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64 HemOnc today | NOVEMBER 25, 2014 | Healio.com/HemOnc

Hematology Drugs in the Pipeline

HemOnc Today presents the most recent information about hematology drugs in the pipeline. Drugs listed here are in phase 2 or phase 3 development for a variety of indications. Clinicians can use this chart as a quick reference to learn about the status of those drugs that may be clinically significant to their practice. To view the entire chart online, go to www.healio.com/hematologypipeline.

Generic name (Brand name, Manufacturer) Indication(s) Development status

12-O-tetradecanoylphorbol-13-acetate (BioSuccess Biotech) acute myelogenous phase 2

A6 peptide (Angstrom Pharmaceuticals) chronic lymphocytic leukemia, small lymphocytic leukemia phase 2

AAV8-hFIX19 (Spark Therapeutics) hemophilia B phase 2

(Lilly) mantle cell lymphoma phase 2

abexinostat (PCI-24781; Pharmacyclics) follicular lymphoma, mantle cell lymphoma phase 2

ABT-199 (AbbVie) acute myelogenous leukemia phase 2 READ PERSPECTIVE on this drug from Jennifer R. chronic lymphocytic leukemia phase 3 Brown, MD, PhD, on page 71.

ACY-1215 (Acetylon) myeloma phase 2

Ad-ISF35 (Memgen) chronic lymphocytic leukemia, diffuse large-cell lymphoma, follicular lym- phase 2 phoma, mantle cell lymphoma, non-Hodgkin’s lymphoma, small lymphocytic lymphoma adeno-associated virus serotype 8 Factor IX gene therapy hemophilia B phase 2 (Asklepios Biopharmaceutical) adeno-associated virus vector, gene therapy (UniQure) hemophilia B phase 2

ADI-PEG 20 (Polaris Pharmaceuticals) acute myelogenous leukemia, non-Hodgkin’s lymphoma phase 2

AEB071 (Novartis) diffuse large B-cell lymphoma phase 2

Aes-103 (AesRx) sickle cell disease phase 2

AFM 13 (Affirmed Therapeutics) Hodgkin’s lymphoma phase 2

(Campath, Ilex Pharmaceuticals) sickle cell disease, T-large granular lymphocyte leukemia phase 2

alisertib (Millennium) peripheral T-cell lymphoma phase 3

Allostim (Immunovative Therapies) leukemia, lymphoma, myeloma phase 2

ALT-803 (Altor Bioscience) hematologic malignancies, myeloma phase 2

(Tolero Pharmaceuticals) acute myelogenous leukemia (relapsed/refractory) phase 2

acute myelogenous leukemia (first-line) phase 3 ambrisentan (Letairis, GlaxoSmithKline) chronic thromboembolic pulmonary hypertension phase 3

AME-133v (Mentrik Biotech) CD20-positive non-Hodgkin’s lymphoma phase 3

AP1903 (Bellicum/Ariad) acute lymphoblastic leukemia, acute myelogenous leukemia, lymphoma phase 2

APTO-253 hematologic malignancies (relapsed/refractory) phase 2

ART-123 (Asahi Kasei Pharma) coagulopathy phase 3

ASP0113 (Astellas) cytomegalovirus reactivation in hematopoietic cell transplant recipients phase 3

ASP1517 (Astellas) anemia in chronic kidney disease patients not on dialysis phase 2

ASP2215 (Astellas) acute myelogenous leukemia phase 2

Erwinia chrysanthemi (Erwinaze, Jazz Pharmaceuticals) acute lymphoblastic leukemia (patients with hypersensitivity to first-line phase 2 asparaginase therapy) AT-101 (Ascenta Therapeutics) chronic lymphocytic leukemia phase 2

