HDAC Program

Histone deacetylase (HDAC) inhibition results in increases and normalization in gene transcription through epigenetic regulation. FORUM Pharmaceuticals is currently investigating how this unique activity may have an important impact on brain diseases that display abnormalities in this gene transcription, which could potentially benefit patients in certain Orphan Drug indications.

FRM-0334 represents an investigational first-in-class, readily brain-penetrant HDAC inhibitor in development expressly for brain diseases that has successfully and safely completed Phase 1 clinical trials. FORUM Pharmaceuticals expects to progress FRM-0334 into an investigational Phase 2 clinical trial in frontotemporal dementia with progranulin mutation later this year.