actinium-225-labeled antibody) Raritan, NJ www.janssen.com
JNJ-70218902 Janssen Research & Development solid tumors Phase I
(T-cell redirecting therapy) Raritan, NJ www.janssen.com
JNJ-73841937 (lazertinib) Janssen Research & Development NSCLC Phase I
(EGFR antagonist) Raritan, NJ www.janssen.com
JNJ-74494550 (cusatuzumab) Argen AML, MDS Phase II
(CD70 antigen inhibitor) Boston, MA www.janssen.com
Janssen Research & Development
Raritan, NJ
JNJ-74699157 Janssen Research & Development solid tumors Phase I completed
(KRAS G12C inhibitor) Raritan, NJ www.janssen.com
JSI-1187 JS InnoPharm solid tumors Phase I
(ERK1/2 inhibitor) Chicago, IL
Medicines in Development: Cancer ǀ 2020 105
Product Name Sponsor Indication Development Status
JTX-2011 (vopratelimab) Jounce Therapeutics urothelial cancer (combination Phase II
(ICOS stimulator) Cambridge, MA therapy), NSCLC (combination therapy) www.jouncetx.com
advanced solid tumors Phase I/II
www.jouncetx.com
JTX-4014 Jounce Therapeutics NSCLC Phase II
(PD-1 inhibitor) Cambridge, MA www.jouncetx.com
K0706 Sun Pharma Advanced Research chronic myeloid leukemia Phase I/II
(Bcr-Abl1 tyrosine kinase inhibitor) Mumbai, India www.sparc.life
KA2237 Karus Therapeutics B-cell lymphoma Phase I
(PI3K inhibitor) Oxfordshire, United Kingdom www.karustherapeutics.com
KAZ954 Novartis solid tumors Phase I
(antineoplastic) East Hanover, NJ www.novartis.com
KD033 Kadmon solid tumors Phase I
(anti-PD-L1/IL-15 fusion protein) New York, NY www.kadmon.com
Kevetrin® Innovation Pharmaceuticals ovarian cancer Phase II
thioureidobutyronitrile Wakefield, MA www.ipharminc.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 106
Product Name Sponsor Indication Development Status
Keytruda® Merck high-risk early stage triple negative application submitted
pembrolizumab Kenilworth, NJ breast cancer (Breakthrough Therapy), www.merck.com
metastatic triple negative breast
cancer
biliary tract cancer, mesothelioma, Phase III
nasopharyngeal cancer, ovarian www.merck.com
cancer, prostate cancer
advanced solid tumors Phase II
www.merck.com
KHK2455 Kyowa Kirin Pharmaceutical urothelial cancer, solid tumors Phase I
(IDO1 inhibitor) Bedminister, NJ www.kkna.kyowakirin.com
Kisqali® Novartis HR-positive HER2-negative Phase III
ribociclib East Hanover, NJ breast cancer (adjuvant) www.novartis.com
KITE-439 Gilead Sciences solid tumors Phase I
(HPV-16 E7 T-cell receptor) Foster City, CA www.gilead.com
Kite Pharma
Santa Monica, CA
KITE-718 Gilead Sciences solid tumors Phase I
(MAGE-A3/A6 T-cell receptor) Foster City, CA www.gilead.com
Kite Pharma
Santa Monica, CA
Medicines in Development: Cancer ǀ 2020 107
Product Name Sponsor Indication Development Status
KN026 Alphamab Biopharmaceuticals HER2-expressing tumors Phase I
(ERBB 2 receptor antagonist) Jiangsu, China www.alphamab.com
K-NK002 Kiadis Pharma prevention of post-transplantation Phase II
(natural killer cell replacement Amsterdam, The Netherlands relapse in AML and MDS www.kiadis.com
therapy) cancers
KO-539 Kura Oncology AML Phase I/II
(menin-MLL Inhibitor) San Diego, CA www.kuraoncology.com
ORPHAN DRUG
KPG-121 Kangpu Biopharmaceuticals prostate cancer Phase I
(angiogenesis inhibitor) Shanghai, China www.kangpugroup.com
KPG-818 Kangpu Biopharmaceuticals hematologic malignancies Phase I
(CRBN protein modulator) Shanghai, China www.kangpugroup.com
KPT-9274 Karyopharm Therapeutics solid tumors Phase I
(PAK4 and NAMPT inhibitor) Newton, MA www.karyopharm.com
KRT-232 Kartos Therapeutics myelofibrosis, polycythemia vera, Phase II
(MDM2 inhibitor) Redwood City, CA Merkel cell carcinoma www.kartosthera.com
AML, B-cell malignancies Phase I
www.kartosthera.com
Medicines in Development: Cancer ǀ 2020 108
Product Name Sponsor Indication Development Status
KUR-501 Kuur Therapeutics neuroblastoma Phase I
(CAR-T NKT therapy) Houston, TX www.kuurtx.com
Baylor College of Medicine
Houston, TX
KUR-502 Kuur Therapeutics B-cell malignancies Phase I
(CAR-T NKT therapy) Houston, TX www.kuurtx.com
Baylor College of Medicine
Houston, TX
KX2-361 Athenex Oncology glioblastoma Phase I
(Src kinase inhibitor) Buffalo, NY www.athenexoncology.com
ORPHAN DRUG
KY1044 Kymab advanced solid tumors Phase I/II
(ICOS stimulator) Cambridge, United Kingdom (combination therapy) www.kymab.com
Kymriah® Novartis 1L high-risk ALL (pediatric and young Phase III
tisagenlecleucel East Hanover, NJ adults), DLBCL (1st relapse), www.novartis.com
follicular lymphoma
DLBCL (combination therapy), Phase I
lymphoma www.novartis.com
Kyprolis® Amgen relapsed multiple myeloma Phase III
carfilzomib Thousand Oaks, CA (weekly dosing), multiple myeloma www.amgen.com
(combination therapy)
Medicines in Development: Cancer ǀ 2020 109
Product Name Sponsor Indication Development Status
L19TNF (fibromun) Philogen 1L soft tissue sarcoma Phase II
(recombinant fusion protein) Siena, Italy www.philogen.com
ORPHAN DRUG
ladiratuzumab vedotin Merck 1L metastatic triple negative Phase II
(antibody-drug conjugate) Kenilworth, NJ breast cancer (+pembrolizumab), www.merck.com
Seagen solid tumors www.seagen.com
Bothell, WA
HR-positive HER2-negative Phase I
metastatic breast cancer www.merck.com
www.seagen.com
LAE001 Laekna Therapeutics castration-resistant prostate cancer Phase I/II
(androgen synthesis enzyme Shanghai, China www.laeknatp.com
inhibitor)
LAM-002 AI Therapeutics NHL (Fast Track) Phase II
(PIKfyve kinase inhibitor) Guilford, CT www.ai-therapeutics.com
ORPHAN DRUG
LAM-003 AI Therapeutics AML Phase I
(HSP90 inhibitor) Guilford, CT www.ai-therapeutics.com
Lartruvo® Eli Lilly pancreatic cancer Phase II
olaratumab Indianapolis, IN www.lilly.com
Medicines in Development: Cancer ǀ 2020 110
Product Name Sponsor Indication Development Status
lasofoxifene Sermonix Pharmaceuticals ER-positive HER2-negative Phase II
(SERM) Columbus, OH breast cancer with ESR1 mutations www.sermonixpharma.com
LB-100 Lixte Biotechnology MDS Phase I/II
(PP2A inhibitor) East Setauket, NY www.lixte.com
L-DOS47 Helix BioPharma pancreatic cancer Phase I/II
(tumor defence breaker) Richmond Hill, Canada www.helixbiopharma.com
Lenvima® + Keytruda® Eisai 1L hepatocellular carcinoma application submitted
lenvatinib + pembrolizumab Woodcliff Lake, NJ (Breakthrough Therapy) www.eisai.com
ORPHAN DRUG Merck www.merck.com
Kenilworth, NJ
1L cisplatin-ineligible bladder cancer Phase III
1L endometrial cancer, www.eisai.com
2L endometrial cancer www.merck.com
(Breakthrough Therapy),
1L head and neck cancer
1L renal cell carcinoma
(Breakthrough Therapy)
1L non-squamous NSCLC,
1L PD-L1-positive NSCLC,
2L NSCLC, 1L melanoma
2L head and neck cancer, Phase II
2L melanoma, advanced solid tumors www.eisai.com
www.merck.com
Medicines in Development: Cancer ǀ 2020 111
Product Name Sponsor Indication Development Status
lenzilumab Humanigen chronic myelomonocytic leukemia Phase III
Burlingame, CA www.humanigen.com
lerociclib EQRx NSCLC Phase I/II
(CDK4/6 inhibitor) Cambridge, MA www.eqrx.com
G1 Therapeutics www.g1therapeutics.com
Research Triangle Park, NC
leronlimab CytoDyn solid tumors Phase I/II
(CCR5 antagonist) Vancouver, WA www.cytodyn.com
triple negative breast cancer Phase I/II
(Fast Track) www.cytodyn.com
LHC165 Novartis solid tumors Phase I
(TLR7 agonist) East Hanover, NJ (combination therapy) www.novartis.com
Medicines in Development: Cancer ǀ 2020 112
Product Name Sponsor Indication Development Status
Libtayo® Regeneron Pharmaceuticals 1L NSCLC application submitted
cemiplimab-rwlc Tarrytown, NY www.sanofi.com
Sanofi
Bridgewater, NJ
cutaneous squamous cell Phase III
carcinoma (adjuvant), 1L NSCLC www.sanofi.com
(combination therapy),
2L cervical cancer (Fast Track)
2L basal cell carcinoma, cutaneous Phase II
squamous cell carcinoma www.sanofi.com
solid tumors Phase I
www.sanofi.com
lifileucel (LN-144) Iovance Biotherapeutics melanoma (Fast Track) Phase III
(TIL autologous cell therapy) San Carlos, CA www.iovance.com
ORPHAN DRUG
melanoma (combination therapy) Phase II
www.iovance.com
lifirafenib (BGB-283) BeiGene B-Raf or K-RAS/N-RAS mutated Phase II
(RAF dimer) Cambridge, MA solid tumors www.beigene.com
Medicines in Development: Cancer ǀ 2020 113
Product Name Sponsor Indication Development Status
LipoCurc™ Signpath Pharma solid tumors Phase I
curcumin liposomal Sandy, UT www.signpathpharma.com
liso-cel® Bristol-Myers Squibb relapsed/refractory B-cell lymphoma application submitted
lisocabtagene maraleucel Princeton, NJ www.bms.com
(CD19 CAR-T cell therapy)
ORPHAN DRUGaggressive large B-cell lymphoma Phase III
(transplant eligible) www.bms.com
3L CLL, 2L pediatric B-cell ALL, Phase II
2L DLBCL (transport ineligible), www.bms.com
3L DLBCL, 3L+ follicular lymphoma,
3L+ marginal zone lymphoma,
2L+ primary CNS lymphoma,
1L high-grade B-cell lymphoma
3L+ mantle cell lymphoma Phase I
www.bms.com
LMP400 Gibson Oncology colorectal cancer, Phase I
(DNA topoisomerase I inhibitor) Miami, FL Ewing sarcoma (pediatric), www.gibsononcology.com
HRD-positive tumors
LMP744 Gibson Oncology solid tumors Phase I
(DNA-topoisomerase I inhibitor) Miami, FL www.gibsononcology.com
Medicines in Development: Cancer ǀ 2020 114
Product Name Sponsor Indication Development Status
LN-145 Iovance Biotherapeutics cervical cancer (Fast Track) Phase III
(TIL autologous cell therapy) San Carlos, CA (Breakthrough Therapy) www.iovance.com
ORPHAN DRUG
head and neck cancer, NSCLC, Phase II
melanoma www.iovance.com
LNS8801 Linnaeus Therapeutics lymphoma, solid tumors Phase I
(GPER agonist) Haddonfield, NJ www.linnaeustx.com
loncastuximab tesirine (ADCT-402) ADC Therapeutics DLBCL application submitted
(anti-CD19 antibody-drug conjugate) Lausanne, Switzerland www.adctherapeutics.com
ORPHAN DRUG
DLBCL, mantle cell lymphoma Phase II
(combination therapy) www.adctherapeutics.com
follicular lymphoma Phase I
www.adctherapeutics.com
Lorbrena® Pfizer 1L ALK-positive NSCLC Phase III
lorlatinib New York, NY www.pfizer.com
ORPHAN DRUG
LOXO-305 LOXO Oncology CL/SLL, NHL Phase I/II
(BTK inhibitor) Indianapolis, IN www.loxooncology.com
Medicines in Development: Cancer ǀ 2020 115
Product Name Sponsor Indication Development Status
LP-100 Lantern Pharma prostate cancer Phase II
Dallas, TX www.lanternpharma.com
LP-108 Newave Pharmaceuticals AML, chronic myelomonocytic Phase I
(Bcl-2 inhibitor) Pleasanton, CA leukemia, MDS
LP-300 Lantern Pharma NSCLC Phase I
(glutaredoxin/thioredoxin modulator) Dallas, TX www.lanternpharma.com
lucitanib Clovis Oncology gynecologic cancer (+nivolumab) Phase II
(angiogenesis inhibitor) Boulder, CO www.clovisoncology.com
ovarian cancer (+rucaparib) Phase I
www.clovisoncology.com
Lumoxiti® AstraZeneca 3L hairy cell leukemia Phase III
moxetumumab pasudotox-tdfk Wilmington, DE www.astrazeneca.com
ORPHAN DRUG
Lutathera® Advanced Accelerator Applications 1L grade 3 gastroentero pancreatic Phase III
177-Lu-oxodotreotide Millburn, NJ neuroendocrine tumors (GEP-NET) www.adacap.com
LVGN6051 Lyvgen Biopharma cancer Phase I
(CD137/4-1BB agonist mAb) Shanghai, China www.lyvgen.com
Medicines in Development: Cancer ǀ 2020 116
Product Name Sponsor Indication Development Status
LXF821 Novartis glioblastoma Phase I
(EGFR CAR-T cell therapy) East Hanover, NJ www.novartis.com
LXH254 Novartis solid tumors Phase I
(cRAF inhibitor) East Hanover, NJ www.novartis.com
LY2880070 Esperas Pharma solid tumors Phase I/II
Montreal, Canada
LY3214996 Eli Lilly cancer Phase I
(ERK inhibitor) Indianapolis, IN www.lilly.com
LY3295668 Eli Lilly neuroblastoma, SCLC Phase I
(aurora A kinase inhibitor) Indianapolis, IN www.lilly.com
LY3405105 Eli Lilly solid tumors Phase I
(CDK7 inhibitor) Indianapolis, IN www.lilly.com
LY3410738 Eli Lilly solid tumors Phase I
(IDH1 inhibitor) Indianapolis, IN www.lilly.com
LY3475070 Eli Lilly cancer Phase I
(CD73 inhibitor) Indianapolis, IN www.lilly.com
Medicines in Development: Cancer ǀ 2020 117
Product Name Sponsor Indication Development Status
LY3484356 Eli Lilly metastatic breast cancer, Phase I
(SERD) Indianapolis, IN endometrial cancer www.lilly.com
LY3527727 Eli Lilly cancer Phase I
(BTK inhibitor) Indianapolis, IN www.lilly.com
LYC-55716 Lycera NSCLC (combination therapy) Phase I
(RORγ agonist) Ann Arbor, MI www.lycera.com
Lynparza® AstraZeneca gBRCA adjuvant breast cancer, Phase III
olaparib Wilmington, DE gBRCA metastatic breast cancer, www.astrazeenca.com
Merck 1L platinum-sensitive colorectal www.merck.com
Kenilworth, NJ cancer, 1L ovarian cancer (combination
therapy), gBRCA PSR ovarian cancer,
1L endometrial cancer, homologous
recombination repair mutation-
positive cancer, pancreatic cancer,
prostate cancer, prostate cancer
(combination therapy),
colorectal cancer (+pembrolizumab),
NSCLC (+pembrolizumab)
breast cancer (+ceralastertib), Phase II
solid tumors (+pembrolizumab) www.astrazeneca.com
www.merck.com
Medicines in Development: Cancer ǀ 2020 118
Product Name Sponsor Indication Development Status
M1774 EMD Serono solid tumors Phase I
(ATR inhibitor) Rockland, MA www.emdserono.com
M3258 EMD Serono multiple myeloma Phase I
(LMP7 inhibitor) Rockland, MA www.emdserono.com
M4344 EMD Serono solid tumors Phase I
(ATR inhibitor) Rockland, MA www.emdserono.com
M6223 EMD Serono solid tumors Phase I
(TIGIT inhibitor) Rockland, MA www.emdserono.com
M8891 EMD Serono solid tumors Phase I
(MetAP2 inhibitor) Rockland, MA www.emdserono.com
magrolimab (GS-4721) Gilead Sciences MDS (Breakthrough Therapy) Phase III
(anti-CD47 mAb) Foster City, CA (Fast Track) www.gilead.com
ORPHAN DRUG
AML (Fast Track), NHL (Fast Track) Phase I
www.gilead.com
MAK683 Novartis cancer Phase I
(EED inhibitor) East Hanover, NJ www.novartis.com
Medicines in Development: Cancer ǀ 2020 119
Product Name Sponsor Indication Development Status
margetuximab MacroGenics HER2-positive breast cancer application submitted
(anti-HER2 mAb) Rockville, MD (Fast Track) www.macrogenics.com
ORPHAN DRUG
HER2-positive gastric/ Phase II
gastroesophageal junction cancer www.macrogenics.com
marizomib Bristol-Myers Squibb newly-diagnosed glioblastoma Phase III
(proteasome inhibitor) Princeton, NJ www.bms.com
ORPHAN DRUG
mavorixafor X4 Pharmaceuticals renal cell carcinoma (+axitinib) Phase II
(CXCR4 inhibitor) Cambridge, MA www.x4pharma.com
Waldenstrom's macroglobulinemia Phase I
