New Phase 2 Trial for Congenital Adrenal Hyperplasia Drug Initiated

A new phase 2 clinical trial to test the efficacy of a new drug to treat the rare inherited disorder congenital adrenal hyperplasia (CAH) will soon be underway.

CAH is caused by a steroidogenic enzyme deficiency that is characterized by overgrowth of the adrenal glands, adrenal insufficiency, and androgen excess. The most frequent form of CAH, responsible for 95% of cases, is a deficiency in the enzyme 21-hydroxylase, which is required for the production of cortisol in the adrenal glands. CAH can lead to severe virilization in females, testicular tumors in men, and infertility.

Millendo Therapeutics, Inc. announced the initiation of the new trial to test the effects of a novel oral drug, ATR-101, in patients with classic CAH which affects anywhere from one in 10,000 to 20,000 births. ATR-101, an adrenal-selective small molecule inhibitor of ACAT1, is also being studied in an ongoing Phase 1 clinical trial in patients with adrenocortical carcinoma (ACC).

ATR-101 is an adrenal-selective small molecule inhibitor of ACAT1 that reduces adrenal steroid production, and, at high doses, induces apoptosis of cells derived from the adrenal cortex. The drug is currently in clinical development for the treatment of adrenocortical carcinoma and CAH, with planned development in endogenous Cushing’s syndrome.

“The initiation of this Phase 2 clinical trial of ATR-101 in classic CAH marks an important milestone in Millendo’s development and is the first of several planned trial initiations for this year as we build a portfolio of novel treatment options for endocrine diseases,” said Julia C. Owens, PhD, Millendo’s president and CEO and an Endocrine Society member, in a statement from the company. “CAH is a serious condition with significant unmet need, where we believe we can provide an important new treatment option.”

The current standard of care for classic CAH is treatment with corticosteroids, which are used to correct the endogenous cortisol deficiency and reduce androgen excess. However, high doses of corticosteroids are required to suppress androgens which can result in serious long-term side effects such as bone loss, growth impairment, and Cushing’s syndrome.

“We look forward to exploring the potential of ATR-101 in classic CAH patients in this Phase 2 clinical trial,” said Endocrine Society member Richard J. Auchus, MD, PhD, professor of internal medicine and pharmacology at the University of Michigan, in the same statement. “A great need exists for alternative treatment options, as the current standard of care for CAH can result in serious long-term side effects, including bone loss, growth impairment and Cushing’s syndrome. Based on the mechanism of action for this compound, we are optimistic that addition of ATR-101 to physiologic doses of corticosteroids can provide improved outcomes for these patients.”

The Phase 2 clinical trial is a multicenter, single-blind, multiple dose study that will assess the efficacy and safety of orally administered ATR-101, in addition to corticosteroids, in patients with classic congenital adrenal hyperplasia resulting from 21-hydroxylase deficiency. The study is designed to determine an effective dose or range of doses with the primary efficacy endpoint assessing the impact of ATR-101 on adrenal steroid/steroid intermediate production as measured by serum 17-hydroxyprogesterone concentration.

For additional information on this clinical trial, please visit


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