SAN DIEGO, June 3, 2024 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) and Diurnal Ltd., a Neurocrine Biosciences company, presented information from its neuroendocrinology pipeline at the Endocrine Society Annual Meeting, ENDO 2024, including primary data just published in The New England Journal of Medicine from its CAHtalyst™ Phase 3 registrational studies of crinecerfont in pediatric and adult patients with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. In addition, Neurocrine Biosciences presented CAHtalog™ Registry data regarding the negative impact of high glucocorticoid (GC) doses and natural history in pediatric and adult CAH patients, as well as disease- and GC-related comorbidities data in CAH patients.
"We were thrilled to share our just published primary CAHtalyst Phase 3 data with ENDO 2024 attendees this past weekend," said Eiry W. Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences. "Crinecerfont offers the potential of a long-awaited new treatment paradigm for endocrinologists in managing CAH. Our CAHtalyst Phase 3 data demonstrate the potential of crinecerfont to reduce elevated androgen levels and lower supraphysiologic glucocorticoid doses while maintaining androgen control in CAH patients of four years and older."
CAHtalystTM Phase 3 Pediatric and Adult DataIn an oral presentation on June 1, Richard Auchus, M.D., Principal Investigator, Professor of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes at the University of Michigan, presented CAHtalyst Phase 3 Adult data demonstrating that the study met its primary and important key secondary endpoints, with 63% of crinecerfont-treated participants achieving reduction in GC dosing to physiologic range (≤ 11mg/m2/day) while maintaining androstenedione control at Week 24 as compared with just 18% with placebo control participants. The data were published in an online edition of The New England Journal of Medicine and will appear in a future print issue of the journal.
In a poster presentation on June 2, Kyriakie Sarafoglou, M.D., Professor, Department of Pediatrics and Department of Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics & Metabolism, at the University of Minnesota, presented CAHtalyst Phase 3 Pediatric data demonstrating that the study met its primary and key secondary endpoints and was well tolerated, showing 29.9% of crinecerfont participants achieved a reduction in GC dosing to a physiologic range (≤11mg/m2/day) at Week 28 while maintaining androgen control versus 0% of placebo participants (Poster #SUN-441). The data were published in an online edition of The New England Journal of Medicine and will appear in a future print issue of the journal.
CAHtalogTM Registry—Negative Impacts of Supraphysiologic Dosing in Patients with CAHA real-world study explored the effects of high GC doses in pediatric and adult patients enrolled in the CAHtalog registry, highlighting the fluctuations in GC dose and androstenedione over time and the relationship between higher GC dose and negative clinical outcomes in patients with CAH (Poster #SUN-685). The findings included:
- Among 44 pediatric patients, 12 (27.3%) received a high GC dose (>15 mg/m2/day), that was associated with:
- Premature adiposity rebound starting at ~2 years of age (usual age is ~6 years)
- Early growth acceleration attributed to advanced bone age, followed by blunted pubertal growth
- In 69 patients (45 pediatric, 34 adult and 10 in both groups) with available comorbidity-related data, hypertensive diseases and metabolic complications were significantly more prevalent with high versus low GC doses
- In pediatric patients, short stature was more common with high versus low GC dose
- In 40 patients (27 pediatric, 14 adult and 1 in both groups) with ≥ 3 matched GC-androstenedione records, 92% had ≥ 1 transition in health state over the median 7-year observation period
- Higher GC dose with androstenedione < upper limit of normal (ULN) was the most common health state observed at the first recorded datapoint (48%), with only 3% maintaining that health state throughout the observation period
- Although 22% of patients presented with lower GC and androstenedione