PhRMA Report 2013: Medicines in Development: Biologics
 

PhRMA Report 2013: Medicines in Development: Biologics

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

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

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

• 338 cancer therapeutics that target several different types of solid tumors, leukemia and lymphoma. Monoclonal
antibodies account for 170 of the 338 products in development.
• 176 candidates in development for an array of infectious diseases, including 134 vaccines.
• 71 medicines for autoimmune diseases, such as lupus, multiple sclerosis and rheumatoid arthritis.
• 58 treatments for cardiovascular diseases, such as congestive heart failure and stroke.
• Other diseases include diabetes, digestive disorders, genetic disorders, neurologic and respiratory disorders.

Definitions for selected product categories and diseases can be found on page 86. For information on the history and
future of biologics research, the science behind the medicines, and a discussion of issues critical to continued discovery
and development of these cutting-edge medicines, please see Biologic Medicines in Development 2013—Overview.

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PhRMA Report 2013: Medicines in Development: Biologics PhRMA Report 2013: Medicines in Development: Biologics Document Transcript

  • 2013 REPORT Medicines in Development Biologics presented by america’s biopharmaceutical research companies Biologics Research Pushing Frontiers of Science With More Than 900 Medicines and Vaccines in Development In the human body there are Biologics In Development 12 trillion cells, By Product Category and 200,000 proteins and Development Phase 25,000 genes 338 Application Submitted Phase III Phase II America’s biopharmaceutical research • treatments for cardiovascular 58 Phase I companies are developing 907 biologics diseases, such as congestive heart 250 targeting more than 100 diseases. Biologics failure and stroke. are developed through biological processes • Other diseases include diabetes, using living cells or organisms. digestive disorders, genetic disorders, This report lists biologics in human neurologic and respiratory disorders. clinical trials or under review by the Definitions for selected product categories U.S. Food and Drug Administration and diseases can be found on page 86. (FDA). A link to the sponsor company’s web site provides more detailed infor- For information on the history and mation on the science and technologies future of biologics research, the science behind each potential product. The 907 behind the medicines, and a discussion medicines and vaccines in development of issues critical to continued discovery include: and development of these cutting-edge medicines, please see Biologic Medicines • cancer therapeutics that target 338 in Development 2013—Overview. 93 several different types of solid tumors, 81 leukemia and lymphoma. Monoclonal The medicines in this report reflect the 69 antibodies account for 170 of the 338 new ways America’s biopharmaceuti- products in development. cal research companies are attacking 46 disease through biotechnology. The 907 • candidates in development for an 176 medicines and vaccines in development 30 array of infectious diseases, including promise to push the frontiers of science 134 vaccines. and bring new treatments to patients for • medicines for autoimmune diseases, 71 our more challenging diseases. such as lupus, multiple sclerosis and e py ins s er y s ine ns ap die rheumatoid arthritis. oth ra ote e er cc the botis h pr va ntian ll t ne nt la ce ina ge na mb clo co no re mo
  • Biologic Medicines in Development Autoimmune Disorders Product Name Sponsor Product Indication Development Phase* Category ABT-122 AbbVie mAb rheumatoid arthritis Phase I North Chicago, IL abbvie.com Actemra® Genentech mAb early rheumatoid arthritis Phase III tocilizumab South San Francisco, CA gene.com Roche ------------------------------------------------ -------------------------------- Nutley, NJ systemic sclerosis Phase II gene.com AGS-009 Argos Therapeutics mAb systemic lupus erythematosus Phase I Durham, NC (SLE) argostherapeutics.com alemtuzumab Genzyme mAb relapsing-remitting multiple application submitted Cambridge, MA sclerosis (Fast Track) genzyme.com Alferon N Injection® Hemispherx Bioscience interferon multiple sclerosis Phase II interferon-alpha-n3 Philadelphia, PA hemispherx.net subcutaneous AMG 557/MEDI-5872 Amgen mAb SLE Phase I Thousand Oaks, CA amgen.com AstraZeneca astrazeneca.com Wilmington, DE medimmune.com MedImmune Gaithersburg, MD AMG 729 Amgen mAb autoimmune diseases Phase I Thousand Oaks, CA amgen.com AMG 811 Amgen mAb discoid lupus erythematosus, Phase I Thousand Oaks, CA SLE amgen.com anti-IL17 mAb Genentech mAb autoimmune disorders Phase I (RG7624) South San Francisco, CA gene.com anti-LINGO Biogen Idec mAb multiple sclerosis Phase II (BIIB033) Weston, MA (see also eye) biogenidec.com ARX424 Ambrx interferon multiple sclerosis Phase I (PEGylated interferon La Jolla, CA ambrx.com beta-1a) atacicept EMD Serono recombinant SLE Phase II/III (TACI-lg) Rockland, MA fusion emdserono.com protein*For more information about a specific medicine or company in the report, please click on the provided link.2 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Autoimmune Disorders Product Name Sponsor Product Indication Development Phase Category ATX-MS-1467 EMD Serono peptide multiple sclerosis Phase I Rockland, MA vaccine emdserono.com AZ01 Allozyne interferon multiple sclerosis Phase I (long-acting pegylated Seattle, WA allozyne.com interferon beta) Benlysta® GlaxoSmithKline mAb SLE Phase III belimumab Rsch. Triangle Park, NC (see also blood, transplantation) gsk.com (subcutaneous) ------------------------------------------------ -------------------------------- systemic scleroderma gsk.com BHT-3009 Bayhill Therapeutics DNA multiple sclerosis Phase II Palo Alto, CA vaccine bayhilltx.com BI-695500 Boehringer-Ingelheim mAb rheumatoid arthritis Phase III (rituximab biosimilar) Pharmaceuticals boehringer-ingelheim. Ridgefield, CT com BI-695501 Boehringer Ingelheim mAb rheumatoid arthritis Phase I (adalimumab biosimilar) Pharmaceuticals boehringer-ingelheim. Ridgefield, CT com BIIB017 Biogen Idec interferon relapsing multiple sclerosis Phase III (PEG-interferon Weston, MA (Fast Track) biogenidec.com beta-1a) BT-061 AbbVie mAb rheumatoid arthritis Phase II North Chicago, IL (see also skin) abbvie.com Biotest biotest.de Dreieich, Germany Cimzia® UCB mAb ankylosing spondylitis, Phase III certolizumab pegol Smyrna, GA juvenile rheumatoid arthritis ucb.com (see also musculoskeletal) clazakizumab Bristol-Myers Squibb mAb rheumatoid arthritis Phase II (anti-IL6) Princeton, NJ (see also digestive, bms.com Alder Biopharmaceuticals musculoskeletal) alderbio.com Bothell, WA CNTO-136 Janssen Biotech mAb rheumatoid arthritis Phase III (sirukumab) Horsham, PA (see also other) janssenbiotech.com CNTO-1959 Janssen Biotech mAb rheumatoid arthritis Phase II Horsham, PA (see also skin) janssenbiotech.comMedicines in Development Biologics 2013 3
  • Biologic Medicines in DevelopmentAutoimmune DisordersProduct Name Sponsor Product Indication Development Phase Categorydaclizumab AbbVie mAb multiple sclerosis (relapsing forms) Phase III North Chicago, IL abbvie.com Biogen Idec biogenidec.com Weston, MAepratuzumab Immunomedics mAb SLE Phase III Morris Plains, NJ (Fast Track) immunomedics.com UCB ucb.com Smyrna, GAGSK1223249 GlaxoSmithKline mAb multiple sclerosis Phase I(NOGO-A mAb) Rsch. Triangle Park, NC (see also neurologic) gsk.comHumira® AbbVie mAb rheumatoid arthritis Phase IIIadalimumab North Chicago, IL (combination therapy) abbvie.com (see also digestive, eye, musculoskeletal, skin, other)Ilaris® Novartis Pharmaceuticals mAb systemic juvenile idiopathic arthritis Phase IIIcanakinumab East Hanover, NJ (see also cardiovascular, diabetes, novartis.com(Orphan Drug) musculoskeletal)ISIS-CRPRx Isis Pharmaceuticals antisense rheumatoid arthritis Phase II Carlsbad, CA (see also cardiovascular) isispharm.commavrilimumab AstraZeneca mAb rheumatoid arthritis Phase II Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MDMEDI-546 AstraZeneca mAb SLE Phase II(anti-IFN-alphaR mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MDMEDI-551 AstraZeneca mAb scleroderma Phase I(anti-CD19 mAb) Wilmington, DE (see also cancer) astrazeneca.com MedImmune medimmune.com Gaithersburg, MDMEDI-570 AstraZeneca mAb SLE Phase I(anti-ICOS mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MD4 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Autoimmune Disorders Product Name Sponsor Product Indication Development Phase Category NeuroVax™ Immune Response Biopharma peptide multiple sclerosis Phase II (IR208) New York, NY vaccine immuneresponsebio pharma.com NN8209 Novo Nordisk mAb rheumatoid arthritis Phase II (anti-C5aR-151 mAb) Princeton, NJ novonordisk.com NN8210 Novo Nordisk mAb rheumatoid arthritis Phase I (anti-C5aR-215 mAb) Princeton, NJ novonordisk.com NN8226 Novo Nordisk mAb rheumatoid arthritis Phase II (anti-IL-20 mAb) Princeton, NJ novonordisk.com NN8765 Novo Nordisk mAb rheumatoid arthritis Phase I (anti-NKG2 mAb) Princeton, NJ novonordisk.com NN8828 Novo Nordisk mAb rheumatoid arthritis Phase II (anti-IL-21 mAb) Princeton, NJ novonordisk.com -------------------------------- -------------------------------- SLE Phase I novonordisk.com Nplate™ Amgen recombinant immune thrombocytopenic Phase III romiplostim Thousand Oaks, CA fusion purpura in children amgen.com (Orphan Drug) protein (see also cancer) NU-100 Nuron Biotech recombinant multiple sclerosis Phase III (interferon beta-1b) Exton, PA interferon nuronbiotech.com ocrelizumab Biogen Idec mAb primary-progressive multiple Phase III (anti-CD20 mAb) Weston, MA sclerosis biogenidec.com Genentech gene.com South San Francisco, CA ------------------------------------------------ --------------------------------- Roche relapsing-remitting multiple Phase III Nutley, NJ sclerosis biogenidec.com gene.com ofatumumab GlaxoSmithKline mAb multiple sclerosis, Phase II (subcutaneous) Rsch. Triangle Park, NC rheumatoid arthritis gsk.com olokizumab UCB mAb rheumatoid arthritis Phase II Smyrna, GA ucb.com Orencia® Bristol-Myers Squibb recombinant early rheumatoid arthritis Phase III abatacept Princeton, NJ fusion protein (see also musculoskeletal, other) bms.comMedicines in Development Biologics 2013 5
  • Biologic Medicines in DevelopmentAutoimmune DisordersProduct Name Sponsor Product Indication Development Phase Categoryotelixizumab GlaxoSmithKline mAb rheumatoid arthritis Phase I(anti-CD3 mAb) Rsch. Triangle Park, NC gsk.comozoralizumab Ablynx mAb rheumatoid arthritis Phase II completed(ATN-103) Ghent, Belgium ablynx.compateclizumab Genentech mAb rheumatoid arthritis Phase II(anti-LT alpha mAb) South San Francisco, CA gene.comPD-360324 Pfizer mAb cutaneous lupus erythematosus Phase I New York, NY pfizer.comPDA-001 Celgene Cellular Therapeutics stem cell rheumatoid arthritis Phase II(cenplacel-L) Summit, NJ therapy (see also digestive) celgene.com ------------------------------------------------ --------------------------------- multiple sclerosis, sarcoidosis Phase I celgene.comPF-04236921 Pfizer mAb SLE Phase II New York, NY (see also digestive) pfizer.com ------------------------------------------------ --------------------------------- rheumatoid arthritis Phase I pfizer.comPF-05280586 Pfizer mAb rheumatoid arthritis Phase I/II(rituximab biosimilar) New York, NY pfizer.complovamer acetate EMD Serono peptide multiple sclerosis Phase I(second-generation Rockland, MA emdserono.compeptide copolymer)Prolia® Amgen mAb rheumatoid arthritis Phase IIdenosumab Thousand Oaks, CA (see also musculoskeletal) amgen.comRAVAX™ Immune Response BioPharma vaccine rheumatoid arthritis Phase IIIrheumatoid arthritis New York, NY immuneresponsebiovaccine pharma.comrontalizumab Genentech mAb SLE Phase II(RG7415) South San Francisco, CA gene.comSAN-300 Santarus mAb rheumatoid arthritis Phase I(anti-VLA-1 antibody) San Diego, CA santarus.com6 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Autoimmune Disorders Product Name Sponsor Product Indication Development Phase Category SAR113244 Sanofi US mAb SLE Phase I (anti-CXCR5 mAb) Bridgewater, NJ sanofi.com sarilumab Regeneron Pharmaceuticals mAb rheumatoid arthritis Phase III (SAR153191) Tarrytown, NY regeneron.com Sanofi US sanofi.com Bridgewater, NJ secukinumab Novartis Pharmaceuticals mAb ankylosing spondylitis, Phase III (AIN457) East Hanover, NJ rheumatoid arthritis novartis.com (see also eye, musculoskeletal, skin) ------------------------------------------------ --------------------------------- multiple sclerosis Phase II novartis.com sifalimumab AstraZeneca mAb SLE Phase II (anti-IFN-alpha mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MD Simponi® Janssen Biotech mAb rheumatoid arthritis application submitted golimumab Horsham, PA (intravenous) janssenbiotech.com (Orphan Drug) (see also digestive) ------------------------------------------------ --------------------------------- juvenile rheumatoid arthritis Phase III (subcutaneous) janssenbiotech.com ----------------------------------------------- --------------------------------- sarcoidosis Phase II completed (subcutaneous) janssenbiotech.com Soliris® Alexion Pharmaceuticals mAb severe or refractory myasthenia Phase II eculizumab Cheshire, CT gravis alxn.com (Orphan Drug) (see also eye, infectious, transplantation) Stelara® Janssen Biotech mAb rheumatoid arthritis, sarcoidosis Phase II ustekinumab Horsham, PA (see also digestive, janssenbiotech.com musculoskeletal, other) tabalumab Eli Lilly mAb SLE Phase III (BAFF inhibitor) Indianapolis, IN (see also cancer) lilly.com Tcelna™ Opexa Therapeutics cell therapy multiple sclerosis Phase II imilecleucel-T The Woodlands, TX vaccine (Fast Track) opexatherapeutics.com Tysarbi® Biogen Idec mAb secondary-progressive multiple Phase III natalizumab Weston, MA sclerosis biogenidec.comMedicines in Development Biologics 2013 7
  • Biologic Medicines in DevelopmentAutoimmune DisordersProduct Name Sponsor Product Indication Development Phase Categoryveltuzumab Immunomedics mAb immune thrombocytopenic Phase II(IMMU-106) Morris Plains, NJ purpura, rheumatoid arthritis immunomedics.com Takeda Pharmaceuticals USA (see also cancer) takeda.com Deerfield, ILVX15 Teva Pharmaceuticals mAb multiple sclerosis Phase I Tikva, Israel (see also cancer) tevapharm.com Vaccinex vaccinex.com Rochester, NYXmAb®5871 Xencor mAb autoimmune disorders Phase I(anti-CD19 mAb) Monrovia, CA xencor.comBlood DisordersProduct Name Sponsor Product Indication Development Phase CategoryACE-536 Acceleron Pharma recombinant anemia Phase I Cambridge, MA fusion acceleronpharma.com Celgene protein celgene.com Summit, NJALXN1102/ALXN1103 Alexion Pharmaceuticals recombinant paroxysmal nocturnal Phase I Cheshire, CT fusion hemoglobinuria alxn.com proteinAMT-060 uniQure gene therapy hemophilia B Phase I/II(Orphan Drug) Amsterdam, The Netherlands uniqure.com St. Jude Children’s Research Hospital Memphis, TNART123 Asahi Kasei Pharma America recombinant disseminated intravascular Phase III(recombinant human Waltham, MA protein coagulation akpamerica.comthrombomodulin alpha)AskBio009 Asklepios BioPharmaceutical gene therapy hemophilia B Phase I/II Chapel Hill, NC askbio.com Baxter International baxter.com Deerfield, ILATryn® GTC Biotherapeutics recombinant heparin resistance in patients under- Phase IIIantithrombin alfa Framingham, MA protein going CABG surgery gtc-bio.com(Orphan Drug)8 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Blood Disorders Product Name Sponsor Product Indication Development Phase Category BAX-111 Baxter International recombinant von Willebrand disease Phase III (recombinant Deerfield, IL protein baxter.com von Willebrand factor) (Orphan Drug) BAX-326 Baxter International recombinant hemophilia B application submitted (rFIX) Deerfield, IL protein baxter.com (Orphan Drug) BAX-817 Baxter International recombinant hemophilia A, hemophilia B Phase I/II (rFVIIa) Deerfield, IL protein baxter.com BAX-855 Baxter International recombinant hemophilia A Phase I (rFVIII) Deerfield, IL protein baxter.com BAY 86-6150 Bayer HealthCare recombinant hemophilia A, hemophilia B Phase II/III (rFVIIa) Pharmaceuticals protein bayerpharma.com Wayne, NJ BAY 94-9027 Bayer HealthCare recombinant hemophilia A Phase II/III (rFVIII) Pharmaceuticals protein bayerpharma.com Wayne, NJ Benlysta® GlaxoSmithKline mAb vasculitis Phase I completed belimumab Rsch. Triangle Park, NC (see also autoimmune, gsk.com (oral) transplantation) beta-globin bluebird bio gene beta-thalassemia, Phase I/II gene therapy Cambridge, MA therapy sickle cell anemia bluebirdbio.com (lentiglobin) INSERM Paris, France BI-655075 Boehringer Ingelheim mAb blood coagulation disorders Phase I Pharmaceuticals boehringer-ingelheim. Ridgefield, CT com CSL-627 CSL Behring recombinant hemophilia A Phase I (rVIII-SC) King of Prussia, PA protein cslbehring.com CSL-654 CSL Behring recombinant hemophilia B Phase II/III (rIX-FP) King of Prussia, PA fusion cslbehring.com (Orphan Drug) protein CSL-689 CSL Behring recombinant hemophilia A, hemophilia B Phase I (rVIIa-FP) King of Prussia, PA fusion cslbehring.com (Orphan Drug) proteinMedicines in Development Biologics 2013 9
  • Biologic Medicines in Development Blood Disorders Product Name Sponsor Product Indication Development Phase Category CYT107 Cytheris recombinant idiopathic CD4-positive Phase I/II (interleukin-7) Rockville, MD interleukin lymphocytopenia cytheris.com (see also cancer, infectious, transplantation) epoetin alfa biosimilar Hospira recombinant anemia Phase III Lake Forest, IL erythropoietin hospira.com erythropoietin gene Medgenics gene therapy anemia Phase II therapy San Francisco, CA medgenics.com factor VIII/ CSL Behring recombinant hemophilia A, Phase III von Willebrand King of Prussia, PA protein von Willebrand disease cslbehring.com factor ferroportin mAb Eli Lilly mAb anemia Phase I Indianapolis, IN lilly.com hepcidin mAb Eli Lilly mAb anemia Phase I Indianapolis, IN lilly.com HM10760A Hanmi Pharmaceutical long-acting anemia Phase I Seoul, South Korea erythropoietin hanmipharm.com human Octapharma USA recombinant hemophilia A Phase II recombinant factor VIII Hoboken, NJ protein octapharma.com IB1001 Inspiration Biopharmaceuticals recombinant hemophilia B application submitted (recombinant factor IX Cambridge, MA protein inspirationbio.com biosimilar) Ipsen Paris, France ISIS-FXIRx Isis Pharmaceuticals antisense clotting disorders Phase II Carlsbad, CA isispharm.com NAV™ therapeutic ReGenX Biosciences gene therapy hemophilia B Phase I/II Washington, DC (see also genetic) regenxbio.com NiCord® Gamida Cell stem cell sickle cell anemia Phase I/II umbilical cord blood Jerusalem, Israel therapy (see also cancer) gamida-cell.com stem cell therapy NN1841 Novo Nordisk recombinant congenital factor XIII deficiency application submitted (rFXIII) Princeton, NJ protein novonordisk.com NN7008 Novo Nordisk recombinant hemophilia A application submitted (turoctocog alfa) Princeton, NJ protein novonordisk.com10 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Blood Disorders Product Name Sponsor Product Indication Development Phase Category NN7088 Novo Nordisk recombinant hemophilia A Phase III (N8-GP, rFVIII Princeton, NJ protein novonordisk.com glycopegylated) NN7999 Novo Nordisk recombinant hemophilia B Phase III (N9-GP, rFIX Princeton, NJ protein novonordisk.com glycopegylated) OBI-1 Inspiration Biopharmaceuticals recombinant hemophilia A Phase III (recombinant Cambridge, MA protein (Fast Track) inspirationbio.com porcine factor VIII) Ipsen (Orphan Drug) Paris, France PF-05280602 Catalyst Biosciences recombinant hemophilia Phase I (rhFVIIa) South San Francisco, CA protein catalystbiosciences.com Pfizer pfizer.com New York, NY PRT4445 Portola Pharmaceuticals recombinant hemorrhage Phase II (FXa inhibitor antidote) South San Francisco, CA protein portola.com recombinant Biogen Idec recombinant hemophilia A Phase III factor VIII-Fc Weston, MA fusion (Fast Track) biogenidec.com (Orphan Drug) protein recombinant Biogen Idec recombinant hemophilia B application submitted factor IX-Fc Weston, MA fusion (Fast Track) biogenidec.com (Orphan Drug) protein ----------------------------------------------- --------------------------------- hemophilia B in children Phase III (Fast Track) biogenidec.com SelG1 Selexys Pharmaceuticals mAb sickle cell anemia Phase I completed Oklahoma City, OK selexys.com sotatercept Acceleron Pharma recombinant anemia in patients with end-stage Phase II (ACE-011) Cambridge, MA fusion renal disease acceleronpharma.com Celgene protein (see also cancer) celgene.com Summit, NJ TT-173 Thrombotargets recombinant blood coagulation disorders Phase I Durham, NC protein thrombotargets.com Wilate® Octapharma USA recombinant surgical blood loss Phase III von Willebrand factor/ Hoboken, NJ protein octapharma.com coagulation factor VIII complex (human)Medicines in Development Biologics 2013 11
