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 • 58 treatments for cardiovascular
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
• 338 cancer therapeutics that target 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
• 176 candidates in development for an 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
• 71 medicines for autoimmune diseases, our more challenging diseases.
such as lupus, multiple sclerosis and
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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

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

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

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

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

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

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

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

 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

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

 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

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

 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

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

 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

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

 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

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

 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

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

 Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

farletuzumab Eisai mAb platinum-sensitive ovarian cancer Phase III

(MORAb-003) Woodcliff Lake, NJ eisai.com
(Orphan Drug) ------------------------------------------------ ---------------------------------
NSCLC Phase II
eisai.com

FG-3019 FibroGen mAb pancreatic cancer Phase II

San Francisco, CA (see also respiratory, other) fibrogen.com

ficlatuzumab AVEO Oncology mAb glioblastoma, lymphoma, Phase I

Cambridge, MA multiple myeloma, solid tumors aveooncology.com

flanvotumab Eli Lilly mAb malignant melanoma Phase I

(TYRP1 protein) Indianapolis, IN lilly.com

folate binding protein-E39 Galena Biopharma peptide endometrial cancer, ovarian cancer Phase I/II
(FBP) Lake Oswego, OR vaccine galenabiopharma.com

FP-1039 Five Prime Therapeutics recombinant solid tumors Phase I

San Francisco, CA fusion fiveprime.com
protein

Fzd7 Bayer HealthCare mAb solid tumors Phase I

(vantictumab) Pharmaceuticals bayerpharma.com
Wayne, NJ oncomed.com
OncoMed Pharmaceuticals
Redwood City, CA

ganitumab Amgen mAb pancreatic cancer Phase III

Thousand Oaks, CA amgen.com
------------------------------------------------ ---------------------------------
breast cancer, colorectal cancer, Phase II
sarcoma amgen.com

GC-33/RG7686 Chugai Pharma USA mAb liver cancer Phase II

Berkeley Heights, NJ chugai-pharm.com
Roche roche.com
Nutley, NJ

GI-4000 GlobeImmune genetically- colorectal cancer, NSCLC, Phase II

Louisville, CO modified pancreatic cancer globeimmune.com
vaccine

GI-6207 GlobeImmune genetically- medullary thyroid cancer Phase II

Louisville, CO modified globeimmune.com
vaccine

GI-6301 GlobeImmune genetically- solid tumors Phase I

(brachyury peptide Louisville, CO modified globeimmune.com
vaccine) vaccine

22 Medicines in Development Biologics 2013

Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

GL-0817 Gliknik peptide head and neck cancer, Phase II

(MAGE-A3 Baltimore, MD vaccine multiple myeloma gliknik.com
immunomodulator)
(Orphan Drug)

GliAtak™ Advantagene gene malignant glioma Phase II

cancer gene therapy Auburndale, MA therapy advantagene.com
(Orphan Drug)

Glionix™ NovaRx antisense glioma Phase I completed

brain vaccine San Diego, CA gene- novarx.com
modified cell
vaccine

GS-6624 Gilead Sciences mAb colorectal cancer, Phase II

(simtuzumab) Foster City, CA pancreatic cancer gilead.com
(see also respiratory, other)

GVAX® Breast BioSante Pharmaceuticals genetically- breast cancer Phase I

cancer immunotherapy Lincolnshire, IL modified biosantepharma.com
vaccine

GVAX® Colorectal BioSante Pharmaceuticals genetically- colorectal cancer Phase I

cancer immunotherapy Lincolnshire, IL modified biosantepharma.com
vaccine

GVAX® Leukemia BioSante Pharmaceuticals genetically- AML, CML Phase II

cancer immunotherapy Lincolnshire, IL modified biosantepharma.com
(Orphan Drug) vaccine

GVAX® Multiple BioSante Pharmaceuticals genetically- multiple myeloma Phase II

Myeloma Lincolnshire, IL modified biosantepharma.com
cancer vaccine
immunotherapeutic

GVAX® Pancreas AduroBiotech genetically- pancreatic cancer Phase II

cancer immunotherapy Berkeley, CA modified adurobiotech.com
(Orphan Drug) BioSante Pharmaceuticals vaccine biosantepharma.com
Lincolnshire, IL

GVAX® Prostate AduroBiotech genetically- prostate cancer Phase III

cancer immunotherapy Berkeley, CA modified (Fast Track) adurobiotech.com
BioSante Pharmaceuticals vaccine biosantepharma.com
Lincolnshire, IL

HCD122 Novartis Pharmaceuticals mAb lymphoma Phase I/II

(anti-CD40 mAb) East Hanover, NJ novartis.com
XOMA xoma.com
Berkeley, CA

HGS-ETR1 GlaxoSmithKline mAb liver cancer, multiple myeloma, Phase II

(mapatumumab) Rsch. Triangle Park, NC NSCLC gsk.com

Medicines in Development Biologics 2013 23

 Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

HS-110 Heat Biologics genetically- NSCLC Phase II

Chapel Hill, NC modified heatbio.com
vaccine

hTERT cancer Merck DNA vaccine cancer Phase I

vaccine Whitehouse Station, NJ merck.com
Vical vical.com
San Diego, CA

HuL2G7 Galaxy Biotech mAb solid tumors Phase I

Sunnyvale, CA galaxybiotech.com

HuM195/rGel Targa Therapeutics mAb AML, CML, myelodysplastic Phase I

San Diego, CA syndromes targatherapeutics.com

huN901-DM1 ImmunoGen mAb SCLC Phase II

(lorvotuzumab Cambridge, MA immunogen.com
mertansine) ----------------------------------------------- ---------------------------------
(Orphan Drug) multiple myeloma, solid tumors Phase I
immunogen.com

HyperAcute® Lung NewLink Genetics DNA NSCLC Phase II/III

turgenpumatucel-L Ames, IA vaccine linkp.com

HyperAcute® Melanoma NewLink Genetics DNA malignant melanoma Phase II

melanoma cancer vaccine Ames, IA vaccine linkp.com

HyperAcute® Pancreas NewLink Genetics DNA pancreatic cancer Phase III

algenpantucel-L Ames, IA vaccine (Fast Track) linkp.com
(Orphan Drug)

icrucumab Eli Lilly mAb bladder cancer, breast cancer, Phase II

(LY3012212) Indianapolis, IN colorectal cancer lilly.com
ImClone Systems
Bridgewater, NJ

ICT-107 ImmunoCellular Therapeutics personalized glioblastoma Phase II

(Orphan Drug) Woodland Hills, CA dendritic cell imuc.com
vaccine

IL-21 Bristol-Myers Squibb interleukin solid tumors Phase I

(BMS-982470) Princeton, NJ bms.com
(Orphan Drug)

IMA901 immatics biotechnologies peptide renal cancer treatment Phase III

(intradermal vaccine) Tübingen, Germany vaccine immatics.com
(Orphan Drug)

24 Medicines in Development Biologics 2013

Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

IMA950 immatics biotechnologies peptide gliobastoma Phase I

(intradermal vaccine) Tübingen, Germany vaccine immatics.com

IMC-gp100 Immunocore recombinant malignant melanoma treatment Phase 0

Oxon, United Kingdom vaccine immunocore.com

IMC-TR1 Eli Lilly mAb solid tumors Phase I

(LY3022859) Indianapolis, IN lilly.com
ImClone Systems
Bridgewater, NJ

IMF-001 ImmunoFrontier recombinant solid tumors Phase I

Tokyo, Japan vaccine immunofrontier.com

IMGN529 ImmunoGen mAb non-Hodgkin lymphoma Phase I

Waltham, MA immunogen.com

IMGN853 ImmunoGen mAb solid tumors Phase I

Waltham, MA immunogen.com

IMMU-102 Immunomedics mAb non-Hodgkin lymphoma Phase I/II

(epratuzumab Y-90) Morris Plains, NJ immunomedics.com

IMT-1012 Immunotope peptide breast cancer, ovarian cancer Phase I

Doylestown, PA immunothera- immunotope.com
peutic
vaccine

inotuzumab Pfizer mAb aggressive non-Hodgkin Phase III

ozogamicin New York, NY lymphoma, ALL pfizer.com
(CMC-544) UCB ucb.com
Smyrna, GA

interleukin-12 OncoSec Medical gene therapy cutaneous T-cell lymphoma, Phase II

gene therapy San Diego, CA malignant melanoma, merkel cell oncosec.com
carcinoma

Iomab-B Actinium Pharmaceuticals mAb AML Phase II

(anti-CD45 mAb) New York, NY actiniumpharmaceuti
cals.com

IRX-2 IRX Therapeutics cytokine head and neck cancer Phase II

New York, NY (Fast Track) irxtherapeutics.com

ISF35 Memgen gene CLL, non-Hodgkin lymphoma Phase II

San Diego, CA vaccine memgenbio.com

ISIS-EIF4ERx Isis Pharmaceuticals antisense NSCLC, pancreatic cancer Phase II

(EIF-4E ASO) Carlsbad, CA isispharm.com

Medicines in Development Biologics 2013 25

 Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

ISIS-STAT3Rx AstraZeneca antisense cancer Phase II

Wilmington, DE astrazeneca.com
Isis Pharmaceuticals isispharm.com
Carlsbad, CA

J 591 Lu-177 BZL Biologics mAb prostate cancer Phase II

New York, NY

JVRS-100 Colby Pharmaceuticals DNA leukemia Phase I

immunotherapeutic San Jose, CA vaccine colbypharma.com

JX-594 Jennerex Biotherapeutics genetically- liver cancer Phase II

San Francisco, CA engineered jennerex.com
virus ------------------------------------------------ ---------------------------------
colorectal cancer Phase I
jennerex.com

JX-929 Jennerex Biotherapeutics genetically- breast cancer, pancreatic cancer Phase I

San Francisco, CA engineered jennerex.com
virus

KB004 KaloBios Pharmaceuticals mAb hematological malignancies Phase I

South San Francisco, CA kalobios.com

LA-EP2006 Sandoz growth factor chemotherapy-related Phase III

(pegfilgrastim biosimilar) Princeton, NJ febrile neutropenia us.sandoz.com

leukemia DNA vaccine Inovio Pharmaceuticals DNA AML, CML Phase II

(WT1) Blue Bell, PA vaccine inovio.com
University of Southampton
Hampshire, United Kingdom

Leukine® Bayer HealthCare granulocyte CLL Phase II

sargramostim Pharmaceuticals macrophage bayerpharma.com
Wayne, NJ colony-
stimulating
factor

LFA102 Novartis Pharmaceuticals mAb breast cancer, prostate cancer Phase I

East Hanover, NJ novartis.com
XOMA
Berkeley, CA

LG631 Lentigen stem cell chemoprotection in patients with Phase I

Gaithersburg, MD therapy glioblastoma lentigen.com

lirilumab Bristol-Myers Squibb mAb cancer Phase I

(anti-KIR) Princeton, NJ bms.com

26 Medicines in Development Biologics 2013

Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

LOR-2040 Lorus Therapeutics antisense AML Phase II

(Orphan Drug) Toronto, Canada lorusthera.com

Lucanix® NovaRx antisense NSCLC Phase III

belagen pumatucel-L San Diego, CA gene-modified (Fast Track) novarx.com
cell vaccine

LY2495655 Eli Lilly mAb cancer cachexia Phase II

(anti-myostatin mAb) Indianapolis, IN (see also musculoskeletal) lilly.com

LY2875358 Eli Lilly mAb cancer Phase II

(c-met mAb) Indianapolis, IN lilly.com

MABp1 XBiotech mAb cancer-related cachexia Phase III

Austin, TX (see also cardiovascular, xbiotech.com
diabetes, skin)
------------------------------------------------ ---------------------------------
advanced cancer Phase I/II
xbiotech.com
------------------------------------------------ ---------------------------------
leukemia Phase I
xbiotech.com

MAGE-A3 GlaxoSmithKline recombinant bladder cancer, melanoma, Phase III

(antigen-specific cancer Rsch. Triangle Park, NC immuno- NSCLC gsk.com
immunotherapeutic) therapeutic
vaccine

MEDI-551 AstraZeneca mAb hematological malignancies Phase II

(anti-CD19 mAb) Wilmington, DE (see also autoimmune) astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD

MEDI-565 Amgen bispecific solid tumors Phase I

(anti-CEA BiTE) Thousand Oaks, CA antibody amgen.com
AstraZeneca astrazeneca.com
Wilmington, DE medimmune.com
MedImmune
Gaithersburg, MD

MEDI-573 AstraZeneca mAb solid tumors Phase II

(anti-IGF mAb) Wilmington, DE astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD

MEDI-575 AstraZeneca mAb glioblastoma, NSCLC Phase II

(anti-PDGFRα mAb) Wilmington, DE astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD

Medicines in Development Biologics 2013 27

 Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

MEDI-0639 AstraZeneca mAb solid tumors Phase I

(anti-DLL-4 mAb) Wilmington, DE astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD

MEDI-3617 AstraZeneca mAb solid tumors Phase I

(anti-ANG-2 mAb) Wilmington, DE astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD

MEDI-4736 AstraZeneca mAb cancer Phase I

(anti-CD274 mAb) Wilmington, DE astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD

MEDI-6469 AgonOx mAb solid tumors Phase I

(anti-OX40 mAb) Portland, OR agonox.com
AstraZeneca astrazeneca.com
Wilmington, DE medimmune.com
MedImmune
Gaithersburg, MD

melanoma vaccine MabVax Therapeutics mAb vaccine malignant melanoma Phase I

San Diego, CA mabvax.com

melanoma vaccine Polynoma vaccine malignant melanoma Phase III

(POL-103A) San Diego, CA polynoma.com

MGA271 MacroGenics mAb solid tumors Phase I

(anti-B7-H3) Rockville, MD macrogenics.com

MGAH22 MacroGenics mAb solid tumors Phase I

(anti-HER2) Rockville, MD macrogenics.com

milatuzumab Immunomedics mAb CLL Phase I

(Orphan Drug) Morris Plains, NJ immunomedics.com

milatuzumab-DOX Immunomedics mAb multiple myeloma Phase I

Morris Plains, NJ immunomedics.com

MINT1526A Genentech mAB solid tumors Phase I

South San Francisco, CA gene.com

MK-3475 Merck mAb malignant melanoma Phase II

Whitehouse Station, NJ merck.com
------------------------------------------------ -------------------------------
NSCLC Phase I
merck.com

28 Medicines in Development Biologics 2013

Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

MKC1106-MT Colby Pharmaceuticals vaccine malignant melanoma Phase II

San Jose, CA colbypharma.com

MLN0264 Millennium Pharmaceuticals mAb gastrointestinal cancer Phase I

(GCC antibody drug Cambridge, MA mlnm.com
conjugate)

MM-111 Merrimack Pharmaceuticals recombinant breast cancer, solid tumors Phase I

Cambridge, MA fusion merrimackpharma.com
protein

mogamulizimab Kyowa Hakko Kirin Pharma mAb cutaneous T-cell lymphoma Phase III
(Orphan Drug) Princeton, NJ kyowa-kirin-pharma.com
------------------------------------------------ ---------------------------------
adult T-cell lymphoma, Phase II
T-cell leukemia kyowa-kirin-pharma.com

MORAb-004 Eisai mAb colorectal cancer, melanoma, Phase II

Woodcliff Lake, NJ sarcoma eisai.com

MORAb-009 Eisai mAb mesothelioma Phase II

(amatuximab) Woodcliff Lake, NJ eisai.com
(Orphan Drug)

moxetumomab AstraZeneca mAb hematological malignancies Phase I

pasudotox Wilmington, DE astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD

Multikine® CEL-SCI growth factor, head and neck cancer Phase III
leukocyte Vienna, VA interferon, cel-sci.com
interleukin cancer interleukin
(Orphan Drug)

MVA-BN® HER2 BN ImmunoTherapeutics cell vaccine breast cancer Phase I

Mountain View, CA bavarian-nordic.com

MVA-BN®-PRO BN ImmunoTherapeutics vaccine prostate cancer Phase I/II

Mountain View, CA bavarian-nordic.com

mycobacterium Bioniche Therapeutics myco- bladder cancer Phase III

cell wall-DNA Pointe-Claire, Canada bacterial (Fast Track) bioniche.com
complex (MCC) DNA

necitumumab Bristol-Myers Squibb mAb NSCLC Phase III

Princeton, NJ bms.com
Eli Lilly lilly.com
Indianapolis, IN
ImClone Systems
Princeton, NJ

Medicines in Development Biologics 2013 29

 Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

neuroblastoma vaccine MabVax Therapeutics mAb vaccine neuroblastoma Phase I

San Diego, CA mabvax.com

NeuVax™ Galena Biopharma peptide breast cancer Phase III

nelipepmut-S Lake Oswego, OR vaccine galenabiopharma.com
------------------------------------------------ ---------------------------------
prostate cancer Phase I/II
galenabiopharma.com

NGR-TNF Molmed recombinant mesothelioma Phase III

(Orphan Drug) Milan, Italy fusion molmed.com
protein

NHS-IL 12 EMD Serono interleukin solid tumors Phase I

Rockland, MA emdserono.com
National Cancer Institute
Bethesda, MD

NiCord® Gamida Cell stem cell hematological malignancies Phase I/II

umbilical cord blood Jerusalem, Israel therapy (see also blood) gamida-cell.com
stem cell therapy

nimotuzumab InnoMab PTE mAb glioma Phase II

(Orphan Drug) Singapore innomab.net

nivolumab Bristol-Myers Squibb mAb melanoma, NSCLC, renal cell Phase III
(anti-PD1) Princeton, NJ carcinoma bms.com
----------------------------------------------- ---------------------------------
solid tumors Phase I/II
bms.com

Nplate™ Amgen recombinant chemotherapy-induced Phase II

romiplostim Thousand Oaks, CA fusion thrombocytopenia amgen.com
protein (see also autoimmune)

NTDDS Periphagen Holdings gene cancer pain Phase II completed

(intradermal Pittsburgh, PA therapy
gene therapy)

