2013 REPORT

America’s biopharmaceutical research companies are developing 907 biologics targeting more than 100 diseases. Biologics are developed through biological processes using living cells or organisms.

This report lists biologics in human clinical trials or under review by the U.S. Food and Drug Administration (FDA). A link to the sponsor company’s web site provides more detailed infor-mation on the science and technologies behind each potential product. The 907 medicines and vaccines in development include:

• 338 cancer therapeutics that target several different types of solid tumors, leukemia and lymphoma. Monoclonal antibodies account for 170 of the 338 products in development.

• 176 candidates in development for an array of infectious diseases, including 134 vaccines.

• 71 medicines for autoimmune diseases, such as lupus, multiple sclerosis and rheumatoid arthritis.

• 58 treatments for cardiovascular diseases, such as congestive heart failure and stroke.

• Other diseases include diabetes, digestive disorders, genetic disorders, neurologic and respiratory disorders.

Definitions for selected product categories and diseases can be found on page 86.

For information on the history and future of biologics research, the science behind the medicines, and a discussion of issues critical to continued discovery and development of these cutting-edge medicines, please see Biologic Medicines in Development 2013—Overview.

The medicines in this report reflect the new ways America’s biopharmaceuti-cal research companies are attacking disease through biotechnology. The 907 medicines and vaccines in development promise to push the frontiers of science and bring new treatments to patients for our more challenging diseases.

Biologics Research Pushing Frontiers of Science With More Than 900 Medicines and Vaccines in Development

Medicines in developMent

Biologicspresented By aMerica’s BiopharMaceutical research coMpanies

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Phase III

Phase II

Phase I

Biologics In Development By Product Category and Development Phase

In the human body there are 12 trillion cells, 200,000 proteins and 25,000 genes

Medicines in Development Biologics 20132

Biologic Medicines in Development

Autoimmune DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase*

ABT-122 AbbVieNorth Chicago, IL

mAb rheumatoid arthritis Phase Iabbvie.com

Actemra® tocilizumab

GenentechSouth San Francisco, CARocheNutley, NJ

mAb early rheumatoid arthritis

------------------------------------------------systemic sclerosis

Phase IIIgene.com--------------------------------Phase IIgene.com

AGS-009 Argos TherapeuticsDurham, NC

mAb systemic lupus erythematosus (SLE)

Phase Iargostherapeutics.com

alemtuzumab GenzymeCambridge, MA

mAb relapsing-remitting multiple sclerosis (Fast Track)

application submittedgenzyme.com

Alferon N Injection®

interferon-alpha-n3subcutaneous

Hemispherx BiosciencePhiladelphia, PA

interferon multiple sclerosis Phase IIhemispherx.net

AMG 557/MEDI-5872 Amgen Thousand Oaks, CA AstraZenecaWilmington, DEMedImmuneGaithersburg, MD

mAb SLE Phase Iamgen.comastrazeneca.commedimmune.com

AMG 729 AmgenThousand Oaks, CA

mAb autoimmune diseases Phase Iamgen.com

AMG 811 Amgen Thousand Oaks, CA

mAb discoid lupus erythematosus, SLE

Phase Iamgen.com

anti-IL17 mAb(RG7624)

GenentechSouth San Francisco, CA

mAb autoimmune disorders Phase Igene.com

anti-LINGO(BIIB033)

Biogen IdecWeston, MA

mAb multiple sclerosis(see also eye)

Phase IIbiogenidec.com

ARX424 (PEGylated interferonbeta-1a)

AmbrxLa Jolla, CA

interferon multiple sclerosis Phase Iambrx.com

atacicept (TACI-lg)

EMD Serono Rockland, MA

recombinantfusionprotein

SLE Phase II/IIIemdserono.com

*For more information about a specific medicine or company in the report, please click on the provided link.

Medicines in Development Biologics 2013 3

Biologic Medicines in Development

Autoimmune DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

ATX-MS-1467 EMD SeronoRockland, MA

peptidevaccine

multiple sclerosis Phase Iemdserono.com

AZ01(long-acting pegylated interferon beta)

AllozyneSeattle, WA

interferon multiple sclerosis Phase Iallozyne.com

Benlysta® belimumab (subcutaneous)

GlaxoSmithKline Rsch. Triangle Park, NC

mAb SLE (see also blood, transplantation)------------------------------------------------systemic scleroderma

Phase IIIgsk.com--------------------------------gsk.com

BHT-3009 Bayhill TherapeuticsPalo Alto, CA

DNA vaccine

multiple sclerosis Phase IIbayhilltx.com

BI-695500(rituximab biosimilar)

Boehringer-IngelheimPharmaceuticalsRidgefield, CT

mAb rheumatoid arthritis Phase IIIboehringer-ingelheim.com

BI-695501(adalimumab biosimilar)

Boehringer IngelheimPharmaceuticalsRidgefield, CT

mAb rheumatoid arthritis Phase Iboehringer-ingelheim.com

BIIB017 (PEG-interferonbeta-1a)

Biogen Idec Weston, MA

interferon relapsing multiple sclerosis (Fast Track)

Phase IIIbiogenidec.com

BT-061 AbbVieNorth Chicago, ILBiotestDreieich, Germany

mAb rheumatoid arthritis (see also skin)

Phase IIabbvie.combiotest.de

Cimzia®

certolizumab pegolUCBSmyrna, GA

mAb ankylosing spondylitis, juvenile rheumatoid arthritis(see also musculoskeletal)

Phase IIIucb.com

clazakizumab(anti-IL6)

Bristol-Myers Squibb Princeton, NJ Alder Biopharmaceuticals Bothell, WA

mAb rheumatoid arthritis (see also digestive, musculoskeletal)

Phase IIbms.comalderbio.com

CNTO-136 (sirukumab)

Janssen BiotechHorsham, PA

mAb rheumatoid arthritis(see also other)

Phase IIIjanssenbiotech.com

CNTO-1959 Janssen BiotechHorsham, PA

mAb rheumatoid arthritis(see also skin)

Phase IIjanssenbiotech.com

Medicines in Development Biologics 20134

Biologic Medicines in Development

Autoimmune DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

daclizumab AbbVie North Chicago, ILBiogen Idec Weston, MA

mAb multiple sclerosis (relapsing forms) Phase IIIabbvie.combiogenidec.com

epratuzumab Immunomedics Morris Plains, NJUCBSmyrna, GA

mAb SLE (Fast Track)

Phase IIIimmunomedics.comucb.com

GSK1223249(NOGO-A mAb)

GlaxoSmithKlineRsch. Triangle Park, NC

mAb multiple sclerosis(see also neurologic)

Phase Igsk.com

Humira® adalimumab

AbbVieNorth Chicago, IL

mAb rheumatoid arthritis (combination therapy) (see also digestive, eye, musculoskeletal, skin, other)

Phase IIIabbvie.com

Ilaris®

canakinumab(Orphan Drug)

Novartis PharmaceuticalsEast Hanover, NJ

mAb systemic juvenile idiopathic arthritis(see also cardiovascular, diabetes, musculoskeletal)

Phase IIInovartis.com

ISIS-CRPRx Isis PharmaceuticalsCarlsbad, CA

antisense rheumatoid arthritis(see also cardiovascular)

Phase IIisispharm.com

mavrilimumab AstraZenecaWilmington, DEMedImmuneGaithersburg, MD

mAb rheumatoid arthritis Phase IIastrazeneca.commedimmune.com

MEDI-546(anti-IFN-alphaR mAb)

AstraZenecaWilmington, DEMedImmuneGaithersburg, MD

mAb SLE Phase IIastrazeneca.commedimmune.com

MEDI-551 (anti-CD19 mAb)

AstraZeneca Wilmington, DE MedImmune Gaithersburg, MD

mAb scleroderma (see also cancer)

Phase Iastrazeneca.commedimmune.com

MEDI-570 (anti-ICOS mAb)

AstraZeneca Wilmington, DE MedImmuneGaithersburg, MD

mAb SLE Phase Iastrazeneca.commedimmune.com

Medicines in Development Biologics 2013 5

Biologic Medicines in Development

Autoimmune DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

NeuroVax™(IR208)

Immune Response BiopharmaNew York, NY

peptide vaccine

multiple sclerosis Phase IIimmuneresponsebio pharma.com

NN8209(anti-C5aR-151 mAb)

Novo NordiskPrinceton, NJ

mAb rheumatoid arthritis Phase IInovonordisk.com

NN8210(anti-C5aR-215 mAb)

Novo NordiskPrinceton, NJ

mAb rheumatoid arthritis Phase Inovonordisk.com

NN8226 (anti-IL-20 mAb)

Novo Nordisk Princeton, NJ

mAb rheumatoid arthritis Phase IInovonordisk.com

NN8765(anti-NKG2 mAb)

Novo NordiskPrinceton, NJ

mAb rheumatoid arthritis Phase Inovonordisk.com

NN8828 (anti-IL-21 mAb)

Novo Nordisk Princeton, NJ

mAb rheumatoid arthritis

--------------------------------SLE

Phase IInovonordisk.com--------------------------------Phase Inovonordisk.com

Nplate™ romiplostim (Orphan Drug)

Amgen Thousand Oaks, CA

recombinant fusion protein

immune thrombocytopenic purpura in children(see also cancer)

Phase IIIamgen.com

NU-100(interferon beta-1b)

Nuron BiotechExton, PA

recombinantinterferon

multiple sclerosis Phase IIInuronbiotech.com

ocrelizumab (anti-CD20 mAb)

Biogen Idec Weston, MA Genentech South San Francisco, CA RocheNutley, NJ

mAb primary-progressive multiple sclerosis

------------------------------------------------relapsing-remitting multiple sclerosis

Phase IIIbiogenidec.comgene.com---------------------------------Phase IIIbiogenidec.comgene.com

ofatumumab(subcutaneous)

GlaxoSmithKlineRsch. Triangle Park, NC

mAb multiple sclerosis, rheumatoid arthritis

Phase IIgsk.com

olokizumab

UCB Smyrna, GA

mAb

rheumatoid arthritis

Phase IIucb.com

Orencia® abatacept

Bristol-Myers Squibb Princeton, NJ

recombinant fusion protein

early rheumatoid arthritis (see also musculoskeletal, other)

Phase IIIbms.com

Medicines in Development Biologics 20136

Biologic Medicines in Development

Autoimmune DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

otelixizumab (anti-CD3 mAb)

GlaxoSmithKline Rsch. Triangle Park, NC

mAb rheumatoid arthritis Phase Igsk.com

ozoralizumab (ATN-103)

AblynxGhent, Belgium

mAb rheumatoid arthritis Phase II completedablynx.com

pateclizumab(anti-LT alpha mAb)

Genentech South San Francisco, CA

mAb rheumatoid arthritis Phase IIgene.com

PD-360324 PfizerNew York, NY

mAb cutaneous lupus erythematosus Phase Ipfizer.com

PDA-001 (cenplacel-L)

Celgene Cellular TherapeuticsSummit, NJ

stem cell therapy

rheumatoid arthritis (see also digestive) ------------------------------------------------multiple sclerosis, sarcoidosis

Phase IIcelgene.com---------------------------------Phase Icelgene.com

PF-04236921 PfizerNew York, NY

mAb SLE (see also digestive)------------------------------------------------rheumatoid arthritis

Phase IIpfizer.com---------------------------------Phase Ipfizer.com

PF-05280586(rituximab biosimilar)

PfizerNew York, NY

mAb rheumatoid arthritis Phase I/IIpfizer.com

plovamer acetate(second-generationpeptide copolymer)

EMD SeronoRockland, MA

peptide multiple sclerosis Phase Iemdserono.com

Prolia®

denosumabAmgen Thousand Oaks, CA

mAb rheumatoid arthritis (see also musculoskeletal)

Phase IIamgen.com

RAVAX™rheumatoid arthritis vaccine

Immune Response BioPharmaNew York, NY

vaccine rheumatoid arthritis Phase IIIimmuneresponsebio pharma.com

rontalizumab(RG7415)

Genentech South San Francisco, CA

mAb SLE Phase IIgene.com

SAN-300 (anti-VLA-1 antibody)

Santarus San Diego, CA

mAb

rheumatoid arthritis Phase Isantarus.com

Medicines in Development Biologics 2013 7

Biologic Medicines in Development

Autoimmune DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

SAR113244(anti-CXCR5 mAb)

Sanofi USBridgewater, NJ

mAb SLE Phase Isanofi.com

sarilumab(SAR153191)

Regeneron PharmaceuticalsTarrytown, NYSanofi US Bridgewater, NJ

mAb rheumatoid arthritis Phase IIIregeneron.comsanofi.com

secukinumab (AIN457)

Novartis Pharmaceuticals East Hanover, NJ

mAb ankylosing spondylitis, rheumatoid arthritis(see also eye, musculoskeletal, skin) ------------------------------------------------multiple sclerosis

Phase IIInovartis.com

---------------------------------Phase IInovartis.com

sifalimumab(anti-IFN-alpha mAb)

AstraZeneca Wilmington, DEMedImmuneGaithersburg, MD

mAb SLE Phase IIastrazeneca.commedimmune.com

Simponi®golimumab(Orphan Drug)

Janssen BiotechHorsham, PA

mAb rheumatoid arthritis(intravenous)(see also digestive)------------------------------------------------juvenile rheumatoid arthritis (subcutaneous)-----------------------------------------------sarcoidosis(subcutaneous)

application submittedjanssenbiotech.com

---------------------------------Phase IIIjanssenbiotech.com---------------------------------Phase II completedjanssenbiotech.com

Soliris®

eculizumab(Orphan Drug)

Alexion PharmaceuticalsCheshire, CT

mAb severe or refractory myasthenia gravis (see also eye, infectious, transplantation)

Phase IIalxn.com

Stelara®

ustekinumabJanssen BiotechHorsham, PA

mAb rheumatoid arthritis, sarcoidosis(see also digestive, musculoskeletal, other)

Phase IIjanssenbiotech.com

tabalumab(BAFF inhibitor)

Eli Lilly Indianapolis, IN

mAb SLE(see also cancer)

Phase IIIlilly.com

Tcelna™ imilecleucel-T

Opexa Therapeutics The Woodlands, TX

cell therapyvaccine

multiple sclerosis (Fast Track)

Phase IIopexatherapeutics.com

Tysarbi®natalizumab

Biogen IdecWeston, MA

mAb secondary-progressive multiple sclerosis

Phase IIIbiogenidec.com

Medicines in Development Biologics 20138

Biologic Medicines in Development

Autoimmune DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

veltuzumab (IMMU-106)

ImmunomedicsMorris Plains, NJ Takeda Pharmaceuticals USADeerfield, IL

mAb immune thrombocytopenic purpura, rheumatoid arthritis (see also cancer)

Phase IIimmunomedics.comtakeda.com

VX15 Teva PharmaceuticalsTikva, IsraelVaccinexRochester, NY

mAb multiple sclerosis(see also cancer)

Phase Itevapharm.comvaccinex.com

XmAb®5871(anti-CD19 mAb)

XencorMonrovia, CA

mAb autoimmune disorders Phase Ixencor.com

BlooD DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

ACE-536 Acceleron PharmaCambridge, MACelgeneSummit, NJ

recombinant fusionprotein

anemia Phase Iacceleronpharma.comcelgene.com

ALXN1102/ALXN1103 Alexion PharmaceuticalsCheshire, CT

recombinant fusionprotein

paroxysmal nocturnal hemoglobinuria

Phase Ialxn.com

AMT-060(Orphan Drug)

uniQureAmsterdam, The NetherlandsSt. Jude Children’s ResearchHospitalMemphis, TN

gene therapy hemophilia B Phase I/IIuniqure.com

ART123 (recombinant human thrombomodulin alpha)

Asahi Kasei Pharma America Waltham, MA

recombinantprotein

disseminated intravascular coagulation

Phase IIIakpamerica.com

AskBio009 Asklepios BioPharmaceuticalChapel Hill, NCBaxter InternationalDeerfield, IL

gene therapy hemophilia B Phase I/IIaskbio.combaxter.com

ATryn®

antithrombin alfa(Orphan Drug)

GTC BiotherapeuticsFramingham, MA

recombinantprotein

heparin resistance in patients under-going CABG surgery

Phase IIIgtc-bio.com

Medicines in Development Biologics 2013 9

Biologic Medicines in Development

BlooD DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

BAX-111(recombinantvon Willebrandfactor)(Orphan Drug)

Baxter InternationalDeerfield, IL

recombinantprotein

von Willebrand disease Phase IIIbaxter.com

BAX-326(rFIX)(Orphan Drug)

Baxter InternationalDeerfield, IL

recombinant protein

hemophilia B application submittedbaxter.com

BAX-817(rFVIIa)

Baxter InternationalDeerfield, IL

recombinant protein

hemophilia A, hemophilia B Phase I/IIbaxter.com

BAX-855(rFVIII)

Baxter InternationalDeerfield, IL

recombinantprotein

hemophilia A Phase Ibaxter.com

BAY 86-6150(rFVIIa)

Bayer HealthCarePharmaceuticalsWayne, NJ

recombinantprotein

hemophilia A, hemophilia B Phase II/IIIbayerpharma.com

BAY 94-9027(rFVIII)

Bayer HealthCarePharmaceuticalsWayne, NJ

recombinantprotein

hemophilia A Phase II/IIIbayerpharma.com

Benlysta®

belimumab(oral)

GlaxoSmithKlineRsch. Triangle Park, NC

mAb vasculitis(see also autoimmune,transplantation)

Phase I completedgsk.com

beta-globingene therapy(lentiglobin)

bluebird bioCambridge, MAINSERMParis, France

genetherapy

beta-thalassemia, sickle cell anemia

Phase I/IIbluebirdbio.com

BI-655075 Boehringer IngelheimPharmaceuticalsRidgefield, CT

mAb blood coagulation disorders Phase Iboehringer-ingelheim.com

CSL-627(rVIII-SC)

CSL BehringKing of Prussia, PA

recombinant protein

hemophilia A Phase Icslbehring.com

CSL-654(rIX-FP)(Orphan Drug)

CSL BehringKing of Prussia, PA

recombinantfusionprotein

hemophilia B Phase II/IIIcslbehring.com

CSL-689(rVIIa-FP)(Orphan Drug)

CSL BehringKing of Prussia, PA

recombinant fusionprotein

hemophilia A, hemophilia B Phase Icslbehring.com

Medicines in Development Biologics 201310

Biologic Medicines in Development

BlooD DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

CYT107(interleukin-7)

CytherisRockville, MD

recombinant interleukin

idiopathic CD4-positivelymphocytopenia(see also cancer, infectious, transplantation)

Phase I/IIcytheris.com

epoetin alfa biosimilar HospiraLake Forest, IL

recombinanterythropoietin

anemia Phase IIIhospira.com

erythropoietin gene therapy

MedgenicsSan Francisco, CA

gene therapy anemia Phase IImedgenics.com

factor VIII/von Willebrandfactor

CSL BehringKing of Prussia, PA

recombinant protein

hemophilia A,von Willebrand disease

Phase IIIcslbehring.com

ferroportin mAb Eli LillyIndianapolis, IN

mAb anemia Phase Ililly.com

hepcidin mAb Eli LillyIndianapolis, IN

mAb anemia Phase Ililly.com

HM10760A Hanmi PharmaceuticalSeoul, South Korea

long-actingerythropoietin

anemia Phase Ihanmipharm.com

humanrecombinant factor VIII

Octapharma USAHoboken, NJ

recombinantprotein

hemophilia A Phase IIoctapharma.com

IB1001(recombinant factor IX biosimilar)

Inspiration BiopharmaceuticalsCambridge, MAIpsenParis, France

recombinantprotein

hemophilia B application submittedinspirationbio.com

ISIS-FXIRx Isis PharmaceuticalsCarlsbad, CA

antisense clotting disorders Phase IIisispharm.com

NAV™ therapeutic ReGenX BiosciencesWashington, DC

gene therapy hemophilia B(see also genetic)

Phase I/IIregenxbio.com

NiCord®

umbilical cord bloodstem cell therapy

Gamida CellJerusalem, Israel

stem cell therapy

sickle cell anemia(see also cancer)

Phase I/IIgamida-cell.com

NN1841(rFXIII)

Novo Nordisk Princeton, NJ

recombinant protein

congenital factor XIII deficiency application submittednovonordisk.com

NN7008(turoctocog alfa)

Novo NordiskPrinceton, NJ

recombinantprotein

hemophilia A application submittednovonordisk.com

Medicines in Development Biologics 2013 11

Biologic Medicines in Development

BlooD DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

NN7088(N8-GP, rFVIII glycopegylated)

Novo NordiskPrinceton, NJ

recombinantprotein

hemophilia A Phase IIInovonordisk.com

NN7999(N9-GP, rFIXglycopegylated)

Novo NordiskPrinceton, NJ

recombinant protein

hemophilia B Phase IIInovonordisk.com

OBI-1(recombinantporcine factor VIII)(Orphan Drug)

Inspiration BiopharmaceuticalsCambridge, MAIpsenParis, France

recombinantprotein

hemophilia A(Fast Track)

Phase IIIinspirationbio.com

PF-05280602(rhFVIIa)

Catalyst BiosciencesSouth San Francisco, CAPfizerNew York, NY

recombinant protein

hemophilia Phase Icatalystbiosciences.compfizer.com

PRT4445(FXa inhibitor antidote)

Portola PharmaceuticalsSouth San Francisco, CA

recombinant protein

hemorrhage Phase IIportola.com

recombinant factor VIII-Fc (Orphan Drug)

Biogen IdecWeston, MA

recombinantfusionprotein

hemophilia A(Fast Track)

Phase IIIbiogenidec.com

recombinant factor IX-Fc (Orphan Drug)

Biogen IdecWeston, MA

recombinant fusion protein

hemophilia B (Fast Track)-----------------------------------------------hemophilia B in children (Fast Track)

application submittedbiogenidec.com---------------------------------Phase IIIbiogenidec.com

SelG1 Selexys PharmaceuticalsOklahoma City, OK

mAb sickle cell anemia Phase I completedselexys.com

sotatercept(ACE-011)

Acceleron Pharma Cambridge, MA Celgene Summit, NJ

recombinantfusionprotein

anemia in patients with end-stage renal disease(see also cancer)

Phase IIacceleronpharma.comcelgene.com

TT-173 ThrombotargetsDurham, NC

recombinant protein

blood coagulation disorders Phase Ithrombotargets.com

Wilate®

von Willebrand factor/coagulation factor VIII complex (human)

Octapharma USAHoboken, NJ

recombinant protein

surgical blood loss Phase IIIoctapharma.com

Medicines in Development Biologics 201312

Biologic Medicines in Development

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

8H9 mAb United Therapeutics Silver Spring, MD

mAb metastatic brain cancer Phase Iunither.com

212-Pb-TCMC- trastuzumab

AREVA MedBethesda, MD

mAb HER2-positive cancer metastasized to the abdominal region

Phase Iarevamed.com

AbGn-7 AbGenomics InternationalLos Altos, CA

mAb solid tumors Phase I

ABIO-0501(TALL-104)

Abiogen Pharma Pisa, Italy

cell therapy chronic myeloid leukemia (CML) Phase IIabiogen.it

ABT-806 AbbVieNorth Chicago, IL

mAb solid tumors Phase Iabbvie.com

Actimab-A(M195 mAb)

Actinium Pharmaceuticals New York, NY

mAb acute myeloid leukemia (AML) Phase I/IIactiniumpharmaceuti cals.com

Adcetris®

brentuximab vedotin(Orphan Drug)

Seattle GeneticsBothell, WA

mAb cutaneous T-cell lymphoma, front-line Hodgkin lymphoma,post-transplant Hodgkin lymphomarelapse prevention------------------------------------------------non-Hodgkin lymphoma,non-lymphoma malignancies, CD30-positive hematologicmalignancies, CD30-positive T-cell lymphoma

