Drug intelligence / Profile preview

givinostat + mechlorethamine

Development stage
Phase 2
Lead developer
Italfarmaco
Modality
Small Molecules
Administration
Oral (for Givinostat), Intravenous (for Mechlorethamine), Topical (mechlorethamine, But Not In Combination)
01

Overview

This is a **combination drug** comprising **givinostat**, a small molecule inhibitor of class I and II histone deacetylases (HDACs), and **mechlorethamine**, an alkylating agent used primarily in hematological malignancies. - **Givinostat** acts as an epigenetic modulator, relaxing chromatin structure by inhibiting HDACs, leading to upregulation of muscle regeneration genes (such as MyoD, Myogenin) and suppression of inflammatory and fibrotic pathways (such as the NF-κB pathway). It reduces fibrosis, inflammation, and promotes muscle repair, with confirmed efficacy and safety in Duchenne muscular dystrophy (DMD)[1][2][3][5]. - **Mechlorethamine** is a DNA-alkylating agent that cross-links DNA, impairing cell replication and leading to cell death; it is mainly used as a chemotherapeutic in lymphomas. There is no established clinical use, approval, or reported development of the specific combination "givinostat + mechlorethamine." No data is available on the mechanism, clinical purpose, or trials for this pairing as a fixed therapy.

02

Targets

DNAHDAC1 (Histone Deacetylase 1)HDAC2 (Histone deacetylase 2)

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