NY-ESO-1 antigen- GlaxoSmithKline recombinant malignant melanoma Phase I

specific cancer Rsch. Triangle Park, NC vaccine gsk.com
immunotherapeutic

obinutuzumab Biogen Idec mAb CLL, diffuse large B-cell lymphoma, Phase III
(GA101) Weston, MA non-Hodgkin lymphoma biogenidec.com
Genentech gene.com
South San Francisco, CA

OHR/AVR118 Ohr Pharmaceutical immune- cachexia associated with cancer Phase II

New York, NY based ohrpharmaceutical.com
therapy

30 Medicines in Development Biologics 2013

Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

olaratumab Eli Lilly mAb glioblastoma Phase II

(LY3012207) Indianapolis, IN lilly.com
ImClone Systems
Princeton, NJ

OMP-52M51 GlaxoSmithKline mAb hematological malignancies Phase I

(anti-Notch 1) Rsch. Triangle Park, NC gsk.com
OncoMed Pharmaceuticals oncomed.com
Redwood City, CA

OMP-59R5 GlaxoSmithKline mAb pancreatic cancer Phase I/II

(anti-Notch 2/3) Rsch. Triangle Park, NC gsk.com
OncoMed Pharmaceuticals oncomed.com
Redwood City, CA

onartuzumab Genentech mAb metastatic NSCLC Phase III

(anti-c-met- mAb) South San Francisco, CA gene.com

Onconase® Tamir Biotechnology ribonuclease NSCLC Phase II

ranpirnase Monmouth Junction, NJ tamirbio.com

Oncoprex™ Genprex gene therapy NSCLC Phase I

TUSC2 nanoparticles Austin, TX genprex.com

OncoVAX® Vaccinogen cell vaccine colorectal cancer Phase I/II

colorectal cancer Frederick, MD (Fast Track) vaccinogeninc.com
vaccine

ONT-10 Oncothyreon vaccine solid tumors Phase I

Seattle, WA oncothyreon.com

Ontak® Eisai recombinant malignant melanoma Phase II

denileukin diftitox Woodcliff Lake, NJ fusion protein eisai.com

OPT-822/OPT-821 Optimer Biotechnology vaccine breast cancer Phase III

Taipei, Taiwan optimerbiotech.com

oregovomab Quest Pharmatech mAb ovarian cancer Phase II

(Orphan Drug) Edmonton, Canada questpharmatech.com

ovarian cancer vaccine MabVax Therapeutics mAb vaccine ovarian cancer Phase II
San Diego, CA mabvax.com

PancAtak™ Advantagene gene therapy pancreatic cancer Phase I/II

cancer gene therapy Auburndale, MA advantagene.com

pbi-shRNA™ STMN1 LP Gradalis gene therapy solid tumors Phase I

Carrollton, TX gradalisinc.com

Medicines in Development Biologics 2013 31

 Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

PEGPH20 Halozyme Therapeutics recombinant solid tumors Phase II

San Diego, CA enzyme halozyme.com

Perjeta™ Genentech mAb early HER2-positive breast cancer Phase III

pertuzumab South San Francisco, CA ----------------------------------------------- gene.com
HER2-positive metastatic breast ---------------------------------
cancer (second-line treatment), Phase II
HER2-positive gastric cancer gene.com

PF-03446962 Pfizer mAb solid tumors Phase I

New York, NY pfizer.com

PF-04605412 Pfizer mAb solid tumors Phase I

New York, NY pfizer.com

PF-04856884 Pfizer fusion renal cancer Phase II

(CVX060) New York, NY peptide pfizer.com
------------------------------------------------ --------------------------------
solid tumors Phase I
pfizer.com

PF-05082566 Pfizer mAb cancer, lymphoma Phase I

New York, NY pfizer.com

PF-05280014 Pfizer mAb metastatic breast cancer Phase I

(trastuzumab biosimilar) New York, NY pfizer.com

polyclonal antibody Cancer Advances polyclonal gastric cancer, pancreatic cancer Phase III
stimulator Durham, NC antibody (see also digestive) canceradvances.com
(Orphan Drug) vaccine ------------------------------------------------ --------------------------------
colorectal cancer Phase II
canceradvances.com

PRAME antigen-specific GlaxoSmithKline recombinant malignant melanoma, NSCLC Phase I

cancer Rsch. Triangle Park, NC vaccine gsk.com
immunotherapeutic

prophage Agenus vaccine glioma Phase II

cancer vaccine (G-series) Lexington, MA agenusbio.com

prophage Agenus vaccine malignant melanoma Phase III

cancer vaccine (M-series) Lexington, MA (Fast Track) agenusbio.com
(Orphan Drug)

prophage Agenus vaccine renal cancer Phase III

cancer vaccine (R-series) Lexington, MA (Fast Track) agenusbio.com

32 Medicines in Development Biologics 2013

Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

ProstAtak™ Advantagene gene therapy prostate cancer Phase III

cancer gene therapy Auburndale, MA (Fast Track) advantagene.com

Prostvac® BN ImmunoTherapeutics recombinant prostate cancer Phase III

recombinant vaccinia Mountain View, CA vaccine bavarian-nordic.com
virus-expressing PSA

Provenge® Dendreon dendritic cell prostate cancer Phase III

sipuleucel-T Seattle, WA vaccine (early-stage disease, recurrent) dendreon.com
------------------------------------------------ ---------------------------------
prostate cancer (early-stage Phase II
disease, neoadjuvant dendreon.com
therapy), prostate cancer
(metastatic, first-line therapy)

PSMA-ADC Progenics Pharmaceuticals mAb prostate cancer Phase II

Tarrytown, NY progenics.com

PT107 Pique Therapeutics vaccine NSCLC Phase II

Durham, NC piquetherapeutics.com

purine nucleoside PNP Therapeutics gene solid tumors Phase I

phosphorylase Birmingham, AL therapy pnptherapeutics.com
gene therapy

PVX-410 OncoPep peptide multiple myeloma Phase I/II

immunotherapeutic Methuen, MA vaccine oncopep.com

QBI-139 Quintessence Biosciences ribonuclease solid tumors Phase I

Madison, WI quintbio.com

ramucirumab Eli Lilly mAb breast cancer, colorectal cancer, Phase III
(LY3009806) Indianapolis, IN gastric cancer lilly.com
ImClone Systems
Princeton, NJ

Redectane® Wilex mAb diagnosis of kidney cancer Phase III

girentuximab I-124 Munich, Germany wilex.com

REGN1400 Regeneron Pharmaceuticals mAb cancer Phase I

Tarrytown, NY regeneron.com

REIC gene therapy Momotaro-Gene gene prostate cancer Phase I

Okayama, Japan therapy mt-gene.com

RG7116 Roche mAb solid tumors Phase I

Nutley, NJ roche.com

RG7155 Roche mAb solid tumors Phase I

Nutley, NJ roche.com

Medicines in Development Biologics 2013 33

 Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

RG7160 Roche mAb colorectal cancer Phase II

(humAb EGFR) Nutley, NJ roche.com

RG7212 Roche mAb solid tumors Phase I

Nutley, NJ roche.com

RG7356 Roche mAb AML, solid tumors Phase I

(anti-CD44 mAb) Nutley, NJ roche.com

RG7458 Genentech mAb ovarian cancer Phase I

(antibody-drug conjugate) South San Francisco, CA gene.com
Seattle Genetics
Bothell, WA

RIGScan™ Navidea Biopharmaceuticals mAb diagnosis of colorectal in clinical trials

radiodiagnostic agent Dublin, OH cancer navidea.com

rilotumumab Amgen mAb colorectal cancer, gastric cancer, Phase II

Thousand Oaks, CA prostate cancer, SCLC amgen.com

rindopepimut Celldex Therapeutics vaccine first-line glioblastoma Phase III

(Orphan Drug) Needham, MA (Fast Track) celldextherapeutics.com
------------------------------------------------ ---------------------------------
second-line glioblastoma Phase II
(Fast Track) celldextherapeutics.com

Rituxan® Genentech mAb diffuse large B-cell lymphoma Phase III

rituximab South San Francisco, CA gene.com

RON8 mAb Eli Lilly mAb cancer Phase I

Indianapolis, IN lilly.com
ImClone Systems
Princeton, NJ

SAR3419 Sanofi US mAb ALL, non-Hodgkin lymphoma Phase II

(maytansin-loaded Bridgewater, NJ sanofi.com
anti-CD19 mAb)

SAR153192 Regeneron Pharmaceuticals mAb cancer Phase I

(REGN 421) Tarrytown, NY regeneron.com
(anti-DLL4 mAb) Sanofi US sanofi.com
Bridgewater, NJ

SAR256212 Merrimack Pharmaceuticals mAb breast cancer Phase II

(MM-121) Cambridge, MA merrimackpharma.com
(anti-ErbB3 mAb) Sanofi US sanofi.com
Bridgewater, NJ ------------------------------------------------ ---------------------------------
solid tumors Phase I
(combination therapy) merrimackpharma.com
sanofi.com

34 Medicines in Development Biologics 2013

Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

SAR307746 Regeneron Pharmaceuticals mAb solid tumors Phase I

(REGN910) Tarrytown, NY regeneron.com
(anti-angiopoietin-2 mAb) Sanofi US sanofi.com
Bridgewater, NJ

SAR566658 Sanofi US mAb DS6-positive solid tumors Phase I

(maytansin-loaded Bridgewater, NJ sanofi.com
anti-DS6)

SAR650984 Sanofi US mAb hematological malignancies Phase I

(anti-CD38 Bridgewater, NJ sanofi.com
naked mAb)

sarcoma MabVax Therapeutics mAb sarcoma Phase II

cancer vaccine San Diego, CA vaccine mabvax.com

SB-313 Sangamo BioSciences cell glioblastoma Phase I

Richmond, CA therapy sangamo.com

SCH-721015 FKD Therapies gene therapy bladder cancer Phase II

Kuopio, Finland

SGN-75 Seattle Genetics mAb non-Hodgkin lymphoma, Phase I

(vorsetuzumab mafodotin) Bothell, WA renal cancer seattlegenetics.com

SL-401 Stemline Therapeutics recombinant AML, myleodysplastic Phase I/II

(Orphan Drug) New York, NY fusion syndromes stemline.com
protein

SL-701 Stemline Therapeutics dendritic cell glioma Phase I/II

New York, NY vaccine stemline.com

small-cell lung cancer MabVax Therapeutics mAb vaccine SCLC Phase I

vaccine San Diego, CA mabvax.com

SNS01-T Senesco Technologies gene diffuse large B-cell lymphoma, Phase I/II
Bridgewater, NJ therapy; RNA mantle cell lymphoma, senesco.com
interference multiple myeloma

sotatercept Acceleron Pharma recombinant chemotherapy-induced Phase II/III

(ACE-011) Cambridge, MA fusion anemia acceleronpharma.com
Celgene protein (see also blood) celgene.com
Summit, NJ

Medicines in Development Biologics 2013 35

 Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

SPI-2012 Hanmi Pharmaceutical growth neutropenia in cancer patients Phase II

(granulocyte colony- Seoul, South Korea factor sppirx.com
stimulating factor, Spectrum Pharmaceuticals
long-acting) Henderson, NV

StemEx® Gamida Cell stem cell hematological malignancies Phase III

carlecortemcel-L Jerusalem, Israel therapy (Fast Track) gamida-cell.com
(Orphan Drug) Teva Pharmaceutical tevapharm.com
Tikva, Israel

Stimuvax® EMD Serono peptide NSCLC Phase III

lioposomal Rockland, MA vaccine (Fast Track) emdserono.com
cancer vaccine Oncothyreon oncothyreon.com
Seattle, WA

Sym004 EMD Serono mAb head and neck cancer Phase II

Rockland, MA emdserono.com
Symphogen symphogen.com
Lyngby, Denmark ------------------------------------------------ ---------------------------------
solid tumors Phase I
emdserono.com
symphogen.com

tabalumab Eli Lilly mAb multiple myeloma Phase III

(BAFF inhibitor) Indianapolis, IN (see also autoimmune) lilly.com

talactoferrin alfa Agennix recombinant NSCLC Phase III completed

(Orphan Drug) Princeton, NJ protein (Fast Track) agennix.com

talminogene Amgen gene therapy malignant melanoma Phase III

laherparepvec Thousand Oaks, CA amgen.com

TeloB-Vax Adamis Pharmaceuticals DNA prostate cancer Phase I

telomerase San Diego, CA vaccine adamispharmaceuticals.
cancer vaccine com

TF2 Immunomedics mAb diagnosis of colorectal cancer Phase I

Morris Plains, NJ immunomedics.com

TG-1101 TG Therapeutics mAb CLL, non-Hodgkin lymphoma Phase I/II

(ublituximab) New York, NY tgtherapeutics.com
(Orphan Drug)

tigatuzumab Daiichi Sankyo mAb breast cancer, liver cancer, Phase II

Parsippany, NJ ovarian cancer, pancreatic cancer dsi.com

TKM-PLK1 Tekmira Pharmaceuticals RNA lymphoma, solid tumors Phase I

Burnaby, Canada interference tekmirapharm.com

36 Medicines in Development Biologics 2013

Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

Toca-511 Tocagen gene therapy glioblastoma Phase I/II

(cytosine deaminase San Diego, CA tocagen.com
gene therapy)

trastuzumab Genentech mAb first-line HER2-positive metastatic Phase III

emtansine South San Francisco, CA breast cancer, third-line HER2-posi- gene.com
(T-DM1) Roche tive metastatic breast cancer roche.com
Nutley, NJ
------------------------------------------------ ---------------------------------
early HER2-positive breast Phase II
cancer, advanced HER2-positive gene.com
gastric cancer roche.com

TRC105 TRACON Pharmaceuticals mAb bladder cancer, liver cancer, Phase II

San Diego, CA ovarian cancer, prostate cancer traconpharma.com
------------------------------------------------ ---------------------------------
solid tumors Phase I
traconpharma.com

tremelimumab AstraZeneca mAb solid tumors Phase II

(anti-CTLA4 mAb) Wilmington, DE astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD

Trivax Activartis Biotech personalized glioblastoma Phase II

cancer vaccine Vienna, Austria dendritic cell activartis.com
vaccine

tumor immunotherapy Genentech mAb solid tumors Phase I

mAb South San Francisco, CA gene.com

TV1-Brain-1 TVAX Biomedical cell vaccine glioblastoma Phase II

Lenexa, KS tvaxbiomedical.com

TV1-Kidney-1 TVAX Biomedical cell vaccine renal cancer Phase II

Lenexa, KS tvaxbiomedical.com

U3-1565 Daiichi Sankyo mAb solid tumors Phase I

Parsippany, NJ dsi.com

urelumab Bristol-Myers Squibb mAb cancer Phase I

(anti-CD137) Princeton, NJ bms.com

Medicines in Development Biologics 2013 37

 Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

V503 Merck vaccine prevention of cervical cancer, Phase III

Whitehouse Station, NJ prevention of vulvovaginal cancer merck.com
(see also infectious)

VAY736 MorphoSys mAb CLL Phase I

Munich, Germany (see also other) morphosys.com
Novartis Pharmaceuticals novartis.com
East Hanover, NJ

VB-111 VBL Therapeutics gene thyroid cancer Phase II

Or Yehuda, Israel therapy vblrx.com
------------------------------------------------ ---------------------------------
glioblastoma Phase I/II
vblrx.com

VB4-845 Viventia Biotechnologies mAb bladder cancer Phase II

(Orphan Drug) Mississauga, Canada viventia.com

Vectibix® Amgen mAb colorectal cancer (first-line therapy, application submitted

panitumumab Thousand Oaks, CA second-line therapy) amgen.com

VEGFR3 mAb Eli Lilly mAb cancer Phase I

(IMC-3C5) Indianapolis, IN lilly.com
ImClone Systems
Bridgewater, NJ

veltuzumab Immunomedics mAb CLL, non-Hodgkin lymphoma Phase I/II

(IMMU-106) Morris Plains, NJ (see also autoimmune) immunomedics.com
(Orphan Drug)

VGX-100 Circadian Technologies mAb solid tumors Phase I

Victoria, Australia circadian.com.au

VGX-3100 Inovio Pharmaceuticals DNA cervical dysplasia Phase II

Blue Bell, PA vaccine inovio.com

volociximab AbbVie mAb NSCLC Phase I completed

North Chicago, IL abbvie.com

VX15 Vaccinex mAb solid tumors Phase I

Rochester, NY (see also autoimmune) vaccinex.com

WT1 antigen- GlaxoSmithKline recombinant AML Phase II

specific cancer Rsch. Triangle Park, NC vaccine gsk.com
immunotherapeutic ------------------------------------------------ ---------------------------------
breast cancer Phase I
gsk.com

38 Medicines in Development Biologics 2013

Biologic Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase
Category

WT2725 Sunovion Pharmaceuticals peptide solid tumors Phase I

Marlborough, MA vaccine sunovion.com

Xgeva® Amgen mAb delay or prevention of bone application submitted

denosumab Thousand Oaks, CA metastases in prostate cancer amgen.com
------------------------------------------------ ---------------------------------
delay or prevention of bone metases Phase III
in breast cancer amgen.com
------------------------------------------------ ---------------------------------
giant cell tumor of the bone Phase II
amgen.com

Xiaflex® BioSpecifics Technologies peptide lipoma Phase II

collagenase clostridium Lynbrook, NY (see also musculoskeletal, biospecifics.com
histolyticum other)

XmAb® Boehringer Ingelheim mAb cancer Phase I

high ADCC mAb Pharmaceuticals boehringer-ingelheim.
Ridgefield, CT com
Xencor xencor.com
Monrovia, CA

XmAb® 2513 Xencor mAb Hodgkin disease, Phase I

anti-CD30 mAb Monrovia, CA T-cell lymphoma xencor.com

XmAb®5574 MorphoSys mAb CLL Phase I

anti-CD19 mAb Munich, Germany morphosys.com
Xencor xencor.com
Monrovia, CA

Y-90 hPAM 4 Immunomedics mAb pancreatic cancer Phase I/II

(IMMU-107) Morris Plains, NJ (Fast Track) immunomedics.com
(Orphan Drug)