Phase IIIseattlegenetics.com

---------------------------------Phase IIseattlegenetics.com

AD-IL-12 ZIOPHARM OncologyNew York, NY

gene therapy malignant melanoma Phase I/IIziopharm.com

ADI-PEG-20 Polaris PharmaceuticalsSan Diego, CA

pegylated protein

liver cancer

------------------------------------------------malignant melanoma, mesothelioma, small-cell lung cancer (SCLC)

Phase IIIpolarispharma.com---------------------------------Phase IIpolarispharma.com

ADXS-HPV AdvaxisPrinceton, NJ

vaccine cervical cancer, cervical intraepithelial neoplasia

Phase IIadvaxis.com

AE08 Antigen ExpressWorcester, MA

peptide vaccine

malignant melanoma Phase Iantigenexpress.com

AE37 Antigen Express Worcester, MA

peptidevaccine

breast cancer Phase IIantigenexpress.com

Medicines in Development Biologics 2013 13

Biologic Medicines in Development

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

AGS-003 Argos Therapeutics Durham, NC

personalized dendritic cell vaccine

metastatic renal cancer (Fast Track)

Phase IIIargostherapeutics.com

ALD-151 CytomedixGaithersburg, MDDuke UniversityDurham, NC

stem cell therapy

leukemia Phase I completedcytomedix.com

AlloStim™cancer vaccine

Immunovative TherapiesShoham, Israel

cell vaccine hematological malignancies, solid tumors

Phase I/IIimmunovative.co.il

Allovectin® velimogene aliplasmid(Orphan Drug)

Vical San Diego, CA

DNA immunothera-peutic

malignant melanoma (Fast Track)

Phase IIIvical.com

ALN-VSP Alnylam PharmaceuticalsCambridge, MA

RNA interference

liver cancer, liver metastases Phase Ialnylam.com

ALT-801 Altor BioScience Miramar, FL

recombinant fusionprotein

AML, bladder cancer, malignant melanoma, multiple myeloma

Phase I/IIaltorbioscience.com

ALT-836 Altor BioScienceMiramar, FLGenentechSouth San Francisco, CA

mAb cancer(see also respiratory)

Phase I/IIaltorbioscience.comgene.com

AME-133v MENTRIK BiotechDallas, TX

mAb non-Hodgkin lymphoma Phase II mentrik.com

AMG 386 Amgen Thousand Oaks, CA

recombinant fusionprotein

fallopian tube cancer, ovarian cancer, peritoneal cancer------------------------------------------------breast cancer, colorectal cancer, gastric cancer, liver cancer, esophageal cancer, renal cancer

Phase IIIamgen.com---------------------------------Phase IIamgen.com

AMG 780 AmgenThousand Oaks, CA

antibody solid tumors Phase Iamgen.com

AMG 820 AmgenThousand Oaks, CA

mAb solid tumors Phase Iamgen.com

AMG 888(U3-1287)

Amgen Thousand Oaks, CA U3 PharmaMartinsried, Germany

mAb non-small-cell lung cancer (NSCLC) Phase IIamgen.com

Medicines in Development Biologics 201314

Biologic Medicines in Development

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

antibody-drug conjugate(RG7600)

GenentechSouth San Francisco, CA

mAb ovarian cancer, pancreatic cancer Phase Igene.com

anti-CD3-anti-HER2/neu-activated T-cells

TransTarget Hillsborough, CABarbara Ann Karmanos Cancer InstituteDetroit, MI

celltherapy

metastatic breast cancer Phase II

anti-CD22 ADC(RG7593)

Genentech South San Francisco, CASeattle GeneticsBothell, WA

mAb diffuse large B-cell lymphoma, non-Hodgkin lymphoma------------------------------------------------chronic lymphocytic leukemia (CLL)

Phase IIgene.com--------------------------------Phase Igene.com

anti-CD45 mAb Actinium PharmaceuticalsNew York, NY

mAb AML Phase Iactiniumpharmaceuti cals.com

anti-CD79b ADC(RG7596)

Genentech South San Francisco, CASeattle GeneticsBothell, WA

mAb diffuse large B-cell lymphoma, non-Hodgkin lymphoma------------------------------------------------CLL

Phase IIgene.com---------------------------------Phase Igene.com

anti-CXCR4 Bristol-Myers SquibbPrinceton, NJ

mAb hematological malignancies Phase Ibms.com

anti-EGFL7 mAb (RG7414)

Genentech South San Francisco, CA

mAb metastatic colorectal cancer, NSCLC------------------------------------------------solid tumors

Phase IIgene.com--------------------------------Phase Igene.com

anti-FGFR3 mAb (RG7444)

GenentechSouth San Francisco, CA

mAb solid tumors Phase Igene.com

anti-HER3/EGFR DAF mAb(RG7597)

Genentech South San Francisco, CA

mAb colorectal cancer, head and neck cancer

Phase IIgene.com

anti-PD-L1 Bristol-Myers Squibb Princeton, NJ

mAb

cancer(see also infectious)

Phase Ibms.com

anti-PD-L1 mAb(RG7446)

GenentechSouth San Francisco, CA

mAb melanoma, solid tumors Phase Igene.com

anti-STEAP1 ADC(RG7450)

GenentechSouth San Francisco, CASeattle GeneticsBothell, WA

mAb prostate cancer Phase Igene.com

Medicines in Development Biologics 2013 15

Biologic Medicines in Development

CanCer and related Conditions

Product Name Sponsor ProductCategory

Indication Development Phase

APN301 (hu14.18-IL2)

Apeiron BiologicsVienna, Austria

mAb malignant melanoma, neuroblastoma in children

Phase II completedapeiron-biologics.com

Archexin™ RX-0201 (Orphan Drug)

Rexahn Pharmaceuticals Rockville, MD

antisense pancreatic cancer Phase IIrexahn.com

Arzerra®

ofatumumab(Orphan Drug)

GlaxoSmithKlineRsch. Triangle Park, NC

mAb CLL (first-line therapy),diffuse large B-cell lymphoma, follicular lymphoma

Phase IIIgsk.com

ASC-101(Orphan Drug)

America Stem CellCarlsbad, CA

stem cell therapy

myeloablation associated with hematological malignancies

Phase I/IIamericastemcell.com

ASG-5ME AgensysSanta Monica, CASeattle GeneticsBothell, WA

mAb-drug conjugate

pancreatic cancer, castration-resistant prostate cancer

Phase Iagensys.comseattlegenetics.com

ASG-22ME AgensysSanta Monica, CASeattle GeneticsBothell, WA

mAb-drug conjugate

solid tumors Phase Iagensys.comseattlegenetics.com

autologous stem cell therapy

California Stem CellIrvine, CA

stem cell therapy

malignant melanoma Phase IIcaliforniastemcell.com

AV-203 AVEO OncologyCambridge, MA

mAb solid tumors Phase Iaveooncology.com

Avastin® bevacizumab (Orphan Drug)

Genentech South San Francisco, CA RocheNutley, NJ

mAb ovarian cancer

------------------------------------------------HER-2 negative-breast cancer, HER-2 positive breast cancer, high- risk carcinoid tumors, glioblastoma multiforme, metastatic ovarian cancer, NSCLC

application submittedgene.com---------------------------------Phase IIIgene.com

AVX701 AlphaVax Rsch. Triangle Park, NC

virus replicon particlevaccine

CEA-expressing colorectal cancer Phase I/IIalphavax.com

AVX901 AlphaVAxRsch. Triangle Park, NC

virus repliconparticle vaccine

HER2-expressing breast cancer Phase Ialphavax.com

B7-2/GM-CSF cancergene therapy(combination immunogene therapy)

NuVax Therapeutics(Radient Pharmaceuticals)Tustin, CA

genetherapy

cancer Phase I radient-pharma.com

Medicines in Development Biologics 201316

Biologic Medicines in Development

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

bavituximab Peregrine Pharmaceuticals Tustin, CA

mAb NSCLC, pancreatic cancer

------------------------------------------------breast cancer, liver cancer, prostate cancer, rectal adenocarcinoma

Phase IIperegrineinc.com--------------------------------Phase I/IIperegrineinc.com

BAX-69 Baxter InternationalDeerfield, IL

mAb solid tumors(see also other)

Phase Ibaxter.com

BAY 79-4620 Bayer HealthCare Pharmaceuticals Wayne, NJ

mAb solid tumors Phase Ibayerpharma.com

BAY 94-9343 Bayer HealthCare PharmaceuticalsWayne, NJ

mAb solid tumors Phase Ibayerpharma.com

BAY 20-10112 AmgenThousand Oaks, CABayer HealthCarePharmaceuticalsWayne, PA

mAb solid tumors Phase Iamgen.combayerpharma.com

BC-819(Orphan Drug)

BioCancell TherapeuticsJerusalem, Israel

genetherapy

bladder cancer, pancreatic cancer (Fast Track)------------------------------------------------ovarian cancer

Phase IIbiocancell.com---------------------------------Phase I/IIbiocancell.com

BHQ880 Novartis Pharmaceuticals East Hanover, NJ

mAb multiple myeloma Phase IInovartis.com

BI-505 (Orphan Drug)

BioInvent InternationalLund, Sweden

mAb multiple myeloma Phase Ibioinvent.com

BI-836845 Boehringer IngelheimPharmaceuticalsRidgefield, CT

mAb solid tumors Phase Iboehringer-ingelheim.com

BiovaxID® B-cell lymphoma vaccine

Biovest InternationalTampa, FL

autologousidiotypevaccine

follicular lymphoma (Fast Track)------------------------------------------------mantle-cell lymphoma

Phase IIIbiovest.com---------------------------------Phase IIbiovest.com

blinatumomab(Orphan Drug)

AmgenThousand Oaks, CA

mAb leukemia and lymphoma Phase IIamgen.com

BP-100-1.01(liposomal Grb-2)

Bio-Path HoldingsHouston, TX

antisense acute lymphocytic leukemia (ALL), AML, CML, myelodysplastic syndromes

Phase Ibiopathholdings.com

Medicines in Development Biologics 2013 17

Biologic Medicines in Development

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

BPX-101 Bellicum PharmaceuticalsHouston, TX

dendritic cellvaccine

prostate cancer Phase I/IIbellicum.com

breast cancer vaccine MabVax TherapeuticsSan Diego, CA

mAb vaccine breast cancer Phase Imabvax.com

BT-062(indatuximab ravtansine)(Orphan Drug)

BiotestDreieich, Germany

mAb multiple myeloma Phase I/IIbiotest.de

BYM338 Novartis PharmaceuticalsEast Hanover, NJ

mAb cancer-related cachexia(see also musculoskeletal)

Phase IInovartis.com

CALAA-01 Calando PharmaceuticalsPasadena, CA

RNAinterference

solid tumors Phase Iarrowheadresearch.com

calaspargase pegol Sigma-Tau PharmaceuticalsGaithersburg, MD

pegylatedenzyme

ALL in adolescents and children Phase IIIsigmatau.com

cancer medicine Eli LillyIndianapolis, IN

biologic cancer Phase Ililly.com

cancer medicine Eli LillyIndianapolis, IN

biologic cancer Phase Ililly.com

cancer vaccine Bayer HealthCare PharmaceuticalsWayne, NJStellar BiotechnologiesPort Hueneme, CA

autologous idiotype vaccine

follicular lymphoma Phase Ibayerpharma.com

cancer vaccine EMD MilliporeBillerica, MA

dendritic cellvaccine

malignant melanoma Phase I completedmillipore.com

cancer vaccine GradalisCarrollton, TXMary Crowley Cancer Research CenterDallas, TX

genetically-modified vaccine

solid tumors Phase Igradalisinc.commarycrowley.org

cancer vaccine Immunitor Vancouver, Canada

vaccine cancer Phase I/IIimmunitor.com

cancer vaccine(AdhTAP)

TapImmuneSeattle, WA

therapeutic vaccine

breast cancer Phase Itapimmune.com

catumaxomab Fresenius Biotech North AmericaWaltham, MA

mAb malignant ascites,ovarian cancer

Phase II completedfresenius-biotech.com

Medicines in Development Biologics 201318

Biologic Medicines in Development

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

CDX-011(glembatumumab vedotin)

Celldex TherapeuticsNeedham, MA

mAb breast cancer (Fast Track), malignant melanoma

Phase IIcelldextherapeutics.com

CDX-1127 Celldex Therapeutics Needham, MA

mAb hematological malignancies, solid tumors

Phase Icelldextherapeutics.com

CDX-1401 Celldex Therapeutics Needham, MA

mAbvaccine

solid tumors Phase I/IIcelldextherapeutics.com

cell therapy Cell MedicaLondon, United KingdomCenter for Cell and Gene TherapyHouston, TX

cell therapy

cancer(see also infectious)

Phase IIcellmedica.co.uk

CEP-37250/KNK-2804 Teva North AmericaNorth Wales, PAKyowa Hakko Kirin PharmaPrinceton, NJ

mAb adenocarcinoma Phase Itevapharm.comkyowa-kirin-pharma.com

CG201 (anti-hCG vaccine)

CG Therapeutics Seattle, WA

antibody vaccine

solid tumors Phase IIcgtherapeutics.com

ch14.18 mAb (Orphan Drug)

United Therapeutics Silver Spring, MD

mAb neuroblastoma Phase IIIunither.com

cixutumumab(LY3012217)

Eli LillyIndianapolis, INImclone SystemsPrinceton, NJ

mAb NSCLC Phase IIlilly.com

CLT-008 Cellerant TherapeuticsSan Carlos, CA

celltherapy

chemotherapy-induced neutropenia(see also transplantation)

Phase I/IIcellerant.com

CNTO-328 (siltuximab)

Janssen BiotechHorsham, PA

mAb giant lymph node hyperplasia, multiple myeloma, myelodysplastic syndromes------------------------------------------------prostate cancer

Phase IIjanssenbiotech.com

---------------------------------Phase Ijanssenbiotech.com

Cotara™ mAb TNT (Orphan Drug)

Peregrine Pharmaceuticals Tustin, CA

mAb recurrent glioblastoma (Fast Track)

Phase IIperegrineinc.com

CRS-207 Aduro BiotechBerkeley, CA

genetically-modifiedvaccine

pancreatic cancer

------------------------------------------------mesothelioma

Phase IIadurobiotech.com---------------------------------Phase Iadurobiotech.com

Medicines in Development Biologics 2013 19

Biologic Medicines in Development

CanCer and related Conditions

Product Name Sponsor ProductCategory

Indication Development Phase

CSF-1R mAb(IMC-CS4)

Eli Lilly Indianapolis, IN ImClone Systems Bridgewater, NJ

mAb solid tumors Phase Ililly.com

CT-011 (pidilizumab)

CureTech Yavne, Israel

mAb AML, colorectal cancer, diffuse large B-cell lymphoma, follicular lymphoma, malignant melanoma

Phase IIcuretechbio.com

CTL019 Novartis East Hanover, NJUniversity of PennsylvaniaPhiladelphia, PA

cell therapy CLL Phase IInovartis.com

custirsen (OGX-011)

OncoGenex TechnologiesVancouver, CanadaTeva PharmaceuticalsTikva, Israel

antisense NSCLC, prostate cancer(Fast Track)

------------------------------------------------first-time NSCLC

Phase IIIoncogenex.comtevapharm.com---------------------------------Phase IIoncogenex.comtevapharm.com

CV-301 BN ImmunoTherapeuticsMountain View, CA

cancer vaccine

metastatic breast cancer Phase IIbavarian-nordic.com

CVac™cancer vaccine MUC-1

Prima BioMedMelbourne, Australia

personalized dendritic cell vaccine

ovarian cancer Phase II/IIIprimabiomed.com.au

CYT107 (interleukin-7)

Cytheris Rockville, MD

recombinant interleukin

malignant melanoma, renal cancer (see also blood, infectious, transplantation)

Phase Icytheris.com

CYT-6091(Aurimune™)

Cytimmune Sciences Rockville, MD

tumor necrosisfactor

solid tumors Phase Icytimmune.com

dalotuzumab (MK-0646)

Merck Whitehouse Station, NJ

mAb

breast cancer, neuroendocrine tumors, NSCLC

Phase IImerck.com

daratumumab Janssen BiotechHorsham, PA

mAb multiple myeloma Phase I/IIjanssenbiotech.com

DC-IL-12 ZIOPHARM OncologyNew York, NY

celltherapy

malignant melanoma Phase Iziopharm.com

DCVax®-Lbrain cancer vaccine(Orphan Drug)

Northwest Biotherapeutics Bethesda, MD

dendritic cell vaccine

glioblastoma Phase IIInwbio.com

Medicines in Development Biologics 201320

Biologic Medicines in Development

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

DEDN6526A(antibody drug conjugate)

GenentechSouth San Francisco, CA

mAb malignant melanoma Phase Igene.com

demcizumab(OMP-21M18)

GlaxoSmithKlineRsch. Triangle Park, NCOncoMed Pharmaceuticals Redwood City, CA

mAb solid tumors Phase Igsk.comoncomed.com

DFRF4539A GenentechSouth San Francisco, CA

mAb multiple myeloma Phase Igene.com

DI17E6(anti-integrin mAb)

EMD Serono Rockland, MA

mAb colorectal cancer, prostate cancer Phase IIemdserono.com

DKN-01 DekkunCambridge, MA

mAb solid tumors Phase I

DN24-02 Dendreon Seattle, WA

personalizeddendritic cell vaccine

HER2-positive urogenital cancer Phase IIdendreon.com

DPX-0907 ImmunovaccineHalifax, Canada

vaccine breast cancer, ovarian cancer,prostate cancer

Phase Iimvaccine.com

DPX-Survivac ImmunovaccineHalifax, Canada

peptide vaccine

ovarian cancer Phase I/IIimvaccine.com

EC17 EndocyteWest Lafayette, IN

vaccine renal cancer (combination therapy)

Phase Iendocyte.com

ecromeximab (KW-2871)

Life Science Pharmaceuticals Darien, CT

mAb metastatic melanoma Phase IIinvestinlife.com

EGEN-001 (Orphan Drug)

EGENHuntsville, AL

genetherapy

ovarian cancer

------------------------------------------------colorectal cancer

Phase IIegeninc.com---------------------------------Phase I/IIegeninc.com

elotuzumab Bristol-Myers SquibbPrinceton, NJAbbVieNorth Chicago, IL

mAb multiple myeloma Phase IIIbms.comabbvie.com

Medicines in Development Biologics 2013 21

Biologic Medicines in Development

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

enavatuzumab AbbVieNorth Chicago, IL

mAb solid tumors Phase I completedabbvie.com

encapsulated cell therapy Metromedia Bio-ScienceNew York, NYRogosin InstituteNew York, NY

celltherapy

colorectal cancer, pancreaticcancer, prostate cancer

Phase IIrogosin.org

encapsulated cell therapy NuvilexSilver Spring, MD

celltherapy

pancreatic cancer Phase IInuvilex.com

ensituximab (NPC-1C)

Neogenix Oncology Great Neck, NY

mAb colorectal cancer,pancreatic cancer

Phase Ineogenix.com

entolimod(CBLB502)

Cleveland BioLabsBuffalo, NY

engineered peptide

radiation damage in cancer patients(see also other)

Phase Icbiolabs.com

epratuzumab Y-90/veltuzumab combination

ImmunomedicsMorris Plains, NJ

mAb non-Hodgkin lymphoma Phase I/IIimmunomedics.com

Erbitux® cetuximab

Bristol-Myers SquibbPrinceton, NJEli LillyIndianapolis, INImClone SystemsPrinceton, NJ

mAb esophageal cancer Phase IIIbms.comlilly.com

ETBX-011 EtubicsSeattle, WA

vector vaccine

colon cancer Phase IIetubics.com

EZN-2968 Enzon PharmaceuticalsPiscataway, NJ

antisense lymphoma, solid tumors Phase Ienzon.com

EZN-4176 Enzon PharmaceuticalsPiscataway, NJ

antisense prostate cancer Phase Ienzon.com

F-627 GeneronShanghai, China

recombinantfusion protein

chemotherapy-induced neutropenia Phase IIgeneronbiomed.com

Fang™ Vaccineautologous tumor cell vaccine

GradalisCarrollton, TX

autologous cell vaccine

solid tumors Phase IIgradalisinc.com

Medicines in Development Biologics 201322

Biologic Medicines in Development

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

farletuzumab (MORAb-003) (Orphan Drug)

EisaiWoodcliff Lake, NJ

mAb platinum-sensitive ovarian cancer

------------------------------------------------NSCLC

Phase IIIeisai.com---------------------------------Phase IIeisai.com

FG-3019 FibroGenSan Francisco, CA

mAb pancreatic cancer (see also respiratory, other)

Phase IIfibrogen.com

ficlatuzumab AVEO OncologyCambridge, MA

mAb glioblastoma, lymphoma, multiple myeloma, solid tumors

Phase Iaveooncology.com

flanvotumab(TYRP1 protein)

Eli LillyIndianapolis, IN

mAb malignant melanoma Phase Ililly.com

folate binding protein-E39 (FBP)

Galena BiopharmaLake Oswego, OR

peptide vaccine

endometrial cancer, ovarian cancer Phase I/IIgalenabiopharma.com

FP-1039 Five Prime Therapeutics San Francisco, CA

recombinantfusionprotein

solid tumors Phase Ifiveprime.com

Fzd7(vantictumab)

Bayer HealthCarePharmaceuticalsWayne, NJOncoMed PharmaceuticalsRedwood City, CA

mAb solid tumors Phase Ibayerpharma.comoncomed.com

ganitumab Amgen Thousand Oaks, CA

mAb pancreatic cancer

------------------------------------------------breast cancer, colorectal cancer,sarcoma

Phase IIIamgen.com---------------------------------Phase IIamgen.com

GC-33/RG7686 Chugai Pharma USABerkeley Heights, NJ Roche Nutley, NJ

mAb liver cancer Phase IIchugai-pharm.comroche.com

GI-4000 GlobeImmuneLouisville, CO

genetically- modified vaccine

colorectal cancer, NSCLC,pancreatic cancer

Phase IIglobeimmune.com

GI-6207 GlobeImmuneLouisville, CO

genetically- modified vaccine

medullary thyroid cancer Phase IIglobeimmune.com

GI-6301(brachyury peptide vaccine)

GlobeImmuneLouisville, CO

genetically-modifiedvaccine

solid tumors Phase Iglobeimmune.com

Medicines in Development Biologics 2013 23

Biologic Medicines in Development

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

GL-0817(MAGE-A3 immunomodulator)(Orphan Drug)

GliknikBaltimore, MD

peptide vaccine

head and neck cancer, multiple myeloma

Phase IIgliknik.com

GliAtak™ cancer gene therapy(Orphan Drug)

Advantagene Auburndale, MA

genetherapy

malignant glioma Phase IIadvantagene.com

Glionix™brain vaccine

NovaRxSan Diego, CA

antisense gene-modified cell vaccine

glioma Phase I completednovarx.com

GS-6624 (simtuzumab)

Gilead Sciences Foster City, CA

mAb colorectal cancer, pancreatic cancer(see also respiratory, other)

Phase IIgilead.com

GVAX® Breast cancer immunotherapy

BioSante PharmaceuticalsLincolnshire, IL

genetically-modified vaccine

breast cancer Phase Ibiosantepharma.com

GVAX® Colorectal cancer immunotherapy

BioSante Pharmaceuticals Lincolnshire, IL

genetically-modifiedvaccine

colorectal cancer Phase Ibiosantepharma.com

GVAX® Leukemia cancer immunotherapy(Orphan Drug)

BioSante PharmaceuticalsLincolnshire, IL

genetically-modified vaccine

AML, CML Phase IIbiosantepharma.com

GVAX® Multiple Myelomacancer immunotherapeutic

BioSante Pharmaceuticals Lincolnshire, IL

genetically-modifiedvaccine

multiple myeloma Phase IIbiosantepharma.com

GVAX® Pancreascancer immunotherapy (Orphan Drug)

