RG2833 (RGFP109) is a brain-penetrant HDAC inhibitor with IC50 of 60 nM and 50 nM for HDAC1 and HDAC3, respectively.

RGFP109 dose-dependently upregulates frataxin mRNA and protein levels in cultures of unstimulated peripheral blood mononuclear cells (PBMC) obtained from FRDA patients.

RG2833 (150 mg/kg) corrects frataxin deficiency and increases histone acetylation in the brain and heart of KIKI mice without acute toxicity signs. RGFP109 ameliorates the disease phenotype of a Friedreich ataxia mouse model. RGFP109 (30 mg/kg p.o.) also alleviates established l-DOPA-induced dyskinesia.