32 HemOnc today | MAY 10, 2016 | Healio.com/HemOnc

Leukemia Drugs in the Pipeline

HemOnc Today presents this guide to drugs in phase 2 or phase 3 development for leukemia. 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. Generic name (Brand name, Manufacturer) Indication(s) Development status

Å6 peptide (Angstrom Pharmaceuticals) chronic lymphocytic leukemia phase 2

acalabrutinib (ACP-196, Acerta Pharma) chronic lymphocytic leukemia (relapsed/refractory, ibrutinib-intolerant disease) phase 2

chronic lymphocytic leukemia (monotherapy for previously treated high-risk phase 3 disease; combination therapy for previously untreated disease) AG-120 (Agios Pharmaceuticals and Celgene) acute myeloid leukemia (combination therapy for newly diagnosed disease) phase 2

AG-221 (Agios Pharmaceuticals and Celgene) acute myeloid leukemia (older patients with IDH2–mutated late-stage phase 3 disease) alvocidib (Tolero Pharmaceuticals) acute myeloid leukemia phase 2

AST-VAC1 (Asterias Biotherapeutics) acute myelogenous leukemia phase 2

Bismab-A2 (Actinium Pharmaceuticals) acute myelogenous leukemia phase 2

BL-8040 (BioLineRx) acute myeloid leukemia phase 2

(Amgen) acute lymphoblastic leukemia (Philadelphia chromosome-positive or BCR- phase 2 ABL–positive disease; patients with B-cell lineage with minimal residual dis- ease; combination therapy for older patients with newly diagnosed disease)

acute lymphoblastic leukemia (relapsed disease; pediatric patients with phase 3 B-cursor disease at high risk for relapse, younger patients with relapsed B-cell disease; combination therapy for patients with newly diagnosed BCR-ABL– negative B-lineage disease) bosutinib (Bosulif, Pfizer) chronic myelogenous leukemia (first-line therapy) phase 3

BP1001 (Bio-Path Holdings) acute myeloid leukemia phase 2

BPX-501 (Bellicum) acute lymphoblastic leukemia, acute myeloid leukemia, chronic lymphocytic phase 2 leukemia, chronic myeloid leukemia calaspargase pegol (Baxalta) acute lymphoblastic leukemia phase 3

carfilzomib (Kyprolis, Amgen) acute lymphoblastic leukemia (combination therapy for children with phase 2 relapsed/refractory disease) CC-486 (Celgene) acute myeloid leukemia (post-induction maintenance) phase 3

cerdulatinib (Portola Pharmaceuticals) chronic lymphocytic leukemia phase 2

CNDO-109 (Fortress Biotech) acute myeloid leukemia phase 2

cordycepin (OVI-123, OncoVista Innovative Therapies) leukemia (TdT–positive refractory disease) phase 2

crenolanib (Arog Pharmaceuticals) acute myeloid leukemia (newly diagnosed disease; maintenance after bone phase 2 marrow transplant)

acute myeloid leukemia (relapsed/refractory disease) phase 3 cytarabine:daunorubicin (CPX-351, Celator) acute myeloid leukemia phase 2

secondary acute myeloid leukemia phase 3 dasatinib (Sprycel, Bristol-Myers Squibb) acute lymphoblastic leukemia, chronic myelogenous leukemia (combination phase 2 therapy)

chronic myeloid leukemia (newly diagnosed disease) phase 3 DFP-10917 (Delta-Fly Pharma) acute lymphocytic leukemia, acute myeloid leukemia phase 2

durvalumab (Celgene) acute myeloid leukemia phase 2

duvelisib (AbbVie and Infinity Pharmaceuticals) chronic lymphocytic leukemia (relapsed/refractory disease) phase 3

entospletinib (Gilead) acute myeloid leukemia (combination therapy), chronic lymphocytic leukemia phase 2

epratuzumab (Immunomedics) leukemia phase 2

ERY-ASP (Erytech Pharma) acute lymphoblastic leukemia (relapsed disease), acute myeloid leukemia phase 2

ganetespib (Synta Pharmaceuticals) acute myeloid leukemia phase 3

gilteritinib (ASP2215, Astellas) acute myeloid leukemia phase 3

glasdegib (PF-0449913, Pfizer) acute myeloid leukemia phase 2

GO-203-2C (Genus Oncology) acute myeloid leukemia (relapsed/refractory disease) phase 2

guadecitabine (SGI-110, Otsuka) acute myeloid leukemia phase 3

ibrutinib (Imbruvica; Pharmacyclics and Janssen) acute lymphoblastic leukemia, acute myelogenous leukemia phase 2

chronic lymphocytic leukemia (combination therapy for frontline treatment) phase 3 idelalisib (Zydelig, Gilead) chronic lymphocytic leukemia (relapsed/refractory disease) phase 3

