2017 Immuno-Oncology Medicines in Development

Adoptive Cell Therapies Drug Name Organization Indication Development Phase

ACTR087 + Unum Therapeutics B-cell lymphoma Phase I (antibody-coupled T-cell receptor Cambridge, MA www.unumrx.com + rituximab)

AFP TCR Adaptimmune liver Phase I (T-cell receptor cell therapy) Philadelphia, PA www.adaptimmune.com anti-BCMA CAR-T cell therapy Juno Therapeutics multiple myeloma Phase I Seattle, WA www.junotherapeutics.com Memorial Sloan Kettering New York, NY anti-CD19 "armored" CAR-T Juno Therapeutics recurrent/relapsed chronic Phase I cell therapy Seattle, WA lymphocytic leukemia (CLL) www.junotherapeutics.com Memorial Sloan Kettering New York, NY anti-CD19 CAR-T cell therapy Intrexon B-cell malignancies Phase I Germantown, MD www.dna.com ZIOPHARM Oncology www.ziopharm.com Boston, MA anti-CD19 CAR-T cell therapy Kite Pharma hematological malignancies Phase I (second generation) Santa Monica, CA www.kitepharma.com National Institute Bethesda, MD

Medicines in Development: Immuno-Oncology 1 Adoptive Cell Therapies Drug Name Organization Indication Development Phase anti-CEA CAR-T therapy Sorrento Therapeutics liver metastases Phase I San Diego, CA www.sorrentotherapeutics.com TNK Therapeutics San Diego, CA anti-PSMA CAR-T cell therapy TNK Therapeutics cancer Phase I San Diego, CA www.sorrentotherapeutics.com Sorrento Therapeutics San Diego, CA

ATA520 Atara Biotherapeutics multiple myeloma, Phase I (WT1-specific T lymphocyte South San Francisco, CA plasma cell leukemia www.atarabio.com cell therapy)

AU101 Aurora BioPharma recurrent glioblastoma, sarcoma, Phase I/II (HER2-CD28 CAR-T cell therapy) Cambridge, MA osteosarcoma www.aurora-biopharma.com

AU105 Aurora BioPharma newly-diagnosed glioblastoma Phase I/II (HER2-CMV CAR-T cell therapy) Cambridge, MA www.aurora-biopharma.com

Medicines in Development: Immuno-Oncology 2 Adoptive Cell Therapies Drug Name Organization Indication Development Phase axicabtagene ciloleucel (KTE-C19) Kite Pharma aggressive non-Hodgkin lymphoma application submitted (CAR-T therapy) Santa Monica, CA www.kitepharma.com ORPHAN DRUG

mantle cell lymphoma, diffuse large Phase II/III B-cell lymphoma (DLBCL), primary www.kitepharma.com mediastinal B-cell lymphoma, transformed

acute lymphoblastic leukemia (ALL), Phase I DLBCL (with a PD-L1 mAb) www.kitepharma.com baltaleucel-T (CMD-003) Cell Medica extranodal natural killer T-cell lymphoma Phase II (T lymphocyte cell therapy) Houston, TX (Fast Track), DLBCL, Hodgkin lymphoma, www.celmedica.co.uk ORPHAN DRUG post-transplant lymphoproliferative disorder bb2121 bluebird bio multiple myeloma Phase I (anti-BCMA CAR-T cell therapy) Cambridge, MA www.bluebirdbio.com ORPHAN DRUG Celgene ww.celgene.com Summit, NJ

BPX-501 (rivogenlecleucel) Bellicum Pharmaceuticals haploidentical hematopoietic Phase I/II (T lymphocyte cell therapy) Houston, TX stem cell transplantation www.bellicum.com (adjunct therapy)

BPX-601 Bellicum Pharmaceuticals non-resectable pancreatic Phase I (GoCAR-T cell therapy) Houston, TX www.bellicum.com

Medicines in Development: Immuno-Oncology 3 Adoptive Cell Therapies Drug Name Organization Indication Development Phase

BPX-701 Bellicum Pharmaceuticals acute myeloid leukemia (AML), Phase I (T-cell receptor based therapy) Houston, TX myelodysplastic syndromes www.bellicum.com

BSK01™ Kiromic AML Phase I/II (cell-based immunotherapy) Houston, TX www.kiromic.com

IMCgp100 Immunocore ocular , cutaneous Phase II (T-cell receptor therapy) Conshohocken, PA melanoma (combination therapy) www.immunocore.com ORPHAN DRUG

JCAR014 Juno Therapeutics B-cell non-Hodgkin lymphoma Phase I (anti-CD19 CAR-T cell therapy) Seattle, WA (with ) www.junotherapeutics.com

JCAR017 Celgene B-cell non-Hodgkin lymphoma Phase I (anti-CD19 CAR-T cell therapy) Summit, NJ () www.celgene.com Juno Therapeutics www.junotherapeutics.com Seattle, WA

JCAR018 Juno Therapeutics ALL (pediatric), non-Hodgkin lymphoma Phase I (anti-CD22 CAR-T cell therapy) Seattle, WA www.junotherapeutics.com

JCAR020 Juno Therapeutics ovarian Phase I (MUC16 CAR-T cell therapy) Seattle, WA www.junotherapeutics.com

JCAR023 Juno Therapeutics neuroblastoma (pediatric) Phase I (anti-CD171 CAR-T cell therapy) Seattle, WA www.junotherapeutics.com

Medicines in Development: Immuno-Oncology 4 Adoptive Cell Therapies Drug Name Organization Indication Development Phase

JCAR024 Juno Therapeutics non-small cell (NSCLC), Phase I (ROR1-directed CAR-T cell therapy) Seattle, WA triple-negative breast www.junotherapeutics.com

JTCR016 Juno Therapeutics AML, chronic myelogenous leukemia (CML) Phase I/II (WT1-specific T lymphocyte therapy) Seattle, WA www.junotherapeutics.com

JTX-2011 Jounce Therapeutics solid tumors Phase II (inducible T-cell CO-stimulator Cambridge, MA www.jouncetx.com activator)

KITE-439 Kite Pharma cervical, head and neck, urogenital Phase I (HPV-16 E7 T-cell receptor therapy) Santa Monica, CA www.kitepharma.com National Cancer Institute Bethesda, MD

KITE- 718 Kite Pharma solid tumors Phase I (MAGE A3/A6 T-cell therapy) Santa Monica, CA www.kitepharma.com

LN-144 Lion Biotechnologies malignant melanoma Phase II (tumor infiltrating lymphocyte San Carlos, CA www.lbio.com therapy) ORPHAN DRUG

LN-145 Lion Biotechnologies cervical, head and neck Phase II (tumor infiltrating lymphocyte San Carlos, CA www.lbio.com therapy)

Medicines in Development: Immuno-Oncology 5 Adoptive Cell Therapies Drug Name Organization Indication Development Phase

MAGE-A10 TCR Adaptimmune bladder, head and neck, Phase I/II (T-cell receptor therapy) Philadelphia, PA melanoma, NSCLC www.adaptimmune.com

MB-101 Mustang Bio glioblastoma Phase I (anti-CD213a2 CAR-T cell therapy) New York, NY www.mustangbio.com

MB-102 Mustang Bio AML Phase I (anti-CD123 CAR-T cell therapy) New York, NY www.mustangbio.com

NKR-2 Celyad hematological malignancies, Phase I/II (CAR-T cell therapy) Boston, MA solid tumors www.celyad.com

NY-ESO-TCR Adaptimmune myxoid round cell liposarcoma, Phase I/II (T-cell receptor therapy) Philadelphia, PA melanoma, multiple myeloma, NSCLC, www.adaptimmune.com ORPHAN DRUG GlaxoSmithKline ovarian, synovial sarcoma Philadelphia, PA

NY-ESO-1 T-cell receptor therapy Kite Pharma solid tumors Phase II Santa Monica, CA www.kitepharma.com National Cancer Institute Bethesda, MD

PNK-007 Celgene AML, multiple myeloma Phase I (natural killer cell therapy) Summit, NJ www.celgene.com

Medicines in Development: Immuno-Oncology 6 Adoptive Cell Therapies Drug Name Organization Indication Development Phase tisagenlecleucel-T Novartis Pharmaceuticals relapsed or refractory acute application submitted (anti-CD19 CAR-T cell therapy) East Hanover, NJ lymphoblastic leukemia www.novartis.com (children and adolescents)

DLBCL Phase II (Breakthrough Therapy) www.novartis.com

TT12 Tessa Therapeutics cervical, oropharyngeal Phase I (HPV-specific T-cell therapy) Singapore www.tessatherapeutics.com

Bispecific Antibodies l Monoclonal Antibodies Drug Name Organization Indication Development Phase

ABBV-428 AbbVie solid tumors Phase I (CD40 ligand stimulant) North Chicago, IL www..com

AFM11 Affimed Therapeutics B-cell non-Hodgkin lymphoma Phase I (bispecific antibody) Heidelberg, Germany www.affimed.com

AFM13 Affimed Therapeutics Hodgkin lymphoma Phase I (CD30 modulator) Heidelberg, Germany www.affimed.com ORPHAN DRUG

AMG 211 (MEDI-565) solid tumors Phase I (anti-CEA/anti-CD3 bispecific Thousand Oaks, CA www.amgen.com antibody) MedImmune www.medimmune.com Gaithersburg, MD

Medicines in Development: Immuno-Oncology 7 Bispecific Antibodies l Monoclonal Antibodies Drug Name Organization Indication Development Phase

AMG 330 Amgen AML Phase I (anti-CD33/anti-CD3 bispecific Thousand Oaks, CA www.amgen.com antibody)

AMG 420 Amgen multiple myeloma Phase I (anti-BCMA/anti-CD3 bispecific Thousand Oaks, CA www.amgen.com antibody)

BI-836880 Boehringer Ingelheim Pharmaceuticals cancer Phase I (anti-VEGF/Ang2 inhibitor Ridgefield, CT www.boehringer-ingelheim.com bispecific nanobody)

Blincyto® Amgen relapsed/refractory Philadelphia Phase II Thousand Oaks, CA chromosome-positive (ph+) and minimal www.amgen.com ORPHAN DRUG residual of ALL, ph+ refractory/relapsed B-cell precursor ALL

DLBCL Phase II www.amgen.com duvortuxizumab (JNJ-64052781) Janssen Research & Development B-cell malignancies Phase I (CD19/CD3 bispecific antibody) Raritan, NJ www.janssen.com

ERY974 Chugai Pharma USA solid tumors Phase I (anti-glypican-3/CD3 bispecific Berkeley Heights, NJ www.chugai-pharma.com antibody)