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category 8H9 mAb United Therapeutics mAb metastatic brain cancer Phase I Silver Spring, MD unither.com 212-Pb-TCMC- AREVA Med mAb HER2-positive cancer metastasized Phase I trastuzumab Bethesda, MD to the abdominal region arevamed.com AbGn-7 AbGenomics International mAb solid tumors Phase I Los Altos, CA ABIO-0501 Abiogen Pharma cell therapy chronic myeloid leukemia (CML) Phase II (TALL-104) Pisa, Italy abiogen.it ABT-806 AbbVie mAb solid tumors Phase I North Chicago, IL abbvie.com Actimab-A Actinium Pharmaceuticals mAb acute myeloid leukemia (AML) Phase I/II (M195 mAb) New York, NY actiniumpharmaceuti cals.com Adcetris® Seattle Genetics mAb cutaneous T-cell lymphoma, Phase III brentuximab vedotin Bothell, WA front-line Hodgkin lymphoma, seattlegenetics.com (Orphan Drug) post-transplant Hodgkin lymphoma relapse prevention ------------------------------------------------ --------------------------------- non-Hodgkin lymphoma, Phase II non-lymphoma malignancies, seattlegenetics.com CD30-positive hematologic malignancies, CD30-positive T-cell lymphoma AD-IL-12 ZIOPHARM Oncology gene therapy malignant melanoma Phase I/II New York, NY ziopharm.com ADI-PEG-20 Polaris Pharmaceuticals pegylated liver cancer Phase III San Diego, CA protein polarispharma.com ------------------------------------------------ --------------------------------- malignant melanoma, Phase II mesothelioma, small-cell polarispharma.com lung cancer (SCLC) ADXS-HPV Advaxis vaccine cervical cancer, cervical Phase II Princeton, NJ intraepithelial neoplasia advaxis.com AE08 Antigen Express peptide malignant melanoma Phase I Worcester, MA vaccine antigenexpress.com AE37 Antigen Express peptide breast cancer Phase II Worcester, MA vaccine antigenexpress.com12 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category AGS-003 Argos Therapeutics personalized metastatic renal cancer Phase III Durham, NC dendritic cell (Fast Track) argostherapeutics.com vaccine ALD-151 Cytomedix stem cell leukemia Phase I completed Gaithersburg, MD therapy cytomedix.com Duke University Durham, NC AlloStim™ Immunovative Therapies cell vaccine hematological malignancies, Phase I/II cancer vaccine Shoham, Israel solid tumors immunovative.co.il Allovectin® Vical DNA malignant melanoma Phase III velimogene San Diego, CA immunothera- (Fast Track) vical.com aliplasmid peutic (Orphan Drug) ALN-VSP Alnylam Pharmaceuticals RNA liver cancer, liver metastases Phase I Cambridge, MA interference alnylam.com ALT-801 Altor BioScience recombinant AML, bladder cancer, malignant Phase I/II Miramar, FL fusion melanoma, multiple myeloma altorbioscience.com protein ALT-836 Altor BioScience mAb cancer Phase I/II Miramar, FL (see also respiratory) altorbioscience.com Genentech gene.com South San Francisco, CA AME-133v MENTRIK Biotech mAb non-Hodgkin lymphoma Phase II Dallas, TX mentrik.com AMG 386 Amgen recombinant fallopian tube cancer, Phase III Thousand Oaks, CA fusion ovarian cancer, peritoneal cancer amgen.com protein ------------------------------------------------ --------------------------------- breast cancer, colorectal cancer, Phase II gastric cancer, liver cancer, amgen.com esophageal cancer, renal cancer AMG 780 Amgen antibody solid tumors Phase I Thousand Oaks, CA amgen.com AMG 820 Amgen mAb solid tumors Phase I Thousand Oaks, CA amgen.com AMG 888 Amgen mAb non-small-cell lung cancer (NSCLC) Phase II (U3-1287) Thousand Oaks, CA amgen.com U3 Pharma Martinsried, GermanyMedicines in Development Biologics 2013 13
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category antibody-drug conjugate Genentech mAb ovarian cancer, pancreatic cancer Phase I (RG7600) South San Francisco, CA gene.com anti-CD3-anti-HER2/ TransTarget cell metastatic breast cancer Phase II neu-activated T-cells Hillsborough, CA therapy Barbara Ann Karmanos Cancer Institute Detroit, MI anti-CD22 ADC Genentech mAb diffuse large B-cell lymphoma, Phase II (RG7593) South San Francisco, CA non-Hodgkin lymphoma gene.com Seattle Genetics ------------------------------------------------ -------------------------------- Bothell, WA chronic lymphocytic leukemia (CLL) Phase I gene.com anti-CD45 mAb Actinium Pharmaceuticals mAb AML Phase I New York, NY actiniumpharmaceuti cals.com anti-CD79b ADC Genentech mAb diffuse large B-cell lymphoma, Phase II (RG7596) South San Francisco, CA non-Hodgkin lymphoma gene.com Seattle Genetics ------------------------------------------------ --------------------------------- Bothell, WA CLL Phase I gene.com anti-CXCR4 Bristol-Myers Squibb mAb hematological malignancies Phase I Princeton, NJ bms.com anti-EGFL7 mAb Genentech mAb metastatic colorectal cancer, Phase II (RG7414) South San Francisco, CA NSCLC gene.com ------------------------------------------------ -------------------------------- solid tumors Phase I gene.com anti-FGFR3 mAb Genentech mAb solid tumors Phase I (RG7444) South San Francisco, CA gene.com anti-HER3/EGFR Genentech mAb colorectal cancer, Phase II DAF mAb South San Francisco, CA head and neck cancer gene.com (RG7597) anti-PD-L1 Bristol-Myers Squibb mAb cancer Phase I Princeton, NJ (see also infectious) bms.com anti-PD-L1 mAb Genentech mAb melanoma, solid tumors Phase I (RG7446) South San Francisco, CA gene.com anti-STEAP1 ADC Genentech mAb prostate cancer Phase I (RG7450) South San Francisco, CA gene.com Seattle Genetics Bothell, WA14 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category APN301 Apeiron Biologics mAb malignant melanoma, Phase II completed (hu14.18-IL2) Vienna, Austria neuroblastoma in children apeiron-biologics.com Archexin™ Rexahn Pharmaceuticals antisense pancreatic cancer Phase II RX-0201 Rockville, MD rexahn.com (Orphan Drug) Arzerra® GlaxoSmithKline mAb CLL (first-line therapy), Phase III ofatumumab Rsch. Triangle Park, NC diffuse large B-cell lymphoma, gsk.com (Orphan Drug) follicular lymphoma ASC-101 America Stem Cell stem cell myeloablation associated with Phase I/II (Orphan Drug) Carlsbad, CA therapy hematological malignancies americastemcell.com ASG-5ME Agensys mAb-drug pancreatic cancer, Phase I Santa Monica, CA conjugate castration-resistant agensys.com Seattle Genetics prostate cancer seattlegenetics.com Bothell, WA ASG-22ME Agensys mAb-drug solid tumors Phase I Santa Monica, CA conjugate agensys.com Seattle Genetics seattlegenetics.com Bothell, WA autologous stem cell California Stem Cell stem cell malignant melanoma Phase II therapy Irvine, CA therapy californiastemcell.com AV-203 AVEO Oncology mAb solid tumors Phase I Cambridge, MA aveooncology.com Avastin® Genentech mAb ovarian cancer application submitted bevacizumab South San Francisco, CA gene.com (Orphan Drug) Roche ------------------------------------------------ --------------------------------- Nutley, NJ HER-2 negative-breast cancer, Phase III HER-2 positive breast cancer, high- gene.com risk carcinoid tumors, glioblastoma multiforme, metastatic ovarian cancer, NSCLC AVX701 AlphaVax virus replicon CEA-expressing colorectal cancer Phase I/II Rsch. Triangle Park, NC particle alphavax.com vaccine AVX901 AlphaVAx virus replicon HER2-expressing breast cancer Phase I Rsch. Triangle Park, NC particle alphavax.com vaccine B7-2/GM-CSF cancer NuVax Therapeutics gene cancer Phase I gene therapy (Radient Pharmaceuticals) therapy radient-pharma.com (combination Tustin, CA immunogene therapy)Medicines in Development Biologics 2013 15
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category bavituximab Peregrine Pharmaceuticals mAb NSCLC, pancreatic cancer Phase II Tustin, CA peregrineinc.com ------------------------------------------------ -------------------------------- breast cancer, liver cancer, prostate Phase I/II cancer, rectal adenocarcinoma peregrineinc.com BAX-69 Baxter International mAb solid tumors Phase I Deerfield, IL (see also other) baxter.com BAY 79-4620 Bayer HealthCare mAb solid tumors Phase I Pharmaceuticals bayerpharma.com Wayne, NJ BAY 94-9343 Bayer HealthCare mAb solid tumors Phase I Pharmaceuticals bayerpharma.com Wayne, NJ BAY 20-10112 Amgen mAb solid tumors Phase I Thousand Oaks, CA amgen.com Bayer HealthCare bayerpharma.com Pharmaceuticals Wayne, PA BC-819 BioCancell Therapeutics gene bladder cancer, pancreatic cancer Phase II (Orphan Drug) Jerusalem, Israel therapy (Fast Track) biocancell.com ------------------------------------------------ --------------------------------- ovarian cancer Phase I/II biocancell.com BHQ880 Novartis Pharmaceuticals mAb multiple myeloma Phase II East Hanover, NJ novartis.com BI-505 BioInvent International mAb multiple myeloma Phase I (Orphan Drug) Lund, Sweden bioinvent.com BI-836845 Boehringer Ingelheim mAb solid tumors Phase I Pharmaceuticals boehringer-ingelheim. Ridgefield, CT com BiovaxID® Biovest International autologous follicular lymphoma Phase III B-cell lymphoma Tampa, FL idiotype (Fast Track) biovest.com vaccine vaccine ------------------------------------------------ --------------------------------- mantle-cell lymphoma Phase II biovest.com blinatumomab Amgen mAb leukemia and lymphoma Phase II (Orphan Drug) Thousand Oaks, CA amgen.com BP-100-1.01 Bio-Path Holdings antisense acute lymphocytic leukemia (ALL), Phase I (liposomal Grb-2) Houston, TX AML, CML, biopathholdings.com myelodysplastic syndromes16 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category BPX-101 Bellicum Pharmaceuticals dendritic cell prostate cancer Phase I/II Houston, TX vaccine bellicum.com breast cancer vaccine MabVax Therapeutics mAb vaccine breast cancer Phase I San Diego, CA mabvax.com BT-062 Biotest mAb multiple myeloma Phase I/II (indatuximab ravtansine) Dreieich, Germany biotest.de (Orphan Drug) BYM338 Novartis Pharmaceuticals mAb cancer-related cachexia Phase II East Hanover, NJ (see also musculoskeletal) novartis.com CALAA-01 Calando Pharmaceuticals RNA solid tumors Phase I Pasadena, CA interference arrowheadresearch.com calaspargase pegol Sigma-Tau Pharmaceuticals pegylated ALL in adolescents and children Phase III Gaithersburg, MD enzyme sigmatau.com cancer medicine Eli Lilly biologic cancer Phase I Indianapolis, IN lilly.com cancer medicine Eli Lilly biologic cancer Phase I Indianapolis, IN lilly.com cancer vaccine Bayer HealthCare autologous follicular lymphoma Phase I Pharmaceuticals idiotype bayerpharma.com Wayne, NJ vaccine Stellar Biotechnologies Port Hueneme, CA cancer vaccine EMD Millipore dendritic cell malignant melanoma Phase I completed Billerica, MA vaccine millipore.com cancer vaccine Gradalis genetically- solid tumors Phase I Carrollton, TX modified gradalisinc.com Mary Crowley Cancer vaccine marycrowley.org Research Center Dallas, TX cancer vaccine Immunitor vaccine cancer Phase I/II Vancouver, Canada immunitor.com cancer vaccine TapImmune therapeutic breast cancer Phase I (AdhTAP) Seattle, WA vaccine tapimmune.com catumaxomab Fresenius Biotech mAb malignant ascites, Phase II completed North America ovarian cancer fresenius-biotech.com Waltham, MAMedicines in Development Biologics 2013 17
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category CDX-011 Celldex Therapeutics mAb breast cancer (Fast Track), Phase II (glembatumumab vedotin) Needham, MA malignant melanoma celldextherapeutics.com CDX-1127 Celldex Therapeutics mAb hematological malignancies, Phase I Needham, MA solid tumors celldextherapeutics.com CDX-1401 Celldex Therapeutics mAb solid tumors Phase I/II Needham, MA vaccine celldextherapeutics.com cell therapy Cell Medica cell cancer Phase II London, United Kingdom therapy (see also infectious) cellmedica.co.uk Center for Cell and Gene Therapy Houston, TX CEP-37250/KNK-2804 Teva North America mAb adenocarcinoma Phase I North Wales, PA tevapharm.com Kyowa Hakko Kirin Pharma kyowa-kirin-pharma.com Princeton, NJ CG201 CG Therapeutics antibody solid tumors Phase II (anti-hCG vaccine) Seattle, WA vaccine cgtherapeutics.com ch14.18 mAb United Therapeutics mAb neuroblastoma Phase III (Orphan Drug) Silver Spring, MD unither.com cixutumumab Eli Lilly mAb NSCLC Phase II (LY3012217) Indianapolis, IN lilly.com Imclone Systems Princeton, NJ CLT-008 Cellerant Therapeutics cell chemotherapy-induced neutropenia Phase I/II San Carlos, CA therapy (see also transplantation) cellerant.com CNTO-328 Janssen Biotech mAb giant lymph node hyperplasia, Phase II (siltuximab) Horsham, PA multiple myeloma, myelodysplastic janssenbiotech.com syndromes ------------------------------------------------ --------------------------------- prostate cancer Phase I janssenbiotech.com Cotara™ Peregrine Pharmaceuticals mAb recurrent glioblastoma Phase II mAb TNT Tustin, CA (Fast Track) peregrineinc.com (Orphan Drug) CRS-207 Aduro Biotech genetically- pancreatic cancer Phase II Berkeley, CA modified adurobiotech.com vaccine ------------------------------------------------ --------------------------------- mesothelioma Phase I adurobiotech.com18 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category CSF-1R mAb Eli Lilly mAb solid tumors Phase I (IMC-CS4) Indianapolis, IN lilly.com ImClone Systems Bridgewater, NJ CT-011 CureTech mAb AML, colorectal cancer, diffuse Phase II (pidilizumab) Yavne, Israel large B-cell lymphoma, follicular curetechbio.com lymphoma, malignant melanoma CTL019 Novartis cell therapy CLL Phase II East Hanover, NJ novartis.com University of Pennsylvania Philadelphia, PA custirsen OncoGenex Technologies antisense NSCLC, prostate cancer Phase III (OGX-011) Vancouver, Canada (Fast Track) oncogenex.com Teva Pharmaceuticals tevapharm.com Tikva, Israel ------------------------------------------------ --------------------------------- first-time NSCLC Phase II oncogenex.com tevapharm.com CV-301 BN ImmunoTherapeutics cancer metastatic breast cancer Phase II Mountain View, CA vaccine bavarian-nordic.com CVac™ Prima BioMed personalized ovarian cancer Phase II/III cancer vaccine MUC-1 Melbourne, Australia dendritic cell primabiomed.com.au vaccine CYT107 Cytheris recombinant malignant melanoma, renal cancer Phase I (interleukin-7) Rockville, MD interleukin (see also blood, infectious, cytheris.com transplantation) CYT-6091 Cytimmune Sciences tumor solid tumors Phase I (Aurimune™) Rockville, MD necrosis cytimmune.com factor dalotuzumab Merck mAb breast cancer, neuroendocrine Phase II (MK-0646) Whitehouse Station, NJ tumors, NSCLC merck.com daratumumab Janssen Biotech mAb multiple myeloma Phase I/II Horsham, PA janssenbiotech.com DC-IL-12 ZIOPHARM Oncology cell malignant melanoma Phase I New York, NY therapy ziopharm.com DCVax®-L Northwest Biotherapeutics dendritic cell glioblastoma Phase III brain cancer vaccine Bethesda, MD vaccine nwbio.com (Orphan Drug)Medicines in Development Biologics 2013 19
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category DEDN6526A Genentech mAb malignant melanoma Phase I (antibody drug conjugate) South San Francisco, CA gene.com demcizumab GlaxoSmithKline mAb solid tumors Phase I (OMP-21M18) Rsch. Triangle Park, NC gsk.com OncoMed Pharmaceuticals oncomed.com Redwood City, CA DFRF4539A Genentech mAb multiple myeloma Phase I South San Francisco, CA gene.com DI17E6 EMD Serono mAb colorectal cancer, prostate cancer Phase II (anti-integrin mAb) Rockland, MA emdserono.com DKN-01 Dekkun mAb solid tumors Phase I Cambridge, MA DN24-02 Dendreon personalized HER2-positive urogenital cancer Phase II Seattle, WA dendritic cell dendreon.com vaccine DPX-0907 Immunovaccine vaccine breast cancer, ovarian cancer, Phase I Halifax, Canada prostate cancer imvaccine.com DPX-Survivac Immunovaccine peptide ovarian cancer Phase I/II Halifax, Canada vaccine imvaccine.com EC17 Endocyte vaccine renal cancer Phase I West Lafayette, IN (combination therapy) endocyte.com ecromeximab Life Science Pharmaceuticals mAb metastatic melanoma Phase II (KW-2871) Darien, CT investinlife.com EGEN-001 EGEN gene ovarian cancer Phase II (Orphan Drug) Huntsville, AL therapy egeninc.com ------------------------------------------------ --------------------------------- colorectal cancer Phase I/II egeninc.com elotuzumab Bristol-Myers Squibb mAb multiple myeloma Phase III Princeton, NJ bms.com AbbVie abbvie.com North Chicago, IL20 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category enavatuzumab AbbVie mAb solid tumors Phase I completed North Chicago, IL abbvie.com encapsulated cell therapy Metromedia Bio-Science cell colorectal cancer, pancreatic Phase II New York, NY therapy cancer, prostate cancer rogosin.org Rogosin Institute New York, NY encapsulated cell therapy Nuvilex cell pancreatic cancer Phase II Silver Spring, MD therapy nuvilex.com ensituximab Neogenix Oncology mAb colorectal cancer, Phase I (NPC-1C) Great Neck, NY pancreatic cancer neogenix.com entolimod Cleveland BioLabs engineered radiation damage in cancer patients Phase I (CBLB502) Buffalo, NY peptide (see also other) cbiolabs.com epratuzumab Y-90/ Immunomedics mAb non-Hodgkin lymphoma Phase I/II veltuzumab combination Morris Plains, NJ immunomedics.com Erbitux® Bristol-Myers Squibb mAb esophageal cancer Phase III cetuximab Princeton, NJ bms.com Eli Lilly lilly.com Indianapolis, IN ImClone Systems Princeton, NJ ETBX-011 Etubics vector colon cancer Phase II Seattle, WA vaccine etubics.com EZN-2968 Enzon Pharmaceuticals antisense lymphoma, solid tumors Phase I Piscataway, NJ enzon.com EZN-4176 Enzon Pharmaceuticals antisense prostate cancer Phase I Piscataway, NJ enzon.com F-627 Generon recombinant chemotherapy-induced neutropenia Phase II Shanghai, China fusion protein generonbiomed.com Fang™ Vaccine Gradalis autologous solid tumors Phase II autologous tumor cell Carrollton, TX cell vaccine gradalisinc.com vaccineMedicines in Development Biologics 2013 21
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category farletuzumab Eisai mAb platinum-sensitive ovarian cancer Phase III (MORAb-003) Woodcliff Lake, NJ eisai.com (Orphan Drug) ------------------------------------------------ --------------------------------- NSCLC Phase II eisai.com FG-3019 FibroGen mAb pancreatic cancer Phase II San Francisco, CA (see also respiratory, other) fibrogen.com ficlatuzumab AVEO Oncology mAb glioblastoma, lymphoma, Phase I Cambridge, MA multiple myeloma, solid tumors aveooncology.com flanvotumab Eli Lilly mAb malignant melanoma Phase I (TYRP1 protein) Indianapolis, IN lilly.com folate binding protein-E39 Galena Biopharma peptide endometrial cancer, ovarian cancer Phase I/II (FBP) Lake Oswego, OR vaccine galenabiopharma.com FP-1039 Five Prime Therapeutics recombinant solid tumors Phase I San Francisco, CA fusion fiveprime.com protein Fzd7 Bayer HealthCare mAb solid tumors Phase I (vantictumab) Pharmaceuticals bayerpharma.com Wayne, NJ oncomed.com OncoMed Pharmaceuticals Redwood City, CA ganitumab Amgen mAb pancreatic cancer Phase III Thousand Oaks, CA amgen.com ------------------------------------------------ --------------------------------- breast cancer, colorectal cancer, Phase II sarcoma amgen.com GC-33/RG7686 Chugai Pharma USA mAb liver cancer Phase II Berkeley Heights, NJ chugai-pharm.com Roche roche.com Nutley, NJ GI-4000 GlobeImmune genetically- colorectal cancer, NSCLC, Phase II Louisville, CO modified pancreatic cancer globeimmune.com vaccine GI-6207 GlobeImmune genetically- medullary thyroid cancer Phase II Louisville, CO modified globeimmune.com vaccine GI-6301 GlobeImmune genetically- solid tumors Phase I (brachyury peptide Louisville, CO modified globeimmune.com vaccine) vaccine22 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category GL-0817 Gliknik peptide head and neck cancer, Phase II (MAGE-A3 Baltimore, MD vaccine multiple myeloma gliknik.com immunomodulator) (Orphan Drug) GliAtak™ Advantagene gene malignant glioma Phase II cancer gene therapy Auburndale, MA therapy advantagene.com (Orphan Drug) Glionix™ NovaRx antisense glioma Phase I completed brain vaccine San Diego, CA gene- novarx.com modified cell vaccine GS-6624 Gilead Sciences mAb colorectal cancer, Phase II (simtuzumab) Foster City, CA pancreatic cancer gilead.com (see also respiratory, other) GVAX® Breast BioSante Pharmaceuticals genetically- breast cancer Phase I cancer immunotherapy Lincolnshire, IL modified biosantepharma.com vaccine GVAX® Colorectal BioSante Pharmaceuticals genetically- colorectal cancer Phase I cancer immunotherapy Lincolnshire, IL modified biosantepharma.com vaccine GVAX® Leukemia BioSante Pharmaceuticals genetically- AML, CML Phase II cancer immunotherapy Lincolnshire, IL modified biosantepharma.com (Orphan Drug) vaccine GVAX® Multiple BioSante Pharmaceuticals genetically- multiple myeloma Phase II Myeloma Lincolnshire, IL modified biosantepharma.com cancer vaccine immunotherapeutic GVAX® Pancreas AduroBiotech genetically- pancreatic cancer Phase II cancer immunotherapy Berkeley, CA modified adurobiotech.com (Orphan Drug) BioSante Pharmaceuticals vaccine biosantepharma.com Lincolnshire, IL GVAX® Prostate AduroBiotech genetically- prostate cancer Phase III cancer immunotherapy Berkeley, CA modified (Fast Track) adurobiotech.com BioSante Pharmaceuticals vaccine biosantepharma.com Lincolnshire, IL HCD122 Novartis Pharmaceuticals mAb lymphoma Phase I/II (anti-CD40 mAb) East Hanover, NJ novartis.com XOMA xoma.com Berkeley, CA HGS-ETR1 GlaxoSmithKline mAb liver cancer, multiple myeloma, Phase II (mapatumumab) Rsch. Triangle Park, NC NSCLC gsk.comMedicines in Development Biologics 2013 23