AduroBiotechBerkeley, CABioSante PharmaceuticalsLincolnshire, IL

genetically-modified vaccine

pancreatic cancer Phase IIadurobiotech.combiosantepharma.com

GVAX® Prostate cancer immunotherapy

AduroBiotechBerkeley, CABioSante PharmaceuticalsLincolnshire, IL

genetically-modified vaccine

prostate cancer (Fast Track)

Phase IIIadurobiotech.combiosantepharma.com

HCD122(anti-CD40 mAb)

Novartis Pharmaceuticals East Hanover, NJ XOMA Berkeley, CA

mAb lymphoma Phase I/IInovartis.comxoma.com

HGS-ETR1 (mapatumumab)

GlaxoSmithKlineRsch. Triangle Park, NC

mAb liver cancer, multiple myeloma, NSCLC

Phase IIgsk.com

Medicines in Development Biologics 201324

Biologic Medicines in Development

CanCer and related Conditions

Product Name Sponsor ProductCategory

Indication Development Phase

HS-110 Heat BiologicsChapel Hill, NC

genetically-modified vaccine

NSCLC Phase IIheatbio.com

hTERT cancervaccine

MerckWhitehouse Station, NJVicalSan Diego, CA

DNA vaccine cancer Phase Imerck.comvical.com

HuL2G7 Galaxy Biotech Sunnyvale, CA

mAb solid tumors Phase Igalaxybiotech.com

HuM195/rGel Targa TherapeuticsSan Diego, CA

mAb AML, CML, myelodysplasticsyndromes

Phase Itargatherapeutics.com

huN901-DM1 (lorvotuzumab mertansine) (Orphan Drug)

ImmunoGen Cambridge, MA

mAb SCLC

-----------------------------------------------multiple myeloma, solid tumors

Phase IIimmunogen.com---------------------------------Phase Iimmunogen.com

HyperAcute® Lung turgenpumatucel-L

NewLink Genetics Ames, IA

DNA vaccine

NSCLC Phase II/IIIlinkp.com

HyperAcute® Melanoma melanoma cancer vaccine

NewLink Genetics Ames, IA

DNA vaccine

malignant melanoma Phase IIlinkp.com

HyperAcute® Pancreas algenpantucel-L(Orphan Drug)

NewLink Genetics Ames, IA

DNA vaccine

pancreatic cancer (Fast Track)

Phase IIIlinkp.com

icrucumab(LY3012212)

Eli LillyIndianapolis, INImClone SystemsBridgewater, NJ

mAb bladder cancer, breast cancer, colorectal cancer

Phase IIlilly.com

ICT-107 (Orphan Drug)

ImmunoCellular Therapeutics Woodland Hills, CA

personalized dendritic cell vaccine

glioblastoma Phase IIimuc.com

IL-21 (BMS-982470)(Orphan Drug)

Bristol-Myers Squibb Princeton, NJ

interleukin solid tumors Phase Ibms.com

IMA901(intradermal vaccine)(Orphan Drug)

immatics biotechnologiesTübingen, Germany

peptide vaccine

renal cancer treatment Phase IIIimmatics.com

Medicines in Development Biologics 2013 25

Biologic Medicines in Development

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

IMA950(intradermal vaccine)

immatics biotechnologiesTübingen, Germany

peptide vaccine

gliobastoma Phase Iimmatics.com

IMC-gp100 ImmunocoreOxon, United Kingdom

recombinant vaccine

malignant melanoma treatment Phase 0immunocore.com

IMC-TR1(LY3022859)

Eli LillyIndianapolis, INImClone SystemsBridgewater, NJ

mAb solid tumors Phase Ililly.com

IMF-001 ImmunoFrontierTokyo, Japan

recombinantvaccine

solid tumors Phase Iimmunofrontier.com

IMGN529 ImmunoGenWaltham, MA

mAb non-Hodgkin lymphoma Phase Iimmunogen.com

IMGN853 ImmunoGenWaltham, MA

mAb solid tumors Phase Iimmunogen.com

IMMU-102 (epratuzumab Y-90)

Immunomedics Morris Plains, NJ

mAb non-Hodgkin lymphoma Phase I/IIimmunomedics.com

IMT-1012 Immunotope Doylestown, PA

peptide immunothera-peuticvaccine

breast cancer, ovarian cancer Phase Iimmunotope.com

inotuzumabozogamicin (CMC-544)

Pfizer New York, NY UCB Smyrna, GA

mAb aggressive non-Hodgkin lymphoma, ALL

Phase IIIpfizer.comucb.com

interleukin-12gene therapy

OncoSec MedicalSan Diego, CA

gene therapy cutaneous T-cell lymphoma, malignant melanoma, merkel cell carcinoma

Phase IIoncosec.com

Iomab-B(anti-CD45 mAb)

Actinium PharmaceuticalsNew York, NY

mAb AML Phase IIactiniumpharmaceuti cals.com

IRX-2 IRX TherapeuticsNew York, NY

cytokine head and neck cancer(Fast Track)

Phase IIirxtherapeutics.com

ISF35 Memgen San Diego, CA

gene vaccine

CLL, non-Hodgkin lymphoma Phase IImemgenbio.com

ISIS-EIF4ERx(EIF-4E ASO)

Isis Pharmaceuticals Carlsbad, CA

antisense NSCLC, pancreatic cancer Phase IIisispharm.com

Medicines in Development Biologics 201326

Biologic Medicines in Development

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

ISIS-STAT3Rx AstraZenecaWilmington, DEIsis PharmaceuticalsCarlsbad, CA

antisense cancer Phase IIastrazeneca.comisispharm.com

J 591 Lu-177 BZL BiologicsNew York, NY

mAb prostate cancer Phase II

JVRS-100 immunotherapeutic

Colby PharmaceuticalsSan Jose, CA

DNA vaccine

leukemia Phase Icolbypharma.com

JX-594 Jennerex BiotherapeuticsSan Francisco, CA

genetically-engineered virus

liver cancer

------------------------------------------------colorectal cancer

Phase IIjennerex.com---------------------------------Phase Ijennerex.com

JX-929 Jennerex BiotherapeuticsSan Francisco, CA

genetically-engineeredvirus

breast cancer, pancreatic cancer Phase Ijennerex.com

KB004 KaloBios PharmaceuticalsSouth San Francisco, CA

mAb hematological malignancies Phase Ikalobios.com

LA-EP2006(pegfilgrastim biosimilar)

SandozPrinceton, NJ

growth factor chemotherapy-relatedfebrile neutropenia

Phase IIIus.sandoz.com

leukemia DNA vaccine(WT1)

Inovio PharmaceuticalsBlue Bell, PAUniversity of SouthamptonHampshire, United Kingdom

DNA vaccine

AML, CML Phase IIinovio.com

Leukine® sargramostim

Bayer HealthCare Pharmaceuticals Wayne, NJ

granulocyte macrophage colony- stimulating factor

CLL Phase IIbayerpharma.com

LFA102 Novartis PharmaceuticalsEast Hanover, NJXOMABerkeley, CA

mAb breast cancer, prostate cancer Phase Inovartis.com

LG631 LentigenGaithersburg, MD

stem cell therapy

chemoprotection in patients with glioblastoma

Phase Ilentigen.com

lirilumab(anti-KIR)

Bristol-Myers SquibbPrinceton, NJ

mAb cancer Phase Ibms.com

Medicines in Development Biologics 2013 27

Biologic Medicines in Development

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

LOR-2040 (Orphan Drug)

Lorus Therapeutics Toronto, Canada

antisense AML Phase II lorusthera.com

Lucanix® belagen pumatucel-L

NovaRx San Diego, CA

antisense gene-modified cell vaccine

NSCLC (Fast Track)

Phase IIInovarx.com

LY2495655(anti-myostatin mAb)

Eli LillyIndianapolis, IN

mAb cancer cachexia(see also musculoskeletal)

Phase IIlilly.com

LY2875358(c-met mAb)

Eli LillyIndianapolis, IN

mAb cancer Phase IIlilly.com

MABp1 XBiotechAustin, TX

mAb cancer-related cachexia(see also cardiovascular, diabetes, skin)------------------------------------------------advanced cancer

------------------------------------------------leukemia

Phase IIIxbiotech.com

---------------------------------Phase I/IIxbiotech.com---------------------------------Phase Ixbiotech.com

MAGE-A3 (antigen-specific cancer immunotherapeutic)

GlaxoSmithKline Rsch. Triangle Park, NC

recombinant immuno-therapeutic vaccine

bladder cancer, melanoma, NSCLC

Phase IIIgsk.com

MEDI-551(anti-CD19 mAb)

AstraZenecaWilmington, DEMedImmuneGaithersburg, MD

mAb hematological malignancies(see also autoimmune)

Phase IIastrazeneca.commedimmune.com

MEDI-565(anti-CEA BiTE)

AmgenThousand Oaks, CAAstraZenecaWilmington, DEMedImmuneGaithersburg, MD

bispecific antibody

solid tumors Phase Iamgen.comastrazeneca.commedimmune.com

MEDI-573 (anti-IGF mAb)

AstraZeneca Wilmington, DE MedImmune Gaithersburg, MD

mAb solid tumors Phase IIastrazeneca.commedimmune.com

MEDI-575 (anti-PDGFRα mAb)

AstraZeneca Wilmington, DE MedImmune Gaithersburg, MD

mAb glioblastoma, NSCLC Phase IIastrazeneca.commedimmune.com

Medicines in Development Biologics 201328

Biologic Medicines in Development

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

MEDI-0639(anti-DLL-4 mAb)

AstraZenecaWilmington, DEMedImmuneGaithersburg, MD

mAb solid tumors Phase Iastrazeneca.commedimmune.com

MEDI-3617(anti-ANG-2 mAb)

AstraZeneca Wilmington, DE MedImmune Gaithersburg, MD

mAb solid tumors Phase Iastrazeneca.commedimmune.com

MEDI-4736(anti-CD274 mAb)

AstraZenecaWilmington, DEMedImmuneGaithersburg, MD

mAb cancer Phase Iastrazeneca.commedimmune.com

MEDI-6469(anti-OX40 mAb)

AgonOxPortland, ORAstraZenecaWilmington, DEMedImmuneGaithersburg, MD

mAb solid tumors Phase Iagonox.comastrazeneca.commedimmune.com

melanoma vaccine MabVax TherapeuticsSan Diego, CA

mAb vaccine malignant melanoma Phase Imabvax.com

melanoma vaccine(POL-103A)

PolynomaSan Diego, CA

vaccine malignant melanoma Phase IIIpolynoma.com

MGA271(anti-B7-H3)

MacroGenicsRockville, MD

mAb solid tumors Phase Imacrogenics.com

MGAH22 (anti-HER2)

MacroGenics Rockville, MD

mAb solid tumors Phase Imacrogenics.com

milatuzumab (Orphan Drug)

Immunomedics Morris Plains, NJ

mAb CLL Phase Iimmunomedics.com

milatuzumab-DOX ImmunomedicsMorris Plains, NJ

mAb multiple myeloma Phase Iimmunomedics.com

MINT1526A GenentechSouth San Francisco, CA

mAB solid tumors Phase Igene.com

MK-3475 MerckWhitehouse Station, NJ

mAb malignant melanoma

------------------------------------------------NSCLC

Phase IImerck.com------------------------------- Phase Imerck.com

Medicines in Development Biologics 2013 29

Biologic Medicines in Development

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

MKC1106-MT Colby PharmaceuticalsSan Jose, CA

vaccine malignant melanoma Phase IIcolbypharma.com

MLN0264(GCC antibody drug conjugate)

Millennium PharmaceuticalsCambridge, MA

mAb gastrointestinal cancer Phase Imlnm.com

MM-111 Merrimack PharmaceuticalsCambridge, MA

recombinant fusionprotein

breast cancer, solid tumors Phase Imerrimackpharma.com

mogamulizimab(Orphan Drug)

Kyowa Hakko Kirin PharmaPrinceton, NJ

mAb cutaneous T-cell lymphoma

------------------------------------------------adult T-cell lymphoma, T-cell leukemia

Phase IIIkyowa-kirin-pharma.com---------------------------------Phase IIkyowa-kirin-pharma.com

MORAb-004 EisaiWoodcliff Lake, NJ

mAb colorectal cancer, melanoma, sarcoma

Phase IIeisai.com

MORAb-009(amatuximab)(Orphan Drug)

EisaiWoodcliff Lake, NJ

mAb mesothelioma Phase IIeisai.com

moxetumomab pasudotox

AstraZenecaWilmington, DEMedImmuneGaithersburg, MD

mAb hematological malignancies Phase Iastrazeneca.commedimmune.com

Multikine® leukocyte interleukin cancer(Orphan Drug)

CEL-SCI Vienna, VA

growth factor, interferon,interleukin

head and neck cancer Phase IIIcel-sci.com

MVA-BN® HER2 BN ImmunoTherapeutics Mountain View, CA

cell vaccine breast cancer Phase Ibavarian-nordic.com

MVA-BN®-PRO BN ImmunoTherapeutics Mountain View, CA

vaccine prostate cancer Phase I/IIbavarian-nordic.com

mycobacterium cell wall-DNA complex (MCC)

Bioniche TherapeuticsPointe-Claire, Canada

myco- bacterialDNA

bladder cancer (Fast Track)

Phase IIIbioniche.com

necitumumab Bristol-Myers Squibb Princeton, NJ Eli Lilly Indianapolis, IN ImClone SystemsPrinceton, NJ

mAb NSCLC Phase IIIbms.comlilly.com

Medicines in Development Biologics 201330

CanCer and related Conditions

Product Name Sponsor ProductCategory

Indication Development Phase

neuroblastoma vaccine MabVax TherapeuticsSan Diego, CA

mAb vaccine neuroblastoma Phase Imabvax.com

NeuVax™ nelipepmut-S

Galena BiopharmaLake Oswego, OR

peptide vaccine

breast cancer

------------------------------------------------prostate cancer

Phase IIIgalenabiopharma.com---------------------------------Phase I/IIgalenabiopharma.com

NGR-TNF(Orphan Drug)

MolmedMilan, Italy

recombinant fusionprotein

mesothelioma Phase IIImolmed.com

NHS-IL 12 EMD SeronoRockland, MANational Cancer InstituteBethesda, MD

interleukin solid tumors Phase Iemdserono.com

NiCord®

umbilical cord blood stem cell therapy

Gamida CellJerusalem, Israel

stem cell therapy

hematological malignancies(see also blood)

Phase I/IIgamida-cell.com

nimotuzumab(Orphan Drug)

InnoMab PTESingapore

mAb glioma Phase IIinnomab.net

nivolumab(anti-PD1)

Bristol-Myers Squibb Princeton, NJ

mAb melanoma, NSCLC, renal cell carcinoma-----------------------------------------------solid tumors

Phase IIIbms.com---------------------------------Phase I/IIbms.com

Nplate™ romiplostim

Amgen Thousand Oaks, CA

recombinant fusion protein

chemotherapy-induced thrombocytopenia(see also autoimmune)

Phase IIamgen.com

NTDDS (intradermalgene therapy)

Periphagen HoldingsPittsburgh, PA

genetherapy

cancer pain Phase II completed

NY-ESO-1 antigen- specific cancer immunotherapeutic

GlaxoSmithKlineRsch. Triangle Park, NC

recombinant vaccine

malignant melanoma Phase Igsk.com

obinutuzumab(GA101)

Biogen IdecWeston, MAGenentechSouth San Francisco, CA

mAb CLL, diffuse large B-cell lymphoma, non-Hodgkin lymphoma

Phase IIIbiogenidec.comgene.com

OHR/AVR118 Ohr PharmaceuticalNew York, NY

immune-basedtherapy

cachexia associated with cancer Phase IIohrpharmaceutical.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 31

Biologic Medicines in Development

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

olaratumab (LY3012207)

Eli LillyIndianapolis, INImClone SystemsPrinceton, NJ

mAb glioblastoma Phase IIlilly.com

OMP-52M51(anti-Notch 1)

GlaxoSmithKlineRsch. Triangle Park, NCOncoMed PharmaceuticalsRedwood City, CA

mAb hematological malignancies Phase Igsk.comoncomed.com

OMP-59R5 (anti-Notch 2/3)

GlaxoSmithKlineRsch. Triangle Park, NCOncoMed Pharmaceuticals Redwood City, CA

mAb pancreatic cancer Phase I/IIgsk.comoncomed.com

onartuzumab (anti-c-met- mAb)

Genentech South San Francisco, CA

mAb metastatic NSCLC Phase IIIgene.com

Onconase® ranpirnase

Tamir BiotechnologyMonmouth Junction, NJ

ribonuclease NSCLC Phase IItamirbio.com

Oncoprex™TUSC2 nanoparticles

GenprexAustin, TX

gene therapy NSCLC Phase Igenprex.com

OncoVAX® colorectal cancer vaccine

Vaccinogen Frederick, MD

cell vaccine colorectal cancer (Fast Track)

Phase I/IIvaccinogeninc.com

ONT-10 OncothyreonSeattle, WA

vaccine solid tumors Phase Ioncothyreon.com

Ontak®

denileukin diftitoxEisaiWoodcliff Lake, NJ

recombinant fusion protein

malignant melanoma Phase IIeisai.com

OPT-822/OPT-821 Optimer BiotechnologyTaipei, Taiwan

vaccine breast cancer Phase IIIoptimerbiotech.com

oregovomab (Orphan Drug)

Quest Pharmatech Edmonton, Canada

mAb ovarian cancer Phase IIquestpharmatech.com

ovarian cancer vaccine MabVax TherapeuticsSan Diego, CA

mAb vaccine ovarian cancer Phase IImabvax.com

PancAtak™ cancer gene therapy

Advantagene Auburndale, MA

gene therapy pancreatic cancer Phase I/IIadvantagene.com

pbi-shRNA™ STMN1 LP GradalisCarrollton, TX

gene therapy solid tumors Phase Igradalisinc.com

Medicines in Development Biologics 201332

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

PEGPH20 Halozyme TherapeuticsSan Diego, CA

recombinant enzyme

solid tumors Phase IIhalozyme.com

Perjeta™ pertuzumab

Genentech South San Francisco, CA

mAb

early HER2-positive breast cancer -----------------------------------------------HER2-positive metastatic breast cancer (second-line treatment), HER2-positive gastric cancer

Phase IIIgene.com---------------------------------Phase IIgene.com

PF-03446962 Pfizer New York, NY

mAb solid tumors Phase Ipfizer.com

PF-04605412 Pfizer New York, NY

mAb solid tumors Phase Ipfizer.com

PF-04856884(CVX060)

PfizerNew York, NY

fusionpeptide

renal cancer

------------------------------------------------solid tumors

Phase IIpfizer.com--------------------------------Phase Ipfizer.com

PF-05082566 PfizerNew York, NY

mAb cancer, lymphoma Phase Ipfizer.com

PF-05280014(trastuzumab biosimilar)

PfizerNew York, NY

mAb metastatic breast cancer Phase Ipfizer.com

polyclonal antibody stimulator(Orphan Drug)

Cancer AdvancesDurham, NC

polyclonal antibodyvaccine

gastric cancer, pancreatic cancer(see also digestive)------------------------------------------------colorectal cancer

Phase IIIcanceradvances.com--------------------------------Phase IIcanceradvances.com

PRAME antigen-specific cancer immunotherapeutic

GlaxoSmithKlineRsch. Triangle Park, NC

recombinant vaccine

malignant melanoma, NSCLC Phase Igsk.com

prophage cancer vaccine (G-series)

AgenusLexington, MA

vaccine glioma Phase IIagenusbio.com

prophage cancer vaccine (M-series) (Orphan Drug)

AgenusLexington, MA

vaccine malignant melanoma (Fast Track)

Phase IIIagenusbio.com

prophage cancer vaccine (R-series)

AgenusLexington, MA

vaccine renal cancer (Fast Track)

Phase IIIagenusbio.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 33

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

ProstAtak™ cancer gene therapy

Advantagene Auburndale, MA

gene therapy prostate cancer (Fast Track)

Phase IIIadvantagene.com

PROSTVAC®

recombinant vaccinia virus-expressing PSA

BN ImmunoTherapeuticsMountain View, CA

recombinantvaccine

prostate cancer Phase IIIbavarian-nordic.com

Provenge® sipuleucel-T

Dendreon Seattle, WA

dendritic cell vaccine

prostate cancer (early-stage disease, recurrent)------------------------------------------------prostate cancer (early-stage disease, neoadjuvanttherapy), prostate cancer (metastatic, first-line therapy)

Phase III dendreon.com---------------------------------Phase IIdendreon.com

PSMA-ADC Progenics Pharmaceuticals Tarrytown, NY

mAb prostate cancer Phase IIprogenics.com

PT107 Pique TherapeuticsDurham, NC

vaccine NSCLC Phase IIpiquetherapeutics.com

purine nucleoside phosphorylase gene therapy

PNP TherapeuticsBirmingham, AL

genetherapy

solid tumors Phase Ipnptherapeutics.com

PVX-410immunotherapeutic

OncoPepMethuen, MA

peptide vaccine

multiple myeloma Phase I/IIoncopep.com

QBI-139 Quintessence BiosciencesMadison, WI

ribonuclease solid tumors Phase Iquintbio.com

ramucirumab(LY3009806)

Eli LillyIndianapolis, INImClone SystemsPrinceton, NJ

mAb breast cancer, colorectal cancer,gastric cancer

Phase IIIlilly.com

Redectane® girentuximab I-124

WilexMunich, Germany

mAb diagnosis of kidney cancer Phase IIIwilex.com

REGN1400 Regeneron PharmaceuticalsTarrytown, NY

mAb cancer Phase Iregeneron.com

REIC gene therapy Momotaro-GeneOkayama, Japan

genetherapy

prostate cancer Phase Imt-gene.com

RG7116 RocheNutley, NJ

mAb solid tumors Phase Iroche.com

RG7155 RocheNutley, NJ

mAb solid tumors Phase Iroche.com

Biologic Medicines in Development

Medicines in Development Biologics 201334

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

RG7160(humAb EGFR)

Roche Nutley, NJ

mAb colorectal cancer Phase IIroche.com

RG7212 RocheNutley, NJ

mAb solid tumors Phase Iroche.com

RG7356(anti-CD44 mAb)

RocheNutley, NJ

mAb AML, solid tumors Phase Iroche.com

RG7458(antibody-drug conjugate)

GenentechSouth San Francisco, CASeattle GeneticsBothell, WA

mAb ovarian cancer Phase Igene.com

RIGScan™radiodiagnostic agent

Navidea BiopharmaceuticalsDublin, OH

mAb diagnosis of colorectal cancer

in clinical trialsnavidea.com

rilotumumab AmgenThousand Oaks, CA

mAb colorectal cancer, gastric cancer, prostate cancer, SCLC

Phase IIamgen.com

rindopepimut(Orphan Drug)

Celldex TherapeuticsNeedham, MA

vaccine first-line glioblastoma (Fast Track)------------------------------------------------second-line glioblastoma (Fast Track)

Phase IIIcelldextherapeutics.com---------------------------------Phase IIcelldextherapeutics.com

Rituxan®

rituximabGenentechSouth San Francisco, CA

mAb diffuse large B-cell lymphoma Phase IIIgene.com

RON8 mAb Eli Lilly Indianapolis, IN ImClone Systems Princeton, NJ

mAb cancer Phase Ililly.com

SAR3419 (maytansin-loaded anti-CD19 mAb)

Sanofi USBridgewater, NJ

mAb ALL, non-Hodgkin lymphoma Phase IIsanofi.com

SAR153192 (REGN 421)(anti-DLL4 mAb)

Regeneron Pharmaceuticals Tarrytown, NY Sanofi USBridgewater, NJ

mAb cancer Phase Iregeneron.comsanofi.com

SAR256212(MM-121)(anti-ErbB3 mAb)

