Biotechnology Presented by America’S Biopharmaceutical Research Companies

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2011 REPORT

Medicines in Development Biotechnology presented by america’s biopharmaceutical research companies

Biotechnology Research Promises to Bolster the Future of Medicine with More Than 900 Medicines and Vaccines in Development

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

300 298

America’s biopharmaceutical research compa- The biotechnology medicines now in develop- nies are using biotechnology to develop 901 ment make use of these and other state-of- medicines and vaccines targeting more than the-art approaches. For example: 100 diseases. Biotechnology medicines are developed through biological processes using • A genetically-modified virus-based living cells or organisms, rather than the tra- vaccine to treat melanoma. ditional chemical synthesis approach. These • A monoclonal antibody for the treatment medicines in development are either in human of cancer and asthma. clinical trials or under review by the U.S. Food and Drug Administration. • An antisense medicine for the treatment of cancer. Biotechnology medicines use many different • A recombinant fusion protein to treat 78 cutting-edge approaches to treat disease. A age-related macular degeneration. 64 monoclonal antibody is a laboratory-made 50 version of the naturally occurring immune These are only a few examples of the new system protein that binds to and neutralizes ways America’s biopharmaceutical research foreign invaders. Interferons are proteins that companies are attacking disease through bio- 23 interfere with the ability of a cell to reproduce. technology. The 901 biotechnology medicines Antisense drugs are medicines that interfere and vaccines in development promise to push with the communication process that tells a the frontiers of science and potentially bring e y cell to produce an unwanted protein. Nano- more and better treatments to patients. technology is now being used in biotechnology vaccines antisens cell therapy as drug-delivery systems, new treatments, and gene therap diagnostics.

recombinant proteins monoclonal antibodies Biotechnology Medicines in Development

Biotechnology Medicines in Development—by Therapeutic Category

Autoimmune Disorders 69 Blood Disorders 32 Cancer/Related Conditions 352 Cardiovascular Disease 59 Diabetes/Related Conditions 24 Digestive Disorders 27 Eye Conditions 20 Genetic Disorders 19 Growth Disorders 5 HIV Infection 39 Infectious Diseases 188 Musculoskeletal Disorders 22 Neurologic Disorders 44 Respiratory Disorders 40 Skin Disorders 28 Transplantation 18 Other Diseases 36

Note: Some medicines are listed in more than one category.

Biotechnology Medicines in Development—by Product Category

Antisense 23 Cell Therapy 64 Gene Therapy 50 Growth Factors 10 Interferons 20 Monoclonal Antibodies (mAb) 300 Recombinant Hormones/Proteins 78 RNA Interference 13 Vaccines 298 Other 45

2 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Autoimmune Disorders

Product Name Sponsor Product Indication Development Phase* Category

A623 Anthera Pharmaceuticals peptide systemic lupus erythematosus Phase II San Mateo, CA fusion (SLE) (650) 931-8868 protein

Actemra® Genentech mAb rheumatoid arthritis Phase III tocilizumab South San Francisco, CA (early-stage disease), (800) 626-3553 ankylosing spondylitis

AGS-009 Argos Therapeutics mAb SLE Phase I Durham, NC (919) 287-6300

AMG 108 Amgen mAb rheumatoid arthritis Phase II completed Thousand Oaks, CA (800) 772-6436

AMG 557 Amgen mAb SLE Phase I Thousand Oaks, CA (800) 772-6436

AMG 811 Amgen mAb discoid lupus erythematosus, Phase I Thousand Oaks, CA SLE (800) 772-6436

Ampligen® Hemispherx Biopharma RNA product chronic fatigue syndrome application submitted rintatolimid Philadelphia, PA (215) 988-0080 (Orphan Drug)

anti-IL6 Bristol-Myers Squibb mAb rheumatoid arthritis Phase II (BMS-945429) Princeton, NJ (800) 332-2056 Alder Biopharmaceuticals (425) 205-2900 Bothell, WA

anti-LT alpha Genentech mAb rheumatoid arthritis Phase II (RG7416) South San Francisco, CA (800) 626-3553

anti-TWEAK Biogen Idec mAb rheumatoid arthritis Phase I completed (BIIB 023) Weston, MA (781) 464-2000 ------lupus nephritis Phase I (781) 464-2000

ARX424 Ambrx interferon multiple sclerosis Phase I (PEGylated interferon La Jolla, CA (858) 875-2400 beta-1a) EMD Serono (800) 298-8088 Rockland, MA

Arzerra® GlaxoSmithKline mAb rheumatoid arthritis Phase II ofatumumab Rsch. Triangle Park, NC (intravenous) (888) 825-5249 (see also cancer) ------rheumatoid arthritis Phase II (subcutaneous) (888) 825-5249

*For more information about a specific medicine in the report, please call the telephone number listed. Medicines in Development Biotechnology 2011 3 Biotechnology Medicines in Development

Autoimmune Disorders

Product Name Sponsor Product Indication Development Phase Category

atacicept EMD Serono recombinant SLE Phase III (TACI-lg) Rockland, MA fusion (800) 283-8088 Zymogenetics protein (206) 442-6000 Seattle, WA ------lupus nephritis Phase I (800) 283-8088 (206) 442-6000

AZ01 Allozyne interferon multiple sclerosis Phase I (peginterferon beta) Seattle, WA (206) 518-5700

BAFF antibody Eli Lilly mAb rheumatoid arthritis, SLE Phase III (tabalumab) Indianapolis, IN (see also cancer, other) (800) 545-5979 ------multiple sclerosis Phase II (800) 545-5979

baminercept Biogen Idec recombinant multiple sclerosis Phase I/II Weston, MA fusion (781) 464-2000 protein

Benlysta® GlaxoSmithKline mAb SLE Phase II belimumab Rsch. Triangle Park, NC (see also blood, transplantation) (888) 825-5249 (subcutaneous) Human Genome Sciences (301) 309-8504 Rockville, MD

BHT-3009 Bayhill Therapeutics DNA multiple sclerosis Phase II completed Palo Alto, CA vaccine (650) 320-2800

BIIB 017 Biogen Idec interferon relapsing multiple sclerosis Phase III (PEG-interferon Weston, MA (Fast Track) (781) 464-2000 beta-1a)

BT-061 Abbott Laboratories mAb rheumatoid arthritis Phase II Abbott Park, IL (see also skin) (847) 937-6100 Biotest www.biotest.de Dreieich, Germany

CAM 3001 AstraZeneca mAb rheumatoid arthritis Phase II (mavrilimumab) Wilmington, DE (800) 236-9933 CSL (301) 398-0000 Victoria, Australia MedImmune Gaithersburg, MD

Campath® Genzyme mAb relapsing-remitting multiple Phase III alemtuzumab Cambridge, MA sclerosis (617) 679-2000 (intravenous) (Fast Track) (see also cancer, transplantation)

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Autoimmune Disorders

Product Name Sponsor Product Indication Development Phase Category

CNTO-136 Janssen Biotech mAb lupus nephritis, rheumatoid arthritis Phase II (sirukumab) Horsham, PA (800) 526-7736

daclizumab Biogen Idec mAb multiple sclerosis—relapsing forms Phase III Weston, MA (781) 464-2000

epratuzumab Immunomedics mAb SLE Phase III Morris Plains, NJ (Fast Track) (973) 605-8200 UCB (see also cancer) Smyrna, GA

fezakinumab Pfizer mAb rheumatoid arthritis Phase II (ILV-094) New York, NY (see also skin) (860) 732-5156

GBR-500 Glenmark Pharmaceuticals mAb multiple sclerosis Phase I Mumbai, India (see also digestive)

gevokizumab XOMA mAb Behcet’s syndrome, Phase II (Orphan Drug) Berkeley, CA rheumatoid arthritis (510) 204-7200 (see also cardiovascular, diabetes)

GSK315234 GlaxoSmithKline mAb rheumatoid arthritis Phase II Rsch. Triangle Park, NC (888) 825-5249

GSK1223249 GlaxoSmithKline mAb multiple sclerosis Phase I Rsch. Triangle Park, NC (see also neurologic) (888) 825-5249

Humira® Abbott Laboratories mAb rheumatoid arthritis Phase III adalimumab Abbott Park, IL (combination therapy) (847) 937-6100 (see also digestive, other)

IL-17 antibody Eli Lilly mAb rheumatoid arthritis Phase II (ixekizumab) Indianapolis, IN (see also skin) (800) 545-5979

Ilaris® Novartis Pharmaceuticals mAb systemic juvenile idiopathic arthritis Phase III canakinumab East Hanover, NJ (see also cardiovascular, diabetes, (888) 669-6682 (Orphan Drug) musculoskeletal, respiratory)

interferon beta-1-a EMD Serono interferon multiple sclerosis Phase I extended release Rockland, MA (800) 283-8088 Flamel Technologies Cedex, France

Medicines in Development Biotechnology 2011 5 Biotechnology Medicines in Development

Autoimmune Disorders

Product Name Sponsor Product Indication Development Phase Category

KB003 KaloBios Pharmaceuticals mAb rheumatoid arthritis Phase II Palo Alto, CA (650) 843-1897

MEDI-545 AstraZeneca mAb SLE Phase II (sifalimumab) Wilmington, DE (see also other) (800) 236-9933 Medarex (609) 430-2880 Princeton, NJ (301) 398-0000 MedImmune Gaithersburg, MD

MEDI-546 AstraZeneca mAb scleroderma Phase I (anti-IFNalphaR mAb) Wilmington, DE (800) 236-9933 Medarex (301) 298-0000 Princeton, NJ MedImmune Gaithersburg, MD

MEDI-551 AstraZeneca mAb scleroderma Phase I (anti-CD19 mAb) Wilmington, DE (see also cancer) (800) 236-9933 MedImmune (301) 298-0000 Gaithersburg, MD

MEDI-570 AstraZeneca mAb SLE Phase I (anti-ICOS mAb) Wilmington, DE (800) 236-9933 MedImmune (301) 298-0000 Gaithersburg, MD

NN8209 Novo Nordisk mAb rheumatoid arthritis Phase II (anti-C5aR) Princeton, NJ (609) 987-5800

NN8226 Novo Nordisk mAb rheumatoid arthritis Phase II (anti-IL-20 antibody) Princeton, NJ (609) 987-5800

NN8555 Novo Nordisk mAb rheumatoid arthritis Phase II Princeton, NJ (see also digestive) (609) 987-5800

NN8828 Novo Nordisk mAb rheumatoid arthritis Phase I (anti-IL-21 mAb) Princeton, NJ (609) 987-5800

Nplate™ Amgen recombinant immune thrombocytopenic Phase III romiplostim Thousand Oaks, CA fusion purpura in children (800) 772-6436 (Orphan Drug) protein (see also cancer)

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Autoimmune Disorders

Product Name Sponsor Product Indication Development Phase Category

NU-100 Nuron Biotech interferon multiple sclerosis Phase I (interferon beta-1b) Exton, PA (610) 968-6700

ocrelizumab Biogen Idec mAb primary progressive multiple Phase III (2nd generation Weston, MA sclerosis (781) 484-2000 anti-CD20) Genentech (800) 626-3553 South San Francisco, CA ------relapsing-remitting multiple Phase II sclerosis (781) 484-2000 (800) 626-3553

olokizumab UCB mAb rheumatoid arthritis Phase II Smyrna, GA (subcutaneous) (770) 970-7500 ------rheumatoid arthritis Phase I/II completed (intravenous) (770) 970-7500

Orencia® Bristol-Myers Squibb recombinant rheumatoid arthritis, SLE in clinical trials abatacept Princeton, NJ protein (800) 332-2056

otelixizumab GlaxoSmithKline mAb myasthenia gravis, Phase II (anti-CD3) Rsch. Triangle Park, NC rheumatoid arthritis (888) 825-5249 Tolerx (see also skin) (617) 354-8100 Cambridge, MA ------Grave’s eye disease Phase I (888) 825-5249 (617) 354-8100

ozoralizumab Pfizer mAb rheumatoid arthritis Phase II (ATN-103) New York, NY (see also digestive, skin) (860) 732-5156 ------ankylosing spondylitis Phase I (860) 732-5156

PDA-001 Celgene Cellular Therapeutics stem cell multiple sclerosis, Phase I/II (cenplacel-L) Summit, NJ therapy rheumatoid arthritis (908) 673-9000 (see also cancer, cardiovascular, digestive)

PEGylated interferon EMD Serono interferon multiple sclerosis Phase I beta-1a Rockland, MA (800) 283-8088

PF-04236921 Pfizer mAb lupus vulgaris, Phase I New York, NY rheumatoid arthritis (860) 732-5156 (see also digestive)

PF-05230905 Pfizer nanobody rheumatoid arthritis Phase I New York, NY (860) 732-5156

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Autoimmune Disorders

Product Name Sponsor Product Indication Development Phase Category

Prolia® Amgen mAb rheumatoid arthritis Phase II completed denosumab Thousand Oaks, CA (see also musculoskeletal) (800) 772-6436

rontalizumab Genentech mAb SLE Phase II (RG7415) South San Francisco, CA (800) 626-3553

SAN-300 Santarus mAb rheumatoid arthritis Phase I (anti-VLA-1 antibody) San Diego, CA (858) 314-5700

SAR153191 sanofi-aventis mAb ankylosing spondylitis, Phase II (anti-IL-6R mAb) Bridgewater, NJ rheumatoid arthritis (800) 633-1610

SBI-087 Emergent BioSolutions peptide rheumatoid arthritis Phase II (PF-05230895) Rockville, MD (301) 795-1800 Pfizer (860) 732-5156 New York, NY ------SLE Phase I (301) 795-1800 (860) 732-5156

secukinumab Novartis Pharmaceuticals mAb multiple sclerosis, Phase II East Hanover, NJ polymyalgia rheumatica, (888) 669-6682 rheumatoid arthritis (see also digestive, eye, skin) ------ankylosing spondylitis Phase I/II (888) 669-6682

Simponi® Janssen Biotech mAb rheumatoid arthritis Phase III golimumab Horsham, PA (intravenous) (800) 526-7736 (Orphan Drug) (see also digestive) ------juvenile rheumatoid arthritis Phase III (subcutaneous) (800) 526-7736 ------sarcoidosis Phase II (subcutaneous) (800) 526-7736

Soliris® Alexion Pharmaceuticals mAb cold agglutinin disease, Phase II eculizumab Cheshire, CT myasthenia gravis (203) 272-2596 (Orphan Drug) (see also blood, eye, neurologic, transplantation, other)

Stelara® Janssen Biotech mAb sarcoidosis Phase II ustekinumab Horsham, PA (see also digestive, skin) (800) 526-7736

Tovaxin® Opexa Therapeutics cell therapy multiple sclerosis Phase II completed autologous T-cell The Woodlands, TX vaccine (281) 272-9331 vaccine

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Autoimmune Disorders

Product Name Sponsor Product Indication Development Phase Category

TRX 1 TolerRx mAb cutaneous lupus erythematosus Phase I completed (anti-CD4 mAb) Cambridge, MA (617) 354-8100

Tysarbi® Biogen Idec mAb multiple sclerosis Phase I natalizumab Weston, MA (intramuscular) (781) 464-2000 ------multiple sclerosis Phase I (subcutaneous) (781) 464-2000

Veldona® Amarillo Biosciences interferon fibromyalgia, polycythemia vera, Phase II human interferon-alpha Amarillo, TX thrombocytosis (806) 376-1741 (Orphan Drug) (see also infectious, respiratory)

veltuzumab Immunomedics mAb immune thrombocytopenic Phase I/II (IMMU-106) Morris Plains, NJ purpura (973) 605-8200 (see also cancer)

VX30 EUSA Pharma mAb rheumatoid arthritis Phase I Langhorne, PA (see also cancer) (800) 833-3533 Vaccinex (585) 271-2700 Rochester, NY

Blood Disorders

Product Name Sponsor Product Indication Development Phase Category

ACE-011 Acceleron Pharma recombinant anemia in patients with Phase II (sotatercept) Cambridge, MA fusion end-stage renal disease (617) 649-9200 Celgene protein (see also cancer, (908) 673-9000 Summit, NJ musculoskeletal, other)

AMT-060 Amsterdam Molecular gene hemophilia B Phase I/II Therapeutics therapy www.amtbiopharma.com Amsterdam, The Netherlands St. Jude Children’s Research Hospital Memphis, TN

anemia protein therapy Medgenics gene anemia Phase I/II Vienna, VA therapy (212) 838-3777

Aranesp® Amgen recombinant anemia in heart failure Phase III darbepoetin alfa Thousand Oaks, CA protein (800) 772-6436

Medicines in Development Biotechnology 2011 9 Biotechnology Medicines in Development

Blood Disorders

Product Name Sponsor Product Indication Development Phase Category

ART-123 Artisan Pharma recombinant disseminated intravascular Phase II completed (recombinant human Waltham, MA protein coagulation (781) 419-1919 thrombomodulin alpha)

ATryn® GTC Biotherapeutics recombinant heparin resistance in patients Phase III antithrombin alfa Framingham, MA protein undergoing CABG surgery (508) 620-9700

BAX-326 Baxter International recombinant hemophilia B Phase I (rFIX) Deerfield, IL protein (800) 422-9837

BAY 86-6150 Bayer HealthCare recombinant hemophilia A, hemophilia B Phase I Pharmaceuticals protein (888) 842-2937 Wayne, NJ

BAY 94-9027 Bayer HealthCare recombinant hemophilia A Phase I Pharmaceuticals protein (888) 842-2937 Wayne, NJ

Benlysta® GlaxoSmithKline mAb vasculitis Phase I belimumab Rsch. Triangle Park, NC (see also autoimmune, (888) 825-5249 (oral) Human Genome Sciences transplantation) (301) 309-8504 Rockville, MD

beta-globin bluebird bio gene beta-thalassemia, Phase I/II gene therapy Cambridge, MA therapy sickle cell anemia (617) 491-5601 INSERM Paris, France

Bosatria™ GlaxoSmithKline mAb Churg-Strauss syndrome Phase I/II mepolizumab Rsch. Triangle Park, NC (see also respiratory) (888) 825-5349 (anti-IL5 mAb)

CNTO-530 Edison Pharmaceuticals recombinant hemoglobinopathies in clinical trials Mountain View, CA fusion (see also other) www.edisonpharma.com protein

CSL-654 CSL Behring recombinant hemophilia B Phase I King of Prussia, PA fusion (610) 878-4000 protein

CYT-107 Cytheris interleukin idiopathic CD4-positive Phase I/II (interleukin-7) Rockville, MD T-lymphocytopenia (301) 231-0450 (see also cancer, HIV, transplantation)

erythropoietin Hanmi Pharmaceutical growth anemia Phase I long-acting Seoul, South Korea factor www.hanmipharm.com

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Blood Disorders

Product Name Sponsor Product Indication Development Phase Category

factor VIII/ CSL Behring recombinant hemophilia A, Phase III von Willebrand King of Prussia, PA protein von Willebrand disease (610) 878-4000 factor

human Octapharma USA recombinant hemophilia A Phase II recombinant Hoboken, NJ protein (201) 604-1130 factor VIII

IB1001 Inspiration Biopharmaceuticals recombinant hemophilia B in infants and Phase III Laguna Niguel, CA protein children www.inspirationbio.com ------hemophilia B Phase II/III www.inspirationbio.com

NN7008 Novo Nordisk recombinant hemophilia A Phase III (octocog alfa) Princeton, NJ protein (800) 727-6500

NN7088 Novo Nordisk recombinant hemophilia A Phase I (N8-GP, rFVIII Princeton, NJ protein (800) 727-6500 glycopegylated)

NN7128 Novo Nordisk recombinant hemophilia with inhibitors Phase II (N7-GP, rFVIIa Princeton, NJ protein (609) 987-5800 glycopegylated)

NN7129 Novo Nordisk recombinant hemophilia with inhibitors Phase I (subcutaneous, Princeton, NJ protein (800) 727-6500 N7-GP, rFVIIa glycopegylated)

NN7999 Novo Nordisk recombinant hemophilia B Phase III (N9-GP, rFIX Princeton, NJ protein (800) 727-6500 glycopegylated)

OBI-1 Inspiration Biopharmaceuticals recombinant hemophilia A Phase III (recombinant Laguna Nigel, CA protein www.inspirationbio.com porcine factor VIII)

recombinant Biogen Idec recombinant hemophilia A Phase II/III factor VIII-Fc Weston, MA fusion (781) 484-2000 (Orphan Drug) protein

Medicines in Development Biotechnology 2011 11 Biotechnology Medicines in Development

Blood Disorders

Product Name Sponsor Product Indication Development Phase Category

recombinant Biogen Idec recombinant hemophilia B Phase II/III factor IX-Fc Weston, MA fusion (781) 484-2000 (Orphan Drug) protein

recombinant Novo Nordisk recombinant congenital factor XIII deficiency application submitted factor XIII Princeton, NJ protein (800) 727-6500 (rFXIII) ------congenital factor XIII deficiency Phase III in children and infants (800) 727-6500 ------postoperative hemorrhage Phase II (800) 727-6500

recombinant Baxter International recombinant von Willebrand disease Phase I von Willebrand Deerfield, IL protein (800) 422-9837 factor

SelG1 Selexys Pharmaceuticals mAb sickle cell anemia Phase I Oklahoma City, OK (405) 319-8195

Soliris® Alexion Pharmaceuticals mAb catastrophic antiphospholid Phase II eculizumab Cheshire, CT syndrome (203) 272-2596 (see also autoimmune, eye, neurologic, transplantation, other)

von Willebrand GTC Biotherapeutics recombinant von Willebrand disease Phase II factor Framingham, MA protein (508) 620-9700 LFB Group Cedex, France

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

8H9 mAb United Therapeutics mAb brain cancer, peritoneal Phase I Silver Spring, MD cancer, soft tissue sarcoma (301) 608-9292

A33 mAb Life Science Pharmaceuticals mAb colorectal cancer Phase I Greenwich, CT (203) 422-6500

abagovomab Menarini mAb ovarian cancer Phase III (Orphan Drug) Florence, Italy vaccine www.menarini.com

ABIO-0501 Abiogen Pharma cell therapy chronic myeloid leukemia (CML) Phase II Pisa, Italy www.abiogen.it

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Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

Ablvax™ Breakthrough Therapeutics peptide CML Phase II VAX100 Greenwich, CT vaccine

ACE-011 Acceleron Pharma recombinant chemotherapy-induced Phase II/III (sotatercept) Cambridge, MA fusion anemia (617) 649-9200 Celgene protein (see also blood, musculoskeletal, (908) 673-9000 Summit, NJ other) ------cancer metastases from Phase II solid tumors (617) 649-9200 (908) 673-9000

adecatumumab EMD Serono mAb breast cancer, colorectal cancer Phase II (MT-201) Rockland, MA (800) 801-8088 Micromet (240) 752-1420 Bethesda, MD

ADXS 11001 Advaxis vaccine cervical intraepithelial neoplasia Phase II Princeton, NJ (609) 452-9813

AE37 Antigen Express peptide breast cancer Phase II Worcester, MA vaccine (508) 852-8783 ------ovarian cancer Phase I (508) 852-8783

AEG 35156 Aegera Therapeutics antisense acute myeloid leukemia (AML) Phase II Montreal, Quebec (514) 288-5532 Idera Pharmaceuticals (617) 679-5500 Cambridge, MA ------B-cell lymphoma, chronic Phase I/II lymphocytic leukemia (CLL) (514) 288-5532 (617) 679-5500

aflibercept Regeneron Pharmaceuticals recombinant second-line metastatic colorectal Phase III (VEGF trap) Tarrytown, NY fusion cancer, second-line non-small-cell (877) 734-6777 sanofi-aventis protein lung cancer (NSCLC), first-line (800) 633-1610 Bridgewater, NJ metastatic prostate cancer ------first-line colorectal cancer Phase II (877) 734-6777 (800) 633-1610

AGS-003 Argos Therapeutics dendritic cell metastatic renal cancer Phase II Durham, NC vaccine (919) 287-6300 Kyowa Hakko Kirin Tokyo, Japan

Medicines in Development Biotechnology 2011 13 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

AGS-005 Argos Therapeutics dendritic cell CLL Phase I/II Durham, NC vaccine (919) 287-6300 Kyowa Hakko Kirin Tokyo, Japan

AGS-1C4D4 Agensys mAb pancreatic cancer Phase II (anti-PSCA mAb) Santa Monica, CA (310) 820-8029

