Pelabresib — Investigational Agent
BET Inhibitor · Phase 3 · MorphoSys (acquired by Novartis)
Mechanism of Action
Pelabresib is a potent and selective small molecule inhibitor of the Bromodomain and Extra-Terminal (BET) family of proteins (BRD2, BRD3, BRD4, and BRDT). It binds to the bromodomains of these epigenetic reader proteins, preventing their interaction with acetylated histones. This blockade suppresses the transcription of key oncogenes, including MYC, and downregulates pro-inflammatory cytokines such as IL-6 and TNF-alpha. In myelofibrosis, this leads to a reduction in megakaryocyte differentiation and attenuation of bone marrow fibrosis.
Investigational Indications
- Myelofibrosis
- Essential Thrombocythemia