Merrimack PharmaceuticalsCambridge, MASanofi USBridgewater, NJ

mAb breast cancer

------------------------------------------------solid tumors(combination therapy)

Phase IImerrimackpharma.comsanofi.com---------------------------------Phase Imerrimackpharma.comsanofi.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 35

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

SAR307746(REGN910)(anti-angiopoietin-2 mAb)

Regeneron PharmaceuticalsTarrytown, NYSanofi USBridgewater, NJ

mAb solid tumors Phase Iregeneron.comsanofi.com

SAR566658 (maytansin-loaded anti-DS6)

Sanofi USBridgewater, NJ

mAb DS6-positive solid tumors Phase Isanofi.com

SAR650984 (anti-CD38 naked mAb)

Sanofi USBridgewater, NJ

mAb hematological malignancies Phase Isanofi.com

sarcomacancer vaccine

MabVax TherapeuticsSan Diego, CA

mAbvaccine

sarcoma Phase IImabvax.com

SB-313 Sangamo BioSciencesRichmond, CA

celltherapy

glioblastoma Phase Isangamo.com

SCH-721015 FKD TherapiesKuopio, Finland

gene therapy bladder cancer Phase II

SGN-75(vorsetuzumab mafodotin)

Seattle GeneticsBothell, WA

mAb non-Hodgkin lymphoma, renal cancer

Phase Iseattlegenetics.com

SL-401 (Orphan Drug)

Stemline Therapeutics New York, NY

recombinantfusionprotein

AML, myleodysplasticsyndromes

Phase I/IIstemline.com

SL-701 Stemline TherapeuticsNew York, NY

dendritic cell vaccine

glioma Phase I/IIstemline.com

small-cell lung cancervaccine

MabVax TherapeuticsSan Diego, CA

mAb vaccine SCLC Phase Imabvax.com

SNS01-T Senesco TechnologiesBridgewater, NJ

genetherapy; RNA interference

diffuse large B-cell lymphoma, mantle cell lymphoma, multiple myeloma

Phase I/IIsenesco.com

sotatercept(ACE-011)

Acceleron Pharma Cambridge, MA Celgene Summit, NJ

recombinantfusionprotein

chemotherapy-induced anemia(see also blood)

Phase II/IIIacceleronpharma.comcelgene.com

Biologic Medicines in Development

Medicines in Development Biologics 201336

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

SPI-2012(granulocyte colony- stimulating factor,long-acting)

Hanmi PharmaceuticalSeoul, South KoreaSpectrum PharmaceuticalsHenderson, NV

growthfactor

neutropenia in cancer patients Phase IIsppirx.com

StemEx® carlecortemcel-L(Orphan Drug)

Gamida Cell Jerusalem, IsraelTeva PharmaceuticalTikva, Israel

stem cell therapy

hematological malignancies(Fast Track)

Phase IIIgamida-cell.comtevapharm.com

Stimuvax® lioposomal cancer vaccine

EMD SeronoRockland, MAOncothyreon Seattle, WA

peptide vaccine

NSCLC(Fast Track)

Phase IIIemdserono.comoncothyreon.com

Sym004 EMD SeronoRockland, MASymphogenLyngby, Denmark

mAb head and neck cancer

------------------------------------------------solid tumors

Phase IIemdserono.comsymphogen.com---------------------------------Phase Iemdserono.comsymphogen.com

tabalumab(BAFF inhibitor)

Eli Lilly Indianapolis, IN

mAb multiple myeloma(see also autoimmune)

Phase IIIlilly.com

talactoferrin alfa (Orphan Drug)

Agennix Princeton, NJ

recombinant protein

NSCLC(Fast Track)

Phase III completedagennix.com

talminogene laherparepvec

Amgen Thousand Oaks, CA

gene therapy malignant melanoma Phase IIIamgen.com

TeloB-Vaxtelomerasecancer vaccine

Adamis PharmaceuticalsSan Diego, CA

DNAvaccine

prostate cancer Phase Iadamispharmaceuticals.com

TF2 Immunomedics Morris Plains, NJ

mAb diagnosis of colorectal cancer Phase Iimmunomedics.com

TG-1101(ublituximab)(Orphan Drug)

TG TherapeuticsNew York, NY

mAb CLL, non-Hodgkin lymphoma Phase I/IItgtherapeutics.com

tigatuzumab Daiichi Sankyo Parsippany, NJ

mAb breast cancer, liver cancer, ovarian cancer, pancreatic cancer

Phase IIdsi.com

TKM-PLK1 Tekmira PharmaceuticalsBurnaby, Canada

RNAinterference

lymphoma, solid tumors Phase Itekmirapharm.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 37

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

Toca-511 (cytosine deaminase gene therapy)

Tocagen San Diego, CA

gene therapy glioblastoma Phase I/IItocagen.com

trastuzumabemtansine (T-DM1)

GenentechSouth San Francisco, CARocheNutley, NJ

mAb first-line HER2-positive metastatic breast cancer, third-line HER2-posi-tive metastatic breast cancer

------------------------------------------------early HER2-positive breast cancer, advanced HER2-positive gastric cancer

Phase IIIgene.comroche.com

---------------------------------Phase IIgene.comroche.com

TRC105 TRACON Pharmaceuticals San Diego, CA

mAb bladder cancer, liver cancer, ovarian cancer, prostate cancer------------------------------------------------solid tumors

Phase IItraconpharma.com---------------------------------Phase Itraconpharma.com

tremelimumab(anti-CTLA4 mAb)

AstraZeneca Wilmington, DEMedImmuneGaithersburg, MD

mAb solid tumors Phase IIastrazeneca.commedimmune.com

Trivaxcancer vaccine

Activartis BiotechVienna, Austria

personalized dendritic cell vaccine

glioblastoma Phase IIactivartis.com

tumor immunotherapy mAb

GenentechSouth San Francisco, CA

mAb solid tumors Phase Igene.com

TV1-Brain-1 TVAX BiomedicalLenexa, KS

cell vaccine glioblastoma Phase IItvaxbiomedical.com

TV1-Kidney-1 TVAX BiomedicalLenexa, KS

cell vaccine renal cancer Phase IItvaxbiomedical.com

U3-1565 Daiichi Sankyo Parsippany, NJ

mAb solid tumors Phase Idsi.com

urelumab(anti-CD137)

Bristol-Myers Squibb Princeton, NJ

mAb cancer Phase Ibms.com

Biologic Medicines in Development

Medicines in Development Biologics 201338

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

V503 MerckWhitehouse Station, NJ

vaccine prevention of cervical cancer, prevention of vulvovaginal cancer(see also infectious)

Phase IIImerck.com

VAY736 MorphoSysMunich, GermanyNovartis PharmaceuticalsEast Hanover, NJ

mAb CLL(see also other)

Phase Imorphosys.comnovartis.com

VB-111 VBL TherapeuticsOr Yehuda, Israel

genetherapy

thyroid cancer

------------------------------------------------glioblastoma

Phase IIvblrx.com---------------------------------Phase I/IIvblrx.com

VB4-845 (Orphan Drug)

Viventia Biotechnologies Mississauga, Canada

mAb bladder cancer Phase IIviventia.com

Vectibix® panitumumab

Amgen Thousand Oaks, CA

mAb colorectal cancer (first-line therapy,second-line therapy)

application submittedamgen.com

VEGFR3 mAb(IMC-3C5)

Eli Lilly Indianapolis, IN ImClone Systems Bridgewater, NJ

mAb cancer Phase Ililly.com

veltuzumab (IMMU-106) (Orphan Drug)

Immunomedics Morris Plains, NJ

mAb CLL, non-Hodgkin lymphoma(see also autoimmune)

Phase I/IIimmunomedics.com

VGX-100 Circadian TechnologiesVictoria, Australia

mAb solid tumors Phase Icircadian.com.au

VGX-3100 Inovio Pharmaceuticals Blue Bell, PA

DNAvaccine

cervical dysplasia Phase IIinovio.com

volociximab AbbVieNorth Chicago, IL

mAb NSCLC Phase I completedabbvie.com

VX15 Vaccinex Rochester, NY

mAb solid tumors(see also autoimmune)

Phase Ivaccinex.com

WT1 antigen- specific cancer immunotherapeutic

GlaxoSmithKlineRsch. Triangle Park, NC

recombinant vaccine

AML

------------------------------------------------breast cancer

Phase IIgsk.com---------------------------------Phase Igsk.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 39

CAnCer AnD relAteD ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

WT2725 Sunovion PharmaceuticalsMarlborough, MA

peptide vaccine

solid tumors Phase Isunovion.com

Xgeva®

denosumabAmgen Thousand Oaks, CA

mAb delay or prevention of bone metastases in prostate cancer------------------------------------------------delay or prevention of bone metases in breast cancer------------------------------------------------giant cell tumor of the bone

application submittedamgen.com---------------------------------Phase IIIamgen.com---------------------------------Phase IIamgen.com

Xiaflex® collagenase clostridiumhistolyticum

BioSpecifics TechnologiesLynbrook, NY

peptide lipoma(see also musculoskeletal,other)

Phase IIbiospecifics.com

XmAb® high ADCC mAb

Boehringer IngelheimPharmaceuticalsRidgefield, CTXencorMonrovia, CA

mAb cancer Phase Iboehringer-ingelheim.comxencor.com

XmAb® 2513 anti-CD30 mAb

Xencor Monrovia, CA

mAb Hodgkin disease, T-cell lymphoma

Phase Ixencor.com

XmAb®5574 anti-CD19 mAb

MorphoSysMunich, GermanyXencor Monrovia, CA

mAb CLL Phase Imorphosys.comxencor.com

Y-90 hPAM 4(IMMU-107)(Orphan Drug)

Immunomedics Morris Plains, NJ

mAb pancreatic cancer (Fast Track)

Phase I/IIimmunomedics.com

Yervoy™ipilimumab (Orphan Drug)

Bristol-Myers Squibb Princeton, NJ

mAb adjuvant melanoma, NSCLC, prostate cancer, SCLC------------------------------------------------gastric cancer, ovarian cancer

Phase IIIbms.com---------------------------------Phase IIbms.com

zanolimumab Emergent BioSolutionsRockville, MD

mAb

peripheral T-cell lymphoma Phase IIemergentbiosolutions.com

Zevalin®

ibritumomab tiuxetanSpectrum PharmaceuticalsHenderson, NV

mAb diffuse large B-cell lymphoma

------------------------------------------------non-Hodgkin lymphoma

Phase IIIsppirx.com---------------------------------Phase IIsppirx.com

Biologic Medicines in Development

Medicines in Development Biologics 201340

CArDiovAsCulAr DiseAse

Product Name Sponsor ProductCategory

Indication Development Phase

3K3A-APC ZZ BiotechHouston, TX

recombinant protein

stroke Phase Izzbiotech.com

ACP-501(rhLCAT)

AlphaCore PharmaAnn Arbor, MI

recombinant protein

atherosclerosis, coronary artery disease

Phase Ialphacorepharma.com

ACY001 ArteriocyteCleveland, OH

stem celltherapy

chronic coronary ischemia Phase I completedarteriocyte.com

adipose stem cell therapy Cytori TherapeuticsSan Diego, CA

stem celltherapy

myocardial ischemia Phase I/IIcytori.com

adult autologous stem cell therapy

Baxter InternationalDeerfield, IL

stem celltherapy

myocardial ischemia

------------------------------------------------critical limb ischemia

Phase IIIbaxter.com---------------------------------Phase I/II completedbaxter.com

adult mesenchymal stem cell therapy

BioCardiaSan Carlos, CA

stem cell therapy

ischemic heart disorders Phase I/IIbiocardia.com

ALD-201 CytomedixGaithersburg, MD

stem cell therapy

ischemic heart failure Phase I completedcytomedix.com

ALD-301 CytomedixGaithersburg, MD

stem celltherapy

critical limb ischemia Phase I/IIcytomedix.com

ALD-401 CytomedixGaithersburg, MD

stem celltherapy

ischemic stroke Phase IIcytomedix.com

allogeneic stem cell therapy

Stemedica Cell TechnologiesSan Diego, CA

stem cell therapy

ischemic stroke Phase I/IIstemedica.com

ALN-PCS Alnylam PharmaceuticalsCambridge, MA

RNA interference

hypercholesterolemia Phase Ialnylam.com

ALO-212 ArteriocyteCleveland, OH

stem celltherapy

critical limb ischemia Phase Iarteriocyte.com

AMG 145 AmgenThousand Oaks, CA

mAb hypercholesterolemia

------------------------------------------------hyperlipoproteinemia type IIa

Phase IIIamgen.com---------------------------------Phase II/IIIamgen.com

AMR-001 Amorcyte Allendale, NJNeoStemNew York, NY

stem cell therapy

myocardial infarction Phase IIamorcyte.comneostem.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 41

CArDiovAsCulAr DiseAse

Product Name Sponsor ProductCategory

Indication Development Phase

Anginera™epicardial angiogenesis patch

TheregenSan Francisco, CA

fibroblastcelltherapy

heart failure, ischemic heart disorders

Phase Itheragen.com

anti-fibrin mAb 3B6/22 Tc-99m

AgenixSydney, Australia

mAb diagnosis of deep vein thrombosis, diagnosis ofpulmonary embolism

Phase IIagenix.com

anti-oxLDL(BI-204/RG7418)

BioInvent InternationalLund, SwedenGenentechSouth San Francisco, CA

mAb atherosclerosis Phase IIgene.com

anti-PCSK9 mAb(RG7652)

GenentechSouth San Francisco, CA

mAb cardiovascular disease Phase IIgene.com

cardiovascular medicine Eli LillyIndianapolis, IN

biologic cardiovascular disease Phase I lilly.com

Collategene®

beperminogene perplasmid

AnGes MGGaithersburg, MD Mitsubishi Tanabe PharmaOsaka, Japan

gene therapy peripheral arterial disease (Fast Track)------------------------------------------------ischemic heart disease

Phase IIanges-mg.com--------------------------------Phase Ianges-mg.com

combination autologousstem cell therapy

TCA Cellular TherapyCovington, LA

stem cell therapy

coronary ischemia,ischemic heart disorders

Phase IItcacellulartherapy.com

CPI-003(intracoronary cardio-sphere-derived stem cell therapy)

CapricorBeverly Hills, CA

stem cell therapy

myocardial infarction Phase Icapricor.com

desmoteplase LundbeckDeerfield, IL

recombinant protein

ischemic stroke (Fast Track)

Phase IIIlundbeck.com

endometrial blood stem cell therapy

MedistemSan Diego, CA

stem cell therapy

heart failure Phase IImedisteminc.com

fibroblast growth factor-1

CardioVascular BioTherapeutics Las Vegas, NV

fibroblast growth factor

severe coronary heart disease (see also skin)

Phase IIcvbt.com

Generx™ alferminogene tadenovec

Cardium Therapeutics San Diego, CA

gene therapy myocardial ischemia (Fast Track)

Phase IIIcardiumthx.com

GGF2(glial growth factor 2)

Acorda TherapeuticsHawthorne, NY

growthfactor

congestive heart failure Phase Iacorda.com

GSK249320 GlaxoSmithKline Rsch. Triangle Park, NC

mAb stroke Phase IIgsk.com

Biologic Medicines in Development

Medicines in Development Biologics 201342

CArDiovAsCulAr DiseAse

Product Name Sponsor ProductCategory

Indication Development Phase

IL-1 ß antibody Eli LillyIndianapolis, IN

mAb cardiovascular disease Phase Ililly.com

Ilaris®

canakinumabNovartis PharmaceuticalsEast Hanover, NJ

mAb secondary prevention of cardiovascular events(see also autoimmune, diabetes, musculoskeletal)

Phase IIInovartis.com

inclacumab(RG1512)

Roche Nutley, NJ

mAb peripheral vascular disease Phase IIroche.com

Iprivask® desirudin

Canyon Pharmaceuticals Columbia, MD

recombinantprotein

prevention of thrombosis in clinical trialscanyonpharma.com

ischemic tolerant allogeneic mesenchymalstem cell therapy

Stemedica Cell TechnologiesSan Diego, CA

stem cell therapy

acute myocardial infarction Phase IIstemedica.com

ISIS-APOCIIIRx Isis PharmaceuticalsCarlsbad, CA

antisense coronary artery disease Phase IIisispharm.com

ISIS-CRPRx Isis PharmaceuticalsCarlsbad, CA

antisense atrial fibrillation, coronary artery disease(see also autoimmune)

Phase IIisispharm.com

ixmyelocel-T(Orphan Drug)

Aastrom BiosciencesAnn Arbor, MI

stem celltherapy

critical limb ischemia(Fast Track)------------------------------------------------dilated cardiomyopathy

Phase IIIaastrom.com---------------------------------Phase IIaastrom.com

MABp1 XBiotechAustin, TX

mAb vascular restinosis(see also cancer, diabetes, skin)

Phase IIxbiotech.com

mesenchymal precursor cell (MPC) product

MesoblastNew York, NY

stem cell therapy

congestive heart failure,myocardial infarction(see also diabetes, musculoskeletal, transplantation)

Phase IImesoblast.com

MultiGeneAngio MultiGene Vascular SystemsNesher, Israel

celltherapy

peripheral arterial disorders Phase I/IImgvs.co.il

MultiStem®

stem cell therapyAthersysCleveland, OH

stem celltherapy

myocardial infarction, stroke(see also digestive, transplantation)

Phase IIathersys.com

Mydicar™ SERCA 2a gene therapy

Celladon La Jolla, CA

gene therapy advanced heart failure class III/IV

Phase IIcelladon.net

MyoCell® muscle stem cell therapy

Bioheart Sunrise, FL

stem celltherapy

congestive heart failure Phase II/IIIbioheartinc.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 43

CardiovasCular disease

Product Name Sponsor ProductCategory

Indication Development Phase

MyoCell® SDF-1muscle stem cell therapy(second-generation)

BioheartSunrise, FL

stem cell therapy

congestive heart failure Phase Ibioheartinc.com

NTx®-265 Stem Cell TherapeuticsToronto, Canada

stem cell therapy

stroke Phase IIstemcellthera.com

PB-1046 PhaseBio PharmaceuticalsMalvern, PA

recombinant fusion protein

essential hypertension Phase Iphasebio.com

PCSK9 adnectin Bristol-Myers SquibbPrinceton, NJ

adnectin cardiovascular disease Phase Ibms.com

PF-04950615(RN316)

PfizerNew York, NY

mAb hypercholesterolemia Phase IIpfizer.com

PF-05335810 PfizerNew York, NY

biologic hypercholesterolemia Phase Ipfizer.com

PLX-PAD Pluristem TherapeuticsHaifa, Israel

stem cell therapy

intermittent claudication Phase IIpluristem.com

Prochymal® remestemcel-L

Osiris Therapeutics Columbia, MD

stem cell therapy

acute myocardial infarction(see also diabetes, digestive, respiratory, transplantation)

Phase IIosiris.com

PRT-201 (Orphan Drug)

Proteon TherapeuticsWaltham, MA

recombinantprotein

vascular access for hemodialysis(Fast Track)------------------------------------------------peripheral vascular disease

Phase IIproteontherapeutics.com---------------------------------Phase Iproteontherapeutics.com

SAR236553/REGN727(anti-PCSK-9 mAb)

Regeneron PharmaceuticalsTarrytown, NYSanofi USBridgewater, NJ

mAb hypercholesterolemia Phase IIIregeneron.comsanofi.com

SB623 SanBioMountain View, CA

stem celltherapy

stroke Phase I/IIsan-bio.com

SDF-1(stromal cell-derivedfactor-1)

Juventas TherapeuticsCleveland, OH

gene therapy critical limb ischemia, heart failure Phase IIjuventasinc.com

Syncria®

albiglutideGlaxoSmithKlineRsch. Triangle Park, NC

recombinant fusion protein

heart failure(see also diabetes)

Phase IIgsk.com

TS01 Thrombolytic Science Interna-tionalCambridge, MA

recombinant protein

acute ischemic stroke Phase Itsillc.net

Biologic Medicines in Development

Medicines in Development Biologics 201344

CArDiovAsCulAr DiseAse

Product Name Sponsor ProductCategory

Indication Development Phase

Vascugel®cell therapy(Orphan Drug)

Shire Regenerative MedicineWayne, PA

celltherapy

peripheral vascular disorder in patients undergoing arteriovenous access procedures for hemodialysis(Fast Track)

Phase IIshire.com

VM-202 (modified hepatocytegrowth factor genetherapy)

VW BioPharmaAtlanta, GA

gene therapy peripheral arterial disease(see also neurologic)------------------------------------------------myocardial ischemia

Phase IIviromed.co.kr---------------------------------Phase Iviromed.co.kr

DiABetes

Product Name Sponsor ProductCategory

Indication Development Phase

BHT-3201 Bayhill Therapeutics Palo Alto, CA

DNAvaccine

type 1 diabetes Phase I completedbayhilltx.com

CVX-096 (PF-04856883)

Pfizer New York, NY

recombinantfusionprotein

type 2 diabetes Phase Ipfizer.com

diabetes medicine Eli LillyIndianapolis, IN

biologic diabetes Phase Ililly.com

diabetes medicine Eli LillyIndianapolis, IN

biologic diabetes Phase Ililly.com

diabetes medicine Eli LillyIndianapolis, IN

biologic diabetes Phase Ililly.com

Diamyd®

autoimmunediabetes vaccine(Orphan Drug)

DiamydPittsburgh, PA

vaccine type 1 diabetes Phase IIIdiamyd.com

dulaglutide(GLP-1)

Eli Lilly Indianapolis, IN

recombinantfusion protein

type 2 diabetes Phase IIIlilly.com

gevokizumab(IL-1B inhibitor mAb)

XOMA Berkeley, CA

mAb type 1 diabetes, type 2 diabetes (see also eye, musculoskeletal, skin)

Phase IIxoma.com

GSK1070806(IL-18 mAb)

GlaxoSmithKlineRsch. Triangle Park, NC

mAb type 2 diabetes(see also digestive)

Phase IIgsk.com

IBC-VS01(insulin B-chain vaccine)

Orban BiotechBrookline, MA

vaccine type I diabetes Phase I completedorbanbiotech.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 45

DiABetes

Product Name Sponsor ProductCategory

Indication Development Phase

Ilaris®

canakinumabNovartis Pharmaceuticals East Hanover, NJ

mAb type 2 diabetes (see also autoimmune, cardiovascular, musculoskeletal)------------------------------------------------type 1 diabetes

Phase II/IIInovartis.com

---------------------------------Phase IInovartis.com

INGAP peptide ExsulinMinneapolis, MN

peptide fragment

type 1 diabetes, type 2 diabetes Phase IIexsulin.com

insulin glargine(rDNA origin)(new formulation)

Sanofi USBridgewater, NJ

recombinantinsulin

type 1 diabetes, type 2 diabetes Phase IIIsanofi.com

insulin lispro/BC106(LY275585)

AdociaLyon, FranceEli LillyIndianapolis, IN

recombinant protein

type 1 diabetes, type 2 diabetes Phase Ililly.com

ISIS-GCCRRx Isis PharmaceuticalsCarlsbad, CA

antisense type 2 diabetes(see also other)

Phase Iisispharm.com

ISIS-GCGRRx Isis PharmaceuticalsCarlsbad, CA

antisense type 2 diabetes Phase Iisispharm.com

ISIS-PTP1BRx(PTP-1B gene inhibitor)

Isis PharmaceuticalsCarlsbad, CA

antisense type 2 diabetes Phase Iisispharm.com

Lantus®

insulin glargine (rDNA origin) injection and lixisenatide combination

Sanofi USBridgewater, NJ

recombinant insulin

type 2 diabetes(see also cardiovascular)

Phase Isanofi.com

LY2605541(novel basal insulin/insulin peglispro)