ALD-151 Aldagen stem cell leukemia Phase I Durham, NC therapy (919) 484-2571

AlloStim™ Immunovative Therapies cell vaccine hematological malignancies, Phase I/II cancer vaccine Shoham, Israel solid tumors www.immunovative.co.il

Allovectin® Vical DNA malignant melanoma Phase III velimogene San Diego, CA immuno- (Fast Track) (858) 646-1100 aliplasmid therapeutic (Orphan Drug)

ALN-VSP Alnylam Pharmaceuticals RNA liver cancer, solid tumors Phase I Cambridge, MA interference (617) 551-8200

ALT-801 Altor BioScience recombinant malignant melanoma, Phase I/II Miramar, FL fusion urogenital cancer (954) 443-8600 protein

ALT-836 Altor BioScience mAb cancer Phase I/II Miramar, FL (see also respiratory) (954) 443-8600

ALXN-6000 Alexion Pharmaceuticals mAb CLL, multiple myeloma Phase I/II (samalizumab) Cheshire, CT (203) 272-2596

amatuximab Morphotek mAb mesothelioma Phase II (MORAb-009) Exton, PA (610) 423-6100

AME-133v MENTRAK Biotech mAb non-Hodgkin’s lymphoma, Phase II completed Dallas, TX urogenital cancer (214) 593-0500

AMG 386 Amgen recombinant fallopian tube cancer, Phase III Thousand Oaks, CA fusion ovarian cancer, peritoneal cancer (800) 772-6436 protein ------breast cancer, colorectal cancer, Phase II gastric cancer, liver cancer, (800) 772-6436 esophageal cancer, renal cancer

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Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

AMG 479 Amgen mAb pancreatic cancer Phase III (ganitumab) Thousand Oaks, CA (800) 772-6436 ------breast cancer, colorectal cancer, Phase II sarcoma (800) 772-6436 ------small-cell lung cancer (SCLC), Phase I/II solid tumors (800) 772-6436

AMG 655 Amgen mAb colorectal cancer Phase II (conatumumab) Thousand Oaks, CA (800) 772-6436 ------pancreatic cancer, sarcoma, Phase I/II solid tumors (800) 772-6436

AMG 780 Amgen antibody solid tumors Phase I Thousand Oaks, CA (800) 772-6436

AMG 820 Amgen antibody solid tumors Phase I Thousand Oaks, CA (800) 772-6436

AMG 888 Amgen mAb NSCLC Phase II (U3-1287) Thousand Oaks, CA (800) 772-6436 U3 Pharma Martinsried, Germany

anti-CD3-anti-HER2/ TransTarget cell metastatic breast cancer Phase II neu-activated T-cells Burlingame, CA therapy

anti-CD22/CD19 Abiogen Pharma mAb acute lymphocytic leukemia (ALL) Phase I mAb-toxin conjugate Pisa, Italy www.abiogen.com University of Texas Southwestern Medical Center Dallas, TX

anti-CD22 mAb- Abiogen Pharma mAb B-cell lymphoma Phase I toxin conjugate Pisa, Italy www.abiogen.com University of Texas Southwestern Medical Center Dallas, TX

anti-CD70 Bristol-Myers Squibb mAb cancer Phase I antibody-drug conjugate Princeton, NJ (800) 332-2056 (ADC) (MDX-1203)

anti-CD137 antibody Bristol-Myers Squibb mAb cancer Phase I (BMS-663513) Princeton, NJ (800) 332-2056

Medicines in Development Biotechnology 2011 15 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

anti-CXCR4 Bristol-Myers Squibb mAb AML Phase I (BMS-936564) Princeton, NJ (800) 332-2056

anti-CXCR4 mAb Eli Lilly mAb cancer Phase I Indianapolis, IN (800) 545-5979

anti-EGFL7 mAb Genentech mAb NSCLC Phase II (RG7414) South San Francisco, CA (800) 626-3553 ------solid tumors Phase I (800) 626-3553

anti-FGFR3 mAb Genentech mAb multiple myeloma, solid tumors Phase I (RG7444) South San Francisco, CA (800) 626-3553

anti-gastrin 17 immunogen Cancer Advances polyclonal gastric cancer, pancreatic cancer Phase III (Orphan Drug) Durham, NC antibody (see also digestive) (919) 361-2162 vaccine

anti-HER3/EGFR Genentech mAb solid tumors Phase I (RG7597) South San Francisco, CA (800) 626-3553

anti-idiotype VaxOnco vaccine prostate cancer Phase II cancer vaccine Seoul, South Korea www.vaxonco.com (105AD7)

anti-IGF-1R mAb Genentech mAb solid tumors Phase I South San Francisco, CA (800) 626-3553

anti-KIR mAb Bristol-Myers Squibb mAb cancer Phase I (IPH-2102) Princeton, NJ (800) 332-2056 Innate Pharma Marseille, France

anti-PD1 Bristol-Myers Squibb mAb advanced solid tumors Phase II (BMS-936558) Princeton, NJ (800) 332-2056

anti-PDL-L1 Bristol-Myers Squibb mAb advanced solid tumors Phase I (BMS-936559) Princeton, NJ (800) 332-2056

ANZ-100 Aduro BioTech vaccine liver cancer Phase I Berkeley, CA (510) 848-4400

APN301 Apeiron Biologics mAb malignant melanoma, Phase II (hu14.18-IL2) Vienna, Austria neuroblastoma in children www.apeironbiologics. com

Archexin™ Rexahn Pharmaceuticals antisense pancreatic cancer Phase II RX-0201 Rockville, MD (240) 268-5300 (Orphan Drug)

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Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

Arzerra® GlaxoSmithKline mAb CLL (first-line therapy), Phase III ofatumumab Rsch. Triangle Park, NC diffuse large B-cell lymphoma (888) 825-5249 (Orphan Drug) (second-line therapy), follicular lymphoma (second-line therapy), non-Hodgkin’s lymphoma (first-line therapy) (see also autoimmune) ------diffuse large B-cell lymphoma Phase II (monotherapy), non-Hodgkin’s (888) 825-5249 lymphoma (first-line therapy), Waldenström’s macroglobulinemia

ASG-5ME Agensys mAb pancreatic cancer, Phase I Santa Monica, CA castration-resistant (310) 820-8029 Seattle Genetics prostate cancer (425) 527-4000 Bothell, WA

ASG-22ME Agensys mAb solid tumors Phase I Santa Monica, CA (310) 820-8029 Seattle Genetics (425) 527-4000 Bothell, WA

astuprotimut-R GlaxoSmithKline recombinant malignant melanoma, NSCLC Phase III Rsch. Triangle Park, NC immuno- (888) 825-5249 therapeutic ------vaccine malignant melanoma Phase II (inoperable/unresectable) (888) 825-5249

autologous idiotype Bayer HealthCare vaccine follicular lymphoma Phase I cancer vaccine Pharmaceuticals (888) 842-2937 Wayne, NJ (805) 488-2147 Stellar Biotechnologies Port Hueneme, CA

autologous tumor cell Champions Oncology vaccine ovarian cancer Phase II vaccine Baltimore, MD (410) 369-0365 ------solid tumors Phase I (410) 369-0365

AV-299 AVEO Pharmaceuticals mAb glioblastoma, lymphoma, Phase I (ficlatuzumab) Cambridge, MA multiple myeloma, solid tumors (617) 299-5000

Avastin® Genentech mAb metastatic breast cancer Phase III bevacizumab and South San Francisco, CA (800) 626-3553 Herceptin® trastuzumab

Medicines in Development Biotechnology 2011 17 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

Avastin® Genentech mAb ovarian cancer application submitted bevacizumab South San Francisco, CA (800) 626-3553 (Orphan Drug) ------breast cancer, carcinoid tumors, Phase III diffuse large B-cell lymphoma, (800) 626-3553 gastric cancer, gliobastoma, head/neck cancer ------cervical cancer, hemangiosarcoma, Phase II malignant melanoma, metastatic (800) 626-3553 brain cancer, glioblastoma, liver cancer, multiple myeloma, non-Hodgkin’s lymphoma ------mesothelioma Phase I/II (800) 626-3553

AVX701 AlphaVax particle prevention of colorectal cancer Phase I/II Rsch. Triangle Park, NC vaccine (919) 595-0400

B7-2/GM-CSF cancer NuVax Therapeutics gene cancer Phase I gene therapy Tustin, CA therapy (714) 505-4461 (combination immunogene therapy)

BAFF antibody Eli Lilly mAb multiple myeloma Phase I (tabalumab) Indianapolis, IN (see also autoimmune, other) (800) 545-5979

BAY 79-4620 Bayer HealthCare mAb solid tumors Phase I Pharmaceuticals (888) 842-2937 Wayne, NJ

BC-819 BioCancell Therapeutics gene bladder cancer Phase II (Orphan Drug) Jerusalem, Israel therapy www.biocancell.com ------ovarian cancer, Phase I/II pancreatic cancer www.biocancell.com

Bexxar® GlaxoSmithKline mAb mantle-cell lymphoma Phase II tositumomab Rsch. Triangle Park, NC (888) 825-5249

BHQ880 Novartis Pharmaceuticals mAb multiple myeloma Phase II East Hanover, NJ (888) 669-6682 ------solid tumors Phase II (888) 669-6682

18 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

BI-505 BioInvent International mAb late-stage multiple myeloma Phase I Lund, Sweden www.bioinvent.com

BIIB 015 Biogen Idec mAb solid tumors Phase I Weston, MA (781) 464-2000

BIIB 022 Biogen Idec mAb liver cancer, NSCLC Phase I Weston, MA (781) 484-2000

BiovaxID® Biovest International autologous follicular lymphoma Phase III B-cell lymphoma Tampa, FL idiotype (Fast Track) (813) 258-6912 vaccine vaccine ------mantle-cell lymphoma Phase II (813) 258-6912

BIW-8962 BioWa mAb multiple myeloma Phase I/II Princeton, NJ (609) 580-7500

BLX 883 Biolex Therapeutics interferon cancer Phase I Pittsboro, NC (see also infectious) (919) 542-9901

BPX-101 Bellicum Pharmaceuticals dendritic cell prostate cancer Phase I/II Houston, TX vaccine www.bellicum.com

breast cancer Quantum Immunologics dendritic cell breast cancer Phase I/II vaccine Tampa, FL vaccine (866) 213-4594

brentuximab vedotin Millennium Pharmaceuticals mAb recurrent Hodgkin’s disease application submitted (Orphan Drug) Cambridge, MA (combination therapy), (800) 390-5663 Seattle Genetics recurrent non-Hodgkin’s (425) 527-4000 Bothell, WA lymphoma ------recurrent Hodgkin’s disease Phase I/II (monotherapy) (800) 390-5663 (Fast Track) (425) 527-4000 ------newly-diagnosed Hodgkin’s Phase I disease, newly-diagnosed (800) 390-5663 non-Hodgkin’s lymphoma (425) 527-4000

BrevaRex™ Quest Pharmatech mAb pancreatic cancer Phase I/II anti-MUC1 Edmonton, Alberta (780) 448-1400 monoclonal cancer antibody

BT-062 Biotest mAb multiple myeloma Phase I/II (Orphan Drug) Dreieich, Germany www.biotest.de

CA9-SCAN Wilex mAb diagnosis of kidney cancer Phase III completed Munich, Germany www.wilex.com

Medicines in Development Biotechnology 2011 19 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

CA-18C3 XBiotech mAb cancer, hematological malignancies Phase I Austin, TX (see also cardiovascular) (512) 386-2900

CALAA-01 Calando Pharmaceuticals RNA solid tumors Phase I Pasadena, CA interference (626) 304-3400

Campath® Genzyme mAb CLL Phase III alemtuzumab Cambridge, MA (see also autoimmune, (617) 252-7500 (intravenous) transplantation) (Orphan Drug)

Campath® Genzyme mAb CLL Phase II alemtuzumab Cambridge, MA (617) 252-7500 (subcutaneous)

cancer vaccine EMD Millipore dendritic cell malignant melanoma Phase I Billerica, MA vaccine (781) 533-6000

cancer vaccine Gradalis genetically- solid tumors Phase I Carrollton, TX modified (214) 442-8100 vaccine

cancer vaccine Immunitor USA vaccine cancer Phase I/II Vancouver, Canada www.immunitor.com

cancer vaccine MabVax Therapeutics mAb sarcoma Phase II San Diego, CA vaccine (858) 259-9405 ------breast cancer Phase I (858) 259-9405

cancer vaccine Mojave Therapeutics recombinant malignant melanoma Phase I/II Hawthorne, NY protein (914) 347-0290 vaccine

cancer vaccine PR1 The Vaccine Company peptide AML Phase III South San Francisco, CA vaccine (650) 244-4100 ------CML, myelodysplastic syndromes Phase II (650) 244-4100

carlumab Janssen Biotech mAb prostate cancer Phase I Horsham, PA (see also respiratory) (800) 526-7736 ------solid tumors Phase I (800) 526-7736

CAT-8015 AstraZeneca mAb hematological malignancies Phase I (moxetumomab Wilmington, DE (800) 236-9933 pasudotox) MedImmune (301) 398-0000 Gaithersburg, MD

20 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

catumaxomab Fresenius Biotech mAb malignant ascites, Phase II North America ovarian cancer www.fresenius-biotech. Waltham, MA com

CB-10-01 Cosmo Pharmaceuticals vaccine malignant melanoma Phase II Lainate, Italy

CDX-011 Celldex Therapeutics mAb breast cancer (Fast Track), Phase II Needham, MA malignant melanoma (781) 433-0771

CDX-1307 Celldex Therapeutics mAb first-line bladder cancer Phase II Needham, MA vaccine (781) 433-0771 ------breast cancer, colorectal cancer, Phase I ovarian cancer, pancreatic cancer (781) 433-0771

CDX-1401 Celldex Therapeutics mAb solid tumors Phase I/II Needham, MA vaccine (781) 433-0771

cell therapy Cell Medica cell cancer Phase II London, United Kingdom therapy (see also infectious) www.cellmedica.co.uk Center for Cell and Gene Therapy (713) 798-1246 Houston, TX

CG-201 CG Therapeutics antibody solid tumors Phase II Seattle, WA vaccine (206) 336-5604

ch14.18 mAb United Therapeutics mAb neuroblastoma in children Phase III Silver Spring, MD (301) 608-9292

Chemophase™ Halozyme Therapeutics recombinant superficial bladder cancer Phase I/II completed recombinant San Diego, CA protein (858) 794-8889 hyaluronidase

chronic MaxCyte cell CLL Phase I/II lymphocytic Gaithersburg, MD vaccine (301) 944-1700 leukemia cell therapy

cintredekin Insys Therapeutics recombinant glioma in adolescents and children Phase I besudotox Lake Forest, IL protein (847) 295-8678 (IL13-PE38QQR) (Orphan Drug)

cixutumumab Eli Lilly mAb cancer Phase II (IMC-A12) Indianapolis, IN (800) 545-5979 ImClone Systems (212) 645-1405 New York, NY

Medicines in Development Biotechnology 2011 21 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

CLT 001 Cellerant Therapeutics cell cancer Phase II San Carlos, CA therapy (650) 232-2122

CLT 008 Cellerant Therapeutics cell neutropenia Phase I/II San Carlos, CA therapy (650) 232-2122 ------hematological malignancies Phase I (650) 232-2122

CNTO-95 Janssen Biotech mAb prostate cancer Phase II completed (intetumumab) Horsham, PA (800) 526-7736 ------malignant melanoma Phase I/II (800) 526-7736 ------solid tumors Phase I completed (800) 526-7736

CNTO-328 Janssen Biotech mAb giant lymph node hyperplasia, Phase II Horsham, PA multiple myeloma (800) 526-7736 ------prostate cancer Phase I (800) 526-7736

Cotara™ Peregrine Pharmaceuticals mAb glioblastoma Phase II mAb TNT Tustin, CA (Fast Track) (714) 508-6000 (Orphan Drug)

CRS-207 Aduro Biotech genetically- solid tumors Phase I Berkeley, CA modified (510) 848-4400 vaccine

CT-011 CureTech mAb AML, colorectal cancer, diffuse Phase II Yavne, Israel large B-cell lymphoma, follicular www.curebiotech.com lymphoma

custirsen OncoGenex Technologies antisense prostate cancer Phase III (OGX-011) Vancouver, Canada (Fast Track) (604) 736-3678 Teva Pharmaceuticals USA (215) 591-3000 North Wales, PA ------NSCLC Phase II (604) 736-3678 (215) 591-3000

22 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

CVac™ Prima BioMed dendritic cell ovarian cancer Phase II cancer vaccine MUC-1 Melbourne, Australia vaccine www.primabiomed. com.au

CYT-107 Cytheris interleukin solid tumors Phase I/II (interleukin-7) Rockville, MD (see also blood, HIV, (301) 231-0450 transplantation) ------malignant melanoma, myeloid Phase I leukemia, renal cancer (301) 231-0450

CYT-500 EUSA Pharma mAb prostate cancer Phase I Langhorne, PA (800) 833-3533

CYT-6091 Cytimmune Sciences tumor solid tumors Phase I Rockville, MD necrosis (301) 825-3041 factor

dalotuzumab Merck mAb neuroendocrine tumors, Phase II (MK-0646) Whitehouse Station, NJ NSCLC (800) 672-6372 ------breast cancer, pancreatic cancer Phase I/II (800) 672-6372

DCVax®-Brain Northwest Biotherapeutics dendritic cell glioblastoma Phase II brain cancer vaccine Bethesda, MD vaccine (240) 497-9024 (Orphan Drug)

DCVax®-L Northwest Biotherapeutics dendritic cell ovarian cancer, peritoneal cancer Phase I/II ovarian cancer vaccine Bethesda, MD vaccine (240) 497-9024

DCVax®-Prostate Northwest Biotherapeutics dendritic cell prostate cancer Phase III prostate cancer vaccine Bethesda, MD vaccine (240) 497-9024

DPX-0907 Immunovaccine vaccine breast cancer, ovarian cancer, Phase I Halifax, Canada prostate cancer www.imvaccine.com

E7777 Eisai fusion peripheral T-cell lymphoma Phase III (denileukin diftitox) Woodcliff Lake, NJ protein (877) 873-4724

EC-17 Endocyte vaccine renal cancer Phase I West Lafayette, IN (combination therapy) (765) 463-7175

ecromeximab Life Science Pharmaceuticals mAb malignant melanoma Phase II Greenwich, CT (203) 422-6500

Medicines in Development Biotechnology 2011 23 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

EGEN-001 EGEN gene fallopian tube cancer, Phase II (Orphan Drug) Huntsville, AL therapy ovarian cancer, peritoneal cancer (256) 512-0988

elotuzumab Bristol-Myers Squibb mAb multiple myeloma Phase III Princeton, NJ (800) 332-2056 Facet Biotech (650) 454-1000 Redwood City, CA

EMD 525797 EMD Serono mAb prostate cancer Phase II/III (DI17E6) Rockland, MA (800) 283-8088

enavatuzumab Facet Biotech mAb solid tumors Phase I Redwood City, CA (650) 454-1000

encapsulated cell therapy Metromedia Bio-Science cell colorectal cancer, pancreatic Phase II New York, NY therapy cancer, prostate cancer (212) 746-1552 Rogosin Institute New York, NY

encapsulated cell therapy Nuvilex cell pancreatic cancer Phase II Scottsdale, AZ therapy (480) 348-8050

ensituximab Neogenix Oncology mAb metastatic colorectal cancer, Phase I Great Neck, NY metastatic pancreatic cancer (516) 482-1200

epratuzumab Immunomedics mAb ALL in children, diffuse large B-cell Phase II (Orphan Drug) Morris Plains, NJ lymphoma, follicular lymphoma (973) 605-8200 UCB (see also autoimmune) Smyrna, GA

Erbitux® Bristol-Myers Squibb mAb colorectal cancer (1st and 3rd-line), in clinical trials cetuximab Princeton, NJ head/neck cancer, NSCLC (800) 332-2056 Eli Lilly (1st and 2nd-line), pediatrics (800) 545-5979 Indianapolis, IN ImClone Systems New York, NY

ertumaxomab Fresenius Biotech North America mAb breast cancer Phase II Waltham, MA www.fresenius-biotech. TRION Pharma com Munich, Germany

24 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

ETBX-011 Etubics vector solid tumors Phase I Seattle, WA vaccine (206) 838-5110

EZN-2968 Enzon Pharmaceuticals antisense lymphoma, solid tumors Phase I Piscataway, NJ (732) 980-4500

EZN-4176 Enzon Pharmaceuticals antisense prostate cancer Phase I Piscataway, NJ (732) 980-4500

F50035 Merck mAb colorectal cancer, NSCLC Phase II Whitehouse Station, NJ (800) 672-6372 Pierre Fabre Paris, France

farletuzumab Morphotek mAb ovarian cancer Phase III (MORAb-003) Exton, PA (610) 423-6100 (Orphan Drug)

FG-3019 FibroGen mAb pancreatic cancer Phase II South San Francisco, CA (see also respiratory) (650) 866-7200

Fibrovax™ Cytokine PharmaSciences fibrocyte cancer Phase I cancer vaccine King of Prussia, PA vaccine (610) 687-1776

FP-1039 Five Prime Therapeutics recombinant endometrial cancer Phase II San Francisco, CA fusion (415) 365-5600 Human Genome Sciences protein (301) 309-8504 Rockville, MD ------late-stage solid tumors Phase I (415) 365-5600 (301) 309-8504

galiximab Biogen Idec mAb non-Hodgkin’s lymphoma Phase II (anti-CD80 mAb) Weston, MA (781) 464-2000

GC-33/RG7686 Chugai Pharma USA mAb liver cancer Phase I Berkeley Heights, NJ (908) 516-1350 Roche (973) 235-5000 Nutley, NJ

Medicines in Development Biotechnology 2011 25 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

GC-1008 Genzyme mAb malignant melanoma, Phase I (fresolimumab) Cambridge, MA renal cancer (617) 252-7500 (see also respiratory, other)

Genasense® Genta antisense CLL (combination therapy) application submitted oblimersen Berkeley Heights, NJ (Fast Track) (908) 286-9800 (Orphan Drug) ------AML, malignant melanoma Phase III (Fast Track) (908) 286-9800 ------NSCLC Phase II/III (combination therapy) (908) 286-9800 ------breast cancer, Phase II non-Hodgkin’s lymphoma (908) 286-9800 ------solid tumors Phase I/II (brief, high-dose IV-fusion) (908) 286-9800 ------solid tumors Phase I (intermittent therapy) (908) 286-9800

GI-4000 GlobeImmune genetically- colorectal cancer, Phase II Louisville, CO modified pancreatic cancer (303) 625-2700 vaccine ------NSCLC Phase I (303) 625-2700

GI-6207 GlobeImmune genetically- cancer Phase II Louisville, CO modified (303) 625-2700 vaccine

GliAtak™ Advantagene gene glioma Phase II cancer gene Boston, MA therapy (617) 916-5445 therapy San Diego, CA (760) 943-8981 (Orphan Drug) ------brain cancer in children Phase I (617) 916-5445 (760) 943-8981

GRNVAC1 Argos Therapeutics dendritic cell AML Phase II Durham, NC vaccine (919) 287-6300 Geron (650) 473-7700 Menlo Park, CA ------malignant melanoma, Phase I/II prostate cancer (919) 287-6300 (650) 473-7700

26 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

GS 6624 Gilead Sciences mAb solid tumors Phase I Foster City, CA (see also respiratory, other) (800) 445-3235

GSK2586881 Apeiron Biologics recombinant cancer Phase I (APN01) Vienna, Austria protein (see also cardiovascular, (888) 825-5249 GlaxoSmithKline diabetes, respiratory) Rsch. Triangle Park, NC

GSK2130597A GlaxoSmithKline recombinant AML Phase II Rsch. Triangle Park, NC vaccine (888) 825-5249

GSK2132231A GlaxoSmithKline recombinant malignant melanoma, NSCLC Phase III (MAGE-A3 antigen cancer Rsch. Triangle Park, NC vaccine (888) 825-5249 immunotherapeutic)

GSK2241658A GlaxoSmithKline recombinant malignant melanoma Phase I (NY-ESO-1 antigen- Rsch. Triangle Park, NC vaccine (888) 825-5249 specific cancer immunotherapeutic)

GSK2302025A GlaxoSmithKline vaccine malignant melanoma Phase I Rsch. Triangle Park, NC (888) 825-5249

GSK2302032A GlaxoSmithKline recombinant malignant melanoma, NSCLC Phase I (PRAME antigen cancer Rsch. Triangle Park, NC vaccine (888) 825-5249 immunotherapeutic)

