NBIX News & Events

Soleno Therapeutics, a Neurocrine Biosciences company, announced late-breaking data at ENDO 2026 showing that resuming treatment with VYKAT XR extended-release tablets for two years after a 16-week randomized withdrawal period was associated with durable improvements in hyperphagia and behavioral symptoms characteristic of Prader-Willi syndrome. Participants who resumed VYKAT XR following a 16-week randomized withdrawal period demonstrated improvements in hyperphagia and behavioral symptoms as early as Week 13, with benefits continuing through 2 years. VYKAT XR demonstrated statistically significant and sustained improvements in hyperphagia and Prader-Willi syndrome-related behaviors for up to 3 years compared to real-world data from the PATH for PWS Natural History Study.

Neurocrine Biosciences announced clinical findings from the first retrospective case series in pediatric and adult patients with classic congenital adrenal hyperplasia due to 11beta-hydroxylase deficiency. This subtype was not previously studied in clinical trials of CRENESSITY 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. Androstenedione and other adrenal hormone levels in 11beta-hydroxylase-deficient patients improved substantially after initiation of CRENESSITY, with greater than90% median reductions in 11-deoxycortisol and 11-deoxycorticosterone. Nearly all patients (14/15) reduced their total glucocorticoid dose with CRENESSITY, and 2 of 5 patients on antihypertensive medications reduced or discontinued these drugs. Findings provide initial clinical insights in patients with classic congenital adrenal hyperplasia due to 11beta-hydroxylase deficiency, a rare subtype not previously studied in clinical trials of CRENESSITY.

Neurocrine Biosciences announced new Phase 3 CAHtalyst Adult study data demonstrating improved cardiometabolic outcomes alongside sustained glucocorticoid dose reduction through up to two years of treatment with Crenessity for classic congenital adrenal hyperplasia. These results, together with additional presentations highlighting improvement in bone outcomes and patient-reported quality of life survey outcomes, were presented at the Endocrine Society's annual meeting, ENDO 2026, in Chicago. Two-year Crenessity data in adults showed improvements in cardiometabolic outcomes of insulin resistance, body composition and body weight alongside sustained reductions in glucocorticoid dose. Among participants who were overweight or obese at baseline, 37% achieved greater than5% reduction in body weight at 2 years, and 43% of those with insulin resistance at baseline were no longer insulin resistant at 2 years. Favorable trends in bone health outcomes were observed with Crenessity treatment, including improvements in bone mineral density with up to 2 years of treatment. Patient-reported survey data from adults in the open-label extension suggested improvements in quality of life outcomes and treatment satisfaction, including emotional well-being, energy levels and confidence in managing their classic congenital adrenal hyperplasia with Crenessity.