Boehringer IngelheimPharmaceuticalsRidgefield, CTEli LillyIndianapolis, IN

insulin hormone

type 1 diabetes, type 2 diabetes Phase IIIboehringer-ingelheim.comlilly.com

LY2963016(new insulin glargineproduct)

Boehringer IngelheimPharmaceuticalsRidgefield, CTEli LillyIndianapolis, IN

insulin hormone

type 1 diabetes, type 2 diabetes Phase IIIboehringer-ingelheim.comlilly.com

MABp1 XBiotechAustin, TX

mAb type 2 diabetes(see also cancer, cardiovascular, skin)

Phase IIxbiotech.com

mesenchymal precursor cell (MPC) product

MesoblastNew York, NY

stem cell therapy

type 2 diabetes(see also cardiovascular, musculoskeletal, transplantation)

Phase IImesoblast.com

Biologic Medicines in Development

Medicines in Development Biologics 201346

DiABetes

Product Name Sponsor ProductCategory

Indication Development Phase

PB-1023(GLP-1 receptor agonist)

PhaseBio PharmaceuticalsMalvern, PA

recombinantfusionprotein

type 2 diabetes Phase IIphasebio.com

PF-05231023 PfizerNew York, NY

biologic type 2 diabetes Phase Ipfizer.com

Prochymal® remestemcel-L (Orphan Drug)

Osiris Therapeutics Columbia, MD

stem cell therapy

type 1 diabetes (see also cardiovascular, digestive,respiratory, transplantation)

Phase IIosiris.com

rHuPH20/insulin Halozyme Therapeutics San Diego, CA

recombinant fusion protein

type 1 diabetes, type 2 diabetes Phase IIhalozyme.com

Syncria®

albiglutideGlaxoSmithKlineRsch. Triangle Park, NC

recombinant fusion protein

type 2 diabetes(see also cardiovascular)

application submittedgsk.com

teplizumab (Orphan Drug)

MacroGenics Rockville, MD

mAb type 1 diabetes Phase IIImacrogenics.com

Digestive DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

alicaforsen Atlantic Healthcare Essex, United Kingdom

antisense pouchitis (Fast Track),distal ulcerative colitis, proximal ulcerative colitis

Phase IIIatlantichc.com

ALV003 Alvine PharmaceuticalsSan Carlos, CA

recombinant protein

celiac disease Phase Ialvinepharma.com

AMG 139/MEDI-2070 AmgenThousand Oaks, CAAstraZenecaWilmington, DE

mAb Crohn’s disease Phase Iamgen.comastrazeneca.com

AMG 181/MEDI-7183 AmgenThousand Oaks, CAAstraZenecaWilmington, DE

mAb Crohn’s disease, ulcerative colitis Phase IIamgen.comastrazeneca.com

anrukinzumab(IMA-638)

PfizerNew York, NY

mAb ulcerative colitis Phase IIpfizer.com

anti-IP10 Bristol-Myers SquibbPrinceton, NJ

mAb Crohn’s disease, ulcerative colitis Phase IIbms.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 47

Digestive DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

clazakizumab(anti-IL6)

Bristol-Myers SquibbPrinceton, NJAlder BiopharmaceuticalsBothell, WA

mAb Crohn’s disease(see also autoimmune, musculoskeletal)

Phase IIbms.comalderbio.com

etrolizumab(rhuMAb-ß7)

GenentechSouth San Francisco, CA

mAb ulcerative colitis Phase IIgene.com

GSK1070806(IL-18 mAb)

GlaxoSmithKlineRsch. Triangle Park, NC

mAb inflammatory bowel disease(see also diabetes)

Phase Igsk.com

HLA-DQ2 peptide vaccine ImmusanTCambridge, MA

peptide vaccine

celiac disease Phase Iimmusant.com

Humira® adalimumab

AbbVieNorth Chicago, IL

mAb Crohn’s disease in children and adolescents (see also autoimmune, eye, musculoskeletal, skin, other)

Phase IIIabbvie.com

MultiStem®

stem cell therapy (PF-05285401)

AthersysCleveland, OHPfizerNew York, NY

stem celltherapy

ulcerative colitis(see also cardiovascular, transplantation)

Phase IIathersys.compfizer.com

NN8717(rFXIII)

Novo NordiskPrinceton, NJ

recombinant protein

ulcerative colitis Phase IInovonordisk.com

PDA-001 (cenplacel-L)

Celgene Cellular TherapeuticsSummit, NJ

stem celltherapy

Crohn’s disease (see also autoimmune)

Phase IIcelgene.com

PF-00547659 PfizerNew York, NY

mAb Crohn’s disease Phase IIpfizer.com

PF-04236921 PfizerNew York, NY

mAb Crohn’s disease (see also autoimmune)

Phase IIpfizer.com

polyclonal antibody stimulator

Cancer AdvancesDurham, NC

polyclonal antibodyvaccine

gastrointestinal reflux disease (GERD)(see also cancer)

Phase IIcanceradvancesinc.com

Prochymal® remestemcel-L

Osiris Therapeutics Columbia, MD

stem cell therapy

Crohn’s disease (Fast Track), gastrointestinal injury from radiation exposure(see also cardiovascular, diabetes, respiratory, transplantation)

Phase IIIosiris.com

Biologic Medicines in Development

Medicines in Development Biologics 201348

Digestive DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

QAX576 Novartis PharmaceuticalsEast Hanover, NJ

mAb rectal fistula in patients with Crohn’s disease(see also respiratory)

Phase IInovartis.com

Remicade®

infliximabJanssen BiotechHorsham, PA

mAb Crohn’s disease (prevention of relapse after surgery resection)

Phase IIIjanssenbiotech.com

SAR252067(anti-LIGHT mAb)

Sanofi USBridgewater, NJ

mAb Crohn’s disease, ulcerative colitis Phase Isanofi.com

SAR339658(VLA2 antagonist)

Sanofi USBridgewater, NJ

mAb inflammatory bowel disease Phase IIsanofi.com

Simponi®golimumab(subcutaneous)

Janssen BiotechHorsham, PA

mAb ulcerative colitis (see also autoimmune)

application submittedjanssenbiotech.com

Stelara®

ustekinumabJanssen BiotechHorsham, PA

mAb Crohn’s disease (see also autoimmune, musculoskeletal, other)

Phase II/IIIjanssenbiotech.com

tralokinumab AstraZenecaWilmington, DEMedImmuneGaithersburg, MD

mAb ulcerative colitis(see also respiratory)

Phase IIastrazeneca.commedimmune.com

vedolizumab(MLN0002)

Takeda Pharmaceuticals Deerfield, IL

mAb Crohn’s disease, ulcerative colitis Phase IIItakeda.com

eye ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

anti-factor D(RG7417)

Genentech South San Francisco, CA

mAb geographic atrophy associated with age-related macular degeneration

Phase IIgene.com

anti-LINGO(BIIB033)

Biogen IdecWeston, MA

mAb optic neuritis (see also autoimmune)

Phase Ibiogenidec.com

AVA-101 Avalanche BiotechnologiesSan Francisco, CA

gene therapy age-related macular degeneration Phase I/IIavalanchebiotech.com

eye disordertherapeutic

MorphoSysMartinsried, GermanyNovartis PharmaceuticalsEast Hanover, NJ

mAb eye disorders Phase Inovartis.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 49

eye ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

Eylea®

afliberceptBayer HealthCare Pharmaceuticals Wayne, NJ Regeneron PharmaceuticalsTarrytown, NY

recombinant fusion protein

diabetic macular edema,macular edema following branch retinal vein occlusion

Phase IIIbayerpharma.comregeneron.com

gevokizumab(IL-1B inhibitor mAb)(Orphan Drug)

XOMABerkeley, CA

mAb intermediate or posterior uveitis(see also diabetes, musculoskeletal, skin)

Phase IIIxoma.com

GSK933776A(anti-B amyloid mAb)

GlaxoSmithKlineRsch. Triangle Park, NC

mAb age-related macular degeneration(see also neurologic)

Phase IIgsk.com

GZ402663(FLT-01) anti-vascular endothelial growth-factor gene therapy

Applied Genetic TechnologiesAlachua, FLGenzymeCambridge, MA

genetherapy

wet (neovascular) age-related macular degeneration

Phase Iagtc.comgenzyme.com

hl-con1 Iconic TherapeuticsAtlanta, GA

recombinantprotein

age-related macular degeneration

Phase Iiconictherapeutics.com

HuCNS-SC®

adult neural stem cell therapy(intraocular)

StemCellsNewark, CA

stem cell therapy

age-related macular degeneration(see also genetic, neurologic)

Phase I/IIstemcellsinc.com

human retinalpigment epithelialcell therapy(Orphan Drug)

Advanced Cell TechnologySanta Monica, CA

stem celltherapy

dry age-related macular degeneration, Stargardt’smacular degeneration

Phase I/IIadvancedcell.com

Humira®

adalimumabAbbVieNorth Chicago, IL

mAb uveitis(see also autoimmune, digestive, musculoskeletal, skin, other)

Phase IIIabbvie.com

iSONEP™ sonepcizumab(intravitreous)

Lpath San Diego, CA

mAb wet age-related macular degeneration

Phase IIlpath.com

iCo-007 iCo TherapeuticsVancouver, Canada

antisense diabetic macular edema Phase II icotherapeutics.com

Biologic Medicines in Development

Medicines in Development Biologics 201350

eye ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

Lucentis® ranibizumab

Genentech South San Francisco, CA RocheNutley, NJ

mAb age-related macular degeneration (0.5 mg PRN)

------------------------------------------------age-related macular degeneration (sustained-delivery), diabetic macular edema (sustained- delivery), retinal vein occlusion

application submittedgene.comroche.com---------------------------------Phase Igene.comroche.com

Mitosol®mitomycinophthalmic

Mobius TherapeuticsSt. Louis, MO

recombinantprotein

prevention of pterygium recurrence after excision surgery

application submittedmobiustherapeutics.com

NT 501 (ciliary neurotrophic factor) (Orphan Drug)

Neurotech PharmaceuticalsCumberland, RI

cell therapy retinitis pigmentosa (Fast Track)------------------------------------------------age-related macular degeneration (Fast Track)

Phase II/IIIneurotechusa.com---------------------------------Phase IIneurotechusa.com

PF-655(RTP801i14)

Quark PharmaceuticalsFremont, CA

RNA interference

wet age-related macular degeneration, diabetic macular edema

Phase IIquarkpharma.com

PF-04382923(RN6G)

PfizerNew York, NY

antibody age-related macular degeneration Phase IIpfizer.com

QPI-1007 Quark PharmaceuticalsFremont, CA

RNAinterference

optic neuropathy Phase Iquarkpharma.com

RetinoStat®angiostatin endostatingene therapy

Oxford BioMedicaOxford, United KingdomSanofi USBridgewater, NJ

genetherapy

wet age-related macular degeneration

Phase Isanofi.com

RU-101(recombinant humanserum albumin)

R-Tech UenoTokyo, Japan

recombinant protein

dry eyes Phase Irtechueno.com

secukinumab(AIN457)

Alcon LabsFort Worth, TXNovartis PharmaceuticalsEast Hanover, NJ

mAb uveitis(see also autoimmune, musculoskeletal, skin)

Phase IIIalcon.comnovartis.com

Soliris®

eculizumabAlexion PharmaceuticalsCheshire, CT

mAb severe or refractory neuromyelitis optica (see also autoimmune, infectious,transplantation)

Phase IIalxn.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 51

eye ConDitions

Product Name Sponsor ProductCategory

Indication Development Phase

StarGen®

Stargardt’s diseasegene therapy(Orphan Drug)

Sanofi USBridgewater, NJ

genetherapy

Stargardt’s disease (juvenile-onset macular dystrophy)

Phase I/IIsanofi.com

genetiC DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

adrenoleucodystrophy gene therapy

bluebird bioCambridge, MAINSERMParis, France

genetherapy

adrenoleucodystrophy Phase I/IIbluebirdbio.com

AGTC-0106 adeno-associated virus vector-medictoten(Orphan Drug)

Applied Genetic Technologies Alachua, FL

gene therapy alpha-1-antitrypsin deficiency Phase IIagtc.com

ALD-601 CytomedixGaithersburg, MD

stem cell therapy

infantile-onset lysosomal storage disease

Phase Icytomedix.com

ALN-TTR02 Alnylam PharmaceuticalsCambridge, MA

RNA interference

TTR-mediated amyloidosis Phase IIalnylam.com

asfotase alfa(Orphan Drug)

Alexion PharmaceuticalsCheshire, CT

recombinantfusionprotein

hypophosphatasia in infants and children(Fast Track)------------------------------------------------hypophosphatasia in adults and adolescents

Phase II/IIIalxn.com

--------------------------------Phase IIalxn.com

AVI-4658(eteplirsen)

Sarepta Therapeutics Cambridge, MA

antisense Duchenne muscular dystrophy Phase IIsareptatherapeutics.com

Biostrophin™ muscular dystrophy gene therapy

Asklêpios BioPharmaceuticals Chapel Hill, NC

gene therapy Duchenne muscular dystrophy Phase Iaskbio.com

BMN-110(Orphan Drug)

BioMarin PharmaceuticalNovato, CA

recombinantenzyme

mucopolysaccharidosis IV (Morquio A syndrome)

Phase IIIbmrn.com

Biologic Medicines in Development

Medicines in Development Biologics 201352

genetiC DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

BMN-701(Orphan Drug)

BioMarin Pharmaceutical Novato, CA

recombinantfusionprotein

glycogen storage disease type II (Pompe disease)

Phase IIbmrn.com

CEQ508(Orphan Drug)

Marina BiotechBothell, WA

RNAinterference

familial adenomatous polyposis

Phase I/IImarinabio.com

GNE lipoplex(muscular dystrophygene therapy)

GradalisCarrollton, TX

gene therapy muscular dystrophy Phase Igradalisinc.com

GSK2402968(PRO-051)(Orphan Drug)

GlaxoSmithKlineRsch. Triangle Park, NCProsensaLeiden, The Netherlands

antisense Duchenne muscular dystrophy Phase IIgsk.comprosensa.eu

GSK2696273 GlaxoSmithKlineRsch. Triangle Park, NC

stem cell therapy

adenosine deaminase severe combined immune deficiency

Phase IIIgsk.com

GZ402665(rhASM)(Orphan Drug)

GenzymeCambridge, MA

recombinantenzyme

Niemann-Pick type B Phase Igenzyme.com

HGT2310(Orphan Drug)

ShireDublin, Ireland

enzymereplacement therapy

Hunter syndrome(mucopolysaccharidosis II)

Phase I/IIshire.com

HuCNS-SC®

adult neural stem cell therapy (intracerebral)

StemCellsNewark, CA

stem cell therapy

Pelizaeus-Merzbacher disease(see also eye, neurologic)

Phase Istemcellsinc.com

ISIS-SMNRx(Orphan Drug)

Biogen IdecWeston, MAIsis PharmaceuticalsCarlsbad, CA

antisense spinal muscular atrophy Phase IIbiogenidec.comisispharm.com

ISIS-TTRRx(Orphan Drug)

GlaxoSmithKlineRsch. Triangle Park, NCIsis PharmaceuticalsCarlsbad, CA

antisense familial amyloid polyneuropathy Phase IIIgsk.comisispharm.com

Kineret®anakinra(Orphan Drug)

SobiArdmore, PA

recombinant protein

cryopyrin-associated periodic syndrome

application submittedsobi.com

KRN-23 Kyowa Hakko Kirin PharmaPrinceton, NJ

mAb X-linked dominant hypophosphatemic rickets

Phase I/IIkyowa-kirin-pharma.com

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Medicines in Development Biologics 2013 53

genetiC DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

Kynamro™mipomersen (Orphan Drug)

Genzyme Cambridge, MA Isis Pharmaceuticals Carlsbad, CA

antisense severe heterozygous familial hypercholesterolemia

Phase IIIgenzyme.comisispharm.com

leber congenitalamaurosis genetherapy(Orphan Drug)

Applied Genetic TechnologiesAlachua, FL

genetherapy

leber congenital amaurosis Phase I/IIagtc.com

NAV™ therapeutic ReGenX BiosciencesWashington, DC

gene therapy Batten disease(see also blood)

Phase IIregenxbio.com

PEG-PAL(Orphan Drug)

BioMarin PharmaceuticalNovato, CA

recombinantenzyme

phenylketonuria Phase IIbmrn.com

PF-06252616 PfizerNew York, NY

biologic muscular dystrophy Phase Ipfizer.com

PRX-102(alpha-galactosidase)

Protalix BiotherapeuticsCarmiel, Israel

recombinant protein

Fabry’s disease Phase I/IIprotalix.com

recombinant human C1 esterase inhibitor(Orphan Drug)

Pharming GroupLeiden, The NetherlandsSantarusSan Diego, CA

recombinant protein

hereditary angioedema(Fast Track)

Phase IIIsantarus.com

sebelipase alfa(Orphan Drug)

Synageva BioPharmaLexington, MA

recombinant protein

Wolman disease(lysosomal acid lipase deficiency)(Fast Track)

Phase II/IIIsynageva.com

UshStat®usher syndrome gene therapy(Orphan Drug)

Sanofi USBridgewater, NJ

gene therapy usher syndrome 1B Phase I/IIsanofi.com

WAS gene therapy Children’s Hospital BostonBoston, MAGenethonÉvry, France

gene therapy Wiskott-Aldrich syndrome Phase I/IIgenethon.fr

Biologic Medicines in Development

Medicines in Development Biologics 201354

infeCtious DiseAses

Product Name Sponsor ProductCategory

Indication Development Phase

ACAM-Cdiff(Clostridium difficiletoxoid vaccine)

Sanofi PasteurSwiftwater, PA

vaccine prevention and treatment of Clostridium difficile infections (Fast Track)

Phase IIsanofipasteur.us

ACE527 TD VaccinesSkorping, Denmark

vaccine prevention of enterotoxigenic Escherichia coli (ETEC) infections

Phase IItdvaccines.com

acellular pertussis combovaccine

Novartis VaccinesCambridge, MA

vaccine diphtheria, pertussis, tetanus Phase Inovartisvaccines.com

ADVAX (DNA vaccine)

Aaron Diamond AIDS Research Center New York, NY International AIDS Vaccine Initiative New York, NYIchor Medical SystemsSan Diego, CA

DNAvaccine

HIV infection prevention

------------------------------------------------HIV infection prevention(new delivery system)

Phase I completedadarc.orgiavi.org---------------------------------Phase I completedadarc.orgiavi.org

AERAS-402/Ad35 Aeras Rockville, MDCrucellLeiden, The Netherlands

recombinant vaccine

tuberculosis prevention Phase I aeras.orgcrucell.com

Agriflu®

influenza virus vaccineNovartis VaccinesCambridge, MA

vaccine influenza virus infections (pediatric)ention)

Phase IIInovartisvaccines.com

AGS-004 Argos Therapeutics Durham, NC

personalized dendritic cell vaccine

HIV infection Phase IIargostherapeutics.com

Alferon LDO®

interferon-alpha-n3oral

Hemispherx BiosciencePhiladelphia, PA

interferon human papillomavirusinfections------------------------------------------------influenza

Phase I/II hemispherx.net---------------------------------Phase Ihemisphrex.net

ALN-RSV01 Alnylam PharmaceuticalsCambridge, MA

RNAinterference

respiratory syncytial virus (RSV) treatment

Phase IIalnylam.com

Anthim™ (ETI-204) (Orphan Drug)

Elusys Therapeutics Pine Brook, NJ

mAb anthrax (Fast Track)

Phase Ielusys.com

anthrax immuneglobulin

CangeneBerwyn, PA

mAb anthrax Phase IIIcangene.com

anti-PD-L1 Bristol-Myers SquibbPrinceton, NJ

mAb hepatitis B(see also cancer)

Phase Ibms.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 55

InfectIous DIseases

Product Name Sponsor ProductCategory

Indication Development Phase

ASP-0113(cytomegalovirus DNA vaccine) (Orphan Drug)

AbbVieNorth Chicago, IL Astellas Pharma US Northbrook, IL Vical San Diego, CA

DNA vaccine

prevention of cytomegalovirus (CMV) infections in stem cell transplantation------------------------------------------------prevention of CMV infections in solid organ transplantation

Phase IIIus.astellas.com

---------------------------------Phase IIus.astellas.com

AVI-7100radavirsen

Sarepta TherapeuticsCambridge, MA

antisense influenza A virus infections Phase Isareptatherapeutics.com

AVI-7288(Orphan Drug)

Sarepta TherapeuticsCambridge, MA

antisense Marburg virusdisease

Phase Isareptatherapeutics.com

AVI-7537 Sarepta TherapeuticsCambridge, MA

antisense post-exposure treatment ofEbola virus infections

Phase Isareptatherapeutics.com

AVX101 (single gene HIV vaccine)

AlphaVax Rsch. Triangle Park, NC

gene vaccine

HIV-1 infection prevention Phase Ialphavax.com

AVX601 AlphaVax Rsch. Triangle Park, NC Novartis VaccinesCambridge, MA

genetically-modified vaccine

prevention of CMV infections Phase Ialphavax.comnovartisvaccines.com

AZD9773(anti-TNF-alpha poly-clonal antibody)

AstraZenecaWilmington, DE

polyclonal antibody

severe sepsis Phase IIastrazeneca.com

Bexsero®

meningococcal vaccine group B

Novartis Vaccines Cambridge, MA

recombinant vaccine

meningococcal group B infections Phase II novartisvaccines.com

BioThrax® anthrax vaccine

Emergent BioSolutions Rockville, MD

vaccine prevention of post-exposure anthrax infection in combination with other antibacterials(Fast Track)

Phase IIemergentbiosolutions.com

botulinum toxin vaccine DynPort Vaccine Frederick, MD

recombinant vaccine

botulism Phase II completedcsc.com/dvc

cell therapy Cell MedicaLondon, United KingdomCenter for Cell and Gene TherapyHouston, TX

celltherapy

viral infections(see also cancer)

Phase I/IIcellmedica.co.uk

chikungunya virusvaccine

InviragenFort Collins, Co

vaccine chikungunya virus infections Phase IIinviragen.com

Biologic Medicines in Development

Medicines in Development Biologics 201356

InfectIous DIseases

Product Name Sponsor ProductCategory

Indication Development Phase

cholera vaccine PaxVaxMenlo Park, CA

live, attenuated vaccine

prevention of cholera infection Phase Ipaxvax.com

Chronvac-C® hepatitis C DNA vaccine

Inovio PharmaceuticalsBlue Bell, PAChronTech PharmaHuddinge, Sweden

gene therapy vaccine

treatment of hepatitis C Phase IIinovio.com

CYT107 (interleukin-7)

Cytheris Rockville, MD

recombinant interleukin

HIV infection treatment(see also blood, cancer, transplantation)

Phase IIcytheris.com

Cytolin® anti-CD8 mAb

CytoDyn Lake Oswego, OR

mAb HIV infection treatment Phase Icytodyn.com

DCVax-001 CelldexNeedham, MARockefeller UniversityNew York, NY

recombinant vaccine

HIV infection prevention and treatment

Phase Icelldextherapeutics.com

dengue vaccine GlaxoSmithKlineRsch. Triangle Park, NC

vaccine dengue Phase Igsk.com

dengue vaccine Sanofi PasteurSwiftwater, PA

vaccine mild to severe dengue fever Phase IIIsanofipasteur.us

DENVax™tetravalent hybrid dengue virus vaccine

InviragenFort Collins, CO

vaccine dengue Phase Iinviragen.com

DermaVir™ Patch DNA topical patch vaccine

Genetic Immunity McLean, VA

DNA vaccine

HIV-1 infection treatment Phase IIgeneticimmunity.com

DNA vaccine(dengue)

Naval Medical Research Center Silver Spring, MDVicalSan Diego, CA

DNAvaccine

prevention of dengue fever Phase Ivical.com

DTP-HepB-polio-Hib(pediatric hexavalentvaccine)(V419)