Medicines in Development: Immuno-Oncology 8 Bispecific Antibodies l Monoclonal Antibodies Drug Name Organization Indication Development Phase (MGD06) MacroGenics AML, myelodysplastic syndromes Phase I (CD123/CD3 bispecific antibody) Rockville, MD www.macrogenics.com ORPHAN DRUG (MM-141) Merrimack Pharmaceuticals pancreatic Phase II (PI3K/AKT/mTOR inhibitor Cambridge, MA www.merrimack.com bispecific antibody)

JNJ-61186372 Janssen Research & Development NSCLC Phase I (EGFR/proto-oncogene protein Raritan, NJ www.janssen.com c-Met bispecific antibody)

JNJ-63709178 Janssen Research & Development AML Phase I (CD123/CD3 bispecific antibody) Raritan, NJ www.janssen.com

LY3164530 Eli Lilly cancer Phase I (EGFR/proto-oncogene protein Indianapolis, IN www.lilly.com c-Met bispecific antibody)

MGD007 MacroGenics metastatic colorectal Phase I (gpA33/CD3 bispecific antibody) Rockville, MD www.macrogenics.com

MGD009 MacroGenics solid tumors Phase I (CD3/B7H3 bispecific antibody) Rockville, MD www.macrogenics.com

Medicines in Development: Immuno-Oncology 9 Bispecific Antibodies l Monoclonal Antibodies Drug Name Organization Indication Development Phase

MOR209/ES414 Aptevo Therapeutics prostate Phase I (PSMA/CD3 bispecific antibody) Seattle, WA www.morphosys.com MorphoSys Planegg, Germany navicixizumab OncoMed Pharmaceuticals colorectal, ovarian Phase I (anti-DLL4/VEGF bispecific antibody) Redwood City, CA www.oncomed.com

OXS-1550 Oxis Biotech non-Hodgkin lymphoma Phase II (bispecific scFv recombinant fusion Tampa, FL www.oxis.com protein-drug conjugate)

PF-06671008 solid tumors Phase I (anti-CD3/P-cadherin bispecific New York, NY www.pfizer.com antibody)

REGN1979 Regeneron Pharmaceuticals cancer Phase I (CD20/CD3 bispecific antibody) Tarrytown, NY www.regeneron.com

RG7386 solid tumors Phase I (fibroblast-activating protein/TRAIL South San Francisco, CA www.gene.com receptor 2 agonist bispecific antibody)

RG7802 Genentech solid tumors Phase I (carcinoembryonic/CD3 antigen South San Francisco, CA www.gene.com inhibitor)

Medicines in Development: Immuno-Oncology 10 Bispecific Antibodies l Monoclonal Antibodies Drug Name Organization Indication Development Phase

RG7828 Genentech hematological malignancies Phase I (CD20/CD3 bispecific antibody) South San Francisco, CA www.gene.com

TF 2-targeted bispecific antibody Immunomedics HER2-negative CEA-expressing breast Phase I/II (TF2 and 68 Ga-IMP-288) Morris Plains, NJ www.immunomedics.com IBC Pharmaceuticals Morris Plains, NJ (RG7221) Genentech metastatic colorectal Phase II (-2 inhibitor/ South San Francisco, CA www.gene.com VEGF-A inhibitor)

XmAb13676 Xencor non-Hodgkin lymphoma Phase I (CD20/CD3 bispecific antibody) Monrovia, CA www.xencor.com

XmAb14045 Xencor AML Phase I (CD3 antigen modulator/IL-3 receptor Monrovia, CA www.xencor.com alpha subunit modulator)

ZW25 Zymeworks ovarian Phase I (two different epitopes of Vancouver, Canada www.zymeworks.com HER2 protein) ORPHAN DRUG

Medicines in Development: Immuno-Oncology 11 Checkpoint Modulators l Monoclonal Antibodies Drug Name Organization Indication Development Phase

AGEN 1884 Agenus solid tumors Phase I (CTLA-4 antagonist) Lexington, MA www.agenusbio.com

AGEN 2034 Agenus solid tumors Phase I/II (PD-1 protein inhibitor) Lexington, MA www.agenusbio.com anti-GITR antibody Bristol-Myers Squibb cancer Phase I (glucocorticoid-induced tumor Princeton, MA www.bms.com necrosis factor receptor) anti-LAG3 (BMS-986016) Bristol-Myers Squibb gastric, NSCLC, renal cell Phase II (CD223 antigen inhibitor) Princeton, NJ www.bms.com

Bavencio® EMD Serono gastric (1L, 3L, 1L maintenance), Phase III Rockland, MA head and neck, NSCLC (1L, 2L), ovarian (1L), www.emdserono.com (anti-PD-L1 mAb) Pfizer platinum-resistant/refractory ovarian, www.pfizer.com ORPHAN DRUG New York, NY renal cell (1L), urothelial (1L, 1L maintenance)

triple negative breast, head and neck, Phase I melanoma, NSCLC www.emdserono.com (with PF-05082566 for all) www.pfizer.com

hematological malignancies, Phase I solid tumors www.emdserono.com www.pfizer.com

Medicines in Development: Immuno-Oncology 12 Checkpoint Modulators l Monoclonal Antibodies Drug Name Organization Indication Development Phase

BGB-A317 BeiGene solid tumors Phase I (PD-1 inhibitor) Cambridge, MA www.beigene.com

BI-754091 Boehringer-Ingelheim Pharmaceuticals solid tumors Phase I (anti-PD-1 mAb) Ridgefield, CT www.boehriner-ingelheim.com

BMS-986178 Bristol-Myers Squibb solid tumors Phase I/II (anti-OX40 mAb) Princeton, NJ (combination therapy) www.bms.com

BMS-986192 Bristol-Myers Squibb NSCLC in clinical trials (anti-PD-L1 mAb) Princeton, NJ www.bms.com

CBT-501 (genolimzumab) CBT Pharmaceuticals solid tumors Phase I (PD-1 protein modulator) Santa Clara, CA www.cbtpharma.com

CX-072 CytomX Therapeutics lymphoma, solid tumors Phase I/II (PD-L1 inhibitor) South San Francisco, CA www.cytomx.com

FAZ053 Novartis Pharmaceuticals solid tumors Phase I (PD-L1 protein inhibitor) East Hanover, NJ www.novartis.com

GSK3174998 GlaxoSmithKline hematological malignancies, Phase I (anti-OX40 mAb) Philadelphia, PA solid tumors www.gsk.com

GWN323 Novartis Pharmaceuticals lymphoma, solid tumors Phase I (anti-GITR mAb) East Hanover, NJ www.novartis.com

Medicines in Development: Immuno-Oncology 13 Checkpoint Modulators l Monoclonal Antibodies Drug Name Organization Indication Development Phase

Imfinzi™ AstraZeneca advanced bladder (2L), Phase III durvalumab Wilmington, DE bladder (1L with ), www.medimmune.com (anti-PD-L1 antibody) head and neck, NSCLC (1L & 3L with tremelimumab), NSCLC stage III (monotherapy), small cell lung cancer (SCLC) (1L with tremelimumab), squamous cell head and neck (1L & 2L with tremelimumab)

solid tumors (monotherapy), squamous Phase II cell head and neck (with AZD5069), www.medimmune.com solid tumors (with MEDI0680), hepatocellular (with tremelimumab), gastric (with tremelimumab)

DLBCL (monotherapy and combination), Phase I melanoma (combination therapy), www.medimmune.com NSCLC (with ), solid tumors (in several combinations)

AstraZeneca acute myeloid leukemia, Phase II Wilmington, DE myelodysplastic syndromes www.celgene.com Celgene www.medimmune.com Summit, NJ

chronic lymphocytic leukemia, Phase I multiple myeloma, non-Hodgkin lymphoma www.celgene.com www.medimmune.com

IMP701 Novartis Pharmaceuticals cancer Phase I (anti-LAG3 mAb) East Hanover, NJ www.novartis.com

Medicines in Development: Immuno-Oncology 14 Checkpoint Modulators l Monoclonal Antibodies Drug Name Organization Indication Development Phase

INCAGN1876 Agenus solid tumors Phase I/II (GITR receptor agonist) Lexington, MA www.agenusbio.com Incyte www.incyte.com Wilmington, DE

INCAGN1949 Agenus solid tumors Phase I/II (OX40 receptor agonist) Lexington, MA www.agenusbio.com Incyte www.incyte.com Wilmington, DE

INCSHR1210 Incyte solid tumors Phase I/I (PD-1 protein inhibitor) Wilmington, DE www.incyte.com

JNJ-63723283 Janssen Research & Development solid tumors Phase I/II (PD-1 inhibitor) Raritan, NJ www.janssen.com

Keytruda® Merck previously treated microsatellite application submitted Kenilworth, NJ instability-high, relapsed or refractory www.merck.com (anti-PD-1 mAb) classical Hodgkin lymphoma, cisplatin- ineligible bladder (1L), metastatic bladder (2L), non-squamous NSCLC (1L, combination therapy)

breast, colorectal, esophageal, gastric, Phase III hepatocellular, multiple myeloma, renal www.merck.com

primary mediastinal large B-cell lymphoma, Phase II nasopharyngeal, ovarian prostate www.merck.com

Medicines in Development: Immuno-Oncology 15 Checkpoint Modulators l Monoclonal Antibodies Drug Name Organization Indication Development Phase

KN035 3D Medicine solid tumors Phase I (anti-PD-L1 antibody) Suzhou, China www.alphamab.com Alphamab Suzhou, China

LAG525 Novartis Pharmaceuticals solid tumors Phase I/II (anti-LAG3 mAb) East Hanover, NJ www.novartis.com Bristol-Myers Squibb acute myeloid leukemia, chronic Phase II (anti-KIR receptor antagonist) Princeton, NJ lymphocytic leukemia, myelodysplastic www.bms.com Innate Pharma syndromes (combination therapy) Marseille, France

solid tumors Phase I/II www.bms.com

LY3300054 Eli Lilly solid tumors Phase I (PD-L1 mAb) Indianapolis, IN www.lilly.com

M7824 EMD Serono solid tumors Phase I (anti-PD-L1/TGFbeta trap) Rockland, MA www.emdserono.com

MBG453 Novartis Pharmaceuticals solid tumors Phase I/II (anti-TIM3 antibody) East Hanover, NJ www.novartis.com

MEDI0562 MedImmune solid tumors Phase I (OX40 receptor agonist) Gaithersburg, MD www.medimmune.com

Medicines in Development: Immuno-Oncology 16 Checkpoint Modulators l Monoclonal Antibodies Drug Name Organization Indication Development Phase

MEDI0680 MedImmune solid tumors Phase I (PD-1 protein inhibitor) Gaithersburg, MD www.medImmune.com

MEDI1873 MedImmune solid tumors Phase I (GITR agonist ) Gaithersburg, MD www.medImmune.com

MGA 012 MacroGenics solid tumors Phase I (PD-1 inhibitor) Rockville, MD www.macrogenics.com

MK-4166 Merck solid tumors Phase I (GITR protein stimulant) Kenilworth, NJ www.merck.com

