#USA #San Diego #Crenessity #Neurocrine Biosciences #CAHtalyst

Exciting Developments in Pediatric CAH Treatment

Neurocrine Biosciences, Inc. (Nasdaq: NBIX) recently unveiled new findings from the exploratory analyses of the Phase 3 CAHtalyst™ Pediatric study. These results highlight critical benefits of their treatment, CRENESSITY™ (crinecerfont), specifically focusing on its effectiveness in managing classic congenital adrenal hyperplasia (CAH) in children. What stands out is that the treatment has successfully enabled pediatric patients to maintain or improve their androgen levels while simultaneously reducing the doses of glucocorticoid medications.

Results Overview

The latest data reveal promising outcomes across various patient subgroups, ensuring that the beneficial effects of CRENESSITY™ are consistent, regardless of demographic factors, baseline androstenedione levels, or initial glucocorticoid doses. This indicates not only the robustness of the treatment but also its versatility in managing CAH's complexities.

At the 2025 Joint Congress of the European Society for Paediatric Endocrinology and the European Society of Endocrinology, Neurocrine Biosciences plans to present these remarkable findings, which could redefine treatment strategies for pediatric patients suffering from this condition.

Understanding Congenital Adrenal Hyperplasia

CAH is a genetic disorder stemming from a deficiency in an enzyme responsible for optimal steroid hormone production. Patients with CAH typically experience an overproduction of androstenedione, leading to various health challenges including abnormal growth patterns, premature puberty, and broader developmental hurdles. Traditional treatment methods have centered around high doses of glucocorticoids. While effective in managing hormone levels, these high doses often come with a suite of adverse side effects.

The Introduction of CRENESSITY™

CRENESSITY™ represents a groundbreaking approach that shifts the focus away from glucocorticoid reliance by utilizing a selective corticotropin-releasing factor type 1 receptor (CRF1) antagonist. This mechanism signifies a shift towards a more targeted treatment, aiming to lower the production of adrenal androgens effectively while mitigating the need for higher glucocorticoid doses.

According to Dr. Eiry W. Roberts, Chief Medical Officer at Neurocrine Biosciences, the potential for CRENESSITY™ to positively influence long-term outcomes is considerable. By enabling patients to maintain their hormone levels without excessive glucocorticoid exposure, the treatment could alleviate the complications often associated with high-steroid use, ranging from metabolic issues to psychological effects like mood swings and memory concerns.

Study Insights

The CAHtalyst Pediatric study incorporated a total of 103 participants, who were randomly assigned either CRENESSITY™ or a placebo over a 28-week period. Key findings showed a significant reduction in androstenedione levels at Week 4, with the treatment demonstrating a steady capacity to reduce glucocorticoid usage while sustaining or improving hormone levels throughout.

• - Androstenedione Levels: At Week 4, CRENESSITY™ led to an average decrease in androstenedione levels compared to placebo, indicating its effectiveness in managing this critical hormone.
• - Glucocorticoid Reduction: By Week 28, patients on CRENESSITY™ had a notable reduction in glucocorticoid doses compared to those on the placebo.

These results collectively suggest that a transition to CRENESSITY™ could be a game-changer for many patients, offering a pathway to a more balanced and healthier treatment regime.

Safety and Tolerability

The CAHtalyst Pediatric study also addressed safety, showing that CRENESSITY™ was well tolerated among participants. The most frequently reported side effects (observed in over 4% of patients) included headaches, abdominal discomfort, fatigue, nasal congestion, and occasional nosebleeds.

Conclusion

The innovative approach of CRENESSITY™ offers hope to children suffering from classic congenital adrenal hyperplasia, with a potential to significantly enhance their quality of life. As Neurocrine Biosciences continues developing solutions for conditions like CAH, the medical community remains optimistic about more effective, less invasive treatment options becoming available. Further analysis and presentations from the CAHtalyst study at international forums will undoubtedly shed more light on the implications and efficiencies of CRENESSITY™, signaling a positive trend for future pediatric CAH therapies.