Neurocrine Biosciences Reports Expansion of Clinical Pipeline

Neurocrine Biosciences, Inc.

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.