The United States Food and Drug Administration (FDA) has granted orphan drug status to Neurocrine Biosciences' NBI-77860, a proprietary corticotropin releasing factor 1 (CRF) receptor antagonist, for the treatment of congenital adrenal hyperplasia (CAH) a disease that affects approximately 20,000-30,000 people in the United States.

"We are very pleased that the FDA has granted NBI-77860 orphan status to treat congenital adrenal hyperplasia, a devastating disease that is a significant challenge for both clinicians and patients," said Malcolm Lloyd-Smith, chief regulatory officer of Neurocrine Biosciences. "This status represents a significant regulatory milestone for the CAH programme and underscores the importance of bringing a safe and effective CAH therapy to market. We look forward to the results from our recently initiated 1401 Study of adolescents with classic CAH, in 2015."

Orphan Drug Designation is granted by the FDA Orphan Drug Designation program for medicines intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. Orphan Drug Designation provides sponsors with development and commercial incentives for such designated compounds and medicines.

Classic CAH is a genetic disorder that results in an enzyme deficiency altering the production of adrenal steroids. Because of this deficiency, the adrenal glands have little to no cortisol biosynthesis resulting in a potentially life-threatening condition. If left untreated, classic CAH can result in salt wasting, dehydration and eventually death. Even with cortisol replacement, persistent elevation of ACTH from the pituitary gland results in excessive androgen levels leading to virilization of females including precocious puberty, menstrual irregularity, short stature, hirsutism, acne and fertility problems.

Corticosteroids are the current standard of care for classic CAH which are used to both correct the endogenous cortisol deficiency and reduce the excessive ACTH levels and androgen excess. However, the dose and duration of steroid use required to suppress ACTH is well above the normal physiological level of cortisol resulting in metabolic syndrome, bone loss, growth impairment, and Cushing's syndrome as common and serious side effects.

NBI-77860 is a potent, selective non-peptide CRF receptor antagonist as demonstrated in a range of in vitro/in vivo assays and human clinical studies. Blockade of CRF receptors at the pituitary has been shown to decrease the release of ACTH, which in turn decreases the production of adrenal steroids including androgens, and potentially the symptoms associated with classic CAH. Lower ACTH levels would also reduce the amount of exogenous corticosteroid necessary for classic CAH patients to thrive avoiding the side-effects currently associated with excessive steroid therapy.

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 day after dosing.

Neurocrine Biosciences, Inc. discovers and develops innovative and life-changing pharmaceuticals, in diseases with high unmet medical needs, through its novel R&D platform, focussed on neurological and endocrine based diseases and disorders.  The Company's two lead late-stage clinical programs are elagolix, a gonadotropin-releasing hormone antagonist for women's health that is partnered with AbbVie Inc., and a wholly owned vesicular monoamine transporter 2 inhibitor for the treatment of movement disorders.  Neurocrine intends to maintain certain commercial rights to its VMAT2 inhibitor for evolution into a fully-integrated pharmaceutical company.

In addition to historical facts, this press release may contain forward-looking statements that involve a number of risks and uncertainties. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties associated with Neurocrine's business and finances in general, as well as risks and uncertainties associated with the Company's CRF program and Company overall. Specifically, the risks and uncertainties the Company faces with respect to the Company's CRF program include, but are not limited to; risk that NBI-77860 will not proceed to later stage clinical trials and risk that the Company's clinical trials will fail to demonstrate that NBI-77860 is safe and effective, risk that NBI-77860 may not replicate the results observed in our prior clinical development, risk that NB.