Opdivo® Bristol-Myers Squib mismatch repair deficient or application submitted Princeton, NJ microsatellite instability-positive www.bms.com ORPHAN DRUG colorectal

esophageal, gastro-esophageal junction, Phase III gastric, glioblastoma, hepatocellular, www.bms.com multiple myeloma, NSCLC (1L), renal cell (1L), SCLC

central nervous system , DLBCL, Phase II follicular lymphoma, hematological www.bms.com malignancies, testicular

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Medicines in Development: Immuno-Oncology 17 Checkpoint Modulators l Monoclonal Antibodies Drug Name Organization Indication Development Phase

Opdivo® Bristol-Myers Squib bladder (combination therapy), Phase I/II nivolumab Princeton, NJ colorectal (combination therapy), www.bms.com ORPHAN DRUG solid tumors (in several combinations)

CML (combination therapy), solid tumors Phase I (with ), solid tumors www.bms.com (with BMS-986207)

Opdivo® / Yervoy® Bristol-Myers Squib head and neck, malignant plural Phase III nivolumab/ Princeton, NJ , NSCLC, renal cell (1L), www.bms.com SCLC

PDR001 Novartis Pharmaceuticals malignant melanoma Phase III (anti-PD-1 antibody) East Hanover, NJ www.novartis.com

nasopharyngeal, neuroendocrine tumors Phase II www.novartis.com

solid tumors, lymphoma Phase I www.novartis.com

PF-04518600 Pfizer cancer (with avelumab) Phase I (OX40 receptor agonist) New York, NY www.pfizer.com

PF-06801591 Pfizer cancer Phase I (anti-PD-1 antibody) New York, NY www.pfizer.com

Medicines in Development: Immuno-Oncology 18 Checkpoint Modulators l Monoclonal Antibodies Drug Name Organization Indication Development Phase

REGN2810 (SAR439684) Regeneron Pharmaceuticals advanced cutaneous squamous cell Phase II (PD-1 inhibitor) Tarrytown, NY www.regeneron.com Sanofi US www.sanofi.com Bridgewater, NJ

cancer Phase I www.regeneron.com www.sanofi.com

REGN3767 Regeneron Pharmaceuticals cancer Phase I (LAG3 antibody) Tarrytown, NY www.regeneron.com

RG7888 Genentech solid tumors, urothelial Phase II (anti-OX40 antibody) South San Francisco, CA www.gene.com

solid tumors (with ) Phase I www.gene.com

Tecentriq® Genentech metastatic NSCLC Phase II atezolizumab South San Francisco, CA www.gene.com

melanoma (with ), Phase I solid tumors (monotherapy), www.gene.com solid tumors (with ) tremelimumab + MEDI0562 MedImmune solid tumors Phase I (anti-CTLA-4 + OX40 receptor agonist) Gaithersburg, MD www.medimmune.com

Medicines in Development: Immuno-Oncology 19 Checkpoint Modulators l Monoclonal Antibodies Drug Name Organization Indication Development Phase

TRX518 Leap Therapeutics solid tumors Phase I (anti-GITR antibody) Cambridge, MA www.leaptx.com

TSR-022 TESARO solid tumors Phase I (anti-TIM-3 mAb) Waltham, MA www.tesaro.com

TSR-042 TESARO solid tumors Phase I (anti-PD-1 mAb) Waltham, MA www.tesarobio.com

Yervoy® Bristol-Myers Squibb squamous NSCLC Phase III ipilimumab Princeton, NJ www.bms.com

Cytokines Drug Name Organization Indication Development Phase

Actimmune® Horizon Pharma breast Phase I/II interferon gamma 1-b Lake Forest, IL www.horizonpharma.com

solid tumors Phase I www.horizonpharma.com

ALKS 4230 solid tumors Phase I (recombinant fusion protein and Waltham, MA www.alkermes.com immunotherapeutic based on IL-2)

Medicines in Development: Immuno-Oncology 20 Cytokines Drug Name Organization Indication Development Phase

ALT-801 Altor BioScience advanced bladder Phase II (tumor antigen-specific T-cell receptor Miramar, FL www.altorbioscience.com linked to IL-2)

non-muscle invasive bladder, Phase I metastatic melanoma www.altorbioscience.com

ALT-803 Altor BioScience non-muscle invasive bladder Phase II (IL-15 superagonist protein complex) Miramar, FL www.altorbioscience.com

hematological malignancies, indolent Phase I non-Hodgkin lymphoma, multiple www.altorbioscience.com myeloma, NSCLC, pancreatic, solid tumors

AM0010 ARMO Biosciences pancreatic ductal adenocarcinoma Phase III (PEGylated IL-10) Redwood City, CA www.armobio.com ORPHAN DRUG

NSCLC, renal cell, solid tumors Phase I www.armobio.com

APN301 Apeiron Biologics neuroblastoma Phase II (mAb hu14.18 linked with IL-2) Vienna, Austria www.apeiron-biologics.com

CDX-301/CDX-1401 Celldex solid tumors Phase II (hematopoietic cytokine [Flt3L)/ Hampton, NJ www.celldex.com NY‑ESO‑1 antibody fusion protein) Cancer Immunotherapy Trials Ntwk. Bethesda, MD

Medicines in Development: Immuno-Oncology 21 Cytokines Drug Name Organization Indication Development Phase cergutuzumab amunaleukin (RG7813) Genentech solid tumors Phase I (immunocytokine comprising a South San Francisco, CA www.gene.com variant of IL-2 targeting CEA)

solid tumors (with atezolizumab) Phase I www.gene.com

CYT-6091 CytImmune Sciences solid tumors Phase I (nanotherapy comprised of cAu Rockville, MD www.cytimmune.com particles, tumor necrosis factor, PEG-Thiol)

DI-Leu16-IL2 Alopexx Oncology non-Hodgkin lymphoma Phase II (immunocytokine fusion protein) Concord, MA www.alopexx.com

GEN-1 Celsion ovarian Phase II (IL-12 gene therapy-nanoparticle) Lawrenceville, NJ www.celsion.com ORPHAN DRUG heterodimeric IL-15 Admune Therapeutics solid tumors Phase I ( stimulant) Danvers, MA www.admune.com

HL143 HanAll Biopharma hepatocellular Phase II (subcutaneous interferon alpha-2b) Rockville, MD www.hanallbiopharma.com

IGN002 ImmunGene non-Hodgkin lymphoma Phase I (anti-CD20 antibody/interferon-alpha) Thousand Oaks, CA www.immungene.com

Medicines in Development: Immuno-Oncology 22 Cytokines Drug Name Organization Indication Development Phase

ImmunoPulse® IL-12 OncoSec Medical triple negative breast, metastatic Phase II interleukin-12 gene therapy San Diego, CA melanoma (Fast Track) www.oncosec.com

IRX-2 IRX Therapeutics head and neck (Fast Track) Phase II (immunostimulant with multiple New York, NY www.irxtherapeutics.com cytokines)

Phase I www.irxtherapeutics.com

M9241 (NHS-IL12) EMD Serono solid tumors Phase I (interleukin-12 stimulant) Rockland, MA www.emdserono.com

MDNA55 Medicenna Therapeutics recurrent glioblastoma (Fast Track) Phase II (IL-4 empowered cytokine) Toronto, Canada www.medicenna.com ORPHAN DRUG

brain metastases, newly-diagnosed Phase I glioblastoma www.medicenna.com

NGR-hTNF MolMed malignant pleural mesothelioma Phase III (recombinant fusion protein) Milan, Italy www.molmed.com ORPHAN DRUG

Medicines in Development: Immuno-Oncology 23 Cytokines Drug Name Organization Indication Development Phase

NKTR 214 Nektar Therapeutics bladder, colorectal, malignant melanoma, Phase I/II (IL-2 receptor beta subunit agonist) San Francisco, CA NSCLC, renal cell (combination therapy) www.nektar.com

solid tumors Phase I www.nektar.com

NM-IL-12 Neumedicines cutaneous T-cell lymphoma Phase II (recombinant IL-12) Pasadena, CA www.neumedicines.com

RG7461 Genentech solid tumors Phase I (IL-2 variant fused with anti-fibroblast South San Francisco, CA www.gene.com activation protein antibody) rSIFN-co Sichuan Huiyang Life Science solid tumors Phase I (interferon) and Technology Chengdu City, China

Oncolytic Virus Therapies Drug Name Organization Indication Development Phase

Ad-VirRx 007 Multivir solid tumors Phase I (engineered oncolytic adenovirus) Houston, TX www.multivir.com

CG0070 Cold Genesys non-muscle invasive bladder Phase II (oncolytic immunotherapy) Santa Ana, CA www.coldgenesys.com

Medicines in Development: Immuno-Oncology 24 Oncolytic Virus Therapies Drug Name Organization Indication Development Phase coxsackievirus (CVA21) Viralytics bladder, lung, melanoma, prostate Phase I/II ORPHAN DRUG Sydney, Australia www.viralytics.com

DNX-2401 DNAtrix glioblastoma (Fast Track) Phase II (oncolytic immunotherapy) Houston, TX (combination therapy) www.dnatrix.com ORPHAN DRUG enadenotucirev PsiOxus Therapeutics solid tumors (with nivolumab) Phase I (Ad11/Ad3 chimeric group B Abingdon, United Kingdom www.psioxus.com adenovirus) Bristol-Myers Squibb www.bms.com Princeton, NJ

GL-ONC1 Genelux peritoneal carcinomatosis Phase I/II (genetically-modified oncolytic virus) San Diego, CA www.genelux.com

solid tumors Phase I www.genelux.com

HF10 Takara Bio USA malignant melanoma Phase II (attenuated mutant of HSV1) Mountain View, CA www.takara-bio.com

Imlygic® Amgen mid- to late-stage malignant melanoma Phase III talimogene laherparepvec Thousand Oaks, CA (combination therapy) www.amgen.com

head and neck, liver Phase I www.amgen.com

Medicines in Development: Immuno-Oncology 25 Oncolytic Virus Therapies Drug Name Organization Indication Development Phase

LOAd703 Lokon Pharma pancreatic Phase I/II (immuno-stimulary oncolytic Uppsala, Sweden www.lokonpharma.com adenovirus) ORPHAN DRUG

ONCOS-102 Targovax advanced or unresectable melanoma Phase I (genetically-modified human Oslo, Sweden progressing after PD-1 blockade www.targovax.com serotype 5/3 adenovirus) pexastimogene devacirepvec (JX-594) SillaJen Biotherapeutics hepatocellular Phase III (engineered strain of oncolytic Seoul, South Korea www.sillajen.com vaccinia poxvirus) Transgene ORPHAN DRUG Strasbourg, France

