Neurocrine Biosciences, Inc. NBIX announced today that NBI-77860, a proprietary corticotropin releasing
factor 1 (CRF) receptor antagonist, has entered clinical trials for the
treatment of classic congenital adrenal hyperplasia (CAH) a disease that
affects approximately 20,000-30,000 people in the United States. The Company
has successfully completed a pilot clinical trial in adults with classic CAH
and is initiating an open-label, single ascending dose trial in approximately
fifteen adolescent females with classic CAH, the 1401 Study.
"We are very pleased with the results from our pilot study of NBI-77860 in
patients that was conducted earlier this year and excited to add another
significant program to our clinical development pipeline," said Christopher F.
O'Brien, Chief Medical Officer of Neurocrine Biosciences. "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."
"I am very pleased with the data generated from this initial exploratory
study; it looks very promising," said Dr. Richard Auchus, Professor of
Internal Medicine, Division of Metabolism, Endocrinology & Diabetes at
University of Michigan Health System. "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."
Results from the Initial Pilot Clinical Study
The pilot clinical trial was a blinded, single-site,
pharmacokinetic/pharmacodynamic study assessing two single, ascending doses of
NBI-77860 against placebo in adult females with refractory CAH. The eight
study participants visited the investigative site for three separate overnight
visits consisting of bedtime dosing with placebo or one of two active doses of
NBI-77860. Each of the visits was separated by a three-week washout period.
Key biomarker measurements included adrenocorticotropic hormone (ACTH),
17-hydroxyprogesterone (17-OHP), androgen, and cortisol levels collected in
the morning after dosing. Data from this initial single dose exploratory study
demonstrated a robust decrease in ACTH and 17-OHP.
A full description of the study results and related data will be presented at
an upcoming scientific meeting.
1401 Study Design
The 1401 study is a Phase I/II open-label, sequential cohort, single ascending
dose pharmacokinetic/pharmacodynamic study assessing three doses of NBI-77860.
The fifteen adolescent females with classic CAH will be split into three
cohorts and each will receive one dose of NBI-77860 at bedtime. Biomarker
measurements include ACTH, 17-OHP, androgen, and cortisol levels collected the
morning after dosing.
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