US-based Millendo Therapeutics has commenced the multicentre, single-blind, multiple dose Phase II clinical trial of its new oral drug candidate, ATR-101, to treat classic congenital adrenal hyperplasia (CAH).

Orally-administered ATR-101 is an adrenal-selective small molecule that inhibits the protein coding gene, Acetyl-CoA Acetyltransferase 1 (ACAT1), and helps in reducing adrenal steroid production.

The Phase II trial aims at evaluating the efficacy and safety of ATR-101, in addition to corticosteroids, in patients suffering from classic CAH resulting from 21-hydroxylase deficiency.

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

The trial focuses on determining an effective dose or range of doses of ATR-101 with the primary efficacy endpoint, evaluating the impact of the drug on adrenal steroid or steroid intermediate production as measured by serum 17 denoting hydroxyprogesterone concentration.

Millendo president and CEO Julia Owen said: “The initiation of this Phase II 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.

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

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It is reported that at a high dosage, ATR-101 triggers apoptosis of cells derived from the adrenal cortex.

University of Michigan Internal Medicine and Pharmacology Professor Richard Auchus said: “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.”

ATR-101 is currently undergoing clinical development to be applied for the treatment of adrenocortical carcinoma (ACC) and congenital adrenal hyperplasia (CAH), with planned development in endogenous Cushing’s syndrome (CS).