ANN ARBOR, Mich.--(BUSINESS WIRE)--Millendo Therapeutics, Inc., a company developing novel therapies for endocrine diseases caused by hormone dysregulation, today announced the online publication of preclinical data on the mechanism of action of ATR-101, a novel, oral drug candidate in development for the treatment of adrenocortical carcinoma (ACC). The studies reported in the manuscript were completed as part of an ongoing collaboration with the laboratory of Dr. Gary Hammer at the University of Michigan. The article titled, “ATR-101, a Selective and Potent Inhibitor of Acyl-CoA Acyltransferase 1, Induces Apoptosis in H295R Adrenocortical Cells and in the Adrenal Cortex of Dogs,” was published in the journal Endocrinology.
As reported in the manuscript, studies demonstrated that selective inhibition of acyl-coenzyme A:cholesterol O-acyltransferase 1 (ACAT1) by ATR-101 increased free cholesterol levels in the H295R human ACC cell line, resulting in dysregulation of endoplasmic reticulum (ER) calcium stores, ER stress, activation of the unfolded protein response, and apoptosis. In an in vivo preclinical model, treatment with ATR-101 decreased adrenocortical steroid production and induced cellular apoptosis that was restricted to the adrenal cortex.
“These studies provide a deeper understanding of the mechanism of action of ATR-101 and its adrenal-specific effects in vivo,” said Julia C. Owens, Ph.D., President and Chief Executive Officer of Millendo. “Based on these data and other preclinical studies to date, we believe that ATR-101 has strong potential to have a positive impact in the treatment of rare endocrine diseases of the adrenal cortex, including congenital adrenal hyperplasia (CAH), Cushing’s syndrome, and adrenocortical carcinoma (ACC).”
The company also announced that Stephen W. Hunt, III, Ph.D., Chief Scientific Officer, will present preclinical data on ATR-101 in a poster preview presentation and poster session at The Endocrine Society’s 98th Annual Meeting & Expo, being held April 1-4 at the Boston Convention and Exhibition Center in Boston, Massachusetts.
Details of the presentations are as follows:
Title: ATR-101, a Selective ACAT1 Inhibitor, Demonstrates Adrenal-Specific Effects in a 13-Week Dog Study
Poster Preview: PP13-3
Date/Time: Saturday, April 2, 2016, 11:30 AM-11:45 AM
Location: Room 160
Poster: SAT 401
Date/Time: Saturday, April 2, 2016, 1:15 PM-3:15 PM
Location: Hall AB1
About Millendo Therapeutics, Inc.
Millendo Therapeutics is focused on developing a portfolio of disease-modifying treatments for endocrine diseases caused by hormone dysregulation. Our product candidates seek to improve the quality of life for patients with orphan and specialty diseases with limited or no approved treatment options. Our clinical programs are designed to address:
- Polycystic Ovary Syndrome (PCOS) – the most common endocrine disease in women
- Adrenocortical Carcinoma (ACC) – a rare endocrine malignancy of the adrenal cortex
- Congenital Adrenal Hyperplasia (CAH) – a recessive genetic defect of cortisol synthesis
- Endogenous Cushing’s Syndrome (CS) – a condition resulting from chronic cortisol excess
Our experienced team is committed to bringing these first-in-class therapies to market.