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 information 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.

1. 2013 REPORT
Medicines in Development
Biologics
presented by america’s biopharmaceutical research companies
Biologics Research Pushing Frontiers of
Science With More Than 900 Medicines and
Vaccines in Development
In the human body
there are
Biologics In Development 12 trillion cells,
By Product Category and 200,000 proteins and
Development Phase 25,000 genes
338
Application
Submitted
Phase III
Phase II
America’s biopharmaceutical research • treatments for cardiovascular
58
Phase I companies are developing 907 biologics diseases, such as congestive heart
250 targeting more than 100 diseases. Biologics failure and stroke.
are developed through biological processes •
Other diseases include diabetes,
using living cells or organisms. digestive disorders, genetic disorders,
This report lists biologics in human neurologic and respiratory disorders.
clinical trials or under review by the Definitions for selected product categories
U.S. Food and Drug Administration and diseases can be found on page 86.
(FDA). A link to the sponsor company’s
web site provides more detailed infor- For information on the history and
mation on the science and technologies future of biologics research, the science
behind each potential product. The 907 behind the medicines, and a discussion
medicines and vaccines in development of issues critical to continued discovery
include: and development of these cutting-edge
medicines, please see Biologic Medicines
• cancer therapeutics that target
338 in Development 2013—Overview.
93
several different types of solid tumors,
81
leukemia and lymphoma. Monoclonal The medicines in this report reflect the
69
antibodies account for 170 of the 338 new ways America’s biopharmaceuti-
products in development. cal research companies are attacking
46 disease through biotechnology. The 907
• candidates in development for an
176 medicines and vaccines in development
30 array of infectious diseases, including promise to push the frontiers of science
134 vaccines. and bring new treatments to patients for
• medicines for autoimmune diseases,
71 our more challenging diseases.
such as lupus, multiple sclerosis and
e
py
ins
s
er
y
s
ine
ns
ap
die
rheumatoid arthritis.
oth
ra
ote
e
er
cc
the
bo
tis
h
pr
va
nti
an
ll t
ne
nt
la
ce
ina
ge
na
mb
clo
co
no
re
mo

2. Biologic Medicines in Development
Autoimmune Disorders
Product Name Sponsor Product Indication Development Phase*
Category
ABT-122 AbbVie mAb rheumatoid arthritis Phase I
North Chicago, IL abbvie.com
Actemra® Genentech mAb early rheumatoid arthritis Phase III
tocilizumab South San Francisco, CA gene.com
Roche ------------------------------------------------ --------------------------------
Nutley, NJ systemic sclerosis Phase II
gene.com
AGS-009 Argos Therapeutics mAb systemic lupus erythematosus Phase I
Durham, NC (SLE) argostherapeutics.com
alemtuzumab Genzyme mAb relapsing-remitting multiple application submitted
Cambridge, MA sclerosis (Fast Track) genzyme.com
Alferon N Injection® Hemispherx Bioscience interferon multiple sclerosis Phase II
interferon-alpha-n3 Philadelphia, PA hemispherx.net
subcutaneous
AMG 557/MEDI-5872 Amgen mAb SLE Phase I
Thousand Oaks, CA amgen.com
AstraZeneca astrazeneca.com
Wilmington, DE medimmune.com
MedImmune
Gaithersburg, MD
AMG 729 Amgen mAb autoimmune diseases Phase I
Thousand Oaks, CA amgen.com
AMG 811 Amgen mAb discoid lupus erythematosus, Phase I
Thousand Oaks, CA SLE amgen.com
anti-IL17 mAb Genentech mAb autoimmune disorders Phase I
(RG7624) South San Francisco, CA gene.com
anti-LINGO Biogen Idec mAb multiple sclerosis Phase II
(BIIB033) Weston, MA (see also eye) biogenidec.com
ARX424 Ambrx interferon multiple sclerosis Phase I
(PEGylated interferon La Jolla, CA ambrx.com
beta-1a)
atacicept EMD Serono recombinant SLE Phase II/III
(TACI-lg) Rockland, MA fusion emdserono.com
protein
*For more information about a specific medicine or company in the report, please click on the provided link.
2 Medicines in Development Biologics 2013

3. Biologic Medicines in Development
Autoimmune Disorders
Product Name Sponsor Product Indication Development Phase
Category
ATX-MS-1467 EMD Serono peptide multiple sclerosis Phase I
Rockland, MA vaccine emdserono.com
AZ01 Allozyne interferon multiple sclerosis Phase I
(long-acting pegylated Seattle, WA allozyne.com
interferon beta)
Benlysta® GlaxoSmithKline mAb SLE Phase III
belimumab Rsch. Triangle Park, NC (see also blood, transplantation) gsk.com
(subcutaneous) ------------------------------------------------ --------------------------------
systemic scleroderma gsk.com
BHT-3009 Bayhill Therapeutics DNA multiple sclerosis Phase II
Palo Alto, CA vaccine bayhilltx.com
BI-695500 Boehringer-Ingelheim mAb rheumatoid arthritis Phase III
(rituximab biosimilar) Pharmaceuticals boehringer-ingelheim.
Ridgefield, CT com
BI-695501 Boehringer Ingelheim mAb rheumatoid arthritis Phase I
(adalimumab biosimilar) Pharmaceuticals boehringer-ingelheim.
Ridgefield, CT com
BIIB017 Biogen Idec interferon relapsing multiple sclerosis Phase III
(PEG-interferon Weston, MA (Fast Track) biogenidec.com
beta-1a)
BT-061 AbbVie mAb rheumatoid arthritis Phase II
North Chicago, IL (see also skin) abbvie.com
Biotest biotest.de
Dreieich, Germany
Cimzia® UCB mAb ankylosing spondylitis, Phase III
certolizumab pegol Smyrna, GA juvenile rheumatoid arthritis ucb.com
(see also musculoskeletal)
clazakizumab Bristol-Myers Squibb mAb rheumatoid arthritis Phase II
(anti-IL6) Princeton, NJ (see also digestive, bms.com
Alder Biopharmaceuticals musculoskeletal) alderbio.com
Bothell, WA
CNTO-136 Janssen Biotech mAb rheumatoid arthritis Phase III
(sirukumab) Horsham, PA (see also other) janssenbiotech.com
CNTO-1959 Janssen Biotech mAb rheumatoid arthritis Phase II
Horsham, PA (see also skin) janssenbiotech.com
Medicines in Development Biologics 2013 3

