Neurocrine Biosciences Reveals Promising CAH Data for Children

Neurocrine Biosciences Unveils Data from Pediatric Study

Neurocrine Biosciences, Inc. (NASDAQ: NBIX) has made a significant announcement regarding its beneficial effects of CRENESSITY™ (crinecerfont) on pediatric patients diagnosed with classic congenital adrenal hyperplasia (CAH). Recent data from the Phase 3 CAHtalyst™ Pediatric study showcases notable reductions in glucocorticoid doses and impressive improvements in various clinical outcomes. This groundbreaking data is set to be presented at the Pediatric Endocrine Society (PES) 2025 Annual Meeting.

Key Findings of the One-Year Data

The study revealed sustained reductions in glucocorticoid doses among pediatric patients treated with CRENESSITY for up to one year. Key hormonal levels, including adrenocorticotropic hormone, 17-hydroxyprogesterone, and androstenedione, were effectively maintained below baseline levels, despite the diminished glucocorticoid doses. Additionally, improvements in clinical outcomes, such as body mass index (BMI) and insulin resistance, were observed, suggesting a multifaceted positive effect on the health of young patients.

In-depth Analysis of the CAHtalyst Pediatric Study

This Phase 3 study stands out as the largest-ever interventional clinical trial focused on classic congenital adrenal hyperplasia. In total, 103 pediatric patients aged four to 17 participated, undergoing a distinct study design that combined both double-blind and open-label treatment phases. The initial 28 weeks involved a randomized controlled trial using CRENESSITY versus placebo, followed by a 24-week phase where all patients were treated with CRENESSITY.

Significant Improvements Observed

Participants demonstrated long-term, sustained reductions in glucocorticoid therapy, which were closely monitored. Remarkably, the percentage of patients achieving glucocorticoid doses within the physiologic range increased slightly from 30% at Week 28 to 32% at Week 52. These results were accompanied by significant reductions in hormone levels indicative of CAH, highlighting CRENESSITY's potential to manage the condition effectively.

CRENESSITY's Profile and Tolerability

CRENESSITY was generally well tolerated by trial participants, with no cases of adrenal crises reported during the study. However, monitoring adverse reactions showed that headaches, abdominal pain, and other side effects occurred more frequently in the treatment group compared to the placebo. Notably, despite high doses of glucocorticoids, severe symptoms related to the condition did not worsen, pointing towards the treatment's overall effectiveness in managing CAH without exacerbating side effects.

Exploring Dosing Regimens

The study also provided insights into evolving glucocorticoid dosing regimens among patients, with many transitioning to less frequent dosing schedules, indicating a potential reduction in treatment burden. These changes may contribute to diminished side effects associated with chronic high-dose glucocorticoid treatments, which have been linked to weight gain and other metabolic issues.

About Congenital Adrenal Hyperplasia

Congenital adrenal hyperplasia is a complex genetic condition caused by an enzyme deficiency that impacts adrenal hormone production, vital for life. In many cases, this deficiency leads to serious health complications if not managed properly. Traditional treatments often involve administering excess glucocorticoids, which can introduce other health risks due to steroid excess.

The Future of CAH Treatment with CRENESSITY

As more data from the CAHtalyst study emerges, it may redefine the treatment landscape for CAH. The ongoing evaluation of CRENESSITY's long-term effects will continue to provide critical insights into managing this condition effectively while minimizing the associated treatment burden.

Frequently Asked Questions

What is CRENESSITY?

CRENESSITY™ (crinecerfont) is an oral treatment designed to reduce excess adrenal hormones in patients with classic congenital adrenal hyperplasia.

What were the results of the Phase 3 CAHtalyst Pediatric Study?

The study found lasting reductions in glucocorticoid doses and significant improvements in clinical outcomes such as BMI and insulin resistance among young patients.

How does CRENESSITY work?

CRENESSITY acts as a selective antagonist for corticotropin-releasing factor receptors, decreasing levels of adrenocorticotropic hormone and consequently lowering excessive adrenal hormone production.

Is CRENESSITY safe for children?

In clinical trials, CRENESSITY was generally well tolerated, with a manageable side effect profile and no reported adrenal crises among participants.

What does this mean for the future treatment of CAH?

These findings suggest that CRENESSITY could transform CAH treatment by allowing safer, more effective hormone management while reducing the need for high-dose glucocorticoids.

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