GVAX® Breast BioSante Pharmaceuticals genetically- breast cancer Phase I cancer immunotherapy Lincolnshire, IL modified (847) 478-0500 vaccine

GVAX® Colorectal BioSante Pharmaceuticals genetically- colorectal cancer Phase I cancer immunotherapy Lincolnshire, IL modified (847) 478-0500 vaccine

GVAX® Leukemia BioSante Pharmaceuticals genetically- AML, CML Phase II cancer immunotherapy Lincolnshire, IL modified (847) 478-0500 (Orphan Drug) vaccine

GVAX® Multiple BioSante Pharmaceuticals genetically- multiple myeloma Phase II Myeloma Lincolnshire, IL modified (847) 478-0500 cancer vaccine immunotherapeutic

GVAX® Pancreatic BioSante Pharmaceuticals genetically- pancreatic cancer Phase II cancer immunotherapy Lincolnshire, IL modified (847) 478-0500 (Orphan Drug) vaccine

Medicines in Development Biotechnology 2011 27 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

GVAX® Prostate BioSante Pharmaceuticals genetically- prostate cancer Phase III cancer immunotherapy Lincolnshire, IL modified (Fast Track) (847) 478-0500 vaccine

HER2-antigen- GlaxoSmithKline recombinant breast cancer Phase II specific cancer Rsch. Triangle Park, NC protein (888) 825-5249 immunotherapeutic

Herceptin® SC Halozyme mAb HER2-positive breast cancer Phase III trastuzumab/rHuPH20 San Diego, CA (858) 794-8889 (subcutaneous) Roche (973) 235-5000 Nutley, NJ

HGS-ETR1 Human Genome Sciences mAb multiple myeloma, NSCLC Phase II (mapatumumab) Rockville, MD (301) 309-8504 ------liver cancer Phase I/II (301) 309-8504

HIV stem cell therapy Benitec stem cell AIDS-related lymphoma Phase I Melbourne, Australia therapy (see also HIV) www.benitec.com City of Hope National (626) 256-4673 Medical Center Duarte, CA

HM10460A Hanmi Pharmaceutical growth neutropenia in cancer patients Phase I (granulocyte colony- Seoul, South Korea factor www.hanmipharm.com stimulating factor, long-acting)

HS-L1 Heat Biologics genetically- NSCLC Phase I Miami Beach, FL modified (305) 432-4684 vaccine

hTERT cancer Merck DNA cancer Phase I vaccine Whitehouse Station, NJ vaccine (800) 632-0000 Vical (858) 646-1100 San Diego, CA

HuM195/rGel Targa Therapeutics mAb AML, CML, myelodysplastic Phase I San Diego, CA syndromes (858) 483-6600

huN901-DM1 ImmunoGen mAb SCLC Phase II (lorvotuzumab Cambridge, MA (617) 995-2500 mertansine) ------(Orphan Drug) multiple myeloma, solid tumors Phase I (617) 995-2500

28 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

HyperAcute® Lung NewLink Genetics DNA NSCLC Phase II lung cancer vaccine Ames, IA vaccine (515) 296-5555

HyperAcute® Melanoma NewLink Genetics DNA malignant melanoma Phase II melanoma cancer vaccine Ames, IA vaccine (515) 296-5555

HyperAcute® Pancreas NewLink Genetics DNA pancreatic cancer Phase III algenpantucel-L Ames, IA vaccine (515) 296-5555 (Orphan Drug)

iboctadekin GlaxoSmithKline recombinant non-Hodgkin’s lymphoma, Phase I completed Rsch. Triangle Park, NC human ovarian cancer (888) 825-5249 cytokine

icrucumab Eli Lilly mAb cancer Phase II (IMC-18F1) Indianapolis, IN (800) 545-5979 ImClone Systems (212) 645-1405 New York, NY

ICT-107 ImmunoCellular Therapeutics dendritic cell glioblastoma Phase II Los Angeles, CA vaccine (818) 992-2907

IDM-2101 Biotech Synergy vaccine NSCLC Phase II San Diego, CA (858) 459-6689

IL-21 Bristol-Myers Squibb interleukin solid tumors Phase I Princeton, NJ (800) 332-2056

IMA-901 immatics biotechnologies peptide renal cancer treatment Phase III (intradermal vaccine) Tübingen, Germany vaccine www.immatics.com

IMC-3C5 Eli Lilly mAb solid tumors Phase I Indianapolis, IN (800) 545-5979 ImClone Systems (212) 645-1405 New York, NY

IMC-3G3 Eli Lilly mAb cancer Phase II (olaratumab) Indianapolis, IN (800) 545-5979 ImClone Systems (212) 645-1405 New York, NY

IMC-20D7S Eli Lilly mAb cancer Phase I Indianapolis, IN (800) 545-5979 ImClone Systems (212) 645-1405 New York, NY

Medicines in Development Biotechnology 2011 29 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

IMC-CS4 Eli Lilly mAb solid tumors Phase I Indianapolis, IN (800) 545-5979 ImClone Systems (212) 645-1405 New York, NY

IMC-gp100 Immunocore recombinant malignant melanoma treatment Phase 0 Oxon, United Kingdom vaccine www.immunocore.com

IMC-RON8 Eli Lilly mAb solid tumors Phase I (narnatumab) Indianapolis, IN (800) 545-5979 ImClone Systems (212) 645-1405 New York, NY

IMF-001 ImmunoFrontier recombinant solid tumors Phase I Tokyo, Japan vaccine www.immunofrontier. com

IMMU-102 Immunomedics mAb non-Hodgkin’s lymphoma Phase I/II (epratuzumab Y-90) Morris Plains, NJ (973) 605-8200

IMMU-105 Immunomedics mAb liver cancer Phase I (tacatuzumab Y-90) Morris Plains, NJ (973) 605-8200

IMMU-111 Immunomedics mAb colorectal cancer Phase II (labetuzumab I-131) Morris Plains, NJ (973) 605-8200

IMP-321 Immutep cell pancreatic cancer Phase I Châtenay-Malabry, France therapy www.immutep.com

IMT-1012 Immunotope peptide breast cancer, ovarian cancer Phase I Doylestown, PA immuno- (215) 253-4180 therapeutic vaccine

INNO-305 CytRx peptide AML, myelodysplastic Phase I immunotherapeutic Los Angeles, CA vaccine syndromes, solid tumors (301) 826-5648

30 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

inotuzumab UCB mAb aggressive non-Hodgkin’s Phase III ozogamicin Smyrna, GA lymphoma (770) 970-7500 (CMC-544) Pfizer (860) 732-5156 New York, NY ------diffuse large B-cell lymphoma, Phase II indolent non-Hodgkin’s (770) 970-7500 lymphoma (860) 732-5156 ------ALL Phase I (770) 970-7500 (860) 732-5156

INSC01 Insception Biosciences stem cell hematologic disorders Phase II Mississauga, Ontario therapy (866) 606-2790

interferon Helix BioPharma interferon cervical dysplasia Phase II completed alpha-2b Aurora, Canada (see also infectious) (905) 841-2300 topical Merck (800) 672-6372 Whitehouse Station, NJ

interleukin-12 OncoSec Medical gene malignant melanoma Phase I gene therapy San Diego, CA therapy www.oncosec.com

ISF35 Memgen gene CLL, non-Hodgkin’s lymphoma Phase I Dallas, TX vaccine (214) 731-3141

ISIS-EIF4ERx Eli Lilly antisense solid tumors Phase II (EIF-4E ASO) Indianapolis, IN (800) 545-5979 Isis Pharmaceuticals (800) 679-4747 Carlsbad, CA

iSONEP™ Lpath mAb solid tumors Phase I completed sonepcizumab San Diego, CA (see also eye) (858) 678-0800 (intravenous)

JVRS-100 Juvaris BioTherapeutics DNA leukemia Phase I immunotherapeutic Burlingame, CA vaccine (650) 259-1800

KB004 KaloBios Pharmaceuticals mAb hematological malignancies Phase I Palo Alto, CA (650) 843-1897

KHK-2866 Kyowa Hakko Kirin Pharma mAb ovarian cancer, solid tumors Phase I Princeton, NJ (609) 919-1100

Medicines in Development Biotechnology 2011 31 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

KRN-330 Kyowa Hakko Kirin Pharma mAb colorectal cancer Phase I/II Princeton, NJ (609) 919-1100

L19-131I Bayer HealthCare mAb brain cancer, hematological Phase III Pharmaceuticals malignancies, solid tumors (888) 842-2937 Wayne, NJ ------Philogen NSCLC Phase I Siena, Italy (888) 842-2937

L19-TNF Bayer HealthCare recombinant malignant melanoma Phase II Pharmaceuticals fusion (888) 842-2937 Wayne, NJ protein ------Philogen solid tumors Phase I/II Siena, Italy (888) 842-2937 ------colorectal cancer Phase I completed (888) 842-2937

leukemia DNA vaccine Inovio Pharmaceuticals DNA AML, CML Phase II Blue Bell, PA vaccine (267) 440-4200 University of Southampton Hampshire, United Kingdom

Leukine® Bayer HealthCare granulocyte- CLL, malignant melanoma, Phase II sargramostim Pharmaceuticals macrophage non-Hodgkin’s lymphoma, (888) 842-2937 Wayne, NJ colony ovarian cancer, prostate cancer

LG740 Lentigen gene ALL, B-cell prolymphocytic Phase I (activated T-cells) Gaithersburg, MD therapy leukemia, CLL, diffuse large (301) 527-4200 University of Pennsylvania B-cell lymphoma, Philadelphia, PA follicular lymphoma, mantle-cell lymphoma

lintuzumab Ac-225 Actinium Pharmaceuticals mAb AML Phase I Florham Park, NY (973) 377-0713

lintuzumab Bi-213 Actinium Pharmaceuticals mAb AML Phase I/II Florham Park, NY (973) 377-0713

LOR-2040 Lorus Therapeutics antisense AML, metastatic breast cancer, Phase II (Orphan Drug) Toronto, Canada colorectal cancer, metastatic (416) 798-1200 renal cancer ------CML, myelodysplastic syndromes Phase I (416) 798-1200

Lucanix® NovaRx genetically- NSCLC Phase III belagen- San Diego, CA modified (Fast Track) (858) 552-8600 pumatucel-L cell-based ------vaccine glioma Phase I (858) 552-8600

32 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

lucatumumab Novartis Pharmaceuticals mAb lymphoma Phase I/II (CHIR 12.12) East Hanover, NJ (888) 669-6682 XOMA (510) 204-7200 Berkeley, CA ------follicular lymphoma Phase I (888) 669-6682 (510) 204-7200

lung cancer vaccine AVAX Technologies autologous NSCLC Phase I/II Philadelphia, PA cell vaccine (215) 241-9760

LY2181308 Eli Lilly antisense AML, NSCLC, prostate cancer Phase II (survivan ASO) Indianapolis, IN (800) 545-5979 Isis Pharmaceuticals (800) 679-4747 Carlsbad, CA

MAXY-G34 Maxygen granulocyte chemotherapy-induced Phase I Redwood City, CA colony- neutropenia (650) 298-5300 stimulating factor

MEDI-551 AstraZeneca mAb hematological malignancies Phase I (anti-CD19 mAb) Wilmington, DE (see also autoimmune) (800) 236-9933 MedImmune (301) 298-0000 Gaithersburg, MD

MEDI-573 AstraZeneca mAb solid tumors Phase I (anti-IGF mAb) Wilmington, DE (800) 236-9933 MedImmune (301) 398-0000 Gaithersburg, MD

MEDI-575 AstraZeneca mAb solid tumors Phase II (anti-PDGFRα mAb) Wilmington, DE (800) 236-9933 MedImmune (301) 398-0000 Gaithersburg, MD

MEDI-3617 AstraZeneca mAb solid tumors Phase I (anti-ANG-2 mAb) Wilmington, DE (800) 236-9933 MedImmune (301) 398-0000 Gaithersburg, MD

melanoma vaccine Ichor Medical Systems DNA malignant melanoma Phase I San Diego, CA vaccine (858) 550-2022 Memorial Sloan-Kettering Cancer Center New York, NY

melanoma vaccine Polynoma vaccine malignant melanoma Phase II (POL-103A) San Diego, CA (858) 205-2501

Medicines in Development Biotechnology 2011 33 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

mesenchymal stem Cephalon stem cell for bone marrow regeneration Phase III cell therapy Frazer, PA therapy in patients undergoing bone (610) 344-0200 (Orphan Drug) Mesoblast marrow transplantation Melbourne, Australia (see also cardiovascular, musculoskeletal)

MetmAb Genentech mAb breast cancer, NSCLC Phase II (RG3638) South San Francisco, CA (800) 626-3553

MGA271 MacroGenics mAb solid tumors Phase I Rockville, MD (301) 251-5172

MGAH22 MacroGenics mAb bladder cancer, breast cancer, Phase I (anti-HER2) Rockville, MD gastric cancer, NSCLC, (301) 251-5172 ovarian cancer

milatuzumab Immunomedics mAb CLL, multiple myeloma, Phase I/II (IMMU-115) Morris Plains, NJ non-Hodgkin’s lymphoma (973) 605-8200 (Orphan Drug)

MKC1106-MT Mannkind vaccine treatment of malignant melanoma Phase II Valencia, CA (661) 775-5300

mogamulizumab Kyowa Hakko Kirin Pharma mAb T-cell lymphoma Phase II (KW-0761) Princeton, NJ (609) 919-1100

MORAb-004 Morphotek mAb melanoma Phase II Exton, PA (610) 423-6100

Multikine® CEL-SCI interleukin head/neck cancer Phase III leukocyte Vienna, VA (703) 506-9460 interleukin cancer (Orphan Drug)

MVA-BN® Breast BN ImmunoTherapeutics cell vaccine breast cancer Phase I Mountain View, CA (650) 681-4660

MVA-BN®-PRO BN ImmunoTherapeutics vaccine prostate cancer Phase I/II Mountain View, CA (650) 681-4660

mycobacterium Bioniche Therapeutics myco- superficial (non-muscle invasive) Phase III cell wall-DNA Pointe-Claire, Canada bacterial bladder cancer (514) 697-6636 complex (MCC) Endo Pharmaceuticals DNA (second-line therapy) Chadd’s Ford, PA (Fast Track)

34 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

naptumomab Active Biotech mAb NSCLC (Fast Track), Phase I completed estafenatox Lund, Sweden pancreatic cancer, renal cancer www.activebiotech.com

necitumumab Bristol-Myers Squibb mAb bladder cancer, NSCLC Phase III (IMC-11F8) Princeton, NJ (800) 332-2056 Eli Lilly (800) 545-5979 Indianapolis, IN (212) 645-1405 ImClone Systems New York, NY

NeuVax™ RXi Pharmaceuticals peptide breast cancer Phase II E75 cancer vaccine Worcester, MA vaccine (508) 767-3861 ------prostate cancer Phase I/II (508) 767-3861

Neuvenge™ Dendreon dendritic cell urogenital cancer Phase II lapuleucel-T Seattle, WA vaccine (206) 256-4545 ------breast cancer, colorectal cancer, Phase I completed ovarian cancer (206) 256-4545

nimotuzumab YM Biosciences USA mAb brain cancer, glioma, Phase II Lehigh Valley, PA prostate cancer (610) 560-0600 ------breast cancer Phase I (610) 560-0600

NP-2 Diamyd Medical gene cancer pain Phase II intradermal Pittsburgh, PA therapy (412) 770-1310 gene therapy

Nplate™ Amgen recombinant thrombocytopenia associated Phase II romiplostim Thousand Oaks, CA fusion with chemotherapy (800) 772-6436 protein (see also autoimmune)

OHR118 Ohr Pharmaceutical immune- cachexia associated with cancer Phase II New York, NY based (212) 682-8452 therapy

Medicines in Development Biotechnology 2011 35 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

Omnitarg™ Genentech mAb HER2-positive metastatic breast Phase III pertuzumab South San Francisco, CA cancer (first-line therapy) (800) 626-3553 Roche ------Nutley, NJ breast cancer (second-line Phase II treatment), pancreatic cancer (800) 626-3553 ------colorectal cancer Phase I/II (800) 626-3553

OMP-18R5 Bayer HealthCare mAb solid tumors Phase I Pharmaceuticals (888) 842-2937 Wayne, NJ (650) 995-8200 OncoMed Pharmaceuticals Redwood City, CA

OMP-21M18 GlaxoSmithKline mAb solid tumors Phase I Rsch. Triangle Park, NC (888) 825-5249 OncoMed Pharmaceuticals (650) 995-8200 Redwood City, CA

OMP-59R5 GlaxoSmithKline mAb late-stage solid tumors Phase I Rsch. Triangle Park, NC (888) 825-5249 OncoMed Pharmaceuticals (650) 995-8200 Redwood City, CA

Onconase® Tamir Biotechnology ribonuclease NSCLC Phase II ranpirnase Monmouth Junction, NJ (732) 823-1003

Oncophage™ Agenus heat shock malignant melanoma Phase III vitespen Lexington, MA protein (Fast Track) (781) 674-4400 (Orphan Drug) ------colorectal cancer, glioma, renal Phase II cancer (781) 674-4400 (Fast Track) ------gastric cancer, pancreatic cancer Phase I/II (781) 674-4400

OncoVAX® Vaccinogen cell vaccine colorectal cancer Phase II colorectal cancer Frederick, MD (combination therapy) (301) 668-8400 vaccine ------colorectal cancer Phase I/II (Fast Track) (301) 668-8400

OncoVEX-GM-CSF Amgen gene head/neck cancer, metastatic Phase III (talminogene Thousand Oaks, CA therapy melanoma (800) 772-6436 laherparepvec)

36 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

Onyvax-P VaxOnco cell prostate cancer Phase II cancer vaccine Seoul, South Korea vaccine www.vaxonco.com

OPT-822 Optimer Pharmaceuticals vaccine breast cancer Phase I San Diego, CA (858) 909-0736

oregovomab Quest Pharmatech mAb ovarian cancer Phase I/II (Orphan Drug) Edmonton, Canada (780) 448-1400

OVax® AVAX Technologies cell ovarian cancer Phase I/II ovarian cancer vaccine Philadelphia, PA vaccine (215) 241-9760

PancAtak™ Advantagene gene pancreatic cancer Phase I cancer gene therapy Boston, MA therapy (617) 916-5445 San Diego, CA (760) 943-8981

PDA-001 Celgene Cellular Therapeutics stem cell hematological malignancies Phase I (cenplacel-L) Summit, NJ therapy (see also autoimmune, (908) 673-9000 cardiovascular, digestive)

Pegasys® Roche interferon cancer metastases, Phase II peginterferon alfa-2a Nutley, NJ renal cancer (973) 235-5000 ------neurofibromatoses Phase I (973) 235-5000

pegdinetanib Bristol-Myers Squibb adnectin colorectal cancer, NSCLC Phase II Princeton, NJ (800) 332-2056

PEG-IntronA® Merck interferon CML Phase II peginterferon alfa-2b Whitehouse Station, NJ (800) 672-6372

PF-03446962 Pfizer mAb late-stage solid tumors Phase I New York, NY (860) 732-5156

PF-04383119 Pfizer mAb cancer pain Phase II (tanezumab) New York, NY (860) 732-5156

PF-04605412 Pfizer mAb solid tumors Phase I New York, NY (860) 732-5156

PF-04856884 Pfizer fusion renal cancer, solid tumors Phase I/II (CovX060) New York, NY peptide (860) 732-5156

PF-05082566 Pfizer mAb cancer, non-Hodgkin’s Phase I New York, NY lymphoma (860) 732-5156

Medicines in Development Biotechnology 2011 37 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

ProstAtak™ Advantagene gene prostate cancer Phase III cancer gene Boston, MA therapy (Fast Track) (617) 916-5445 therapy San Diego, CA (760) 943-8981

prostate cancer Novartis Pharmaceuticals vaccine prostate cancer Phase I/II vaccine East Hanover, NJ (treatment) (888) 669-6682 Pepscan Therapeutics Lelystad, The Netherlands

prostate cancer Progenics Pharmaceuticals recombinant prostate cancer Phase I vaccine Tarrytown, NY vaccine (914) 789-2800

Prostvac®-VF BN ImmunoTherapeutics recombinant prostate cancer Phase II recombinant vaccinia Mountain View, CA vaccine (650) 681-4660 virus-expressing PSA

Provenge® Dendreon dendritic cell prostate cancer Phase III completed sipuleucel-T Seattle, WA vaccine (early-stage disease, recurrent) (877) 256-4545 ------prostate cancer Phase II (early-stage disease, neoadjuvant (877) 256-4545 therapy)

PSMA-ADC Progenics Pharmaceuticals mAb prostate cancer Phase I Tarrytown, NY (914) 789-2800

PSMA-VRP Progenics Pharmaceuticals gene prostate cancer Phase I Tarrytown, NY vaccine (914) 789-2800

PTI-188 Pain Therapeutics mAb malignant melanoma Phase I completed San Mateo, CA (650) 624-8200

purine nucleoside PNP Therapeutics gene solid tumors Phase I phosphorylase Birmingham, AL therapy www.pnptherapeutics. gene therapy com

ramucirumab Eli Lilly mAb breast cancer, colorectal cancer, Phase III (IMC-1121B) Indianapolis, IN gastric cancer, liver cancer, (800) 545-5979 ImClone Systems NSCLC (212) 645-1405 New York, NY

REIC gene therapy Crucell gene prostate cancer Phase I Leiden, The Netherlands therapy www.crucell.com Momotaro-Gene www.mt-gene.com Okayama, Japan

Rencarex® Prometheus Laboratories mAb non-metastatic, clear-cell Phase III girentuximab San Diego, CA renal cell cancer (888) 423-5227 (Orphan Drug) Wilex www.wilex.com Munich, Germany

38 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

Reximmune-C® Epeius Biotechnologies gene breast cancer Phase I/II San Marino, CA vaccine (626) 441-6695

Rexin-G™ Epeius Biotechnologies gene osteosarcoma Phase II targeted nanomedicine San Marino, CA therapy (626) 441-6695 (Orphan Drug) ------metastatic breast cancer, Phase I/II pancreatic cancer (Fast Track), (626) 441-6695 soft tissue sarcoma

RG3637 Genentech mAb Hodgkin’s disease Phase I/II (lebrikizumab) South San Francisco, CA (see also respiratory) (800) 626-3553

RG7159 Biogen Idec mAb CLL, non-Hodgkin’s lymphoma Phase III (GA101) Weston, MA (781) 484-2000 Genentech (800) 626-3553 South San Francisco, CA

RG7160 Roche mAb colorectal cancer Phase II (humAb EGFR) Nutley, NJ (973) 235-5000

RG7334 Roche mAb glioblastoma, liver cancer, Phase I (anti-PLGF mAb) Nutley, NJ solid tumors (973) 235-5000

RG7347 Genentech mAb solid tumors Phase I completed (IgG1 mAb) South San Francisco, CA (800) 626-3553

RG7356 Roche mAb solid tumors Phase I (anti-CD44 mAb) Nutley, NJ (847) 937-5000

RG7446 Genentech mAb solid tumors Phase I (tumor immunotherapy) South San Francisco, CA (800) 626-3553

RG7450 Genentech mAb prostate cancer Phase I (antibody-drug conjugate) South San Francisco, CA (800) 626-3553 Seattle Genetics (425) 527-4000 Bothell, WA

RG7458 Genentech mAb ovarian cancer Phase I (antibody-drug conjugate) South San Francisco, CA (800) 626-3553 Seattle Genetics (425) 527-4000 Bothell, WA

RG7593 Genentech mAb hematological malignancies Phase I (anti-CD22 mAb) South San Francisco, CA (800) 626-3553

Medicines in Development Biotechnology 2011 39 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

RG7594 Genentech mAb solid tumors Phase I (anti-angiogenic) South San Francisco, CA (800) 626-3553

RG7596 Genentech mAb recurrent CLL, Phase I (anti-CD22 ADC) South San Francisco, CA recurrent non-Hodgkin’s lymphoma (800) 626-3553 Seattle Genetics (425) 527-4000 Bothell, WA

RIGScan® CR Neoprobe mAb diagnosis of colorectal application submitted radiodiagnostic agent Dublin, OH cancer (800) 793-0079

rindopepimut Celldex Therapeutics vaccine newly diagnosed glioblastoma Phase II (Orphan Drug) Needham, MA (Fast Track) (781) 433-0771 ------solid tumors Phase I (781) 433-0771

