Study reveals challenges in managing congenital adrenal hyperplasia

SAN DIEGO - A new analysis of real-world data shows that approximately 95% of patients with classic congenital adrenal hyperplasia (CAH) experience either high glucocorticoid exposure or elevated androgen levels during treatment, Neurocrine Biosciences, Inc. (NASDAQ:NBIX) reported Tuesday. The company, currently valued at $13.37 billion and maintaining strong financial health according to InvestingPro metrics, has demonstrated impressive revenue growth of 21.73% over the last twelve months.

The findings, presented at the Endocrine Society’s Annual Meeting in San Francisco, highlight significant variability in glucocorticoid treatment regimens and frequent changes in health status over time in both pediatric and adult patients. With a robust current ratio of 3.13 and moderate debt levels, Neurocrine Biosciences appears well-positioned to continue its research and development initiatives.

The analysis used data from CAHtalog, a comprehensive U.S. registry of CAH patients, examining 98 individuals including 37 adults, 42 children and adolescents, and 19 people who contributed data as both children and adults.

Researchers tracked glucocorticoid doses and androstenedione levels over time, categorizing patients into four health states based on medication dose and hormone levels. Among patients eligible for the health state transition analysis, 88.9% experienced at least one health state change, with 58.7% undergoing three or more changes.

"The CAHtalog data revealed just how dynamic and unpredictable disease control can be for people with classic congenital adrenal hyperplasia," said Sanjay Keswani, Chief Medical Officer at Neurocrine Biosciences, according to the press release.

The study found that even when patients achieved optimal control with low glucocorticoid doses and normal androstenedione levels, this state was often temporary. Nearly all patients (95.2%) entered a suboptimal health state at least once during their follow-up period.

Classic CAH is a rare genetic condition causing enzyme deficiency that alters adrenal steroid hormone production. While glucocorticoid therapy is essential to replace cortisol deficiency, achieving hormonal control often requires supraphysiologic doses, which can lead to serious complications.

The analysis was conducted using data collected prior to the commercial availability of CRENESSITY (crinecerfont), Neurocrine’s CAH treatment, and does not include any CRENESSITY treatment data.

In other recent news, Neurocrine Biosciences announced positive one-year data for its CRENESSITY treatment, showing significant reductions in glucocorticoid doses and improved clinical outcomes for adults with classic congenital adrenal hyperplasia. The Phase 3 CAHtalyst Adult study revealed a 25% reduction in glucocorticoid doses for continuous treatment and a 30% reduction for those switching from placebo. Meanwhile, Goldman Sachs initiated coverage on Neurocrine Biosciences with a Buy rating, highlighting the company’s growth prospects and setting a price target of $182. UBS also raised its price target for the company to $174, citing positive indicators for the CRENESSITY drug launch and projecting higher-than-consensus revenue estimates. Additionally, Neurocrine reported new data indicating that its drug Ingrezza significantly reduced cognitive and motor burdens in adults with Huntington’s disease chorea. Further analyses showed improvements in health-related quality of life for older adults with tardive dyskinesia treated with Ingrezza, demonstrating its potential benefits beyond motor symptom control. These developments underscore Neurocrine’s ongoing efforts to enhance treatment options for patients with complex neurological and endocrine disorders.

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