MerckWhitehouse Station, NJSanofi PasteurSwiftwater, PA

vaccine diphtheria, tetanus, pertussis, hepatitis B, poliomyelitis, Haemophilus influenzae type b

Phase IIImerck.comsanofipasteur.us

Ebola vaccine CrucellLeiden, The Netherlands

DNA vaccine

prevention of Ebola virus infections Phase Icrucell.com

Biologic Medicines in Development

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InfectIous DIseases

Product Name Sponsor ProductCategory

Indication Development Phase

Ebola virus vaccine GenPharMt. Pleasant, SC

vaccine Ebola virus infections in clinical trialsgenphar.com

enterovirus 71 vaccine(INV21)

InviragenFort Collins, CO

vaccine prevention of head, foot and mouth disease

Phase Iinviragen.com

EP1300(malaria DNAvaccine)

VaxOnco Seoul, South Korea National Institute of Allergyand Infectious DiseasesBethesda, MD

DNAvaccine

prevention of malaria infection Phase I

FGI-101-1A6 Functional GeneticsGaithersburg, MD

mAb influenza Phase I completedfunctional-genetics.com

Fluad®

influenza virus vaccineNovartis VaccinesCambridge, MA

vaccine influenza virus infections in the elderly and children

Phase IIInovartisvaccines.com

FluBIok® derived from recombinant hemagglutinin (rHA)

Protein Sciences Meriden, CT

recombinant vaccine

prevention of influenza virus infections in infants and children

Phase I/IIproteinsciences.com

Fludase® DAS-181 inhalation

NexBio San Diego, CA

recombinant fusion protein

influenza A virus H1N1 infections,prevention of influenza virusinfections

Phase II completednexbio.com

FluLaval® QIVinfluenza virus vaccine

GlaxoSmithKlineRsch. Triangle Park, NC

vaccine prevention of influenzainfections

application submitted gsk.com

FluNhance®

influenza virus vaccine(rNA)

Protein SciencesMeriden, CT

recombinant vaccine

influenza virus infections Phase IIIproteinsciences.com

Fluzone® QIV IMquadrivalent influenza virus vaccine

Sanofi Pasteur Swiftwater, PA

vaccine seasonal influenza virus infections application submittedsanofipasteur.us

foravirumab CrucellLeiden, The Netherlands

mAb post-exposure prevention of rabies Phase IIcrucell.com

GI-5005 GlobeImmuneLouisville, CO

genetically-modified vaccine

hepatitis C Phase IIglobeimmune.com

Biologic Medicines in Development

Medicines in Development Biologics 201358

infeCtious DiseAses

Product Name Sponsor ProductCategory

Indication Development Phase

GSK692342(Mtb72F/AS02A)

AerasRockville, MDGlaxoSmithKlineRsch. Triangle Park, NC

recombinant vaccine

prevention of tuberculosis Phase IIgsk.com

GSK1437173A(varicella zoster vaccine)

GlaxoSmithKlineRsch. Triangle Park, NC

recombinant vaccine

prevention of varicella zostervirus infections

Phase IIIgsk.com

GSK1492903A (cytomegalovirus glycoprotein vaccine)

GlaxoSmithKline Rsch. Triangle Park, NC

recombinant vaccine

prevention of CMV infections Phase I gsk.com

GSK2189242A(Streptococcus pneumoniae recombinant-conjugated vaccine)

GlaxoSmithKlineRsch. Triangle Park, NC

recombinant vaccine

prevention of pneumococcalinfections

Phase IIgsk.com

GSK2654909A GlaxoSmithKlineRsch. Triangle Park, NC

vaccine prevention of influenza A virus H9N2subtype (avian flu)

Phase Igsk.com

GSK2654911A GlaxoSmithKlineRsch. Triangle Park, NC

vaccine prevention of influenza A virus H9N2subtype (avian flu)

Phase Igsk.com

GSK2830930A GlaxoSmithKlineRsch. Triangle Park, NC

vaccine prevention of pneumococcal infections

Phase Igsk.com

GSK2838497A GlaxoSmithKlineRsch. Triangle Park, NC

vaccine prevention of Haemophilus infections

Phase Igsk.com

HBV-002 Hawaii BiotechAiea, Hawaii

recombinant vaccine

prevention of West Nile virus infections

Phase I completedhibiotech.com

hepatitis C immune globulin(Orphan Drug)

Biotest PharmaceuticalsBoca Raton, FL

polyclonal antibody

hepatitis C prevention Phase IIbiotestpharma.com

hepatitis C vaccine therapy

OkairosBasel, Switzerland

gene vaccine prevention of hepatitis C Phase I/IIokairos.com

Heplisav™hepatitis B vaccine

Dynavax TechnologiesBerkeley, CA

DNA vaccine

prevention of hepatitis B application submitteddynavax.com

HerpV(herpes vaccine)

AgenusLexington, MA

recombinant protein vaccine

treatment of herpes simplex virus infections

Phase IIagenusbio.com

Hexaxim™ DTP-HepB-polio-Hib vaccine

Sanofi Pasteur Swiftwater, PA

vaccine diphtheria, tetanus, pertussis, hepa-titis B, poliomyelitis,Haemophilus influenzae type b

application submittedsanofipasteur.us

Biologic Medicines in Development

Medicines in Development Biologics 2013 59

infeCtious DiseAses

Product Name Sponsor ProductCategory

Indication Development Phase

HIV gene therapy AdaptimmunePhiladelphia, PACardiff UniversityCardiff, WalesUniversity of PennsylvaniaPhiladelphia, PA

gene therapy HIV infection Phase Iadaptimmune.com

HIV recombinant vaccine GlaxoSmithKlineRsch. Triangle Park, NC

recombinant vaccine

HIV infection prevention Phase Igsk.com

HIV recombinant vaccine GlaxoSmithKlineRsch. Triangle Park, NC

recombinant vaccine

HIV infection treatment Phase IIgsk.com

HIV vaccine Crucell Leiden, The Netherlands Beth Israel DeaconessMedical CenterBoston, MAInternational AIDS VaccineInitiativeNew York, NY

recombinantvaccine

HIV infection prevention Phase Icrucell.com

HIV vaccine GeoVax Smyrna, GA

vaccine HIV infection prevention Phase IIgeovax.com

HIV vaccine GeoVax Smyrna, GA

vaccine HIV infection treatment Phase I/IIgeovax.com

HIV vaccine Massachusetts General HospitalBoston, MAOpal TherapeuticsParkvill, Australia

vaccine HIV infection Phase I

HIV vaccine Novartis Vaccines Cambridge, MA

vaccine HIV infection Phase Inovartisvaccines.com

HIV vaccine PaxVaxSan Diego, CA

oral vaccine HIV infection prevention in clinical trialspaxvax.com

HIV vaccine(MAG pDNA)

Profectus BiosciencesBaltimore, MD

DNA vaccine

HIV infection prevention Phase Iprofectusbiosciences.com

HIV vaccine(rVSV)

Profectus BiosciencesBaltimore, MD

recombinant vaccine

HIV-1 infection prevention Phase Iprofectusbiosciences.com

HIV-1 gene therapy(MazF)

Takara BioShiga, Japan

gene therapy HIV-1 infection Phase Itakara-bio.com

Biologic Medicines in Development

Medicines in Development Biologics 201360

InfectIous DIseases

Product Name Sponsor ProductCategory

Indication Development Phase

HIV-1 vaccine(SAV001)

SumagenSeoul, South Korea

vaccine HIV-1 infection Phase Isumagen.co.kr

HIVAX™replication-defectiveHIV-1 vaccine

GeneCure BiotechnologiesNorcross, GA

vaccine HIV-1 infection Phase Igenecure.com

HSV-2 Tx Genocea BiosciencesCambridge, MA

T-cell vaccine treatment of herpes simplex virus 2 infections

Phase I/IIgenocea.com

human interferon alpha oral(Orphan Drug)

Amarillo Biosciences Amarillo, TX

interferon hepatitis C, influenza Phase IIamarbio.com

ibalizumab (TMB-355)

TaiMed Biologics USAIrvine, CA

mAb HIV-1 infection (intravenous)(Fast Track)------------------------------------------------HIV-1 infection (subcutaneous)

Phase IItaimedbiologics.com

---------------------------------Phase Itaimedbiologics.com

ID-93/GLA-SE Aeras Global TB Vaccine FoundationRockville, MDInfectious Disease Research InstituteSeattle, WA

recombinant fusionprotein vaccine

tuberculosis Phase Iaeras.orgidri.org

Imvamune®

smallpox vaccineBavarian NordicKvistgaard, Denmark

vaccine smallpox Phase IIbavarian-nordic.com

influenza A virus H1N1vaccine

CEL-SCIVienna, VA

peptide vaccine

prevention of influenza A virus H1N1 subtype

Phase Icel-sci.com

influenza A virus H1N1vaccine

iBioNewark, DE

vaccine prevention of influenza A virusH1N1 subtype

Phase Iibioinc.com

influenza A virus H1N1 vaccine

MedicagoQuebec, Canada

VLP vaccine influenza A virus H1N1 subtype Phase Imedicago.com

influenza A virus H1N1 vaccine (FVH-1)

Inovio PharmaceuticalsBlue Bell, PA

DNA vaccine influenza A virus H1N1 subtypeprevention

Phase Iinovio.com

influenza A virus H1N1 vaccine (li-key hybrid peptide vaccine)

Antigen ExpressWorcester, MA

peptide vaccine

influenza A virus H1N1 infections Phase Iantigenexpress.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 61

InfectIous DIseases

Product Name Sponsor ProductCategory

Indication Development Phase

influenza A virus H5N1cell-cultured based vaccine

GlaxoSmithKlineRsch. Triangle Park, NC

cell-cultured vaccine

prevention of pandemic influenza A virus H5N1 subtype infections

Phase Igsk.com

influenza A virus H5N1 intranasal vaccine

NanotherapeuticsAlachua, FL

vaccine prevention of influenza A virus H5N1 subtype

Phase Inanotherapeutics.com

influenza A virus H5N1 vaccine

iBioNewark, DE

vaccine influenza A virus H5N1 subtype Phase Iibioinc.com

influenza A virus H5N1 vaccine

Inovio PharmaceuticalsBlue Bell, PA

vaccine prevention of influenza A virus H5N1 subtype

Phase Iinovio.com

influenza A virus H5N1 vaccine

MedicagoQuebec, Canada

VLP vaccine influenza A virus H5N1 subtype Phase Imedicago.com

influenza A virus H5N1 vaccine

Novartis VaccinesCambridge, MA

vaccine prevention of influenza A virus H5N1 subtype

Phase IInovartisvaccines.com

influenza A virus H5N1 vaccine

NovavaxRockville, MD

vaccine prevention of influenza A virusH5N1 subtype

Phase IInovavax.com

influenza A virus H5N1 vaccine

PaxVaxSan Diego, CA

vaccine prevention of influenza A virusH5N1 subtype

Phase Ipaxvax.com

influenza A virus H5N1 vaccine

VaxartSan Francisco, CA

vaccine prevention of influenza A virusH5N1 subtype

Phase Ivaxart.com

influenza A virus H5N1 vaccine(li-key hybrid peptide vaccine)

Antigen ExpressWorcester, MA

peptide vaccine

influenza A virus H5N1 subtype Phase Iantigenexpress.com

influenza A virus H9N2 vaccine

Baxter InternationalDeerfield, IL

vaccine influenza A virus H9N2 subtype Phase I/IIbaxter.com

influenza virus-like particle vaccine

NovavaxRockville, MD

vaccine prevention of influenza virus infections

Phase IInovavax.com

influenza virus vaccine(intradermal)

CrucellLeiden, The Netherlands

vaccine prevention of influenza virus infections

Phase IIcrucell.com

Influvac® TCcell culture-derivedinfluenza vaccine

AbbVieNorth Chicago, IL

cell-cultured vaccine

influenza virus infections (prevention)

Phase Iabbvie.com

Biologic Medicines in Development

Medicines in Development Biologics 201362

Biologic Medicines in Development

infeCtious DiseAses

Product Name Sponsor ProductCategory

Indication Development Phase

interferon-alpha-2b HanAll BiopharmaSeoul, South Korea

interferon hepatitis C Phase IIhanallbiopharma.com

interferon alpha-2binfusion

MedtronicMinneapolis, MN

interferon hepatitis C Phase II completedmedtronic.com

Ixiaro®

Japanese encephalitisvaccine

IntercellVienna, AustriaNovartis Vaccines Cambridge, MA

vaccine prevention of Japanese encephalitisin children

application submittedintercell.comnovartisvaccines.com

KB001-A(antibody-fragment product)

KaloBios Pharmaceuticals South San Francisco, CA Sanofi PasteurSwiftwater, PA

mAb Pseudomonas infections in cysticfibrosis patients (KalBios only)------------------------------------------------prevention of ventilator-associated pneumonia

Phase I/IIkalobios.com---------------------------------Phase Ikalobios.comsanofipasteur.us

KD-247 KaketsukenKumamoto, Japan

mAb HIV-1 infection Phase Ikaketsuken.or.jp

LIQ-001 Liquidia TechnologiesRsch. Triangle Park, NC

vaccine influenza virus infections Phase I liquidia.com

Lyme disease vaccine Baxter HealthcareDeerfield, IL

recombinant vaccine

prevention of Lyme disease Phase I/IIbaxter.com

malaria vaccine CrucellLeiden, The NetherlandsGlaxoSmithKlineRsch. Triangle Park, NC

recombinant vaccine

malaria Phase I/IIcrucell.comgsk.com

malaria vaccine GenVecGaithersburg, MDU.S. Naval Medical Research CenterSilver Spring, MD

recombinant vaccine

malaria Phase I/IIgenvec.com

malaria vaccine SanariaRockville, MD

vaccine prevention of malaria Phase I/IIsanaria.com

Marburg virus DNA vaccine

GenPharMt. Pleasant, SC

vaccine Marburg virus disease Phase Igenphar.com

MBL-HCV1 MassBiologicsBoston, MA

mAb hepatitis C Phase IIumassmed.edu/ massbiologics

Medicines in Development Biologics 2013 63

Biologic Medicines in Development

infeCtious DiseAses

Product Name Sponsor ProductCategory

Indication Development Phase

MEDI-550(pandemic influenza virus vaccine)

AstraZenecaWilmington, DEMedImmuneGaithersburg, MD

vaccine pandemic influenza prevention Phase Iastrazeneca.commedimmune.com

MEDI-557 (RSV mAb- extended half-life)

AstraZenecaWilmington, DEMedImmuneGaithersburg, MD

mAb prevention of RSV infections in high risk adults

Phase Iastrazeneca.commedimmune.com

MEDI-559(RSV vaccine)

AstraZenecaWilmington, DEMedImmuneGaithersburg, MD

vaccine prevention of RSV infections(pediatric)

Phase I/IIastrazeneca.commedimmune.com

MenABCWY(meningococcal vaccine groups A,C,Y,W-135 conjugate)

Novartis Vaccines Cambridge, MA

vaccine meningococcal infections Phase IInovartisvaccines.com

meninge ACYW conjugate vaccine(2nd-generation meningococcal conjugateinfant vaccine)

Sanofi PasteurSwiftwater, PA

vaccine meningococcal infections Phase IIsanofipasteur.us

meningococcal vaccine groups A,C,Y,W-135 conjugate

GlaxoSmithKlineRsch. Triangle Park, NC

vaccine meningococcal infections Phase IIIgsk.com

meningococcal vaccine groups A,C,Y,W-135 conjugate

JN-International MedicalOmaha, NE

vaccine meningococcal group A,C,Y,W-135 infections

Phase Ijn-vaccines.org

Menveo®

meningococcal vaccine groups A,C,Y, W-135 conjugate vaccine

Novartis VaccinesCambridge, MA

vaccine meningococcal groups A,C,Y,W-35 infection in infants (ages 2–12 months)

application submittednovartisvaccines.com

miravirsen(SPC3649)

Santaris Pharma A/S USASan Diego, CA

antisense hepatitis C Phase IIsantaris.com

MK-3415A(actoxumab/ bezlotoxumab)

MerckWhitehouse Station, NJ

mAb Clostridium difficile infections Phase IIImerck.com

MMR vaccine live(GSK209762)

GlaxoSmithKlineRsch. Triangle Park, NC

vaccine prevention of measles, mumps, rubella

Phase IIIgsk.com

MnB rLP2086 (PF-05212366)

PfizerNew York, NY

recombinantvaccine

adolescent and young adult meningitis B

Phase IIIpfizer.com

Medicines in Development Biologics 201364

Biologic Medicines in Development

infeCtious DiseAses

Product Name Sponsor ProductCategory

Indication Development Phase

MVA-BN®

HIV multiantigen vaccineBavarian Nordic Washington, DC

vaccine HIV-1 infection prevention and treatment

Phase I/IIbavarian-nordic.com

NB-001 NanoBio Ann Arbor, MI

nano-emulsion

recurrent herpes simplex virus infections

Phase IIInanobio.com

NB-002 NanoBio Ann Arbor, MI

nano-emulsion

distal subungual onychomycosis of the toe

Phase IInanobio.com

NB-1008(intranasal)

NanoBioAnn Arbor, MI

nano-emulsion vaccine

prevention of influenza virus infections

Phase I completednanobio.com

NDV-3 NovaDigm TherapeuticsGrand Forks, ND

recombinantvaccine

prevention of candidiasis, prevention of methicillin-resistantStaphylococcus aureus (MRSA) infections

Phase Inovadigm.net

norovirus virus-like particle vaccineintranasal

LigoCyte PharmaceuticalsBozeman, MT

vaccine norovirus infections Phase I/IIligocyte.com

Nuthrax™anthrax vaccine adsorbed with CPG 7909 adjuvant

Emergent BioSolutions Rockville, MD

vaccine anthrax (Fast Track)

Phase Iemergentbiosolutions.com

PanBlok™influenza A virus H5N1 vaccine

Protein SciencesMeriden, CT

vaccine prevention of influenza A virusH5N1 subtype infections

Phase I/IIproteinsciences.com

peginterferon lambda-1a Bristol-Myers SquibbPrinceton, NJ

interferon hepatitis C

------------------------------------------------hepatitis B

Phase IIIbms.com---------------------------------Phase IIbms.com

Pennvax®-B DNA vaccine (clade B)

Inovio Pharmaceuticals Blue Bell, PA

DNAvaccine

HIV infection prevention and treatment

Phase Iinovio.com

Pennvax®-G DNA vaccine (clades A, C, D)

Inovio Pharmaceuticals Blue Bell, PA

DNAvaccine

HIV infection prevention Phase Iinovio.com

PF-06290510(SA4Ag)

PfizerNew York, NY

vaccine Staphylococcus aureus infections Phase IIpfizer.com

Medicines in Development Biologics 2013 65

Biologic Medicines in Development

infeCtious DiseAses

Product Name Sponsor ProductCategory

Indication Development Phase

PF-06425090(Clostridium difficile vaccine)

PfizerNew York, NY

vaccine Clostridium difficile colitis Phase Ipfizer.com

plague injectable vaccine DynPort VaccineFrederick, MD

vaccine Yersinia infections Phase IIcsc.com/dvc

Preflucel™seasonal influenza virus vaccine

Baxter InternationalDeerfield, ILDynPort VaccineFrederick, MD

vaccine prevention of influenza virus infections

Phase IIIbaxter.comcsc.com/dvc

Prepandrix™H5N1 (pre-)pandemicinfluenza virus vaccine

GlaxoSmithKlineRsch. Triangle Park, NC

vaccine prevention of influenza A virus H5N1 subtype for pandemic use------------------------------------------------prevention of influenza A virus H5N1 subtype for pandemic use in children and adolescents

application submittedgsk.com---------------------------------Phase IIIgsk.com

PreviThrax™recombinant protective antigen anthrax vaccine

Emergent BiosolutionsRockville, MD

recombinant vaccine

anthrax(Fast Track)

Phase IIemergentbiosolutions.com

PRO 140 CytoDynLake Oswego, OR

mAb HIV infection Phase II completedcytodyn.com

Quadracel®DTP, polio vaccine

Sanofi PasteurSwiftwater, PA

vaccine diphtheria, tetanus, pertussis, poliomyelitis (4-6 years of age)

Phase IIIsanofipasteur.us

rabies VRVg vaccine (purified vero rabies vaccine)

Sanofi PasteurSwiftwater, PA

vaccine prevention of rabies Phase IIsanofipasteur.us

respiratory syncytial virus VLP vaccine

NovavaxRockville, MD

recombinant vaccine

prevention of RSV infections Phase IInovavax.com

Rivax™ricin vaccine(Orphan Drug)

SoligenixPrinceton, NJ

vaccine ricin poisoning(pre-exposure)

Phase Isoligenix.com

rotavirus vaccine(live attenuated tetrava-lent oral vaccine)

Sanofi PasteurSwiftwater, PA

vaccine rotavirus Phase Isanofipasteur.us

SAR279356(anti-PNAG mAB)

Sanofi USBridgewater, NJ

mAb prevention of bacterial infections Phase IIsanofi.com

Medicines in Development Biologics 201366

Biologic Medicines in Development

infeCtious DiseAses

Product Name Sponsor ProductCategory

Indication Development Phase

SB-728-T Sangamo BioSciencesRichmond, CA

gene therapy

HIV infection Phase IIsangamo.com

Soliris®

eculizumab(Orphan Drug)

Alexion PharmaceuticalsCheshire, CT

mAb Shiga toxin E. coli-related hemolytic uremic syndrome (STEC-HUS)(see also autoimmune, eye, transplantation)

Phase IIalxn.com

Stealth Vector®

HGTV-43™ antisense gene medicine

Enzo Therapeutics New York, NY

antisense HIV-1 infection treatment Phase I/IIenzo.com

streptococcal B vaccine conjugate

Novartis VaccinesCambridge, MA

mAb prevention of streptococcal Binfections

Phase Inovartisvaccines.com

Streptococcus pneumoniae vaccine (meningitis and pneumonia vaccine)

Sanofi PasteurSwiftwater, PA

vaccine treatment of meningitis and pneumonia

Phase Isanofipasteur.us

TCN-032(IgG mAb)

Theraclone SciencesSeattle, WA

mAb influenza A virus infections Phase Itheraclone-sciences.com

TCN-202 Theraclone SciencesSeattle, WA

mAb CMV infections Phase Itheraclone-sciences.com

TDENV-PIV(tetravalent vaccine)

GlaxoSmithKlineRsch. Triangle Park, NC

vaccine dengue Phase Igsk.com

TG-4040 TransgeneCedex, France

recombinant vaccine

hepatitis C Phase IItransgene.fr

Thravixa™fully human anthrax mAb(Orphan Drug)

Emergent BioSolutionsRockville, MD

mAb post-exposure treatment of anthrax(Fast Track)

Phase I completedemergentbiosolutions.com

TKM-Ebola Tekmira PharmaceuticalsBurnaby, Canada

RNA interference

Ebola virus infections Phase Itekmirapharm.com

tuberculosis vaccine(recombinant subunit vaccine)

Sanofi PasteurSwiftwater, PA

recombinant vaccine

tuberculosis Phase Isanofipasteur.us

tularemia vaccine DynaPort VaccineFrederick, MD

vaccine tularemia Phase I completedcsc.com/dvc

TUTI-16(lipoprotein vaccine)

ThymonShort Hills, NJ

vaccine HIV-1 infection treatment Phase I/II

Medicines in Development Biologics 2013 67

Biologic Medicines in Development

InfectIous DIseases

Product Name Sponsor ProductCategory

Indication Development Phase

UB-421 United Biomedical Hauppauge, NY

mAb HIV-1 infection treatment Phase IIunitedbiomedical.com

V114(pneumococcal 15-valentvaccine conjugate)

