Definition

An orally bioavailable, non-steroidal, potent, reversible, dual inhibitor of cytochrome P450 17 (CYP17 or CYP17A1) and CYP11B2, with potential antiandrogen and antineoplastic activities. Upon oral administration, LAE001 inhibits the enzymatic activity of CYP17A1 in both the testes and adrenal glands, thereby inhibiting androgen production. This may decrease androgen-dependent growth signaling and may inhibit cell proliferation of androgen-dependent tumor cells. LAE001 also inhibits the enzymatic activity of CYP11B2, thereby inhibiting aldosterone production. This may reduce the elevated aldosterone levels resulting from CYP17 inhibition and androgen deprivation, leading to a reduction in mineralocorticoid side effects including cardiovascular complications. The cytochrome P450 enzyme CYP17A1, localized to the endoplasmic reticulum, exhibits both 17alpha-hydroxylase and 17,20-lyase activities, and plays a key role in the steroidogenic pathway that produces steroidal hormones. The cytochrome P450 enzyme CYP11B2, aldosterone synthase, is an enzyme that plays a key role in aldosterone biosynthesis.