Clinical Trial Initiated to Evaluate Cushing’s Syndrome Drug

A Phase 2 clinical trial evaluating the safety and efficacy of a novel oral drug candidate for patients with endogenous Cushing’s syndrome was announced by the drug’s manufacturer, Millendo Therapeutics, Inc.

The drug, ATR-101,  is an adrenal-selective small molecule inhibitor of ACAT1. It is also being studied in an ongoing Phase 2 clinical trial in patients with classic congenital adrenal hyperplasia (CAH) and a Phase 1 clinical trial in patients with adrenocortical carcinoma (ACC).

“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,” according to Endocrine Society member Julia C. Owens, PhD, president and CEO of Millendo. “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 orally-administered ATR-101 in patients with endogenous Cushing’s syndrome. The primary efficacy endpoint will assess the impact of ATR-101 on urinary free cortisol (UFC) concentration, and secondary endpoints will evaluate the impact of ATR-101 on adrenal steroids/steroid intermediates and adrenocorticotropic hormone (ACTH).

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

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