4. Biologic Medicines in Development
Autoimmune Disorders
Product Name Sponsor Product Indication Development Phase
Category
daclizumab AbbVie mAb multiple sclerosis (relapsing forms) Phase III
North Chicago, IL abbvie.com
Biogen Idec biogenidec.com
Weston, MA
epratuzumab Immunomedics mAb SLE Phase III
Morris Plains, NJ (Fast Track) immunomedics.com
UCB ucb.com
Smyrna, GA
GSK1223249 GlaxoSmithKline mAb multiple sclerosis Phase I
(NOGO-A mAb) Rsch. Triangle Park, NC (see also neurologic) gsk.com
Humira® AbbVie mAb rheumatoid arthritis Phase III
adalimumab North Chicago, IL (combination therapy) abbvie.com
(see also digestive, eye,
musculoskeletal, skin, other)
Ilaris® Novartis Pharmaceuticals mAb systemic juvenile idiopathic arthritis Phase III
canakinumab East Hanover, NJ (see also cardiovascular, diabetes, novartis.com
(Orphan Drug) musculoskeletal)
ISIS-CRPRx Isis Pharmaceuticals antisense rheumatoid arthritis Phase II
Carlsbad, CA (see also cardiovascular) isispharm.com
mavrilimumab AstraZeneca mAb rheumatoid arthritis Phase II
Wilmington, DE astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD
MEDI-546 AstraZeneca mAb SLE Phase II
(anti-IFN-alphaR mAb) Wilmington, DE astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD
MEDI-551 AstraZeneca mAb scleroderma Phase I
(anti-CD19 mAb) Wilmington, DE (see also cancer) astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD
MEDI-570 AstraZeneca mAb SLE Phase I
(anti-ICOS mAb) Wilmington, DE astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD
4 Medicines in Development Biologics 2013

5. Biologic Medicines in Development
Autoimmune Disorders
Product Name Sponsor Product Indication Development Phase
Category
NeuroVax™ Immune Response Biopharma peptide multiple sclerosis Phase II
(IR208) New York, NY vaccine immuneresponsebio
pharma.com
NN8209 Novo Nordisk mAb rheumatoid arthritis Phase II
(anti-C5aR-151 mAb) Princeton, NJ novonordisk.com
NN8210 Novo Nordisk mAb rheumatoid arthritis Phase I
(anti-C5aR-215 mAb) Princeton, NJ novonordisk.com
NN8226 Novo Nordisk mAb rheumatoid arthritis Phase II
(anti-IL-20 mAb) Princeton, NJ novonordisk.com
NN8765 Novo Nordisk mAb rheumatoid arthritis Phase I
(anti-NKG2 mAb) Princeton, NJ novonordisk.com
NN8828 Novo Nordisk mAb rheumatoid arthritis Phase II
(anti-IL-21 mAb) Princeton, NJ novonordisk.com
-------------------------------- --------------------------------
SLE Phase I
novonordisk.com
Nplate™ Amgen recombinant immune thrombocytopenic Phase III
romiplostim Thousand Oaks, CA fusion purpura in children amgen.com
(Orphan Drug) protein (see also cancer)
NU-100 Nuron Biotech recombinant multiple sclerosis Phase III
(interferon beta-1b) Exton, PA interferon nuronbiotech.com
ocrelizumab Biogen Idec mAb primary-progressive multiple Phase III
(anti-CD20 mAb) Weston, MA sclerosis biogenidec.com
Genentech gene.com
South San Francisco, CA ------------------------------------------------ ---------------------------------
Roche relapsing-remitting multiple Phase III
Nutley, NJ sclerosis biogenidec.com
gene.com
ofatumumab GlaxoSmithKline mAb multiple sclerosis, Phase II
(subcutaneous) Rsch. Triangle Park, NC rheumatoid arthritis gsk.com
olokizumab UCB mAb rheumatoid arthritis Phase II
Smyrna, GA ucb.com
Orencia® Bristol-Myers Squibb recombinant early rheumatoid arthritis Phase III
abatacept Princeton, NJ fusion protein (see also musculoskeletal, other) bms.com
Medicines in Development Biologics 2013 5

