Endocrine Abstracts

MLE4901 (previously AZD4901) is a potent and selective neurokinin 3 receptor (NK3R) antagonist being developed for the treatment of polycystic ovary syndrome (PCOS). Clinical studies indicate the compound negatively regulates the hypothalamus-pituitary-gonadotropin (HPG) axis to reduce pituitary luteinizing hormone and gonadal sex steroids. To understand the longer-term in vivo effects of MLE4901, a 13-week safety study was carried out. Groups of 3 male and female dog...

ATR-101 is a selective Acyl-CoA: cholesterol acyltransferase 1 (ACAT1) inhibitor in development for the treatment of diseases of the adrenal cortex including rare endocrine diseases, such as congenital adrenal hyperplasia (CAH) and Cushing’s syndrome (CS), and in adrenocortical carcinoma (ACC). ATR-101 has been shown to inhibit adrenal steroidogenesis at low doses and cause apoptosis at high doses. To better understand the adrenal-specific effects of ATR-101, in vivo<...

Cushing’s syndrome (CS) in humans shares many similarities with its counterpart in dogs in terms of etiology (pituitary versus adrenal causes), clinical signs, and pathophysiologic sequelae. ATR-101 is a novel small molecule therapeutic currently in clinical development for the treatment of congenital adrenal hyperplasia and adrenocortical carcinoma in humans. ATR-101 is an adrenal-selective inhibitor of ACAT1 (acyl coenzyme A:cholesterol acyltransferase 1). ACAT1 catalyz...