AT7519 (Astex/Novartis) myeloma phase 2

ATIR (Kiadis Pharma) haploidentical stem cell transplantation with rapid immune reconstitution phase 2 without severe graft-versus-host-disease (E5501, Eisai) thrombocytopenia during interferon therapy phase 2

thrombocytopenia in chronic liver disease phase 3 (Vidaza, Celgene) acute myelogenous leukemia, phase 3 HemOnc today | NOVEMBER 25, 2014 | Healio.com/HemOnc 65

Hematology Drugs in the Pipeline

Generic name (Brand name, Manufacturer) Indication(s) Development status

BAX 855 (Baxter Healthcare) hemophilia A phase 3

BAY 94-9027 (Bayer) hemophilia A phase 3

BB305 (LentiGlobin, Bluebird Bio) beta thalassemia, sickle cell disease phase 2

(Treanda, Teva) mantle cell lymphoma (relapsed/refractory) phase 2

mantle cell lymphoma, non-Hodgkin’s lymphoma phase 3 BGJ398 (Novartis) hematologic malignancies phase 2

BI 811283 (Boehringer Ingelheim) acute myelogenous leukemia phase 2

BiovaxID (Biovest International) follicular non-Hodgkin’s lymphoma, mantle cell lymphoma, other B-cell phase 3 malignancies birinapant (TL32711, TetraLogic Pharmaceuticals) myelodysplastic syndrome phase 2

Bismab-A (Actinium Pharmaceuticals) acute myelogenous leukemia phase 2

BL-8040 (BioLineRx) acute myelogenous leukemia phase 2

(AMG 103, Amgen) diffuse large B-cell lymphoma, non-Hodgkin’s lymphoma phase 2

acute myelogenous leukemia phase 3 (Velcade, Millennium) diffuse large B-cell lymphoma phase 2

mantle cell lymphoma phase 3 bosutinib (Bosulif, Pfizer) Philadelphia chromosome-positive chronic myeloid leukemia phase 3

BPX-501 (Bellicum) acute lymphoblastic leukemia, acute myelogenous leukemia, bone marrow phase 2 transplantation in hematologic malignancies, lymphoma (Adcetris; Seattle Genetics, Millennium) CD30-positive non-Hodgkin’s lymphoma (relapsed/refractory), CD30-positive phase 2 non-lymphoma malignancies, diffuse large B-cell lymphoma (front-line), Hodgkin’s lymphoma in patients aged 60 years or older (front-line)

cutaneous T-cell lymphoma, Hodgkin’s lymphoma (front-line and post-trans- phase 3 plant), CD30-positive mature T-cell lymphoma BT-062 (ImmunoGen) myeloma phase 2

calaspargase pegol (EZN-2285, Sigma-Tau Pharmaceuticals) acute lymphoblastic leukemia phase 3

captisol-enabled (Spectrum Pharmaceuticals) myeloma prior to autologous stem cell transplant phase 2

(Kyprolis, Amgen/Onyx) myeloma phase 3

CC-486 (Celgene) acute myelogenous leukemia (post-induction maintenance), myelodysplastic phase 3 syndrome cerdulatinib (Portola Pharmaceuticals) chronic lymphocytic leukemia, non-Hodgkin’s lymphoma phase 2

CLT-008 (Cellerant Therapeutics) acute myelogenous leukemia phase 2

CMD-003 (Cell Medica) Epstein-Barr virus lymphoma phase 2

CNDO-109 (Coronado Biosciences) acute myelogenous leukemia, myeloma phase 2

Factor VIIa, recombinant (rEVO Biologics) hemophilia A, hemophilia B phase 3

(BAY 80-6946, Bayer) non-Hodgkin’s lymphoma phase 2

crenolanib (Arog Pharmaceuticals) acute myelogenous leukemia with FLT3 mutations (relapsed/refractory) phase 2

