2013 REPORT More Than 240 Medicines in Development for Leukemia, Lymphoma and Other Blood Cancers Every 4 minutes a person is diagnosed with leukemia, Medicines in Development lymphoma or For Leading Blood Cancers myeloma; Accounting for Application Submitted 9% of all cancers Phase III diagnosed each year Phase II Biopharmaceutical research companies • 15 each for myeloproliferative neo- Phase I are developing 241 medicines for blood plasms, such as myelofibrosis, poly- cancers—leukemia, lymphoma and cythemia vera and essential throm- 97 98 myeloma. This report lists medicines in bocythemia; and for myelodysplastic human clinical trials or under review by syndromes, which are diseases affect- the U.S. Food and Drug Administration ing the blood and bone marrow. (FDA). These medicines in development offer The medicines in development include: hope for greater survival for the thou- sands of Americans who are affected by • 98 for lymphoma, including Hodgkin these cancers of the blood. and non-Hodgkin lymphoma, which 52 affect nearly 80,000 Americans each Definitions for the cancers listed in this year. report and other terms can be found on page 27. Links to sponsor company web • 97 for leukemia, including the four sites provide more information on the major types, which affect nearly potential products. 50,000 people in the United States 24 each year. For information on the value of medi- cines, an in-depth look at current in- • 52 for myeloma, a cancer of the novation and key medical breakthroughs plasma cells, which impacts more benefiting blood cancer patients, please than 22,000 people each year in the see Medicines in Development for Leu- United States. kemia and Lymphoma 2013—Overview. emia • 24 medicines are targeting hema- tological malignancies, which affect Leuk ymphoma Myeloma L bone marrow, blood and lymph Malignancies Hematological nodes. Medicines in Development for Leukemia & Lymphoma Hematological Malignancies Product Name Sponsor Indication Development Phase* Adcetris® Millennium Pharmaceuticals treatment of CD30-positive Phase II brentuximab vedotin Cambridge, MA hematological malignancies millennium.com (Orphan Drug) Seattle Genetics (see also lymphoma) seattlegenetics.com Bothell, WA AMG 319 Amgen hematological malignancies Phase I (PI3K-delta inhibitor) Thousand Oaks, CA amgen.com anti-CD22 antibody-drug Genentech hematological malignancies Phase II conjugate South San Francisco, CA (see also lymphoma) gene.com (DCDT 2980S) anti-CD79b antibody-drug Genentech hematological malignancies Phase II conjugate South San Francisco, CA (see also lymphoma) gene.com (DCDS 4501A) AR-42 Arno Therapeutics hematological malignancies Phase I/II (pan-DAC inhibitor) Flemington, NJ arnothera.com AZD1208 AstraZeneca hematological malignancies Phase I (PIM kinase inhibitor) Wilmington, DE astrazeneca.com AZD9150 AstraZeneca hematological malignancies Phase I (STAT3 inhibitor) Wilmington, DE astrazeneca.com belinostat Spectrum Pharmaceuticals hematological malignancies Phase I (HDAC inhibitor) Henderson, NV (see also lymphoma) sppirx.com CB3304 Cougar Biotechnology hematological malignancies Phase I (noscapine) Raritan, NJ cougarbiotechnology.com darinaparsin ZIOPHARM Oncology hematological malignancies Phase II (ZIO-101) New York, NY (see also lymphoma, myeloma) ziopharm.com fostamatinib AstraZeneca hematological malignancies Phase II (Syk inhibitor) Wilmington, DE astrazeneca.com Rigel Pharmaceuticals rigel.com South San Francisco, CA GS-9973 Gilead Sciences B-cell hematological malignancies Phase I (Syk inhibitor) Foster City, CA gilead.com *For more information about a specific medicine or company in the report, please click on the provided link. 2 Medicines in Development Leukemia & Lymphoma 2013 Medicines in Development for Leukemia & Lymphoma Hematological Malignancies Product Name Sponsor Indication Development Phase Jakafi® Incyte hematological malignancies Phase II ruxolitinib Wilmington, DE incyte.com MEDI-551 AstraZeneca hematological malignancies Phase II (anti-CD19 mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MD MLN4924 Millennium Pharmaceuticals hematological malignancies Phase I (pevonedistat) Cambridge, MA millennium.com MLN8237 Millennium Pharmaceuticals hematological malignancies Phase II (alisertib) Cambridge, MA (see also lymphoma) millennium.com MLN9708 Millennium Pharmaceuticals hematological malignancies Phase I (ixazomib) Cambridge, MA (see also myeloma) millennium.com (second-generation proteasome inhibitor) moxetumomab pasudotox AstraZeneca hematological malignancies Phase I (anti-CD22 recombinant Wilmington, DE astrazeneca.com immunotoxin) MedImmune medimmune.com Gaithersburg, MD nivolumab Bristol-Myers Squibb hematological malignancies Phase I (anti-PD-1) Princeton, NJ bms.com oprozomib Onyx Pharmaceuticals hematological malignancies Phase I South San Francisco, CA onyx.com RG7112 Roche hematological malignancies Phase I (MDM2 antagonist) Nutley, NJ roche.com RG7388 Roche hematological malignancies Phase I (proto oncogene protein-c mdm2 Nutley, NJ roche.com inhibitor) SAR405838 Ascenta Therapeutics hematological malignancies Phase I (HDM2/p53 antagonist) Malvern, PA ascenta.com Sanofi US sanofi.com Bridgewater, NJ SAR650984 ImmunoGen hematological malignancies Phase I (anti-CD38 naked mAb) Waltham, MA immunogen.com Sanofi US sanofi.com Bridgewater, NJ Medicines in Development Leukemia & Lymphoma 2013 3 Medicines in Development for Leukemia & Lymphoma Leukemia Product Name Sponsor Indication Development Phase ABT-199/GDC-0199/RG7601 AbbVie relapsed/refractory chronic Phase I (Bcl-2 inhibitor) North Chicago, IL lymphocytic leukemia (CLL) abbvie.com Genentech (see also lymphoma, myeloma) gene.com South San Francisco, CA roche.com Roche Nutley, NJ Actimab-A™ Actinium Pharmaceuticals acute myeloid leukemia (AML) Phase I/II lintuzumab Ac-225 New York, NY actiniumpharmaceuticals.com aminopterin Syntrix Biosystems pediatric acute lymphoblastic Phase II Auburn, WA leukemia (ALL) syntrixbio.com Arzerra® GlaxoSmithKline first-line CLL, relapsed CLL Phase III ofatumumab Rsch. Triangle Park, NC (see also lymphoma) gsk.com (Orphan Drug) AT-101 Ascenta Therapeutics CLL Phase II completed (pan Bcl-2 inhibitor) Malvern, PA (see also lymphoma) ascenta.com AT-406 Ascenta Therapeutics AML Phase I (multi-IAP inhibitor) Malvern, PA ascenta.com Debiopharm debiopharm.com Lausanne, Switzerland bafetinib CytRx CLL Phase II completed Los Angeles, CA cytrx.com BI-811283 Boehringer Ingelheim Pharmaceuticals AML Phase I/II (aurora B kinase inhibitor) Ridgefield, CT boehringer-ingelheim.com BI-836826 Boehringer Ingelheim Pharmaceuticals CLL Phase I Ridgefield, CT boehringer-ingelheim.com BI-836858 Boehringer Ingelheim Pharmaceuticals AML Phase I Ridgefield, CT boehringer-ingelheim.com birinapant TetraLogic Pharmaceuticals AML Phase I/II Malvern, PA (see also lymphoma) tetralogicpharma.com Bismab-A™ Actinium Pharmaceuticals AML Phase I/II lintuzumab Bi-213 New York, NY actiniumpharmaceuticals.com 4 Medicines in Development Leukemia & Lymphoma 2013 Medicines in Development for Leukemia & Lymphoma Leukemia Product Name Sponsor Indication Development Phase blinatumomab Amgen ALL Phase II (AMG 103) Thousand Oaks, CA (see also lymphoma) amgen.com BMS-906024 Bristol-Myers Squibb T-cell ALL Phase I (notch inhibitor) Princeton, NJ (see also lymphoma) bms.com BMS-936564 Bristol-Myers Squibb AML, CLL, diffuse large Phase I (anti-CXCR4) Princeton, NJ B-cell leukemia bms.com (see also lymphoma, myeloma) Bosulif® Pfizer first-line Ph+ chronic myeloid Phase III bosutinib New York, NY leukemia (CML) pfizer.com BP-100-1.01 Bio-Path Holdings ALL, AML, CML Phase I (liposomal Grb-2) Houston, TX (see also myelodysplastic) biopathholdings.com (Orphan Drug) calaspargase pegol Sigma-Tau Pharmaceuticals ALL in adolescents and children Phase III (Orphan Drug) Gaithersburg, MD sigmatau.com CC-292 Celgene CLL Phase I (Btk inhiitor) Summit, NJ (see also lymphoma) celgene.com CC-486 Celgene post-induction AML maintenance Phase II (nucleoside analogue) Summit, NJ (see also myelodysplastic) celgene.com Ceplene® EpiCept AML Phase III histamine Tarrytown, NY epicept.com CNDO-109 Coronado Biosciences AML Phase II (tumor-activated natural Burlington, MA coronadobiosciences.com killer cells) (Orphan Drug) CPX-351 Celator Pharmaceuticals AML Phase III (cytarabine/daunorubicin) Princeton, NJ celatorpharma.com (Orphan Drug) crenolanib AROG Pharmaceuticals AML Phase II (CP-868-596) Dallas, TX arogpharma.com CTL019 Novartis Pharmaceuticals CLL Phase II (CAR T-cell therapy) East Hanover, NJ novartis.com University of Pennsylvania Philadelphia, PA CWP232291 JW Pharmaceutical AML Phase I Seoul, South Korea Medicines in Development Leukemia & Lymphoma 2013 5 Medicines in Development for Leukemia & Lymphoma Leukemia Product Name Sponsor Indication Development Phase Dacogen® Eisai AML application submitted decitabine Woodcliff Lake, NJ eisai.com (Orphan Drug) -------------------------------------- ----------------------------------------- pediatric AML Phase II eisai.com DFP-10917 Delta-Fly Pharma ALL, AML Phase I/II (cell cycle inhibitor) Tokushima, Japan dinaciclib Merck CLL Phase III (SOK inhibitor) Whitehouse Station, NJ merck.com (Orphan Drug) elesclomol Synta Pharmaceuticals AML Phase I Lexington, MA syntapharma.com ENMD-2076 EntreMed AML Phase I completed (Orphan Drug) Rockville, MD (see also myeloma) entremed.com entinostat Syndax Pharmaceuticals leukemia Phase II Waltham, MA (see also lymphoma) syndax.com EPZ-5676 Epizyme leukemia