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category HS-110 Heat Biologics genetically- NSCLC Phase II Chapel Hill, NC modified heatbio.com vaccine hTERT cancer Merck DNA vaccine cancer Phase I vaccine Whitehouse Station, NJ merck.com Vical vical.com San Diego, CA HuL2G7 Galaxy Biotech mAb solid tumors Phase I Sunnyvale, CA galaxybiotech.com HuM195/rGel Targa Therapeutics mAb AML, CML, myelodysplastic Phase I San Diego, CA syndromes targatherapeutics.com huN901-DM1 ImmunoGen mAb SCLC Phase II (lorvotuzumab Cambridge, MA immunogen.com mertansine) ----------------------------------------------- --------------------------------- (Orphan Drug) multiple myeloma, solid tumors Phase I immunogen.com HyperAcute® Lung NewLink Genetics DNA NSCLC Phase II/III turgenpumatucel-L Ames, IA vaccine linkp.com HyperAcute® Melanoma NewLink Genetics DNA malignant melanoma Phase II melanoma cancer vaccine Ames, IA vaccine linkp.com HyperAcute® Pancreas NewLink Genetics DNA pancreatic cancer Phase III algenpantucel-L Ames, IA vaccine (Fast Track) linkp.com (Orphan Drug) icrucumab Eli Lilly mAb bladder cancer, breast cancer, Phase II (LY3012212) Indianapolis, IN colorectal cancer lilly.com ImClone Systems Bridgewater, NJ ICT-107 ImmunoCellular Therapeutics personalized glioblastoma Phase II (Orphan Drug) Woodland Hills, CA dendritic cell imuc.com vaccine IL-21 Bristol-Myers Squibb interleukin solid tumors Phase I (BMS-982470) Princeton, NJ bms.com (Orphan Drug) IMA901 immatics biotechnologies peptide renal cancer treatment Phase III (intradermal vaccine) Tübingen, Germany vaccine immatics.com (Orphan Drug)24 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category IMA950 immatics biotechnologies peptide gliobastoma Phase I (intradermal vaccine) Tübingen, Germany vaccine immatics.com IMC-gp100 Immunocore recombinant malignant melanoma treatment Phase 0 Oxon, United Kingdom vaccine immunocore.com IMC-TR1 Eli Lilly mAb solid tumors Phase I (LY3022859) Indianapolis, IN lilly.com ImClone Systems Bridgewater, NJ IMF-001 ImmunoFrontier recombinant solid tumors Phase I Tokyo, Japan vaccine immunofrontier.com IMGN529 ImmunoGen mAb non-Hodgkin lymphoma Phase I Waltham, MA immunogen.com IMGN853 ImmunoGen mAb solid tumors Phase I Waltham, MA immunogen.com IMMU-102 Immunomedics mAb non-Hodgkin lymphoma Phase I/II (epratuzumab Y-90) Morris Plains, NJ immunomedics.com IMT-1012 Immunotope peptide breast cancer, ovarian cancer Phase I Doylestown, PA immunothera- immunotope.com peutic vaccine inotuzumab Pfizer mAb aggressive non-Hodgkin Phase III ozogamicin New York, NY lymphoma, ALL pfizer.com (CMC-544) UCB ucb.com Smyrna, GA interleukin-12 OncoSec Medical gene therapy cutaneous T-cell lymphoma, Phase II gene therapy San Diego, CA malignant melanoma, merkel cell oncosec.com carcinoma Iomab-B Actinium Pharmaceuticals mAb AML Phase II (anti-CD45 mAb) New York, NY actiniumpharmaceuti cals.com IRX-2 IRX Therapeutics cytokine head and neck cancer Phase II New York, NY (Fast Track) irxtherapeutics.com ISF35 Memgen gene CLL, non-Hodgkin lymphoma Phase II San Diego, CA vaccine memgenbio.com ISIS-EIF4ERx Isis Pharmaceuticals antisense NSCLC, pancreatic cancer Phase II (EIF-4E ASO) Carlsbad, CA isispharm.comMedicines in Development Biologics 2013 25
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category ISIS-STAT3Rx AstraZeneca antisense cancer Phase II Wilmington, DE astrazeneca.com Isis Pharmaceuticals isispharm.com Carlsbad, CA J 591 Lu-177 BZL Biologics mAb prostate cancer Phase II New York, NY JVRS-100 Colby Pharmaceuticals DNA leukemia Phase I immunotherapeutic San Jose, CA vaccine colbypharma.com JX-594 Jennerex Biotherapeutics genetically- liver cancer Phase II San Francisco, CA engineered jennerex.com virus ------------------------------------------------ --------------------------------- colorectal cancer Phase I jennerex.com JX-929 Jennerex Biotherapeutics genetically- breast cancer, pancreatic cancer Phase I San Francisco, CA engineered jennerex.com virus KB004 KaloBios Pharmaceuticals mAb hematological malignancies Phase I South San Francisco, CA kalobios.com LA-EP2006 Sandoz growth factor chemotherapy-related Phase III (pegfilgrastim biosimilar) Princeton, NJ febrile neutropenia us.sandoz.com leukemia DNA vaccine Inovio Pharmaceuticals DNA AML, CML Phase II (WT1) Blue Bell, PA vaccine inovio.com University of Southampton Hampshire, United Kingdom Leukine® Bayer HealthCare granulocyte CLL Phase II sargramostim Pharmaceuticals macrophage bayerpharma.com Wayne, NJ colony- stimulating factor LFA102 Novartis Pharmaceuticals mAb breast cancer, prostate cancer Phase I East Hanover, NJ novartis.com XOMA Berkeley, CA LG631 Lentigen stem cell chemoprotection in patients with Phase I Gaithersburg, MD therapy glioblastoma lentigen.com lirilumab Bristol-Myers Squibb mAb cancer Phase I (anti-KIR) Princeton, NJ bms.com26 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category LOR-2040 Lorus Therapeutics antisense AML Phase II (Orphan Drug) Toronto, Canada lorusthera.com Lucanix® NovaRx antisense NSCLC Phase III belagen pumatucel-L San Diego, CA gene-modified (Fast Track) novarx.com cell vaccine LY2495655 Eli Lilly mAb cancer cachexia Phase II (anti-myostatin mAb) Indianapolis, IN (see also musculoskeletal) lilly.com LY2875358 Eli Lilly mAb cancer Phase II (c-met mAb) Indianapolis, IN lilly.com MABp1 XBiotech mAb cancer-related cachexia Phase III Austin, TX (see also cardiovascular, xbiotech.com diabetes, skin) ------------------------------------------------ --------------------------------- advanced cancer Phase I/II xbiotech.com ------------------------------------------------ --------------------------------- leukemia Phase I xbiotech.com MAGE-A3 GlaxoSmithKline recombinant bladder cancer, melanoma, Phase III (antigen-specific cancer Rsch. Triangle Park, NC immuno- NSCLC gsk.com immunotherapeutic) therapeutic vaccine MEDI-551 AstraZeneca mAb hematological malignancies Phase II (anti-CD19 mAb) Wilmington, DE (see also autoimmune) astrazeneca.com MedImmune medimmune.com Gaithersburg, MD MEDI-565 Amgen bispecific solid tumors Phase I (anti-CEA BiTE) Thousand Oaks, CA antibody amgen.com AstraZeneca astrazeneca.com Wilmington, DE medimmune.com MedImmune Gaithersburg, MD MEDI-573 AstraZeneca mAb solid tumors Phase II (anti-IGF mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MD MEDI-575 AstraZeneca mAb glioblastoma, NSCLC Phase II (anti-PDGFRα mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MDMedicines in Development Biologics 2013 27
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category MEDI-0639 AstraZeneca mAb solid tumors Phase I (anti-DLL-4 mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MD MEDI-3617 AstraZeneca mAb solid tumors Phase I (anti-ANG-2 mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MD MEDI-4736 AstraZeneca mAb cancer Phase I (anti-CD274 mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MD MEDI-6469 AgonOx mAb solid tumors Phase I (anti-OX40 mAb) Portland, OR agonox.com AstraZeneca astrazeneca.com Wilmington, DE medimmune.com MedImmune Gaithersburg, MD melanoma vaccine MabVax Therapeutics mAb vaccine malignant melanoma Phase I San Diego, CA mabvax.com melanoma vaccine Polynoma vaccine malignant melanoma Phase III (POL-103A) San Diego, CA polynoma.com MGA271 MacroGenics mAb solid tumors Phase I (anti-B7-H3) Rockville, MD macrogenics.com MGAH22 MacroGenics mAb solid tumors Phase I (anti-HER2) Rockville, MD macrogenics.com milatuzumab Immunomedics mAb CLL Phase I (Orphan Drug) Morris Plains, NJ immunomedics.com milatuzumab-DOX Immunomedics mAb multiple myeloma Phase I Morris Plains, NJ immunomedics.com MINT1526A Genentech mAB solid tumors Phase I South San Francisco, CA gene.com MK-3475 Merck mAb malignant melanoma Phase II Whitehouse Station, NJ merck.com ------------------------------------------------ ------------------------------- NSCLC Phase I merck.com28 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category MKC1106-MT Colby Pharmaceuticals vaccine malignant melanoma Phase II San Jose, CA colbypharma.com MLN0264 Millennium Pharmaceuticals mAb gastrointestinal cancer Phase I (GCC antibody drug Cambridge, MA mlnm.com conjugate) MM-111 Merrimack Pharmaceuticals recombinant breast cancer, solid tumors Phase I Cambridge, MA fusion merrimackpharma.com protein mogamulizimab Kyowa Hakko Kirin Pharma mAb cutaneous T-cell lymphoma Phase III (Orphan Drug) Princeton, NJ kyowa-kirin-pharma.com ------------------------------------------------ --------------------------------- adult T-cell lymphoma, Phase II T-cell leukemia kyowa-kirin-pharma.com MORAb-004 Eisai mAb colorectal cancer, melanoma, Phase II Woodcliff Lake, NJ sarcoma eisai.com MORAb-009 Eisai mAb mesothelioma Phase II (amatuximab) Woodcliff Lake, NJ eisai.com (Orphan Drug) moxetumomab AstraZeneca mAb hematological malignancies Phase I pasudotox Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MD Multikine® CEL-SCI growth factor, head and neck cancer Phase III leukocyte Vienna, VA interferon, cel-sci.com interleukin cancer interleukin (Orphan Drug) MVA-BN® HER2 BN ImmunoTherapeutics cell vaccine breast cancer Phase I Mountain View, CA bavarian-nordic.com MVA-BN®-PRO BN ImmunoTherapeutics vaccine prostate cancer Phase I/II Mountain View, CA bavarian-nordic.com mycobacterium Bioniche Therapeutics myco- bladder cancer Phase III cell wall-DNA Pointe-Claire, Canada bacterial (Fast Track) bioniche.com complex (MCC) DNA necitumumab Bristol-Myers Squibb mAb NSCLC Phase III Princeton, NJ bms.com Eli Lilly lilly.com Indianapolis, IN ImClone Systems Princeton, NJMedicines in Development Biologics 2013 29
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category neuroblastoma vaccine MabVax Therapeutics mAb vaccine neuroblastoma Phase I San Diego, CA mabvax.com NeuVax™ Galena Biopharma peptide breast cancer Phase III nelipepmut-S Lake Oswego, OR vaccine galenabiopharma.com ------------------------------------------------ --------------------------------- prostate cancer Phase I/II galenabiopharma.com NGR-TNF Molmed recombinant mesothelioma Phase III (Orphan Drug) Milan, Italy fusion molmed.com protein NHS-IL 12 EMD Serono interleukin solid tumors Phase I Rockland, MA emdserono.com National Cancer Institute Bethesda, MD NiCord® Gamida Cell stem cell hematological malignancies Phase I/II umbilical cord blood Jerusalem, Israel therapy (see also blood) gamida-cell.com stem cell therapy nimotuzumab InnoMab PTE mAb glioma Phase II (Orphan Drug) Singapore innomab.net nivolumab Bristol-Myers Squibb mAb melanoma, NSCLC, renal cell Phase III (anti-PD1) Princeton, NJ carcinoma bms.com ----------------------------------------------- --------------------------------- solid tumors Phase I/II bms.com Nplate™ Amgen recombinant chemotherapy-induced Phase II romiplostim Thousand Oaks, CA fusion thrombocytopenia amgen.com protein (see also autoimmune) NTDDS Periphagen Holdings gene cancer pain Phase II completed (intradermal Pittsburgh, PA therapy gene therapy) NY-ESO-1 antigen- GlaxoSmithKline recombinant malignant melanoma Phase I specific cancer Rsch. Triangle Park, NC vaccine gsk.com immunotherapeutic obinutuzumab Biogen Idec mAb CLL, diffuse large B-cell lymphoma, Phase III (GA101) Weston, MA non-Hodgkin lymphoma biogenidec.com Genentech gene.com South San Francisco, CA OHR/AVR118 Ohr Pharmaceutical immune- cachexia associated with cancer Phase II New York, NY based ohrpharmaceutical.com therapy30 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category olaratumab Eli Lilly mAb glioblastoma Phase II (LY3012207) Indianapolis, IN lilly.com ImClone Systems Princeton, NJ OMP-52M51 GlaxoSmithKline mAb hematological malignancies Phase I (anti-Notch 1) Rsch. Triangle Park, NC gsk.com OncoMed Pharmaceuticals oncomed.com Redwood City, CA OMP-59R5 GlaxoSmithKline mAb pancreatic cancer Phase I/II (anti-Notch 2/3) Rsch. Triangle Park, NC gsk.com OncoMed Pharmaceuticals oncomed.com Redwood City, CA onartuzumab Genentech mAb metastatic NSCLC Phase III (anti-c-met- mAb) South San Francisco, CA gene.com Onconase® Tamir Biotechnology ribonuclease NSCLC Phase II ranpirnase Monmouth Junction, NJ tamirbio.com Oncoprex™ Genprex gene therapy NSCLC Phase I TUSC2 nanoparticles Austin, TX genprex.com OncoVAX® Vaccinogen cell vaccine colorectal cancer Phase I/II colorectal cancer Frederick, MD (Fast Track) vaccinogeninc.com vaccine ONT-10 Oncothyreon vaccine solid tumors Phase I Seattle, WA oncothyreon.com Ontak® Eisai recombinant malignant melanoma Phase II denileukin diftitox Woodcliff Lake, NJ fusion protein eisai.com OPT-822/OPT-821 Optimer Biotechnology vaccine breast cancer Phase III Taipei, Taiwan optimerbiotech.com oregovomab Quest Pharmatech mAb ovarian cancer Phase II (Orphan Drug) Edmonton, Canada questpharmatech.com ovarian cancer vaccine MabVax Therapeutics mAb vaccine ovarian cancer Phase II San Diego, CA mabvax.com PancAtak™ Advantagene gene therapy pancreatic cancer Phase I/II cancer gene therapy Auburndale, MA advantagene.com pbi-shRNA™ STMN1 LP Gradalis gene therapy solid tumors Phase I Carrollton, TX gradalisinc.comMedicines in Development Biologics 2013 31
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category PEGPH20 Halozyme Therapeutics recombinant solid tumors Phase II San Diego, CA enzyme halozyme.com Perjeta™ Genentech mAb early HER2-positive breast cancer Phase III pertuzumab South San Francisco, CA ----------------------------------------------- gene.com HER2-positive metastatic breast --------------------------------- cancer (second-line treatment), Phase II HER2-positive gastric cancer gene.com PF-03446962 Pfizer mAb solid tumors Phase I New York, NY pfizer.com PF-04605412 Pfizer mAb solid tumors Phase I New York, NY pfizer.com PF-04856884 Pfizer fusion renal cancer Phase II (CVX060) New York, NY peptide pfizer.com ------------------------------------------------ -------------------------------- solid tumors Phase I pfizer.com PF-05082566 Pfizer mAb cancer, lymphoma Phase I New York, NY pfizer.com PF-05280014 Pfizer mAb metastatic breast cancer Phase I (trastuzumab biosimilar) New York, NY pfizer.com polyclonal antibody Cancer Advances polyclonal gastric cancer, pancreatic cancer Phase III stimulator Durham, NC antibody (see also digestive) canceradvances.com (Orphan Drug) vaccine ------------------------------------------------ -------------------------------- colorectal cancer Phase II canceradvances.com PRAME antigen-specific GlaxoSmithKline recombinant malignant melanoma, NSCLC Phase I cancer Rsch. Triangle Park, NC vaccine gsk.com immunotherapeutic prophage Agenus vaccine glioma Phase II cancer vaccine (G-series) Lexington, MA agenusbio.com prophage Agenus vaccine malignant melanoma Phase III cancer vaccine (M-series) Lexington, MA (Fast Track) agenusbio.com (Orphan Drug) prophage Agenus vaccine renal cancer Phase III cancer vaccine (R-series) Lexington, MA (Fast Track) agenusbio.com32 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category ProstAtak™ Advantagene gene therapy prostate cancer Phase III cancer gene therapy Auburndale, MA (Fast Track) advantagene.com Prostvac® BN ImmunoTherapeutics recombinant prostate cancer Phase III recombinant vaccinia Mountain View, CA vaccine bavarian-nordic.com virus-expressing PSA Provenge® Dendreon dendritic cell prostate cancer Phase III sipuleucel-T Seattle, WA vaccine (early-stage disease, recurrent) dendreon.com ------------------------------------------------ --------------------------------- prostate cancer (early-stage Phase II disease, neoadjuvant dendreon.com therapy), prostate cancer (metastatic, first-line therapy) PSMA-ADC Progenics Pharmaceuticals mAb prostate cancer Phase II Tarrytown, NY progenics.com PT107 Pique Therapeutics vaccine NSCLC Phase II Durham, NC piquetherapeutics.com purine nucleoside PNP Therapeutics gene solid tumors Phase I phosphorylase Birmingham, AL therapy pnptherapeutics.com gene therapy PVX-410 OncoPep peptide multiple myeloma Phase I/II immunotherapeutic Methuen, MA vaccine oncopep.com QBI-139 Quintessence Biosciences ribonuclease solid tumors Phase I Madison, WI quintbio.com ramucirumab Eli Lilly mAb breast cancer, colorectal cancer, Phase III (LY3009806) Indianapolis, IN gastric cancer lilly.com ImClone Systems Princeton, NJ Redectane® Wilex mAb diagnosis of kidney cancer Phase III girentuximab I-124 Munich, Germany wilex.com REGN1400 Regeneron Pharmaceuticals mAb cancer Phase I Tarrytown, NY regeneron.com REIC gene therapy Momotaro-Gene gene prostate cancer Phase I Okayama, Japan therapy mt-gene.com RG7116 Roche mAb solid tumors Phase I Nutley, NJ roche.com RG7155 Roche mAb solid tumors Phase I Nutley, NJ roche.comMedicines in Development Biologics 2013 33
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category RG7160 Roche mAb colorectal cancer Phase II (humAb EGFR) Nutley, NJ roche.com RG7212 Roche mAb solid tumors Phase I Nutley, NJ roche.com RG7356 Roche mAb AML, solid tumors Phase I (anti-CD44 mAb) Nutley, NJ roche.com RG7458 Genentech mAb ovarian cancer Phase I (antibody-drug conjugate) South San Francisco, CA gene.com Seattle Genetics Bothell, WA RIGScan™ Navidea Biopharmaceuticals mAb diagnosis of colorectal in clinical trials radiodiagnostic agent Dublin, OH cancer navidea.com rilotumumab Amgen mAb colorectal cancer, gastric cancer, Phase II Thousand Oaks, CA prostate cancer, SCLC amgen.com rindopepimut Celldex Therapeutics vaccine first-line glioblastoma Phase III (Orphan Drug) Needham, MA (Fast Track) celldextherapeutics.com ------------------------------------------------ --------------------------------- second-line glioblastoma Phase II (Fast Track) celldextherapeutics.com Rituxan® Genentech mAb diffuse large B-cell lymphoma Phase III rituximab South San Francisco, CA gene.com RON8 mAb Eli Lilly mAb cancer Phase I Indianapolis, IN lilly.com ImClone Systems Princeton, NJ SAR3419 Sanofi US mAb ALL, non-Hodgkin lymphoma Phase II (maytansin-loaded Bridgewater, NJ sanofi.com anti-CD19 mAb) SAR153192 Regeneron Pharmaceuticals mAb cancer Phase I (REGN 421) Tarrytown, NY regeneron.com (anti-DLL4 mAb) Sanofi US sanofi.com Bridgewater, NJ SAR256212 Merrimack Pharmaceuticals mAb breast cancer Phase II (MM-121) Cambridge, MA merrimackpharma.com (anti-ErbB3 mAb) Sanofi US sanofi.com Bridgewater, NJ ------------------------------------------------ --------------------------------- solid tumors Phase I (combination therapy) merrimackpharma.com sanofi.com34 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category SAR307746 Regeneron Pharmaceuticals mAb solid tumors Phase I (REGN910) Tarrytown, NY regeneron.com (anti-angiopoietin-2 mAb) Sanofi US sanofi.com Bridgewater, NJ SAR566658 Sanofi US mAb DS6-positive solid tumors Phase I (maytansin-loaded Bridgewater, NJ sanofi.com anti-DS6) SAR650984 Sanofi US mAb hematological malignancies Phase I (anti-CD38 Bridgewater, NJ sanofi.com naked mAb) sarcoma MabVax Therapeutics mAb sarcoma Phase II cancer vaccine San Diego, CA vaccine mabvax.com SB-313 Sangamo BioSciences cell glioblastoma Phase I Richmond, CA therapy sangamo.com SCH-721015 FKD Therapies gene therapy bladder cancer Phase II Kuopio, Finland SGN-75 Seattle Genetics mAb non-Hodgkin lymphoma, Phase I (vorsetuzumab mafodotin) Bothell, WA renal cancer seattlegenetics.com SL-401 Stemline Therapeutics recombinant AML, myleodysplastic Phase I/II (Orphan Drug) New York, NY fusion syndromes stemline.com protein SL-701 Stemline Therapeutics dendritic cell glioma Phase I/II New York, NY vaccine stemline.com small-cell lung cancer MabVax Therapeutics mAb vaccine SCLC Phase I vaccine San Diego, CA mabvax.com SNS01-T Senesco Technologies gene diffuse large B-cell lymphoma, Phase I/II Bridgewater, NJ therapy; RNA mantle cell lymphoma, senesco.com interference multiple myeloma sotatercept Acceleron Pharma recombinant chemotherapy-induced Phase II/III (ACE-011) Cambridge, MA fusion anemia acceleronpharma.com Celgene protein (see also blood) celgene.com Summit, NJMedicines in Development Biologics 2013 35
