- Neurocrine Biosciences (NASDAQ: NBIX) presented one-year data from Phase 3 CAHtalyst studies highlighting the efficacy of CRENESSITY® (crinecerfont) in managing weight-related outcomes for classic congenital adrenal hyperplasia (CAH) patients. - The study included 103 pediatric and 182 adult patients, showing significant improvements in body mass index (BMI) and insulin resistance, with a favorable safety profile. - 27% of overweight/obese adults achieved more than a 5% weight reduction by month 12, and 27% of pediatric patients achieved at least a 0.2 reduction in BMI SDS.
Neurocrine Biosciences, Inc. (NASDAQ: NBIX) released promising results from its Phase 3 CAHtalyst program at the Endocrine Society's Annual Meeting, ENDO 2025, held in San Francisco. The data demonstrated that CRENESSITY® (crinecerfont) led to significant improvements in weight-related outcomes in patients with classic congenital adrenal hyperplasia (CAH) over the span of one year.
The trials enrolled 103 pediatric patients aged between 4 and 17 years, alongside 182 adult patients aged 18 to 58. Key findings highlighted included a substantial reduction in body mass index (BMI) and insulin resistance across both demographics. Specifically, 27% of adults achieved a weight reduction of more than 5% by the end of the 12-month period, while 27% of pediatric patients realized at least a 0.2 reduction in BMI standard deviation scores (SDS).
CRENESSITY® was noted for its favorable safety profile, with side effects being mostly mild to moderate and transient. Commonly reported adverse effects in adults included fatigue, headache, and dizziness, whereas children experienced headaches, stomach pain, and tiredness.
Dr. Sanjay Keswani, Chief Medical Officer at Neurocrine Biosciences, emphasized that the use of CRENESSITY® in conjunction with glucocorticoid therapy is setting new standards in the management of classic CAH by significantly improving cardiometabolic outcomes while allowing for reduced steroid doses.
