Neurocrine will present a new post hoc analysis of Phase 2 data of the investigational drug crinecerfont in adolescent patients with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency. The analysis suggests that adolescents with classic CAH who have more elevated baseline hormone levels may have the potential for a greater response to treatment with crinecerfont and may experience a reduction in androgen levels across a broad range of glucocorticoid doses. These new data will be presented at the 61st Annual European Society for Pediatric Endocrinology Meeting in The Hague Sept. 21-23. A strong correlation was found between baseline hormone concentration and change from baseline to Day 14 for 17-OHP, ACTH and A4, with the greatest reductions observed in participants with the highest baseline hormone levels. These data suggest adolescents with higher baseline hormone concentrations may have the potential for a greater response to treatment with crinecerfont. However, there was no correlation between baseline GC dose and treatment response, which suggests that androgen reduction might occur across a broad range of GC doses in this population. In the Phase 2 study, crinecerfont was generally well tolerated in adolescents, with no serious adverse events or discontinuations due to adverse events.
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