Azacitidine is a pyrimidine nucleoside analogue with anti-neoplastic activity. It differs from cytosine by the presence of nitrogen in the C5-position, key in its hypomethylating activity.[A1406,A1413,A1415] Two main mechanisms of action have been proposed fo…

Myelodysplastic syndromes (MDS) are a group of hematopoietic neoplasms that give rise to variable cytopenias progressing to secondary acute myeloid leukemia (sAML), which is invariably fatal if untreated.[A215082, A215092, A215097] Hypomethylating agents such…

CD70 is a ligand expressed on the surface of activated lymphocytes and mature dendritic cells that, in its binding to CD27 receptors, plays an important role in cell proliferation and survival. An overexpression of CD70 can result in the proliferation of mali…

DCLL9718S is an antibody-drug conjugate targeting C-type lectin-like molecule-1 (CLL-1). DCLL9718S is under investigation in clinical trial NCT03298516 (A Study of DCLL9718S in Participants With Relapsed or Refractory Acute Myeloid Leukemia (AML) or DCLL9718S…

Myelodysplastic syndromes (MDS) are a heterogeneous group of hematopoietic neoplasms with variable underlying etiology and presentation, including neutropenia and thrombocytopenia. Further mutations leading to increased proliferation of cancerous cells can ev…

evorpacept is under investigation in clinical trial NCT04755244 (A Study of Evorpacept (ALX148) With Venetoclax and Azacitidine for Acute Myeloid Leukemia (ASPEN-05)).

FF-10501-01 is a novel, selective inosine monophosphate dehydrogenase (IMPDH) inhibitor with anticancer activity.[A257306] FF-10501-01 is under investigation in clinical trial NCT03486353 (A Study of FF-10501-01 in Combination With Azacitidine in Patients Wit…

INCB-057643 is under investigation in clinical trial NCT02959437 (Azacitidine Combined With Pembrolizumab and Epacadostat in Subjects With Advanced Solid Tumors (ECHO-206)).

Ivosidenib is a first-in-class isocitrate dehydrogenase-1 (IDH1) inhibitor. IDH1 is an enzyme that is often mutated and overexpressed in some cancers, leading to aberrant cell growth and proliferation.[A248750] Ivosidenib inhibits mutated IDH1, blocking the e…

Jaktinib is a JAK 1, JAK 2 and JAK 3 inhibitor, being developed by Suzhou Zelgen Biopharmaceuticals. Jaktinib is under investigation in clinical trial NCT04866056 (Jaktinib and Azacitidine in Treating Patients With MDS With MF or MDS/MPN With MF.).

Lemzoparlimab is under investigation in clinical trial NCT04912063 (Study to Evaluate Adverse Events and Movement of Lemzoparlimab in Body When Used Intravenously (IV) With Azacitidine Subcutaneously or IV and Venetoclax Orally in Participants With Acute Myel…

Letaplimab is under investigation in clinical trial NCT04485052 (Efficacy and Safety Evaluation of IBI188 in Combination With Azacitidine in Treatment of Patients With Acute Myeloid Leukemia).

Milademetan is under investigation in clinical trial NCT02319369 (Safety, Tolerability and Pharmacokinetics of Milademetan Alone and With 5-Azacitidine (AZA) in Acute Myelogenous Leukemia (AML) or High-Risk Myelodysplastic Syndrome (MDS)).

PLX51107 is a potent and selective inhibitor of the bromodomain and extraterminal (BET) protein family.[A257246,A257251] PLX51107 is under investigation in clinical trial NCT04022785 (PLX51107 and Azacitidine in Treating Patients With Acute Myeloid Leukemia o…

Sovleplenib is a novel, investigational, selective small molecule inhibitor for oral administration targeting spleen tyrosine kinase (Syk). It is under investigation in clinical trial NCT03483948 (Phase I Study of Hmpl-523+azacitidine in Elderly Patients With…

IMM01 is a recombinant human signal regulatory protein ? (SIRP?) IgG 1 fusion protein.[A257261] IMM01 is under investigation in clinical trial NCT05140811 (A Study of IMM01 Combined With Azacitidine in Patients With Acute Myeloid Leukemia and Myelodysplastic …