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Neurocrine Biosciences Reports One-Year Data at ENDO 2025

Neurocrine Biosciences, Inc. (Nasdaq: NBIX) recently unveiled promising one-year data from the CAHtalyst™ Adult study of CRENESSITY® (crinecerfont) at the Endocrine Society's Annual Meeting, ENDO 2025, held in San Francisco. This research highlights the sustained efficacy of CRENESSITY in adult patients suffering from classic congenital adrenal hyperplasia (CAH).

Groundbreaking Findings on Efficacy

The one-year findings from the CAHtalyst Adult study are crucial as they demonstrate the long-term benefits of treatments for classic CAH. The data indicates that patients on CRENESSITY could achieve more physiologic glucocorticoid doses, while maintaining crucial hormone levels within acceptable ranges. Both adrenocorticotropic hormone (ACTH) and 17-hydroxyprogesterone levels remained stable or below baseline levels, indicating a significant improvement in hormonal balance for these patients.

Dr. Sanjay Keswani, Chief Medical Officer at Neurocrine Biosciences, emphasized the significance of these findings: "The results from the pivotal CAHtalyst clinical trial continue to reinforce the critical role of CRENESSITY in managing classic CAH. These one-year data illustrate CRENESSITY's capacity to effectively address ACTH and adrenal steroid imbalances in adults, allowing for lower and more physiologic steroid dosing along with improved clinical outcomes."

Study Design and Participant Demographics

The Phase 3 CAHtalyst Adult study marked a significant advance in CAH treatment research, incorporating 182 adult participants aged between 18 to 58 years. The study comprised a rigorous 24-week double-blinded, placebo-controlled phase, followed by an open-label phase of another 24 weeks. Participants initially maintained stable glucocorticoid doses during the first four weeks, after which doses were gradually reduced towards more physiologic levels, guided by patient tolerance.

Throughout this study, there were noteworthy changes in serum levels of crucial hormones, as it assessed the effects of up to one year of CRENESSITY treatment. Impressively, participants who sustained treatment with CRENESSITY demonstrated substantial reductions in their glucocorticoid doses, without compromising on the maintenance of androstenedione (A4) levels.

Outcomes of Interest

Data analysis from the study illuminated lasting reductions of glucocorticoid doses—up to a 30% decrease observed over the treatment period. In contrast, A4 levels were either maintained or improved under these reduced glucocorticoid doses. Moreover, the group receiving continuous CRENESSITY therapy reported a remarkably stable proportion of participants achieving glucocorticoid doses within the physiological range.

Moreover, patients recorded enhancements in both insulin resistance metrics and hirsutism symptoms among female participants, suggesting broader health benefits from the treatment.

Understanding CAH and CRENESSITY's Role

Congenital adrenal hyperplasia is a rare genetic disorder stemming from enzyme deficiencies that impact adrenal hormone production. CRENESSITY addresses these imbalances effectively. It operates as a potent and selective antagonist for corticotropin-releasing factor type 1 receptors (CRF1), managing both ACTH and adrenal androgen levels without relying solely on glucocorticoids. This innovative approach supports healthier management of the condition with fewer side effects typically associated with high-dose steroid treatments.

The findings from the CAHtalyst Adult study not only hold significance for current patients but also pave the way for newly diagnosed individuals seeking effective treatment pathways for classic CAH. In addition, Neurocrine Biosciences is expected to share additional data on weight-related outcomes from the trial, enhancing our understanding of the impact of CRENESSITY in comprehensive patient care.

Conclusion

As Neurocrine Biosciences continues to push the envelope in CAH treatment paradigms, the promising results from this large-scale study showcase CRENESSITY as a remarkable therapeutic option for adults. The data presented at ENDO 2025 undoubtedly serve as a critical stepping stone toward improving the management of classic congenital adrenal hyperplasia.