Neurocrine Biosciences (NBIX) Presents First Retrospective Case Series of CRENESSITY

Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced clinical findings from the first retrospective case series in pediatric and adult patients with classic congenital adrenal hyperplasia (CAH) due to 11β‑hydroxylase deficiency. This subtype was not previously studied in clinical trials of CRENESSITY^® (crinecerfont) and is the second most common form of classic CAH after 21-hydroxylase deficiency, accounting for approximately 5% of cases. The findings were presented at the Endocrine Society's annual meeting, ENDO 2026, in Chicago.

Like other forms of CAH, 11β‑hydroxylase deficiency (11β‑OHD) is characterized by cortisol deficiency and excess adrenal androgens. However, 11β‑OHD is uniquely associated with the accumulation of the adrenal steroid precursors 11-deoxycortisol (11-dF) and 11-deoxycorticosterone (DOC). These hormonal imbalances can contribute to distinct clinical features, including hypertension and other long‑term complications. In this retrospective case series, reductions in androstenedione (A4) were observed across all patients with elevated baseline levels following initiation of CRENESSITY. Steroid precursors also decreased, with values reaching within normal ranges, and blood pressure improved in adult patients.

"Patients with 11β‑hydroxylase deficiency represent a complex subgroup within classic congenital adrenal hyperplasia, with limited data available to guide treatment decisions for these individuals," said Sanjay Keswani, M.D., Chief Medical Officer, Neurocrine Biosciences. "This first case series provides preliminary clinical insights on the use of CRENESSITY to target ACTH and potentially improve hormonal control in patients with 11β‑hydroxylase deficiency, highlighting our ongoing commitment to advancing care for the entire classic CAH community."

This retrospective case series outlines clinical responses to CRENESSITY treatment in 15 pediatric (n=11) and adult (n=4) patients with classic CAH due to 11β‑OHD. Across all patients with elevated A4 and/or adrenal steroid precursors at baseline, initiation of CRENESSITY was associated with reduction of levels.

• A4 and precursor normalization were observed as early as one month following treatment initiation, including in patients whose hormone excess had persisted despite prior supraphysiologic glucocorticoid (GC) therapy.
  • Among patients with elevated hormones at baseline, median decreases of -92% (DOC, n=5), -95% (11‑dF, n=7), and -65% (A4, n=3) were observed after CRENESSITY initiation.

• Among patients receiving antihypertensive treatment, two of five were able to reduce or discontinue these medications.
• Following the initiation of CRENESSITY, 14 of 15 patients were able to reduce their total GC dose.

"Patients with 11β-hydroxylase deficiency often experience both androgen excess and elevated adrenal steroid precursors that can contribute to hypertension and other complications, making disease management particularly challenging," said Kyriakie Sarafoglou, M.D., Professor, Department of Pediatrics and Department of Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics & Metabolism, University of Minnesota. "Given the rarity of this condition, data from 15 patients gives clinicians meaningful insight into the potential role of CRENESSITY in managing this challenging form of classic congenital adrenal hyperplasia."

This case series provides early, foundational evidence supporting the efficacy and safety of CRENESSITY treatment in patients with classic CAH due to 11β-OHD. Although CRENESSITY is approved as an adjunctive treatment to GCs for patients with classic CAH regardless of enzyme deficiency, there is limited evidence on its use in this rare subtype. These findings support further exploration of CRENESSITY in this classic CAH subtype and reinforce Neurocrine's commitment to supporting patients across the full spectrum of classic CAH and other rare endocrine diseases.

Presentations at the ENDO 2026 annual meeting included:

CAHtalyst^® Adult Study Two-Year Results

Title: Weight-Related Outcomes and Insulin Resistance in Adults with Classic Congenital Adrenal Hyperplasia: 2-Year Results from the CAHtalyst Adult Study (Oral Presentation #ORF32-07)
Authors: Oksana Hamidi, D.O., et al

Title: Adults with Classic Congenital Adrenal Hyperplasia Taking Crinecerfont Demonstrated Sustained Decreases in Glucocorticoid Doses: 2-Year Results from the CAHtalyst Adult Study (Poster Presentation #SUN-458)
Authors: Irina Bancos, M.D., et al

Title: A Cross-sectional Survey on Quality of Life of Adults with Classic Congenital Adrenal Hyperplasia in the United States Participating in CAHtalyst Adult Open-Label Extension Study (Poster Presentation #SUN-467)
Authors: Sonal Vaid, M.D., et al

Title: Bone Outcomes in Adults with Classic Congenital Adrenal Hyperplasia Treated with Crinecerfont for Up to 2 Years in CAHtalyst Adult Study (Poster Presentation #SUN-468)
Authors: Maria Vogiatzi, M.D., et al

CAHtalyst Pediatric Study Two-Year Results

Title: Characterization of Children and Adolescents with Classic Congenital Adrenal Hyperplasia Who Had Slowed Bone Age Progression and Improved Height Prediction with Crinecerfont (Oral Presentation #ORF32-05)
Authors: Maria Vogiatzi, M.D., et al

Title: Long-term Crinecerfont Treatment Reduced ACTH and 17-Hydroxyprogesterone — Clinical Outcomes in Children and Adolescents with Classic Congenital Adrenal Hyperplasia: 2-Year Results from CAHtalyst Pediatric (Poster Presentation #SAT-465)
Authors: Natalie Nokoff, M.D., et al

Title: Long-term Crinecerfont Enables Sustained Decreases in Glucocorticoid Doses — Clinical Outcomes in Children and Adolescents with Classic Congenital Adrenal Hyperplasia: 2-Year Results from CAHtalyst Pediatric (Poster Presentation #SUN-465)
Authors: Kyriakie Sarafoglou, M.D., et al

Additional Presentations

Title: Long-Term Risk of Cardiometabolic Comorbidities Associated with Glucocorticoid Exposure and Androgen Control in Classic Congenital Adrenal Hyperplasia: A Cox Proportional Hazards Analysis from the CAHtalog Registry ("New Therapies and Perspectives for Congenital Adrenal Hyperplasia and Adrenal Insufficiency" Rapid Fire Presentation #ORF32-02 and Poster Presentation #MON-495)
Authors: Oksana Lekarev, D.O., et al

Title: Crinecerfont Treatment of Classic Congenital Adrenal Hyperplasia Due to 11β-Hydroxylase Deficiency: A Case Series (Poster Presentation #SAT-466)
Authors: Kyriakie Sarafoglou, M.D., et al

Title: A Modified Delphi Panel of U.S. Endocrinologists to Align on Minimum Clinically Important Difference in Glucocorticoid Dose and Other Key Considerations in Classic Congenital Adrenal Hyperplasia (Poster Presentation #SAT-459)
Authors: Ahmed Khattab, M.D., et al

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