www.x4pharma.com
MB-101 Mustang Bio glioblastoma, glioma Phase I
(IL-BR3α2 CAR-T cell therapy) Worcester, MA www.mustangbio.com
City of Hope Medical Center
Duarte, CA
MB-102 Mustang Bio AML, blastic plasmacytoid dendritic Phase I/II
(anti-CD123 CAR-T cell therapy) Worcester, MA cell neoplasm, high-risk MDS www.mustangbio.com
ORPHAN DRUG City of Hope Medical Center
Duarte, CA
Medicines in Development: Cancer ǀ 2020 120
Product Name Sponsor Indication Development Status
MB-103 Mustang Bio brain or leptomeningeal Phase I
(HER2 CAR-T cell therapy) Worcester, MA metastases www.mustangbio.com
City of Hope Medical Center
Duarte, CA
MB-104 Mustang Bio multiple myeloma Phase I
(CS1 CAR-T cell therapy) Worcester, MA www.mustangbio.com
City of Hope Medical Center
Duarte, CA
MB-105 Mustang Bio metastatic castration-resistant Phase I
(PSCA CAR-T cell therapy) Worcester, MA prostate cancer www.mustangbio.com
City of Hope Medical Center
Duarte, CA
MB-106 Mustang Bio B-cell NHL Phase I/II
(CD20 CAR-T cell therapy) Worcester, MA www.mustangbio.com
Fred Hutchinson Cancer Center
Seattle, WA
MB-108 (C134) Mustang Bio glioblastoma Phase I
(genetically modified oncolytic virus) New York, NY www.mustangbio.com
ORPHAN DRUG Nationwide Children's Hospital
Columbus, OH
Medicines in Development: Cancer ǀ 2020 121
Product Name Sponsor Indication Development Status
MBG453 (sabatolimab) Novartis high-risk MDS Phase III
(TIM3 antagonist) East Hanover, NJ www.novartis.com
unfit AML Phase II
www.novartis.com
MCC1 TCR bluebird bio Merkel cell carcinoma Phase I
(autologous TCR T-cell therapy) Cambridge, MA www.bluebirdbio.com
MCLA-117 Merus AML Phase I
(CLEC12AxCD3 bispecific antibody) Cambridge, MA www.merus.nl
MCLA-128 (zenocutuzumab) Merus metastatic breast cancer Phase II
(HER2xHER3 bispecific antibody) Cambridge, MA www.merus.nl
ORPHAN DRUG
solid tumors harboring an NRG1 fusion Phase I
www.merus.nl
MCLA-145 Incyte lymphoma, solid tumors Phase I
(PD-L1xCD137 bispecific antibody) Wilmington, DE www.incyte.com
Merus
Cambridge, MA
MCLA-158 Merus solid tumors Phase I
(Lgr5xEGFR bispecific antibody) Cambridge, MA www.merus.nl
Medicines in Development: Cancer ǀ 2020 122
Product Name Sponsor Indication Development Status
MCM998 Novartis multiple myeloma (+LXG250) Phase I
(BCMA CAR-T cell therapy) East Hanover, NJ www.novartis.com
MCY-M11 MaxCyte mesothelioma, fallopian tube cancer, Phase I
(mRNA gene therapy) Gaithersburg, MD ovarian cancer, peritoneal cancer www.maxcyte.com
MDNA55 Medicenna Therapeutics recurrent glioblastoma (Fast Track), Phase II
(protein synthesis inhibitor) Houston, TX brain metastases, newly-diagnosed www.medicenna.com
ORPHAN DRUG glioblastoma
ME-401 (zandelisib) MEI Pharma follicular lymphoma (Fast Track) Phase II
(PI3Kδ inhibitor) San Diego, CA www.meipharma.com
Kyowa Kirin
Bedminster, NJ
MEI Pharma B-cell malignancies Phase I
San Diego, CA www.meipharma.com
BeiGene
Cambridge, MA
MEDI0457 AstraZeneca HPV-associated head and neck Phase I/II
(therapeutic vaccine) Wilmington, DE cancer (+durvalumab) www.astrazeneca.com
MEDI1191 AstraZeneca solid tumors Phase I
(mRNA therapeutic) Wilmington, DE www.astrazeneca.com
Moderna Therapeutics
Cambridge, MA
Medicines in Development: Cancer ǀ 2020 123
Product Name Sponsor Indication Development Status
MEDI2228 AstraZeneca multiple myeloma Phase I
(immunoconjugate) Wilmington, DE www.astrazeneca.com
MEDI5395 AstraZeneca solid tumors Phase I
(oncolytic virus therapy) Wilmington, DE www.astrazeneca.com
MEDI5752 AstraZeneca solid tumors Phase II
(PD-1xCTLA-4 bispecific antibody) Wilmington, DE www.astrazeneca.com
renal cell carcinoma Phase I
www.astrazeneca.com
Melblez Kit Delcath Systems hepatic-dominant ocular melanoma Phase III
melphalan drug delivery system New York, NY (Fast Track) www.delcath.com
ORPHAN DRUG
intrahepatic cholangiocarcinoma Phase II/III
www.delcath.com
melflufen Oncopeptides multiple myeloma application submitted
(INN melphalan flufenamide) Waltham, MA (+ dexamethasone) www.oncopeptides.se
ORPHAN DRUG
MEN1611 Menarini colorectal cancer Phase I/II
(PI3K inhibitor) Florence, Italy www.menarini.com
Medicines in Development: Cancer ǀ 2020 124
Product Name Sponsor Indication Development Status
MGC018 MacroGenics solid tumors Phase I
(B7-H3 antibody-drug conjugate) Rockville, MD www.macrogenics.com
MGD013 (tebotelimab) MacroGenics hematologic malignancies, Phase II
(PD-1xLAG3 bispecific antibody) Rockville, MD solid tumors www.macrogenics.com
MGD019 MacroGenics solid tumors Phase I
(PD-1xCTLA-4 bispecific antibody) Rockville, MD www.macrogenics.com
MGTA-145 Magenta Therapeutics hematologic malignancies Phase I
(stem cell therapy) Cambridge, MA www.magentatx.com
MGTA-456 (spanlecortemlocel) Magenta Therapeutics hematologic malignancies Phase II
(stem cell therapy) Cambridge, MA www.magentatx.com
mifepristone Corcept Therapeutics GR-positive triple negative breast Phase II
(glucocorticoid/progesterone Menlo Park, CA cancer (+nab-paclitaxel), www.corcept.com
receptor antagonist) HER2-negative breast cancer
(+pembrolizumab), castration-
resistant prostate cancer
(+enzalutamide)
MIK665 (S64315) Novartis AML (combination therapy) Phase I
(MCL1 inhibitor) East Hanover, NJ www.novartis.com
Servier
Suresnes, France
Medicines in Development: Cancer ǀ 2020 125
Product Name Sponsor Indication Development Status
minnelide Minneamrita Therapeutics advanced solid tumors Phase I
(HSP70 inhibitor) Tampa, FL
mirdametinib + lifirafenib BeiGene RAS/RAF mutant solid tumors Phase I/II
(MEK inhibitor/RAF dimer inhibitor) Cambridge, MA www.beigene.com
SpringWorks Therapeutics www.springworkstx.com
Stamford, CT
mirvetuximab soravtansine ImmunoGen ovarian cancer (monotherapy) Phase III
(antibody-drug conjugate) Waltham, MA www.immunogen.com
ORPHAN DRUG
ovarian cancer (combination therapy) Phase II
www.immunogen.com
MK-0482 Merck solid tumors Phase I
Kenilworth, NJ www.merck.com
MK-1026 (ARQ-531) Merck hematologic malignancies Phase II
(BTK inhibitor) Kenilworth, NJ www.merck.com
MK-1308 Merck melanoma (+pembrolizumab), Phase II
(CTLA4 inhibitor) Kenilworth, NJ NSCLC (+pembrolizumab) www.merck.com
MK-1454 Merck head and neck cancer Phase II
(MPYS protein stimulant) Kenilworth, NJ (+pembrolizumab) www.merck.com
Medicines in Development: Cancer ǀ 2020 126
Product Name Sponsor Indication Development Status
MK-2118 Merck lymphoma, solid tumors Phase I
Kenilworth, NJ www.merck.com
MK-4280 Merck hematologic malignancies Phase II
(CD223 antigen inhibitor) Kenilworth, NJ (+pembrolizumab), www.merck.com
NSCLC (+pembrolizumab)
MK-4830 Merck solid tumors Phase I
(ILT4 inhibitor) Kenilworth, NJ www.merck.com
MK-5890 Merck NSCLC (+pembrolizumab) Phase II
(anti-CD27 antibody) Kenilworth, NJ www.merck.com
MK-6482 Merck renal cell carcinoma Phase III
(HIF-2α inhibitor) Kenilworth, NJ (Breakthrough Therapy) www.merck.com
ORPHAN DRUG
MK-7162 Merck solid tumors (+pembrolizumab) Phase I
Kenilworth, NJ www.merck.com
MK-7684 (vibostolimab) Merck melanoma (+pembrolizumab), Phase II
(anti-TIGIT antibody) Kenilworth, NJ NSCLC (+pembrolizumab) www.merck.com
MNTX Bausch Health pancreatic cancer Phase II/III
(methylnaltrexone bromide) Bridgewater, NJ www.bauschhealth.com
Medicines in Development: Cancer ǀ 2020 127
Product Name Sponsor Indication Development Status
mobocertinib (TAK-788) Takeda 1L/2L NSCLC with EGFR exon 20 Phase III
(ERBB 2 receptor antagonist) Deerfield, IL insertion mutation (Breakthrough www.takeda.com
ORPHAN DRUG Therapy), HER2-mutant NSCLC
ModraDoc006/r Modra Pharmaceuticals metastatic prostate cancer Phase II
(docetaxel plus ritonavir) Amsterdam, Netherlands www.modrapharmaceuticals.com
momelotinib Sierra Oncology myelofibrosis (Fast Track) Phase III
(JAK1/JAK2/ACVR1 inhibitor) Vancouver, Canada www.sierraoncology.com
ORPHAN DRUG
Monjuvi® Incyte CLL/SLL Phase II
tafasitamab-cxix Wilmington, DE www.incyte.com
ORPHAN DRUG MorphoSys www.morphosys.com
Boston, MA
MORAb-202 Eisai solid tumors Phase I/II
(antibody-drug conjugate) Woodcliff Lake, NJ www.eisai.com
motixafortide (BL-8040) BioLineRx advanced pancreatic cancer Phase II
(CXCR4 inhibitor) Modi’in, Israel (+pembrolizumab) www.biolinerx.com
ORPHAN DRUG
motolimod Bristol-Myers Squibb head and neck squamous cell Phase I
(TLR8 agonist) Princeton, NJ carcinoma www.bms.com
Medicines in Development: Cancer ǀ 2020 128
Product Name Sponsor Indication Development Status
MRG002 Miracogen solid tumors Phase I/II
(antibody-drug conjugate) Shanghai, China www.miracogen.com
mRNA-2416 Moderna Therapeutics ovarian cancer Phase II
(mRNA-OX40L) Cambridge, MA www.modernatx.com
lymphoma, solid tumors Phase I
www.modernatx.com
mRNA-2752 Moderna Therapeutics lymphoma, solid tumors Phase I
(mRNA-OX40L/IL-23/IL-36ƴ) Cambridge, MA www.modernatx.com
mRNA-4157 Moderna Therapeutics melanoma (+ pembrolizumab) Phase II
(personalized cancer vaccine) Cambridge, MA www.modernatx.com
Merck
Kenilworth, NJ
solid tumors Phase I
www.modernatx.com
mRNA-5671 Moderna Therapeutics colorectal cancer, NSCLC, Phase I
(KRAS vaccine) Cambridge, MA pancreatic cancer (monotherapy www.modernatx.com
Merck and with pembrolizumab)
Kenilworth, NJ
MRTX849 Mirati Therapeutics advanced cancers with KRAS G12C Phase I/II
(KRAS G12C inhibitor) San Diego, CA mutation www.mirati.com
Medicines in Development: Cancer ǀ 2020 129
Product Name Sponsor Indication Development Status
MRx0518 4D Pharma pancreatic cancer, Phase I
(live biotherapeutic) Leeds, United Kingdom solid tumors (combination therapy) www.4dpharmaplc.com
MRX-2843 Meryx advanced solid tumors Phase I
(FLT-3/MerTK inhibitor) Chapel Hill, NC www.meryx.com
MS-553 MingSight Pharmaceuticals CLL/SLL Phase I
(PKCß inhibitor) San Diego, CA
MSB2311 Transcenta advanced solid tumors Phase I
(anti-PD-L1 mAb) Suzhou, China www.transcenta.com
MSC-1 AstraZeneca solid tumors Phase I
(LIF inhibitor) Wilmington, Delaware www.astrazeneca.com
MSI-1436 DepYmed HER2-positive breast cancer Phase I
(PTP-1B inhibitor) Farmingdale, NY www.depymedinc.com
MT-3724 Molecular Templates DLBCL, NHL Phase II
(anti-CD20 engineered toxinbody) Austin, TX www.mtem.com
MT-5111 Molecular Templates HER2-positive solid tumors Phase I
(anti-HER2 engineered toxinbody) Austin, TX www.mtem.com
Medicines in Development: Cancer ǀ 2020 130
Product Name Sponsor Indication Development Status
MultiTAA (MT-401) Marker Therapeutics AML Phase II
(TAA-specific T cell therapy) Houston, TX www.markertherapeutics.com
ORPHAN DRUG
ALL, lymphoma, multiple myeloma, Phase I/II
pancreatic cancer, sarcoma www.markertherapeutics.com
Multikine CEL-SCI head and neck cancer Phase III
leukocyte interleukin Vienna, VA www.cel-sci.com
ORPHAN DRUG
MV-NIS Vyriad bladder cancer Phase I
(intravesical therapy with an Rochester, MN www.vyriad.com
attenuated measles virus)
MVA-BM Brachyury Bavarian-Nordic solid tumors Phase I
vaccine (intravenous) Morrisville, NC www.bavarian-nordic.com
MVI-118 Madison Vaccines prostate cancer (+pembrolizumab) Phase II
(DNA cancer vaccine) Madison, WI www.madisonvaccines.com
MVI-816 Madison Vaccines prostate cancer (+pembrolizumab) Phase II
(DNA plasmid vaccine) Madison, WI www.madisonvaccines.com
MYL-14020 Mylan cervical cancer, colorectal cancer, application submitted
(bevacizumab biosimilar) Pittsburgh, PA NSCLC, glioblastoma, renal cell www.mylan.com
carcinoma
Medicines in Development: Cancer ǀ 2020 131
Product Name Sponsor Indication Development Status
nab -sirolimus (ABI-009) AADi Bioscience perivascular epithelioid cell application submitted
(nanoparticle albumin-bound Palisades, CA carcinoma (Breakthrough Therapy) www.aadibio.com
mTOR inhibitor)
ORPHAN DRUGneuroendocrine tumors Phase II
newly-diagnosed glioblastoma, www.aadibio.com
recurrent high-grade glioma
(combination therapy), pan-tumor
(TSC1/2 mutation)
1L colorectal cancer Phase I/II
(combination therapy) www.aadibio.com
soft tissue sarcoma (combination
therapy), nonadipocytic soft tissue
sarcoma (combination therapy)
solid tumors (pediatric) Phase I
(combination therapy) www.aadibio.com
nadofaragene firadenovec FerGene high-grade BCG unresponsive application submitted
(IFNA2B gene therapy) Cambridge, MA non-muscle invasive bladder cancer www.fergene.com
Trizell (Fast Track) (Breakthrough Therapy)
London, United Kingdom
malignant pleural mesothelioma Phase III
www.fergene.com
Medicines in Development: Cancer ǀ 2020 132
Product Name Sponsor Indication Development Status
nalotimagene carmaleucel (HSV-Tk) AGC Biologics acute leukemia Phase III
(ex vivo gene therapy) Bothell, WA www.agcbio.com
ORPHAN DRUG
namodenoson (CF102) Can-Fite BioPharma hepatocellular carcinoma Phase II
(A3AR agonist) Waltham, MA (Fast Track) www.canfite.com
ORPHAN DRUG
nanatinostat (VRx-396) Viracta Therapeutics EBV-associated lymphoma Phase I/II
(HDAC inhibitor) Cardiff, CA (combination therapy) (Fast Track) www.viracta.com
ORPHAN DRUG
NanoDoce® NanOlogy high-risk non-muscle invasive Phase I
submicron particle docetaxel Fort Worth, TX bladder cancer, muscle invasive www.nanology.us
bladder cancer
NanoPac® NanOlogy ovarian cancer, locally advanced Phase II
submicron particle paclitaxel Fort Worth, TX pancreatic cancer, mucinous cystic www.nanology.us
ORPHAN DRUG pancreatic neoplasms, prostate
cancer
napabucasin Sumitomo Dainippon colorectal cancer Phase III
(oncogenic pathways inhibitor) Pharma Oncology (combination therapy) www.sdponcology.com
Cambridge, MA
advanced solid tumors Phase I/II
(monotherapy and combinations) www.sdponcology.com
Medicines in Development: Cancer ǀ 2020 133
Product Name Sponsor Indication Development Status
naratuximab emtansine (DEBIO 1562) Debiopharm DLBCL, NHL Phase II
(CD37 protein inhibitor) Lausanne, Switzerland www.debiopharm.com
ORPHAN DRUG
navicixizumab (OMP-305B83) OncXerna Therapeutics ovarian cancer (Fast Track) Phase I
(DLL4/VEGF-A inhibitor) Waltham, MA www.oncxerna.com
navitoclax AbbVie myelofibrosis Phase III
(Bcl-XL/Bcl-2 protein inhibitor) North Chicago, IL www.abbvie.com
NBE-002 NBE-Therapeutics advanced solid tumors Phase I/II
(immune-stimulatory antibody-drug Basel, Switzerland www.nbe-therapeutics.com
conjugate)
NBF-006 Nitto Biopharma colorectal cancer, NSCLC, Phase I
(siRNA-based lipid nanoparticle) San Diego, CA pancreatic cancer www.nittobiopharma.com
NBM-BMX softgel capsules NatureWise Biotech & Medicals solid tumors Phase I
(HDAC8 protein inhibitor) Taipei, Taiwan www.naturewise.com.tw
NBTXR3 nanoparticles Nanobiotix prostate cancer (combination Phase I/II
Cambridge, MA therapy) www.nanobiotix.com
advanced solid tumors Phase I
www.nanobiotix.com
Medicines in Development: Cancer ǀ 2020 134
Product Name Sponsor Indication Development Status
NC318 NextCure solid tumors Phase I/II
(T lymphocyte stimulant) Beltsville, MD www.nextcure.com
NC410 NextCure solid tumors Phase I/II
(T lymphocyte stimulant) Beltsville, MD www.nextcure.com
NC-6004 NanoCarrier solid tumors, including NSCLC, Phase I/II
(cisplatin micelle) Chiba, japan head and neck cancer, biliary cancer, www.nanocarrier.co.jp
ORPHAN DRUG bladder cancer
NC-6300 NanoCarrier soft tissue sarcoma, solid tumors Phase I/II
(epirubicin micelle formulation) Chiba, Japan www.nanocarrier.co.jp
ORPHAN DRUG
nelipepimut-S Sellas Life Sciences HER2-positive breast cancer Phase II completed
(E75 cancer vaccine) New York, NY (combination therapy) (Fast Track) www.sellaslifesciences.com
NEO-100 NeOnc Technologies glioblastoma Phase I/II
perillyl alcohol intranasal Los Angeles, CA www.neonctech.com
ORPHAN DRUG
NEO-102 (ensituximab) Precision Biologics metastatic colorectal cancer, Phase II completed
(chimeric mAb) Bethesda, MD metastatic pancreatic cancer www.precision-biologics.com
ORPHAN DRUG
NEO-201 Precision Biologics solid tumors Phase I
(IgG1 mAb) Bethesda, MD www.precision-biologics.com
Medicines in Development: Cancer ǀ 2020 135
Product Name Sponsor Indication Development Status
NEO-PV-01 BioNTech US NSCLC (combination therapy) Phase I
(personalized cancer vaccine) Cambridge, MA www.biontech.de
NeoTCR-P1 PACT Pharma solid tumors Phase I
(adoptive cell therapy) South San Francisco, CA www.pactpharma.com
Nerlynx® Puma Biotechnology HER2-positive metastatic breast Phase II
neratinib Los Angeles, CA cancer (monotherapy and www.pumabiotechnology.com
combination therapy),
HER2-mutant breast cancer
nerofe Immune System Key AML, MDS Phase I
(tumor cells apoptosis factor) Jerusalem, Israel www.immunesk.com
ORPHAN DRUG
NEXI-001 NexImmune AML, MDS Phase I/II
(nanoparticle adoptive cellular therapy) Gaithersburg, MD www.neximmune.com
NEXI-002 NexImmune multiple myeloma Phase I/II
(nanoparticle adoptive cellular therapy) Gaithersburg, MD www.neximune.com
NG-350A PsiOxus Therapeutics solid tumors Phase I
(gene therapy) Oxfordshire, United Kingdom www.psioxus.com
NG-641 PsiOxus Therapeutics solid tumors Phase I
(gene therapy) Oxfordshire, United Kingdom www.psioxus.com
Medicines in Development: Cancer ǀ 2020 136
Product Name Sponsor Indication Development Status
NGM120 NGM Biopharmaceuticals advanced solid tumors Phase I
(GFRAL antagonistic antibody) South San Francisco, CA www.ngmbio.com
NGR-TNF AGC Biologics mesothelioma Phase III completed
(recombinant fusion protein) Bothell, WA www.agcbio.com
ORPHAN DRUG
Ninlaro® Takeda newly-diagnosed multiple myeloma Phase III
iIxazomib Deerfield, IL (maintenance therapy) www.takeda.com
r/r multiple myeloma (triplet Tx), Phase I/II
r/r multiple myeloma (double Tx) www.takeda.com
nirogacestat SpringWorks Therapeutics desmoid tumors (adult) Phase III
(gamma secretase inhibitor) Stamford, CT (Breakthrough Therapy) (Fast Track) www.springworkstx.com
ORPHAN DRUG
desmoid tumors (pediatric) Phase II
www.springworkstx.com
multiple myeloma Phase I
(combination therapy) www.springworkstx.com
NIR178 Novartis cancer (+PD-1) Phase II
(Ad2AR inhibitor) East Hanover, NJ www.novartis.com
Medicines in Development: Cancer ǀ 2020 137
Product Name Sponsor Indication Development Status
NIS793 Novartis solid tumors (+PD-1) Phase I
(TGFBI inhibitor) East Hanover, NJ www.novartis.com
nivatrotamab Y-mAbs Therapeutics GD2-positive solid tumors Phase I
(GD2xCD3 bispecific antibody) New York, NY www.ymabs.com
ORPHAN DRUG
NIZ985 Novartis solid tumors Phase I
(IL-15 agonist) East Hanover, NJ www.novartis.com
NJH395 Novartis solid tumors Phase I
(immune stimulator East Hanover, NJ www.novartis.com
antibody conjugate)
NKTR-255 Nektar Therapeutics colorectal cancer (combination Phase II
(IL-15 agonist) San Francisco, CA therapy), head and neck cancer www.nektar.com
(combination therapy), multiple
myeloma and NHL (monotherapy
and combination therapy)
NKTR-262 Nektar Therapeutics advanced solid tumors Phase I
(PEG-conjugated TLR7/8 agonist) San Francisco, CA www.nektar.com
NKX101 NKarta AML, MDS Phase I
(CAR-T NK cell therapy) South San Francisco, CA www.nkartatx.com
Medicines in Development: Cancer ǀ 2020 138
Product Name Sponsor Indication Development Status
NM21-1480 Numab Therapeutics advanced solid tumors Phase I
(tri-specific antibody) Wädenswil, Switzerland www.numab.com
NMS-03305293 Nerviano Medical Sciences advanced solid tumors Phase I
(PARP inhibitor) Nerviano, Italy www.nervianoms.com
NOUS-209 Nouscom solid tumors Phase I
(neoantigen genetic vaccine) Basel, Switzerland www.nouscom.com
NP-G2-044 Novita Pharmaceuticals solid tumors Phase I
(cell movement inhibitor) New York, NY
NT-I7 NeoImmuneTech glioblastoma (multiple doses), Phase I/II
(efineptakin alfa) Rockville, MD solid tumors (combination therapy), www.neoimmunetech.com
high-risk skin cancer (combination
therapy), triple negative breast
cancer (combination therapy)
glioblastoma (single dose) Phase I
www.neoimmunetech.com
NT219 TyrNovo (Kitov) metastatic solid tumors Phase I/II
(STAT3 and IRS1/2 inhibitor) Tel Aviv, Israel www.kitovpharma.com
Medicines in Development: Cancer ǀ 2020 139
Product Name Sponsor Indication Development Status
Nubeqa® Bayer Pharmaceuticals metastatic hormone-sensitive Phase III
darolutamide Whippany, NJ prostate cancer, prostate cancer www.pharma.bayer.com
adjuvant
NUC-1031 NuCana biliary tract cancer Phase III
(fosgemcitabine palabenamide) Newton, MA www.nucana.com
ORPHAN DRUG
NUC-3373 NuCana colorectal cancer Phase I
(fosifloxuridine nafalbenamide) Newton, MA www.nucana.com
NZV930 Novartis solid tumors Phase I
(CD73 antagonist) East Hanover, NJ (combination therapy) www.novartis.com
Surface Oncology
Cambridge, MA
OBI-3424 OBI Pharma hepatocellular carcinoma, Phase I/II
(cytotoxic prodrug) San Diego, CA castration-resistant prostate cancer www.obipharma.com
ORPHAN DRUG
OBI-888 OBI Pharma solid tumors Phase I/II
(anti-Globo H mAb) San Diego, CA www.obipharma.com
OBI-999 OBI Pharma solid tumors Phase I/II
(antibody-drug conjugate) San Diego, CA www.obipharma.com
Medicines in Development: Cancer ǀ 2020 140
Product Name Sponsor Indication Development Status
OBP-301 (telomelysin) Oncolys BioPharma esophagogastric adenocarcinoma Phase II
(oncolytic type 5 adenovirus) Edison, NJ www.oncolys.com
OBP-801 Oncolys BioPharma solid tumors Phase I
(HDAC inhibitor) Edison, NJ www.oncolys.com
OBT076 Oxford BioTherapeutics CD205-positive malignancies, Phase I
(antibody-drug conjugate) San Jose, CA HER2-negative breast cancer www.oxfordbiotherapeutics.com
ofranergene obadenovec (VB-111) VBL Therapeutics platinum-resistant ovarian cancer Phase III
(targeted gene therapy) Modi'in, Israel www.vblrx.com
OKI 179 OnKure Therapeutics solid tumors Phase I
(HDAC inhibitor) Boulder, CO www.onkuretherapeutics.com
OKN-007 (disufenton) Oblato recurrent glioblastoma Phase II
(oxygen radical scavenger) Princeton, NJ (combination therapy)
ORPHAN DRUG
oleclumab AstraZeneca EGFR mutant NSCLC (+osimertinib) Phase I
Wilmington, DE www.asgtgrazeneca.com
Medicines in Development: Cancer ǀ 2020 141
Product Name Sponsor Indication Development Status
omburtamab Y-mAbs Therapeutics CNS/leptomeningeal metastases Phase II
(B7-H3 thernostic mAb) New York, NY from neuroblastoma (pediatric) (131I) www.ymabs.com
ORPHAN DRUG (Breakthrough Therapy),
desmoplastic small round cell
tumor (pediatric) (131I)
CNS/leptomeningeal metastases Phase I
(intrathecal immunotherapy) (131I), www.ymabs.com
diffuse intrinsic pontine glioma
(pediatric) (124I), medulloblastoma
(pediatric) (177-Lu)
omidubicel Gamida Cell hematologic malignancies Phase III
(stem cell therapy) Jerusalem, Israel (Breakthrough Therapy) www.gamida-cell.com
ORPHAN DRUG
ONA-XR (onapristone ER) Context Therapeutics granulosa cell ovarian cancer Phase II
(progesterone receptor antagonist) Philadelphia, PA (Fast Track), metastatic breast cancer, www.contexttherapeutics.com
recurrent endometrial cancer
progesterone-positive cancer Phase I
www.contexttherapeutics.com
Medicines in Development: Cancer ǀ 2020 142
Product Name Sponsor Indication Development Status
ONC201 Oncoceutics H3 K27M-mutant gliomas (adult), Phase II
(DRD2 antagonist/ClpP agonist) Philadelphia, PA recurrent glioblastoma, midline glioma www.oncoceutics.com
ORPHAN DRUG (Fast Track), endometrial cancer
H3 K27M-mutant gliomas (pediatric) Phase I
www.oncoceutics.com
ONC206 Oncoceutics CNS cancer Phase I
(DRD2 antagonist/ClpP agonist) Philadelphia, PA www.oncoceutics.com
ONC-392 OncoImmune NSCLC, advanced solid tumors Phase I
(CTLA4 inhibitor) Rockville, MD www.oncoimmune.com
ONCOS-102 Targovax melanoma Phase I
(oncolytic adenovirus therapy) Oslo, Norway www.targovax.com
OncoVax® Vaccinogen colorectal cancer (Fast Track) Phase III
autologous cancer vaccine Savannah, GA www.vaccinogen-oncovax.com
ONCR-177 Oncorus solid tumors Phase I
(oncolytic virus therapy) Cambridge, MA www.oncorus.com
Onivyde® Ipsen SCLC (Fast Track), 1L pancreatic Phase III
irinotecan liposome injection Cambridge, MA ductal adenocarcinoma (Fast Track) www.ipsen.com
Medicines in Development: Cancer ǀ 2020 143
Product Name Sponsor Indication Development Status
ONO-7475 Ono Pharmaceutical acute leukemia Phase I
(Axl/Mer inhibitor) Osaka, Japan www.ono.co.jp
Onureg® Bristol-Myers Squibb AML (maintenance) Phase III
axacitidine tablets Princeton, NJ www.bms.com
MDS Phase II
www.bms.com
onvansertib Cardiff Oncology AML, metastatic castration-resistant Phase II
(PLK1 inhibitor) San Diego, CA prostate cancer www.cardiffoncology.com
ORPHAN DRUG
KRAS-mutant colorectal cancer Phase I
(Fast Track) www.cardiffoncology.com
OP-1250 Olema Pharmaceuticals HR-positive HER2-negative Phase I/II
(ER antagonist/SERD) San Francisco, CA breast cancer www.olema.com
OPB-111077 Otsuka America Pharmaceutical hematologic malignancies Phase I
(STAT inhibitor) Rockville, MD www.otsuka.com
Medicines in Development: Cancer ǀ 2020 144
Product Name Sponsor Indication Development Status
Opdivo® Bristol-Myers Squibb 1L head and neck cancer, 1L locally- Phase III
nivolumab Princeton, NJ advanced head and neck cancer, ww.bms.com
1L glioblastoma, adjuvant esophageal/
gastroesophageal cancer,
1L hepatocellular carcinoma,
adjuvant hepatocellular carcinoma,
adjuvant bladder cancer,
neoadjuvant NSCLC, unresectable
NSCLC, adjuvant renal cell carcinoma,
adjuvant gastric cancer, adjuvant
melanoma, metastatic castration-
resistant prostate cancer,
neoadjuvant ER-positive
HER2-negative breast cancer,
refractory Hodgkin lymphoma,
high-risk non-muscle bladder
cancer (+BCG)
1L colorectal cancer, pan-tumor Phase II
TMB high, pediatric solid tumors, www.bms.com
Hodgkin lymphoma (pediatric),
DLBCL, follicular lymphoma,
primary testicular lymphoma,
neoadjuvant ER-positive HER2-
negative breast cancer
hematologic malignancies, Phase I
solid tumors www.bms.com
Medicines in Development: Cancer ǀ 2020 145
Product Name Sponsor Indication Development Status
Opdivo® + bempegaldesleukin Bristol-Myers Squibb muscle invasive bladder cancer, Phase III
nivolumab + bempegaldesleukin Princeton, NJ 1L melanoma, 1L renal cell www.bms.com
Nektar Therapeutics carcinoma www.nektar.com
San Francisco, CA
1L bladder cancer Phase II
www.bms.com
www.nektar.com
Opdivo® + Cabometyx® Bristol-Myers Squibb advanced renal cell carcinoma application submitted
nivolumab + cabozantinib Princeton, NJ www.bms.com
Exelixis www.exelixis.com
Alameda, CA
Opdivo® + Empliciti® Bristol-Myers Squibb multiple myeloma Phase II
nivolumab + elotuzumab Princeton, NJ www.bms.com
Opdivo® + linrodostat Bristol-Myers Squibb neoadjuvant muscle invasive bladder Phase III
nivolumab + linrodostat Princeton, NJ cancer, 1L metastatic melanoma www.bms.com
solid tumors Phase II
www.bms.com
Medicines in Development: Cancer ǀ 2020 146
Product Name Sponsor Indication Development Status
Opdivo® + relatilmab Bristol-Myers Squibb 1L melanoma Phase III
nivolumab + relatilmab Princeton, NJ www.bms.com
solid tumors Phase II
www.bms.com
Opdivo® + Yervoy® Bristol-Myers Squibb 1L head and neck cancer, Phase III
nivolumab + ipilimumab Princeton, NJ 1L esophageal cancer, www.bms.com
1L hepatocellular carcinoma,
1L bladder cancer, adjuvant renal
cell carcinoma, metastatic renal
cell carcinoma (+cabozantinib),
1L gastric cancer, adjuvant melanoma,
EGFR-mutant NSCLC, 1L NSCLC,
unresectable NSCLC
metastatic castration-resistant Phase II
prostate cancer, solid tumors www.bms.com
oregovomab OncoQuest Pharmaceuticals ovarian cancer Phase III
(anti-CA-125 antibody) Edmonton, Canada www.oncoquestinc.com
ORPHAN DRUG
ORIC-101 ORIC Pharmaceuticals prostate cancer (combination therapy), Phase I
(glucocorticoid receptor antagonist) South San Francisco, CA solid tumors (combination therapy) www.oricpharma.com
Medicines in Development: Cancer ǀ 2020 147
Product Name Sponsor Indication Development Status
ORIN1001 Orinove metastatic breast cancer Phase I/II
(IRE1a inhibitor) Newbury Park, CA www.orinove.com
orva-cel (JCARH125) Bristol-Myers Squibb multiple myeloma Phase II
(orvacabtagene autoleucel) Princeton, NJ www.bms.com
(BCMA CAR-T cell therapy)
ORPHAN DRUG
OS2966 OncoSynergy recurrent glioblastoma Phase I
(anti-CD29 beta1 integrin) Stamford, CT www.oncosynergy.com
ORPHAN DRUG
OSE-2101 OSE Immunotherapeutics NSCLC Phase III
(cancer vaccine) Nantes, France www.ose-immuno.com
ORPHAN DRUG
OT-82 OncoTartis lymphoma Phase I
(NAMPT inhibitor) Buffalo, NY www.oncotartis.com
OT-101 Mateon/Oncotelic glioblastoma, melanoma, Phase II completed
(anti-TGF-B2 antisense) Agoura Hills, CA pancreatic www.mateon.com
OTS167 OncoTherapy Science breast cancer, triple negative Phase I
(MELK inhibitor) Kawasaki, Japan breast cancer, hematologic www.oncotherapy.co.jp
malignancies
Medicines in Development: Cancer ǀ 2020 148