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category SPI-2012 Hanmi Pharmaceutical growth neutropenia in cancer patients Phase II (granulocyte colony- Seoul, South Korea factor sppirx.com stimulating factor, Spectrum Pharmaceuticals long-acting) Henderson, NV StemEx® Gamida Cell stem cell hematological malignancies Phase III carlecortemcel-L Jerusalem, Israel therapy (Fast Track) gamida-cell.com (Orphan Drug) Teva Pharmaceutical tevapharm.com Tikva, Israel Stimuvax® EMD Serono peptide NSCLC Phase III lioposomal Rockland, MA vaccine (Fast Track) emdserono.com cancer vaccine Oncothyreon oncothyreon.com Seattle, WA Sym004 EMD Serono mAb head and neck cancer Phase II Rockland, MA emdserono.com Symphogen symphogen.com Lyngby, Denmark ------------------------------------------------ --------------------------------- solid tumors Phase I emdserono.com symphogen.com tabalumab Eli Lilly mAb multiple myeloma Phase III (BAFF inhibitor) Indianapolis, IN (see also autoimmune) lilly.com talactoferrin alfa Agennix recombinant NSCLC Phase III completed (Orphan Drug) Princeton, NJ protein (Fast Track) agennix.com talminogene Amgen gene therapy malignant melanoma Phase III laherparepvec Thousand Oaks, CA amgen.com TeloB-Vax Adamis Pharmaceuticals DNA prostate cancer Phase I telomerase San Diego, CA vaccine adamispharmaceuticals. cancer vaccine com TF2 Immunomedics mAb diagnosis of colorectal cancer Phase I Morris Plains, NJ immunomedics.com TG-1101 TG Therapeutics mAb CLL, non-Hodgkin lymphoma Phase I/II (ublituximab) New York, NY tgtherapeutics.com (Orphan Drug) tigatuzumab Daiichi Sankyo mAb breast cancer, liver cancer, Phase II Parsippany, NJ ovarian cancer, pancreatic cancer dsi.com TKM-PLK1 Tekmira Pharmaceuticals RNA lymphoma, solid tumors Phase I Burnaby, Canada interference tekmirapharm.com36 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category Toca-511 Tocagen gene therapy glioblastoma Phase I/II (cytosine deaminase San Diego, CA tocagen.com gene therapy) trastuzumab Genentech mAb first-line HER2-positive metastatic Phase III emtansine South San Francisco, CA breast cancer, third-line HER2-posi- gene.com (T-DM1) Roche tive metastatic breast cancer roche.com Nutley, NJ ------------------------------------------------ --------------------------------- early HER2-positive breast Phase II cancer, advanced HER2-positive gene.com gastric cancer roche.com TRC105 TRACON Pharmaceuticals mAb bladder cancer, liver cancer, Phase II San Diego, CA ovarian cancer, prostate cancer traconpharma.com ------------------------------------------------ --------------------------------- solid tumors Phase I traconpharma.com tremelimumab AstraZeneca mAb solid tumors Phase II (anti-CTLA4 mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MD Trivax Activartis Biotech personalized glioblastoma Phase II cancer vaccine Vienna, Austria dendritic cell activartis.com vaccine tumor immunotherapy Genentech mAb solid tumors Phase I mAb South San Francisco, CA gene.com TV1-Brain-1 TVAX Biomedical cell vaccine glioblastoma Phase II Lenexa, KS tvaxbiomedical.com TV1-Kidney-1 TVAX Biomedical cell vaccine renal cancer Phase II Lenexa, KS tvaxbiomedical.com U3-1565 Daiichi Sankyo mAb solid tumors Phase I Parsippany, NJ dsi.com urelumab Bristol-Myers Squibb mAb cancer Phase I (anti-CD137) Princeton, NJ bms.comMedicines in Development Biologics 2013 37
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category V503 Merck vaccine prevention of cervical cancer, Phase III Whitehouse Station, NJ prevention of vulvovaginal cancer merck.com (see also infectious) VAY736 MorphoSys mAb CLL Phase I Munich, Germany (see also other) morphosys.com Novartis Pharmaceuticals novartis.com East Hanover, NJ VB-111 VBL Therapeutics gene thyroid cancer Phase II Or Yehuda, Israel therapy vblrx.com ------------------------------------------------ --------------------------------- glioblastoma Phase I/II vblrx.com VB4-845 Viventia Biotechnologies mAb bladder cancer Phase II (Orphan Drug) Mississauga, Canada viventia.com Vectibix® Amgen mAb colorectal cancer (first-line therapy, application submitted panitumumab Thousand Oaks, CA second-line therapy) amgen.com VEGFR3 mAb Eli Lilly mAb cancer Phase I (IMC-3C5) Indianapolis, IN lilly.com ImClone Systems Bridgewater, NJ veltuzumab Immunomedics mAb CLL, non-Hodgkin lymphoma Phase I/II (IMMU-106) Morris Plains, NJ (see also autoimmune) immunomedics.com (Orphan Drug) VGX-100 Circadian Technologies mAb solid tumors Phase I Victoria, Australia circadian.com.au VGX-3100 Inovio Pharmaceuticals DNA cervical dysplasia Phase II Blue Bell, PA vaccine inovio.com volociximab AbbVie mAb NSCLC Phase I completed North Chicago, IL abbvie.com VX15 Vaccinex mAb solid tumors Phase I Rochester, NY (see also autoimmune) vaccinex.com WT1 antigen- GlaxoSmithKline recombinant AML Phase II specific cancer Rsch. Triangle Park, NC vaccine gsk.com immunotherapeutic ------------------------------------------------ --------------------------------- breast cancer Phase I gsk.com38 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Cancer and Related Conditions Product Name Sponsor Product Indication Development Phase Category WT2725 Sunovion Pharmaceuticals peptide solid tumors Phase I Marlborough, MA vaccine sunovion.com Xgeva® Amgen mAb delay or prevention of bone application submitted denosumab Thousand Oaks, CA metastases in prostate cancer amgen.com ------------------------------------------------ --------------------------------- delay or prevention of bone metases Phase III in breast cancer amgen.com ------------------------------------------------ --------------------------------- giant cell tumor of the bone Phase II amgen.com Xiaflex® BioSpecifics Technologies peptide lipoma Phase II collagenase clostridium Lynbrook, NY (see also musculoskeletal, biospecifics.com histolyticum other) XmAb® Boehringer Ingelheim mAb cancer Phase I high ADCC mAb Pharmaceuticals boehringer-ingelheim. Ridgefield, CT com Xencor xencor.com Monrovia, CA XmAb® 2513 Xencor mAb Hodgkin disease, Phase I anti-CD30 mAb Monrovia, CA T-cell lymphoma xencor.com XmAb®5574 MorphoSys mAb CLL Phase I anti-CD19 mAb Munich, Germany morphosys.com Xencor xencor.com Monrovia, CA Y-90 hPAM 4 Immunomedics mAb pancreatic cancer Phase I/II (IMMU-107) Morris Plains, NJ (Fast Track) immunomedics.com (Orphan Drug) Yervoy™ Bristol-Myers Squibb mAb adjuvant melanoma, NSCLC, Phase III ipilimumab Princeton, NJ prostate cancer, SCLC bms.com (Orphan Drug) ------------------------------------------------ --------------------------------- gastric cancer, ovarian cancer Phase II bms.com zanolimumab Emergent BioSolutions mAb peripheral T-cell lymphoma Phase II Rockville, MD emergentbiosolutions. com Zevalin® Spectrum Pharmaceuticals mAb diffuse large B-cell lymphoma Phase III ibritumomab tiuxetan Henderson, NV sppirx.com ------------------------------------------------ --------------------------------- non-Hodgkin lymphoma Phase II sppirx.comMedicines in Development Biologics 2013 39
  • Biologic Medicines in Development Cardiovascular Disease Product Name Sponsor Product Indication Development Phase Category 3K3A-APC ZZ Biotech recombinant stroke Phase I Houston, TX protein zzbiotech.com ACP-501 AlphaCore Pharma recombinant atherosclerosis, Phase I (rhLCAT) Ann Arbor, MI protein coronary artery disease alphacorepharma.com ACY001 Arteriocyte stem cell chronic coronary ischemia Phase I completed Cleveland, OH therapy arteriocyte.com adipose stem cell therapy Cytori Therapeutics stem cell myocardial ischemia Phase I/II San Diego, CA therapy cytori.com adult autologous stem cell Baxter International stem cell myocardial ischemia Phase III therapy Deerfield, IL therapy baxter.com ------------------------------------------------ --------------------------------- critical limb ischemia Phase I/II completed baxter.com adult mesenchymal stem BioCardia stem cell ischemic heart disorders Phase I/II cell therapy San Carlos, CA therapy biocardia.com ALD-201 Cytomedix stem cell ischemic heart failure Phase I completed Gaithersburg, MD therapy cytomedix.com ALD-301 Cytomedix stem cell critical limb ischemia Phase I/II Gaithersburg, MD therapy cytomedix.com ALD-401 Cytomedix stem cell ischemic stroke Phase II Gaithersburg, MD therapy cytomedix.com allogeneic stem Stemedica Cell Technologies stem cell ischemic stroke Phase I/II cell therapy San Diego, CA therapy stemedica.com ALN-PCS Alnylam Pharmaceuticals RNA hypercholesterolemia Phase I Cambridge, MA interference alnylam.com ALO-212 Arteriocyte stem cell critical limb ischemia Phase I Cleveland, OH therapy arteriocyte.com AMG 145 Amgen mAb hypercholesterolemia Phase III Thousand Oaks, CA amgen.com ------------------------------------------------ --------------------------------- hyperlipoproteinemia type IIa Phase II/III amgen.com AMR-001 Amorcyte stem cell myocardial infarction Phase II Allendale, NJ therapy amorcyte.com NeoStem neostem.com New York, NY40 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Cardiovascular Disease Product Name Sponsor Product Indication Development Phase Category Anginera™ Theregen fibroblast heart failure, ischemic Phase I epicardial angiogenesis San Francisco, CA cell heart disorders theragen.com patch therapy anti-fibrin mAb Agenix mAb diagnosis of deep vein Phase II 3B6/22 Tc-99m Sydney, Australia thrombosis, diagnosis of agenix.com pulmonary embolism anti-oxLDL BioInvent International mAb atherosclerosis Phase II (BI-204/RG7418) Lund, Sweden gene.com Genentech South San Francisco, CA anti-PCSK9 mAb Genentech mAb cardiovascular disease Phase II (RG7652) South San Francisco, CA gene.com cardiovascular medicine Eli Lilly biologic cardiovascular disease Phase I Indianapolis, IN lilly.com Collategene® AnGes MG gene therapy peripheral arterial disease Phase II beperminogene Gaithersburg, MD (Fast Track) anges-mg.com perplasmid Mitsubishi Tanabe Pharma ------------------------------------------------ -------------------------------- Osaka, Japan ischemic heart disease Phase I anges-mg.com combination autologous TCA Cellular Therapy stem cell coronary ischemia, Phase II stem cell therapy Covington, LA therapy ischemic heart disorders tcacellulartherapy.com CPI-003 Capricor stem cell myocardial infarction Phase I (intracoronary cardio- Beverly Hills, CA therapy capricor.com sphere-derived stem cell therapy) desmoteplase Lundbeck recombinant ischemic stroke Phase III Deerfield, IL protein (Fast Track) lundbeck.com endometrial blood stem Medistem stem cell heart failure Phase II cell therapy San Diego, CA therapy medisteminc.com fibroblast growth CardioVascular BioTherapeutics fibroblast severe coronary heart disease Phase II factor-1 Las Vegas, NV growth factor (see also skin) cvbt.com Generx™ Cardium Therapeutics gene therapy myocardial ischemia Phase III alferminogene tadenovec San Diego, CA (Fast Track) cardiumthx.com GGF2 Acorda Therapeutics growth congestive heart failure Phase I (glial growth factor 2) Hawthorne, NY factor acorda.com GSK249320 GlaxoSmithKline mAb stroke Phase II Rsch. Triangle Park, NC gsk.comMedicines in Development Biologics 2013 41
  • Biologic Medicines in Development Cardiovascular Disease Product Name Sponsor Product Indication Development Phase Category IL-1 ß antibody Eli Lilly mAb cardiovascular disease Phase I Indianapolis, IN lilly.com Ilaris® Novartis Pharmaceuticals mAb secondary prevention of Phase III canakinumab East Hanover, NJ cardiovascular events novartis.com (see also autoimmune, diabetes, musculoskeletal) inclacumab Roche mAb peripheral vascular disease Phase II (RG1512) Nutley, NJ roche.com Iprivask® Canyon Pharmaceuticals recombinant prevention of thrombosis in clinical trials desirudin Columbia, MD protein canyonpharma.com ischemic tolerant Stemedica Cell Technologies stem cell acute myocardial infarction Phase II allogeneic mesenchymal San Diego, CA therapy stemedica.com stem cell therapy ISIS-APOCIIIRx Isis Pharmaceuticals antisense coronary artery disease Phase II Carlsbad, CA isispharm.com ISIS-CRPRx Isis Pharmaceuticals antisense atrial fibrillation, Phase II Carlsbad, CA coronary artery disease isispharm.com (see also autoimmune) ixmyelocel-T Aastrom Biosciences stem cell critical limb ischemia Phase III (Orphan Drug) Ann Arbor, MI therapy (Fast Track) aastrom.com ------------------------------------------------ --------------------------------- dilated cardiomyopathy Phase II aastrom.com MABp1 XBiotech mAb vascular restinosis Phase II Austin, TX (see also cancer, diabetes, skin) xbiotech.com mesenchymal precursor Mesoblast stem cell congestive heart failure, Phase II cell (MPC) product New York, NY therapy myocardial infarction mesoblast.com (see also diabetes, musculoskeletal, transplantation) MultiGeneAngio MultiGene Vascular Systems cell peripheral arterial disorders Phase I/II Nesher, Israel therapy mgvs.co.il MultiStem® Athersys stem cell myocardial infarction, stroke Phase II stem cell therapy Cleveland, OH therapy (see also digestive, transplantation) athersys.com Mydicar™ Celladon gene therapy advanced heart failure Phase II SERCA 2a gene therapy La Jolla, CA class III/IV celladon.net MyoCell® Bioheart stem cell congestive heart failure Phase II/III muscle stem cell therapy Sunrise, FL therapy bioheartinc.com42 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Cardiovascular Disease Product Name Sponsor Product Indication Development Phase Category MyoCell® SDF-1 Bioheart stem cell congestive heart failure Phase I muscle stem cell therapy Sunrise, FL therapy bioheartinc.com (second-generation) NTx®-265 Stem Cell Therapeutics stem cell stroke Phase II Toronto, Canada therapy stemcellthera.com PB-1046 PhaseBio Pharmaceuticals recombinant essential hypertension Phase I Malvern, PA fusion protein phasebio.com PCSK9 adnectin Bristol-Myers Squibb adnectin cardiovascular disease Phase I Princeton, NJ bms.com PF-04950615 Pfizer mAb hypercholesterolemia Phase II (RN316) New York, NY pfizer.com PF-05335810 Pfizer biologic hypercholesterolemia Phase I New York, NY pfizer.com PLX-PAD Pluristem Therapeutics stem cell intermittent claudication Phase II Haifa, Israel therapy pluristem.com Prochymal® Osiris Therapeutics stem cell acute myocardial infarction Phase II remestemcel-L Columbia, MD therapy (see also diabetes, digestive, osiris.com respiratory, transplantation) PRT-201 Proteon Therapeutics recombinant vascular access for hemodialysis Phase II (Orphan Drug) Waltham, MA protein (Fast Track) proteontherapeutics.com ------------------------------------------------ --------------------------------- peripheral vascular disease Phase I proteontherapeutics.com SAR236553/REGN727 Regeneron Pharmaceuticals mAb hypercholesterolemia Phase III (anti-PCSK-9 mAb) Tarrytown, NY regeneron.com Sanofi US sanofi.com Bridgewater, NJ SB623 SanBio stem cell stroke Phase I/II Mountain View, CA therapy san-bio.com SDF-1 Juventas Therapeutics gene therapy critical limb ischemia, heart failure Phase II (stromal cell-derived Cleveland, OH juventasinc.com factor-1) Syncria® GlaxoSmithKline recombinant heart failure Phase II albiglutide Rsch. Triangle Park, NC fusion protein (see also diabetes) gsk.com TS01 Thrombolytic Science Interna- recombinant acute ischemic stroke Phase I tional protein tsillc.net Cambridge, MAMedicines in Development Biologics 2013 43
  • Biologic Medicines in Development Cardiovascular Disease Product Name Sponsor Product Indication Development Phase Category Vascugel® Shire Regenerative Medicine cell peripheral vascular disorder in Phase II cell therapy Wayne, PA therapy patients undergoing arteriovenous shire.com (Orphan Drug) access procedures for hemodialysis (Fast Track) VM-202 VW BioPharma gene therapy peripheral arterial disease Phase II (modified hepatocyte Atlanta, GA (see also neurologic) viromed.co.kr growth factor gene ------------------------------------------------ --------------------------------- therapy) myocardial ischemia Phase I viromed.co.kr Diabetes Product Name Sponsor Product Indication Development Phase Category BHT-3201 Bayhill Therapeutics DNA type 1 diabetes Phase I completed Palo Alto, CA vaccine bayhilltx.com CVX-096 Pfizer recombinant type 2 diabetes Phase I (PF-04856883) New York, NY fusion pfizer.com protein diabetes medicine Eli Lilly biologic diabetes Phase I Indianapolis, IN lilly.com diabetes medicine Eli Lilly biologic diabetes Phase I Indianapolis, IN lilly.com diabetes medicine Eli Lilly biologic diabetes Phase I Indianapolis, IN lilly.com Diamyd® Diamyd vaccine type 1 diabetes Phase III autoimmune Pittsburgh, PA diamyd.com diabetes vaccine (Orphan Drug) dulaglutide Eli Lilly recombinant type 2 diabetes Phase III (GLP-1) Indianapolis, IN fusion protein lilly.com gevokizumab XOMA mAb type 1 diabetes, type 2 diabetes Phase II (IL-1B inhibitor mAb) Berkeley, CA (see also eye, musculoskeletal, xoma.com skin) GSK1070806 GlaxoSmithKline mAb type 2 diabetes Phase II (IL-18 mAb) Rsch. Triangle Park, NC (see also digestive) gsk.com IBC-VS01 Orban Biotech vaccine type I diabetes Phase I completed (insulin B-chain vaccine) Brookline, MA orbanbiotech.com44 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Diabetes Product Name Sponsor Product Indication Development Phase Category Ilaris® Novartis Pharmaceuticals mAb type 2 diabetes Phase II/III canakinumab East Hanover, NJ (see also autoimmune, novartis.com cardiovascular, musculoskeletal) ------------------------------------------------ --------------------------------- type 1 diabetes Phase II novartis.com INGAP peptide Exsulin peptide type 1 diabetes, type 2 diabetes Phase II Minneapolis, MN fragment exsulin.com insulin glargine Sanofi US recombinant type 1 diabetes, type 2 diabetes Phase III (rDNA origin) Bridgewater, NJ insulin sanofi.com (new formulation) insulin lispro/BC106 Adocia recombinant type 1 diabetes, type 2 diabetes Phase I (LY275585) Lyon, France protein lilly.com Eli Lilly Indianapolis, IN ISIS-GCCRRx Isis Pharmaceuticals antisense type 2 diabetes Phase I Carlsbad, CA (see also other) isispharm.com ISIS-GCGRRx Isis Pharmaceuticals antisense type 2 diabetes Phase I Carlsbad, CA isispharm.com ISIS-PTP1BRx Isis Pharmaceuticals antisense type 2 diabetes Phase I (PTP-1B gene inhibitor) Carlsbad, CA isispharm.com Lantus® Sanofi US recombinant type 2 diabetes Phase I insulin glargine Bridgewater, NJ insulin (see also cardiovascular) sanofi.com (rDNA origin) injection and lixisenatide combination LY2605541 Boehringer Ingelheim insulin type 1 diabetes, type 2 diabetes Phase III (novel basal insulin/ Pharmaceuticals hormone boehringer-ingelheim. insulin peglispro) Ridgefield, CT com Eli Lilly lilly.com Indianapolis, IN LY2963016 Boehringer Ingelheim insulin type 1 diabetes, type 2 diabetes Phase III (new insulin glargine Pharmaceuticals hormone boehringer-ingelheim. product) Ridgefield, CT com Eli Lilly lilly.com Indianapolis, IN MABp1 XBiotech mAb type 2 diabetes Phase II Austin, TX (see also cancer, cardiovascular, xbiotech.com skin) mesenchymal precursor Mesoblast stem cell type 2 diabetes Phase II cell (MPC) product New York, NY therapy (see also cardiovascular, mesoblast.com musculoskeletal, transplantation)Medicines in Development Biologics 2013 45