MerckWhitehouse Station, NJ

vaccine prevention of pneumococcal infections

Phase IImerck.com

V212 MerckWhitehouse Station, NJ

vaccine prevention of herpes zoster Phase IIImerck.com

V503 MerckWhitehouse Station, NJ

vaccine prevention of human papillomavirusinfections(see also cancer)

Phase IIImerck.com

Vacc-4x Bionor PharmaOslo, Norway

vaccine HIV-1 infection treatment Phase IIbionorpharma.com

VAX102(flagellin.HuM2e)

VaxInnateCranbury, NJ

recombinant fusionprotein vaccine

prevention of influenza A virus Phase I/II completedvaxinnate.com

VAX125(flagellin.HuHa)

VaxInnateCranbury, NJ

recombinant fusionprotein vaccine

prevention of influenza A virus Phase II completedvaxinnate.com

VAX128 Emergent BioSolutionsRockville, MDVaxInnateCranbury, NJ

recombinant vaccine

prevention of influenza A virus H1N1 subtype

Phase Iemergentbipsolutions.comvaxinnate.com

VAX161 Emergent BioSolutionsRockville, MDVaxInnateCranbury, NJ

recombinant vaccine

prevention of influenza A virus H5N1 subtype

Phase Iemergentbiosolutions.comvaxinnate.com

VaxiGrip®

quadrivalent inactivated influenza vaccine

Sanofi PasteurSwiftwater, PA

vaccine seasonal influenza virus infections Phase IIIsanofipasteur.us

VGX-3400 Inovio Pharmaceuticals Blue Bell, PA

DNAvaccine

prevention of influenza A virus H5N1 subtype

Phase Iinovio.com

visceral leishmaniasis vaccine

Infectious DiseaseResearch InstituteSeattle, WA

vaccine visceral leishmaniasis Phase Iidri.org

Medicines in Development Biologics 201368

infeCtious DiseAses

Product Name Sponsor ProductCategory

Indication Development Phase

XOMA 3AB XOMABerkeley, CANational Institute of Allergy and Infectious DiseasesBethesda, MD

mAb botulism Phase Ixoma.com

Zostavax®

varicella zoster virus vaccine live

MerckWhitehouse Station, NJ

vaccine prevention of herpes zoster Phase Imerck.com

musCuloskeletAl DisorDers/Arthritis

Product Name Sponsor ProductCategory

Indication Development Phase

ABT-981 AbbVieNorth Chicago, IL

mAb osteoarthritis Phase Iabbvie.com

AMG 167 AmgenThousand Oaks, CA

antibody metabolic bone diseases Phase Iamgen.com

AMG 745 Amgen Thousand Oaks, CA

mAb muscular atrophy Phase Iamgen.com

AMG 827(brodalumab)

AstraZenecaWilmington, DEAmgenThousand Oaks, CA

mAb psoriatic arthritis(see also respiratory, skin)

Phase IIastrazeneca.comamgen.com

blosozumab(LY2541546)

Eli LillyIndianapolis, IN

mAb osteoporosis Phase IIlilly.com

BYM338 Novartis PharmaceuticalsEast Hanover, NJ

mAb sporadic inclusion body myositis,muscular atrophy(see also cancer)

Phase IInovartis.com

Chondrogen®

mesenchymal stem cell therapy for meniscal repair

Osiris Therapeutics Columbia, MD

stem cell therapy

knee injuries, prevention of osteoarthritis

Phase IIosiris.com

Cimzia® certolizumab pegol

UCB Smyrna, GA

mAb psoriatic arthritis(see also autoimmune)

Phase IIIucb.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 69

musCuloskeletAl DisorDers/Arthritis

Product Name Sponsor ProductCategory

Indication Development Phase

clazakizumab(anti-IL6)

Bristol-Myers SquibbPrinceton, NJAlder BiopharmaceuticalsBothell, WA

mAb psoriatic arthritis(see also autoimmune, digestive)

Phase IIbms.comalderbio.com

Dysport®abobotulinumtoxinA

IpsenParis, France

bacterial protein

focal spasticity of upper and lower limb

Phase III ipsen.com

FGF-18 (sprifermin)

EMD Serono Rockland, MA

growth factor acute cartilage injury of the knee Phase IIemdserono.com

gevokizumab(IL-1B inhibitor mAb)

XOMABerkeley, CA

mAb osteoarthritis of the hand(see also diabetes, eye, skin)

Phase IIxoma.com

Humira®

adalimumabAbbVieNorth Chicago, IL

mAb spondylarthritis(see also autoimmune, digestive, eye, skin, other)

Phase IIIabbvie.com

Ilaris®

canakinumabNovartis Pharmaceuticals East Hanover, NJ

mAb gouty arthritis(see also autoimmune, cardiovascular, diabetes)------------------------------------------------osteoarthritis, polymyalgiarheumatica

application submittednovartis.com

---------------------------------Phase IInovartis.com

ixekizumab(IL-17 antibody)

Eli LillyIndianapolis, IN

mAb psoriatic arthritis(see also skin)

Phase IIIlilly.com

juvenile chondrocyte grafts

ISTO Technologies St. Louis, MO

celltherapy

cartilage disorders Phase IIIisotech.com

KTP-001(recombinant human matrix metalloproteinase)

KaketsukenKumamoto, JapanTeijin PharmaTokyo, Japan

recombinant protein

intervertebral disc displacement Phase I/IIkaketsuken.or.jpteijin-pharma.co.jp

LY2495655(anti-myostatin mAb)

Eli LillyIndianapolis, IN

mAb disuse muscular atrophy(see also cancer)

Phase IIlilly.com

MACI®matrix-induced autologous chondrocyte implantation

GenzymeCambridge, MA

cell therapy articular cartilage defects Phase IIIgenzyme.com

MCS110 Novartis PharmaceuticalsEast Hanover, NJ

mAb synovitis Phase IInovartis.com

Biologic Medicines in Development

Medicines in Development Biologics 201370

musCuloskeletAl DisorDers/Arthritis

Product Name Sponsor ProductCategory

Indication Development Phase

mesenchymal precursor cell (MPC) product

MesoblastNew York, NY

stem cell therapy

spinal fusion, spondylosis(see also cardiovascular, diabetes, transplantation)

Phase IImesoblast.com

mesenchymal stem cell therapy

MedipostSeoul, South Korea

stem cell therapy

osteoarthritis Phase I/IImedi-post.com

NeoCart® autologous chondrocyteimplantation

HistogenicsWaltham, MA

celltherapy

cartilage disorders Phase IIIhistogenics.com

Orencia®

abataceptBristol-Myers SquibbPrinceton, NJ

recombinant fusionprotein

psoriatic arthritis(see also autoimmune, other)

Phase IIbms.com

Prolia®

denosumabAmgen Thousand Oaks, CA

mAb male osteoporosis(see also autoimmune)

Phase IIIamgen.com

romosozumab(AMG 785)

AmgenThousand Oaks, CA

mAb postmenopausal osteoporosis Phase IIIamgen.com

SAR391786(REGN1033)

Regeneron PharmaceuticalsTarrytown, NYSanofi USBridgewater, NJ

mAb treatment of muscle atrophy post-orthopedic surgery

Phase Iregeneron.comsanofi.com

secukinumab (AIN 457)

Novartis Pharmaceuticals East Hanover, NJ

mAb

psoriatic arthritis(see also autoimmune, eye, skin)------------------------------------------------polymyalgia rheumatica

Phase IIInovartis.com---------------------------------Phase IInovartis.com

SI-6603 SeikagakuTokyo, Japan

enzyme intervertebral disc displacement

Phase IIseikagaku.co.jp

Stelara®

ustekinumab Janssen BiotechHorsham, PA

mAb psoriatic arthritis(see also autoimmune, digestive, other)

application submittedjanssenbiotech.com

tanezumab PfizerNew York, NY

mAb osteoarthritis(see also neurologic)

Phase IIIpfizer.com

teriparatide(recombinant PTH 1-34)

Eli LillyIndianapolis, IN

recombinant hormone

healing of fractures in patients withosteoporosis

Phase IIIlilly.com

TG-C TissueGene Rockville, MD

celltherapy

osteoarthritis Phase IItissuegene.com

Xiaflex®

collagenase clostridiumhistolyticum

Auxilium PharmaceuticalsChesterbrook, PABioSpecifics Technologies

peptide frozen shoulder(see also cancer, other)

Phase IIauxilium.combiospecifics.com

Biologic Medicines in Development

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neurologiC DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

AAB-002 Janssen Alzheimer Immunotherapy South San Francisco, CA PfizerNew York, NY

mAb Alzheimer’s disease Phase 0janimm.compfizer.com

AAB-003/PF-05236812 Janssen Alzheimer Immunotherapy South San Francisco, CA PfizerNew York, NY

mAb Alzheimer’s disease Phase Ijanimm.compfizer.com

AAV1-FS344(gene therapy-delivered myostatin inhibitor)(Orphan Drug)

Milo BiotechnologyCleveland, OH

gene therapy Becker musculardystrophy

Phase I/IImilobiotechnology.com

ABT-110 AbbVieNorth Chicago, IL

mAb chronic pain Phase IIabbvie.com

ACC-001(PF-05236806)

Janssen Alzheimer ImmunotherapySouth San Francisco, CAPfizerNew York, NY

peptide vaccine

Alzheimer’s disease Phase IIjanimm.compfizer.com

AD02 vaccine AFFiRisVienna, AustriaGlaxoSmithKlineRsch. Triangle Park, NC

vaccine Alzheimer’s disease Phase IIaffiris.comgsk.com

AD03 vaccine AFFiRisVienna, AustriaGlaxoSmithKlineRsch. Triangle Park, NC

vaccine Alzheimer’s disease Phase Iaffiris.comgsk.com

AGN-214868(senrebotase)

AllerganIrvine, CA

recombinantprotein

postherpetic neuralgia(see also other)

Phase IIallergan.com

ALD403 Alder BiopharmaceuticalsBothell, WA

mAb prevention of migraine Phase Ialderbio.com

ATI355(anti-Nogo-A mAb)(Orphan Drug)

Novartis PharmaceuticalsEast Hanover, NJ

mAb spinal cord injury Phase Inovartis.com

Biologic Medicines in Development

Medicines in Development Biologics 201372

Biologic Medicines in Development

Neurologic DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

AYX-1 AdynxxSan Francisco, CA

oglionucleo-tide

postoperative pain Phase Iadynxx.com

BA-210 (Orphan Drug)

BioAxone BiosciencesCambridge, MA

recombinant fusion protein

spinal cord injury Phase IIbioaxonebio.com

BAN2401(amyloid beta-protein inhibitor)

BioArctic NeuroscienceStockholm, SwedenEisaiWoodcliff Lake, NJ

mAb Alzheimer’s disease Phase Ibioarctic.seeisai.com

bapineuzumab (subcutaneous)(AAB-001)

Janssen Alzheimer ImmunotherapySouth San Francisco, CA PfizerNew York, NY

mAb Alzheimer’s disease Phase IIjanimm.compfizer.com

Botox®

onabotulinumtoxinAAllerganIrvine, CA

bacterial protein

motor neuron disease(see also other)

Phase IIIallergan.com

CAD106 Cytos BiotechologyZurich, SwitzerlandNovartis PharmaceuticalsEast Hanover, NJ

therapeutic vaccine

Alzheimer’s disease Phase IIcytos.comnovartis.com

CERE-110 (AAV-NGF)

Ceregene San Diego, CA

gene therapy Alzheimer’s disease Phase IIceregene.com

CERE-120 (AAV-NTN)

Ceregene San Diego, CA

gene therapy Parkinson’s disease Phase IIceregene.com

chondrocyte tissue grafts

ISTO Technologies St. Louis, MO

celltherapy

back pain Phase Iisotech.com

crenezumab(anti-Abeta)

GenentechSouth San Francisco, CA

mAb Alzheimer’s disease Phase IIgene.com

fulranumab Janssen Research & DevelopmentRaritan, NJ

mAb cancer pain Phase IIjanssenrnd.com

GDNF-producing adultstem cell therapy(Orphan Drug)

BrainStorm Cell TherapeuticsNew York, NY

stem celltherapy

amyotrophic lateral sclerosis(ALS)

Phase I/IIbrainstorm-cell.com

GSK1223249(NOGO-A mAb)

GlaxoSmithKlineRsch. Triangle Park, NC

mAb ALS (see also autoimmune)

Phase Igsk.com

GSK933776A (anti-B amyloid mAb)

GlaxoSmithKline Rsch. Triangle Park, NC

mAb Alzheimer’s disease (see also eye)

Phase I completedgsk.com

Medicines in Development Biologics 2013 73

neurologiC DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

GZ404477(AAV-hAADC gene therapy)

Genzyme Cambridge, MA

gene therapy Parkinson’s disease Phase Igenzyme.com

HuCNS-SC®

adult neural stem cell therapy(intraosseous)

StemCellsNewark, CA

stem cell therapy

thoracic spinal cord injury(see also eye, genetic)

Phase I/IIstemcellsinc.com

LY2951742(CGRP peptide)

Arteaus TherapeuticsCambridge, MAEli LillyIndianapolis, IN

mAb migraine prevention Phase IIlilly.com

MEDI-5117(anti-IL-6 mAb)

AstraZenecaWilmington, DEMedImmuneGaithersburg, MD

mAb osteoarthritis pain Phase Iastrazeneca.commedimmune.com

neurological disordersstem cell therapy

TCA Cellular TherapyCovington, LA

stem cell therapy

ALS, spinal cord injuries Phase Itcacellulartherapy.com

NSI-566(Orphan Drug)

NeuralstemRockville, MD

stem celltherapy

ALS Phase Ineuralstem.com

RG1450 (gantenerumab)

Roche Nutley, NJ

mAb

prodromal Alzheimer’s disease Phase II/IIIroche.com

RN-307(anti-CGRP mAb)

Labrys BiologicsSan Francisco, CA

mAb migraine Phase II

SAR228810(anti-protofibrillar AB mAb)

Sanofi USBridgewater, NJ

mAb Alzheimer’s disease Phase Isanofi.com

solanezumab(LY2062430)

Eli Lilly Indianapolis, IN

mAb Alzheimer’s disease Phase IIIlilly.com

tanezumab PfizerNew York, NY

mAb chronic pain(see also musculoskeletal)

Phase IIpfizer.com

UB-311immunotherapeutic

United BiomedicalHauppauge, NY

vaccine Alzheimer’s disease Phase IIunitedbiomedical.com

V950 Merck Whitehouse Station, NJ

vaccine Alzheimer’s disease Phase I completedmerck.com

VM-202(modified hepatocytegrowth factor genetherapy)

VM BioPharmaAtlanta, GA

genetherapy

diabetic neuropathy(see also cardiovascular)

Phase IIviromed.co.kr

Biologic Medicines in Development

Medicines in Development Biologics 201374

neurologiC DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

Xeomin®

incobotulinumtoxinAMerz PharmaceuticalsGreensboro, NC

bacterial protein

post-stroke spasticity

-----------------------------------------------sialorrhea in patients withParkinson’s disease and ALS

Phase IIImerzusa.com---------------------------------Phase IImerzusa.com

respirAtory DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

ALT-836 Altor BioScience Miramar, FL GenentechSouth San Francisco, CA

mAb acute lung injury, adult respiratory distress syndrome(see also cancer)

Phase IIaltorbioscience.comgene.com

AMG 157/MEDI-9929 Amgen Thousand Oaks, CA AstraZenecaWilmington, DE

mAb asthma Phase Iamgen.comastrazeneca.com

AMG 761 AmgenThousand Oaks, CA

mAb asthma Phase Iamgen.com

AMG 827 (brodalumab)

Amgen Thousand Oaks, CA AstraZenecaWilmington, DE

mAb asthma(see also musculoskeletal, skin)

Phase IIamgen.comastrazeneca.com

ASM8 PharmaxisExton, PA

antisense asthma Phase IIpharmaxis.com.au

benralizumab AstraZenecaWilmington, DEMedImmuneGaithersburg, MD

mAb asthma, chronic obstructive pulmonary disease (COPD)

Phase IIastrazeneca.commedimmune.com

Bosatria™ mepolizumab

GlaxoSmithKline Rsch. Triangle Park, NC

mAb asthma Phase IIIgsk.com

carlumab Janssen Biotech Horsham, PA

mAb pulmonary fibrosis Phase IIjanssenbiotech.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 75

respirAtory DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

CNTO-3157 Janssen BiotechHorsham, PA

mAb asthma Phase Ijanssenbiotech.com

CNTO-5825 Janssen BiotechHorsham, PA

mAb allergic asthma Phase Ijanssenbiotech.com

CYT003 Cytos BiotechnologyZurich, Switzerland

vaccine allergic asthma Phase IIcytos.com

Excellair™syk kinase silencer

ZaBeCor PharmaceuticalsBala Cynwyd, PA

RNAinterference

asthma Phase IIzabecor.com

FG-3019 FibroGen San Francisco, CA

mAb idiopathic pulmonary fibrosis(see also cancer, other)

Phase IIfibrogen.com

grass pollen extract StallergenesCedex, France

immuno-therapy

seasonal allergic rhinitis Phase IIIstallergenes.com

GS-6624 (simtuzumab)

Gilead Sciences Foster City, CA

mAb idiopathic pulmonary fibrosis(see also cancer, other)

Phase Igilead.com

GSK2586881(APN01)

Apeiron BiologicsVienna, AustriaGlaxoSmithKlineRsch. Triangle Park, NC

recombinantprotein

acute lung injury Phase Igsk.com

KB003 KaloBios PharmaceuticalsSouth San Francisco, CA

mAb severe asthma Phase IIkalobios.com

MEDI-4212(anti-IgE mAb)

AstraZenecaWilmington, DEMedImmuneGaithersburg, MD

mAb asthma Phase Iastrazeneca.commedimmune.com

MEDI-7814(anti-C5/C5a mAb)

AstraZenecaWilmington, DEMedImmuneGaithersburg, MD

mAb COPD Phase Iastrazeneca.commedimmune.com

MEDI-8968(anti-IL-1R mAb)

AstraZenecaWilmington, DEMedImmuneGaithersburg, MD

mAb COPD Phase IIastrazeneca.commedimmune.com

Biologic Medicines in Development

Medicines in Development Biologics 201376

respirAtory DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

MK-3641ragweed allergy vaccine (sublingual)

MerckWhitehouse Station, NJ

immuno-therapy vaccine

ragweed pollen-induced rhinoconjunctivitis

Phase IIImerck.com

MK-7243grass pollen allergy tablet vaccine

MerckWhitehouse Station, NJ

immuno-therapy

grass pollen-induced allergic rhinoconjunctivitis

Phase IIImerck.com

MK-8237house dust mite allergy vaccine tablet

MerckWhitehouse Station, NJ

vaccine allergic asthma, allergic rhinitis, allergic rhinoconjunctivitis

Phase IImerck.com

Pollinex® Quattroinjectable MPL allergy vaccine

Allergy TherapeuticsWest Sussex, United Kingdom

vaccine grass pollen hypersensitivity, ragweed pollen hypersensitivity-----------------------------------------------tree pollen hypersensitivity

Phase IIIallergytherapeutics.com---------------------------------Phase IIallergytherapeutics.com

PRM-151(rhPTX-2 for injection)(Orphan Drug)

PromediorLexington, MA

recombinantprotein

idiopathic pulmonary fibrosis(see also other)

Phase Ipromedior.com

Prochymal®remestemcel-L

Osiris TherapeuticsColumbia, MD

stem celltherapy

COPD(see also cardiovascular, diabetes, digestive, transplantation)

Phase IIosiris.com

QAX576 Novartis Pharmaceuticals East Hanover, NJ

mAb asthma, idiopathic pulmonary fibrosis (see also digestive)

Phase IInovartis.com

QBX258 Novartis PharmaceuticalsEast Hanover, NJ

mAb asthma Phase IInovartis.com

QGE031 Novartis PharmaceuticalsEast Hanover, NJ

mAb allergic asthma (see also skin)

Phase IInovartis.com

quilizumab(anti-M1 prime mAb)

Genentech South San Francisco, CA

mAb allergic asthma, allergic rhinitis Phase IIgene.com

ragweed allergy immunotherapy(sublingual)

StallergenesCedex, France

vaccine seasonal allergic rhinitis Phase Istallergenes.com

ragweed allergy vaccine (sublingual)

Greer LaboratoriesLenoir, NC

vaccine seasonal allergic rhinitis Phase IIIgreerlabs.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 77

respirAtory DisorDers

Product Name Sponsor ProductCategory

Indication Development Phase

reslizumab(Orphan Drug)

CephalonFrazer, PA

mAb asthma, eosinophilic esophagitis in adolescents and children

Phase IIIcephalon.com

RG3637(lebrikizumab)

GenentechSouth San Francisco, CARocheNutley, NJ

mAb severe asthma Phase IIIgene.comroche.com

SAR156597(bispecific interleukin-4/interleukin-13 mAb)

Sanofi USBridgewater, NJ

mAb idiopathic pulmonary fibrosis Phase IIsanofi.com

SAR231893 (anti-IL4 mAb)

Regeneron PharmaceuticalsTarrytown, NYSanofi USBridgewater, NJ

mAb asthma (see also skin)

Phase IIregeneron.comsanofi.com

STX-100 Biogen IdecWeston, MA

mAb idiopathic pulmonary fibrosis Phase IIbiogenidec.com

tralokinumab AstraZenecaWilmington, DEMedImmune Gaithersburg, MD

mAb asthma (see also digestive)

Phase IIastrazeneca.commedimmune.com

skin DiseAsesmedicines for skin cancer are listed under Cancer

Product Name Sponsor ProductCategory

Indication Development Phase

AbGn-168H AbGenomics InternationalLos Altos, CA

mAb plaque psoriasis Phase I completedabgenomics.com

Amevive®

alefaceptAstellas Pharma USDeerfield, IL

recombinant fusionprotein

psoriasis in adolescents Phase II completedus.astellas.com

AMG 827 (brodalumab)

Amgen Thousand Oaks, CA AstraZenecaWilmington, DE

mAb psoriasis (see also musculoskeletal, respiratory)

Phase IIamgen.comastrazeneca.com

ANT-1207(botulinum toxin A)

AnteriosNew York, NY

bacterial protein,nanotechnol-ogy

acne vulgaris Phase II

Biologic Medicines in Development

Medicines in Development Biologics 201378

skin DiseAsesmedicines for skin cancer are listed under Cancer

Product Name Sponsor ProductCategory

Indication Development Phase

BT-061 AbbVieNorth Chicago, ILBiotestDreieich, Germany

mAb plague psoriasis(see also autoimmune)

Phase IIabbvie.combiotest.de

CNTO-1959 Janssen BiotechHorsham, PA

mAb plaque psoriasis(see also autoimmune)

Phase IIjanssenbiotech.com

CureXcell™white blood cell therapy

MacrocureTikva, Israel

cell therapy diabetic foot ulcer Phase I/IImacrocure.com

Enbrel® etanercept

Amgen Thousand Oaks, CA

recombinant fusion protein

plague psoriasis in adolescents and children

application submittedamgen.com

fibroblast growth factor-1

CardioVascular Therapeutics Las Vegas, NV

fibroblast growth factor

wounds in diabetes(see also cardiovascular)

Phase IIcvbt.com

GBT 009 Garnet BioTherapeuticsMalvern, PA

celltherapy

scar prevention Phase IIgarnetbio.com

gevokizumab(IL-1B inhibitor mAb)

XOMABerkeley, CA

mAb acne vulgaris(see also diabetes, eye, musculoskeletal)

Phase IIxoma.com

HP802-247 (allogeneic human skin replacement)

Healthpoint Biotherapeutics Fort Worth, TX

celltherapy

leg ulcers

------------------------------------------------Mohs micrographic surgical wounds

Phase IIIhealthpointbio.com---------------------------------Phase IIhealthpointbio.com