Product Name Sponsor Indication Development Status
ovapuldencel-T (AVOVA-1) AIVITA Biomedical ovarian cancer Phase II
(tumor stem cell specific Irvine, CA www.aivitabiomedical.com
dendritic cell vaccine)
OXi4503 Mateon/Oncotelic AML (Fast Track), MDS Phase I/II completed
(combretastatin A1 phosphate) Agoura Hills, CA www.mateon.com
ORPHAN DRUG
P1101 PharmaEssentia polycythemia vera application submitted
(ropeginterferon alfa-2b) Burlington, MA www.pharmaessentia.com
ORPHAN DRUG
PAC-1 Vanquish Oncology advanced solid tumors Phase I
(procaspase activating compound-1) Champaign, IL www.vanquishoncology.com
pacritinib CTI BioPharma myelofibrosis (Fast Track) Phase III
(JAK2/IRAK1 inhibitor) Seattle, WA www.ctibiopharma.com
ORPHAN DRUG
Padcev® Astellas 1L metastatic urothelial cancer Phase III
enfortumab vedotin-ejfv Northbrook, IL (+pembrolizumab), metastatic www.astellas.com
Seagen urothelial cancer (pretreated), www.seagen.com
Bothell, WA muscle invasive bladder cancer
(+pembrolizumab)
1L muscle invasive bladder cancer Phase II
(+pembrolizumab), urothelial www.astellas.com
cancer, solid tumors www.seagen.com
Medicines in Development: Cancer ǀ 2020 149
Product Name Sponsor Indication Development Status
pamiparib (BGB-290) BeiGene 1L platinum-sensitive gastric cancer Phase II
(PARP inhibitor) Cambridge, MA (maintenance) www.beigene.com
solid tumors (monotherapy and Phase I
combination therapy), glioblastoma www.beigene.com
pamrevlumab (RG-3019) Fibrogen pancreatic cancer (combination Phase III
(anti-CTGF antibody) San Francisco, CA therapy (Fast Track) www.fibrogen.com
ORPHAN DRUG
panobinostat (MTX110) Midatech Pharma DIPG (pediatric), medulloblastoma Phase I
(HDAC inhibitor) Cardiff, United Kingdom www.midatechpharam.com
ORPHAN DRUG
parsaclisib Incyte follicular lymphoma, marginal zone Phase II
(PI3Kδ inhibitor) Wilmington, DE lymphoma, mantle cell lymphoma www.incyte.com
patidegib PellePharm basal cell carcinoma Phase II
(SMO protein inhibitor) San Francisco, CA www.pellepharm.com
PBCAR0191 Precision Biosciences B-cell ALL (Fast Track), NHL Phase I/II
(anti-CD19 CAR-T cell therapy) Durham, NC www.precisionbiosciences.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 150
Product Name Sponsor Indication Development Status
PBCAR20A Precision Biosciences CLL/SLL, NHL Phase I/II
(anti-CD20 CAR-T cell therapy) Durham, NC www.precisionbiosciences.com
ORPHAN DRUG
PBCAR269A Precision Biosciences multiple myeloma (Fast Track) Phase I/II
(CAR-T cell therapy) Durham, NC www.precisionbiosciences.com
ORPHAN DRUG
P-BCMA-101 Poseida Therapeutics multiple myeloma Phase II
(CAR-T cell therapy) San Diego, CA www.poseida.com
ORPHAN DRUG
PBF-1129 Palobiofarma NSCLC Phase I
(A2b receptor antagonist) Barcelona, Spain www.palobiofarma.com
PBI-05204 Phoenix Biotechnology pancreatic cancer Phase II
(Nerium oleander extract) San Antonio, TX www.phoenixbiotechnology.com
pbi-shRNA™ Gradalis Ewing's sarcoma Phase I
EWS/FLT1 type 1 lipoplex Carrollton, TX www.gradalisinc.com
PC14586 PMV Pharmaceuticals p53 Y22OC-mutated solid tumors Phase I/II
(p53 reactivator) Cranbury, NJ (tumor agnostic) (Fast Track) www.pmvpharma.com
Medicines in Development: Cancer ǀ 2020 151
Product Name Sponsor Indication Development Status
PD-L1 t-haNK ImmunityBio 1L+ pancreatic cancer Phase II
(CAR-T NK cell therapy) Culver City, CA (+aldoxorubicin and N-803), www.nantkwest.com
NantKwest 3L NSCLC (+N-803 and checkpoint
Culver City, CA inhibitor)
2L triple negative breast cancer Phase I
(+N-803) www.nantkwest.com
PDS0101 PDS Biotechnology 1L metastatic head and neck cancer, Phase II
(cancer immunotherapy) Florham Park, NJ cervical cancer www.pdsbiotech.com
HPV-associated malignancies Phase I/II
www.pdsbiotech.com
pegargiminase Polaris Pharmaceuticals mesothelioma (combination therapy) Phase II/III
(ADI-PEG-20) San Diego, CA www.polarispharma.com
ORPHAN DRUG
advanced solid tumors Phase I
www.polarispharma.com
pegzilarginase (AEB1102) Aeglea Biotherapeutics hematologic malignancies, Phase I
(recombinant enzyme Austin, TX advanced solid tumors www.aegleabio.com
replacement therapeutic)
pelareorep Oncolytics Biotech HR-positive HER2-negative breast Phase II
(immuno-oncolytic virus) Calgary, Canada cancer www.oncolyticsbiotech.com
Medicines in Development: Cancer ǀ 2020 152
Product Name Sponsor Indication Development Status
Pemazyre® Incyte bladder cancer, solid tumors, Phase II
pemigatinib Wilmington, DE 8p11 myeloproliferative neoplasms www.incyte.com
ORPHAN DRUG (Breakthrough Therapy)
PEN-221 Tarveda Therapeutics SSTR2-expressing solid tumors, Phase I/II
(SSTR2 modulator) Watertown, MA including neuroendocrine tumors www.tarvedatx.com
and SCLC
PEN-866 Tarveda Therapeutics solid tumors Phase I/II
(Topo-1 inhibitor) Watertown, MA www.tarvedatx.com
PEPI-CMV Annias Immunotherapeutics malignant glioma, recurrent Phase I
(dendritic cell vaccine) Chapel Hill, NC medulloblastoma www.anniasimmuno.com
pepinemab Vaccinex NSCLC (combination therapy) Phase II
(anti-SEMA4D mAb) Rochester, NY www.vaccinex.com
peposertib (M3814) EMD Serono rectal cancer Phase II
(DNA-PK inhibitor) Rockland, MA www.emdserono.com
solid tumors Phase I
www.emdserono.com
Medicines in Development: Cancer ǀ 2020 153
Product Name Sponsor Indication Development Status
Pexa-Vec SillaJen Biotherapeutics renal cell carcinoma (combination Phase III
(pexastimogene devacirepvec) Busan, South Korea therapy), liver cancer www.sillajen.com
(oncolytic vaccinia poxvirus)
ORPHAN DRUG
pevonedistat (TAK-924) Takeda unfit AML, HR-negative MDS Phase III
(NEDD8 inhibitor) Deerfield, IL (Breakthrough Therapy) www.takeda.com
ORPHAN DRUG
PF-05082566 Pfizer cancer (+axicabtagene ciloleucel) Phase I
(CD137 agonist) New York, NY www.pfizer.com
PF-05212384 Pfizer metastatic breast cancer Phase I
(mTOR/PI3K inhibitor) New York, NY www.pfizer.com
PF-06804103 Pfizer solid tumors Phase I
(HER2 antibody-drug conjugate) New York, NY www.pfizer.com
PF-06821497 Pfizer lymphoma, solid tumors Phase I
(EZH2 inhibitor) New York, NY www.pfizer.com
PF-06863135 Pfizer multiple myeloma Phase I
(BCMA-CD3 bispecific antibody) New York, NY www.pfizer.com
PF-06873600 Pfizer metastatic breast cancer Phase I
(CDK-2/4/6 inhibitor) New York, NY www.pfizer.com
Medicines in Development: Cancer ǀ 2020 154
Product Name Sponsor Indication Development Status
PF-06936308 Pfizer solid tumors Phase I
(therapeutic vaccine) New York, NY www.pfizer.com
PF-06939999 Pfizer solid tumors Phase I
(PRMT5 inhibitor) New York, NY www.pfizer.com
PF-06940434 Pfizer solid tumors Phase I
(integrin αV/β8 antagonist) New York, NY www.pfizer.com
PF-06952229 Pfizer solid tumors Phase I
(TGFBR1 inhibitor) New York, NY www.pfizer.com
PF-07062119 Pfizer solid tumors Phase I
(GUCY2cxCD3 bispecific antibody) New York, NY www.pfizer.com
PF-07104091 Pfizer metastatic breast cancer Phase I
(CDK2 inhibitor) New York, NY www.pfizer.com
PF-07220060 Pfizer metastatic breast cancer Phase I
(CDK4 inhibitor) New York, NY www.pfizer.com
PF-07265807 Pfizer solid tumors Phase I
(AXL/MERTK inhibitor) New York, NY www.pfizer.com
Medicines in Development: Cancer ǀ 2020 155
Product Name Sponsor Indication Development Status
PHE885 Novartis multiple myeloma Phase I
(BCMA CAR-T cell therapy) East Hanover, NJ www.novartis.com
PIN-2 PIN Pharma solid tumors Phase I
(immune modulator) New York, NY www.pinpharma.com
Piqray® Novartis 2L/3L head and neck squamous Phase III
alpelisib East Hanover, NJ cell carcinoma, ovarian cancer, www.novartis.com
HER2-positive breast cancer,
triple negative breast cancer
plinabulin BeyondSpring Pharmaceuticals 2L/3L NSCLC (combination therapy) Phase III
New York, NY www.beyongspringpharma.com
PLX2853 Daiichi Sankyo AML, solid tumors Phase I
(BET inhibitor) Basking Ridge, NJ www.dsi.com
PLX8394 Novellus solid tumors Phase I/II
(BRAF inhibitor) Berkeley, CA www.novellusbio.com
PLX9486 Cogent Biosciences gastrointestinal stromal tumors Phase I/II
(KIT inhibitor) Cambridge, MA www.cogentbio.com
PMD-026 Phoenix Molecular Designs metastatic breast cancer, Phase I
(RSK inhibitor) San Diego, CA triple negative breast cancer www.phoenixmd.com
Medicines in Development: Cancer ǀ 2020 156
Product Name Sponsor Indication Development Status
Polivy® Roche/Genentech 1L DLBCL (Breakthrough Therapy) Phase III
polatuzumab vedotin-piiq South San Francisco, CA www.roche.com
ORPHAN DRUG
polyclonal antibody stimulator Cancer Advances gastric cancer Phase III
(gastrin inhibitor) Durham, NC www.canceradvances.com
ORPHAN DRUG
PolyPEPI 1018 Treos Bio colorectal cancer Phase I/II
(cancer vaccine) London, United Kingdom www.treosbio.com
Pomalyst® Bristol-Myers Squibb glioblastoma (pediatric) Phase II
pomalidomide Princeton, NJ www.bms.com
poziotinib Spectrum Pharmaceuticals EGFR or HER2 Exon 20 Phase II
(TKI inhibitor) Henderson, NV mutation-positive NSCLC www.sppirx.com
P-PSMA-101 Poseida Therapeutics prostate cancer Phase I
(CAR-T cell therapy) San Diego, CA www.poseida.com
pracinostat MEI Pharma high-risk MDS (combination therapy) Phase II
(HDAC inhibitor) San Diego, CA www.meipharma.com
ORPHAN DRUG Helsinn
Lugano, Switzerland
Medicines in Development: Cancer ǀ 2020 157
Product Name Sponsor Indication Development Status
prexigebersen Bio-Path AML, CML Phase II
(GRB2 adaptor protein inhibitor) Bellaire, TX www.biopathholdings.com
ORPHAN DRUG
PRGN-2009 Precigen solid tumors Phase I
(immunotherapy vaccine) Germantown, MD www.orecigen.com
PRGN-3005 Precigen ovarian cancer Phase I
(UltraCAR-T cell therapy) Germantown, MD www.precigen.com
pritumumab Nascent Biotech brain cancer Phase I
(IgG antibody) San Diego, CA www.nascentbiotech.com
ORPHAN DRUG
Prophage™ Agenus newly-diagnosed glioblastoma Phase II
autologous cancer vaccine Lexington, MA www.agenusbio.com
ORPHAN DRUG
Provenge® Dendreon newly-diagnosed prostate cancer Phase III
sipuleucel-T Seal Beach, CA www.dendreon.com
proxalutamide (GT0918) Suzhou Kintor Pharmaceutical metastatic castration-resistant Phase II
(androgen receptor antagonist) Jiangsu, China prostate cancer www.kintor.com.cn
PRS-343 Pieris Pharmaceuticals solid tumors Phase I
(4-1BB/HER2 bispecific antibody) Boston, MA www.pieris.com
Medicines in Development: Cancer ǀ 2020 158
Product Name Sponsor Indication Development Status
PRT543 Prelude Therapeutics hematologic malignancies, Phase I
(PRMT5 protein inhibitor) Wilmington, DE solid tumors www.preludetx.com
PRT811 Prelude Therapeutics recurrent glioma, solid tumors Phase I
(PRMT5 protein inhibitor) Wilmington, DE www.preludetx.com
PRT1419 Prelude Therapeutics hematologic malignancies Phase I
(MCL1 protein inhibitor) Wilmington, DE www.preludetx.com
PRV111 Privo Technologies oral squamous call carcinoma Phase I/II
(cisplatin transmucosal patch) Peabody, MA www.privotechnologies.com
PSMA CAR-T cell therapy Tmunity Therapeutics prostate cancer Phase I
Philadelphia, PA www.tmunity.com
PSMA TTC Progenics Pharmaceuticals prostate cancer Phase II
(thorium-227 labeled PSMA-targeted New York, NY www.progenics.com
antibody therapeutic)
PT-112 Phosplatin Therapeutics advanced solid tumors, Phase I
(apoptosis stimulant) New York, NY multiple myeloma www.phosplatin.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 159
Product Name Sponsor Indication Development Status
PT2385 Merck Von Hippel-Lindau disease Phase II
(HIF-2α inhibitor) Kenilworth, NJ associated with clear cell renal www.merck.com
cell carcinoma
PTC299 PTC Therapeutics AML Phase I
(DHODH inhibitor) South Plainfield, NJ www.ptcbio.com
PTC596 PTC Therapeutics DIPG, leiomyosarcoma (Fast Track) Phase I
(BMI1 inhibitor) South Plainfield, NJ www.ptcbio.com
ORPHAN DRUG
PTX-200 (triciribine) Prescient Therapeutics acute leukemia Phase I/II
(Akt inhibitor) South Melbourne, Australia www.ptxtherapeutics.com
PTX-35 Pelican Therapeutics solid tumors Phase I
(TNFRSF25 agonist) San Antonio, TX www.pelicantherapeutics.com
PU-H71 Samus Therapeutics myelofibrosis, breast cancer Phase I
(HSP90 heat shock protein inhibitor) Topsfield, MA www.samustherapeutics.com
ORPHAN DRUG
PV-10 Provectus Biopharmaceuticals metastatic cancer to the liver, Phase I
(rose bengal sodium) Knoxville, TN metastatic melanoma www.provectusbio.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 160
Product Name Sponsor Indication Development Status
PVSRIPO Istari Oncology glioblastoma (Breakthrough Therapy), Phase II
(recombinant polio virus vaccine) Morrisville, NC (monotherapy and combination www.istarioncology.com
ORPHAN DRUG therapy), refractory melanoma
recurrent glioma (pediatric), Phase I
invasive breast cancer, www.istarioncology.com
unresectable melanoma
PVX-410 OncoPep triple negative breast cancer, Phase I
(multi-peptide therapeutic cancer vaccine)Boston, MA smoldering multiple myeloma www.oncopep.com
ORPHAN DRUG
QBS10072S Quadriga BioSciences solid tumors Phase I
(bifunctional amino acid analogue Los Altos, CA www.quadrigabiosciences.com
targeting LAT1)
Qinlock™ Deciphera Pharmaceuticals 2L gastrointestinal stomal tumors Phase III
ripretinib Waltham, MA www.deciphera.com
solid tumors Phase I
www.deciphera.com
quaratusugene ozeplasmid (GPX-001) Genprex NSCLC (Fast Track) Phase I/II
(nanoparticle gene therapy) Austin, TX www.genprex.com
Medicines in Development: Cancer ǀ 2020 161
Product Name Sponsor Indication Development Status
quizartinib Daiichi Sankyo 1L AML (Breakthrough Therapy) Phase III
(FLT3 inhibitor) Basking Ridge, NJ (Fast Track) www.dsi.com
ORPHAN DRUG
RAPA-201 Rapa Therapeutics multiple myeloma Phase II
(CAR-T cell therapy) Rockville, MD
RBN-2397 Ribon Therapeutics solid tumors Phase I
(PARP7 inhibitor) Cambridge, MA www.ribontx.com
rebastinib (DCC-2036) Deciphera Pharmaceuticals solid tumors (combination therapy) Phase I/II
(TIE2 selective inhibitor) Waltham, MA www.deciphera.com
Reblozyl® Accleron Pharma MDS (ESA naïve) Phase III
luspatercept-aamt Cambridge, MA www.bms.com
Bristol-Myers Squibb
Princeton, NJ
REGN1979 (adronextamab) Regeneron Pharmaceuticals B-cell non-Hodgkin lymphoma Phase II
(CD20xCD3 bispecific antibody) Tarrytown, NY www.regeneron.com
ORPHAN DRUG
B-cell malignancies Phase I
www.regeneron.com
REGN3767 Regeneron Pharmaceuticals hematologic malignancies, Phase I
(LAG3 inhibitor) Tarrytown, NY solid tumors www.regeneron.com