Reolysin® Oncolytics Biotech NSCLC Phase II pelareorep Calgary, Canada www.oncolyticsbiotech.com ORPHAN DRUG

colorectal, glioma, multiple myeloma, Phase I pancreatic www.oncolyticsbiotech.com telomelysin Oncolys Biopharma melanoma Phase II (telomerase-selective oncolytic Tokyo, Japan www.oncolys.com virus)

VSV-IFNß-NIS Vyriad advanced solid tumors Phase I (recombinant vesicular stomatitis Rochester, MN www.vyriad.com virus)

Medicines in Development: Immuno-Oncology 26 Vaccines Drug Name Organization Indication Development Phase adagloxad simolenin OBI Pharma metastatic breast Phase II/III (cancer immunotherapy) Taipei, Taiwan www.obipharma.com

ADXS-HER2 Advaxis metastatic HER2-expressing solid tumors Phase I (cancer immunotherapy) Princeton, NJ www.advaxis.com

ADXS-PSA Advaxis metastatic castration-resistant Phase I/II (cancer immunotherapy) Princeton, NJ prostate (monotherapy) www.advaxis.com

Advaxis metastatic prostate (in combination Phase I/II Princeton, NJ with pembrolizumab) www.advaxis.com Merck Kenilworth, NJ

AE37 Antigen Express newly-diagnosed breast Phase II (Ii-key hybrid cancer vaccine) Wellesley, MA www.antigenexpress.com

metastatic prostate (monotherapy) Phase I www.antigenexpress.com

AE37/GP2 peptide vaccine Antigen Express breast Phase I (Ii-key hybrid cancer vaccine) Wellesley, MA www.antigenexpress.com

AlloStim® Immunovative Therapies metastatic colorectal Phase II T-cell therapy-device combination Jerusalem, Israel www.immunovative.com

Medicines in Development: Immuno-Oncology 27 Vaccines Drug Name Organization Indication Development Phase

AST-VAC1 Asterias Biotherapeutics AML Phase II (dendritic cell vaccine) Fremont, CA www.asteriasbiotherapeutics.com

AutoSynVax™ Agenus cancer (prevention) Phase I autologous synthetic vaccine Lexington, MA www.agenusbio.com

AVX 701 AlphaVax CEA-expressing colorectal Phase I/II (alphavirus VEE cancer vaccine) Durham, NC www.alphavax.com

AVX 901 AlphaVax HER2-expressing breast Phase I (alphavirus VEE cancer vaccine) Durham, NC www.alphavax.com axalimogene filolisbac (AXAL) Advaxis high-risk locally advanced cervical Phase III (cancer immunotherapy) Princeton, NJ (Fast Track) www.advaxis.com ORPHAN DRUG

metastatic cervical, metastatic head Phase II and neck, metastatic anal www.advaxis.com

BB-MPI-03 Benovus Bio AML, multiple myeloma, Phase I (RNA-derived cancer vaccine) Atlanta, GA myelodysplastic syndromes www.benovousbio.com

BI 1361849 Boehringer Ingelheim Pharmaceuticals NSCLC Phase II (RNA cancer vaccine) Ridgefield, CT www.boehringer-ingelheim.com

BriaVax™ BriaCell Therapeutics breast cancer Phase I/II SV-BR-1 GM cancer vaccine Berkeley, CA www.briacell.com

Medicines in Development: Immuno-Oncology 28 Vaccines Drug Name Organization Indication Development Phase

CMB305 Immune Design soft tissue sarcoma Phase II (prime-boost imunotherapy) Seattle, WA www.immunedesign.com ORPHAN DRUG

CMP-001 Checkmate Pharmaceuticals malignant melanoma (with pembrolizumab) Phase I (virus-like particle cancer vacicne) Cambridge, MA www.checkmatepharma.com vaccine Targeted Diagnostics & Therapeutics colorectal Phase I (therapeutic cancer vaccine) Exton, PA www.viralgeneinc.com Viral Gene Philadelphia, PA

CRS-207 Aduro Biotech ovarian (combination therapy) Phase I/II (Listeria monocytogenes Berkeley, CA www.aduro.com cancer vaccine) Incyte ORPHAN DRUG Wilmington, DE

Aduro Biotech gastric (combination therapy), Phase I Berkeley, CA mesothelioma (combination therapy) www.aduro.com

CV301 Bavarian Nordic NSCLC (with nivolumab) Phase II (CEA/MUC-1 targeted immunotherapy) Washington, DC www.bavarian-nordic.com

DCVax®-Direct Northwest Biotherapeutics solid tumors Phase I/II personalized immune therapy Bethesda, MD www.nwbio.com

Medicines in Development: Immuno-Oncology 29 Vaccines Drug Name Organization Indication Development Phase

DCVax®-L Northwest Biotherapeutics newly-diagnosed glioblastoma Phase III personalized immune therapy Bethesda, MD www.nwbio.com ORPHAN DRUG

metastatic ovarian Phase I completed www.nwbio.com

DCVax®-Prostate Northwest Biotherapeutics prostate Phase II completed personalized immune therapy Bethesda, MD www.nwbio.com

DPV-001 UbiVac definitely-treated stage III NSCLC Phase II (tumor-derived, autophagosome- Portland, OR www.ubivac.com enriched cancer vaccine)

advanced prostate Phase I www.ubivac.com

DPX E7 Immunovaccine HPV-related anal, cervical, head and Phase I/II (peptide cancer vaccine) Halifax, Canada neck www.imvaccine.com

DPX Survivac Immunovaccine ovarian Phase I (peptide cancer vaccine) Halifax, Canada (combination therapy) www.imvaccine.com ORPHAN DRUG

DSP-7888 Sunovion Pharmaceuticals hematologic malignancies, solid tumors Phase I (peptide cancer vaccine) Marlborough, MA www.sunovion.com

Medicines in Development: Immuno-Oncology 30 Vaccines Drug Name Organization Indication Development Phase

Elenagen™ CureLab Oncology solid tumors Phase I/II p62 anti-cancer DNA Vaccine Canton, MA www.curelaboncology.com

EP-101 EpiThany breast Phase I (type-1 T helper cell stimulant) Seattle, WA www.epithany.com

EP-201 EpiThany ovarian Phase I (type-1 T helper cell stimulant) Seattle, WA www.epithany.com

EP-301 EpiThany breast Phase I (type-1 T helper cell stimulant) Seattle, WA www.epithany.com

ERC1671 Epitopoietic Research glioblastoma Phase II (allogeneic/autologous cell vaccine) Pasadena, CA www.erc-immunotherapy.com ORPHAN DRUG

ETBX-011 Etubics solid tumors Phase I/II (Ad5 CEA cancer vaccine) Seattle, WA www.etubics.com

ETBX-021 Etubics breast Phase I (Ad5 HER2/neu cancer vaccine) Seattle, WA www.etubics.com NantBioScience Culver City, CA

GALE-301 endometrial, Phase II (folate binding protein E39-targeted San Ramon, CA www.galenabiopharma.com peptide vaccine) ORPHAN DRUG

Medicines in Development: Immuno-Oncology 31 Vaccines Drug Name Organization Indication Development Phase

GALE-301 + GALE-302 Galena Biopharma breast, ovarian Phase I/II (immunogenic peptide vaccine San Ramon, CA www.galenabiopharma.com combination) ORPHAN DRUG

galinpepimut-S Sellas Life Sciences AML (Fast Track), Phase II (WT1 cancer vaccine) New York, NY mesothelioma (Fast Track), www.sellaslifesciences.com ORPHAN DRUG multiple myeloma

CML, ovarian (with nivolumab) Phase I/II www.sellaslifesciences.com

GI-6207 Celgene medullary thyroid cancer Phase II (CEA inhibitor cancer vaccine) Summit, NJ www.celgene.com GlobeImmune www.globeimmune.com Louisville, CO

GI-6301 Celgene chordoma Phase II (brachyury peptide cancer vaccine) Summit, NJ www.globeimmune.com GlobeImmune Louisville, CO advanced cancer Phase I www.globeimmune.com

GL 0817 Gliknik head and neck, squamous cell Phase II (peptide immunomodulator) Baltimore, MD www.gliknik.com

GRN 1201 GreenPeptide melanoma Phase I (peptide cancer vaccine) Kurume, Japan www.green-peptide.com

Medicines in Development: Immuno-Oncology 32 Vaccines Drug Name Organization Indication Development Phase

ICT-107 ImmunoCellular Therapeutics newly-diagnosed glioblastoma Phase III (dendritic cell vaccine) Calabasas, CA www.imuc.com ORPHAN DRUG

ICT-121 ImmunoCellular Therapeutics recurrent glioblastoma Phase I (dendritic cell vaccine) Calabasas, CA www.imuc.com

IMT1012 Immunotope breast, ovarian Phase I (multi-peptide antigen Doylestown, PA www.immunotope.com cancer vaccine)

INO-1400 Inovio Pharmaceuticals breast, lung, pancreatic Phase I (hTERT DNA cancer vaccine) Plymouth Meeting, PA www.inovio.com

INO-3106 Inovio Pharmaceuticals aerodigestive Phase I (E6, E7 DNA cancer vaccine) Plymouth Meeting, PA www.inovio.com

INO-3112 Inovio Pharmaceuticals cervical, head and neck Phase I/II (E6, E7 DNA cancer vaccine) Plymouth Meeting, PA www.inovio.com MedImmune www.medimmune.com Gaithersburg, MD

INO-5150 Inovio Pharmaceuticals prostate Phase I (PSA, PSMA DNA cancer vaccine) Plymouth Meeting, PA www.inovio.com

INT230-6 Intensity Therapeutics cancer Phase I/II (cisplatin based intratumoral Westport, CT www.intensitytherapeutics.com cancer vaccine)

Medicines in Development: Immuno-Oncology 33 Vaccines Drug Name Organization Indication Development Phase

JNJ-64041757 (ADU-214) Aduro Biotech lung Phase I (Listeria monocytogenes Berkeley, CA www.aduro.com immunotherapy) Janssen Research & Development www.janssen.com Raritan, NJ

JNJ-64041809 (ADU-741) Aduro Biotech prostate Phase I (Listeria monocytogenes Berkeley, CA www.aduro.com immunotherapy) Janssen Research & Development www.janssen.com Raritan, NJ

LAMP-Vax + pp65 DC therapy Immunomic Therapeutics newly-diagnosed glioblastoma Phase I/II (DNA immunotherapeutic vaccine) Rockville, MD www.immunomix.com

LV305 Immune Design breast, malignant melanoma, NSCLC, Phase I (RNA cancer vaccine) Seattle, WA ovarian, sarcoma www.immunedesign.com ORPHAN DRUG

MUC-1 CD40L dendritic cell vaccine MicroVAX solid tumors Phase I Manassas, VA

MVA-BN brachyury vaccine Bavarian Nordic cancer Phase I (cancer immunotherapy) Washington, DC www.bavarian-nordic.com

