Neurocrine reports two-year Phase 3 CAHtalyst Pediatric data showing sustained hormone control, reduced glucocorticoid use, and improved metabolic outcomes with crinecerfont in pediatric CAH.
Written By: Dr. Preethi Putti, PharmD
Reviewed By: Pharmacally Editorial Team
Neurocrine Biosciences has reported new two-year data from the Phase 3 CAHtalyst Pediatric study (NCT04806451) showing sustained hormone control, reduced glucocorticoid exposure, and improved metabolic outcomes in children and adolescents with classic congenital adrenal hyperplasia (CAH).
The results, presented at the Pediatric Endocrine Society 2026 Annual Meeting, extend previously reported one-year findings and are based on 86 patients aged 4–17 years who continued treatment in the open-label extension of the trial.
Sanjay Keswani M.D., Chief Medical Officer, Neurocrine Biosciences noted that treatment with CRENESSITY (crinecerfont) over two years led to sustained reductions in androgen levels and glucocorticoid use in pediatric CAH, with improved hormonal control linked to better clinical outcomes, including body mass index and insulin resistance.
Treatment maintained reductions in key hormonal markers while enabling lower glucocorticoid dosing. Mean adrenocorticotropic hormone (ACTH) levels decreased by 157 pg/mL from a baseline of 329 pg/mL, and 17-hydroxyprogesterone (17-OHP) levels declined by 1,924 ng/dL from 8,682 ng/dL, measured pre-dose. Mean daily glucocorticoid dose was reduced by 3.2 mg/m²/day, supporting a shift toward more physiologic replacement without loss of disease control.
Clinical manifestations of androgen excess improved. Acne severity decreased over time, and among males with elevated androstenedione-to-testosterone ratios at baseline, 36% achieved normalization by Month 24. In female patients, hirsutism scores remained largely stable despite reduced steroid exposure and progression through puberty.
Metabolic parameters also improved. Among patients who were overweight or obese at baseline, 60% achieved clinically meaningful reductions in BMI, and 23% reached a BMI below the 85th percentile. Among those with insulin resistance at baseline, 61% no longer met criteria for insulin resistance after two years.
CRENESSITY was generally well tolerated, with more than 80% of patients remaining on treatment at two years and no new safety signals observed.
Mimi Kim, principal investigator of the CAHtalyst Pediatric study, stated that in pediatric CAH, excess androgens and prolonged high-dose glucocorticoid use can negatively affect growth, cardiometabolic health, and quality of life. She added that sustained androgen control with reduced glucocorticoid dosing using crinecerfont may support improved long-term outcomes.
Neurocrine previously reported two-year data from the CAHtalyst Adult study showing sustained glucocorticoid dose reductions while maintaining androgen control in adults with classic CAH.
CRENESSITY is an oral corticotropin-releasing factor type 1 (CRF-1) receptor antagonist that reduces ACTH and adrenal androgen production through a non-glucocorticoid mechanism. Classic CAH, most commonly due to 21-hydroxylase deficiency, is characterized by impaired cortisol production and androgen excess and is typically managed with glucocorticoids that often require supraphysiologic doses and are associated with long-term complications.
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About the Writer
Dr.Preethi Putti, PharmD is a pharmaceutical researcher with experience in healthcare and pharmaceutical market research and competitive intelligence. She specializes in analyzing drug pipelines, clinical data, and industry trends and translating complex scientific data into clear and structured medical content. Strong foundation in clinical research, data interpretation, and evidence-based healthcare analysis. Committed to advancing a global career in clinical research and healthcare innovation.