Medicines in Development: Cancer ǀ 2020 162
Product Name Sponsor Indication Development Status
REGN4018 Regeneron Pharmaceuticals ovarian cancer (monotherapy), Phase I
(MUCxCD3 bispecific antibody) Tarrytown, NY ovarian cancer (+cemiplimab) www.regeneron.com
Sanofi www.sanofi.com
Bridgewater, NJ
REGN5093 Regeneron Pharmaceuticals MET-altered advanced NSCLC Phase I
(METxMET bispecific antibody) Tarrytown, NY www.regeneron.com
REGN5458 Regeneron Pharmaceuticals multiple myeloma Phase II
(BCMAxCD3 bispecific antibody) Tarrytown, NY www.regeneron.com
Sanofi www.sanofi.com
Bridgewater, NJ
REGN5459 Regeneron Pharmaceuticals multiple myeloma Phase I
(BCMAxCD3 bispecific antibody) Tarrytown, NY www.regeneron.com
Sanofi www.sanofi.com
Bridgewater, NJ
REGN5678 Regeneron Pharmaceuticals prostate cancer Phase I
(PSMAxCD28 antibody) Tarrytown, NY www.regeneron.com
REGN6569 Regeneron Pharmaceuticals solid tumors Phase I
(GITR antibody) Tarrytown, NY www.regeneron.com
REIC gene therapy (MTG201) Momotaro-Gene malignant pleural mesothelioma Phase II
(AAV-based gene therapy) Okayama, Japan www.mt-gene.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 163
Product Name Sponsor Indication Development Status
relacorilant Corcept Therapeutics pancreatic cancer (+nab-paclitaxel) Phase III
(glucocorticoid receptor II antagonist) Menlo Park, CA www.corcept.com
ORPHAN DRUG Corcept Therapeutics
ovarian cancer (+mab-pacitaxel), Phase II
castration-resistant prostate cancer www.corcept.com
(+enzalutamide)
adrenocortical cancer Phase I
(+pembrolizumab) www.corcept.com
relatlimab Bristol-Myers Squibb hematologic malignancies, Phase I
(LAG-3 inhibitor) Princeton, NJ solid tumors www.bms.com
relugolix Myovant Sciences advanced prostate cancer application submitted
(LHRH receptor antagonist) Brisbane, CA www.myovant.com
REM-001 Kintara Therapeutics cutaneous metastatic breast cancer Phase III
(photodynamic therapy) San Diego, CA www.kintara.com
RenovoCath™/Gemcitabine RenovoRx locally-advanced pancreatic Phase III
trans-arterial micro-perfusion Los Altos, CA cancer www.renovorx.com
delivery system
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 164
Product Name Sponsor Indication Development Status
repotrectinib (TPX-0005) Turning Point Therapeutics ROS1+ advanced NSCLC (Fast Track), Phase I/II
(ROS1/TRK inhibitor) San Diego, CA NTRK+ advanced solid tumors www.tptherapeutics.com
ORPHAN DRUG (Fast Track), ALK,ROS1,NTRK-altered
soldi tumors (up to 25 years)
Retevmo™ Eli Lilly 1L RET-mutant NSCLC, Phase III
selpercatinib Indianapolis, IN 1L RET-mutant thyroid cancer www.lilly.com
RET-mutant solid tumors Phase I/II
www.lilly.com
retifanlimab Incyte NSCLC, squamous cell anal carcinoma Phase III
(anti-PD-1 mAb) Wilmington, DE www.incyte.com
MacroGenics
Rockville, MD
Merkel cell carcinoma, Phase II
MSI-H endometrial cancer www.incyte.com
RG6062 (glofitamab) Roche/Genentech hematologic malignancies Phase I
(bispecific mAb) South San Francisco, CA www.roche.com
RG6076 Roche/Genentech hematologic malignancies Phase I
(CD19-4-1BBL) South San Francisco, CA www.roche.com
Medicines in Development: Cancer ǀ 2020 165
Product Name Sponsor Indication Development Status
RG6114 (GDC-0077) Roche/Genentech HR-positive HER2-negative breast Phase III
(PI3K inhibitor) South San Francisco, CA cancer www.roche.com
breast cancer (combination therapy), Phase I
solid tumors www.roche.com
RG6115 (RO7119929) Roche/Genentech hepatocellular carcinoma Phase I
(TLR7 agonist) South San Francisco, CA www.roche.com
RG6139 Roche/Genentech solid tumors Phase I
(PD-1xLAG3 bispecific antibody) South San Francisco, CA www.roche.com
RG6160 (BFCR4350A) Roche/Genentech multiple myeloma Phase I
(FcRH5xCD3 bispecific antibody) South San Francisco, CA www.roche.com
RG6171 (GDC-9545) Roche/Genentech ER-positive, HER2-negative Phase III
(SERD) South San Francisco, CA metastatic breast cancer www.roche.com
1L ER-positive, HER2-negative breast Phase I
cancer www.roche.com
Medicines in Development: Cancer ǀ 2020 166
Product Name Sponsor Indication Development Status
RG6180 (BNT122) BioNTech metastatic melanoma Phase II
(mRNA cancer vaccine) Mainz, Germany (+pembrolizumab) www.biontech.de
Roche/Genentech www.roche.com
South San Francisco, CA
solid tumors Phase I
www.biontech.de
www.roche.com
RG6185 (GDC-5573) Roche/Genentech solid tumors Phase I
(pan-RAF inhibitor) South San Francisco, CA www.roche.com
Hanmi Pharmaceutical
Seoul, South Korea
RG6194 (BTRC4017A) Roche/Genentech breast cancer Phase I
(HER2xCD3 bispecific antibody) South San Francisco, CA www.roche.com
RG6279 Roche/Genentech solid tumors Phase I
(PD-1/IL2v) South San Francisco, CA www.roche.com
RG6286 (BLYG8824A) Roche/Genentech colorectal cancer Phase I
South San Francisco, CA www.roche.com
RG6290 (IMC-C103C) Immunocore MAGE4-expressing solid tumors Phase I
(MAGE-A4 ImmTac) Conshohocken, PA www.roche.com
Roche/Genentech
South San Francisco, CA
Medicines in Development: Cancer ǀ 2020 167
Product Name Sponsor Indication Development Status
RG6292 Roche/Genentech solid tumors Phase I
(CD25 mAb) South San Francisco, CA www.roche.com
RG6296 (AFM-26) Affimed multiple myeloma Phase I
(BCMA x CD16a bispecific antibody) Heidelberg, Germany www.roche.com
Roche/Genentech
South San Francisco, CA
RG6330 (GDC-6036) Roche/Genentech solid tumors with KRAS G12C Phase I
(KRAS G12C inhibitor) South San Francisco, CA mutation www.roche.com
RG7440 (ipatasertib) Roche/Genentech 1L triple negative breast cancer, Phase III
(pan-Akt inhibitor) South San Francisco, CA 1L castration-resistant prostate www.roche.com
cancer, HR-positive breast cancer
triple negative breast cancer, Phase I
prostate cancer, solid tumors www.roche.com
RG7461 (simlukafusp alfa) Roche/Genentech solid tumors Phase I
(FAP-IL1v fusion protein) South San Francisco, CA www.roche.com
RG7769 Roche/Genentech solid tumors Phase I
(PD-1xTIM3 bispecific antibody) South San Francisco, CA www.roche.com
RG7802 (cibisatamab) Roche/Genentech solid tumors (+atezolizumab) Phase I
(CD3 antigen inhibitor) South San Francisco, CA www.roche.com
Medicines in Development: Cancer ǀ 2020 168
Product Name Sponsor Indication Development Status
RG7827 Roche/Genentech solid tumors Phase I
(FAP-4-1BBL fusion protein) South San Francisco, CA www.roche.com
RG7828 (mosunetuzumab) Roche/Genentech hematologic malignancies Phase I
(CD20xCD3 bispecific antibody) South San Francisco, CA www.gene.com
RG7876 (selicrelumab) Roche/Genentech solid tumors Phase I
(anti-CD40 mAb) South San Francisco, CA www.roche.com
RGX-104 Rgenix 1L non-squamous NSCLC, 2L SCLC Phase I/II
(LXR agonist) New York, NY (combination therapy) www.rgenix.com
RGX-202 Rgenix gastrointestinal cancer Phase I/II
(cancer metabolism therapeutic) New York, NY (combination therapy) www.rgenix.com
rigosertib Onconova Therapeutics KRAS-positive NSCLC Phase I/II
(RAS inhibitor) Newtown, PA www.onconova.com
RiMO-301 RiMO Therapeutics advanced solid tumors Phase I
Chicago, IL
RIVAL-01 (Tbio-6517/TAK-605) Takeda solid tumors Phase I/II
(oncolytic immunotherapy/vaccine) Deerfield, IL www.turnstonebio.com
Turnstone Biologics
New York, NY
Medicines in Development: Cancer ǀ 2020 169
Product Name Sponsor Indication Development Status
rivoceranib Elevar Therapeutics gastric cancer Phase III
(tyrosine kinase inhibitor) Salt Lake City, UT www.elevartherapeutics.com
ORPHAN DRUG
metastatic colorectal cancer Phase I/II
(combination therapy), www.elevartherapeutics.com
adenoid cystic carcinoma
rivogenlecleucel (BPX-501) Bellicum Pharmaceuticals hematologic malignancies Phase I/II completed
(allogeneic polyclonal T-cell therapy) Houston, TX www.bellicum.com
RLY-1971 Relay Therapeutics solid tumors Phase I
(SHP2 inhibitor) Cambridge, MA www.relaytx.com
RLY-4008 Relay Therapeutics solid tumors Phase I
(FGFR2 inhibitor) Cambridge, MA www.relaytx.com
RNL™ Plus Therapeutics recurrent glioblastoma Phase I/II
BMEDA-chelated rhenium Austin, TX (Fast Track) www.plustherapeutics.com
nanoliposome
ORPHAN DRUG
rogaratinib Bayer Pharmaceutical urogenital cancer Phase II
(pan-FGFR inhibitor) Whippany, NJ www.pharma.bayer.com
solid tumors Phase I
www.pharma.bayer.com
Medicines in Development: Cancer ǀ 2020 170
Product Name Sponsor Indication Development Status
ropidoxuridine (IPdR) Shuttle Pharmaceuticals sarcoma Phase I
(radiation sensitizer) Rockville, MD www.shuttlepharma.com
RP1 Replimune cutaneous squamous cell carcinoma Phase I
(oncolytic immunotherapy) Woburn, MA (monotherapy and combination www.replimune.com
therapy), solid tumors (monotherapy
and combination therapy)
RP2 Replimune solid tumors (monotherapy and Phase I
(oncolytic immunotherapy) Woburn, MA (combination therapy) www.replimune.com
RP-3500 Repare Therapeutics solid tumors Phase I/II
(ATR inhibitor) Cambridge, MA www.reparerx.com
RPTR-147 Repertoire Immune Medicines lymphoma (Fast Track), Phase I
(primed T-cell therapy) Cambridge, MA solid tumors (Fast Track) www.repertoire.com
RRx-001 EpicentRx SCLC Phase III
(CD47-SIRPα inhibitor) La Jolla, CA www.epicentrx.com
ORPHAN DRUG
colorectal cancer, neuroendocrine Phase II
tumors, ovarian cancer www.epicentrx.com
glioblastoma Phase I
www.epicentrx.com
Medicines in Development: Cancer ǀ 2020 171
Product Name Sponsor Indication Development Status
RTX-240 Rubius Therapeutics lymphoma, solid tumors Phase I/II
Cambridge, MA www.rubiustx.com
Rubraca® Clovis Oncology BRCA-mutant ovarian cancer Phase III
rucaparib Boulder, CO (Breakthrough Therapy), www.clovisoncology.com
ORPHAN DRUG ovarian cancer (+nivolumab),
castration-resistant prostate cancer
HRR-mutant solid tumors Phase II
www.clovisoncology.com
solid tumors (+sacituzumab Phase I
govitecan) www.clovisoncology.com
RV001V RhoVac prostate cancer (FastTrack) Phase II
(RhoC peptide vaccine) Lund, Sweden www.rhovac.com
RX108 NeuPharma solid tumors Phase I
(sodium-potassium-chloride Suzhou, China www.neupharma.com
symporter inhibitor)
SAIT101 Archigen Biotech follicular lymphoma Phase III
(rituximab biosimilar) Cambridge, United Kingdom www.archigenbio.com
Medicines in Development: Cancer ǀ 2020 172
Product Name Sponsor Indication Development Status
sapacitabine Cyclacel Pharmaceuticals 1L AML (70 years and older) Phase III
(cell cycle inhibitor) Berkeley Heights, NJ www.cyclacel.com
ORPHAN DRUG
AML (combination therapy), Phase I/II
MDS (combination therapy), www.cyclacel.com
BRCA-mutant breast cancer
SAR408701 Sanofi NSCLC (+ramucirumab) Phase III
(anti-CEACAM5 ADC) Bridgewater, NJ www.sanofi.com
2L/3L NSCLC Phase II
www.sanofi.com
SAR439459 Sanofi advanced solid tumors Phase I
(anti-TGFß mAb) Bridgewater, NJ www.sanofi.com
SAR439859 (amcenestrant) Sanofi 1L ER-positive HER2-negative Phase III
(SERD) Bridgewater, NJ breast cancer (combination therapy) www.sanofi.com
2/3L metastatic breast cancer, Phase II
early breast cancer, metastatic www.sanofi.com
breast cancer (combination therapy)
SAR440234 Sanofi leukemia Phase I
(T cell engager multi-specific mAb) Bridgewater, NJ www.sanofi.com
Medicines in Development: Cancer ǀ 2020 173
Product Name Sponsor Indication Development Status
SAR441000 BioNTech solid tumors Phase I
(cytokine mRNA) Mainz, Germany www.sanofi.com
Sanofi
Bridgewater, NJ
SAR442085 Sanofi multiple myeloma Phase I
(anti-CD38 mAb Fc engineered) Bridgewater, NJ www.sanofi.com
SAR442257 Sanofi non-Hodgkin lymphoma, Phase I
(CD38xCD28xCD3 trispecific mAb) Bridgewater, NJ multiple myeloma www.sanofi.com
SAR442720 (RMC-4630) Revolution Medicines solid tumors (+pembrolizumab), Phase I
(SHP2 inhibitor) Redwood City, CA solid tumors (+cobimetinib) www.revmed.com
Sanofi www.sanofi.com
Bridgewater, NJ
SAR444245 (THOR-707) Sanofi solid tumors Phase I
(non-alpha IL1) Bridgewater, NJ www.sanofi.com
Medicines in Development: Cancer ǀ 2020 174
Product Name Sponsor Indication Development Status
Sarclisa® Sanofi newly-diagnosed multiple myeloma Phase III
isatuximab-irfc Bridgewater, NJ (transplant eligible), 2L relapsing/ www.sanofi.com
refractory multiple myeloma,
1L newly-diagnosed multiple myeloma
(transplant ineligible), smoldering
multiple myeloma
colorectal cancer (+atezolizumab), Phase II
AML (pediatric), ALL (pediatric) www.sanofi.com
Sanofi lymphoma (+cemiplimab) Phase II
Bridgewater, NJ www.sanofi.com
Regeneron www.regeneron.com
Tarrytown, NY
sasanlimab (PF-06801591) Pfizer non-muscle invasive bladder cancer Phase III
(anti-PD-1) New York, NY (+BCG), www.pfizer.com
NSCLC (combination therapy) Phase I/II
www.pfizer.com
SB8 Samsung Bioepis NSCLC application submitted
(bevacizumab biosimilar) Incheon, Korea www.samsungbioepis.com
SB 11285 F-star Therapeutics solid tumors Phase I
(STING agonist) Cambridge, MA www.f-star.com
Medicines in Development: Cancer ǀ 2020 175
Product Name Sponsor Indication Development Status
SBP-101 Panbela Therapeutics pancreatic cancer (Fast Track) Phase I
(polyamine analogue) Waconia, MN www.panbela.com
ORPHAN DRUG
SBT6050 Silverback Therapeutics HER2-expressing solid tumors Phase I
(TLR8 agonist) Seattle, WA www.silverbacktx.com
SCO-088 Sun Pharma Advanced Research CML Phase II
(Bcr-Abl inhibitor) Mumbai, India www.sparc.com
SCO-120 Sun Pharma Advanced Research breast cancer Phase I
(SERD) Mumbai, India www.sparc.com
SD-101 TriSalus Life Sciences uveal melanoma, Phase I
(TLR9 agonist) Westminster, CO liver metastases www.trisaluslifesciences.com
SDX-7320 SynDevRx advanced solid tumors Phase I
(MetAP2 inhibitor) Cambridge, MA www.syndevrx.com
SEA-BCMA Seagen multiple myeloma Phase I
(anti-BCMA mAb) Bothell, WA www.seagen.com
SEA-CD40 Seagen pancreatic cancer Phase I
(anti-CD40 mAb) Bothell, WA www.seagen.com
Medicines in Development: Cancer ǀ 2020 176
Product Name Sponsor Indication Development Status
SEA-CD70 Seagen AML, MDS Phase I
(anti-CD70) Bothell, WA www.seagen.com
SEA-TGT Seagen lymphoma, solid tumors Phase I
(TIGIT inhibitor) Bothell, WA www.seagen.com
seclidemstat Salarius Pharmaceuticals Ewing's sarcoma (Fast Track) Phase I
(LSD1 inhibitor) Houston, TX advanced solid tumors www.salariuspharma.com
ORPHAN DRUG
SEL120 Ryvu Therapeutics AML, MDS Phase I
(CDK8/19 inhibitor) Kraków, Poland www.ryvu.com
ORPHAN DRUG
SEL24/MEN1703 Menarini AML Phase I/II
(PIM/FLT3 inhibitor) Florence, Italy www.menarini.com