CSL654 rIX-FP (CSL Behring) hemophilia phase 3

CTL019 (Novartis) acute lymphoblastic leukemia phase 2

: (CPX-351, Celator Pharmaceuticals) acute lymphoblastic leukemia, myelodysplastic syndrome phase 2

acute myelogenous leukemia phase 3 (BAY 94-9027, Bayer) hemophilia phase 3

(HuMax-CD38; Genmab, Janssen) myeloma (relapsed/refractory) phase 3

darbepoetin alfa (Aranesp, Amgen) myelodysplastic syndrome phase 3 66 HemOnc today | NOVEMBER 25, 2014 | Healio.com/HemOnc

Hematology Drugs in the Pipeline

Generic name (Brand name, Manufacturer) Indication(s) Development status

neutropenia, sickle cell disease phase 2

deferasirox (Exjade, Novartis) chronic iron overload due to transfusion-dependent anemias, transfusion- phase 2 dependent beta-thalassemia denosumab (Xgeva, Amgen) cancer-related bone damage in patients with myeloma phase 3

DFP-10917 (Delta-Fly Pharma) acute lymphoblastic leukemia, acute myelogenous leukemia phase 2

DKN-01 (HealthCare Pharmaceuticals) myeloma phase 2

(IPI-145, Infinity Pharmaceuticals) non-Hodgkin’s lymphoma phase 2

chronic lymphocytic leukemia, follicular lymphoma phase 3 (HuLuc63; Bristol-Myers Squibb, AbbVie) myeloma phase 3

(Promacta/Revolade, GlaxoSmithKline) acute myelogenous leukemia, aplastic anemia, myelodysplastic syndrome phase 2

(SNDX-275, Syndax) acute myelogenous leukemia, Hodgkin’s lymphoma phase 2

EP-100 (Esperance Pharmaceuticals) hematologic malignancies phase 2

epoetin alfa neonatal anemia phase 2

epratuzumab (Immunomedics) leukemia, lymphoma phase 2

EPZ-6438 (Epizyme) non-Hodgkin’s lymphoma phase 2

everolimus (Afinitor, Novartis) diffuse large B-cell lymphoma phase 2

ezatiostat HCI (Telintra, Telik) myelodysplastic syndrome, severe chronic idiopathic neutropenia phase 2

fenretinide intravenous (CerRx) lymphoma (adult) phase 2

ferroportin (LY2928057, Lilly) anemia phase 2

FF-10501 (Boston Strategics/FujiFilm Pharmaceuticals) hematologic malignancies phase 2

filanesib (ARRY-520, Array Biopharma) myeloma (relapsed/refractory) phase 2 READ PERSPECTIVE on this drug from Cristina Gasparetto, MD, on page 72.

(Mundipharma) peripheral T-cell lymphoma (recurrent/refractory) phase 2

(R935788, Rigel) persistent/chronic immune thrombocytopenic purpura phase 3

FPI-01 (Formula Pharmaceuticals) acute lymphoblastic leukemia, acute myelogenous leukemia phase 2

gabapentin sickle cell disease phase 2

galunisertib (LY2157299, Lilly) myelodysplastic syndrome phase 2

ganetespib (Synta Pharmaceuticals) acute myelogenous leukemia phase 2

(Mylotarg, Wyeth) acute promyelocytic leukemia phase 2

acute myelogenous leukemia phase 3 GreenGene F (Green Cross Corporation) hemophilia A phase 3

GS-9973 (Gilead) chronic lymphocytic leukemia, diffuse large B-cell lymphoma, follicular phase 2 lymphoma, mantle cell lymphoma, non-Hodgkin’s lymphoma GSK1278863 (GlaxoSmithKline) anemia phase 2

GSK2696273 (GlaxoSmithKline) severe combined immune deficiency phase 3

GSK2696274 (GlaxoSmithKline) Wiskott-Aldrich syndrome phase 2

HSC835 (Novartis) hematologic malignancies, single umbilical cord transplant phase 2

HSV-Tk (MolMed) acute leukemia (high risk) phase 3

human-cl rh Factor VIII (Octapharma) severe hemophilia A phase 3 HemOnc today | NOVEMBER 25, 2014 | Healio.com/HemOnc 67

