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2020 Medicines in Development ꟷ

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 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 Venture US metastatic , Phase II (PARP inhibitor) Scottsdale, AZ advanced 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 , 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 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 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 Phase I/II (genetically-engineered cell therapy San Diego, CA (+high dose and www.angiocrinebioscience.com derived from human umbilical vein autologous stem cell transplantation), tissue) , 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 ) 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..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 (AML), Phase I (surviving TCR/CD3 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 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

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 inhibitor) San Diego, CA www.sorrentotherapeutics.com

ABL001 (asciminib) 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 ( biosimilar) North Chicago, IL non- (NHL) www.abbvie.com 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 DRUG relapsed/refractory AML Phase I (combination therapy) www.actinumpharma.com

Actimab-M (CD33) Actinium Pharmaceuticals 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 Phase I/II (purine nucleoside phosphorylase Birmingham, AL www.pnptherapeutics.com therapy) adagloxad simolenin OBI Pharma triple negative breast cancer Phase III (cancer ) 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..com

Adcetris® Seagen DLBCL (combination therapy) Phase III 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), , 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 () 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 (+-rwlc) Phase II (DNA-based in vivo gene therapy) Boston, MA (Fast Track) www.ziopharm.com ORPHAN DRUG

glioblastoma (monotherapy), Phase I glioblastoma (+), www.ziopharm.com pediatric brain tumors, diffuse intrinsic pontine (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 (+) 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, 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/ ALK-positive NSCLC (adjuvant) Phase III South San Francisco, CA www.roche.com

Aliqopa™ Pharmaceuticals NHL (combination therapy) Phase III 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 (-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 , WA www.alpineimmunesciences.com dual )

Medicines in Development: Cancer ǀ 2020 14 Product Name Sponsor Indication Development Status

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 inhibitor) Burlingame, CA www.alxoncology.com

lymphoma, solid tumors Phase I www.alxoncology.com

(MORAb-009) Eisai 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 ) 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 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/ 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 ) 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) , 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 ( 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 ( 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 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 (/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 (-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 () 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 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 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 Raritan, NJ tumor agnostic (FGFR gene alteration) www.janssen.com

Bavencio® EMD Serono 1L NSCLC Phase III Rockland, MA www.emdserono.com 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

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 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 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 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 ( stimulant) San Francisco, CA NSCLC (+pembrolizumab) www.nektar.com berubicin CNS Pharmaceuticals glioblastoma Phase I (type II DNA ) 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) Oslo, Norway Track), NHL www.nordicnanovector.com satetraxetan ORPHAN DRUG

BGB-10188 BeiGene RAF-mutant solid tumors Phase I (PI3K) Cambridge, MA www..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 implant Alessa Therapeutics prostate cancer Phase I ( 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-) 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 ) 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 Research Triangle Park, NC www.gsk.com

Blincyto® Amgen ALL (pediatric) Phase III 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 + 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 Phase III 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 (-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 ( 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 Alameda, CA prostate cancer (combination therapy), www.exelixis.com ORPHAN DRUG 1L renal cell carcinoma (combination therapy) (Breakthrough Therapy), (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 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 (ADCT-301) ADC Therapeutics Hodgkin lymphoma Phase II (antibody-drug conjugate) Lausanne, Switzerland www.adctherapeutics.com Copenhagen, Denmark solid tumors Phase I www.adctherapeutics.com

camsirubicin (MNPR-201) Monopar Therapeutics soft tissue sarcoma Phase II ( 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 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 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 + ) 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 (+) 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 (+), Phase I (A2AR inhibitor) Burlingame, CA renal cell carcinoma (+), 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

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 DRUG head 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 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 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 DRUG colon cancer risk reduction Phase III www.canprevent.com

CPX-POM CicloMed solid tumors Phase I (mitosis inhibitor) Kansas City, MO www.ciclomed.com 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 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 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 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-.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 ORPHAN DRUG doranidazole Shuttle Pharmaceuticals pancreatic cancer Phase I (cell wall inhibitor) Rockville, MD www.shuttlepharma.com 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- 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 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) 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- 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) 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 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 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 DRUG post-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 (+) 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 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 ) 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- antibody, Cambridge, MA www.fujifilmpharma.com 90Y conjugate) 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 ( biosimilar) Cambridge, United Kingdom www.centusbiotherapeutics.com 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 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 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 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 Eleison Pharmaceuticals pancreatic cancer (Fast Track) Phase III ( conjugate of ifosfamide) Princeton, NJ www.eleison-pharma.com ORPHAN DRUG