selitrectinib Bayer Pharmaceuticals solid tumors Phase I
(TRK inhibitor) Whippany, NJ www.pharma.bayer.com
ORPHAN DRUG
sEphB4-HSA VasGene Therapeutics urothelial cancer (combination Phase II
(recombinant fusion protein) Los Angeles, CA therapy), Kaposi's sarcoma
Seprehvir™ Sorrento Therapeutics solid tumors Phase I
selumetinib San Diego, CA www.sorrentotherapeutics.com
Medicines in Development: Cancer ǀ 2020 177
Product Name Sponsor Indication Development Status
SER-401 Seres Therapeutics metastatic melanoma Phase I
(microbiome therapy) Cambridge, MA www.serestherapeutics.com
seribantumab Elevation Oncology NRG1 gene fusion-positive Phase II
(anti-HER3 mAb) New York, NY advanced solid tumors www.elevationoncology.com
seviprotimut-L Polynoma metastatic melanoma (Fast Track) Phase III
(melanoma antigens vaccine) San Diego, CA www.polynoma.com
ORPHAN DRUG
seviteronel (INO-464) Innocrin Pharmaceuticals breast cancer (Fast Track), castration- Phase II
(CYP17 lyase / AR inhibitor) Durham, NC resistant prostate cancer (Fast Track) www.innocrinpharma.org
SF1126 SignalRx Pharmaceuticals hepatocellular carcinoma Phase I
(PI3K/mTOR inhibitor) Omaha, NE www.signalrx.com
SGN-B6A Seagen solid tumors Phase I
(antibody-drug conjugate) Bothell, WA www.seagen.com
SGN-CD30C Seagen multiple myeloma Phase I
(antibody-drug conjugate) Bothell, WA www.seagen.com
SGN-CD228A Seagen solid tumors Phase I
(antibody-drug conjugate) Bothell, WA www.seagen.com
Medicines in Development: Cancer ǀ 2020 178
Product Name Sponsor Indication Development Status
SGT-53 SynerGene Therapeutics pancreatic cancer Phase II
(p53 DNA gene therapy) Potomac, MD
solid tumors (pediatric) Phase I
SGX301 Soligenix cutaneous T-cell lymphoma Phase III
(photodynamic therapy) Princeton, NJ (Fast Track) www.soligenix.com
ORPHAN DRUG
SHR-1258 (pyrotinib) Hengrui Therapeutics HER2-positive solid tumors Phase I
(HER2 tyrosine kinase inhibitor) Princeton, NJ www.hengruitx.com
SI-B001 SystImmune epithelium squamous cell cancer Phase I
(EGFRxHER3 bispecific antibody) Redmond, WA www.systimmune.com
SI-B003 SystImmune solid tumors Phase I
(bispecific antibody) Redmond, WA www.systimmune.com
siG12D-LODER Silenseed pancreatic cancer Phase II
(antisense K-ras RNA gene therapy) Jerusalem, Israel
silmitasertib (CX-4945) Senhwa Biosciences cholangiocarcinoma Phase I/II
(CK2 inhibitor) Taipei, Taiwan www.senhwabiosciences.com
ORPHAN DRUG
basal cell carcinoma Phase I
www.senhwabiosciences.com
Medicines in Development: Cancer ǀ 2020 179
Product Name Sponsor Indication Development Status
sintilimab (IBI308) Innovent Biologics advanced/metastatic solid Phase I/II
(anti-PD-1 mAb) Jiangsu, China malignancies www.innoventbio.com
sitravatinib Mirati Therapeutics 2L/3L NSCLC (+PD-1) Phase III
(multi kinase inhibitor) San Diego, CA www.mirati.com
bladder cancer (+PD-1), head and Phase II
neck cancer (+PD-1), clear cell www.mirati.com
renal carcinoma (+PD-1)
clear cell renal carcinoma Phase I
(+nivolumab and ipilimumab) www.mirati.com
SKB264 Klus Pharma solid tumors Phase I
(TROP2 antibody-drug conjugate) Cranbury, NJ www.kluspharma.com
SKI-G-801 Genosco AML Phase I
(FLT3/AXL inhibitor) Cambridge, MA www.genosco.com
SL-172154 Shattuck Labs ovarian cancer Phase I
(SIRPα-Fc-CD40L) Durham, NC www.shattucklabs.com
SL-701 Stemline Therapeutics glioblastoma Phase I
(antigen vaccine) New York, NY www.stemline.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 180
Product Name Sponsor Indication Development Status
SL-801 Stemline Therapeutics solid tumors Phase I
(XPO-1 inhibitor) New York, NY www.stemline.com
SLATE Gritstone Oncology NSCLC Phase I/II
(neoantigen-based immunotherapy) Emeryville, CA www.gritstoneoncology.com
Sleeping Beauty TCR ZIOPHARM Oncology solid tumors Phase I
(T cell receptor therapy) Boston, MA www.ziopharm.com
Sleeping Beauty CAR-TCR ZIOPHARM Oncology leukemia, lymphoma Phase I
(CAR-T cell therapy) Boston, MA www.ziopharm.com
SM08502 Samumed solid tumors Phase I
(CLK2/3 inhibitor) San Diego, CA www.samumed.com
SM09419 Samumed hematologic malignancies Phase I
(pan-CLK inhibitor/Wnt modulator) San Diego, CA www.samumed.com
SM-88 (racemetyrosine) Tyme Technologies pancreatic cancer Phase III
(cancer metabolism-based Bedminster, NJ www.tymeinc.com
therapeutic)
ORPHAN DRUGprostate cancer, Phase II
metastatic sarcomas www.tymeinc.com
Medicines in Development: Cancer ǀ 2020 181
Product Name Sponsor Indication Development Status
SNDX-5613 Syndax Pharmaceuticals AML, MLLr leukemia Phase I
(menin inhibitor) Waltham, MA www.syndax.com
ORPHAN DRUG
SNK-01 NKMax America NSCLC (+pembrolizumab) Phase I/II
(autologous NK cell therapy) Santa Ana, CA www.nkmaxamerica.com
solid tumors Phase I
www.nkmaxamerica.com
SNX281 Silicon Therapeutics lymphoma, solid tumors Phase I
(STING agonist) Boston, MA www.silicontx.com
SNS-301 Sensei Biotherapeutics head and neck cancer Phase I/II
(nanoparticle cancer vaccine) Gaithersburg, MD www.senseibio.com
SO-C101 Sotio solid tumors Phase I
(recombinant fusion protein) Boston, MA www.sotio.com
Sofusa® Sorrento Therapeutics cutaneous T-cell lymphoma Phase I
lymphatic delivery system San Diego, CA www.sorrentotherapeutics.com
SOR-C13 Soricimed Biopharma cancer Phase I
(TRPV6 inhibitor) Moncton, Canada www.soricimed.com
Medicines in Development: Cancer ǀ 2020 182
Product Name Sponsor Indication Development Status
sotorasib Amgen NSCLC Phase III
(KRAS inhibitor) Thousand Oaks, CA www.amgen.com
ORPHAN DRUG
advanced colorectal cancer, Phase II
solid tumors www.amgen.com
spartalizumab (PDR001) Novartis metastatic BRAF-V600-positive Phase III
(anti-PD-1 antibody) East Hanover, NJ melanoma (combination therapy) www.novartis.com
metastatic melanoma Phase II
(combination therapy) www.novartis.com
AML (combination therapy), Phase I
solid tumors (combination therapy) www.novartis.com
SPL-108 Splash Pharmaceuticals ovarian cancer Phase II
San Diego, CA www.splashpharma.com
ovarian cancer (combination therapy) Phase I
www.splashpharma.com
SPYK04 Chugai Pharma USA solid tumors Phase I
Berkeley Heights, NJ www.chugai-pharma.com
Medicines in Development: Cancer ǀ 2020 183
Product Name Sponsor Indication Development Status
SQ3370 Shasqi solid tumors Phase I
(SQL70 biopolymer/SPQ33 prodrug) San Francisco, CA www.shasqi.com
SQZ622 Novartis AML Phase I
(CD123xCD3 bispecific antibody) East Hanover, NJ www.novartis.com
SQZ-PBMC-HPV Roche/Genentech HPV-positive solid tumors Phase I
(autologous cell therapy) South San Francisco, CA www.roche.com
SQZ Biotechnologies www.sqzbiotech.com
Watertown, MA
SRF388 Surface Oncology advanced solid tumors, including Phase I
(anti-IL27 mAb) Cambridge, MA renal cell carcinoma, liver cancer www.surfaceoncology.com
ORPHAN DRUG (Fast Track)
SRF617 Surface Oncology advanced solid tumors Phase I
(anti-CD39 mAb) Cambridge, MA www.surfaceoncology.com
SRK-181 Scholar Rock solid tumors Phase I
(TGFβ1 inhibitor) Cambridge, MA www.scholarrock.com
ST101 Sapience Therapeutics solid tumors Phase I
(C/EBPβ inhibitor) Harrison, NY www.sapiencetherapeutics.com
Medicines in Development: Cancer ǀ 2020 184
Product Name Sponsor Indication Development Status
stenoparib Allarity Therapeutics ovarian cancer Phase II
(PARP inhibitor) Scottsdale, AZ www.allarity.com
STING agonist Boehringer Ingelheim solid tumors Phase I
Ridgefield, CT www.boehringer-ingelheim.com
Stivarga® Bayer Pharmaceuticals glioblastoma Phase III
regorafenib Whippany, NJ www.pharma.bayer.com
metastatic colorectal cancer Phase II
(+nivolumab) www.pharma.bayer.com
solid tumors Phase I
www.pharma.bayer.com
STMN1 Gradalis solid tumors Phase I
(RNA interference) Carrollton, TX www.gradalisinc.com
STP1002 ST Pharm advanced solid tumors Phase I
(KRAS inhibitor) Seoul, South Korea www.stpharm.co.kr
STP705 Sirnaomics cutaneous squamous cell Phase I/II
(siRNA therapeutic) Gaithersburg, MD carcinoma www.sirnaomics.com
Medicines in Development: Cancer ǀ 2020 185
Product Name Sponsor Indication Development Status
STRO-001 Sutro Biopharma B-cell malignancies Phase I
(anti-CD74 antibody-drug conjugate) South San Francisco, CA www.sutrobio.com
ORPHAN DRUG
STRO-002 Sutro Biopharma endometrial cancer, ovarian cancer Phase I
(antibody-drug conjugate) South San Francisco, CA www.sutrobio.com
SUBA-itraconazole Inhibitor Therapeutics basal cell carcinoma nevus syndrome, Phase II
ORPHAN DRUG Tampa, FL prostate cancer www.inhibitortx.com
SurVaxM® MimiVax newly-diagnosed glioblastoma Phase III
(SVN53-67/M57-KLH peptide vaccine) Buffalo, NY www.mimvax.com
ORPHAN DRUG
recurrent glioblastoma Phase II
www.mimivax.com
medulloblastoma (pediatric), Phase I
high grade glioma (pediatric), www.mimivax.com
multiple myeloma,
neuroendocrine tumors
SV-BR-1-GM cancer vaccine BriaCell Therapeutics breast cancer (combination therapy) Phase I/II
(allogeneic genetically-engineered Berkeley, CA www.briacell.com
cell vaccine)
Medicines in Development: Cancer ǀ 2020 186
Product Name Sponsor Indication Development Status
SVV-001 Seneca Therapeutics solid tumors, Phase II
(oncolytic virus therapy) Blue Bell, PA neuroendocrine tumors www.senecatherapeutics.com
SX-682 Syntrix Biosystems MDS, melanoma (+pembrolizumab) Phase I
(CXCR1/CXCR2 inhibitor) Auburn, WA www.syntrixbio.com
SY-1425 (tamibarotene) Syros Pharmaceuticals AML, MDS Phase II
(RARα agonist) Cambridge, MA www.syros.com
ORPHAN DRUG
SY-5609 Syros Pharmaceuticals solid tumors Phase I
(CDK7 inhibitor) Cambridge, MA www.syros.com
Sym015 Symphogen solid tumors Phase I
(c-Met inhibitor) Ballerup, Denmark www.symphogen.com
Sym021 Symphogen lymphoma, solid tumors Phase I
(anti-PD-1 mAB) Ballerup, Denmark (+Sym022) www.symphogen.com
lymphoma, solid tumors Phase I
(+Sym023) www.symphogen.com
Sym022 Symphogen lymphoma, solid tumors Phase I completed
(anti-TIM3) Ballerup, Denmark www.symphogen.com
Medicines in Development: Cancer ǀ 2020 187
Product Name Sponsor Indication Development Status
Sym023 Symphogen lymphoma, solid tumors Phase I completed
(anti-LAG3) Ballerup, Denmark www.symphogen.com
SYN004 Synermore Biologics colorectal cancer, Phase I
(EGFR inhibitor) Taipei City, Taiwan head and neck cancer www.synermore.com
SYN125 Synermore Biologics colorectal cancer, Phase I
(anti-PD-1 mAb) Taipei City, Taiwan head and neck cancer www.synermore.com
SYNB1891 Synlogic lymphoma, solid tumors Phase I
(STING agonist) Cambridge, MA www.synlogictx.com
T3011 ImmVira Pharma cutaneous or subcutaneous Phase I
(genetically-engineered recombinant Hong Kong malignancies www.immvira-theravir.com
herpes simplex virus)
TAB001 (toripalimab) Junshi Biosciences advanced malignancies Phase I
(anti-PD-1 antibody) Shanghai, China www.junshipharma.com
TopAlliance Biosciences
Rockville, MD
TAB004 (JS004) Junshi Biosciences advanced solid tumors Phase I
(BTLA protein modulator) Shanghai, China www.junshipharma.com
TopAlliance Biosciences
Rockville, MD
Medicines in Development: Cancer ǀ 2020 188
Product Name Sponsor Indication Development Status
tab-cel® (tabelecleucel) Atara Biotherapeutics EBV-associated post transplant Phase III
(EBV-specific T cell immunotherapy) South San Francisco, CA lymphoproliferative disorder www.atarabio.com
ORPHAN DRUG (EBV+PTLD) following hematopoietic
cell transplant, EBV-PTLD following
solid organ transplant
(Breakthrough Therapy)
EBV-associated nasopharyngeal Phase I/II
carcinoma, EBV-associated cancer www.atarabio.com
Tabrecta™ Novartis NSCLC (Breakthrough Therapy), Phase II
capmatinib East Hanover, NJ EGFR wild-type NSCLC, solid tumors www.novartis.com
ORPHAN DRUG
Taclantis™ Sun Pharma Advanced Research breast cancer application submitted
paclitaxel injection concentrate Mumbai, India www.sparc.life
for suspension
TAEK-VAC-HerBy Bavarian Nordic HER2-expressing solid tumors Phase I/II
(cancer vaccine) Morrisville, NC www.bavarian-nordic.com
Medicines in Development: Cancer ǀ 2020 189
Product Name Sponsor Indication Development Status
Tagrisso® AstraZeneca adjuvant EGFR-mutant NSCLC, Phase III
osimertinib Wilmington, DE stage 3 EGFR-mutant NSCLC, www.astrazeneca.com
1L advanced EGFR-mutant NSCLC
(combination therapy)
EGFR-mutant NSCLC Phase II
www.astrazeneca.com
TAK-007 Takeda CD19-positive hematologic Phase I/II
(CAR-NK cell therapy) Deerfield, IL malignancies www.takeda.com
TAK-102 Takeda solid tumors Phase I
(CAR-T cell therapy) Deerfield, IL www.takeda.com
TAK-169 Molecular Templates multiple myeloma Phase I
(dimeric fusion protein) Austin, TX www.takeda.com
ORPHAN DRUG Takeda
Deerfield, IL
TAK-252 (SL-279252) Shattuck Labs solid tumors Phase I
(PD1-Fc-OX40L) Durham, NC www.shattucklabs.com
ORPHAN DRUG Takeda www.takeda.com
Deerfield, IL
TAK-573 Takeda multiple myeloma, solid tumors Phase I
(CD38 antigen modulator) Deerfield, IL www.takeda.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 190
Product Name Sponsor Indication Development Status
TAK-676 Takeda solid tumors Phase I
(STING agonist) Deerfield, IL www.takeda.com
TAK-940 Takeda CD19-positive hematologic Phase I
Deerfield, IL malignancies www.takeda.com
TAK-981 Takeda non-Hodgkin lymphoma, Phase I/II
(SUMO inhibitor) Deerfield, IL solid tumors www.takeda.com
talabostat (BXCL701) BioXcel Therapeutics neuroendocrine cancer, Phase II
(DPP8/9, CD26, FAP inhibitor) New Haven, CT advanced solid tumors www.bioxceltherapeutics.com
ORPHAN DRUG
pancreatic cancer Phase I/II
www.bioxceltherapeutics.com
Talzenna® Pfizer 1L metastatic castration-resistant Phase III
talazoparib New York, NY prostate cancer (+enzalutamide) www.pfizer.com
2L metastatic castration-resistant Phase II
prostate cancer, germline BRCA- www.pfizer.com
mutant locally advanced triple
negative breast cancer
TAR-200 Janssen Research & Development muscle invasive urothelial Phase I
(gemcitabine-releasing Raritan, NJ cancer (Fast Track) www.janssen.com
intravesical system)
Medicines in Development: Cancer ǀ 2020 191
Product Name Sponsor Indication Development Status
tarloxotinib Rain Therapeutics NSCLC, solid tumors Phase II
(pan-HER inhibitor) Newark, CA www.rainthera.com
TAS-114 Taiho Oncology NSCLC Phase II
(dUTPase inhibitor) Princeton, NJ www.taihooncology.com
TAS-116 Taiho Oncology solid tumors Phase I
(HSP90 inhibitor) Princeton, NJ www.taihooncology.com
