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pevonedistat
- Development stage
- Phase 3
- Lead developer
- Takeda
- Modality
- Small Molecules
01
Overview
Pevonedistat is a first-in-class small molecule inhibitor of the NEDD8 activating enzyme (NAE), developed primarily as an antineoplastic agent. By binding to and inhibiting NAE, pevonedistat disrupts the neddylation pathway, which is essential for the activation of cullin-RING ligases involved in protein degradation. This inhibition leads to disruption of cell cycle progression and survival in cancer cells, resulting in apoptosis and reduced tumor proliferation. Pevonedistat has been investigated mainly for hematologic malignancies such as higher-risk myelodysplastic syndromes (HR-MDS), acute myeloid leukemia (AML), chronic myelomonocytic leukemia (CMML), and various solid tumors. It has received orphan drug status for AML and MDS[1][3][5][7].
- Other names
- pevonedistatum
02
Targets
NAE (NEDD8-activating enzyme)
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