Imprime PGG (Biothera) chronic lymphocytic leukemia phase 2 HemOnc today | MAY 10, 2016 | Healio.com/HemOnc 33

Leukemia Drugs in the Pipeline

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

(Pfizer) acute lymphoblastic leukemia phase 3

Iomab-B (Actinium Pharmaceuticals) acute myeloid leukemia (transplant conditioning for elderly patients with phase 3 relapsed/refractory disease) JCAR015 (Juno Therapeutics) acute lymphoblastic leukemia (pediatric) phase 2

JNJ-56022473 (CSL and Janssen) acute myeloid leukemia phase 2

lenalidomide (Revlimid, Celgene) chronic lymphocytic leukemia (maintenance) phase 3

-Ac225 (Actinium Pharmaceuticals) acute myeloid leukemia (combination therapy for older patients) phase 2

midostaurin (PKC412, Novartis) acute myelogenous leukemia phase 3

mogamulizumab (KW-0761, Kyowa Hakko Kirin) adult T-cell leukemia phase 2

MOR208 (Morphosys) acute lymphoblastic leukemia, chronic lymphocytic leukemia phase 2

(AstraZeneca) hairy cell leukemia phase 3

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

(Opdivo, Bristol-Myers Squibb) acute myeloid leukemia, chronic lymphocytic leukemia (combination phase 2 therapy) (Genentech) chronic lymphocytic leukemia (front-line therapy) phase 2

(Arzerra, Novartis) acute lymphoblastic leukemia (combination therapy for CD20–positive phase 2 disease), chronic lymphocytic leukemia (combination therapy for first-line treatment and previously treated disease)

chronic lymphocytic leukemia (maintenance therapy; combination therapy phase 3 for relapsed disease) otlertuzumab (Emergent BioSolutions) chronic lymphocytic leukemia phase 2

pacritinib (Baxalta) acute myeloid leukemia (combination therapy) phase 2

ponatinib (Iclusig, Ariad Pharmaceuticals) acute lymphoblastic leukemia (Philadelphia chromosome-positive or phase 2 BCR-ABL–positive disease), acute myeloid leukemia (intermediate-risk FLT3- ITD–positive disease in first complete remission), chronic myeloid leukemia (frontline therapy in accelerated phase; second-line therapy)

chronic myeloid leukemia (imatinib-resistant disease in chronic phase) phase 3 pracinostat (MEI Pharma) acute myeloid leukemia (frontline therapy, elderly patients) phase 2

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

quizartinib (Daiichi Sankyo) acute myeloid leukemia phase 3

biosimilar (GP2013, Sandoz) chronic lymphocytic leukemia phase 3

RP-323 (Rich Pharmaceuticals) acute myeloid leukemia phase 2

sapacitabine (CYC682, Cyclacel Pharmaceuticals) acute myeloid leukemia phase 3

selinexor (KPT-330, Karyopharm Therapeutics) acute myeloid leukemia phase 2

SGI-110 (Astex Pharmaceuticals) acute myelogenous leukemia phase 2

SL-401 (Stemline Therapeutics) acute myeloid leukemia phase 2

TG-1101 (TG Therapeutics) chronic lymphocytic leukemia (combination therapy) phase 3

tisagenlecleucel-T (CTL019, Novartis) acute lymphoblastic leukemia (pediatric) phase 2

TK008 (MolMed) acute leukemia (high-risk disease) phase 3

tosedostat (CTI Biopharma) acute myeloid leukemia (relapsed/refractory disease) phase 2

(SGN-CD33A) acute myeloid leukemia (relapsed disease) phase 2

acute myeloid leukemia (frontline therapy for older patients) phase 3 venetoclax (ABT-199/GDC-019; AbbVie and Roche) acute myelogenous leukemia phase 2

chronic lymphocytic leukemia (frontline therapy; relapsed/refractory disease; phase 3 unfit patients) volasertib (Boehringer Ingelheim) acute myeloid leukemia phase 3

vosaroxin (Qinprezo) acute myeloid leukemia (frontline therapy; relapsed/refractory disease) 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.