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Obafistat is an investigational small molecule inhibitor of aldo-keto reductase family 1 member C3 (AKR1C3, also known as type 5 17β-hydroxysteroid dehydrogenase), an enzyme involved in the activation of androgens in peripheral tissues such as adipose tissue. Originally developed by Bayer, obafistat is being co-developed in collaboration with Imperial College London for the treatment of polycystic ovary syndrome (PCOS), also referred to as polyendocrine metabolic ovarian syndrome (PMOS). By inhibiting AKR1C3, the drug aims to lower active androgen levels and improve metabolic health in women with PCOS. It is currently being evaluated in the Phase 1 AKROPAT trial, a randomized, double-blind, placebo-controlled dose-escalation study in obese adult women with PCOS.
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