Millendo Starts Phase 2 Trial Of New Drug For Cushing’s Syndrome

ANN ARBOR — The Ann Arbor pharmaceutical developer Millendo Therapeutics Inc. has announced the initiation of a Phase 2 clinical trial evaluating the safety and efficacy of its drug candidate ATR-101 in patients with endogenous Cushing’s syndrome.

Endogenous Cushing’s syndrome is a rare endocrine disorder characterized by increased cortisol production from the adrenal glands, which is the result of a pituitary, adrenal or ectopic tumor. The chronic cortisol excess in Cushing’s syndrome can cause a multitude of issues for the patient such as weight gain, fatigue, hypertension, diabetes, bone loss, and neurologic symptoms. With chronic exposure to higher than normal levels of cortisol, patients may also exhibit cognitive impairment and mood disorders. In men, symptoms can also include decreased fertility and erectile dysfunction, while women may demonstrate menstrual irregularities and facial hair growth.

Endogenous CS affects approximately 20,000 people in the United States. It most commonly affects people who are 20 to 50 years of age, and women are three times more likely than men to have CS.

ATR-101 is also being studied in an ongoing Phase 2 clinical trial in patients with classic congenital adrenal hyperplasia (CAH), which can cause sexual and growth abnormalities, and a Phase 1 clinical trial in patients with adrenocortical carcinoma (ACC), a rare cancer of the adrenal gland.

“The initiation of this Phase 2 clinical trial of ATR-101 in endogenous CS marks an important milestone in the development of this product candidate in endocrine diseases where current therapies are insufficient,” said Julia C. Owens, Ph.D., Millendo president and CEO. “There are numerous etiologies of CS and, in patients for whom surgical intervention is not successful, current treatment options are limited. We are hopeful that the adrenal-selective effects of ATR-101 will provide a new treatment option for patients with endogenous CS, regardless of the underlying causes.”

The Phase 2 clinical trial is a two-part multicenter study consisting of a six-week open-label intra-subject dose-escalation period and a four-week double-blind randomized withdrawal period to assess the efficacy and safety of ATR-101 in patients with endogenous Cushing’s syndrome.

The multicenter, international study will be conducted at sites in the United States and the United Kingdom. For additional information on this clinical trial, please visit, identifier number NCT03053271.

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.

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