Oral ACTH Antagonist


ACTH acts through the melanocortin type 2 receptor (MC2R) that is primarily expressed in the adrenal gland. This is the first reported oral, selective ACTH antagonist discovered by our team in development for the treatment of Cushing’s disease and other diseases of excess ACTH, such as congenital adrenal hyperplasia.

For the Treatment of Cushing’s Disease


Cushing’s disease results from a pituitary tumor that secretes excess ACTH, which in turn causes the downstream synthesis and over-secretion of cortisol by the adrenal glands. Cortisol is the body’s main stress hormone and excess amounts can cause significant increases in mortality and morbidity.

For the Treatment of Congenital Adrenal Hyperplasia (CAH)


CAH encompasses a set of disorders caused by genetic mutations that result in impaired cortisol synthesis. This lack of cortisol leads to a breakdown of feedback mechanisms and results in persistently high levels of ACTH, which in turn causes overstimulation of the adrenal cortex. The resulting adrenal hyperplasia and over-secretion of other steroids (particularly androgens) and steroid precursors can lead to a variety of effects, from improper gonadal development to life-threatening dysregulation of mineralocorticoids.

We have ongoing efforts to advance the first nonpeptide product candidate to antagonize the peptide adrenocorticotrophic hormone (ACTH) designed for the treatment of Cushing’s disease, CAH, and other diseases of ACTH excess.