2013年在研白血病和淋巴瘤药物

20 13 R EPO RT Biopharmaceutical research companies are developing 241 medicines for blood cancers—leukemia, lymphoma and myeloma. This report lists medicines in human clinical trials or under review by the U.S. Food and Drug Administration (FDA). The medicines in development include: • 98 for lymphoma, including Hodgkin and non-Hodgkin lymphoma, which affect nearly 80,000 Americans each year. • 97 for leukemia, including the four major types, which affect nearly 50,000 people in the United States each year. • 52 for myeloma, a cancer of the plasma cells, which impacts more than 22,000 people each year in the United States. • 24 medicines are targeting hema- tological malignancies, which affect bone marrow, blood and lymph nodes. • 15 each for myeloproliferative neo- plasms, such as myelofibrosis, poly- cythemia vera and essential throm- bocythemia; and for myelodysplastic syndromes, which are diseases affect- ing the blood and bone marrow. These medicines in development offer hope for greater survival for the thou- sands of Americans who are affected by these cancers of the blood. Definitions for the cancers listed in this report and other terms can be found on page 27. Links to sponsor company web sites provide more information on the potential products. For information on the value of medi- cines, an in-depth look at current in- novation and key medical breakthroughs benefiting blood cancer patients, please see Medicines in Development for Leu- kemia and Lymphoma 2013—Overview. More Than 240 Medicines in Development for Leukemia, Lymphoma and Other Blood Cancers M ye lo m a Le uk em ia 24 97 98 Ly m ph om a 52 He m at ol og ica l M ali gn an cie s Application Submitted Phase III Phase II Phase I Medicines in Development For Leading Blood Cancers Every 4 minutes a person is diagnosed with leukemia, lymphoma or myeloma; Accounting for 9% of all cancers diagnosed each year Medicines in Development Leukemia & Lymphoma 20132 Medicines in Development for Leukemia & Lymphoma *For more information about a specific medicine or company in the report, please click on the provided link. Hematological Malignancies Product Name Sponsor Indication Development Phase* Adcetris® brentuximab vedotin (Orphan Drug) Millennium Pharmaceuticals Cambridge, MA Seattle Genetics Bothell, WA treatment of CD30-positive hematological malignancies (see also lymphoma) Phase II millennium.com seattlegenetics.com AMG 319 (PI3K-delta inhibitor) Amgen Thousand Oaks, CA hematological malignancies Phase I amgen.com anti-CD22 antibody-drug conjugate (DCDT 2980S) Genentech South San Francisco, CA hematological malignancies (see also lymphoma) Phase II gene.com anti-CD79b antibody-drug conjugate (DCDS 4501A) Genentech South San Francisco, CA hematological malignancies (see also lymphoma) Phase II gene.com AR-42 (pan-DAC inhibitor) Arno Therapeutics Flemington, NJ hematological malignancies Phase I/II arnothera.com AZD1208 (PIM kinase inhibitor) AstraZeneca Wilmington, DE hematological malignancies Phase I astrazeneca.com AZD9150 (STAT3 inhibitor) AstraZeneca Wilmington, DE hematological malignancies Phase I astrazeneca.com belinostat (HDAC inhibitor) Spectrum Pharmaceuticals Henderson, NV hematological malignancies (see also lymphoma) Phase I sppirx.com CB3304 (noscapine) Cougar Biotechnology Raritan, NJ hematological malignancies Phase I cougarbiotechnology.com darinaparsin (ZIO-101) ZIOPHARM Oncology New York, NY hematological malignancies (see also lymphoma, myeloma) Phase II ziopharm.com fostamatinib (Syk inhibitor) AstraZeneca Wilmington, DE Rigel Pharmaceuticals South San Francisco, CA hematological malignancies Phase II astrazeneca.com rigel.com GS-9973 (Syk inhibitor) Gilead Sciences Foster City, CA B-cell hematological malignancies Phase I gilead.com Medicines in Development Leukemia & Lymphoma 2013 3 Medicines in Development for Leukemia & Lymphoma Hematological Malignancies Product Name Sponsor Indication Development Phase Jakafi® ruxolitinib Incyte Wilmington, DE hematological malignancies Phase II incyte.com MEDI-551 (anti-CD19 mAb) AstraZeneca Wilmington, DE MedImmune Gaithersburg, MD hematological malignancies Phase II astrazeneca.com medimmune.com MLN4924 (pevonedistat) Millennium Pharmaceuticals Cambridge, MA hematological malignancies Phase I millennium.com MLN8237 (alisertib) Millennium Pharmaceuticals Cambridge, MA hematological malignancies (see also lymphoma) Phase II millennium.com MLN9708 (ixazomib) (second-generation proteasome inhibitor) Millennium Pharmaceuticals Cambridge, MA hematological malignancies (see also myeloma) Phase I millennium.com moxetumomab pasudotox (anti-CD22 recombinant immunotoxin) AstraZeneca Wilmington, DE MedImmune Gaithersburg, MD hematological malignancies Phase I astrazeneca.com medimmune.com nivolumab (anti-PD-1) Bristol-Myers Squibb Princeton, NJ hematological malignancies Phase I bms.com oprozomib Onyx Pharmaceuticals South San Francisco, CA hematological malignancies Phase I onyx.com RG7112 (MDM2 antagonist) Roche Nutley, NJ hematological malignancies Phase I roche.com RG7388 (proto oncogene protein-c mdm2 inhibitor) Roche Nutley, NJ hematological malignancies Phase I roche.com SAR405838 (HDM2/p53 antagonist) Ascenta Therapeutics Malvern, PA Sanofi US Bridgewater, NJ hematological malignancies Phase I ascenta.com sanofi.com SAR650984 (anti-CD38 naked mAb) ImmunoGen Waltham, MA Sanofi US Bridgewater, NJ hematological malignancies Phase I immunogen.com sanofi.com Medicines in Development Leukemia & Lymphoma 20134 Medicines in Development for Leukemia & Lymphoma Leukemia Product Name Sponsor Indication Development Phase ABT-199/GDC-0199/RG7601 (Bcl-2 inhibitor) AbbVie North Chicago, IL Genentech South San Francisco, CA Roche Nutley, NJ relapsed/refractory chronic lymphocytic leukemia (CLL) (see also lymphoma, myeloma) Phase I abbvie.com gene.com roche.com Actimab-A™ lintuzumab Ac-225 Actinium Pharmaceuticals New York, NY acute myeloid leukemia (AML) Phase I/II actiniumpharmaceuticals.com aminopterin Syntrix Biosystems Auburn, WA pediatric acute lymphoblastic leukemia (ALL) Phase II syntrixbio.com Arzerra® ofatumumab (Orphan Drug) GlaxoSmithKline Rsch. Triangle Park, NC first-line CLL, relapsed CLL (see also lymphoma) Phase III gsk.com AT-101 (pan Bcl-2 inhibitor) Ascenta Therapeutics Malvern, PA CLL (see also lymphoma) Phase II completed ascenta.com AT-406 (multi-IAP inhibitor) Ascenta Therapeutics Malvern, PA Debiopharm Lausanne, Switzerland AML Phase I ascenta.com debiopharm.com bafetinib CytRx Los Angeles, CA CLL Phase II completed cytrx.com BI-811283 (aurora B kinase inhibitor) Boehringer Ingelheim Pharmaceuticals Ridgefield, CT AML Phase I/II boehringer-ingelheim.com BI-836826 Boehringer Ingelheim Pharmaceuticals Ridgefield, CT CLL Phase I boehringer-ingelheim.com BI-836858 Boehringer Ingelheim Pharmaceuticals Ridgefield, CT AML Phase I boehringer-ingelheim.com birinapant TetraLogic Pharmaceuticals Malvern, PA AML (see also lymphoma) Phase I/II tetralogicpharma.com Bismab-A™ lintuzumab Bi-213 Actinium Pharmaceuticals New York, NY AML Phase I/II actiniumpharmaceuticals.com Medicines in Development Leukemia & Lymphoma 2013 5 Leukemia Product Name Sponsor Indication Development Phase blinatumomab (AMG 103) Amgen Thousand Oaks, CA ALL (see also lymphoma) Phase II amgen.com BMS-906024 (notch inhibitor) Bristol-Myers Squibb Princeton, NJ T-cell ALL (see also lymphoma) Phase I bms.com BMS-936564 (anti-CXCR4) Bristol-Myers Squibb Princeton, NJ AML, CLL, diffuse large B-cell leukemia (see also lymphoma, myeloma) Phase I bms.com Bosulif® bosutinib Pfizer New York, NY first-line Ph+ chronic myeloid leukemia (CML) Phase III