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azacitidine + panobinostat
- Development stage
- Unknown
- Lead developer
- Novartis
- Modality
- Small Molecules
- Administration
- Oral, Subcutaneous, Intravenous
01
Overview
Azacitidine + panobinostat is an investigational combination therapy targeting epigenetic mechanisms in hematologic malignancies. Azacitidine is a hypomethylating agent that inhibits DNA methyltransferase, leading to DNA demethylation and reactivation of tumor suppressor genes. Panobinostat is a pan-histone deacetylase (HDAC) inhibitor that increases histone acetylation, promoting an open chromatin structure and inducing cell cycle arrest or apoptosis. This dual approach aims to synergistically reprogram the cancer cell epigenome. The combination has been primarily evaluated for acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and chronic myelomonocytic leukemia (CMML). While early phase studies showed promise, a randomized phase 2b trial indicated limited efficacy benefit over azacitidine monotherapy in certain populations. Recent research also explores its role in chemosensitizing KMT2A-rearranged pediatric AML by modulating NF-kB signaling.
- Brand names
- FarydakVidaza
- Other names
- aza-pano
02
Targets
DNMT1 (DNA (cytosine-5)-methyltransferase 1)
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