Belgian company Augustine Therapeutics has initiated subject dosing in the randomised Phase I trial of AGT-100216, a selective histone deacetylase 6 (HDAC6) inhibitor, targeting the treatment of Charcot-Marie-Tooth disease (CMT).
The first-in-human, double-blind, placebo-controlled trial is aimed at assessing the safety, tolerability, pharmacodynamics and pharmacokinetics of the therapy in healthy adult volunteers.
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This two-part trial combines single ascending and multiple ascending dose evaluations of this oral therapy.
Augustine noted that AGT-100216 inhibits HDAC6, which is said to preserve the enzyme’s beneficial non-catalytic functions.
The company’s strategy involves a non-hydroxamate, non-hydrazide producing chemotype, which is expected to circumvent the limitations associated with other chemotypes and is tailored for chronic conditions.
In addition to targeting CMT, the company plans to explore the potential of its HDAC6 inhibitors in treating other diseases, including neurodegenerative and cardio-metabolic conditions.
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By GlobalDataCMT represents a diverse group of hereditary peripheral neuropathies, marked by progressive damage to the nerves of the legs, feet, arms and hands.
Augustine Therapeutics CEO Gerhard Koenig said: “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.
“We are well-positioned to progress AGT-100216 through clinical development for CMT and to also advance our pipeline of next-generation HDAC6 inhibitors in significant cardio-metabolic and neurodegenerative diseases.”
This March, the company secured funding in an oversubscribed Series A financing round, raising €78m ($85m) investment spearheaded by Jeito Capital and Novo Holdings.
The round also included contributions from Eli Lilly, Asabys Partners, AdBio Partners, PMV, VIB, V-Bio Ventures, Gemma Frisius Fund, the US-based CMT Research Foundation, and Newton Biocapital.