6. Biologic Medicines in Development
Autoimmune Disorders
Product Name Sponsor Product Indication Development Phase
Category
otelixizumab GlaxoSmithKline mAb rheumatoid arthritis Phase I
(anti-CD3 mAb) Rsch. Triangle Park, NC gsk.com
ozoralizumab Ablynx mAb rheumatoid arthritis Phase II completed
(ATN-103) Ghent, Belgium ablynx.com
pateclizumab Genentech mAb rheumatoid arthritis Phase II
(anti-LT alpha mAb) South San Francisco, CA gene.com
PD-360324 Pfizer mAb cutaneous lupus erythematosus Phase I
New York, NY pfizer.com
PDA-001 Celgene Cellular Therapeutics stem cell rheumatoid arthritis Phase II
(cenplacel-L) Summit, NJ therapy (see also digestive) celgene.com
------------------------------------------------ ---------------------------------
multiple sclerosis, sarcoidosis Phase I
celgene.com
PF-04236921 Pfizer mAb SLE Phase II
New York, NY (see also digestive) pfizer.com
------------------------------------------------ ---------------------------------
rheumatoid arthritis Phase I
pfizer.com
PF-05280586 Pfizer mAb rheumatoid arthritis Phase I/II
(rituximab biosimilar) New York, NY pfizer.com
plovamer acetate EMD Serono peptide multiple sclerosis Phase I
(second-generation Rockland, MA emdserono.com
peptide copolymer)
Prolia® Amgen mAb rheumatoid arthritis Phase II
denosumab Thousand Oaks, CA (see also musculoskeletal) amgen.com
RAVAX™ Immune Response BioPharma vaccine rheumatoid arthritis Phase III
rheumatoid arthritis New York, NY immuneresponsebio
vaccine pharma.com
rontalizumab Genentech mAb SLE Phase II
(RG7415) South San Francisco, CA gene.com
SAN-300 Santarus mAb rheumatoid arthritis Phase I
(anti-VLA-1 antibody) San Diego, CA santarus.com
6 Medicines in Development Biologics 2013

7. Biologic Medicines in Development
Autoimmune Disorders
Product Name Sponsor Product Indication Development Phase
Category
SAR113244 Sanofi US mAb SLE Phase I
(anti-CXCR5 mAb) Bridgewater, NJ sanofi.com
sarilumab Regeneron Pharmaceuticals mAb rheumatoid arthritis Phase III
(SAR153191) Tarrytown, NY regeneron.com
Sanofi US sanofi.com
Bridgewater, NJ
secukinumab Novartis Pharmaceuticals mAb ankylosing spondylitis, Phase III
(AIN457) East Hanover, NJ rheumatoid arthritis novartis.com
(see also eye, musculoskeletal, skin)
------------------------------------------------ ---------------------------------
multiple sclerosis Phase II
novartis.com
sifalimumab AstraZeneca mAb SLE Phase II
(anti-IFN-alpha mAb) Wilmington, DE astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD
Simponi® Janssen Biotech mAb rheumatoid arthritis application submitted
golimumab Horsham, PA (intravenous) janssenbiotech.com
(Orphan Drug) (see also digestive)
------------------------------------------------ ---------------------------------
juvenile rheumatoid arthritis Phase III
(subcutaneous) janssenbiotech.com
----------------------------------------------- ---------------------------------
sarcoidosis Phase II completed
(subcutaneous) janssenbiotech.com
Soliris® Alexion Pharmaceuticals mAb severe or refractory myasthenia Phase II
eculizumab Cheshire, CT gravis alxn.com
(Orphan Drug) (see also eye, infectious,
transplantation)
Stelara® Janssen Biotech mAb rheumatoid arthritis, sarcoidosis Phase II
ustekinumab Horsham, PA (see also digestive, janssenbiotech.com
musculoskeletal, other)
tabalumab Eli Lilly mAb SLE Phase III
(BAFF inhibitor) Indianapolis, IN (see also cancer) lilly.com
Tcelna™ Opexa Therapeutics cell therapy multiple sclerosis Phase II
imilecleucel-T The Woodlands, TX vaccine (Fast Track) opexatherapeutics.com
Tysarbi® Biogen Idec mAb secondary-progressive multiple Phase III
natalizumab Weston, MA sclerosis biogenidec.com
Medicines in Development Biologics 2013 7

8. Biologic Medicines in Development
Autoimmune Disorders
Product Name Sponsor Product Indication Development Phase
Category
veltuzumab Immunomedics mAb immune thrombocytopenic Phase II
(IMMU-106) Morris Plains, NJ purpura, rheumatoid arthritis immunomedics.com
Takeda Pharmaceuticals USA (see also cancer) takeda.com
Deerfield, IL
VX15 Teva Pharmaceuticals mAb multiple sclerosis Phase I
Tikva, Israel (see also cancer) tevapharm.com
Vaccinex vaccinex.com
Rochester, NY
XmAb®5871 Xencor mAb autoimmune disorders Phase I
(anti-CD19 mAb) Monrovia, CA xencor.com
Blood Disorders
Product Name Sponsor Product Indication Development Phase
Category
ACE-536 Acceleron Pharma recombinant anemia Phase I
Cambridge, MA fusion acceleronpharma.com
Celgene protein celgene.com
Summit, NJ
ALXN1102/ALXN1103 Alexion Pharmaceuticals recombinant paroxysmal nocturnal Phase I
Cheshire, CT fusion hemoglobinuria alxn.com
protein
AMT-060 uniQure gene therapy hemophilia B Phase I/II
(Orphan Drug) Amsterdam, The Netherlands uniqure.com
St. Jude Children’s Research
Hospital
Memphis, TN
ART123 Asahi Kasei Pharma America recombinant disseminated intravascular Phase III
(recombinant human Waltham, MA protein coagulation akpamerica.com
thrombomodulin alpha)
AskBio009 Asklepios BioPharmaceutical gene therapy hemophilia B Phase I/II
Chapel Hill, NC askbio.com
Baxter International baxter.com
Deerfield, IL
ATryn® GTC Biotherapeutics recombinant heparin resistance in patients under- Phase III
antithrombin alfa Framingham, MA protein going CABG surgery gtc-bio.com
(Orphan Drug)
8 Medicines in Development Biologics 2013

