|Tildacerfont||Adult Classic Congenital Adrenal Hyperplasia||CAHmelia-203 and CAHmelia-204, evaluating adrenal androgen control, glucocorticoid reduction and related clinical outcomes, are in progress|
|Pediatric Classic Congenital Adrenal Hyperplasia||Phase 2 trial in children planned in 2021|
|Polycystic Ovary Syndrome||Filing of U.S. IND and initiation of Phase 2 proof of concept trial planned in 2021|
Tildacerfont is a 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 on the pituitary gland, inhibiting excessive production of ACTH, and ultimately limiting the production of adrenal androgens such as androstenedione, a precursor to testosterone.