Vivian Lin, MD, Executive Medical Director of Medical Affairs at Neurocrine, discusses the CAHtalyst clinical trials in adults with congenital adrenal hyperplasia (CAH).
CAH refers to a group of genetic conditions that affect the adrenal gland production of hormones, particularly, the production of cortisol and androgen hormones. Persons with severe forms of the condition can be dramatically impacted. For example, females with a severe form of the condition may have ambiguous genitalia at birth and if not properly diagnosed, develop dehydration, poor feeding, diarrhea, vomiting and other health problems soon after. People with milder forms may not be diagnosed with the condition until adolescence or adulthood when they experience early signs of puberty or fertility problems.
The CAHtalyst clinical trials were two phase 3 studies analyzing the changes in glucocorticoid doses with A4 levels in adult and pediatric patients with CAH. Crinecerfont is a first-in-class corticotropin releasing factor type 1 receptor antagonist approved by the U.S. Food and Drug Administration (FDA) as an adjunct to glucocorticoid replacement to control androgens in patients with CAH. Post hoc and one year results were presented on the CAHtalyst adult patients at the 2025 ENDO conference.
At baseline, all patients were taking supraphysiologic glucocorticoids. At week 24, 63% achieved a physiologic glucocorticoid dose (less than or equal to 11 mg/m2/d) while maintaining or improving A4 with crinecerfont, versus 18% in the placebo group. In the post hoc analysis, 50% had A4 levels higher than the upper limit of normal at baseline. At week 24, 82% of patients on crinecerfont achieved a physiologic glucocorticoid dose regardless of A4 level, compared to 37% in the placebo group. Of the crinecerfont patients, 53% had A4 levels less than or equal to the upper limit of normal, compared to 43% in the placebo group. Additionally, 49% of placebo-treated patients had A4 levels greater than the upper limit of normal despite supraphysiologic glucocorticoid dosing, compared to 11% with crinecerfont.
To learn more about CAH and other rare endocrine disorders, visit https://checkrare.com/diseases/endocrine-disorders/