Hematology Drugs in the Pipeline

Generic name (Brand name, Manufacturer) Indication(s) Development status

sickle cell disease phase 3

ibrutinib (Imbruvica; Pharmacyclics, Janssen) follicular lymphoma, myeloma, Waldenstrom’s macroglobulinemia phase 2

chronic lymphocytic leukemia (combination treatment in relapsed/refractory phase 3 patients, single-agent treatment in treatment-naive patients), diffuse large B-cell lymphoma, mantle cell lymphoma (combination treatment in treat- ment-naive patients, single-agent treatment in relapsed/refractory patients), Waldenstrom’s macroglobulinemia (combination treatment in previously treated adults) (Zydelig, Gilead) chronic lymphocytic leukemia (front-line), indolent non-Hodgkin’s lymphoma phase 3

imetelstat (Geron) essential thrombocythemia, myelofibrosis, polycythemia vera phase 2

IMO-8400 (Idera Pharmaceuticals) Waldenstrom’s macroglobulinemia phase 2

Imprime PGG (Biothera) chronic lymphocytic leukemia, indolent B-cell non-Hodgkin’s lymphomas phase 2 (relapsed/refractory) (Pfizer) acute lymphoblastic leukemia phase 3

interleukin-12 gene therapy (OncoSec) cutaneous T-cell lymphoma phase 2

Iomab-B (Actinium Pharmaceuticals) hematopoietic stem cell transplantation in acute lymphoblastic leukemia phase 2

ISIS-STAT3Rx (Isis Pharmaceuticals/AstraZeneca) diffuse large-B cell lymphoma phase 2

(MLN9708, Millennium) follicular lymphoma phase 2

amyloidosis, myeloma (relapsed/refractory) phase 3 JAK2 inhibitor (LY2784544, Lilly) myeloproliferative neoplasms phase 2

JCAR014 (Juno Therapeutics) acute lymphoblastic leukemia, chronic lymphocytic leukemia (refractory), phase 2 non-Hodgkin’s lymphoma JCAR017 (Juno Therapeutics) leukemia phase 2

JNJ-26481585 (Janssen) cutaneous T-cell lymphoma phase 2

JNJ-40346527 (Janssen) Hodgkin’s lymphoma phase 2

KB004 (KaloBios Pharmaceuticals) hematologic malignancies phase 2

KPT-330 (Karyopharm Therapeutics) acute myelogenous leukemia (elderly patients, second-line) phase 2

KTE-C19 CAR (Kite Pharma) diffuse large B-cell lymphoma phase 2

lenalidomide (Revlimid, Celgene) diffuse large B-cell lymphoma (first-line) phase 2

chronic lymphocytic leukemia (maintenance, second-line), diffuse large phase 3 B-cell lymphoma (maintenance), follicular lymphoma, indolent lymphoma, myeloma (maintenance), myelodysplastic syndrome LGH447 (Novartis) myeloma phase 2

lipegfilgrastim (Lonquex, Teva) elderly patients with aggressive B-cell non-Hodgkin’s lymphoma at high risk phase 3 for R-CHOP21–induced neutropenia Ac-225 (Actinium Pharmaceuticals) acute myelogenous leukemia (first-line) phase 2

luspatercept (ACE-536; Celgene, Acceleron) sickle cell anemia phase 2

losartan anemia, beta-thalassemia, myelodysplastic syndrome phase 2

marizomib (Triphase Accelerator) myeloma phase 2

MDGN-201 (Medgenics) renal anemia phase 2

MEDI-551 (AstraZeneca) chronic lymphocytic leukemia, diffuse large B-cell lymphoma phase 2

midostaurin (PKC412, Novartis) acute lymphoblastic leukemia, acute myelogenous leukemia phase 2

