Neurocrine reports lasting benefits of CRENESSITY in CAH study

SAN DIEGO - Neurocrine Biosciences, Inc. (NASDAQ:NBIX), a $11.9 billion biopharmaceutical company with impressive revenue growth of 21.7% over the last twelve months, has presented new Phase 3 study data indicating that CRENESSITY™ (crinecerfont) sustains reductions in glucocorticoid doses and improves clinical outcomes for pediatric patients with classic congenital adrenal hyperplasia (CAH) over a year-long treatment period. According to InvestingPro analysis, the company appears undervalued based on its Fair Value calculations, with 9 analysts recently revising their earnings estimates upward. The findings from the CAHtalyst™ Pediatric study were shared at the Pediatric Endocrine Society (PES) 2025 Annual Meeting in National Harbor, MD.

The study involved 103 pediatric patients aged four to 17 diagnosed with classic CAH. Throughout the 28-week double-blind, placebo-controlled period, followed by a 24-week open-label period, patients received CRENESSITY or a placebo. During both phases, glucocorticoid doses were initially stabilized and then reduced while monitoring adrenocorticotropic hormone, 17-hydroxyprogesterone, and androstenedione levels. The company’s strong financial position, with a current ratio of 3.13 and moderate debt levels, positions it well to support ongoing clinical development programs.

Key results showed that reductions in higher glucocorticoid doses were maintained for up to one year. Among patients who started on CRENESSITY, 32% achieved glucocorticoid doses within the physiological range at week 52, comparable to the 30% at week 28. Furthermore, clinical outcomes such as body mass index and insulin resistance improved, with no worsening of hirsutism in female participants.

CRENESSITY was generally well tolerated, with headache being the most common adverse reaction. No adrenal crises were reported during the double-blind, placebo-controlled period.

Additional data presented at the meeting highlighted changes in glucocorticoid regimens, suggesting that treatment with CRENESSITY could decrease the frequency of hydrocortisone dosing and enable a transition to dexamethasone- and prednisolone-free regimens.

The CAHtalyst Pediatric study forms part of the largest interventional clinical trial program for classic CAH to date. CRENESSITY received FDA approval in December 2024 based on data from the CAHtalyst Phase 3 studies. The ongoing open-label extension treatment portions of these studies continue to evaluate the long-term safety and efficacy of the medication.

CRENESSITY, a corticotropin-releasing factor type 1 receptor antagonist, aims to control excess adrenocorticotropic hormone and adrenal androgens without the use of glucocorticoids. It is available in capsule form and as an oral solution, with dosages tailored for both adult and pediatric patients.

This report is based on a press release statement from Neurocrine Biosciences, Inc. The company maintains a "GREAT" financial health score according to InvestingPro analysis, with robust profitability metrics and strong cash flow generation. For deeper insights into NBIX’s financial performance and growth potential, including additional ProTips and comprehensive analysis, investors can access the detailed Pro Research Report available on InvestingPro.

In other recent news, Neurocrine Biosciences has reported significant findings from a Phase 4 study on their treatment for tardive dyskinesia, INGREZZA, showing notable improvements in patients’ quality of life. This comes as the company continues to receive attention from analysts, with Cantor Fitzgerald maintaining an Overweight rating and a $170 price target, despite adjusting some financial projections. UBS has raised its price target for Neurocrine to $152, maintaining a Buy rating, citing confidence in the growth prospects of INGREZZA and the upcoming launch of Crenessity. Stifel also reiterated a Buy rating with a $166 target, highlighting strong performance from both INGREZZA and Crenessity, which exceeded market expectations. Additionally, RBC Capital Markets increased their price target to $145, maintaining an Outperform rating, pointing to strong sales momentum and a promising start for Crenessity. These developments reflect a positive outlook on Neurocrine Biosciences’ recent commercial performance and future growth potential.

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