pfizer.com BP-100-1.01 (liposomal Grb-2) (Orphan Drug) Bio-Path Holdings Houston, TX ALL, AML, CML (see also myelodysplastic) Phase I biopathholdings.com calaspargase pegol (Orphan Drug) Sigma-Tau Pharmaceuticals Gaithersburg, MD ALL in adolescents and children Phase III sigmatau.com CC-292 (Btk inhiitor) Celgene Summit, NJ CLL (see also lymphoma) Phase I celgene.com CC-486 (nucleoside analogue) Celgene Summit, NJ post-induction AML maintenance (see also myelodysplastic) Phase II celgene.com Ceplene® histamine EpiCept Tarrytown, NY AML Phase III epicept.com CNDO-109 (tumor-activated natural killer cells) (Orphan Drug) Coronado Biosciences Burlington, MA AML Phase II coronadobiosciences.com CPX-351 (cytarabine/daunorubicin) (Orphan Drug) Celator Pharmaceuticals Princeton, NJ AML Phase III celatorpharma.com crenolanib (CP-868-596) AROG Pharmaceuticals Dallas, TX AML Phase II arogpharma.com CTL019 (CAR T-cell therapy) Novartis Pharmaceuticals East Hanover, NJ University of Pennsylvania Philadelphia, PA CLL Phase II novartis.com CWP232291 JW Pharmaceutical Seoul, South Korea AML Phase I Medicines in Development for Leukemia & Lymphoma Medicines in Development Leukemia & Lymphoma 20136 Leukemia Product Name Sponsor Indication Development Phase Dacogen® decitabine (Orphan Drug) Eisai Woodcliff Lake, NJ AML -------------------------------------- pediatric AML application submitted eisai.com ----------------------------------------- Phase II eisai.com DFP-10917 (cell cycle inhibitor) Delta-Fly Pharma Tokushima, Japan ALL, AML Phase I/II dinaciclib (SOK inhibitor) (Orphan Drug) Merck Whitehouse Station, NJ CLL Phase III merck.com elesclomol Synta Pharmaceuticals Lexington, MA AML Phase I syntapharma.com ENMD-2076 (Orphan Drug) EntreMed Rockville, MD AML (see also myeloma) Phase I completed entremed.com entinostat Syndax Pharmaceuticals Waltham, MA leukemia (see also lymphoma) Phase II syndax.com EPZ-5676 (DOT1L inhibitor) Epizyme Cambridge, MA leukemia Phase I epizyme.com Erwinaze™ asparaginase Erwinia chrysanthemi (IV formulation) Jazz Pharmaceuticals Palo Alto, CA ALL Phase I jazzpharma.com forodesine (PNP inhibitor) (Orphan Drug) BioCryst Pharmaceuticals Durham, NC CLL (see also lymphoma) Phase II biocryst.com GNKG-168 (TLR9 agonist) SBI Biotech Tokyo, Japan CLL Phase I sbibiotech.jp GS-9820 (PI3K delta inhibitor) Gilead Sciences Foster City, CA CLL (see also lymphoma) Phase I gilead.com hTERT RNA vaccine BioTime Alameda, CA AML Phase II biotimeinc.com HuM195/rGel (lintuzumab-gelonin conjugate) Targa Therapeutics San Diego, CA AML, CML (see also myelodysplastic) Phase I targatherapeutics.com ibrutinib (Orphan Drug) Janssen Biotech Horsham, PA Pharmacyclics Sunnyvale, CA CLL (Fast Track) (see also lymphoma, myeloma) Phase III janssenbiotech.com pharmacyclics.com Medicines in Development for Leukemia & Lymphoma Medicines in Development Leukemia & Lymphoma 2013 7 Leukemia Product Name Sponsor Indication Development Phase ibrutinib companion diagnostic Abbott Laboratories Abbott Park, IL Janssen Biotech Horsham, PA Pharmacyclics Sunnyvale, CA CLL (diagnosis) in clinical trials abbott.com janssenbiotech.com pharmacyclics.com Iclusig® ponatinib (Orphan Drug) ARIAD Pharmaceuticals Cambridge, MA newly-diagnosed CML -------------------------------------- AML Phase III ariad.com ----------------------------------------- Phase II ariad.com idelalisib (PI3K delta inhibitor) Gilead Sciences Foster City, CA CLL (see also lymphoma) Phase III gilead.com Imprime PGG® immunotherapeutic Biothera Eagan, MN first-line CLL (see also lymphoma) Phase I/II biothera.com inotuzumab ozogamicin Pfizer New York, NY ALL (see also lymphoma) Phase III pfizer.com Iomab™-B anti-CD45 mAb Actinium Pharmaceuticals New York, NY AML Phase II actiniumpharmaceuticals.com ISF35 (recombinant CD40 ligand immunotherapy) Memgen San