- (Immunomedics) chronic lymphocytic leukemia, non-Hodgkin’s lymphoma phase 2

mocetinostat (Mirati Therapeutics) diffuse large B-cell lymphoma, myelodysplastic syndrome phase 2

mogamulizumab injection (KW-0761, Kyowa Hakko Kirin) adult T-cell leukemia, adult T-cell lymphoma, cutaneous T-cell lymphoma phase 2 68 HemOnc today | NOVEMBER 25, 2014 | Healio.com/HemOnc

Hematology Drugs in the Pipeline

Generic name (Brand name, Manufacturer) Indication(s) Development status

molidustat (BAY 85-3934, Bayer) anemia phase 2

momelotinib (Gilead) myelofibrosis phase 3 READ PERSPECTIVE on this drug from Ruben A. Mesa, MD, on page 71.

montelukast sickle cell anemia phase 2

MOR208 (MorphoSys) acute lymphoblastic leukemia, chronic lymphocytic leukemia, non-Hodgkin’s phase 2 lymphoma (Pfizer) hemophilia A phase 3

(AstraZeneca) hairy cell leukemia phase 3

N-Acetylcysteine sickle cell disease phase 3

NiCord (Gamida Cell) acute lymphoblastic leukemia, acute myelogenous leukemia, myelodysplas- phase 2 tic syndrome, sickle cell disease

hematologic malignancies phase 3 nilotinib (Tasigna, Novartis) chronic myeloid leukemia (treatment-free remission study) phase 3

(Opdivo, Bristol-Myers Squibb) diffuse large B-cell lymphoma, follicular lymphoma, Hodgkin’s lymphoma, phase 2 READ PERSPECTIVE non-Hodgkin’s lymphoma on this drug from Jonathon B. Cohen, MD, MS, on page 71.

NNC 0129-0000-1003 (Novo Nordisk) hemophilia A phase 3

nonacog beta pegol (N9-GP, Novo Nordisk) hemophilia B phase 3

non-Hodgkin’s lymphoma vaccine (XEME Biopharma) non-Hodgkin’s follicular lymphoma phase 2

NY-ESO-1/LAGE-1-specific T cells (Adaptimmune) myeloma phase 2

(Gazyva, Genentech/Roche) diffuse large B-cell lymphoma, indolent non-Hodgkin’s lymphoma (front-line phase 3 and refractory), chronic lymphocytic leukemia (front-line) (Arzerra; GlaxoSmithKline, Genmab) Waldenstrom’s macroglobulinemia phase 2

chronic lymphocytic leukemia (relapsed), diffuse large B-cell lymphoma phase 3 (relapsed), follicular lymphoma (relapsed and refractory) omacetaxine mepesuccinate (Synribo, Teva) chronic myeloid leukemia phase 2

OMS-1120 (Oncosec) cutaneous T-cell lymphoma phase 2

(Amgen/Onyx) myeloma, Waldenstrom’s macroglobulinemia phase 2

(TRU-016, Emergent Biosolutions) chronic lymphocytic leukemia phase 2

pacritinib (SB1518: CTI Biopharma, Baxter) acute myelogenous leukemia (relapsed) phase 2 READ PERSPECTIVE on this drug from Ruben A. myelodysplastic syndrome phase 3 Mesa, MD, on page 71.

READ PERSPECTIVE on this drug from Srikanth Nagalla, MBBS, MS, FACP, on page 72.

(LBH589, Novartis) acute myelogenous leukemia, diffuse large B-cell lymphoma, Hodgkin’s phase 2 lymphoma, myelodysplastic syndrome, myelofibrosis, myeloma, non-Hodgkin’s lymphoma pegylated recombinant Factor VIII, long-acting (Baxter) hemophilia phase 3

PF-04449913 (Pfizer) acute myelogenous leukemia phase 2

pidilizumab (CureTech) myeloma phase 2

(DCDT2980S/RG7593; Genentech) diffuse large B-cell lymphoma, non-Hodgkin’s lymphoma phase 2