Rituxan® Biogen Idec mAb diffuse large B-cell lymphoma Phase III rituximab Weston, MA (see also skin, transplantation) (781) 464-2000 Genentech (800) 626-3553 South San Francisco, CA

Rituxan® SC Halozyme mAb non-Hodgkin’s lymphoma Phase III rituximab/rHuPH20 San Diego, CA (858) 794-8889 (subcutaneous) Roche (973) 235-5000 Nutley, NJ

robatumumab Merck mAb colorectal cancer, osteosarcoma, Phase II Whitehouse Station, NJ sarcoma, solid tumors in children, (800) 672-6372 solid tumors

SAR3419 sanofi-aventis mAb non-Hodgkin’s lymphoma Phase I (maytansin-loaded Bridgewater, NJ (800) 633-1610 anti-CD19 mAb)

SAR153192 Regeneron Pharmaceuticals mAb cancer Phase I (anti-DLL4 mAb) Tarrytown, NY (877) 734-6777 sanofi-aventis (800) 833-1610 Bridgewater, NJ

SAR256212 Merrimack Pharmaceuticals mAb breast cancer Phase II (MM-121) Cambridge, MA (617) 441-1000 (anti-ErbB3 mAb) sanofi-aventis (800) 633-1610 Bridgewater, NJ ------ovarian cancer, solid tumors Phase I (617) 441-1000 (800) 633-1610

40 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

SAR307746 Regeneron Pharmaceuticals mAb cancer Phase I (REGN910) Tarrytown, NY (877) 734-6777 (anti-angiopoietin-2 mAb) sanofi-aventis (800) 633-1810 Bridgewater, NJ

SAR566658 sanofi-aventis mAb solid tumors Phase I (maytansin-loaded Bridgewater, NJ (800) 633-1810 anti-DS6)

SAR650984 sanofi-aventis mAb hematological malignancies Phase I (anti-CD38 Bridgewater, NJ (800) 633-1810 naked mAb)

SB-313 Sangamo BioSciences cell glioblastoma Phase I Richmond, CA therapy (510) 970-6000

SBC-014 Synageva BioPharma growth chemotherapy-induced neutropenia Phase II Lexington, MA factor (781) 357-9900

SGN-75 Seattle Genetics mAb non-Hodgkin’s lymphoma, Phase I Bothell, WA renal cancer (425) 527-4000

sibrotuzumab Life Science Pharmaceuticals mAb colorectal cancer, Phase II Greenwich, CT head/neck cancer, NSCLC (203) 422-6500

SL-401 Stemline Therapeutics recombinant AML, myleodysplastic Phase I/II New York, NY fusion syndromes (212) 831-1111 protein

SL-701 Stemline Therapeutics dendritic cell glioma Phase I/II New York, NY vaccine (212) 831-1111

SNS01-T Senesco Technologies gene multiple myeloma Phase I/II Bridgewater, NJ therapy (908) 864-4444

StemEx® Gamida Cell stem cell hematological malignancies Phase II/III carlecortemcel-L Jerusalem, Israel therapy (Fast Track) (215) 591-3000 (Orphan Drug) Teva Pharmaceutical North Wales, PA

Stimuvax® Oncothyreon vaccine NSCLC Phase III lioposomal Bellevue, WA (Fast Track) (425) 450-0370 cancer vaccine

TAK-701 Millennium Pharmaceuticals mAb solid tumors Phase I Cambridge, MA (800) 390-5663

talactoferrin alfa Agennix recombinant NSCLC Phase III (Orphan Drug) Houston, TX protein (Fast Track) (713) 552-1091 (see also infectious)

Medicines in Development Biotechnology 2011 41 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

Tarvacin™ Peregrine Pharmaceuticals mAb NSCLC, pancreatic cancer Phase II bavituximab Tustin, CA (see also HIV, infectious) (714) 508-6000 ------liver cancer, solid tumors Phase I/II (714) 508-6000

T-cell gene therapy Immunomedics gene colorectal cancer Phase I Morris Plains, NJ therapy (973) 605-8200

TeloB-Vax Adamis Pharmaceuticals DNA prostate cancer Phase I telomerase San Diego, CA vaccine www.adamispharma. cancer vaccine com

TF2 Immunomedics mAb diagnosis of colorectal cancer Phase I Morris Plains, NJ (973) 605-8200

TG 4001 Transgene gene cervical intraepithelial neoplasia Phase II (tipapkinogene sovacivec) Strasbourg, France vaccine (301) 816-5404 Kensington, MD

tigatuzumab Daiichi Sankyo mAb breast cancer, liver cancer, Phase II Parsippany, NJ ovarian cancer, pancreatic cancer (973) 359-2600 ------colorectal cancer, lymphoma Phase I (973) 359-2600

TKM-PLK1 Alnylam Pharmaceuticals RNA lymphoma, solid tumors Phase I Cambridge, MA interference (617) 551-8200 Tekmira Pharmaceuticals (604) 419-3200 Burnaby, Canada

TNFerade™ GenVec gene colorectal cancer, Phase II golnerminogene Gaithersburg, MD therapy esophageal cancer, (877) 943-6832 pradenovac malignant melanoma ------head/neck cancer Phase I/II (877) 943-6832

Toca-511 Tocagen gene glioblastoma Phase I/II (cytosine deaminase San Diego, CA therapy (858) 412-8400 gene therapy)

trastuzumab-DM1 Genentech mAb advanced metastatic HER2- Phase III (RG3502) South San Francisco, CA positive breast cancer, (800) 626-3553 metastatic HER2-positive breast cancer ------early HER2-positive breast Phase II cancer (800) 626-3553

42 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

TRC093 TRACON Pharmaceuticals mAb solid tumors Phase I completed San Diego, CA (858) 550-0780

TRC105 TRACON Pharmaceuticals mAb breast cancer, prostate cancer Phase I/II San Diego, CA ------(858) 550-0780 solid tumors ------Phase I (858) 550-0780

Trivax Trimed Biotech dendritic cell glioblastoma Phase II cancer vaccine Vienna, Austria vaccine www.trimed-biotech.com ------renal cancer in clinical trials www.trimed-biotech.com

TroVax® Oxford BioMedica gene prostate cancer Phase II Oxford, United Kingdom vaccine

TRX518 Tolerx mAb malignant melanoma Phase I Cambridge, MA (617) 354-8100

TVAX cancer vaccine TVAX Biomedical cell vaccine glioma Phase II Lenexa, KS (913) 492-2221 ------breast cancer, colorectal cancer, Phase I/II completed lung cancer, malignant melanoma, (913) 492-2221 ovarian cancer, prostate cancer ------renal cancer in clinical trials (913) 492-2221

U3-1565 Amgen mAb solid tumors Phase I Thousand Oaks, CA (800) 772-6436 U3 Pharma Martinsried, Germany

umbilical cord blood Gamida Cell stem cell hematological malignancies Phase I/II stem cell therapy Jerusalem, Israel therapy

V503 Merck vaccine prevention of cervical cancer, Phase III Whitehouse Station, NJ prevention of vulvovaginal cancer (800) 672-6372 (see also infectious)

V505 Merck vaccine cervical cancer Phase II completed Whitehouse Station, NJ (see also infectious) (800) 672-6372

Medicines in Development Biotechnology 2011 43 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

V930/V932 Merck DNA cancer Phase I completed Whitehouse Station, NJ vaccine (800) 672-6372 Vical (858) 646-1100 San Diego, CA

V935 Geron vaccine solid tumors Phase I Menlo Park, CA (650) 473-7700 Merck (800) 672-6372 Whitehouse Station, NJ

VB-111 VBL Therapeutics gene thyroid cancer Phase II Or Yehuda, Israel therapy www.vblrx.com ------glioblastoma, Phase I/II solid tumors www.vblrx.com

VB4-845 Viventia Biotechnologies mAb bladder cancer, Phase II (Orphan Drug) Mississauga, Canada head/neck cancer (905) 362-2973 (Fast Track)

Vectibix® Amgen mAb colorectal cancer (first-line therapy, application submitted panitumumab Thousand Oaks, CA second-line therapy) (805) 447-1000 ------head/neck cancer Phase III (first-line therapy) (805) 447-1000 ------biliary cancer, head/neck cancer Phase II (second-line therapy), esophageal (805) 447-1000 cancer, NSCLC

veglin VasGene Therapeutics antisense cancer, Kaposi’s sarcoma, Phase I completed (VEGF antisense) Los Angeles, CA mesothelioma, renal cancer (323) 221-7818

veltuzumab Immunomedics mAb CLL, non-Hodgkin’s lymphoma Phase I/II (IMMU-106) Morris Plains, NJ (see also autoimmune) (973) 605-8200

verpasep caltespen Akela Pharma recombinant cervical intraepithelial neoplasia Phase I completed (HspE7) Austin, TX vaccine (512) 834-0449

VGX-3100 Inovio Pharmaceuticals DNA cervical intraepithelial neoplasia Phase II Blue Bell, PA vaccine (see also infectious) (877) 446-6846

volociximab Biogen Idec mAb NSCLC, ovarian cancer, Phase II Weston, MA peritoneal cancer (781) 464-2000 Facet Biotech (see also eye) Redwood City, CA

44 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

Voraxaze™ BTG International recombinant chemoprotection application submitted glucarpidase Brentwood, TN enzyme (615) 327-1027 (Orphan Drug)

VX15 Vaccinex mAb solid tumors Phase I Rochester, NY (585) 271-2700

VX30 EUSA Pharma mAb hematological Phase I Langhorne, PA malignancies (800) 833-3533 Vaccinex (see also autoimmune) (585) 271-2700 Rochester, NY

Xgeva™ Amgen mAb prevention of bone metastases application submitted denosumab Thousand Oaks, CA in men with castration-resistant (805) 447-1000 prostate cancer ------prevention of metastatic bone Phase III cancer (805) 447-1000 ------multiple myeloma Phase II (805) 447-1000

XmAb™2513 Xencor mAb Hodgkin’s disease, Phase I Monrovia, CA T-cell lymphoma (626) 305-5900

XmAb®5574 Xencor mAb CLL Phase I (MOR-208) Monrovia, CA (626) 305-5900 MorphoSys Munich, Germany

Y-90 hPAM 4 Immunomedics mAb pancreatic cancer Phase I/II (IMMU-107) Morris Plains, NJ (Fast Track) (973) 605-8200 (Orphan Drug)

Yervoy™ Bristol-Myers Squibb mAb adjuvant melanoma, metastatic in clinical trials ipilimumab Princeton, NJ melanoma, gastric cancer, NSCLC, (800) 332-2056 (Orphan Drug) ovarian cancer, prostate cancer, SCLC

zanolimumab Emergent BioSolutions mAb cutaneous T-cell lymphoma Phase III (Orphan Drug) Rockville, MD (Fast Track) (301) 795-1800 ------peripheral T-cell lymphoma Phase II (301) 795-1800

ZIN-ATI-001 Intrexon gene malignant melanoma Phase I New York, NY therapy (646) 214-0700 ZIOPHARM Oncology New York, NY

Medicines in Development Biotechnology 2011 45 Biotechnology Medicines in Development

Cancer and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

ZIN-CTI-001 Intrexon cell malignant melanoma Phase I New York, NY therapy (646) 214-0700 ZIOPHARM Oncology New York, NY

ZRx-101 ZelleRx cell malignant melanoma, Phase I Chicago, IL therapy multiple myeloma, renal cancer (312) 243-5200 (see also transplantation)

Cardiovascular Disease

Product Name Sponsor Product Indication Development Phase Category

Activase® Genentech protein acute ischemic stroke Phase III alteplase South San Francisco, CA (extended-time treatment window) (800) 626-3553

ACY001 Arteriocyte stem cell chronic coronary ischemia Phase I Cleveland, OH therapy (216) 456-9640

adipose stem cell therapy Cytori Therapeutics stem cell myocardial infarction, Phase I San Diego, CA therapy myocardial ischemia (877) 470-8000

adult autologous stem cell Baxter International stem cell myocardial ischemia Phase II completed therapy Deerfield, IL therapy (800) 422-9837 ------critical limb ischemia Phase I/II completed (800) 422-9837

ALD-201 Aldagen stem cell ischemic heart failure Phase I completed Durham, NC therapy (919) 484-2571

ALD-301 Aldagen stem cell critical limb ischemia Phase I/II Durham, NC therapy (919) 484-2571

ALD-401 Aldagen stem cell ischemic stroke Phase II Durham, NC therapy (919) 484-2571

ALO-212 Arteriocyte stem cell critical limb ischemia Phase I Cleveland, OH therapy (216) 456-9640

AMG 145 Amgen mAb hypercholesterolemia Phase I Thousand Oaks, CA (800) 772-6436

AMR-001 Amorcyte cell myocardial infarction Phase I Allendale, NJ therapy (201) 883-5300 Progenitor Cell Therapy Allendale, NJ

46 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Cardiovascular Disease

Product Name Sponsor Product Indication Development Phase Category

anti-fibrin mAb AGEN Biomedical mAb diagnosis of deep vein Phase II 3B6/22 Tc-99m Sydney, Australia thrombosis, diagnosis of www.agenix.com pulmonary embolism

beperminogene AnGes MG gene peripheral arterial disease Phase II perplasmid Gaithersburg, MD therapy (Fast Track) www.anges.mg.com ------ischemic heart disease Phase I www.anges.mg.com

CA-18C3 XBiotech mAb prevention of vascular restenosis Phase II Austin, TX after surgical intervention (512) 386-2900 (see also cancer)

coronary artery disease Biocardia gene coronary artery disease Phase II gene therapy San Carlos, CA therapy (800) 624-1179

desmoteplase Lundbeck Research USA recombinant ischemic stroke Phase III Paramus, NJ protein (Fast Track) (201) 261-1331

epicardial angiogenesis Theregen fibroblast heart failure, ischemic Phase I patch San Francisco, CA cell heart disorders (415) 439-8320 therapy

fibroblast growth CardioVascular BioTherapeutics fibroblast angina pectoris Phase II factor-1 Las Vegas, NV growth (see also skin) (702) 248-1174 factor

Generx™ Cardium Therapeutics gene myocardial ischemia Phase III alferminogene tadenovec San Diego, CA therapy (Fast Track) (858) 436-1000

gevokizumab XOMA mAb cardiovascular disorders Phase II Berkeley, CA (see also autoimmune, diabetes) (510) 204-7200

GGF2 Acorda Therapeutics growth congestive heart failure Phase I (glial growth factor 2) Hawthorne, NY factor (914) 347-4300

GSK249320 GlaxoSmithKline mAb stroke Phase II Rsch. Triangle Park, NC (see also neurologic) (888) 825-5249

GSK2586881 Apeiron Biologics recombinant heart failure, hypertension, Phase I (APN01) Vienna, Austria protein myocardial infarction (888) 825-5249 GlaxoSmithKline (see also cancer, diabetes, Rsch. Triangle Park, NC respiratory)

HIF-1 alpha Genzyme gene peripheral arterial disease Phase II gene therapy Cambridge, MA therapy (617) 252-7500

IL-1 ß antibody Eli Lilly mAb cardiovascular disease Phase I Indianapolis, IN (800) 545-5979

Medicines in Development Biotechnology 2011 47 Biotechnology Medicines in Development

Cardiovascular Disease

Product Name Sponsor Product Indication Development Phase Category

Ilaris® Novartis Pharmaceuticals mAb secondary prevention of Phase III canakinumab East Hanover, NJ cardiovascular events (888) 669-6682 (see also autoimmune, diabetes, musculoskeletal, respiratory)

Iprivask® Canyon Pharmaceuticals recombinant prevention of thrombosis in clinical trials desirudin Hunt Valley, MD protein (888) 434-7003

ISIS-APOCIIIRx Isis Pharmaceuticals antisense hypertriglyceridemia Phase I Carlsbad, CA (800) 679-4747

JVS-100 Juventus Therapeutics gene heart failure Phase I Cleveland, OH therapy www.juventusinc.com

Kalbitor® Dyax recombinant angiotensin converting enzyme Phase II ecallantide Cambridge, MA protein (ACE), inhibitor-induced (617) 225-2500 angioedema

Lantus® sanofi-aventis recombinant reduction of cardiovascular Phase III insulin glargine (rDNA Bridgewater, NJ insulin insulin morbidity and mortality (800) 633-1610 origin) injection

LU AA24493 Lundbeck Research growth stroke Phase I Paramus, NJ factor (see also genetic) (201) 261-1331

mesenchymal Cephalon stem cell congestive heart failure, Phase II stem cell therapy Frazer, PA therapy myocardial infarction (610) 344-0200 Mesoblast (see also cancer, musculoskeletal) Melbourne, Australia

mipomersen Genzyme antisense hypercholesterolemia, Phase III (Orphan Drug) Cambridge, MA hyperlipoproteinemia type IIa (617) 252-7500 Isis Pharmaceuticals (800) 679-ISIS Carlsbad, CA

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

MultiStem® Angiotech Pharmaceuticals stem cell myocardial infarction Phase I completed stem cell therapy Vancouver, Canada therapy (see also digestive, transplantation) (604) 221-7676 Athersys (216) 431-9900 Cleveland, OH ------stroke Phase I (604) 221-7676 (216) 431-9900

Mydicar™ Celladon gene congestive heart failure Phase I/II SERCA 2a gene therapy La Jolla, CA therapy (class III/IV) (858) 366-4288 AmpliPhi Biosciences (206) 623-7612 Seattle, WA

48 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Cardiovascular Disease

Product Name Sponsor Product Indication Development Phase Category

MyoCell® Bioheart cell congestive heart failure Phase II/III cell-based heart Sunrise, FL therapy (954) 835-1500 damage therapy (tissue regeneration)

nitric oxide synthase MaxCyte gene pulmonary hypertension Phase I gene therapy Gaithersburg, MD therapy (301) 944-1700 Northern Therapeutics (514) 842-0071 Montreal, Canada

NTx®-265 Stem Cell Therapeutics growth stroke Phase II completed Calgary, Canada factor (403) 245-5495

NX-CP105 Neuronyx stem cell myocardial infarction Phase I Malvern, PA therapy (610) 240-4150

PDA-001 Celgene Cellular Therapeutics stem cell ischemic stroke Phase II (cenplacel-L) Summit, NJ therapy (see also autoimmune, cancer, (908) 673-9000 digestive)

PF-04950615 Pfizer unspecified hypercholesterolemia Phase II (RN316) New York, NY (860) 732-5156

PLX-PAD Pluristem Therapeutics stem cell critical limb ischemia Phase I completed Haifa, Israel therapy www.pluristem.com

Prochymal® Osiris Therapeutics stem cell myocardial infarction Phase II remestemcel-L Baltimore, MD therapy (see also diabetes, digestive, (410) 522-5005 respiratory, transplantation)

PRT-201 Proteon Therapeutics recombinant vascular access for hemodialysis Phase II (Orphan Drug) Waltham, MA protein (781) 890-0102

PVS-10200 Pervasis Therapeutics cell therapy peripheral arterial disorders Phase I/II Cambridge, MA (617) 621-3407

Resten-MP™ Cook Pharmica antisense coronary artery restinosis Phase I Bloomington, IN (877) 312-2665

Resten-NG™ Cook Pharmica antisense coronary artery restenosis Phase II Bloomington, IN (877) 312-2665

RG1512 Roche mAb peripheral vascular disease Phase II (P-selectin humAb) Nutley, NJ (973) 235-5000

RG7418 Genentech mAb atherosclerosis Phase II completed (anti-oxLDL) South San Francisco, CA (800) 626-3553

Medicines in Development Biotechnology 2011 49 Biotechnology Medicines in Development

Cardiovascular Disease

Product Name Sponsor Product Indication Development Phase Category

SAR236553 Regeneron Pharmaceuticals mAb hypercholesterolemia Phase II (REGN727) Tarrytown, NY (877) 734-6777 (anti-PCSK-9 mAb) sanofi-aventis (800) 633-1810 Bridgewater, NJ

SB-623 SanBio stem cell stroke Phase I/II Mountain View, CA therapy (650) 625-8965

StemedicaMCT™ Stemedica Cell Technologies stem cell stroke Phase I/II stem cell therapy San Diego, CA therapy (858) 658-0910

Syncria® GlaxoSmithKline recombinant heart failure Phase II albiglutide Rsch. Triangle Park, NC fusion (see also diabetes) (888) 825-5249 protein

tissue repair stem Aastrom Biosciences stem cell critical limb ischemia Phase II cell therapy implant Ann Arbor, MI therapy (Fast Track), (734) 930-5555 (ixmyelocel-T) dilated cardiomyopathy (Orphan Drug) (see also musculoskeletal)

TKM-ApoB Tekmira Pharmaceuticals RNA hypercholesterolemia Phase I Burnaby, Canada interference (604) 419-3200

Vascugel® Pervasis Therapeutics cell peripheral vascular disorder Phase II cell therapy Cambridge, MA therapy in patients undergoing (617) 621-3407 (Orphan Drug) arteriovenous access procedures for hemodialysis (Fast Track) ------peripheral arterial disorder in Phase I patients undergoing peripheral (617) 621-3407 bypass surgery for peripheral arterial disease

VM-202 ViroMed gene peripheral arterial disease Phase II (modified hepatocyte Seoul, South Korea therapy (see also diabetes) growth factor gene ------therapy) myocardial ischemia Phase I completed

Xigris® Eli Lilly recombinant prevention of thrombosis in Phase II completed drotrecogin alfa Indianapolis, IN protein patients with end-stage renal (800) 545-5979 disease on dialysis

50 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Diabetes and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

anti-TGF-beta-1 Eli Lilly mAb diabetes Phase II mAb Indianapolis, IN (800) 545-5979

BHT-3201 Bayhill Therapeutics DNA type 1 diabetes Phase I (RG7426) Palo Alto, CA vaccine (650) 320-2800 Genentech (800) 626-3553 South San Francisco, CA

Diamyd® Diamyd vaccine type 1 diabetes Phase III autoimmune Pittsburgh, PA (412) 488-0348 diabetes vaccine (Orphan Drug)

gevokizumab XOMA mAb type 2 diabetes Phase II Berkeley, CA (see also autoimmune, (510) 204-7200 cardiovascular)

GLP-1 FC Eli Lilly recombinant type 2 diabetes Phase III (dulaglutide) Indianapolis, IN fusion (800) 545-5979 protein

Glymera™ PhaseBio Pharmaceuticals recombinant type 2 diabetes Phase I/II PB1023 Malvern, PA fusion (610) 981-6500 protein

GSK2586881 Apeiron Biologics recombinant diabetes nephropathy Phase I (APN01) Vienna, Austria protein (see also cancer, cardiovascular, (888) 825-5249 GlaxoSmithKline respiratory) Rsch. Triangle Park, NC

IBC-VS01 Orban Biotech vaccine type I diabetes Phase I completed (insulin B-chain vaccine) Brookline, MA (774) 571-2616

Ilaris® Novartis Pharmaceuticals mAb type 2 diabetes Phase II/III canakinumab East Hanover, NJ (see also autoimmune, (888) 669-6682 cardiovascular, musculoskeletal, respiratory)

INGAP peptide Exsulin peptide type 1 diabetes, type 2 diabetes Phase II Burnsville, MN fragment (952) 898-5814

ISIS-PTP1BRx Isis Pharmaceuticals antisense type 2 diabetes Phase I Carlsbad, CA (800) 679-4747

KUR-211 Baxter International growth diabetic foot ulcers Phase II Deerfield, IL factor (800) 422-9837 Kuros Biosurgery Zurich, Switzerland

Medicines in Development Biotechnology 2011 51 Biotechnology Medicines in Development

Diabetes and Related Conditions

Product Name Sponsor Product Indication Development Phase Category

Lantus® sanofi-aventis recombinant type 2 diabetes Phase I insulin glargine Bridgewater, NJ insulin (800) 633-1610 (rDNA origin) injection and lixisenatide combination

PF-04856883 Pfizer recombinant type 2 diabetes Phase I (CVX-096) New York, NY fusion (860) 732-5156 protein

PF-05212389 Pfizer unspecified type 2 diabetes Phase I (OAP-189) New York, NY (see also other) (860) 732-5156

PF-05231023 Pfizer unspecified type 2 diabetes Phase I New York, NY (860) 732-5156

Prochymal® Osiris Therapeutics stem cell type 1 diabetes Phase II remestemcel-L Baltimore, MD therapy (see also cardiovascular, digestive, (410) 522-5005 (Orphan Drug) respiratory, transplantation)

