Neurocrine presents real-world CAH patient journey data at nursing symposium

Neurocrine Biosciences (Nasdaq: NBIX) presented real-world data at the Endocrine Nurses Society Annual Symposium in Phoenix on Oct. 16 showing how the clinical and daily impact of classic congenital adrenal hyperplasia (CAH) varies among individuals and changes throughout a patient's life.

The analysis drew on de-identified electronic medical records from 10 adults in the CAHtalog registry, a U.S.-based CAH patient registry developed by Neurocrine in partnership with the CARES Foundation. The dataset comprised more than 2,800 CAH-relevant events representing more than 300 patient-years of medical history. Large language model-assisted analysis was used to construct patient journeys, which were then reviewed and validated by the study team.

Patient records showed that challenges began in infancy with diagnosis and treatment, progressed to growth and bone-age monitoring in childhood, and expanded to puberty-related symptoms and reproductive concerns in adolescence and young adulthood. In adulthood, patients faced a wider range of health concerns along with increased imaging and testing needs. Glucocorticoid dose adjustments and stress dosing were documented across all 10 patients throughout their lives.

Records also captured impacts beyond clinical complications, including disruptions to school, work, and daily activities. The analysis identified some sex-specific findings, including menstrual concerns among female patients and testicular adrenal rest tumor concerns among male patients.

The company noted the analysis was descriptive and qualitative, with some observations based on one or two individuals, and the findings should not be interpreted as prevalence estimates or evidence of causality.

Neurocrine also presented two-year results from its CAHtalyst pediatric and adult studies at the same symposium, examining outcomes associated with crinecerfont, sold under the brand name Crenessity, which received FDA approval in December 2024 for the treatment of classic CAH.

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