(Pixuvri, CTI Biopharma) diffuse large B-cell lymphoma, non-Hodgkin’s lymphoma phase 3

plasma-derived AT-III concentrate (Grifols Biologicals) antithrombin III deficiency phase 3

plerixafor (Mozobil, Genzyme) major sickle cell syndrome phase 2

plitidepsin (Aplidin, PharmaMar) myeloma (combination treatment), T-cell lymphoma phase 2

myeloma (single-agent treatment) phase 3 HemOnc today | NOVEMBER 25, 2014 | Healio.com/HemOnc 69

Hematology Drugs in the Pipeline

Generic name (Brand name, Manufacturer) Indication(s) Development status

PLX3397 (Plexxikon) acute myelogenous leukemia phase 2

PMO1183 (PharmaMar) acute leukemia phase 2

PNT2258 (ProNAi) non-Hodgkin’s lymphoma phase 2

(DCDS4501A/RG7596; Genentech/Roche) diffuse large B-cell lymphoma, non-Hodgkin’s lymphoma phase 2

polyethylene glycol recombinant adenosine deaminase (EZN-2279, enzyme replacement therapy for adenosine deaminase deficiency in patients phase 3 Sigma-Tau Pharmaceuticals) with severe combined immunodeficiency disease who are not candidates for or have failed bone marrow transplantation ponatinib (Ariad) acute myelogenous leukemia, Philadelphia chromosome-positive acute phase 2 lymphoblastic leukemia pracinostat (MEI Pharma) acute myelogenous leukemia, myelodysplastic syndrome phase 2

injection (Folotyn, Spectrum Pharmaceuticals) peripheral T-cell lymphoma (front-line) phase 2

prasugrel (Effient; Daiichi Sankyo, Lilly) sickle cell disease phase 3

PRI-724 (Prism Pharma) acute myelogenous leukemia, chronic myeloid leukemia phase 2

Prochymal (Osiris Therapeutics) graft-versus-host disease after hematopoietic stem cell transplant phase 3

PVX-410 (OncoPep) smoldering myeloma phase 2

quizartinib (Ambit Biosciences) acute myelogenous leukemia phase 3

recombinant Factor VIII (CSL Behring) hemophilia A phase 3

recombinant Factor VIII (BAY81-8973, Bayer) hemophilia A phase 3

recombinant Factor IX (BAX 326, Baxter) hemophilia B phase 3

recombinant Factor IX albumin fusion protein (CSL Behring) hemophilia B phase 3

recombinant Factor IX Fc protein (BIIB 029, Biogen Idec) hemophilia B phase 3

recombinant protein-free Factor VIII (Baxter Healthcare) hemophilia A phase 3

regadenoson (Lexiscan, Astellas) sickle cell anemia phase 2

RG6013 (Roche/Chugai) hemophilia A phase 2

RG7601 (Roche/AbbVie/WEHI) chronic lymphocytic leukemia (relapsed/refractory with 17p deletion), diffuse phase 2 large B-cell lymphoma (front-line)

chronic lymphocytic leukemia (relapsed/refractory) in combination with phase 3 MabThera and RGI-2001 (Regimmune) graft-versus-host disease associated with hematopoietic stem cell transplantation phase 2

rigosertib (Onconova Therapeutics) myelodysplastic syndrome phase 3

rivaroxaban (Xarelto, Janssen) prevention of symptomatic venous thromboembolism and VTE-related death phase 3 in high-risk, medically ill patients rivipansel (GMI-1070) vaso-occlusive crisis associated with sickle cell disease phase 2

rocilinostat (Acetylon Pharmaceuticals) myeloma phase 2

(Istodax, Celgene) peripheral T-cell lymphoma (first-line) phase 3

injection (AMG531; Amgen, Kyowa Hakko Kirin) aplastic anemia phase 2

roxadustat (AstraZeneca) anemia in chronic kidney disease, anemia in end-stage renal disease phase 3

ruxolitinib (Jakafi, Incyte) chronic lymphocytic leukemia, chronic myeloid leukemia, thalassemia major phase 2

polycythemia vera phase 3 (Cyclacel) chronic lymphocytic leukemia, myelodysplastic syndrome phase 2

acute myelogenous leukemia phase 3 SAR245409 (XL765; Exelixis, Sanofi) leukemia, non-Hodgkin’s lymphoma phase 2

