HDAC6 is a well-known target. We’ve got a breakthrough approach.
Histone DeACetylase 6 (HDAC6) is not like other HDACs. Its effects are not mediated through the nucleus nor does it directly affect epigenetic pathways. It’s a cytoplasmic regulator implicated in everything from microtubule transport to autophagy.
Microtubule Transport
Chaperone
Response
Autophagy &
Lipophagy
Aggresome Formation & Protein Clearance
Oxidative
Stress
Neurite Outgrowth & Regeneration
Innate Immunity & Inflammation
Decades of research and numerous animal studies have validated its therapeutic potential, but to date extensive industry efforts to drug it with hydroxamic acid and oxadiazole-based inhibitors have been suboptimal — not because the target was wrong, but because the molecules were not suitable.
At Augustine Therapeutics, we’ve solved these challenges with a novel non-hydroxamate, non-hydrazide producing chemotype: selective, safe, and built for chronic disease.
A single target with broad opportunities across neurologic and cardio-metabolic diseases
Our lead program, AGT-100216, is the first selective HDAC6 inhibitor for long-term treatment of Charcot-Marie-Tooth Disease (CMT), but our pipeline doesn’t stop there. We’re advancing a portfolio designed to maximize the therapeutic potential of HDAC6, targeting peripheral neuropathies, neurodegenerative and cardio-metabolic diseases.
The first peripherally-restricted, selective HDAC6i for Charcot-Marie-Tooth disease (CMT), currently in regulatory non-clinical development.
A novel class of peripherally-restricted, orally bioavailable HDAC6i for cardiometabolic diseases, currently in advanced lead optimization.
A brain-penetrant HDAC6i for neurodegenerative diseases like ALS, currently in lead optimization.
“Our mapping of the HDACi landscape has made us confident that Augustine’s innovative and rigorous approach to medicinal chemistry has yielded molecules with potential to be best-in-class. HDAC6 inhibition shows great promise in many indications, and we are enthused to start our collaboration with Augustine’s top-tier management team”











Decades of experience, one bold mission
Our leadership combines deep scientific expertise with proven drug development success. With experience across pharma, biotech, and academia, we are pioneering a new generation of HDAC6 inhibitors that are selective, safe, and effective.










