Neurocrine Biosciences Unveils Promising Two-Year Data for CRENESSITY in Congenital Adrenal Hyperplasia

Neurocrine Biosciences, Inc. has officially revealed groundbreaking two-year data from the Phase 3 CAHtalyst® Adult study, demonstrating the efficacy of CRENESSITY® (crinecerfont) in adults with classic congenital adrenal hyperplasia (CAH). This research, presented at the 2026 Annual Meeting of the American Association of Clinical Endocrinology, showcases sustained and substantial reductions in glucocorticoid doses while effectively maintaining androgen control.

Key Findings of the Study

The study's results are promising, showing that approximately 70% of adult patients treated with CRENESSITY achieved and sustained glucocorticoid dosing within the physiologic range at the two-year mark. Specifically, the data indicated that 75% of patients who initially took dexamethasone were able to transition off this treatment, allowing for a more physiologic glucocorticoid regimen without compromising androgen control, a significant advancement in CAH management.

Dr. Sanjay Keswani, Chief Medical Officer at Neurocrine Biosciences, emphasized the importance of these findings. He remarked, "For decades, managing classic congenital adrenal hyperplasia has relied heavily on supraphysiologic glucocorticoid dosing, exposing patients to numerous long-term health risks. Our two-year findings indicate that CRENESSITY provides effective androgen control while allowing for significant reductions in glucocorticoid exposure."

At 24 months, around 69% of participants managed to keep their glucocorticoid doses to physiologic levels (≤11 mg/m²/day of hydrocortisone equivalents), with numerous patients eliminating non-physiologic glucocorticoid types. Notably, among those initially taking dexamethasone, 75% shifted to a treatment regimen free of this medication. This impressive reduction in glucocorticoid doses was achieved without negatively affecting androgen control, reaffirming CRENESSITY's safety profile.

Long-Term Benefits of CRENESSITY

Long-term exposure to high doses of glucocorticoids can result in severe health complications, including cardiometabolic issues, decreased bone density, and mental health concerns. Richard J. Auchus, M.D., a principal investigator for the CAHtalyst Adult study, highlighted the critical nature of these results, stating, "Years of supraphysiologic glucocorticoid exposure can lead to severe long-term health issues that typically develop with traditional CAH treatments."

The study not only demonstrates the efficacy of CRENESSITY in reducing glucocorticoid exposure but also emphasizes the absence of new safety concerns or tolerability issues, indicating that it represents a reliable long-term treatment option. Over 80% of the participants remained in the study for the full two years, underscoring high treatment satisfaction.

Additional Future Developments

Neurocrine Biosciences is expected to present further data on clinical endpoints and outcomes related to CRENESSITY at various upcoming medical conferences, continuing its commitment to advancing care options for patients with congenital adrenal hyperplasia.

Understanding Congenital Adrenal Hyperplasia

For context, congenital adrenal hyperplasia is a genetic disorder caused by an enzyme deficiency, impacting the production of adrenal steroid hormones. The condition leads to insufficient cortisol production and, in many cases, aldosterone due to severe enzyme deficiencies. As a result, patients often produce excess androgens, which can cause various health complications if left untreated.

CRENESSITY, an oral selective corticotropin-releasing factor type 1 receptor antagonist, works by effectively controlling excess adrenocorticotropic hormone (ACTH) and androgens through a mechanism that does not rely on glucocorticoids. This represents a significant step forward in CAH treatment, allowing for lower and more physiologically appropriate glucocorticoid dosing, thereby enhancing the overall quality of life for individuals affected by this challenging condition.

Ultimately, the results presented by Neurocrine Biosciences signal a transformative advancement in managing congenital adrenal hyperplasia, potentially changing the lives of countless patients who have long lived under the burden of high-dose glucocorticoid therapy.