CMT news out of Belgium: Augustine Therapeutics has officially dosed the first participant in a Phase I clinical trial of AGT-100216, the company’s experimental drug in development for Charcot-Marie-Tooth disease (CMT).
According to the company, AGT-100216 is an HDAC6 inhibitor (HDAC6i). HDAC6, or histone deacetylase 6, is a cellular enzyme involved in multiple pathways linked to nerve health and function.
The Phase I trial will focus on safety, tolerability, and pharmacokinetics (how the drug moves through the body) in healthy volunteers. Phase I trials are a foundation of clinical research. They help determine whether a new drug is safe enough to move forward into studies involving people with the disease it’s intended to treat. .
“The initiation of our first clinical trial is a major milestone for Augustine,” said Gerhard Koenig, PhD, Augustine Therapeutics’ CEO. “Decades of research have validated the therapeutic potential of HDAC6 as a target, but efforts to drug it to date have been sub-optimal. Augustine is developing a new generation of HDAC6 inhibitors, like AGT-100216, with a unique mechanism of action shown to be selective, safe, and effective in pre-clinical trials.” .
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Published on: May 30, 2025