Yervoy™ Bristol-Myers Squibb mAb adjuvant melanoma, NSCLC, Phase III

ipilimumab Princeton, NJ prostate cancer, SCLC bms.com
(Orphan Drug) ------------------------------------------------ ---------------------------------
gastric cancer, ovarian cancer Phase II
bms.com

zanolimumab Emergent BioSolutions mAb peripheral T-cell lymphoma Phase II

Rockville, MD emergentbiosolutions.
com

Zevalin® Spectrum Pharmaceuticals mAb diffuse large B-cell lymphoma Phase III
ibritumomab tiuxetan Henderson, NV sppirx.com
------------------------------------------------ ---------------------------------
non-Hodgkin lymphoma Phase II
sppirx.com

Medicines in Development Biologics 2013 39

 Biologic Medicines in Development

Cardiovascular Disease
Product Name Sponsor Product Indication Development Phase
Category

3K3A-APC ZZ Biotech recombinant stroke Phase I

Houston, TX protein zzbiotech.com

ACP-501 AlphaCore Pharma recombinant atherosclerosis, Phase I

(rhLCAT) Ann Arbor, MI protein coronary artery disease alphacorepharma.com

ACY001 Arteriocyte stem cell chronic coronary ischemia Phase I completed

Cleveland, OH therapy arteriocyte.com

adipose stem cell therapy Cytori Therapeutics stem cell myocardial ischemia Phase I/II
San Diego, CA therapy cytori.com

adult autologous stem cell Baxter International stem cell myocardial ischemia Phase III
therapy Deerfield, IL therapy baxter.com
------------------------------------------------ ---------------------------------
critical limb ischemia Phase I/II completed
baxter.com

adult mesenchymal stem BioCardia stem cell ischemic heart disorders Phase I/II
cell therapy San Carlos, CA therapy biocardia.com

ALD-201 Cytomedix stem cell ischemic heart failure Phase I completed

Gaithersburg, MD therapy cytomedix.com

ALD-301 Cytomedix stem cell critical limb ischemia Phase I/II

Gaithersburg, MD therapy cytomedix.com

ALD-401 Cytomedix stem cell ischemic stroke Phase II

Gaithersburg, MD therapy cytomedix.com

allogeneic stem Stemedica Cell Technologies stem cell ischemic stroke Phase I/II
cell therapy San Diego, CA therapy stemedica.com

ALN-PCS Alnylam Pharmaceuticals RNA hypercholesterolemia Phase I

Cambridge, MA interference alnylam.com

ALO-212 Arteriocyte stem cell critical limb ischemia Phase I

Cleveland, OH therapy arteriocyte.com

AMG 145 Amgen mAb hypercholesterolemia Phase III

Thousand Oaks, CA amgen.com
------------------------------------------------ ---------------------------------
hyperlipoproteinemia type IIa Phase II/III
amgen.com

AMR-001 Amorcyte stem cell myocardial infarction Phase II

Allendale, NJ therapy amorcyte.com
NeoStem neostem.com
New York, NY

40 Medicines in Development Biologics 2013

Biologic Medicines in Development

Cardiovascular Disease
Product Name Sponsor Product Indication Development Phase
Category

Anginera™ Theregen fibroblast heart failure, ischemic Phase I

epicardial angiogenesis San Francisco, CA cell heart disorders theragen.com
patch therapy

anti-fibrin mAb Agenix mAb diagnosis of deep vein Phase II

3B6/22 Tc-99m Sydney, Australia thrombosis, diagnosis of agenix.com
pulmonary embolism

anti-oxLDL BioInvent International mAb atherosclerosis Phase II

(BI-204/RG7418) Lund, Sweden gene.com
Genentech
South San Francisco, CA

anti-PCSK9 mAb Genentech mAb cardiovascular disease Phase II

(RG7652) South San Francisco, CA gene.com

cardiovascular medicine Eli Lilly biologic cardiovascular disease Phase I

Indianapolis, IN lilly.com

Collategene® AnGes MG gene therapy peripheral arterial disease Phase II

beperminogene Gaithersburg, MD (Fast Track) anges-mg.com
perplasmid Mitsubishi Tanabe Pharma ------------------------------------------------ --------------------------------
Osaka, Japan ischemic heart disease Phase I
anges-mg.com

combination autologous TCA Cellular Therapy stem cell coronary ischemia, Phase II
stem cell therapy Covington, LA therapy ischemic heart disorders tcacellulartherapy.com

CPI-003 Capricor stem cell myocardial infarction Phase I

(intracoronary cardio- Beverly Hills, CA therapy capricor.com
sphere-derived stem cell
therapy)

desmoteplase Lundbeck recombinant ischemic stroke Phase III

Deerfield, IL protein (Fast Track) lundbeck.com

endometrial blood stem Medistem stem cell heart failure Phase II

cell therapy San Diego, CA therapy medisteminc.com

fibroblast growth CardioVascular BioTherapeutics fibroblast severe coronary heart disease Phase II
factor-1 Las Vegas, NV growth factor (see also skin) cvbt.com

Generx™ Cardium Therapeutics gene therapy myocardial ischemia Phase III

alferminogene tadenovec San Diego, CA (Fast Track) cardiumthx.com

GGF2 Acorda Therapeutics growth congestive heart failure Phase I

(glial growth factor 2) Hawthorne, NY factor acorda.com

GSK249320 GlaxoSmithKline mAb stroke Phase II

Rsch. Triangle Park, NC gsk.com

Medicines in Development Biologics 2013 41

 Biologic Medicines in Development

Cardiovascular Disease
Product Name Sponsor Product Indication Development Phase
Category

IL-1 ß antibody Eli Lilly mAb cardiovascular disease Phase I

Indianapolis, IN lilly.com

Ilaris® Novartis Pharmaceuticals mAb secondary prevention of Phase III

canakinumab East Hanover, NJ cardiovascular events novartis.com
(see also autoimmune, diabetes,
musculoskeletal)

inclacumab Roche mAb peripheral vascular disease Phase II

(RG1512) Nutley, NJ roche.com

Iprivask® Canyon Pharmaceuticals recombinant prevention of thrombosis in clinical trials

desirudin Columbia, MD protein canyonpharma.com

ischemic tolerant Stemedica Cell Technologies stem cell acute myocardial infarction Phase II
allogeneic mesenchymal San Diego, CA therapy stemedica.com
stem cell therapy

ISIS-APOCIIIRx Isis Pharmaceuticals antisense coronary artery disease Phase II

Carlsbad, CA isispharm.com

ISIS-CRPRx Isis Pharmaceuticals antisense atrial fibrillation, Phase II

Carlsbad, CA coronary artery disease isispharm.com
(see also autoimmune)

ixmyelocel-T Aastrom Biosciences stem cell critical limb ischemia Phase III
(Orphan Drug) Ann Arbor, MI therapy (Fast Track) aastrom.com
------------------------------------------------ ---------------------------------
dilated cardiomyopathy Phase II
aastrom.com

MABp1 XBiotech mAb vascular restinosis Phase II

Austin, TX (see also cancer, diabetes, skin) xbiotech.com

mesenchymal precursor Mesoblast stem cell congestive heart failure, Phase II

cell (MPC) product New York, NY therapy myocardial infarction mesoblast.com
(see also diabetes,
musculoskeletal, transplantation)

MultiGeneAngio MultiGene Vascular Systems cell peripheral arterial disorders Phase I/II
Nesher, Israel therapy mgvs.co.il

MultiStem® Athersys stem cell myocardial infarction, stroke Phase II

stem cell therapy Cleveland, OH therapy (see also digestive, transplantation) athersys.com

Mydicar™ Celladon gene therapy advanced heart failure Phase II

SERCA 2a gene therapy La Jolla, CA class III/IV celladon.net

MyoCell® Bioheart stem cell congestive heart failure Phase II/III

muscle stem cell therapy Sunrise, FL therapy bioheartinc.com

42 Medicines in Development Biologics 2013

Biologic Medicines in Development

Cardiovascular Disease
Product Name Sponsor Product Indication Development Phase
Category

MyoCell® SDF-1 Bioheart stem cell congestive heart failure Phase I

muscle stem cell therapy Sunrise, FL therapy bioheartinc.com
(second-generation)

NTx®-265 Stem Cell Therapeutics stem cell stroke Phase II

Toronto, Canada therapy stemcellthera.com

PB-1046 PhaseBio Pharmaceuticals recombinant essential hypertension Phase I

Malvern, PA fusion protein phasebio.com

PCSK9 adnectin Bristol-Myers Squibb adnectin cardiovascular disease Phase I

Princeton, NJ bms.com

PF-04950615 Pfizer mAb hypercholesterolemia Phase II

(RN316) New York, NY pfizer.com

PF-05335810 Pfizer biologic hypercholesterolemia Phase I

New York, NY pfizer.com

PLX-PAD Pluristem Therapeutics stem cell intermittent claudication Phase II

Haifa, Israel therapy pluristem.com

Prochymal® Osiris Therapeutics stem cell acute myocardial infarction Phase II

remestemcel-L Columbia, MD therapy (see also diabetes, digestive, osiris.com
respiratory, transplantation)

PRT-201 Proteon Therapeutics recombinant vascular access for hemodialysis Phase II

(Orphan Drug) Waltham, MA protein (Fast Track) proteontherapeutics.com
------------------------------------------------ ---------------------------------
peripheral vascular disease Phase I
proteontherapeutics.com

SAR236553/REGN727 Regeneron Pharmaceuticals mAb hypercholesterolemia Phase III

(anti-PCSK-9 mAb) Tarrytown, NY regeneron.com
Sanofi US sanofi.com
Bridgewater, NJ

SB623 SanBio stem cell stroke Phase I/II

Mountain View, CA therapy san-bio.com

SDF-1 Juventas Therapeutics gene therapy critical limb ischemia, heart failure Phase II
(stromal cell-derived Cleveland, OH juventasinc.com
factor-1)

Syncria® GlaxoSmithKline recombinant heart failure Phase II

albiglutide Rsch. Triangle Park, NC fusion protein (see also diabetes) gsk.com

TS01 Thrombolytic Science Interna- recombinant acute ischemic stroke Phase I

tional protein tsillc.net
Cambridge, MA

Medicines in Development Biologics 2013 43

 Biologic Medicines in Development

Cardiovascular Disease
Product Name Sponsor Product Indication Development Phase
Category

Vascugel® Shire Regenerative Medicine cell peripheral vascular disorder in Phase II

cell therapy Wayne, PA therapy patients undergoing arteriovenous shire.com
(Orphan Drug) access procedures for hemodialysis
(Fast Track)

VM-202 VW BioPharma gene therapy peripheral arterial disease Phase II

(modified hepatocyte Atlanta, GA (see also neurologic) viromed.co.kr
growth factor gene ------------------------------------------------ ---------------------------------
therapy) myocardial ischemia Phase I
viromed.co.kr

Diabetes
Product Name Sponsor Product Indication Development Phase
Category

BHT-3201 Bayhill Therapeutics DNA type 1 diabetes Phase I completed

Palo Alto, CA vaccine bayhilltx.com

CVX-096 Pfizer recombinant type 2 diabetes Phase I

(PF-04856883) New York, NY fusion pfizer.com
protein

diabetes medicine Eli Lilly biologic diabetes Phase I

Indianapolis, IN lilly.com

diabetes medicine Eli Lilly biologic diabetes Phase I

Indianapolis, IN lilly.com

diabetes medicine Eli Lilly biologic diabetes Phase I

Indianapolis, IN lilly.com

Diamyd® Diamyd vaccine type 1 diabetes Phase III

autoimmune Pittsburgh, PA diamyd.com
diabetes vaccine
(Orphan Drug)

dulaglutide Eli Lilly recombinant type 2 diabetes Phase III

(GLP-1) Indianapolis, IN fusion protein lilly.com

gevokizumab XOMA mAb type 1 diabetes, type 2 diabetes Phase II

(IL-1B inhibitor mAb) Berkeley, CA (see also eye, musculoskeletal, xoma.com
skin)

GSK1070806 GlaxoSmithKline mAb type 2 diabetes Phase II

(IL-18 mAb) Rsch. Triangle Park, NC (see also digestive) gsk.com

IBC-VS01 Orban Biotech vaccine type I diabetes Phase I completed

(insulin B-chain vaccine) Brookline, MA orbanbiotech.com

44 Medicines in Development Biologics 2013

Biologic Medicines in Development

Diabetes
Product Name Sponsor Product Indication Development Phase
Category

Ilaris® Novartis Pharmaceuticals mAb type 2 diabetes Phase II/III

canakinumab East Hanover, NJ (see also autoimmune, novartis.com
cardiovascular, musculoskeletal)
------------------------------------------------ ---------------------------------
type 1 diabetes Phase II
novartis.com

INGAP peptide Exsulin peptide type 1 diabetes, type 2 diabetes Phase II

Minneapolis, MN fragment exsulin.com

insulin glargine Sanofi US recombinant type 1 diabetes, type 2 diabetes Phase III
(rDNA origin) Bridgewater, NJ insulin sanofi.com
(new formulation)

insulin lispro/BC106 Adocia recombinant type 1 diabetes, type 2 diabetes Phase I

(LY275585) Lyon, France protein lilly.com
Eli Lilly
Indianapolis, IN

ISIS-GCCRRx Isis Pharmaceuticals antisense type 2 diabetes Phase I

Carlsbad, CA (see also other) isispharm.com

ISIS-GCGRRx Isis Pharmaceuticals antisense type 2 diabetes Phase I

Carlsbad, CA isispharm.com

ISIS-PTP1BRx Isis Pharmaceuticals antisense type 2 diabetes Phase I

(PTP-1B gene inhibitor) Carlsbad, CA isispharm.com

Lantus® Sanofi US recombinant type 2 diabetes Phase I

insulin glargine Bridgewater, NJ insulin (see also cardiovascular) sanofi.com
(rDNA origin) injection and
lixisenatide combination

LY2605541 Boehringer Ingelheim insulin type 1 diabetes, type 2 diabetes Phase III
(novel basal insulin/ Pharmaceuticals hormone boehringer-ingelheim.
insulin peglispro) Ridgefield, CT com
Eli Lilly lilly.com
Indianapolis, IN

LY2963016 Boehringer Ingelheim insulin type 1 diabetes, type 2 diabetes Phase III
(new insulin glargine Pharmaceuticals hormone boehringer-ingelheim.
product) Ridgefield, CT com
Eli Lilly lilly.com
Indianapolis, IN

MABp1 XBiotech mAb type 2 diabetes Phase II

Austin, TX (see also cancer, cardiovascular, xbiotech.com
skin)

mesenchymal precursor Mesoblast stem cell type 2 diabetes Phase II

cell (MPC) product New York, NY therapy (see also cardiovascular, mesoblast.com
musculoskeletal, transplantation)

Medicines in Development Biologics 2013 45

 Biologic Medicines in Development

Diabetes
Product Name Sponsor Product Indication Development Phase
Category

PB-1023 PhaseBio Pharmaceuticals recombinant type 2 diabetes Phase II

(GLP-1 receptor agonist) Malvern, PA fusion phasebio.com
protein

PF-05231023 Pfizer biologic type 2 diabetes Phase I

New York, NY pfizer.com

Prochymal® Osiris Therapeutics stem cell type 1 diabetes Phase II

remestemcel-L Columbia, MD therapy (see also cardiovascular, digestive, osiris.com
(Orphan Drug) respiratory, transplantation)

rHuPH20/insulin Halozyme Therapeutics recombinant type 1 diabetes, type 2 diabetes Phase II

San Diego, CA fusion halozyme.com
protein

Syncria® GlaxoSmithKline recombinant type 2 diabetes application submitted

albiglutide Rsch. Triangle Park, NC fusion (see also cardiovascular) gsk.com
protein

teplizumab MacroGenics mAb type 1 diabetes Phase III

(Orphan Drug) Rockville, MD macrogenics.com

Digestive Disorders
Product Name Sponsor Product Indication Development Phase
Category

alicaforsen Atlantic Healthcare antisense pouchitis (Fast Track), Phase III

Essex, United Kingdom distal ulcerative colitis, atlantichc.com
proximal ulcerative colitis

ALV003 Alvine Pharmaceuticals recombinant celiac disease Phase I

San Carlos, CA protein alvinepharma.com

AMG 139/MEDI-2070 Amgen mAb Crohn’s disease Phase I

Thousand Oaks, CA amgen.com
AstraZeneca astrazeneca.com
Wilmington, DE

AMG 181/MEDI-7183 Amgen mAb Crohn’s disease, ulcerative colitis Phase II

Thousand Oaks, CA amgen.com
AstraZeneca astrazeneca.com
Wilmington, DE

anrukinzumab Pfizer mAb ulcerative colitis Phase II

(IMA-638) New York, NY pfizer.com

anti-IP10 Bristol-Myers Squibb mAb Crohn’s disease, ulcerative colitis Phase II

Princeton, NJ bms.com

46 Medicines in Development Biologics 2013

Biologic Medicines in Development

Digestive Disorders
Product Name Sponsor Product Indication Development Phase
Category

clazakizumab Bristol-Myers Squibb mAb Crohn’s disease Phase II

(anti-IL6) Princeton, NJ (see also autoimmune, bms.com
Alder Biopharmaceuticals musculoskeletal) alderbio.com
Bothell, WA

etrolizumab Genentech mAb ulcerative colitis Phase II

(rhuMAb-ß7) South San Francisco, CA gene.com

GSK1070806 GlaxoSmithKline mAb inflammatory bowel disease Phase I

(IL-18 mAb) Rsch. Triangle Park, NC (see also diabetes) gsk.com

HLA-DQ2 peptide vaccine ImmusanT peptide celiac disease Phase I

Cambridge, MA vaccine immusant.com

Humira® AbbVie mAb Crohn’s disease in children and Phase III

adalimumab North Chicago, IL adolescents abbvie.com
(see also autoimmune, eye,
musculoskeletal, skin, other)