Diego, CA CLL (see also lymphoma) Phase II memgenbio.com JVRS-100 (DNA vaccine) Colby Pharmaceutical San Jose, CA leukemia Phase I colbypharma.com KX01 (non-ATP competitive Src kinase inhibitor) Kinex Pharmaceuticals Buffalo, NY AML, CML Phase I kinexpharma.com LDE225 (erismodegib) Novartis Pharmaceuticals East Hanover, NJ CML Phase I novartis.com lestaurtinib (CEP-701) Cephalon (Teva) Horsham, PA AML (see also myeloproliferative) Phase II tevapharm.com lirilumab (anti-Kir) Bristol-Myers Squibb Princeton, NJ AML Phase II bms.com Medicines in Development for Leukemia & Lymphoma Medicines in Development Leukemia & Lymphoma 20138 Leukemia Product Name Sponsor Indication Development Phase LOR-2040 (ribonucleotide reductase inhibitor) Lorus Therapeutics Toronto, Canada AML Phase II lorusthera.com LY2090314 (GSK3-beta inhibitor) Eli Lilly Indianapolis, IN AML, acute promyelocytic leukemia (APL) Phase II lilly.com Marqibo® vincristine liposomal (Orphan Drug) Talon Therapeutics South San Francisco, CA ALL in the elderly (Fast Track) Phase III talontx.com milatuzumab (anti-CD74 mAb) (Orphan Drug) Immunomedics Morris Plains, NJ CLL Phase I/II immunomedics.com MK-8242 (MDM2 inhibitor) Merck Whitehouse Station, NJ AML Phase I merck.com mogamulizumab (anti-CCR4 mAb) (Orphan Drug) Kyowa Hakko Kirin Tokyo, Japan adult T-cell leukemia (see also lymphoma) Phase II kyowa-kirin.com navitoclax (bcl-2 inhibitor) AbbVie North Chicago, IL CLL Phase II abbvie.com obinutuzumab (anti-CD20 mAb) Biogen Idec Weston, MA Genentech South San Francisco, CA Roche Nutley, NJ first-line CLL (see also lymphoma) Phase III biogenidec.com gene.com roche.com OXi4503 (Orphan Drug) OXiGENE South San Francisco, CA refractory AML Phase I oxigene.com perifosine AEterna Zentaris Basking Ridge, NJ CLL Phase II aezsinc.com PF-04449913 Pfizer New York, NY AML Phase I pfizer.com PKC412 (midostaurin) Novartis Pharmaceuticals East Hanover, NJ AML (see also myeloproliferative) Phase III novartis.com Medicines in Development for Leukemia & Lymphoma Medicines in Development Leukemia & Lymphoma 2013 9 Leukemia Product Name Sponsor Indication Development Phase PKC412 (midostaurin) companion diagnostic Invivoscribe Technologies San Diego, CA Novartis Pharmaceuticals East Hanover, NJ AML (diagnosis) in clinical trials invivoscribe.com novartis.com PLX3397 Plexxikon Berkeley, CA AML (see also lymphoma) Phase II plexxikon.com PM01183 (cell-cycle inhibitor) PharmaMar Madrid, Spain leukemia Phase I pharmamar.com PR104 Proacta La Jolla, CA AML Phase I/II proacta.com PRI-724 (CBP/β-catenin inhibitor) Prism Pharma Tokyo, Japan AML, CML Phase I/II prismbiolab.com Promacta® eltrombopag GlaxoSmithKline Rsch. Triangle Park, NC AML (see also myelodysplastic) Phase II gsk.com quizartinib (FLT3 inhibitor) Ambit Biosciences San Diego, CA relapsed/refractory AML -------------------------------------- first-line AML (combination therapy), post-HSCT maintenance in AML Phase II ambitbio.com ----------------------------------------- Phase I ambitbio.com Revlimid® lenalidomide (Orphan Drug) Celgene Summit, NJ CLL (initial treatment and maintenance) (see also lymphoma, myelodysplastic) Phase III celgene.com Revlimid® lenalidomide and Vidaza® azacitidine injection (Orphan Drug) Celgene Summit, NJ AML Phase II celgene.com RG7356 (anti-CD44 mAb) Roche Nutley, NJ AML Phase I roche.com sapacitabine (Orphan Drug) Cyclacel Pharmaceuticals Berkeley Heights, NJ AML (see also myelodysplastic) -------------------------------------- CLL Phase III cyclacel.com ----------------------------------------- Phase II cyclacel.com SAR3419 (maytansin-loaded anti-CD19 mAb) ImmunoGen Waltham, MA Sanofi US Bridgewater, MA ALL (see also lymphoma) Phase II immunogen.com sanofi.com Medicines in Development for Leukemia & Lymphoma Medicines in Development Leukemia & Lymphoma 201310 