SAR3419 (ImmunoGen/Sanofi) diffuse large B-cell lymphoma phase 2 70 HemOnc today | NOVEMBER 25, 2014 | Healio.com/HemOnc

Hematology Drugs in the Pipeline

Generic name (Brand name, Manufacturer) Indication(s) Development status

SAR650984 (ImmunoGen) myeloma phase 2

SelG1 (Selexys Pharmaceuticals) sickle cell disease phase 2

SG2000 (Spirogen) acute myelogenous leukemia, chronic lymphocytic leukemia phase 2

SGI110 (Astex) acute myelogenous leukemia, myelodysplastic syndrome phase 2

(Sylvant, Janssen) myeloma phase 2

simtuzumab (Gilead) myelofibrosis phase 2

simvastatin sickle cell disease phase 2

sirolimus sickle cell disease phase 2

SL-401 (Stemline Therapeutics) acute myelogenous leukemia, myelodysplastic syndrome phase 2

SNS01-T (Sevion Therapeutics) diffuse large B-cell lymphoma, mantle cell lymphoma, myeloma phase 2

sonidegib (LDE225, Novartis) myelofibrosis phase 2

sotatercept (ACE-011; Celgene, Acceleron) anemia, beta-thalassemia, myelodysplastic syndrome phase 2

StemEx (Gamida Cell) hematologic malignancies phase 3

tetradecanoylphorbol acetate (Rich Pharmaceuticals) acute myelogenous leukemia phase 2

TG-1101 (TG Therapeutics) chronic lymphocytic leukemia, mantle cell lymphoma phase 2

TH-302 (Threshold Pharmaceuticals) myeloma phase 2

ticagrelor (Brilinta, AstraZeneca) sickle cell disease phase 2

arterial thrombosis phase 3 tosedostat (CTI Pharma) acute myelogenous leukemia, myelodysplastic syndrome phase 2

trenonacog alpha (IB 1001, Emergent Biosolutions) hemophilia B phase 3

pegol (N8-GP, Novo Nordisk) hemophilia A phase 3

TZ101 (Targazyme) cord blood transplantation for hematologic malignancies phase 2

umbilical cord blood cells expanded with multipotent progenitor transplantation in patients with hematologic malignancies phase 3 cells (Mesoblast) (ABT-888, AbbVie) lymphoma, myeloma phase 2

(Immunomedics) immune thrombocytopenia, non-Hodgkin’s lymphoma phase 2

vepoloxamer (MST-188, Mast Therapeutics) vaso-occlusive crisis in patients with sickle cell disease phase 3 READ PERSPECTIVE on this drug from Susan P. Perrine, MD, on page 73.

sulfate liposome injection (Marqibo, Spectrum Pharma- non-Hodgkin’s lymphoma, Philadelphia chromosome-negative acute lym- phase 3 ceuticals) phoblastic leukemia (front-line, elderly patients) vismodegib (Erivedge; Roche/Curis) acute myelogenous leukemia phase 2

VLX1570 (Vivolux) myeloma (relapsed/refractory) phase 2

volasertib (BI 6727, Boehringer Ingelheim) acute myelogenous leukemia phase 3

(Zolinza, Merck) sickle cell anemia, sickle cell disease phase 2

myeloma phase 3 vosaroxin (Qinprezo) acute myelogenous leukemia (front-line), myelodysplastic syndrome phase 2

acute myelogenous leukemia (relapsed/refractory) phase 3

Information in this chart was compiled from the Pharmaceutical Research and Manufacturers of America, NIH (www.clinicaltrials.gov), corporate websites and the databases of HemOnc Today. The publisher or editors do not assume responsibility for any errors or omissions.