  • Biologic Medicines in Development Diabetes Product Name Sponsor Product Indication Development Phase Category PB-1023 PhaseBio Pharmaceuticals recombinant type 2 diabetes Phase II (GLP-1 receptor agonist) Malvern, PA fusion phasebio.com protein PF-05231023 Pfizer biologic type 2 diabetes Phase I New York, NY pfizer.com Prochymal® Osiris Therapeutics stem cell type 1 diabetes Phase II remestemcel-L Columbia, MD therapy (see also cardiovascular, digestive, osiris.com (Orphan Drug) respiratory, transplantation) rHuPH20/insulin Halozyme Therapeutics recombinant type 1 diabetes, type 2 diabetes Phase II San Diego, CA fusion halozyme.com protein Syncria® GlaxoSmithKline recombinant type 2 diabetes application submitted albiglutide Rsch. Triangle Park, NC fusion (see also cardiovascular) gsk.com protein teplizumab MacroGenics mAb type 1 diabetes Phase III (Orphan Drug) Rockville, MD macrogenics.com Digestive Disorders Product Name Sponsor Product Indication Development Phase Category alicaforsen Atlantic Healthcare antisense pouchitis (Fast Track), Phase III Essex, United Kingdom distal ulcerative colitis, atlantichc.com proximal ulcerative colitis ALV003 Alvine Pharmaceuticals recombinant celiac disease Phase I San Carlos, CA protein alvinepharma.com AMG 139/MEDI-2070 Amgen mAb Crohn’s disease Phase I Thousand Oaks, CA amgen.com AstraZeneca astrazeneca.com Wilmington, DE AMG 181/MEDI-7183 Amgen mAb Crohn’s disease, ulcerative colitis Phase II Thousand Oaks, CA amgen.com AstraZeneca astrazeneca.com Wilmington, DE anrukinzumab Pfizer mAb ulcerative colitis Phase II (IMA-638) New York, NY pfizer.com anti-IP10 Bristol-Myers Squibb mAb Crohn’s disease, ulcerative colitis Phase II Princeton, NJ bms.com46 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Digestive Disorders Product Name Sponsor Product Indication Development Phase Category clazakizumab Bristol-Myers Squibb mAb Crohn’s disease Phase II (anti-IL6) Princeton, NJ (see also autoimmune, bms.com Alder Biopharmaceuticals musculoskeletal) alderbio.com Bothell, WA etrolizumab Genentech mAb ulcerative colitis Phase II (rhuMAb-ß7) South San Francisco, CA gene.com GSK1070806 GlaxoSmithKline mAb inflammatory bowel disease Phase I (IL-18 mAb) Rsch. Triangle Park, NC (see also diabetes) gsk.com HLA-DQ2 peptide vaccine ImmusanT peptide celiac disease Phase I Cambridge, MA vaccine immusant.com Humira® AbbVie mAb Crohn’s disease in children and Phase III adalimumab North Chicago, IL adolescents abbvie.com (see also autoimmune, eye, musculoskeletal, skin, other) MultiStem® Athersys stem cell ulcerative colitis Phase II stem cell therapy Cleveland, OH therapy (see also cardiovascular, athersys.com (PF-05285401) Pfizer transplantation) pfizer.com New York, NY NN8717 Novo Nordisk recombinant ulcerative colitis Phase II (rFXIII) Princeton, NJ protein novonordisk.com PDA-001 Celgene Cellular Therapeutics stem cell Crohn’s disease Phase II (cenplacel-L) Summit, NJ therapy (see also autoimmune) celgene.com PF-00547659 Pfizer mAb Crohn’s disease Phase II New York, NY pfizer.com PF-04236921 Pfizer mAb Crohn’s disease Phase II New York, NY (see also autoimmune) pfizer.com polyclonal antibody Cancer Advances polyclonal gastrointestinal reflux Phase II stimulator Durham, NC antibody disease (GERD) canceradvancesinc.com vaccine (see also cancer) Prochymal® Osiris Therapeutics stem cell Crohn’s disease (Fast Track), Phase III remestemcel-L Columbia, MD therapy gastrointestinal injury from osiris.com radiation exposure (see also cardiovascular, diabetes, respiratory, transplantation)Medicines in Development Biologics 2013 47
  • Biologic Medicines in Development Digestive Disorders Product Name Sponsor Product Indication Development Phase Category QAX576 Novartis Pharmaceuticals mAb rectal fistula in patients with Crohn’s Phase II East Hanover, NJ disease novartis.com (see also respiratory) Remicade® Janssen Biotech mAb Crohn’s disease (prevention Phase III infliximab Horsham, PA of relapse after surgery resection) janssenbiotech.com SAR252067 Sanofi US mAb Crohn’s disease, ulcerative colitis Phase I (anti-LIGHT mAb) Bridgewater, NJ sanofi.com SAR339658 Sanofi US mAb inflammatory bowel disease Phase II (VLA2 antagonist) Bridgewater, NJ sanofi.com Simponi® Janssen Biotech mAb ulcerative colitis application submitted golimumab Horsham, PA (see also autoimmune) janssenbiotech.com (subcutaneous) Stelara® Janssen Biotech mAb Crohn’s disease Phase II/III ustekinumab Horsham, PA (see also autoimmune, janssenbiotech.com musculoskeletal, other) tralokinumab AstraZeneca mAb ulcerative colitis Phase II Wilmington, DE (see also respiratory) astrazeneca.com MedImmune medimmune.com Gaithersburg, MD vedolizumab Takeda Pharmaceuticals mAb Crohn’s disease, ulcerative colitis Phase III (MLN0002) Deerfield, IL takeda.com Eye Conditions Product Name Sponsor Product Indication Development Phase Category anti-factor D Genentech mAb geographic atrophy associated with Phase II (RG7417) South San Francisco, CA age-related macular gene.com degeneration anti-LINGO Biogen Idec mAb optic neuritis Phase I (BIIB033) Weston, MA (see also autoimmune) biogenidec.com AVA-101 Avalanche Biotechnologies gene therapy age-related macular degeneration Phase I/II San Francisco, CA avalanchebiotech.com eye disorder MorphoSys mAb eye disorders Phase I therapeutic Martinsried, Germany novartis.com Novartis Pharmaceuticals East Hanover, NJ48 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Eye Conditions Product Name Sponsor Product Indication Development Phase Category Eylea® Bayer HealthCare recombinant diabetic macular edema, Phase III aflibercept Pharmaceuticals fusion protein macular edema following branch bayerpharma.com Wayne, NJ retinal vein occlusion regeneron.com Regeneron Pharmaceuticals Tarrytown, NY gevokizumab XOMA mAb intermediate or posterior uveitis Phase III (IL-1B inhibitor mAb) Berkeley, CA (see also diabetes, musculoskeletal, xoma.com (Orphan Drug) skin) GSK933776A GlaxoSmithKline mAb age-related macular degeneration Phase II (anti-B amyloid mAb) Rsch. Triangle Park, NC (see also neurologic) gsk.com GZ402663 Applied Genetic Technologies gene wet (neovascular) age-related Phase I (FLT-01) Alachua, FL therapy macular degeneration agtc.com anti-vascular Genzyme genzyme.com endothelial growth-factor Cambridge, MA gene therapy hl-con1 Iconic Therapeutics recombinant age-related macular Phase I Atlanta, GA protein degeneration iconictherapeutics. com HuCNS-SC® StemCells stem cell age-related macular degeneration Phase I/II adult neural stem Newark, CA therapy (see also genetic, neurologic) stemcellsinc.com cell therapy (intraocular) human retinal Advanced Cell Technology stem cell dry age-related macular Phase I/II pigment epithelial Santa Monica, CA therapy degeneration, Stargardt’s advancedcell.com cell therapy macular degeneration (Orphan Drug) Humira® AbbVie mAb uveitis Phase III adalimumab North Chicago, IL (see also autoimmune, digestive, abbvie.com musculoskeletal, skin, other) iSONEP™ Lpath mAb wet age-related macular Phase II sonepcizumab San Diego, CA degeneration lpath.com (intravitreous) iCo-007 iCo Therapeutics antisense diabetic macular edema Phase II Vancouver, Canada icotherapeutics.comMedicines in Development Biologics 2013 49
  • Biologic Medicines in Development Eye Conditions Product Name Sponsor Product Indication Development Phase Category Lucentis® Genentech mAb age-related macular degeneration application submitted ranibizumab South San Francisco, CA (0.5 mg PRN) gene.com Roche roche.com Nutley, NJ ------------------------------------------------ --------------------------------- age-related macular degeneration Phase I (sustained-delivery), diabetic gene.com macular edema (sustained- roche.com delivery), retinal vein occlusion Mitosol® Mobius Therapeutics recombinant prevention of pterygium recurrence application submitted mitomycin St. Louis, MO protein after excision surgery mobiustherapeutics.com ophthalmic NT 501 Neurotech Pharmaceuticals cell therapy retinitis pigmentosa Phase II/III (ciliary neurotrophic Cumberland, RI (Fast Track) neurotechusa.com factor) ------------------------------------------------ --------------------------------- (Orphan Drug) age-related macular Phase II degeneration neurotechusa.com (Fast Track) PF-655 Quark Pharmaceuticals RNA wet age-related macular Phase II (RTP801i14) Fremont, CA interference degeneration, diabetic macular quarkpharma.com edema PF-04382923 Pfizer antibody age-related macular degeneration Phase II (RN6G) New York, NY pfizer.com QPI-1007 Quark Pharmaceuticals RNA optic neuropathy Phase I Fremont, CA interference quarkpharma.com RetinoStat® Oxford BioMedica gene wet age-related macular Phase I angiostatin endostatin Oxford, United Kingdom therapy degeneration sanofi.com gene therapy Sanofi US Bridgewater, NJ RU-101 R-Tech Ueno recombinant dry eyes Phase I (recombinant human Tokyo, Japan protein rtechueno.com serum albumin) secukinumab Alcon Labs mAb uveitis Phase III (AIN457) Fort Worth, TX (see also autoimmune, alcon.com Novartis Pharmaceuticals musculoskeletal, skin) novartis.com East Hanover, NJ Soliris® Alexion Pharmaceuticals mAb severe or refractory neuromyelitis Phase II eculizumab Cheshire, CT optica alxn.com (see also autoimmune, infectious, transplantation)50 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Eye Conditions Product Name Sponsor Product Indication Development Phase Category StarGen® Sanofi US gene Stargardt’s disease Phase I/II Stargardt’s disease Bridgewater, NJ therapy (juvenile-onset macular dystrophy) sanofi.com gene therapy (Orphan Drug) Genetic Disorders Product Name Sponsor Product Indication Development Phase Category adrenoleucodystrophy bluebird bio gene adrenoleucodystrophy Phase I/II gene therapy Cambridge, MA therapy bluebirdbio.com INSERM Paris, France AGTC-0106 Applied Genetic Technologies gene therapy alpha-1-antitrypsin deficiency Phase II adeno-associated virus Alachua, FL agtc.com vector-medictoten (Orphan Drug) ALD-601 Cytomedix stem cell infantile-onset lysosomal storage Phase I Gaithersburg, MD therapy disease cytomedix.com ALN-TTR02 Alnylam Pharmaceuticals RNA TTR-mediated amyloidosis Phase II Cambridge, MA interference alnylam.com asfotase alfa Alexion Pharmaceuticals recombinant hypophosphatasia in infants Phase II/III (Orphan Drug) Cheshire, CT fusion and children alxn.com protein (Fast Track) ------------------------------------------------ -------------------------------- hypophosphatasia in adults and Phase II adolescents alxn.com AVI-4658 Sarepta Therapeutics antisense Duchenne muscular dystrophy Phase II (eteplirsen) Cambridge, MA sareptatherapeutics.com Biostrophin™ Asklêpios BioPharmaceuticals gene therapy Duchenne muscular dystrophy Phase I muscular dystrophy Chapel Hill, NC askbio.com gene therapy BMN-110 BioMarin Pharmaceutical recombinant mucopolysaccharidosis IV Phase III (Orphan Drug) Novato, CA enzyme (Morquio A syndrome) bmrn.comMedicines in Development Biologics 2013 51
  • Biologic Medicines in Development Genetic Disorders Product Name Sponsor Product Indication Development Phase Category BMN-701 BioMarin Pharmaceutical recombinant glycogen storage disease type II Phase II (Orphan Drug) Novato, CA fusion (Pompe disease) bmrn.com protein CEQ508 Marina Biotech RNA familial adenomatous Phase I/II (Orphan Drug) Bothell, WA interference polyposis marinabio.com GNE lipoplex Gradalis gene therapy muscular dystrophy Phase I (muscular dystrophy Carrollton, TX gradalisinc.com gene therapy) GSK2402968 GlaxoSmithKline antisense Duchenne muscular dystrophy Phase II (PRO-051) Rsch. Triangle Park, NC gsk.com (Orphan Drug) Prosensa prosensa.eu Leiden, The Netherlands GSK2696273 GlaxoSmithKline stem cell adenosine deaminase severe Phase III Rsch. Triangle Park, NC therapy combined immune deficiency gsk.com GZ402665 Genzyme recombinant Niemann-Pick type B Phase I (rhASM) Cambridge, MA enzyme genzyme.com (Orphan Drug) HGT2310 Shire enzyme Hunter syndrome Phase I/II (Orphan Drug) Dublin, Ireland replacement (mucopolysaccharidosis II) shire.com therapy HuCNS-SC® StemCells stem cell Pelizaeus-Merzbacher disease Phase I adult neural stem Newark, CA therapy (see also eye, neurologic) stemcellsinc.com cell therapy (intracerebral) ISIS-SMNRx Biogen Idec antisense spinal muscular atrophy Phase II (Orphan Drug) Weston, MA biogenidec.com Isis Pharmaceuticals isispharm.com Carlsbad, CA ISIS-TTRRx GlaxoSmithKline antisense familial amyloid polyneuropathy Phase III (Orphan Drug) Rsch. Triangle Park, NC gsk.com Isis Pharmaceuticals isispharm.com Carlsbad, CA Kineret® Sobi recombinant cryopyrin-associated application submitted anakinra Ardmore, PA protein periodic syndrome sobi.com (Orphan Drug) KRN-23 Kyowa Hakko Kirin Pharma mAb X-linked dominant Phase I/II Princeton, NJ hypophosphatemic rickets kyowa-kirin-pharma.com52 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Genetic Disorders Product Name Sponsor Product Indication Development Phase Category Kynamro™ Genzyme antisense severe heterozygous familial Phase III mipomersen Cambridge, MA hypercholesterolemia genzyme.com (Orphan Drug) Isis Pharmaceuticals isispharm.com Carlsbad, CA leber congenital Applied Genetic Technologies gene leber congenital amaurosis Phase I/II amaurosis gene Alachua, FL therapy agtc.com therapy (Orphan Drug) NAV™ therapeutic ReGenX Biosciences gene therapy Batten disease Phase II Washington, DC (see also blood) regenxbio.com PEG-PAL BioMarin Pharmaceutical recombinant phenylketonuria Phase II (Orphan Drug) Novato, CA enzyme bmrn.com PF-06252616 Pfizer biologic muscular dystrophy Phase I New York, NY pfizer.com PRX-102 Protalix Biotherapeutics recombinant Fabry’s disease Phase I/II (alpha-galactosidase) Carmiel, Israel protein protalix.com recombinant human C1 Pharming Group recombinant hereditary angioedema Phase III esterase inhibitor Leiden, The Netherlands protein (Fast Track) santarus.com (Orphan Drug) Santarus San Diego, CA sebelipase alfa Synageva BioPharma recombinant Wolman disease Phase II/III (Orphan Drug) Lexington, MA protein (lysosomal acid lipase deficiency) synageva.com (Fast Track) UshStat® Sanofi US gene therapy usher syndrome 1B Phase I/II usher syndrome gene Bridgewater, NJ sanofi.com therapy (Orphan Drug) WAS gene therapy Children’s Hospital Boston gene therapy Wiskott-Aldrich syndrome Phase I/II Boston, MA genethon.fr Genethon Évry, FranceMedicines in Development Biologics 2013 53
  • Biologic Medicines in Development Infectious Diseases Product Name Sponsor Product Indication Development Phase Category ACAM-Cdiff Sanofi Pasteur vaccine prevention and treatment of Phase II (Clostridium difficile Swiftwater, PA Clostridium difficile infections sanofipasteur.us toxoid vaccine) (Fast Track) ACE527 TD Vaccines vaccine prevention of enterotoxigenic Phase II Skorping, Denmark Escherichia coli (ETEC) infections tdvaccines.com acellular pertussis combo Novartis Vaccines vaccine diphtheria, pertussis, tetanus Phase I vaccine Cambridge, MA novartisvaccines.com ADVAX Aaron Diamond AIDS DNA HIV infection prevention Phase I completed (DNA vaccine) Research Center vaccine adarc.org New York, NY iavi.org International AIDS Vaccine ------------------------------------------------ --------------------------------- Initiative HIV infection prevention Phase I completed New York, NY (new delivery system) adarc.org Ichor Medical Systems iavi.org San Diego, CA AERAS-402/Ad35 Aeras recombinant tuberculosis prevention Phase I Rockville, MD vaccine aeras.org Crucell crucell.com Leiden, The Netherlands Agriflu® Novartis Vaccines vaccine influenza virus infections (pediatric) Phase III influenza virus vaccine Cambridge, MA ention) novartisvaccines.com AGS-004 Argos Therapeutics personalized HIV infection Phase II Durham, NC dendritic cell argostherapeutics.com vaccine Alferon LDO® Hemispherx Bioscience interferon human papillomavirus Phase I/II interferon-alpha-n3 Philadelphia, PA infections hemispherx.net oral ------------------------------------------------ --------------------------------- influenza Phase I hemisphrex.net ALN-RSV01 Alnylam Pharmaceuticals RNA respiratory syncytial virus (RSV) Phase II Cambridge, MA interference treatment alnylam.com Anthim™ Elusys Therapeutics mAb anthrax Phase I (ETI-204) Pine Brook, NJ (Fast Track) elusys.com (Orphan Drug) anthrax immune Cangene mAb anthrax Phase III globulin Berwyn, PA cangene.com anti-PD-L1 Bristol-Myers Squibb mAb hepatitis B Phase I Princeton, NJ (see also cancer) bms.com54 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Infectious Diseases Product Name Sponsor Product Indication Development Phase Category ASP-0113 AbbVie DNA prevention of cytomegalovirus Phase III (cytomegalovirus DNA North Chicago, IL vaccine (CMV) infections in stem cell us.astellas.com vaccine) Astellas Pharma US transplantation (Orphan Drug) Northbrook, IL ------------------------------------------------ --------------------------------- Vical prevention of CMV infections in solid Phase II San Diego, CA organ transplantation us.astellas.com AVI-7100 Sarepta Therapeutics antisense influenza A virus infections Phase I radavirsen Cambridge, MA sareptatherapeutics.com AVI-7288 Sarepta Therapeutics antisense Marburg virus Phase I (Orphan Drug) Cambridge, MA disease sareptatherapeutics.com AVI-7537 Sarepta Therapeutics antisense post-exposure treatment of Phase I Cambridge, MA Ebola virus infections sareptatherapeutics.com AVX101 AlphaVax gene HIV-1 infection prevention Phase I (single gene HIV vaccine) Rsch. Triangle Park, NC vaccine alphavax.com AVX601 AlphaVax genetically- prevention of CMV infections Phase I Rsch. Triangle Park, NC modified alphavax.com Novartis Vaccines vaccine novartisvaccines.com Cambridge, MA AZD9773 AstraZeneca polyclonal severe sepsis Phase II (anti-TNF-alpha poly- Wilmington, DE antibody astrazeneca.com clonal antibody) Bexsero® Novartis Vaccines recombinant meningococcal group B infections Phase II meningococcal vaccine Cambridge, MA vaccine novartisvaccines.com group B BioThrax® Emergent BioSolutions vaccine prevention of post-exposure Phase II anthrax vaccine Rockville, MD anthrax infection in combination emergentbiosolutions.com with other antibacterials (Fast Track) botulinum toxin vaccine DynPort Vaccine recombinant botulism Phase II completed Frederick, MD vaccine csc.com/dvc cell therapy Cell Medica cell viral infections Phase I/II London, United Kingdom therapy (see also cancer) cellmedica.co.uk Center for Cell and Gene Therapy Houston, TX chikungunya virus Inviragen vaccine chikungunya virus infections Phase II vaccine Fort Collins, Co inviragen.comMedicines in Development Biologics 2013 55
  • Biologic Medicines in Development Infectious Diseases Product Name Sponsor Product Indication Development Phase Category cholera vaccine PaxVax live, prevention of cholera infection Phase I Menlo Park, CA attenuated paxvax.com vaccine Chronvac-C® Inovio Pharmaceuticals gene therapy treatment of hepatitis C Phase II hepatitis C DNA Blue Bell, PA vaccine inovio.com vaccine ChronTech Pharma Huddinge, Sweden CYT107 Cytheris recombinant HIV infection treatment Phase II (interleukin-7) Rockville, MD interleukin (see also blood, cancer, cytheris.com transplantation) Cytolin® CytoDyn mAb HIV infection treatment Phase I anti-CD8 mAb Lake Oswego, OR cytodyn.com DCVax-001 Celldex recombinant HIV infection prevention and Phase I Needham, MA vaccine treatment celldextherapeutics.com Rockefeller University New York, NY dengue vaccine GlaxoSmithKline vaccine dengue Phase I Rsch. Triangle Park, NC gsk.com dengue vaccine Sanofi Pasteur vaccine mild to severe dengue fever Phase III Swiftwater, PA sanofipasteur.us DENVax™ Inviragen vaccine dengue Phase I tetravalent hybrid dengue Fort Collins, CO inviragen.com virus vaccine DermaVir™ Patch Genetic Immunity DNA HIV-1 infection treatment Phase II DNA topical patch vaccine McLean, VA vaccine geneticimmunity.com DNA vaccine Naval Medical DNA prevention of dengue fever Phase I (dengue) Research Center vaccine vical.com Silver Spring, MD Vical San Diego, CA DTP-HepB-polio-Hib Merck vaccine diphtheria, tetanus, pertussis, Phase III (pediatric hexavalent Whitehouse Station, NJ hepatitis B, poliomyelitis, merck.com vaccine) Sanofi Pasteur Haemophilus influenzae type b sanofipasteur.us (V419) Swiftwater, PA Ebola vaccine Crucell DNA prevention of Ebola virus infections Phase I Leiden, The Netherlands vaccine crucell.com56 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Infectious Diseases Product Name Sponsor Product Indication Development Phase Category Ebola virus vaccine GenPhar vaccine Ebola virus infections in clinical trials Mt. Pleasant, SC genphar.com enterovirus 71 vaccine Inviragen vaccine prevention of head, foot Phase I (INV21) Fort Collins, CO and mouth disease inviragen.com EP1300 VaxOnco DNA prevention of malaria infection Phase I (malaria DNA Seoul, South Korea vaccine vaccine) National Institute of Allergy and Infectious Diseases Bethesda, MD FGI-101-1A6 Functional Genetics mAb influenza Phase I completed Gaithersburg, MD functional-genetics.com Fluad® Novartis Vaccines vaccine influenza virus infections Phase III influenza virus vaccine Cambridge, MA in the elderly and children novartisvaccines.com FluBIok® Protein Sciences recombinant prevention of influenza virus Phase I/II derived from recombinant Meriden, CT vaccine infections in infants and children proteinsciences.com hemagglutinin (rHA) Fludase® NexBio recombinant influenza A virus H1N1 infections, Phase II completed DAS-181 inhalation San Diego, CA fusion protein prevention of influenza virus nexbio.com infections FluLaval® QIV GlaxoSmithKline vaccine prevention of influenza application submitted influenza virus vaccine Rsch. Triangle Park, NC infections gsk.com FluNhance® Protein Sciences recombinant influenza virus infections Phase III influenza virus vaccine Meriden, CT vaccine proteinsciences.com (rNA) Fluzone® QIV IM Sanofi Pasteur vaccine seasonal influenza virus infections application submitted quadrivalent influenza Swiftwater, PA sanofipasteur.us virus vaccine foravirumab Crucell mAb post-exposure prevention of rabies Phase II Leiden, The Netherlands crucell.com GI-5005 GlobeImmune genetically- hepatitis C Phase II Louisville, CO modified globeimmune.com vaccineMedicines in Development Biologics 2013 57