RG7685 Roche peptide type 2 diabetes Phase I (GIP/GLP-1 dual agonist) Nutley, NJ (973) 235-4000

rHuPH20/insulin Halozyme Therapeutics recombinant type 1 diabetes, type 2 diabetes Phase II San Diego, CA fusion (858) 794-8889 protein

SB-509 Sangamo BioSciences DNA-binding diabetic neuropathies Phase II Richmond, CA protein (see also neurologic) (510) 970-6000

Syncria® GlaxoSmithKline recombinant type 2 diabetes Phase III albiglutide Rsch. Triangle Park, NC fusion (see also cardiovascular) (888) 825-5249 protein

teplizumab MacroGenics mAb type 1 diabetes Phase III (anti-CD3 mAb) Rockville, MD (301) 251-5172

TGF-ß antibody Eli Lilly mAb chronic renal disease Phase II (LY2382770) Indianapolis, IN (800) 545-5979

VM-202 ViroMed gene diabetic nephropathy Phase I/II (modified hepatocyte Seoul, South Korea therapy (see also cardiovascular) growth factor gene therapy)

52 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Digestive Disorders

Product Name Sponsor Product Indication Development Phase Category

alicaforsen Atlantic Healthcare antisense pouchitis (Fast Track), Phase III Cambridge, MA distal ulcerative colitis, www.atlantiche.com proximal ulcerative colitis

AMG 139 Amgen mAb Crohn’s disease Phase I Thousand Oaks, CA (see also skin) (800) 772-6436

AMG 181 Amgen antibody ulcerative colitis Phase I Thousand Oaks, CA (800) 772-6436

AMG 827 Amgen mAb Crohn’s disease Phase II (brodalumab) Thousand Oaks, CA (see also respiratory, skin) (800) 772-6436

anti-CCR5 mAb Human Genome Sciences mAb ulcerative colitis Phase I Rockville, MD (301) 309-8504

anti-gastrin 17 immunogen Cancer Advances polyclonal GERD Phase II Durham, NC antibody (see also cancer) (919) 361-2162 vaccine

anti-IP10 Bristol-Myers Squibb mAb ulcerative colitis Phase II (BMS-936557) Princeton, NJ (800) 332-2056

Avonex® Biogen Idec interferon ulcerative colitis Phase II completed interferon beta-1a Weston, MA (781) 464-2000

Cimzia® UCB mAb Crohn’s disease in children and Phase II certolizumab pegol Smyrna, GA adolescents (770) 970-7500 (see also musculoskeletal)

GBR-500 Glenmark Pharmaceuticals mAb inflammatory bowel disease Phase I Mumbai, India (see also autoimmune)

Humira® Abbott Laboratories mAb ulcerative colitis application submitted adalimumab Abbott Park, IL (see also autoimmune, other) (847) 937-6100 ------Crohn’s disease in children and Phase III adolescents (847) 937-6100

MultiStem® Athersys stem cell ulcerative colitis Phase II stem cell therapy Cleveland, OH therapy (see also cardiovascular, (216) 431-9900 (PF-05285401) Pfizer transplantation) (860) 732-5156 New York, NY

NN8555 Novo Nordisk mAb Crohn’s disease Phase II Princeton, NJ (see also autoimmune) (609) 987-5800

ozoralizumab Pfizer mAb Crohn’s disease Phase I (ATN-103) New York, NY (see also autoimmune, skin) (860) 732-5156

Medicines in Development Biotechnology 2011 53 Biotechnology Medicines in Development

Digestive Disorders

Product Name Sponsor Product Indication Development Phase Category

PDA-001 Celgene Cellular Therapeutics stem cell Crohn’s disease Phase II (cenplacel-L) Summit, NJ therapy (see also autoimmune, cancer, (908) 673-9000 cardiovascular)

PF-00547659 Pfizer mAb Crohn’s disease Phase II New York, NY (860) 732-5156

PF-04236921 Pfizer mAb Crohn’s disease Phase II New York, NY (see also autoimmune) (860) 732-5156

PF-05230900 Pfizer unspecified Crohn’s disease Phase I (ATR-107) New York, NY (860) 732-5156

PF-05230917 Pfizer mAb ulcerative colitis Phase II (anrukinzumab) New York, NY (860) 732-5156

Prochymal® Osiris Therapeutics stem cell Crohn’s disease Phase III remestemcel-L Baltimore, MD therapy (Fast Track) (410) 522-5005 (see also cardiovascular, diabetes, respiratory, transplantation)

Remicade® Janssen Biotech mAb ulcerative colitis in children application submitted infliximab Horsham, PA (see also skin) (800) 526-7736 (Orphan Drug) ------Crohn’s disease Phase III (prevention of relapse) (800) 526-7736

rhuMAb-ß7 Genentech mAb ulcerative colitis Phase I (RG7413) South San Francisco, CA (800) 626-3553

secukinumab Novartis Pharmaceuticals mAb Crohn’s disease Phase II East Hanover, NJ (see also autoimmune, eye, skin) (888) 669-6682

Simponi® Janssen Biotech mAb ulcerative colitis Phase III golimumab Horsham, PA (see also autoimmune) (800) 526-7736 (subcutaneous)

Stelara® Janssen Biotech mAb Crohn’s disease Phase II/III ustekinumab Horsham, PA (see also autoimmune, skin) (800) 526-7736

teduglutide NPS Pharmaceuticals peptide short bowel syndrome Phase III (Orphan Drug) Salt Lake City, UT (801) 583-4939

vedolizumab Millennium Pharmaceuticals mAb Crohn’s disease, ulcerative colitis Phase III Cambridge, MA (800) 589-9005

54 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Eye Conditions

Product Name Sponsor Product Indication Development Phase Category

AAV2sFLT-01 Applied Genetic Technologies gene wet (neovascular) age-related Phase I (anti-vascular Alachua, FL therapy macular degeneration (386) 462-2204 endothelial growth-factor Genzyme (617) 252-7500 gene therapy) Cambridge, MA

eye disorder MorphoSys mAb eye disorders Phase I therapeutic Germany (888) 669-6682 Novartis Pharmaceuticals East Hanover, NJ

Eylea™ Bayer HealthCare recombinant age-related macular application submitted aflibercept Pharmaceuticals fusion degeneration (888) 842-2937 Wayne, NJ protein (877) 734-6777 Regeneron Pharmaceuticals ------Tarrytown, NY diabetic macular edema, Phase III retinal vascular occlusion (888) 842-2937 (877) 734-6777

GSK933776A GlaxoSmithKline mAb age-related macular degeneration Phase II Rsch. Triangle Park, NC (see also neurologic) (888) 825-5249

human retinal Advanced Cell Technology stem cell dry age-related macular Phase I/II pigment epithelial Santa Monica, CA therapy degeneration, Stargardt’s (310) 576-0611 cell therapy macular degeneration

iSONEP™ Lpath mAb retinal detachment secondary Phase I/II sonepcizumab San Diego, CA to wet age-related macular (858) 678-0800 (intravitreous) degeneration or polypoidal choroidal vasculopathy (see also cancer) ------age-related macular Phase I completed degeneration (858) 678-0800

Lucentis® Genentech mAb diabetic macular edema, Phase III ranibizumab South San Francisco, CA degenerative myopia (800) 626-3553 (see also genetic)

mitomycin Mobius Therapeutics recombinant adjunctive therapy in glaucoma application submitted ophthalmic St. Louis, MO protein surgery, prevention of pterygium (314) 615-6930 recurrence after excision surgery

NT 501 Neurotech USA cell therapy retinitis pigmentosa Phase II/III (ciliary neurotrophic Lincoln, RI (Fast Track) (401) 333-3880 factor) ------(Orphan Drug) age-related macular Phase II degeneration (401) 333-3880 (Fast Track)

PF-04382923 Pfizer antibody age-related macular degeneration Phase I (RN6G) New York, NY (860) 732-5156

Medicines in Development Biotechnology 2011 55 Biotechnology Medicines in Development

Eye Conditions

Product Name Sponsor Product Indication Development Phase Category

PF-04523655 Pfizer RNA age-related macular degeneration, Phase II New York, NY interference diabetic macular edema (860) 732-5156

PRM-151 Promedior recombinant prevention of scars in patients Phase II (rhPTX-2 for Malvern, PA protein with glaucoma following (610) 560-1435 injection) trabeculectomy (see also respiratory)

QPI-1007 Quark Pharmaceuticals RNA eye disorders Phase I Fremont, CA interference (510) 402-4020

RetinoStat® Oxford BioMedica gene wet age-related macular Phase I/II angiostatin endostatin Oxford, United Kingdom therapy degeneration (800) 633-1610 gene therapy sanofi-aventis Bridgewater, NJ

RG7417 Genentech mAb age-related macular Phase I/II (anti-factor D) South San Francisco, CA degeneration (800) 626-3553

secukinumab Novartis Pharmaceuticals mAb uveitis Phase III East Hanover, NJ (see also autoimmune, (888) 669-6682 digestive, skin)

Soliris® Alexion Pharmaceuticals mAb age-related macular degeneration, Phase II eculizumab Cheshire, CT neuromyelitis optica (203) 272-2596 (see also autoimmune, blood, neurologic, transplantation, other)

StarGen™ Oxford BioMedica gene Stargardt’s disease Phase I/II Stargardt’s disease Oxford, United Kingdom therapy (juvenile-onset macular dystrophy) gene therapy (Orphan Drug)

stem cell therapy Stemedica Cell Technologies stem diabetic retinopathy, in clinical trials San Diego, CA cell glaucoma, macular (858) 658-0910 therapy degeneration, retinitis pigmentosa

volociximab Ophthotech mAb age-related macular degeneration Phase I Princeton, NJ (wet form) (609) 945-6050 (see also cancer)

56 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Genetic Disorders

Product Name Sponsor Product Indication Development Phase Category

adrenoleucodystrophy bluebird bio gene adrenoleucodystrophy Phase I/II gene therapy Cambridge, MA therapy (617) 491-5601 INSERM Paris, France

AGTC-0106 Applied Genetic Technologies gene alpha-1-antitrypsin deficiency Phase II adeno-associated virus Alachua, FL therapy (386) 462-2204 vector-medictoten (Orphan Drug)

ALD-601 Aldagen stem cell infantile-onset lysosomal storage Phase I Durham, NC therapy disease (919) 484-2571

BMN-701 BioMarin Pharmaceutical recombinant glycogen storage disease type II Phase I/II (Orphan Drug) Novato, CA fusion (Pompe disease) (415) 506-6700 protein

CEQ508 Marina Biotech RNA familial adenomatous Phase I/II Bothell, WA interference polyposis (425) 908-3600

conestat alfa Pharming Group recombinant hereditary angioedema application submitted (C1 inhibitor) Leiden, The Netherlands protein (Fast Track) www.pharming.com (Orphan Drug) Santarus (see also transplantation) (858) 314-5700 San Diego, CA

DNA nanoparticle Copernicus Therapeutics gene cystic fibrosis Phase I/II gene therapy Cleveland, OH therapy (216) 231-0227 (Orphan Drug)

ENB-0040 Enobia Pharma recombinant hypophosphatasia in adults, Phase II (asfotase alfa) Cambridge, MA fusion adolescents and children www.enobia.com protein (Fast Track)

GSK2696273 GlaxoSmithKline stem cell adenosine deaminase Phase III Rsch. Triangle Park, NC gene severe combined (888) 825-5249 therapy immune deficiency

HuCNS-SC® StemCells stem cell Pelizaeus-Merzbacher disease Phase I adult neural stem Newark, CA therapy (510) 456-4000 cell therapy

ISIS-TTRRx Isis Pharmaceuticals antisense familial amyloid polyneuropathy Phase I Carlsbad, CA (800) 679-4747

KRN-23 Kyowa Hakko Kirin Pharma mAb X-linked dominant Phase I/II Princeton, NJ hypophosphatemic rickets (609) 919-1100

Medicines in Development Biotechnology 2011 57 Biotechnology Medicines in Development

Genetic Disorders

Product Name Sponsor Product Indication Development Phase Category

leber congenital Applied Genetic Technologies gene leber congenital amaurosis Phase I/II amaurosis gene Alachua, FL therapy (386) 462-2204 therapy (Orphan Drug)

liprotamase Eli Lilly enzyme treatment of malabsorption application submitted (Orphan Drug) Indianapolis, IN replacement as a result of pancreatic therapy (800) 545-5979 therapy insufficiency (Fast Track)

LU AA24493 Lundbeck Research growth Friedrich’s ataxia Phase II Paramus, NJ factor (see also cardiovascular) (201) 261-1331

Lucentis® Genentech mAb hereditary hemorrhagic Phase I/II ranibizumab South San Francisco, CA telangiectasia (800) 626-3553 (see also eye)

NAV™ therapeutic ReGenX Biosciences gene Batten disease Phase I Washington, DC therapy (neuronal ceroid lipofuscinosis) (202) 785-7438

taliglucerase alfa Pfizer enzyme Gaucher’s disease application submitted New York, NY replacement (Fast Track) (860) 732-5156 therapy

tgAAG-76 AmpliPhi Biosciences gene Leber’s hereditary Phase I/II gene therapy Seattle, WA therapy optic atrophy (206) 623-7612

Growth Disorders

Product Name Sponsor Product Indication Development Phase Category

Increlex® Tercica recombinant short stature in children Phase III mecasermin Brisbane, CA growth with primary IGF-1 deficiency (650) 624-4900 (Orphan Drug) factor (evaluating daily dosing regimens)

somatropin LG Life Sciences recombinant somatropin deficiency Phase III (sustained-release) Seoul, South Korea human growth hormone

58 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Growth Disorders

Product Name Sponsor Product Indication Development Phase Category

somatropin Pacira Pharmaceuticals growth growth disorders Phase I (sustained-release) San Diego, CA hormone (858) 625-2424

somatropin/ Genentech recombinant short stature in children Phase II mecasermin South San Francisco, CA growth (800) 626-3553 combination Tercica hormone, (650) 624-4900 Brisbane, CA recombinant growth factor

VRS-317 Versartis recombinant somatropin deficiency Phase I (human growth hormone) Mountain View, CA fusion in adults (650) 639-8580 protein

HIV Infection

Product Name Sponsor Product Indication Development Phase Category

Ad26.ENVA.01 Crucell recombinant HIV infection prevention Phase I HIV-1 recombinant Leiden, The Netherlands vaccine (212) 847-1111 vaccine Beth Israel Deaconess Medical Center Boston, MA International AIDS Vaccine Initiative New York, NY

ADVAX Aaron Diamond AIDS DNA HIV infection prevention Phase I (DNA vaccine) Research Center vaccine (212) 448-5000 New York, NY (212) 847-1111 International AIDS Vaccine ------Initiative HIV infection prevention Phase I New York, NY (new delivery system) (212) 448-5000 Ichor Medical Systems (212) 847-1111 San Diego, CA

AGS-004 Argos Therapeutics dendritic cell HIV infection Phase II Durham, NC vaccine (919) 287-6300 Kyowa Hakko Kirin Tokyo, Japan

anti-HIV-1 mAb Polymun Scientific mAb HIV infection treatment Phase II (2G12, 2F5, 4E10) Vienna, Austria www.polymun.com

Medicines in Development Biotechnology 2011 59 Biotechnology Medicines in Development

HIV Infection

Product Name Sponsor Product Indication Development Phase Category

AVX101 AlphaVax gene HIV-1 infection Phase I completed Rsch. Triangle Park, NC vaccine (919) 595-0400

CDX-2401 Celldex mAb HIV infection Phase I Needham, MA vaccine (781) 433-0771 Rockefeller University New York, NY

CYT-107 Cytheris interleukin HIV infection Phase II (interleukin-7) Rockville, MD (see also blood, cancer, (301) 231-0450 transplantation)

Cytolin® CytoDyn mAb HIV infection Phase I anti-CD8 mAb Santa Fe, NM (505) 988-5520

DermaVir™ Patch Genetic Immunity DNA HIV-1 infections Phase I/II HIV DNA topical vaccine McLean, VA vaccine (703) 287-8720

DP-6001 CytRx DNA prevention of HIV infection Phase I (HIV DNA vaccine) Los Angeles, CA vaccine (310) 826-5648

EP1043 VaxOnco recombinant prevention of HIV-1 Phase I completed Seoul, South Korea vaccine infection www.vaxonco.com

EP1090 VaxOnco DNA HIV-1 infection prevention and Phase I/II Seoul, South Korea vaccine treatment www.vaxonco.com

EP1232/EP1233 Bavarian Nordic recombinant HIV infection prevention Phase I Washington, DC vaccine (202) 536-1581 VaxOnco www.vaxonco.com Seoul, South Korea

HGTV43™ Enzo Therapeutics antisense HIV-1 infection Phase I/II New York, NY (212) 583-0100

HIV combination GlaxoSmithKline recombinant HIV-1 infection prevention Phase I completed vaccine Rsch. Triangle Park, NC vaccine (888) 825-5249 (gp120/NefTat/ASO2A)

HIV stem cell therapy Benitec stem HIV infection Phase I Melbourne, Australia cell (see also cancer) www.benitec.com City of Hope National therapy (626) 256-4673 Medical Center Duarte, CA

60 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

HIV Infection

Product Name Sponsor Product Indication Development Phase Category

HIV T-cell therapy Benitec cell HIV infection Phase 0 Melbourne, Australia therapy www.benitec.com City of Hope National (626) 256-4673 Medical Center Duarte, CA

HIV vaccine GeoVax vaccine HIV infection prevention Phase II Smyrna, GA (678) 384-7220 ------HIV-1 treatment Phase I (678) 384-7220

HIV vaccine GlaxoSmithKline vaccine HIV infections Phase II Rsch. Triangle Park, NC (888) 825-5249

HIV vaccine Massachusetts General Hospital vaccine HIV infection Phase I Boston, MA (617) 726-2000 Opal Therapeutics Parkvill, Australia

HIV vaccine Novartis Vaccines & Diagnostics vaccine HIV infection Phase I Cambridge, MA (617) 871-7000

HIV vaccine GlaxoSmithKline recombinant HIV infection Phase II (gag/pol/nef) Rsch. Triangle Park, NC vaccine (888) 825-5249

HIV vaccine Profectus Biosciences DNA HIV infection prevention Phase I (MAG pDNA) Baltimore, MD vaccine (866) 938-8559

HIV vaccine Profectus Biosciences recombinant HIV infection prevention Phase I (rVSV) Baltimore, MD vaccine (866) 938-8559

ibalizumab TaiMed Biologics mAb HIV infection Phase II (TNX-355) Taipei, Taiwan (Fast Track)

KD-247 Kaketsuken mAb HIV-1 infection Phase I Kumamoto, Japan www.kaketsuken.or.jp

lexgenleucel-T VIRxSYS gene HIV infection Phase II (VRX496) Gaithersburg, MD therapy (301) 987-0480

MVA-BN® Bavarian Nordic vaccine HIV-1 infection prevention and Phase I/II HIV multiantigen vaccine Washington, DC treatment (202) 536-1581

Medicines in Development Biotechnology 2011 61 Biotechnology Medicines in Development

HIV Infection

Product Name Sponsor Product Indication Development Phase Category

Pennvax™-B Inovio Pharmaceuticals DNA HIV infection prevention and Phase I DNA vaccine Blue Bell, PA vaccine treatment (877) 446-6846 (clade B)

Pennvax™-G Inovio Pharmaceuticals DNA HIV infection prevention Phase I DNA vaccine Blue Bell, PA vaccine (877) 446-6846 (clades A, C, D)

PRO 140 Progenics Pharmaceuticals mAb HIV infection Phase II Tarrytown, NY (914) 789-2800

SB-728-T Sangamo BioSciences gene HIV infection Phase I/II Richmond, CA therapy (510) 970-6000

Tarvacin™ Peregrine Pharmaceuticals mAb HIV infection in patients co-infected Phase I bavituximab Tustin, CA with hepatitis C (714) 508-6000 (see also cancer, infectious)

TUTI-16 Thymon vaccine HIV-1 infection Phase I Short Hills, NJ

UB-421 United Biomedical mAb HIV-1 infection in Phase I Hauppauge, NY treatment-naive patients (631) 273-2828

V520/V526 Merck gene HIV infection Phase I Whitehouse Station, NJ vaccine (800) 672-6372

Vacc-4x Bionor Pharma vaccine treatment of HIV-1 infection Phase II Bethesda, MD (301) 571-9493

VRC-HIVADV014-00-VP GenVec gene HIV-1 infection prevention Phase II Gaithersburg, MD vaccine (240) 632-0740 Vaccine Research Center (NIAID) www.vrc.nih.gov Bethesda, MD

VRC-HIVADV027-00-VP GenVec gene HIV infection prevention Phase I Gaithersburg, MD vaccine (240) 632-0740 Vaccine Research Center (NIAID) www.vrc.nih.gov Bethesda, MD

62 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Infectious Diseases

Product Name Sponsor Product Indication Development Phase Category

ABthrax™ Human Genome Sciences mAb anthrax application submitted raxibacumab Rockville, MD (301) 309-8504 (Orphan Drug)

ACAM-Cdiff sanofi pasteur vaccine Clostridium difficile infections Phase II (Clostridium difficile Swiftwater, PA (800) 822-2463 toxoid vaccine)

ACE527 ACE Biosciences vaccine prevention of traveler’s diarrhea Phase II Odense, Denmark caused by Escherichia coli www.acebiosciences. TD Vaccines infections com Skorping, Denmark www.atdvaccines.com

AERAS-402/Ad35 Aeras Global recombinant tuberculosis Phase I TB Vaccine Foundation vaccine (301) 547-2900 Rockville, MD Crucell Leiden, The Netherlands

Aflunov™ Novartis Pharmaceuticals cell-cultured prevention of influenza A virus Phase II H5N1 pre-pandemic East Hanover, NJ vaccine H5N1 subtype infections (888) 669-6682 influenza vaccine

Alferon N Injection® Hemispherx Biopharma interferon West Nile virus prevention Phase II interferon alfa-n3 Philadelphia, PA (215) 988-0080

Alferon N LDO® Hemispherx Biopharma interferon human papillomavirus infections Phase I/II interferon alfa-n3 Philadelphia, PA (215) 988-0080 ------influenza virus infections Phase I (215) 988-0080

ALN-RSV01 Alnylam Pharmaceuticals RNA respiratory syncytial virus (RSV) Phase II Cambridge, MA interference treatment (617) 551-8200

Anthim™ Elusys Therapeutics mAb anthrax Phase I (ETI-204) Pine Brook, NJ (Fast Track) (973) 808-0222 (Orphan Drug)

anthrax biodefense BioSante Pharmaceuticals vaccine anthrax Phase I/II vaccine (rPA) Lincolnshire, IL (847) 478-0500 U.S. Army Medical Research www.usamriid.army.mil Institute of Infectious Diseases Frederick, MD

anthrax immune Cangene mAb anthrax Phase II completed globulin Winnipeg, Canada (204) 275-4200

anthrax vaccine Buckler Biodefense vaccine anthrax in clinical trials Allentown, NY www.bucklerdefense. com

Medicines in Development Biotechnology 2011 63 Biotechnology Medicines in Development

Infectious Diseases

Product Name Sponsor Product Indication Development Phase Category

anthrax vaccine PharmAthene recombinant anthrax Phase II (second generation) Annapolis, MD vaccine (410) 269-2600

anthrax vaccine Intercell USA vaccine anthrax Phase I (transdermal) Gaithersburg, MD (301) 556-4500

ANZ-521 Aduro Biotech vaccine hepatitis C Phase I Berkeley, CA (510) 848-4400

AV 7909 Emergent BioSolutions vaccine anthrax Phase I Rockville, MD (Fast Track) (301) 795-0400

AVI-6002 AVI BioPharma antisense post-exposure treatment of Phase I Bothell, WA Ebola virus infections (425) 354-5038

AVI-6003 AVI BioPharma antisense treatment of Marburg virus Phase I Bothell, WA infections (425) 354-5038

AVI-7100 AVI BioPharma RNA influenza A virus infections Phase I Bothell, WA vaccine (425) 354-5038

AVP-21D9 Emergent BioSolutions mAb post-exposure treatment of anthrax Phase I Rockville, MD (Fast Track) (301) 795-1800

AVX502 AlphaVax vaccine influenza virus infections Phase I/II Rsch. Triangle Park, NC (919) 595-0400

