Tildacerfont is a second-generation CRF1 receptor antagonist designed to potentially offer markedly improved disease control and reduced steroid burden in patients with classic congenital adrenal hyperplasia (CAH) and other diseases characterized by elevated levels of adrenocorticotropic hormone (ACTH). Tildacerfont binds to CRF1 receptors, inhibiting excessive production of ACTH, and ultimately limiting the production of adrenal androgens such as androstenedione in patients with CAH and PCOS. Tildacerfont also has the potential to address hyperactive brain CRF neurotransmission and aberrant functioning of the HPA axis in patients with MDD by blocking CRF1 receptors in the brain to address hyperactive corticotropin-releasing factor (CRF) neurotransmission and CRF1 receptor signal transduction.