9. Biologic Medicines in Development
Blood Disorders
Product Name Sponsor Product Indication Development Phase
Category
BAX-111 Baxter International recombinant von Willebrand disease Phase III
(recombinant Deerfield, IL protein baxter.com
von Willebrand
factor)
(Orphan Drug)
BAX-326 Baxter International recombinant hemophilia B application submitted
(rFIX) Deerfield, IL protein baxter.com
(Orphan Drug)
BAX-817 Baxter International recombinant hemophilia A, hemophilia B Phase I/II
(rFVIIa) Deerfield, IL protein baxter.com
BAX-855 Baxter International recombinant hemophilia A Phase I
(rFVIII) Deerfield, IL protein baxter.com
BAY 86-6150 Bayer HealthCare recombinant hemophilia A, hemophilia B Phase II/III
(rFVIIa) Pharmaceuticals protein bayerpharma.com
Wayne, NJ
BAY 94-9027 Bayer HealthCare recombinant hemophilia A Phase II/III
(rFVIII) Pharmaceuticals protein bayerpharma.com
Wayne, NJ
Benlysta® GlaxoSmithKline mAb vasculitis Phase I completed
belimumab Rsch. Triangle Park, NC (see also autoimmune, gsk.com
(oral) transplantation)
beta-globin bluebird bio gene beta-thalassemia, Phase I/II
gene therapy Cambridge, MA therapy sickle cell anemia bluebirdbio.com
(lentiglobin) INSERM
Paris, France
BI-655075 Boehringer Ingelheim mAb blood coagulation disorders Phase I
Pharmaceuticals boehringer-ingelheim.
Ridgefield, CT com
CSL-627 CSL Behring recombinant hemophilia A Phase I
(rVIII-SC) King of Prussia, PA protein cslbehring.com
CSL-654 CSL Behring recombinant hemophilia B Phase II/III
(rIX-FP) King of Prussia, PA fusion cslbehring.com
(Orphan Drug) protein
CSL-689 CSL Behring recombinant hemophilia A, hemophilia B Phase I
(rVIIa-FP) King of Prussia, PA fusion cslbehring.com
(Orphan Drug) protein
Medicines in Development Biologics 2013 9

10. Biologic Medicines in Development
Blood Disorders
Product Name Sponsor Product Indication Development Phase
Category
CYT107 Cytheris recombinant idiopathic CD4-positive Phase I/II
(interleukin-7) Rockville, MD interleukin lymphocytopenia cytheris.com
(see also cancer, infectious,
transplantation)
epoetin alfa biosimilar Hospira recombinant anemia Phase III
Lake Forest, IL erythropoietin hospira.com
erythropoietin gene Medgenics gene therapy anemia Phase II
therapy San Francisco, CA medgenics.com
factor VIII/ CSL Behring recombinant hemophilia A, Phase III
von Willebrand King of Prussia, PA protein von Willebrand disease cslbehring.com
factor
ferroportin mAb Eli Lilly mAb anemia Phase I
Indianapolis, IN lilly.com
hepcidin mAb Eli Lilly mAb anemia Phase I
Indianapolis, IN lilly.com
HM10760A Hanmi Pharmaceutical long-acting anemia Phase I
Seoul, South Korea erythropoietin hanmipharm.com
human Octapharma USA recombinant hemophilia A Phase II
recombinant factor VIII Hoboken, NJ protein octapharma.com
IB1001 Inspiration Biopharmaceuticals recombinant hemophilia B application submitted
(recombinant factor IX Cambridge, MA protein inspirationbio.com
biosimilar) Ipsen
Paris, France
ISIS-FXIRx Isis Pharmaceuticals antisense clotting disorders Phase II
Carlsbad, CA isispharm.com
NAV™ therapeutic ReGenX Biosciences gene therapy hemophilia B Phase I/II
Washington, DC (see also genetic) regenxbio.com
NiCord® Gamida Cell stem cell sickle cell anemia Phase I/II
umbilical cord blood Jerusalem, Israel therapy (see also cancer) gamida-cell.com
stem cell therapy
NN1841 Novo Nordisk recombinant congenital factor XIII deficiency application submitted
(rFXIII) Princeton, NJ protein novonordisk.com
NN7008 Novo Nordisk recombinant hemophilia A application submitted
(turoctocog alfa) Princeton, NJ protein novonordisk.com
10 Medicines in Development Biologics 2013

11. Biologic Medicines in Development
Blood Disorders
Product Name Sponsor Product Indication Development Phase
Category
NN7088 Novo Nordisk recombinant hemophilia A Phase III
(N8-GP, rFVIII Princeton, NJ protein novonordisk.com
glycopegylated)
NN7999 Novo Nordisk recombinant hemophilia B Phase III
(N9-GP, rFIX Princeton, NJ protein novonordisk.com
glycopegylated)
OBI-1 Inspiration Biopharmaceuticals recombinant hemophilia A Phase III
(recombinant Cambridge, MA protein (Fast Track) inspirationbio.com
porcine factor VIII) Ipsen
(Orphan Drug) Paris, France
PF-05280602 Catalyst Biosciences recombinant hemophilia Phase I
(rhFVIIa) South San Francisco, CA protein catalystbiosciences.com
Pfizer pfizer.com
New York, NY
PRT4445 Portola Pharmaceuticals recombinant hemorrhage Phase II
(FXa inhibitor antidote) South San Francisco, CA protein portola.com
recombinant Biogen Idec recombinant hemophilia A Phase III
factor VIII-Fc Weston, MA fusion (Fast Track) biogenidec.com
(Orphan Drug) protein
recombinant Biogen Idec recombinant hemophilia B application submitted
factor IX-Fc Weston, MA fusion (Fast Track) biogenidec.com
(Orphan Drug) protein ----------------------------------------------- ---------------------------------
hemophilia B in children Phase III
(Fast Track) biogenidec.com
SelG1 Selexys Pharmaceuticals mAb sickle cell anemia Phase I completed
Oklahoma City, OK selexys.com
sotatercept Acceleron Pharma recombinant anemia in patients with end-stage Phase II
(ACE-011) Cambridge, MA fusion renal disease acceleronpharma.com
Celgene protein (see also cancer) celgene.com
Summit, NJ
TT-173 Thrombotargets recombinant blood coagulation disorders Phase I
Durham, NC protein thrombotargets.com
Wilate® Octapharma USA recombinant surgical blood loss Phase III
von Willebrand factor/ Hoboken, NJ protein octapharma.com
coagulation factor VIII
complex (human)
Medicines in Development Biologics 2013 11

