Millendo Therapeutics has begun a Phase llb clinical trial to investigate the efficacy and safety of nevanimibe for the treatment of patients with classic congenital adrenal hyperplasia (CAH).

CAH is a rare inherited endocrine disease, which is the result of a genetic mutation preventing cortisol synthesis and is characterised by overgrowth of the adrenal glands, adrenal insufficiency, and other factors.

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The new open-label, intra-subject, dose-escalation trial aims to include up to 24 adult CAH patients who are on the current standard of care, glucocorticoids.

During the course of the trial, patients will be treated with nevanimibe for 12 consecutive weeks starting with a dose of 1,000mg BID.

Depending on 17 hydroxyprogesterone (17-OHP) levels, the dose will be increased to 1,500mg BID or 2,000mg BID.

“Our Phase llb trial will explore longer durations of treatment and higher doses of nevanimibe with the objective of enabling more patients to achieve hormonal control.”

The trial’s primary objective is to evaluate the percentage of patients that achieve 17-OHP levels less than or equal to two times the upper limit of normal (ULN).

Its secondary objectives comprise evaluation of other adrenal hormones, including androgens.

Millendo Therapeutics president and CEO Julia Owens said: “We are encouraged by the positive Phase ll efficacy and safety results of nevanimibe that were presented at the Endocrine Society’s annual meeting in March, in which these data demonstrated reductions in key steroids and steroid precursors, including promising reductions in 17-OHP, a key measure of disease control.

“Our Phase llb trial will explore longer durations of treatment and higher doses of nevanimibe with the objective of enabling more patients to achieve hormonal control.”

Nevanimibe, formerly ATR-101, is an adrenal-selective inhibitor of acylCoA:cholesterol acyltransferase 1 (ACAT1) enzyme that catalyses the transformation of free cholesterol into cholesterol ester.