Humira®

adalimumabAbbVieNorth Chicago, IL

mAb hidradenitis suppurativa(see also autoimmune, digestive, eye, musculoskeletal, other)

Phase IIIabbvie.com

ixekizumab(IL-17 antibody)

Eli Lilly Indianapolis, IN

mAb psoriasis(see also musculoskeletal)

Phase IIIlilly.com

JVS-100(stromal cell-derived factor-1)

SironRX TherapeuticsCleveland, OH

genetherapy

wound healing related to cardiovascular surgery

Phase Isironrx.com

KUR-211 (bioactive wound-healing foam)

Baxter InternationalDeerfield, IL Kuros BiosurgeryZurich, Switzerland

growthfactor

diabetic foot ulcers Phase IIbaxter.comkuros.ch

Biologic Medicines in Development

Medicines in Development Biologics 2013 79

skin DiseAsesmedicines for skin cancer are listed under Cancer

Product Name Sponsor ProductCategory

Indication Development Phase

laViv™azficel-T

Fibrocell ScienceExton, PA

fibroblastcelltherapy

scars Phase II/IIIfibrocellscience.com

MABp1 XBiotechAustin, TX

mAb acne, psoriasis(see also cancer, cardiovascular, diabetes)

Phase IIxbiotech.com

MK-3222(tildrakizumab)

Merck Whitehouse Station, NJ

mAb plaque psoriasis Phase IIImerck.com

PF-06473871 (EXC 001) PfizerNew York, NY

antisense dermal scarring Phase IIpfizer.com

QGE031 Novartis PharmaceuticalsEast Hanover, NJ

mAb atopic dermatitis(see also respiratory)

Phase IInovartis.com

REGN846 Regeneron Pharmaceuticals Tarrytown, NY

mAb atopic dermatitis Phase I/IIregeneron.com

RT001(botulinum toxin A)

Revance TherapeuticsNewark, CA

bacterial protein

lateral canthal lines(see also other)

Phase IIrevance.com

RXI-109 RXi PharmaceuticalsWestborough, MA

RNA interference

dermal scarring Phase Irxipharma.com

SAR231893(anti-IL4 mAb)

Regeneron PharmaceuticalsTarrytown, NYSanofi USBridgewater, NJ

mAb atopic dermatitis(see also respiratory)

Phase IIregeneron.comsanofi.com

SDF-1 SironRXCleveland, OH

gene therapy wound healing and reduction of scarring

Phase Isironrx.com

secukinumab(AIN457)

Novartis PharmaceuticalsEast Hanover, NJ

mAb plaque psoriasis(see also autoimmune, eye, musculoskeletal)

Phase IIInovartis.com

StrataGraft® tissue repair cell therapy(Orphan Drug)

Stratatech Madison, WI

celltherapy

chronic wounds Phase I/IIstratatechcorp.com

TD101 TransDermSanta Cruz, CA

RNAinterference

pachyonychia congenita Phase Itransderminc.com

Xolair® omalizumab

Genentech South San Francisco, CA Novartis PharmaceuticalsEast Hanover, NJ

mAb chronic idiopathic urticaria Phase IIIgene.comnovartis.com

Biologic Medicines in Development

Medicines in Development Biologics 201380

trAnsplAntAtion

Product Name Sponsor ProductCategory

Indication Development Phase

ASKP-1240 Astellas Pharma USDeerfield, ILKyowa Hakko Kirin PharmaPrinceton, NJ

mAb prevention of organ transplant rejection

Phase IIus.astellas.comkyowa-kirin-pharma.com

Benlysta®

belimumab(intravenous)

GlaxoSmithKlineRsch. Triangle Park, NC

mAb immunosuppression(see also autoimmune, blood)

Phase IIgsk.com

CaspaCIDe™ DLIT-cell replacementtherapy

Bellicum PharmaceuticalsHouston, TX

celltherapy

graft-versus-host disease in patients with late-stage cancer undergoing bone marrow transplantation

Phase I/IIbellicum.com

CDX-301(Fms-like tyrosinekinase 3 ligand)

Celldex TherapeuticsNeedham, MA

recombinant protein

hematopoietic stem cell transplantation

Phase Icelldextherapeutics.com

CLT-008 Cellerant TherapeuticsSan Carlos, CA

cell therapy facilitate engraftment in core blood transplants(see also cancer)

Phase Icellerant.com

CYT107(interleukin-7)

CytherisRockville, MD

recombinant interleukin

prevention of bone marrowtransplant rejection(see also blood, cancer, infectious)

Phase Icytheris.com

mesenchymal precursor cell (MPC) product(Orphan Drug)

MesoblastNew York, NY

stem celltherapy

for bone marrow regeneration in patients undergoing bonemarrow transplantation(see also cardiovascular, diabetes,musculoskeletal)

Phase IIImesoblast.com

MultiStem®

stem cell therapy(Orphan Drug)

AthersysCleveland, OH

stem celltherapy

graft-versus-host disease(see also cardiovascular, digestive)

Phase Iathersys.com

NX001(Orphan Drug)

NephrxKalamazoo, MI

growthfactor

delayed graft function in renal transplant patients

Phase Inephrx.com

Prochymal® remestemcel-L(Orphan Drug)

Osiris Therapeutics Columbia, MD

stem cell therapy

graft-versus-host disease (Fast Track)(see also cardiovascular, diabetes, digestive, respiratory)

Phase IIIosiris.com

QPI 1002(I5NP)(Orphan Drug)

Quark PharmaceuticalsFremont, CA

RNAinterference

prevention of delayed graftfunction(see also other)

Phase IIquarkpharma.com

Soliris®

eculizumabAlexion PharmaceuticalsCheshire, CT

mAb presensitized kidney transplant (acute humoral rejection)(see also autoimmune,eye, infectious)

Phase IIalxn.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 81

trAnsplAntAtion

Product Name Sponsor ProductCategory

Indication Development Phase

TOL101(Orphan Drug)

Tolera TherapeuticsKalamazoo, MI

mAb prevention of transplant rejection Phase I/IItolera.com

other

Product Name Sponsor ProductCategory

Indication Development Phase

AC607(allogeneic multi-potentstem cell therapy)(Orphan Drug)

AlloCureBurlington, MA

stem celltherapy

acute kidney injury(Fast Track)

Phase IIallocure.com

AGN-214868(senrebotase)

AllerganIrvine, CA

recombinantprotein

overactive bladder with urinary incontinence(see also neurologic)

Phase IIallergan.com

ALD-451 CytomedixGaithersburg, MD

stem cell therapy

cognition disorders following treatment for malignant glioma

Phase Icytomedix.com

Ampligen® rintatolimid(Orphan Drug)

Hemispherx Biopharma Philadelphia, PA

RNA product chronic fatigue syndrome application submittedhemispherx.net

antibody therapeutic MorphoSysMunich, GermanyNovartis PharmaceuticalsEast Hanover, NJ

mAb undisclosed Phase Imorphosys.comnovartis.com

antibody therapeutic MorphoSysMunich, GermanyNovartis PharmaceuticalsEast Hanover, NJ

mAb undisclosed Phase IImorphosys.comnovartis.com

antibody therapeutic(BI-1)

MorphoSysMunich, GermanyBoehringer-Ingelheim PharmaceuticalsRidgefield, CT

mAb undisclosed Phase Imorphosys.comboehringer-ingelheim.com

anti-IL31 Bristol-Myers SquibbPrinceton, NJ

mAb immunology Phase Ibms.com

Biologic Medicines in Development

Medicines in Development Biologics 201382

other

Product Name Sponsor ProductCategory

Indication Development Phase

anti-TWEAK (BIIB 023)

Biogen Idec Weston, MA

mAb lupus nephritis Phase IIbiogenidec.com

BAX-69 Baxter InternationalDeerfield, IL

mAb lupus nephritis(see also cancer)

Phase Ibaxter.com

Botox® onabotulinumtoxinA

AllerganIrvine, CA

bacterial protein

idiopathic overactivebladder(see also neurologic)------------------------------------------------benign prostatic hyperplasia

application submittedallergan.com

---------------------------------Phase II allergan.com

cat allergy vaccine CircassiaOxford, United Kingdom

vaccine hypersensitivity in patients withrhinoconjunctivitis caused by cat allergy

Phase IIIcircassia.co.uk

CDX-1135 Celldex TherapeuticsNeedham, MA

recombinantprotein

kidney disorders Phase Icelldextherapeutics.com

CM-AT CureMarkRye, NY

enzymereplacementtherapy

autistic disorder(Fast Track)

Phase IIIcuremark.com

CNTO-5 Janssen BiotechHorsham, PAMorphoSysMunich, Germany

mAb inflammation Phase Ijanssenbiotech.commorphosys.com

CNTO-136(sirukumab)

Janssen BiotechHorsham, PA

mAb lupus nephritis(see also autoimmune)

Phase IIjanssenbiotech.com

ELAD®

human-cell-based bioartificial liver(Orphan Drug)

Vital TherapiesSan Diego, CA

cell therapy

liver failure Phase II/IIIvitaltherapies.com

entolimod(CBLB502)(Orphan Drug)

Cleveland BioLabsBuffalo, NY

engineered peptide

acute radiation syndrome(Fast Track)(see also cancer)

Phase Icbiolabs.com

FB 301 Cytovance BiologicsOklahoma City, OKFountain BiopharmaTaipei, Taiwan

mAb hypersensitivity(IgE-mediated allergic diseases)

in clinical trialscytovance.com

FG-3019 FibroGenSan Francisco, CA

mAb liver fibrosis due to chronic hepatitis B infection(see also cancer, respiratory)

Phase IIfibrogen.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 83

other

Product Name Sponsor ProductCategory

Indication Development Phase

food allergyimmunotherapy

DBV TechnologiesParis, France

topical vaccine

peanut hypersensitivity Phase IIdbv-technologies.com

fresolimumab(TGFß antagonist)

GenzymeCambridge, MA

mAb fibrosis Phase IIgenzyme.com

GS-6624 (simtuzumab)

Gilead Sciences Foster City, CA

mAb liver fibrosis, myelofibrosis(see also cancer, respiratory)

Phase IIgilead.com

GSK1070806(anti-interleukin 18 mAb)

GlaxoSmithKlineRsch. Triangle Park, NC

mAb metabolic disorders Phase IIgsk.com

hMaxi-K gene therapy

Ion Channel Innovations New York, NY

gene therapy overactive bladder Phase I completedionchannelinnovations.com

Humira® adalimumab

AbbVieNorth Chicago, IL

mAb interstitial cystitis (see also autoimmune, digestive, eye, musculoskeletal, skin)

Phase IIIabbvie.com

ISIS-FGFR4Rx Isis PharmaceuticalsCarlsbad, CA

antisense obesity Phase IIisispharm.com

ISIS-GCCRRx Isis PharmaceuticalsCarlsbad, CA

antisense Cushing syndrome(see also diabetes)

Phase Iisispharm.com

JRC-LAMP-vax Immunomic TherapeuticsLancaster, PA

DNA vaccine Japanese red cedar pollen-inducedrhinoconjunctivitis

Phase Iimmunomix.com

kidney disease medicine Eli LillyIndianapolis, IN

biologic chronic kidney disease Phase I lilly.com

liprotamase(LY3031642)(Orphan Drug)

Eli LillyIndianapolis, IN

enzyme replacement therapy

treatment of exocrine pancreatic enzyme insufficiency, including in cystic fibrosis patients(Fast Track)

application submittedlilly.com

liraglutide (NN8022)

Novo Nordisk Princeton, NJ

recombinantpeptide

obesity Phase IIInovonordisk.com

liver cell therapy(Orphan Drug)

CytonetWeinheim, Germany

celltherapy

urea cycle disorders in children and infants

Phase IIcytonet.de

LY2382770 (TGF-ß antibody)

Eli LillyIndianapolis, IN

mAb diabetic nephropathy Phase IIlilly.com

Biologic Medicines in Development

Medicines in Development Biologics 201384

other

Product Name Sponsor ProductCategory

Indication Development Phase

mAb GenentechSouth San Francisco, CA

mAb metabolic disorders Phase Igene.com

Meth-mAb InterveXion TherapeuticsLittle Rock, AR

mAb methamfetamine abuse Phase Iintervexion.com

metreleptin Bristol-Myers SquibbPrinceton, NJ

recombinant hormone

lipodystrophy application submittedbms.com

Natpara® recombinant human parathyroid hormone (rhPTH)(Orphan Drug)

NPS Pharmaceuticals Salt Lake City, UT

recombinant hormone

hypoparathyroidism Phase IIInpsp.com

Neo-Urinary Conduit™ autologous culturedurological tissue cells

Tengion Winston-Salem, NC

cell therapy

cystectomy due to bladder cancer Phase Itengion.com

NIC002 Novartis Pharmaceuticals East Hanover, NJ

vaccine smoking withdrawal Phase IInovartis.com

Orencia® abatacept

Bristol-Myers SquibbPrinceton, NJ

recombinant fusionprotein

lupus nephritis(see also autoimmune, musculoskeletal)

Phase IIIbms.com

PF-05402536 PfizerNew York, NY

vaccine smoking cessation Phase Ipfizer.com

PRM-151(rhPTX-2 for injection)

PromediorLexington, MA

recombinantprotein

myelofibrosis(see also respiratory)

Phase Ipromedior.com

prohibitin targeting peptide-1

Ablaris TherapeuticsPasadena, CA

homing peptide

obesity Phase Iablaris.com

PRT-105 Protalix BiotherapeuticsCarmiel, Israel

recombinant enzyme

poisoning Phase I completedprotalix.com

QPI-1002(I5Np)

Quark PharmaceuticalsFremont, CA

RNAinterference

prevention of renal failure in patients undergoing cardiovascular surgery(see also transplantation)

Phase I/IIquarkpharma.com

recombinant humanhepatocyte growth factor

Kringle PharmaOsaka, Japan

recombinantgrowth factor

acute renal failure Phase Ikringle-pharma.com

REGN728 Regeneron PharmaceuticalsTarrytown, NY

mAb undisclosed Phase Iregeneron.com

Biologic Medicines in Development

Medicines in Development Biologics 2013 85

other

Product Name Sponsor ProductCategory

Indication Development Phase

RT001(botulinum toxin A)

Revance TherapeuticsNewark, CA

bacterial protein

hyperhidrosis(see also skin)

Phase IIrevance.com

SEL-068 Selecta BiosciencesWatertown, MA

nanoparticlevaccine

smoking withdrawal Phase Iselectabio.com

somatropin biosimilar(sustained-release)

LG Life Sciences Seoul, South Korea

recombinant human growth hormone

somatropin deficiency Phase IIIlgls.com

Stelara®

ustekinumabJanssen BiotechHorsham, PA

mAb primary biliary cirrhosis(see also autoimmune, digestive,musculoskeletal)

Phase IIjanssenbiotech.com

TA-CD Celtic Pharma Hamilton, Bermuda

immuno- therapeutic vaccine

cocaine abuse Phase IIceltictherapeutic.com

UMK-121 ProteonomixHawthorne, NJ

stem cell therapy

liver failure Phase Iproteonomix.com

VAY736 MorphoSysMunich, GermanyNovartis PharmaceuticalsEast Hanover, NJ

mAb inflammation(see also cancer)

Phase Imorphosys.comnovartis.com

VEN100(recombinant human lactoferrin)

Ventria BioscienceFort Collins, CO

recombinant protein

antibiotic-associated diarrhea Phase IIventria.com

VRS-317(human growth hormone)

VersartisRedwood City, CA

recombinanthuman growthhormone

somatropin deficiency Phase I/IIversartis.com

Xiaflex®

collagenase clostridiumhistolyticum(Orphan Drug)

Auxilium PharmaceuticalsChesterbrook, PABioSpecifics TechnologiesLynbrook, NY

peptide Peyronie’s disease(see also cancer, musculoskeletal)

------------------------------------------------cellulite

application submittedauxilium.combiospecifics.com---------------------------------Phase Iauxilium.combiospecifics.com

Biologic Medicines in Development

Medicines in Development Biologics 201386

adjuvant—Substance or drug that aids an-other substance in its action.

angioedema—Swelling of the mucous mem-branes, tissues beneath the skin, or an internal organ due to an allergic reaction.

ankylosing spondylitis—An inflammatory disorder of unknown cause that primarily affects the spine. The vertebrae may fuse together and form a rigid back that is impos-sible to bend.

antisense—An antisense drug is the mirror or complementary image of a small segment of messenger RNA (mRNA), the substance that carries instructions (“sense”) from the genes to the cell’s protein-making machinery. The anti-sense drug readily binds to the mRNA strand, keeping it from transmitting its instructions to the cell, and thus inhibiting the production of an unwanted protein.

application submitted—An application for marketing has been submitted by the company to the Food and Drug Administration (FDA).

biotechnology—The collection of processes that involve the use of biological systems. For some of the industries, these processes involve the use of genetically engineered organisms. For the purpose of this report, only those products that involve recombinant DNA, monoclonal antibody/hybridoma, continuous cell lines, cellular therapy, gene therapy and vaccines technology are included.

Clostridium difficile—A bacterium that produces an irritating toxin that causes a form of colitis characterized by profuse, watery diar-rhea with cramps and low-grade fever.

colony stimulating factor (CSF)—Protein responsible for controlling the production of white blood cells.

Crohn’s disease—A subacute chronic gastro-intestinal disorder, involving the small intestine, characterized by patchy deep ulcers that may cause fistulas and a narrowing and thickening of the bowel.

cytomegalovirus (CMV)—A DNA virus related to the herpes virus, affecting mostly neonatal infants and immunocompromised individuals. CMV can occur without symptoms or result in mild flu-like symptoms.

erythropoietin—Hormone that stimulates red blood cell production in the bone marrow.

Fast Track—A process designed to facilitate the development and expedite the review of drugs to treat serious diseases and fill an unmet medical need. The status is assigned by the U.S. Food and Drug Administration. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious diseases. Generally, determining factors include whether the drug will have an impact on such factors 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. Filling an unmet medical need is defined as pro-viding a therapy where none exists or providing a therapy which may be potentially superior to existing therapy. Once a drug receives Fast Track designation, early and frequent commu-nication between the FDA and a drug company is encouraged throughout the entire drug de-velopment and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.

gene therapy—Therapy at the intracellular level to replace or inactivate the effects of disease-causing genes or to augment normal gene functions to overcome illness.

glioblastoma—The most common primary brain tumor and one of the most aggressive forms of brain cancer, primarily affecting adults over the age of 50.

growth factors—Factors responsible for regulating cell proliferation (rapid and repeated reproduction), function and differentiation.

growth hormone—Pituitary hormone that stimulates the growth of long bones in prepu-bertal children.

hemophilia A and B—Hemophilia A, the “clas-sic” hemophilia, is a genetic bleeding disorder due to deficiency of the coagulation factor VIII. Hemophilia B, or “Christmas” disease, is caused by deficiency of coagulation factor IX.

hepatitis—Inflammation of the liver with accom-panying liver cell damage or death, caused most often by viral infection, e.g., hepatitis B, and C.

herpes simplex virus—A strain of herpes virus that may lie dormant in nerve tissue and

can be reactivated to produce painful sores of the anus or genitals.

human papillomavirus—Viral agent of warts, believed to be contagious and usually harm-less, but it can lead to cervical cancer.

interferon—A glycoprotein naturally produced by cells that interferes with the ability of a virus to reproduce after it invades the body.

interleukin—An endogenous substance that stimulates the production of different types of white blood cells or leukocytes.

ischemia—Insufficient supply of blood to an organ or tissue, which can cause organ dam-age such as an ischemic stroke.

lymphoma—Cancers in which the cells of lym-phoid tissue, found mainly in the lymph nodes and spleen, multiply unchecked. Lymphomas fall into two categories: One is called Hodgkin disease, characterized by a particular kind of abnormal cell. All others are called non-Hodgkin lymphomas, which vary in their malignancy according to the nature and activity of the abnormal cells.

mAb (monoclonal antibodies)—Large protein molecules produced by white blood cells that seek out and destroy harmful foreign sub-stances.

macular degeneration—A progressive disor-der that affects the central part of the retina, causing gradual loss of vision. It is a painless condition, usually affects both eyes and is com-mon in the elderly.

metastases/metastatic—Cancers that have spread from the primary or original cancer site. Cancer cells can break away from a tumor and travel to other areas of the body through the bloodstream or the lymph system. They are of-ten more difficult to treat then primary tumors.

multiple myeloma—A malignant condition char-acterized by uncontrolled proliferation of plasma cells (a class of white blood cells) in bone marrow. Symptoms include pain and destruction of bone tissue, numbness and paralysis, kidney damage, anemia, and frequent infections.

myelodysplastic syndromes (MDS)—Blood disorders that ultimately are fatal. Patients usually succumb to infections or bleeding. The term “preleukemia” has been used to describe these disorders.

Glossary

Medicines in Development Biologics 2013 87

myeloid—Pertaining to, derived from, or mani-festing certain features of the bone marrow. In some cases, myeloid also pertains to certain types of non-lymphocyte white blood cells found in the bone marrow.

myocardial infarction—Damage to the heart muscle caused by stoppage or impair-ment of blood flow to the heart, also known as heart attack.

neuroblastoma—A tumor of the adrenal glands or sympathetic nervous system (the part of the nervous system responsible for certain automatic body functions, such as the “fight or flight response”).

neutropenia—An abnormally low neutrophil count (certain white blood cells).

Orphan Drug—A drug to treat a disease that has a patient population of 200,000 or less in the United States, or a disease that has a patient population of more than 200,000 and a development cost that will not be recovered from sales in the United States.

peripheral vascular disorder—The obstruc-tion of blood supply to the extremities, particu-larly the legs, caused by atherosclerosis.

Phase 0—First-in-human trials conducted in accordance with FDA’s 2006 guidance on exploratory Investigational New Drug (IND) studies designed to speed up development of promising drugs by establishing very early on whether the agent behaves in human subjects as was anticipated from preclinical studies.

Phase I—Researchers test the drug in a small group of people, usually between 20 and 80 healthy adult volunteers, to evaluate its initial safety and tolerability profile, determine a safe dosage range, and identify potential side effects.

Phase II—The drug is given to volunteer patients, usually between 100 and 300, to see if it is effective, identify an optimal dose, and to further evaluate its short-term safety.

Phase III—The drug is given to a larger, more diverse patient population, often involving be-tween 1,000 and 3,000 patients (but sometime many more thousands), to generate statistically significant evidence to confirm its safety and effectiveness. They are the longest studies, and usually take place in multiple sites around the world.

pulmonary fibrosis—Scarring and thickening of lung tissue.

respiratory syncytial virus (RSV) disease—One of the most important causes of lower respiratory tract disease in children, account-ing for more than 90 percent of cases of bronchiolitis.

restenosis—A condition where an artery plugs up again following treatment to open it up.

sepsis—A condition associated with a serious bacterial infection of the blood.

systemic lupus erythematosus (SLE)—The most serious form of lupus, a chronic autoim-mune disorder causing inflammation and damage to multiple organs.

thrombocytopenia—A reduction in the number of platelet cells in the blood, which causes a tendency to bleed, especially from the smaller blood vessels.

ulcerative colitis—A chronic inflammation and ulceration of the lining of the colon and rectum. It causes bloody diarrhea and mainly involves the left colon.

uveitis—Inflammation of the uvea, the middle layer of the eye.

Glossary

The content of this report has been obtained through public, government and industry sources, and the Adis “R&D Insight” database based on the latest information. Report current as of February 7, 2013. The information in this report 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 web site.A publication of PhRMA’s Communications & Public Affairs Department. (202) 835-3460www.phrma.org | www.innovation.org | www.pparx.org | www.buysafedrugs.infoProvided as a Public Service by PhRMA. Founded in 1958 as the Pharmaceutical Manufacturers Association.Copyright © 2013 by the Pharmaceutical Research and Manufacturers of America. Permission to reprint is awarded if proper credit is given.

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