MVA p53 vaccine Tara Immuno-Oncology solid tumors Phase I (modified vaccinia virus ankara New York, NY vaccine expressing p53)

Medicines in Development: Immuno-Oncology 34 Vaccines Drug Name Organization Indication Development Phase

M-VAX AVAX Technologies melanoma Phase II (autologous tumor cell vaccine) Philadelphia, PA www.avax-tech.com ORPHAN DRUG

MVI-118 Madison Vaccines metastatic prostate Phase I (AR plasmind DNA vaccine) Madison, WI www.madisonvaccinesinc.com

MVI-816 Madison Vaccines biochemically recurrent prostate Phase II (plasmid DNA vaccine) Madison, WI www.madisonvaccinesinc.com

metastatic castration-resistant prostate Phase I (combination therapy) www.madisonvaccinesinc.com

NEO-PV-01 Bristol-Myers Squibb bladder, melanoma, NSCLC Phase I (personalized neoantigen vaccine) Princeton, NJ (with nivolumab) www.neontherapeutics.com Neon Therapeutics Cambridge, MA

NeuVax® Galena Biopharma triple negative HER2-positive breast Phase II nelipepimut S San Ramon, CA (combination therapy), HER2-positive www.galenabiopharma.com (peptide cancer vaccine) ductal carcinoma in situ of breast (combination therapy)

gastric Phase I www.galenabiopharma.com

Oncoquest™-CLL XEME Biopharma chronic lymphocytic leukemia Phase I chronic lymphocytic leukemia vaccine Lombard, IL www.xemebiopharma.com

Medicines in Development: Immuno-Oncology 35 Vaccines Drug Name Organization Indication Development Phase

Oncoquest-L® XEME Biopharma non-Hodgkin lymphoma Phase I completed personalized cancer vaccine Lombard, IL www.xemebiopharma.com

OncoVAX® Vaccinogen colorectal (Fast Track) Phase III colorectal cancer vaccine Baltimore, MD www.vaccinogeninc.com

OSE-2101 OSE Immunotherapeutics NSCLC Phase III (neo-epitopes cancer vaccine) Nantes, France www.ose-immuno.com

O-VAX AVAX Technologies ovarian Phase I/II (autologous tumor cell-based vaccine) Philadelphia, PA www.avax-tech.com ovapuldencel-T AiVita Biomedical ovarian Phase II Irvine, CA www.aivitabiomedical.com

PAN-301-1 Panacea Pharmaceuticals prostate Phase I (nanoparticle-based cancer vaccine) Gaithersburg, MD www.panaceapharma.com

PDS0101 PDS Biotechnology HPV-related cancer Phase I (cancer immunotherapy) North Brunswick, NJ www.pdsbiotech.com

PEPIDH1M Annias Immunotherapeutics glioblastoma Phase I/II (dendritic cell cancer vaccine) Chapel Hill, NC www.anniasimmuno.com

PF-06753512 Pfizer prostate Phase I (cancer immunotherapy) New York, NY www.pfizer.com

Medicines in Development: Immuno-Oncology 36 Vaccines Drug Name Organization Indication Development Phase polyclonal antibody stimulator Cancer Advances gastric, pancreatic Phase III (fusion peptide) Durham, NC www.canceradvancesinc.com ORPHAN DRUG

colorectal Phase II www.canceradvancesinc.com

Prophage™ Agenus newly-diagnosed glioblastoma Phase II heat shock protein vaccine Lexington, MA www.agenusbio.com ORPHAN DRUG

ProscaVax OncBioMune Pharmaceuticals prostate cancer Phase I PSA/IL-2/GM-CSF vaccine Baton Rouge, LA www.oncbiomune.com

PROSTVAC Bavarian Nordic metastatic castration-resistant Phase III rilimogene galvacirepvec- Morrisville, NC prostate cancer (monotherapy) www.bavarian-nordic.com rilimogene glafolivec Bristol-Myers Squibb (Fast Track) www.bms.com Princeton, NJ

PSMA VRP AlphaVax prostate Phase I (viral vector vaccine) Durham, NC www.alphavax.com

PT 107 Pique Therapeutics NSCLC Phase II (allogeneic B7.1/HLA-A1 transfected Durham, NC www.piquetherapeutics.com tumor cell vaccine)

PVX-410 OncoPep smoldering multiple myeloma Phase I (peptide cancer vaccine) North Andover, MA www.oncopep.com

Medicines in Development: Immuno-Oncology 37 Vaccines Drug Name Organization Indication Development Phase

REIC gene therapy Momotaro-Gene localized prostate Phase I Okayama, Japan www.mt-gene.com

RG6180 Genentech cancer Phase I (personalized mRNA cancer vaccine) South San Francisco, CA www.gene.com

rocapuldencel-T Argos Therapeutics renal cell carcinoma (Fast Track) Phase III (dendritic cell vaccine) Durham, NC www.argostherapeutics.com seviprotimut-L Polynoma melanoma Phase III (polyvalent antigen vaccine) San Diego, CA www.polynoma.com

SL-701 Stemline Therapeutics glioblastoma (adults) Phase II (alpha-type 1 dendritic New York, NY www.stemline.com cell-based vaccine) ORPHAN DRUG stapuldencel-T SOTIO prostate Phase III (autologous dendritic cell vaccine) Prague, Czech Republic www.sotio.com

SurVaxM MimiVax glioblastoma Phase II peptide mimic vaccine Rochester, NY www.mimivax.com

multiple myeloma Phase I www.mimivax.com

Medicines in Development: Immuno-Oncology 38 Vaccines Drug Name Organization Indication Development Phase tergenpumatucel-L + indoximod + NewLink Genetics NSCLC (combination therapy) Phase I/II Ames, IA www.newlinkgenetics.com (tumor cell vaccine)

TG4010 Transgene NSCLC (with nivolumab) Phase II (MVA-MUC1-IL2 cancer vaccine) Paris, France www.transgene.fr Bristol-Myers Squibb Princeton, NJ

TLPLDC Cancer Insight melanoma Phase II/III (tumor lysate particle loaded San Antonio, TX www.cancerinsight.com dendritic cell vaccine) Elios Therapeutics Austin, TX ovarian Phase I/II www.cancerinsight.com

TPIV 110 TapImmune HER2/neu-positive breast Phase I (peptide antigen cancer vaccine) Jacksonville, FL www.tapimmune.com

TPIV 200 TapImmune platinum-sensitive ovarian (Fast Track), Phase II (peptide antigen cancer vaccine) Jacksonville, FL platinum-resistant ovarian (combination www.tapimmune.com ORPHAN DRUG therapy), triple negative breast

TV-Kidney-1 TVAX Biomedical renal cell Phase II (personalized cancer cell vaccine Olathe, KS www.tvaxbiomedical.com and killer T-cell therapy)

Medicines in Development: Immuno-Oncology 39 Vaccines Drug Name Organization Indication Development Phase

TV-Brain-1 TVAX Biomedical glioblastoma Phase II (personalized cancer cell vaccine Olathe, KS www.tvaxbiomedical.com and killer T-cell therapy) vesigenurtucel-L (HS-410) Heat Biologics non-muscle invasive bladder Phase II (cytotoxic T-lymphocyte stimulant Durham, NC (Fast Track) www.heatbiologics.com vaccine)

VGX-3100 Inovio Pharmaceuticals cervical intraepithelial neoplasia Phase II (therapeutic DNA vaccine) Plymouth Meeting, PA (high grade HPV-caused pre-) www.inovio.com viagenpumatucel-L (HS-110) Heat Biologics NSCLC Phase II (allogeneic vaccine) Durham, NC www.heatbio.com

Vigil™ Gradalis advanced ovarian Phase II genetically-modified autologous Dallas, TX www.gradalisinc.com tumor cell vaccine

advanced breast, Ewing's sarcoma, Phase I advanced NSCLC, advanced melanoma www.gradalisinc.com

WT2725 Sunovion Pharmaceuticals hematological malignancies, solid tumors Phase I (peptide cancer vaccine) Marlborough, MA www.sunovion.com

Medicines in Development: Immuno-Oncology 40 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

131-I-MIP-1095 Progenics Pharmaceuticals metastatic castration-resistant prostate Phase I (PSMA-targeting ADC) New York, NY www.progenics.com

177 Lu PSMA-617 targeted RadioMedix metastatic castration-resistant Phase II radioligand therapy Houston, TX prostate cancer www.radiomedix.com Excel Diagnostics and Nuclear Oncology Houston, TX University of California-Los Angeles Los Angeles, CA

8H9 I-124 mAb Y-mAbs Therapeutics diffuse intrinsic pontine glioma Phase I/II ORPHAN DRUG New York, NY www.ymabs.com

8H9 I-131 mAb Y-mAbs Therapeutics leptomeningeal tumor from Phase II ORPHAN DRUG New York, NY neuroblastoma www.ymabs.com

desmoplastic small round cell tumor Phase I/II www.ymabs.com

ABBV-181 AbbVie solid tumors Phase I North Chicago, IL www.abbvie.com

ABBV-399 AbbVie solid tumors Phase I (c-met inhibitor) North Chicago, IL www.abbvie.com

ABBV-838 AbbVie multiple myeloma Phase I (CD319 antigen inhibitor) North Chicago, IL www.abbvie.com

Medicines in Development: Immuno-Oncology 41 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

ABP 215 NSCLC application submitted (bevacizumab biosimilar) Parsippany, NJ www.allergan.com Amgen www.amgen.com Thousand Oaks, CA

ABP 798 Allergan non-Hodgkin lymphoma, CLL Phase III (rituximab biosimilar) Parsippany, NJ www.allergan.com Amgen www.amgen.com Thousand Oaks, CA

ABP 980 Allergan HER2-positive breast, HER2-positive gastric Phase III ( biosimilar) Parsippany, NJ www.allergan.com Amgen www.amgen.com Thousand Oaks, CA

ABT-414 AbbVie glioblastoma Phase II () North Chicago, IL www.abbvie.com ORPHAN DRUG

pediatric brain tumors Phase I www.abbvie.com

Actimab-A Actinium Pharmaceuticals newly-diagnosed AML in patients over Phase II (CD33 antigen inhibitor) New York, NY 60 years of age www.actiniumpharma.com ORPHAN DRUG

Actimab-M Actinium Pharmaceuticals relapsed/refractory multiple myeloma Phase I (CD33 antigen inhibitor) New York, NY www.actiniumpharma.com

Medicines in Development: Immuno-Oncology 42 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

Adcetris® Seattle Genetics cutaneous T-cell lymphoma, Hodgkin Phase III Bothell, WA lymphoma (1L), CD30-expressing www.seattlegenetics.conm ORPHAN DRUG mature T-cell lymphoma (1L)

CD30-expressing DLBCL, Hodgkin lymphoma Phase II in patients 60 and older, Hodgkin lymphoma www.seattlegenetics.com (2L), relapsed non-Hodgkin lymphoma