MultiStem® Athersys stem cell ulcerative colitis Phase II

stem cell therapy Cleveland, OH therapy (see also cardiovascular, athersys.com
(PF-05285401) Pfizer transplantation) pfizer.com
New York, NY

NN8717 Novo Nordisk recombinant ulcerative colitis Phase II

(rFXIII) Princeton, NJ protein novonordisk.com

PDA-001 Celgene Cellular Therapeutics stem cell Crohn’s disease Phase II

(cenplacel-L) Summit, NJ therapy (see also autoimmune) celgene.com

PF-00547659 Pfizer mAb Crohn’s disease Phase II

New York, NY pfizer.com

PF-04236921 Pfizer mAb Crohn’s disease Phase II

New York, NY (see also autoimmune) pfizer.com

polyclonal antibody Cancer Advances polyclonal gastrointestinal reflux Phase II

stimulator Durham, NC antibody disease (GERD) canceradvancesinc.com
vaccine (see also cancer)

Prochymal® Osiris Therapeutics stem cell Crohn’s disease (Fast Track), Phase III
remestemcel-L Columbia, MD therapy gastrointestinal injury from osiris.com
radiation exposure
(see also cardiovascular, diabetes,
respiratory, transplantation)

Medicines in Development Biologics 2013 47

 Biologic Medicines in Development

Digestive Disorders
Product Name Sponsor Product Indication Development Phase
Category

QAX576 Novartis Pharmaceuticals mAb rectal fistula in patients with Crohn’s Phase II
East Hanover, NJ disease novartis.com
(see also respiratory)

Remicade® Janssen Biotech mAb Crohn’s disease (prevention Phase III

infliximab Horsham, PA of relapse after surgery resection) janssenbiotech.com

SAR252067 Sanofi US mAb Crohn’s disease, ulcerative colitis Phase I

(anti-LIGHT mAb) Bridgewater, NJ sanofi.com

SAR339658 Sanofi US mAb inflammatory bowel disease Phase II

(VLA2 antagonist) Bridgewater, NJ sanofi.com

Simponi® Janssen Biotech mAb ulcerative colitis application submitted

golimumab Horsham, PA (see also autoimmune) janssenbiotech.com
(subcutaneous)

Stelara® Janssen Biotech mAb Crohn’s disease Phase II/III

ustekinumab Horsham, PA (see also autoimmune, janssenbiotech.com
musculoskeletal, other)

tralokinumab AstraZeneca mAb ulcerative colitis Phase II

Wilmington, DE (see also respiratory) astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD

vedolizumab Takeda Pharmaceuticals mAb Crohn’s disease, ulcerative colitis Phase III
(MLN0002) Deerfield, IL takeda.com

Eye Conditions
Product Name Sponsor Product Indication Development Phase
Category

anti-factor D Genentech mAb geographic atrophy associated with Phase II

(RG7417) South San Francisco, CA age-related macular gene.com
degeneration

anti-LINGO Biogen Idec mAb optic neuritis Phase I

(BIIB033) Weston, MA (see also autoimmune) biogenidec.com

AVA-101 Avalanche Biotechnologies gene therapy age-related macular degeneration Phase I/II
San Francisco, CA avalanchebiotech.com

eye disorder MorphoSys mAb eye disorders Phase I

therapeutic Martinsried, Germany novartis.com
Novartis Pharmaceuticals
East Hanover, NJ

48 Medicines in Development Biologics 2013

Biologic Medicines in Development

Eye Conditions
Product Name Sponsor Product Indication Development Phase
Category

Eylea® Bayer HealthCare recombinant diabetic macular edema, Phase III

aflibercept Pharmaceuticals fusion protein macular edema following branch bayerpharma.com
Wayne, NJ retinal vein occlusion regeneron.com
Regeneron Pharmaceuticals
Tarrytown, NY

gevokizumab XOMA mAb intermediate or posterior uveitis Phase III

(IL-1B inhibitor mAb) Berkeley, CA (see also diabetes, musculoskeletal, xoma.com
(Orphan Drug) skin)

GSK933776A GlaxoSmithKline mAb age-related macular degeneration Phase II

(anti-B amyloid mAb) Rsch. Triangle Park, NC (see also neurologic) gsk.com

GZ402663 Applied Genetic Technologies gene wet (neovascular) age-related Phase I

(FLT-01) Alachua, FL therapy macular degeneration agtc.com
anti-vascular Genzyme genzyme.com
endothelial growth-factor Cambridge, MA
gene therapy

hl-con1 Iconic Therapeutics recombinant age-related macular Phase I

Atlanta, GA protein degeneration iconictherapeutics.
com

HuCNS-SC® StemCells stem cell age-related macular degeneration Phase I/II

adult neural stem Newark, CA therapy (see also genetic, neurologic) stemcellsinc.com
cell therapy
(intraocular)

human retinal Advanced Cell Technology stem cell dry age-related macular Phase I/II
pigment epithelial Santa Monica, CA therapy degeneration, Stargardt’s advancedcell.com
cell therapy macular degeneration
(Orphan Drug)

Humira® AbbVie mAb uveitis Phase III

adalimumab North Chicago, IL (see also autoimmune, digestive, abbvie.com
musculoskeletal, skin, other)

iSONEP™ Lpath mAb wet age-related macular Phase II

sonepcizumab San Diego, CA degeneration lpath.com
(intravitreous)

iCo-007 iCo Therapeutics antisense diabetic macular edema Phase II

Vancouver, Canada icotherapeutics.com

Medicines in Development Biologics 2013 49

 Biologic Medicines in Development

Eye Conditions
Product Name Sponsor Product Indication Development Phase
Category

Lucentis® Genentech mAb age-related macular degeneration application submitted

ranibizumab South San Francisco, CA (0.5 mg PRN) gene.com
Roche roche.com
Nutley, NJ ------------------------------------------------ ---------------------------------
age-related macular degeneration Phase I
(sustained-delivery), diabetic gene.com
macular edema (sustained- roche.com
delivery), retinal vein occlusion

Mitosol® Mobius Therapeutics recombinant prevention of pterygium recurrence application submitted

mitomycin St. Louis, MO protein after excision surgery mobiustherapeutics.com
ophthalmic

NT 501 Neurotech Pharmaceuticals cell therapy retinitis pigmentosa Phase II/III

(ciliary neurotrophic Cumberland, RI (Fast Track) neurotechusa.com
factor) ------------------------------------------------ ---------------------------------
(Orphan Drug) age-related macular Phase II
degeneration neurotechusa.com
(Fast Track)

PF-655 Quark Pharmaceuticals RNA wet age-related macular Phase II

(RTP801i14) Fremont, CA interference degeneration, diabetic macular quarkpharma.com
edema

PF-04382923 Pfizer antibody age-related macular degeneration Phase II

(RN6G) New York, NY pfizer.com

QPI-1007 Quark Pharmaceuticals RNA optic neuropathy Phase I

Fremont, CA interference quarkpharma.com

RetinoStat® Oxford BioMedica gene wet age-related macular Phase I

angiostatin endostatin Oxford, United Kingdom therapy degeneration sanofi.com
gene therapy Sanofi US
Bridgewater, NJ

RU-101 R-Tech Ueno recombinant dry eyes Phase I

(recombinant human Tokyo, Japan protein rtechueno.com
serum albumin)

secukinumab Alcon Labs mAb uveitis Phase III

(AIN457) Fort Worth, TX (see also autoimmune, alcon.com
Novartis Pharmaceuticals musculoskeletal, skin) novartis.com
East Hanover, NJ

Soliris® Alexion Pharmaceuticals mAb severe or refractory neuromyelitis Phase II

eculizumab Cheshire, CT optica alxn.com
(see also autoimmune, infectious,
transplantation)

50 Medicines in Development Biologics 2013

Biologic Medicines in Development

Eye Conditions
Product Name Sponsor Product Indication Development Phase
Category

StarGen® Sanofi US gene Stargardt’s disease Phase I/II

Stargardt’s disease Bridgewater, NJ therapy (juvenile-onset macular dystrophy) sanofi.com
gene therapy
(Orphan Drug)

Genetic Disorders
Product Name Sponsor Product Indication Development Phase
Category

adrenoleucodystrophy bluebird bio gene adrenoleucodystrophy Phase I/II

gene therapy Cambridge, MA therapy bluebirdbio.com
INSERM
Paris, France

AGTC-0106 Applied Genetic Technologies gene therapy alpha-1-antitrypsin deficiency Phase II

adeno-associated virus Alachua, FL agtc.com
vector-medictoten
(Orphan Drug)

ALD-601 Cytomedix stem cell infantile-onset lysosomal storage Phase I

Gaithersburg, MD therapy disease cytomedix.com

ALN-TTR02 Alnylam Pharmaceuticals RNA TTR-mediated amyloidosis Phase II

Cambridge, MA interference alnylam.com

asfotase alfa Alexion Pharmaceuticals recombinant hypophosphatasia in infants Phase II/III

(Orphan Drug) Cheshire, CT fusion and children alxn.com
protein (Fast Track)
------------------------------------------------ --------------------------------
hypophosphatasia in adults and Phase II
adolescents alxn.com

AVI-4658 Sarepta Therapeutics antisense Duchenne muscular dystrophy Phase II

(eteplirsen) Cambridge, MA sareptatherapeutics.com

Biostrophin™ Asklêpios BioPharmaceuticals gene therapy Duchenne muscular dystrophy Phase I

muscular dystrophy Chapel Hill, NC askbio.com
gene therapy

BMN-110 BioMarin Pharmaceutical recombinant mucopolysaccharidosis IV Phase III

(Orphan Drug) Novato, CA enzyme (Morquio A syndrome) bmrn.com

Medicines in Development Biologics 2013 51

 Biologic Medicines in Development

Genetic Disorders
Product Name Sponsor Product Indication Development Phase
Category

BMN-701 BioMarin Pharmaceutical recombinant glycogen storage disease type II Phase II

(Orphan Drug) Novato, CA fusion (Pompe disease) bmrn.com
protein

CEQ508 Marina Biotech RNA familial adenomatous Phase I/II

(Orphan Drug) Bothell, WA interference polyposis marinabio.com

GNE lipoplex Gradalis gene therapy muscular dystrophy Phase I

(muscular dystrophy Carrollton, TX gradalisinc.com
gene therapy)

GSK2402968 GlaxoSmithKline antisense Duchenne muscular dystrophy Phase II

(PRO-051) Rsch. Triangle Park, NC gsk.com
(Orphan Drug) Prosensa prosensa.eu
Leiden, The Netherlands

GSK2696273 GlaxoSmithKline stem cell adenosine deaminase severe Phase III

Rsch. Triangle Park, NC therapy combined immune deficiency gsk.com

GZ402665 Genzyme recombinant Niemann-Pick type B Phase I

(rhASM) Cambridge, MA enzyme genzyme.com
(Orphan Drug)

HGT2310 Shire enzyme Hunter syndrome Phase I/II

(Orphan Drug) Dublin, Ireland replacement (mucopolysaccharidosis II) shire.com
therapy

HuCNS-SC® StemCells stem cell Pelizaeus-Merzbacher disease Phase I

adult neural stem Newark, CA therapy (see also eye, neurologic) stemcellsinc.com
cell therapy
(intracerebral)

ISIS-SMNRx Biogen Idec antisense spinal muscular atrophy Phase II

(Orphan Drug) Weston, MA biogenidec.com
Isis Pharmaceuticals isispharm.com
Carlsbad, CA

ISIS-TTRRx GlaxoSmithKline antisense familial amyloid polyneuropathy Phase III

(Orphan Drug) Rsch. Triangle Park, NC gsk.com
Isis Pharmaceuticals isispharm.com
Carlsbad, CA

Kineret® Sobi recombinant cryopyrin-associated application submitted

anakinra Ardmore, PA protein periodic syndrome sobi.com
(Orphan Drug)

KRN-23 Kyowa Hakko Kirin Pharma mAb X-linked dominant Phase I/II
Princeton, NJ hypophosphatemic rickets kyowa-kirin-pharma.com

52 Medicines in Development Biologics 2013

Biologic Medicines in Development

Genetic Disorders
Product Name Sponsor Product Indication Development Phase
Category

Kynamro™ Genzyme antisense severe heterozygous familial Phase III

mipomersen Cambridge, MA hypercholesterolemia genzyme.com
(Orphan Drug) Isis Pharmaceuticals isispharm.com
Carlsbad, CA

leber congenital Applied Genetic Technologies gene leber congenital amaurosis Phase I/II
amaurosis gene Alachua, FL therapy agtc.com
therapy
(Orphan Drug)

NAV™ therapeutic ReGenX Biosciences gene therapy Batten disease Phase II

Washington, DC (see also blood) regenxbio.com

PEG-PAL BioMarin Pharmaceutical recombinant phenylketonuria Phase II

(Orphan Drug) Novato, CA enzyme bmrn.com

PF-06252616 Pfizer biologic muscular dystrophy Phase I

New York, NY pfizer.com

PRX-102 Protalix Biotherapeutics recombinant Fabry’s disease Phase I/II

(alpha-galactosidase) Carmiel, Israel protein protalix.com

recombinant human C1 Pharming Group recombinant hereditary angioedema Phase III

esterase inhibitor Leiden, The Netherlands protein (Fast Track) santarus.com
(Orphan Drug) Santarus
San Diego, CA

sebelipase alfa Synageva BioPharma recombinant Wolman disease Phase II/III

(Orphan Drug) Lexington, MA protein (lysosomal acid lipase deficiency) synageva.com
(Fast Track)

UshStat® Sanofi US gene therapy usher syndrome 1B Phase I/II

usher syndrome gene Bridgewater, NJ sanofi.com
therapy
(Orphan Drug)

WAS gene therapy Children’s Hospital Boston gene therapy Wiskott-Aldrich syndrome Phase I/II
Boston, MA genethon.fr
Genethon
Évry, France

Medicines in Development Biologics 2013 53

 Biologic Medicines in Development

Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category

ACAM-Cdiff Sanofi Pasteur vaccine prevention and treatment of Phase II

(Clostridium difficile Swiftwater, PA Clostridium difficile infections sanofipasteur.us
toxoid vaccine) (Fast Track)

ACE527 TD Vaccines vaccine prevention of enterotoxigenic Phase II

Skorping, Denmark Escherichia coli (ETEC) infections tdvaccines.com

acellular pertussis combo Novartis Vaccines vaccine diphtheria, pertussis, tetanus Phase I
vaccine Cambridge, MA novartisvaccines.com

ADVAX Aaron Diamond AIDS DNA HIV infection prevention Phase I completed
(DNA vaccine) Research Center vaccine adarc.org
New York, NY iavi.org
International AIDS Vaccine ------------------------------------------------ ---------------------------------
Initiative HIV infection prevention Phase I completed
New York, NY (new delivery system) adarc.org
Ichor Medical Systems iavi.org
San Diego, CA

AERAS-402/Ad35 Aeras recombinant tuberculosis prevention Phase I

Rockville, MD vaccine aeras.org
Crucell crucell.com
Leiden, The Netherlands

Agriflu® Novartis Vaccines vaccine influenza virus infections (pediatric) Phase III
influenza virus vaccine Cambridge, MA ention) novartisvaccines.com

AGS-004 Argos Therapeutics personalized HIV infection Phase II

Durham, NC dendritic cell argostherapeutics.com
vaccine

Alferon LDO® Hemispherx Bioscience interferon human papillomavirus Phase I/II

interferon-alpha-n3 Philadelphia, PA infections hemispherx.net
oral ------------------------------------------------ ---------------------------------
influenza Phase I
hemisphrex.net

ALN-RSV01 Alnylam Pharmaceuticals RNA respiratory syncytial virus (RSV) Phase II

Cambridge, MA interference treatment alnylam.com

Anthim™ Elusys Therapeutics mAb anthrax Phase I

(ETI-204) Pine Brook, NJ (Fast Track) elusys.com
(Orphan Drug)

anthrax immune Cangene mAb anthrax Phase III

globulin Berwyn, PA cangene.com

anti-PD-L1 Bristol-Myers Squibb mAb hepatitis B Phase I

Princeton, NJ (see also cancer) bms.com

54 Medicines in Development Biologics 2013

Biologic Medicines in Development

Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category

ASP-0113 AbbVie DNA prevention of cytomegalovirus Phase III

(cytomegalovirus DNA North Chicago, IL vaccine (CMV) infections in stem cell us.astellas.com
vaccine) Astellas Pharma US transplantation
(Orphan Drug) Northbrook, IL ------------------------------------------------ ---------------------------------
Vical prevention of CMV infections in solid Phase II
San Diego, CA organ transplantation us.astellas.com

AVI-7100 Sarepta Therapeutics antisense influenza A virus infections Phase I

radavirsen Cambridge, MA sareptatherapeutics.com

AVI-7288 Sarepta Therapeutics antisense Marburg virus Phase I

(Orphan Drug) Cambridge, MA disease sareptatherapeutics.com

AVI-7537 Sarepta Therapeutics antisense post-exposure treatment of Phase I

Cambridge, MA Ebola virus infections sareptatherapeutics.com

AVX101 AlphaVax gene HIV-1 infection prevention Phase I

(single gene HIV vaccine) Rsch. Triangle Park, NC vaccine alphavax.com

AVX601 AlphaVax genetically- prevention of CMV infections Phase I

Rsch. Triangle Park, NC modified alphavax.com
Novartis Vaccines vaccine novartisvaccines.com
Cambridge, MA

AZD9773 AstraZeneca polyclonal severe sepsis Phase II

(anti-TNF-alpha poly- Wilmington, DE antibody astrazeneca.com
clonal antibody)

