AGT100216

AGT100216 is a first-in-class, peripherally restricted and selective small molecule inhibitor of histone deacetylase 6 (HDAC6), developed by Augustine Therapeutics. It is being advanced as a novel therapeutic for Charcot-Marie-Tooth disease (CMT) and other peripheral neuropathies. The drug acts by inhibiting HDAC6, an enzyme implicated in neurodegeneration and cellular aging processes. Preclinical studies have demonstrated that AGT100216 can halt disease progression, reverse the disease phenotype, and rescue axonal integrity in CMT models in a dose-dependent manner. The compound has shown potent oral anti-inflammatory activity through attenuation of NLRP3 inflammasome activation. As of early 2025, it is entering Phase I/II clinical trials for CMT[1][2][3][5][6][7][8].

Other names

Selective HDAC6 inhibitors Augustine Therapeutics