Drug intelligence / Profile preview

CPI-482

Development stage
Preclinical
Lead developer
Novartis
Modality
Small Molecules
01

Overview

**CPI-482** is a small molecule inhibitor of lysine-specific demethylase 1 (LSD1, also known as KDM1A), an epigenetic enzyme that regulates gene expression by removing methyl groups from histone H3 lysine 4 (H3K4me). Developed by Constellation Pharmaceuticals, it suppresses megakaryocyte formation, reduces spleen volume, bone marrow fibrosis, and improves survival in preclinical mouse models of myelofibrosis and leukemia. Named as a development candidate in 2021 for hematologic malignancies, particularly myeloproliferative neoplasms (MPNs), it leverages translational research on epigenetic mechanisms in myeloid cells, though it was in preclinical stages as of 2020-2022 and later deprioritized following company acquisition.[1][2][3][7]

02

Targets

KDM1A (LSD1)

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