Neurocrine Biosciences Shares Promising Results from CAHtalyst Pediatric Study at PES 2025 Annual Meeting

Neurocrine Biosciences Presents Significant Findings from CAHtalyst Pediatric Study



Neurocrine Biosciences, Inc. (NASDAQ: NBIX) has announced that it will present new findings from its Phase 3 CAHtalyst Pediatric study at the upcoming Pediatric Endocrine Society (PES) Annual Meeting, taking place from May 15-18, 2025, in National Harbor, MD. This presentation is highly anticipated due to the promising results showcasing the medication CRENESSITY™ (crinecerfont), aimed at treating classic congenital adrenal hyperplasia (CAH).

The Phase 3 trial analyzed pediatric patients aged between 4 and 17 years who were treated with CRENESSITY for up to one year. This innovative study revealed significant reductions in glucocorticoid doses while simultaneously achieving improvements in key clinical outcomes. Notably, the levels of adrenocorticotropic hormone (ACTH), 17-hydroxyprogesterone, and androstenedione remained below baseline levels in patients despite the reduction in glucocorticoid therapy, which is crucial for managing CAH effectively.

In the study, there were substantial improvements recorded in the body mass index (BMI) and insulin resistance among patients treated with CRENESSITY. Dr. Eiry W. Roberts, Chief Medical Officer of Neurocrine Biosciences, remarked that these exciting results underscore the potential of CRENESSITY to revolutionize treatment options for young patients affected by this condition. The treatment approach not only focuses on alleviating hormonal imbalances but also allows for a significant decrease in glucocorticoid doses, which are traditionally required to manage this disorder.

The findings are particularly important as previous treatments often required high doses of glucocorticoids, which led to several health complications, including weight gain, diabetes, and cardiovascular issues. The CAHtalyst study is considered the most extensive interventional clinical trial program for CAH to date, involving a total of 103 pediatric patients. It included both a double-blind placebo-controlled (DBPC) period and an open-label (OL) phase where all patients received the treatment.

During the trial, the initial 28 weeks involved a stable glucocorticoid dose, followed by a carefully monitored reduction towards more physiologic levels based on how each patient tolerated the change. Remarkably, the reduction in glucocorticoid dosage was maintained for up to one year, showcasing not only the efficacy of CRENESSITY but also its safety. Notably, improvements were observed in the metabolic health of the participants, with specific attention to body weight and insulin sensitivity, which are crucial factors in the long-term management of CAH.

Moreover, the overall tolerability of CRENESSITY was favorable, with minimal reports of adverse reactions. The most commonly reported side effects included headache and abdominal pain, typically mild and resolved quickly without requiring treatment discontinuation. Importantly, no adrenal crises were reported during the double-blind period, which raises confidence in the medication's safety profile.

At the PES Annual Meeting, Neurocrine will not only present these significant outcomes from the CAHtalyst Pediatric study but will also share insights into broader research on treatment patterns among pediatric patients diagnosed with CAH. Additional analyses will provide a deeper understanding of the improvements in health states and allocations of glucocorticoid therapy.

With the relentless pursuit of innovative treatments addressing childhood health issues, the implications of the CAHtalyst study are profound. By enabling more physiologic glucocorticoid dosing while maintaining optimal androgen levels, CRENESSITY represents a major step forward in enhancing the quality of life for children living with CAH.

In conclusion, the results from Neurocrine Biosciences' Phase 3 CAHtalyst Pediatric study signal hope and optimism for better therapeutic regimens for children affected by classic congenital adrenal hyperplasia. As the PES 2025 Annual Meeting approaches, the medical community eagerly anticipates further details on the ability of CRENESSITY to reshape the treatment landscape for pediatric patients grappling with this complex endocrine condition.

Topics Health)

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