AVX601 AlphaVax genetically- prevention of cytomegalovirus Phase I Rsch. Triangle Park, NC modified (CMV) infections (919) 595-0400 Novartis Pharmaceuticals vaccine (888) 669-6682 East Hanover, NJ

AZD9773 AstraZeneca polyclonal severe sepsis Phase II (anti-TNF-alpha polyclonal Wilmington, DE antibody (800) 236-9933 antibody)

BioThrax® Emergent BioSolutions vaccine prevention of post-exposure in clinical trials anthrax vaccine Rockville, MD anthrax infection in combination (301) 795-1800 with other antibacterials (Fast Track)

BLX-883 Biolex Therapeutics interferon hepatitis C Phase I Pittsboro, NC (see also cancer) (919) 542-9901

BLX-883 Biolex Therapeutics interferon hepatitis C Phase II (controlled-release) Pittsboro, NC (919) 542-9901

64 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Infectious Diseases

Product Name Sponsor Product Indication Development Phase Category

botulinum toxin vaccine DynPort Vaccine recombinant botulism Phase II Frederick, MD vaccine (301) 607-5000

cell therapy Cell Medica cell viral infections Phase I/II London, United Kingdom therapy (see also cancer) www.cellmedica.co.uk Center for Cell and Gene Therapy (713) 798-1246 Houston, TX

chikungunya virus Inviragen vaccine chikungunya virus infections Phase II vaccine Fort Collins, Co (970) 672-4918

cholera/enterotoxigenic Vaccine Technologies vaccine prevention of cholera, prevention of Phase I Escherichia coli vaccine Wellesly, MA Escherichia coli infections (781) 489-3388

cholera vaccine Vaccine Technologies vaccine cholera Phase II Wellesly, MA (781) 489-3388

Chronvac-C® Inovio Biomedical gene hepatitis C Phase II hepatitis C DNA San Diego, CA therapy (877) 446-6846 vaccine Tripep vaccine Huddinge, Sweden

Civacir™ Biotest Pharmaceuticals polyclonal hepatitis C prevention Phase II hepatitis C Boca Raton, FL antibody (800) 327-7106 immune globulin (Orphan Drug)

coxsackievirus vaccine Inviragen vaccine coxsackievirus infections Phase I (INV21) Fort Collins, CO (970) 672-4918

dengue vaccine sanofi pasteur vaccine moderate to severe Phase III Swiftwater, PA dengue fever (800) 822-2463

dengue vaccine GlaxoSmithKline vaccine dengue Phase II Rsch. Triangle Park, NC (888) 825-5249

DNA vaccine Astellas Pharma US DNA prevention of CMV Phase II (cytomegalovirus) Deerfield, IL vaccine infections (800) 695-4321 (Orphan Drug) Vical (858) 646-1100 San Diego, CA

DNA vaccine U.S. Naval Medical DNA prevention of dengue fever Phase I (dengue) Research Center vaccine (858) 646-1100 Silver Spring, MD Vical San Diego, CA

Medicines in Development Biotechnology 2011 65 Biotechnology Medicines in Development

Infectious Diseases

Product Name Sponsor Product Indication Development Phase Category

DNA vaccine Vaccine Research Center (NIAID) DNA prevention of Ebola virus Phase I completed (Ebola) Bethesda, MD vaccine infections www.vrc.nih.gov Vical (858) 646-1100 San Diego, CA

DNA vaccine Vical DNA prevention of influenza A Phase I (influenza H1N1) San Diego, CA vaccine (H1N1 subtype) virus infection (858) 646-1100

DNA vaccine Vical DNA prevention of influenza A Phase I (influenza H5N1) San Diego, CA vaccine (H5N1 subtype) virus (858) 646-1100 infections

DNA vaccine U.S. Naval Medical DNA prevention of malaria Phase I/II (malaria) Research Center vaccine (858) 646-1100 Silver Spring, MD Vical San Diego, CA

DNA vaccine Vaccine Research Center (NIAID) DNA prevention of coronavirus infections Phase I completed (SARS) Bethesda, MD vaccine www.vrc.nih.gov Vical (858) 646-1100 San Diego, CA

DNA vaccine Vaccine Research Center (NIAID) DNA prevention of West Nile virus Phase I (West Nile) Bethesda, MD vaccine infection www.vrc.nih.gov Vical (858) 646-1100 San Diego, CA

DTP-HepB-polio-Hib Merck vaccine diphtheria, tetanus, pertussis, Phase III (pediatric hexavalent Whitehouse Station, NJ hepatitis B, poliomyelitis, (800) 672-6372 vaccine) sanofi pasteur Haemophilus influenzae type b (800) 822-2463 (V419) Swiftwater, PA

DV-601 Dynavax Technologies vaccine treatment of hepatitis B Phase I Emeryville, CA (510) 848-5100

Ebola vaccine Crucell DNA prevention of Ebola virus infections Phase I Leiden, The Netherlands vaccine www.crucell.com

Ebola virus vaccine GenPhar vaccine Ebola virus infections in clinical trials Mt. Pleasant, SC (843) 884-0120

EP1300 VaxOnco DNA prevention of malaria infection Phase I (malaria DNA Seoul, South Korea vaccine www.vaxonco.com vaccine) National Institute of Allergy and Infectious Diseases Bethesda, MD

FGI-101-1A6 Functional Genetics mAb viral infections Phase I Gaithersburg, MD (240) 631-6790

66 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Infectious Diseases

Product Name Sponsor Product Indication Development Phase Category

FluBIok® Protein Sciences recombinant prevention of influenza virus application submitted derived from recombinant Meriden, CT vaccine infections in adults and children (203) 686-0800 hemagglutinin (rHA) (Fast Track)

Fludase® NexBio recombinant influenza A virus H1N1 infections, Phase II DAS-181 inhalation San Diego, CA fusion prevention of influenza virus (858) 452-2631 protein infections

FluLaval™ GlaxoSmithKline vaccine influenza virus infections in children Phase III influenza vaccine Rsch. Triangle Park, NC (888) 825-5249

FluNhance® Protein Sciences recombinant influenza virus infections Phase II completed influenza virus vaccine Meriden, CT vaccine (203) 686-0800 (rNA)

Fluvirin® Novartis Vaccines & Diagnostics vaccine prevention of influenza A virus Phase II/III influenza A virus H1N1 Cambridge, MA H1N1 subtype in children (617) 871-7000 vaccine

Fluzone® ID sanofi pasteur vaccine seasonal influenza virus infections application submitted intradermal Swiftwater, PA (800) 822-2463 micro-injection vaccine

Fluzone® QIV sanofi pasteur vaccine influenza virus infections Phase III quadrivalent inactivated Swiftwater, PA (800) 822-2463 influenza vaccine

foravirumab Crucell mAb post-exposure prevention of rabies Phase II (rabies vaccine) Leiden, The Netherlands vaccine www.crucell.com sanofi pasteur (800) 822-2463 Swiftwater, PA

GI-5005 GlobeImmune genetically- hepatitis C Phase II Louisville, CO modified (303) 625-2700 vaccine

GSK692342 GlaxoSmithKline recombinant prevention of tuberculosis Phase II (Mtb72F/AS02A) Rsch. Triangle Park, NC vaccine (888) 825-5249

GSK1437173A GlaxoSmithKline recombinant prevention of varicella zoster Phase III (varicella zoster vaccine) Rsch. Triangle Park, NC vaccine virus infections (888) 825-5249

GSK1492903A GlaxoSmithKline recombinant prevention of CMV infections Phase I completed (cytomegalovirus Rsch. Triangle Park, NC vaccine (888) 825-5249 glycoprotein vaccine)

Medicines in Development Biotechnology 2011 67 Biotechnology Medicines in Development

Infectious Diseases

Product Name Sponsor Product Indication Development Phase Category

GSK2189242A GlaxoSmithKline recombinant prevention of pneumococcal Phase II (Streptococcus Rsch. Triangle Park, NC vaccine infections (888) 825-5249 pneumoniae recombinant- conjugated vaccine)

GSK2202083A GlaxoSmithKline vaccine prevention of Neisseria Phase II (heptavalent combination Rsch. Triangle Park, NC meningitis C, Haemophilus (888) 825-5249 vaccine) influenzae type b, hepatitis B, diphtheria, tetanus, pertussis and poliomyelitis

GSK2231395A GlaxoSmithKline vaccine prevention of Haemophilus and Phase I Rsch. Triangle Park, NC pneumococcal infections (888) 825-5249

GSK2254232A GlaxoSmithKline recombinant prevention of Haemophilus and Phase I completed Rsch. Triangle Park, NC vaccine pneumococcal infections (888) 825-5249

GSK2282512A GlaxoSmithKline vaccine prevention of influenza virus Phase III Rsch. Triangle Park, NC infections (888) 825-5249

GSK2321138A GlaxoSmithKline vaccine prevention of influenza virus Phase III Rsch. Triangle Park, NC infections (888) 825-5249

GSK2392102A GlaxoSmithKline vaccine staphylococcal infections Phase I Rsch. Triangle Park, NC (888) 825-5249

H5N1 influenza virus Novavax vaccine prevention of influenza A virus Phase II vaccine Rockville, MD H5N1 subtype (240) 268-2000

H5N1 influenza virus Vaxart vaccine prevention of influenza A virus Phase I vaccine San Francisco, CA H5N1 subtype (415) 437-0132

H5N1 influenza virus Nanotherapeutics vaccine influenza virus infections Phase I vaccine Alachua, FL (386) 462-9663 intranasal

HBV-002 Hawaii Biotech recombinant prevention of West Nile virus Phase I completed Aiea, Hawaii vaccine infections (808) 486-5533

Helicobacter pylori Novartis Vaccines & Diagnostics vaccine Helicobacter infections Phase I vaccine Cambridge, MA (617) 871-7000

hepatitis B DNA vaccine Pfizer DNA hepatitis B treatment Phase I New York, NY vaccine (860) 732-5156

hepatitis B vaccine Emergent BioSolutions vaccine hepatitis B treatment Phase II Rockville, MD (301) 795-1800

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Infectious Diseases

Product Name Sponsor Product Indication Development Phase Category

hepatitis C vaccine Novartis Vaccines & Diagnostics DNA hepatitis C treatment Phase I/II Cambridge, MA vaccine (617) 871-7000

hepatitis C vaccine Virionics vaccine prevention and treatment of Phase I Scottsdale, AZ hepatitis C www.virionics.com

Heplisav™ Dynavax Technologies DNA prevention of hepatitis B Phase III hepatitis B vaccine Emeryville, CA vaccine (510) 848-5100

HerpV Agenus recombinant herpes simplex virus Phase I completed (herpes vaccine) New York, NY protein infections (866) 805-8994 vacccine

Hexaxim™ sanofi pasteur vaccine diphtheria, tetanus, pertussis, Phase III DTP-HepB-polio-Hib Swiftwater, PA hepatitis B, poliomyelitis, (800) 822-2463 vaccine Haemophilus influenzae type b

human papillomavirus National Cancer Institute recombinant human papillomavirus infections Phase II vaccine Bethesda, MD vaccine (240) 268-2000 Novavax Rockville, MD

IC-43 Intercell USA fusion prevention of Pseudomonas Phase II Gaithersburg, MD protein infections (301) 556-4500 vaccine

Imvamune® Bavarian Nordic vaccine smallpox Phase II smallpox vaccine Washington, DC (202) 536-1581 ------smallpox in treatment-naive patients Phase I/II (202) 536-1581

influenza A virus H1N1 CEL-SCI vaccine prevention of influenza A virus in clinical trials vaccine Vienna, VA H1N1 subtype (703) 506-9460

influenza A virus H1N1 CSL Behring vaccine prevention of influenza A virus Phase II vaccine King of Prussia, PA H1N1 subtype in the elderly (610) 878-4000

influenza A virus H1N1 GlaxoSmithKline vaccine prevention of influenza A virus Phase II vaccine Rsch. Triangle Park, NC H1N1 subtype in infants and (888) 825-5249 children

influenza A virus H1N1 iBio vaccine prevention of influenza A virus Phase I vaccine Newark, DE H1N1 subtype (302) 355-0650

influenza A virus H1N1 Medicago vaccine influenza A virus H1N1 subtype Phase I vaccine Quebec, Canada (418) 658-9393

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 (888) 825-5249 vaccine

Medicines in Development Biotechnology 2011 69 Biotechnology Medicines in Development

Infectious Diseases

Product Name Sponsor Product Indication Development Phase Category

influenza A virus H5N1 Baxter International vaccine prevention of influenza A virus Phase I vaccine Deerfield, IL H5N1 subtype (800) 422-9837

influenza A virus H5N1 iBio vaccine influenza A virus H5N1 subtype Phase I vaccine Newark, DE (302) 355-0650

influenza A virus vaccine Baxter International vaccine influenza A virus H9N2 subtype Phase I/II H9N2 Deerfield, IL (800) 422-9837

influenza virus-like Novavax vaccine prevention of influenza virus Phase II particle vaccine Rockville, MD infections (240) 268-2000

influenza virus vaccine Antigen Express vaccine influenza virus H5N1 infections Phase I Worcester, MA (508) 852-8783

influenza virus vaccine Crucell vaccine prevention of influenza virus Phase II Leiden, The Netherlands infections www.crucell.com

influenza virus vaccine Dynavax DNA prevention of influenza virus Phase I Berkeley, CA vaccine infections (510) 848-5100

influenza virus vaccine Novartis Vaccines & Diagnostics vaccine influenza virus infections Phase III (fluad) Cambridge, MA in the elderly (617) 871-7000

influenza virus vaccine Vaxin vaccine influenza virus infections Phase I intranasal Birmingham, AL (205) 413-8238

Influvac® TC Abbott Laboratories cell-cultured influenza virus infections Phase I cell culture-derived Abbott Park, IL vaccine (prevention) (847) 937-6100 influenza vaccine

interferon-alpha-2b HanAll Biopharma interferon hepatitis C Phase I/II Seoul, South Korea (301) 738-3986 Rockville, MD

interferon alpha-2b Medtronic interferon hepatitis C Phase II infusion Minneapolis, MN (800) 633-8766

interferon alpha-2b BioSante Pharmaceuticals interferon hepatitis B, hepatitis C in clinical trials inhalation Lincolnshire, IL (847) 478-0500

interferon alpha-2b Helix BioPharma interferon human papillomavirus infections Phase II topical Aurora, Canada (see also cancer) (905) 841-2300 Merck (800) 672-6372 Whitehouse Station, NJ

70 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Infectious Diseases

Product Name Sponsor Product Indication Development Phase Category

interferon-omega Intarcia Therapeutics interferon hepatitis C Phase II Emeryville, CA (510) 652-2600

Ixiaro® Intercell vaccine prevention of Japanese encephalitis Phase III Japanese encephalitis Vienna, Austria in children (617) 871-7000 vaccine Novartis Vaccines & Diagnostics Cambridge, MA

KB001 KaloBios Pharmaceuticals mAb pseudomonal infections Phase I/II completed Palo Alto, CA (650) 843-1897

LIQ-001 Liquidia Technologies vaccine influenza virus infections Phase I Rsch. Triangle Park, NC (919) 328-4400

malaria vaccine BioSante Pharmaceuticals DNA prevention of malaria Phase I/II Lincolnshire, IL vaccine (847) 478-0500 U.S. Naval Medical Research Center Silver Spring, MD

malaria vaccine Crucell recombinant malaria Phase I Leiden, The Netherlands vaccine www.crucell.com

malaria vaccine GenVec recombinant malaria Phase I/II Gaithersburg, MD vaccine (877) 943-6832 U.S. Naval Medical Research Center Silver Spring, MD

malaria vaccine GlaxoSmithKline recombinant prevention of malaria Phase I/II Rsch. Triangle Park, NC vaccine (888) 825-5249 Walter Reed Army Institute of Research Washington, DC

malaria vaccine Sanaria vaccine malaria Phase I/II Rockville, MD (301) 770-3222

Marburg virus GenPhar genetic Marburg virus disease in clinical trials DNA vaccine Mt. Pleasant, SC vaccine (843) 884-0120

MEDI-534 AstraZeneca vaccine prevention of parainfluenza virus Phase I/II (RSV/PIV-3 vaccine) Wilmington, DE infection, prevention of RSV (800) 236-9933 MedImmune infection (301) 398-0000 Gaithersburg, MD

MEDI-550 AstraZeneca vaccine pandemic influenza prevention Phase I (pandemic influenza virus Wilmington, DE (800) 238-9933 vaccine) MedImmune (301) 398-0000 Gaithersburg, MD

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Infectious Diseases

Product Name Sponsor Product Indication Development Phase Category

MEDI-557 AstraZeneca mAb RSV infections Phase I (RSV mAb- Wilmington, DE (800) 236-9933 extended half-life) MedImmune (301) 398-0000 Gaithersburg, MD

MEDI-559 AstraZeneca vaccine prevention of RSV infections Phase I/II (RSV vaccine) Wilmington, DE (800) 236-9933 MedImmune (301) 398-0000 Gaithersburg, MD

MEDI-3250 AstraZeneca vaccine seasonal influenza application submitted (live, attenuated intranasal Wilmington, DE (800) 236-9933 influenza virus vaccine, MedImmune (301) 398-0000 quadrivalent) Gaithersburg, MD

Menactra® sanofi pasteur vaccine meningococcal disease in infants application submitted (meningococcal conjugate Swiftwater, PA and toddlers 9-12 months (800) 822-2463 vaccine)

MenHibrix® GlaxoSmithKline vaccine prevention of Haemophilus application submitted Hib-meningococcal Rsch. Triangle Park, NC infections, meningococcal group C (888) 825-5249 vaccine groups C and Y and group Y infections conjugate

meninge ACYW conjugate sanofi pasteur vaccine meningococcal infections Phase II vaccine Swiftwater, PA (800) 822-2463 (2nd-generation meningococcal conjugate infant vaccine)

meningococcal group B Pfizer recombinant adolescent meningitis Phase II vaccine New York, NY vaccine (860) 732-5156 (PF-05212366) ------infant meningitis Phase I (860) 732-5156

meningococcal vaccine Novartis Vaccines & Diagnostics recombinant meningococcal group B infections Phase II completed group B Cambridge, MA vaccine (617) 871-7000

meningococcal vaccine Novartis Vaccines & Diagnostics vaccine meningococcal infections Phase II groups A,C,Y,W-135 Cambridge, MA (617) 871-7999 conjugate

meningococcal vaccine GlaxoSmithKline vaccine meningococcal infections Phase III groups A,C,Y,W-135 Rsch. Triangle Park, NC (888) 825-5249 conjugate

72 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Infectious Diseases

Product Name Sponsor Product Indication Development Phase Category

Menveo® Novartis Vaccines & Diagnostics vaccine meningococcal groups A,C,Y,W-135 application submitted meningococcal vaccine Cambridge, MA infection in children and infants (617) 871-7999 groups A,C,Y, W-135 conjugate vaccine

MGAWN1 MacroGenics mAb West Nile virus infections Phase I Rockville, MD (301) 251-5172

MK-3415A Merck mAb Clostridium difficile-associated Phase II Whitehouse Station, NJ diarrhea (800) 672-6372

MMR vaccine live GlaxoSmithKline vaccine prevention of measles, mumps, Phase II Rsch. Triangle Park, NC rubella (888) 825-5249

Mosquirix™ GlaxoSmithKline recombinant prevention of malaria Phase III malaria vaccine Rsch. Triangle Park, NC vaccine (888) 825-5249

motavizumab AstraZeneca mAb early and late treatment of RSV in Phase II Gaithersburg, MD children older than 1 year (301) 398-0000 MedImmune Gaithersburg, MD

NB-001 NanoBio nano- recurrent herpes simplex virus Phase III Ann Arbor, MI emulsion infections (734) 302-4000

NB-002 NanoBio nano- distal subungual onychomycosis Phase II Ann Arbor, MI emulsion of the toe (734) 302-4000

NB-1008 NanoBio nano- prevention of influenza virus Phase I completed (intranasal) Ann Arbor, MI emulsion infections (734) 302-4000 vaccine

NDV3 NovaDigm vaccine prevention of candidiasis, Phase I Grand Forks, ND prevention of methicillin-resistant (701) 757-5161 Staphylococcus aureus (MRSA) infections

norovirus bivalent vaccine LigoCyte Pharmaceuticals vaccine norovirus infections Phase I Bozeman, MT (405) 585-2733

norovirus monovalent LigoCyte Pharmaceuticals vaccine norovirus infections Phase I/II vaccine Bozeman, MT (405) 585-2733 intranasal

Medicines in Development Biotechnology 2011 73 Biotechnology Medicines in Development

Infectious Diseases

Product Name Sponsor Product Indication Development Phase Category

Optaflu® Novartis Vaccines & Diagnostics vaccine prevention of influenza virus Phase III influenza virus vaccine Cambridge, MA infections (617) 871-7000

pagibaximab Biosynexus mAb prevention of staphylococcal Phase II/III (Orphan Drug) Gaithersburg, MD infection in neonates (301) 330-5800

PanBlok™ Protein Sciences vaccine prevention of influenza A virus Phase I/II influenza A virus H5N1 Meriden, CT H5N1 subtype infections (203) 686-0800 vaccine

parvovirus B19 vaccine Meridian Bioscience vaccine parvovirus infections Phase I/II Cincinnati, OH (513) 271-3700

PEG-interferon lambda Bristol-Myers Squibb interferon hepatitis C Phase II Princeton, NJ (800) 332-2056

PF-05230894 Pfizer vaccine staphylococcal infections Phase I (SA3Ag) New York, NY (860) 732-5156

PHP Curacyte nitric septic shock Phase III (pyridoxalated hemoglobin Chapel Hill, NC oxide (919) 405-4002 polyoxyethylene) scavenger

plague injectable vaccine DynPort Vaccine vaccine Yersinia infections Phase II Frederick, MD (301) 607-5000

Preflucel™ Baxter International vaccine prevention of influenza virus Phase III seasonal influenza virus Deerfield, IL infections (800) 422-9837 vaccine

Prepandrix™ GlaxoSmithKline vaccine influenza A virus H5N1 subtype Phase III H5N1 (pre-)pandemic Rsch. Triangle Park, NC prevention for pre-pandemic use (888) 825-5249 influenza virus vaccine

PreviThrax™ Emergent Biosolutions recombinant anthrax Phase II recombinant protective Rockville, MD vaccine (Fast Track) (301) 795-1800 anthrax antigen

Prevnar-13® Pfizer vaccine prevention of pneumococcal application submitted pneumococcal vaccine New York, NY infections in the elderly (860) 732-5156 conjugate 13-valent

Pseudomonas aeruginosa sanofi pasteur antibody prevention of ventilator-associated Phase I antibody fragment Swiftwater, PA vaccine pneumonia (800) 822-2463 vaccine

Quadracel® sanofi pasteur vaccine diphtheria, tetanus, pertussis, Phase III DTP, polio vaccine Swiftwater, PA poliomyelitis; 4-6 years of age (800) 822-2463

74 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Infectious Diseases

Product Name Sponsor Product Indication Development Phase Category

rabies VRVg vaccine sanofi pasteur vaccine rabies Phase II (purified vero rabies Swiftwater, PA (800) 822-2463 vaccine)

respiratory syncytial virus Novavax vaccine prevention of RSV infections Phase I VLP vaccine Rockville, MD (240) 268-2000

ricin biodefense vaccine BioSante Pharmaceuticals vaccine ricin poisoning Phase I/II Lincolnshire, IL (847) 478-0500 U.S. Army Medical Research www.usamriid.army.mil Institute of Infectious Diseases Frederick, MD

ricin vaccine Soligenix vaccine prevention of ricin poisoning Phase I Princeton, NJ (609) 538-8200

rotavirus vaccine GlaxoSmithKline vaccine prevention of rotavirus infections Phase III (liquid) Rsch. Triangle Park, NC (888) 825-5249

rotavirus vaccine sanofi pasteur vaccine rotavirus Phase I (live attenuated tetravalent Swiftwater, PA (800) 822-2463 oral vaccine)

rotavirus vaccine 1321 Bharat Biotech vaccine prevention of rotavirus infections Phase I Hyderabad, India in infants www.bharatbiotech.com

SAR279356 Alopexx Pharmaceuticals mAb serious infections Phase I (anti-PNAG mAB) Cambridge, MA (617) 945-2510 sanofi-aventis (800) 633-1610 Bridgewater, NJ