  • Biologic Medicines in Development Infectious Diseases Product Name Sponsor Product Indication Development Phase Category GSK692342 Aeras recombinant prevention of tuberculosis Phase II (Mtb72F/AS02A) Rockville, MD vaccine gsk.com GlaxoSmithKline Rsch. Triangle Park, NC GSK1437173A GlaxoSmithKline recombinant prevention of varicella zoster Phase III (varicella zoster vaccine) Rsch. Triangle Park, NC vaccine virus infections gsk.com GSK1492903A GlaxoSmithKline recombinant prevention of CMV infections Phase I (cytomegalovirus Rsch. Triangle Park, NC vaccine gsk.com glycoprotein vaccine) GSK2189242A GlaxoSmithKline recombinant prevention of pneumococcal Phase II (Streptococcus Rsch. Triangle Park, NC vaccine infections gsk.com pneumoniae recombinant- conjugated vaccine) GSK2654909A GlaxoSmithKline vaccine prevention of influenza Phase I Rsch. Triangle Park, NC A virus H9N2 gsk.com subtype (avian flu) GSK2654911A GlaxoSmithKline vaccine prevention of influenza Phase I Rsch. Triangle Park, NC A virus H9N2 gsk.com subtype (avian flu) GSK2830930A GlaxoSmithKline vaccine prevention of pneumococcal Phase I Rsch. Triangle Park, NC infections gsk.com GSK2838497A GlaxoSmithKline vaccine prevention of Haemophilus Phase I Rsch. Triangle Park, NC infections gsk.com HBV-002 Hawaii Biotech recombinant prevention of West Nile virus Phase I completed Aiea, Hawaii vaccine infections hibiotech.com hepatitis C immune Biotest Pharmaceuticals polyclonal hepatitis C prevention Phase II globulin Boca Raton, FL antibody biotestpharma.com (Orphan Drug) hepatitis C vaccine Okairos gene vaccine prevention of hepatitis C Phase I/II therapy Basel, Switzerland okairos.com Heplisav™ Dynavax Technologies DNA prevention of hepatitis B application submitted hepatitis B vaccine Berkeley, CA vaccine dynavax.com HerpV Agenus recombinant treatment of herpes simplex virus Phase II (herpes vaccine) Lexington, MA protein infections agenusbio.com vaccine Hexaxim™ Sanofi Pasteur vaccine diphtheria, tetanus, pertussis, hepa- application submitted DTP-HepB-polio-Hib Swiftwater, PA titis B, poliomyelitis, sanofipasteur.us vaccine Haemophilus influenzae type b58 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Infectious Diseases Product Name Sponsor Product Indication Development Phase Category HIV gene therapy Adaptimmune gene therapy HIV infection Phase I Philadelphia, PA adaptimmune.com Cardiff University Cardiff, Wales University of Pennsylvania Philadelphia, PA HIV recombinant vaccine GlaxoSmithKline recombinant HIV infection prevention Phase I Rsch. Triangle Park, NC vaccine gsk.com HIV recombinant vaccine GlaxoSmithKline recombinant HIV infection treatment Phase II Rsch. Triangle Park, NC vaccine gsk.com HIV vaccine Crucell recombinant HIV infection prevention Phase I Leiden, The Netherlands vaccine crucell.com Beth Israel Deaconess Medical Center Boston, MA International AIDS Vaccine Initiative New York, NY HIV vaccine GeoVax vaccine HIV infection prevention Phase II Smyrna, GA geovax.com HIV vaccine GeoVax vaccine HIV infection treatment Phase I/II Smyrna, GA geovax.com HIV vaccine Massachusetts General Hospital vaccine HIV infection Phase I Boston, MA Opal Therapeutics Parkvill, Australia HIV vaccine Novartis Vaccines vaccine HIV infection Phase I Cambridge, MA novartisvaccines.com HIV vaccine PaxVax oral vaccine HIV infection prevention in clinical trials San Diego, CA paxvax.com HIV vaccine Profectus Biosciences DNA HIV infection prevention Phase I (MAG pDNA) Baltimore, MD vaccine profectusbiosciences.com HIV vaccine Profectus Biosciences recombinant HIV-1 infection prevention Phase I (rVSV) Baltimore, MD vaccine profectusbiosciences.com HIV-1 gene therapy Takara Bio gene therapy HIV-1 infection Phase I (MazF) Shiga, Japan takara-bio.comMedicines in Development Biologics 2013 59
  • Biologic Medicines in Development Infectious Diseases Product Name Sponsor Product Indication Development Phase Category HIV-1 vaccine Sumagen vaccine HIV-1 infection Phase I (SAV001) Seoul, South Korea sumagen.co.kr HIVAX™ GeneCure Biotechnologies vaccine HIV-1 infection Phase I replication-defective Norcross, GA genecure.com HIV-1 vaccine HSV-2 Tx Genocea Biosciences T-cell vaccine treatment of herpes simplex virus 2 Phase I/II Cambridge, MA infections genocea.com human interferon Amarillo Biosciences interferon hepatitis C, influenza Phase II alpha oral Amarillo, TX amarbio.com (Orphan Drug) ibalizumab TaiMed Biologics USA mAb HIV-1 infection Phase II (TMB-355) Irvine, CA (intravenous) taimedbiologics.com (Fast Track) ------------------------------------------------ --------------------------------- HIV-1 infection Phase I (subcutaneous) taimedbiologics.com ID-93/GLA-SE Aeras Global TB Vaccine recombinant tuberculosis Phase I Foundation fusion aeras.org Rockville, MD protein idri.org Infectious Disease Research vaccine Institute Seattle, WA Imvamune® Bavarian Nordic vaccine smallpox Phase II smallpox vaccine Kvistgaard, Denmark bavarian-nordic.com influenza A virus H1N1 CEL-SCI peptide prevention of influenza A virus Phase I vaccine Vienna, VA vaccine H1N1 subtype cel-sci.com influenza A virus H1N1 iBio vaccine prevention of influenza A virus Phase I vaccine Newark, DE H1N1 subtype ibioinc.com influenza A virus H1N1 Medicago VLP vaccine influenza A virus H1N1 subtype Phase I vaccine Quebec, Canada medicago.com influenza A virus H1N1 Inovio Pharmaceuticals DNA vaccine influenza A virus H1N1 subtype Phase I vaccine (FVH-1) Blue Bell, PA prevention inovio.com influenza A virus H1N1 Antigen Express peptide influenza A virus H1N1 infections Phase I vaccine (li-key hybrid Worcester, MA vaccine antigenexpress.com peptide vaccine)60 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Infectious Diseases Product Name Sponsor Product Indication Development Phase Category influenza A virus H5N1 GlaxoSmithKline cell-cultured prevention of pandemic influenza Phase I cell-cultured based Rsch. Triangle Park, NC vaccine A virus H5N1 subtype infections gsk.com vaccine influenza A virus H5N1 Nanotherapeutics vaccine prevention of influenza A virus H5N1 Phase I intranasal vaccine Alachua, FL subtype nanotherapeutics.com influenza A virus H5N1 iBio vaccine influenza A virus H5N1 subtype Phase I vaccine Newark, DE ibioinc.com influenza A virus H5N1 Inovio Pharmaceuticals vaccine prevention of influenza A virus H5N1 Phase I vaccine Blue Bell, PA subtype inovio.com influenza A virus H5N1 Medicago VLP vaccine influenza A virus H5N1 subtype Phase I vaccine Quebec, Canada medicago.com influenza A virus H5N1 Novartis Vaccines vaccine prevention of influenza A virus Phase II vaccine Cambridge, MA H5N1 subtype novartisvaccines.com influenza A virus H5N1 Novavax vaccine prevention of influenza A virus Phase II vaccine Rockville, MD H5N1 subtype novavax.com influenza A virus H5N1 PaxVax vaccine prevention of influenza A virus Phase I vaccine San Diego, CA H5N1 subtype paxvax.com influenza A virus H5N1 Vaxart vaccine prevention of influenza A virus Phase I vaccine San Francisco, CA H5N1 subtype vaxart.com influenza A virus H5N1 Antigen Express peptide influenza A virus H5N1 subtype Phase I vaccine Worcester, MA vaccine antigenexpress.com (li-key hybrid peptide vaccine) influenza A virus H9N2 Baxter International vaccine influenza A virus H9N2 subtype Phase I/II vaccine Deerfield, IL baxter.com influenza virus-like Novavax vaccine prevention of influenza virus Phase II particle vaccine Rockville, MD infections novavax.com influenza virus vaccine Crucell vaccine prevention of influenza virus Phase II (intradermal) Leiden, The Netherlands infections crucell.com Influvac® TC AbbVie cell-cultured influenza virus infections Phase I cell culture-derived North Chicago, IL vaccine (prevention) abbvie.com influenza vaccineMedicines in Development Biologics 2013 61
  • Biologic Medicines in Development Infectious Diseases Product Name Sponsor Product Indication Development Phase Category interferon-alpha-2b HanAll Biopharma interferon hepatitis C Phase II Seoul, South Korea hanallbiopharma.com interferon alpha-2b Medtronic interferon hepatitis C Phase II completed infusion Minneapolis, MN medtronic.com Ixiaro® Intercell vaccine prevention of Japanese encephalitis application submitted Japanese encephalitis Vienna, Austria in children intercell.com vaccine Novartis Vaccines novartisvaccines.com Cambridge, MA KB001-A KaloBios Pharmaceuticals mAb Pseudomonas infections in cystic Phase I/II (antibody-fragment South San Francisco, CA fibrosis patients (KalBios only) kalobios.com product) Sanofi Pasteur ------------------------------------------------ --------------------------------- Swiftwater, PA prevention of ventilator-associated Phase I pneumonia kalobios.com sanofipasteur.us KD-247 Kaketsuken mAb HIV-1 infection Phase I Kumamoto, Japan kaketsuken.or.jp LIQ-001 Liquidia Technologies vaccine influenza virus infections Phase I Rsch. Triangle Park, NC liquidia.com Lyme disease vaccine Baxter Healthcare recombinant prevention of Lyme disease Phase I/II Deerfield, IL vaccine baxter.com malaria vaccine Crucell recombinant malaria Phase I/II Leiden, The Netherlands vaccine crucell.com GlaxoSmithKline gsk.com Rsch. Triangle Park, NC malaria vaccine GenVec recombinant malaria Phase I/II Gaithersburg, MD vaccine genvec.com U.S. Naval Medical Research Center Silver Spring, MD malaria vaccine Sanaria vaccine prevention of malaria Phase I/II Rockville, MD sanaria.com Marburg virus GenPhar vaccine Marburg virus disease Phase I DNA vaccine Mt. Pleasant, SC genphar.com MBL-HCV1 MassBiologics mAb hepatitis C Phase II Boston, MA umassmed.edu/ massbiologics62 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Infectious Diseases Product Name Sponsor Product Indication Development Phase Category MEDI-550 AstraZeneca vaccine pandemic influenza prevention Phase I (pandemic influenza virus Wilmington, DE astrazeneca.com vaccine) MedImmune medimmune.com Gaithersburg, MD MEDI-557 AstraZeneca mAb prevention of RSV infections in Phase I (RSV mAb- Wilmington, DE high risk adults astrazeneca.com extended half-life) MedImmune medimmune.com Gaithersburg, MD MEDI-559 AstraZeneca vaccine prevention of RSV infections Phase I/II (RSV vaccine) Wilmington, DE (pediatric) astrazeneca.com MedImmune medimmune.com Gaithersburg, MD MenABCWY Novartis Vaccines vaccine meningococcal infections Phase II (meningococcal vaccine Cambridge, MA novartisvaccines.com groups A,C,Y,W-135 conjugate) meninge ACYW conjugate Sanofi Pasteur vaccine meningococcal infections Phase II vaccine Swiftwater, PA sanofipasteur.us (2nd-generation meningococcal conjugate infant vaccine) meningococcal vaccine GlaxoSmithKline vaccine meningococcal infections Phase III groups A,C,Y,W-135 Rsch. Triangle Park, NC gsk.com conjugate meningococcal vaccine JN-International Medical vaccine meningococcal group A,C,Y,W-135 Phase I groups A,C,Y,W-135 Omaha, NE infections jn-vaccines.org conjugate Menveo® Novartis Vaccines vaccine meningococcal groups A,C,Y,W-35 application submitted meningococcal vaccine Cambridge, MA infection in infants (ages 2–12 novartisvaccines.com groups A,C,Y, W-135 months) conjugate vaccine miravirsen Santaris Pharma A/S USA antisense hepatitis C Phase II (SPC3649) San Diego, CA santaris.com MK-3415A Merck mAb Clostridium difficile infections Phase III (actoxumab/ Whitehouse Station, NJ merck.com bezlotoxumab) MMR vaccine live GlaxoSmithKline vaccine prevention of measles, mumps, Phase III (GSK209762) Rsch. Triangle Park, NC rubella gsk.com MnB rLP2086 Pfizer recombinant adolescent and young adult Phase III (PF-05212366) New York, NY vaccine meningitis B pfizer.comMedicines in Development Biologics 2013 63
  • Biologic Medicines in Development Infectious Diseases Product Name Sponsor Product Indication Development Phase Category MVA-BN® Bavarian Nordic vaccine HIV-1 infection prevention and Phase I/II HIV multiantigen vaccine Washington, DC treatment bavarian-nordic.com NB-001 NanoBio nano- recurrent herpes simplex virus Phase III Ann Arbor, MI emulsion infections nanobio.com NB-002 NanoBio nano- distal subungual onychomycosis Phase II Ann Arbor, MI emulsion of the toe nanobio.com NB-1008 NanoBio nano- prevention of influenza virus Phase I completed (intranasal) Ann Arbor, MI emulsion infections nanobio.com vaccine NDV-3 NovaDigm Therapeutics recombinant prevention of candidiasis, Phase I Grand Forks, ND vaccine prevention of methicillin-resistant novadigm.net Staphylococcus aureus (MRSA) infections norovirus virus-like LigoCyte Pharmaceuticals vaccine norovirus infections Phase I/II particle vaccine Bozeman, MT ligocyte.com intranasal Nuthrax™ Emergent BioSolutions vaccine anthrax Phase I anthrax vaccine adsorbed Rockville, MD (Fast Track) emergentbiosolutions.com with CPG 7909 adjuvant PanBlok™ Protein Sciences vaccine prevention of influenza A virus Phase I/II influenza A virus H5N1 Meriden, CT H5N1 subtype infections proteinsciences.com vaccine peginterferon lambda-1a Bristol-Myers Squibb interferon hepatitis C Phase III Princeton, NJ bms.com ------------------------------------------------ --------------------------------- hepatitis B Phase II bms.com Pennvax®-B Inovio Pharmaceuticals DNA HIV infection prevention and Phase I DNA vaccine Blue Bell, PA vaccine treatment inovio.com (clade B) Pennvax®-G Inovio Pharmaceuticals DNA HIV infection prevention Phase I DNA vaccine Blue Bell, PA vaccine inovio.com (clades A, C, D) PF-06290510 Pfizer vaccine Staphylococcus aureus infections Phase II (SA4Ag) New York, NY pfizer.com64 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Infectious Diseases Product Name Sponsor Product Indication Development Phase Category PF-06425090 Pfizer vaccine Clostridium difficile colitis Phase I (Clostridium difficile New York, NY pfizer.com vaccine) plague injectable vaccine DynPort Vaccine vaccine Yersinia infections Phase II Frederick, MD csc.com/dvc Preflucel™ Baxter International vaccine prevention of influenza virus Phase III seasonal influenza virus Deerfield, IL infections baxter.com vaccine DynPort Vaccine csc.com/dvc Frederick, MD Prepandrix™ GlaxoSmithKline vaccine prevention of influenza A virus H5N1 application submitted H5N1 (pre-)pandemic Rsch. Triangle Park, NC subtype for pandemic use gsk.com influenza virus vaccine ------------------------------------------------ --------------------------------- prevention of influenza A virus Phase III H5N1 subtype for pandemic use in gsk.com children and adolescents PreviThrax™ Emergent Biosolutions recombinant anthrax Phase II recombinant protective Rockville, MD vaccine (Fast Track) emergentbiosolutions.com antigen anthrax vaccine PRO 140 CytoDyn mAb HIV infection Phase II completed Lake Oswego, OR cytodyn.com Quadracel® Sanofi Pasteur vaccine diphtheria, tetanus, pertussis, Phase III DTP, polio vaccine Swiftwater, PA poliomyelitis (4-6 years of age) sanofipasteur.us rabies VRVg vaccine Sanofi Pasteur vaccine prevention of rabies Phase II (purified vero rabies Swiftwater, PA sanofipasteur.us vaccine) respiratory syncytial virus Novavax recombinant prevention of RSV infections Phase II VLP vaccine Rockville, MD vaccine novavax.com Rivax™ Soligenix vaccine ricin poisoning Phase I ricin vaccine Princeton, NJ (pre-exposure) soligenix.com (Orphan Drug) rotavirus vaccine Sanofi Pasteur vaccine rotavirus Phase I (live attenuated tetrava- Swiftwater, PA sanofipasteur.us lent oral vaccine) SAR279356 Sanofi US mAb prevention of bacterial infections Phase II (anti-PNAG mAB) Bridgewater, NJ sanofi.comMedicines in Development Biologics 2013 65
  • Biologic Medicines in Development Infectious Diseases Product Name Sponsor Product Indication Development Phase Category SB-728-T Sangamo BioSciences gene HIV infection Phase II Richmond, CA therapy sangamo.com Soliris® Alexion Pharmaceuticals mAb Shiga toxin E. coli-related hemolytic Phase II eculizumab Cheshire, CT uremic syndrome (STEC-HUS) alxn.com (Orphan Drug) (see also autoimmune, eye, transplantation) Stealth Vector® Enzo Therapeutics antisense HIV-1 infection treatment Phase I/II HGTV-43™ New York, NY enzo.com antisense gene medicine streptococcal B vaccine Novartis Vaccines mAb prevention of streptococcal B Phase I conjugate Cambridge, MA infections novartisvaccines.com Streptococcus Sanofi Pasteur vaccine treatment of meningitis and Phase I pneumoniae vaccine Swiftwater, PA pneumonia sanofipasteur.us (meningitis and pneumonia vaccine) TCN-032 Theraclone Sciences mAb influenza A virus infections Phase I (IgG mAb) Seattle, WA theraclone-sciences.com TCN-202 Theraclone Sciences mAb CMV infections Phase I Seattle, WA theraclone-sciences.com TDENV-PIV GlaxoSmithKline vaccine dengue Phase I (tetravalent vaccine) Rsch. Triangle Park, NC gsk.com TG-4040 Transgene recombinant hepatitis C Phase II Cedex, France vaccine transgene.fr Thravixa™ Emergent BioSolutions mAb post-exposure treatment of anthrax Phase I completed fully human anthrax mAb Rockville, MD (Fast Track) emergentbiosolutions.com (Orphan Drug) TKM-Ebola Tekmira Pharmaceuticals RNA Ebola virus infections Phase I Burnaby, Canada interference tekmirapharm.com tuberculosis vaccine Sanofi Pasteur recombinant tuberculosis Phase I (recombinant subunit Swiftwater, PA vaccine sanofipasteur.us vaccine) tularemia vaccine DynaPort Vaccine vaccine tularemia Phase I completed Frederick, MD csc.com/dvc TUTI-16 Thymon vaccine HIV-1 infection treatment Phase I/II (lipoprotein vaccine) Short Hills, NJ66 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Infectious Diseases Product Name Sponsor Product Indication Development Phase Category UB-421 United Biomedical mAb HIV-1 infection treatment Phase II Hauppauge, NY unitedbiomedical.com V114 Merck vaccine prevention of pneumococcal Phase II (pneumococcal 15-valent Whitehouse Station, NJ infections merck.com vaccine conjugate) V212 Merck vaccine prevention of herpes zoster Phase III Whitehouse Station, NJ merck.com V503 Merck vaccine prevention of human papillomavirus Phase III Whitehouse Station, NJ infections merck.com (see also cancer) Vacc-4x Bionor Pharma vaccine HIV-1 infection treatment Phase II Oslo, Norway bionorpharma.com VAX102 VaxInnate recombinant prevention of influenza A virus Phase I/II completed (flagellin.HuM2e) Cranbury, NJ fusion vaxinnate.com protein vaccine VAX125 VaxInnate recombinant prevention of influenza A virus Phase II completed (flagellin.HuHa) Cranbury, NJ fusion vaxinnate.com protein vaccine VAX128 Emergent BioSolutions recombinant prevention of influenza A virus H1N1 Phase I Rockville, MD vaccine subtype emergentbipsolutions. VaxInnate com Cranbury, NJ vaxinnate.com VAX161 Emergent BioSolutions recombinant prevention of influenza A virus H5N1 Phase I Rockville, MD vaccine subtype emergentbiosolutions. VaxInnate com Cranbury, NJ vaxinnate.com VaxiGrip® Sanofi Pasteur vaccine seasonal influenza virus infections Phase III quadrivalent inactivated Swiftwater, PA sanofipasteur.us influenza vaccine VGX-3400 Inovio Pharmaceuticals DNA prevention of influenza A virus Phase I Blue Bell, PA vaccine H5N1 subtype inovio.com visceral leishmaniasis Infectious Disease vaccine visceral leishmaniasis Phase I vaccine Research Institute idri.org Seattle, WAMedicines in Development Biologics 2013 67
