A New CMT Treatment Is One Step Closer
Augustine Therapeutics Begins Phase I Trial of AGT‑100216
Exciting news for the CMT community! TRIAD partner, Augustine Therapeutics has officially dosed the first human participant in their Phase I clinical trial of AGT‑100216—a potential disease-modifying therapy for Charcot-Marie-Tooth disease.
This trial is the first-ever clinical study of a peripherally restricted, selective HDAC6 inhibitor for CMT. AGT‑100216 is designed to target the root causes of nerve damage with fewer side effects, giving hope for safer, long-term treatment options.
What’s happening now?
The Phase I trial focuses on healthy volunteers to test:
- Safety & tolerability
- How the drug behaves in the body (pharmacokinetics)
- Early signs of effectiveness (pharmacodynamics)
Why it matters:
CMT affects 1 in 2,500 people, and there are currently no approved treatments that slow or stop the disease. This milestone is a huge step forward in bringing a targeted therapy closer to those who need it most.
Next steps?
Pending results, Augustine plans to move into patient-centric trials. With strong backing from global investors and scientific leaders, this program is gaining momentum—and fast.
Learn more about the trial here
Thank you for being part of the movement to cure CMT. Every step forward starts with YOU.
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