- Neurocrine Biosciences (NBIX, Financial) showcases promising Phase 3 data for CRENESSITY™ (crinecerfont) in CAH treatment.
- CRENESSITY achieves 90% response rate among pediatric patients for hormonal balance versus 21% on placebo.
- Significant improvements in reproductive hormones observed in adult males with classic congenital adrenal hyperplasia.
Neurocrine Biosciences (NBIX) has presented new data highlighting the efficacy of CRENESSITY™ (crinecerfont) in treating classic congenital adrenal hyperplasia (CAH). The findings were disclosed during the 2025 American Association of Clinical Endocrinology Annual Meeting in Orlando.
In pediatric patients, 90% on CRENESSITY achieved targets for androstenedione reduction or glucocorticoid dose reduction, a notable improvement compared to just 21% on placebo. Additionally, 30% of these patients reached physiologic glucocorticoid doses while maintaining or improving their androstenedione levels, an outcome not achieved by any participants on placebo.
Adult males with CAH exhibited remarkable progress in reproductive hormone normalization. At Month 12, 65% of adult males experienced normalization in luteinizing hormone levels, while 24% achieved normalization in the androstenedione-to-testosterone ratio, all while glucocorticoid doses were substantially reduced.
Despite the overall positive results, two adult participants (1.6%) experienced adrenal crisis while on CRENESSITY, highlighting a rare but serious potential risk. The most common adverse effects reported included headache (25%), fatigue (25%), and abdominal pain (13%).
CRENESSITY's unique approach as a corticotropin-releasing factor type 1 receptor antagonist marks a significant advancement in CAH treatment, providing a dual mechanism that reduces excess androgens and enables a reduction in glucocorticoid doses. These findings position CRENESSITY as a transformative option for addressing the longstanding challenges associated with CAH, potentially setting a new standard of care in this domain.
