Drug intelligence / Profile preview

bomedemstat + tretinoin

Development stage
Unknown
Lead developer
Merck
Modality
Small Molecules
Administration
Oral
01

Overview

This drug is a **combination of two active agents: bomedemstat and tretinoin (all-trans retinoic acid)**. - **Bomedemstat** is an orally administered small molecule inhibitor of lysine-specific demethylase 1 (LSD1/KDM1A), being investigated for its ability to disrupt abnormal hematopoietic cell proliferation by modifying epigenetic regulation. It is in late-stage development and has demonstrated efficacy in multiple myeloproliferative neoplasms (MPNs), acute myeloid leukemia (AML), and myelodysplastic syndromes (MDS)[1][2][3][4][5][8]. - **Tretinoin (all-trans retinoic acid, ATRA)** is a retinoid that acts primarily by binding to retinoic acid receptors (RARs), modulating gene expression, and promoting cellular differentiation. It is well established as a treatment for acute promyelocytic leukemia (APL) and various dermatologic conditions. This combination is being explored in high-risk AML and high-risk MDS. The rationale is that LSD1 inhibition enhances differentiation effects, and ATRA/tretinoin synergistically increases leukemic cell differentiation and apoptosis[5][6].

Other names
all-trans retinoic acid
02

Targets

KDM1A (LSD1)

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