ASH Clinical News Focus on Myeloid Malignancies | Page 11
Hypomethylating Agents
Guadecitabine: a DNA methyltransferase inhibitor for the
treatment of higher-risk MDS or AML (phase I/II)
CAR T-Cell Therapies
CD123 CAR T cells: CD123-directed autologous modified T cells
for the treatment of relapsed/refractory CD123-positive AML or
BPDCN (phase I)
CD33 CAR T cells: CD33-directed allogeneic modified T cells
for the treatment of relapsed/refractory CD33-positive AML
(phase I)
Tipifarnib: a farnesyltransferase inhibitor for the treatment of
newly diagnosed AML (phase II/III)
Imetelstat: a first-in-class telomerase inhibitor for the treatment
of myelofibrosis and MDS (phase II)
Ivosidenib: an IDH1 inhibitor for the treatment of IDH2-
mutant relapsed/refractory and newly diagnosed AML (phase I)
IDH305: an IDH1 inhibitor for the treatment of IDH-mutant,
newly diagnosed AML (phase I)
ASTX727: an oral cytidine deaminase inhibitor for the treatment
of MDS (phase I/II)
H3B-8800: an SF3b modulator for the treatment of MDS, AML,
and CMML (phase I)
Tamibarotene: a synthetic retinoid for the treatment of
relapsed/refractory AML and MDS (phase II)
HDAC Inhibitor
Pracinostat: an HDAC inhibitor for the treatment of newly
diagnosed AML, myelofibrosis, and MDS (phase II)
Belinostat: an HDAC inhibitor for the treatment of relapsed/
refractory AML and MDS (phase I)
Entinostat: an HDAC inhibitor for the treatment of previously
treated MDS (phase I) and relapsed/refractory AML and CMML
(phase II)
CD7 CAR T cells: CD7-directed allogeneic modified T cells for
the treatment of relapsed/refractory AML (phase I/II)
Recombinant Fusion Proteins
Luspatercept: a recombinant fusion protein for the treatment of
anemia in MDS and β-thalassemia (phase III)
Sotatercept: a recombinant fusion protein for the treatment of
anemia in MDS, non-CMML, and MPNs (phase II)
Immune Checkpoint Inhibitors
Nivolumab: an anti-PD-1 monoclonal antibody for the
treatment of relapsed/refractory AML (phase II) and MDS
(phase II)
Pembrolizumab: an anti-PD-1 monoclonal antibody for
the treatment of newly diagnosed AML (phase II), relapsed/
refractory AML (phase I/II), and advanced MPNs (phase II)
Atezolizumab: an anti-PD-1 monoclonal antibody for the
treatment of relapsed/refractory and treatment-naïve AML
(phase I/II) and relapsed/refractory CMML (phase I/II)
CC-90002: an anti-CD47 antibody for the treatment of relapsed/
refractory AML or high-risk MDS (phase I)
Hu5F9-G4: an anti-CD47 monoclonal antibody for the
treatment of relapsed/refractory AML (phase I)
May 2017
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