12. Biologic Medicines in Development
Cancer and Related Conditions
Product Name Sponsor Product Indication Development Phase
Category
8H9 mAb United Therapeutics mAb metastatic brain cancer Phase I
Silver Spring, MD unither.com
212-Pb-TCMC- AREVA Med mAb HER2-positive cancer metastasized Phase I
trastuzumab Bethesda, MD to the abdominal region arevamed.com
AbGn-7 AbGenomics International mAb solid tumors Phase I
Los Altos, CA
ABIO-0501 Abiogen Pharma cell therapy chronic myeloid leukemia (CML) Phase II
(TALL-104) Pisa, Italy abiogen.it
ABT-806 AbbVie mAb solid tumors Phase I
North Chicago, IL abbvie.com
Actimab-A Actinium Pharmaceuticals mAb acute myeloid leukemia (AML) Phase I/II
(M195 mAb) New York, NY actiniumpharmaceuti
cals.com
Adcetris® Seattle Genetics mAb cutaneous T-cell lymphoma, Phase III
brentuximab vedotin Bothell, WA front-line Hodgkin lymphoma, seattlegenetics.com
(Orphan Drug) post-transplant Hodgkin lymphoma
relapse prevention
------------------------------------------------ ---------------------------------
non-Hodgkin lymphoma, Phase II
non-lymphoma malignancies, seattlegenetics.com
CD30-positive hematologic
malignancies, CD30-positive T-cell
lymphoma
AD-IL-12 ZIOPHARM Oncology gene therapy malignant melanoma Phase I/II
New York, NY ziopharm.com
ADI-PEG-20 Polaris Pharmaceuticals pegylated liver cancer Phase III
San Diego, CA protein polarispharma.com
------------------------------------------------ ---------------------------------
malignant melanoma, Phase II
mesothelioma, small-cell polarispharma.com
lung cancer (SCLC)
ADXS-HPV Advaxis vaccine cervical cancer, cervical Phase II
Princeton, NJ intraepithelial neoplasia advaxis.com
AE08 Antigen Express peptide malignant melanoma Phase I
Worcester, MA vaccine antigenexpress.com
AE37 Antigen Express peptide breast cancer Phase II
Worcester, MA vaccine antigenexpress.com
12 Medicines in Development Biologics 2013

13. Biologic Medicines in Development
Cancer and Related Conditions
Product Name Sponsor Product Indication Development Phase
Category
AGS-003 Argos Therapeutics personalized metastatic renal cancer Phase III
Durham, NC dendritic cell (Fast Track) argostherapeutics.com
vaccine
ALD-151 Cytomedix stem cell leukemia Phase I completed
Gaithersburg, MD therapy cytomedix.com
Duke University
Durham, NC
AlloStim™ Immunovative Therapies cell vaccine hematological malignancies, Phase I/II
cancer vaccine Shoham, Israel solid tumors immunovative.co.il
Allovectin® Vical DNA malignant melanoma Phase III
velimogene San Diego, CA immunothera- (Fast Track) vical.com
aliplasmid peutic
(Orphan Drug)
ALN-VSP Alnylam Pharmaceuticals RNA liver cancer, liver metastases Phase I
Cambridge, MA interference alnylam.com
ALT-801 Altor BioScience recombinant AML, bladder cancer, malignant Phase I/II
Miramar, FL fusion melanoma, multiple myeloma altorbioscience.com
protein
ALT-836 Altor BioScience mAb cancer Phase I/II
Miramar, FL (see also respiratory) altorbioscience.com
Genentech gene.com
South San Francisco, CA
AME-133v MENTRIK Biotech mAb non-Hodgkin lymphoma Phase II
Dallas, TX mentrik.com
AMG 386 Amgen recombinant fallopian tube cancer, Phase III
Thousand Oaks, CA fusion ovarian cancer, peritoneal cancer amgen.com
protein ------------------------------------------------ ---------------------------------
breast cancer, colorectal cancer, Phase II
gastric cancer, liver cancer, amgen.com
esophageal cancer, renal cancer
AMG 780 Amgen antibody solid tumors Phase I
Thousand Oaks, CA amgen.com
AMG 820 Amgen mAb solid tumors Phase I
Thousand Oaks, CA amgen.com
AMG 888 Amgen mAb non-small-cell lung cancer (NSCLC) Phase II
(U3-1287) Thousand Oaks, CA amgen.com
U3 Pharma
Martinsried, Germany
Medicines in Development Biologics 2013 13