Bexsero® Novartis Vaccines recombinant meningococcal group B infections Phase II

meningococcal vaccine Cambridge, MA vaccine novartisvaccines.com
group B

BioThrax® Emergent BioSolutions vaccine prevention of post-exposure Phase II

anthrax vaccine Rockville, MD anthrax infection in combination emergentbiosolutions.com
with other antibacterials
(Fast Track)

botulinum toxin vaccine DynPort Vaccine recombinant botulism Phase II completed

Frederick, MD vaccine csc.com/dvc

cell therapy Cell Medica cell viral infections Phase I/II

London, United Kingdom therapy (see also cancer) cellmedica.co.uk
Center for Cell and Gene Therapy
Houston, TX

chikungunya virus Inviragen vaccine chikungunya virus infections Phase II

vaccine Fort Collins, Co inviragen.com

Medicines in Development Biologics 2013 55

 Biologic Medicines in Development

Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category

cholera vaccine PaxVax live, prevention of cholera infection Phase I

Menlo Park, CA attenuated paxvax.com
vaccine

Chronvac-C® Inovio Pharmaceuticals gene therapy treatment of hepatitis C Phase II

hepatitis C DNA Blue Bell, PA vaccine inovio.com
vaccine ChronTech Pharma
Huddinge, Sweden

CYT107 Cytheris recombinant HIV infection treatment Phase II

(interleukin-7) Rockville, MD interleukin (see also blood, cancer, cytheris.com
transplantation)

Cytolin® CytoDyn mAb HIV infection treatment Phase I

anti-CD8 mAb Lake Oswego, OR cytodyn.com

DCVax-001 Celldex recombinant HIV infection prevention and Phase I

Needham, MA vaccine treatment celldextherapeutics.com
Rockefeller University
New York, NY

dengue vaccine GlaxoSmithKline vaccine dengue Phase I

Rsch. Triangle Park, NC gsk.com

dengue vaccine Sanofi Pasteur vaccine mild to severe dengue fever Phase III
Swiftwater, PA sanofipasteur.us

DENVax™ Inviragen vaccine dengue Phase I

tetravalent hybrid dengue Fort Collins, CO inviragen.com
virus vaccine

DermaVir™ Patch Genetic Immunity DNA HIV-1 infection treatment Phase II

DNA topical patch vaccine McLean, VA vaccine geneticimmunity.com

DNA vaccine Naval Medical DNA prevention of dengue fever Phase I

(dengue) Research Center vaccine vical.com
Silver Spring, MD
Vical
San Diego, CA

DTP-HepB-polio-Hib Merck vaccine diphtheria, tetanus, pertussis, Phase III

(pediatric hexavalent Whitehouse Station, NJ hepatitis B, poliomyelitis, merck.com
vaccine) Sanofi Pasteur Haemophilus influenzae type b sanofipasteur.us
(V419) Swiftwater, PA

Ebola vaccine Crucell DNA prevention of Ebola virus infections Phase I

Leiden, The Netherlands vaccine crucell.com

56 Medicines in Development Biologics 2013

Biologic Medicines in Development

Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category

Ebola virus vaccine GenPhar vaccine Ebola virus infections in clinical trials
Mt. Pleasant, SC genphar.com

enterovirus 71 vaccine Inviragen vaccine prevention of head, foot Phase I

(INV21) Fort Collins, CO and mouth disease inviragen.com

EP1300 VaxOnco DNA prevention of malaria infection Phase I

(malaria DNA Seoul, South Korea vaccine
vaccine) National Institute of Allergy
and Infectious Diseases
Bethesda, MD

FGI-101-1A6 Functional Genetics mAb influenza Phase I completed

Gaithersburg, MD functional-genetics.com

Fluad® Novartis Vaccines vaccine influenza virus infections Phase III

influenza virus vaccine Cambridge, MA in the elderly and children novartisvaccines.com

FluBIok® Protein Sciences recombinant prevention of influenza virus Phase I/II

derived from recombinant Meriden, CT vaccine infections in infants and children proteinsciences.com
hemagglutinin (rHA)

Fludase® NexBio recombinant influenza A virus H1N1 infections, Phase II completed

DAS-181 inhalation San Diego, CA fusion protein prevention of influenza virus nexbio.com
infections

FluLaval® QIV GlaxoSmithKline vaccine prevention of influenza application submitted

influenza virus vaccine Rsch. Triangle Park, NC infections gsk.com

FluNhance® Protein Sciences recombinant influenza virus infections Phase III

influenza virus vaccine Meriden, CT vaccine proteinsciences.com
(rNA)

Fluzone® QIV IM Sanofi Pasteur vaccine seasonal influenza virus infections application submitted
quadrivalent influenza Swiftwater, PA sanofipasteur.us
virus vaccine

foravirumab Crucell mAb post-exposure prevention of rabies Phase II

Leiden, The Netherlands crucell.com

GI-5005 GlobeImmune genetically- hepatitis C Phase II

Louisville, CO modified globeimmune.com
vaccine

Medicines in Development Biologics 2013 57

 Biologic Medicines in Development

Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category

GSK692342 Aeras recombinant prevention of tuberculosis Phase II

(Mtb72F/AS02A) Rockville, MD vaccine gsk.com
GlaxoSmithKline
Rsch. Triangle Park, NC

GSK1437173A GlaxoSmithKline recombinant prevention of varicella zoster Phase III

(varicella zoster vaccine) Rsch. Triangle Park, NC vaccine virus infections gsk.com

GSK1492903A GlaxoSmithKline recombinant prevention of CMV infections Phase I

(cytomegalovirus Rsch. Triangle Park, NC vaccine gsk.com
glycoprotein vaccine)

GSK2189242A GlaxoSmithKline recombinant prevention of pneumococcal Phase II

(Streptococcus Rsch. Triangle Park, NC vaccine infections gsk.com
pneumoniae recombinant-
conjugated vaccine)

GSK2654909A GlaxoSmithKline vaccine prevention of influenza Phase I

Rsch. Triangle Park, NC A virus H9N2 gsk.com
subtype (avian flu)

GSK2654911A GlaxoSmithKline vaccine prevention of influenza Phase I

Rsch. Triangle Park, NC A virus H9N2 gsk.com
subtype (avian flu)

GSK2830930A GlaxoSmithKline vaccine prevention of pneumococcal Phase I

Rsch. Triangle Park, NC infections gsk.com

GSK2838497A GlaxoSmithKline vaccine prevention of Haemophilus Phase I

Rsch. Triangle Park, NC infections gsk.com

HBV-002 Hawaii Biotech recombinant prevention of West Nile virus Phase I completed
Aiea, Hawaii vaccine infections hibiotech.com

hepatitis C immune Biotest Pharmaceuticals polyclonal hepatitis C prevention Phase II

globulin Boca Raton, FL antibody biotestpharma.com
(Orphan Drug)

hepatitis C vaccine Okairos gene vaccine prevention of hepatitis C Phase I/II

therapy Basel, Switzerland okairos.com

Heplisav™ Dynavax Technologies DNA prevention of hepatitis B application submitted

hepatitis B vaccine Berkeley, CA vaccine dynavax.com

HerpV Agenus recombinant treatment of herpes simplex virus Phase II

(herpes vaccine) Lexington, MA protein infections agenusbio.com
vaccine

Hexaxim™ Sanofi Pasteur vaccine diphtheria, tetanus, pertussis, hepa- application submitted
DTP-HepB-polio-Hib Swiftwater, PA titis B, poliomyelitis, sanofipasteur.us
vaccine Haemophilus influenzae type b

58 Medicines in Development Biologics 2013

Biologic Medicines in Development

Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category

HIV gene therapy Adaptimmune gene therapy HIV infection Phase I

Philadelphia, PA adaptimmune.com
Cardiff University
Cardiff, Wales
University of Pennsylvania
Philadelphia, PA

HIV recombinant vaccine GlaxoSmithKline recombinant HIV infection prevention Phase I

Rsch. Triangle Park, NC vaccine gsk.com

HIV recombinant vaccine GlaxoSmithKline recombinant HIV infection treatment Phase II

Rsch. Triangle Park, NC vaccine gsk.com

HIV vaccine Crucell recombinant HIV infection prevention Phase I

Leiden, The Netherlands vaccine crucell.com
Beth Israel Deaconess
Medical Center
Boston, MA
International AIDS Vaccine
Initiative
New York, NY

HIV vaccine GeoVax vaccine HIV infection prevention Phase II

Smyrna, GA geovax.com

HIV vaccine GeoVax vaccine HIV infection treatment Phase I/II

Smyrna, GA geovax.com

HIV vaccine Massachusetts General Hospital vaccine HIV infection Phase I

Boston, MA
Opal Therapeutics
Parkvill, Australia

HIV vaccine Novartis Vaccines vaccine HIV infection Phase I

Cambridge, MA novartisvaccines.com

HIV vaccine PaxVax oral vaccine HIV infection prevention in clinical trials
San Diego, CA paxvax.com

HIV vaccine Profectus Biosciences DNA HIV infection prevention Phase I

(MAG pDNA) Baltimore, MD vaccine profectusbiosciences.com

HIV vaccine Profectus Biosciences recombinant HIV-1 infection prevention Phase I

(rVSV) Baltimore, MD vaccine profectusbiosciences.com

HIV-1 gene therapy Takara Bio gene therapy HIV-1 infection Phase I
(MazF) Shiga, Japan takara-bio.com

Medicines in Development Biologics 2013 59

 Biologic Medicines in Development

Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category

HIV-1 vaccine Sumagen vaccine HIV-1 infection Phase I

(SAV001) Seoul, South Korea sumagen.co.kr

HIVAX™ GeneCure Biotechnologies vaccine HIV-1 infection Phase I

replication-defective Norcross, GA genecure.com
HIV-1 vaccine

HSV-2 Tx Genocea Biosciences T-cell vaccine treatment of herpes simplex virus 2 Phase I/II
Cambridge, MA infections genocea.com

human interferon Amarillo Biosciences interferon hepatitis C, influenza Phase II

alpha oral Amarillo, TX amarbio.com
(Orphan Drug)

ibalizumab TaiMed Biologics USA mAb HIV-1 infection Phase II

(TMB-355) Irvine, CA (intravenous) taimedbiologics.com
(Fast Track)
------------------------------------------------ ---------------------------------
HIV-1 infection Phase I
(subcutaneous) taimedbiologics.com

ID-93/GLA-SE Aeras Global TB Vaccine recombinant tuberculosis Phase I

Foundation fusion aeras.org
Rockville, MD protein idri.org
Infectious Disease Research vaccine
Institute
Seattle, WA

Imvamune® Bavarian Nordic vaccine smallpox Phase II

smallpox vaccine Kvistgaard, Denmark bavarian-nordic.com

influenza A virus H1N1 CEL-SCI peptide prevention of influenza A virus Phase I

vaccine Vienna, VA vaccine H1N1 subtype cel-sci.com

influenza A virus H1N1 iBio vaccine prevention of influenza A virus Phase I

vaccine Newark, DE H1N1 subtype ibioinc.com

influenza A virus H1N1 Medicago VLP vaccine influenza A virus H1N1 subtype Phase I
vaccine Quebec, Canada medicago.com

influenza A virus H1N1 Inovio Pharmaceuticals DNA vaccine influenza A virus H1N1 subtype Phase I
vaccine (FVH-1) Blue Bell, PA prevention inovio.com

influenza A virus H1N1 Antigen Express peptide influenza A virus H1N1 infections Phase I
vaccine (li-key hybrid Worcester, MA vaccine antigenexpress.com
peptide vaccine)

60 Medicines in Development Biologics 2013

Biologic Medicines in Development

Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category

influenza A virus H5N1 GlaxoSmithKline cell-cultured prevention of pandemic influenza Phase I

cell-cultured based Rsch. Triangle Park, NC vaccine A virus H5N1 subtype infections gsk.com
vaccine

influenza A virus H5N1 Nanotherapeutics vaccine prevention of influenza A virus H5N1 Phase I
intranasal vaccine Alachua, FL subtype nanotherapeutics.com

influenza A virus H5N1 iBio vaccine influenza A virus H5N1 subtype Phase I
vaccine Newark, DE ibioinc.com

influenza A virus H5N1 Inovio Pharmaceuticals vaccine prevention of influenza A virus H5N1 Phase I
vaccine Blue Bell, PA subtype inovio.com

influenza A virus H5N1 Medicago VLP vaccine influenza A virus H5N1 subtype Phase I
vaccine Quebec, Canada medicago.com

influenza A virus H5N1 Novartis Vaccines vaccine prevention of influenza A virus Phase II
vaccine Cambridge, MA H5N1 subtype novartisvaccines.com

influenza A virus H5N1 Novavax vaccine prevention of influenza A virus Phase II

vaccine Rockville, MD H5N1 subtype novavax.com

influenza A virus H5N1 PaxVax vaccine prevention of influenza A virus Phase I

vaccine San Diego, CA H5N1 subtype paxvax.com

influenza A virus H5N1 Vaxart vaccine prevention of influenza A virus Phase I

vaccine San Francisco, CA H5N1 subtype vaxart.com

influenza A virus H5N1 Antigen Express peptide influenza A virus H5N1 subtype Phase I
vaccine Worcester, MA vaccine antigenexpress.com
(li-key hybrid peptide
vaccine)

influenza A virus H9N2 Baxter International vaccine influenza A virus H9N2 subtype Phase I/II
vaccine Deerfield, IL baxter.com

influenza virus-like Novavax vaccine prevention of influenza virus Phase II

particle vaccine Rockville, MD infections novavax.com

influenza virus vaccine Crucell vaccine prevention of influenza virus Phase II

(intradermal) Leiden, The Netherlands infections crucell.com

Influvac® TC AbbVie cell-cultured influenza virus infections Phase I

cell culture-derived North Chicago, IL vaccine (prevention) abbvie.com
influenza vaccine

Medicines in Development Biologics 2013 61

 Biologic Medicines in Development

Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category

interferon-alpha-2b HanAll Biopharma interferon hepatitis C Phase II

Seoul, South Korea hanallbiopharma.com

interferon alpha-2b Medtronic interferon hepatitis C Phase II completed

infusion Minneapolis, MN medtronic.com

Ixiaro® Intercell vaccine prevention of Japanese encephalitis application submitted

Japanese encephalitis Vienna, Austria in children intercell.com
vaccine Novartis Vaccines novartisvaccines.com
Cambridge, MA

KB001-A KaloBios Pharmaceuticals mAb Pseudomonas infections in cystic Phase I/II

(antibody-fragment South San Francisco, CA fibrosis patients (KalBios only) kalobios.com
product) Sanofi Pasteur ------------------------------------------------ ---------------------------------
Swiftwater, PA prevention of ventilator-associated Phase I
pneumonia kalobios.com
sanofipasteur.us

KD-247 Kaketsuken mAb HIV-1 infection Phase I

Kumamoto, Japan kaketsuken.or.jp

LIQ-001 Liquidia Technologies vaccine influenza virus infections Phase I

Rsch. Triangle Park, NC liquidia.com

Lyme disease vaccine Baxter Healthcare recombinant prevention of Lyme disease Phase I/II
Deerfield, IL vaccine baxter.com

malaria vaccine Crucell recombinant malaria Phase I/II

Leiden, The Netherlands vaccine crucell.com
GlaxoSmithKline gsk.com
Rsch. Triangle Park, NC

malaria vaccine GenVec recombinant malaria Phase I/II

Gaithersburg, MD vaccine genvec.com
U.S. Naval Medical Research
Center
Silver Spring, MD

malaria vaccine Sanaria vaccine prevention of malaria Phase I/II

Rockville, MD sanaria.com

Marburg virus GenPhar vaccine Marburg virus disease Phase I

DNA vaccine Mt. Pleasant, SC genphar.com

MBL-HCV1 MassBiologics mAb hepatitis C Phase II

Boston, MA umassmed.edu/
massbiologics

62 Medicines in Development Biologics 2013

Biologic Medicines in Development

Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category

MEDI-550 AstraZeneca vaccine pandemic influenza prevention Phase I

(pandemic influenza virus Wilmington, DE astrazeneca.com
vaccine) MedImmune medimmune.com
Gaithersburg, MD

MEDI-557 AstraZeneca mAb prevention of RSV infections in Phase I

(RSV mAb- Wilmington, DE high risk adults astrazeneca.com
extended half-life) MedImmune medimmune.com
Gaithersburg, MD

MEDI-559 AstraZeneca vaccine prevention of RSV infections Phase I/II

(RSV vaccine) Wilmington, DE (pediatric) astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD

MenABCWY Novartis Vaccines vaccine meningococcal infections Phase II

(meningococcal vaccine Cambridge, MA novartisvaccines.com
groups A,C,Y,W-135
conjugate)

meninge ACYW conjugate Sanofi Pasteur vaccine meningococcal infections Phase II

vaccine Swiftwater, PA sanofipasteur.us
(2nd-generation
meningococcal conjugate
infant vaccine)

meningococcal vaccine GlaxoSmithKline vaccine meningococcal infections Phase III

groups A,C,Y,W-135 Rsch. Triangle Park, NC gsk.com
conjugate

meningococcal vaccine JN-International Medical vaccine meningococcal group A,C,Y,W-135 Phase I

groups A,C,Y,W-135 Omaha, NE infections jn-vaccines.org
conjugate

Menveo® Novartis Vaccines vaccine meningococcal groups A,C,Y,W-35 application submitted

meningococcal vaccine Cambridge, MA infection in infants (ages 2–12 novartisvaccines.com
groups A,C,Y, W-135 months)
conjugate vaccine

miravirsen Santaris Pharma A/S USA antisense hepatitis C Phase II

(SPC3649) San Diego, CA santaris.com

MK-3415A Merck mAb Clostridium difficile infections Phase III

(actoxumab/ Whitehouse Station, NJ merck.com
bezlotoxumab)

MMR vaccine live GlaxoSmithKline vaccine prevention of measles, mumps, Phase III
(GSK209762) Rsch. Triangle Park, NC rubella gsk.com

MnB rLP2086 Pfizer recombinant adolescent and young adult Phase III
(PF-05212366) New York, NY vaccine meningitis B pfizer.com

Medicines in Development Biologics 2013 63

 Biologic Medicines in Development

Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category

MVA-BN® Bavarian Nordic vaccine HIV-1 infection prevention and Phase I/II
HIV multiantigen vaccine Washington, DC treatment bavarian-nordic.com

