Also known as: AZA
Azacitidine is an antineoplastic nucleoside analogue medicine used in myelodysplastic syndrome and myeloid leukemias. It is marketed under brands including Vidaza and Onureg. Its activity includes hypomethylating and cytotoxic effects depending on dosing context.
A phase 3 trial of azacitidine and chidamide plus CHOP in untreated PTCL showed a significant overall survival improvement but no significant gains in response rate or progression-free survival. The study suggests molecular profiling could guide future therapies.
The FDA has cleared an IND for Amphista's novel BRD9 degrader AMX-883 for AML, approved Lantheus' new prostate cancer imaging agent Pylarify TruVu, and accepted Biofrontera's sNDA for Ameluz PDT in superficial basal cell carcinoma. These regulatory milestones span hematologic and solid tumor areas, addressing significant unmet medical needs.
Omeros reported initial nonhuman primate data for OncotoX-AML in acute myeloid leukemia. One treatment course reduced myeloid progenitor cells by up to 99% and was well tolerated.
Over 10 companies are developing 12+ pipeline therapies for Neuromyelitis Optica Spectrum Disorder, including Roche's satralizumab Phase III pediatric trial and multiple anti-CD20 monoclonal antibody candidates.
Faron Pharmaceuticals announces acceptance of bexmarilimab Phase I/II data for poster presentation at BSH 2026, while launching a new Phase II BEAM-X study evaluating the drug in AML patients post-stem cell transplantation.
Faron Pharmaceuticals announces Phase II BEAM-X trial evaluating bexmarilimab plus azacitidine in MRD-positive AML patients after stem cell transplantation. The Nordic AML Group-led trial will enroll 24 patients with first results expected 12-15 months after enrollment begins in Q3 2026.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07773909 |
Venetoclax With Hypomethylating Agents in Newly Diagnosed AML Unfit for Intensive Chemotherapy |
COMPLETED | PHASE4 |
| NCT07770698 |
A Study of Sonrotoclax (BGB-11417) in Children With Relapsed or Refractory Acute Myeloid Leukemia and B-cell Acute Lymphoblastic Leukemia |
NOT_YET_RECRUITING | PHASE1/PHASE2 |
| NCT07754799 |
A Randomized Phase II Selection Trial of Venetoclax-Based Induction Intensity in Newly Diagnosed Acute Myeloid Leukemia |
NOT_YET_RECRUITING | PHASE2 |
| NCT07743112 |
A Phase 2 Study of MAX-40279 Combined With Venetoclax and Azacitidine in Unfit Newly Diagnosed Acute Myeloid Leukemia |
RECRUITING | PHASE2 |
| NCT07735117 |
Mitoxantrone Hydrochloride Liposome, Cytarabine, G-CSF Plus Venetoclax vs. Azacitidine Plus Venetoclax for MDS-IB2 and Secondary/Elderly AML |
NOT_YET_RECRUITING | PHASE3 |
| NCT07722767 |
A Phase 1b Study of CLN-049 in Combination With Azacitidine and Venetoclax in AML Patients |
NOT_YET_RECRUITING | PHASE1 |
| NCT07691450 |
Belinostat in Combination With Azacitidine or Pralatrexate for the Treatment of Relapse or Refractory T-cell Lymphoma |
NOT_YET_RECRUITING | PHASE1 |
| NCT07679334 |
Phase I Trial of Pacritinib in Combination With Venetoclax and Azacitidine for the Treatment of Accelerated and Blast Phase Myeloproliferative Neoplasms |
NOT_YET_RECRUITING | PHASE1 |
| NCT07672769 |
Bexmarilimab + Azacitidine Versus Placebo + Azacitidine in Participants With Treatment-naïve Higher-risk Myelodysplastic Syndromes |
NOT_YET_RECRUITING | PHASE2 |
| NCT07645469 |
FH-WT1-E50 TCR T Cells With Azacitidine for the Treatment of Minimal Residual Disease Positive Acute Myeloid Leukemia |
RECRUITING | PHASE1 |
