Drug intelligence / Profile preview

MBF-015

Development stage
Phase 2
Lead developer
Medibiofarma
Modality
Small Molecules
Administration
Oral
01

Overview

MBF-015 is a small molecule drug developed as a potent inhibitor of CoREST/HDAC1/2, designed for the treatment of synaptopathies such as Huntington's disease, multiple sclerosis, Alzheimer's disease, and Parkinson's disease. It acts by inhibiting histone deacetylase 1 and 2 (HDAC1/2), enzymes involved in transcriptional repression through chromatin remodeling. By blocking these enzymes, MBF-015 promotes synaptic regrowth and regulates gene expression relevant to neurodegenerative diseases. The compound freely crosses the blood-brain barrier and has demonstrated significant induction of synaptic regrowth in animal models. It is administered orally and has shown favorable pharmacokinetics and tolerability in preclinical studies. MBF-015 is currently in Phase II clinical trials for Huntington’s disease[1][3][5].

02

Targets

HDAC1 (Histone Deacetylase 1)RCOR1 (CoREST Complex)

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