NB-001 NanoBio nano- recurrent herpes simplex virus Phase III

Ann Arbor, MI emulsion infections nanobio.com

NB-002 NanoBio nano- distal subungual onychomycosis Phase II

Ann Arbor, MI emulsion of the toe nanobio.com

NB-1008 NanoBio nano- prevention of influenza virus Phase I completed

(intranasal) Ann Arbor, MI emulsion infections nanobio.com
vaccine

NDV-3 NovaDigm Therapeutics recombinant prevention of candidiasis, Phase I

Grand Forks, ND vaccine prevention of methicillin-resistant novadigm.net
Staphylococcus aureus (MRSA)
infections

norovirus virus-like LigoCyte Pharmaceuticals vaccine norovirus infections Phase I/II

particle vaccine Bozeman, MT ligocyte.com
intranasal

Nuthrax™ Emergent BioSolutions vaccine anthrax Phase I

anthrax vaccine adsorbed Rockville, MD (Fast Track) emergentbiosolutions.com
with CPG 7909 adjuvant

PanBlok™ Protein Sciences vaccine prevention of influenza A virus Phase I/II

influenza A virus H5N1 Meriden, CT H5N1 subtype infections proteinsciences.com
vaccine

peginterferon lambda-1a Bristol-Myers Squibb interferon hepatitis C Phase III

Princeton, NJ bms.com
------------------------------------------------ ---------------------------------
hepatitis B Phase II
bms.com

Pennvax®-B Inovio Pharmaceuticals DNA HIV infection prevention and Phase I

DNA vaccine Blue Bell, PA vaccine treatment inovio.com
(clade B)

Pennvax®-G Inovio Pharmaceuticals DNA HIV infection prevention Phase I

DNA vaccine Blue Bell, PA vaccine inovio.com
(clades A, C, D)

PF-06290510 Pfizer vaccine Staphylococcus aureus infections Phase II

(SA4Ag) New York, NY pfizer.com

64 Medicines in Development Biologics 2013

Biologic Medicines in Development

Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category

PF-06425090 Pfizer vaccine Clostridium difficile colitis Phase I

(Clostridium difficile New York, NY pfizer.com
vaccine)

plague injectable vaccine DynPort Vaccine vaccine Yersinia infections Phase II

Frederick, MD csc.com/dvc

Preflucel™ Baxter International vaccine prevention of influenza virus Phase III

seasonal influenza virus Deerfield, IL infections baxter.com
vaccine DynPort Vaccine csc.com/dvc
Frederick, MD

Prepandrix™ GlaxoSmithKline vaccine prevention of influenza A virus H5N1 application submitted

H5N1 (pre-)pandemic Rsch. Triangle Park, NC subtype for pandemic use gsk.com
influenza virus vaccine ------------------------------------------------ ---------------------------------
prevention of influenza A virus Phase III
H5N1 subtype for pandemic use in gsk.com
children and adolescents

PreviThrax™ Emergent Biosolutions recombinant anthrax Phase II

recombinant protective Rockville, MD vaccine (Fast Track) emergentbiosolutions.com
antigen anthrax vaccine

PRO 140 CytoDyn mAb HIV infection Phase II completed

Lake Oswego, OR cytodyn.com

Quadracel® Sanofi Pasteur vaccine diphtheria, tetanus, pertussis, Phase III

DTP, polio vaccine Swiftwater, PA poliomyelitis (4-6 years of age) sanofipasteur.us

rabies VRVg vaccine Sanofi Pasteur vaccine prevention of rabies Phase II

(purified vero rabies Swiftwater, PA sanofipasteur.us
vaccine)

respiratory syncytial virus Novavax recombinant prevention of RSV infections Phase II

VLP vaccine Rockville, MD vaccine novavax.com

Rivax™ Soligenix vaccine ricin poisoning Phase I

ricin vaccine Princeton, NJ (pre-exposure) soligenix.com
(Orphan Drug)

rotavirus vaccine Sanofi Pasteur vaccine rotavirus Phase I

(live attenuated tetrava- Swiftwater, PA sanofipasteur.us
lent oral vaccine)

SAR279356 Sanofi US mAb prevention of bacterial infections Phase II

(anti-PNAG mAB) Bridgewater, NJ sanofi.com

Medicines in Development Biologics 2013 65

 Biologic Medicines in Development

Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category

SB-728-T Sangamo BioSciences gene HIV infection Phase II

Richmond, CA therapy sangamo.com

Soliris® Alexion Pharmaceuticals mAb Shiga toxin E. coli-related hemolytic Phase II

eculizumab Cheshire, CT uremic syndrome (STEC-HUS) alxn.com
(Orphan Drug) (see also autoimmune, eye,
transplantation)

Stealth Vector® Enzo Therapeutics antisense HIV-1 infection treatment Phase I/II
HGTV-43™ New York, NY enzo.com
antisense gene medicine

streptococcal B vaccine Novartis Vaccines mAb prevention of streptococcal B Phase I

conjugate Cambridge, MA infections novartisvaccines.com

Streptococcus Sanofi Pasteur vaccine treatment of meningitis and Phase I

pneumoniae vaccine Swiftwater, PA pneumonia sanofipasteur.us
(meningitis and
pneumonia vaccine)

TCN-032 Theraclone Sciences mAb influenza A virus infections Phase I

(IgG mAb) Seattle, WA theraclone-sciences.com

TCN-202 Theraclone Sciences mAb CMV infections Phase I

Seattle, WA theraclone-sciences.com

TDENV-PIV GlaxoSmithKline vaccine dengue Phase I

(tetravalent vaccine) Rsch. Triangle Park, NC gsk.com

TG-4040 Transgene recombinant hepatitis C Phase II

Cedex, France vaccine transgene.fr

Thravixa™ Emergent BioSolutions mAb post-exposure treatment of anthrax Phase I completed

fully human anthrax mAb Rockville, MD (Fast Track) emergentbiosolutions.com
(Orphan Drug)

TKM-Ebola Tekmira Pharmaceuticals RNA Ebola virus infections Phase I

Burnaby, Canada interference tekmirapharm.com

tuberculosis vaccine Sanofi Pasteur recombinant tuberculosis Phase I

(recombinant subunit Swiftwater, PA vaccine sanofipasteur.us
vaccine)

tularemia vaccine DynaPort Vaccine vaccine tularemia Phase I completed

Frederick, MD csc.com/dvc

TUTI-16 Thymon vaccine HIV-1 infection treatment Phase I/II

(lipoprotein vaccine) Short Hills, NJ

66 Medicines in Development Biologics 2013

Biologic Medicines in Development

Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category

UB-421 United Biomedical mAb HIV-1 infection treatment Phase II

Hauppauge, NY unitedbiomedical.com

V114 Merck vaccine prevention of pneumococcal Phase II

(pneumococcal 15-valent Whitehouse Station, NJ infections merck.com
vaccine conjugate)

V212 Merck vaccine prevention of herpes zoster Phase III

Whitehouse Station, NJ merck.com

V503 Merck vaccine prevention of human papillomavirus Phase III

Whitehouse Station, NJ infections merck.com
(see also cancer)

Vacc-4x Bionor Pharma vaccine HIV-1 infection treatment Phase II

Oslo, Norway bionorpharma.com

VAX102 VaxInnate recombinant prevention of influenza A virus Phase I/II completed

(flagellin.HuM2e) Cranbury, NJ fusion vaxinnate.com
protein
vaccine

VAX125 VaxInnate recombinant prevention of influenza A virus Phase II completed

(flagellin.HuHa) Cranbury, NJ fusion vaxinnate.com
protein
vaccine

VAX128 Emergent BioSolutions recombinant prevention of influenza A virus H1N1 Phase I

Rockville, MD vaccine subtype emergentbipsolutions.
VaxInnate com
Cranbury, NJ vaxinnate.com

VAX161 Emergent BioSolutions recombinant prevention of influenza A virus H5N1 Phase I

Rockville, MD vaccine subtype emergentbiosolutions.
VaxInnate com
Cranbury, NJ vaxinnate.com

VaxiGrip® Sanofi Pasteur vaccine seasonal influenza virus infections Phase III
quadrivalent inactivated Swiftwater, PA sanofipasteur.us
influenza vaccine

VGX-3400 Inovio Pharmaceuticals DNA prevention of influenza A virus Phase I

Blue Bell, PA vaccine H5N1 subtype inovio.com

visceral leishmaniasis Infectious Disease vaccine visceral leishmaniasis Phase I

vaccine Research Institute idri.org
Seattle, WA

Medicines in Development Biologics 2013 67

 Biologic Medicines in Development

Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category

XOMA 3AB XOMA mAb botulism Phase I

Berkeley, CA xoma.com
National Institute of Allergy and
Infectious Diseases
Bethesda, MD

Zostavax® Merck vaccine prevention of herpes zoster Phase I

varicella zoster Whitehouse Station, NJ merck.com
virus vaccine live

Musculoskeletal Disorders/Arthritis
Product Name Sponsor Product Indication Development Phase
Category

ABT-981 AbbVie mAb osteoarthritis Phase I

North Chicago, IL abbvie.com

AMG 167 Amgen antibody metabolic bone diseases Phase I

Thousand Oaks, CA amgen.com

AMG 745 Amgen mAb muscular atrophy Phase I

Thousand Oaks, CA amgen.com

AMG 827 AstraZeneca mAb psoriatic arthritis Phase II

(brodalumab) Wilmington, DE (see also respiratory, skin) astrazeneca.com
Amgen amgen.com
Thousand Oaks, CA

blosozumab Eli Lilly mAb osteoporosis Phase II

(LY2541546) Indianapolis, IN lilly.com

BYM338 Novartis Pharmaceuticals mAb sporadic inclusion body myositis, Phase II

East Hanover, NJ muscular atrophy novartis.com
(see also cancer)

Chondrogen® Osiris Therapeutics stem cell knee injuries, prevention of Phase II

mesenchymal stem cell Columbia, MD therapy osteoarthritis osiris.com
therapy for meniscal
repair

Cimzia® UCB mAb psoriatic arthritis Phase III

certolizumab pegol Smyrna, GA (see also autoimmune) ucb.com

68 Medicines in Development Biologics 2013

Biologic Medicines in Development

Musculoskeletal Disorders/Arthritis
Product Name Sponsor Product Indication Development Phase
Category

clazakizumab Bristol-Myers Squibb mAb psoriatic arthritis Phase II

(anti-IL6) Princeton, NJ (see also autoimmune, bms.com
Alder Biopharmaceuticals digestive) alderbio.com
Bothell, WA

Dysport® Ipsen bacterial focal spasticity of upper Phase III

abobotulinumtoxinA Paris, France protein and lower limb ipsen.com

FGF-18 EMD Serono growth factor acute cartilage injury of the knee Phase II
(sprifermin) Rockland, MA emdserono.com

gevokizumab XOMA mAb osteoarthritis of the hand Phase II

(IL-1B inhibitor mAb) Berkeley, CA (see also diabetes, eye, skin) xoma.com

Humira® AbbVie mAb spondylarthritis Phase III

adalimumab North Chicago, IL (see also autoimmune, abbvie.com
digestive, eye, skin, other)

Ilaris® Novartis Pharmaceuticals mAb gouty arthritis application submitted

canakinumab East Hanover, NJ (see also autoimmune, novartis.com
cardiovascular, diabetes)
------------------------------------------------ ---------------------------------
osteoarthritis, polymyalgia Phase II
rheumatica novartis.com

ixekizumab Eli Lilly mAb psoriatic arthritis Phase III

(IL-17 antibody) Indianapolis, IN (see also skin) lilly.com

juvenile chondrocyte ISTO Technologies cell cartilage disorders Phase III

grafts St. Louis, MO therapy isotech.com

KTP-001 Kaketsuken recombinant intervertebral disc displacement Phase I/II

(recombinant human Kumamoto, Japan protein kaketsuken.or.jp
matrix metalloproteinase) Teijin Pharma teijin-pharma.co.jp
Tokyo, Japan

LY2495655 Eli Lilly mAb disuse muscular atrophy Phase II

(anti-myostatin mAb) Indianapolis, IN (see also cancer) lilly.com

MACI® Genzyme cell therapy articular cartilage defects Phase III

matrix-induced Cambridge, MA genzyme.com
autologous
chondrocyte implantation

MCS110 Novartis Pharmaceuticals mAb synovitis Phase II

East Hanover, NJ novartis.com

Medicines in Development Biologics 2013 69

 Biologic Medicines in Development

Musculoskeletal Disorders/Arthritis
Product Name Sponsor Product Indication Development Phase
Category

mesenchymal precursor Mesoblast stem cell spinal fusion, spondylosis Phase II

cell (MPC) product New York, NY therapy (see also cardiovascular, diabetes, mesoblast.com
transplantation)

mesenchymal stem cell Medipost stem cell osteoarthritis Phase I/II

therapy Seoul, South Korea therapy medi-post.com

NeoCart® Histogenics cell cartilage disorders Phase III

autologous chondrocyte Waltham, MA therapy histogenics.com
implantation

Orencia® Bristol-Myers Squibb recombinant psoriatic arthritis Phase II

abatacept Princeton, NJ fusion (see also autoimmune, bms.com
protein other)

Prolia® Amgen mAb male osteoporosis Phase III

denosumab Thousand Oaks, CA (see also autoimmune) amgen.com

romosozumab Amgen mAb postmenopausal osteoporosis Phase III

(AMG 785) Thousand Oaks, CA amgen.com

SAR391786 Regeneron Pharmaceuticals mAb treatment of muscle atrophy Phase I

(REGN1033) Tarrytown, NY post-orthopedic surgery regeneron.com
Sanofi US sanofi.com
Bridgewater, NJ

secukinumab Novartis Pharmaceuticals mAb psoriatic arthritis Phase III

(AIN 457) East Hanover, NJ (see also autoimmune, eye, skin) novartis.com
------------------------------------------------ ---------------------------------
polymyalgia rheumatica Phase II
novartis.com

SI-6603 Seikagaku enzyme intervertebral disc Phase II

Tokyo, Japan displacement seikagaku.co.jp

Stelara® Janssen Biotech mAb psoriatic arthritis application submitted

ustekinumab Horsham, PA (see also autoimmune, digestive, janssenbiotech.com
other)

tanezumab Pfizer mAb osteoarthritis Phase III

New York, NY (see also neurologic) pfizer.com

teriparatide Eli Lilly recombinant healing of fractures in patients with Phase III
(recombinant PTH 1-34) Indianapolis, IN hormone osteoporosis lilly.com

TG-C TissueGene cell osteoarthritis Phase II

Rockville, MD therapy tissuegene.com

Xiaflex® Auxilium Pharmaceuticals peptide frozen shoulder Phase II

collagenase clostridium Chesterbrook, PA (see also cancer, other) auxilium.com
histolyticum BioSpecifics Technologies biospecifics.com

70 Medicines in Development Biologics 2013

Biologic Medicines in Development

Neurologic Disorders
Product Name Sponsor Product Indication Development Phase
Category

AAB-002 Janssen Alzheimer mAb Alzheimer’s disease Phase 0

Immunotherapy janimm.com
South San Francisco, CA pfizer.com
Pfizer
New York, NY

AAB-003/PF-05236812 Janssen Alzheimer mAb Alzheimer’s disease Phase I

Immunotherapy janimm.com
South San Francisco, CA pfizer.com
Pfizer
New York, NY

AAV1-FS344 Milo Biotechnology gene therapy Becker muscular Phase I/II

(gene therapy-delivered Cleveland, OH dystrophy milobiotechnology.com
myostatin inhibitor)
(Orphan Drug)

ABT-110 AbbVie mAb chronic pain Phase II

North Chicago, IL abbvie.com

ACC-001 Janssen Alzheimer peptide Alzheimer’s disease Phase II

(PF-05236806) Immunotherapy vaccine janimm.com
South San Francisco, CA pfizer.com
Pfizer
New York, NY

AD02 vaccine AFFiRis vaccine Alzheimer’s disease Phase II

Vienna, Austria affiris.com
GlaxoSmithKline gsk.com
Rsch. Triangle Park, NC

AD03 vaccine AFFiRis vaccine Alzheimer’s disease Phase I

Vienna, Austria affiris.com
GlaxoSmithKline gsk.com
Rsch. Triangle Park, NC

AGN-214868 Allergan recombinant postherpetic neuralgia Phase II

(senrebotase) Irvine, CA protein (see also other) allergan.com

ALD403 Alder Biopharmaceuticals mAb prevention of migraine Phase I

Bothell, WA alderbio.com

ATI355 Novartis Pharmaceuticals mAb spinal cord injury Phase I

(anti-Nogo-A mAb) East Hanover, NJ novartis.com
(Orphan Drug)

Medicines in Development Biologics 2013 71

 Biologic Medicines in Development

Neurologic Disorders
Product Name Sponsor Product Indication Development Phase
Category

AYX-1 Adynxx oglionucleo- postoperative pain Phase I

San Francisco, CA tide adynxx.com

BA-210 BioAxone Biosciences recombinant spinal cord injury Phase II

(Orphan Drug) Cambridge, MA fusion protein bioaxonebio.com

BAN2401 BioArctic Neuroscience mAb Alzheimer’s disease Phase I

(amyloid beta-protein Stockholm, Sweden bioarctic.se
inhibitor) Eisai eisai.com
Woodcliff Lake, NJ

bapineuzumab Janssen Alzheimer mAb Alzheimer’s disease Phase II

(subcutaneous) Immunotherapy janimm.com
(AAB-001) South San Francisco, CA pfizer.com
Pfizer
New York, NY

Botox® Allergan bacterial motor neuron disease Phase III

onabotulinumtoxinA Irvine, CA protein (see also other) allergan.com

CAD106 Cytos Biotechology therapeutic Alzheimer’s disease Phase II

Zurich, Switzerland vaccine cytos.com
Novartis Pharmaceuticals novartis.com
East Hanover, NJ

