GPCR

• MC2 is the adrenocorticotropic hormone (ACTH) receptor and is almost exclusively expressed in the adrenal cortex
• ACTH is a key component of the hypothalamic-pituitary-adrenal (HPA) axis and can trigger cortisol and androgen production
• Human genetics provides support that blocking MC2 significantly decreases the production of cortisol and androgens without significant side effects

Diseases of ACTH excess, including congenital adrenal hyperplasia and ACTH-dependent Cushing’s

• Mallappa, A. & Merke, D., Management challenges and therapeutic advances in congenital adrenal hyperplasia (2022). Nature Reviews Endocrinology 18, 337-352
• Reincke, M. & Fleseriu, M., Cushing syndrome: A review (2023). Journal of the American Medical Association 330, 170-181
• O’Riordan, S. et al., A novel variant of familial glucocorticoid deficiency prevalent among the Irish Traveler population (2008). The Journal of Clinical Endocrinology and Metabolism 93, 2896-9

The focus of our program has been to increase the receptor residency time of our molecules to make them resistant to competition by the endogenous hormone, allowing us to treat all patients, including those with very high ACTH levels

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