TAS-120 (futibatinib) Taiho Oncology breast cancer, cholangiocarcinoma, Phase II
(FGFR inhibitor) Princeton, NJ FGFR aberration cancers www.taihooncology.com
ORPHAN DRUG
TAS-0728 Taiho Oncology solid tumors Phase I/II
(HER2 inhibitor) Princeton, NJ www.taihooncology.com
TAS-1440 Taiho Oncology AML Phase I
(LSD1 inhibitor) Princeton, NJ www.taihooncology.com
TAS-3681 Taiho Oncology prostate cancer Phase I
(androgen receptor antagonist) Princeton, NJ www.taihooncology.com
tasquinimod Active Biotech multiple myeloma Phase I
(oral immunomodulatory therapy) Lund, Sweden www.activebiotech.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 192
Product Name Sponsor Indication Development Status
TAVO (tavokinogene telseplasmid) OncoSec Medical stage III/IV melanoma Phase II
(IL-12 gene therapy) San Diego, CA (+pembrolizumab) (Fast Track), www.oncosec.com
ORPHAN DRUG triple negative breast cancer
(+pembrolizumab)
TAVT-45 Tavanta Therapeutics prostate cancer Phase II
(abiraterone acetate enhanced Media, PA www.tavanta.com
formulation)
Tazverik™ Epizyme 2L follicular lymphoma Phase III
tazemetostat Cambridge, MA 1L epithelioid sarcoma www.epizyme.com
ORPHAN DRUG
1L follicular lymphoma, 1L DLBCL Phase II
(Fast Track) www.epizyme.com
castration-resistant prostate cancer, Phase I
INI-1 negative tumors (pediatric) www.epizyme.com
TBR-760 Tiburio Therapeutics non-functioning pituitary adenoma Phase II
(dopamine/somastatin agonist) Cambridge, MA www.tiburio.com
TBX-3400 Taiga Biotechnologies melanoma Phase I
(cell-based immunotherapy) Aurora, CO www.taigabiotech.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 193
Product Name Sponsor Indication Development Status
TC-110 TCR2 Therapeutics ALL, DLBCL, NHL Phase I/II
(T-cell therapy) Cambridge, MA www.tcr2.com
TC-210 TCR2 Therapeutics cholangiosarcoma, Phase I/II
(T-cell therapy) Cambridge, MA malignant pleural mesothelioma, www.tcr2.com
ORPHAN DRUG NSCLC, ovarian cancer
Tecartus™ Gilead Sciences adult ALL, pediatric ALL Phase II
brexucabtagene autoleucel Foster City, CA www.gilead.com
Kite Pharma
Santa Monica, CA
CLL Phase I
www.gilead.com
Tecentriq® Roche/Genentech adjuvant NSCLC, non-muscle invasive Phase III
atezolizumab South San Francisco, CA bladder cancer, neoadjuvant www.roche.com
HER2-positive breast cancer,
1L triple negative breast cancer,
head and neck cancer, hepato-
cellular carcinoma, renal cell
carcinoma, 1L NSCLC, 1L urothelial
cancer
NSCLC (subcutaneous) Phase II
www.roche.com
various solid tumors (combination Phase I
therapy) www.roche.com
Medicines in Development: Cancer ǀ 2020 194
Product Name Sponsor Indication Development Status
telaglenastat Calithera Biosciences renal cell carcinoma Phase III
(glutaminase inhibitor) South San Francisco, CA (+cabozantinib) www.calithera.com
NSCLC, renal cell carcinoma, Phase II
solid tumors (combination therapy) www.calithera.com
telisotuzumab vedotin (ABBV-399) AbbVie NSCLC Phase II
(antibody-drug conjugate) North Chicago, IL www.abbvie.com
tenalisib (RPG6530) Rhizen Pharmaceuticals T-cell lymphoma (Fast Track) Phase I/II
(PI3K inhibitor) Fort Washington, PA www.rhizen.com
ORPHAN DRUG
tepotinib EMD Serono NSCLC (METex14 skipping) application submitted
(MET inhibitor) Rockland, MA (Breakthrough Therapy) www.emdserono.com
NSCLC (MET amplified) Phase II
www.emdserono.com
terameprocol Erimos high-grade glioma Phase I
(BIRC5 inhibitor) Houston, TX www.erimos.com
Medicines in Development: Cancer ǀ 2020 195
Product Name Sponsor Indication Development Status
tesetaxel Odonate Therapeutics HER2-negative, HR-positive metastatic Phase III
(taxane derivative) San Diego, CA breast cancer with prior taxane www.odonate.com
HER2-negative, HR-positive metastatic Phase II
breast cancer without prior taxane, www.odonate.com
metastatic triple negative breast cancer,
HER2-negative metastatic breast cancer
(65 years and older)
TG-1701 TG Therapeutics B-cell malignancies Phase I
(BTK inhibitor) New York, NY www.tgtherapeutics.com
TG-1801 TG Therapeutics B-cell malignancies Phase I
(CD47xCD19 bispecific antibody) New York, NY www.tgtherapeutics.com
TG4050 Transgene ovarian cancer Phase I
(viral-based neoantigen Boston, MA www.transgene.com
therapeutic vaccine)
ThermoDox® Celsion primary liver cancer (Fast Track) Phase III
doxorubicin liposomal Lawrenceville, NJ www.celsion.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 196
Product Name Sponsor Indication Development Status
Tibsovo® Agios Pharmaceuticals cholangiocarcinoma (Fast Track), Phase III
ivosidenib Cambridge, MA 1L intensive chemotherapy-eligible www.agios.com
ORPHAN DRUG AML, 1L intensive chemotherapy-
ineligible AML
MDS Phase I
www.agios.com
tidutamab (XmAb-18087) Xencor gastrointestinal stromal tumors, Phase I
(SSTR2xCD3 bispecific antibody) Monrovia, CA neuroendocrine tumors www.xencor.com
tilsotolimod (IMO-2125) Idera Pharmaceuticals anti-PD-1 refractory advanced Phase III
(TLR9 agonist) Exton, PA melanoma (combination therapy) www.iderapharma.com
ORPHAN DRUG (Fast Track)
microsatellite instability-high Phase II
colorectal cancer (combination www.iderapharma.com
therapy)
resistant solid tumors Phase I
www.iderapharma.com
tinostamustine (EDO-S101) Mundipharma-EDO hematologic malignancies, Phase I
(HDAC inhibitor) Cambridge, United Kingdom solid tumors www.mundipharmaresearch.com
ORPHAN DRUG
Medicines in Development: Cancer ǀ 2020 197
Product Name Sponsor Indication Development Status
tipifarnib Kura Oncology HRAS-mutant head and neck cancer Phase III
(farnesyltransferase/CXCL12 inhibitor) San Diego, CA www.kuraoncology.com
tiragolumab (RG6058) Roche/Genentech 1L SCLC (+atezolizumab), 1L PD-L1- Phase III
(anti-TIGIT mAb) South San Francisco, CA positive NSCLC (+atezolizumab), www.roche.com
locally advanced esophageal
cancer (+atezolizumab), stage III
unresectable NSCLC (+atezolizumab)
NSCLC (+atezolizumab), Phase II
cervical cancer (+atezolizumab) www.roche.com
hematologic malignancies Phase I
www.roche.com
tirapazamine Teclison liver cancer (combination therapy) Phase II
(type II DNA topoisomerase inhibitor) Montclair, NJ www.teclison.com
Medicines in Development: Cancer ǀ 2020 198
Product Name Sponsor Indication Development Status
tislelizumab BeiGene 1L gastric cancer (combination Phase III
(anti-PD-1 mAB) Cambridge, MA therapy), 2L advanced esophageal www.beigene.com
ORPHAN DRUG squamous cell carcinoma,
1L esophageal squamous cell
carcinoma (combination therapy),
1L/2L/3L hepatocellular carcinoma,
2L NSCLC
NK/T-cell lymphoma Phase II
www.beigene.com
B-cell malignancies, solid tumors Phase I
www.beigene.com
tisotumab vedotin Seagen platinum-resistant ovarian cancer, Phase II
(antibody-drug conjugate) Bothell, WA 1L/2L cervical cancer, www.seagen.com
recurrent metastatic cervical cancer,
metastatic solid tumors
tivozanib AVEO Oncology renal cell carcinoma application submitted
(VEGF/TKI inhibitor) www.aveooncology.com
renal cell carcinoma (combination Phase I/II
therapy), liver cancer (combination www.aveooncology.com
therapy)
Medicines in Development: Cancer ǀ 2020 199
Product Name Sponsor Indication Development Status
TJC4 (TJ011133) I-MAB Biopharma lymphoma, solid tumors Phase I
(anti-CD47 mAb) Gaithersburg, MD www.i-mabbiopharma.com
TJ4309 (TJ00439) I-MAB Biopharma solid tumors Phase I
(anti-CD72 mAb) Gaithersburg, MD www.i-mabbiopharma.com
TRACON Pharmaceuticals www.traconpharma.com
San Diego, CA
TK216 Oncternal Therapeutics Ewing's sarcoma (Fast Track) Phase I
(proto oncogene protein c-ets/ San Diego, CA www.oncternal.com
c-fli-1 inhibitor)
ORPHAN DRUG
TLC178 Taiwan Liposome cancer Phase I
(vinorelbine liposomal) San Francisco, CA www.tlcbio.com
TLPLDC Elios Therapeutics melanoma Phase II
(autologous dendritic cell vaccine) Austin, TX www.eliostherapeutics.com
TNB-383B AbbVie multiple myeloma Phase I
(BCMA x CD3 bispecific antibody) North Chicago, IL www.abbvie.com
ORPHAN DRUG Teneobio
Newark, CA
TNB-486 Teneobio B-cell lymphoma Phase I
(CD19xCD3 bispecific antibody) Newark, CA www.abbvie.com
Medicines in Development: Cancer ǀ 2020 200
Product Name Sponsor Indication Development Status
TnMUC-1 CAR-T cell therapy Tmunity Therapeutics TnMUC-positive solid tumors Phase I
Philadelphia, PA www.tmmunity.com
TNO155 Novartis solid tumors (monotherapy and Phase I
(SHP2 inhibitor) East Hanover, NJ combination therapy) www.novartis.com
tomivosertib (eFT-508) Effector Therapeutics solid tumors (combination therapy) Phase II
(MNK1/2 inhibitor) San Diego, CA www.effector.com
advanced breast cancer Phase I
(combination therapy) www.effector.com
TOOKAD® Steba Biotech intermediate-risk prostate cancer Phase III
padeliporfin Luxembourg City, Luxembourg www.stebabiotech.com
Tozaride® Andarix Pharmaceuticals solid tumors Phase I/II
Rhenium-188 P2046 Somerville, MA www.andarix.com
TP-0184 Sumitomo Dainippon advanced solid tumors Phase I
(ACVR1 inhibitor) Pharma Oncology www.sdppncology.com
Cambridge, MA
TP-1287 Sumitomo Dainippon advanced solid tumors Phase I
(CDK9 inhibitor) Pharma Oncology www.sdppncology.com
Cambridge, MA
Medicines in Development: Cancer ǀ 2020 201
Product Name Sponsor Indication Development Status
TP-1454 Sumitomo Dainippon advanced solid tumors Phase I
(PKM2 activator) Pharma Oncology www.sdppncology.com
Cambridge, MA
TP-3654 Sumitomo Dainippon myelofibrosis, Phase I
(PIM inhibitor) Pharma Oncology advanced solid tumors www.sdppncology.com
Cambridge, MA
TPIV100 Marker Therapeutics HER2-positive breast cancer Phase I/II
(therapeutic cancer vaccine) Houston, TX www.markertherapeutics.com
TPIV200 Marker Therapeutics triple negative breast cancer Phase II
(peptide dendritic vaccine) Houston, TX www.markertherapeutics.com
ORPHAN DRUG
TPST-1120 Tempest Therapeutics solid tumors Phase I
(PPARα antagonist) South San Francisco, CA www.tempesttx.com
TPST-1495 Tempest Therapeutics solid tumors Phase I
(EP4/EP2 dual antagonist) South San Francisco, CA www.tempesttx.com
TPX-0022 Turning Point Therapeutics MET-positive advanced solid tumors Phase I
(MET/CSF1R/SRC inhibitor) San Diego, CA www.tptherapeutics.com
TPX-0046 Turning Point Therapeutics RET-positive advanced solid tumors Phase I
(RET/SRC inhibitor) San Diego, CA www.tptherapeutics.com
Medicines in Development: Cancer ǀ 2020 202
Product Name Sponsor Indication Development Status
TR1801-ADC Tanabe Research Laboratories USA solid tumors Phase I
(antibody-drug conjugate) San Diego, CA www.trlusa.com
trastuzumab duocarmazine (SYD985) Byondis metastatic breast cancer (Fast Track) Phase III
(antibody-drug conjugate) Nijmegen, The Netherlands www.byondis.com
metastatic endometrial cancer Phase II
www.byondis.com
TRC102 TRACON Pharmaceuticals mesothelioma Phase II
(DNA repair inhibitor) San Diego, CA www.traconpharma.com
solid tumors Phase I
www.traconpharma.com
TRC253 TRACON Pharmaceuticals prostate cancer Phase II
(androgen receptor antagonist) San Diego, CA www.traconpharma.com
trilaciclib G1 Therapeutics 1L/2L/3L SCLC (combination therapy) application submitted
(CDK4/CDK6 inhibitor) Research Triangle Park, NC (Breakthrough Therapy) www.g1therapeutics.com
triple negative breast cancer Phase II
(combination therapy), neoadjuvant www.g1therapeutics.com
breast cancer
Medicines in Development: Cancer ǀ 2020 203
Product Name Sponsor Indication Development Status
TRK-950 Toray advanced solid tumors Phase I
(mAb) Tokyo, Japan www.toray.com
Trodelvy™ Immunomedics 3L metastatic urothelial cancer Phase III
sacituzumab govitecan-hziy Morris Plains, NJ (Fast Track), HR-positive www.immunomedics.com
HER2-negative metastatic breast
cancer
metastatic urothelial cancer Phase II
(combination therapy), www.immunomedics.com
1L metastatic triple negative breast
cancer (combination therapy),
head and neck cancer, metastatic
NSCLC (combination therapy), NSCLC
metastatic triple negative breast Phase I
cancer, metastatic urothelial www.immunomedics.com
cancer, ovarian cancer
TRPH-222 Bristol-Myers Squibb B-cell non-Hodgkin lymphoma Phase I
(CD22 antibody-drug conjugate) Princeton, NJ www.triphaseco.com
Triphase Accelerator
La Jolla, CA
TRS003 Teruisi colorectal cancer, NSCLC Phase I
(bevacizumab biosimilar) Zhejiang, China www.teruisipharm.com
Medicines in Development: Cancer ǀ 2020 204
Product Name Sponsor Indication Development Status
TSC Diffusion Pharmaceuticals glioblastoma Phase III
(trans sodium crocetinate) Charlottesville, VA www.diffusionpharma.com
ORPHAN DRUG
brain metastases, pancreatic cancer Phase I
www.diffusionpharma.com
TSD-001 LIPAC Oncology non-muscle invasive bladder caner Phase I/II
(proliposomal intravesical paclitaxel) Pomona, CA www.lipaconcology.com
TST001 Transcenta solid tumors Phase I
(CLDN18.2 mAb) Suzhou, China www.transcenta.com
TT-00420 TransThera Biosciences solid tumors Phase I
(multi kinase inhibitor) Nanjing, China www.transtherabio.com
TTAC-0001 PharmAbcine recurrent glioblastoma Phase II
(anti-VEGFR2 mAb) Daejeon, South Korea www.pharmabcine.com
ORPHAN DRUG
TTC-352 TTC Oncology metastatic ER-positive breast cancer Phase I
(estrogen receptor alpha agonist) Minneapolis, MN www.ttconcology.com
TTI-101 Tvardi Therapeutics advanced solid tumors Phase I
(STAT3 inhibitor) Houston, TX www.tvarditherapeutics.com
Medicines in Development: Cancer ǀ 2020 205
Product Name Sponsor Indication Development Status
TTI-621 Trillium Therapeutics hematologic malignancies, Phase I
(CD47 inhibitor) Cambridge, MA solid tumors www.trilliumtherapeutics.com
TTI-622 Trillium Therapeutics hematologic malignancies Phase I
(CD47 inhibitor) Cambridge, MA www.trilliumtherapeutics.com
TTX-030 AbbVie advanced lymphoma, Phase I
(anti-CD39 mAb) North Chicago, IL advanced solid tumors www.abbvie.com
Tizona Therapeutics
South San Francisco, CA
TTX-080 Tizona Therapeutics advanced solid tumors Phase I
(HLA-G inhibitor) South San Francisco, CA www.tizonatx.com
tucidinostat (HBI-8000) HUYA Bioscience melanoma, NSCLC, renal cell Phase I/II
(HDAC inhibitor) San Diego, CA carcinoma www.huyabio.com
Tukysa® Merck HER2-positive metastatic breast Phase III
tucatinib Kenilworth, NJ cancer (combination therapy) www.seagen.com
ORPHAN DRUG Seagen
Bothell, WA
HER2-positive colorectal cancer Phase II
(combination therapy) www.seagen.com
1L HER2-positive gastric cancer Phase I
www.seagen.com
