Neurocrine Biosciences Presents New Analysis of CAHtalog® Registry Showing Most Patients with Classic Congenital Adrenal Hyperplasia Experienced High Glucocorticoid Exposure, Suboptimal Disease Control or Both at ENDO 2025

• Approximately 95% of adult and pediatric patients were exposed to supraphysiologic doses of glucocorticoids and/or experienced elevated androstenedione levels at least once during their treatment journey
• Real-world findings underscore long-term challenges of managing classic congenital adrenal hyperplasia and highlight clinical need for therapies to reduce androgen excess without high glucocorticoid doses

SAN DIEGO, July 15, 2025 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced results from a new analysis using real-world data from CAHtalog®, a comprehensive registry of patients with classic congenital adrenal hyperplasia in the United States. The analysis demonstrated significant variability in glucocorticoid treatment regimens and frequent changes in health status and outcomes over time in both pediatric and adult patients. These results were presented at the Endocrine Society's Annual Meeting, ENDO 2025, in San Francisco.

"The CAHtalog data revealed just how dynamic and unpredictable disease control can be for people with classic congenital adrenal hyperplasia, with most experiencing elevated glucocorticoid exposure and/or loss of androgen control at multiple points throughout their lives," said Sanjay Keswani, M.D., Chief Medical Officer, Neurocrine Biosciences. "This analysis emphasizes that achieving disease control today, often with the use of high-dose glucocorticoids, offers no guarantee of maintaining disease control long term and underscores the ongoing complexity of managing classic congenital adrenal hyperplasia over a lifetime."

While glucocorticoid (GC) therapy is essential to replace cortisol deficiency in patients with classic congenital adrenal hyperplasia (CAH), achieving hormonal control using GC therapy alone requires supraphysiologic doses to reduce elevated adrenocorticotropic hormone (ACTH) and androgen levels. Developed in partnership with the CARES Foundation, the CAHtalog (Congenital Adrenal Hyperplasia: Patient and Clinical Outcomes in Real-World Practice Settings) registry was established to characterize longitudinal treatment patterns, natural history and impact of CAH. This latest analysis specifically examined patterns of GC use and androstenedione (A4) levels over time in pediatric and adult participants.

In this most recent analysis, data were examined from 98 patients, including 37 adults, 42 children and adolescents and 19 individuals who contributed data as both children and adults. Importantly, this analysis was conducted using data collected prior to the commercial availability of CRENESSITY® (crinecerfont) and does not include any CRENESSITY treatment data. GC treatment records were paired with the nearest available A4 laboratory values. GC doses were classified as "higher" (pediatric: >11 mg/m2/d hydrocortisone equivalents [HCe]; adult: >20 mg/d HCe) or "lower" (pediatric: ≤11 mg/m2/d HCe; adult ≤20 mg/d HCe). A4 levels were categorized as "higher" (≥upper limit of normal [ULN]) or "lower" (