Ann Arbor Pharma Starts Trial Of Drug For Adrenal Disease

ANN ARBOR — The pharmaceutical startup Millendo Therpeutics Inc. announced the start of a Phase 2 clinical trial of its new drug candidate ATR-101 to treat classic congenital adrenal hyperplasia (CAH), a rare inherited endocrine disorder characterized by overgrowth of the adrenal glands.

ATR-101 is also being studied in an ongoing Phase 1 clinical trial in patients with adrenocortical carcinoma (ACC).

“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, president and CEO of Millendo. “CAH is a serious condition with significant unmet need, where we believe we can provide an important new treatment option.”

Added Richard J. Auchus, M.D., professor of internal medicine and pharmacology at the University of Michigan: “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. 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. For additional information on this clinical trial, visit

In Phase 1 trials, researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects. In Phase 2 trials, the drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety. And in Phase 3, the drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.

CAH is a rare inherited endocrine disorder 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 percent 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 — the development of male physical characteristics — in women, testicular tumors in men, and infertility.

The incidence of classic CAH is one every 10,000 to 20,000 births, and the current standard of care 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 (characterized by fatty deposits around the face and upper back, weight gain around the midsection, and fat loss from the arms and legs).

ATR-101reduces adrenal steroid production, and, at high doses, induces the death of cells derived from the adrenal cortex.

Millendo Therapeutics is focused on developing a portfolio of disease-modifying treatments for endocrine diseases caused by hormone dysregulation. Besides CAH, the company’s research is directed to treatments for Polycystic Ovary Syndrome (PCOS), the most common endocrine disease in women; Endogenous Cushing’s Syndrome (CS) – a condition resulting from chronic cortisol excess; and Adrenocortical Carcinoma (ACC) – a rare endocrine malignancy of the adrenal cortex.

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