Leukemia Product Name Sponsor Indication Development Phase SGI110 (DNMT1 inhibitor) Astex Pharmaceuticals Dublin, CA AML (see also myelodysplastic) Phase II astx.com SGN-CD19A (CD19 antibody-drug conjugate) Seattle Genetics Bothell, WA ALL (see also lymphoma) Phase I seattlegenetics.com SL-401 (recombinant fusion protein) (Orphan Drug) Stemline Therapeutics New York, NY relapsed/refractory AML, relapsed/ refractory blastic plasmacytoid dendritic cell neoplasm (see also myelodysplastic) Phase I/II stemline.com Sprycel dasatinib Bristol-Myers Squibb Princeton, NJ leukemia in children and adolescents Phase II bms.com tamibarotene (Orphan Drug) CytRx Los Angeles, CA acute promyelocytic leukemia (Fast Track) Phase II cytrx.com Tasigna® nilotinib Novartis Pharmaceuticals East Hanover, NJ ALL (adolescents and children), CML (adolescents and children) Phase II novartis.com TCN-P (AKT inhibitor) Cahaba Pharmaceuticals Princeton, NJ AML Phase I cahabapharma.com Temodar® temozolomide Merck Whitehouse Station, NJ AML Phase II completed merck.com tetradecanoylphorbol acetate (TPA) (PD-616) Biosuccess Biotech Beverly Hills, CA AML Phase I/II biosuccessbiotech.com TG02 (multi-kinase inhibitor) Tragara Pharmaceuticals Carlsbad, CA acute leukemia, CLL (see also myeloma) Phase I tragarapharma.com TH-302 (hypoxia-targeted drug) EMD Serono Rockland, MA Threshold Pharmaceuticals South San Francisco, CA advanced leukemia (see also myeloma) Phase I emdserono.com thresholdpharm.com tosedostat Cell Therapeutics Seattle, WA Chroma Therapeutics Oxon, United Kingdom AML Phase II celltherapeutics.com Treanda® bendamustine Cephalon (Teva) Frazer, PA ALL, AML (see also lymphoma, myeloma) Phase I/II tevapharm.com TRU-016 (anti-CD37 mAb) (Orphan Drug) Emergent BioSolutions Rockville, MD CLL Phase II emergentbiosolutions.com Medicines in Development for Leukemia & Lymphoma Medicines in Development Leukemia & Lymphoma 2013 11 Leukemia Product Name Sponsor Indication Development Phase ublituximab (TG-1101) (Orphan Drug) TG Therapeutics New York, NY CLL (see also lymphoma) Phase I/II tgtherapeutics.com VAY736 (anti-BAFFR mAb) MorphoSys Martinsried, Germany Novartis Pharmaceuticals East Hanover, NJ CLL Phase I morphosys.com novartis.com veltuzumab (Orphan Drug) Immunomedics Morris Plains, NJ CLL (see also lymphoma) Phase II immunomedics.com volasertib Boehringer Ingelheim Pharmaceuticals Ridgefield, CT AML Phase III boehringer-ingelheim.com vosaroxin Sunesis Pharmaceuticals South San Francisco, CA AML Phase III sunesis.com WT1 immunotherapeutic (antigen-specific cancer immunotherapeutic/ASCI) GlaxoSmithKline Rsch. Triangle Park, NC AML Phase II gsk.com XmAb® 5574/MOR208 (anti-CD19 mAb) MorphoSys Martinsried, Germany Xenvor Monrovia, CA CLL Phase I morphosys.com xencor.com Lymphoma Product Name Sponsor Indication Development Phase abexinostat (HDAC inhibitor) Pharmacyclics Sunnyvale, CA follicular lymphoma, mantle cell lymphoma Phase II pharmacyclics.com ABT-199/GDC-0199/RG7601 (Bcl-2 inhibitor) AbbVie North Chicago, IL Genentech South San Francisco, CA Roche Nutley, NJ non-Hodgkin lymphoma (see also leukemia, myeloma) Phase I abbvie.com gene.com roche.com Medicines in Development for Leukemia & Lymphoma Medicines in Development Leukemia & Lymphoma 201312 Medicines in Development for Leukemia & Lymphoma Lymphoma Product Name Sponsor Indication Development Phase Adcetris® brentuximab vedotin (Orphan Drug) Millennium Pharmaceuticals Cambridge, MA Seattle Genetics Bothell, WA Hodgkin lymphoma (post-transplant relapse prevention), relapsed CD30-positive cutaneous T-cell lymphoma, first-line Hodgkin lymphoma, first-line CD30-positive mature T-cell lymphoma (see also hematological) -------------------------------------- relapsed/refractory CD30-positive non-Hodgkin lymphoma, CD30- positive lymphoma malignancies, first-line H