CERE-110 Ceregene gene therapy Alzheimer’s disease Phase II

(AAV-NGF) San Diego, CA ceregene.com

CERE-120 Ceregene gene therapy Parkinson’s disease Phase II

(AAV-NTN) San Diego, CA ceregene.com

chondrocyte tissue ISTO Technologies cell back pain Phase I

grafts St. Louis, MO therapy isotech.com

crenezumab Genentech mAb Alzheimer’s disease Phase II

(anti-Abeta) South San Francisco, CA gene.com

fulranumab Janssen Research mAb cancer pain Phase II

& Development janssenrnd.com
Raritan, NJ

GDNF-producing adult BrainStorm Cell Therapeutics stem cell amyotrophic lateral sclerosis Phase I/II
stem cell therapy New York, NY therapy (ALS) brainstorm-cell.com
(Orphan Drug)

GSK1223249 GlaxoSmithKline mAb ALS Phase I

(NOGO-A mAb) Rsch. Triangle Park, NC (see also autoimmune) gsk.com

GSK933776A GlaxoSmithKline mAb Alzheimer’s disease Phase I completed

(anti-B amyloid mAb) Rsch. Triangle Park, NC (see also eye) gsk.com

72 Medicines in Development Biologics 2013

Biologic Medicines in Development

Neurologic Disorders
Product Name Sponsor Product Indication Development Phase
Category

GZ404477 Genzyme gene therapy Parkinson’s disease Phase I

(AAV-hAADC gene Cambridge, MA genzyme.com
therapy)

HuCNS-SC® StemCells stem cell thoracic spinal cord injury Phase I/II
adult neural stem Newark, CA therapy (see also eye, genetic) stemcellsinc.com
cell therapy
(intraosseous)

LY2951742 Arteaus Therapeutics mAb migraine prevention Phase II

(CGRP peptide) Cambridge, MA lilly.com
Eli Lilly
Indianapolis, IN

MEDI-5117 AstraZeneca mAb osteoarthritis pain Phase I

(anti-IL-6 mAb) Wilmington, DE astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD

neurological disorders TCA Cellular Therapy stem cell ALS, spinal cord injuries Phase I
stem cell therapy Covington, LA therapy tcacellulartherapy.com

NSI-566 Neuralstem stem cell ALS Phase I

(Orphan Drug) Rockville, MD therapy neuralstem.com

RG1450 Roche mAb prodromal Alzheimer’s disease Phase II/III

(gantenerumab) Nutley, NJ roche.com

RN-307 Labrys Biologics mAb migraine Phase II

(anti-CGRP mAb) San Francisco, CA

SAR228810 Sanofi US mAb Alzheimer’s disease Phase I

(anti-protofibrillar AB Bridgewater, NJ sanofi.com
mAb)

solanezumab Eli Lilly mAb Alzheimer’s disease Phase III

(LY2062430) Indianapolis, IN lilly.com

tanezumab Pfizer mAb chronic pain Phase II

New York, NY (see also musculoskeletal) pfizer.com

UB-311 United Biomedical vaccine Alzheimer’s disease Phase II

immunotherapeutic Hauppauge, NY unitedbiomedical.com

V950 Merck vaccine Alzheimer’s disease Phase I completed

Whitehouse Station, NJ merck.com

VM-202 VM BioPharma gene diabetic neuropathy Phase II

(modified hepatocyte Atlanta, GA therapy (see also cardiovascular) viromed.co.kr
growth factor gene
therapy)

Medicines in Development Biologics 2013 73

 Biologic Medicines in Development

Neurologic Disorders
Product Name Sponsor Product Indication Development Phase
Category

Xeomin® Merz Pharmaceuticals bacterial post-stroke spasticity Phase III

incobotulinumtoxinA Greensboro, NC protein merzusa.com
----------------------------------------------- ---------------------------------
sialorrhea in patients with Phase II
Parkinson’s disease and ALS merzusa.com

Respiratory Disorders
Product Name Sponsor Product Indication Development Phase
Category

ALT-836 Altor BioScience mAb acute lung injury, adult respiratory Phase II
Miramar, FL distress syndrome altorbioscience.com
Genentech (see also cancer) gene.com
South San Francisco, CA

AMG 157/MEDI-9929 Amgen mAb asthma Phase I

Thousand Oaks, CA amgen.com
AstraZeneca astrazeneca.com
Wilmington, DE

AMG 761 Amgen mAb asthma Phase I

Thousand Oaks, CA amgen.com

AMG 827 Amgen mAb asthma Phase II

(brodalumab) Thousand Oaks, CA (see also musculoskeletal, skin) amgen.com
AstraZeneca astrazeneca.com
Wilmington, DE

ASM8 Pharmaxis antisense asthma Phase II

Exton, PA pharmaxis.com.au

benralizumab AstraZeneca mAb asthma, chronic obstructive Phase II

Wilmington, DE pulmonary disease (COPD) astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD

Bosatria™ GlaxoSmithKline mAb asthma Phase III

mepolizumab Rsch. Triangle Park, NC gsk.com

carlumab Janssen Biotech mAb pulmonary fibrosis Phase II

Horsham, PA janssenbiotech.com

74 Medicines in Development Biologics 2013

Biologic Medicines in Development

Respiratory Disorders
Product Name Sponsor Product Indication Development Phase
Category

CNTO-3157 Janssen Biotech mAb asthma Phase I

Horsham, PA janssenbiotech.com

CNTO-5825 Janssen Biotech mAb allergic asthma Phase I

Horsham, PA janssenbiotech.com

CYT003 Cytos Biotechnology vaccine allergic asthma Phase II

Zurich, Switzerland cytos.com

Excellair™ ZaBeCor Pharmaceuticals RNA asthma Phase II

syk kinase silencer Bala Cynwyd, PA interference zabecor.com

FG-3019 FibroGen mAb idiopathic pulmonary fibrosis Phase II

San Francisco, CA (see also cancer, other) fibrogen.com

grass pollen extract Stallergenes immuno- seasonal allergic rhinitis Phase III
Cedex, France therapy stallergenes.com

GS-6624 Gilead Sciences mAb idiopathic pulmonary fibrosis Phase I

(simtuzumab) Foster City, CA (see also cancer, other) gilead.com

GSK2586881 Apeiron Biologics recombinant acute lung injury Phase I

(APN01) Vienna, Austria protein gsk.com
GlaxoSmithKline
Rsch. Triangle Park, NC

KB003 KaloBios Pharmaceuticals mAb severe asthma Phase II

South San Francisco, CA kalobios.com

MEDI-4212 AstraZeneca mAb asthma Phase I

(anti-IgE mAb) Wilmington, DE astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD

MEDI-7814 AstraZeneca mAb COPD Phase I

(anti-C5/C5a mAb) Wilmington, DE astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD

MEDI-8968 AstraZeneca mAb COPD Phase II

(anti-IL-1R mAb) Wilmington, DE astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD

Medicines in Development Biologics 2013 75

 Biologic Medicines in Development

Respiratory Disorders
Product Name Sponsor Product Indication Development Phase
Category

MK-3641 Merck immuno- ragweed pollen-induced Phase III

ragweed allergy vaccine Whitehouse Station, NJ therapy rhinoconjunctivitis merck.com
(sublingual) vaccine

MK-7243 Merck immuno- grass pollen-induced allergic Phase III

grass pollen allergy tablet Whitehouse Station, NJ therapy rhinoconjunctivitis merck.com
vaccine

MK-8237 Merck vaccine allergic asthma, allergic rhinitis, Phase II

house dust mite Whitehouse Station, NJ allergic rhinoconjunctivitis merck.com
allergy vaccine tablet

Pollinex® Quattro Allergy Therapeutics vaccine grass pollen hypersensitivity, Phase III
injectable MPL allergy West Sussex, United Kingdom ragweed pollen hypersensitivity allergytherapeutics.com
vaccine ----------------------------------------------- ---------------------------------
tree pollen hypersensitivity Phase II
allergytherapeutics.com

PRM-151 Promedior recombinant idiopathic pulmonary fibrosis Phase I

(rhPTX-2 for injection) Lexington, MA protein (see also other) promedior.com
(Orphan Drug)

Prochymal® Osiris Therapeutics stem cell COPD Phase II

remestemcel-L Columbia, MD therapy (see also cardiovascular, osiris.com
diabetes, digestive, transplantation)

QAX576 Novartis Pharmaceuticals mAb asthma, idiopathic pulmonary Phase II

East Hanover, NJ fibrosis novartis.com
(see also digestive)

QBX258 Novartis Pharmaceuticals mAb asthma Phase II

East Hanover, NJ novartis.com

QGE031 Novartis Pharmaceuticals mAb allergic asthma Phase II

East Hanover, NJ (see also skin) novartis.com

quilizumab Genentech mAb allergic asthma, allergic rhinitis Phase II

(anti-M1 prime mAb) South San Francisco, CA gene.com

ragweed allergy Stallergenes vaccine seasonal allergic rhinitis Phase I

immunotherapy Cedex, France stallergenes.com
(sublingual)

ragweed allergy vaccine Greer Laboratories vaccine seasonal allergic rhinitis Phase III
(sublingual) Lenoir, NC greerlabs.com

76 Medicines in Development Biologics 2013

Biologic Medicines in Development

Respiratory Disorders
Product Name Sponsor Product Indication Development Phase
Category

reslizumab Cephalon mAb asthma, eosinophilic esophagitis in Phase III

(Orphan Drug) Frazer, PA adolescents and children cephalon.com

RG3637 Genentech mAb severe asthma Phase III

(lebrikizumab) South San Francisco, CA gene.com
Roche roche.com
Nutley, NJ

SAR156597 Sanofi US mAb idiopathic pulmonary fibrosis Phase II

(bispecific interleukin-4/ Bridgewater, NJ sanofi.com
interleukin-13 mAb)

SAR231893 Regeneron Pharmaceuticals mAb asthma Phase II

(anti-IL4 mAb) Tarrytown, NY (see also skin) regeneron.com
Sanofi US sanofi.com
Bridgewater, NJ

STX-100 Biogen Idec mAb idiopathic pulmonary fibrosis Phase II

Weston, MA biogenidec.com

tralokinumab AstraZeneca mAb asthma Phase II

Wilmington, DE (see also digestive) astrazeneca.com
MedImmune medimmune.com
Gaithersburg, MD

Skin Diseases
medicines for skin cancer are listed under Cancer

Product Name Sponsor Product Indication Development Phase

Category

AbGn-168H AbGenomics International mAb plaque psoriasis Phase I completed

Los Altos, CA abgenomics.com

Amevive® Astellas Pharma US recombinant psoriasis in adolescents Phase II completed

alefacept Deerfield, IL fusion us.astellas.com
protein

AMG 827 Amgen mAb psoriasis Phase II

(brodalumab) Thousand Oaks, CA (see also musculoskeletal, amgen.com
AstraZeneca respiratory) astrazeneca.com
Wilmington, DE

ANT-1207 Anterios bacterial acne vulgaris Phase II

(botulinum toxin A) New York, NY protein,
nanotechnol-
ogy

Medicines in Development Biologics 2013 77

 Biologic Medicines in Development

Skin Diseases
medicines for skin cancer are listed under Cancer
Product Name Sponsor Product Indication Development Phase
Category

BT-061 AbbVie mAb plague psoriasis Phase II

North Chicago, IL (see also autoimmune) abbvie.com
Biotest biotest.de
Dreieich, Germany

CNTO-1959 Janssen Biotech mAb plaque psoriasis Phase II

Horsham, PA (see also autoimmune) janssenbiotech.com

CureXcell™ Macrocure cell therapy diabetic foot ulcer Phase I/II

white blood cell therapy Tikva, Israel macrocure.com

Enbrel® Amgen recombinant plague psoriasis in adolescents application submitted

etanercept Thousand Oaks, CA fusion protein and children amgen.com

fibroblast growth CardioVascular Therapeutics fibroblast wounds in diabetes Phase II

factor-1 Las Vegas, NV growth factor (see also cardiovascular) cvbt.com

GBT 009 Garnet BioTherapeutics cell scar prevention Phase II

Malvern, PA therapy garnetbio.com

gevokizumab XOMA mAb acne vulgaris Phase II

(IL-1B inhibitor mAb) Berkeley, CA (see also diabetes, eye, xoma.com
musculoskeletal)

HP802-247 Healthpoint Biotherapeutics cell leg ulcers Phase III

(allogeneic human Fort Worth, TX therapy healthpointbio.com
skin replacement) ------------------------------------------------ ---------------------------------
Mohs micrographic surgical wounds Phase II
healthpointbio.com

Humira® AbbVie mAb hidradenitis suppurativa Phase III

adalimumab North Chicago, IL (see also autoimmune, digestive, abbvie.com
eye, musculoskeletal, other)

ixekizumab Eli Lilly mAb psoriasis Phase III

(IL-17 antibody) Indianapolis, IN (see also musculoskeletal) lilly.com

JVS-100 SironRX Therapeutics gene wound healing related to Phase I

(stromal cell-derived Cleveland, OH therapy cardiovascular surgery sironrx.com
factor-1)

KUR-211 Baxter International growth diabetic foot ulcers Phase II

(bioactive wound-healing Deerfield, IL factor baxter.com
foam) Kuros Biosurgery kuros.ch
Zurich, Switzerland

78 Medicines in Development Biologics 2013

Biologic Medicines in Development

Skin Diseases
medicines for skin cancer are listed under Cancer
Product Name Sponsor Product Indication Development Phase
Category

laViv™ Fibrocell Science fibroblast scars Phase II/III

azficel-T Exton, PA cell fibrocellscience.com
therapy

MABp1 XBiotech mAb acne, psoriasis Phase II

Austin, TX (see also cancer, cardiovascular, xbiotech.com
diabetes)

MK-3222 Merck mAb plaque psoriasis Phase III

(tildrakizumab) Whitehouse Station, NJ merck.com

PF-06473871 (EXC 001) Pfizer antisense dermal scarring Phase II

New York, NY pfizer.com

QGE031 Novartis Pharmaceuticals mAb atopic dermatitis Phase II

East Hanover, NJ (see also respiratory) novartis.com

REGN846 Regeneron Pharmaceuticals mAb atopic dermatitis Phase I/II

Tarrytown, NY regeneron.com

RT001 Revance Therapeutics bacterial lateral canthal lines Phase II

(botulinum toxin A) Newark, CA protein (see also other) revance.com

RXI-109 RXi Pharmaceuticals RNA dermal scarring Phase I

Westborough, MA interference rxipharma.com

SAR231893 Regeneron Pharmaceuticals mAb atopic dermatitis Phase II

(anti-IL4 mAb) Tarrytown, NY (see also respiratory) regeneron.com
Sanofi US sanofi.com
Bridgewater, NJ

SDF-1 SironRX gene therapy wound healing and reduction of Phase I

Cleveland, OH scarring sironrx.com

secukinumab Novartis Pharmaceuticals mAb plaque psoriasis Phase III

(AIN457) East Hanover, NJ (see also autoimmune, novartis.com
eye, musculoskeletal)

StrataGraft® Stratatech cell chronic wounds Phase I/II

tissue repair cell Madison, WI therapy stratatechcorp.com
therapy
(Orphan Drug)

TD101 TransDerm RNA pachyonychia congenita Phase I

Santa Cruz, CA interference transderminc.com

Xolair® Genentech mAb chronic idiopathic urticaria Phase III

omalizumab South San Francisco, CA gene.com
Novartis Pharmaceuticals novartis.com
East Hanover, NJ

Medicines in Development Biologics 2013 79

 Biologic Medicines in Development

Transplantation
Product Name Sponsor Product Indication Development Phase
Category

ASKP-1240 Astellas Pharma US mAb prevention of organ transplant Phase II

Deerfield, IL rejection us.astellas.com
Kyowa Hakko Kirin Pharma kyowa-kirin-pharma.com
Princeton, NJ

Benlysta® GlaxoSmithKline mAb immunosuppression Phase II

belimumab Rsch. Triangle Park, NC (see also autoimmune, blood) gsk.com
(intravenous)

CaspaCIDe™ DLI Bellicum Pharmaceuticals cell graft-versus-host disease in Phase I/II

T-cell replacement Houston, TX therapy patients with late-stage cancer bellicum.com
therapy undergoing bone marrow
transplantation

CDX-301 Celldex Therapeutics recombinant hematopoietic stem cell Phase I

(Fms-like tyrosine Needham, MA protein transplantation celldextherapeutics.com
kinase 3 ligand)

CLT-008 Cellerant Therapeutics cell therapy facilitate engraftment in core Phase I

San Carlos, CA blood transplants cellerant.com
(see also cancer)

CYT107 Cytheris recombinant prevention of bone marrow Phase I

(interleukin-7) Rockville, MD interleukin transplant rejection cytheris.com
(see also blood, cancer, infectious)

mesenchymal precursor Mesoblast stem cell for bone marrow regeneration Phase III
cell (MPC) product New York, NY therapy in patients undergoing bone mesoblast.com
(Orphan Drug) marrow transplantation
(see also cardiovascular, diabetes,
musculoskeletal)

MultiStem® Athersys stem cell graft-versus-host disease Phase I

stem cell therapy Cleveland, OH therapy (see also cardiovascular, digestive) athersys.com
(Orphan Drug)

NX001 Nephrx growth delayed graft function in Phase I

(Orphan Drug) Kalamazoo, MI factor renal transplant patients nephrx.com

Prochymal® Osiris Therapeutics stem cell graft-versus-host disease Phase III

remestemcel-L Columbia, MD therapy (Fast Track) osiris.com
(Orphan Drug) (see also cardiovascular, diabetes,
digestive, respiratory)

QPI 1002 Quark Pharmaceuticals RNA prevention of delayed graft Phase II

(I5NP) Fremont, CA interference function quarkpharma.com
(Orphan Drug) (see also other)

Soliris® Alexion Pharmaceuticals mAb presensitized kidney transplant Phase II

eculizumab Cheshire, CT (acute humoral rejection) alxn.com
(see also autoimmune,
eye, infectious)

80 Medicines in Development Biologics 2013

Biologic Medicines in Development

Transplantation
Product Name Sponsor Product Indication Development Phase
Category