  • Biologic Medicines in Development Infectious Diseases Product Name Sponsor Product Indication Development Phase Category XOMA 3AB XOMA mAb botulism Phase I Berkeley, CA xoma.com National Institute of Allergy and Infectious Diseases Bethesda, MD Zostavax® Merck vaccine prevention of herpes zoster Phase I varicella zoster Whitehouse Station, NJ merck.com virus vaccine live Musculoskeletal Disorders/Arthritis Product Name Sponsor Product Indication Development Phase Category ABT-981 AbbVie mAb osteoarthritis Phase I North Chicago, IL abbvie.com AMG 167 Amgen antibody metabolic bone diseases Phase I Thousand Oaks, CA amgen.com AMG 745 Amgen mAb muscular atrophy Phase I Thousand Oaks, CA amgen.com AMG 827 AstraZeneca mAb psoriatic arthritis Phase II (brodalumab) Wilmington, DE (see also respiratory, skin) astrazeneca.com Amgen amgen.com Thousand Oaks, CA blosozumab Eli Lilly mAb osteoporosis Phase II (LY2541546) Indianapolis, IN lilly.com BYM338 Novartis Pharmaceuticals mAb sporadic inclusion body myositis, Phase II East Hanover, NJ muscular atrophy novartis.com (see also cancer) Chondrogen® Osiris Therapeutics stem cell knee injuries, prevention of Phase II mesenchymal stem cell Columbia, MD therapy osteoarthritis osiris.com therapy for meniscal repair Cimzia® UCB mAb psoriatic arthritis Phase III certolizumab pegol Smyrna, GA (see also autoimmune) ucb.com68 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Musculoskeletal Disorders/Arthritis Product Name Sponsor Product Indication Development Phase Category clazakizumab Bristol-Myers Squibb mAb psoriatic arthritis Phase II (anti-IL6) Princeton, NJ (see also autoimmune, bms.com Alder Biopharmaceuticals digestive) alderbio.com Bothell, WA Dysport® Ipsen bacterial focal spasticity of upper Phase III abobotulinumtoxinA Paris, France protein and lower limb ipsen.com FGF-18 EMD Serono growth factor acute cartilage injury of the knee Phase II (sprifermin) Rockland, MA emdserono.com gevokizumab XOMA mAb osteoarthritis of the hand Phase II (IL-1B inhibitor mAb) Berkeley, CA (see also diabetes, eye, skin) xoma.com Humira® AbbVie mAb spondylarthritis Phase III adalimumab North Chicago, IL (see also autoimmune, abbvie.com digestive, eye, skin, other) Ilaris® Novartis Pharmaceuticals mAb gouty arthritis application submitted canakinumab East Hanover, NJ (see also autoimmune, novartis.com cardiovascular, diabetes) ------------------------------------------------ --------------------------------- osteoarthritis, polymyalgia Phase II rheumatica novartis.com ixekizumab Eli Lilly mAb psoriatic arthritis Phase III (IL-17 antibody) Indianapolis, IN (see also skin) lilly.com juvenile chondrocyte ISTO Technologies cell cartilage disorders Phase III grafts St. Louis, MO therapy isotech.com KTP-001 Kaketsuken recombinant intervertebral disc displacement Phase I/II (recombinant human Kumamoto, Japan protein kaketsuken.or.jp matrix metalloproteinase) Teijin Pharma teijin-pharma.co.jp Tokyo, Japan LY2495655 Eli Lilly mAb disuse muscular atrophy Phase II (anti-myostatin mAb) Indianapolis, IN (see also cancer) lilly.com MACI® Genzyme cell therapy articular cartilage defects Phase III matrix-induced Cambridge, MA genzyme.com autologous chondrocyte implantation MCS110 Novartis Pharmaceuticals mAb synovitis Phase II East Hanover, NJ novartis.comMedicines in Development Biologics 2013 69
  • Biologic Medicines in Development Musculoskeletal Disorders/Arthritis Product Name Sponsor Product Indication Development Phase Category mesenchymal precursor Mesoblast stem cell spinal fusion, spondylosis Phase II cell (MPC) product New York, NY therapy (see also cardiovascular, diabetes, mesoblast.com transplantation) mesenchymal stem cell Medipost stem cell osteoarthritis Phase I/II therapy Seoul, South Korea therapy medi-post.com NeoCart® Histogenics cell cartilage disorders Phase III autologous chondrocyte Waltham, MA therapy histogenics.com implantation Orencia® Bristol-Myers Squibb recombinant psoriatic arthritis Phase II abatacept Princeton, NJ fusion (see also autoimmune, bms.com protein other) Prolia® Amgen mAb male osteoporosis Phase III denosumab Thousand Oaks, CA (see also autoimmune) amgen.com romosozumab Amgen mAb postmenopausal osteoporosis Phase III (AMG 785) Thousand Oaks, CA amgen.com SAR391786 Regeneron Pharmaceuticals mAb treatment of muscle atrophy Phase I (REGN1033) Tarrytown, NY post-orthopedic surgery regeneron.com Sanofi US sanofi.com Bridgewater, NJ secukinumab Novartis Pharmaceuticals mAb psoriatic arthritis Phase III (AIN 457) East Hanover, NJ (see also autoimmune, eye, skin) novartis.com ------------------------------------------------ --------------------------------- polymyalgia rheumatica Phase II novartis.com SI-6603 Seikagaku enzyme intervertebral disc Phase II Tokyo, Japan displacement seikagaku.co.jp Stelara® Janssen Biotech mAb psoriatic arthritis application submitted ustekinumab Horsham, PA (see also autoimmune, digestive, janssenbiotech.com other) tanezumab Pfizer mAb osteoarthritis Phase III New York, NY (see also neurologic) pfizer.com teriparatide Eli Lilly recombinant healing of fractures in patients with Phase III (recombinant PTH 1-34) Indianapolis, IN hormone osteoporosis lilly.com TG-C TissueGene cell osteoarthritis Phase II Rockville, MD therapy tissuegene.com Xiaflex® Auxilium Pharmaceuticals peptide frozen shoulder Phase II collagenase clostridium Chesterbrook, PA (see also cancer, other) auxilium.com histolyticum BioSpecifics Technologies biospecifics.com70 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Neurologic Disorders Product Name Sponsor Product Indication Development Phase Category AAB-002 Janssen Alzheimer mAb Alzheimer’s disease Phase 0 Immunotherapy janimm.com South San Francisco, CA pfizer.com Pfizer New York, NY AAB-003/PF-05236812 Janssen Alzheimer mAb Alzheimer’s disease Phase I Immunotherapy janimm.com South San Francisco, CA pfizer.com Pfizer New York, NY AAV1-FS344 Milo Biotechnology gene therapy Becker muscular Phase I/II (gene therapy-delivered Cleveland, OH dystrophy milobiotechnology.com myostatin inhibitor) (Orphan Drug) ABT-110 AbbVie mAb chronic pain Phase II North Chicago, IL abbvie.com ACC-001 Janssen Alzheimer peptide Alzheimer’s disease Phase II (PF-05236806) Immunotherapy vaccine janimm.com South San Francisco, CA pfizer.com Pfizer New York, NY AD02 vaccine AFFiRis vaccine Alzheimer’s disease Phase II Vienna, Austria affiris.com GlaxoSmithKline gsk.com Rsch. Triangle Park, NC AD03 vaccine AFFiRis vaccine Alzheimer’s disease Phase I Vienna, Austria affiris.com GlaxoSmithKline gsk.com Rsch. Triangle Park, NC AGN-214868 Allergan recombinant postherpetic neuralgia Phase II (senrebotase) Irvine, CA protein (see also other) allergan.com ALD403 Alder Biopharmaceuticals mAb prevention of migraine Phase I Bothell, WA alderbio.com ATI355 Novartis Pharmaceuticals mAb spinal cord injury Phase I (anti-Nogo-A mAb) East Hanover, NJ novartis.com (Orphan Drug)Medicines in Development Biologics 2013 71
  • Biologic Medicines in Development Neurologic Disorders Product Name Sponsor Product Indication Development Phase Category AYX-1 Adynxx oglionucleo- postoperative pain Phase I San Francisco, CA tide adynxx.com BA-210 BioAxone Biosciences recombinant spinal cord injury Phase II (Orphan Drug) Cambridge, MA fusion protein bioaxonebio.com BAN2401 BioArctic Neuroscience mAb Alzheimer’s disease Phase I (amyloid beta-protein Stockholm, Sweden bioarctic.se inhibitor) Eisai eisai.com Woodcliff Lake, NJ bapineuzumab Janssen Alzheimer mAb Alzheimer’s disease Phase II (subcutaneous) Immunotherapy janimm.com (AAB-001) South San Francisco, CA pfizer.com Pfizer New York, NY Botox® Allergan bacterial motor neuron disease Phase III onabotulinumtoxinA Irvine, CA protein (see also other) allergan.com CAD106 Cytos Biotechology therapeutic Alzheimer’s disease Phase II Zurich, Switzerland vaccine cytos.com Novartis Pharmaceuticals novartis.com East Hanover, NJ CERE-110 Ceregene gene therapy Alzheimer’s disease Phase II (AAV-NGF) San Diego, CA ceregene.com CERE-120 Ceregene gene therapy Parkinson’s disease Phase II (AAV-NTN) San Diego, CA ceregene.com chondrocyte tissue ISTO Technologies cell back pain Phase I grafts St. Louis, MO therapy isotech.com crenezumab Genentech mAb Alzheimer’s disease Phase II (anti-Abeta) South San Francisco, CA gene.com fulranumab Janssen Research mAb cancer pain Phase II & Development janssenrnd.com Raritan, NJ GDNF-producing adult BrainStorm Cell Therapeutics stem cell amyotrophic lateral sclerosis Phase I/II stem cell therapy New York, NY therapy (ALS) brainstorm-cell.com (Orphan Drug) GSK1223249 GlaxoSmithKline mAb ALS Phase I (NOGO-A mAb) Rsch. Triangle Park, NC (see also autoimmune) gsk.com GSK933776A GlaxoSmithKline mAb Alzheimer’s disease Phase I completed (anti-B amyloid mAb) Rsch. Triangle Park, NC (see also eye) gsk.com72 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Neurologic Disorders Product Name Sponsor Product Indication Development Phase Category GZ404477 Genzyme gene therapy Parkinson’s disease Phase I (AAV-hAADC gene Cambridge, MA genzyme.com therapy) HuCNS-SC® StemCells stem cell thoracic spinal cord injury Phase I/II adult neural stem Newark, CA therapy (see also eye, genetic) stemcellsinc.com cell therapy (intraosseous) LY2951742 Arteaus Therapeutics mAb migraine prevention Phase II (CGRP peptide) Cambridge, MA lilly.com Eli Lilly Indianapolis, IN MEDI-5117 AstraZeneca mAb osteoarthritis pain Phase I (anti-IL-6 mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MD neurological disorders TCA Cellular Therapy stem cell ALS, spinal cord injuries Phase I stem cell therapy Covington, LA therapy tcacellulartherapy.com NSI-566 Neuralstem stem cell ALS Phase I (Orphan Drug) Rockville, MD therapy neuralstem.com RG1450 Roche mAb prodromal Alzheimer’s disease Phase II/III (gantenerumab) Nutley, NJ roche.com RN-307 Labrys Biologics mAb migraine Phase II (anti-CGRP mAb) San Francisco, CA SAR228810 Sanofi US mAb Alzheimer’s disease Phase I (anti-protofibrillar AB Bridgewater, NJ sanofi.com mAb) solanezumab Eli Lilly mAb Alzheimer’s disease Phase III (LY2062430) Indianapolis, IN lilly.com tanezumab Pfizer mAb chronic pain Phase II New York, NY (see also musculoskeletal) pfizer.com UB-311 United Biomedical vaccine Alzheimer’s disease Phase II immunotherapeutic Hauppauge, NY unitedbiomedical.com V950 Merck vaccine Alzheimer’s disease Phase I completed Whitehouse Station, NJ merck.com VM-202 VM BioPharma gene diabetic neuropathy Phase II (modified hepatocyte Atlanta, GA therapy (see also cardiovascular) viromed.co.kr growth factor gene therapy)Medicines in Development Biologics 2013 73
  • Biologic Medicines in Development Neurologic Disorders Product Name Sponsor Product Indication Development Phase Category Xeomin® Merz Pharmaceuticals bacterial post-stroke spasticity Phase III incobotulinumtoxinA Greensboro, NC protein merzusa.com ----------------------------------------------- --------------------------------- sialorrhea in patients with Phase II Parkinson’s disease and ALS merzusa.com Respiratory Disorders Product Name Sponsor Product Indication Development Phase Category ALT-836 Altor BioScience mAb acute lung injury, adult respiratory Phase II Miramar, FL distress syndrome altorbioscience.com Genentech (see also cancer) gene.com South San Francisco, CA AMG 157/MEDI-9929 Amgen mAb asthma Phase I Thousand Oaks, CA amgen.com AstraZeneca astrazeneca.com Wilmington, DE AMG 761 Amgen mAb asthma Phase I Thousand Oaks, CA amgen.com AMG 827 Amgen mAb asthma Phase II (brodalumab) Thousand Oaks, CA (see also musculoskeletal, skin) amgen.com AstraZeneca astrazeneca.com Wilmington, DE ASM8 Pharmaxis antisense asthma Phase II Exton, PA pharmaxis.com.au benralizumab AstraZeneca mAb asthma, chronic obstructive Phase II Wilmington, DE pulmonary disease (COPD) astrazeneca.com MedImmune medimmune.com Gaithersburg, MD Bosatria™ GlaxoSmithKline mAb asthma Phase III mepolizumab Rsch. Triangle Park, NC gsk.com carlumab Janssen Biotech mAb pulmonary fibrosis Phase II Horsham, PA janssenbiotech.com74 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Respiratory Disorders Product Name Sponsor Product Indication Development Phase Category CNTO-3157 Janssen Biotech mAb asthma Phase I Horsham, PA janssenbiotech.com CNTO-5825 Janssen Biotech mAb allergic asthma Phase I Horsham, PA janssenbiotech.com CYT003 Cytos Biotechnology vaccine allergic asthma Phase II Zurich, Switzerland cytos.com Excellair™ ZaBeCor Pharmaceuticals RNA asthma Phase II syk kinase silencer Bala Cynwyd, PA interference zabecor.com FG-3019 FibroGen mAb idiopathic pulmonary fibrosis Phase II San Francisco, CA (see also cancer, other) fibrogen.com grass pollen extract Stallergenes immuno- seasonal allergic rhinitis Phase III Cedex, France therapy stallergenes.com GS-6624 Gilead Sciences mAb idiopathic pulmonary fibrosis Phase I (simtuzumab) Foster City, CA (see also cancer, other) gilead.com GSK2586881 Apeiron Biologics recombinant acute lung injury Phase I (APN01) Vienna, Austria protein gsk.com GlaxoSmithKline Rsch. Triangle Park, NC KB003 KaloBios Pharmaceuticals mAb severe asthma Phase II South San Francisco, CA kalobios.com MEDI-4212 AstraZeneca mAb asthma Phase I (anti-IgE mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MD MEDI-7814 AstraZeneca mAb COPD Phase I (anti-C5/C5a mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MD MEDI-8968 AstraZeneca mAb COPD Phase II (anti-IL-1R mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MDMedicines in Development Biologics 2013 75
  • Biologic Medicines in Development Respiratory Disorders Product Name Sponsor Product Indication Development Phase Category MK-3641 Merck immuno- ragweed pollen-induced Phase III ragweed allergy vaccine Whitehouse Station, NJ therapy rhinoconjunctivitis merck.com (sublingual) vaccine MK-7243 Merck immuno- grass pollen-induced allergic Phase III grass pollen allergy tablet Whitehouse Station, NJ therapy rhinoconjunctivitis merck.com vaccine MK-8237 Merck vaccine allergic asthma, allergic rhinitis, Phase II house dust mite Whitehouse Station, NJ allergic rhinoconjunctivitis merck.com allergy vaccine tablet Pollinex® Quattro Allergy Therapeutics vaccine grass pollen hypersensitivity, Phase III injectable MPL allergy West Sussex, United Kingdom ragweed pollen hypersensitivity allergytherapeutics.com vaccine ----------------------------------------------- --------------------------------- tree pollen hypersensitivity Phase II allergytherapeutics.com PRM-151 Promedior recombinant idiopathic pulmonary fibrosis Phase I (rhPTX-2 for injection) Lexington, MA protein (see also other) promedior.com (Orphan Drug) Prochymal® Osiris Therapeutics stem cell COPD Phase II remestemcel-L Columbia, MD therapy (see also cardiovascular, osiris.com diabetes, digestive, transplantation) QAX576 Novartis Pharmaceuticals mAb asthma, idiopathic pulmonary Phase II East Hanover, NJ fibrosis novartis.com (see also digestive) QBX258 Novartis Pharmaceuticals mAb asthma Phase II East Hanover, NJ novartis.com QGE031 Novartis Pharmaceuticals mAb allergic asthma Phase II East Hanover, NJ (see also skin) novartis.com quilizumab Genentech mAb allergic asthma, allergic rhinitis Phase II (anti-M1 prime mAb) South San Francisco, CA gene.com ragweed allergy Stallergenes vaccine seasonal allergic rhinitis Phase I immunotherapy Cedex, France stallergenes.com (sublingual) ragweed allergy vaccine Greer Laboratories vaccine seasonal allergic rhinitis Phase III (sublingual) Lenoir, NC greerlabs.com76 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Respiratory Disorders Product Name Sponsor Product Indication Development Phase Category reslizumab Cephalon mAb asthma, eosinophilic esophagitis in Phase III (Orphan Drug) Frazer, PA adolescents and children cephalon.com RG3637 Genentech mAb severe asthma Phase III (lebrikizumab) South San Francisco, CA gene.com Roche roche.com Nutley, NJ SAR156597 Sanofi US mAb idiopathic pulmonary fibrosis Phase II (bispecific interleukin-4/ Bridgewater, NJ sanofi.com interleukin-13 mAb) SAR231893 Regeneron Pharmaceuticals mAb asthma Phase II (anti-IL4 mAb) Tarrytown, NY (see also skin) regeneron.com Sanofi US sanofi.com Bridgewater, NJ STX-100 Biogen Idec mAb idiopathic pulmonary fibrosis Phase II Weston, MA biogenidec.com tralokinumab AstraZeneca mAb asthma Phase II Wilmington, DE (see also digestive) astrazeneca.com MedImmune medimmune.com Gaithersburg, MD Skin Diseases medicines for skin cancer are listed under Cancer Product Name Sponsor Product Indication Development Phase Category AbGn-168H AbGenomics International mAb plaque psoriasis Phase I completed Los Altos, CA abgenomics.com Amevive® Astellas Pharma US recombinant psoriasis in adolescents Phase II completed alefacept Deerfield, IL fusion us.astellas.com protein AMG 827 Amgen mAb psoriasis Phase II (brodalumab) Thousand Oaks, CA (see also musculoskeletal, amgen.com AstraZeneca respiratory) astrazeneca.com Wilmington, DE ANT-1207 Anterios bacterial acne vulgaris Phase II (botulinum toxin A) New York, NY protein, nanotechnol- ogyMedicines in Development Biologics 2013 77
  • Biologic Medicines in Development Skin Diseases medicines for skin cancer are listed under Cancer Product Name Sponsor Product Indication Development Phase Category BT-061 AbbVie mAb plague psoriasis Phase II North Chicago, IL (see also autoimmune) abbvie.com Biotest biotest.de Dreieich, Germany CNTO-1959 Janssen Biotech mAb plaque psoriasis Phase II Horsham, PA (see also autoimmune) janssenbiotech.com CureXcell™ Macrocure cell therapy diabetic foot ulcer Phase I/II white blood cell therapy Tikva, Israel macrocure.com Enbrel® Amgen recombinant plague psoriasis in adolescents application submitted etanercept Thousand Oaks, CA fusion protein and children amgen.com fibroblast growth CardioVascular Therapeutics fibroblast wounds in diabetes Phase II factor-1 Las Vegas, NV growth factor (see also cardiovascular) cvbt.com GBT 009 Garnet BioTherapeutics cell scar prevention Phase II Malvern, PA therapy garnetbio.com gevokizumab XOMA mAb acne vulgaris Phase II (IL-1B inhibitor mAb) Berkeley, CA (see also diabetes, eye, xoma.com musculoskeletal) HP802-247 Healthpoint Biotherapeutics cell leg ulcers Phase III (allogeneic human Fort Worth, TX therapy healthpointbio.com skin replacement) ------------------------------------------------ --------------------------------- Mohs micrographic surgical wounds Phase II healthpointbio.com Humira® AbbVie mAb hidradenitis suppurativa Phase III adalimumab North Chicago, IL (see also autoimmune, digestive, abbvie.com eye, musculoskeletal, other) ixekizumab Eli Lilly mAb psoriasis Phase III (IL-17 antibody) Indianapolis, IN (see also musculoskeletal) lilly.com JVS-100 SironRX Therapeutics gene wound healing related to Phase I (stromal cell-derived Cleveland, OH therapy cardiovascular surgery sironrx.com factor-1) KUR-211 Baxter International growth diabetic foot ulcers Phase II (bioactive wound-healing Deerfield, IL factor baxter.com foam) Kuros Biosurgery kuros.ch Zurich, Switzerland78 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Skin Diseases medicines for skin cancer are listed under Cancer Product Name Sponsor Product Indication Development Phase Category laViv™ Fibrocell Science fibroblast scars Phase II/III azficel-T Exton, PA cell fibrocellscience.com therapy MABp1 XBiotech mAb acne, psoriasis Phase II Austin, TX (see also cancer, cardiovascular, xbiotech.com diabetes) MK-3222 Merck mAb plaque psoriasis Phase III (tildrakizumab) Whitehouse Station, NJ merck.com PF-06473871 (EXC 001) Pfizer antisense dermal scarring Phase II New York, NY pfizer.com QGE031 Novartis Pharmaceuticals mAb atopic dermatitis Phase II East Hanover, NJ (see also respiratory) novartis.com REGN846 Regeneron Pharmaceuticals mAb atopic dermatitis Phase I/II Tarrytown, NY regeneron.com RT001 Revance Therapeutics bacterial lateral canthal lines Phase II (botulinum toxin A) Newark, CA protein (see also other) revance.com RXI-109 RXi Pharmaceuticals RNA dermal scarring Phase I Westborough, MA interference rxipharma.com SAR231893 Regeneron Pharmaceuticals mAb atopic dermatitis Phase II (anti-IL4 mAb) Tarrytown, NY (see also respiratory) regeneron.com Sanofi US sanofi.com Bridgewater, NJ SDF-1 SironRX gene therapy wound healing and reduction of Phase I Cleveland, OH scarring sironrx.com secukinumab Novartis Pharmaceuticals mAb plaque psoriasis Phase III (AIN457) East Hanover, NJ (see also autoimmune, novartis.com eye, musculoskeletal) StrataGraft® Stratatech cell chronic wounds Phase I/II tissue repair cell Madison, WI therapy stratatechcorp.com therapy (Orphan Drug) TD101 TransDerm RNA pachyonychia congenita Phase I Santa Cruz, CA interference transderminc.com Xolair® Genentech mAb chronic idiopathic urticaria Phase III omalizumab South San Francisco, CA gene.com Novartis Pharmaceuticals novartis.com East Hanover, NJMedicines in Development Biologics 2013 79