14. Biologic Medicines in Development
Cancer and Related Conditions
Product Name Sponsor Product Indication Development Phase
Category
antibody-drug conjugate Genentech mAb ovarian cancer, pancreatic cancer Phase I
(RG7600) South San Francisco, CA gene.com
anti-CD3-anti-HER2/ TransTarget cell metastatic breast cancer Phase II
neu-activated T-cells Hillsborough, CA therapy
Barbara Ann Karmanos
Cancer Institute
Detroit, MI
anti-CD22 ADC Genentech mAb diffuse large B-cell lymphoma, Phase II
(RG7593) South San Francisco, CA non-Hodgkin lymphoma gene.com
Seattle Genetics ------------------------------------------------ --------------------------------
Bothell, WA chronic lymphocytic leukemia (CLL) Phase I
gene.com
anti-CD45 mAb Actinium Pharmaceuticals mAb AML Phase I
New York, NY actiniumpharmaceuti
cals.com
anti-CD79b ADC Genentech mAb diffuse large B-cell lymphoma, Phase II
(RG7596) South San Francisco, CA non-Hodgkin lymphoma gene.com
Seattle Genetics ------------------------------------------------ ---------------------------------
Bothell, WA CLL Phase I
gene.com
anti-CXCR4 Bristol-Myers Squibb mAb hematological malignancies Phase I
Princeton, NJ bms.com
anti-EGFL7 mAb Genentech mAb metastatic colorectal cancer, Phase II
(RG7414) South San Francisco, CA NSCLC gene.com
------------------------------------------------ --------------------------------
solid tumors Phase I
gene.com
anti-FGFR3 mAb Genentech mAb solid tumors Phase I
(RG7444) South San Francisco, CA gene.com
anti-HER3/EGFR Genentech mAb colorectal cancer, Phase II
DAF mAb South San Francisco, CA head and neck cancer gene.com
(RG7597)
anti-PD-L1 Bristol-Myers Squibb mAb cancer Phase I
Princeton, NJ (see also infectious) bms.com
anti-PD-L1 mAb Genentech mAb melanoma, solid tumors Phase I
(RG7446) South San Francisco, CA gene.com
anti-STEAP1 ADC Genentech mAb prostate cancer Phase I
(RG7450) South San Francisco, CA gene.com
Seattle Genetics
Bothell, WA
14 Medicines in Development Biologics 2013

15. Biologic Medicines in Development
Cancer and Related Conditions
Product Name Sponsor Product Indication Development Phase
Category
APN301 Apeiron Biologics mAb malignant melanoma, Phase II completed
(hu14.18-IL2) Vienna, Austria neuroblastoma in children apeiron-biologics.com
Archexin™ Rexahn Pharmaceuticals antisense pancreatic cancer Phase II
RX-0201 Rockville, MD rexahn.com
(Orphan Drug)
Arzerra® GlaxoSmithKline mAb CLL (first-line therapy), Phase III
ofatumumab Rsch. Triangle Park, NC diffuse large B-cell lymphoma, gsk.com
(Orphan Drug) follicular lymphoma
ASC-101 America Stem Cell stem cell myeloablation associated with Phase I/II
(Orphan Drug) Carlsbad, CA therapy hematological malignancies americastemcell.com
ASG-5ME Agensys mAb-drug pancreatic cancer, Phase I
Santa Monica, CA conjugate castration-resistant agensys.com
Seattle Genetics prostate cancer seattlegenetics.com
Bothell, WA
ASG-22ME Agensys mAb-drug solid tumors Phase I
Santa Monica, CA conjugate agensys.com
Seattle Genetics seattlegenetics.com
Bothell, WA
autologous stem cell California Stem Cell stem cell malignant melanoma Phase II
therapy Irvine, CA therapy californiastemcell.com
AV-203 AVEO Oncology mAb solid tumors Phase I
Cambridge, MA aveooncology.com
Avastin® Genentech mAb ovarian cancer application submitted
bevacizumab South San Francisco, CA gene.com
(Orphan Drug) Roche ------------------------------------------------ ---------------------------------
Nutley, NJ HER-2 negative-breast cancer, Phase III
HER-2 positive breast cancer, high- gene.com
risk carcinoid tumors, glioblastoma
multiforme, metastatic ovarian
cancer, NSCLC
AVX701 AlphaVax virus replicon CEA-expressing colorectal cancer Phase I/II
Rsch. Triangle Park, NC particle alphavax.com
vaccine
AVX901 AlphaVAx virus replicon HER2-expressing breast cancer Phase I
Rsch. Triangle Park, NC particle alphavax.com
vaccine
B7-2/GM-CSF cancer NuVax Therapeutics gene cancer Phase I
gene therapy (Radient Pharmaceuticals) therapy radient-pharma.com
(combination Tustin, CA
immunogene therapy)
Medicines in Development Biologics 2013 15

16. Biologic Medicines in Development
Cancer and Related Conditions
Product Name Sponsor Product Indication Development Phase
Category
bavituximab Peregrine Pharmaceuticals mAb NSCLC, pancreatic cancer Phase II
Tustin, CA peregrineinc.com
------------------------------------------------ --------------------------------
breast cancer, liver cancer, prostate Phase I/II
cancer, rectal adenocarcinoma peregrineinc.com
BAX-69 Baxter International mAb solid tumors Phase I
Deerfield, IL (see also other) baxter.com
BAY 79-4620 Bayer HealthCare mAb solid tumors Phase I
Pharmaceuticals bayerpharma.com
Wayne, NJ
BAY 94-9343 Bayer HealthCare mAb solid tumors Phase I
Pharmaceuticals bayerpharma.com
Wayne, NJ
BAY 20-10112 Amgen mAb solid tumors Phase I
Thousand Oaks, CA amgen.com
Bayer HealthCare bayerpharma.com
Pharmaceuticals
Wayne, PA
BC-819 BioCancell Therapeutics gene bladder cancer, pancreatic cancer Phase II
(Orphan Drug) Jerusalem, Israel therapy (Fast Track) biocancell.com
------------------------------------------------ ---------------------------------
ovarian cancer Phase I/II
biocancell.com
BHQ880 Novartis Pharmaceuticals mAb multiple myeloma Phase II
East Hanover, NJ novartis.com
BI-505 BioInvent International mAb multiple myeloma Phase I
(Orphan Drug) Lund, Sweden bioinvent.com
BI-836845 Boehringer Ingelheim mAb solid tumors Phase I
Pharmaceuticals boehringer-ingelheim.
Ridgefield, CT com
BiovaxID® Biovest International autologous follicular lymphoma Phase III
B-cell lymphoma Tampa, FL idiotype (Fast Track) biovest.com
vaccine vaccine ------------------------------------------------ ---------------------------------
mantle-cell lymphoma Phase II
biovest.com
blinatumomab Amgen mAb leukemia and lymphoma Phase II
(Orphan Drug) Thousand Oaks, CA amgen.com
BP-100-1.01 Bio-Path Holdings antisense acute lymphocytic leukemia (ALL), Phase I
(liposomal Grb-2) Houston, TX AML, CML, biopathholdings.com
myelodysplastic syndromes
16 Medicines in Development Biologics 2013