TOL101 Tolera Therapeutics mAb prevention of transplant rejection Phase I/II

(Orphan Drug) Kalamazoo, MI tolera.com

Other
Product Name Sponsor Product Indication Development Phase
Category

AC607 AlloCure stem cell acute kidney injury Phase II

(allogeneic multi-potent Burlington, MA therapy (Fast Track) allocure.com
stem cell therapy)
(Orphan Drug)

AGN-214868 Allergan recombinant overactive bladder with urinary Phase II

(senrebotase) Irvine, CA protein incontinence allergan.com
(see also neurologic)

ALD-451 Cytomedix stem cell cognition disorders following Phase I

Gaithersburg, MD therapy treatment for malignant glioma cytomedix.com

Ampligen® Hemispherx Biopharma RNA product chronic fatigue syndrome application submitted
rintatolimid Philadelphia, PA hemispherx.net
(Orphan Drug)

antibody therapeutic MorphoSys mAb undisclosed Phase I

Munich, Germany morphosys.com
Novartis Pharmaceuticals novartis.com
East Hanover, NJ

antibody therapeutic MorphoSys mAb undisclosed Phase II

Munich, Germany morphosys.com
Novartis Pharmaceuticals novartis.com
East Hanover, NJ

antibody therapeutic MorphoSys mAb undisclosed Phase I

(BI-1) Munich, Germany morphosys.com
Boehringer-Ingelheim boehringer-ingelheim.com
Pharmaceuticals
Ridgefield, CT

anti-IL31 Bristol-Myers Squibb mAb immunology Phase I

Princeton, NJ bms.com

Medicines in Development Biologics 2013 81

 Biologic Medicines in Development

Other
Product Name Sponsor Product Indication Development Phase
Category

anti-TWEAK Biogen Idec mAb lupus nephritis Phase II

(BIIB 023) Weston, MA biogenidec.com

BAX-69 Baxter International mAb lupus nephritis Phase I

Deerfield, IL (see also cancer) baxter.com

Botox® Allergan bacterial idiopathic overactive application submitted

onabotulinumtoxinA Irvine, CA protein bladder allergan.com
(see also neurologic)
------------------------------------------------ ---------------------------------
benign prostatic hyperplasia Phase II
allergan.com

cat allergy vaccine Circassia vaccine hypersensitivity in patients with Phase III
Oxford, United Kingdom rhinoconjunctivitis caused by cat circassia.co.uk
allergy

CDX-1135 Celldex Therapeutics recombinant kidney disorders Phase I

Needham, MA protein celldextherapeutics.com

CM-AT CureMark enzyme autistic disorder Phase III

Rye, NY replacement (Fast Track) curemark.com
therapy

CNTO-5 Janssen Biotech mAb inflammation Phase I

Horsham, PA janssenbiotech.com
MorphoSys morphosys.com
Munich, Germany

CNTO-136 Janssen Biotech mAb lupus nephritis Phase II

(sirukumab) Horsham, PA (see also autoimmune) janssenbiotech.com

ELAD® Vital Therapies cell liver failure Phase II/III

human-cell-based San Diego, CA therapy vitaltherapies.com
bioartificial liver
(Orphan Drug)

entolimod Cleveland BioLabs engineered acute radiation syndrome Phase I

(CBLB502) Buffalo, NY peptide (Fast Track) cbiolabs.com
(Orphan Drug) (see also cancer)

FB 301 Cytovance Biologics mAb hypersensitivity in clinical trials

Oklahoma City, OK (IgE-mediated allergic diseases) cytovance.com
Fountain Biopharma
Taipei, Taiwan

FG-3019 FibroGen mAb liver fibrosis due to chronic Phase II

San Francisco, CA hepatitis B infection fibrogen.com
(see also cancer, respiratory)

82 Medicines in Development Biologics 2013

Biologic Medicines in Development

Other
Product Name Sponsor Product Indication Development Phase
Category

food allergy DBV Technologies topical peanut hypersensitivity Phase II

immunotherapy Paris, France vaccine dbv-technologies.com

fresolimumab Genzyme mAb fibrosis Phase II

(TGFß antagonist) Cambridge, MA genzyme.com

GS-6624 Gilead Sciences mAb liver fibrosis, myelofibrosis Phase II

(simtuzumab) Foster City, CA (see also cancer, respiratory) gilead.com

GSK1070806 GlaxoSmithKline mAb metabolic disorders Phase II

(anti-interleukin 18 mAb) Rsch. Triangle Park, NC gsk.com

hMaxi-K Ion Channel Innovations gene therapy overactive bladder Phase I completed
gene therapy New York, NY ionchannelinnovations.
com

Humira® AbbVie mAb interstitial cystitis Phase III

adalimumab North Chicago, IL (see also autoimmune, digestive, abbvie.com
eye, musculoskeletal, skin)

ISIS-FGFR4Rx Isis Pharmaceuticals antisense obesity Phase II

Carlsbad, CA isispharm.com

ISIS-GCCRRx Isis Pharmaceuticals antisense Cushing syndrome Phase I

Carlsbad, CA (see also diabetes) isispharm.com

JRC-LAMP-vax Immunomic Therapeutics DNA vaccine Japanese red cedar pollen-induced Phase I
Lancaster, PA rhinoconjunctivitis immunomix.com

kidney disease medicine Eli Lilly biologic chronic kidney disease Phase I
Indianapolis, IN lilly.com

liprotamase Eli Lilly enzyme treatment of exocrine pancreatic application submitted

(LY3031642) Indianapolis, IN replacement enzyme insufficiency, including in lilly.com
(Orphan Drug) therapy cystic fibrosis patients
(Fast Track)

liraglutide Novo Nordisk recombinant obesity Phase III

(NN8022) Princeton, NJ peptide novonordisk.com

liver cell therapy Cytonet cell urea cycle disorders in children Phase II
(Orphan Drug) Weinheim, Germany therapy and infants cytonet.de

LY2382770 Eli Lilly mAb diabetic nephropathy Phase II

(TGF-ß antibody) Indianapolis, IN lilly.com

Medicines in Development Biologics 2013 83

 Biologic Medicines in Development

Other
Product Name Sponsor Product Indication Development Phase
Category

mAb Genentech mAb metabolic disorders Phase I

South San Francisco, CA gene.com

Meth-mAb InterveXion Therapeutics mAb methamfetamine abuse Phase I

Little Rock, AR intervexion.com

metreleptin Bristol-Myers Squibb recombinant lipodystrophy application submitted

Princeton, NJ hormone bms.com

Natpara® NPS Pharmaceuticals recombinant hypoparathyroidism Phase III

recombinant human Salt Lake City, UT hormone npsp.com
parathyroid hormone
(rhPTH)
(Orphan Drug)

Neo-Urinary Conduit™ Tengion cell cystectomy due to bladder cancer Phase I

autologous cultured Winston-Salem, NC therapy tengion.com
urological tissue cells

NIC002 Novartis Pharmaceuticals vaccine smoking withdrawal Phase II

East Hanover, NJ novartis.com

Orencia® Bristol-Myers Squibb recombinant lupus nephritis Phase III

abatacept Princeton, NJ fusion (see also autoimmune, bms.com
protein musculoskeletal)

PF-05402536 Pfizer vaccine smoking cessation Phase I

New York, NY pfizer.com

PRM-151 Promedior recombinant myelofibrosis Phase I

(rhPTX-2 for injection) Lexington, MA protein (see also respiratory) promedior.com

prohibitin targeting Ablaris Therapeutics homing obesity Phase I

peptide-1 Pasadena, CA peptide ablaris.com

PRT-105 Protalix Biotherapeutics recombinant poisoning Phase I completed

Carmiel, Israel enzyme protalix.com

QPI-1002 Quark Pharmaceuticals RNA prevention of renal failure in patients Phase I/II
(I5Np) Fremont, CA interference undergoing cardiovascular surgery quarkpharma.com
(see also transplantation)

recombinant human Kringle Pharma recombinant acute renal failure Phase I

hepatocyte growth factor Osaka, Japan growth factor kringle-pharma.com

REGN728 Regeneron Pharmaceuticals mAb undisclosed Phase I

Tarrytown, NY regeneron.com

84 Medicines in Development Biologics 2013

Biologic Medicines in Development

Other
Product Name Sponsor Product Indication Development Phase
Category

RT001 Revance Therapeutics bacterial hyperhidrosis Phase II

(botulinum toxin A) Newark, CA protein (see also skin) revance.com

SEL-068 Selecta Biosciences nanoparticle smoking withdrawal Phase I

Watertown, MA vaccine selectabio.com

somatropin biosimilar LG Life Sciences recombinant somatropin deficiency Phase III

(sustained-release) Seoul, South Korea human lgls.com
growth
hormone

Stelara® Janssen Biotech mAb primary biliary cirrhosis Phase II

ustekinumab Horsham, PA (see also autoimmune, digestive, janssenbiotech.com
musculoskeletal)

TA-CD Celtic Pharma immuno- cocaine abuse Phase II

Hamilton, Bermuda therapeutic celtictherapeutic.com
vaccine

UMK-121 Proteonomix stem cell liver failure Phase I

Hawthorne, NJ therapy proteonomix.com

VAY736 MorphoSys mAb inflammation Phase I

Munich, Germany (see also cancer) morphosys.com
Novartis Pharmaceuticals novartis.com
East Hanover, NJ

VEN100 Ventria Bioscience recombinant antibiotic-associated diarrhea Phase II

(recombinant human Fort Collins, CO protein ventria.com
lactoferrin)

VRS-317 Versartis recombinant somatropin deficiency Phase I/II

(human growth hormone) Redwood City, CA human versartis.com
growth
hormone

Xiaflex® Auxilium Pharmaceuticals peptide Peyronie’s disease application submitted

collagenase clostridium Chesterbrook, PA (see also cancer, musculoskeletal) auxilium.com
histolyticum BioSpecifics Technologies biospecifics.com
(Orphan Drug) Lynbrook, NY ------------------------------------------------ ---------------------------------
cellulite Phase I
auxilium.com
biospecifics.com

Medicines in Development Biologics 2013 85

 Glossary

adjuvant—Substance or drug that aids an- erythropoietin—Hormone that stimulates red can be reactivated to produce painful sores of
other substance in its action. blood cell production in the bone marrow. the anus or genitals.
angioedema—Swelling of the mucous mem- Fast Track—A process designed to facilitate human papillomavirus—Viral agent of warts,
branes, tissues beneath the skin, or an internal the development and expedite the review believed to be contagious and usually harm-
organ due to an allergic reaction. of drugs to treat serious diseases and fill an less, but it can lead to cervical cancer.
unmet medical need. The status is assigned
ankylosing spondylitis—An inflammatory interferon—A glycoprotein naturally produced
by the U.S. Food and Drug Administration.
disorder of unknown cause that primarily by cells that interferes with the ability of a virus
The purpose is to get important new drugs
affects the spine. The vertebrae may fuse to reproduce after it invades the body.
to the patient earlier. Fast Track addresses a
together and form a rigid back that is impos-
broad range of serious diseases. Generally, interleukin—An endogenous substance that
sible to bend.
determining factors include whether the drug stimulates the production of different types of
antisense—An antisense drug is the mirror or will have an impact on such factors as survival, white blood cells or leukocytes.
complementary image of a small segment of day-to-day functioning, or the likelihood that
the disease, if left untreated, will progress from ischemia—Insufficient supply of blood to an
messenger RNA (mRNA), the substance that
a less severe condition to a more serious one. organ or tissue, which can cause organ dam-
carries instructions (“sense”) from the genes to
Filling an unmet medical need is defined as pro- age such as an ischemic stroke.
the cell’s protein-making machinery. The anti-
sense drug readily binds to the mRNA strand, viding a therapy where none exists or providing lymphoma—Cancers in which the cells of lym-
keeping it from transmitting its instructions to a therapy which may be potentially superior phoid tissue, found mainly in the lymph nodes
the cell, and thus inhibiting the production of an to existing therapy. Once a drug receives Fast and spleen, multiply unchecked. Lymphomas
unwanted protein. Track designation, early and frequent commu- fall into two categories: One is called Hodgkin
nication between the FDA and a drug company disease, characterized by a particular kind
application submitted—An application for is encouraged throughout the entire drug de- of abnormal cell. All others are called non-
marketing has been submitted by the company velopment and review process. The frequency Hodgkin lymphomas, which vary in their
to the Food and Drug Administration (FDA). of communication assures that questions and malignancy according to the nature and activity
biotechnology—The collection of processes issues are resolved quickly, often leading to of the abnormal cells.
that involve the use of biological systems. earlier drug approval and access by patients.
mAb (monoclonal antibodies)—Large protein
For some of the industries, these processes gene therapy—Therapy at the intracellular molecules produced by white blood cells that
involve the use of genetically engineered level to replace or inactivate the effects of seek out and destroy harmful foreign sub-
organisms. For the purpose of this report, only disease-causing genes or to augment normal stances.
those products that involve recombinant DNA, gene functions to overcome illness.
monoclonal antibody/hybridoma, continuous macular degeneration—A progressive disor-
cell lines, cellular therapy, gene therapy and glioblastoma—The most common primary der that affects the central part of the retina,
vaccines technology are included. brain tumor and one of the most aggressive causing gradual loss of vision. It is a painless
forms of brain cancer, primarily affecting adults condition, usually affects both eyes and is com-
Clostridium difficile—A bacterium that over the age of 50. mon in the elderly.
produces an irritating toxin that causes a form
of colitis characterized by profuse, watery diar- growth factors—Factors responsible for metastases/metastatic—Cancers that have
rhea with cramps and low-grade fever. regulating cell proliferation (rapid and repeated spread from the primary or original cancer site.
reproduction), function and differentiation. Cancer cells can break away from a tumor and
colony stimulating factor (CSF)—Protein
growth hormone—Pituitary hormone that travel to other areas of the body through the
responsible for controlling the production of
stimulates the growth of long bones in prepu- bloodstream or the lymph system. They are of-
white blood cells.
bertal children. ten more difficult to treat then primary tumors.
Crohn’s disease—A subacute chronic gastro-
hemophilia A and B—Hemophilia A, the “clas- multiple myeloma—A malignant condition char-
intestinal disorder, involving the small intestine,
sic” hemophilia, is a genetic bleeding disorder acterized by uncontrolled proliferation of plasma
characterized by patchy deep ulcers that may
due to deficiency of the coagulation factor cells (a class of white blood cells) in bone
cause fistulas and a narrowing and thickening
VIII. Hemophilia B, or “Christmas” disease, is marrow. Symptoms include pain and destruction
of the bowel.
caused by deficiency of coagulation factor IX. of bone tissue, numbness and paralysis, kidney
cytomegalovirus (CMV)—A DNA virus related damage, anemia, and frequent infections.
to the herpes virus, affecting mostly neonatal hepatitis—Inflammation of the liver with accom-
panying liver cell damage or death, caused most myelodysplastic syndromes (MDS)—Blood
infants and immunocompromised individuals.
often by viral infection, e.g., hepatitis B, and C. disorders that ultimately are fatal. Patients
CMV can occur without symptoms or result in
usually succumb to infections or bleeding. The
mild flu-like symptoms.
herpes simplex virus—A strain of herpes term “preleukemia” has been used to describe
virus that may lie dormant in nerve tissue and these disorders.

86 Medicines in Development Biologics 2013

Glossary

myeloid—Pertaining to, derived from, or mani- Phase I—Researchers test the drug in a small systemic lupus erythematosus (SLE)—The
festing certain features of the bone marrow. In group of people, usually between 20 and 80 most serious form of lupus, a chronic autoim-
some cases, myeloid also pertains to certain healthy adult volunteers, to evaluate its initial mune disorder causing inflammation and
types of non-lymphocyte white blood cells damage to multiple organs.
safety and tolerability profile, determine a
found in the bone marrow.
safe dosage range, and identify potential side thrombocytopenia—A reduction in the
myocardial infarction—Damage to the effects. number of platelet cells in the blood, which
heart muscle caused by stoppage or impair- causes a tendency to bleed, especially from
ment of blood flow to the heart, also known Phase II—The drug is given to volunteer the smaller blood vessels.
as heart attack. patients, usually between 100 and 300, to see ulcerative colitis—A chronic inflammation and
neuroblastoma—A tumor of the adrenal if it is effective, identify an optimal dose, and to ulceration of the lining of the colon and rectum.
glands or sympathetic nervous system (the further evaluate its short-term safety. It causes bloody diarrhea and mainly involves
part of the nervous system responsible for the left colon.
certain automatic body functions, such as the Phase III—The drug is given to a larger, more
diverse patient population, often involving be- uveitis—Inflammation of the uvea, the middle
“fight or flight response”).
layer of the eye.
tween 1,000 and 3,000 patients (but sometime
neutropenia—An abnormally low neutrophil
count (certain white blood cells). many more thousands), to generate statistically
significant evidence to confirm its safety and
Orphan Drug—A drug to treat a disease that
effectiveness. They are the longest studies,
has a patient population of 200,000 or less
in the United States, or a disease that has a and usually take place in multiple sites around
patient population of more than 200,000 and the world.
a development cost that will not be recovered pulmonary fibrosis—Scarring and thickening
from sales in the United States. of lung tissue.
peripheral vascular disorder—The obstruc- respiratory syncytial virus (RSV) disease—
tion of blood supply to the extremities, particu- One of the most important causes of lower
larly the legs, caused by atherosclerosis. respiratory tract disease in children, account-
Phase 0—First-in-human trials conducted ing for more than 90 percent of cases of
in accordance with FDA’s 2006 guidance on bronchiolitis.
exploratory Investigational New Drug (IND) restenosis—A condition where an artery plugs
studies designed to speed up development of up again following treatment to open it up.
promising drugs by establishing very early on
whether the agent behaves in human subjects sepsis—A condition associated with a serious
as was anticipated from preclinical studies. bacterial infection of the blood.

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) 835-3460
www.phrma.org | www.innovation.org | www.pparx.org | www.buysafedrugs.info
Provided 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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Medicines in Development Biologics 2013 87