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Millendo Therapeutics Announces Initiation of Phase 2 Clinical Trial of ATR-101 in Patients with Endogenous Cushing's Syndrome


Therapeutics, Inc.
, a clinical-stage biopharmaceutical company
focused on developing novel treatments for endocrine diseases caused by
hormone dysregulation, today announced the initiation of a Phase 2
clinical trial evaluating the safety and efficacy of a novel oral drug
candidate, ATR-101, in patients with endogenous Cushing's syndrome (CS),
a rare endocrine disease characterized by increased cortisol production
from the adrenal glands. ATR-101, an adrenal-selective small molecule
inhibitor of ACAT1, 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," said Julia C. Owens, Ph.D., President and Chief Executive
Officer 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 6-week open-label intra-subject dose-escalation period and a 4-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
identifier number NCT03053271.

About Endogenous Cushing's Syndrome

Endogenous Cushing's syndrome (CS) 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. Untreated CS can be
life-threatening, and many cases can be cured through surgery, but for
those cases that are refractory to surgery, medical management can be
challenging. New broad-acting agents are needed, as currently approved
treatments are only indicated for subsets of CS patients and are limited
by side effects.

About ATR-101

ATR-101, an adrenal-selective small molecule inhibitor of ACAT1, reduces
adrenal steroid production, and, at high doses, induces apoptosis of
cells derived from the adrenal cortex. ATR-101 is currently in clinical
development for the treatment of congenital adrenal hyperplasia (CAH),
endogenous Cushing's syndrome (CS), and adrenocortical carcinoma (ACC).

About Millendo Therapeutics, Inc.

Millendo Therapeutics is focused on developing novel treatments for
endocrine diseases caused by hormone dysregulation. Our mission is to
build a leading endocrine company that creates distinct and
transformative treatments for a wide range of diseases where there is a
significant unmet medical need. We are advancing two product candidates
in five indications: MLE4901, designed to address Polycystic Ovary
Syndrome (PCOS) and Vasomotor Symptoms (VMS), and ATR-101 for the
treatment of Classic Congenital Adrenal Hyperplasia (CAH), Endogenous
Cushing's Syndrome (CS), and Adrenocortical Carcinoma (ACC).

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