17. Biologic Medicines in Development
Cancer and Related Conditions
Product Name Sponsor Product Indication Development Phase
Category
BPX-101 Bellicum Pharmaceuticals dendritic cell prostate cancer Phase I/II
Houston, TX vaccine bellicum.com
breast cancer vaccine MabVax Therapeutics mAb vaccine breast cancer Phase I
San Diego, CA mabvax.com
BT-062 Biotest mAb multiple myeloma Phase I/II
(indatuximab ravtansine) Dreieich, Germany biotest.de
(Orphan Drug)
BYM338 Novartis Pharmaceuticals mAb cancer-related cachexia Phase II
East Hanover, NJ (see also musculoskeletal) novartis.com
CALAA-01 Calando Pharmaceuticals RNA solid tumors Phase I
Pasadena, CA interference arrowheadresearch.com
calaspargase pegol Sigma-Tau Pharmaceuticals pegylated ALL in adolescents and children Phase III
Gaithersburg, MD enzyme sigmatau.com
cancer medicine Eli Lilly biologic cancer Phase I
Indianapolis, IN lilly.com
cancer medicine Eli Lilly biologic cancer Phase I
Indianapolis, IN lilly.com
cancer vaccine Bayer HealthCare autologous follicular lymphoma Phase I
Pharmaceuticals idiotype bayerpharma.com
Wayne, NJ vaccine
Stellar Biotechnologies
Port Hueneme, CA
cancer vaccine EMD Millipore dendritic cell malignant melanoma Phase I completed
Billerica, MA vaccine millipore.com
cancer vaccine Gradalis genetically- solid tumors Phase I
Carrollton, TX modified gradalisinc.com
Mary Crowley Cancer vaccine marycrowley.org
Research Center
Dallas, TX
cancer vaccine Immunitor vaccine cancer Phase I/II
Vancouver, Canada immunitor.com
cancer vaccine TapImmune therapeutic breast cancer Phase I
(AdhTAP) Seattle, WA vaccine tapimmune.com
catumaxomab Fresenius Biotech mAb malignant ascites, Phase II completed
North America ovarian cancer fresenius-biotech.com
Waltham, MA
Medicines in Development Biologics 2013 17

18. Biologic Medicines in Development
Cancer and Related Conditions
Product Name Sponsor Product Indication Development Phase
Category
CDX-011 Celldex Therapeutics mAb breast cancer (Fast Track), Phase II
(glembatumumab vedotin) Needham, MA malignant melanoma celldextherapeutics.com
CDX-1127 Celldex Therapeutics mAb hematological malignancies, Phase I
Needham, MA solid tumors celldextherapeutics.com
CDX-1401 Celldex Therapeutics mAb solid tumors Phase I/II
Needham, MA vaccine celldextherapeutics.com
cell therapy Cell Medica cell cancer Phase II
London, United Kingdom therapy (see also infectious) cellmedica.co.uk
Center for Cell and Gene Therapy
Houston, TX
CEP-37250/KNK-2804 Teva North America mAb adenocarcinoma Phase I
North Wales, PA tevapharm.com
Kyowa Hakko Kirin Pharma kyowa-kirin-pharma.com
Princeton, NJ
CG201 CG Therapeutics antibody solid tumors Phase II
(anti-hCG vaccine) Seattle, WA vaccine cgtherapeutics.com
ch14.18 mAb United Therapeutics mAb neuroblastoma Phase III
(Orphan Drug) Silver Spring, MD unither.com
cixutumumab Eli Lilly mAb NSCLC Phase II
(LY3012217) Indianapolis, IN lilly.com
Imclone Systems
Princeton, NJ
CLT-008 Cellerant Therapeutics cell chemotherapy-induced neutropenia Phase I/II
San Carlos, CA therapy (see also transplantation) cellerant.com
CNTO-328 Janssen Biotech mAb giant lymph node hyperplasia, Phase II
(siltuximab) Horsham, PA multiple myeloma, myelodysplastic janssenbiotech.com
syndromes
------------------------------------------------ ---------------------------------
prostate cancer Phase I
janssenbiotech.com
Cotara™ Peregrine Pharmaceuticals mAb recurrent glioblastoma Phase II
mAb TNT Tustin, CA (Fast Track) peregrineinc.com
(Orphan Drug)
CRS-207 Aduro Biotech genetically- pancreatic cancer Phase II
Berkeley, CA modified adurobiotech.com
vaccine ------------------------------------------------ ---------------------------------
mesothelioma Phase I
adurobiotech.com
18 Medicines in Development Biologics 2013

