Drug intelligence / Profile preview

Ro 08-2750

Development stage
Preclinical
Modality
Small Molecules
01

Overview

Ro 08-2750 is a small molecule inhibitor originally developed by Hoffmann-La Roche. It was initially identified as a non-peptide antagonist of nerve growth factor (NGF) that selectively prevents NGF from binding to the p75 neurotrophin receptor (p75NTR) over TrkA at low concentrations, and inhibits both at higher concentrations. More recently, Ro 08-2750 has been characterized as a competitive inhibitor of the RNA-binding proteins Musashi-1 (MSI1) and Musashi-2 (MSI2), disrupting their RNA-binding activity. It has been investigated in preclinical research for its potential therapeutic effects in various cancers, including acute myeloid leukemia (AML), mantle cell lymphoma (MCL), and triple-negative breast cancer (TNBC), where it impairs cancer stemness, cell survival, and chemoresistance.

Other names
2,3,4,10-tetrahydro-7,10-dimethyl-2,4-dioxobenzo[g]pteridine-8-carboxaldehyde7,10-dimethyl-2,4-dioxo-2,3,4,10-tetrahydrobenzo[g]pteridine-8-carbaldehyde
02

Targets

NGF (Nerve growth factor)MSI1 (RNA-binding protein Musashi homolog 1)MSI2 (Musashi RNA-binding protein 2)

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