ADCT-301 ADC Therapeutics AML, lymphoma Phase I (antibody-drug conjugate) Murray Hill, NJ www.adctherapeutics.com

ADCT-402 ADC Therapeutics ALL, B-cell non-Hodgkin lymphoma Phase I (antibody-drug conjugate) Murray Hill, NJ www.adctherapeutics.com

ADCT-502 ADC Therapeutics HER2-expressing solid tumors Phase I (antibody-drug conjugate) Murray Hill, NJ www.adctherapeutics.com

AGS-16C3F Agensys renal cell Phase II (antibody-drug conjugate Santa Monica, CA www.agensys.com targeting ENPP3)

AGS-62P1 Agensys AML Phase I (antibody-drug conjugate) Santa Monica, CA www.agensys.com

AGS-67E Agensys AML, lymphoid malignancies Phase I (antibody-drug conjugate) Santa Monica, CA www.agensys.com

Medicines in Development: Immuno-Oncology 43 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase (MORAb-009) Eisai mesothelioma Phase II (IgG1 mAb) Woodcliff Lake, NJ www.eisai.com ORPHAN DRUG Morphotek Exton, PA

AMG 224 Amgen multiple myeloma Phase I (antibody-drug conjugate) Thousand Oaks, CA www.amgen.com

AMG 820 Amgen solid tumors Phase I (c-fms inhibitor) Thousand Oaks, CA www.amgen.com (GS-5745) gastric Phase III (MMP9 mAb inhibitor) Foster City, CA www.gilead.com

solid tumors Phase I www.gilead.com anetumab ravtansine Bayer HealthCare Pharmaceuticals mesothelioma Phase II (mesothelin-targeted ADC) Whippany, NJ www.pharma.bayer.com ORPHAN DRUG

solid tumors Phase I www.pharma.bayer.com angiopoietin 2 mAb Eli Lilly cancer Phase I Indianapolis, IN www.lilly.com

Medicines in Development: Immuno-Oncology 44 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase anti-CD73 mAb Bristol-Myers Squib cancer Phase I Princeton, NJ www.bms.com anti-MUC1 AR20.5 OncoQuest pancreatic Phase I/II (chemo-enhanced immunotherapy) Edmonton, Canada www.oncoquestinc.com

APX005M Apexigen malignant melanoma, solid tumors Phase I/II (CD40 agonist) San Carlos, CA www.apexigen.com

ARX788 Ambrx HER2-overexpressing breast Phase I (antibody-drug conjugate) La Jolla, CA www.ambrx.com

Arzerra® Novartis Pharmaceuticals follicular lymphoma Phase III East Hanover, NJ www.novartis.com

ASG-15ME Astellas metastatic urothelial Phase I (antibody-drug conjugate) Northbrook, IL www.astellas.com Seattle Genetics www.seattlegenetics.com Bothell, WA

AV-203 AVEO Oncology solid tumors Phase I (ERBB-3 receptor antagonist) Cambridge, MA www.aveooncology.com

Avastin® Genentech newly-diagnosed glioblastoma, application submitted bevacizumab South San Francisco, CA relapsed platinum-sensitive ovarian www.gene.com ORPHAN DRUG

Medicines in Development: Immuno-Oncology 45 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

AVID100 Formation Biologics solid tumors Phase I/II (epidermal modulator) Austin, TX www.formationbiologics.com

B-701 BioClin Therapeutics metastatic bladder (2L) Phase II (type-3-FGF receptor antagonist) San Ramon, CA www.bioclintherapeutics.com

urothelial (with atezolizumab) Phase I www.bioclintherapeutics.com Peregrine Pharmaceuticals previously-treated NSCLC Phase III (chimeric mAb) Tustin, CA (combination therapy) www.peregrineinc.com

hepatocellular (combination therapy), Phase II head and neck (combination therapy), www.peregrineinc.com squamous cell

glioblastoma, rectal Phase I www.peregrineinc.com

BAY-1129980 Bayer HealthCare Pharmaceuticals solid tumors Phase I (C4.4a antibody-drug conjugate) Whippany, NJ www.pharma.bayer.com bevacizumab biosimilar Biocon cancer Phase I Bangalore, India www.biocon.com www.mylan.com Canonsburg, PA

Medicines in Development: Immuno-Oncology 46 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase bevacizumab biosimilar Oncobiologics colorectal, lung Phase I Cranbury, NJ www.oncobiologics.com

BHQ880 Novartis Pharmaceuticals smoldering multiple myeloma Phase II (DKK1 protein inhibitor) East Hanover, NJ www.novartis.com

BI-695500 Boehringer Ingelheim Pharmaceuticals follicular lymphoma Phase III (rituximab biosimilar) Ridgefield, CT www.boehringer-ingelheim.com

BI-695502 Boehringer Ingelheim Pharmaceuticals brain metastases Phase II (bevacizumab biosimilar) Ridgefield, CT www.boehringer-ingelheim.com

BI-836826 Boehringer Ingelheim Pharmaceuticals cancer Phase I Ridgefield, CT www.boehringer-ingelheim.com Xencor www.xencor.com Monrovia, CA

BI-836858 Boehringer Ingelheim Pharmaceuticals AML, myelodysplastic syndromes Phase I/II (CD33 antigen inhibitor) Ridgefield, CT www.boehringer-ingelheim.com ORPHAN DRUG Xencor www.xencor.com Monrovia, CA

BMS-986012 Bristol-Myers Squibb SCLC Phase II (anti-fucosyl GM1) Princeton, NJ www.bms.com

BMS-986148 Bristol-Myers Squibb solid tumors Phase I (mesothelin ADC) Princeton, NJ www.bms.com

Medicines in Development: Immuno-Oncology 47 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

BMS-986207 Bristol-Myers Squibb solid tumors Phase I/II (anti-TIGIT) Princeton, NJ www.bms.com

BMS-986218 Bristol-Myers Squibb solid tumors Phase I/II Princeton, NJ www.bms.com OncoMed Pharmaceuticals colorectal Phase I (anti-notch-1 mAb) Redwood City, CA www.oncomed.com cabiralizumab Bristol-Myers Squibb solid tumors Phase I (CSF/M-CSF receptor antagonist) Princeton, NJ www.bms.com Five Prime Therapeutics www.fiveprime.com South San Francisco, CA

CC-90002 Celgene AML, myelodysplastic syndromes, Phase I (CD47 antigen inhibitor) Summit, NJ solid tumors www.celgene.com

CDX-014 Celldex Therapeutics renal cell Phase I/II (antibody-drug conjugate) Hampton, NJ www.celldex.com

CDX-0158 Celldex Therapeutics gastrointestinal stromal tumors, Phase I (antibody-drug conjugate) Hampton, NJ KIT-positive tumors www.celldex.com

CDX-3379 Celldex Therapeutics solid tumors Phase I (ERBB-3 receptor antagonist) Hampton, NJ www.celldex.com

Medicines in Development: Immuno-Oncology 48 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase Oncternal Therapeutics CLL Phase I (receptor protein-tyrosine San Diego, CA www.oncternal.com kinase antagonist)

CJM112 Novartis Pharmaceuticals solid tumors Phase I (IL-17 inhibitor) East Hanover, NJ www.novartis.com codrituzumab (RG7686) Genentech metastatic liver Phase II (glypican 3 inhibitor) South San Francisco, CA www.gene.com

coltuximab ravtansine ImmunoGen non-Hodgkin lymphoma Phase II (CD19 antigen inhibitor) Waltham, MA www.immunogen.com

Cotara® Peregrine Pharmaceuticals glioblastoma Phase II derlotuximan biotin Tustin, CA www.peregrineinc.com (mAb conjugated to 131-iodine) ORPHAN DRUG

CT-P6 Celltrion breast Phase I completed (trastuzumab biosimilar) Incheon, South Korea www.tevausa.com Teva Pharmaceuticals North wales, PA

Medicines in Development: Immuno-Oncology 49 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

Cyramza® Eli Lilly bladder, gastric, hepatocellular, NSCLC Phase III Indianapolis, IN www.lilly.com ORPHAN DRUG

advanced or metastatic biliary Phase II www.lilly.com

NSCLC (combination therapy) Phase I www.lilly.com

Darzalex® Janssen Research & Development multiple myeloma (1L combination Phase III Raritan, NJ therapy) www.janssen.com ORPHAN DRUG

DLBCL, follicular lymphoma, mantle-cell Phase II lymphoma www.janssen.com Seattle Genetics DLBCL Phase II (anti-CD19 protein) Bothell, WA www.seattlegenetics.com depatuxizumab AbbVie solid tumors Phase I (EGFR receptor antagonist) North Chicago, IL www.abbvie.com United Therapeutics SCLC Phase II/III (anti-GD2 mAb) Silver Spring, MD www.unither.com

Medicines in Development: Immuno-Oncology 50 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

DKN-01 Leap Therapeutics cholangiocarcinoma, esophageal Phase I/II (anti-DKK1 antibody) Cambridge, MA www.leaptx.com

NSCLC Phase I www.leaptx.com

DS-8201 Daiichi Sankyo breast (Fast Track), gastric Phase I (anti-HER2 antibody-drug conjugate) Parsippany, NJ www.dsi.com

DS-8273 Daiichi Sankyo colorectal Phase I (anti-DR5 antibody) Parsippany, NJ www.daiichisankyo.com (MEDI-573) MedImmune metastatic breast cancer Phase II (-like growth factor inhibitor) Gaithersburg, MD www.medimmune.com (RG7155) Genentech solid tumors (combination therapy) Phase I (M-CSF receptor antagonist) South San Francisco, CA www.gene.com Eli Lilly cancer Phase II (proto oncogene protein Indianapolis, IN www.lilly.com c-Met inhibitor)

Empliciti® Bristol-Myers Squibb multiple myeloma (1L) Phase III Princeton, NJ www.bms.com ORPHAN DRUG

Medicines in Development: Immuno-Oncology 51 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase Agensys urogenital Phase II (ADC targeting nectin-4) Santa Monica, CA www.agensys.com Seattle Genetics www.seattlegenetics.com Bothell, WA MacroGenics solid tumors Phase I (CD276 protein inhibitor) Rockville, MD www.macrogenics.com Precision Biologics colorectal, pancreatic Phase II (neoplasm antigen inhibitor) Great Neck, NY www.precision-biologics.com ORPHAN DRUG Immunomedics ALL (pediatric) Phase III (anti-CD22 mAb) Morris Plains, NJ www.immunomedics.com ORPHAN DRUG (MORAb-003) Eisai platinum-sensitive ovarian, NSCLC Phase II (folate receptor 1 antagonist) Woodcliff Lake, NJ www.eisai.com ORPHAN DRUG Morphotek Exton, PA

