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RT-312 is an orally bioavailable small molecule inhibitor of 11-beta-hydroxysteroid dehydrogenase type 1 (11β-HSD1) being developed by Retex AU Pty Ltd for the treatment of autosomal dominant polycystic kidney disease (ADPKD). The enzyme 11β-HSD1 is responsible for the intracellular conversion of inactive cortisone into active cortisol; in the context of ADPKD, elevated local cortisol levels are thought to promote cyst growth, fluid secretion, and inflammation. By selectively inhibiting this enzyme, RT-312 aims to reduce renal cortisol production and slow disease progression. The compound is currently undergoing Phase 1 clinical evaluation in healthy participants to establish its safety, tolerability, and pharmacokinetic profile.
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