Neurocrine Biosciences Reports Positive Interim Results from Phase II Study of NBI-74788 in Adults with Classic Congenital Adrenal Hyperplasia
"Patients with classic CAH currently have limited treatment options besides more and more glucocorticoids. The management of their genetic disorder is complex due to the highly variable clinical features and response to therapy, which also changes over time. Most patients receive supraphysiologic doses of glucocorticoids chronically to manage their disease, which frequently leads to serious long-term health consequences," said
Classic CAH is a rare genetic disorder caused by a deficiency of the 21-hydroxylase enzyme, which alters the production of cortisol and other adrenal steroids, leading to adrenal insufficiency, overgrowth of the adrenal glands, and excess androgen levels. Classic CAH can lead to adrenal crisis, virilization, hirsutism, precocious puberty, fertility problems and abnormal growth. The standard of care for classic CAH requires a lifelong regimen with high-dose corticosteroids, which cause additional serious long-term clinical problems, including bone loss, Cushing's syndrome and metabolic issues.
"We are very encouraged by the positive interim results of NBI-74788 in this Phase II proof-of-concept study demonstrating the pharmacological effects of CRF1 antagonism on key biomarkers in patients with CAH," said Eiry W. Roberts, M.D., Chief Medical Officer at
NBI-74788 Phase II Study Design
The NBI-74788 Phase II clinical study is an open-label, multiple-dose, dose-escalation study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of NBI-74788 in approximately 30 adults with a documented medical diagnosis of classic 21-hydroxylase deficiency CAH. The study includes a sequential-cohort design with three NBI-74788 dose cohorts, with each dose administered for 14 consecutive days.
NBI-74788 is a proprietary, potent, selective, orally-active, non-peptide corticotropin-releasing factor type 1 (CRF1) receptor antagonist under evaluation for the treatment of classic CAH. Blockade of CRF receptors in 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 associated with CAH. Research suggests that lowering ACTH levels could reduce the amount of corticosteroid treatment necessary for CAH patients to thrive and avoid the effects associated with long-term steroid therapy.
About Classic Congenital Adrenal Hyperplasia (CAH)
Classic CAH is a genetic disorder that results in an enzyme deficiency that alters 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 adrenocorticotropic hormone (ACTH) from the pituitary gland results in excessive androgen levels leading to virilization and menstrual irregularities in females; both males and females may also experience precocious puberty, short stature, hirsutism, acne and fertility problems. Classic CAH is a disease that affects approximately 20,000 to 30,000 people in
There are currently no non-steroidal
About Neurocrine Biosciences
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-74788 and the Phase II study in general, including the risk that NBI-74788 will not be found to be safe and/or effective, or that final results of the study will replicate the interim results. Specifically, the risks and uncertainties the Company faces for NBI-74788 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 and/or interim 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; risks that NBI-74788 may not obtain regulatory approvals; or that the
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Neurocrine Biosciences, Inc., Navjot Rai (Media & Investors), 858-617-7623, IR@neurocrine.com