Neurocrine Reports 6.5-Point HQ-CT Benefit for VYKAT XR and 95% Hormone Cuts in CAH
NBIX•Neurocrine’s VYKAT XR extended-release tablets produced sustained hyperphagia improvements in Prader-Willi patients, achieving 6.2–6.5 point HQ-CT score differences versus natural history controls over three years and a –6.3 mean change two years after randomized withdrawal. Crinecerfont in 15 CAH patients cut 11-deoxycortisol by 95% and deoxycorticosterone by 92%, allowing 14 patients to lower glucocorticoid doses.
1. VYKAT XR Long-Term Efficacy Data
Neurocrine’s Phase 3 program showed that PWS participants who resumed VYKAT XR after a 16-week randomized withdrawal achieved a mean –6.3 reduction in HQ-CT Total Score by two years, compared with –3.1 in continuously treated patients. Versus PATH real-world controls, VYKAT XR delivered 6.2–6.5 point HQ-CT advantages at Years 1–3, plus significant gains across all six PWSP behavioral domains, stable BMI and no new safety signals.
2. Crinecerfont Case Series in 11β-Hydroxylase CAH
A retrospective case series of 15 classic CAH patients with 11β-hydroxylase deficiency reported median reductions of 95% in 11-deoxycortisol, 92% in deoxycorticosterone and 65% in androstenedione within one month of crinecerfont initiation. Fourteen patients lowered their glucocorticoid doses, and two adults discontinued antihypertensives, indicating potential for improved hormonal control and reduced comorbidity risk in this rare CAH subtype.
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