Shigella vaccine Emergent BioSolutions vaccine Shigella infections Phase I Rockville, MD (301) 795-1800

staphylococcal BioSante Pharmaceuticals vaccine staphylococcal infections Phase I/II biodefense vaccine Lincolnshire, IL (847) 478-0500 (rSEB) U.S. Army Medical Research www.usamriid.army.mil Institute of Infectious Diseases Frederick, MD

Staphylococcus aureus GlaxoSmithKline vaccine gram-positive infections, Phase I/II vaccine conjugate Rsch. Triangle Park, NC staphylococcal infections (888) 825-5249 pentavalent

streptococcal B vaccine Emergent BioSolutions vaccine streptococcal infections Phase I Rockville, MD (301) 795-1800

Medicines in Development Biotechnology 2011 75 Biotechnology Medicines in Development

Infectious Diseases

Product Name Sponsor Product Indication Development Phase Category

streptococcal B vaccine Novartis Vaccines & Diagnostics mAb prevention of streptococcal Phase I/II conjugate Cambridge, MA infections (617) 871-7000

Streptococcus sanofi pasteur vaccine Streptococcus pneumoniae Phase I pneumoniae vaccine Swiftwater, PA (800) 822-2463 (meningitis and pneumonia vaccine)

talactoferrin alfa Agennix recombinant sepsis Phase II/III Houston, TX protein (see also cancer) (713) 552-1091 ------nosocomial infections in Phase I/II infants (713) 552-1091

Tarvacin™ Peregrine Pharmaceuticals mAb hepatitis C in patients co-infected Phase I bavituximab Tustin, CA with HIV infection (714) 508-6000 (see also cancer, HIV)

tetravalent hybrid dengue Inviragen vaccine dengue Phase I virus vaccine Fort Collins, CO (970) 672-4918

TG-4040 Transgene recombinant hepatitis C Phase II Strasbourg, France vaccine (301) 816-5404 Kensington, MD

tuberculosis vaccine sanofi pasteur recombinant tuberculosis Phase I (recombinant subunit Swiftwater, PA vaccine (800) 822-2463 vaccine)

tularemia vaccine DynaPort Vaccine vaccine tularemia Phase I Frederick, MD (301) 607-5000

Typhella™ Emergent BioSolutions vaccine prevention of typhoid Phase II typhoid vaccine Rockville, MD (301) 795-1800

typhoid vaccine Novartis Vaccines & Diagnostics vaccine prevention of typhoid Phase II Cambridge, MA (617) 871-7000

urinary tract infections Xanodyne Pharmaceuticals vaccine urinary tract infections Phase II vaccine Newport, KY (859) 371-6383 (transmucosal)

V212 Merck vaccine prevention of herpes zoster Phase III Whitehouse Station, NJ (800) 672-6372

76 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Infectious Diseases

Product Name Sponsor Product Indication Development Phase Category

V502 Merck vaccine human papillomavirus infections Phase II Whitehouse Station, NJ (800) 672-6372

V503 Merck vaccine prevention of human papillomavirus Phase III Whitehouse Station, NJ infections (800) 672-6372 (see also cancer)

V505 Merck vaccine human papillomavirus infections Phase II completed Whitehouse Station, NJ (see also cancer) (800) 672-6372

V1114 Merck vaccine prevention of pneumococcal Phase II (pneumococcal 15-valent Whitehouse Station, NJ infections in infants (800) 673-6273 vaccine conjugate)

VAX-102 VaxInnate recombinant prevention of influenza A virus Phase I/II Cranbury, NJ fusion infections (609) 860-2260 protein vaccine

VAX-125 VaxInnate recombinant influenza A virus infections Phase II Cranbury, NJ fusion in elderly patients (609) 860-2260 protein vaccine

VAX-128 VaxInnate recombinant prevention of influenza A virus Phase I Cranbury, NJ fusion H1N1 subtype (609) 860-2260 protein vaccine

Veldona® Amarillo Biosciences interferon human papillomavirus Phase II human interferon-alpha Amarillo, TX infections (806) 376-1741 (Orphan Drug) (see also autoimmune, respiratory)

VGX-3100 Inovio Pharmaceuticals DNA human papillomavirus Phase II Blue Bell, PA vaccine (see also cancer) (877) 446-6846

VGX-3400 Inovio Pharmaceuticals DNA prevention of influenza A virus Phase I Blue Bell, PA vaccine H5N1 subtype (877) 446-6846

XOMA 3AB XOMA mAb botulism Phase I Berkeley, CA (510) 204-7200

yellow fever vaccine Xcellerex vaccine prevention of yellow fever Phase I Marlborough, MA (508) 480-9235

Medicines in Development Biotechnology 2011 77 Biotechnology Medicines in Development

Musculoskeletal Disorders/Arthritis

Product Name Sponsor Product Indication Development Phase Category

ACE-011 Acceleron Pharma recombinant postmenopausal osteoporosis Phase I (sotatercept) Cambridge, MA fusion (see also blood, cancer, other) (617) 649-9200 Celgene protein (908) 673-9000 Summit, NJ

AMG 167 Amgen antibody metabolic bone diseases Phase I Thousand Oaks, CA (800) 772-6436

AMG 745 Amgen protein/ muscular atrophy Phase I Thousand Oaks, CA peptibody (800) 772-6436

AMG 785 Amgen mAb fracture treatment, Phase II Thousand Oaks, CA postmenopausal osteoporosis (800) 772-6436

Arcalyst™ Regeneron Pharmaceuticals recombinant gout Phase III rilonacept Tarrytown, NY fusion (877) 734-6777 protein

Biocart™ ProChon Biotech cell cartilage lesions of the knee Phase II autologous cartilage Woburn, MA therapy (781) 305-5035 system

Chondrogen® Osiris Therapeutics stem cell knee injuries Phase I/II mesenchymal stem cell Baltimore, MD therapy (410) 522-5005 therapy for meniscal repair

Cimzia® UCB mAb psoriatic arthritis, spondylitis Phase III certolizumab pegol Smyrna, GA (see also digestive) (770) 970-7500

FGF-18 EMD Serono growth acute cartilage injury of the knee Phase II (fibroblast growth factor) Rockland, MA factor (800) 283-8088 ------osteoarthritis Phase I (800) 283-8088

Ilaris® Novartis Pharmaceuticals mAb gouty arthritis application submitted canakinumab East Hanover, NJ (see also autoimmune, (888) 669-6682 cardiovascular, diabetes, respiratory) ------osteoarthritis, polymyalgia Phase II rheumatica (888) 669-6682

juvenile chondrocyte ISTO Technologies cell cartilage disorders Phase I/II grafts St. Louis, MO therapy (314) 995-6049

LY2495655 Eli Lilly mAb muscular atrophy Phase I Indianapolis, IN (800) 545-5979

78 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Musculoskeletal Disorders/Arthritis

Product Name Sponsor Product Indication Development Phase Category

LY2541546 Eli Lilly mAb postmenopausal osteoporosis Phase II Indianapolis, IN (800) 545-5979

mesenchymal stem Cephalon stem cell spinal fusion Phase II cell therapy Frazer, PA therapy (see also cancer, cardiovascular) (610) 344-0200 Mesoblast Melbourne, Australia

musculoskeletal MorphoSys mAb musculoskeletal disorders Phase I therapeutic antibody Munich, Germany (888) 669-6682 Novartis Pharmaceuticals East Hanover, NJ

NeoCart® Histogenics cell cartilage disorders Phase III autologous chondrocyte Waltham, MA therapy (781) 547-7900 implantation

NN8765 Novo Nordisk mAb rheumatoid arthritis Phase I Princeton, NJ (609) 987-5800

pegadricase 3SBio recombinant gout, hyperuricemia associated Phase I Shenyang, China protein with tumour lysis syndrome www.3sbio.com

PF-04840082 Pfizer mAb osteoporosis Phase I New York, NY (860) 732-5156

Prolia® Amgen mAb treatment-induced bone loss application submitted denosumab Thousand Oaks, CA in patients undergoing hormonal (805) 447-1000 ablation therapy for either prostate or breast cancer (see also autoimmune) ------male osteoporosis, prevention of Phase III osteoporosis (805) 447-1000

TG-C TissueGene cell osteoarthritis Phase I Rockville, MD therapy (301) 921-6000

tissue repair stem Aastrom Biosciences stem cell osteonecrosis Phase III cell therapy implant Ann Arbor, MI therapy (see also cardiovascular) (734) 930-5555 (ixmyelocel-T) ------(Orphan Drug) alveolar bone loss, non-union Phase I/II fracture, vertebral fracture (734) 930-5555

Medicines in Development Biotechnology 2011 79 Biotechnology Medicines in Development

Neurologic Disorders

Product Name Sponsor Product Indication Development Phase Category

AAB-002 Janssen Alzheimer mAb Alzheimer’s disease Phase 0 Immunotherapy (800) 817-5286 South San Francisco, CA (860) 732-5156 Pfizer New York, NY

AAB-003 Janssen Alzheimer mAb Alzheimer’s disease Phase I (PF-05236812) Immunotherapy (800) 817-5286 South San Francisco, CA (860) 732-5156 Pfizer New York, NY

ACC-001 Janssen Alzheimer peptide Alzheimer’s disease Phase II (vanutide cridificar) Immunotherapy vaccine (800) 817-5286 South San Francisco, CA (860) 732-5156 Pfizer New York, NY

ACC-002 Janssen Alzheimer peptide Alzheimer’s disease Phase 0 Immunotherapy vaccine (800) 817-5286 South San Francisco, CA (860) 732-5156 Pfizer New York, NY

ACE-031 Acceleron Pharam recombinant Duchenne muscular dystrophy Phase II Cambridge, MA fusion (617) 649-9200 protein

AD02 vaccine AFFiRis vaccine Alzheimer’s disease Phase II Vienna, Austria (888) 825-5249 GlaxoSmithKline Rsch. Triangle Park, NC

AD03 vaccine AFFiRis vaccine Alzheimer’s disease Phase I Vienna, Austria (888) 825-5249 GlaxoSmithKline Rsch. Triangle Park, NC

AGN-214868 Allergan recombinant postherpetic neuralgia Phase II Irvine, CA protein (see also other) (949) 788-6700

ALT 1102 Antisense Therapeutics antisense multiple sclerosis Phase II Victoria, Australia (800) 679-4747 Isis Pharmaceuticals Carlsbad, CA

ATI355 Novartis Pharmaceuticals mAb spinal cord injury Phase I (anti-Nogo-A mAb) East Hanover, NJ (888) 669-6682

80 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Neurologic Disorders

Product Name Sponsor Product Indication Development Phase Category

AV 201 Genzyme gene Parkinson’s disease Phase I/II Cambridge, MA therapy (617) 252-7500

AVI-4658 AVI BioPharma RNA Duchenne muscular dystrophy Phase I/II (eteplirsen) Bothell, WA interference (425) 354-5038

BA-210 Alseres Pharmaceuticals recombinant spinal cord injury Phase II (Orphan Drug) Hopkinton, MA fusion (508) 497-2360 BioAxone protein Montreal, Quebec

bapineuzumab Janssen Alzheimer mAb Alzheimer’s disease Phase III (AAB-001) Immunotherapy (intravenous) (800) 817-5286 South San Francisco, CA (Fast Track) (860) 732-5156 Pfizer ------New York, NY Alzheimer’s disease Phase II (subcutaneous) (800) 817-5286 (860) 732--5156

Biostrophin™ Asklêpios BioPharmaceuticals gene Duchenne muscular Phase I muscular dystrophy Chapel Hill, NC therapy dystrophy (919) 968-2727 gene therapy

CAD106 Cytos Biotechology vaccine Alzheimer’s disease Phase II Zurich, Switzerland (888) 669-6682 Novartis Pharmaceuticals East Hanover, NJ

CERE-110 Ceregene gene Alzheimer’s disease Phase II San Diego, CA therapy (858) 458-8800

CERE-120 Ceregene gene Parkinson’s disease Phase II San Diego, CA therapy (858) 458-8800

chondrocyte tissue ISTO Technologies cell back pain Phase I grafts St. Louis, MO therapy (314) 995-6049

DVM™ DaVinci Biosciences stem cell spinal cord injury in clinical trials bone marrow stem Costa Mesa, CA therapy www.dvbiosciences.com cell therapy

fulranumab Johnson & Johnson mAb back pain Phase II Pharmaceutical Research (800) 817-5286 & Development Raritan, NJ

GDNF-producing adult BrainStorm Cell Therapeutics stem cell amyotrophic lateral sclerosis Phase I/II stem cell therapy New York, NY therapy (212) 557-7200

Medicines in Development Biotechnology 2011 81 Biotechnology Medicines in Development

Neurologic Disorders

Product Name Sponsor Product Indication Development Phase Category

GNE lipoplex Gradalis gene muscular dystrophy Phase I (muscular dystrophy Carrollton, TX therapy (214) 442-8100 gene therapy)

GRNOPC 1 Geron stem cell spinal cord injury Phase I (human embryonic Menlo Park, CA therapy (650) 473-7700 stem cell [hESC]-derived cell therapy)

GSK249320 GlaxoSmithKline mAb central nervous system (CNS) Phase I Rsch. Triangle Park, NC trauma (888) 825-5249 (see also cardiovascular)

GSK1223249 GlaxoSmithKline mAb amytrophic lateral sclerosis Phase I Rsch. Triangle Park, NC (see also autoimmune) (888) 825-5249

GSK2402968 GlaxoSmithKline antisense Duchenne muscular dystrophy Phase I (PRO-051) Rsch. Triangle Park, NC (888) 825-5249 Prosensa Leiden, The Netherlands

GSK933776A GlaxoSmithKline mAb Alzheimer’s disease Phase I Rsch. Triangle Park, NC (see also eye) (888) 825-5249

Neo-Bladder Augment™ Tengion cell neurogenic bladder Phase II autologous cultured East Norriton, PA therapy www.tengion.com urological tissue cells

NLX P101 Neurologix gene Parkinson’s disease Phase II Fort Lee, NJ therapy (201) 592-6451

NSI-566RSC Neuralstem stem cell amyotrophic lateral sclerosis Phase I Rockville, MD therapy (301) 366-4960

PF-04360365 Pfizer mAb Alzheimer’s disease Phase II (ponezumab) New York, NY (860) 732-5156

PF-04427429 Pfizer unspecified migraine Phase I New York, NY (860) 732-5156

PG-110 Abbott Laboratories mAb pain Phase I completed Abbott Park, IL (847) 937-6100

ProCord Proneuron Biotechnologies cell complete spinal cord injury Phase II autologous activated New York, NY therapy (212) 758-9763 macrophage therapy (Orphan Drug)

RG1450 Roche mAb Alzheimer’s disease Phase II (gantenerumab) Nutley, NJ (973) 235-5000

82 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Neurologic Disorders

Product Name Sponsor Product Indication Development Phase Category

RG3039 Repligen protein spinal muscular atrophy Phase I Waltham, MA stimulant (Fast Track) (800) 622-2259

RG7412 AC Immune mAb Alzheimer’s disease Phase II (anti-Abeta) Lausanne, Switzerland (800) 626-3553 Genentech South San Francisco, CA

SB-509 Sangamo BioSciences DNA-binding amyotrophic lateral sclerosis Phase II Richmond, CA protein (see also diabetes) (510) 970-6000

solanezumab Eli Lilly mAb Alzheimer’s disease Phase III Indianapolis, IN (800) 545-5979

Soliris® Alexion Pharmaceuticals mAb multifocal motor neuropathy Phase II eculizumab Cheshire, CT (see also autoimmune, blood, (203) 272-2596 eye, transplantation, other)

stem cell therapy Stemedica Cell Technologies stem cell Parkinson’s disease in clinical trials San Diego, CA therapy (858) 658-0910

UB-311 United Biomedical vaccine Alzheimer’s disease Phase I Hauppauge, NY (631) 273-2828

V950 Merck vaccine Alzheimer’s disease Phase I Whitehouse Station, NJ (800) 672-6372

Respiratory Disorders

Product Name Sponsor Product Indication Development Phase Category

Aerovant™ Aerovance recombinant acute asthma Phase II pitrakinra Berkeley, CA protein (510) 549-5500

ALT-836 Altor BioScience mAb acute lung injury, adult respiratory Phase II Miramar, FL distress syndrome (954) 443-8600 (see also cancer)

AMG 157 Amgen mAb asthma Phase I Thousand Oaks, CA (see also skin) (800) 772-6436

AMG 761 Amgen mAb asthma Phase I Thousand Oaks, CA (800) 772-6436

Medicines in Development Biotechnology 2011 83 Biotechnology Medicines in Development

Respiratory Disorders

Product Name Sponsor Product Indication Development Phase Category

AMG 827 Amgen mAb asthma Phase II (brodalumab) Thousand Oaks, CA (see also digestive, skin) (800) 772-6436

anti-M1 prime mAb Genentech mAb allergic asthma, allergic rhinitis Phase II (RG7449) South San Francisco, CA (800) 626-3553

ASM8 Pharmaxis antisense asthma Phase II completed Exton, PA (610) 363-5120

Bosatria™ GlaxoSmithKline mAb asthma Phase II mepolizumab Rsch. Triangle Park, NC (see also blood) (888) 825-5249 (anti-IL5 mAb) ------(Orphan Drug) eosinophilic esophagitis in Phase I/II adolescents and children (888) 825-5249

carlumab Janssen Biotech mAb pulmonary fibrosis Phase I Horsham, PA (see also cancer) (800) 526-7736

CAT-354 AstraZeneca mAb asthma Phase II (tralokinumab) Wilmington, DE (800) 236-9933 MedImmune (301) 398-0000 Gaithersburg, MD

cat allergy vaccine Greer Laboratories vaccine perennial allergic rhinitis due to Phase I (sublingual) Lenoir, NC cat hair allergy (800) 378-3906

CNTO-3157 Janssen Biotech mAb asthma Phase I Horsham, PA (800) 526-7736

CNTO-5825 Janssen Biotech mAb allergic asthma Phase I Horsham, PA (800) 526-7736

Excellair™ ZoBeCor Pharmaceuticals RNA asthma Phase II syk kinase silencer Bala Cynwyd, PA interference (610) 660-7733

FG-3019 FibroGen mAb idiopathic pulmonary fibrosis Phase II South San Francisco, CA (see also cancer) (650) 866-7200

GC-1008 Genzyme mAb pulmonary fibrosis Phase I (fresolimumab) Cambridge, MA (see also cancer, other) (617) 252-7500

grass pollen allergy tablet Merck vaccine seasonal allergic rhinitis Phase III vaccine Whitehouse Station, NJ (800) 672-6372 (SCH-697243)

grass pollen extract Stallergenes vaccine seasonal allergic rhinitis Phase III Cedex, France www.stallergenes.com

84 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Respiratory Disorders

Product Name Sponsor Product Indication Development Phase Category

GS 6624 Gilead Sciences mAb idiopathic pulmonary fibrosis Phase I Foster City, CA (see also cancer, other) (800) 445-3235

GSK679586 GlaxoSmithKline mAb asthma Phase II completed Rsch. Triangle Park, NC (888) 825-5249

GSK2586881 Apeiron Biologics recombinant adult respiratory distress syndrome Phase I (APN01) Vienna, Austria protein (see also cancer, cardiovascular, (888) 825-5249 GlaxoSmithKline diabetes) Rsch. Triangle Park, NC

house dust mite Greer Laboratories vaccine allergic rhinitis Phase I allergy vaccine Lenoir, NC (800) 419-7302

house dust mite Merck vaccine allergic asthma Phase II allergy vaccine tablet Whitehouse Station, NJ (800) 672-6372 (SCH-900237)

Ilaris® Novartis Pharmaceuticals mAb chronic obstructive pulmonary Phase I/II completed canakinumab East Hanover, NJ disease (COPD) (888) 669-6682 (see also autoimmune, cardiovascular, diabetes, musculoskeletal)

injectable MPL allergy Allergy Therapeutics vaccine prevention of seasonal allergic Phase III vaccine West Sussex, United Kingdom rhinitis www.allergytherapeutics. com

MEDI-528 AstraZeneca mAb asthma Phase II (enokizumab) Wilmington, DE (800) 236-9933 Ligand Pharmaceuticals (858) 550-7500 La Jolla, CA (301) 398-0000 MedImmune Gaithersburg, MD

MEDI-563 AstraZeneca mAb asthma Phase II (benralizumab) Wilmington, DE (800) 236-9933 MedImmune (301) 398-0000 Gaithersburg, MD

MPL103 Allergy Therapeutics vaccine grass and ragweed allergies Phase III West Sussex, United Kingdom

PRM-151 Promedior recombinant idiopathic pulmonary fibrosis Phase I (rhPTX-2 for injection) Malvern, PA protein (see also eye) (610) 560-1435

Prochymal® Osiris Therapeutics stem cell COPD Phase II remestemcel-L Baltimore, MD therapy (see also cardiovascular, (410) 522-5005 diabetes, digestive, transplantation)

Medicines in Development Biotechnology 2011 85 Biotechnology Medicines in Development

Respiratory Disorders

Product Name Sponsor Product Indication Development Phase Category

QAX576 Novartis Pharmaceuticals mAb allergic rhinitis, eosinophilic Phase II East Hanover, NJ esophagitis, pulmonary fibrosis (888) 669-6682 (see also skin)

QGE031 Novartis Pharmaceuticals mAb asthma Phase I East Hanover, NJ (888) 669-6682

ragweed allergy Stallergenes vaccine seasonal allergic rhinitis Phase I immunotherapy Cedex, France www.stallergenes.com (sublingual)

ragweed allergy vaccine Greer Laboratories vaccine seasonal allergic rhinitis Phase III (sublingual) Lenoir, NC (800) 378-3906

ragweed allergy vaccine Merck vaccine seasonal allergic rhinitis Phase III (sublingual) Whitehouse Station, NJ (800) 672-6372

reslizumab Cephalon mAb asthma, eosinophilic esophagitis in Phase III (Orphan Drug) Frazer, PA adolescents and children (610) 344-0200

RG3637 Genentech mAb allergic asthma, asthma Phase II (lebrikizumab) South San Francisco, CA (see also cancer) (800) 626-3553

SAR156597 sanofi-aventis mAb idiopathic pulmonary fibrosis Phase I (bispecific interleukin-4/ Bridgewater, NJ (800) 633-1610 interleukin-13 mAb)

SAR231893 Regeneron Pharmaceuticals mAb asthma Phase II (anti-IL4 mAb) Tarrytown, NY (see also skin) (877) 734-6777 sanofi-aventis (800) 633-1610 Bridgewater, NJ

Veldona® Amarillo Biosciences interferon COPD, pulmonary fibrosis Phase II human interferon-alpha Amarillo, TX (see also autoimmune, infectious) (806) 376-1741

Skin Diseases

Product Name Sponsor Product Indication Development Phase Category

AbGn-168 Boehringer Ingelheim mAb plaque psoriasis Phase I Pharmaceuticals (800) 243-0127 Ridgefield, CT

Amevive® Astellas Pharma US recombinant psoriasis in adolescents Phase II alefacept Deerfield, IL fusion (800) 695-4321 protein

86 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Skin Diseases

Product Name Sponsor Product Indication Development Phase Category

AMG 139 Amgen mAb psoriasis Phase I Thousand Oaks, CA (see also digestive) (800) 772-6436

AMG 157 Amgen mAb atopic dermatitis Phase I Thousand Oaks, CA (see also respiratory) (800) 772-6436

AMG 827 Amgen mAb psoriasis Phase II (brodalumab) Thousand Oaks, CA (see also digestive, respiratory) (800) 772-6436

BT-061 Abbott Laboratories mAb plague psoriasis Phase II Abbott Park, IL (see also autoimmune) (847) 937-6100 Biotest www.biotest.de Dreieich, Germany

Enbrel® Amgen recombinant plague psoriasis in adolescents application submitted etanercept Thousand Oaks, CA fusion and children (800) 772-6436 protein (see also transplantation, other)

fezakinumab Pfizer mAb psoriasis Phase I (ILV-094) New York, NY (see also autoimmune) (860) 732-5156

fibroblast Fibrocell Science fibroblast scars Phase II/III cell therapy Exton, PA cell (see also other) (484) 713-6000 therapy

fibroblast growth CardioVascular Therapeutics fibroblast wounds in diabetes Phase II factor-1 Las Vegas, NV growth (see also cardiovascular) (702) 248-1174 factor

GBT 009 Garnet BioTherapeutics cell scar prevention Phase II Malvern, PA therapy (484) 395-2440

HP802-247 Healthpoint Biotherapeutics cell leg ulcers, skin ulcers Phase II (allogeneic human Fort Worth, TX therapy (800) 441-8227 skin replacement)

