Drug Name: FRM-0334
Company: Forum Pharmaceuticals (formerly EnVivo Pharmaceuticals)
Location: US-Massachusetts
Drug Type: Small Molecule
Conditions: FTD
Mechanism Type: Epigenetics, HDAC inhibitor
Mechanism: FRM-0334 is a brain penetrant histone deacetylase (HDAC) inhibitor which is being developed as a therapy for frontotemporal dementia (FTD) due to progranulin haploinsufficiency. FRM-0334 has been shown to increase progranulin expression in rodents and in cells derived from patients carrying granulin mutations.
U.S. Status for ALS: N/A

[1] Selectivity and Kinetic Requirements of HDAC Inhibitors as Progranulin Enhancers for Treating Frontotemporal Dementia She, A. et al., Cell Chem Biol. 2017 Jul 20;24(7):892-906.e5.
[2] HDAC Program. Forum Pharmaceuticals, 2015. Accessed 9 Mar 2016 from
[3] Study to Assess the Safety, Tolerability, and Pharmacodynamic (PD) Effects of FRM-0334 in Subjects With Prodromal to Moderate Frontotemporal Dementia With Granulin Mutation., 6 May 2015. Accessed 9 Mar 2016 from

Last updated February 8th, 2018

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