19. Biologic Medicines in Development
Cancer and Related Conditions
Product Name Sponsor Product Indication Development Phase
Category
CSF-1R mAb Eli Lilly mAb solid tumors Phase I
(IMC-CS4) Indianapolis, IN lilly.com
ImClone Systems
Bridgewater, NJ
CT-011 CureTech mAb AML, colorectal cancer, diffuse Phase II
(pidilizumab) Yavne, Israel large B-cell lymphoma, follicular curetechbio.com
lymphoma, malignant melanoma
CTL019 Novartis cell therapy CLL Phase II
East Hanover, NJ novartis.com
University of Pennsylvania
Philadelphia, PA
custirsen OncoGenex Technologies antisense NSCLC, prostate cancer Phase III
(OGX-011) Vancouver, Canada (Fast Track) oncogenex.com
Teva Pharmaceuticals tevapharm.com
Tikva, Israel ------------------------------------------------ ---------------------------------
first-time NSCLC Phase II
oncogenex.com
tevapharm.com
CV-301 BN ImmunoTherapeutics cancer metastatic breast cancer Phase II
Mountain View, CA vaccine bavarian-nordic.com
CVac™ Prima BioMed personalized ovarian cancer Phase II/III
cancer vaccine MUC-1 Melbourne, Australia dendritic cell primabiomed.com.au
vaccine
CYT107 Cytheris recombinant malignant melanoma, renal cancer Phase I
(interleukin-7) Rockville, MD interleukin (see also blood, infectious, cytheris.com
transplantation)
CYT-6091 Cytimmune Sciences tumor solid tumors Phase I
(Aurimune™) Rockville, MD necrosis cytimmune.com
factor
dalotuzumab Merck mAb breast cancer, neuroendocrine Phase II
(MK-0646) Whitehouse Station, NJ tumors, NSCLC merck.com
daratumumab Janssen Biotech mAb multiple myeloma Phase I/II
Horsham, PA janssenbiotech.com
DC-IL-12 ZIOPHARM Oncology cell malignant melanoma Phase I
New York, NY therapy ziopharm.com
DCVax®-L Northwest Biotherapeutics dendritic cell glioblastoma Phase III
brain cancer vaccine Bethesda, MD vaccine nwbio.com
(Orphan Drug)
Medicines in Development Biologics 2013 19

20. Biologic Medicines in Development
Cancer and Related Conditions
Product Name Sponsor Product Indication Development Phase
Category
DEDN6526A Genentech mAb malignant melanoma Phase I
(antibody drug conjugate) South San Francisco, CA gene.com
demcizumab GlaxoSmithKline mAb solid tumors Phase I
(OMP-21M18) Rsch. Triangle Park, NC gsk.com
OncoMed Pharmaceuticals oncomed.com
Redwood City, CA
DFRF4539A Genentech mAb multiple myeloma Phase I
South San Francisco, CA gene.com
DI17E6 EMD Serono mAb colorectal cancer, prostate cancer Phase II
(anti-integrin mAb) Rockland, MA emdserono.com
DKN-01 Dekkun mAb solid tumors Phase I
Cambridge, MA
DN24-02 Dendreon personalized HER2-positive urogenital cancer Phase II
Seattle, WA dendritic cell dendreon.com
vaccine
DPX-0907 Immunovaccine vaccine breast cancer, ovarian cancer, Phase I
Halifax, Canada prostate cancer imvaccine.com
DPX-Survivac Immunovaccine peptide ovarian cancer Phase I/II
Halifax, Canada vaccine imvaccine.com
EC17 Endocyte vaccine renal cancer Phase I
West Lafayette, IN (combination therapy) endocyte.com
ecromeximab Life Science Pharmaceuticals mAb metastatic melanoma Phase II
(KW-2871) Darien, CT investinlife.com
EGEN-001 EGEN gene ovarian cancer Phase II
(Orphan Drug) Huntsville, AL therapy egeninc.com
------------------------------------------------ ---------------------------------
colorectal cancer Phase I/II
egeninc.com
elotuzumab Bristol-Myers Squibb mAb multiple myeloma Phase III
Princeton, NJ bms.com
AbbVie abbvie.com
North Chicago, IL
20 Medicines in Development Biologics 2013

21. Biologic Medicines in Development
Cancer and Related Conditions
Product Name Sponsor Product Indication Development Phase
Category
enavatuzumab AbbVie mAb solid tumors Phase I completed
North Chicago, IL abbvie.com
encapsulated cell therapy Metromedia Bio-Science cell colorectal cancer, pancreatic Phase II
New York, NY therapy cancer, prostate cancer rogosin.org
Rogosin Institute
New York, NY
encapsulated cell therapy Nuvilex cell pancreatic cancer Phase II
Silver Spring, MD therapy nuvilex.com
ensituximab Neogenix Oncology mAb colorectal cancer, Phase I
(NPC-1C) Great Neck, NY pancreatic cancer neogenix.com
entolimod Cleveland BioLabs engineered radiation damage in cancer patients Phase I
(CBLB502) Buffalo, NY peptide (see also other) cbiolabs.com
epratuzumab Y-90/ Immunomedics mAb non-Hodgkin lymphoma Phase I/II
veltuzumab combination Morris Plains, NJ immunomedics.com
Erbitux® Bristol-Myers Squibb mAb esophageal cancer Phase III
cetuximab Princeton, NJ bms.com
Eli Lilly lilly.com
Indianapolis, IN
ImClone Systems
Princeton, NJ
ETBX-011 Etubics vector colon cancer Phase II
Seattle, WA vaccine etubics.com
EZN-2968 Enzon Pharmaceuticals antisense lymphoma, solid tumors Phase I
Piscataway, NJ enzon.com
EZN-4176 Enzon Pharmaceuticals antisense prostate cancer Phase I
Piscataway, NJ enzon.com
F-627 Generon recombinant chemotherapy-induced neutropenia Phase II
Shanghai, China fusion protein generonbiomed.com
Fang™ Vaccine Gradalis autologous solid tumors Phase II
autologous tumor cell Carrollton, TX cell vaccine gradalisinc.com
vaccine
Medicines in Development Biologics 2013 21