Based on Preclinical Observations, Two Clinical Studies of NBI-77860 Halted by the Company Prior to Patient Enrollment
"Out of an abundance of caution, we halted the implementation of these studies and notified the
The two clinical studies that were halted include a single dose study in adolescent females with classic congenital adrenal hyperplasia and a multiple dose study in adults with classic congenital adrenal hyperplasia. The Company had not enrolled any subjects in either study and accordingly, there have been no adverse events reported.
The Company has also been informed by the
About Classic Congenital Adrenal Hyperplasia (CAH)
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.
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 product candidate NBI-77860 in general, including the risk that NBI-77860 will not be found to be safe and effective. Specifically, the risks and uncertainties the Company faces for NBI-77860 include risks that development activities may not be completed on time or at all; risks that clinical development activities may be delayed for regulatory or other reasons, may not be successful or replicate previous clinical trial results, or may not be predictive of real-world results or of results in subsequent clinical trials; risks that regulatory submissions may not occur or be submitted in a timely manner; risk that NBI-77860 candidates may not obtain regulatory approval; or that the