FF21101 Fujifilm Pharmaceuticals U.S.A. solid tumors Phase I (radio-labelled anti-cadherin-3 mAb) Cambridge, MA www.fujifilmusa.com

FG-3019 FibroGen pancreatic Phase II () San Francisco, CA www.fibrogen.com

Medicines in Development: Immuno-Oncology 52 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase AVEO Oncology AML, head and neck Phase I Cambridge, MA www.aveooncology.com Biodesix Boulder, CO

FPA144 Five Prime Therapeutics bladder, gastric Phase I (FGFR2b receptor antagonist) South San Francisco, ca www.fiveprime.com ORPHAN DRUG NantCell Ewing's sarcoma in clinical trials ORPHAN DRUG Culver City, CA www.nantworks.com

Gazyva® Genentech hematological malignancies (multiple Phase I oblinutuzumab South San Francisco, CA combination regimens) www.gene.com

Celldex triple negative breast (Fast Track), Phase II Hampton, NJ metastatic melanoma www.celldex.com glypican3-ADC Bristol-Myers Squibb cancer Phase I Princeton, NJ www.bms.com

GP2013 Sandoz CLL, DLBCL, follicular lymphoma Phase III (rituximab biosimilar) Princeton, NJ www.sandoz.com

GSK2857916 GlaxoSmithKline multiple myeloma Phase I (beta cell maturation antigen ADC) Philadelphia, PA www.gsk.com

Medicines in Development: Immuno-Oncology 53 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

GSK3359609 GlaxoSmithKline solid tumors Phase I (induced T-cell costimulatory agonist Philadelphia, PA www.gsk.com antibody)

HKT288 Novartis Pharmaceuticals solid tumors Phase I (cadherin-6 antibody-drug conjugate) East Hanover, NJ www.novartis.com

Hu3F8 mAb Y-mAbs Therapeutics refractory/relapsed neuroblastoma Phase II (GD2 ganglioside inhibitor) New York, NY www.ymabs.com ORPHAN DRUG

osteosarcoma (pediatric) Phase I/II www.ymabs.com

Hu5F9-G4 Forty Seven colorectal cancer, solid tumors Phase I/II (CD47 antigen inhibitor) Menlo Park, CA (combination therapy), relapsed/refractory www.fortyseveninc.com B-cell non-Hodgkin lymphoma

AML, solid tumors Phase I www.fortyseveninc.com

HuL2G7 Galaxy Biotech solid tumors Phase I completed (anti-HGF ) Sunnyvale, CA www.galaxybiotech.com

HuMax-IL8 Bristol-Myers Squibb solid tumors Phase I (IL-8 inhibitor) Princeton, NJ www.bms.com

Medicines in Development: Immuno-Oncology 54 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

ICON-1 Iconic Therapeutics uveal melanoma Phase I (immunoconjugate proteins to South San Francisco, CA www.iconictherapeutics.com tissue proteins) ifabotuzumab KaloBios Pharmaceuticals AML, myelodysplastic syndromes Phase II (EphA3 receptor antagonist) Brisbane, CA www.kalobios.com

IGN523 Igenica AML Phase I (CD98 antigen inhibitor) Burlingame, CA www.igencia.com

IMGN529 ImmunoGen non-Hodgkin lymphoma Phase II (antibody-drug conjugate) Waltham, MA www.immunogen.com

IMGN779 ImmunoGen AML Phase I (CD33-targeting DNA-alkylating ADC) Waltham, MA www.immunogen.com

IMMU-114 Immunomedics hematological malignancies Phase I (anti-HLA-DR antigen) Morris Plains, NJ www.immunomedics.com Biotest multiple myeloma Phase II ORPHAN DRUG Dreieich, Germany www.biotest.com

Pfizer ALL (Breakthrough Therapy) application submitted ORPHAN DRUG New York, NY www.pfizer.com

Medicines in Development: Immuno-Oncology 55 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

Iomab-B Actinium Pharmaceuticals bone marrow transplant in patients with Phase III CD45 antigen inhibitor New York, NY AML (age 55 and over) www.actiniumpharma.com ORPHAN DRUG

IPH4102 Innate Pharma cutaneous T-cell lymphoma Phase I (cytotoxic mAb) Marseille, France www.innatepharma.com Sanofi US relapsed/refractory multiple myeloma Phase III (anti-CD38 naked mAb) Bridgewater, NJ www.sanofi.com ORPHAN DRUG

JNJ-56022473 Janssen Research & Development acute myeloid leukemia Phase II (IL-3-receptor-alpha-subunit Raritan, NJ www.janssen.com antagonist)

JNJ-61610588 Janssen Research & Development solid tumors Phase I (VISTA inhibitor) Raritan, NJ www.janssen.com

JNJ-64457107 Janssen Research & Development solid tumors Phase I (CD40 antigen stimulant) Raritan, NJ www.janssen.com

Kadcyla® Genentech early HER2-positive breast (with Phase III South San Francisco, CA ), HER2-positive breast (3L), www.gene.com HER2-positive breast (adjuvant)

HER2-positive metastatic breast (2L) Phase II (with atezolizumab) www.gene.com

Medicines in Development: Immuno-Oncology 56 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase govitecan Immunomedics colorectal Phase II (anti-CEACAM5-SN-38) Morris Plains, NJ www.immunomedics.com

Lartruvo™ Eli Lilly cancer (pediatric) Phase I Indianapolis, IN www.lilly.com

L-DOS47 Helix Biopharma NSCLC Phase I (immunoconjugate) Toronto, Canada www.helixbiopharma.com KaloBios Pharmaceuticals chronic myelomonocytic leukemia Phase I/II (GM-CSF antagonist) Brisbane, CA www.kalobios.com satetraxetan Nordic Nanovector DLBCL Phase I (lutetium-17 conjugates to a mAb) Oslo, Norway www.nordicnanovector.com ORPHAN DRUG

LY3022855 Eli Lilly cancer Phase I (CSF-1R mAb) Indianapolis, IN www.lilly.com

LY3076226 Eli Lilly cancer Phase I (FGFR3 antibody-drug conjugate) Indianapolis, IN www.lilly.com MacroGenics breast Phase III (anti-HER2 antibody) Rockville, MD www.macrogenics.com

gastric Phase II www.macrogenics.com

Medicines in Development: Immuno-Oncology 57 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

MCS110 Novartis Pharmaceuticals triple negative breast Phase I (IgG1 kappa mAb) East Hanover, NJ (combination therapy) www.novartis.com

MEDI3726 (ADCT-401) ADC Therapeutics prostate Phase I (PSMA antibody-drug conjugate) Murray Hill, NJ www.adctherapeutics.com MedImmune www.medimmune.com Gaithersburg, MD

MEDI4276 MedImmune solid tumors Phase I (HER2 antibody-drug conjugate) Gaithersburg, MD www.medImmune.com

MEDI9447 MedImmune solid tumors Phase I (anti-CD37 mAb) Gaithersburg, MD www.medimmune.com mirvetuximab soravtansine ImmunoGen platinum-resistant ovarian Phase III (antibody-drug conjugate) Waltham, MA www.immunogen.com ORPHAN DRUG

MM-151 Merrimack Pharmaceuticals solid tumors Phase I (EGFR antagonist) Cambridge, MA www.merrimack.com mogamulizumab Kyowa Kirin cutaneous T-cell lymphoma Phase III (CCR4 receptor antagonist) Bedminster, NJ www.kyowa-kirin.com ORPHAN DRUG

adult T-cell leukemia / lymphoma, Phase I/II solid tumors www.kyowa-kirin.com

Medicines in Development: Immuno-Oncology 58 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase monalizumab Innate Pharma CLL, head and neck (combination therapy) Phase I/II (NK cell lectin-like receptor Marseille, France www.innatepharma.com subfamily C antagonist)

Innate Pharma solid tumors Phase I Marseille, France (with durvalumab) www.innatepharma.com MedImmune www.medimmune.com Gaithersburg, MD

MOR208 MorphoSys chronic lymphocytic leukemia, Phase II (CD19 antigen inhibitor) Planegg, Germany diffuse large B-cell lymphoma (Fast Track) www.morphosys.com ORPHAN DRUG

MORAb-066 Eisai solid tumors Phase I (IgG mAb) Woodcliff Lake, NJ www.eisai.com Morphotek www.morphotek.com Exton, PA MedImmune hairy cell leukemia Phase III (CD22/recombinant immunotoxin) Gaithersburg, MD www.medimmune.com ORPHAN DRUG

MT-3724 Molecular Templates B-cell malignancies Phase I (modified toxins fused to single-chain San Diego, CA www.moleculartemplates.com antibody variable fragments)

MVT-1075 MabVax Therapeutics pancreatic Phase I () San Diego, CA www.mabvax.com

Medicines in Development: Immuno-Oncology 59 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

MVT-5873 MabVax Therapeutics colorectal, pancreatic Phase I (GD2 ganglioside modulator) San Diego, CA www.mabvax.com

Mylotarg® Pfizer acute myeloid leukemia (1L) application submitted New York, NY www.pfizer.com Eli Lilly NSCLC (combination therapy) Phase I (EGFR inhibitor) Indianapolis, IN www.lilly.com

NIS793 Novartis Pharmaceuticals solid tumors Phase I/II (anti-TGF-beta mAb) East Hanover, NJ www.novartis.com

OMP-313M32 OncoMed Pharmaceuticals solid tumors Phase I (anti-TIGHT antibody) Redwood City, CA www.oncomed.com (MORAb-004) Eisai melanoma Phase II (CD248 antigen inhibitor) Woodcliff Lake, NJ www.eisai.com Morphotek Exton, PA Eleven Biotherapeutics bladder Phase III (mAb conjugated to cytotoxic agent) Cambridge, MA www.elevenbio.com ORPHAN DRUG

head and neck (Fast Track) Phase II www.elevenbio.com

Medicines in Development: Immuno-Oncology 60 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase OncoQuest ovarian Phase II (anti-idiotype mAb) Edmonton, Canada www.oncoquestinc.com ORPHAN DRUG Aptevo Therapeutics CLL Phase II (CD37 protein inhibitor) Seattle, WA www.aptevotherapeutics.com ORPHAN DRUG

PCA062 Novartis Pharmaceuticals p-CAD positive solid tumors Phase I (antibody-drug conjugate) East Hanover, NJ www.novartis.com

Perjeta® Genentech early HER2-positive breast, early breast Phase III pertuzumab South San Francisco, CA (adjuvant), advanced HER2-positive gastric www.gene.com

PF-05082566 Pfizer solid tumors Phase I (4-1BB agonist antibody) New York, NY www.pfizer.com

solid tumors (combination therapy) Phase I www.pfizer.com

PF-05280014 Pfizer metastatic breast cancer Phase III (trastuzumab biosimilar) New York, NY www.pfizer.com

PF-05280586 Pfizer follicular lymphoma Phase III (rituximab biosimilar) New York, NY www.pfizer.com

