NBI-77860 is a potent, selective non-peptide CRF receptor antagonist as showed in a range of in vitro/in vivo assays and human clinical trials.
Blockade of CRF receptors at the pituitary has been shown to decrease the release of adrenocorticotropic hormone (ACTH), which in turn decreases the production of adrenal steroids including androgens, and potentially the symptoms related with classic CAH.
According to the company, lower ACTH levels would also reduce the amount of exogenous corticosteroid needed for classic CAH patients to thrive avoiding the side-effects currently associated with excessive steroid therapy.
Recently, the company has initiated a open-label, sequential cohort, single ascending dose pharmacokinetic/pharmacodynamic Phase I/II trial (1401 study) designed to evaluate three doses of NBI-77860.
Neurocrine Biosciences chief regulatory officer Malcolm Lloyd-Smith said: "This status represents a significant regulatory milestone for the CAH program 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."
The trial will include 15 adolescent females with classic CAH who will be split into three groups and each will be given one dose of NBI-77860 at bedtime.