GMI-1359 GlycoMimetics HR-positive metastatic breast cancer Phase I (E-/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 ( 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 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 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- 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 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 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 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 (-like-growth-factor St. Paul, MN www.igfoncology.com -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 (/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 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 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 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

IMGN632 ImmunoGen ALL, AML, blastic plasmacytoid Phase I/II (CD123 antibody drug conjugate) Waltham, MA dendritic cell (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 (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 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 , 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 ( antagonist) Rockville, MD www.innate-pharma.com

Medicines in Development: Cancer ǀ 2020 101 Product Name Sponsor Indication Development Status

IPI-549 () 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 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 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 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 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 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 + 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 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 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..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 DRUG aggressive 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 (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 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 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 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 () Princeton, NJ www.bms.com ORPHAN DRUG mavorixafor X4 Pharmaceuticals renal cell carcinoma (+) 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 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 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 (+ ) 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 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 Sierra Oncology myelofibrosis (Fast Track) Phase III (JAK1/JAK2/ACVR1 inhibitor) Vancouver, Canada www.sierraoncology.com ORPHAN DRUG

Monjuvi® Incyte CLL/SLL Phase II -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 DRUG neuroendocrine 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 , 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 ( 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 Los Angeles, CA cancer (monotherapy and www.pumabiotechnology.com combination therapy), HER2-mutant breast cancer nerofe 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 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 AstraZeneca EGFR mutant NSCLC (+) 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 (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 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 + 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 + 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 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 ) 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 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 -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 (RG-3019) Fibrogen pancreatic cancer (combination Phase III (anti-CTGF antibody) San Francisco, CA therapy (Fast Track) www.fibrogen.com ORPHAN DRUG (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 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..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 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 -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 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 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 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 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 () 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 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 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 Cyclacel Pharmaceuticals 1L AML (70 years and older) Phase III ( 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 (+) 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 -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 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 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 vaccine) San Diego, CA www.polynoma.com ORPHAN DRUG (INO-464) Innocrin Pharmaceuticals breast cancer (Fast Track), castration- Phase II (CYP17 / 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 (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 DRUG prostate 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 Amgen NSCLC Phase III (KRAS inhibitor) Thousand Oaks, CA www.amgen.com ORPHAN DRUG

advanced colorectal cancer, Phase II solid tumors www.amgen.com (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 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 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 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 ( 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 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 Odonate Therapeutics HER2-negative, HR-positive metastatic Phase III ( 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 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 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 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 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 ( 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 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 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 -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 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 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 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 (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 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 () Phase III 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 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 and 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 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 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 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 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 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, alone or in combination with one or more other drugs, to treat a serious or life threatening disease or condition and preliminary clinical evidence indicates that it may demonstrate substantial improvement over existing therapies on one or more clinically-significant endpoints, such as substantial treatment effects observed early in clinical development. If a drug or biologic is designated as a Breakthrough Therapy, the FDA will expedite the development and review. With this designation, all Fast Track features convey to the medicine.

Fast Track—Upon request by a sponsor, the FDA can grant this designation to facilitate the development and expedite the review of a drug or biologic to treat a serious condition and fill an unmet medical need. When considering a biopharmaceutical company’s request for Fast Track designation for an investigational drug or biologic, the FDA evaluates whether it will affect factors such as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one, and whether a condition can be adequately addressed by available therapy. With Fast Track designation, early and frequent communication between the FDA and the biopharmaceutical company is encouraged throughout the entire drug development and review process to help to quickly resolve any questions or issues that arise, potentially leading to an earlier approval and access by patients.

Orphan Designation—Upon request by a sponsor, the FDA can grant special status (“orphan status”) to a drug or biologic to treat a rare disease or condition. In order to receive an orphan designation, a qualifying drug or biologic must be intended for the treatment, diagnosis, or prevention of a rare disease or condition that affects 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.

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