Medicines in Development: Immuno-Oncology 61 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

PF-06439535 Pfizer NSCLC Phase III (bevacizumab biosimilar) New York, NY www.pfizer.com

PF-06647020 Pfizer solid tumors Phase I (PTK7 protein inhibitor) New York, NY www.pfizer.com Stemcentrx South San Francisco, CA

PF-06647263 Pfizer solid tumors Phase I ( A4 antibody-drug conjugate) New York, NY www.pfizer.com

PF-06688992 Pfizer multiple myeloma Phase I (GD3 antibody-drug conjugate) New York, NY www.pfizer.com

PF-06747143 Pfizer AML Phase I (CXCR4 receptor antagonist) New York, NY www.pfizer.com (MDV9300) Medivation DLBCL Phase II New York, NY www.medivation.com Genentech hematological malignancies Phase II (anti-CD79b antibody) South San Francisco, CA www.gene.com Nascent Biotech glioma Phase II (natural human IgG1κ antibody) Vero Beach, FL www.nascentbiotech.com ORPHAN DRUG

Medicines in Development: Immuno-Oncology 62 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

Resimmune® Angimmune cutaneous T-cell lymphoma, Phase I/II anti-CD3 immunotoxin Rockville, MD malignant melanoma www.angimmune.com

RG6058 Genentech solid tumors Phase I (anti-TIGIT mAb) South San Francisco, CA www.gene.com

RG7876 Genentech solid tumors (with atezolizumab), Phase I (CD40 antigen stimulant) South San Francisco, CA solid tumors (with vanucizumab) www.gene.com

RG7882 Genentech ovarian, pancreatic Phase I (anti-MUC16 antibody-drug conjugate) South San Francisco, CA www.gene.com

RG7986 Genentech non-Hodgkin lymphoma Phase I (antibody-drug conjugate) South San Francisco, CA www.gene.com

Rituxan® Genentech CLL (subcutaneous formulation) application submitted rituximab South San Francisco, CA www.gene.com

relapsed/refractory CLL Phase III (with venetoclax) www.gene.com

relapsed/refractory follicular lymphoma Phase II (with venetoclax) www.gene.com

RM-1929 Aspyrian Therapeutics head and neck Phase I (antibody-drug conjugate) San Diego, CA www.aspyriantherapeutics.com

Medicines in Development: Immuno-Oncology 63 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase rosmantuzumab OncoMed Pharmaceuticals colorectal Phase I (RSPO3 protein inhibitor) Redwood City, CA www.oncomed.com

Rova-T AbbVie SCLC Phase II (rovalpituzumab tesirine) North Chicago, IL www.abbvie.com ORPHAN DRUG Stemcentrx South San Francisco, CA neuroendocrine tumors Phase I www.abbvie.com (IMMU-132) Immunomedics metastatic triple negative breast Phase II (antibody-drug conjugate) Morris Plains, NJ (Fast Track & Breakthrough Therapy), www.immunomedics.com solid tumors AML Phase II (CD200 antigen inhibitor) New Haven, CT www.alexion.com

solid tumors Phase I www.alexion.com

SAR408701 Sanofi US solid tumors Phase I (maytansin-loaded anti-CEACAM5 Bridgewater, NJ www.sanofi.com mAb)

SAR428926 Sanofi US solid tumors Phase I (mattansin-loaded anti-Lamp1 mAb) Bridgewater, NJ www.sanofi.com

Medicines in Development: Immuno-Oncology 64 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

SAR566658 Sanofi US solid tumors Phase I (maytansin-loaded anti-CA6 mAb) Bridgewater, NJ www.sanofi.com

SC-002 AbbVie solid tumors Phase I (antibody-drug conjugate) North Chicago, IL www.abbvie.com Stemcentrx South San Francisco, CA

SC-003 AbbVie solid tumors Phase I (antibody-drug conjugate) North Chicago, IL www.abbvie.com Stemcentrx South San Francisco, CA

SC-006 AbbVie solid tumors Phase I (antibody-drug conjugate) North Chicago, IL www.abbvie.com Stemcentrx South San Francisco, CA

SEA-CD40 Seattle Genetics metastatic or unresectable solid tumors, Phase I (antibody-drug conjugate) Bothell, WA hematologic malignancies www.seattlegenetics.com Merrimack Pharmaceuticals colorectal, head and neck, Phase II (ERBB-3 receptor antagonist) Cambridge, MA NSCLC (combination therapy) (Fast Track) www.merrimack.com

SGN-2FF Seattle Genetics solid tumors Phase I (antibody-drug conjugate) Bothell, WA www.seattlegenetics.com

Medicines in Development: Immuno-Oncology 65 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

SGN-CD19B Seattle Genetics non-Hodgkin lymphoma Phase I (antibody-drug conjugate) Bothell, WA www.seattlegenetics.com

SGN-CD123A Seattle Genetics AML Phase I (antibody-drug conjugate) Bothell, WA www.seattlegenetics.com

SGN-CD352A Seattle Genetics multiple myeloma Phase I (antibody-drug conjugate) Bothell, WA www.seattlegenetics.com

SGN-LIV1A Seattle Genetics metastatic breast Phase I (antibody-drug conjugate) Bothell, WA www.seattlegenetics.com

SNDX-6352 Syndax Pharmaceuticals solid tumors Phase I (anti-CSF-1R) Waltham, MA www.syndax.com

STI-001 Sorrento Therapeutics colorectal Phase III ( biobetter) San Diego, CA www.sorrentotherapeutics.com

Sylvant® Janssen Research & Development smoldering multiple myeloma Phase II Raritan, NJ www.janssen.com (interleukin-6 inhibitor)

Sym004 Symphogen colorectal, glioblastoma Phase II (/modotuximab) Somerville, NJ www.symphogen.com

squamous NSCLC (combination therapy) Phase I colorectal (combination therapy) www.symphogen.com

Medicines in Development: Immuno-Oncology 66 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

Sym013 Symphogen cancer Phase I/II (EGFR/ERBB-2/ERBB-3 receptor Somerville, NJ www.symphogen.com antagonist)

Sym015 Symphogen solid tumors Phase I/II ( Somerville, NJ www.symphogen.com receptor antagonist)

SYN004 Synermore Biologics solid tumors Phase I (EGFR inhibitor) Taipei, Taiwan www.synermore.com

TAK-202 (plozalizumab) Takeda Oncology solid tumors, melanoma (with checkpoint Phase I (CCR2 receptor antagonist) Cambridge, MA inhibitor) www.takeda.com OncoMed Pharmaceuticals pancreatic, SCLC Phase II (anti-notch-2/3 Mab) Redwood City, CA www.oncomed.com ORPHAN DRUG

TB-403 BioInvent International relapsed/refractory medulloblastoma Phase II (placenta growth factor inhibitor) Lund, Sweden www.bioinvent.se Oncurious www.oncurious.com Leuven, Belgium Neuroblastoma and Medulloblastoma Translational Research Consortium Grand Rapids, MI tisotumab vedotin Genmab solid tumors Phase I/II (HuMax-TF antibody-drug conjugate) Princeton, NJ www.genmab.com

Medicines in Development: Immuno-Oncology 67 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase trastuzumab biosimilar Biocon metastatic HER2-positive breast application submitted (ERBB-2 receptor antagonist) Bangalore, India www.biocon.com Mylan www.mylan.com Canonsburg, PA

TRC105 TRACON Pharmaceuticals hemangiosarcoma Phase III (ENG protein inhibitor) San Diego, CA www.traconpharma.com ORPHAN DRUG

hepatocellular, renal cell (Fast Track), Phase II gestational trophoblastic neoplasia www.traconpharma.com

breast, lung Phase I www.traconpharma.com (TG-1101) TG Therapeutics high-risk CLL (combination therapy), Phase III (CD20 antigen inhibitor) New York, NY DLBCL (combination therapy) www.tgtherapeutics.com ORPHAN DRUG Bristol-Myers Squibb AML Phase I (CXCR4 receptor antagonist) Princeton, NJ www.bms.com Bristol-Myers Squibb advanced solid tumors, advanced Phase II (CD137 antigen agonist) Princeton, NJ B-cell non-Hodgkin lymphoma www.bms.com

Medicines in Development: Immuno-Oncology 68 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase Seattle Genetics AML (elderly) Phase III (CD33 antigen inhibitor/cytoadhesin Bothell, WA www.seattlegenetics.com receptor antagonist/DNA cross linking agent) ORPHAN DRUG myelodysplastic syndromes Phase II www.seattlegenetics.com

AML (1L) Phase I www.seattlegenetics.com Celldex Therapeutics glioblastoma, colorectal, head and neck Phase II (CD27 antigen inhibitor) Hampton, NJ www.celldex.com Immunomedics non-Hodgkin lymphoma Phase II (CD20 antigen inhibitor) Morris Plains, NJ www.immunomedics.com

VX15 Vaccinex NSCLC Phase I (CD10 antigen inhibitor) Rochester, NY www.vaccinex.com xentuzumab (BI-836845) Boehringer Ingelheim Pharmaceuticals prostate Phase I/II (somatomedin inhibitor) Ridgefield, CT www.boehringer-ingelheim.com

Xgeva® Amgen delay or prevention of bone metastases Phase III Thousand Oaks, CA in patients with adjuvant breast www.amgen.com

Medicines in Development: Immuno-Oncology 69 Antibody-Drug Conjugates & Tumor Antigen Specific Antibodies l Monocloncal Antibodies Drug Name Organization Indication Development Phase

Xilonix™ XBiotech colorectal cancer (Fast Track) Phase III anti-IL-1-alpha mAb Austin, TX www.xbiotech.com

NSCLC Phase II www.xbiotech.com

XMT-1522 Mersana Therapeutics breast Phase I (anti-HER2 antibody) Cambridge, MA www.mersana.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 May 26, 2017. The medicines in this listing include medicines being developed by U.S.-based companies conducting trials in the United States and abroad, PhRMA-member companies conducting trials in the United States and abroad, and foreign companies conducting clinical trials in the United States. Some products may not be in active clinical trials. The information may not be comprehensive. For more, specific information about a particular product, contact the individual company directly or go to www.clinicaltrials.gov. The entire series of Medicines in Development is available on PhRMA's website, www.phrma.org.

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.

Medicines in Development: Immuno-Oncology 70 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 .

Phase II—The investigational drug or biologic is given to volunteer patients, usually between 100 and 500, to determine whether it is effective, identify an optimal dose, and to further evaluate its short-term safety.

Phase III—The investigational drug or biologic is given to a larger, more diverse patient population, often involving between 1,000 and 5,000 patients (but sometimes many more), to generate statistically significant evidence to confirm its safety and effectiveness. Phase III studies are the longest studies and usually take place in multiple sites around the world.

Medicines in Development: Immuno-Oncology 71