Neurocrine reports positive one-year data for CAH treatment

SAN DIEGO - Neurocrine Biosciences, Inc. (NASDAQ:NBIX), a profitable biotech company with a market capitalization of $13.2 billion and impressive revenue growth of 22% over the last twelve months, announced today that its CRENESSITY (crinecerfont) treatment demonstrated lasting reductions in glucocorticoid doses and improved clinical outcomes in adults with classic congenital adrenal hyperplasia (CAH) after one year of treatment.

The data, presented at the Endocrine Society’s Annual Meeting in San Francisco, showed that adult patients achieved more physiologic glucocorticoid doses while maintaining or improving hormone levels. These results complement previously reported one-year data from the pediatric study.

The Phase 3 CAHtalyst Adult study included 182 patients aged 18 to 58 years. Patients receiving continuous CRENESSITY treatment for one year showed a 25% reduction in mean glucocorticoid dose, while those who switched from placebo to CRENESSITY showed a 30% reduction. According to InvestingPro analysis, Neurocrine maintains strong financial health with a current ratio of 3.13, indicating robust capability to fund ongoing clinical development. Get access to 8 more exclusive ProTips and comprehensive financial analysis with InvestingPro.

The study also demonstrated improvements in insulin resistance and hirsutism in female participants. Adrenocorticotropic hormone and 17-hydroxyprogesterone levels remained at or below baseline despite substantial reductions in glucocorticoid doses.

"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," said Sanjay Keswani, Chief Medical Officer at Neurocrine Biosciences, according to the press release.

CRENESSITY was approved by the U.S. Food and Drug Administration in December 2024 for use with glucocorticoids to control androgen levels in adults and children 4 years and older with classic CAH. The most common side effects in adults included headache and fatigue, which were generally mild to moderate. With earnings per share of $2.96 and analysts projecting continued profitability, InvestingPro’s Fair Value analysis suggests the stock is currently undervalued. Discover detailed valuation metrics and growth potential in the comprehensive Pro Research Report, available exclusively to subscribers.

Classic CAH is a rare genetic condition affecting the production of adrenal steroid hormones, requiring lifelong treatment with glucocorticoids. Traditional high-dose glucocorticoid treatment can lead to serious complications, including metabolic issues, cardiovascular disease, and osteoporosis.

In other recent news, Neurocrine Biosciences has caught the attention of investors and analysts alike with several key developments. UBS has raised its price target for Neurocrine to $174 from $152, citing positive indicators for the company’s Crenessity drug launch, and maintains a Buy rating. UBS projects second-quarter revenue for Crenessity at $25 million, surpassing the consensus estimate of $22 million. Goldman Sachs also initiated coverage on Neurocrine with a Buy rating, highlighting a projected 11% compound annual growth rate through 2030. They noted that Neurocrine’s current trading values reflect market concerns despite its promising growth profile.

Neurocrine recently reported new patient data showing that its drug Ingrezza significantly reduced cognitive and motor-related burdens in adults with Huntington’s disease chorea. Additionally, analyses presented at a conference showed that older adults with tardive dyskinesia experienced significant improvements in quality of life when treated with Ingrezza. In corporate news, Neurocrine appointed Lewis Choi as its new Chief Information Officer, bringing over 25 years of experience in IT and AI to the company. Choi’s appointment comes at a pivotal time, with the ongoing launch of Crenessity and growth of Ingrezza.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.