Medicines in Development: Cancer ǀ 2020 206
Product Name Sponsor Indication Development Status
TVI-Brain-1 TVAX Biomedical glioblastoma (Fast Track) Phase II
(personalized cancer cell vaccine Olathe, KS www.tvaxbiomedical.com
and T-cell therapy)
ORPHAN DRUG
U3-1402 Daiichi Sankyo HER3-positive colorectal cancer Phase II
(anti-HER3 antibody-drug conjugate) Basking Ridge, NJ www.dsi.com
HER3-positive breast cancer, Phase I
NSCLC www.dsi.com
ublituximab TG Therapeutics high-risk CLL (combination therapy) application submitted
(anti-CD20 mAb) New York, NY www.tgtherapeutics.com
ORPHAN DRUG
UCART 123 Cellectis AML Phase I
(CAR-T cell therapy) New York, NY www.cellectis.com
UCART22 Cellectis B-cell ALL Phase I
(CAR-T cell therapy) New York, NY www.cellectis.com
UCARTCS1 Cellectis multiple myeloma Phase I
(CAR-T cell therapy) New York, NY www.cellectis.com
Medicines in Development: Cancer ǀ 2020 207
Product Name Sponsor Indication Development Status
umbralisib TG Therapeutics marginal zone lymphoma application submitted
(PI3Kδ/CK1 inhibitor) New York, NY (Breakthrough Therapy), www.tgtherapeutics.com
ORPHAN DRUG follicular lymphoma
1L CLL (combination therapy) Phase III
www.tgtherapeutics.com
CLL (combination therapy) Phase II
NHL (combination therapy) www.tgtherapeutics.com
uproleselan (GMI-1271) GlycoMimetics AML (Breakthrough Therapy) Phase III
(E-selectin inhibitor) Rockville, MD (Fast Track) www.glycomimetics.com
ORPHAN DRUG
newly-diagnosed AML Phase II
www.glycomimetics.com
UV1 vaccine Ultimovacs metastatic melanoma Phase II
(hTERT peptide vaccine) Oslo, Norway (+nivolumab & ipilimumab) www.ultimovacs.com
metastatic melanoma Phase I
(+pembrolizumab) www.ultimovacs.com
V937 Merck melanoma, cutaneous squamous Phase II
(oncolytic virus vaccine) Kenilworth, NJ cell carcinoma, breast cancer, www.merck.com
ORPHAN DRUG head and neck cancer
Medicines in Development: Cancer ǀ 2020 208
Product Name Sponsor Indication Development Status
V938 Merck advanced metastatic or recurrent Phase I
Kenilworth, NJ malignancies (+pembrolizumab) www.merck.com
VAC2 Lineage Cell Therapeutics NSCLC Phase I
(dendritic cell vaccine) Carlsbad, CA www.lineagecell.com
Cancer Research UK
London, United Kingdom
vactosertib (TEW-7197) Medpacto urothelial cancer Phase II
(TGF-β antagonist) Seoul, South Korea www.medpacto.com
MDS Phase I/II
www.medpacto.com
VAL-083 Kintara Therapeutics glioblastoma (Fast Track) Phase II
(chemotherapeutic) San Diego, CA www.kintara.com
ORPHAN DRUG
VAX014 Vaxiion Therapeutics non-muscle invasive bladder cancer Phase I
(bacterial mini-cell recombinant San Diego, CA www.vaxiion.com
non-antibody)
VAY736 (ianalumab + ibrutinib) Novartis hematologic malignancies Phase I
(BAFF-R inhibitor/BTK inhibitor) East Hanover, NJ www.novartis.com
Medicines in Development: Cancer ǀ 2020 209
Product Name Sponsor Indication Development Status
VBI-1901 VBI Vaccines glioblastoma Phase II
(enveloped virus-like particle vaccine) Cambridge, MA www.vbivaccines.com
VE800 Vedanta Biosciences solid tumors Phase I
(live biotherapeutic product) Cambridge, MA www.vedantabio.com
vecabrutinib (SNS-062) Sunesis Pharmaceuticals B-cell malignancies Phase I/II
(BTK inhibitor) South San Francisco, CA www.sunesis.com
veliparib (ABT-888) AbbVie BRCA breast cancer, NSCLC Phase III
(PARP inhibitor) North Chicago, IL ovarian cancer www.abbvie.com
ORPHAN DRUG
Venclexta® AbbVie MDS, multiple myeloma Phase III
venetoclax North Chicago, IL www.abbvie.com
Genentech www.gene.com
South San Francisco, CA
1L MDS (combination therapy), Phase II
2L HR-positive breast cancer www.abbvie.com
(combination therapy), multiple www.gene.com
myeloma (combination therapy)
ALL, AML, MDS, hematologic Phase I
malignancies (pediatric), solid tumors www.abbvie.com
www.gene.com
Medicines in Development: Cancer ǀ 2020 210
Product Name Sponsor Indication Development Status
VERU-111 Veru metastatic castration resistant Phase I/II
(tubulin polymerization inhibitor) Miami, FL prostate cancer www.verupharma.com
Verzenio® Eli Lilly breast cancer (adjuvant therapy) Phase III
abemaciclib Indianapolis, IN www.lilly.com
prostate cancer Phase II
www.lilly.com
viagenpumatucel-L HS-110) Heat Biologics NSCLC Phase II
(allogeneic tumor cell vaccine) Morrisville, NC www.heatbio.com
VIC-1911 VITRAC Therapeutics solid tumors Phase I
(aurora A kinase inhibitor) Boston, MA www.vitractherapeutics.com
Vicineum™ Sesen Bio BCG-unresponsive high-risk application submitted
oportuzumab monatox Cambridge, MA non-muscle invasive bladder cancer www.sesenbio.com
ORPHAN DRUG (Fast Track)
head and neck squamous cell Phase II
carcinoma (Fast Track) www.sesenbio.com
BCG-unresponsive high-risk Phase I
non-muscle invasive bladder www.sesenbio.com
cancer (+durvalumab)
Medicines in Development: Cancer ǀ 2020 211
Product Name Sponsor Indication Development Status
Vigil™ Gradalis Ewing's sarcoma Phase III
(genetically-modified autologous Dallas, TX www.gradalisinc.com
tumor cell vaccine and bi-shRNAfurin)
melanoma, ovarian cancer, Phase II
gynecologic cancer www.gradalisinc.com
ovarian cancer (IO combination), Phase I
breast cancer (IO combination) www.gradalisinc.com
VIMO-001 (alpha-TEA) Veana Therapeutics breast cancer Phase I
(alpha-tocopheroloxyacetic acid) Portland, OR www.veana-therapeutics.com
VLS-101 VelosBio hematologic malignancies Phase I
(antibody-drug conjugate) San Diego, CA www.velosbio.com
ORPHAN DRUG
VMD-928 VM Oncology lymphoma, solid tumors Phase I
(TrkA antagonist) Freemont, CA www.vmdiscovery.com
VOB560 Novartis cancer Phase I
(Bcl-2 inhibitor) East Hanover, NJ www.novartis.com
vorasidenib (AG-881) Agios Pharmaceuticals low-grade glioma Phase III
(pan-IDHm inhibitor) Cambridge, MA www.agiosbio.com
Medicines in Development: Cancer ǀ 2020 212
Product Name Sponsor Indication Development Status
vorolanib Xcovery renal cell carcinoma Phase III
(PDGFR/VEGF inhibitor) Palm Beach Gardens, FL www.xcovery.com
melanoma, solid tumors Phase II
www.xcovery.com
voruciclib MEI Pharma AML, B-cell malignancies Phase I
(CDK-9 inhibitor) San Diego, CA www.meipharma.com
Voyager-V1 Vyriad metastatic colorectal cancer Phase II
(genetically-modified vesicular Rochester, MN (combination therapy), www.vyriad.com
stomatitis virus) head and neck cancer
(combination therapy)
solid tumors Phase I
www.vyriad.com
VPM087 (gevokizumab) Novartis 1L colorectal cancer, 1L renal cell Phase I
(IL-1b antagonist) East Hanover, NJ carcinoma www.novartis.com
VS-6766 Verastem Oncology KRAS-mutant NSCLC, Phase II
(RAF/MEK inhibitor) Boston, MA low-grade serous ovarian cancer www.verastem.com
VT1021 Vigeo Therapeutics advanced solid tumors Phase I
(Tsp-1 agonist) Cambridge, MA www.vigeotherapeutics.com
Medicines in Development: Cancer ǀ 2020 213
Product Name Sponsor Indication Development Status
VTR-297 Vanda Pharmaceuticals hematologic malignancies Phase I
(HDAC inhibitor) Washington, DC www.vandapharma.com
Vyxeos® Jazz Pharmaceuticals untreated AML Phase I
daunorubicin and cytarabine Palo Alto, CA www.jazzpharma.com
WNT974 Novartis solid tumors Phase I
(porcupine inhibitor) East Hanover, NJ www.novartis.com
WP1066 Moleculin Biotech glioblastoma, pediatric brain tumors Phase I
(HIF-1/c-Myc inhibitor) Houston, TX www.moleculin.com
ORPHAN DRUG
WP1220 Moleculin Biotech cutaneous T-cell lymphoma Phase I
(STAT3 inhibitor) Houston, TX www.moleculin.com
WVT078 Novartis multiple myeloma Phase I
East Hanover, NJ www.novartis.com
Xalkori® Pfizer systemic ALK-positive anaplastic application submitted
crizotinib New York, NY large cell lymphoma (pediatric) www.pfizer.com
(Breakthrough Therapy)
Medicines in Development: Cancer ǀ 2020 214
Product Name Sponsor Indication Development Status
xentuzumab (BI 836845) Boehringer Ingelheim HR-positive HER2-negative Phase II
(IGF-1/2 inhibitor) Ridgefield, CT metastatic breast cancer www.boehinger-ingelheim.com
breast cancer, NSCLC Phase I
www.boehringer-ingelheim.com
Xermelo® TerSera Therapeutics biliary tract cancer Phase II
telotristat etiprate Lake Forest, IL www.tersera.com
XL092 Exelixis solid tumors Phase I
(TK inhibitor) Alameda, CA www.exelixis.com
XL888 Exelixis advanced gastrointestinal cancer Phase I
(HSP90 inhibitor) Alameda, CA www.exelixis.com
XmAb13676 (plamotamab) Xencor B-cell malignancies Phase I
(CD20xCD3 bispecific antibody) Monrovia, CA www.xencor.com
XmAb-14045 (vibecotamab) Xencor AML, hematologic malignancies Phase I
(CD123xCD3 bispecific antibody) Monrovia, CA www.xencor.com
XmAb20717 Xencor solid tumors Phase I
(PD-1xCTLA4 bispecific antibody) Monrovia, CA www.xencor.com
XmAb22841 Xencor solid tumors Phase I
(CTLA4xLAG3 bispecific antibody) Monrovia, CA www.xencor.com
Medicines in Development: Cancer ǀ 2020 215
Product Name Sponsor Indication Development Status
XmAb23104 Xencor solid tumors Phase I
(PD-1xICOS bispecific antibody) Monrovia, CA www.xencor.com
XmAB24306 (RG6323) Roche/Genentech solid tumors Phase I
(IL15xIL15Ra-Fc bispecific antibody) South San Francisco, CA www.xencor.com
Xencor
Monrovia, CA
XMT-1536 Mersana Therapeutics NSCLC, ovarian cancer (Fast Track) Phase I
(antibody-drug conjugate) Cambridge, MA www.mersana.com
XMT-1592 Mersana Therapeutics NSCLC, ovarian cancer Phase I
(antibody-drug conjugate) Cambridge, MA www.mersana.com
Xofigo® Bayer Pharmaceuticals NSCLC with bone metastases Phase I/II
radium Ra 223 dichloride Whippany, NJ (+pembrolizumab) www.pharma.bayer.com
Xospata® Astellas Pharma US post-chemotherapy maintenance Phase III
gilteritinib Northbrook, IL AML, post-hematopoietic stem cell www.astellas.com
ORPHAN DRUG transplant maintenance AML,
newly-diagnosed AML with high and
low intensity induction of
chemotherapy, AML (pediatric)
Medicines in Development: Cancer ǀ 2020 216
Product Name Sponsor Indication Development Status
Xpovio® Karyopharm Therapeutics relapsed/refractory multiple myeloma, Phase III
selinexor Newton, MA DLBCL, liposarcoma, endometrial www.karyopharm.com
ORPHAN DRUG cancer
glioblastoma, myelofibrosis, Phase II
1L multiple myeloma www.karyopharm.com
glioblastoma (combination therapy), Phase I
colorectal cancer, myelofibrosis, www.karyopharm.com
(combination therapy), DLBCL
(combination therapy)
Xtandi® Astellas Pharma non-metastatic high-risk castration Phase III
enzalutamide Northbrook, IL sensitive prostate cancer www.astellas.com
Pfizer www.pfizer.com
New York, NY
Yeliva® RedHill Biopharma cholangiocarcinoma Phase II
opaganib Raleigh, NC www.redhillbio.com
ORPHAN DRUG
YE-NEO-001 NantBioScience solid tumors Phase I
(NANT neoepitope yeast vaccine) Culver City, CA www.nantworks.com
Medicines in Development: Cancer ǀ 2020 217
Product Name Sponsor Indication Development Status
Yescarta® Gilead Sciences 2L BLBCL Phase III
axicabtagene ciloleucel Foster City, CA www.gilead.com
Kite Pharma
Santa Monica, CA
indolent non-Hodgkin lymphoma, Phase II
1L DLBCL, 3L DLBCL (combination www.gilead.com
therapy)
3L DLBCL (combination therapy) Phase I
www.gilead.com
YIV-906 Yiviva advanced hepatocellular carcinoma Phase II
(Chinese herbal botanical New York, NY www.yiviva.com
formulation)
ORPHAN DRUG
YTB323 Novartis hematologic malignancies Phase I
(CD19 CAR-T cell therapy) East Hanover, NJ (mono & combination therapy) www.novartis.com
zalifrelimab (AGEN1884) Agenus advanced or refractory solid tumors, Phase I/II
(anti-CTLA-4 antibody) Lexington, MA cervical cancer www.agenusbio.com
Medicines in Development: Cancer ǀ 2020 218
Product Name Sponsor Indication Development Status
zanidatamab (ZW25) Zymeworks HER2-positive/HR-positive advanced Phase II
(HER2 x HER2 bispecific antibody) Seattle, WA breast cancer, HER2-expressing www.zymeworks.com
ORPHAN DRUG gastroesophageal adenocarcinoma
(Fast Track), HER2-amplified biliary
tract cancer (Breakthrough Therapy)
(Fast Track)
HER2-expressing cancers Phase I
www.zymeworks.com
Zejula® GlaxoSmithKline 1L maintenance ovarian cancer Phase III
niraparib Research triangle Park, NC (combination therapy) www.gsk.com
ZEN-3694 Zenith Epigenetics prostate cancer (combination therapy) Phase II
(BET inhibitor) San Francisco, CA triple negative breast cancer www.zenithepigenetics.com
(combination therapy)
Zepzelca™ PharmaMar relapsed SCLC Phase III
lurbinectedin Madrid, Spain www.pharmar.com
Jazz Pharmaceuticals www.jazzphama.com
Palo Alto, CA
ZL-1201 ZAI Lab advanced solid tumors Phase I
(anti-CD47 antibody) Shanghai, China www.zailaboratory.com
ZN-c3 Zentalis Pharmaceuticals solid tumors Phase I/II
(WEE1 inhibitor) New York, NY www.zentalis.com
Medicines in Development: Cancer ǀ 2020 219
Product Name Sponsor Indication Development Status
ZN-c5 Zentalis Pharmaceuticals breast cancer Phase I/II
(SERD) New York, NY www.zentalis.com
ZN-e4 Zentalis Pharmaceuticals NSCLC Phase I/II
(EGFR antagonist) New York, NY www.zentalis.com
zolbetuximab Astellas Pharma gastric/gastroesophageal junction Phase III
(anti-claudin 18.2 mAb) Northbrook, IL adenocarcinoma www.astellas.com
ORPHAN DRUG
pancreatic adenocarcinoma Phase II
www.astellas.com
zotiraciclib (TG02) Adastra Pharmaceuticals anaplastic astrocytoma, Phase I
(multi kinase inhibitor) Princeton, NJ recurrent glioblastoma www.adastrarx.com
ORPHAN DRUG
ZW49 Zymeworks HER2-expressing tumors Phase I
(HER2 x HER2 bispecific antibody) Seattle, WA www.zymeworks.com
The content of this report has been obtained through public, government and industry sources, and the Springer "Adis Insight” database based on the latest
information. Report current as of December 4, 2020. 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. Some products may not be in active clinical trials. 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: www.phrma.org.
Medicines in Development: Cancer ǀ 2020 220
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, aloneor 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 maydemonstrate substantial improvement over existing therapies on one or more clinically-significant endpoints, such as substantial treatment effects observed early inclinical 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 usually fewer than 200,000 people in the United States.
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.
Medicines in Development: Cancer ǀ 2020 221
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