Neurocrine Biosciences has presented new one-year data from its Phase III CAHtalyst Adult study of Crenessity (crinecerfont) for classic congenital adrenal hyperplasia (CAH).
The data revealed that the therapy enabled subjects to achieve more physiological glucocorticoid doses while adrenocorticotropic hormone, 17-hydroxyprogesterone, and androstenedione levels stayed at or below baseline.
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The CAHtalyst Adult study, which involved 182 subjects aged between 18 and 58 years, was part of the interventional clinical trial programme for CAH.
The trial comprised a 24-week double-blind, placebo-controlled (DBPC) period followed by an open-label (OL) period of the same duration.
During the DBPC period, subjects with classic CAH currently on supraphysiologic glucocorticoid (GC) doses were randomised and given either the therapy or a placebo. While in the OL period, the therapy was given to all subjects.
Throughout both periods, GC doses were initially kept stable for four weeks. They were then reduced as tolerated toward more physiologic levels while still improving or maintaining androstenedione (A4) relative to baseline of Day 1.
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By GlobalDataThe data presented at the Endocrine Society’s Annual Meeting, ENDO 2025, in San Francisco, US, assessed the impact of up to one year of the therapy on serum A4, adrenocorticotropic hormone (ACTH), and 17-hydroxyprogesterone (7-OHP) levels, as well as GC doses and clinical outcomes.
Patients notably experienced a lasting reduction in high GC doses, with improved A4 levels.
In addition to the hormone level and GC dosing improvements, the study observed enhancements in insulin resistance and in hirsutism among females for those who received the therapy for up to one year.
Neurocrine Biosciences chief medical officer Sanjay Keswani said: “Results from the pivotal CAHtalyst clinical trial programme continue to reinforce the critical role of Crenessity in the management of classic CAH.
“These one-year data show the lasting ability of Crenessity to effectively manage the ACTH and adrenal steroid imbalances in adults while allowing for lower, more physiologic steroid dosing and improved clinical outcomes.”
Crenessity is an oral corticotropin-releasing factor type 1 receptor antagonist that demonstrated a good safety profile, with fatigue and headache being the common side effects in the two trial periods.
In 2023, Neurocrine reported positive top-line outcomes from the Phase III CAHtalyst Pediatric trial of crinecerfont for CAH due to 21-hydroxylase deficiency.
