- Neurocrine Biosciences' (NBIX, Financial) Phase 3 CAHtalyst Pediatric study shows CRENESSITY™ significantly reduces glucocorticoid doses while maintaining hormonal balance.
- CRENESSITY improved androstenedione levels, with a -6.9 nmol/L reduction at Week 4 versus a +2.5 nmol/L increase with placebo.
- The drug maintained a well-tolerated profile with common side effects such as headache and abdominal pain.
Neurocrine Biosciences (NBIX) has unveiled promising results from the Phase 3 CAHtalyst™ Pediatric study, highlighting the efficacy of their product CRENESSITY™ (crinecerfont) in managing classic congenital adrenal hyperplasia (CAH) in pediatric patients. The trial involved 103 participants, with 69 receiving CRENESSITY and 34 on placebo over a 28-week period.
Key findings revealed that CRENESSITY significantly reduced the levels of androstenedione—a pivotal adrenal androgen—by -6.9 nmol/L compared to an increase of +2.5 nmol/L in the placebo group at the four-week mark. Moreover, the study marked a noteworthy decrease in required glucocorticoid dosages, with a reduction of -18.0% relative to a +5.6% increase observed in the placebo group by Week 28.
Eiry W. Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences, commented on the impact stating, "By enabling patients to maintain or improve their androgen levels while reducing reliance on high-dose glucocorticoids, CRENESSITY has potential to meaningfully enhance long-term outcomes."
The comprehensive trial also indicated that CRENESSITY was generally well-tolerated among participants. Common adverse reactions included headache, abdominal pain, fatigue, nasal congestion, and nosebleed, occurring in over 4% of patients. These findings are set to be presented at the 2025 Joint Congress of the European Society for Paediatric Endocrinology and the European Society of Endocrinology in Copenhagen, Denmark.