IL-17 antibody Eli Lilly mAb psoriasis Phase II (ixekizumab) Indianapolis, IN (see also autoimmune) (800) 545-5979

MK-3222 Merck mAb plaque psoriasis Phase II Whitehouse Station, NJ (800) 672-6372

NF-kappa-B decoy Transcription Factor Therapeutics oligo- atopic dermatitis Phase I/II oligonucleotide Irvine, CA nucleotide

otelixizumab GlaxoSmithKline mAb psoriasis Phase I completed (anti-CD3) Rsch. Triangle Park, NC (see also autoimmune) (888) 825-5249 ToleRx (617) 354-8100 Cambridge, MA

Medicines in Development Biotechnology 2011 87 Biotechnology Medicines in Development

Skin Diseases

Product Name Sponsor Product Indication Development Phase Category

ozoralizumab Pfizer mAb psoriatic arthritis Phase I (ATN-103) New York, NY (see also autoimmune, digestive) (860) 732-5156

QAX576 Novartis Pharmaceuticals mAb keloids after shave removal Phase II East Hanover, NJ (see also respiratory) (888) 669-6682

Remicade® Janssen Biotech mAb pyoderma in patients with Phase II infliximab Horsham, PA inflammatory bowel disease (800) 526-7736 (see also digestive)

RG4934 Roche mAb psoriatic arthritis Phase I (anti-IL-17 mAb) Nutley, NJ (973) 235-5000

Rituxan® Genentech mAb dermatomyositis Phase II rituximab South San Francisco, CA (see also cancer, transplantation) (800) 626-3553 Biogen Idec (781) 464-2000 Weston, MA

SAR231893 Regeneron Pharmaceuticals mAb atopic dermatitis Phase I (anti-IL4 mAb) Tarrytown, NY (see also respiratory) (914) 345-7400 sanofi-aventis (800) 633-1610 Bridgewater, NJ

SAR302532 Regeneron Pharmaceuticals mAb atopic dermatitis Phase I (REGN846) Tarrytown, NY (877) 734-6777 sanofi-aventis (800) 633-1810 Bridgewater, NJ

secukinumab Novartis Pharmaceuticals mAb psoriatic arthritis Phase II East Hanover, NJ (see also autoimmune, digestive, (888) 669-6682 eye) ------plaque psoriasis Phase I (888) 669-6682

Stelara® Janssen Biotech mAb psoriatic arthritis Phase III ustekinumab Horsham, PA (see also autoimmune, digestive) (800) 526-7736

StrataGraft® Stratatech cell chronic wounds Phase I/II tissue repair cell Madison, WI therapy (608) 441-2750 therapy

TD101 TransDerm RNA pachyonychia congenital Phase I Santa Cruz, CA interference (831) 420-1684

Xolair® Genentech mAb urticaria Phase III omalizumab South San Francisco, CA (see also other) (800) 626-3553 Novartis Pharmaceuticals (888) 669-6682 East Hanover, NJ

88 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Transplantation

Product Name Sponsor Product Indication Development Phase Category

ASKP-1240 Astellas Pharma US mAb transplant rejection Phase II Deerfield, IL (800) 695-4321 Kyowa Hakko Kirin (858) 952-7000 La Jolla, CA

Benlysta® GlaxoSmithKline mAb immunosuppression Phase II belimumab Rsch. Triangle Park, NC (see also autoimmune, blood) (888) 825-5249 (intravenous) Human Genome Sciences (301) 309-8504 Rockville, MD

Campath® Genzyme mAb graft-versus-host disease Phase I alemtuzumab Cambridge, MA (see also autoimmune, cancer) (617) 252-7500 (intravenous)

CaspaCIDe™ DLI Bellicum Pharmaceuticals cell graft-versus-host disease in Phase I T-cell replacement Houston, TX therapy patients with late-stage cancer www.bellicum.com therapy undergoing bone marrow transplantation

CDX-1135 Celldex recombinant allotransplant rejection Phase II Needham, MA protein (see also other) (781) 433-0771

conestat alfa Pharming Group recombinant renal transplant rejection Phase II (C1 inhibitor) Leiden, The Netherlands protein (see also genetic) www.pharming.com (Orphan Drug) Sanatrus (858) 314-5700 San Diego, CA ------delayed graft function Phase I www.pharming.com (858) 314-5700

CYT-107 Cytheris interleukin prevention of bone marrow Phase I (interleukin-7) Rockville, MD transplant rejection (301) 231-0450 (see also blood, cancer, HIV)

Enbrel® Amgen recombinant graft-versus-host disease Phase II etanercept Thousand Oaks, CA fusion (see also skin, other) (800) 772-6436 protein

MultiStem® Athersys stem cell graft-versus-host disease Phase I stem cell therapy Cleveland, OH therapy (see also cardiovascular, digestive) (216) 431-9900 (Orphan Drug)

Nulojix™ Bristol-Myers Squibb recombinant solid organ transplant rejection in clinical trials belatacept Princeton, NJ fusion (800) 332-2056 protein

Medicines in Development Biotechnology 2011 89 Biotechnology Medicines in Development

Transplantation

Product Name Sponsor Product Indication Development Phase Category

NX-001 Nephrx growth delayed graft function in Phase I (Orphan Drug) Kalamazoo, MI factor renal transplant patients (269) 372-8703

Prochymal® Osiris Therapeutics stem cell graft-versus-host disease Phase III remestemcel-L Baltimore, MD therapy (Fast Track) (410) 522-5005 (Orphan Drug) (see also cardiovascular, diabetes, digestive, respiratory)

QPI-1002 Quark Pharmaceuticals RNA prevention of delayed graft Phase II (Orphan Drug) Fremont, CA interference function (510) 402-4020 (see also other)

Rituxan® Genentech mAb graft-versus-host disease Phase II rituximab South San Francisco, CA (see also cancer, skin) (800) 626-3553 Biogen Idec (718) 464-2000 Weston, MA

Soliris® Alexion Pharmaceuticals mAb prevention of renal transplant Phase II eculizumab Cheshire, CT rejection (203) 272-2596 (see also autoimmune, blood, eye, neurologic, other)

TOL-101 Tolera Therapeutics mAb prevention of transplant rejection Phase I/II Kalamazoo, MI (877) 986-5372

YSPSL Y’s Therapeutics recombinant reperfusion injury associated with Phase II (Orphan Drug) Tokyo, Japan fusion liver transplantation www.ysthera.com protein ------delayed graft function associated Phase I/II with kidney transplantation www.ysthera.com

ZRx-101 ZelleRx cell therapy graft-versus-host disease Phase I Chicago, IL (see also cancer) (312) 243-5200

Other

Product Name Sponsor Product Indication Development Phase Category

ACE-011 Acceleron Pharma recombinant end-stage renal disease Phase I (sotatercept) Cambridge, MA fusion (see also blood, cancer, (617) 649-9200 Celgene protein musculoskeletal) (908) 673-9000 Summit, NJ

AGN-214868 Allergan recombinant overactive bladder with urinary Phase II Irvine, CA protein incontinence (949) 788-6700 (see also neurologic)

90 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Other

Product Name Sponsor Product Indication Development Phase Category

allogeneic multi-potent AlloCure stem cell acute kidney injury Phase I stem cell therapy Burlington, MA therapy (781) 425-5380

ALN-TTR01 Alnylam Pharmaceuticals RNA amyloidosis Phase I Cambridge, MA interference (617) 551-8200

AMG 191 Amgen mAb inflammation Phase I Thousand Oaks, CA (800) 772-6436

antibody therapeutic MorphoSys mAb undisclosed Phase II Munich, Germany (888) 669-6682 Novartis Pharmaceuticals East Hanover, NJ

anti-IL-23 mAb Merck mAb inflammation Phase I Whitehouse Station, NJ (800) 672-6372

BAFF antibody Eli Lilly mAb end-stage renal disease Phase II (tabalumab) Indianapolis, IN (see also autoimmune, cancer) (800) 545-5979

cat allergy immunotherapy Planet Biopharmaceuticals immuno- cat allergy (hypersensitivity) Phase II (sublingual) Ridgefield, CT therapy (800) 255-3749

CDX-1135 Celldex Therapeutics recombinant kidney disorders Phase I Needham, MA protein (see also transplantation) (781) 433-0771

CNTO-530 Edison Pharmaceuticals recombinant mitochondrial disorders in clinical trials Mountain View, CA fusion (see also blood) www.edisonpharma.com protein

ELAD® Vital Therapies cell liver failure Phase II/III extracorporeal liver San Diego, CA therapy (858) 673-6840 assist device (Orphan Drug)

Enbrel® Amgen recombinant metabolic syndrome Phase II etanercept Thousand Oaks, CA fusion (see also skin, transplantation) (800) 772-6436 protein

fibroblast cell therapy Fibrocell Science fibroblast periodontal disorders Phase II Exton, PA cell therapy (see skin) (484) 713-6000

food allergy DBV Technologies vaccine peanut hypersensitivity Phase I immunotherapy Paris, France www.dbv-technologies. com

GC-1008 Genzyme mAb focal segmental Phase I (fresolimumab) Cambridge, MA glomerulosclerosis (617) 252-7500 (see also cancer, respiratory)

Medicines in Development Biotechnology 2011 91 Biotechnology Medicines in Development

Other

Product Name Sponsor Product Indication Development Phase Category

GS 6624 Gilead Sciences mAb myelofibrosis Phase II Foster City, CA (see also cancer, respiratory) (800) 445-3235

GSK1070806 GlaxoSmithKline mAb metabolic disorders Phase I (anti-interleukin 18 mAb) Rsch. Triangle Park, NC (888) 825-5249

HLA-DQ2 peptide vaccine ImmusanT peptide celiac disease Phase I Cambridge, MA vaccine www.immusant.com

hMaxi-K Ion Channel Innovations gene erectile dysfunction, Phase I gene therapy New York, NY therapy overactive bladder (336) 713-7296

Humira® Abbott Laboratories mAb interstitial cystitis Phase III adalimumab Abbott Park, IL (see also autoimmune, digestive) (847) 937-6100

iontophoretic warts gene NB Therapeutics gene non-genital warts Phase II therapy Bristol, PA therapy (866) 957-6200

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

MEDI-545 AstraZeneca mAb myositis Phase II (sifalimumab) Wilmington, DE (see also autoimmune) (800) 236-9933 Medarex (609) 430-2880 Princeton, NJ (301) 398-0000 MedImmune Gaithersburg, MD

microencapsulated Curalogic immuno- cat allergy Phase II cat hair extract Copenhagen, Denmark therapy

NIC002 Novartis Pharmaceuticals vaccine smoking withdrawal Phase II East Hanover, NJ (888) 669-6682

NicVax™ GlaxoSmithKline vaccine smoking withdrawal Phase II nicotine abuse vaccine Rsch. Triangle Park, IL (Fast Track) (888) 825-5249 Nabi Biopharmaceuticals (800) 685-1766 Boca Raton, FL

PF-05212389 Pfizer unspecified obesity Phase I (OAP-189) New York, NY (see also diabetes) (860) 732-5156

92 Medicines in Development Biotechnology 2011 Biotechnology Medicines in Development

Other

Product Name Sponsor Product Indication Development Phase Category

Preos® NPS Pharmaceuticals recombinant hypoparathyroidism Phase III recombinant human Salt Lake City, UT hormone (801) 583-4939 parathyroid hormone (rhPTH) (Orphan Drug)

QPI-1002 Quark Pharmaceuticals RNA prevention of renal failure in Phase I/II Fremont, CA interference patients undergoing cardiovascular (510) 402-4020 surgery (see also transplantation)

REGN728 Regeneron Pharmaceuticals mAb undisclosed Phase I Tarrytown, NY (877) 734-6777 sanofi-aventis (800) 633-1810 Bridgewater, NJ

Soliris® Alexion Pharmaceuticals mAb atypical hemolytic uremic application submitted eculizumab Cheshire, CT syndrome (203) 272-2596 (Orphan Drug) ------type II membranoproliferative Phase II glomerulonephritis (203) 272-2596 (see also autoimmune, blood, eye, neurologic, transplantation)

STX-100 Stromedix mAb fibrosis Phase I completed Cambridge, MA (617) 674-8400

TA-CD Celtic Pharma immuno- cocaine abuse Phase II Hamilton, Bermuda therapeutic (441) 299-7440 vaccine

Victoza® Novo Nordisk recombinant obesity Phase III liraglutide Princeton, NJ peptide (609) 987-5800 (rDNA origin) injection

Xolair® Genentech mAb peanut allergy Phase II omalizumab South San Francisco, CA (see also skin) (800) 626-3553 Novartis Pharmaceuticals (888) 669-6682 East Hanover, NJ

Medicines in Development Biotechnology 2011 93 Glossary

adjunct—An auxiliary treatment that is sec- infants and immunocompromised individuals. heat shock protein—A natural adjuvant (a ondary to the main treatment. CMV can occur without symptoms or result in substance that enhances the immune response mild flu-like symptoms. to an antigen) linked to molecular signals or adjuvant—Substance or drug that aids an- antigenic peptides, which are capable of stimu- other substance in its action. erythropoietin—Hormone that stimulates red lating an immune response. The heat shock blood cell production in the bone marrow. protein (HSP) core is an invariable molecule angioedema—Swelling of the mucous mem- that remains the same for each patient; the an- branes, tissues beneath the skin, or an internal Fast Track—A process designed to facilitate tigenic peptides are patient- and tumor-specific organ due to an allergic reaction. the development and expedite the review or pathogen-specific. When purified in a certain of drugs to treat serious diseases and fill an way, HSP-peptide complexes constitute the ankylosing spondylitis—An inflammatory unmet medical need. The status is assigned “fingerprint” of the cell from which they were disorder of unknown cause that primarily by the U.S. Food and Drug Administration. derived. When injected into a patient, these affects the spine. The vertebrae may fuse The purpose is to get important new drugs antigenic peptides are re-expressed on the together and form a rigid back that is impos- to the patient earlier. Fast Track addresses a surface of important immune cells—macro- sible to bend. broad range of serious diseases. Generally, phages—that play a major role in activating the determining factors include whether the drug antisense—An antisense drug is the mirror or immune system, resulting in a much more po- will have an impact on such factors as survival, complementary image of a small segment of tent immune response than the one achieved day-to-day functioning, or the likelihood that messenger RNA (mRNA), the substance that by the expression of these same peptides by the disease, if left untreated, will progress carries instructions (“sense”) from the genes to the tumor or pathogen-infected cell. from a less severe condition to a more serious the cell’s protein-making machinery. The anti- one. Filling an unmet medical need is defined sense drug readily binds to the mRNA strand, hemophilia A and B—Hemophilia A, the “clas- as providing a therapy where none exists or keeping it from transmitting its instructions to sic” hemophilia, is a genetic bleeding disorder providing a therapy which may be potentially the cell, and thus inhibiting the production of an due to deficiency of the coagulation factor superior to existing therapy. Once a drug unwanted protein. VIII. Hemophilia B, or “Christmas” disease, is receives Fast Track designation, early and caused by deficiency of coagulation factor IX. application submitted—An application for frequent communication between the FDA and marketing has been submitted by the company a drug company is encouraged throughout the hepatitis—Inflammation of the liver with ac- to the Food and Drug Administration (FDA). entire drug development and review process. companying liver cell damage or death, caused The frequency of communication assures that most often by viral infection, e.g., hepatitis B, biotechnology—The collection of processes questions and issues are resolved quickly, of- and C. that involve the use of biological systems. ten leading to earlier drug approval and access For some of the industries, these processes by patients. herpes simplex virus—A strain of herpes involve the use of genetically engineered virus that may lie dormant in nerve tissue and organisms. For the purpose of this report, only Gaucher’s disease—An inherited disease can be reactivated to produce painful sores of those products that involve recombinant DNA, caused by a lack or deficiency of an enzyme the anus or genitals. monoclonal antibody/hybridoma, continuous (glucocerebrosidase). Primarily affects the liver, human papillomavirus—Viral agent of warts, cell lines, cellular therapy, gene therapy and spleen and bone marrow, usually resulting in believed to be contagious and usually harm- vaccines technology are included. death. less, but it can lead to cervical cancer. Clostridium difficile—A bacterium that gene therapy—Therapy at the intracellular interferon—A glycoprotein naturally produced produces an irritating toxin that causes a form level to replace or inactivate the effects of by cells that interferes with the ability of a virus of colitis characterized by profuse, watery diar- disease-causing genes or to augment normal to reproduce after it invades the body. rhea with cramps and low-grade fever. gene functions to overcome illness. interleukin—An endogenous substance that colony stimulating factor (CSF)—Protein glioblastoma—The most common primary stimulates the production of different types of responsible for controlling the production of brain tumor and one of the most aggressive white blood cells or leukocytes. white blood cells. forms of brain cancer, primarily affecting adults over the age of 50. ischemia—Insufficient supply of blood to an Crohn’s disease—A subacute chronic gastro- organ or tissue, which can cause organ dam- intestinal disorder, involving the small intestine, growth factors—Factors responsible for age such as an ischemic stroke. characterized by patchy deep ulcers that may regulating cell proliferation (rapid and repeated reproduction), function and differentiation. cause fistulas and a narrowing and thickening lymphoma—Cancers in which the cells of of the bowel. growth hormone—Pituitary hormone that lymphoid tissue, found mainly in the lymph nodes and spleen, multiply unchecked. cytomegalovirus (CMV)—A DNA virus related stimulates the growth of long bones in prepu- Lymphomas fall into two categories: One is to the herpes virus, affecting mostly neonatal bertal children.

94 Medicines in Development Biotechnology 2011 Glossary

called Hodgkin’s disease, characterized by a myocardial infarction—Damage to the pulmonary hypertension—High blood pres- particular kind of abnormal cell. All others are heart muscle caused by stoppage or impair- sure in the arteries that supply blood to the called non-Hodgkin’s lymphomas, which vary ment of blood flow to the heart, also known lungs. in their malignancy according to the nature and as heart attack. activity of the abnormal cells. pulmonary fibrosis—Scarring and thickening neuroblastoma—A tumor of the adrenal of lung tissue. mAb (monoclonal antibodies)—Large protein glands or sympathetic nervous system (the molecules produced by white blood cells that part of the nervous system responsible for respiratory syncytial virus (RSV) disease— seek out and destroy harmful foreign substances. certain automatic body functions, such as the One of the most important causes of lower “fight or flight response”). respiratory tract disease in children, account- macular degeneration—A progressive disor- ing for more than 90 percent of cases of der that affects the central part of the retina, neutropenia—An abnormally low neutrophil bronchiolitis. causing gradual loss of vision. It is a painless count (certain white blood cells). condition, usually affects both eyes and is com- restenosis—A condition where an artery plugs mon in the elderly. Orphan Drug—A drug to treat a disease that up again following treatment to open it up. has a patient population of 200,000 or less metastases/metastatic—Cancers that have in the United States, or a disease that has a sepsis—A condition associated with a serious spread from the primary or original cancer site. patient population of more than 200,000 and bacterial infection of the blood. Cancer cells can break away from a tumor and a development cost that will not be recovered short bowel syndrome—The malabsorptive travel to other areas of the body through the from sales in the United States. condition that arises after significant segments bloodstream or the lymph system. They are of- of the small intestine are removed. ten more difficult to treat then primary tumors. peripheral vascular disorder—The obstruc- tion of blood supply to the extremities, particu- systemic lupus erythematosus (SLE)—The multiple myeloma—A malignant condition char- larly the legs, caused by atherosclerosis. most serious form of lupus, a chronic autoim- acterized by uncontrolled proliferation of plasma mune disorder causing inflammation and cells (a class of white blood cells) in bone Phase 0—First-in-human trials conducted damage to multiple organs. marrow. Symptoms include pain and destruction in accordance with FDA’s 2006 guidance on exploratory Investigational New Drug (IND) of bone tissue, numbness and paralysis, kidney thrombocytopenia—A reduction in the studies designed to speed up development of damage, anemia, and frequent infections. number of platelet cells in the blood, which promising drugs by establishing very early on causes a tendency to bleed, especially from myelodysplastic syndromes (MDS)—Blood whether the agent behaves in human subjects the smaller blood vessels. disorders that ultimately are fatal. Patients as was anticipated from preclinical studies. usually succumb to infections or bleeding. The ulcerative colitis—A chronic inflammation and Phase I—Safety testing and pharmacological term “preleukemia” has been used to describe ulceration of the lining of the colon and rectum. profiling in humans. these disorders. It causes bloody diarrhea and mainly involves the left colon. myeloid—Pertaining to, derived from, or mani- Phase II—Effectiveness and safety testing in humans. festing certain features of the bone marrow. In uveitis—Inflammation of the uvea, the middle some cases, myeloid also pertains to certain Phase III—Extensive clinical trials to demon- layer of the eye. types of non-lymphocyte white blood cells strate safety and efficacy in humans. found in the bone marrow.

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 August 11, 2011. 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 © 2011 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 Biotechnology 2011 95 The Drug Discovery, Development and Approval Process

It takes 10-15 years on average for an experimental drug to travel from the lab to U.S. patients. Only five in 5,000 compounds that enter preclinical testing make it to human testing. One of these five tested in people is approved.

Clinical Trials

Discovery/ Preclinical Testing Phase I Phase II Phase III FDA Phase IV

Years 6.5 1.5 2 3.5 1.5

20 to 100 100 to 500 1,000 to 5,000 Test Laboratory and

healthy patient patient at FDA Population animal studies volunteers volunteers volunteers Additional Review post- process/ marketing Confirm Assess safety, Evaluate approval testing

File IND at FDA Determine effectiveness, biological activity effective- required Purpose safety monitor adverse and formulations ness, look for File NDA/BLA by FDA and dosage reactions from side effects long-term use

5,000 Success compounds 5 1 Rate evaluated enter trials approved

The Drug Development and Approval Process The U.S. system of new drug approvals is Administration (FDA) to begin to test the drug hospitals. Physicians monitor patients closely to perhaps the most rigorous in the world. in people. The IND shows results of previous confirm efficacy and identify adverse events. experiments; how, where and by whom the It takes 10-15 years, on average, for an experi- New Drug Application (NDA)/Biologic new studies will be conducted; the chemical mental drug to travel from lab to U.S. patients, License Application (BLA). Following the structure of the compound; how it is thought according to the Tufts Center for the Study of completion of all three phases of clinical trials, to work in the body; any toxic effects found in Drug Development, based on drugs approved a company analyzes all of the data and files an the animal studies; and how the compound from 1994 through 1998. Only five in 5,000 NDA or BLA with FDA if the data successfully is manufactured. All clinical trials must be compounds that enter preclinical testing make demonstrate both safety and effectiveness. reviewed and approved by the Institutional it to human testing. And only one of those five The applications contain all of the scientific Review Board (IRB) where the trials will be is approved for sale. information that the company has gathered. conducted. Progress reports on clinical trials Applications typically run 100,000 pages or On average, it costs a company $1.2 billion, must be submitted at least annually to FDA and more. The average review time for the 26 new including the cost of failures, to get one new the IRB. medicines approved by the FDA in 2010 was medicine from the laboratory to U.S. patients, Clinical Trials, Phase I. These tests usually 14.1 months. according to a 2007 study by the Tufts Center involve about 20 to 100 healthy volunteers. The for the Study of Drug Development. Approval. Once FDA approves an NDA or tests study a drug’s safety profile, including the BLA, the new medicine becomes available Once a new compound has been identified in safe dosage range. The studies also determine for physicians to prescribe. A company must the laboratory, medicines are developed as how a drug is absorbed, distributed, metabo- continue to submit periodic reports to FDA, follows: lized, and excreted as well as the duration of including any cases of adverse reactions and its action. Preclinical Testing. A pharmaceutical com- appropriate quality-control records. For some pany conducts laboratory and animal studies Clinical Trials, Phase II. In this phase, medicines, FDA requires additional trials to show biological activity of the compound controlled trials of approximately 100 to 500 (Phase IV) to evaluate long-term effects. against the targeted disease, and the com- volunteer patients (people with the disease) Discovering and developing safe and effective pound is evaluated for safety. assess a drug’s effectiveness and determine new medicines is a long, difficult, and expensive the early side effect profile. Investigational New Drug Application (IND). process. Pharmaceutical companies invested an After completing preclinical testing, a company Clinical Trials, Phase III. This phase usually estimated $67.4 billion in research and develop- files an IND with the U.S. Food and Drug involves 1,000 to 5,000 patients in clinics and ment in 2010.