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Neurocrine Biosciences launches trial for congenital adrenal hyperplasia therapy
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After completing a pilot clinical trial in adults with classic congenital adrenal hyperplasia, Neurocrine Biosciences, Inc. announced a trial for its proprietary corticotropin releasing factor 1 receptor antagonist in adolescents, according to a statement from the company.
The open-label, sequential cohort, single-ascending dose trial, known as the 1401 Study, for the drug NBI-77860 will involve approximately 15 adolescent girls with classic congenital adrenal hyperplasia (CAH), according to the statement.
“I am very pleased with the data generated from this initial exploratory study; it looks very promising,” said Richard Auchus, MD, PhD, of the Division of Metabolism, Endocrinology and Diabetes, University of Michigan Health System, said in the statement. “This treatment strategy, if validated in future clinical trials, has the potential to revolutionize the treatment for CAH patients and make an important difference in their lives.”
The blinded, single-site, pharmacokinetic/pharmacodynamic pilot trial compared two single-ascending doses of NBI-77860 vs. placebo in eight adult females with refractory CAH. During three separate overnight visits at an investigative site, patients received placebo or one of two active doses of NBI-77860 at bedtime.
Visits were separated by 3-week washout periods. Key biomarkers adrenocorticotropic hormone (ACTH), 17-hydroxyprogesterone (17-OHP), androgen and cortisol levels were collected in the morning after dosing. A robust decrease in ACTH and 17-OHP were observed.
The 1401 phase 1/2 pharmacokinetic/pharmacodynamic study assesses three doses of NBI-77860 among three cohorts; each patient receives a single dose of NBI-77860 at bedtime. ACTH, 17-OHP, androgen and cortisol levels will also be collected the morning after dosing.
“We have requested orphan drug status from the FDA while we continue to expand our preclinical and clinical efforts around NBI-77860 in classic CAH,” Christopher F. O’Brien, Chief Medical Officer of Neurocrine Biosciences, said in the statement.