  • Biologic Medicines in Development Transplantation Product Name Sponsor Product Indication Development Phase Category ASKP-1240 Astellas Pharma US mAb prevention of organ transplant Phase II Deerfield, IL rejection us.astellas.com Kyowa Hakko Kirin Pharma kyowa-kirin-pharma.com Princeton, NJ Benlysta® GlaxoSmithKline mAb immunosuppression Phase II belimumab Rsch. Triangle Park, NC (see also autoimmune, blood) gsk.com (intravenous) CaspaCIDe™ DLI Bellicum Pharmaceuticals cell graft-versus-host disease in Phase I/II T-cell replacement Houston, TX therapy patients with late-stage cancer bellicum.com therapy undergoing bone marrow transplantation CDX-301 Celldex Therapeutics recombinant hematopoietic stem cell Phase I (Fms-like tyrosine Needham, MA protein transplantation celldextherapeutics.com kinase 3 ligand) CLT-008 Cellerant Therapeutics cell therapy facilitate engraftment in core Phase I San Carlos, CA blood transplants cellerant.com (see also cancer) CYT107 Cytheris recombinant prevention of bone marrow Phase I (interleukin-7) Rockville, MD interleukin transplant rejection cytheris.com (see also blood, cancer, infectious) mesenchymal precursor Mesoblast stem cell for bone marrow regeneration Phase III cell (MPC) product New York, NY therapy in patients undergoing bone mesoblast.com (Orphan Drug) marrow transplantation (see also cardiovascular, diabetes, musculoskeletal) MultiStem® Athersys stem cell graft-versus-host disease Phase I stem cell therapy Cleveland, OH therapy (see also cardiovascular, digestive) athersys.com (Orphan Drug) NX001 Nephrx growth delayed graft function in Phase I (Orphan Drug) Kalamazoo, MI factor renal transplant patients nephrx.com Prochymal® Osiris Therapeutics stem cell graft-versus-host disease Phase III remestemcel-L Columbia, MD therapy (Fast Track) osiris.com (Orphan Drug) (see also cardiovascular, diabetes, digestive, respiratory) QPI 1002 Quark Pharmaceuticals RNA prevention of delayed graft Phase II (I5NP) Fremont, CA interference function quarkpharma.com (Orphan Drug) (see also other) Soliris® Alexion Pharmaceuticals mAb presensitized kidney transplant Phase II eculizumab Cheshire, CT (acute humoral rejection) alxn.com (see also autoimmune, eye, infectious)80 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Transplantation Product Name Sponsor Product Indication Development Phase Category TOL101 Tolera Therapeutics mAb prevention of transplant rejection Phase I/II (Orphan Drug) Kalamazoo, MI tolera.com Other Product Name Sponsor Product Indication Development Phase Category AC607 AlloCure stem cell acute kidney injury Phase II (allogeneic multi-potent Burlington, MA therapy (Fast Track) allocure.com stem cell therapy) (Orphan Drug) AGN-214868 Allergan recombinant overactive bladder with urinary Phase II (senrebotase) Irvine, CA protein incontinence allergan.com (see also neurologic) ALD-451 Cytomedix stem cell cognition disorders following Phase I Gaithersburg, MD therapy treatment for malignant glioma cytomedix.com Ampligen® Hemispherx Biopharma RNA product chronic fatigue syndrome application submitted rintatolimid Philadelphia, PA hemispherx.net (Orphan Drug) antibody therapeutic MorphoSys mAb undisclosed Phase I Munich, Germany morphosys.com Novartis Pharmaceuticals novartis.com East Hanover, NJ antibody therapeutic MorphoSys mAb undisclosed Phase II Munich, Germany morphosys.com Novartis Pharmaceuticals novartis.com East Hanover, NJ antibody therapeutic MorphoSys mAb undisclosed Phase I (BI-1) Munich, Germany morphosys.com Boehringer-Ingelheim boehringer-ingelheim.com Pharmaceuticals Ridgefield, CT anti-IL31 Bristol-Myers Squibb mAb immunology Phase I Princeton, NJ bms.comMedicines in Development Biologics 2013 81
  • Biologic Medicines in Development Other Product Name Sponsor Product Indication Development Phase Category anti-TWEAK Biogen Idec mAb lupus nephritis Phase II (BIIB 023) Weston, MA biogenidec.com BAX-69 Baxter International mAb lupus nephritis Phase I Deerfield, IL (see also cancer) baxter.com Botox® Allergan bacterial idiopathic overactive application submitted onabotulinumtoxinA Irvine, CA protein bladder allergan.com (see also neurologic) ------------------------------------------------ --------------------------------- benign prostatic hyperplasia Phase II allergan.com cat allergy vaccine Circassia vaccine hypersensitivity in patients with Phase III Oxford, United Kingdom rhinoconjunctivitis caused by cat circassia.co.uk allergy CDX-1135 Celldex Therapeutics recombinant kidney disorders Phase I Needham, MA protein celldextherapeutics.com CM-AT CureMark enzyme autistic disorder Phase III Rye, NY replacement (Fast Track) curemark.com therapy CNTO-5 Janssen Biotech mAb inflammation Phase I Horsham, PA janssenbiotech.com MorphoSys morphosys.com Munich, Germany CNTO-136 Janssen Biotech mAb lupus nephritis Phase II (sirukumab) Horsham, PA (see also autoimmune) janssenbiotech.com ELAD® Vital Therapies cell liver failure Phase II/III human-cell-based San Diego, CA therapy vitaltherapies.com bioartificial liver (Orphan Drug) entolimod Cleveland BioLabs engineered acute radiation syndrome Phase I (CBLB502) Buffalo, NY peptide (Fast Track) cbiolabs.com (Orphan Drug) (see also cancer) FB 301 Cytovance Biologics mAb hypersensitivity in clinical trials Oklahoma City, OK (IgE-mediated allergic diseases) cytovance.com Fountain Biopharma Taipei, Taiwan FG-3019 FibroGen mAb liver fibrosis due to chronic Phase II San Francisco, CA hepatitis B infection fibrogen.com (see also cancer, respiratory)82 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Other Product Name Sponsor Product Indication Development Phase Category food allergy DBV Technologies topical peanut hypersensitivity Phase II immunotherapy Paris, France vaccine dbv-technologies.com fresolimumab Genzyme mAb fibrosis Phase II (TGFß antagonist) Cambridge, MA genzyme.com GS-6624 Gilead Sciences mAb liver fibrosis, myelofibrosis Phase II (simtuzumab) Foster City, CA (see also cancer, respiratory) gilead.com GSK1070806 GlaxoSmithKline mAb metabolic disorders Phase II (anti-interleukin 18 mAb) Rsch. Triangle Park, NC gsk.com hMaxi-K Ion Channel Innovations gene therapy overactive bladder Phase I completed gene therapy New York, NY ionchannelinnovations. com Humira® AbbVie mAb interstitial cystitis Phase III adalimumab North Chicago, IL (see also autoimmune, digestive, abbvie.com eye, musculoskeletal, skin) ISIS-FGFR4Rx Isis Pharmaceuticals antisense obesity Phase II Carlsbad, CA isispharm.com ISIS-GCCRRx Isis Pharmaceuticals antisense Cushing syndrome Phase I Carlsbad, CA (see also diabetes) isispharm.com JRC-LAMP-vax Immunomic Therapeutics DNA vaccine Japanese red cedar pollen-induced Phase I Lancaster, PA rhinoconjunctivitis immunomix.com kidney disease medicine Eli Lilly biologic chronic kidney disease Phase I Indianapolis, IN lilly.com liprotamase Eli Lilly enzyme treatment of exocrine pancreatic application submitted (LY3031642) Indianapolis, IN replacement enzyme insufficiency, including in lilly.com (Orphan Drug) therapy cystic fibrosis patients (Fast Track) liraglutide Novo Nordisk recombinant obesity Phase III (NN8022) Princeton, NJ peptide novonordisk.com liver cell therapy Cytonet cell urea cycle disorders in children Phase II (Orphan Drug) Weinheim, Germany therapy and infants cytonet.de LY2382770 Eli Lilly mAb diabetic nephropathy Phase II (TGF-ß antibody) Indianapolis, IN lilly.comMedicines in Development Biologics 2013 83
  • Biologic Medicines in Development Other Product Name Sponsor Product Indication Development Phase Category mAb Genentech mAb metabolic disorders Phase I South San Francisco, CA gene.com Meth-mAb InterveXion Therapeutics mAb methamfetamine abuse Phase I Little Rock, AR intervexion.com metreleptin Bristol-Myers Squibb recombinant lipodystrophy application submitted Princeton, NJ hormone bms.com Natpara® NPS Pharmaceuticals recombinant hypoparathyroidism Phase III recombinant human Salt Lake City, UT hormone npsp.com parathyroid hormone (rhPTH) (Orphan Drug) Neo-Urinary Conduit™ Tengion cell cystectomy due to bladder cancer Phase I autologous cultured Winston-Salem, NC therapy tengion.com urological tissue cells NIC002 Novartis Pharmaceuticals vaccine smoking withdrawal Phase II East Hanover, NJ novartis.com Orencia® Bristol-Myers Squibb recombinant lupus nephritis Phase III abatacept Princeton, NJ fusion (see also autoimmune, bms.com protein musculoskeletal) PF-05402536 Pfizer vaccine smoking cessation Phase I New York, NY pfizer.com PRM-151 Promedior recombinant myelofibrosis Phase I (rhPTX-2 for injection) Lexington, MA protein (see also respiratory) promedior.com prohibitin targeting Ablaris Therapeutics homing obesity Phase I peptide-1 Pasadena, CA peptide ablaris.com PRT-105 Protalix Biotherapeutics recombinant poisoning Phase I completed Carmiel, Israel enzyme protalix.com QPI-1002 Quark Pharmaceuticals RNA prevention of renal failure in patients Phase I/II (I5Np) Fremont, CA interference undergoing cardiovascular surgery quarkpharma.com (see also transplantation) recombinant human Kringle Pharma recombinant acute renal failure Phase I hepatocyte growth factor Osaka, Japan growth factor kringle-pharma.com REGN728 Regeneron Pharmaceuticals mAb undisclosed Phase I Tarrytown, NY regeneron.com84 Medicines in Development Biologics 2013
  • Biologic Medicines in Development Other Product Name Sponsor Product Indication Development Phase Category RT001 Revance Therapeutics bacterial hyperhidrosis Phase II (botulinum toxin A) Newark, CA protein (see also skin) revance.com SEL-068 Selecta Biosciences nanoparticle smoking withdrawal Phase I Watertown, MA vaccine selectabio.com somatropin biosimilar LG Life Sciences recombinant somatropin deficiency Phase III (sustained-release) Seoul, South Korea human lgls.com growth hormone Stelara® Janssen Biotech mAb primary biliary cirrhosis Phase II ustekinumab Horsham, PA (see also autoimmune, digestive, janssenbiotech.com musculoskeletal) TA-CD Celtic Pharma immuno- cocaine abuse Phase II Hamilton, Bermuda therapeutic celtictherapeutic.com vaccine UMK-121 Proteonomix stem cell liver failure Phase I Hawthorne, NJ therapy proteonomix.com VAY736 MorphoSys mAb inflammation Phase I Munich, Germany (see also cancer) morphosys.com Novartis Pharmaceuticals novartis.com East Hanover, NJ VEN100 Ventria Bioscience recombinant antibiotic-associated diarrhea Phase II (recombinant human Fort Collins, CO protein ventria.com lactoferrin) VRS-317 Versartis recombinant somatropin deficiency Phase I/II (human growth hormone) Redwood City, CA human versartis.com growth hormone Xiaflex® Auxilium Pharmaceuticals peptide Peyronie’s disease application submitted collagenase clostridium Chesterbrook, PA (see also cancer, musculoskeletal) auxilium.com histolyticum BioSpecifics Technologies biospecifics.com (Orphan Drug) Lynbrook, NY ------------------------------------------------ --------------------------------- cellulite Phase I auxilium.com biospecifics.comMedicines in Development Biologics 2013 85
  • Glossary adjuvant—Substance or drug that aids an- erythropoietin—Hormone that stimulates red can be reactivated to produce painful sores of other substance in its action. blood cell production in the bone marrow. the anus or genitals. angioedema—Swelling of the mucous mem- Fast Track—A process designed to facilitate human papillomavirus—Viral agent of warts, branes, tissues beneath the skin, or an internal the development and expedite the review believed to be contagious and usually harm- organ due to an allergic reaction. of drugs to treat serious diseases and fill an less, but it can lead to cervical cancer. unmet medical need. The status is assigned ankylosing spondylitis—An inflammatory interferon—A glycoprotein naturally produced by the U.S. Food and Drug Administration. disorder of unknown cause that primarily by cells that interferes with the ability of a virus The purpose is to get important new drugs affects the spine. The vertebrae may fuse to reproduce after it invades the body. to the patient earlier. Fast Track addresses a together and form a rigid back that is impos- broad range of serious diseases. Generally, interleukin—An endogenous substance that sible to bend. determining factors include whether the drug stimulates the production of different types of antisense—An antisense drug is the mirror or will have an impact on such factors as survival, white blood cells or leukocytes. complementary image of a small segment of day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from ischemia—Insufficient supply of blood to an messenger RNA (mRNA), the substance that a less severe condition to a more serious one. organ or tissue, which can cause organ dam- carries instructions (“sense”) from the genes to Filling an unmet medical need is defined as pro- age such as an ischemic stroke. the cell’s protein-making machinery. The anti- sense drug readily binds to the mRNA strand, viding a therapy where none exists or providing lymphoma—Cancers in which the cells of lym- keeping it from transmitting its instructions to a therapy which may be potentially superior phoid tissue, found mainly in the lymph nodes the cell, and thus inhibiting the production of an to existing therapy. Once a drug receives Fast and spleen, multiply unchecked. Lymphomas unwanted protein. Track designation, early and frequent commu- fall into two categories: One is called Hodgkin nication between the FDA and a drug company disease, characterized by a particular kind application submitted—An application for is encouraged throughout the entire drug de- of abnormal cell. All others are called non- marketing has been submitted by the company velopment and review process. The frequency Hodgkin lymphomas, which vary in their to the Food and Drug Administration (FDA). of communication assures that questions and malignancy according to the nature and activity biotechnology—The collection of processes issues are resolved quickly, often leading to of the abnormal cells. that involve the use of biological systems. earlier drug approval and access by patients. mAb (monoclonal antibodies)—Large protein For some of the industries, these processes gene therapy—Therapy at the intracellular molecules produced by white blood cells that involve the use of genetically engineered level to replace or inactivate the effects of seek out and destroy harmful foreign sub- organisms. For the purpose of this report, only disease-causing genes or to augment normal stances. those products that involve recombinant DNA, gene functions to overcome illness. monoclonal antibody/hybridoma, continuous macular degeneration—A progressive disor- cell lines, cellular therapy, gene therapy and glioblastoma—The most common primary der that affects the central part of the retina, vaccines technology are included. brain tumor and one of the most aggressive causing gradual loss of vision. It is a painless forms of brain cancer, primarily affecting adults condition, usually affects both eyes and is com- Clostridium difficile—A bacterium that over the age of 50. mon in the elderly. produces an irritating toxin that causes a form of colitis characterized by profuse, watery diar- growth factors—Factors responsible for metastases/metastatic—Cancers that have rhea with cramps and low-grade fever. regulating cell proliferation (rapid and repeated spread from the primary or original cancer site. reproduction), function and differentiation. Cancer cells can break away from a tumor and colony stimulating factor (CSF)—Protein growth hormone—Pituitary hormone that travel to other areas of the body through the responsible for controlling the production of stimulates the growth of long bones in prepu- bloodstream or the lymph system. They are of- white blood cells. bertal children. ten more difficult to treat then primary tumors. Crohn’s disease—A subacute chronic gastro- hemophilia A and B—Hemophilia A, the “clas- multiple myeloma—A malignant condition char- intestinal disorder, involving the small intestine, sic” hemophilia, is a genetic bleeding disorder acterized by uncontrolled proliferation of plasma characterized by patchy deep ulcers that may due to deficiency of the coagulation factor cells (a class of white blood cells) in bone cause fistulas and a narrowing and thickening VIII. Hemophilia B, or “Christmas” disease, is marrow. Symptoms include pain and destruction of the bowel. caused by deficiency of coagulation factor IX. of bone tissue, numbness and paralysis, kidney cytomegalovirus (CMV)—A DNA virus related damage, anemia, and frequent infections. to the herpes virus, affecting mostly neonatal hepatitis—Inflammation of the liver with accom- panying liver cell damage or death, caused most myelodysplastic syndromes (MDS)—Blood infants and immunocompromised individuals. often by viral infection, e.g., hepatitis B, and C. disorders that ultimately are fatal. Patients CMV can occur without symptoms or result in usually succumb to infections or bleeding. The mild flu-like symptoms. herpes simplex virus—A strain of herpes term “preleukemia” has been used to describe virus that may lie dormant in nerve tissue and these disorders.86 Medicines in Development Biologics 2013
  • Glossarymyeloid—Pertaining to, derived from, or mani- Phase I—Researchers test the drug in a small systemic lupus erythematosus (SLE)—Thefesting certain features of the bone marrow. In group of people, usually between 20 and 80 most serious form of lupus, a chronic autoim-some cases, myeloid also pertains to certain healthy adult volunteers, to evaluate its initial mune disorder causing inflammation andtypes of non-lymphocyte white blood cells damage to multiple organs. safety and tolerability profile, determine afound in the bone marrow. safe dosage range, and identify potential side thrombocytopenia—A reduction in themyocardial infarction—Damage to the effects. number of platelet cells in the blood, whichheart muscle caused by stoppage or impair- causes a tendency to bleed, especially fromment of blood flow to the heart, also known Phase II—The drug is given to volunteer the smaller blood vessels.as heart attack. patients, usually between 100 and 300, to see ulcerative colitis—A chronic inflammation andneuroblastoma—A tumor of the adrenal if it is effective, identify an optimal dose, and to ulceration of the lining of the colon and rectum.glands or sympathetic nervous system (the further evaluate its short-term safety. It causes bloody diarrhea and mainly involvespart of the nervous system responsible for the left colon.certain automatic body functions, such as the Phase III—The drug is given to a larger, more diverse patient population, often involving be- uveitis—Inflammation of the uvea, the middle“fight or flight response”). layer of the eye. tween 1,000 and 3,000 patients (but sometimeneutropenia—An abnormally low neutrophilcount (certain white blood cells). many more thousands), to generate statistically significant evidence to confirm its safety andOrphan Drug—A drug to treat a disease that effectiveness. They are the longest studies,has a patient population of 200,000 or lessin the United States, or a disease that has a and usually take place in multiple sites aroundpatient population of more than 200,000 and the world.a development cost that will not be recovered pulmonary fibrosis—Scarring and thickeningfrom sales in the United States. of lung tissue.peripheral vascular disorder—The obstruc- respiratory syncytial virus (RSV) disease—tion of blood supply to the extremities, particu- One of the most important causes of lowerlarly the legs, caused by atherosclerosis. respiratory tract disease in children, account-Phase 0—First-in-human trials conducted ing for more than 90 percent of cases ofin accordance with FDA’s 2006 guidance on bronchiolitis.exploratory Investigational New Drug (IND) restenosis—A condition where an artery plugsstudies designed to speed up development of up again following treatment to open it up.promising drugs by establishing very early onwhether the agent behaves in human subjects sepsis—A condition associated with a seriousas was anticipated from preclinical studies. bacterial infection of the blood.The content of this report has been obtained through public, government and industry sources, and the Adis “R&D Insight” database based on thelatest information. Report current as of February 7, 2013. The information in this report may not be comprehensive. For more specific informationabout a particular product, contact the individual company directly or go to www.clinicaltrials.gov. The entire series of Medicines in Development isavailable on PhRMA’s web site.A publication of PhRMA’s Communications & Public Affairs Department. (202) 835-3460www.phrma.org | www.innovation.org | www.pparx.org | www.buysafedrugs.infoProvided as a Public Service by PhRMA. Founded in 1958 as the Pharmaceutical Manufacturers Association.Copyright © 2013 by the Pharmaceutical Research and Manufacturers of America. Permission to reprint is awarded if proper credit is given.Pharmaceutical Research and Manufacturers of America • 950 F Street, NW, Washington, DC 20004Medicines in Development Biologics 2013 87