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GV-100 is a novel, orally bioavailable, selective histone deacetylase 6 (HDAC6) inhibitor being developed by Gilva Therapeutics for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD). HDAC6 is an enzyme implicated in the regulation of cyst formation, inflammation, and fibrosis, which are central to the progression of ADPKD. GV-100 is a small molecule quinazolinone derivative that has shown potent and selective inhibition of HDAC6 (IC50 = 1.18 nM) in preclinical models. In May 2026, Gilva Therapeutics received approval in Australia to initiate a Phase 1 first-in-human clinical trial, designed as a randomized, double-blind, placebo-controlled single and multiple ascending dose (SAD/MAD) study in healthy adults to evaluate safety, pharmacokinetics, and potential drug-drug interactions.
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