Key takeaways:
- Crinecerfont enabled many youngsters with congenital adrenal hyperplasia (CAH) to realize reductions in glucocorticoid dose and androstenedione stage.
- The drug improved hormone ranges for adults with CAH.
ORLANDO — In sufferers with basic congenital adrenal hyperplasia, crinecerfont conferred significant reductions in glucocorticoid dose and androstenedione stage in youngsters and improved reproductive hormone ranges in adults, new information present.
New findings from the CAHtalyst pediatric research and the CAHtalyst grownup research of crinecerfont (Crenessity, Neurocrine Biosciences), which was authorized by the FDA in December, have been offered on the American Affiliation of Medical Endocrinology Annual Scientific and Medical Convention.
New information from the CAHtalyst pediatric and grownup research revealed advantages of crinecerfont for individuals with basic congenital adrenal hyperplasia.
CAHtalyst pediatric research
As Healio beforehand reported, within the CAHtalyst pediatric research, youngsters assigned crinecerfont have been extra more likely to have reductions in glucocorticoid dose and androstenedione stage than these assigned placebo at 28 weeks.
Within the presentation on the AACE Annual Scientific and Medical Convention, Patricia Y. Fechner, MD, medical director of Seattle Kids’s Congenital Adrenal Hyperplasia Middle and professor of pediatrics on the College of Washington Faculty of Medication, stated by week 4, 74% of the crinecerfont group had androstenedione ranges under the higher restrict of regular in contrast with 12% of the placebo group.
Patricia Y. Fechner
The proportion of sufferers attaining at the very least a 30% discount of androstenedione ranges between baseline and week 4 was 85% within the crinecerfont group and 18% within the placebo group, whereas the share bettering from at the very least 1.5 occasions the higher restrict of regular to under the higher restrict of regular was 61% within the crinecerfont group and 0% within the placebo group, she stated.
Those that had a discount in androstenedione ranges by week 4 have been extra more likely to have a discount in glucocorticoid dose by week 28 (crinecerfont, –18%; placebo, +6%; least squares imply distinction, –24 proportion factors; 95% CI, –30 to –17; P < .0001), she stated.
She additionally stated 30% of the crinecerfont group reached a physiologic dose of glucocorticoids at week 28 whereas sustaining or bettering androstenedione ranges, however no little one within the placebo group completed that.
A discount in glucocorticoid dose of at the very least 2.5 mg/m2 per day by week 28 occurred in 51% of the crinecerfont group and three% of the placebo group, whereas the share of sufferers who achieved that or a physiologic dose (outlined as < 11 mg/m2 per day) was 57% within the crinecerfont group and three% within the placebo group, Fechner stated.
The proportion of sufferers who achieved at the very least one threshold associated to glucocorticoid dose and/or androstenedione stage was 90% within the crinecerfont group and 21% within the placebo group, she stated.
“Crinecerfont was effectively tolerated, with few severe adversarial occasions and no adrenal disaster,” Fechner stated in the course of the presentation. “[The study] demonstrates that clinicians can deal with decreasing androgens, reducing glucocorticoid dose or each primarily based on the person affected person’s remedy objectives.”
CAHtalyst grownup research
In new information from the CAHtalyst grownup research, the crinecerfont group was extra more likely to have improved ranges of reproductive hormones than the placebo group, Sonal Vaid, MD, doctor scientist on the NIH Medical Middle, stated throughout a presentation.
At week 24 within the randomized section and 12 months within the open-label section, male sufferers assigned crinecerfont have been extra more likely to have normalization of luteinizing hormone than these assigned placebo (week 24, 47% vs. 22%; month 12, 65% vs. 44%), and at 12 months, male sufferers assigned crinecerfont have been extra more likely to have normalization of follicle-stimulating hormone (36% vs. 14%), she stated.
Amongst male sufferers with elevated androstenedione-to-testosterone ratio at baseline, the crinecerfont group was extra probably than the placebo group to realize normalization by week 24 (19% vs. 5%), whereas 24% in each teams achieved normalization at 12 months within the open-label research, she stated.
At 24 weeks, feminine sufferers within the crinecerfont group have been extra more likely to obtain normalization of testosterone ranges (11% vs. 0%) and progesterone ranges (14% vs. 6%), and a few sufferers from each teams achieved normalization at 12 months regardless of considerably diminished glucocorticoid doses, Vaid stated.
The most typical adversarial occasions related to crinecerfont have been headache and fatigue, she stated.
“Crinecerfont, a novel therapeutic choice for decreasing extra androgens and enabling the reducing of glucocorticoids to extra physiologic doses, may symbolize a promising method to bettering reproductive hormones in adults with [congenital adrenal hyperplasia],” Vaid stated in the course of the presentation.
Reference:
- Vaid S, et al. Crinecerfont improves reproductive hormones in basic congenital adrenal hyperplasia: 1-year outcomes from the CAHtalyst grownup research. Introduced at: American Affiliation of Medical Endocrinology Annual Scientific and Medical Convention; Might 15-17, 2025; Orlando.
Sources/Disclosures
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Fechner PY, et al. Crinecerfont permits for extra physiologic glucocorticoid remedy with higher reductions of androstenedione in pediatric sufferers with basic congenital adrenal hyperplasia: Analyses of particular person affected person information from the CAHtalyst pediatric research. Introduced at: American Affiliation of Medical Endocrinology Annual Scientific and Medical Convention; Might 15-17, 2025; Orlando.
Disclosures:
The research have been funded by Neurocrine Biosciences. Fechner studies receiving analysis grants from Diurnal Ltd. (now Neurocrine UK Ltd.), Neurocrine Biosciences and Spruce Biosciences; serving on an advisory board for Neurocrine Biosciences; and consulting for Eton Prescription drugs. Vaid studies no related monetary disclosures.
